Fibrocell Science Inc
Annual Report 2017

Plain-text annual report

UNITED STATES SECURITIES AND EXCHANGE COMMISSIONWashington, D.C. 20549________________________________________________________________FORM 10-Ký Annual Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 For the fiscal year ended December 31, 2017OR¨ Transition Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934________________________________________________________________Fibrocell Science, Inc.(Exact name of registrant as specified in its Charter.) Delaware001-3156487-0458888(State or other jurisdiction of incorporation)(Commission File Number)(I.R.S. Employer Identification No.) 405 Eagleview BoulevardExton, Pennsylvania 19341(Address of principal executive offices, including zip code)(484) 713-6000(Registrant’s telephone number, including area code)Securities registered pursuant to Section 12(b) of the Act:Title of Each ClassName of each exchange on which registeredCommon Stock, $.001 par value The Nasdaq Stock Market LLC Securities registered pursuant to Section 12(g) of the Act: None____________________________________________________________________________________________________________Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes o No ýIndicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. Yes o No ýIndicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934, as amended, during thepreceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ý No o Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Web site, if any, every Interactive Data File required to be submitted andposted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit andpost such files). Yes ý No oIndicate by check mark if disclosure of delinquent filers pursuant to Item 405 of Regulation S-K (§ 229.405) is not contained herein, and will not be contained, to the best ofregistrant’s knowledge, in definitive proxy or information statements incorporated by reference in Part III of this Form 10-K or any amendment to this Form 10-K. oIndicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company or an emerging growth company.See the definitions of “large accelerated filer,” “accelerated filer”, “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act. Large accelerated filer ¨Accelerated filer ¨Non-accelerated filer ¨ (Do not check if a smaller reporting company)Smaller reporting company x Emerging growth company ¨ If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financialaccounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨Indicate by check mark whether the registrant is a shell company (as defined in the Exchange Act Rule 12b-2). Yes o No ýThe aggregate market value of the registrant’s common stock held by non-affiliates was $36.7 million as of June 30, 2017 (the last business day of the registrant’s most recentlycompleted second fiscal quarter), based on a total of 9,161,161 shares of common stock held by non-affiliates and on a closing price of $4.01 as reported on the Nasdaq CapitalMarket on June 30, 2017.As of March 6, 2018, there were 28,356,351 shares of the registrant’s common stock, par value $0.001 per share, outstanding.DOCUMENTS INCORPORATED BY REFERENCE Portions of the registrant’s definitive proxy statement for its 2018 annual meeting of stockholders are incorporated by reference into Part III of this Form 10-K where indicated.Such definitive proxy statement will be filed with the U.S. Securities and Exchange Commission within 120 days after the year ended December 31, 2017. TABLE OF CONTENTSPage NOTE REGARDING FORWARD-LOOKING STATEMENTS1 PART I ITEM 1.BUSINESS3ITEM 1A.RISK FACTORS22ITEM 1B.UNRESOLVED STAFF COMMENTS50ITEM 2.PROPERTIES50ITEM 3.LEGAL PROCEEDINGS50ITEM 4.MINE SAFETY DISCLOSURE50 PART II ITEM 5.MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OFEQUITY SECURITIES51ITEM 6.SELECTED FINANCIAL DATA51ITEM 7.MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS53ITEM 7A.QUANTITATIVE AND QUALITATIVE DISCLOSURE ABOUT MARKET RISK64ITEM 8.FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA64ITEM 9.CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE64ITEM 9A.CONTROLS AND PROCEDURES64ITEM 9B.OTHER INFORMATION65 PART III ITEM 10.DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE66ITEM 11.EXECUTIVE COMPENSATION66ITEM 12.SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDERMATTERS66ITEM 13.CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTOR INDEPENDENCE66ITEM 14.PRINCIPAL ACCOUNTANT FEES AND SERVICES66 PART IV ITEM 15.EXHIBITS AND FINANCIAL STATEMENT SCHEDULES67ITEM 16.FORM 10-K SUMMARY69 SIGNATURE PAGE70_________________________Unless the context otherwise indicates, references in this Annual Report on Form 10-K to “Fibrocell,” “the Company,” “we,” “us” and “our” refer toFibrocell Science, Inc. and its subsidiaries.Fibrocell, Fibrocell Science and LAVIV® are trademarks of Fibrocell. Other trademarks, trade names and service marks appearing in this AnnualReport on Form 10-K are the property of their respective owners.Unless otherwise indicated, all share amounts and the exercise or conversion price of any of our securities reflect, as applicable, the occurrence of a1-for-25 reverse split of our common stock that occurred on April 30, 2013 and the occurrence of a 1-for-3 reverse split of our common stock that occurred onMarch 10, 2017.Table of ContentsNOTE REGARDING FORWARD-LOOKING STATEMENTS This Annual Report on Form 10-K (this Form 10-K) contains “forward-looking statements” within the meaning of the Private Securities LitigationReform Act of 1995. These statements include, among others, statements about:•our expectation that our existing cash resources, will be sufficient to enable us to fund our operations into the first quarter of 2019;•future expenses and capital expenditures;•our estimates regarding expenses, future revenues, capital requirements and needs for, and ability to obtain, additional financing;•our plans to address our future capital requirements and the consequences of failing to do so;•our plans to resolve our noncompliance with the minimum bid price requirements of the Nasdaq Capital Market (Nasdaq) listing rules and theconsequences of failing to do so;•our need to raise substantial additional capital to fund our operations;•our plans to execute enrollment of pediatric patients in the Phase 2 portion of our Phase 1/2 clinical trial of FCX-007 in the first quarter of 2018;•our plans to report interim data from patients from our Phase 1/2 clinical trial for FCX-007 in the second quarter of 2018;•our expectation to begin dosing patients in the Phase 2 portion of our Phase 1/2 clinical trial of FCX-007 in the second quarter of 2018;•our expectation to complete enrollment of patients for our Phase 1/2 clinical trial of FCX-007 in the third quarter of 2018;•our plans to report interim data from patients from our Phase 1/2 clinical trial for FCX-007 in the first quarter of 2019;•our expectation to initiate enrollment in a Phase 1/2 clinical trial of FCX-013 in the third quarter of 2018;•our product development goals under our collaborations with Intrexon Corporation for our product candidates;•the potential benefits of Fast Track, Orphan Drug and Rare Pediatric Disease designations;•the potential advantages of our product candidates and technologies; and•the effect of legal and regulatory developments;as well as other statements relating to our future operations, financial performance or financial condition, prospects or other future events. Forward-lookingstatements appear primarily in the sections of this Form 10-K entitled “Item 1—Business,” “Item 1A—Risk Factors,” “Item 7—Management’s Discussion andAnalysis of Financial Condition and Results of Operations,” “Item 7A—Quantitative and Qualitative Disclosures About Market Risk,” and “Item 8—Financial Statements and Supplementary Data.” In some cases, you can identify forward-looking statements by words such as “may,” “will,” “could,”“would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing,” “scheduled” andsimilar expressions, although not all forward-looking statements contain these identifying words.Forward-looking statements are based upon current expectations and assumptions and are subject to a number of known and unknown risks,uncertainties and other factors that could cause actual results to differ materially and adversely from those expressed or implied by such statements. Factorsthat could cause or contribute to such differences include, but are not limited to, those discussed in this Form 10-K and in particular the risks anduncertainties discussed under "Item 1A—Risk Factors" of this Form 10-K. As a result, you should not place undue reliance on forward-looking statements.Additionally, the forward-looking statements contained in this Form 10-K represent our views only as of the date of this Form 10-K (or any earlierdate indicated in such statement). While we may update certain forward-looking statements from time to time, we specifically disclaim any obligation to doso, even if new information becomes available in the future. However, you are advised to consult any further disclosures we make on related subjects in theperiodic and current reports thatwe file with the Securities and Exchange Commission. The foregoing cautionary statements are intended to qualify all forward-looking statements wherever they may appear in this Form 10-K. For allforward-looking statements, we claim protection of the safe harbor for the forward-looking statements contained in the Private Securities Litigation ReformAct of 1995. 1Table of ContentsThis Form 10-K also contains estimates, projections and other information concerning our industry, our business, and the markets for certaindiseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that isbased on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstancesmay differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, we obtained this industry, business,market and other data from reports, research surveys, studies and similar data prepared by market research firms and other third parties, industry, medical andgeneral publications, government data and similar sources.2Table of ContentsPart IItem 1. BusinessOverviewWe are an autologous cell and gene therapy company focused on translating personalized biologics into medical breakthroughs for diseasesaffecting the skin and connective tissue. Our distinctive approach to personalized biologics is based on our proprietary autologous fibroblast technology.Fibroblasts are the most common cell in skin and connective tissue and are responsible for synthesizing extracellular matrix proteins, including collagen andother growth factors, that provide structure and support. Because fibroblasts naturally reside in the localized environment of the skin and connective tissue,they represent an ideal delivery vehicle for proteins targeted to these areas. We target the underlying cause of disease by using fibroblast cells from a patient’sskin and genetically modifying them to create localized therapies that are compatible with the unique biology of the patient (i.e., which are autologous).We are focused on discovering and developing localized therapies for diseases affecting the skin and connective tissue, where there are high unmetneeds, to improve the lives of patients and their families. In that regard, we commit significant resources to our research and development programs.Currently, all of our research and development operations and focus are on gaining regulatory approvals to commercialize our product candidates in theUnited States; however, we may seek to expand into international markets in the future.Our current pipeline consists of the following product candidates which we are developing in collaboration with Intrexon Corporation (Intrexon): Our most advanced product candidate, FCX-007, has entered the Phase 2 portion of a Phase 1/2 clinical trial for the treatment of recessive dystrophicepidermolysis bullosa (RDEB). Our second gene therapy product candidate, FCX-013, is in development for the treatment of moderate to severe localizedscleroderma. We submitted an investigational new drug (IND) application for FCX-013 to the United States Food and Drug Administration (FDA) in January2018 and in March 2018, the FDA allowed the IND to progress to clinical trials. We expect to initiate enrollment for an open label, single arm Phase 1/2clinical trial in third quarter of 2018. In addition, we have a third program in the research phase for the treatment of arthritis and related conditions. Seefurther discussion of our gene therapy product candidates under the heading “Development Programs” included within section “Item 1—Business” of thisForm 10-K.3Table of ContentsOur StrategyOur strategy is to develop and commercialize transformational therapies for diseases affecting the skin and connective tissue to improve the lives ofpatients and their families. Key elements of our strategy are:•Leveraging our proprietary autologous fibroblast technology and patented manufacturing process;•Advancing our clinical stage gene therapy product candidate, FCX-007, through human clinical trials;•Advancing our gene therapy product candidate, FCX-013, into and through human clinical trials;•Advancing our research stage gene therapy program focused on arthritis and related conditions through research and into pre-clinical development;and•Leveraging our FDA-compliant current Good Manufacturing Practices (cGMP) manufacturing facility and our expertise in cell therapymanufacturing to advance the development of our autologous cell and gene therapy pipeline.Our PlatformOur proprietary autologous fibroblast technology is the foundation for creating personalized biologics for diseases of the skin and connective tissue.This technology uses a patented manufacturing process, which involves collecting small skin biopsies from patients, isolating cells and expanding them inculture, transducing the fibroblast cells with an integrative lentiviral vector to express a targeted protein, followed by continued expansion of the genemodified cells in culture. In this manner, each patient is treated with cells that were cultivated from his or her own dermal tissue (i.e., autologous).The Science of Autologous FibroblastsFibroblasts are the basis of our personalized biologics platform because they are the most common cell in skin and connective tissue and areresponsible for synthesizing extracellular matrix proteins, including collagen and other growth factors, that provide structure and support.Personalized Biologics Approach Because fibroblasts naturally reside in the localized environment of the skin and connective tissue, they represent an ideal delivery vehicle forproteins targeted to these areas. Utilizing our autologous fibroblast technology, we use a patient’s fibroblast cells to create localized gene therapies that arecompatible with the unique biology of the patient and have the potential to address the underlying cause of disease. 4Table of ContentsWe believe our personalized biologics approach provides the following distinct advantages for creating gene therapies:•Localized administration—avoids side effects typically associated with systemic therapy•Reduced rejection and immunogenicity concerns—because autologous fibroblasts are compatible with the unique biology of each patient•Fibroblast cells are genetically modified ex vivo—to enable testing for safety and confirmation of protein expression levels prior toadministration to the patient•Demonstrated expertise in manufacturing our fibroblast cell therapy We are developing all of our gene therapy product candidates in collaboration with Intrexon, a leader in synthetic biology. Through ourcollaboration with Intrexon, we have access to:•Intrexon’s proprietary vector technology, which is designed to facilitate the assembly and delivery of the necessary target gene constructs fordelivery to autologous fibroblast cells. Access to this technology allows us to rapidly screen and construct genetic therapeutic solutions.•Intrexon’s proprietary RheoSwitch Therapeutic System® (RTS®) technology. The RTS® biologic switch is activated by an orally-administeredcompound (veledimex) to control level and timing of protein expression in those diseases where such control is ideal.5Table of ContentsDevelopment ProgramsOur development programs are focused on diseases affecting the skin and connective tissue for which there are high unmet needs. Our programsconsist of the following:Program Potential Indication StatusFCX-007 RDEB Phase 1/2FCX-013 Moderate to severe localized scleroderma Post-INDResearch Program Arthritis ResearchFCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)RDEB is the most severe form of dystrophic epidermolysis bullosa (DEB), a congenital, progressive, devastatingly painful and debilitating geneticdisorder that often leads to death. RDEB is caused by a mutation of the COL7A1 gene, the gene which encodes for type VII collagen (COL7), a protein thatforms anchoring fibrils. Anchoring fibrils hold together the layers of skin, and without them, skin layers separate causing severe blistering, open wounds andscarring in response to friction, including normal daily activities like rubbing or scratching. Children who inherit this condition are often called “butterflychildren” because their skin can be as fragile as a butterfly’s wings. We estimate that there are approximately 1,100 - 2,500 RDEB patients in the U.S.Currently, treatments for RDEB address only the sequelae, including daily bandaging (which can cost a patient in excess of $10,000 per month), hydrogeldressings, antibiotics, feeding tubes and surgeries.Our lead product candidate, FCX-007, is in clinical development for the treatment of RDEB. FCX-007 is a genetically-modified autologousfibroblast that encodes the gene for COL7 for localized treatment of RDEB and is being developed in collaboration with Intrexon. By genetically modifyingautologous fibroblasts ex vivo to produce COL7, culturing them and then treating blisters and wounds locally via injection, FCX-007 offers the potential toaddress the underlying cause of the disease by providing high levels of COL7 directly to the affected areas, thereby avoiding systemic treatment. In addition,we believe the autologous nature of the cells, localized delivery, use of an integrative vector and the low turnover rate of the protein will contribute to long-term persistence of the COL7 produced by FCX-007.FCX-007 has received Orphan Drug Designation for the treatment of DEB, including RDEB, Rare Pediatric Disease Designation for the treatment ofRDEB and fast track designation for the treatment of RDEB from the FDA.Phase 1/2 Trial of FCX-007 for RDEBThe primary objective of this open-label trial is to evaluate the safety of FCX-007 in RDEB patients. Additionally, the trial will assess (i) thepharmacology of FCX-007 through the presence of vector DNA or COL7 mRNA evaluation of COL7 expression and/or the presence of anchoring fibrils and(ii) the efficacy of FCX-007 through intra-subject paired analysis of target wound areas by comparing FCX-007 treated wounds to untreated wounds in Phase1 and to wounds administered with sterile saline in Phase 2 through the evaluation of digital imaging of wounds. Prior to treating pediatric patients in thistrial, we were required to and obtained allowance from the FDA by submitting evidence of FCX-007 safety and benefit in the adult6Table of Contentspatients and data from our completed pre-clinical toxicology study. After submission of the requested data, the FDA granted allowance to include pediatricpatients in the clinical trial in January 2018.Four patients are enrolled in the Phase 1 portion of the clinical trial and continue to progress through follow-up study visits. In September 2017, wereported interim results from the Phase 1 portion of the Phase 1/2 clinical trial of FCX-007. Three adult NC1+ patients were dosed with a single intradermalinjection session of FCX-007 in the margins of and across targeted wounds, as well as in separate intact skin sites. Five wounds were treated on the threepatients, ranging in size from 4.4cm2 to 13.1cm2. Data from these patients show FCX-007 was well-tolerated through 12 weeks post-administration. Therewere no serious adverse events and no product related adverse events reported.Our study is currently designed to recruit two additional patients in the NC1- (patients who do not express the first non-collagenous portion of theCOL7 protein) cohort of the Phase 1 portion of the clinical trial. The most recent scientific thinking and the rarity of NC1- patients compared to NC1+, i.e.,the difficulty in recruiting this group, we are seeking a modification in the protocol to close out the Phase 1 cohort of this clinical trial without including anyadditional patients. The clinical trial protocol is designed to allow a cohort to move into the Phase 2 portion of the trial even if the other cohort is stillenrolling or in the follow-up evaluation period; therefore, the modification of the protocol for the trial does not impact ongoing enrollment of Phase 2patients. As data is collected, progress will be reported.The targeted wounds of the three adult patients were evaluated during a monitoring period prior to dosing and were observed to be open for up toeight months. Compared to the baseline measurement collected at Day 0 before the single intradermal injection session of FCX-007, at four weeks post-administration 100% (5/5) of wounds were ≥ 75% healed. At 12 weeks post-administration, 80% (4/5) of wounds were ≥ 70% healed. The wound that was <70% healed from the 12 week data set was biopsied by the investigator in the middle of the wound bed rather than on the wound edge, which we believe mayhave contributed to the wound’s instability. We also monitored and evaluated untreated wounds of similar size to the matched treated wounds on the samepatient. Compared to the baseline measurement collected at Day 0, at four weeks and 12 weeks post-administration 80% (4/5) of wounds were < 25% healed.We will continue to monitor all treated and untreated wounds throughout follow-up visits.Various pharmacology signals for vector DNA, COL7 mRNA, or COL7 protein expression were detected throughout the data set in each patient forone or more assays up to 12 weeks post-administration (qPCR, electron microscopy or immunofluorescence). Anchoring fibrils have not been detected todate, whereas expressed COL7 mRNA and COL7 protein have been confirmed in multiple patient samples including one that detected linear expression ofCOL7 at the basement membrane zone. The Data Safety Monitoring Board (DSMB) for the trial reviewed the interim data and concluded that safety andpotential benefit were established, and allowed continuation of enrollment and dosing.We completed dosing of the fourth adult patient and performed additional dosing of existing adult patients in the Phase 1 portion of the trial in thefourth quarter of 2017. We expect to report additional interim adult data and provide a trial update in the second quarter of 2018, which includes presentingat the 7th International Investigative Dermatology meeting in May 2018.We plan to enroll six patients ages seven and older in the Phase 2 portion of the clinical trial. One RDEB adult patient has been enrolled as the firstpatient in Phase 2 and dosing of this patient is expected to occur in the second quarter of 2018. With the allowance from the FDA, we will now includeenrollment of pediatric patients. We expect to complete enrollment of Phase 2 patients in the third quarter of 2018. We plan to report another interim datareadout and trial update in the first quarter of 2019.We plan to use the existing data from the Phase 1 portion of the Phase 1/2 clinical trial to also support a petition for Regenerative MedicineAdvanced Therapy or Breakthrough Therapy Designation for FCX-007.We have designated our existing, cGMP cell therapy manufacturing facility in Exton, PA as the production site for FCX-007 in our IND application.FCX-007 drug product dosed in the fourth quarter of 2017 was produced and distributed from our Exton, PA facility. The facility will be used for theremaining clinical and future commercial manufacture of FCX-007, with capacity to serve the U.S. market for RDEB. The approximately 13,000 square footfacility previously supported commercial autologous fibroblast manufacturing, with multiple FDA inspections conducted at the site. The facility includescleanroom cell therapy manufacturing, quality control testing, cryogenic storage, shipping/receiving and warehousing space.7Table of ContentsFCX-013 for Moderate to Severe Localized SclerodermaLocalized scleroderma is a chronic autoimmune skin disorder that manifests as excess production of extracellular matrix, specifically collagen,resulting in thickening of the skin and connective tissue. Localized scleroderma encompasses several subtypes which are classified based on the depth andpattern of the lesion(s). The moderate to severe forms of the disorder include any subtype that affects function or produces symptoms of discomfort, tightnessand pain. Current treatments for localized scleroderma include systemic or topical corticosteroids which target inflammation, UVA light therapy and physicaltherapy. There are few treatment options to address excessive collagen accumulation in the skin and connective tissue. We estimate that there areapproximately 90,000 patients in the U.S. considered to have moderate to severe localized scleroderma.Our second gene therapy product candidate, FCX-013, is in development for the treatment of moderate to severe localized scleroderma. FCX-013 isan autologous fibroblast genetically-modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), the protein responsible for breakingdown collagen. FCX-013 incorporates Intrexon’s proprietary RheoSwitch Therapeutic System® (RTS®), a biologic switch activated by an orally administeredcompound (veledimex) to control protein expression at the site of localized scleroderma lesions. FCX-013 is designed to be injected under the skin at thelocation of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation. With theFCX-013 therapy, the patient will take an oral compound (veledimex) to facilitate protein expression. Once the fibrosis is resolved, the patient will stoptaking the oral compound which will halt further MMP-1 production.We previously completed a proof-of-concept study for FCX-013 in which the primary objective was to determine whether FCX-013 had thepotential to reduce dermal thickness in fibrotic tissue. In this study, FCX-013 was evaluated in a bleomycin-induced scleroderma model utilizing severecombined immunodeficiency (SCID) mice. Data from the study demonstrated that FCX-013 reduced dermal thickness of fibrotic tissue to levels similar tothat of the non-bleomycin treated control and further reduced the thickness of the sub-dermal muscle layer. Based upon these data and the FDA’s feedback toour pre-IND briefing package, we advanced FCX-013 into a pre-clinical dose-ranging study which has been completed.In December 2017, we completed a good laboratory practice (GLP) toxicology/biodistribution study that assessed FCX-013 in a bleomycin fibrosismodel using immunocompromised (NOD/SCID) mice. Data from this study showed no test article-related clinical observations, body weight changes,changes in clinical pathology parameters, gross observations or organ weight change. In addition, there was no significant vector biodistribution to targetorgans.We submitted an IND for FCX-013 to FDA in January 2018, and in March 2018, the FDA allowed the IND to progress to clinical trials. We expect toinitiate enrollment for an open label, single arm Phase 1/2 clinical trial in the third quarter of 2018. The primary objective of the trial is to evaluate the safetyof FCX-013. Secondary analyses consist of several fibrosis assessments including histology, skin scores, ultrasound and additional measurements of targetedsclerotic lesions and control sites at various time points up to 16 weeks post-administration of FCX-013. Ten patients with any subtype of localizedscleroderma are targeted for enrollment (approximately 5 patients per Phase). The Phase 1 portion will enroll adult patients, and dosing for the first three adultpatients will be staggered prior to dosing the rest of the trial’s population. Fibrocell intends to include pediatric patients in the Phase 2 portion of the trialafter submission and approval of safety and activity data from the adult Phase 1 patients to the FDA and the DSMB for the trial. We plan to manufacture FCX-013 at our Exton, PA cGMP manufacturing facility.FCX-013 has received Orphan Drug Designation from the FDA for the treatment of localized scleroderma and Rare Pediatric Disease Designation formoderate to severe localized scleroderma.Gene Therapy Research Program for Arthritis and Related ConditionsArthritis is a broad term that covers a group of more than 100 different types of diseases that affect the joints, as well as connective tissues andorgans, including the skin. According to the Centers for Disease Control and Prevention, arthritis—characterized by joint inflammation, pain and decreasedrange of motion—is the United States’ most common cause of disability affecting more than 52 million adults as well as 300,000 children at a cost exceeding$120 billion.Our third gene therapy program is in the research phase and is focused on the treatment of arthritis and related conditions. Our goal is to deliver aprotein therapy locally to the joint to provide sustained efficacy while avoiding key side effects typically associated with systemic therapy.8Table of ContentsIntrexon Collaborations2012 Exclusive Channel Collaboration Agreement (2012 ECC)In October 2012, we entered into an Exclusive Channel Collaboration Agreement, with Intrexon, which was amended in June 2013 and January2014 (as amended, the 2012 ECC) pursuant to which we are Intrexon’s exclusive channel collaborator in the research, development and commercialization ofproducts in the following areas (the 2012 Fields):•the enhanced production and purification of autologous fibroblasts (without genetic modification) for all aesthetic and therapeutic indications;•the enhanced production and purification of autologous dermal cells (without genetic modification) for aesthetic and therapeutic treatment ofdermal, vocal cord, and periodontal indications;•the development of genetically modified autologous fibroblasts for all aesthetic and therapeutic indications where an autologous fibroblastitself is the principal effector of the product in contrast to the use of autologous fibroblasts as the source of expression of a systemicallyavailable therapeutic protein in which that protein (and not the fibroblast) is the principal therapeutic effector;•the development of genetically modified autologous dermal cells for aesthetic and therapeutic treatment of dermal, vocal cord, and periodontalindications;•autologous fibroblasts genetically modified to express a therapeutic protein and/or bioactive ribonucleic acid for the treatment of autoimmuneand non-infectious inflammatory disorders that manifest in cutaneous tissues, fascia and/or muscle; and•autologous human fibroblasts with gene therapy to express bioactive Tenascin-X locally to correct connective tissue disorders associated withEhlers-Danlos Syndrome (hypermobility type).Pursuant to the terms of the 2012 ECC, Intrexon has granted us a license to use its proprietary technologies and other intellectual property toresearch, develop and commercialize products in the 2012 Fields within the United States. We are responsible for all costs incurred in connection with theresearch, development and commercialization of products under the 2012 ECC and own all clinical data, regulatory filings and regulatory approvals relatingto such products. We engage Intrexon for support services for the research and development of products under the 2012 ECC, and reimburse Intrexon for itscost for time and materials for such services. We are required to pay Intrexon quarterly cash royalties on all products developed under the 2012 ECC in an amount equal to 7% of aggregatequarterly net sales up to $25 million, plus 14% on aggregate quarterly net sales greater than $25 million. We are also required to pay Intrexon half of anysublicensing revenues we receive from third parties in consideration for sublicenses granted by us with respect to products developed under the 2012 ECC,but only to the extent such sublicensing revenues are not included in net sales subject to royalties. Sales from other products that we develop andcommercialize outside of the 2012 ECC are not subject to royalty payments unless we are able to reduce the product’s cost of goods sold through the 2012ECC, in which case, we are required to pay quarterly cash royalties on such products equal to one-third of the cost of goods sold savings less any suchsavings developed by us outside of the 2012 ECC. The 2012 ECC may be terminated by Intrexon if we fail to exercise diligent efforts in developing products through the collaboration or if we electnot to pursue the development of a therapy identified by Intrexon within the 2012 Field and that qualifies as a “Superior Therapy” as defined in the 2012ECC. Upon such termination, the products covered by the 2012 ECC in active and ongoing Phase II clinical trials or later stage development shall beentitled to be continued by us with a continuation of the related milestone, royalty and other payment obligations for such products, and all rights toproducts covered by the 2012 ECC still in an earlier stage of development shall revert to Intrexon.In September 2015, we and Intrexon entered into a letter of agreement pursuant to which we mutually agreed to terminate our collaboration withrespect to the development of potential therapies to treat Ehlers-Danlos Syndrome (hypermobility type) due to technical hurdles. As a result, we no longerhave any rights or obligations under the 2012 ECC with respect to the development of “autologous human fibroblasts genetically modified to expressbioactive Tenascin-X locally to correct connective tissue disorders”.Currently, we are in development of two gene therapy product candidates, FCX-007 and FCX-013, under the 2012 ECC, as more fully describedunder the heading “Development Programs” within “Item 1—Business” of this Form 10-K.9Table of Contents2015 Exclusive Channel Collaboration Agreement (2015 ECC)In December 2015, we entered into an additional Exclusive Channel Collaboration Agreement with Intrexon (the 2015 ECC) pursuant to which weare Intrexon’s exclusive channel collaborator in the research, development and commercialization of products for the treatment of chronic inflammation anddegenerative diseases of human joints through intra-articular or other local administration of genetically-modified fibroblasts (the 2015 Field). Thecollaboration leverages our autologous fibroblast technology with Intrexon’s synthetic biology technology to identify and develop cell-based therapeuticsthat will be genetically modified to express one or more proteins at sites of joint inflammation. We believe this treatment approach has the potential toovercome the limitations of existing therapies for chronic inflammation and degenerative diseases of the joint, including arthritis and related conditions.Pursuant to the terms of the 2015 ECC, Intrexon has granted us a license to use its proprietary technologies and other intellectual property todevelop and commercialize products in the 2015 Field throughout the world. We are responsible for all costs incurred in connection with the research,development and commercialization of products under the 2015 ECC and own all clinical data, regulatory filings and regulatory approvals relating to suchproducts. We engage Intrexon for support services in connection with the research and development of products under the 2015 ECC, and reimburse Intrexonfor its cost for time and materials for such services.In consideration for the license and the other rights that we receive under the 2015 ECC, we paid Intrexon an up-front technology access fee of $10million in cash in January 2016. For each product that we develop under the 2015 ECC, we are required to pay Intrexon development milestones of up to $30million for the first product developed under the 2015 ECC (and development milestones up to $55 million for each subsequent product developed under the2015 ECC) and commercialization milestones of up to $22.5 million, for each product developed, a low double-digit royalty on our net sales of suchproducts and half of any sublicensing revenues we receive from third parties in consideration for sublicenses granted by us with respect to such products butonly to the extent such sublicensing revenues are not included in net sales subject to royalties.The 2015 ECC may be terminated by Intrexon if we fail to exercise diligent efforts in developing products through the collaboration or if we electnot to pursue the development of a therapy identified by Intrexon within the 2015 Field and that qualifies as a “Superior Therapy” as defined in the 2015ECC. Upon such termination, the products covered by the 2015 ECC in active and ongoing Phase II clinical trials or later stage development shall beentitled to be continued by us with a continuation of the related milestone, royalty and other payment obligations for such products, and all rights toproducts covered by the 2015 ECC still in an earlier stage of development shall revert to Intrexon.To date, we have only conducted research for a gene therapy product candidate for arthritis and related conditions under the 2015 ECC. We havedeferred further development under the 2015 ECC in order to focus our efforts and our resources on our ongoing development of FCX-007 and FCX-013.ManufacturingWe lease and operate our own manufacturing facility located in Exton, Pennsylvania. We have historically used this facility to manufacture our non-genetically modified products and during 2016 began using this facility for pre-clinical manufacturing of our gene therapy product candidate, FCX-013. Wedesignated our Exton, PA cGMP manufacturing facility in Exton, PA as the production site for FCX-007 in the fourth quarter of 2017, and are currentlyproducing the drug product for the FCX-007 clinical trial at that facility. We also plan to manufacture FCX-013 at the same location. Previously weoutsourced certain manufacturing of our genetically-modified product candidate, FCX-007, to a contract manufacturer with a facility located in MountainView, California. We adhere to FDA cGMP regulations. We believe that we have adequate manufacturing capacity to satisfy our pre-clinical and clinicaldemands.The fibroblast cells that constitute our product candidates are cultured by our proprietary cGMP manufacturing process, beginning with thecollection of skin biopsies from the patient’s skin. Fibroblasts are extracted from the biopsies and cultured using standard culture techniques to increase thecell population. A viral transduction is then performed to introduce targeted genes to the cells. The fibroblasts are then further expanded and cryopreservedfor storage. When a treatment is requested, the cells are thawed, washed and prepared for shipment.All component parts, including raw materials and other supplies utilized in our manufacturing process are available from various third partysuppliers and manufacturers in quantities adequate to meet our needs. We seek to ensure continuity of supply of such component parts, raw materials andsupplies using a strategy of dual sourcing, where possible. Some of our raw materials are currently sourced from one vendor; however, alternate vendors areavailable should they be required, although we would need sufficient lead time to qualify those vendors. 10Table of ContentsWe use certain hazardous chemicals and biological materials in our manufacturing process which are subject to a variety of federal, state and locallaws and regulations governing, among other matters, the use, generation, manufacture, transportation, storage, handling, disposal of and human exposure tothese materials, including regulation by governmental regulatory agencies, such as the Occupational Safety and Health Administration and the U.S.Environmental Protection Agency. We incur capital and operating expenditures and other costs in the ordinary course of our business in complying withthese laws and regulations. We dispose of minimal hazardous biological waste as a result of our manufacturing process.Intellectual PropertyWe believe that patents, trademarks, copyrights and other proprietary rights are important to our business. We also rely on trade secrets, know-howand continuing technological innovations to develop and maintain our competitive position. We seek to protect our intellectual property rights by a varietyof means, including obtaining patents, maintaining trade secrets and proprietary know-how and technological innovation to operate without infringing onthe proprietary rights of others and to prevent others from infringing on our proprietary rights. As of December 31, 2017, we own or license 11 issued U.S. patents, 8 pending U.S. patent applications, 1 granted foreign patent, 1 pendinginternational patent and 14 pending foreign patent applications. Our issued patents and patent applications primarily cover the method of using autologouscell fibroblasts for the repair of skin and soft tissue defects and the use of autologous fibroblast cells for tissue regeneration. In particular, we own an issuedpatent in the U.S. that is directed to methods of long-term augmentation of subcutaneous or dermal tissue by injecting an effective amount of a suspension ofautologous passaged dermal fibroblasts into subadjacent tissue, which is set to expire in July 2020. In addition, we own an issued U.S. patent, an issuedAustralian patent, and pending applications in Canada, China, Europe, India, Japan, South Korea, Hong Kong and the U.S. directed to dosage formulationsfor injection containing particular amounts of autologous human fibroblasts and uses thereof, which naturally expire in 2030 and 2031. We also ownpending applications in the U.S. and several foreign countries related to topical formulations of autologous dermal fibroblasts and uses thereof, the earliest ofwhich, if issued, would naturally expire in 2027. We have also in-licensed from Intrexon Corporation an international application directed to compositionsand methods for treating Type VII collagen deficiencies which, if issued, would expire in 2037. We have also in-licensed from Intrexon Corporation U.S.provisional applications directed to delivery of autologous cells comprising matrix metalloproteinase-1 for treating scleroderma which, if a non-provisionalapplication would issue therefrom, would expire in 2038.CompetitionThere is significant competition in the biopharmaceutical industry which can be attributed to companies ranging from small specializedbiotechnology firms to large well-established pharmaceutical companies. More specifically, there are many companies currently competing in drugdevelopment for new therapies for the treatment of diseases affecting the skin, connective tissue and joints, our focus area. Some of our competitors havesubstantially greater financial resources and larger research and development organizations. In addition, our experience in clinical trials, obtaining FDA andother regulatory approvals, manufacturing and commercialization of products may be more limited. Product competition is based on a variety of factors, including but not limited to: product safety, efficacy, convenience of dosing, availability, price,as well as brand recognition. Our product candidates, if approved for commercial use, will contend with treatments offered by our competitors. Although webelieve the autologous nature and localized treatment approach of our product candidates provide advantages over our competitors, existing and newtreatments may also possess certain advantages. Additionally, the development of other drug technologies and methods of treating diseases are occurring at arapid pace. These developments may render our products or technologies obsolete or noncompetitive. Currently, we believe the primary competitors for ourproduct candidates are as follows:FCX-007 for RDEB. Our product candidate FCX-007 is being developed for the treatment of RDEB. Current treatments for RDEB, which includebandaging, antibiotics, feeding tubes, and surgery (hand and esophageal), only address the symptoms of this disorder. There are currently no productsapproved by the FDA for the treatment of RDEB. We are aware of a potentially competing product candidate, EB-101, which is a genetically-modifiedkeratinocyte graft being developed by Abeona Therapeutics (Abeona). Following presentation of Phase 1/2 clinical data at the Society of InvestigativeDermatology conference in 2017, Abeona received guidance from the FDA to initiate a Pivotal Phase 3 trial in 2018. In addition, we are aware of severalother products in development for the treatment of various forms of epidermolysis bullosa (including DEB and RDEB). 11Table of ContentsFCX-013 for Moderate to Severe Localized Scleroderma. Our product candidate FCX-013 is being developed for the treatment of moderate to severelocalized scleroderma. Current treatments for localized scleroderma include systemic or topical corticosteroids which target inflammation, UVA light therapy,and physical therapy. There are few treatment options to address excessive collagen accumulation in the skin and connective tissue. There are currently noproducts approved by the FDA for the treatment of localized scleroderma. We are aware of a potentially competing product, ECCS-50 Cellular Therapy,which is being developed by Cytori Therapeutics (Cytori), for the treatment of systemic scleroderma that affects the hands. Cytori recently released data fromits clinical trial and is working with statisticians and study investigators in preparation for publication of these results in a peer-reviewed journal. We are alsoaware that miRagen Therapeutics has a product candidate, MRG-201, which utilizes microRNA biology and is in a Phase 1 clinical trial for the treatment ofsystemic and localized scleroderma.Research and DevelopmentWe expense research and development costs as they are incurred. For the years ended December 31, 2017 and 2016, we incurred total research anddevelopment expenses of approximately $12.2 million and $12.1 million, respectively. Additionally, for the year ended December 31, 2016, we incurredexpenses of less than $0.1 million related to a research and development agreement that we have with a third party to investigate potential new non-pharmaceutical applications for our conditioned fibroblast media technology. Expenses pertaining to this research and development agreement are classifiedunder the caption “Cost of collaboration revenue” in the Consolidated Statements of Operations.Government RegulationWe are subject to extensive government regulation, principally by the FDA and state and local authorities in the United States and by comparableagencies in foreign countries. Governmental authorities in the United States extensively regulate the pre-clinical and clinical testing, safety, efficacy,research, development, manufacturing, labeling, storage, record-keeping, advertising, promotion, import, export, marketing and distribution, among otherthings, of pharmaceutical and biologic products under various federal laws including the Federal Food, Drug and Cosmetic Act (FFDCA), the Public HealthService Act (PHSA) and under comparable laws by the states and in most foreign countries.Domestic Regulation In the United States, the FDA, under the FFDCA, the PHSA, and other federal statutes and regulations, subjects pharmaceutical and biologic productsto rigorous review. If we do not comply with applicable requirements, we may be subjected to various enforcement actions, such as product seizures andcourt injunctions, the government may refuse to approve our marketing applications, and we could even be criminally prosecuted in certain circumstances. The FDA also has the authority to suspend or revoke a Biologics License Application (BLA), issue adverse publicity, and take other measures if we fail tocomply with applicable regulatory standards.FDA Approval ProcessTo obtain approval of a new drug product from the FDA, we must, among other requirements, submit data demonstrating its safety, purity, andpotency, which includes efficacy, as well as detailed information on the manufacture and composition of the product candidate. In most cases, this entailsextensive laboratory tests and pre-clinical and clinical trials. This testing and the preparation of necessary applications and processing of those applicationsby the FDA are expensive and typically take many years to complete. The FDA may deny our applications or may not act quickly or favorably in reviewingthese applications, and we may encounter significant difficulties or costs in our efforts to obtain FDA approvals that could delay or preclude us frommarketing any products we may develop. The FDA also may require post-marketing testing and surveillance to monitor the effects of approved products orplace conditions on any approvals that could restrict the commercial applications of these products. Regulatory authorities may withdraw product approvalsif we fail to comply with regulatory standards or if we encounter problems following initial marketing. With respect to patented products or technologies,delays imposed by the governmental approval process may materially reduce the period during which we may have the exclusive right to exploit theproducts or technologies.The FDA does not apply a single regulatory scheme to human cells and tissues and products derived from human cells and tissue. On a product-by-product basis, the FDA may regulate such products as drugs, biologic products, or medical devices, in addition to regulating them as human cells, tissues, orcellular or tissue-based products (HCT/Ps), depending on whether or not the particular product triggers any of an enumerated list of regulatory factors. Afundamental difference in the treatment of products under these classifications is that the FDA permits certain HCT/Ps, sometimes referred to as section 361HCT/Ps, that do not trigger any of those regulatory factors to be commercially distributed without regulatory approval. In12Table of Contentscontrast, HCT/P products that trigger those factors, such as if they are more than minimally manipulated, are regulated as drugs, biologics, or medical devicesand require FDA approval prior to commercial distribution. We have determined that our product candidates trigger regulatory factors that make thembiologic products, in addition to HCT/Ps, and consequently, we must obtain approval from the FDA before marketing such products and must also satisfy allregulatory requirements for HCT/Ps.The process required by the FDA before a new drug or biologic product may be marketed in the United States generally involves the following:•completion of pre-clinical laboratory tests or studies and formulation studies;•submission to the FDA of an IND application for a new drug or biologic product, which must become effective before human clinical trials maybegin;•performance of adequate and well-controlled human clinical trials to establish the safety and efficacy of the proposed drug, or safety, purity, andpotency of the proposed biologic product for its intended use;•detailed information on product characterization and manufacturing process; and•submission and approval of a New Drug Application (NDA) for a drug, or a BLA for a biologic product.Pre-clinical tests include laboratory evaluation of product chemistry formulation and stability, as well as animal and other studies to evaluatetoxicity. Under FDA regulations, the results of any pre-clinical testing, together with manufacturing information and analytical data, are submitted to theFDA as part of an IND application. The FDA requires a 30-day waiting period after the filing of each IND application before clinical trials may begin, in orderto ensure that human research patients will not be exposed to unreasonable health risks. At any time during this 30-day period or at any time thereafter, theFDA may halt proposed or ongoing clinical trials, may authorize trials only on specified terms, or may require additional trials. The IND application processmay become extremely costly and substantially delay development of our products. Moreover, positive results of pre-clinical tests will not necessarilyforecast positive results in clinical trials. The sponsor typically conducts human clinical trials in three sequential phases, which may overlap. These phases generally include the following:•Phase 1: The product candidate is usually first introduced into healthy humans or, on occasion, into patients, and is tested for safety, dosagetolerance, absorption, distribution, excretion and metabolism;•Phase 2: The product candidate is introduced into a limited patient population to:•assess its efficacy in specific, targeted indications;•assess dosage tolerance and optimal dosage; and•identify possible adverse effects and safety risks.