ANNUAL REPORT
2019
Advancing Critical Care Medicine and Helping Patients
We are Passionate About our Mission to Help Patients
Athersys is a clinical-stage biotechnology company developing novel and proprietary best-in-class
therapies designed to extend and enhance the quality of human life. Our focus is on the