•Phase 3: These are commonly referred to as pivotal studies. If a product candidate is found to have an acceptable safety profile and to bepotentially effective in Phase 2 clinical trials, clinical trials in Phase 3 will be initiated to further demonstrate clinical efficacy, optimal dosageand safety within an expanded and diverse patient population at geographically dispersed clinical trial sites; and•If the FDA does ultimately approve the product candidate, it may require post-marketing testing, including potentially expensive Phase 4studies, to confirm or further evaluate its safety and effectiveness. Continued ability to commercialize the product may be based on thesuccessful completion of these additional studies.Before proceeding with a trial, the sponsor may seek a written agreement from the FDA regarding the design, size, and conduct of a clinical trial. This is known as a Special Protocol Assessment (SPA). Among other things, SPAs can cover clinical trials for pivotal studies whose data will form theprimary basis to establish a product’s efficacy. SPAs thus help establish up-front agreement with the FDA about the adequacy of a clinical trial design tosupport a regulatory approval, but the agreement is not binding if new circumstances arise. Even if the FDA agrees to a SPA, the agreement may be changedby the sponsor or the FDA on written agreement by either parties, or if a senior FDA official determines that a substantial scientific issue essential todetermining the safety or effectiveness of the product was identified after the testing began. There is no guarantee that a study will ultimately be adequate tosupport an approval, even if the study is subject to a SPA. The FDA retains significant latitude and discretion in interpreting the terms of the SPA and thedata and results from any study that is the subject of the SPA. The FDA may revoke or alter its SPA agreement under the following circumstances:13Table of Contents•a substantial scientific issue essential to determining the safety or efficacy of the drug has been identified after testing has begun;•the protocol that was agreed upon with the FDA has not been followed by a sponsor;•the relevant data, assumptions, or information provided by a sponsor in a request for a SPA change are found to be false or misleading, or arefound to exclude relevant facts; or•the FDA and sponsor agree in writing to modify the protocol and such modification is intended to improve the study.Clinical trials must meet requirements for Institutional Review Board (IRB) oversight, patient informed consent and the FDA’s Good ClinicalPractice (GCP) requirements. Prior to commencement of each clinical trial, the sponsor must submit to the FDA a clinical plan, or protocol, accompanied bythe approval of the committee responsible for overseeing clinical trials at the clinical trial sites. The FDA or the IRB at each institution at which a clinicaltrial is being performed may order the temporary or permanent discontinuation of a clinical trial at any time if it believes that the clinical trial is not beingconducted in accordance with FDA requirements or presents an unacceptable risk to the clinical trial patients. Data safety monitoring boards, which monitorcertain studies to protect the welfare of study patients, may also require that a clinical trial be discontinued or modified.The sponsor must submit to the FDA the results of the pre-clinical and clinical trials, together with, among other things, detailed information on themanufacturing and composition of the product, and proposed labeling, in the form of an NDA, or, in the case of a biologic product, a BLA. The applicantmust also submit with the NDA or BLA a substantial user fee payment, unless a waiver or reduction applies. In some cases, a sponsor may be able to expandthe indications in an approved NDA or BLA through a submission of a Prior Approval Supplement. Each NDA or BLA submitted for FDA approval is usuallyreviewed for administrative completeness and reviewability within 60 days following submission of the application. If deemed complete, the FDA will “file”the NDA or BLA, thereby triggering substantive review of the application. The FDA can refuse to file any NDA or BLA that it deems incomplete or notproperly reviewable. Once the submission has been accepted for filing, the FDA will review the application and will usually respond to the applicant inaccordance with performance goals the FDA, industry and Congress have negotiated for the review of NDAs and BLAs. For NDAs for new molecular entitydrugs and for original BLA submissions, the review period is currently six months from the filing date of the application for priority applications and tenmonths from the filing date for standard applications. The review process is often significantly extended by FDA requests for additional information, pre-clinical studies or clinical trials, or clarification, or by changes to the application submitted by the applicant in the form of amendments. The FDA may referapplications for novel product candidates which present difficult questions of safety or efficacy to an advisory committee, typically a panel that includesclinicians and other experts, for review, evaluation, and a recommendation as to whether the application should be approved and under what conditions. TheFDA is not bound by the recommendations of an advisory committee, but it considers such recommendations carefully when making decisions.Before approving an NDA or BLA, the FDA generally inspects the facilities at which the product is manufactured and will not approve the productunless the manufacturing facilities are in compliance with cGMP requirements which govern the manufacture, holding and distribution of a product. Manufacturers of human cellular or tissue-based biologics also must comply with the FDA’s Good Tissue Practices, as applicable, and the general biologicproduct standards. It is possible that our product candidates will not successfully proceed through this approval process or that the FDA will not approve them in anyspecific period of time, or at all. The FDA may deny or delay approval of applications that do not meet applicable regulatory criteria, or if the FDAdetermines that the clinical data do not adequately establish the safety and efficacy of the product. Satisfaction of FDA pre-market approval requirements fora new biologic product is a process that may take a number of years and the actual time required may vary substantially based upon the type, complexity andnovelty of the product or disease. The FDA reviews these applications and, when and if it decides that adequate data are available to show that the product isboth safe and effective and that other applicable requirements have been met, approves the drug or biologic product for marketing. Government regulationmay delay or prevent marketing of potential products for a considerable period of time and imposes costly procedures upon our activities. Success in earlystage clinical trials does not assure success in later stage clinical trials. Data obtained from clinical activities are not always conclusive and may besusceptible to varying interpretations that could delay, limit or prevent regulatory approval. Upon approval, a product candidate may be marketed only forthose indications approved in the NDA or BLA and will be subject to labeling and promotional requirements or limitations, including warnings, precautions,contraindications and use limitations, which could materially impact profitability. Once approved, the FDA may withdraw the product approval ifcompliance with pre- and post-market regulatory standards and requirements are not maintained or if safety, efficacy or other problems occur after the productreaches the marketplace.14Table of ContentsThe FDA may, during its review of an NDA or BLA, ask for additional study data. If the FDA does ultimately approve the product, approval may besubject to limitations based on the FDA’s interpretation of the existing pre-clinical and clinical data and the FDA may require post-marketing testing,including potentially expensive Phase IV studies, to confirm or otherwise further evaluate the safety and effectiveness of the product. The FDA also mayrequire, as a condition to approval or continued marketing of a drug, a risk evaluation and mitigation strategy (REMS) to ensure that the benefits of a drug orbiologic product outweigh its risks. A REMS can include additional educational materials for healthcare professionals and patients such as MedicationGuides and Patient Package Inserts, a plan for communicating information to healthcare professionals, and restricted distribution of the product. In addition,the FDA may, in some circumstances, impose restrictions on the use of the product, which may be difficult and expensive to administer and may require priorapproval of promotional materials. Following approval, the FDA may require labeling changes or impose new post-approval study, risk management, ordistribution restriction requirements.The FDA has developed several programs intended to expedite the development and review of drugs that address unmet medical needs for serious orlife threatening conditions, especially when the drugs are the first available treatment or have advantages over existing treatments:•Accelerated Approval. The FDA may grant accelerated approval to drugs or biologic products that treat serious or life-threatening illnesses andthat provide meaningful therapeutic benefits to patients over existing treatments. Under this program, the FDA may approve a product based onsurrogate endpoints or clinical endpoints that can be measured earlier than mortality or irreversible morbidity. When approval is based onsurrogate endpoints or clinical endpoints that can be measured earlier than mortality or irreversible morbidity, the sponsor will be required toconduct additional post-approval clinical trials to verify and describe clinical benefit. Under the agency’s accelerated approval regulations, ifthe FDA concludes that a product that has been shown to be effective can be safely used only if distribution or use is restricted, it may requirecertain post-marketing restrictions as necessary to assure safe use. In addition, for products approved under accelerated approval, sponsors willbe required to submit all copies of their promotional materials, including advertisements, to the FDA at least thirty days prior to initialdissemination unless otherwise informed by the FDA. After a hearing, the FDA may withdraw a previously granted accelerated approval if, forinstance, post-marketing studies fail to verify any clinical benefit, it becomes clear that restrictions on the distribution of the product areinadequate to ensure its safe use, or if a sponsor fails to comply with the conditions of the accelerated approval.•Breakthrough Therapy. The FDA may grant “breakthrough therapy” status to drugs or biologic products designed to treat, alone or incombination with another drug(s) or biologic(s), a serious or life-threatening disease or condition and for which preliminary evidence suggests asubstantial improvement on clinically-meaningful endpoints over existing therapies. Such products need not address an unmet need, but arenevertheless eligible for expedited review if they offer the potential for an improvement over existing therapies. Breakthrough therapy statusentitles the sponsor to earlier and more frequent meetings with the FDA regarding the development of nonclinical and clinical data and permitsthe FDA to offer product development or regulatory advice for the purpose of potentially shortening the time to product approval. The FDA mayrescind breakthrough therapy designation if it believes the designated product no longer meets the qualifying criteria. Breakthrough therapystatus does not guarantee that a product will be developed or reviewed more quickly and does not ensure FDA approval.•Fast Track. The FDA may grant “fast track” status to drugs or biologic products that are intended to treat serious diseases or illness anddemonstrate the potential to fill an unmet medical need. Fast track is a process designed to expedite the review of such products by providing,among other things, more frequent meetings with the FDA to discuss the product’s development plan, more frequent written correspondencefrom the FDA about trial design, potential eligibility for accelerated approval, and rolling review, which allows submission of individuallycompleted sections of a NDA or BLA for the FDA’s review before the entire filing is completed. Fast track status does not ensure that a productwill be developed more quickly or receive FDA approval more quickly, if at all.•Priority Review. The FDA may grant “priority review” status to products that, if approved, would be significant improvements in safety oreffectiveness of the treatment, diagnosis or prevention of serious conditions. Priority review is intended to reduce the time it takes for the FDAto review an NDA or BLA.•Regenerative Medicine Advanced Therapy. A product may be eligible for regenerative medicine advanced therapy (RMAT) designation if:a.The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell andtissue product, or any combination product using such therapies or products, except15Table of Contentsfor those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations;b.The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; andc.Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or conditionAdvantages of the RMAT designation include all of the benefits of the fast track breakthrough designation programs, including early interactionswith sponsors.Additionally, there are various incentives to support development and approval of certain product candidates, including, but is not limited to,orphan drug designation and rare pediatric disease designation.Orphan Drug DesignationUnder the U.S. Orphan Drug Act, the FDA may grant orphan drug designation to drugs or biologic products intended to treat a “rare disease orcondition,” which is generally defined as having a prevalence of less than 200,000 individuals in the U.S. Orphan drug designation must be requested beforesubmitting an NDA or BLA for the product. Orphan drug designation does not shorten the regulatory review and approval process, nor does it provide anyadvantage in the regulatory review and approval process. However, if an orphan drug later receives approval for the indication for which it has designation,the relevant regulatory authority may not approve any other applications to market the same drug for the same indication, except in very limitedcircumstances, for seven years in the U.S. Although obtaining approval to market a product with orphan drug exclusivity may be advantageous, we cannot becertain:•that we will be the first to obtain approval for any drug for which we obtain orphan drug designation;•that orphan drug designation will result in any commercial advantage or reduce competition; or•that the limited exceptions to this exclusivity will not be invoked by the relevant regulatory authority.Additionally, orphan drug exclusive marketing rights may be lost under certain conditions, such as if the request for designation was materiallydefective or if the manufacturer is unable to assure sufficient quantity of the drug.FCX-007 and FCX-013 have received Orphan Drug Designation from the FDA, for the treatment of DEB (including RDEB) and localizedscleroderma, respectively.Rare Pediatric Disease DesignationFCX-007 has received Rare Pediatric Disease Designation from the FDA for the treatment of RDEB and FCX-013 has received Rare Pediatric DiseaseDesignation from the FDA for the treatment of moderate to severe localized scleroderma. The FDA generally defines a “rare pediatric disease” as a disease thataffects fewer than 200,000 individuals in the U.S. primarily under the age of 18 years old. Under the FDA’s Rare Pediatric Disease Priority Review Voucher(PRV) program, upon the approval of an NDA or BLA for a product for the treatment of a rare pediatric disease that is serious or life-threatening, the sponsorof such application is eligible for a Rare Pediatric Disease Priority Review Voucher. Currently, the Priority Review Voucher can be used to obtain priorityreview for any subsequent NDA or BLA and may be sold or transferred an unlimited number of times. Under the 21st Century Cures Act, Congress extendedthe PRV program for rare pediatric diseases through 2020. A drug designated as a drug for a rare pediatric disease by September 30, 2020, and approved bySeptember 30, 2022, may receive a voucher. Because this program has been subject to criticism, including by the FDA, it is possible that even if we obtainapproval for FCX-007 and FCX-013 and qualify for such a PRV, the program may no longer be in effect at the time of approval. Ongoing FDA Requirements and Post-Marketing ObligationsAll approved drug products are subject to continuing regulation by the FDA, including record-keeping requirements, reporting of adverseexperiences with the product, sampling and distribution requirements, notifying the FDA and gaining its approval of certain manufacturing or labelingchanges, complying with certain electronic records and signature requirements, submitting periodic reports to the FDA, maintaining and providing updatedsafety and efficacy information to the FDA, and complying with FDA promotion and advertising requirements. Failure to comply with the statutory andregulatory requirements can subject a manufacturer to possible legal or regulatory action, such as warning letters, suspension of manufacturing, seizure ofproduct, injunctive action, criminal prosecution, or civil penalties.16Table of ContentsThe FDA may require post-marketing studies or clinical trials to develop additional information regarding the safety of a product. These studies ortrials may involve continued testing of a product and development of data, including clinical data, about the product’s effects in various populations and anyside effects associated with long-term use. The FDA may require post-marketing studies or trials to investigate possible or known serious risks or signals ofserious risks, or to identify unexpected serious risks, and may require periodic status reports if new safety information develops. Failure to conduct thesestudies in a timely manner may result in substantial civil penalties, or withdrawal of product approval.Also, newly discovered or developed safety or efficacy data may require changes to a product’s approved labeling, including the addition of newwarnings and contraindications, additional pre-clinical studies or clinical trials, or even in some instances, withdrawal of the approval. Violations ofregulatory requirements at any stage, including after approval, may result in various adverse consequences, including the FDA’s withdrawal of an approvedproduct from the market, other voluntary or FDA-initiated action that could delay or restrict further marketing, and the imposition of civil fines and criminalpenalties against the manufacturer or the NDA or BLA holder. In addition, later discovery of previously unknown problems may result in restrictions on theproduct, manufacturer or NDA or BLA holder, including withdrawal of the product from the market. The labeling, advertising, promotion, marketing and distribution of a drug or biologic product also must be in compliance with FDA requirementswhich include, among other things, promotional activities, standards and regulations for direct-to-consumer advertising, promotional activities involving theinternet, and industry sponsored scientific and educational activities. In general, all product promotion must be consistent with the labeling approved by theFDA for such product, contain a balanced presentation of information on the product’s uses, benefits, risks, and important safety information and limitationson use, and otherwise not be false or misleading. The FDA has very broad enforcement authority, and failure to abide by these regulations can result inpenalties, including the issuance of a warning letter directing a company to correct deviations from regulatory standards and enforcement actions that caninclude seizures, injunctions and criminal prosecution. Failure to comply with applicable FDA requirements and restrictions also may subject a company toadverse publicity and enforcement action by the U.S. Department of Justice (DOJ) or the Office of the Inspector General of the U.S. Department of Health andHuman Services (HHS) as well as state authorities. This could subject the company to a range of penalties that could have a significant commercial impact,including civil and criminal fines and agreements that materially restrict the manner in which a company promotes or distributes its products.Drug and biologic product manufacturers and their subcontractors are required to register their establishments with the FDA and certain stateagencies, and to list their products with the FDA. The FDA periodically inspects manufacturing facilities in the United States and abroad in order to assurecompliance with the applicable cGMP regulations and other requirements. These cGMP requirements apply to all stages of the manufacturing process,including production, processing, sterilization, packaging, labeling, storage and shipment. Facilities also are subject to inspections by other federal, foreign,state and local agencies. In complying with the cGMP regulations, manufacturers must continue to assure that the product meets applicable specifications,regulations and other post-marketing requirements. Failure to comply with these requirements subjects the manufacturer to possible legal, regulatory orvoluntary action, such as suspension of manufacturing or recall or seizure of product.Sponsors and their third-party contractors are also subject to various laws and regulations governing laboratory practices, the experimental use ofanimals and the use and disposal of hazardous or potentially hazardous substances in connection with their research. The FDA has regulatory andenforcement power to disqualify nonclinical laboratory studies performed by a violative facility from being considered by FDA in support of any applicationfor a research or marketing permit; to publicly disclose the fact of such disqualification; and to pursue any other available and appropriate judicialproceeding or regulatory action, such as court-ordered injunctions denial or withdrawal of regulatory approvals and referral to other federal, state or localgovernment law enforcement or regulatory agencies.Furthermore, new government requirements may be established that could delay or prevent regulatory approval of our products under development,or affect the conditions under which approved products are marketed. 17Table of ContentsHIPAA RequirementsOther federal legislation may affect our ability to obtain certain health information in conjunction with our research activities. We may be subject todata privacy and security regulation by both the federal government and the states in which we conduct our business. The Health Insurance Portability andAccountability Act of 1996 (HIPAA), as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (HITECH), and itsimplementing regulations, imposes obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission ofindividually identifiable health information. HIPAA also prohibits knowingly and willfully falsifying, concealing or covering up a material fact or makingany materially false, fictitious or fraudulent statements or representation, or making or using any false writing or document knowing the same to contain anymaterially false, fictitious or fraudulent statement or entry in connection with the delivery of or payment for healthcare benefits, items or services.In addition, numerous federal and state laws and regulations that address privacy and data security, including state data breach notification laws,state health information privacy laws, and federal and state consumer protection laws (e.g., Section 5 of the Federal Trade Commission Act), govern thecollection, use, disclosure and protection of health-related and other personal information. Failure to comply with data protection laws and regulations couldresult in government enforcement actions and create liability for us (which could include civil and/or criminal penalties), private litigation and/or adversepublicity that could negatively affect our business.Failure to achieve and sustain compliance with applicable federal and state privacy, security and fraud laws could result in government enforcementactions and create liability for us (which could include civil and/or criminal penalties), private litigation and/or adverse publicity that could negatively affectour operating results and business.Other U.S. Regulatory RequirementsIn the United States, the research, manufacturing, distribution, sale, and promotion of drug and biologic products are potentially subject toregulation by various federal, state and local authorities in addition to the FDA, including the Centers for Medicare and Medicaid Services (CMS), otherdivisions of the HHS (e.g., the Office of Inspector General), the DOJ and individual U.S. Attorney offices within the DOJ, and state and local governments. For example, sales, marketing and scientific/educational grant programs must comply with the federal Anti-Kickback Statute, the False Claims Act and otherfederal and state fraud and abuse laws, as described in detail below.If we successfully commercialize any of our products, we may participate in the Medicaid Drug Rebate Program. Participation is required for federalfunds to be available for our products that we successfully commercialize under Medicaid and Medicare Part B. Under the Medicaid Drug Rebate Program,we would be required to pay a rebate to each state Medicaid program for our covered outpatient drugs that are dispensed to Medicaid beneficiaries and paidfor by a state Medicaid program as a condition of having federal funds being made available to the states for our drugs under Medicaid and Part B of theMedicare program.Federal law requires that any company that participates in the Medicaid Drug Rebate Program also participate in the Public Health Service’s 340Bdrug pricing program in order for federal funds to be available for the manufacturer’s drugs under Medicaid and Medicare Part B. The 340B drug pricingprogram requires participating manufacturers to agree to charge statutorily-defined covered entities no more than the 340B “ceiling price” for themanufacturer’s covered outpatient drugs. These 340B covered entities include a variety of community health clinics and other entities that receive healthservices grants from the Public Health Service, as well as hospitals that serve a disproportionate share of low-income patients.Under Section 603 of the Veterans Health Care Act of 1992 (VHCA), drug companies that participate in Medicaid or Medicare Part B are required tooffer their “covered drugs” (biologic products and innovator drugs) for sale on a Federal Supply Schedule (FFS) contract at a statutorily reduced price to fourfederal agencies including the U.S. Department of Veterans Affairs, the U.S. Department of Defense, the Public Health Service and the Coast Guard.Participation under Section 603 the VHCA requires submission of pricing data and calculation of discounts pursuant to complex statutory formulas, as wellas the entry into government procurement contracts governed by the Federal Acquisition Regulation. In addition, pursuant to regulations issued by theDepartment of Defense TRICARE Management Activity, now the Defense Health Agency, to implement Section 703 of the National Defense AuthorizationAct for Fiscal Year 2008, manufacturers are required to pay rebates on “covered drug” prescriptions dispensed to TRICARE beneficiaries by TRICAREnetwork retail pharmacies. All of these activities are also potentially subject to federal and state consumer protection, unfair competition, and other laws.In March 2010, President Obama signed into law the Affordable Care Act, as amended by the Health Care and Education Reconciliation Act of 2010(together, the Affordable Care Act). The Affordable Care Act substantially changed the18Table of Contentsway healthcare is financed by both governmental and private insurers, and significantly impacts the pharmaceutical industry. The Affordable Care Actintended to broaden access to health insurance, reduce or constrain the growth of healthcare spending, enhance remedies against fraud and abuse, add newtransparency requirements for health care and health insurance industries, impose new taxes and fees on the health industry and impose additional healthpolicy reforms. The Affordable Care Act has resulted in, and we expect it will continue to result in, downward pressure on coverage and the price of productscovered by Medicare and other government programs. Any reduction in reimbursement from Medicare and other government programs may result in a similarreduction in payments and coverage from private payors. The implementation of cost containment measures or other healthcare reforms may prevent us frombeing able to generate revenue, attain profitability, or commercialize our products. In addition, certain legislative changes to and regulatory changes underthe Affordable Care Act have occurred in the 115th United States Congress and under the Trump Administration. For instance, the Bipartisan Budget Act of2018 increased the Affordable Care Act required manufacturer point-of-sale discount from 50% to 70% off the negotiated price for Medicare Part Dbeneficiaries during their coverage gap period beginning in 2019. It is possible that there will be further legislation or regulation that could harm ourbusiness, financial condition and results of operations.At such time as we market, sell and distribute any products for which we obtain marketing approval, it is possible that our business activities couldbe subject to scrutiny and enforcement under one or more federal or state health care fraud and abuse laws and regulations, which could affect our ability tooperate our business. These include the following fraud and abuse laws and regulations:•The federal Anti-Kickback Statute prohibits, among other things, knowingly and willfully offering, paying, soliciting or receiving remunerationto induce or in return for purchasing, leasing, ordering or arranging for the purchase, lease or order of any healthcare item or servicereimbursable under Medicare, Medicaid or other federally financed healthcare programs. This statute has been interpreted to apply toarrangements between pharmaceutical manufacturers on one hand and prescribers, purchasers, and formulary managers on the other. Althoughthere are a number of statutory exemptions and regulatory safe harbors protecting some business arrangements from prosecution, the exemptionsand safe harbors are drawn narrowly and practices that involve remuneration intended to induce prescribing, purchasing or recommending maybe subject to scrutiny if they do not qualify for an exemption or safe harbor. Our practices may not in all cases meet all of the criteria for safeharbor protection from federal Anti-Kickback Statute liability. The Affordable Care Act, among other things, clarified that liability may beestablished under the federal Anti-Kickback Statute without proving actual knowledge of the statute or specific intent to violate it. In addition,the Affordable Care Act provides that the government may assert that a claim including items or services resulting from a violation of the federalAnti-Kickback Statute constitutes a false or fraudulent claim for purposes of the civil False Claims Act (discussed below) or the civil monetarypenalties statute, which imposes penalties against any person who is determined to have presented or caused to be presented a claim to a federalhealth program that the person knows or should know is for an item or service that was not provided as claimed or is false or fraudulent.•The federal civil False Claims Act prohibits any person from, among other things, knowingly presenting, or causing to be presented, a false orfraudulent claim for payment of government funds; knowingly making, using, or causing to be made or used a false record or statement materialto a false or fraudulent claim to the federal government; or knowingly concealing or knowingly and improperly avoiding, decreasing orconcealing an obligation to pay money to the federal government. The False Claims Act also permits a private individual acting as a“whistleblower” to bring actions on behalf of the federal government alleging violations of the statute and to share in any monetary recovery.Many pharmaceutical and other healthcare companies have been investigated and have reached substantial financial settlements with thefederal government under the civil False Claims Act for a variety of alleged improper marketing activities, including: providing free product tocustomers with the expectation that the customers would bill federal programs for the product; providing sham consulting fees, grants, freetravel and other benefits to physicians to induce them to prescribe the company’s products; and inflating prices reported to private pricepublication services, which are used to set drug payment rates under government healthcare programs. In addition, in recent years thegovernment has pursued civil False Claims Act cases against a number of pharmaceutical companies for causing false claims to be submitted asa result of the marketing of their products for unapproved, and thus non-reimbursable, uses. False Claims Act liability is potentially significantin the healthcare industry because the statute provides for treble damages and mandatory penalties in the tens of thousands of dollars.Pharmaceutical and other healthcare companies also are subject to other federal false claim laws, including, among others, federal criminalhealthcare fraud and false statement statutes that extend to non-government health benefit programs.•HIPAA created federal criminal statutes that prohibit among other actions, knowingly and willfully executing, or attempting to execute, ascheme to defraud any healthcare benefit program, including private third-party payors,19Table of Contentsknowingly and willfully embezzling or stealing from a healthcare benefit program, willfully obstructing a criminal investigation of a healthcareoffense, and knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulentstatement in connection with the delivery of or payment for healthcare benefits, items or services.•Analogous state laws and regulations, such as state anti-kickback and false claims laws, may apply to items or services reimbursed underMedicaid and other state programs or, in several states, apply regardless of the payor. Some state laws also require pharmaceutical companies toreport expenses relating to the marketing and promotion of pharmaceutical products and to report gifts and payments to certain healthcareproviders in the states. Other states prohibit providing meals to prescribers or other marketing related activities. Other states restrict the ability ofmanufacturers to offer co-pay support to patients for certain prescription drugs. Still other states and cities require identification or licensing ofsales representatives. In addition, California, Connecticut, Nevada and Massachusetts require pharmaceutical companies to implementcompliance programs or marketing codes of conduct.•The federal Physician Payment Sunshine Act, being implemented as the Open Payments Program, requires certain pharmaceutical manufacturersto engage in extensive tracking of payments and other transfers of value to physicians and teaching hospitals, and to submit such data to CMS,which will then make all of this data publicly available on the CMS website. Pharmaceutical manufacturers with products for which payment isavailable under Medicare, Medicaid or the State Children’s Health Insurance Program are required to track reportable payments and must submita report to CMS on or before the 90th day of each calendar year disclosing reportable payments made in the previous calendar year. Failure tocomply with the reporting obligations may result in civil monetary penalties.•The federal Foreign Corrupt Practices Act of 1997 and other similar anti-bribery laws in other jurisdictions generally prohibit companies andtheir intermediaries from providing money or anything of value to officials of foreign governments, foreign political parties, or internationalorganizations with the intent to obtain or retain business or seek a business advantage. Recently, there has been a substantial increase in anti-bribery law enforcement activity by U.S. regulators, with more frequent and aggressive investigations and enforcement proceedings by bothDOJ and the U.S. Securities and Exchange Commission (SEC). Violations of United States or foreign laws or regulations could result in theimposition of substantial fines, interruptions of business, loss of supplier, vendor or other third-party relationships, termination of necessarylicenses and permits and other legal or equitable sanctions. Other internal or government investigations or legal or regulatory proceedings,including lawsuits brought by private litigants, may also follow as a consequence.Violations of any of the laws described above or any other governmental regulations are punishable by significant civil, criminal and administrativepenalties, damages, fines and exclusion from government-funded healthcare programs, such as Medicare and Medicaid. Although compliance programs canmitigate the risk of investigation and prosecution for violations of these laws, the risks cannot be entirely eliminated. Moreover, achieving and sustainingcompliance with applicable federal and state privacy, security and fraud laws may prove costly.International RegulationThe regulation of our product candidates outside of the United States varies by country. Certain countries regulate human tissue products as apharmaceutical product, which would require us to make extensive filings and obtain regulatory approvals before selling our product candidates. Certainother countries classify our product candidates as human tissue for transplantation but may restrict its import or sale. Other countries may have noapplication regulations regarding the import or sale of products similar to our product candidates, creating uncertainty as to what standards we may berequired to meet.EmployeesAs of December 31, 2017, we had 20 full-time employees, all located in the United States. Of these full-time employees, 15 are engaged in research,development and manufacturing (including facilities) functions and 5 are engaged in finance, legal, human resources, information technology, and othergeneral administrative functions. None of our employees are covered by a collective bargaining agreement, and we consider our relations with our employeesto be good. 20Table of ContentsCorporate InformationWe were incorporated under the laws of the State of Delaware in September 1992. Our corporate office is located at 405 Eagleview Boulevard,Exton, Pennsylvania 19341. Our telephone number is (484) 713-6000. We maintain an Internet website at www.fibrocell.com. The information contained onour website is not incorporated by reference into this Form 10-K.We file reports, proxy and information statements and other information with the SEC. We make available free of charge under the “Investors—SECFilings” section of our website all of our filings with the SEC, including our annual reports on Form 10-K, quarterly reports on Form 10-Q, current reports onForm 8-K, proxy statements and amendments to such documents, each of which is provided on our website as soon as reasonably practicable after weelectronically file the information with the SEC.The public may read and copy any materials we file with the SEC at the SEC’s Public Reference Room at 100 F Street, NE, Washington, DC 20549.The public may obtain information on the operation of the Public Reference Room by calling the SEC at 1-800-SEC-0330. Additionally, the SEC maintainsa website (www.sec.gov) that contains reports, proxy and information statements, and other information regarding issuers that file electronically with theSEC, including us.21Table of ContentsItem 1A. Risk FactorsOur business is subject to substantial risks and uncertainties. The occurrence of any of the following risks and uncertainties, either alone or taken together,could materially and adversely affect our business, financial condition, results of operations or prospects. In these circumstances, the market price of ourcommon stock could decline and you may lose all or part of your investment. The risks and uncertainties described below are not the only ones we face.Risks and uncertainties of general applicability and additional risks and uncertainties not currently known to us or that we currently deem to be immaterialmay also materially and adversely affect our business, financial condition, results of operations or prospects.Risks Related to our Financial Position and Need for Additional CapitalWe need to obtain additional capital to continue as a going concern. If we are unable to obtain sufficient capital, we will need to curtail and reduce ouroperations and costs, and modify our business strategy.Our principal sources of liquidity are cash and cash equivalents of $17.4 million as of December 31, 2017. As of December 31, 2017, we hadworking capital of $13.5 million. We believe that our existing cash and cash equivalents will be sufficient to fund our operations into the first quarter of2019. However, changing circumstances may cause us to consume capital faster than we currently anticipate, and we may need to spend more money thancurrently expected because of such circumstances.To meet our capital needs, we are considering multiple alternatives, including but not limited to, equity financings, debt financings, corporatecollaborations, partnerships and other strategic transactions and funding opportunities. However, there can be no assurance that we will be able to completeany such transaction on acceptable terms or otherwise. These factors raise substantial doubt about our ability to continue as a going concern. Consequently,the audit report prepared by our independent registered public accounting firm relating to our Consolidated Financial Statements for the year endedDecember 31, 2017 includes a paragraph related to the substantial doubt about our ability to continue as a going concern.If we raise additional funds by issuing equity securities, our stockholders will experience dilution. Debt financing, if available, will result inincreased fixed payment obligations and may involve agreements that include covenants limiting or restricting our ability to take specific actions, such asincurring additional debt, making capital expenditures or declaring dividends. Any debt financing or additional equity that we raise may contain terms, suchas liquidation, dividends and other rights or preferences, which are not favorable to us or our stockholders. If we raise additional funds through collaborationor partnership arrangements with third parties, it may be necessary to relinquish valuable rights to our technologies, future revenue streams or productcandidates or to grant licenses on terms that may not be favorable to us.If we are unable to raise additional capital in sufficient amounts or on terms acceptable to us, we will need to curtail and reduce our operations andcosts, and modify our business strategy which may require us to, among other things:•significantly delay, scale back or discontinue the development or commercialization of one or more of our product candidates or one or more ofour other research and development initiatives;•seek collaborators for one or more of our current or future product candidates at an earlier stage than otherwise would be desirable or on termsthat are less favorable than might otherwise be available;•sell or license on unfavorable terms our rights to technologies or product candidates that we otherwise would seek to develop or commercializeourselves; or•seek bankruptcy protection which may result in the termination of agreements pursuant to which we license important intellectual propertyrights including our exclusive collaboration agreements with Intrexon.We have incurred significant losses since our inception and anticipate that we will continue to incur losses in the future.We have incurred losses since our inception, have not generated significant revenue from commercial sales of our products, and have never beenprofitable. Investment in drug development is highly speculative because it entails substantial upfront capital expenditures and significant risk that a productcandidate will fail to gain regulatory approval or become commercially viable. We continue to incur significant research, development and other expensesrelated to our ongoing operations including development of our product candidates and operation of our manufacturing facility. As a result, we are notprofitable and have incurred losses in each period since we emerged from bankruptcy in September 2009. For the year ended December 31, 2017, we had anet loss of $16.2 million and used $17.0 million in operating activities and had an accumulated deficit of $178.8 million as of December 31, 2017.22Table of ContentsWe expect to continue to incur significant expenses and increasing operating losses for the foreseeable future. We anticipate that our expenses willcontinue to be significant if and as we:•continue our research and pre-clinical and clinical development of our product candidates;•initiate additional pre-clinical, clinical or other studies or trials for our product candidates, including under our collaboration agreements withIntrexon;•continue or expand our collaborations with Intrexon and our other collaborators;•further develop the manufacturing process for our product candidates;•continue to maintain a cGMP manufacturing facility;•change or add additional manufacturers or suppliers;•seek regulatory approvals for our product candidates that successfully complete clinical trials;•establish a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain regulatory approval;•seek to identify and validate additional product candidates;•acquire or in-license other product candidates and technologies;•maintain, protect and expand our intellectual property portfolio;•attract and retain skilled personnel;•create additional infrastructure to support our product development and planned future commercialization efforts; and•experience any delays or encounter issues with any of the above.The net losses we incur may fluctuate significantly from quarter to quarter and year to year, such that a period-to-period comparison of our results ofoperations may not be a good indication of our future performance. In any particular quarter or quarters, our operating results could be below the expectationsof securities analysts or investors, which could cause our stock price to decline.We do not generate significant revenues from product sales and may never be profitable.Our ability to generate revenue and achieve profitability depends on our ability, alone or with strategic collaboration partners, to successfullycomplete the development of, and obtain the regulatory approvals necessary for, the manufacture and commercialization of our product candidates. We donot anticipate generating significant revenues from product sales for the foreseeable future, if ever. Our ability to generate future revenues from product salesdepends heavily on our success in:•completing research and pre-clinical and clinical development of our product candidates;•seeking and obtaining regulatory approvals for product candidates for which we complete clinical trials;•developing a sustainable, scalable, reproducible, and transferable manufacturing process for our product candidates;•establishing and maintaining supply and manufacturing relationships with third parties that can provide adequate (in amount and quality)products and services to support clinical development and the market demand for our product candidates, if approved;•launching and commercializing product candidates for which we obtain regulatory approval, either by collaborating with a partner or, iflaunched independently, by establishing a sales force, marketing, sales operations and distribution infrastructure;•obtaining market acceptance of our product candidates and cell therapy and gene therapy as viable treatment options;•addressing any competing technological and market developments;•implementing additional internal systems and infrastructure, as needed;•identifying and validating new product candidates;•negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter;23Table of Contents•maintaining, protecting and expanding our portfolio of intellectual property rights, including patents, trade secrets and know-how; and•attracting, hiring and retaining qualified personnel.Even if one or more of the product candidates that we develop is approved for commercial sale, we anticipate incurring significant costs associatedwith commercializing any approved product candidate. Our expenses could increase beyond expectations if we are required by the FDA or other regulatoryagencies, domestic or foreign, to perform clinical trials or other studies in addition to those that we currently anticipate. Even if we are able to generaterevenues from the sale of any approved products, we may not become profitable and may need to obtain additional funding to continue operations.We will seek to raise additional funds in the future, which may be dilutive to stockholders or impose operational restrictions.We will need to raise additional capital in the future to help fund our clinical trials, our collaboration efforts with Intrexon and for the developmentand commercialization of our product candidates. If we raise additional capital through the issuance of equity securities, the percentage ownership of ourcurrent stockholders will be reduced. We may also issue equity as part of license issue fees to our licensors, to compensate consultants or to settle outstandingpayables. Our stockholders may experience additional dilution in net book value per share and any additional equity securities may have rights, preferencesand privileges senior to those of the holders of our common stock. Debt financing, if available, will result in increased fixed payment obligations and mayinvolve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capitalexpenditures or declaring dividends. Any debt financing or additional equity that we raise may contain terms, such as liquidation and other preferences,which are not favorable to us or our stockholders. If we raise additional funds through corporate collaboration, partnership or other strategic transactions, itmay be necessary to relinquish valuable rights to our product candidates, our technologies or future revenue streams or to grant licenses or sell assets on termsthat may not be favorable to us. If we cannot raise additional funds, we will have to delay our development activities or cease operations.Our failure to comply with the restrictive covenants or other terms of our outstanding convertible notes, including as a result of events beyond ourcontrol, could result in a default under the notes that could materially and adversely affect the ongoing viability of our business.On September 7, 2016, we issued an aggregate of approximately $18.1 million in principal of convertible promissory notes (each a Note andcollectively, the Notes) and accompanying warrants to purchase an aggregate of 6,029,174 shares of common stock (the Private Placement Warrants) in aprivate placement (the 2016 Private Placement) to institutional and accredited investors (each an Investor and collectively, the Investors). The Notes bearinterest at 4% per annum and have a stated maturity date of the earlier of (i) September 7, 2026 and (ii) one-hundred and eighty (180) days after the date onwhich our product candidate, FCX-007, is approved by the FDA for the treatment of RDEB. Each individual Note holder has the right to require us to repayall or any portion of the unpaid principal from time to time on or after September 7, 2021 (such right, a Put Right). With respect to accrued and unpaidinterest on the Note, each Note holder may elect, at any time and from time to time, to have any accrued and unpaid interest converted into shares of ourcommon stock. In addition, each Note holder may elect to accelerate the repayment of all unpaid principal and accrued interest under such holder’s Noteupon consummation of a specified change of control transaction or occurrence of certain events of default (as specified in the Notes), including, among otherthings:•our default in a payment obligation under the Notes;•our default in a payment obligation under our other debt in excess of $5 million;•our breach of the restrictive covenants or other terms of the Notes;•certain specified insolvency and bankruptcy-related events; and•our common stock ceasing to be listed or quoted on Nasdaq or another national securities exchange.In addition, upon an event of default, the base interest rate (excluding any additional interest) for the Notes automatically increases to twelvepercent (12%) per annum. Subject to any applicable cure period set forth in the Notes, all amounts outstanding with respect to the Notes (principal andaccrued interest) would become due and payable immediately upon an event of default. We cannot assure you that our assets or cash flow would be sufficientto fully repay our obligations under the Notes if the obligations thereunder are accelerated upon any events of default. Further, if we are unable to repay,refinance or restructure our obligations under the Notes, the holders of such Notes could proceed to protect and enforce their rights under the Notes byexercising such remedies as are available to the holders thereunder and in respect thereof under applicable law, either by suit in equity or by action at law, orboth, whether for specific performance of any covenant or other24Table of Contentsagreement contained in the Notes or in aid of the exercise of any power granted in the Notes. The foregoing would materially and adversely affect theongoing viability of our business.We are subject to restrictive covenants that may restrict our ability to pursue business strategies that are in our long-term best interests.The Notes and Purchase Agreement (as defined below) for the sale of our Series A Preferred Stock (as defined below) contain a number of restrictivecovenants that impose significant restrictions on us and may limit our ability to engage in acts that may be in our long-term best interests. Subject to certainlimited exceptions, the Notes and Purchase Agreement include covenants restricting, among other things, our ability to:•pay cash dividends or make distributions on our capital stock or redeem or repurchase our capital stock;•create, assume or suffer to exist at any time any lien upon any of our properties or assets;•assign any accounts or other right to receive income;•incur any senior and pari passu debt;•enter into transactions with affiliates other than on terms and conditions approved by a majority of the disinterested members of our board ofdirectors (the Board); and•use the proceeds of the 2016 Private Placement or Series A Preferred Stock Offering (as defined below) for any purpose other than solely for thecontinued pre-clinical and clinical development of our product candidates and for other general corporate purposes.In addition, a breach of any of these restrictive covenants could result in a default under the Notes, entitling the holders to declare the Notes,together with accrued and unpaid interest and other amounts payable thereunder, to be immediately due and payable.Provisions of the Notes and certain of our outstanding common stock purchase warrants provide for certain potential payments to the holders of suchNotes and common stock purchase warrants that could impede a sale of the Company.The Private Placement Warrants we issued in the 2016 Private Placement give each holder the option to receive a cash payment based on a Black-Scholes valuation upon our change of control. We are required, at the warrant holder’s option, exercisable at any time concurrently with, or within 30 daysafter, the announcement of a change of control, to repurchase the Private Placement Warrants from the applicable holder by paying to the holder an amount ofcash equal to the value of the unexercised portion of such holder's warrant as determined in accordance with the Black-Scholes option pricing model and theterms of the Private Placement Warrants.The December 2017 Common Warrants (as defined below) we issued in the December 2017 Offering (as defined below) give the holders the optionto receive a cash payment based on a Black-Scholes valuation upon our change of control. We are required, at the holder’s option, exercisable at any timeconcurrently with, or within 30 days after, the announcement of a change of control, to repurchase the December 2017 Common Warrants from the applicableholder by paying to the holder an amount of cash equal to the value of the unexercised portion of such holder’s warrant as determined in accordance with theBlack-Scholes option pricing model and the terms of the December 2017 Common Warrants.In addition, upon consummation of a specified change of control transaction, each holder of a Note may elect to accelerate the repayment of allunpaid principal and accrued interest under such holder’s Note. If a holder does not elect to have us prepay its Note upon such change of control transaction,then we may prepay the Notes, in an amount equal to one hundred one percent (101%) of the outstanding principal due under the Notes (together withaccrued and unpaid interest due thereon). These provisions may make it more costly for a potential acquirer to engage in a business combination transactionwith us. Provisions that have the effect of discouraging, delaying or preventing a change in control could limit the opportunity for our stockholders toreceive a premium for their shares of our common stock and could also affect the price that some investors are willing to pay for our common stock.We may be subject to payment of liquidated damages if we fail to file and maintain an effective registration statement with respect to the securities coveredunder the registration rights agreements that we entered into in connection with the 2016 Private Placement.In connection with the 2016 Private Placement, we entered into a registration rights agreement (the Registration Rights Agreement) with theinvestors that participated in the offering. The Registration Rights Agreement contains demand and piggyback registration rights requiring us to registershares of our common stock issuable upon the conversion of the Notes or the exercise of the Private Placement Warrants and any other shares of our commonstock held by the investors for resale under25Table of Contentsthe Securities Act of 1933, as amended. If we fail, under certain circumstances as described in the Registration Rights Agreement, to file and maintain aneffective registration statement with respect to the securities covered under the Registration Rights Agreement, we have agreed to pay liquidated damages toeach investor in an amount equal to one percent (1.0%) of the aggregate amount invested by such investor pursuant to the Notes then owned thereby for each30-day period or pro rata for any portion thereof during which the failure to file or keep a registration statement effective continues.We have a significant number of outstanding convertible notes, convertible preferred stock, warrants and stock options, and future sales of underlyingshares of our common stock may cause substantial dilution to our existing stockholders.We issued an aggregate of $18.1 million in principal of Notes and Private Placement Warrants to purchase a total of 6,029,174 shares of our commonstock in connection with the 2016 Private Placement. Each Private Placement Warrant has a five year term ending on September 7, 2021 and is initiallyexercisable at $4.50 per share beginning March 8, 2017. Holders of the Notes have the right to convert unpaid principal of the Notes into shares of ourcommon stock at any time at conversion prices ranging from $3.40875 to $3.67875 per share (the Conversion Price). The Notes bear interest at four percent(4%) per annum which we may elect to pay in cash or accrue. If we elect for interest to accrue, then (i) we may elect to repay any such accrued and unpaidinterest in cash at any time and from time to time and (ii) each holder of a Note may elect to have us repay any such accrued and unpaid interest by deliveringsuch number of shares of our common stock equal to (x) the amount of the accrued and unpaid interest to be repaid, divided by (y) the greater of (i) the lastclosing bid price of a share of our common stock as reported on Nasdaq on the date of such election and (ii) the applicable Conversion Price. CommencingSeptember 8, 2016, we have elected to accrue interest.On March 7, 2017, we entered into a Securities Purchase Agreement (the Purchase Agreement) with certain of our existing investors pursuant towhich we issued and sold a total of 8,000 units (the Units) for a purchase price of $1,000 per Unit, with each Unit consisting of (i) one share of our Series AConvertible Preferred Stock (Series A Preferred Stock) and (ii) a warrant to purchase 429 shares of our common stock (the March 2017 Warrants) (collectively,the Series A Preferred Stock Offering). Each share of Series A Preferred Stock has an initial stated value of $1,000 and is convertible into shares of ourcommon stock at a conversion price of $2.3271 per share of common stock, subject to adjustment for stock splits, stock dividends, stock combinations,recapitalizations or similar events. Holders of the Series A Preferred Stock are also entitled to receive cumulative dividends at a rate per share of 4% perannum (with such dividend rate increasing to 8% per annum on the five year anniversary of the original issuance of the Series A Preferred Stock), with suchdividends compounded quarterly by increasing the stated value of the Series A Preferred. Each March 2017 Warrant has an exercise price of $2.53773 pershare, is exercisable six months after the date of issuance and expires five years from the date of issuance.On December 7, 2017, we entered into an underwriting agreement (the HCW Underwriting Agreement) with H.C. Wainwright & Co., LLC (HCW),pursuant to which we issued and sold 7,714,156 shares of our common stock, pre-funded warrants to purchase an aggregate of 5,922,208 shares of ourcommon stock (December 2017 Pre-Funded Warrants) and common stock purchase warrants to purchase up to an aggregate of 14,046,950 shares of ourcommon stock (the December 2017 Common Warrants), which amount of common stock purchase warrants includes warrants to purchase up to 410,586shares of Common Stock pursuant to the partial exercise of HCW’s option to purchase additional common stock purchase warrants . Each share of ourcommon stock or December 2017 Pre-Funded Warrant, as applicable, was sold together with a December 2017 Common Warrant to purchase one share of ourcommon stock at a combined effective price to the public of $0.77 per share and accompanying December 2017 Common Warrant (collectively, theDecember 2017 Offering). Each December 2017 Common Warrant has an exercise price of $0.77 per share, was exercisable upon the date of issuance andexpires five years from the date of issuance.As additional compensation, we issued warrants to HCW to purchase 436,364 shares of our common stock (the December 2017 UnderwriterWarrants). Each December 2017 Underwriter Warrant has an exercise price of $0.9625 per share, was exercisable as of the date of the HCW UnderwritingAgreement, and will expire five years after the date of the HCW Underwriting Agreement.Subject to adjustment upon certain corporate events, including stock dividends, stock splits and distributions of cashor other assets to stockholders:•up to 5,280,370 shares of our common stock could be issuable by us in connection with the conversion of principal under the Notes; plus•up to 2,581,843 shares of our common stock could be issuable by us in satisfaction of our interest payment obligations under the Notes; plus•up to 6,029,174 shares of our common stock could be issuable by us in connection with the exercise of the Private Placement Warrants; plus26Table of Contents•up to 3,432,000 shares of our common stock could be issuable by us in connection with the conversion of the shares of Series A Preferred Stock; plus•up to 3,437,334 shares of our common stock could be issuable by us in connection with the exercise of the March 2017 Warrants; plus•up to 2,416,104 shares of common stock could be issuable by us in connection with the exercise of the December 2017 Pre-Funded Warrants; plus•up to 436,364 shares of our common stock could be issuable by us in connection with the exercise of the December 2017 Underwriter Warrants; plus•up to 14,046,950 shares of our common stock could be issuable by us in connection with the exercise of the December 2017 Common Warrants.The exercise of the Private Placement Warrants, the March 2017 Warrants, the December 2017 Pre-Funded Warrants, the December 2017 UnderwriterWarrants or the December 2017 Common Warrants or the conversion of the Notes or Series A Preferred Stock may cause substantial dilution to our existingstockholders.If we are unable to regain compliance with the listing requirements of Nasdaq, our common stock may be delisted from the Nasdaq Capital Market whichcould have a material adverse effect on our financial condition and could make it more difficult for you to sell your shares.Our common stock is listed on the Nasdaq Capital Market, and we are therefore subject to its continued listing requirements, including requirementswith respect to the market value of publicly-held shares, market value of listed shares, minimum bid price per share, and minimum stockholders’ equity,among others, and requirements relating to board and committee independence. If we fail to satisfy one or more of the requirements, we may be delisted fromthe Nasdaq Capital Market.On January 23, 2018, we received notice (the Notice) from the Nasdaq Stock Market LLC that we are not in compliance with Nasdaq Listing Rule5550(a)(2), as the minimum bid price of our common stock has been below $1.00 per share for 30 consecutive business days. The Notice has no immediateeffect on the listing of our common stock, which will continue to trade at this time on the Nasdaq Capital Market under the symbol “FCSC.”In accordance with Nasdaq Listing Rule 5810(c)(3)(A), we have a period of 180 calendar days, or until July 23, 2018, to regain compliance with theminimum bid price requirement. To regain compliance, the closing bid price of our common stock must meet or exceed $1.00 per share for at least tenconsecutive business days during this 180 calendar day period. In the event we do not regain compliance by July 23, 2018, we may be eligible for anadditional 180 calendar day grace period if we meet the continued listing requirement for market value of publicly held shares ($1 million) and all otherinitial listing standards which require, among other things, that we have at least $5 million of stockholders’ equity or at least $4 million of stockholders’equity and $50 million market value of listed shares. If we fail to regain compliance during the applicable period, we will receive notification from Nasdaqthat our common stock is subject to delisting. At that time we may then appeal the delisting determination to a Hearings Panel. Such notification will have noimmediate effect on our listing on the Nasdaq Capital Market, nor will it have an immediate effect on the trading of our common stock pending such hearing.There can be no assurance, however, that we will be able to regain compliance with Nasdaq’s minimum bid price requirement. If we regain compliance withthe Nasdaq’s minimum bid price requirement, there can be no assurance that we will be able to maintain compliance with the continued listing requirementsfor the Nasdaq Capital Market, or that our common stock will not be delisted from the Nasdaq Capital Market in the future. In addition, we may be unable tomeet other applicable listing requirements of the Nasdaq Capital Market, including maintaining minimum levels of stockholders’ equity or market values ofour common stock in which case, our common stock could be delisted notwithstanding our ability to demonstrate compliance with the minimum bid pricerequirement.Delisting from the Nasdaq Capital Market may adversely affect our ability to raise additional financing through the public or private sale of equitysecurities, may significantly affect the ability of investors to trade our securities and may negatively affect the value and liquidity of our common stock.Delisting also could have other negative results, including the potential loss of employee confidence, the loss of institutional investors or interest in businessdevelopment opportunities. Moreover, a delisting of our common stock could result in an event of default under the Notes issued in the 2016 PrivatePlacement.If we are delisted from the Nasdaq Capital Market and we are not able to list our common stock on another exchange, our common stock could bequoted on the OTC Bulletin Board or in the “pink sheets.” As a result, we could face significant adverse consequences including, among others:27Table of Contents•a limited availability of market quotations for our securities;•a determination that our common stock is a “penny stock” which will require brokers trading in our common stock to adhere to more stringent rulesand possibly result in a reduced level of trading activity in the secondary trading market for our securities;•a limited amount of news and little or no analyst coverage for us;•we would no longer qualify for exemptions from state securities registration requirements, which may require us to comply with applicable statesecurities laws; and•a decreased ability to issue additional securities (including pursuant to short-form registration statements on Form S-3) or obtain additionalfinancing in the future.If our stockholders’ equity falls below $2.5 million, our common stock may be subject to delisting from NasdaqNasdaq has the authority, pursuant to Nasdaq Listing Rule 5550(b)(1), to delist our common stock if our stockholders’ equity falls below $2.5million. As of December 31, 2017, our stockholders’ equity was $9.0 million. If our stockholders equity is hereafter reduced below $2.5 million as a result ofoperating losses or for other reasons, we will fail to meet Nasdaq’s stockholders’ equity requirement. If that occurs, or if we are unable to demonstrate toNasdaq’s satisfaction that we will be able to sustain compliance with this requirement, Nasdaq may delist our common stock. In addition, even if we regaintechnical compliance with the stockholders’ equity requirement, we will have to continue to meet other objective and subjective listing requirements tocontinue to be listed on the Nasdaq Capital Market, including the requirement that our common stock continues to trade above $1.00. For the year endedDecember 31, 2017, we incurred a net loss of $16.2 million and used $17.0 million in operating activities and had an accumulated deficit of $178.8 millionas of December 31, 2017.We are actively monitoring our stockholders’ equity and will consider any and all options available to us to maintain compliance. There can be noassurance, however, that we will be able to maintain compliance and meet Nasdaq’s minimum stockholders’ equity requirements. The alternatives to tradingon the Nasdaq Capital Market or another national securities exchange are generally considered to be less efficient and less broad-based than the nationalsecurities exchanges and the liquidity of our common stock will likely be reduced. In addition, if at any time we are not listed on the Nasdaq Capital Market(or similar national securities exchange), then each holder of our Notes will have the option to declare the Notes held by each holder immediately due andpayable, which would drain our financial resources, have a material adverse effect on our financial condition and make it exceedingly difficult to continue asa going concern.If our common stock becomes subject to the penny stock rules, it would become more difficult to trade our shares.The SEC has adopted rules that regulate broker-dealer practices in connection with transactions in penny stocks. Penny stocks are generally equitysecurities with a price of less than $5.00, other than securities registered on certain national securities exchanges or authorized for quotation on certainautomated quotation systems, provided that current price and volume information with respect to transactions in such securities is provided by the exchangeor system. If we do not retain our listing on the Nasdaq Capital Market and if the price of our common stock is less than $5.00, our common stock may bedeemed a penny stock. The penny stock rules require a broker-dealer, before a transaction in a penny stock not otherwise exempt from those rules, to deliver astandardized risk disclosure document containing specified information. In addition, the penny stock rules require that before effecting any transaction in apenny stock not otherwise exempt from those rules, a broker-dealer must make a special written determination that the penny stock is a suitable investmentfor the purchaser and receive (i) the purchaser’s written acknowledgment of the receipt of a risk disclosure statement; (ii) a written agreement to transactionsinvolving penny stocks; and (iii) a signed and dated copy of a written suitability statement. These disclosure requirements may have the effect of reducingthe trading activity in the secondary market for our common stock, and therefore stockholders may have difficulty selling their shares.We have a limited operating history, which may make it difficult for you to evaluate the success of our business to date and to assess our future viability.We have a limited operating history and our primary business activities consist of research, pre-clinical development and conducting clinical trials,pursuing our collaborations with Intrexon and previously commercializing LAVIV (azficel-T) which had previously been approved by the FDA in June 2011for the improvement of the appearance of moderate to severe nasolabial fold wrinkles in adults. As such, our historical financial data is of limited value inestimating future operating expenses. Our budgeted expense levels are based in part on our expectations concerning the costs of our research, pre-clinicaldevelopment, clinical trials and our collaborations with Intrexon, which depend on the success of such activities, and our ability to effectively and efficientlyconduct such research, pre-clinical development, clinical trials and our expectations related to our efforts to achieve FDA approval with respect to ourproduct candidates. Our limited operating history and clinical trial experience make these costs difficult to forecast accurately. We may be unable to adjustour operations in a timely manner to28Table of Contentscompensate for any unexpected increase in costs. Further, our fixed manufacturing costs and operating expenses may increase significantly as we expand ouroperations. Accordingly, a significant increase in costs could have an immediate and material adverse effect on our business, results of operations andfinancial condition.We may acquire other assets or businesses, or form collaborations or make investments in other companies or technologies that could harm our operatingresults, dilute our stockholders’ ownership, incur debt or cause us to incur significant expense.As part of our business strategy, we may pursue acquisitions of assets or businesses, or strategic alliances and collaborations, to expand our existingtechnologies and operations. We may not identify or complete these transactions in a timely manner, on a cost-effective basis, or at all, and we may notrealize the anticipated benefits of any such transaction, any of which could have a detrimental effect on our financial condition, results of operations and cashflows. We may not be able to find suitable acquisition candidates, and if we make any acquisitions, we may not be able to integrate these acquisitionssuccessfully into our existing business and we may incur debt or assume unknown or contingent liabilities in connection therewith. Integration of anacquired company or assets may also disrupt ongoing operations, require the hiring of additional personnel and the implementation of additional internalsystems and infrastructure, especially the acquisition of commercial assets, and require management resources that would otherwise focus on developing ourexisting business. We may not be able to find suitable collaboration partners or identify other investment opportunities, and we may experience losses relatedto any such investments.To finance any acquisitions or collaborations, we may choose to issue debt or shares of our common stock as consideration. Any such issuance ofshares would dilute the ownership of our stockholders. If the price of our common stock is low or volatile, we may not be able to acquire other assets orcompanies or fund a transaction using our stock as consideration. Alternatively, it may be necessary for us to raise additional capital for acquisitions throughpublic or private financings. Additional capital may not be available on terms that are favorable to us, or at all. In addition, the Notes issued in the 2016Private Placement restrict or limit our ability to incur or assume additional indebtedness.Risks Related to Clinical Development, Regulatory Approval and Commercialization of Our Product CandidatesOur product candidates are based on novel technology, which makes it difficult to predict the time and cost of product development and subsequentlyobtaining regulatory approval. At the moment, only a small number of gene therapy products have been approved in the United States and only two genetherapy products have been approved in the European Union.Our product candidates, are based on novel technology. Our future success depends on the successful development of this therapeutic approach.There can be no assurance that any development problems we experience in the future related to our product candidates will not cause significant delays orunanticipated costs, or that such development problems can be solved. We may also experience delays in developing a sustainable, reproducible andcommercial-scale manufacturing process or transferring that process to commercial partners, which may prevent us from completing our clinical trials orcommercializing our product candidates on a timely or profitable basis, if at all.In addition, the clinical trial requirements of the FDA, the European Medicines Agency (the EMA), and other regulatory agencies and the criteriathese regulators use to determine the safety and efficacy of a product candidate vary substantially according to the type, complexity, novelty and intendeduse and market of the potential products. The regulatory approval process for gene therapy product candidates such as ours can be more expensive and takelonger than for other, better known or more extensively studied pharmaceutical or other product candidates. At the moment, only a small number of genetherapy products have been approved in the Western world, which makes it difficult to determine how long it will take or how much it will cost to obtainregulatory approvals for our product candidates in the United States, the European Union or other jurisdictions. Approvals by the EMA may not be indicativeof what the FDA may require for approval and vice versa.Regulatory requirements governing gene and cell therapy products have evolved and may continue to change in the future. For example, the FDAhas established the Office of Tissues and Advanced Therapies within its Center for Biologics Evaluation and Research, or CBER, to consolidate the review ofgene therapy and related products, and established the Cellular, Tissue and Gene Therapies Advisory Committee to advise CBER on its review. In addition,the Recombinant DNA Advisory Committee, or RAC, of the U.S. National Institutes of Health, or NIH, reviews human gene transfer protocols when anoversight body, such as an Institutional Review Board, has determined that the protocol would significantly benefit from RAC review and when the protocolmeets certain criteria. The NIH Director may also select a protocol unilaterally for RAC review if it presents significant scientific, societal, or ethical concerns.Although the FDA decides whether individual gene therapy protocols may proceed, the RAC review process can impede the initiation of a clinical trial, evenif the FDA has reviewed the29Table of Contentsclinical trial and approved its initiation. Furthermore, all institutions and clinical trial sites in the United States that are subject to the NIH Guidelines forResearch Involving Recombinant or Synthetic Nucleic Acid Molecules, or NIH Guidelines, must follow those Guidelines and all RAC recommendations, orrisk losing NIH funding for such research or needing NIH pre-approval before conducting such research. Institutions subject to the NIH Guidelines include allinstitutions that receive any funding from NIH for research involving recombinant or synthetic nucleic acid molecules, as well as institutions subject tofederal or state regulations, local ordinances, or agency guidelines that require compliance with the NIH Guidelines, and institutions receiving support fromfederal agencies or private funders who condition such support on compliance with the NIH Guidelines. In addition, the FDA can put an IND on clinical holdfor numerous reasons, such as if the information in such IND is not sufficient to assess the risks in pediatric patients. Before a clinical trial can begin at anyinstitution, that institution’s institutional review board, or IRB, and its Institutional Biosafety Committee will have to review the proposed clinical trial toassess the safety of the trial. Moreover, serious adverse events, or SAEs, or developments in clinical trials of gene therapy product candidates conducted byothers may cause the FDA or other regulatory bodies to initiate a clinical hold on our clinical trials or otherwise to change the requirements for approval ofany of our product candidates.These regulatory review agencies, committees and advisory groups and the new requirements and guidelines they promulgate may lengthen theregulatory review process, require us to perform additional or larger studies, increase our development costs, lead to changes in regulatory positions andinterpretations, delay or prevent approval and commercialization of these treatment candidates or lead to significant post-approval studies, limitations orrestrictions. As we advance our product candidates, we will be required to consult with these regulatory and advisory groups and comply with applicablerequirements and guidelines. If we fail to do so, we may be required to delay or discontinue development of our product candidates. Delay or failure toobtain, or unexpected costs in obtaining, the regulatory approval necessary to bring a potential product to market could decrease our ability to generatesufficient product revenue to maintain our business.The results seen in pre-clinical studies and early stage clinical trials of our product candidates may not be replicated in humans.Although we have seen positive results in pre-clinical studies of FCX-007 and FCX-013 and interim data from the Phase 1 portion of our Phase 1/2clinical trial of FCX-007, we may not see positive results when these and any other product candidates undergo clinical trials in humans. Pre-clinical studiesare not designed to test the efficacy of a product candidate in humans, but rather to:•test short-term safety;•establish biological plausibility;•identify biologically active dose levels;•establish feasibility and reasonable safety of the investigational product’s proposed clinical route of administration;•identify physiologic parameters that can guide clinical monitoring; and/or•establish proof of concept, or the feasibility and rationale for use of an investigational product in the targeted patient population.Success in pre-clinical studies does not ensure that later studies or any clinical trials will be successful nor does it predict future results. The rate offailure in drug development is quite high, and many companies in the biopharmaceutical industry have suffered significant setbacks in advanced clinicaltrials, even after obtaining promising results in pre-clinical studies and earlier clinical trials. Product candidates may fail to show desired safety and efficacywhen used with human patients. Negative or inconclusive results from any of our ongoing pre-clinical studies could result in delays, modifications, orabandonment of clinical trials and the termination of our development of a product candidate.In previous clinical trials involving viral vectors for gene therapy, some patients experienced serious adverse events, including the development ofleukemia due to vector-related insertional oncogenesis. If our vectors demonstrate a similar effect, we may be required to halt or delay further clinicaldevelopment of our product candidates.A significant risk in any gene therapy product based on viral vectors is that the vector will insert in or near cancer-causing oncogenes leading touncontrolled clonal proliferation of mature cancer cells in the patient. For example, in 2003, 20 patients treated for X-linked severe combinedimmunodeficiency in two gene therapy trials using a murine, or mouse-derived, gamma-retroviral vector showed correction of the disease, but the trials wereterminated after five patients developed leukemia (four of whom were subsequently cured). The cause of these adverse events was shown to be insertionaloncogenesis, which is the process whereby the corrected gene inserts in or near a gene that is important in a critical cellular process like growth or division,and this insertion results in the development of a cancer (often leukemia). Using molecular diagnostic techniques, it30Table of Contentswas determined that clones from these patients showed retrovirus insertion in proximity to the promoter of the LMO2 proto-oncogene. Earlier generationretroviruses like the one used in these two trials have been shown to preferentially integrate in regulatory regions of genes that control cell growth. These well-publicized adverse events led to the development of new viral vectors, such as lentiviral vectors like the ones we utilize for FCX-007 andFCX-013, with potentially improved safety profiles and also the requirement of enhanced safety monitoring in gene therapy clinical trials, including routineperformance of vector copy number analysis on all production lots to monitor the number of insertion events per cell. Notwithstanding the potential safetyimprovements of lentiviral vectors, the risk of insertional oncogenesis remains a significant concern for gene therapy and we cannot assure that it will notoccur in any of our clinical trials. There is also the potential risk of delayed adverse events following exposure to gene therapy products due to persistentbiological activity of the genetic material or other components of products used to carry the genetic material. The FDA has stated that lentiviral vectorspossess characteristics that may pose risks of delayed adverse events. If any such adverse events occur, further advancement of our clinical trials could behalted or delayed, which would have a material adverse effect on our business and operations.We may find it difficult to enroll patients in our clinical trials, which could delay or prevent clinical trials of our product candidates.Identifying and qualifying patients to participate in clinical trials of our product candidates is critical to our success. The timing of our clinical trialsdepends on the speed at which we can recruit patients to participate in testing our product candidates. We have experienced delays in some of our clinicaltrials, and we may experience similar delays in the future. If patients are unwilling to participate in our clinical trials because of negative publicity fromadverse events in cell and gene therapies or for other reasons, including competitive clinical trials for similar patient populations, the timeline for recruitingpatients, conducting clinical trials and obtaining regulatory approval of potential products may be delayed. These delays could result in increased costs,delays in advancing our product candidates, delays in testing the effectiveness of our technology or termination of the clinical trials altogether.We may not be able to identify, recruit and enroll a sufficient number of patients, or those with required or desired characteristics to achievediversity in a clinical trial or complete our clinical trials in a timely manner. Patient enrollment is affected by a variety factors including, among others:•severity of the disease under investigation;•design of the study protocol;•prevalence of the disease/size of the patient population;•eligibility criteria for the clinical trial in question;•perceived risks and benefits of the product candidate under study;•proximity and availability of clinical trial sites for prospective patients;•availability of competing therapies and clinical trials;•efforts to facilitate timely enrollment in clinical trials;•patient referral practices of physicians; and•ability to monitor patients adequately during and after treatment.Our study is currently designed to recruit two additional patients in the NC1- (patients who do not express the first non-collagenous portion of theCOL7 protein) cohort of the Phase 1 portion of the clinical trial. The most recent scientific thinking and the rarity of NC1- patients compared to NC1+, i.e.,the difficultly in recruiting this group, we are seeking a modification in the protocol to close out the Phase 1 cohort of this clinical trial without includingany additional patients. The clinical trial protocol is designed to allow a cohort to move into the Phase 2 portion of the trial even if the other cohort is stillenrolling or in the follow-up evaluation period. Therefore, the modification of the protocol for the trial does not impact ongoing enrollment of Phase 2patients. If the FDA objects to this modification, we will continue to attempt to enroll NC1- patients. As data is collected, progress will be reported.Our current product candidates are being developed to treat rare diseases with limited patient pools from which to draw for clinical trials and theprocess of finding and diagnosing patients may prove costly. We have estimated that there are approximately 1,100 to 2,500 U.S. patients with RDEB andapproximately 90,000 patients in the US considered to have moderate to severe localized scleroderma. We may not be able to initiate or continue clinicaltrials if we cannot enroll a31Table of Contentssufficient number of eligible patients to participate in the clinical trials required by the FDA or other regulatory agencies. If we have difficulty enrolling asufficient number of patients to conduct our clinical trials as planned, we may need to delay, limit or terminate ongoing or planned clinical trials, any ofwhich would have an adverse effect on our business.Clinical trials may fail to demonstrate the safety or efficacy of our product candidates, which could prevent or significantly delay regulatory approval ofour product candidates and harm our business.Prior to receiving approval to commercialize any of our product candidates, we must demonstrate with substantial evidence from well-controlledclinical trials, and to the satisfaction of the FDA and other regulatory authorities in the United States and abroad, if applicable, that such product candidate isboth safe and effective. We will need to demonstrate such product candidate’s efficacy and monitor its safety throughout the process. If our current or futureclinical trials are unsuccessful, regulatory approval of our product candidates could be delayed or prevented and our business could be harmed.All of our product candidates are subject to the risks of failure inherent in drug development. The results of early-stage clinical trials of our productcandidates do not necessarily predict the results of later-stage clinical trials. Product candidates in later-stage clinical trials may fail to demonstrate desiredsafety and efficacy traits despite having successfully progressed through initial clinical testing. Even if we believe the data collected from clinical trials ofour product candidates is promising, this data may not be sufficient to support approval by the FDA or any other U.S. or foreign regulatory approval. TheFDA may also reject any of our completed clinical trials as inadequate to support approval if the trial design does not include specific safety monitoringmeasures. Pre-clinical and clinical data can be interpreted in different ways. Accordingly, FDA officials could reach different conclusions in assessing suchdata than we do which could delay, limit or prevent regulatory approval. In addition, the FDA, other regulatory authorities, our IRB or we may suspend orterminate clinical trials at any time.Obtaining FDA and other regulatory approvals is complex, time consuming and expensive, and the outcomes are uncertain.The process of obtaining FDA and other regulatory approvals is time consuming, expensive and difficult. Clinical trials are required to establish thesafety and efficacy of product candidates. Applications to market product candidates must be submitted to the FDA which must be reviewed for approval andapproved by the FDA before product candidates may be marketed and clinical trials, manufacturing, and the marketing of products, if approved, are subject tostrict regulatory compliance. The commencement and completion of clinical trials for any of our product candidates could be delayed or prevented by avariety of factors, including:•delays in obtaining regulatory approvals to commence a study or trial;•delays in identifying and reaching agreement on acceptable terms with prospective clinical trial sites;•delays or failures in obtaining approval of our clinical trial protocol from an IRB to conduct a clinical trial at a prospective study site;•delays in the enrollment of patients;•manufacturing difficulties;•failure of our clinical trials and clinical investigators to be in compliance with the FDA’s GCP;•failure of our third-party contract research organizations, clinical site organizations or other clinical trial managers, to satisfy their contractualduties, comply with regulations or meet expected deadlines;•lack of efficacy during clinical trials; or•unforeseen safety issues.We do not know whether our clinical trials will need to be restructured or will be completed on schedule, if at all, or whether they will provide datanecessary to support regulatory approval. Significant delays in clinical trials will impede our ability to commercialize our product candidates and generaterevenue, and could significantly increase our development costs.In addition, we utilize bovine-sourced materials to manufacture our product candidates. It is possible that future FDA regulations may require us tochange the source of the bovine-sourced materials we use in our product candidates or to cease using bovine-sourced materials. If we are required to usealternative materials in our product candidates, and in the event that such alternative materials are available to us, or if we choose to change the materialsused in our product candidates in the future, we would need to validate the new manufacturing process and run comparability trials with any reformulatedproduct candidate, which could delay future clinical trials and the submission for regulatory approval of our product candidates and negatively impact thedevelopment and potential commercialization of our product candidates.32Table of ContentsIf we fail to obtain the necessary regulatory approvals, or if such approvals are limited, we will not be able to commercialize our product candidates, andwe will not generate product revenues.Even if we comply with all FDA pre-approval regulatory requirements, the FDA may determine that our product candidates are not safe or effective,and we may never obtain regulatory approval for such product candidates. If we fail to obtain regulatory approval for some or all of our product candidates,we will have fewer commercial products, if any, and correspondingly lower product revenues, if any. Even if our product candidates receive regulatoryapproval, such approval may involve limitations on the indications and conditions of use or marketing claims for our products. Further, later discovery ofpreviously unknown problems or AEs could result in additional regulatory restrictions, including withdrawal of products and addition of warnings or otherstatements on the product label.In jurisdictions outside the United States, we must receive marketing authorizations from the appropriate regulatory authorities beforecommercializing our product candidates. Regulatory approval processes outside the United States generally include requirements and risks similar to, and inmany cases in excess of, the risks associated with FDA approval.Our failure to comply with extensive governmental regulation may significantly affect our operating results.Even if we obtain regulatory approval for some or all of our product candidates, we will continue to be subject to extensive ongoing requirements bythe FDA, as well as by a number of foreign, national, state and local agencies. These regulations will impact many aspects of our operations, includingtesting, research and development, manufacturing, safety, efficacy, labeling, storage, quality control, AE reporting, import and export, record keeping,approval, distribution, advertising and promotion of our future products. We must also submit new or supplemental applications and obtain FDA approval forcertain changes to an approved product, product labeling or manufacturing process. Application holders must also submit advertising and other promotionalmaterial to the FDA and report on ongoing clinical trials. The FDA enforces post-marketing regulatory requirements, including the cGMP requirements,through periodic unannounced inspections. We do not know whether we will pass any future FDA inspections. Failure to pass an inspection could disrupt,delay or shut down our manufacturing operations. Failure to comply with applicable regulatory requirements could result in, among other things:•administrative or judicial enforcement actions;•changes to advertising;•failure to obtain regulatory approvals for our product candidates;•revocation or suspension of regulatory approvals of products;•product seizures or recalls;•court-ordered injunctions;•import detentions;•delay, interruption or suspension of product manufacturing, distribution, marketing and sales; or•civil or criminal sanctions.The discovery of previously unknown problems with any of our future approved products may result in restrictions on such products, includingwithdrawal from the market. In addition, the FDA may revisit and change its prior determinations with regard to the safety or efficacy of our future approvedproducts. If the FDA’s position changes, we may be required to change our labeling or cease to manufacture and market our future approved products. Evenprior to any formal regulatory action, we could voluntarily decide to cease the distribution and sale or recall any of our future approved products if concernsabout their safety or efficacy develop.In their regulation of advertising and promotion, the FDA may issue correspondence alleging that some advertising or promotional practices arefalse, misleading or deceptive. The FDA is authorized to impose a wide array of sanctions on companies for such advertising and promotion practices, whichcould result in any of the following:•incurring substantial expenses, including fines, penalties, legal fees and costs to comply with the FDA’s requirements;•changes in the methods of marketing and selling products;•taking FDA mandated corrective action, which may include placing advertisements or sending letters to physicians rescinding previousadvertisements or promotions; or33Table of Contents•disruption in the distribution of products and loss of sales until compliance with the FDA’s position is obtained.Improper promotional activities may also lead to investigations by federal or state prosecutors, and result in criminal and civil penalties. If webecome subject to any of the above requirements, it could be damaging to our reputation and restrict our ability to sell or market our future approvedproducts, and our business condition could be adversely affected. We may also incur significant expenses in defending ourselves.Physicians may prescribe pharmaceutical or biologic products for uses that are not described in a product’s labeling or differ from those tested by usand approved by the FDA. While such “off-label” uses are common and the FDA does not regulate physicians’ choice of treatments, the FDA does restrict amanufacturer’s communications on the subject of off-label use. Companies cannot promote FDA-approved pharmaceutical or biologic products for off-labeluses, but under certain limited circumstances they may disseminate to practitioners’ articles published in peer-reviewed journals. To the extent allowed bythe FDA, we may disseminate peer-reviewed articles on our future approved products to practitioners. If, however, our activities fail to comply with the FDA’sregulations or guidelines, we may be subject to warnings from, or enforcement action by, the FDA or other regulatory or law enforcement authorities.Our sales, marketing, and scientific/educational grant programs, if any in the future, must also comply with applicable requirements of variousfederal and state anti-fraud and abuse laws and regulations including the federal Anti-Kickback Statute, the federal civil False Claims Act, HIPAA’s anti-fraudprovisions, the federal Physician Payment Sunshine Act (Open Payment Program), and similar federal and state fraud and abuse laws and regulations.Additional information about the scope of these requirements is offered under “Other U.S. Regulatory Requirements” in the Government Regulatory sectionabove.Depending on the circumstances, failure to meet post-approval requirements can result in criminal prosecution, fines or other penalties, injunctions,recall or seizure of products, total or partial suspension of production, denial or withdrawal of pre-marketing product approvals, or refusal to allow us to enterinto supply contracts, including government contracts. Any government investigation of alleged violations of law could require us to expend significanttime and resources in response, and could generate negative publicity.We are subject to significant regulation with respect to the manufacturing of our product candidates.Components of a finished therapeutic product approved for commercial sale or used in late-stage clinical trials must be manufactured in accordancewith cGMP. These regulations govern manufacturing processes and procedures and the implementation and operation of quality systems to control andassure the quality of investigational products and products approved for sale. Our facilities and quality systems and the facilities and quality systems of someor all of our third party manufacturers and suppliers must pass inspection for compliance with the applicable regulations as a condition of FDA approval ofour products. In addition, the FDA may, at any time, audit or inspect a manufacturing facility, including our manufacturing facility or our associated qualitysystems for compliance with the regulations applicable to the activities being conducted. The FDA also may, at any time following approval of a product forsale, audit our manufacturing facilities or those of our third party manufacturers. If any such inspection or audit identifies a failure to comply with applicableregulations or if a violation of our product specifications or applicable regulation occurs independent of such an inspection or audit, we or the FDA mayrequire remedial measures that may be costly and/or time consuming for us or a third party to implement and that may include the temporary or permanentsuspension of a clinical trial or commercial sales, recalls, market withdrawals, seizures or the temporary or permanent closure of a facility. Any such remedialmeasures imposed upon us or third parties with whom we contract could materially harm our business.If we fail to obtain or maintain orphan drug exclusivity for any of our product candidates, our competitors may sell products to treat the same conditionsand our operations will be adversely impacted.As part of our business strategy, we have obtained FDA Orphan Drug Designation for FCX-007 and FCX-013. Under the Orphan Drug Act, the FDAmay designate a product as an orphan drug if it is intended to treat a rare disease or condition, defined as a patient population of fewer than 200,000 in theU.S. The first company to obtain FDA approval for a designated orphan drug for a given rare disease receives marketing exclusivity for use of that drug for thestated condition for a period of seven years. Orphan drug exclusive marketing rights may be lost if the FDA later determines that the request for designationwas materially defective or if the manufacturer is unable to assure sufficient quantity of the drug or access to the biologic. Because the extent and scope of patent protection for some of our product candidates is limited, Orphan Drug Designation is especially important forour product candidates that are eligible for Orphan Drug Designation. For eligible product candidates, we plan to rely, in part, on the exclusivity period underthe Orphan Drug Act to maintain a competitive34Table of Contentsposition. If we do not obtain orphan drug exclusivity for our product candidates that do not have broad patent protection, our competitors may then sell thesame drug or biologic to treat the same condition which could adversely affect our operations.Even though we have obtained Orphan Drug Designation for FCX-007 and FCX-013 and even if we obtain Orphan Drug Designation for otherpotential product candidates in the future, due to the uncertainties associated with developing biopharmaceutical products, we may not be the first to obtainregulatory approval for any particular orphan indication, which means that we may not obtain orphan drug exclusivity and could also potentially be blockedfrom approval until the first product’s orphan drug exclusivity period expires. Further, even if we obtain orphan drug exclusivity for a product, thatexclusivity may not effectively protect the product from competition because different drugs can be approved for the same condition. Even after an orphandrug is approved and granted orphan drug exclusivity, the FDA can subsequently approve the same drug or biologic for the same condition if the FDAconcludes that the later drug or biologic is safer, more effective or makes a major contribution to patient care. Orphan Drug Designation does not shorten theregulatory review and approval process, nor does it provide any additional opportunities for review and guidance from the FDA during the review andapproval process.Even if we were to obtain approval for FCX-007 or FCX-013 with the Rare Pediatric Disease Designation, the Rare Pediatric Disease PRV Program mayno longer be in effect at the time of such approval.FCX-007 has received Rare Pediatric Disease Designation from the FDA for the treatment of RDEB and FCX-013 has received Rare Pediatric DiseaseDesignation from the FDA for the treatment of localized scleroderma. The FDA defines a “rare pediatric disease” as a disease that affects fewer than 200,000individuals in the U.S. primarily under the age of 18 years old. Under the FDA’s Rare Pediatric Disease PRV program, upon the approval of a NDA or BLA forthe treatment of a rare pediatric disease, the sponsor of such application would be eligible for a Rare Pediatric Disease PRV that can be used to obtain priorityreview for a subsequent NDA or BLA. The PRV may be sold or transferred an unlimited number of times. Congress has extended the PRV program untilSeptember 30, 2020. This program has been subject to criticism, including by the FDA, and it is possible that even if we obtain approval for FCX-007 andFCX-013 and qualify for such a PRV, the program may no longer be in effect at the time of approval.We are largely dependent on the future commercial success of our product candidates. Our ability to generate revenues and become profitable will depend in large part on the future commercial success of our product candidates. If anyproduct that we commercialize in the future does not gain an adequate level of acceptance among physicians, patients and third parties, we may not generatesignificant product revenues or become profitable. Market acceptance of our products that we commercialize, by physicians, patients and third party payorswill depend on a number of factors, some of which are beyond our control, including:•The efficacy, safety and other potential advantages in relation to alternative treatments;•The relative convenience and ease of administration;•The availability of adequate coverage or reimbursement by third parties, such as insurance companies and other healthcare payors, and bygovernment healthcare programs, including Medicare and Medicaid;•The prevalence and severity of adverse events;•The cost of treatment in relation to alternative treatments, including generic or biosimilar products;•The extent and strength of our third party manufacturer and supplier support;•The extent and strength of marketing and distribution support;•The limitations or warnings contained in a product’s FDA approved labeling; and•Distribution and use restrictions imposed by the FDA or to which we agree as part of a mandatory REMS or voluntary risk management planFor example, even if our products have been approved by the FDA, physicians and patients may not immediately be receptive to them and may beslow to adopt them. In addition, even though we believe our product candidates have significant advantages to other treatment options, because no head-to-head trials comparing our product candidates to competing products will have been conducted, the prescribing information approved by the FDA would notcontain claims that our product is safer or more effective than competitive products. Accordingly, promotion of our products will not reflect any comparativeadvantages that may exist. If our products do not achieve an adequate level of acceptance among physicians, patients and third party payors, we may notgenerate meaningful revenues and we may not become profitable.35Table of ContentsIn order to commercialize any of our product candidates, we will need to increase our manufacturing capacity and improve our manufacturingcapabilities, which will require significant expenditures and regulatory approval.We currently have limited manufacturing capacity. In order to commercialize any of our product candidates, we will need to increase ourmanufacturing capacity. We are developing enhancements and alternatives to our current manufacturing process. If we have difficulties in increasing ourmanufacturing capacity and improving our capabilities, we will be limited in our ability to manufacture and commercialize our product candidates, if theyare approved for marketing; and we may not be able to decrease our manufacturing costs. These difficulties could adversely affect our financial performanceand damage our reputation. Even if we are successful in developing such enhancements or finding alternatives to our current process, such manufacturingchanges will require additional expenditures, for which we may be required to seek external financing. In addition, our ability to increase our manufacturingcapacity or modify our manufacturing processes will be subject to additional FDA review and approval.Negative public opinion and increased regulatory scrutiny of gene therapies may damage public perception of our product candidates or adversely affectour ability to conduct our business or obtain regulatory approvals for our product candidates.Public perception may be influenced by claims that gene therapies are unsafe, and gene therapies may not gain the acceptance of the public or themedical community. In particular, our success will depend upon physicians specializing in the treatment of those diseases that our product candidates targetprescribing treatments that involve the use of our product candidates in lieu of, or in addition to, existing treatments they are already familiar with and forwhich greater clinical data may be available. More restrictive government regulations or negative public opinion would have a negative effect on ourbusiness or financial condition and may delay or impair the development and commercialization of our product candidates or demand for any products wemay develop. Adverse events in our clinical trials, even if not ultimately attributable to our product candidates and the resulting publicity could result inincreased governmental regulation, unfavorable public perception, potential regulatory delays in the testing or approval of our potential product candidates,stricter labeling requirements for those product candidates that are approved and a decrease in demand for any such product candidates.Future sales of our products are subject to adequate coverage, pricing and reimbursement from third-party payors, which are subject to increasing andintense pressure from political, social, competitive and other sources. Our inability to obtain and maintain adequate coverage, pricing or reimbursement,could have an adverse effect on our business.Future sales of our product candidates, should they receive regulatory approval and be commercialized, are dependent, in large part, on theavailability and extent of coverage, pricing and reimbursement from government health administration authorities, private health insurers and otherorganizations. When a new pharmaceutical or biologic product is approved, the availability of government and private reimbursement for that product maybe uncertain, as is the pricing and amount for which that product will be reimbursed. The manner and level at which reimbursement is provided for servicesrelated to our product candidates (e.g., for administration of our products to patients) is also important. Inadequate reimbursement for such services may leadto physician resistance and adversely affect our ability to market or sell our products.Pricing and reimbursement for our products and services related to our products may be adversely affected by a number of factors, including:•changes in federal, state or foreign government regulations or private third-party payors’ reimbursement policies;•pressure by employers on private health insurance plans to reduce costs; and•consolidation and increasing assertiveness of payors, including managed care organizations, health insurers, pharmacy benefit managers,government health administration authorities, private health insurers and other organizations, seeking price discounts or rebates inconnection with the placement of our products on their formularies and, in some cases, the imposition of restrictions on access or coverageof particular drugs or biologics pricing determined based on perceived value.Our failure to maintain adequate coverage, pricing, or reimbursement for our products and services related to our products would have an adverseeffect on our business, revenues and results of operation, could curtail or eliminate our ability to adequately fund research and development programs for thediscovery and commercialization of new product candidates, and could cause a decline in our stock price. Drug pricing and other health care costs are under significant scrutiny in the U.S. and are subject to intense political and societal pressures which weanticipate will continue and escalate on a global basis. As a result, our business and reputation36Table of Contentsmay be harmed, our stock price may be adversely impacted and experience periods of volatility, and our results of operations may be adversely impacted.If the market opportunities for our product candidates are smaller than we believe they are, our results of operations may be adversely affected and ourbusiness may suffer.We focus our research and product development on treatments of diseases affecting the skin and connective tissue. Our projections of both thenumber of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment with ourproduct candidates, are based on estimates. These estimates may prove to be incorrect and new studies or clinical trials may change the estimated incidenceor prevalence of these diseases. The number of patients in the U.S. and elsewhere may turn out to be lower than expected, may not be otherwise amenable totreatment with our products, or new patients may become increasingly difficult to identify or gain access to, all of which would adversely affect our results ofoperations and our business.If any of our approved products were to become the subject of problems related to their efficacy, safety, or otherwise, our business would be seriouslyharmed.Any of our product candidates that may be approved by the FDA will be subject to continual review by the FDA, and we cannot assure you thatnewly discovered or developed safety issues will not arise. For all of our product candidates, the FDA has required us to pay special attention to potentialskin cancer and hypersensitivity reactions at the site of injection and, while we have seen no issues to date, we cannot rule out that issues may arise in thefuture. With the use of any newly marketed drug by a wider patient population, serious AEs may occur from time to time that initially do not appear to relateto the drug itself. Any safety issues could cause us to suspend or cease marketing of our approved products, cause us to modify how we market our approvedproducts, subject us to substantial liabilities, and adversely affect our financial condition and business.If physicians do not follow our established protocols, the efficacy and safety of our product candidates may be adversely affected.We are dependent on physicians and other healthcare professionals to follow our established protocols as to the administration and the handling ofour product candidates in connection with our clinical trials, and we will continue to be dependent on physicians and other healthcare professionals to followsuch protocols if and when our product candidates are commercialized. The treatment protocol requires each physician to verify the patient’s name and dateof birth with the patient and the patient records immediately prior to injection. In the event more than one patient’s cells are delivered to a physician or wedeliver the wrong patient’s cells to the physician, which has occurred in the past, it is the physician’s obligation to follow the treatment protocol and assurethat the patient is treated with the correct cells. If the physicians and other healthcare professionals do not follow our protocol, the efficacy and safety of ourproduct candidates may be adversely affected.Our product candidates may face competition in the future from other pharmaceutical, medical device and biotechnology companies that may havesuperior products, manufacturing capabilities, financial resources or marketing position.The Biologics Price Competition and Innovation Act of 2009 (BPCIA), established an abbreviated pathway for the approval of follow-on biologicalproducts. This abbreviated regulatory pathway establishes legal authority for the FDA to review and approve biosimilar biologics, including the possibledesignation of a biosimilar as “interchangeable” to an existing brand product, or reference product. Under the BPCIA, an application for a biosimilar productcannot be approved by the FDA until 12 years after the reference product was approved under a BLA. These market and data exclusivities do not preventanother company from developing a product that is highly similar to the original branded product, generating its own data and seeking approval. Market anddata exclusivity only assures that another company cannot rely upon the data within the innovator’s application to support the biosimilar product’s approval.FDA’s implementation and interpretation of the BPCIA continues to evolve and could have a material adverse effect on the future commercialprospects for our product candidates, if they are approved.We may be liable for product liability claims not covered by insurance.Physicians, patients and clinical trial participants who have used our products in the past or who use them in the future may bring product liabilityclaims against us. While we have taken, and continue to take, what we believe are appropriate precautions, we may be unable to avoid significant liabilityexposure. We currently keep in force product liability insurance, although such insurance may not be adequate to fully cover any potential claims or maylapse in accordance with its terms prior37Table of Contentsto the assertion of claims. We may be unable to obtain product liability insurance in the future, or we may be unable to do so on acceptable terms. Anyinsurance we obtain or have obtained in the past may not provide adequate coverage against any asserted claims. In addition, regardless of merit or eventualoutcome, product liability claims may result in:•diversion of management’s time and attention;•expenditure of large amounts of cash on legal fees, expenses and payment of damages;•decreased demand for our products or any of our future products and services; or•injury to our reputation.If we are the subject of any future product liability claims, our business could be adversely affected, and if these claims are in excess of insurancecoverage, if any, that we may possess, our financial position will suffer.Risks Related to Our Dependence on Third PartiesWe will incur additional expenses in connection with our exclusive channel collaboration agreements with Intrexon.Pursuant to our exclusive channel collaboration agreements with Intrexon, we are responsible for future research, development andcommercialization expenses of product candidates developed under such collaborations, including FCX-007, FCX-013 and our gene therapy program forarthritis and related conditions, the effect of which we expect will increase the level of our overall research and development expenses going forward.Although all manufacturing, pre-clinical studies and human clinical trials are expensive and difficult to design and implement, costs associated with themanufacturing, research and development of gene therapy product candidates are generally greater in comparison to small molecule product candidates. Wehave added personnel and expect to add additional personnel, either directly or through consulting arrangements, to support our exclusive channelcollaborations with Intrexon.Because development activities are determined pursuant to a joint steering committee comprised of Intrexon and ourselves and we have limitedexperience, future development costs associated with our product candidates may be difficult to anticipate and exceed our expectations. Our actual cashrequirements may vary materially from our current expectations for a number of other factors that may include, but are not limited to, unanticipated technicalchallenges, changes in the focus and direction of our development activities or adjustments necessitated by changes in the competitive landscape in whichwe operate. If we are unable to continue to financially support such collaborations due to our own working capital constraints, we may be forced todiscontinue the collaborations or delay our activities.We may not be able to retain the exclusive rights licensed to us by Intrexon to develop and commercialize our product candidates.Pursuant to our exclusive channel collaboration agreements, we are using Intrexon’s technology in connection with all of our product candidates. The collaboration agreements grant us a license to use patents and other intellectual property of Intrexon in connection with the research, development, andcommercialization of collaboration products within “Fields” that we set forth above in the “Item 1. Business - Intrexon Collaboration”.The exclusive channel collaboration agreements may be terminated by Intrexon if we fail to exercise diligent efforts in developing products throughthe collaborations or if we elect not to pursue the development of a therapy in a “Field” identified by Intrexon that is a “Superior Therapy” as defined in thecollaboration agreements. Upon such termination, the product candidates covered by the applicable exclusive channel collaboration agreement in activeand ongoing Phase II or III clinical trials or later stage development through the exclusive channel collaboration agreement shall be entitled to be continuedby us with a continuation of the related royalties for such product candidates, and all rights to products covered by the exclusive channel collaborationagreement still in an earlier stage of development shall revert to Intrexon.There can be no assurance that we will be able to successfully perform under the exclusive channel collaboration agreements and if any of theagreements are terminated it may prevent us from achieving our business objectives and our business may be harmed.38Table of ContentsWe depend on third parties to conduct our pre-clinical studies and clinical trials, which may result in costs and delays that prevent us from obtainingregulatory approval or successfully commercializing our product candidates.We engage third parties to perform various aspects of our pre-clinical studies and clinical trials. For instance, we obtain viral vector from a solesource supplier in connection with the clinical development of FCX-007 and FCX-013. We depend on these third parties to perform these activities on atimely basis in accordance with the protocol, good laboratory practices, good clinical practices, and other regulatory requirements. Our reliance on these thirdparties for pre-clinical and clinical development activities reduces our control over these activities. Accordingly, if these parties do not successfully carry outtheir contractual duties or obligations or meet expected deadlines, our pre-clinical studies and clinical trials may be extended, delayed, terminated or our datamay be rejected by the FDA. For example, if our sole source supplier of viral vector in connection with the clinical development of FCX-007 or FCX-013were to cease to be able to supply viral vector to us, or decline to supply viral vector to us, our FCX-007 and FCX-013 programs would be delayed until weobtained an alternative source, which could take a considerable length of time. If it became necessary to replace a third party that was assisting with one ofour pre-clinical studies or clinical trials, we believe that there are a number of other third-party contractors that could be engaged to continue these activities,although it may result in a delay of the applicable pre-clinical study or clinical trial. If there are delays in testing or obtaining regulatory approvals as a resultof a third party’s failure to perform, our drug discovery and development costs will likely increase, and we may not be able to obtain regulatory approval foror successfully commercialize our product candidates.We have limited manufacturing capacity and any manufacturing difficulties, disruptions or delays could adversely affect our ability to conduct ourclinical trials.Manufacturing biologic products is difficult, complex and highly regulated. During 2016, we began to manufacture the pre-clinical supply of ourFCX-013 product candidate in our facility in Exton, PA. We lease and operate our own manufacturing facility located in Exton, Pennsylvania. We havehistorically used this facility to manufacture our non-genetically modified products and during 2016 began using this facility for pre-clinical manufacturingof our gene therapy product candidate, FCX-013. We designated our Exton, PA cGMP manufacturing facility in Exton, PA as the production site for FCX-007 in the fourth quarter of 2017, and are currently producing drug product for the FCX-013 clinical trial at that facility. We also plan to manufacture FCX-013 at the same location. Our ability to adequately and timely manufacture and supply our product candidates is dependent on the operation of our solefacility which may be impacted by, among other things:•availability, performance, or contamination of raw materials and components used in the manufacturing process, particularly those for which wehave no other source or supplier;•capacity of our facility and those of contract manufacturer;•the performance of information technology systems;•compliance with regulatory requirements;•inclement weather and natural disasters;•changes in forecasts of future demand for product components;•timing and actual number of production runs for product components;•potential facility contamination by microorganisms or viruses;•updating of manufacturing specifications; and•product quality success rates and yields.If the efficient manufacture and supply of our product candidates is interrupted, we may experience delayed shipments or supply constraints, whichmay materially impact our ongoing and future pre-clinical studies and clinical trials.Our manufacturing processes and those of our contract manufacturer must undergo a potentially lengthy FDA approval process, as well as otherregulatory approval processes, and are subject to continued review by the FDA and other regulatory authorities. It is a multi-year process to build and licensea new manufacturing facility and it can take significant time to qualify and license a contract manufacturer.If regulatory authorities determine that we or our contract manufacturer or certain of our third-party service providers have violated regulations or ifthey restrict, suspend or revoke our prior approvals, they could prohibit us from manufacturing our products or conducting clinical trials or selling ourmarketed products until we or the affected third-party service providers comply, or indefinitely. Because our third-party service providers are subject to theFDA and, potentially, in the future, foreign39Table of Contentsregulatory authorities, alternative qualified third-party service providers may not be available on a timely basis or at all. If we or our third-party serviceproviders cease or interrupt production or if our third-party service providers fail to supply materials, products or services to us, we may experience delayedshipments, and supply constraints for our products.Our research, development and manufacturing operations depend on two facilities for all of our product candidates. If one or more of such facilities isdestroyed or is out of operation for a substantial period of time, our business may be adversely impacted.We currently conduct our research, development and manufacturing operations related to our product candidates in our facility located in Exton,Pennsylvania. Previously we outsourced certain manufacturing of our genetically-modified product candidate FCX-007, to a contract manufacturer with afacility located in Mountain View, California.If regulatory, manufacturing or other problems require us to discontinue production at our Exton, PA facility, we will not be able to have supplies forour pre-clinical studies and clinical trials, which would adversely impact our business. If the facility or the equipment in it is significantly damaged ordestroyed by fire, flood, power loss or similar events, we may not be able to quickly or inexpensively replace our or our contract manufacturer’s facility. Inthe event of a temporary or protracted loss of either facility or equipment, we might not be able to transfer manufacturing to a third party. Even if we couldtransfer manufacturing to a third party, the shift would likely be expensive and time-consuming, particularly since the new facility would need to complywith necessary regulatory requirements.Risks Related to Our Intellectual PropertyIf we or our licensors are unable to protect our intellectual property rights or if our intellectual property rights are inadequate for our technologies andproduct candidates, our competitive position could be harmed.Our commercial success will depend in large part on our, and our licensors, ability to obtain and maintain patent and other intellectual propertyprotection in the U.S. and other countries with respect to our proprietary technology and our product candidates. We rely on trade secret, patent, copyrightand trademark laws, and confidentiality, licensing and other agreements with employees and third parties, all of which offer only limited protection. We seekto protect our proprietary position by filing and prosecuting patent applications in the U.S. and abroad related to our novel technologies and productcandidates that are important to our business.The patent positions of biopharmaceutical companies generally are highly uncertain, involve complex legal and factual questions and have inrecent years been the subject of much litigation. As a result, the issuance, scope, validity, enforceability and commercial value of our patents, including thosepatent rights licensed to us by third parties, are highly uncertain. The steps we or our licensors have taken to protect our proprietary rights may not beadequate to preclude misappropriation of our proprietary information or infringement of our intellectual property rights, both inside and outside the U.S.Further, the examination process may require us or our licensors to narrow the claims for our pending patent applications, which may limit the scope of patentprotection that may be obtained if these applications issue. The rights already granted under any of our currently issued patents or those licensed to us andthose that may be granted under future issued patents may not provide us with the proprietary protection or competitive advantages we are seeking. If we orour licensors are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection obtained is notsufficient, our competitors could develop and commercialize technology and products similar or superior to ours, and our ability to successfullycommercialize our technology and products may be adversely affected. It is also possible that we or our licensors will fail to identify patentable aspects ofinventions made in the course of our development and commercialization activities before it is too late to obtain patent protection on them.With respect to patent rights, we do not know whether any of the pending patent applications for any of our therapies will result in the issuance ofpatents that protect our technology or products, or if any of our or our licensors’ issued patents will effectively prevent others from commercializingcompetitive technologies and product candidates. Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patentapplications in the U.S. and other jurisdictions are typically not published until 18 months after filing or in some cases not at all, until they are issued as apatent. Therefore, we cannot be certain that we or our licensors were the first to make the inventions claimed in our owned or licensed patents or pendingpatent applications, or that we or our licensors were the first to file for patent protection of such inventions.Our issued patents, those that may be issued in the future or those licensed or acquired by us, may be challenged, invalidated or circumvented, andthe rights granted under any issued patent may not provide us with proprietary protection or competitive advantages against competitors with similartechnology. In particular, we do not know if competitors will be able40Table of Contentsto design variations on our treatment methods to circumvent our current and anticipated patent claims. Furthermore, competitors may independently developsimilar technologies or duplicate any technology developed by us.Our pending applications cannot be enforced against third parties practicing the technology claimed in such applications unless and until a patentissues from such applications. Because the issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, issued patents thatwe own or have licensed from third parties may be challenged in the courts or patent offices in the U.S. and abroad. Such challenges may result in the loss ofpatent protection, the narrowing of claims in such patents or the invalidity or unenforceability of such patents, which could limit our ability to stop othersfrom using or commercializing similar or identical technology and products, or limit the duration of the patent protection for our technology and products.Protecting against the unauthorized use of our or our licensor’s patented technology, trademarks and other intellectual property rights is expensive, difficultand may in some cases not be possible. In some cases, it may be difficult or impossible to detect third-party infringement or misappropriation of ourintellectual property rights, even in relation to issued patent claims, and proving any such infringement may be even more difficult.Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain andcould harm our business.Our commercial success depends upon our ability to develop, manufacture, and if approved, market and sell our product candidates and to use ourrelated proprietary technologies. We may become party to, or threatened with, future adversarial proceedings or litigation regarding intellectual propertyrights with respect to our product candidates, including interference, post grant review, inter partes review or derivation proceedings before the U.S. Patentand Trademark Office, or USPTO. Third parties may assert infringement claims against us based on existing patents or patents that may be granted in thefuture. If we are found to infringe a third party’s intellectual property rights, we could be required to obtain a license from such third party in order to be ableto commercialize any of our product candidates that obtain regulatory approval. However, we may not be able to obtain any required license oncommercially reasonable terms or at all. Under certain circumstances, we could be forced, including by court order, to cease commercializing our futureapproved products and then expend time and funding to redesign such products so that such products do not infringe others’ patents while still allowing usto compete in the market with a substantially similar product. In addition, in any such proceeding or litigation, we could be found liable for monetarydamages. A finding of infringement could prevent us from commercializing any of our product candidates that obtain regulatory approval or force us to ceasesome of our business operations, which could materially harm our business. Any claims by third parties that we have misappropriated their confidentialinformation or trade secrets could have a similar negative impact on our business. In addition, our involvement in any of these proceedings may cause us toincur substantial costs and result in diversion of management and technical personnel. Furthermore, parties making claims against us may be able to obtaininjunctive or other equitable relief that could effectively block our ability to develop, commercialize and sell products, and could result in the award ofsubstantial damages against us.We believe that use of our product candidates in clinical trials falls within the scope of the exemptions provided by 35 U.S.C. Section 271(e) in theU.S., which exempts from patent infringement liability activities reasonably related to the development and submission of information to the FDA. As ourproduct candidates progress toward regulatory approval and commercialization, the possibility of a patent infringement claim against us increases. Weattempt to ensure that our product candidates and the methods we employ to manufacture them, as well as the methods for their use we intend to promote, donot infringe other parties’ patents and other proprietary rights. There can be no assurance they do not, however, and competitors or other parties may assertthat we infringe their proprietary rights in any event.We may not be able to protect our intellectual property rights throughout the world.Filing, prosecuting and defending patents on all of our product candidates throughout the world would be prohibitively expensive, and our or ourlicensors’ intellectual property rights in some countries outside the U.S. can be less extensive than those in the U.S. In addition, the laws and practices ofsome foreign countries do not protect intellectual property rights to the same extent as federal and state laws in the U.S. Consequently, we and our licensorsmay not be able to prevent third parties from practicing our and our licensors’ inventions in all countries outside the U.S., or from selling or importingproducts made using our and our licensors’ inventions in and into the U.S. or other jurisdictions. Competitors may use our technologies in jurisdictions wherewe have not obtained patent protection to develop their own products, and may export otherwise infringing products to territories where we or our licensorshave patent protection, but where enforcement is not as strong as that in the U.S. These products may compete with our products in jurisdictions where we donot have any issued patents and our patent claims or other intellectual property rights may not be effective or sufficient to prevent them from so competing.Many companies have encountered significant problems in protecting and defending intellectual property rights in certain foreign jurisdictions. Thelegal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents and other intellectual propertyprotection, particularly those relating to biopharmaceuticals, which could41Table of Contentsmake it difficult for us to stop the infringement of our or our licensor’s patents or marketing of competing products in violation of our proprietary rightsgenerally in those countries. Proceedings to enforce our patent rights in foreign jurisdictions could result in substantial cost and divert our efforts andattention from other aspects of our business, could put our and our licensors’ patents at risk of being invalidated or interpreted narrowly and our and ourlicensors’ patent applications at risk of not issuing and could provoke third parties to assert claims against us or our licensors. We or our licensors may notprevail in any lawsuits that we or our licensors initiate and the damages or other remedies awarded, if any, may not be commercially meaningful.The laws of certain foreign countries may not protect our rights to the same extent as the laws of the U.S., and these foreign laws may also be subjectto change. For example, methods of treatment and manufacturing processes may not be patentable in certain jurisdictions, and the requirements forpatentability may differ in certain countries, particularly developing countries. Furthermore, competitors may challenge the scope, validity or enforceabilityof our or our licensors’ patents, requiring us or our licensors to engage in complex, lengthy and costly litigation or other proceedings. Biopharmaceuticalcompanies may develop, seek approval for, and launch biosimilar versions of our products. Many countries, including European Union countries, India,Japan and China, have compulsory licensing laws under which a patent owner may be compelled under certain circumstances to grant licenses to thirdparties. In those countries, we and our licensors may have limited remedies if patents are infringed or if we or our licensors are compelled to grant a license toa third party, which could materially diminish the value of those patents. This could limit our potential revenue opportunities. Accordingly, our and ourlicensors’ efforts to enforce intellectual property rights around the world may be inadequate to obtain a significant commercial advantage from theintellectual property that we own or license.Patent terms may be inadequate to protect our competitive position on our product candidates for an adequate amount of time.Given the amount of time required for the development, testing and regulatory review of our product candidates, patents protecting such productcandidates might expire before or shortly after such product candidates are commercialized. We expect to seek extensions of patent terms in the U.S. and, ifavailable, in other countries where we are prosecuting patents. In the U.S., the Drug Price Competition and Patent Term Restoration Act of 1984 permits apatent term extension of up to five years beyond the normal expiration of the patent, which is limited to the approved indication (or any additionalindications approved during the period of extension). However, the applicable authorities, including the FDA and the USPTO in the U.S., and any equivalentregulatory authority in other countries, may not agree with our assessment of whether such extensions are available, and may refuse to grant extensions to ourpatents, or may grant more limited extensions than we request. If this occurs, our competitors may be able to take advantage of our investment indevelopment and clinical trials by referencing our clinical and pre-clinical data and launch their product earlier than might otherwise be the case.Changes in patent law, including recent patent reform legislation, could increase the uncertainties and costs surrounding the prosecution of our patentapplications and the enforcement or defense of our issued patents.As is the case with other biopharmaceutical companies, our success is heavily dependent on intellectual property, particularly patents. Obtainingand enforcing patents in the biopharmaceutical industry involve technological and legal complexity, and obtaining and enforcing pharmaceutical patents iscostly, time-consuming, and inherently uncertain. Changes in either the patent laws or interpretation of the patent laws in the U.S. and other countries maydiminish the value of our patents or narrow the scope of our patent protection. For example, the U.S. Supreme Court has ruled on several patent cases inrecent years, either narrowing the scope of patent protection available in certain circumstances or weakening the rights of patent owners in certain situations.In addition to increasing uncertainty with regard to our and our licensors’ ability to obtain patents in the future, this combination of events has createduncertainty with respect to the value of patents, once obtained. Depending on decisions by Congress, the federal courts, and the USPTO, the laws andregulations governing patents could change in unpredictable ways that would weaken our and our licensors’ ability to obtain new patents or to enforceexisting patents and patents we and our licensors may obtain in the future. Recent patent reform legislation could increase the uncertainties and costssurrounding the prosecution of our and our licensors’ patent applications and the enforcement or defense of our or our licensors’ issued patents.In September 2011, the Leahy-Smith America Invents Act (the Leahy-Smith Act), was signed into law. The Leahy-Smith Act includes a number ofsignificant changes to U.S. patent law. These include provisions that affect the way patent applications will be prosecuted and may also affect patentlitigation. In particular, under the Leahy-Smith Act, the U.S. transitioned in March 2013 to a “first to file” system in which the first inventor to file a patentapplication will be entitled to the patent. Third parties are allowed to submit prior art before the issuance of a patent by the USPTO and may become involvedin opposition, derivation, reexamination, post grant review, inter-partes review or interference proceedings challenging our patent rights or the patent rightsof our licensors. An adverse determination in any such submission, proceeding or litigation could reduce the scope of, or invalidate, our or our licensors’patent rights, which could adversely affect our competitive position.42Table of ContentsThe Leahy-Smith Act and its implementation could increase the uncertainties and costs surrounding the prosecution of our patent applications and theenforcement or defense of our issued patents and those licensed to us.Obtaining and maintaining our patent protection depends on compliance with various procedural, document submissions, fee payment and otherrequirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with theserequirements.Periodic maintenance fees on any issued patent are due to be paid to the USPTO and foreign patent agencies in several stages over the lifetime of thepatent. The USPTO and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and othersimilar provisions during the patent application process. While an inadvertent lapse can in many cases be cured by payment of a late fee or by other means inaccordance with the applicable rules, there are situations in which noncompliance can result in abandonment or lapse of the patent or patent application,resulting in partial or complete loss of patent rights in the relevant jurisdiction. Non-compliance events that could result in abandonment or lapse of a patentor patent application include, but are not limited to, failure to respond to official actions within prescribed time limits, non-payment of fees and failure toproperly legalize and submit formal documents. If we or our licensors fail to maintain the patents and patent applications covering our product candidates,our competitive position would be adversely affected.If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.To protect our proprietary know-how and our inventions for which patents may be unobtainable or difficult to obtain, and to maintain ourcompetitive position, we rely on trade secret protection and confidentiality agreements. To this end, it is our general policy to require our employees,consultants, advisors, and contractors to enter into agreements that prohibit the disclosure of confidential information and, where applicable, requiredisclosure and assignment to us of the ideas, developments, discoveries, and inventions important to our business. These agreements may not provideadequate protection for our trade secrets, know-how or other proprietary information in the event of any unauthorized use or disclosure or the lawfuldevelopment by others of such information. Moreover, we may not be able to obtain adequate remedies for any breaches of these agreements. Our tradesecrets may also be obtained by third parties by other means, such as breaches of our physical or computer security systems. Enforcing a claim that a partyillegally disclosed or misappropriated a trade secret is difficult, expensive and time-consuming, and the outcome is unpredictable. In addition, some courtsinside and outside the United States are less willing or unwilling to protect trade secrets. If any of our trade secrets were to be lawfully obtained orindependently developed by a competitor, we would have no right to prevent them, or those to whom they communicate it, from using that technology orinformation to compete with us. If any of our trade secrets, know-how or other proprietary information is disclosed, the value of our trade secrets, know-howand other proprietary rights would be significantly impaired and our business and competitive position would suffer.We may become involved in lawsuits to protect or enforce our intellectual property, which could be expensive, time consuming and unsuccessful and havea material adverse effect on the success of our business.Competitors may infringe our patents or misappropriate or otherwise violate our intellectual property rights. To counter infringement orunauthorized use, litigation may be necessary in the future to enforce or defend our intellectual property rights, to protect our trade secrets or to determine thevalidity and scope of our own intellectual property rights or the proprietary rights of others. Also, third parties may initiate legal proceedings against us orour licensors to challenge the validity or scope of intellectual property rights we own or control. These proceedings can be expensive and time consuming.Many of our current and potential competitors have the ability to dedicate substantially greater resources to defend their intellectual property rights than wecan. Accordingly, despite our efforts, we may not be able to prevent third parties from infringing upon or misappropriating our intellectual property.Litigation could result in substantial costs and diversion of management resources, which could harm our business and financial results. In addition, in aninfringement proceeding, a court may decide that a patent owned by or licensed to us is invalid or unenforceable, or may refuse to stop the other party fromusing the technology at issue on the grounds that our patents do not cover the technology in question. An adverse result in any litigation proceeding couldput one or more of our patents at risk of being invalidated, held unenforceable or interpreted narrowly. Furthermore, because of the substantial amount ofdiscovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised bydisclosure during this type of litigation. There could also be public announcements of the results of hearings, motions or other interim proceedings ordevelopments. If securities analysts or investors perceive these results to be negative, it could have a material adverse effect on the price of shares of ourcommon stock.43Table of ContentsWe may be subject to claims by third parties asserting that our licensors, employees or we have misappropriated their intellectual property, or claimingownership of what we regard as our own intellectual property.Many of our employees and our licensors’ employees, including our senior management, were previously employed at universities or at otherbiotechnology or pharmaceutical companies, including our competitors or potential competitors. Although we try to ensure that our employees do not usethe proprietary information or know-how of others in their work for us, we may be subject to claims that we or these employees have used or disclosedintellectual property, including trade secrets or other proprietary information, of any such third party. Litigation may be necessary to defend against suchclaims. If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights or personnel orsustain damages. Such intellectual property rights could be awarded to a third party, and we could be required to obtain a license from such third party tocommercialize our technology or products. Such a license may not be available on commercially reasonable terms or at all. Even if we are successful indefending against such claims, litigation could result in substantial costs and be a distraction to management.Intellectual property rights do not necessarily address all potential threats to our competitive advantage.The degree of future protection afforded by our intellectual property rights is uncertain because intellectual property rights have limitations, andmay not adequately protect our business, or permit us to maintain our competitive advantage. The following examples are illustrative:•others may be able to make biologics that are the same as or similar to our product candidates, but that are not covered by the claims of thepatents that we own or have exclusively licensed;•we or our licensors or any strategic partners might not have been the first to make the inventions covered by the issued patents or pendingpatent applications that we own or have exclusively licensed;•we or our licensors might not have been the first to file patent applications covering certain of our inventions;•others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our intellectualproperty rights;•it is possible that our pending patent applications will not lead to issued patents;•issued patents that we own or have exclusively licensed may not provide us with any competitive advantages, or may be held invalid orunenforceable as a result of legal challenges;•our competitors might conduct research and development activities in the U.S. and other countries that provide a safe harbor from patentinfringement claims for certain research and development activities, as well as in countries where we do not have patent rights and then use theinformation learned from such activities to develop competitive products for sale in our major commercial markets;•we may not develop additional proprietary technologies that are patentable; and•the patents of others may have an adverse effect on our business.Risks Related to Business OperationsWe are dependent on our executives and other key professionals and the loss of any of these individuals could harm our business.We are dependent on the efforts of our executives and other key scientific, manufacturing and quality personnel. The loss of any of theseindividuals, or our inability to recruit and train additional key personnel in a timely manner, could materially and adversely affect our business and our futureprospects. A loss of one or more of our current executives or other key professionals could severely and negatively impact our operations. All of ouremployees, including our chief executive officer, are employed “at-will,” and any of them may elect to pursue other opportunities at any time. We have nopresent intention of obtaining key man life insurance on any of our executive officers or key professionals.We may need to attract, train and retain additional experienced executives and other key professionals in the future.In the future, we may need to seek additional executives and other key professionals. There is a high demand for experienced executive, scientific,manufacturing and quality personnel in our industry. We do not know whether we will be able to attract, train and retain such experienced personnel in thefuture, which could have a material adverse effect on our business, financial condition and results of operations.44Table of ContentsOur business may be adversely affected by current and potential future healthcare reforms.In the U.S., federal and state legislatures, health agencies and third-party payors continue to focus on containing the cost of health care. Legislativeand regulatory proposals and enactments to reform health care insurance programs could significantly influence the manner in which our product candidates,if approved, are prescribed and purchased. For example, provisions of the Patient Protection and Affordable Care Act (PPACA) have resulted in changes in theway health care is paid for by both governmental and private insurers, including increased rebates owed by manufacturers under the Medicaid Drug RebateProgram, annual fees and taxes on manufacturers of certain branded prescription drugs, the requirement that manufacturers participate in a discount programfor certain outpatient drugs under Medicare Part D and the expansion of the number of hospitals eligible for discounts under Section 340B of the PublicHealth Service Act. In addition, certain legislative changes to and regulatory changes under the Affordable Care Act have occurred in the 115th United StatesCongress and under the Trump Administration. For instance, the Bipartisan Budget Act of 2018 increased the PPACA required manufacturer point-of-salediscount from 50% to 70% off the negotiated price for Medicare Part D beneficiaries during their coverage gap period beginning in 2019. Further legislativechanges to and regulatory changes under the PPACA remain possible.There is also significant economic pressure on state budgets that may result in states increasingly seeking to achieve budget savings throughmechanisms that limit coverage or payment for certain drugs. In recent years, some states have considered legislation and ballot initiatives that would controlthe prices of drugs, including laws to allow importation of pharmaceutical products from lower cost jurisdictions outside the U.S. and laws intended toimpose price controls on state drug purchases. State Medicaid programs are increasingly requesting manufacturers to pay supplemental rebates and requiringprior authorization by the state program for use of any drug for which supplemental rebates are not being paid. Government efforts to reduce Medicaidexpenses may lead to increased use of managed care organizations by Medicaid programs. This may result in managed care organizations influencingprescription decisions for a larger segment of the population and a corresponding constraint on prices and reimbursement for our product candidates, ifapproved. In addition, under the PPACA, as states implement their health care marketplaces or operate under the federal exchange, the impact on drugmanufacturers will depend in part on the formulary and benefit design decisions made by insurance sponsors or plans participating in these programs. It ispossible that we may need to provide discounts or rebates to such plans in order to maintain favorable formulary access for our future product candidates, ifapproved, which could have an adverse impact on our sales and results of operations.If we fail to comply with federal and state healthcare laws, including fraud and abuse and health information privacy and security laws, we could facesubstantial penalties and our business, results of operations, financial condition and prospects could be adversely affected.As a biopharmaceutical company, even though we do not bill directly to Medicare, Medicaid or other third-party payors, certain federal and statehealthcare laws and regulations pertaining to fraud and abuse and patients’ rights are and will be applicable to our business. For example, we could be subjectto healthcare fraud and abuse and patient privacy regulation by both the federal government and the states in which we conduct our business. The laws thatmay affect our ability to operate include, among others:•The federal Anti-Kickback Statute, which constrains our business activities, including our marketing practices, educational programs, pricingpolicies, and relationships with healthcare providers or other entities, by prohibiting, among other things, soliciting, receiving, offering orpaying remuneration, directly or indirectly, to induce, or in return for, either the referral of an individual or the purchase or recommendation ofan item or service reimbursable under a federal healthcare program, such as the Medicare and Medicaid programs;•Federal civil false claims laws and civil monetary penalty laws, which prohibit, individuals or entities from, among other things, knowinglypresenting, or causing to be presented, claims for payment of government funds, or other third-party payors that are false or fraudulent. Criminalprosecution is also possible for making or presenting a false or fictitious or fraudulent claim to the federal government;•HIPAA’s anti-fraud provisions, which prohibit, among other things, executing a scheme to defraud any healthcare benefit program or makingfalse statements relating to healthcare matters;•HIPAA’s privacy and security provisions, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009(HITECH), and its implementing regulations, which imposes certain requirements relating to the privacy, security and transmission ofindividually identifiable health information;•The federal Physician Payment Sunshine Act (implemented as the Open Payments program), which requires pharmaceutical manufacturers toreport annually to CMS for certain “transfers of value” made to teaching hospitals and physicians and any ownership and investment interestsheld by physicians and their immediate family members and applicable group purchasing organizations during the preceding calendar year; and45Table of Contents•State and foreign law equivalents of each of the above federal laws, such as anti-kickback and false claims laws which may apply to items orservices reimbursed by any third-party payor, including commercial insurers; state laws that require pharmaceutical companies to comply withthe industry’s voluntary compliance guidelines and the applicable compliance guidance promulgated by the federal government that otherwiserestricts certain payments that may be made to healthcare providers and entities; state laws that require drug manufacturers to report informationrelated to payments and other transfer of value to physicians and other healthcare providers and entities; and state and foreign laws governingthe privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and often arenot preempted by HIPAA, thus complicating compliance efforts.Additional information about the scope of these requirements is offered under “Other U.S. Regulatory Requirements” in the Government Regulatorysection above. Because of the breadth of these laws and the narrowness of the statutory exceptions and safe harbors available, it is possible that some of ourbusiness activities could be subject to challenge under one or more of such laws. For example, the federal Anti-Kickback Statute is subject to evolvinginterpretations and has been applied by government enforcement officials to a number of common business arrangements in the pharmaceutical industry. Thefederal government has enforced the Anti-Kickback Statute to reach large settlements with pharmaceutical manufacturers based on allegedly sham consultantarrangements with physicians and other arrangements that are common in our industry. The government can establish a violation of the Anti-KickbackStatute without proving that a person or entity had actual knowledge of the law or specific intent to violate it. There are a number of statutory exceptions andregulatory safe harbors protecting certain common activities from prosecution or other regulatory sanctions, but the exceptions and safe harbors are drawnnarrowly. Failure to meet all of the requirements of a particular statutory exception or regulatory safe harbor does not make the conduct per se illegal underthe Anti-Kickback Statute, but the legality of the arrangement will be evaluated on a case-by- case basis based on the totality of the facts and circumstances.In addition, a claim to a federal health care program that includes items or services resulting from a violation of the federal Anti-Kickback Statute constitutesa false or fraudulent claim for purposes of the federal False Claims Act. In addition, settlements with DOJ or other law enforcement agencies have forcedhealthcare companies to agree to additional compliance and reporting requirements as part of a consent decree or corporate integrity agreement.To the extent that any of our product candidates is ultimately sold in a foreign country, we may be subject to similar foreign laws and regulations.If we or our operations are found to be in violation of any of the laws described above or any other governmental regulations that apply to us, wemay be subject to penalties, including administrative, civil and criminal penalties, damages, fines, exclusion from participation in United States federal orstate health care programs, such as Medicare and Medicaid, and the curtailment or restructuring of our operations any of which could materially adverselyaffect our ability to operate our business and our financial results. Although compliance programs can mitigate the risk of investigation and prosecution forviolations of these laws, the risks cannot be entirely eliminated. Any action against us for violation of these laws, even if we successfully defend against it,could cause us to incur significant legal expenses and divert our management’s attention from the operation of our business. Moreover, achieving andsustaining compliance with applicable federal and state privacy, security and fraud laws may prove costly.If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that couldhave a material adverse effect on the success of our business.We are subject to numerous environmental, health and safety laws and regulations, including those governing manufacturing and laboratoryprocedures and the handling, use, storage, treatment and disposal of hazardous materials and wastes. Our operations involve the use of hazardous andflammable materials, including chemicals and biological materials. Our operations also produce hazardous waste products. We generally contract with thirdparties for the disposal of these materials and wastes. We cannot eliminate the risk of contamination or injury from these materials. In the event ofcontamination or injury resulting from our use of hazardous materials, we could be held liable for any resulting damages, and any liability could exceed ourresources. We also could incur significant costs associated with civil or criminal fines and penalties.Although we maintain workers’ compensation insurance to cover us for costs and expenses we may incur due to injuries to our employees resultingfrom the use of hazardous materials or other work-related injuries, this insurance may not provide adequate coverage against potential liabilities. In addition,we may incur substantial costs in order to comply with current or future environmental, health and safety laws and regulations. These current or future lawsand regulations may impair our research, development or production efforts. Failure to comply with these laws and regulations also may result in substantialfines, penalties or other sanctions.46Table of ContentsOur business and operations would suffer in the event of computer system failures.Despite the implementation of security measures, our internal computer systems, and those of our contract research organizations, contractmanufacturing organization, and other third parties on which we rely, are vulnerable to damage from computer viruses, unauthorized access, natural disasters,terrorism, war and telecommunication and electrical failures. System failures, accidents or security breaches could cause interruptions in our operations or theunauthorized transfer of our proprietary information, and could result in a material disruption of our research, pre-clinical and clinical activities and businessoperations, in addition to possibly requiring substantial expenditures of resources to remedy. If such an event were to occur and cause interruptions in ouroperations, it could result in a material disruption of our development programs. For example, the loss of clinical trial data from completed or ongoingclinical trials could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. To the extent thatany disruption or security breach was to result in a loss of or damage to our data or applications, or inappropriate disclosure of confidential or proprietaryinformation, we could incur liability and the further development of our product candidates could be delayed.Our ability to use net operating loss carryforwards to reduce future tax payments may be limited or restricted.We have generated significant net operating loss carryforwards (NOLs) as a result of our incurrence of losses and our conduct of research activitiessince inception. We generally are able to carry NOLs forward to reduce our tax liability in future years. However, our ability to utilize the NOLs is subject tothe rules of Sections 382 and 383 of the Internal Revenue Code of 1986, as amended (the Code). Those sections generally restrict the use of NOLs after an“ownership change.” An ownership change occurs if, among other things, the stockholders (or specified groups of stockholders) who own or have owned,directly or indirectly, 5% or more of a corporation’s common stock or are otherwise treated as 5% stockholders under Section 382 of the Code and the UnitedStates Treasury Department regulations promulgated thereunder increase their aggregate percentage ownership of that corporation’s stock by more than 50percentage points over the lowest percentage of the stock owned by these stockholders over the applicable testing period. In the event of an ownershipchange, Section 382 imposes an annual limitation on the amount of taxable income a corporation may offset with NOL carry forwards and Section 383imposes an annual limitation on the amount of tax a corporation may offset with carry forwards. Any unused annual limitation may be carried over to lateryears until the applicable expiration date for the respective NOL carry forwards. We have completed several financings since our inception which we believehave resulted in “ownership changes” within the meaning of Section 382. We may also experience ownership changes in the future as a result of additionalfinancings and subsequent shifts in our stock ownership. As a result, our NOLs may be subject to limitations and we may be required to pay taxes earlier andin larger amounts than would be the case if our NOLs were freely usable.Risks Related to Ownership of our Common StockThe trading price of the shares of our common stock has been highly volatile, and purchasers of our common stock could incur substantial losses.Our stock began trading on NYSE MKT on May 17, 2013 and then on the Nasdaq Capital Market on August 29, 2014. Between May 17, 2013 andDecember 31, 2017, our common stock has traded between $0.61 and $22.80. Our stock price could be subject to wide fluctuations in response to a variety offactors, which include:•whether our clinical trials can be conducted within the timeframe that we expect and whether such trials will yield positive results;•whether our collaborations with Intrexon can be advanced with positive results within the timeframe and budget that we expect;•changes in laws or regulations applicable to our products or product candidates, including but not limited to clinical trial requirements forapprovals;•unanticipated serious safety concerns related to the use of our product candidates;•a decision to initiate a clinical trial, not to initiate a clinical trial or to terminate an existing clinical trial;•our ability to increase our manufacturing capacity and reduce our manufacturing costs through the improvement of our manufacturing process,our ability to validate any such improvements with the relevant regulatory agencies and our ability to accomplish the foregoing on a timelybasis;•adverse regulatory decisions;•the introduction of new products or technologies offered by us or our competitors;47Table of Contents•negative public opinion or perception of cell and gene therapies;•the inability to effectively manage our growth;•actual or anticipated variations in quarterly operating results;•the failure to meet or exceed the estimates and projections of the investment community;•the perception of the biopharmaceutical industry by the public, legislatures, regulators and the investment community;•the overall performance of the U.S. equity capital markets and general political and economic conditions;•announcements of significant acquisitions, strategic partnerships, joint ventures, collaborations or capital commitments by us or ourcompetitors;•disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain patent protection forour technologies;•additions or departures of key personnel;•the trading volume of our common stock; and•other events or factors, many of which are beyond our control.In addition, the stock market in general, and the stock of biotechnology companies in particular, have experienced extreme price and volumefluctuations that have often been unrelated or disproportionate to the operating performance of these companies. Broad market and industry factors maynegatively affect the market price of our common stock, regardless of our actual operating performance.Randal J. Kirk and certain of his affiliates (including Intrexon) own a substantial percentage of our common stock and will be able to exert significantinfluence over matters subject to stockholder approval.As of March 6, 2018, Randal J. Kirk and certain of his affiliates (including Intrexon, our collaboration partner on our gene therapy programs)beneficially owned approximately 8.3 million shares, or approximately 29%, of our common stock, excluding common stock underlying the Notes andPrivate Placement Warrants issued in connection with the 2016 Private Placement, the Series A Preferred Stock, the March 2017 Warrants and the December2017 Common Warrants. If Randal J. Kirk and certain affiliates exercised the convertible securities or warrants acquired in the September 2016 PrivatePlacement, the Series A Preferred Stock Offering and the December 2017 offering, they would receive, in the aggregate, (i) approximately 2.3 million sharesof our common stock pursuant to exercise of the Private Placement Warrants, (ii) approximately 2.0 million shares of common stock underlying $6,762,500outstanding principal amount of Notes, (iii) approximately 0.1 million shares of common stock underlying accrued interest on the Notes, (iv) approximately1.3 million shares of common stock upon conversion of the Series A Preferred Stock (v) approximately 1.3 million shares of common stock pursuant to theexercise of the March 2017 Warrants and (vi) approximately 2.7 million shares of common stock pursuant to the exercise of the December 2017 CommonWarrants, resulting in the beneficial ownership of approximately 47% of our common stock.Mr. Kirk and his affiliates may have interests that conflict with our other stockholders and, if acting together, have the ability to significantlyinfluence the outcome of matters submitted to our stockholders for approval, including the election and removal of directors and any merger, consolidationor sale of all or substantially all of our assets. This concentration of ownership could delay or prevent any acquisition of our company on terms that otherstockholders may desire.Additionally, two of our directors, Julian Kirk (who is the son of Randal J. Kirk) and Marcus Smith, are employees of Third Security, LLC, which isan affiliate of Randal J. Kirk.Our operating results may fluctuate significantly in the future, which may cause our results to fall below the expectations of securities analysts,stockholders and investors.Our operating results may fluctuate significantly in the future as a result of a variety of factors, many of which are outside of our control. Thesefactors include, but are not limited to:•the timing, implementation and cost of our research, pre-clinical studies and clinical trials;•expenses in connection with our exclusive channel collaboration agreements with Intrexon;•the timely and successful implementation of improved manufacturing processes;48Table of Contents•our ability to attract and retain personnel with the necessary strategic, technical and creative skills required for effective operations;•the amount and timing of expenditures by practitioners and their patients;•introduction of new technologies;•product liability litigation, class action and derivative action litigation, or other litigation;•the amount and timing of capital expenditures and other costs relating to the expansion of our operations;•the state of the debt and/or equity capital markets at the time of any proposed offering we choose to initiate;•our ability to successfully integrate new acquisitions into our operations;•government regulation and legal developments regarding our product candidates in the United States and in the foreign countries in which wemay operate in the future; and•general economic conditions.As a strategic response to changes in the competitive environment, we may from time to time make pricing, service, technology or marketingdecisions or business or technology acquisitions that could have a material adverse effect on our operating results. Due to any of these factors, our operatingresults may fall below the expectations of securities analysts, stockholders and investors in any future period, which may cause our stock price to decline.Future sales of our common stock may depress our stock price.The market price of our common stock could decline as a result of sales of substantial amounts of our common stock in the public market, or as aresult of the perception that these sales could occur, which could occur if we issue a large number of shares of common stock (or securities convertible intoour common stock) in connection with a future financing, as our common stock is trading at low levels. These factors could make it more difficult for us toraise funds through future offerings of common stock or other equity securities. In addition to our common stock outstanding, as of December 31, 2017, wehad warrants and stock options outstanding that were exercisable for a total of 28,353,679 shares of our common stock.Holders of our outstanding preferred shares have dividend, liquidation and other rights that are senior to the rights of the holders of our common shares.Upon our liquidation, dissolution or winding up, the holders of the Series A Preferred Stock are entitled to receive out of our assets, whether capitalor surplus, an amount equal to such holder’s then stated value for each share of Series A Preferred Stock before any distribution to the holders of the commonstock, any class or series of preferred stock and all other common stock equivalents other than those securities which are explicitly senior or pari passu to theSeries A Preferred Stock in redemption, distribution of assets upon a liquidation or dividends. If there are insufficient assets to pay in full such amounts, thenthe available assets will be ratably distributed to the holders of the Series A Preferred Stock in accordance with the respective amounts that would be payableon such shares if all amounts payable thereon were paid in full. This will reduce the remaining amount of our assets, if any, available to distribute to holdersof our common stock.We have not declared any dividends on our common stock to date, and we have no intention of declaring dividends in the foreseeable future.The decision to pay cash dividends on our common stock rests with our Board and will depend on our earnings, unencumbered cash, capitalrequirements and financial condition. We do not anticipate declaring any dividends in the foreseeable future, as we intend to use any excess cash to fund ouroperations. Investors in our common stock should not expect to receive dividend income on their investment, and investors will be dependent on theappreciation of our common stock to earn a return on their investment.Provisions in our charter documents could prevent or delay stockholders’ attempts to replace or remove current members of our Board.Our charter documents provide for staggered terms for the members of our Board. Our Board is divided into three staggered classes, and each directorserves a term of three years. At stockholders’ meetings, only those directors comprising one of the three classes will have completed their term and be subjectto re-election or replacement.49Table of ContentsIn addition, our Board is authorized to issue “blank check” preferred stock, with designations, rights and preferences as they may determine.Accordingly, our Board has in the past and may in the future, without stockholder approval, issue shares of preferred stock with dividend, liquidation,conversion, voting or other rights that could adversely affect the voting power or other rights of the holders of our common stock. This type of preferred stockcould also be issued to discourage, delay or prevent a change in our control.The use of a staggered Board and the ability to issue “blank check” preferred stock are traditional anti-takeover measures. These provisions in ourcharter documents make it difficult for a majority stockholder to gain control of the Board and of our company. These provisions may be beneficial to ourmanagement and our Board in a hostile tender offer and may have an adverse impact on stockholders who may want to participate in such a tender offer, orwho may want to replace some or all of the members of our Board.Provisions in our bylaws provide for indemnification of officers and directors, which could require us to direct funds away from our business and thedevelopment of our product candidates.Our bylaws provide for the indemnification of our officers and directors. We have in the past and may in the future be required to advance costsincurred by an officer or director and to pay judgments, fines and expenses incurred by an officer or director, including reasonable attorneys’ fees, as a resultof actions or proceedings in which our officers and directors are involved by reason of being or having been an officer or director of our company. Funds paidin satisfaction of judgments, fines and expenses may be funds we need for the operation of our business and the development of our product candidates,thereby affecting our ability to attain profitability.An active market for our common stock may not be sustained.In the past, we have had a limited, volatile and sporadic public trading market for our common stock. Although our common stock is listed on theNasdaq Capital Market, an active trading market for our common stock may not be sustained, especially given the large percentage of our common stockheld by our affiliates. If an active market for our common stock is not sustained, it may be difficult for our stockholders to sell shares without depressing themarket price for our common stock.If securities or industry analysts do not publish research or publish inaccurate or unfavorable research about our business, our stock price and tradingvolume could decline.The trading market for our common stock depends in part on the research and reports that securities or industry analysts publish about us or ourbusiness. We do not have any control over these analysts. If one or more of the analysts who cover us downgrade our stock or publish inaccurate orunfavorable research about our business, our stock price would likely decline. If one or more of these analysts cease coverage of our company or fail topublish reports on us regularly, demand for our stock could decrease, which might cause our stock price and trading volume to decline.Item 1B. Unresolved Staff Comments Not applicable.Item 2. Properties Our corporate office and manufacturing facility are located at 405 Eagleview Boulevard, Exton, Pennsylvania. This location consists ofapproximately 17,500 square feet of manufacturing and laboratory space and 69,000 square feet of office space, which we lease pursuant to a lease agreementthat expires on March 31, 2023. We believe this facility is suitable for our current needs.Item 3. Legal ProceedingsWe are not a party to any pending legal proceedings.Item 4. Mine Safety DisclosureNot applicable. 50Table of ContentsPart IIItem 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity SecuritiesMarket InformationOur common stock is traded on the Nasdaq Capital Market under the symbol “FCSC.” The following table sets forth, for the indicated periods, thehigh and low intra-day sales prices per share for our common stock, adjusted to reflect the effect of the reverse stock split of our common stock on March 10,2017, for the two most recent years, as reported on the Nasdaq Capital Market. High LowYear Ended December 31, 2017 First Quarter$3.51 $1.86Second Quarter$4.64 $1.80Third Quarter$4.17 $2.41Fourth Quarter$3.29 $0.61Year Ended December 31, 2016 First Quarter$13.86 $6.12Second Quarter$11.34 $2.73Third Quarter$4.14 $2.10Fourth Quarter$3.15 $1.56The closing price of our common stock on March 6, 2018 was $0.62 as reported on the Nasdaq Capital Market.Holders of RecordAs of March 6, 2018, there were 28,356,351 shares of our common stock outstanding. There were approximately 35 holders of record at March 6,2018. Because many of our shares are held by brokers and other institutions on behalf of stockholders, we are unable to estimate the total number ofstockholders represented by these record holders.DividendsWe have never declared or paid any cash dividend on our common stock and our Board does not intend to do so in the foreseeable future. Thedeclaration and payment of dividends in the future, of which there can be no assurance, will be determined by the Board in light of conditions then existing,including earnings, financial condition, capital requirements and other factors. In addition, our outstanding Notes and our outstanding Series A PreferredStock each restrict our ability to pay cash dividends on our equity securities.Securities Authorized for Issuance under Equity Compensation PlansInformation regarding securities authorized for issuance under equity compensation plans is incorporated by reference into the information in PartIII, Item 12 of this Form 10-K.Recent Sales of Unregistered SecuritiesAll information regarding the issuance of our securities during the year ended December 31, 2017 have been previously disclosed in current reportswe have filed on Form 8-K or in quarterly reports we have filed on Form 10-Q. We did not issue any unregistered equity securities during the quarter endedDecember 31, 2017.Purchases of Equity Securities by the Issuer and Affiliated PurchasersWe did not repurchase any of our equity securities during the year ended December 31, 2017.51Table of Contents Item 6. Selected Financial Data We are a smaller reporting company as defined by Rule 12b-2 of the Securities Exchange Act of 1934 and are not required to provide theinformation under this item.52Table of ContentsItem 7. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe following discussion and analysis of our financial condition and results of operations should be read in conjunction with our audited consolidatedfinancial statements and the related notes included in Part IV of this Form 10-K.OverviewWe are an autologous cell and gene therapy company focused on translating personalized biologics into medical breakthroughs for diseasesaffecting the skin and connective tissue. Our distinctive approach to personalized biologics is based on our proprietary autologous fibroblast technology.Fibroblasts are the most common cell in skin and connective tissue and are responsible for synthesizing extracellular matrix proteins, including collagen andother growth factors, that provide structure and support. Because fibroblasts naturally reside in the localized environment of the skin and connective tissue,they represent an ideal delivery vehicle for proteins targeted to these areas. We target the underlying cause of disease by using fibroblast cells from a patient’sskin and genetically modifying them to create localized therapies that are compatible with the unique biology of the patient (i.e., which are autologous).We are focused on discovering and developing localized therapies for diseases affecting the skin and connective tissue, where there are high unmetneeds, to improve the lives of patients and their families. In that regard, we commit significant resources to our research and development programs.Currently, all of our research and development operations and focus are on gaining regulatory approvals to commercialize our product candidates in theUnited States; however, we may seek to expand into international markets in the futureDevelopment ProgramsOur current pipeline consists of the following product candidates, which we are developing in collaboration with Intrexon:FCX-007 is our clinical-stage, gene therapy product candidate for the treatment of RDEB, a congenital and progressive orphan skin disease causedby the deficiency of COL7. FCX-007 is a genetically-modified autologous fibroblast that encodes the gene for COL7 for localized treatment of RDEB and isbeing developed in collaboration with Intrexon. By genetically modifying autologous fibroblasts ex vivo to produce COL7, culturing them and then treatingwounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to theaffected areas, thereby avoiding systemic treatment.FCX-007 is currently in a Phase 1/2 clinical trial. Four patients are enrolled in the Phase 1 portion of the clinical trial and continue to progressthrough follow-up study visits. We completed dosing of the fourth adult patient and performed additional dosing of existing adult patients in the Phase 1portion of the trial in the fourth quarter of 2017. We expect to report additional interim adult data and provide a trial update in the second quarter of 2018,which includes presenting at the 7th International Investigative Dermatology meeting in May 2018. We have designated our existing, cGMP cell therapymanufacturing facility in Exton, PA as the production site for FCX-007 in our IND application. FCX-007 drug product dosed in the fourth quarter of 2017was produced and distributed from our Exton, PA facility.Prior to treating pediatric patients in this trial, we were required to and obtained allowance from the FDA by submitting evidence of FCX-007 safetyand benefit in the adult patients and data from our completed pre-clinical toxicology study. After submission of the requested data, the FDA grantedallowance to include pediatric patients in the clinical trial in January 2018. We plan to enroll six patients ages seven and older in the Phase 2 portion of theclinical trial. One RDEB adult patient has been enrolled as the first patient in Phase 2 and dosing of this patient is expected to occur in the second quarter of2018. With allowance from the FDA, we will now include enrollment of pediatric patients. We expect to complete enrollment of Phase 2 patients in the thirdquarter of 2018. We plan to report another interim data readout and trial update in the first quarter of 2019. FCX-007 has received Orphan Drug Designation for the treatment of DEB, including RDEB, Rare Pediatric Disease Designation for the treatment ofRDEB and fast track designation for the treatment of RDEB from the FDA.In addition, our second gene therapy candidate, FCX-013 is in development for the treatment of moderate to severe localized scleroderma, whichmanifests as excess production of extracellular matrix, specifically collagen, resulting in thickening of the skin and connective tissue. FCX-013 is designedto be injected under the skin at the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce matrix metalloproteinase 1(MMP-1) to break down excess collagen accumulation. We previously completed a proof-of-concept study and pre-clinical dose-ranging study for FCX-013.In December 2017, we completed a GLP toxicology/biodistribution study. We submitted an IND application for FCX-013 to the53Table of ContentsFDA in January 2018, and in March 2018, the FDA allowed the IND to progress to clinical trials. We expect to initiate enrollment for an open label, singlearm Phase 1/2 clinical trial in the third quarter of 2018. We plan to manufacture FCX-013 at our Exton, PA cGMP manufacturing facility.FCX-013 has received Orphan Drug Designation from the FDA for the treatment of localized scleroderma and Rare Pediatric Disease Designation formoderate to severe localized scleroderma.Gene Therapy Research Program for Arthritis and Related ConditionsWe expanded our collaboration with Intrexon to pursue the research, development and commercialization of products for the treatment of chronicinflammation and degenerative diseases of human joints through intra-articular or other local administration of genetically modified fibroblasts. We arecurrently in the research phase for a gene therapy to treat arthritis and related conditions under this collaboration. Our goal is to deliver a protein therapylocally to the joint to provide sustained efficacy while avoiding key side effects typically associated with systemic therapy.See “Item 1—Business” within Part I of this Form 10-K for additional details regarding our development programs, research programs, andcollaboration agreements.54Table of ContentsCritical Accounting PoliciesThe following discussion and analysis of financial condition and results of operations are based upon our Consolidated Financial Statements, whichhave been prepared in conformity with U.S. generally accepted accounting principles (GAAP). Preparing financial statements requires us to make estimatesand assumptions that affect the reported amounts of assets, liabilities, revenues and expenses. Estimates are based on our historical operations, our futurebusiness plans and projected financial results, the terms of existing contracts, our observance of trends in the industry, information provided by our customersand information available from other outside sources, as appropriate. These estimates and assumptions are affected by the application of our accountingpolicies. Critical accounting policies and practices are both important to the portrayal of a company’s financial condition and results of operations, andrequire management’s most difficult, subjective or complex judgments, often as a result of the need to make estimates about the effects of matters that areinherently uncertain. Actual results could differ from such estimates due to changes in economic factors or other conditions that are outside the control ofmanagement. A summary of our significant accounting policies is more fully described in Note 3 of the Consolidated Financial Statements contained in thisForm 10-K.Warrant LiabilityThe Company accounts for stock warrants as either equity instruments, derivative liabilities, or liabilities in accordance with ASC 480,Distinguishing Liabilities from Equity (ASC 480), depending on the specific terms of the warrant agreement. Stock warrants are accounted for as a derivativein accordance with ASC 815, Derivatives and Hedging (ASC 815) if the stock warrants contain “down-round protection” or other terms that could potentiallyrequire “net cash settlement” and therefore, do not meet the scope exception for treatment as a derivative. Since “down-round protection” is not an input intothe calculation of the fair value of the warrants, the warrants cannot be considered indexed to the Company’s own stock which is a requirement for the scopeexception as outlined under ASC 815. Warrant instruments that could potentially require “net cash settlement” in the absence of express language precludingsuch settlement and those which include “down-round provisions” are initially classified as derivative liabilities at their estimated fair values, regardless ofthe likelihood that such instruments will ever be settled in cash. The Company will continue to classify the fair value of the warrants that contain “down-round protection” and “net cash settlement” as a liability until the warrants are exercised, expire or are amended in a way that would no longer require thesewarrants to be classified as a liability. Warrants that the Company may be required to redeem through payment of cash or other assets outside its control areclassified as liabilities pursuant to ASC 480 and are initially and subsequently measured at their estimated fair values. For additional discussion on warrants,see Note 8.Debt Issued With WarrantsThe Company considers guidance within ASC 470-20, Debt (ASC 470), ASC 480, and ASC 815 when accounting for the issuance of convertibledebt with detachable warrants. As described above under the caption “Warrant Liability”, the Company classifies stock warrants as either equity instruments,derivative liabilities, or liabilities depending on the specific terms of the warrant agreement. In circumstances in which debt is issued with liability-classifiedwarrants, the proceeds from the issuance of convertible debt are first allocated to the warrants at their full estimated fair value and established as both aliability and a debt discount. The remaining proceeds, as further reduced by discounts created by the bifurcation of embedded derivatives and beneficialconversion features, are allocated to the debt. The Company accounts for debt as liabilities measured at amortized cost and amortizes the resulting debtdiscount from the allocation of proceeds, to interest expense using the effective interest method over the expected term of the debt instrument pursuant toASC 835, Interest (ASC 835).Embedded Derivatives. The Company considers whether there are any embedded features in debt instruments that require bifurcation and separateaccounting as derivative financial instruments pursuant to ASC 815. Embedded derivatives are initially and subsequently measured at fair value. See Note 7for additional discussion on the embedded derivatives associated with the Company’s convertible notes.Beneficial Conversion Feature. If the amount allocated to the convertible debt results in an effective per share conversion price less than the fairvalue of the Company’s common stock on the commitment date, the intrinsic value of this beneficial conversion feature is recorded as a discount to theconvertible debt with a corresponding increase to additional paid in capital. The beneficial conversion feature discount is equal to the difference between theeffective conversion price and the fair value of the Company’s common stock at the commitment date, unless limited by the remaining proceeds allocated tothe debt. See Note 7 for additional discussion on the beneficial conversion feature associated with the Company’s convertible notes. 55Table of ContentsDebt Issuance Costs. The Company follows the guidance under Accounting Standards Update (ASU) 2015-03, Interest - Imputation of Interest(Subtopic 835-30): Simplifying the Presentation of Debt Issuance Costs (ASU 2015-03) for accounting for debt issuance costs. The Company allocates debtissuance costs between the debt and the warrants on the same basis as proceeds were allocated. The Company expenses issuance costs allocated to thewarrants and presents the issuance costs allocated to the debt as a direct reduction from the carrying amount of the debt liability in the balance sheet.However, if debt issuance costs exceed the carrying amount of the debt, issuance costs are recorded to additional paid-in capital as a reduction of thebeneficial conversion feature. As of December 31, 2017, the Company’s debt issuance costs are presented in additional paid-in capital as a reduction of thebeneficial conversion feature and are being amortized to interest expense (despite their classification in additional paid-in capital) using the effective interestrate method over the expected term of the debt pursuant to ASC 835.Results of OperationsComparison of Years Ending December 31, 2017 and 2016Revenue and Cost of RevenueRevenue and cost of revenue were comprised of the following: Year Ended December 31, 2017 vs 2016 Change ($ in thousands)2017 2016 $% Revenue from product sales$— $337 $(337)(100.0)%(1)Collaboration revenue— 18 (18)(100.0)%(1)Total revenue— 355 (355)(100.0)% Cost of product sales— 696 (696)(100.0)%(1)Cost of collaboration revenue— 1 (1)(100.0)%(1)Total cost of revenue— 697 (697)(100.0)% Gross loss$— $(342) 342(100.0)% (1)As a result of the wind-down of azficel-T operations in the second half of 2016, we have no products for sale. Therefore in the year ended December31, 2017 we had no revenues or related cost of revenue.Research and Development ExpensesFor each of our research and development programs, we incur both direct and indirect expenses. We track direct research and development expensesby program, which include third party costs such as contract research, consulting and pre-clinical development costs and clinical trial and manufacturingcosts. We do not allocate indirect research and development expenses, which may include regulatory, laboratory (equipment and supplies), personnel,facility, process development and other overhead costs (including depreciation and amortization), to specific programs, as these expenses are to be deployedacross all of our product candidates. We expect research and development costs to continue to be significant for the foreseeable future as a result of our pre-clinical studies and clinical trials, as well as our ongoing collaborations with Intrexon. 56Table of ContentsDirect research and development costs, by major program, and indirect research and development costs, by major component, were as follows: Year Ended December 31,2017 vs 2016 Change ($ in thousands)2017 2016 $% Direct costs: FCX-0074,350 3,216 1,13435.3 % (1)FCX-0133,117 1,534 1,583103.2 % (2)Other72 366 (294)(80.3)% (3)Total direct costs7,539 5,116 2,42347.4 % Indirect costs: Regulatory costs91 762 (671)(88.1)% (4)Intangible amortization— 231 (231)(100.0)% (5)Compensation and related expenses2,031 3,267 (1,236)(37.8)% (6)Process development7 1,014 (1,007)(99.3)% (7)Other indirect R&D costs2,564 1,734 83047.9 % (8)Total indirect costs4,693 7,008 (2,315)(33.0)% Total research and development expenses$12,232 $12,124 $1080.9 % (1)Costs for our FCX-007 program increased approximately $1.1 million, or 35.3%, for the year ended December 31, 2017 compared to 2016 dueprimarily to costs associated with the Phase 1 portion of our Phase 1/2 clinical trial for FCX-007 in adults which began in the second quarter of 2016and continued throughout 2017.Through December 31, 2017, we have incurred approximately $24.8 million in direct research and development costs related to this program, life-to-date, which include non-cash expenses of $6.9 million in stock issuance costs associated with the 2012 ECC with Intrexon. Other costs includeproduct and assay development, key opinion leader development, pre-clinical studies and manufacturing, the design of the Phase 1/2 clinical trialprotocol and recruiting subjects, clinical product manufacturing, statistical analyses, report generation and future clinical trial costs.(2)Costs for our FCX-013 program for the year ended December 31, 2017 increased approximately $1.6 million, or 103.2% compared to 2016 dueprimarily to costs associated with a pre-clinical dose-ranging study and a toxicology study.Through December 31, 2017, we have incurred approximately $13.9 million in direct research and development costs related to this program, life-to-date, which include non-cash expenses of $6.4 million in stock issuance costs with the 2012 ECC with Intrexon. Other costs include product andassay development and pre-clinical work, including execution of our proof-of concept study, a pre-clinical dose-ranging study and a toxicologystudy. Going forward, research and development investments for this program are expected to support ongoing product and assay development, pre-clinical study execution, key opinion leader development, National Institutes of Health Recombinant DNA Advisory Committee (NIH RAC)meeting preparation expenses, and the design and execution of clinical trials.(3)Costs for our other programs decreased approximately $0.3 million or 80.3%, for the year ended December 31, 2017 compared to 2016. The azficel-T for chronic dysphonia program was discontinued at June 30, 2016 and the costs recorded since then relate to specific close out activities of theprogram.(4)Regulatory costs decreased approximately $0.7 million, or 88.1%, for the year ended December 31, 2017 compared to 2016 due primarily to adecrease in costs incurred with the FDA for fees levied under the Prescription Drug User Fee Act (PDUFA). The decrease in fees resulted from ourdecision to wind-down azficel-T (including LAVIV), which began in the fourth quarter of 2016, exempted us from being assessed annual productregistration and establishment fees imposed under PDUFA, which resulted in substantial cost savings.(5)Intangible asset amortization decreased approximately $0.2 million, or 100.0%, for the year ended December 31, 2017 compared to 2016 due to theimpairment of our intangible assets during the second quarter of 2016 which resulted in no amortization expense during the second half of 2016 orall of 2017. See Note 3 in the accompanying Notes to the Consolidated Financial Statements contained in this Form 10-K for further details.(6)Compensation and related expenses decreased approximately $1.2 million, or 37.8%, for the year ended December 31, 2017 compared to 2016, dueprimarily to decreases in salaries, benefits and bonus expense resulting from the reduction in workforce associated with the wind-down of azficel-Toperations which occurred in June 2016.57Table of Contents(7)Process development costs decreased approximately $1.0 million, or 99.3%, for the year ended December 31, 2017 compared to 2016, as a resultprimarily of internal process development work being halted in June 2016 in connection with the wind-down of azficel-T operations and relatedrestructuring initiatives.(8)Other indirect R&D costs increased approximately $0.8 million, or 47.9%, for the year ended December 31, 2017 compared to 2016, due primarily tothe inclusion of costs for overhead items that were previously part of the cost of products sold computation.Selling, General and Administrative ExpensesSelling, general and administrative expenses were comprised of the following: Year Ended December 31, 2017 vs 2016 Change ($ in thousands)2017 2016 $% Compensation and related expenses$1,764 $4,695 (2,931)(62.4)% (1)Professional fees2,103 2,161 (58)(2.7)% (2)Facilities and related expenses and other2,882 2,917 (35)(1.2)% (3)Total selling, general and administrative expenses$6,749 $9,773 $(3,024)(30.9)% (1)Compensation and related expenses decreased approximately $2.9 million, or 62.4% for the year ended December 31, 2017 compared to 2016. Thedecrease is due primarily to reductions in employee count and their related expenses, all as part of a reduction in workforce at June 30, 2016, andmanagement reorganization and reduction later into 2016 and early 2017.(2)Professional fees decreased approximately $0.06 million, or 2.7%, for the year ended December 31, 2017 compared to 2016. This decrease isattributable primarily to lower levels of business activity.(3)Facilities and related expenses were approximately $2.9 million for both of the years ending December 31, 2017 and 2016.Intangible Asset Impairment ExpenseDuring the year ended December 31, 2016 we recorded a non-cash impairment charge of approximately $3.9 million to write off our intangibleassets in connection with our decision to wind-down azficel-T (including LAVIV). No such charges were incurred during 2017. See Note 3 in theaccompanying Notes to the Consolidated Financial Statements contained in this Form 10-K for further details.Restructuring CostsDuring the year ended December 31, 2016 we recorded restructuring costs totaling approximately $0.3 million. Restructuring costs were comprisedof employee severance and benefit related charges associated with our reduction in workforce in June 2016 and non-cash impairment charges against thecarrying values of equipment with no alternative future use. No such costs were incurred during 2017. See Part I, Item 1, "Business —Wind-down of azficel-TOperations" of this Form 10-K and Note 12 in the accompanying Notes to the Consolidated Financial Statements contained in Part IV of this Form 10-K forfurther details.Warrant Revaluation IncomeDuring the years ended December 31, 2017 and 2016, we recorded non-cash income of approximately $4.9 million and $11.9 million, respectively,for warrant revaluation income in our Consolidated Statements of Operations. Due to the nature and inputs of the model used to assess the fair value of ouroutstanding warrants, it is not abnormal to experience significant fluctuations from year to year. These fluctuations were due to a variety of factors includingchanges in our stock price, changes in the remaining contractual life of the warrants, and changes in management’s estimated probability of certain eventsoccurring that would impact the warrants. Warrant revaluation income for 2017 and 2016 was driven primarily by decreases in both our stock price and theremaining contractual term of the warrants.58Table of ContentsDerivative Revaluation ExpenseDuring the years ended December 31, 2017 and 2016, we recorded non-cash derivative revaluation expense of approximately $1.4 million and $0.5million, respectively, for derivative liability revaluation charges in our Consolidated Statement of Operations related to a compound bifurcated derivativeinitially recorded in September 2016 in connection with the 2016 Private Placement. Derivative valuation expense was primarily the result of the decrease inour stock price during the year ended December 31, 2017. See Note 7 in the accompanying Notes to the Consolidated Financial Statements contained in PartIV of this Form 10-K for further details.Interest ExpenseDuring the years ended December 31, 2017 and 2016, we recorded interest expense of approximately $0.8 million and $0.2 million, respectively, inour Consolidated Statement of Operations related to the Notes that we issued in the 2016 Private Placement which bear interest at 4% per annum. No suchexpenses were incurred in prior periods. See Note 7 in the accompanying Notes to the Consolidated Financial Statements contained in Part IV of this Form10-K for further details.Net LossNet loss increased approximately $0.8 million to $16.2 million for the year ended December 31, 2017, as compared to $15.3 million for the yearended December 31, 2016. The increase was due primarily to an overall net decrease in operating expenses of approximately $7.9 million, as more fullydescribed at the component level above, offset by a decrease in warrant revaluation income of approximately $7.0 million, increased derivative revaluationexpense of approximately $0.9 million and increased interest expense of approximately $0.6 million.59Table of ContentsFinancial Condition, Liquidity and Capital ResourcesFinancial ConditionWe have experienced losses since our inception. As of December 31, 2017, we had an accumulated deficit of approximately $178.8 million. Theprocess of developing and commercializing our product candidates requires significant research and development efforts and clinical trial work, as well assignificant manufacturing and process development. These activities, together with our selling, general and administrative expenses, are expected tocontinue to result in significant operating losses for the foreseeable future. Additionally, to fund our operations, we issued convertible promissory notes in anaggregate amount of approximately $18.1 million, which bear interest at 4% per annum, in connection with the 2016 Private Placement as more fullydescribed under the heading “Contractual Obligations” below and in Note 7 in the accompanying Notes to the Consolidated Financial Statements containedin Part IV of this Form 10-K.Our financial condition is summarized below as of the following dates: As of December 31, Change($ in thousands)2017 2016 $%Cash and cash equivalents$17,417 $17,515 $(98)(0.6)% Working capital: Total current assets$17,902 $18,028 $(126)(0.7)%Less: Total current liabilities4,425 2,987 1,43848.1 %Net working capital$13,477 $15,041 $(1,564)(10.4)% Convertible notes payable (gross principal)$18,003 $18,088 $(85)(0.5)%Liquidity and Capital ResourcesOur principal sources of liquidity are cash and cash equivalents of approximately $17.4 million as of December 31, 2017. As of December 31, 2017,we had net working capital of approximately $13.5 million which decreased approximately $1.6 million, or 10.4%, from December 31, 2016. We believe thatour existing cash and cash equivalents, will be sufficient to fund our operations into the first quarter of 2019; however, changing circumstances may cause usto consume capital faster than we currently anticipate, and we may need to spend more money than currently expected because of such circumstances. Wewill require additional capital to fund operations beyond that point and prior to our business achieving significant net cash from operations. Our futurecapital requirements may be substantial, and will depend on many factors, including, but not limited to:•the cost of clinical activities and outcomes related to our Phase 1/2 clinical trial for FCX-007;•the costs of clinical activities related to FCX-013, for which we received FDA allowance for our IND in the first quarter of 2018;•the cost of additional pre-clinical studies and clinical trials in order to obtain regulatory approvals for our product candidates;•the cost of regulatory submissions, as well as the preparation, initiation and execution of clinical trials in potential new clinical indications; and•the cost of filing, surveillance around, prosecuting, defending and enforcing patent claims. To meet our capital needs, we consider multiple alternatives, including but not limited to equity financings, debt financings, corporatecollaborations, partnerships and other strategic transactions and funding opportunities. However, there is no assurance that we will be able to complete anysuch transaction or obtain the additional required capital on acceptable terms or otherwise. Furthermore, the covenants under our convertible notes limit ourability to obtain additional debt financing. If we raise additional funds by issuing equity securities, our stockholders will experience dilution. Debtfinancing, if available, will result in increased fixed payment obligations and may involve agreements that include covenants limiting or restricting ourability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. Any debt or equity financing that wecomplete may contain terms, such as liquidation and other preferences, which are not favorable to us or our stockholders. If we raise additional funds throughcollaboration or partnership arrangements with third parties, it may60Table of Contentsbe necessary to relinquish valuable rights to our technologies, future revenue streams or product candidates or to grant licenses on terms that may not befavorable to us.If we are unable to raise additional capital in sufficient amounts or on terms acceptable to us, we will need to curtail and reduce our operations andcosts and modify our business strategy which may require us to, among other things:•significantly delay, scale back or discontinue the development or commercialization of one or more of our product candidates or one or more of ourother research and development initiatives;•seek collaborators for one or more of our current or future product candidates at an earlier stage than otherwise would be desirable or on terms thatare less favorable than might otherwise be available; or•sell or license on unfavorable terms our rights to technologies or product candidates that we otherwise would seek to develop or commercializeourselves.Additionally, failure to obtain the necessary capital in a timely manner could require us to seek bankruptcy protection or result in our breach ordefault under agreements on which our business relies or pursuant to which we obtain valuable rights which could result in, among other things, the potentialacceleration of payments thereunder or the termination of such agreements.These factors raise substantial doubt about our ability to continue as a going concern. Consequently, the audit report prepared by our independentregistered public accounting firm relating to our Consolidated Financial Statements for the year ended December 31, 2017 includes a paragraph related to thesubstantial doubt about our ability to continue as a going concern.Nasdaq Deficiency NoticeOn January 23, 2018, we received (the “Notice”) from Nasdaq that we are not in compliance with Nasdaq Listing Rule 5550(a)(2), as the minimumbid price of our common stock has been below $1.00 per share for 30 consecutive business days. The Notice has no immediate effect on the listing of ourcommon stock, which will continue to trade at this time on the Nasdaq Capital Market under the symbol “FCSC.”In accordance with Nasdaq Listing Rule 5810(c)(3)(A), we have a period of 180 calendar days, or until July 23, 2018, to regain compliance with theminimum bid price requirement. To regain compliance, the closing bid price of our common stock must meet or exceed $1.00 per share for at least tenconsecutive business days during this 180 calendar day period. In the event we do not regain compliance by July 23, 2018, we may be eligible for anadditional 180 calendar day grace period if we meet the continued listing requirement for market value of publicly held shares ($1 million) and all otherinitial listing standards which require, among other things, that we have at least $5 million of stockholders’ equity or at least $4 million of stockholders’equity and $50 million market value of listed shares. If we fail to regain compliance during the applicable period, we will receive notification from Nasdaqthat our common stock is subject to delisting. At that time we may then appeal the delisting determination to a Hearings Panel. Such notification will have noimmediate effect on our listing on the Nasdaq Capital Market, nor will it have an immediate effect on the trading of our common stock pending such hearing.There can be no assurance, however, that we will be able to regain compliance with Nasdaq’s minimum bid price requirement. If we regain compliance withthe Nasdaq’s minimum bid price requirement, there can be no assurance that we will be able to maintain compliance with the continued listing requirementsfor the Nasdaq Capital Market, or that our common stock will not be delisted from the Nasdaq Capital Market in the future. In addition, we may be unable tomeet other applicable listing requirements of the Nasdaq Capital Market, including maintaining minimum levels of stockholders’ equity or market values ofour common stock in which case, our common stock could be delisted notwithstanding our ability to demonstrate compliance with the minimum bid pricerequirement.We intend to monitor the closing bid price of our common stock and consider options to resolve our noncompliance with the minimum bid pricerequirement.Nasdaq Equity RequirementNasdaq has the authority, pursuant to Nasdaq Listing Rule 5550(b)(1), to delist our common stock if our stockholders’ equity falls below $2.5million. As of December 31, 2017, our stockholders’ equity was $9.0 million. If our stockholders equity is hereafter reduced below $2.5 million as a result ofoperating losses or for other reasons, we will fail to meet Nasdaq’s stockholders’ equity requirement. If that occurs, or if we are unable to demonstrate toNasdaq’s satisfaction that we will be able to sustain compliance with this requirement, Nasdaq may delist our common stock. In addition, even if we regaintechnical compliance with the stockholders’ equity requirement, we will have to continue to meet other objective and subjective listing61Table of Contentsrequirements to continue to be listed on the Nasdaq Capital Market, including the requirement that our common stock continues to trade above $1.00. Forthe year ended December 31, 2017, we incurred a net loss of $16.2 million and used $17.0 million in operating activities and had an accumulated deficit of$178.8 as of December 31, 2017.We are actively monitoring our stockholders’ equity and will consider any and all options available to us to maintain compliance. There can be noassurance, however, that we will be able to maintain compliance and meet Nasdaq’s minimum stockholders’ equity requirements. 2017 Series A Preferred Stock OfferingOn March 8, 2017, we completed the Series A Convertible Preferred Stock Offering for the sale of $8.0 million of our Series A Preferred Stock andthe March 2017 Warrants to certain of our existing investors, including certain related parties (including Intrexon). After deducting offering expenses, netproceeds from the offering excluding the proceeds, if any, from the exercise of the March 2017 Warrants, was approximately $7.6 million.2017 Common Stock and Warrant OfferingOn December 11, 2017, we completed the December 2017 Offering for the sale of $10.5 million of shares of our common stock, detachable warrantsto the general public and the December 2017 Underwriter Warrants. After deducting offering expenses, net proceeds from the December 2017 Offeringexcluding the proceeds, if any, from the exercise of the warrants, was approximately $9.3 million. Also, see Risks Related to Our Financial Position and Need for Additional Capital included within Part I, Item 1A, “Risk Factors” of this Form 10-K.Cash FlowsThe following table summarizes our cash flow activity: Year Ended December 31, 2017 vs 2016 Change($ in thousands)2017 2016 $%Net cash flows provided by (used in): Operating activities$(17,037) $(29,390) $12,353(42.0)%Investing activities$(433) $(252) $(181)71.8 %Financing activities$17,372 $17,889 $(517)(2.9)%Operating Activities. Cash used in operating activities during the year ended December 31, 2017 was approximately $17.0 million, a decrease ofapproximately $12.4 million over the year ended December 31, 2016. This decrease was due primarily to the $10 million up-front technology access feepayment to Intrexon in January 2016 in connection with the 2015 ECC, for which there was no comparable payment in 2017, as well as decreased operatingcosts related to the reduction in workforce at June 30, 2016.Investing Activities. Cash used in investing activities during the year ended December 31, 2017, increased by approximately $0.2 million over the yearended December 31, 2016. The amount in both periods related primarily to the purchases of equipment and leasehold improvements. Financing Activities. Cash provided by financing activities during the year ended December 31, 2017 was approximately $17.4 million, a decrease ofapproximately $0.5 million as compared to the year ended December 31, 2016. The decrease was due primarily to net proceeds from the two offerings in2017, raising approximately $16.9 million, as compared to the 2016 Private Placement of approximately $17.9 million. See additional information regardingthe two 2017 offerings in Note 9, Equity, See additional information regarding the 2016 Private Placement included under the heading “ContractualObligations” below and in Note 7 in the accompanying Notes to the Consolidated Financial Statements contained in Part IV of this Form 10-K. Off-Balance Sheet TransactionsWe do not engage in material off-balance sheet transactions.62Table of ContentsContractual ObligationsThe following table summarizes our contractual obligations and commercial commitments as of December 31, 2017 and the effects such obligationsare expected to have on our liquidity and cash flows in future periods: Payments due by period($ in thousands)Total 2018 2019 2020 2021 2022 2023 andthereafterOperating lease obligations (1)$7,451 $1,254 $1,416 $1,471 $1,471 $1,471 $368Debt obligations (2)21,968 — — — 21,968 — —Total (3)$29,419 $1,254 $1,416 $1,471 $23,439 $1,471 $368(1)Operating lease obligations are stated based on the amended lease agreement for our office, warehouse and laboratory facility executed in February 2012.(2)Obligations under the Notes issued in connection with the 2016 Private Placement which includes principal and accrued interest through September 7, 2021, based onstated fixed rates, as we have elected to accrue interest. The Notes have a maturity date of the earlier of (i) September 7, 2026 and (ii) one-hundred and eighty (180) daysafter the date on which our product candidate, FCX-007, is approved by the FDA for the treatment of RDEB. However, each Note holder has the right to require us torepay all or any portion of the unpaid principal and accrued interest from time to time on or after September 7, 2021. See details under the sub-heading “2016 PrivatePlacement” below.(3)This table does not include (a) any milestone payments which may become payable to third parties under license agreements as the timing and likelihood of such paymentsare not known, (b) any royalty payments to third parties as the amounts of such payments, timing and/or the likelihood of such payments are not known, and (c) contractsthat are entered into in the ordinary course of business which are not material in the aggregate in any period presented above.2016 Private PlacementIn September 2016, we issued an aggregate of approximately $18.1 million in principal of Notes and accompanying Private Placement Warrants topurchase an aggregate of 6,029,174 shares of common stock in a private placement to the Investors, including certain related parties (including Intrexon)which were issued an aggregate of approximately $6.8 million in principal of Notes and accompanying Private Placement Warrants to purchase an aggregateof 2,254,168 shares of our common stock.The Notes bear interest at four percent (4%) per annum. Interest is earned daily and compounded quarterly and, at our election at the beginning ofeach quarter, shall accrue or be paid in cash. If we elect to have interest accrue, such interest will not be added to the principal amount of the Notes but suchinterest shall be subject to additional interest at the rate of four percent (4%) per annum, compounded quarterly, and shall be due and payable upon theearliest of the conversion of the Notes, exercise of the Put Right, exercise of the Prepayment Right or the Maturity Date (in each case, as defined below).Additionally, if we elect for interest to accrue, then (i) we may elect to repay any such accrued and unpaid interest in cash at any time and from time to timeand (ii) each Investor may elect to have us repay any such accrued and unpaid interest by delivering such number of shares of common stock equal to (x) theamount of the accrued and unpaid interest to be repaid, divided by (y) the greater of (i) the last closing bid price of a share of common stock as reported onNasdaq on the date of such election and (ii) the applicable Conversion Price.All unpaid principal of each Investor’s Note is convertible, at any time and from time to time, at the option of such Investor into shares of commonstock at the conversion price, ($3.40875,to $3.52875) and accrued interest is convertible at the greater of (x) the conversion price or (y) the last closing bidprice of a share of common stock as reported on the Nasdaq Capital Market at the time of such Investor’s execution of the Purchase Agreement, plus$0.12625.The Notes have a maturity date of the earlier of (i) September 7, 2026 and (ii) one-hundred and eighty (180) days after the date on which our productcandidate, FCX-007, is approved by the FDA for the treatment of RDEB. Each individual Note holder has the right to require us to repay all or any portion ofthe unpaid principal and accrued and unpaid interest from time to time on or after September 7, 2021. Such Put Right must be exercised by such Note holderby delivering written notice to us no later than one-hundred and eighty (180) days prior to such exercise date of such Put Right. In addition, uponconsummation of a specified change of control transaction or the occurrence of certain events of default, as defined in the Notes, each Note holder may electto accelerate the repayment of all unpaid principal and accrued interest under such holder’s Note. If an Investor does not elect to have us prepay its Note uponsuch change of control transaction, then we may prepay the Notes, in an amount equal to one hundred one percent (101%) of the outstanding principal dueunder the Notes (together with accrued and unpaid interest due thereon) (the Prepayment Right). Additionally, upon the occurrence of certain events ofdefault, as defined63Table of Contentsin the Notes, each Investor may elect to accelerate the repayment of all unpaid principal and accrued interest under each Note and the Notes provide forautomatic redemption upon the occurrence of certain bankruptcy related events of default, as defined in the Notes.Collaborations with Related PartyWe are party to two separate exclusive channel collaboration agreements with Intrexon, a related party, pursuant to which we became Intrexon’sexclusive channel collaborator in the research, development and commercialization of certain products as defined in the respective agreements. Inconnection with these exclusive channel collaboration agreements, we engage Intrexon for support services for the research and development of productcandidates covered under the respective agreements and reimburses Intrexon for its cost for time and materials for such services. For the years ended December 31, 2017 and 2016, we incurred expenses of $5.7 million and $3.7 million, respectively, for payments to Intrexon. Asof December 31, 2017 and 2016, we had outstanding payables with Intrexon of $2.3 million and $0.9 million, respectively. For additional details, see information within Part I, Item 1—Business, under the heading “Intrexon Collaborations” and Note 14, Related PartyTransactions, to the Consolidated Financial Statements, included in Part IV of this Form 10-K.Recently Issued Accounting PronouncementsSee Note 3, Summary of Significant Accounting Policies, in the Notes to the Consolidated Financial Statements included in Part IV of this Form 10-K for discussion on recently issued accounting pronouncements.Item 7A. Quantitative and Qualitative Disclosure about Market Risk Not applicable.Item 8. Financial Statements and Supplementary DataThe information required by Item 8 including the financial statements and notes thereto, and report of the independent registered public accountingfirm thereon, are included in this Form 10-K as set forth in the “Index to Consolidated Financial Statements” on page F-1.Item 9. Changes in and Disagreements with Accountants on Accounting and Financial Disclosure.Not applicable.Item 9A. Controls and ProceduresEvaluation of Disclosure Controls and ProceduresOur management, including our principal executive officer and principal financial officer, has evaluated the effectiveness of the design andoperation of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (theExchange Act)), as of the end of the period covered by this Form 10-K. Based upon that evaluation, our Chief Executive Officer (our principal executiveofficer and principal financial officer), concluded that, as of December 31, 2017, our disclosure controls and procedures were effective to provide reasonableassurance that (a) the information required to be disclosed by us in the reports that we file or submit under the Exchange Act is recorded, processed,summarized and reported within the time periods specified in the SEC’s rules and forms, and (b) such information is accumulated and communicated to ourmanagement, including our Chief Executive Officer (our principal executive officer and principal financial officer), as appropriate to allow timely decisionsregarding required disclosure. In designing and evaluating our disclosure controls and procedures, our management recognized that any controls and procedures, no matter howwell designed and operated, can provide only reasonable assurance of achieving the desired control64Table of Contentsobjectives, and our management necessarily was required to apply its judgment in evaluating the cost-benefit relationship of possible controls andprocedures.Management’s Report on Internal Control over Financial ReportingManagement is responsible for establishing and maintaining adequate internal control over financial reporting, as such term is defined in ExchangeAct Rules 13a-15(f) and 15d-15(f). Under the supervision and with the participation of our management, including our principal executive officer, who alsoserves as our principal financial officer, we conducted an evaluation of the effectiveness of our internal control over financial reporting, based on theframework in the Internal Control - Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission (COSO2013).Our internal control over financial reporting is a process designed to provide reasonable assurance regarding the reliability of financial reporting andthe preparation of financial statements for external purposes in accordance with generally accepted accounting principles in the United States of America.Our internal control over financial reporting includes those policies and procedures that (i) pertain to the maintenance of records that, in reasonable detail,accurately and fairly reflect the transactions and dispositions of our assets; (ii) provide reasonable assurance that transactions are recorded as necessary topermit preparation of financial statements in accordance with generally accepted accounting principles, and our receipts and expenditures are being madeonly in accordance with authorizations of our management and directors; and (iii) provide reasonable assurance regarding prevention or timely detection ofunauthorized acquisition, use, or disposition of our assets that could have a material effect on the financial statements.Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements. Also, projections of anyevaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degreeof compliance with the policies or procedures may deteriorate.Based on our evaluation under the framework in COSO 2013, our management concluded that our internal control over financial reporting waseffective as of December 31, 2017.This Form 10-K does not include an attestation report of our independent registered public accounting firm regarding internal control over financialreporting. Management’s report was not subject to the attestation by our independent registered public accounting firm because smaller reporting companiesare exempt from this requirement.Changes in Internal Control over Financial ReportingThere were no changes in our internal control over financial reporting during the quarter ended December 31, 2017 that have materially affected, orare reasonably likely to materially affect, our internal control over financial reporting.Item 9B. Other Information None. 65Table of ContentsPart IIIItem 10. Directors, Executive Officers and Corporate GovernanceThe information required by this item is incorporated by reference to our Proxy Statement for the 2018 Annual Meeting of Stockholders to be filedwith the Securities and Exchange Commission within 120 days of the fiscal year ended December 31, 2017.Our Board has adopted a written Code of Business Conduct and Ethics applicable to all officers, directors and employees, which is available on ourwebsite (www.fibrocell.com) under “Corporate Governance” within the “Investors” section. We intend to satisfy the disclosure requirement under Item 5.05of Form 8-K regarding any amendment to, or waiver from, a provision of this Code and by posting such information on the website address and locationspecified above.Item 11. Executive CompensationThe information required by this item is incorporated by reference to our Proxy Statement for the 2018 Annual Meeting of Stockholders to be filedwith the Securities and Exchange Commission within 120 days of the fiscal year ended December 31, 2017.Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters The information required by this item is incorporated by reference to our Proxy Statement for the 2018 Annual Meeting of Stockholders to be filedwith the Securities and Exchange Commission within 120 days of the fiscal year ended December 31, 2017. Item 13. Certain Relationships and Related Transactions, and Director IndependenceThe information required by this item is incorporated by reference to our Proxy Statement for the 2018 Annual Meeting of Stockholders to be filedwith the Securities and Exchange Commission within 120 days of the fiscal year ended December 31, 2017.Item 14. Principal Accountant Fees and ServicesThe information required by this item is incorporated by reference to our Proxy Statement for the 2018 Annual Meeting of Stockholders to be filedwith the Securities and Exchange Commission within 120 days of the fiscal year ended December 31, 2017.66Table of ContentsPart IV Item 15. Exhibits and Financial Statement Schedule(a) (1) Consolidated Financial Statements.The Consolidated Financial Statements are filed as part of this report. See the Index to the Consolidated Financial Statements on page F-1.(2) Consolidated Financial Statement Schedule.Schedules are omitted because they are not applicable, or are not required, or because the information is included in the Consolidated FinancialStatements and Notes thereto.(3) The exhibits listed under Item 15(b), which are incorporated herein by reference, are filed or furnished as part of this report or are incorporated intothis report by reference.(b) Exhibits.EXHIBITNO. IDENTIFICATION OF EXHIBIT2.1 Debtors’ First Amended Joint Plan of Reorganization dated July 30, 2009 and Disclosure Statement (incorporated by reference to asExhibit 10.2 to our Quarterly Report on Form 10-Q for the quarter ended June 30, 2009, filed on August 12, 2009 and as Exhibit 99.1to our Form 8-K, filed September 2, 2009)3.1 Restated Certificate of Incorporation (incorporated by reference to Exhibit 3.1 to our Current Report on Form 8-K, filed December 13,2012)3.2 Certificate of Amendment of the Restated Certificate of Incorporation filed April 26, 2013 (incorporated by reference to Exhibit 3.1 toour Current Report on Form 8-K, filed April 29, 2013)3.3 Certificate of Amendment to the Company’s Restated Certificate of Incorporation, as amended, filed July 19, 2013 (incorporated byreference to Exhibit 3.1 to our Current Report on Form 8-K filed July 22, 2013)3.4 Certificate of Amendment of the Restated Certificate of Incorporation filed July 12, 2016 (incorporated by reference to Exhibit 3.1 toour Quarterly Report on Form 10-Q for the quarter ended June 30, 2016, filed August 4, 2016)3.5 Certificate of Amendment of the Restated Certificate of Incorporation of Fibrocell Science, Inc., as amended, dated March 10, 2017(incorporated by reference to Exhibit 3.1 to our Current Report on Form 8-K, filed on March 10, 2017)3.6 Certificate of Designation of Preferences, Rights and Limitations of Series A Convertible Preferred Stock) incorporated by reference toExhibit 3.1 to our Current Report on Form 8-K, filed on March 8, 2017)3.7 Fourth Amended and Restated Bylaws (incorporated by reference to Exhibit 3.1 to our Quarterly Report on Form 10-Q for the quarterended March 31, 2015, filed on May 8, 2015)3.8 Amendment to Fourth Amended and Restated Bylaws (incorporated by reference to Exhibit 3.2 to our Quarterly Report on Form 10-Qfor the quarter ended March 31, 2015, filed May 8, 2015)4.1 Form of Common Stock Certificate (incorporated by reference to Exhibit 4.1 to our Quarterly Report on Form 10-Q for the quarterended September 30, 2009, filed November 23, 2009)4.2 Form of Common Stock Purchase Warrant used for the Series E Preferred Stock offering (incorporated by reference to Exhibit 4.1 toour Current Report on Form 8-K, filed May 14, 2012)4.3 Form of Amended and Restated Common Stock Purchase Warrant issued to our prior 12.5% Note holders (incorporated by reference toExhibit 10.5 to our Current Report on Form 8-K, filed October 9, 2012)4.4 Form of Convertible Promissory Note (incorporated by reference to Exhibit 4.1 to our Current Report on Form 8-K, filed September 8,2016)4.5 Form of Common Stock Purchase Warrant (incorporated by reference to Exhibit 4.2 to our Current Report on Form 8-K, filedSeptember 8, 2016)4.6 Form of Common Stock Purchase Warrant (incorporated by reference to Exhibit 4.1 to our Current Report on Form 8-K, filed on March8, 2017)4.7 Form of Common Stock Purchase Warrant (incorporated by reference to Exhibit 4.2 to our Current Report on Form 8-K, filedDecember 11, 2017)4.8 Form of Pre-Funded Common Stock Purchase Warrant (incorporated by reference to Exhibit 4.3 to our Current Report on Form 8-K,filed December 11, 2017)67Table of Contents4.9 Form of Underwriter’s Common Stock Purchase Warrant issued in December 2017 offering (incorporated by reference to Exhibit 4.1 toour Current Report on Form 8-K, filed December 11, 2017)10.1 Lease Agreement between Isolagen, Inc. and The Hankin Group dated April 7, 2005 (incorporated by reference to Exhibit 10.1 to ourCurrent Report on Form 8-K, filed April 12, 2005)10.2 Amendment to Lease Agreement between Fibrocell Science, Inc. and The Hankin Group dated February 17, 2012 (incorporated byreference to Exhibit 10.17 to our Annual Report on Form 10-K for the fiscal year ended December 31, 2011, filed March 30, 2012)10.3 Securities Purchase Agreement dated October 5, 2012 (incorporated by reference to Exhibit 10.1 to our Current Report on Form 8-K,filed October 9, 2012)10.4 Registration Rights Agreement dated October 5, 2012 (incorporated by reference to Exhibit 10.2 to our Current Report on Form 8-K,filed October 9, 2012)10.5 Stock Issuance Agreement dated October 5, 2012 between the Company and Intrexon Corporation (incorporated by reference toExhibit 10.3 to our Current Report on Form 8-K, filed October 9, 2012)10.6 Amendment and Conversion Agreement dated October 5, 2012 between the Company and the Holders of the Company’s Notes(incorporated by reference to Exhibit 10.4 to our Current Report on Form 8-K, filed October 9, 2012)10.7 Exclusive Channel Collaboration Agreement between Intrexon Corporation and Fibrocell Science, Inc. (incorporated by reference toExhibit 10.21 to our Annual Report on Form 10-K for the fiscal year ended December 31, 2012, filed April 1, 2013)10.8 First Amendment to Exclusive Channel Collaboration Agreement between the Company and Intrexon Corporation dated June 28,2013 (incorporated by reference to Exhibit 10.1 to our Current Report on Form 8-K, filed July 1, 2013)10.9 Supplemental Stock Issuance Agreement between the Company and Intrexon Corporation dated June 28, 2013 (incorporated byreference to Exhibit 10.2 to our Current Report on Form 8-K, filed July 1, 2013)10.10 Second Amendment to Exclusive Channel Collaboration Agreement between the Company and Intrexon Corporation datedJanuary 10, 2014 (incorporated by reference to Exhibit 10.1 to our Current Report on Form 8-K, filed January 13, 2014)10.11 Supplemental Stock Issuance Agreement between the Company and Intrexon Corporation dated January 10, 2014 (incorporated byreference to Exhibit 10.2 to our Current Report on Form 8-K, filed January 13, 2014)10.12 Letter Agreement to Exclusive Channel Collaboration Agreement, as amended, between Fibrocell Science, Inc. and IntrexonCorporation dated September 29, 2015 (incorporated by reference to Exhibit 10.2 to our Quarterly Report on Form 10-Q for thequarter ended September 30, 2015, filed November 5, 2015)10.13tExclusive Channel Collaboration Agreement, dated December 31, 2015, between Fibrocell Science, Inc. and Intrexon Corporation(incorporated by reference to Exhibit 99.1 to our Current Report on Form 8-K, filed January 4, 2016)10.14UFibrocell Science, Inc. 2009 Equity Incentive Plan, as amended and restated as of March 11, 2017 (incorporated by reference toExhibit 10.1 to our Current Report on Form 8-K, filed June 20, 2014)10.15 Form of Nonqualified Stock Option Agreement for Employee Grants under Fibrocell Science, Inc. 2009 Equity Incentive Plan(incorporated by reference to Exhibit 10.2 to our Quarterly Report on Form 10-Q for the quarter ended March 31, 2015, filed May 8,2015)10.16 Form of Nonqualified Stock Option Agreement for Director Grants under Fibrocell Science, Inc. 2009 Equity Incentive Plan(incorporated by reference to Exhibit 10.3 to our Quarterly Report on Form 10-Q for the quarter ended March 31, 2015, filed May 8,2015)10.17 Form of Incentive Stock Option Agreement for Employee Grants under Fibrocell Science, Inc. 2009 Equity Incentive Plan(incorporated by reference to Exhibit 10.4 to our Quarterly Report on Form 10-Q for the quarter ended March 31, 2015, filed May 8,2015)10.18UEmployment Agreement between the Company and Michael F. Marino dated June 1, 2015 (incorporated by reference to Exhibit 10.1to our Form 10-Q for the quarter ended June 30, 2015, filed August 7, 2015)10.19UEmployment Agreement between the Company and John Maslowski dated September 14, 2015 (incorporated by reference toExhibit 10.1 to our Current Report on Form 8-K, filed September 16, 2015)10.20UOffer Letter by and between the Company and John M. Maslowski dated December 18, 2016 (incorporated by reference to Exhibit10.2 to our Current Report on Form 8-K, filed December 19, 2016)10.21USeparation Agreement and General Release by and between the Company and Michael F. Marino dated January 25, 2017(incorporated by reference to Exhibit 10.1 to our Current Report on Form 8-K, filed January 26, 2017)10.22USeparation Agreement and General Release by and between the Company and Kimberly M. Smith dated March 3, 2017 (incorporatedby reference to Exhibit 10.1 to our Current Report on Form 8-K, filed March 3, 2017)10.23 Agreement for the Purchase and Sale of Convertible Debt and Common Stock Warrants dated August 9, 2016 (incorporated byreference to Exhibit 10.1 to our Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, filed on November 3, 2016)68Table of Contents10.24 Form of Registration Rights Agreement (incorporated by reference to Exhibit 10.1 to our Current Report on Form 8-K, filed onSeptember 8, 2016)10.25 Controlled Equity Offering Sales Agreement by and between the Company and Cantor Fitzgerald & Co. dated January 21, 2016(incorporated by reference to Exhibit 10.1 of our Current Report on Form 8-K, filed January 21, 2016)10.26 Form of Securities Purchase Agreement by and between the Company and other signatories thereto dated March 7, 2017 (incorporatedby reference to Exhibit 10.1 to our Current Report on Form 8-K, filed on March 8, 201710.27 Engagement Letter, dated May 8, 2017, by and between the Company and AC Lordi (incorporated by reference to Exhibit 10.1 to ourCurrent Report on Form 8-K, filed May 9, 2017) *21 List of Subsidiaries*23 Consent of PricewaterhouseCoopers LLP*31 Certification of Principal Executive Officer and Principal Financial Officer pursuant to Rule 13a-14(a) and 15d-14(a), required underSection 302 of the Sarbanes-Oxley Act of 2002*32 Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350 as adopted pursuant toSection 906 of the Sarbanes-Oxley Act of 2002101.INS XBRL Instance Document101.SCH XBRL Taxonomy Extension Schema Document101.CAL XBRL Taxonomy Extension Calculation Linkbase Document101.LAB XBRL Taxonomy Extension Label Linkbase Document101.PRE XBRL Taxonomy Extension Presentation Linkbase Document101.DEF XBRL Taxonomy Extension Definition Linkbase Document*Filed herewith.UIndicates management contract or compensatory plan or arrangement.tConfidential treatment has been granted as to certain portions of this exhibit pursuant to Rule 406 of the Securities Act of 1933, as amended, orRule 24b-2 of the Securities Exchange Act of 1934, as amended.Item 16. Form 10-K SummaryNot applicable.69Table of ContentsSIGNATURESPursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signedon its behalf by the undersigned, thereunto duly authorized.FIBROCELL SCIENCE, INC. By:/s/ John M. MaslowskiJohn M. MaslowskiPresident and Chief Executive Officer Date: March 19, 2018Pursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed below by the following persons on behalf of theRegistrant and in the capacities and on the date indicated.SignatureTitleDate /s/ John M. MaslowskiPresident and Chief Executive OfficerMarch 19, 2018John M. Maslowski(Principal Executive Officer, Principal Financial Officer and PrincipalAccounting Officer) /s/ Douglas J. SwirskyChairman of the BoardMarch 19, 2018Douglas J. Swirsky /s/ Kelvin MooreDirectorMarch 19, 2018Kelvin Moore /s/ Marc MazurDirectorMarch 19, 2018Marc Mazur /s/ Julian KirkDirectorMarch 19, 2018Julian Kirk /s/ Marcus SmithDirectorMarch 19, 2018Marcus Smith /s/ Christine St.ClareDirectorMarch 19, 2018Christine St.Clare70Table of ContentsFibrocell Science, Inc.Index to Consolidated Financial Statements PAGE Report of Independent Registered Public Accounting FirmF 2 Consolidated Balance Sheets as of December 31, 2017 and 2016F 3 Consolidated Statements of Operations for the years ended December 31, 2017 and 2016F 4 Consolidated Statements of Stockholders’ Equity for the years ended December 31, 2017 and 2016F 5 Consolidated Statements of Cash Flows for the years ended December 31, 2017 and 2016F 6 Notes to Consolidated Financial StatementsF 7F 1Table of ContentsReport of Independent Registered Public Accounting FirmTo the Board of Directors and Stockholders of Fibrocell Science, Inc.Opinion on the Financial StatementsWe have audited the accompanying consolidated balance sheets of Fibrocell Science, Inc. and its subsidiaries as of December 31, 2017 and 2016, and therelated consolidated statements of operations, stockholders’ equity and cash flows for the years then ended, including the related notes (collectively referredto as the “consolidated financial statements”). In our opinion, the consolidated financial statements present fairly, in all material respects, the financialposition of the Company as of December 31, 2017 and 2016, and the results of its operations and its cash flows for the years then ended in conformity withaccounting principles generally accepted in the United States of America.Substantial Doubt About the Company’s Ability to Continue as a Going ConcernThe accompanying consolidated financial statements have been prepared assuming that the Company will continue as a going concern. As discussed in Note1 to the consolidated financial statements, the Company has suffered recurring losses from operations, has an accumulated deficit and cash outflows fromoperating activities that raise substantial doubt about its ability to continue as a going concern. Management's plans in regard to these matters are alsodescribed in Note 1. The financial statements do not include any adjustments that might result from the outcome of this uncertainty.Basis for OpinionThese consolidated financial statements are the responsibility of the Company’s management. Our responsibility is to express an opinion on the Company’sconsolidated financial statements based on our audits. We are a public accounting firm registered with the Public Company Accounting Oversight Board(United States) (“PCAOB”) and are required to be independent with respect to the Company in accordance with the U.S. federal securities laws and theapplicable rules and regulations of the Securities and Exchange Commission and the PCAOB.We conducted our audits of these consolidated financial statements in accordance with the standards of the PCAOB. Those standards require that we plan andperform the audit to obtain reasonable assurance about whether the consolidated financial statements are free of material misstatement, whether due to error orfraud. The Company is not required to have, nor were we engaged to perform, an audit of its internal control over financial reporting. As part of our audits weare required to obtain an understanding of internal control over financial reporting but not for the purpose of expressing an opinion on the effectiveness ofthe Company's internal control over financial reporting. Accordingly, we express no such opinion.Our audits included performing procedures to assess the risks of material misstatement of the consolidated financial statements, whether due to error or fraud,and performing procedures that respond to those risks. Such procedures included examining, on a test basis, evidence regarding the amounts and disclosuresin the consolidated financial statements. Our audits also included evaluating the accounting principles used and significant estimates made by management,as well as evaluating the overall presentation of the consolidated financial statements. We believe that our audits provide a reasonable basis for our opinion./s/PricewaterhouseCoopers LLPPhiladelphia, PennsylvaniaMarch 19, 2018We have served as the Company's auditor since 2015. F 2Table of ContentsFibrocell Science, Inc.Consolidated Balance Sheets($ in thousands, except share data) As of December 31, 2017 2016Assets Current assets: Cash and cash equivalents$17,417 $17,515Prepaid expenses and other current assets485 513Total current assets17,902 18,028Property and equipment, net of accumulated depreciation of $1,919 and $1,561, respectively1,470 1,489Other assets39 65Total assets$19,411 $19,582 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable$862 $440Related party payable2,303 942Accrued expenses1,260 1,551Warrant liability, current— 54Total current liabilities4,425 2,987Convertible promissory notes, net of debt discount of $18,003 and $18,088, respectively (see Note 7)— —Accrued interest payable967 228Warrant liability, long term1,073 5,980Derivative liability3,136 1,735Deferred rent803 791Total liabilities10,404 11,721 Commitments and contingencies (Note 16) Stockholders’ equity: Preferred stock, $0.001 par value; 5,000,000 shares authorized; 8,000 shares issued and outstanding as of December 31, 2017;5,000,000 shares authorized, no shares issued and outstanding as of December 31, 2016; aggregate liquidation preference of$8,264 at December 31, 2017— —Common stock, $0.001 par value; 150,000,000 shares authorized, 25,940,247 shares issued andoutstanding as of December 31, 2017; 150,000,000 shares authorized, 14,688,135 shares issuedand outstanding as of December 31, 201626 15Additional paid-in capital187,784 170,409Accumulated deficit(178,803) (162,563)Total stockholders’ equity9,007 7,861Total liabilities and stockholders’ equity$19,411 $19,582 The accompanying notes are an integral part of these consolidated financial statements.F 3Table of ContentsFibrocell Science, Inc.Consolidated Statements of Operations($ in thousands, except share and per share data) Year Ended December 31, 2017 2016Revenue from product sales$— $337Collaboration revenue— 18Total revenue— 355Cost of product sales— 696Cost of collaboration revenue— 1Total cost of revenue— 697Gross loss— (342)Research and development expenses6,512 8,400Research and development expenses - related party5,720 3,724Selling, general and administrative expenses6,749 9,773Intangible asset impairment expense— 3,905Restructuring costs— 335Operating loss(18,981) (26,479)Other income (expense): Warrant revaluation income4,920 11,884Derivative revaluation expense(1,407) (462)Interest expense(828) (228)Other income (expense), net56 (7)Loss before income taxes(16,240) (15,292)Income tax benefit— —Net loss(16,240) $(15,292) Dividend paid in-kind to preferred stockholders(264) — Deemed dividend on preferred stock (see Note 9)(4,099) — Net loss attributable to common stockholders$(20,603) $(15,292) Per Share Information: Net loss — Basic$(1.33) $(1.04)— Diluted$(1.33) $(1.18) Weighted average number of common shares outstanding — Basic15,454,199 14,641,528— Diluted15,460,118 14,647,534The accompanying notes are an integral part of these consolidated financial statements. F 4Table of ContentsFibrocell Science, Inc.Consolidated Statements of Stockholders’ Equity($ in thousands, except share data) Preferred Stock Common Stock Additionalpaid-incapital Accumulateddeficit Total Equity Shares Amount Shares Amount Balance, December 31, 2015— $— 14,634,855 $15 $161,359 $(147,171) $14,203Cumulative effect from adoption of newaccounting standard (Note 3)— — — — 100 (100) —Stock-based compensation expense— — — — 1,933 — 1,933Issuance of shares under “At-The-Market”equity program, net of offering costs— — 53,280 — — — —Intrinsic value of beneficial ownershipconversion feature, net of issuance costs(Note 7)— — — — 7,017 — 7,017Net loss— — — — — (15,292) (15,292)Balance, December 31, 2016— $— 14,688,135 $15 $170,409 $(162,563) $7,861Issuance of Series A convertible preferredstock with detachable warrants net ofissuance costs of $3778,000 — — — 7,623 — 7,623Stock-based compensation expense— — — — 322 — 322Exercise of liability-classified warrants— — 6,941 — 41 — 41Conversion of promissory notes— — 24,911 — 95 — 95Issuance of common stock with detachablewarrants net of issuance costs $1,175— — 11,220,260 11 9,294 — 9,305Net loss— — — — — (16,240) (16,240)Balance, December 31, 20178,000 $— 25,940,247 $26 $187,784 $(178,803) $9,007 The accompanying notes are an integral part of these consolidated financial statements.F 5Table of ContentsFibrocell Science, Inc.Consolidated Statements of Cash Flows($ in thousands) Year Ended December 31, 2017 2016Cash flows from operating activities: Net loss$(16,240) $(15,292)Adjustments to reconcile net loss to net cash used in operating activities: Stock-based compensation expense322 1,933Warrant liability revaluation income(4,920) (11,884)Derivative liability revaluation expense1,407 462Loss on disposal or impairment of property and equipment40 69Depreciation and amortization384 564Amortization of discount on convertible debt converted to common shares86 —Intangible asset impairment— 3,905Recovery of doubtful accounts— (12)Loss on write-down of inventory— 356Decrease (increase) in operating assets: Accounts receivable— 12Inventory— 126Prepaid expenses and other current assets28 796Other assets26 (65)Increase (decrease) in operating liabilities: Accounts payable69 (139)Related party payable1,361 (9,778)Accrued expenses and deferred rent(342) (214)Accrued interest payable742 228Deferred revenue— (457)Net cash used in operating activities(17,037) (29,390)Cash flows from investing activities: Purchase of property and equipment(433) (253)Proceeds from the sale of property and equipment— 1Net cash used in investing activities(433) (252)Cash flows from financing activities: Proceeds from private placement, net7,623 17,933Proceeds from common stock offering, net9,749 —Payment of deferred offering costs— (42)Principal payments on capital lease obligations— (2)Net cash provided by financing activities17,372 17,889Net decrease in cash and cash equivalents(98) (11,753)Cash and cash equivalents, beginning of period17,515 29,268Cash and cash equivalents, end of period$17,417 $17,515 Supplemental disclosures of cash flow information: Non-cash investing and financing activities: Property and equipment in accounts payable$29 $57Offering costs in accounts payable and accrued expenses$444 $23Reduction of warrant liability upon cashless exercise of warrants$41 $—Reduction of accrued interest payable upon cashless exercise of promissory notes$3 $—Reduction in derivative liability upon cashless exercise of promissory notes$6 $—Cashless exercise of promissory notes$85 $—Dividend paid in-kind to preferred stockholders$264 $—Deemed dividend on preferred stock$4,099 $— The accompanying notes are an integral part of these consolidated financial statements. F 6Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial StatementsNote 1. Business and OrganizationOrganizationFibrocell Science, Inc. (as used herein, “we,” “us,” “our,” “Fibrocell” or the “Company”) is the parent company of Fibrocell Technologies, Inc.(Fibrocell Tech). Fibrocell Tech is the parent company of Isolagen International, S.A., a company organized under the laws of Switzerland (IsolagenSwitzerland). The Company’s international activities are currently immaterial.Effective April 1, 2016, Fibrocell Science Hong Kong Limited (Fibrocell Hong Kong), a company organized under the laws of Hong Kong andformer subsidiary of Fibrocell, was dissolved. As this entity had no historical financial or operational activities, the impact of the dissolution did not, and isnot expected to have, a material impact on the Company’s present or future consolidated financial statements.Business OverviewFibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs. The Company is focusedon discovering and developing therapies for the localized treatment of diseases affecting the skin and connective tissue. All of the Company’s productcandidates incorporate its proprietary autologous fibroblast technology. The Company’s research and development efforts focus on gaining regulatoryapprovals of its product candidates in the United States.Liquidity and Financial ConditionThe Company expects to continue to incur losses and will require additional capital to advance its product candidates through development tocommercialization. For the year ended December 31, 2017, the Company incurred a net loss of approximately $16.2 million, had an accumulated deficit of$178.8 million and used approximately $17.0 million in cash for operations. As of December 31, 2017, the Company had cash and cash equivalents ofapproximately $17.4 million and working capital of approximately $13.5 million. The Company believes that its cash and cash equivalents at December 31,2017, will be sufficient to fund operations into the first quarter of 2019. The Company will require additional capital to fund operations beyond that point.To meet its capital needs, the Company intends to raise additional capital through debt or equity financings, collaborations, partnerships or other strategictransactions. However, there can be no assurance that the Company will be able to complete any such transaction on acceptable terms or otherwise. Thefailure of the Company to obtain sufficient funds on acceptable terms when needed could have a material adverse effect on the Company’s business, results ofoperations and financial condition. These conditions raise substantial doubt about its ability to continue as a going concern. The accompanyingconsolidated financial statements do not include any adjustments that might result from the outcome of this uncertainty. Accordingly, the consolidatedfinancial statements have been prepared on a basis that assumes the company will continue as a going concern and which contemplates the realization ofassets and satisfaction of liabilities and commitments in the ordinary course of business.On January 23, 2018, the Company received notice from the Nasdaq Stock Market LLC that the Company is not in compliance with Nasdaq ListingRule 5550(a)(2), as the minimum bid price of the Company’s common stock has been below $1.00 per share for 30 consecutive business days. The Notice hasno immediate effect on the listing of the Company’s common stock, which will continue to trade at this time on the Nasdaq Capital Market under the symbol“FCSC.”In accordance with Nasdaq Listing Rule 5810(c)(3)(A), the Company has a period of 180 calendar days, or until July 23, 2018, to regain compliancewith the minimum bid price requirement. To regain compliance, the closing bid price of the Company’s common stock must meet or exceed $1.00 per sharefor at least ten consecutive business days during this 180 calendar day period. In the event the Company does not regain compliance by July 23, 2018, theCompany may be eligible for an additional 180 calendar day grace period if it meets the continued listing requirement for market value of publicly heldshares ($1 million) and all other initial listing standards for the Nasdaq Capital Market, with the exception of the minimum bid price, and provides writtennotice to Nasdaq of its intention to cure the deficiency during the second compliance period by effecting a reverse stock split, if necessary. If the Companydoes not regain compliance within the allotted compliance period(s), Nasdaq will provide notice that the Company’s common stock will be subject todelisting from the Nasdaq Capital Market. In that event, the Company may appeal such delisting determination to a hearings panel.F 7Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 1. Business and Organization (continued)The Company intends to monitor the closing bid price of its common stock and consider options to resolve its noncompliance with the minimum bidprice requirement.Nasdaq has the authority, pursuant to Nasdaq Listing Rule 5550(b)(1), to delist our common stock if our stockholders’ equity falls below $2.5million. As of December 31, 2017, our stockholders’ equity was $9.0 million. If our stockholders equity is hereafter reduced below $2.5 million as a result ofoperating losses or for other reasons, we will fail to meet Nasdaq’s stockholders’ equity requirement. If that occurs, or if we are unable to demonstrate toNasdaq’s satisfaction that we will be able to sustain compliance with this requirement, Nasdaq may delist our common stock. In addition, even if we regaintechnical compliance with the stockholders’ equity requirement, we will have to continue to meet other objective and subjective listing requirements tocontinue to be listed on the Nasdaq Capital Market, including the requirement that our common stock continues to trade above $1.00.We are actively monitoring our stockholders’ equity and will consider any and all options available to us to maintain compliance. There can be noassurance, however, that we will be able to maintain compliance and meet Nasdaq’s minimum stockholders’ equity requirements. F 8Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial StatementsNote 2. Basis of PresentationGeneralThe accompanying Consolidated Financial Statements have been prepared in accordance with U.S. generally accepted accounting principles(GAAP) and include the accounts of Fibrocell and its wholly owned subsidiaries. The accompanying Consolidated Financial Statements should be read inconjunction with the Notes to the Consolidated Financial Statements.All intercompany accounts and transactions have been eliminated in consolidation. The Company’s foreign operations are immaterial and it has nounrealized gains or losses from the sale of investments. As a result, it does not have any items that would be classified as other comprehensive income in sucha statement.ReclassificationsOn March 10, 2017, the Company implemented a one-for-three reverse split of its issued and outstanding shares of common stock (the Reverse StockSplit), as authorized at a special meeting of stockholders on March 1, 2017. The Reverse Stock Split became effective on March 10, 2017 at 5:00 pm and theCompany’s common stock began trading on The Nasdaq Capital Market on a post-split basis at the open of business on March 13, 2017. As of a result of theReverse Stock Split, every three shares of the Company’s issued and outstanding common stock were combined into one share of its common stock, except tothe extent that the Reverse Stock Split resulted in any of the Company’s stockholders owning a fractional share, which was rounded up to the next highestwhole share. In connection with the Reverse Stock Split, there was no change in the nominal par value per share of $0.001. The Reverse Stock Split waseffectuated in order to increase the per share trading price of the Company’s common stock to satisfy the $1.00 minimum bid price requirement for continuedlisting on The Nasdaq Capital Market. By letter dated March 27, 2017, The Nasdaq Capital Market Listing Qualification Department, confirmed that theCompany’s common stock was in compliance with listing requirements.All share and per share amounts of common stock, options and warrants in the accompanying financial statements have been restated for all periodsto give retroactive effect to the Reverse Stock Split. Accordingly, the Consolidated Statement of Stockholders’ Equity reflects the impact of the ReverseStock Split by reclassifying from “Common Stock” to “Additional paid-in capital” an amount equal to the par value of the decreased shares resulting fromthe Reverse Stock Split.Note 3. Summary of Significant Accounting PoliciesUse of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates, judgments, and assumptions that affectthe reported amounts of assets, liabilities, equity, revenues and expenses, and related disclosure of contingencies in the accompanying ConsolidatedFinancial Statements and Notes. In addition, management’s assessment of the Company’s ability to continue as a going concern involves the estimation ofthe amount and timing of future cash inflows and outflows. On an ongoing basis, the Company evaluates its estimates, judgments and methodologies. TheCompany bases its estimates on historical experience and on various other assumptions that are believed to be reasonable. Actual results may differmaterially from those estimates.Segment InformationThe Company has determined that it operates in only one segment, as it only reports operational results on an aggregate basis to its chief operatingdecision maker. Additionally, all of the Company’s research development activities occur in, and assets are located in, the United States.Cash and Cash EquivalentsThe Company considers highly liquid investments with an original maturity of three months or less when purchased to be cash equivalents.F 9Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 3. Summary of Significant Accounting Policies (continued)Concentration of Credit RiskFinancial instruments that potentially subject the Company to concentrations of credit risk are limited to the Company’s cash and cash equivalents.As of December 31, 2017, the Company maintains its operating cash with one major U.S. domestic bank and the remainder of its cash and cash equivalents asa money market fund with one major global bank. Federal insurance coverage on operating cash amounted to $250,000 per depositor at each financialinstitution, and the Company’s non-interest bearing cash balances may exceed federally insured limits. The terms of these deposits are on demand tominimize risk. The Company has not incurred losses related to these deposits.Property and EquipmentProperty and equipment is carried at acquisition cost less accumulated depreciation, subject to review for impairment whenever events or changes incircumstances indicate that the carrying amount of the asset may not be recoverable as described further under the heading “Impairment of Long-livedAssets” below. The cost of normal, recurring, or periodic repairs and maintenance activities related to property and equipment are expensed as incurred. Thecost for planned major maintenance activities, including the related acquisition or construction of assets, is capitalized if the repair will result in futureeconomic benefits.Depreciation is computed on a straight-line basis over the estimated useful life of the respective assets, which are summarized as follows:Property and equipment category Useful lifeComputer equipment and software 3 yearsLaboratory equipment 6 yearsFurniture and fixtures 10 yearsLeasehold improvements Lesser of remaining lease term or life of assetWhen an asset is disposed of, the associated cost and accumulated depreciation is removed from the related accounts on the Company’sConsolidated Balance Sheet with any resulting gain or loss included in the Company’s Consolidated Statement of Operations.Intangible AssetsIntangible assets were research and development assets related to the Company’s primary study on azficel-T that were capitalized on the balancesheet upon emergence from bankruptcy. The portion of the reorganization value which was attributed to identifiable intangible assets was $6.3 million.Azficel-T had two target indications: the Company’s FDA-approved product LAVIV® and a clinical development program for azficel-T for the treatment ofvocal cord scarring resulting in chronic or severe dysphonia. Effective January 1, 2012, the Company launched LAVIV and as a result, the research anddevelopment intangible assets related to the Company’s primary study were considered to be finite-lived intangible assets and began amortizing over 12years, the estimated useful life of the assets which was analogous with the exclusivity period granted to the Company under the BLA.Finite-lived intangible assets are recorded at cost, net of accumulated amortization, and if applicable, impairment charges. Amortization of finite-lived intangible assets is provided over their estimated useful lives on a straight-line basis. The Company reviews the estimated remaining useful life of itsintangible assets on an annual basis with any changes, if applicable, accounted for prospectively. Additionally, finite-lived intangible assets are reviewed forimpairment whenever events or changes in circumstances indicate that the carrying amount of the asset may not be recoverable as described further under theheading "Impairment of Long-lived Assets" below. Amortization expense for the year ended December 31, 2016 was approximately $0.2 million. For the yearended December 31, 2017, the Company did not have any intangible assets nor any amortization expense of them. See below for discussion of impairmentcharges incurred.F 10Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 3. Summary of Significant Accounting Policies (continued)Impairment of Long-Lived AssetsIn accordance with Financial Accounting Standards Board (FASB) Accounting Standards Codification (ASC) 360-10-35, Impairment or Disposal ofLong-Lived Assets, the Company reviews its long-lived assets and identifiable finite-lived intangible assets for impairment whenever events or changes incircumstances indicate that the carrying amount of an asset may not be recoverable (i.e., impaired). Once an impairment is determined, the actual impairmentrecognized is the difference between the carrying amount and the fair value (less costs to sell for assets to be disposed of) as estimated using one of thefollowing approaches: income, cost and/or market. Fair value using the income approach is determined primarily using a discounted cash flow model thatuses the estimated cash flows associated with the asset or asset group under review, discounted at a rate commensurate with the risk involved. Fair valueutilizing the cost approach is determined based on the replacement cost of the asset reduced for, among other things, depreciation and obsolescence. Fairvalue, utilizing the market approach, benchmarks the fair value against the carrying amount.In June 2016, based on its failure to achieve primary efficacy endpoints for its Phase II clinical trial of azficel-T for the treatment of vocal cordscarring, the Company determined to wind-down its azficel-T operations as more fully described in Note 12. As a result, management concluded that theCompany’s intangible assets had become fully impaired. Accordingly, a non-cash impairment charge of approximately $3.9 million was recorded during thesecond quarter of 2016 and is included in the Consolidated Statement of Operations for the year ended December 31, 2016, as a result of the impairment, thecarrying value of the intangible asset was $0 as of December 31, 2017 and 2016, respectively.Warrant LiabilityThe Company accounts for stock warrants as either equity instruments, derivative liabilities, or liabilities in accordance with ASC 480,Distinguishing Liabilities from Equity (ASC 480), depending on the specific terms of the warrant agreement. Stock warrants are accounted for as a derivativein accordance with ASC 815, Derivatives and Hedging (ASC 815) if the stock warrants contain “down-round protection” or other terms that could potentiallyrequire “net cash settlement” and therefore, do not meet the scope exception for treatment as a derivative. Since “down-round protection” is not an input intothe calculation of the fair value of the warrants, the warrants cannot be considered indexed to the Company’s own stock which is a requirement for the scopeexception as outlined under ASC 815. Warrant instruments that could potentially require “net cash settlement” in the absence of express language precludingsuch settlement and those which include “down-round provisions” are initially classified as derivative liabilities at their estimated fair values, regardless ofthe likelihood that such instruments will ever be settled in cash. The Company will continue to classify the fair value of the warrants that contain “down-round protection” and “net cash settlement” as a liability until the warrants are exercised, expire or are amended in a way that would no longer require thesewarrants to be classified as a liability. Warrants that the Company may be required to redeem through payment of cash or other assets outside its control areclassified as liabilities pursuant to ASC 480 and are initially and subsequently measured at their estimated fair values. For additional discussion on warrants,see Note 8.Debt Issued With WarrantsThe Company considers guidance within ASC 470-20, Debt (ASC 470), ASC 480, and ASC 815 when accounting for the issuance of convertibledebt with detachable warrants. As described above under the caption “Warrant Liability”, the Company classifies stock warrants as either equity instruments,derivative liabilities, or liabilities depending on the specific terms of the warrant agreement. In circumstances in which debt is issued with liability-classifiedwarrants, the proceeds from the issuance of convertible debt are first allocated to the warrants at their full estimated fair value and established as both aliability and a debt discount. The remaining proceeds, as further reduced by discounts created by the bifurcation of embedded derivatives and beneficialconversion features, are allocated to the debt. The Company accounts for debt as liabilities measured at amortized cost and amortizes the resulting debtdiscount from the allocation of proceeds, to interest expense using the effective interest method over the expected term of the debt instrument pursuant toASC 835, Interest (ASC 835).Embedded Derivatives. The Company considers whether there are any embedded features in debt instruments that require bifurcation and separateaccounting as derivative financial instruments pursuant to ASC 815. Embedded derivatives are initially and subsequently measured at fair value. See Note 7for additional discussion on the embedded derivatives associated with the Company’s convertible notes.Beneficial Conversion Feature. If the amount allocated to the convertible debt results in an effective per share conversion price less than the fairvalue of the Company’s common stock on the commitment date, the intrinsic value of thisF 11Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 3. Summary of Significant Accounting Policies (continued)beneficial conversion feature is recorded as a discount to the convertible debt with a corresponding increase to additional paid in capital. The beneficialconversion feature discount is equal to the difference between the effective conversion price and the fair value of the Company’s common stock at thecommitment date, unless limited by the remaining proceeds allocated to the debt. See Note 7 for additional discussion on the beneficial conversion featureassociated with the Company’s convertible notes.Debt Issuance Costs. The Company follows the guidance under Accounting Standards Update (ASU) 2015-03, Interest - Imputation of Interest(Subtopic 835-30): Simplifying the Presentation of Debt Issuance Costs (ASU 2015-03) for accounting for debt issuance costs. The Company allocates debtissuance costs between the debt and the warrants on the same basis as proceeds were allocated. The Company expenses issuance costs allocated to thewarrants and presents the issuance costs allocated to the debt as a direct reduction from the carrying amount of the debt liability in the balance sheet.However, if debt issuance costs exceed the carrying amount of the debt, issuance costs are recorded to additional paid-in capital as a reduction of thebeneficial conversion feature. As of December 31, 2017, the Company’s debt issuance costs are presented in additional paid-in capital as a reduction of thebeneficial conversion feature and are being amortized to interest expense (despite their classification in additional paid-in capital) using the effective interestrate method over the expected term of the debt pursuant to ASC 835.Revenue RecognitionRevenue from Product Sales. In June 2011, the FDA approved the Company’s BLA for LAVIV for the improvement of the appearance of moderate tosevere nasolabial fold wrinkles in adults. The Company recognizes revenue from product sales in accordance with ASC 605, Revenue Recognition (ASC605). In general, ASC 605 requires that four basic criteria must be met before revenue can be recognized: (1) persuasive evidence of an arrangement exists,(2) delivery has occurred or services rendered, (3) the fee is fixed and determinable and (4) collectability is reasonably assured. Prepayments on product sales are generally received at three different stages of the treatment: (1) the biopsy stage, (2) the cell harvest stage, and (3)the injection stage. As one full course of LAVIV therapy includes three series of injections, prepayments are deferred and revenue is recognized on a proratabasis as each of the three series of injections is shipped to the physician. In connection with the wind-down of azficel-T operations during 2016 as more fullydescribed in Note 12, the Company wrote off all remaining raw materials and work-in-process inventories as of September 30, 2016. Collaboration Revenue. The Company follows ASC 605-25, Revenue Recognition – Multiple-Element Arrangements (ASC 605-25) and ASC 808,Collaborative Arrangements, if applicable, to determine the recognition of revenue under its collaborative research, development and commercializationagreements. The terms of these agreements generally contain multiple elements, or deliverables, which may include (i) grants of licenses, or options to obtainlicenses, to our intellectual property, (ii) research and development services, (iii) clinical and commercial manufacturing, and/or (iv) participation on jointresearch and/or joint development committees. The payments the Company may receive under these arrangements typically include one or more of thefollowing: non-refundable, up-front license fees; funding of research and/or development efforts including proof-of-concept studies and productdevelopment; amounts due upon the achievement of specified objectives or milestones such as obtaining patents, trademarks and certain regulatoryapprovals, and achievement of commercialization of products; and/or royalties on future product sales.Each of the required deliverables under such an arrangement are evaluated, in accordance with ASC 605-25, to determine whether it qualifies as aseparate unit of accounting based on whether the deliverable has “stand-alone value” to the customer. The arrangement’s consideration that is fixed ordeterminable is then allocated to each separate unit of accounting based on the relative selling price of each deliverable. In general, the considerationallocated to each unit of accounting is recognized as the related goods or services are delivered, limited to the consideration that is not contingent uponfuture deliverables. Collaboration revenue is recognized on a gross basis, in accordance with the criteria set forth in ASC 605-45, Revenue Recognition:Principal Agent Considerations. Collaboration revenue for the year ended December 31, 2016 is related to a research and development agreement that the Company has with a thirdparty to investigate potential new non-pharmaceutical applications for the Company’s conditioned fibroblast media technology. Revenue recognized to datefrom this collaboration relates to an upfront license fee that was amortized over the estimated total contract period and a proof-of-concept study which wascompleted in 2015.F 12Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 3. Summary of Significant Accounting Policies (continued)The Company will recognize future milestone payments when earned provided that (1) the milestone event is substantive in that it can only beachieved based in whole or in part on either the Company’s performance or on the occurrence of a specific outcome resulting from the Company’sperformance and its achievability was not reasonably assured at the inception of the agreement; (2) the Company does not have ongoing performanceobligations related to the achievement of the milestone; and (3) it would result in the receipt of additional payments. A milestone payment is consideredsubstantive if all of the following conditions are met: (a) the milestone payment is non-refundable; (b) achievement of the milestone was not reasonablyassured at the inception of the arrangement; (c) substantive effort is involved to achieve the milestone; and (d) the amount of the milestone payment appearsreasonable in relation to the effort expended, the other milestones in the arrangement and the related risk associated with the achievement of the milestone.Cost of RevenueCost of revenue includes expenses related to revenue from product sales and collaboration revenue.Cost of Product Sales. Costs include the expense to manufacture LAVIV, including direct and indirect costs. Costs incurred for shipping andhandling during the biopsy stage (to/from physicians) are included in cost of product sales. Costs related to shipping and handling of injections (tophysicians) are included in selling, general and administrative expenses. Cost of Collaboration Revenue. Costs directly related to deliverables in a revenue-generating collaboration are charged to cost of collaboration revenue as incurred.Research and Development ExpensesResearch and development costs are expensed as incurred and include employee salaries and benefits, costs incurred with third party contractors toperform research, conduct clinical trials, develop and manufacture drug materials and delivery devices, and a portion of facilities costs. Research anddevelopment expenses also include costs to manufacture product for clinical trial use and to develop manufacturing, cell collection and logistical processimprovements. Clinical trial costs are a significant component of research and development expenses, often with third party service providers. Invoicing from thirdparty contractors for services performed can lag several months. The Company accrues the costs of services rendered in connection with third party contractoractivities based on its estimate of management fees, site management and monitoring costs and data management costs incurred in a given period.Stock-Based CompensationThe Company follows ASC 718, Compensation – Stock Compensation (ASC 718), or ASC 505-50, Equity – Equity Based Payments to Non-Employees, where applicable. The Company accounts for stock-based awards to employees using the fair value based method to determine compensation forall arrangements where shares of stock or equity instruments are issued for compensation. In addition, the Company accounts for stock-based compensationto non-employees in accordance with the accounting guidance for equity instruments that are issued to entities or persons other than employees. TheCompany uses a Black-Scholes option-pricing model to determine the fair value of each option grant as of the date of grant for expense incurred. The Black-Scholes option pricing model requires inputs for risk-free interest rate, dividend yield, expected stock price volatility and expected term of the options. Thevalue of the award that is ultimately expected to vest based on the achievement of a performance condition (i.e., service period) is recognized as expense on astraight-line basis over the requisite service period. See Note 11 for additional details.Previously, ASC 718 required forfeitures to be estimated at the time of grant and revised, if necessary, in subsequent periods if actual forfeituresdiffered from those estimates. In the first quarter of 2016, the Company adopted ASU 2016-09, Compensation - Stock Compensation (Topic 718):Improvements to Employee Share-Based Payment Accounting (ASU 2016-09), which allows an entity to elect as an accounting policy either to continue toestimate the total number of awards for which the requisite service period will not be rendered or to account for forfeitures when they occur. In connectionwith the adoption of this ASU, the Company made an accounting policy election to account for forfeitures as they occur and applied this change inaccounting policy on a modified retrospective basis. As a result, the Company recorded a cumulative effect adjustment to retained earnings in 2016 whichresulted in an increase to accumulated deficit of $0.1 million with an offsetting increase to additional paid-in capital (zero net total equity impact) as of thedate of adoption, principally related to additional stock compensation expense that would have been recognized on unvested outstanding optionsunadjusted for estimated forfeitures.F 13Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 3. Summary of Significant Accounting Policies (continued)Restructuring CostsRestructuring charges are primarily comprised of severance costs related to workforce reductions, contract termination and wind-down costs, assetimpairments and costs of decommissioning the Company’s azficel-T manufacturing facility. In accordance with ASC 420, Exit or Disposal Cost Obligations,the Company recognizes restructuring charges when the liability has been incurred, except for one-time employee termination benefits that are incurred overtime. Generally, one-time employee termination benefits (i.e., severance costs) are accrued at the date management has committed to a plan of terminationand employees have been notified of their termination dates and expected severance payments. Other costs, including but not limited to, contract terminationand wind-down costs and manufacturing facility decommissioning costs, will be recorded as incurred. Asset impairment charges have been, and will be,recognized when management has concluded that the assets have been impaired in accordance with ASC 360-10-35, Impairment or Disposal of Long-LivedAssets, or other applicable authoritative guidance. See Note 12 for additional details.Income TaxesAn asset and liability approach is used for financial accounting and reporting for income taxes. Deferred income taxes arise from temporarydifferences between income tax and financial reporting and principally relate to recognition of revenue and expenses in different periods for financial and taxaccounting purposes and are measured using currently enacted tax rates and laws. In addition, a deferred tax asset can be generated by a net operating losscarryover. If it is more likely than not that some portion or all of a deferred tax asset will not be realized, a valuation allowance is recognized.In the event the Company is charged interest or penalties related to income tax matters, the Company would record such interest as interest expenseand would record such penalties as other expense in the Consolidated Statements of Operations. No such charges have been incurred by the Company. Foreach of the years ended December 31, 2017 and 2016, the Company had no uncertain tax positions. See Note 13 for additional details.Loss Per Share DataBasic loss per share is computed by dividing net loss for the period by the weighted average number of shares of common stock outstanding duringthat period. The diluted loss per share calculation gives effect to dilutive stock options, warrants, convertible notes and other potentially dilutive commonstock equivalents outstanding during the period. Diluted loss per share is based on the if-converted method or the treasury stock method, as applicable, andincludes the effect from the potential issuance of common stock, such as shares issuable pursuant to the conversion of convertible notes and the exercise ofstock options and warrants, assuming the exercise of all “in-the-money” common stock equivalents based on the average market price during the period.Common stock equivalents have been excluded where their inclusion would be anti-dilutive. See Note 15 for additional details. Recently Issued Accounting PronouncementsIn July 2017, the FASB issued ASU No. 2017-11, “Earnings Per Share (Topic 260), Distinguishing Liabilities from Equity (Topic 480) andDerivatives and Hedging (Topic 815): Part 1 - Accounting for Certain Financial Instruments with Down Round Features and Part 2 - Replacement of theIndefinite Deferral for Mandatorily Redeemable Financial Instruments of Certain Nonpublic Entities and Certain Mandatorily Redeemable NoncontrollingInterests with Scope Exception”. Part 1 of ASU No. 2017-11 addresses the complexity of accounting for certain financial instruments with down roundfeatures. Down round features are features of certain equity-linked instruments (or embedded features) that result in the strike price being reduced on the basisof the pricing of future equity offerings. Current accounting guidance creates cost and complexity for entities that issue financial instruments (such aswarrants and convertible instruments) with down round features that require fair value measurement of the entire instrument or conversion option. Part II ofASU No. 2017-11 addresses the difficulty of navigating Topic 480, Distinguishing Liabilities from Equity, because of the existence of extensive pendingcontent in the FASB Accounting Standards Codification®. This pending content is the result of the indefinite deferral of accounting requirements aboutmandatorily redeemable financial instruments of certain nonpublic entities and certain mandatorily redeemable noncontrolling interests. For public businessentities, the amendments in Part I of this update are effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2018.The amendments in Part II of this update do not require any transition guidance because those amendments do not have an accounting effect. The Companycurrently does not have any outstanding financial instruments with down round provisions, and therefore the impact of the adoption of this standard on itsConsolidated Financial Statements, will not be material.F 14Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 3. Summary of Significant Accounting Policies (continued)In May 2017, the FASB issued ASU 2017-09, “Scope of Modification Accounting,” which clarifies the application of stock based accountingguidance when a change is made to the terms or conditions of a share-based payment award. The guidance is effective for the Company beginning in the firstquarter of fiscal year 2018. Early adoption is permitted. The Company does not anticipate any material impacts of adopting this guidance on its ConsolidatedFinancial Statements. In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842), which is intended to increase transparency and comparability amongorganizations by recognizing lease assets and lease liabilities on the balance sheet (including by lessees for those leases classified as operating leases underprevious GAAP) and disclosing key information about leasing arrangements. The guidance is effective for public companies with annual reporting periodsbeginning after December 15, 2018, including interim periods within that reporting period. Earlier application is permitted. While the Company is currentlyassessing the full impact this ASU will have on its Consolidated Financial Statements, the Company believes the primary impact upon adoption will be therecognition, on a discounted basis, of its minimum commitments under the current noncancelable operating lease, as amended, for its Exton, PA facility,resulting in the recording of right of use assets and lease obligations. The Company does not anticipate any other material impacts to its ConsolidatedFinancial Statements. Current minimum commitments under noncancelable operating leases are disclosed in Note 16 of these Notes. From time to time, new accounting pronouncements are issued by the FASB and rules are issued by the SEC that we adopt as of the specified date.Unless otherwise noted, management does not believe that any other recently issued accounting pronouncements issued by the FASB or guidance issued bythe SEC had, or is expected to have, a material impact on the Company’s present or future consolidated financial statements.F 15Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial StatementsNote 4. InventoryFor the years ended December 31, 2017 and 2016, the Company had no inventory on hand.Inventories have historically been recorded at the lower of cost or market value, with cost determined under specific identification and on the first-in, first-out (FIFO) method. Inventories consisted of raw materials and work-in-process intended for use in the manufacture of LAVIV, which was approved bythe FDA in 2011 for the improvement of nasolabial fold wrinkles in adults. Raw materials that could be used either for manufacturing pre-clinical and clinicalproduct candidates or the production of commercial products were expensed as research and development costs when selected for use in pre-clinical orclinical manufacturing operations. As a result of the wind-down of the Company’s azficel-T operations, more fully described in Note 12, the Companywrote off all remaining raw materials and work-in-process inventories as of September 30, 2016. Total inventory write-offs of approximately $0.4 million are included in cost of product sales in the Company’s Consolidated Statement ofOperations for the year ended December 31, 2016. For the year ended December 31, 2017, raw materials purchased for pre-clinical and clinical trials werecharged to R&D expense as incurred.Note 5. Property and EquipmentProperty and equipment consisted of the following as of: December 31,($ in thousands)2017 2016Laboratory equipment$1,514 $1,429Computer equipment and software318 313Furniture and fixtures44 44Leasehold improvements1,412 1,228Construction-in-process101 36Total property and equipment, gross3,389 3,050Less: Accumulated depreciation(1,919) (1,561)Total property and equipment, net$1,470 $1,489Depreciation expense was approximately $0.4 million and $0.3 million for the years ended December 31, 2017 and 2016, respectively.Note 6. Accrued ExpensesAccrued expenses consisted of the following as of: December 31,($ in thousands)2017 2016Accrued professional fees$322 $526Accrued compensation462 631Accrued other476 394Total accrued expenses$1,260 $1,551F 16Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial StatementsNote 7. Convertible Notes2016 Private PlacementIn September 2016, the Company issued an aggregate of $18,087,500 in principal of convertible promissory notes (each, a Note and collectively,the Notes) and accompanying warrants to purchase an aggregate of 6,029,174 shares of common stock (each a Warrant and collectively, the Warrants) in aprivate placement to institutional and accredited investors (each an Investor and collectively, the Investors).The Notes bear interest at four percent (4%) per annum. Interest is earned daily and compounded quarterly and, at the election of the Company at thebeginning of each quarter, shall accrue or be paid in cash. If the Company elects to have interest accrue, such interest will not be added to the principalamount of the Notes but such interest shall be subject to additional interest at the rate of four percent (4%) per annum, compounded quarterly, and shall bedue and payable upon the earliest of the conversion of the Notes, exercise of the Put Right, exercise of the Prepayment Right or the Maturity Date (in eachcase, as defined below). Additionally, if the Company elects for interest to accrue, then (i) the Company may elect to repay any such accrued and unpaidinterest in cash at any time and from time to time and (ii) each Investor may elect to have the Company repay any such accrued and unpaid interest bydelivering such number of shares of common stock equal to (x) the amount of the accrued and unpaid interest to be repaid, divided by (y) the greater of (i) thelast closing bid price of a share of Common Stock as reported on Nasdaq on the date of such election and (ii) the Conversion Price (as defined below). As ofDecember 31, 2017, the Company has elected to accrue interest.All unpaid principal of each Investor’s Note is convertible, at any time and from time to time, at the option of such Investor into shares of commonstock at a range of $3.40875 to $3.67875 (the “conversion price”), and any unpaid interest at the greater of (x) the conversion price and (y) the last closingbid price of a share of common stock as reported on Nasdaq at the time of such Investor’s execution of the Purchase Agreement plus $0.12625.The Notes have a maturity date of the earlier of (i) September 7, 2026 and (ii) one-hundred and eighty (180) days after the date on which theCompany’s product candidate, FCX-007, is approved by the United States Food and Drug Administration for the treatment of recessive dystrophicepidermolysis bullosa (the Maturity Date). Each Investor has the right to require the Company to repay all or any portion of the unpaid principal and accruedand unpaid interest from time to time on or after September 7, 2021 (such right, a Put Right). Such Put Right must be exercised by such Investor by deliveringwritten notice to the Company no later than one-hundred and eighty (180) days prior to such exercise date of such Put Right. In addition, uponconsummation of a specified change of control transaction, each Investor may elect to accelerate the repayment of all unpaid principal and accrued interestunder such Investor’s Note. If an Investor does not elect to have the Company prepay its Note upon such change of control transaction, then the Companymay prepay the Notes, in an amount equal to one hundred one percent (101%) of the outstanding principal due under the Notes (together with accrued andunpaid interest due thereon) (the Prepayment Right). Additionally, upon the occurrence of certain Events of Default, as defined in the Notes, each Investormay elect to accelerate the repayment of all unpaid principal and accrued interest under each Note and the Notes provide for automatic redemption upon theoccurrence of certain bankruptcy related Events of Default, as defined in the Notes.Accounting for Convertible Notes and Embedded DerivativesThe Company accounts for debt as liabilities measured at amortized cost and amortizes the resulting debt discount from allocation of proceeds tointerest expense using the effective interest method over the expected term of the Notes pursuant to ASC 835, Interest (ASC 835). See Note 3 for discussion of the Company’s policies for accounting for debt with detachable warrants. In connection with the issuance of the Notesand Warrants, the Company recorded a debt discount of approximately $18.1 million based on an allocation of proceeds to the Warrants of approximately$9.6 million, an allocation to bifurcated derivatives (which consist of a contingent put option upon a change of control or acceleration upon event of default(the Contingent Put Option) and a contingent call option upon a change of control (the Contingent Call Option) included in the Notes) of approximately$1.3 million, and a beneficial conversion feature of approximately $7.2 million, before issuance costs, based on the difference between the fair value of theunderlying common stock at the commitment date of each Note transaction and the effective conversion price of the Notes, as limited by the proceedsallocated to the Notes. F 17Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 7. Convertible Notes (continued)Convertible promissory notes outstanding were as follows:($ in thousands)December 31,2017 2016Convertible promissory notes$18,003 $18,088Debt discount - warrants(9,598) (9,643)Debt discount - compound bifurcated derivatives(1,267) (1,273)Debt discount - beneficial conversion feature(7,138) (7,172)Convertible promissory notes, net$— $—The debt discount and issuance costs are amortized using the effective interest method over five years, the expected term of the Notes. Amortizationof the debt discounts included in interest expense in the Consolidated Statement of Operations for the year ended December 31, 2017 and December 31, 2016was approximately $0.1 million and $0. Based on an effective yield of approximately 1157% resulting from the Notes being initially recorded at a fulldiscount, the Company will not recognize any material amounts of amortization until years 2020 and 2021. The amortization of debt discount recorded inthe year ended December 31, 2017 was related to the conversion of notes into shares.Assumptions Used in Determining Fair Value of Compound Bifurcated DerivativeThe Company utilizes a binomial lattice model to value its bifurcated derivatives included in the Notes. ASC 815 does not permit an issuer toaccount separately for individual derivative terms and features embedded in hybrid financial instruments that require bifurcation and liability classificationas derivative financial instruments. Rather, such terms and features must be combined together and fair valued as a single, compound embedded derivative.The Company selected a binomial lattice model to value the compound embedded derivative because it believes this technique is reflective of all significantassumptions that market participants would likely consider in negotiating the transfer of the Notes. Such assumptions include, among other inputs:Volatility. The Company estimates stock price volatility based on the Company’s historical stock price performance over a period of time thatmatches the volume-weighted average expected remaining life of the Notes.Risk-free interest rate. The risk-free interest rate is based on the U.S. Treasury zero-coupon yield curve in effect at the valuation date commensuratewith the expected remaining life assumption.Expected remaining life. The expected life of the Notes is assumed to be equivalent to their remaining contractual term.Dividend rate. The dividend rate is based on the historical rate, which the Company anticipates will remain at zero.Scenarios. The probability of complex features of the compound bifurcated derivative being triggered is subjective (no observable inputs oravailable market data) and based on internal and external information known to management at the valuation date. Such assumptions include,among other inputs, probabilities related to a change of control and when it might occur as well as probabilities related to a default under theprovisions of the Notes and when it might occur.Changes to the key assumptions or to the scenarios used in the valuation model, including the probability of key events, such as a change of controltransaction, could have a material impact to the overall valuation of the compound bifurcated derivative liability. Additionally, there are other embeddedfeatures of the Notes requiring bifurcation, other than the Contingent Put Option and the Contingent Call Option, which had no value at December 31, 2017or December 31, 2016, due to management’s estimates of the likelihood of certain events, but that may have value in the future should those estimateschange.F 18Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 7. Convertible Notes (continued)The estimated fair value of the compound bifurcated derivative is determined using Level 2 and Level 3 inputs. Significant inputs and assumptionsused in the binomial lattice model for the derivative liability are as follows:($ in thousands except per share data)December 31, 2017December 31, 2016Calculated aggregate value$3,136$1,735Closing price per share of common stock$0.64$1.89Contractual remaining term8 years, 8 months9 years, 8 monthsContractual interest rate4.0%4.0%Volume-weighted average conversion rate$3.40933$3.40985Risk-free interest rate (term structure)1.28% - 2.40%.44% - 2.45%Dividend yield——Credit RatingCCCCCredit Spread36.98%33.27%Volatility99.0%99.9%The foregoing compound bifurcated derivative was recorded at its estimated fair value at the date of issuance, with subsequent changes in estimatedfair value recorded in derivative revaluation expense in the Company’s Consolidated Statement of Operations. The change in estimated fair value of theCompany’s derivative liability for the years ended December 31, 2017 and December 31, 2016, resulted in non-cash expense of approximately $1.4 millionand $0.5 million respectively.F 19Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial StatementsNote 8. WarrantsThe Company accounts for common stock warrants as equity instruments, derivative liabilities, or liabilities, depending on the specific terms of thewarrant agreement. See Note 3 for further details on accounting policies related to the Company’s stock warrants.In connection with various financing transactions, the Company has issued warrants to purchase the Company’s common stock. In December 2017,the Company issued (i) pre-funded warrants to purchase an aggregate of 5,922,208 shares of the Company’s common stock and (ii) common stock purchasewarrants to purchase up to an aggregate of 14,046,950 shares of the Company’s common stock including warrants to purchase up to 410,586 shares, issuedpursuant to the partial exercise of the underwriters option to purchase additional common stock purchase warrants. Each pre-funded warrant was sold togetherwith a common stock purchase warrant to purchase one share of the Company’s common stock at a combined effective price of $0.77 per share andaccompanying warrant. Each common stock purchase warrant has an exercise price of $0.77 per share, was exercisable upon the date of issuance and expiresfive years from the date of issuance. As additional compensation, the Company issued warrants to the underwriter to purchase 436,364 shares of theCompany’s common stock. Each such warrant has an exercise price of $0.9625 per share, and was exercisable as of the date of the underwriting agreement,and will expire five years after the date of the underwriting agreement, all as more fully described in Note 9.In March 2017, the Company issued warrants to purchase 3,437,334 shares of its common stock in connection with the Company’s public offeringof convertible preferred stock and warrants (each a Series A Warrant and collectively, the Series A Warrants), more fully described in Note 10. Each warranthas an exercise price of $2.54, was exercisable six months after the date of issuance and will expire five years from the date of issuance.In September 2016, the Company issued warrants to purchase 6,029,174 shares of its common stock for an exercise price of $4.50 per share toinvestors in connection with a private placement of convertible debt securities as more fully discussed in Note 7. The warrants are exercisable at any timebeginning six months after issuance through five years after issuance. The Company classified these warrants as liabilities based on the guidance in ASC 480,as the warrants contain a provision that could result in the Company’s redemption of the warrants outside its control for cash equal to the value of thewarrants calculated using a Black-Scholes option pricing model.F 20Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 8. Warrants (continued)The following table summarizes the Company’s outstanding warrants to purchase common stock as of: Number of Warrants December 31, 2017 December 31, 2016 Exercise Price Expiration DatesLiability-classified Warrants Issued in Series E Preferred Stock offering (1)— 71,430 $2.10 Dec 2017Issued with June 2012 Convertible Notes375,194 375,194 $7.50 Jun 2018Issued in Series E Preferred Stock offering523,045 523,045 $22.50 Dec 2018Issued with September 2016 Convertible Notes6,029,174 6,029,174 $4.50 Sep 2021 6,927,413 6,998,843 Equity-classified Warrants Issued in 2017 Series A Preferred Stock Offering3,437,334 — $2.54 Mar 2022Issued in 2017 Common Stock Offering - common warrants14,046,950 — $0.77 Dec 2022Issued in 2017 Common Stock Offering - underwriter warrants436,364 — $0.9625 Dec 2022Issued in 2017 Common Stock Offering - pre-funded warrants2,416,104 — $0.01 No exp 20,336,752 — Total outstanding warrants27,264,165 6,998,843 (1)As a result of the anti-dilution provisions contained in the warrants, the exercise price for warrants issued in connection with the Company’s Series E Preferred Stockoffering was decreased from $2.50 per warrant share to $2.10 and from $2.10 per warrant share to $0.77 and the number of warrant shares was increased by 154,288 and80,197 during 2016 and 2017, respectively.The table below is a summary of the Company’s warrant activity for the year ended December 31, 2017. Number of warrants Liability-classified Equity-classified Total Weighted-averageexercise priceOutstanding at December 31, 20166,998,843 — 6,998,843 $5.98Granted— 23,842,856 23,842,856 0.84Adjustments (1)80,197 80,197 0.77Exercised(25,000) (3,506,104) (3,531,104) 0.02Expired(126,627) — (126,627) 0.77Outstanding at December 31, 20176,927,413 20,336,752 27,264,165 $2.26(1) See footnote 1 above.Accounting for Liability-classified WarrantsThe foregoing warrants are recorded as liabilities at their estimated fair value at the date of issuance, with subsequent changes in estimated fair valuerecorded in warrant revaluation income in the Company’s Consolidated Statement of Operations in each subsequent period. The change in estimated fairvalue of the Company’s warrant liability for the years ended December 31, 2017 and 2016 resulted in non-cash income of $4.9 million and $11.9 million,respectively. Additionally, the warrants are classified as either current or non-current on the Company’s Consolidated Balance Sheet based on theircontractual expiration date. The Company utilizes the Monte Carlo simulation valuation method to value its liability-classified warrants.F 21Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 8. Warrants (continued)Assumptions Used in Determining Fair Value of WarrantsThe estimated fair value of warrants is determined using Level 2 and Level 3 inputs which is further discussed in Note 10. Inherent in the MonteCarlo simulation valuation method are the following assumptions:Volatility. The Company estimates stock price volatility based on the Company’s historical stock price performance over a period of time thatmatches the volume-weighted average expected remaining life of the warrants.Risk-free interest rate. The risk-free interest rate is based on the U.S. Treasury zero-coupon yield curve in effect at the valuation date commensuratewith the expected remaining life assumption.Expected remaining life. The expected life of the warrants is assumed to be equivalent to their remaining contractual term.Dividend rate. The dividend rate is based on the historical rate, which the Company anticipates will remain at zero.Scenarios. The probability of complex features of the warrants being triggered is subjective (no observable inputs or available market data) andbased on internal and external information known to management at the valuation date. Such assumptions include, among other inputs,probabilities related to a change of control and when it might occur as well as probabilities related to a default under the provisions of the Notes andwhen it might occur.Changes to the key assumptions or to the scenarios used in the valuation model, including the probability of key events, such as a change of controltransaction, could have a material impact to the overall valuation of the warrant liability.The following table summarizes the calculated aggregate fair values, along with the assumptions utilized in each calculation: ($ in thousands, except per share data)December 31, 2017 December 31, 2016Calculated aggregate value$1,073 $6,034Weighted average exercise price per share$6.02 $5.98Closing price per share of common stock$0.64 $1.89Volatility92.2% 85.6%Weighted average remaining expected life3 years, 4 months 4 years, 3 monthsRisk-free interest rate2.00% 1.75%Dividend yield— —Note 9. EquityPreferred StockThe Company is authorized to issue 5,000,000 shares of preferred stock, at a par value of $0.001 per share, in one or more series and to fix the rights,preferences, privileges, and restrictions thereof. These rights, preferences and privileges could include dividend rights, conversion rights, voting rights, termsof redemption, liquidation preferences, sinking fund terms and the number of shares constituting any series or the designation of such series, any or all ofwhich may be greater than the rights of common stock. The issuance of the Company’s preferred stock could adversely affect the voting power of holders ofcommon stock and the likelihood that such holders will receive dividend payments and payments upon liquidation. In addition, the issuance of preferredstock could have the effect of delaying, deferring or preventing a change of control of the Company or other corporate action.Series A Convertible Preferred StockIn March 2017, the Board authorized the issuance of 8,000 shares of preferred stock designated as Series A Convertible Preferred Stock (the Series APreferred Stock). The rights, preferences and privileges of the Series A PreferredF 22Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 9. Equity (continued)Stock is set forth in the Certificate of Designation of Preferences, Rights and Limitations of Series A Convertible Preferred Stock dated March 7, 2017(Certificate of Designation).On March 7, 2017, the Company entered into a securities purchase agreement with certain of its existing accredited investors pursuant to which theCompany agreed to sell a total of 8,000 units (the Units) for a purchase price of $1,000 per Unit, with each Unit consisting of (i) one share of the Company’sSeries A Preferred Stock, with an initial stated value of $1,000 and is convertible into 429 shares of the Company’s common stock with a conversion price of$2.3271 and (ii) a warrant to purchase up to the number of shares of common stock equal to 100% of the conversion shares issuable on March 7, 2017pursuant to the shares of Series A Preferred Stock purchased by each investor (collectively, the 2017 Series A Preferred Stock Offering). The 2017 Series APreferred Stock Offering closed on March 8, 2017 and resulted in gross proceeds of $8.0 million, before deducting offering costs. The proceeds from the 2017 Series A Preferred Stock Offering (including offering costs) were allocated between the Series A Warrants and Series APreferred Stock issued in the transaction based upon their respective fair values using the relative fair value (proportional) method. The fair value of the SeriesA Preferred Stock issued was calculated as the sum of (i) the value of the Series A Preferred Stock as if it had been converted into common stock on theissuance date and (ii) the value of a perpetual annuity paying a 4% dividend rate in conversion shares for five years and 8% thereafter. In connection with thevaluation, the following assumptions were used: risk free interest rate of 3.15%, credit spread of 31.27% and a market yield of 34.42%. The application of therelative fair value method resulted in an allocation of gross proceeds to the Series A Preferred Stock of approximately $1.3 million, net of discounts of $3.0million attributed to the warrants (See Note 5) and $3.7 million from a beneficial conversion feature. The discount attributed to the beneficial conversionfeature was immediately amortized as the Series A Preferred Stock has no stated redemption date and is convertible at the issuance date. For the year endedDecember 31, 2017, the Company recognized approximately $4.1 million of amortization of the discount on the Series A Preferred Stock as deemeddividends charged to additional paid-in capital (in the absence of retained earnings). The value of the beneficial conversion feature is calculated as thedifference between the effective conversion price of the Series A Preferred Stock and the fair market value of the common stock into which the Series APreferred Stock are convertible at the commitment date.The discount attributed to the warrants is being accreted using the effective interest method and charged as a deemed dividend to additional paid-capital (in the absence of retained earnings), over the five-year period of the Series A Preferred Stock in which the stated dividend rate is 4%. For the yearended December 31, 2017 the Company recognized approximately$0.3 million in deemed dividends due to the accretion of the warrant discount.The 2017 Series A Preferred Stock Offering securities purchase agreement contains customary representations, warranties, and agreements by theCompany. The securities purchase agreement also contains customary prohibitions on certain Company payments, the incurrence of certain senior and paripassu debt, certain affiliate transactions and the incurrence of certain liens.Holders of the Series A Preferred Stock are entitled to receive cumulative dividends at a rate per share of 4% per annum (with such dividend rateincreasing to 8% per annum on the five-year anniversary of the original issuance of the Series A Preferred Stock), with such dividends compounded quarterlyand payable only by way by increasing the stated value of the Series A Preferred Stock in accordance with the terms of the Certificate of Designation. For theyear ended December 31, 2017 cumulative dividends paid in-kind to holders of the Series A Preferred Stock were approximately $0.3 million.Shares of Series A Preferred Stock generally have no voting rights, except as required by law; provided, however, that without the prior writtenconsent of the holders of at least 70% of the then outstanding shares of Series A Preferred Stock, the Company may not: (i) alter or change adversely thepowers, preferences or rights given to the Series A Preferred Stock or alter or amend the Certificate of Designation; (ii) amend the Company’s certificate ofincorporation or other charter documents in any manner that adversely affects any rights of a holder of the Series A Preferred Stock; (iii) authorize or createany class of stock ranking as to redemption, distribution of assets upon liquidation or dividends senior to, or otherwise pari passu with, the Series A PreferredStock; (iv) declare or make any dividends other than dividend payments or other distributions payable solely in the Common Stock; or (v) enter into anyagreement with respect to any of the foregoing.Upon a liquidation, dissolution or winding up of the Company, the holders of the Series A Preferred Stock are entitled to receive out of theCompany’s assets, whether capital or surplus, an amount equal to such holder’s then stated value for each share of Series A Preferred Stock before anydistribution to the holders of the Common Stock, any class or series of preferred stock and all other Common Stock equivalents other than those securitieswhich are explicitly senior or pari passu to the SeriesF 23Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 9. Equity (continued)A Preferred Stock in redemption, distribution of assets upon a liquidation or dividends. If there are insufficient assets to pay in full such amounts, then theavailable assets will be ratably distributed to the holders of the Series A Preferred Stock in accordance with the respective amounts that would be payable onsuch shares if all amounts payable thereon were paid in full.Common StockIn July 2016, the Company amended its Restated Certificate of Incorporation, as amended, to increase the number of shares of common stock thatthe Company is authorized to issue from 100,000,000 to 150,000,000.On March 10, 2017, the Company implemented a one-for-three reverse split of its issued and outstanding shares of common stock (the Reverse StockSplit), as authorized at a special meeting of stockholders on March 1, 2017. The Reverse Stock Split became effective on March 10, 2017 at 5:00 pm and theCompany’s common stock began trading on The Nasdaq Capital Market on a post-split basis at the open of business on March 13, 2017. As of a result of theReverse Stock Split, every three shares of the Company’s issued and outstanding common stock were combined into one share of its common stock, except tothe extent that the Reverse Stock Split resulted in any of the Company’s stockholders owning a fractional share, which was rounded up to the next highestwhole share. In connection with the Reverse Stock Split, there was no change in the nominal par value per share of $0.001. The Reverse Stock Split waseffectuated in order to increase the per share trading price of the Company’s common stock to satisfy the $1.00 minimum bid price requirement for continuedlisting on The Nasdaq Capital Market. By letter dated March 27, 2017, The Nasdaq Capital Market Listing Qualification Department, confirmed that theCompany’s common stock was in compliance with listing requirements.December 2017 Public OfferingOn December 7, 2017, the Company entered into an underwriting agreement (the “Underwriting Agreement”) with H.C. Wainwright & Co., LLC(“HCW”), relating to the sale of 7,714,156 shares of its common stock, par value $0.001 per share (the “Common Stock”), pre-funded warrants to purchase anaggregate of 5,922,208 shares of common stock and common warrants to purchase up to an aggregate of 13,636,364 shares of common stock (the “Offering”).Each share of common stock or pre-funded warrant, as applicable, was sold together with a common warrant to purchase one share of common stock at acombined effective price to the public of $0.77 per share and accompanying common warrant. At December 31, 2017, 3,506,104 of the pre-funded warrantshad been exercised and converted to shares of common stock.Pursuant to the HCW Underwriting Agreement, the Company granted HCW a thirty day option, which option ended on January 6, 2018, to purchaseup to 2,045,454 additional shares of Common Stock at a purchase price of $0.76 per share and/or common warrants to purchase up to an aggregate of2,045,454 shares of Common Stock at a purchase price of $0.01 per common warrant with an exercise price of $0.77 per share, less the underwriting discountsand commissions. On December 8, 2017, HCW partially exercised this option by purchasing common warrants to purchase 410,586 shares of common stock.As additional compensation, the Company issued warrants to HCW to purchase 436,364 shares of common stock (the “Underwriter Warrants”). TheUnderwriter Warrants have an exercise price of $0.9625 per share, will be exercisable for five years from the date of the HCW Underwriting Agreement andmay be exercised on a cashless basis in certain circumstances specified therein. The Company and HCW completed the Offering on December 11, 2017, resulting in approximately $9.3 million of net proceeds to the Companyafter deducting the underwriter’s discounts and commissions and other estimated offering expenses payable by the Company.The common warrants are exercisable immediately at an exercise price of $0.77 per share and will expire five years from the date of issuance. Thepre-funded warrants are exercisable immediately at an exercise price of $0.01 per share and may be exercised until they are exercised in full. The UnderwriterWarrants have an exercise price of $0.9625 per share and will expire five years from the date of the HCW Underwriting Agreement. The exercise price andnumber of shares of Common Stock issuable upon exercise of the common warrants, pre-funded warrants and Underwriter Warrants will be subject toadjustment in the event of any stock split, reverse stock split, stock dividend, recapitalization, reorganization or similar transaction, among other events asdescribed in the common warrants and pre-funded warrants.In the event of certain transactions involving a sale of the Company, each holder of common warrants has the right, exercisable at its option, torequire the Company to purchase such holder’s common warrants at a price determined using a BlackScholes option pricing model as described in thecommon warrants. The shares of Common Stock or pre-funded warrants,F 24Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 9. Equity (continued)as applicable, and the accompanying common warrants could only be purchased together in this Offering but were issued separately.F 25Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial StatementsNote 10. Fair Value MeasurementsAssets and Liabilities Measured at Fair Value on a Recurring BasisThe Company follows the guidance in ASC 820, Fair Value Measurement, to account for financial assets and liabilities measured on a recurringbasis. Fair value is the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at themeasurement date. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use inpricing an asset or liability. The Company uses a fair value hierarchy, which distinguishes between assumptions based on market data (observable inputs) andan entity’s own assumptions (unobservable inputs). The guidance requires fair value measurements be classified and disclosed in one of the following threecategories:•Level 1: Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities;•Level 2: Quoted prices in markets that are not active or inputs which are observable, either directly or indirectly, for substantially the full termof the asset or liability;•Level 3: Prices or valuation techniques that require inputs that are both significant to the fair value measurement and unobservable (i.e.,supported by little or no market activity). Determining which category an asset or liability falls within the hierarchy requires significant judgment. The Company evaluates its hierarchydisclosures each reporting period. There were no transfers between Level 1, 2 and 3 duringeach of the years ended December 31, 2017 and 2016.The following fair value hierarchy table presents information about each major category of the Company’s financial assets and liabilities measuredat fair value on a recurring basis as of December 31, 2017 and 2016: December 31, 2017($ in thousands) Level 1 Level 2 Level 3 TotalAssets: Cash and cash equivalents$14,670 $— $— $14,670Total Assets$14,670 $— $— $14,670Liabilities: Warrant liability$— $— $1,073 $1,073Derivative liability— — 3,136 3,136Total Liabilities$— $— $4,209 $4,209 December 31, 2016($ in thousands) Level 1 Level 2 Level 3 TotalAssets: Cash and cash equivalents$17,515 $— $— $17,515Total Assets$17,515 $— $— $17,515Liabilities: Warrant liability$— $— $6,034 $6,034Derivative liability— — 1,735 1,735Total Liabilities$— $— $7,769 $7,769F 26Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 10. Fair Value Measurements (continued)Changes in Level 3 Liabilities Measured at Fair Value on a Recurring BasisCommon Stock Warrants - Warrant LiabilityThe reconciliation of the Company’s warrant liability measured at fair value on a recurring basis using unobservable inputs (Level 3) is as follows:($ in thousands)Warrant LiabilityBalance at December 31, 2015$8,275Issuance of warrants (3)9,643Expiration of warrants (2)(1,910)Change in fair value of warrant liability(9,974)Balance at December 31, 2016$6,034Exercise of warrants (1)(41)Change in fair value of warrant liability(4,920)Balance at December 31, 2017$1,073(1)Warrants were exercised under the cashless exercise method pursuant to the corresponding warrant agreements. As a result of such exercises, the Company issued 6,941shares of common stock. Consequently, these instruments were no longer classified as liabilities. These common stock warrants were remeasured to their fair value as of theexercise date with the change in fair value recorded to the Company’s Consolidated Statement of Operations. The fair value related to the shares issued in connection with theexercised warrants was reclassified from a liability to additional paid-in capital in the Company’s Consolidated Balance Sheets.(2)Represents the fair value as of the beginning of the year for warrants expiring during the year and has been recorded to warrant revaluation income in the Company’sConsolidated Statement of Operations for the respective year end.(3)Represents the fair value of warrants on the issuance date.The fair value of the warrant liability is based on Level 3 inputs. For this liability, the Company developed its own assumptions that do not haveobservable inputs or available market data to support the fair value. See Note 8 for further discussion of the warrant liability.Bifurcated Compound Derivative - Derivative LiabilityThe reconciliation of the derivative liability measured at fair value on a recurring basis using unobservable inputs(Level 3) was as follows:($ in thousands)Derivative LiabilityBalance at December 31, 2015$— Issuance of convertible notes (1)1,273Change in fair value of derivative liability462Balance at December 31, 2016$1,735 Derivative liability to equity upon note conversion (2)(6) Change in fair value of derivative liability1,407Balance at December 31, 2017$3,136(1)Represents fair value of embedded derivatives on the issuance date.(2)Convertible notes were converted to shares of common stock pursuant to the corresponding convertible note agreements. As a result of such conversions, the Companyissued 24,911 shares of common stock. Consequently, these instruments were no longer classified as liabilities. These embedded derivatives were remeasured to their fairvalue as of the exercise date with the change in fair value recorded to the Company’s Consolidated Statement of Operations. The fair value related to the shares issued inconnection with the converted notes was reclassified from a liability to additional paid-in capital in the Company’s Consolidated Balance Sheets.F 27Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 10. Fair Value Measurements (continued)Effect of the Company’s Stock Price and Volatility Assumptions on the Calculation of Fair Value of Financial Instruments Measured on a RecurringBasisCommon Stock Warrants - Warrant LiabilityThe fair value of the Company’s warrant liability is based on Level 3 inputs. As discussed in Note 8, the Company uses a Monte Carlo simulationvaluation method to value its liability-classified warrants. The determination of fair value as of the reporting date is affected by the Company’s stock price aswell as assumptions regarding a number of subjective variables that do not have observable inputs or available market data to support the fair value. Thesevariables include, but are not limited to, expected stock price volatility over the term of the warrants and the risk-free interest rate. The primary factorsaffecting the fair value of the warrant liability are the Company’s stock price and volatility as well as certain assumptions by the Company as to thelikelihood of provisions to the underlying warrant agreements being triggered. The methods described above and in Note 8 may produce a fair valuecalculation that may not be indicative of net realizable value or reflective of future fair values. Furthermore, while the Company believes its valuationmethod is appropriate and consistent with other market participants, the use of different methodologies or assumptions to determine the fair value couldresult in a different fair value measurement at the reporting date.Bifurcated Compound Derivative - Derivative LiabilityThe fair value of the derivative liability is based on Level 3 inputs. As discussed in Note 7, the Company uses a binomial lattice model to value thecompound embedded derivative bifurcated from the Notes. The determination of fair value as of the reporting date is affected by the Company’s stock priceas well as assumptions regarding a number of subjective variables that do not have observable inputs or available market data to support the fair value. Thesevariables include, but are not limited to, expected stock price volatility, changes in interest rates, assumptions regarding the adjusted conversion prices in theNotes, and early redemption or conversion of the Notes. The methods described above and in Note 7 may produce a fair value calculation that may not beindicative of net realizable value or reflective of future fair values. Furthermore, while the Company believes its valuation method is appropriate andconsistent with other market participants, the use of different methodologies or assumptions to determine the fair value could result in a different fair valuemeasurement at the reporting date.Fair Value of Certain Financial Assets and LiabilitiesThe Company believes that the fair values of its current assets and liabilities approximate their reported carrying amounts. The fair value of the long-term convertible promissory notes was approximately $11.2 million at December 31, 2017, and $13.9 million at December 31, 2016, compared to a carryingvalue of $0, as a result of unamortized debt discounts, for both periods.F 28Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial StatementsNote 11. Stock-Based Compensation2009 Equity Incentive PlanThe Company’s Board of Directors (the Board) adopted the 2009 Equity Incentive Plan (as amended to date, the Plan) effective September 3, 2009. The Plan is intended to further align the interests of the Company and its stockholders with its employees, including its officers, non-employee directors,consultants and advisers by providing equity-based incentives. The Plan allows for the issuance of up to 2,533,333 shares of the Company’s common stock. In addition, as of December 31, 2017, there were 8,334 options outstanding that were issued outside the Plan to consultants in 2013.The types of awards that may be granted under the Plan include options (both non-qualified stock options and incentive stock options), stockappreciation rights, stock awards, stock units, and other stock-based awards. The term of each award is determined by the Compensation Committee of theBoard at the time each award is granted, provided that the term of the option does not exceed ten years. Vesting schedules for stock options vary, butgenerally vest 25% per year, over four years for employee options and on the one-year anniversary date for non-employee director options. The Plan had1,430,736 options available for grant as of December 31, 2017.Accounting for Stock-Based CompensationThe Company recognizes non-cash compensation expense for stock-based awards based on their grant date fair value, determined using the Black-Scholes option-pricing model. During the years ended December 31, 2017 and 2016, the weighted average fair market value of options granted was $2.12and $3.66, respectively.Total stock-based compensation expense recognized using the straight-line attribution method and included in operating expenses in the theCompany’s Consolidated Statements of Operations was approximately $0.3 million and $1.9 million for the years ended December 31, 2017 and 2016,respectively.Assumptions Used in Determining Fair Value of Stock OptionsInherent in the Black-Scholes option-pricing model are the following assumptions:Volatility. The Company estimates stock price volatility based on the Company’s historical stock price performance over a period of time thatmatches the expected term of the stock options.Risk-free interest rate. The risk-free interest rate is based on the U.S. Treasury yield curve in effect at the time of grant commensurate with theexpected term assumption.Expected term. The expected term of stock options granted is based on an estimate of when options will be exercised in the future. The Companyapplied the simplified method of estimating the expected term of the options, described in the SEC’s Staff Accounting Bulletins 107 and 110, ashistorical experience is not indicative of expected behavior in the future. The expected term, calculated under the simplified method, is applied togroups of stock options that have similar contractual terms. Using this method, the expected term is determined using the average of the vestingperiod and the contractual life of the stock options granted.Dividend rate. The dividend rate is based on the historical rate, which the Company anticipates will remain at zero.F 29Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 11. Stock-Based Compensation (continued)The fair market value of the stock options at the date of grant was estimated using the Black-Scholes option-pricing model with the followingweighted average assumptions for the years ended December 31: 2017 2016Expected life (1)5 years, 11 months 6 years, 2 monthsInterest rate1.9% 1.5%Dividend yield— —Volatility88.7% 92.4%(1)The Company uses the simplified method for estimating the stock option term.Stock Option ActivityThe following table summarizes stock option activity for the years ended December 31, 2017 and 2016:($ in thousands, except share and per share data) Number of shares Weighted-average exercise price Weighted-average remaining contractual term (in years) Aggregate intrinsic valueOutstanding at December 31, 2015 1,044,723 $18.69 8 years $1,630Granted 528,479 4.80 Expired (11,840) 11.84 Forfeited (281,983) 8.49 Outstanding at December 31, 2016 1,279,379 $15.16 7 years, 2 months $—Granted 295,000 2.88 Expired (211,773) 21.35 Forfeited (273,093) 9.50 Outstanding at December 31, 2017 (1) 1,089,513 $12.06 7 years, 3 months $—Exercisable at December 31, 2017 665,334 $17.48 6 years, 1 month $— (1)Includes both vested stock options as well as unvested stock options for which the requisite service period has not been rendered but that are expected to vest based onachievement of a service condition.The total fair value of options vested during the years ended December 31, 2017 and 2016 was $0.5 million and $2.4 million, respectively.Additionally, as of December 31, 2017, there was approximately $0.9 million of unrecognized compensation expense related to non-vested stock optionswhich is expected to be recognized over a weighted-average period of 2.0 years.During the years ended December 31, 2017 and December 31, 2016, there were no exercises of vested stock options.F 30Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial StatementsNote 12. Restructuring CostsIn June 2016, the Company determined to wind-down its azficel-T operations at the Company’s Exton, PA facility and to reduce the workforce thatsupports such operations. This decision enables the Company to focus its resources towards development of its gene therapy product candidates.Restructuring-related charges for the year ended December 31, 2016 were comprised of approximately $0.3 million of employee severance andbenefit-related charges and less than $0.1 million of asset impairments. No such charges were incurred during the year ended December 31, 2017.The restructuring and asset impairment activity for the years ended December 31, 2017, and 2016, was as follows:($ in thousands) Employee Severance andBenefits Asset Impairments TotalAccrued restructuring balance as of December 31, 2015 $— $— $—Additional accruals 301 34 335Cash payments (282) — (282)Non-cash settlements — (34) (34)Accrued restructuring balance as of December 31, 2016 19 — 19Cash payments (19) — (19)Accrued restructuring balance as of December 31, 2017 $— $— $—The restructuring-related charges incurred during the year ended December 31, 2016 related to employee severance and benefits resulting from thereduction-in-workforce and the impairment of property and equipment. In connection with the reduction-in-workforce, approximately 50% of the Company’semployees were terminated, primarily in the areas of manufacturing and quality operations. The accrued restructuring balance as of December 31, 2016relates to employee severance and benefits which are expected to be paid in the first quarter of 2017 and is recorded as a current liability within accruedexpenses in the Company’s Consolidated Balance Sheet. Additionally, the Company recognized inventory write-offs in cost of product sales related to thewind-down of its azficel-T (including LAVIV) operations as described in Note 4. Note 13. Income TaxesFibrocell Science, Inc. and Fibrocell Technologies, Inc. file a consolidated U.S. federal income tax return, and file U.S. state income tax returns inseveral jurisdictions as well. In general, the U.S. federal and state income tax returns remain open to examination by taxing authorities for tax years beginningin 2014 to present. However, if and when the Company claims net operating loss (NOL) carryforwards from years prior to 2014 against future taxable income,those losses may be examined by the taxing authorities. The Company’s foreign subsidiaries file income tax returns in their respective jurisdictions.The components of the income tax expense (benefit) related to operations, were as follows: Year ended December 31,($ in thousands)2017 2016U.S. Federal: Current$— $—Deferred— —U.S. State: Current— —Deferred— —Income tax expense (benefit)$— $—F 31Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 13. Income Taxes (continued)The reconciliation between income tax expense (benefit) at the U.S. federal statutory rate and the amount recorded in the accompanyingConsolidated Financial Statements were as follows: Year ended December 31,($ in thousands)2017 2016Tax benefit at U.S. federal statutory rate$(5,684) $(5,353)Increase in domestic valuation allowance5,914 10,162State income taxes benefit before valuation allowance, net of federal benefit561 (1,160)Warrant revaluation income and other financing costs(898) (3,742)Credits(904) (366)Stock-based compensation61 239Return to provision true-ups127 220Capital loss carryforward expiration817 —Impact of federal rate change34,463 —Impact of federal rate change on valuation allowance(34,463) —Other6 —Income tax expense (benefit)$— $—The components of the Company’s net deferred tax assets and liabilities at December 31, 2017 and 2016 were as follows: Year ended December 31,($ in thousands)2017 2016Deferred tax liabilities: Convertible notes$2,821 $4,263Total deferred tax liabilities$2,821 $4,263Deferred tax assets: Loss carryforwards$60,428 $85,263Intangible assets68 117Capital loss carryforward— 852Property and equipment606 1,067License fees4,419 7,776Accrued expenses and other401 549Stock-based compensation2,753 4,059Credits1,436 418Total deferred tax assets before valuation allowance70,111 100,101Less: valuation allowance(67,290) (95,838)Total deferred tax assets$2,821 $4,263Net deferred tax assets$— $—As of December 31, 2017, the Company had generated U.S. net operating loss carryforwards of approximately $237.9 million which expire from2018 to 2037 and U.S. federal R&D credits of $1.4 million which expire from 2033 to 2037. The NOL carryforwards are available to reduce future taxableincome. However, the NOL carryforwards may be, or become subject to, an annual limitation in the event of certain cumulative changes in the ownershipinterest of significant stockholders over a three year period in excess of 50%, as defined under Sections 382 and 383 of the Internal Revenue Code of 1986, asamended, as well as similar state tax provisions. This could limit the amount of NOL’s that the Company can utilize annually to offset future taxable incomeor tax liabilities. The amount of the annual limitation, if any, will be determined based on the value of the Company immediately prior to an ownershipchange. Subsequent ownership changes may further affect the limitation in future years. If and when the Company utilizes the NOL carryforwards in a futureperiod, it will perform an analysis to determine the effect, if any, of these loss limitation rules on the NOL carryforward balances. Additionally, U.S. tax lawslimit the time during which these carryforwards may be applied against future taxes, therefore, the Company may not be able to takeF 32Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 13. Income Taxes (continued)full advantage of these carryforwards for federal income tax purposes. In addition, the Company has NOL carryforwards in certain non-U.S. jurisdictions ofapproximately $0.3 million. However, it is not expected that these non-U.S. loss carryforwards will ever be utilized, so they are not included in thecomponents of deferred taxes listed above. Finally, there are no unremitted earnings in foreign jurisdictions, so no provision for taxes thereupon is required.As the Company has had cumulative losses and there is no assurance of future taxable income, valuation allowances have been recorded to fullyoffset deferred tax assets at December 31, 2017 and 2016. The valuation allowance decreased by $28.5 million in 2017, which was partly due to a $34.4million decrease due to the reduction of the federal corporate tax rate and partly due to a $5.9 million increase primarily due to the impact of the net lossesincurred in 2017. The valuation allowance increased by $5.8 million in 2016, primarily due to the impact of the net losses incurred during that year.As of December 22, 2017, the United States enacted tax reform legislation “known as H.R. 1”, commonly referred to as the “Tax Cuts and Jobs Act”(TCJA or the “Act”), resulting in significant modifications to existing law. Among other changes, the TCJA permanently lowers the corporate federal incometax rate to 21% from the existing maximum rate of 35% effective for tax years beginning after December 31, 2017. As a result of the reduction of thecorporate federal income tax rate to 21%, U.S. GAAP requires companies to revalue their deferred tax assets and deferred tax liabilities as of the date ofenactment, with the resulting tax effects accounted for in the reporting period of enactment. As a result of this revaluation, the Company incurred $34.5million income tax expense in continuing operations with a corresponding reduction in the valuation allowance. Therefore, there was no impact on theCompany’s consolidated statements of operations or statements of financial position from the reduction in the federal tax rate. The other provisions of theTCJA did not have a material impact on the consolidated financial statements.In response to the enactment of the TCJA, the SEC staff issued Staff Accounting Bulletin No. 118 (“SAB 118”), which provides guidance onaccounting for the tax effects of the Act. SAB 118 provides a measurement period that should not extend beyond one year from the Act enactment date forcompanies to complete the accounting under ASC 740. In accordance with SAB 118, the Company has recorded a provisional estimate in these financialstatements for the effect of the corporate tax rate change. The effect of the change in federal corporate tax rate from 35% to 21% is subject to change based onresolution of estimates used in determining the amounts of deferred tax assets and liabilities that were remeasured.Note 14. Related Party TransactionsOverview of Related PartiesThe Company and Intrexon Corporation (Intrexon) are parties to two distinct exclusive channel collaboration agreements, as more fully describedbelow. Pursuant to these agreements, the Company engages Intrexon for support services for the research and development of product candidates coveredunder the respective agreements and reimburses Intrexon for its cost for time and materials for such work. Additionally, the Company’s future commitmentspursuant to the agreements include cash royalties and various developmental and commercial milestone payments as more fully described below.For the years ended December 31, 2017 and 2016, the Company incurred expenses of $5.7 million and $3.7 million, respectively, with Intrexon. Ofthe expenses incurred during the 2017 period, $1.3 million related to direct expenses for work performed by Intrexon and $4.4 million related to pass-throughcosts for work performed under the 2012 ECC. Of the expenses incurred during the 2016 period, $1.2 million related to direct expenses for work performed byIntrexon and $2.5 million related to pass-through costs, both for work performed under the 2012 ECC. As of December 31, 2017 and 2016, the Company had outstanding payables with Intrexon of $2.3 million and $0.9 million, respectively. Inconnection with the 2015 ECC, in consideration for the license and the other rights that the Company receives under the agreement, the Company paidIntrexon an up-front technology access fee of $10 million in cash in January 2016.In the second quarter of 2017, Intrexon notified the Company that it had received invoices for approximately $1.1 million in charges from a vendorwho provides services to Intrexon and which are passed-through to the Company under the 2012 ECC. Additional charges have been presented since thesecond quarter, and the total of disputed charges at December 31, 2017, was approximately $1.4 million. Intrexon is disputing the volume and nature of thesecharges and has not invoiced theF 33Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 14. Related Party Transactions (continued)Company for these charges as of December 31, 2017. The Company has recorded approximately $0.7 million of such charges as its best estimate of theamount owed.Randal J. Kirk is the chairman of the board and chief executive officer of Intrexon and, together with his affiliates, owns more than 50% of Intrexon’scommon stock. Affiliates of Randal J. Kirk (including Intrexon) own approximately 29% of the Company’s common stock. Additionally, two of theCompany’s directors, Julian Kirk (who is the son of Randal J. Kirk) and Marcus Smith, are employees of Third Security, LLC, which is an affiliate of Randal J.Kirk.Affiliates of Randal J. Kirk (including Intrexon) participated in the Company’s private placement of convertible debt securities in September 2016,more fully described in Note 7, and were issued an aggregate of $6,762,500 in principal of Notes and accompanying Warrants to purchase an aggregate of2,254,168 shares of common stock. Affiliates of Randal J. Kirk (including Intrexon) participated in the Company’s March 2017 Series A ConvertiblePreferred Stock offering, more fully described in Note 9, and were issued an aggregate of 3,016 shares of convertible preferred stock and accompanyingwarrants to purchase 1,295,875 shares of common stock. Additionally, affiliates of Randal J. Kirk (including Intrexon) participated in the Company’sDecember 2017 public offering, and were issued an aggregate of 2,727,273 shares of common stock and accompanying warrants to purchase 2,727,273 sharesof common stock.Intrexon Collaboration - 2012 ECCIn October 2012, the Company entered into an Exclusive Channel Collaboration Agreement with Intrexon which was amended in June 2013 andJanuary 2014 (as amended, the 2012 ECC) pursuant to which the Company is Intrexon’s exclusive channel collaborator in the research, development andcommercialization of products in the following fields (the 2012 Fields):•the enhanced production and purification of autologous fibroblasts, without gene therapy, for all aesthetic and therapeutic indications;•the enhanced production and purification of autologous dermal cells, without gene therapy, for aesthetic and therapeutic treatment of dermal,vocal cord, and periodontal indications;•the development of our gene therapies applied to autologous fibroblasts for all aesthetic and therapeutic indications;•the development of our gene therapies applied to autologous dermal cells for aesthetic and therapeutic treatment of dermal, vocal cord, andperiodontal indications;•autologous human fibroblasts with gene therapy to express a therapeutic protein and/or bioactive ribonucleic acid for the treatment ofautoimmune and non-infectious inflammatory disorders that manifest in cutaneous tissues, fascia and/or muscle; and•autologous human fibroblasts with gene therapy to express bioactive Tenascin-X locally to correct connective tissue disorders associated withEhlers-Danlos Syndrome (hypermobility type).Pursuant to the terms of the 2012 ECC, Intrexon has granted the Company a license to use its proprietary technologies and other intellectualproperty to research, develop and commercialize products in the 2012 Fields within the United States.The Company is responsible for all costs incurred in connection with the research, development and commercialization of products under the 2012ECC and will own all clinical data, regulatory filings and regulatory approvals relating to such products. The Company engages Intrexon for support servicesfor the research and development of products under the 2012 ECC and reimburses Intrexon for its cost for time and materials for such services.In September 2015, the Company and Intrexon entered into a letter of agreement pursuant to which the parties mutually agreed to terminate theircollaboration with respect to the development of potential therapies to treat Ehlers-Danlos Syndrome (hypermobility type) due to technical hurdles. As aresult, the Company no longer has any rights or obligations under the 2012 ECC with respect to the development of “autologous human fibroblastsgenetically modified to express bioactive Tenascin-X locally to correct connective tissue disorders”.The Company is required to pay Intrexon quarterly cash royalties on all products developed under the 2012 ECC in an amount equal to 7% onaggregate quarterly net sales up to $25 million, plus 14% on aggregate quarterly net sales greater than $25 million. The Company is also required to payIntrexon half of any sublicensing revenues that it receives from third partiesF 34Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 14. Related Party Transactions (continued)in consideration for sublicenses granted by the Company with respect to products developed under the 2012 ECC, but only to the extent such sublicensingrevenues are not included in net sales subject to royalties. Sales from LAVIV (azficel-T), including new indications, or other products that the Companydevelops and commercializes outside of the 2012 ECC are not subject to royalty payments unless the Company is able to reduce the product’s cost of goodssold through the 2012 ECC, in which case, the Company is required to pay quarterly cash royalties on such products equal to one third of such cost of goodssold savings. No royalties have been paid to date in connection with the 2012 ECC.Intrexon Collaboration - 2015 ECCIn December 2015, the Company entered into an additional Exclusive Channel Collaboration Agreement with Intrexon (the 2015 ECC) pursuant towhich the Company is Intrexon’s exclusive channel collaborator in the research, development and commercialization of products for the treatment of chronicinflammatory and degenerative diseases of human joints through intra-articular or other local administration of genetically modified fibroblasts (the 2015Field). Pursuant to the terms of the 2015 ECC, Intrexon has granted the Company a license to use its proprietary technologies and other intellectualproperty to develop and commercialize collaboration products in the 2015 Field throughout the world. The Company is responsible for all costs incurred inconnection with the development and commercialization of collaboration products and will own all clinical data, regulatory filings and regulatory approvalsrelating to such products. The Company engages Intrexon for support services in connection with the research and development of products under the 2015ECC and reimburses Intrexon for its cost for time and materials for such services.In consideration for the license and the other rights that the Company receives under the 2015 ECC, the Company paid Intrexon an up-fronttechnology access fee of $10 million in cash in January 2016. For each collaboration product the Company develops under the 2015 ECC, the Company isrequired to pay Intrexon development milestones of up to $30 million and commercialization milestones of up to $22.5 million, a low double-digit royaltyon its net sales of such products and half of any sublicensing revenues received from third parties for such products. No royalties or milestone payments havebeen paid to date in connection with the 2015 ECC.F 35Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial StatementsNote 15. Loss Per ShareDetails in the computation of basic and diluted loss per share were as follows: For the Year Ended December 31,($ in thousands except share and per share data)2017 2016Loss per share — Basic: Net loss$(16,240) $(15,292)Less: Dividend paid in-kind to preferred stockholders(264) —Less: Deemed dividend on preferred stock(4,099) —Net loss attributable to common stockholders - basic$(20,603) $(15,292) Numerator for basic loss per share$(20,603) $(15,292)Denominator for basic loss per share15,454,199 14,641,528Basic loss per common share$(1.33) $(1.04) Loss per share — Diluted: Numerator for basic loss per share$(20,603) $(15,292)Adjust: Warrant revaluation income for dilutive warrants(34) (1,958)Numerator for diluted loss per share$(20,637) $(17,250) Denominator for basic loss per share15,454,199 14,641,528Plus: Incremental shares underlying “in the money” warrants outstanding5,919 6,006Denominator for diluted loss per share15,460,118 14,647,534Diluted loss per common share$(1.33) $(1.18) The following potentially dilutive securities have been excluded from the computations of diluted weighted-average shares outstanding, as theireffect would be anti-dilutive: For the Year Ended December 31, 2017 2016“In the money” stock options212,494 150,120“Out of the money” stock options769,881 1,218,563“In the money” warrants44,991 17,858“Out of the money” warrants10,529,782 4,382,445Shares underlying convertible notes5,292,853 5,304,533Shares underlying accrued interest on convertible notes203,702 40,137Shares underlying convertible preferred stock3,494,000 —Note 16. Commitments and ContingenciesLeasesOn April 6, 2005, the Company entered into a non-cancellable operating lease (the Lease) for its office, warehouse and laboratory facilities in Exton,Pennsylvania. The lease agreement had an original term of 8 years. On February 17, 2012, the Company entered into an amended and restated lease (theAmended Lease) for an additional term of 10 years through the year 2023. The Lease and the Amended Lease provide for rent payments escalating on aperiodic basis. In accordance with ASC 840-20, Operating Leases, the Company accounts for total minimum payments under the lease on a straight-linebasis over the life of the lease. The difference between actual rent payments and payments accounted for using the straight-line basis are reflected as deferredrent on the Company’s Consolidated Balance Sheets. The Company has the option to renew the lease for an additional 5 years at fair market value. Rentalexpense totaled approximately $1.6 million for both the years ended December 31, 2017 and 2016.F 36Table of ContentsFibrocell Science, Inc.Notes to Consolidated Financial Statements Note 16. Commitments and Contingencies (continued)Collaboration with Related Party (Intrexon)The Company is a party to two separate exclusive channel collaboration agreements with Intrexon, a related party. Pursuant to the agreements, theCompany is Intrexon’s exclusive channel collaborator in the research, development and commercialization of products in certain defined fields. TheCompany is required to pay future royalties, as well as development and commercialization milestones, under these agreements. See Note 14 for additionaldetails.Contractual ObligationsThe following table summarizes the Company’s minimum contractual obligations as of December 31, 2017: Payments due by period($ in thousands)Total 2018 2019 2020 2021 2022 2023 andthereafterOperating lease obligations (1)7,451 1,254 1,416 1,471 1,471 1,471 368Debt obligations (2)21,968 — — — 21,968 — —Total (3)$29,419 $1,254 $1,416 $1,471 $23,439 $1,471 $368(1)Operating lease obligations are stated based on the Amended Lease agreement for the office, warehouse and laboratory facilities executed in February 2012. (2)Obligations under the Notes issued in connection with the 2016 Private Placement which includes principal and accrued interest through September 7, 2021, based onstated fixed rates, as the Company has elected to accrue interest. The Notes have a maturity date of the earlier of (i) September 7, 2026 and (ii) one-hundred and eighty(180) days after the date on which the Company’s product candidate, FCX-007, is approved by the FDA for the treatment of RDEB. However, each Note holder has theright to require the Company to repay all or any portion of the unpaid principal and accrued interest from time to time on or after September 7, 2021. See details withinNote 7.(3)This table does not include (a) any milestone payments which may become payable to third parties under license agreements as the timing and likelihood of such paymentsare not known, (b) any royalty payments to third parties as the amounts of such payments, timing and/or the likelihood of such payments are not known, and (c) contractsthat are entered into in the ordinary course of business which are not material in the aggregate in any period presented above.F 37Exhibit 21List of SubsidiariesFibrocell Technologies, Inc., a Delaware corporation (wholly owned by Fibrocell Science, Inc.)Isolagen International, S.A., a company organized under the laws of Switzerland (wholly owned by Fibrocell Technologies, Inc.)Exhibit 23 Consent of Independent Registered Public Accounting Firm Fibrocell Science, Inc.Exton, PennsylvaniaWe hereby consent to the incorporation by reference in the Registration Statements on Form S-3 (No. 333-185463, No. 333-185466 and No. 333-209077),Form S-8 (No. 333-172776, No. 333-190650, No. 333-196964 and No. 333-212827) and Form S-1 (No. 333-221375) of our report dated March 19, 2018relating to the financial statements, which appears in this Form 10-K. /s/ PricewaterhouseCoopers Philadelphia, PennsylvaniaMarch 19, 2018Exhibit 31 OFFICER’S CERTIFICATION PURSUANT TOSECTION 302 OF THE SARBANES-OXLEY ACT OF 2002 I, John M. Maslowski, Chief Executive Officer of Fibrocell Science, Inc., certify that: 1. I have reviewed this Annual Report on Form 10-K of Fibrocell Science, Inc.; 2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make thestatements made, in light of the circumstances under which such statements were made not misleading with respect to the period covered by this report; 3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects thefinancial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report; 4. The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined inExchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for theregistrant and have: a)Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, toensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities,particularly during the period in which this report is being prepared;b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under oursupervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for externalpurposes in accordance with generally accepted accounting principles;c)Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about theeffectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; andd)Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recentfiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect,the registrant’s internal control over financial reporting; and5. The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to theregistrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions): a)All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonablylikely to adversely affect the registrant’s ability to record, process, summarize and report financial information; andb)Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal controlover financial reporting. Dated:March 19, 2018By:/s/ John M. Maslowski John M. Maslowski President and Chief Executive Officer (Principal Executive Officer, Principal Financial Officer and PrincipalAccounting Officer)Exhibit 32 CERTIFICATION PURSUANT TO18 U.S.C. SECTION 1350, AS ADOPTED PURSUANT TOSECTION 906 OF THE SARBANES-OXLEY ACT OF 2002 Pursuant to 18 U.S.C. Section 1350, I, John M. Maslowski, Chief Executive Officer of Fibrocell Science, Inc. (the "Company"), hereby certify that, to the bestof my knowledge: i.the Annual Report on Form 10-K of the Company for the year ended December 31, 2017, as filed with the Securities and ExchangeCommission on the date hereof (the "Report") fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of1934, as amended; andii. the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of theCompany. Dated: March 19, 2018 By:/s/ John M. MaslowskiJohn M. MaslowskiPresident and Chief Executive Officer (Principal Executive Officer, Principal Financial Officer and PrincipalAccounting Officer)Fibrocell Science, Inc. A signed original of this written statement required by Section 906 has been provided to Fibrocell Science, Inc. and will be retained by FibrocellScience, Inc. and furnished to the Securities and Exchange Commission or its staff upon request.

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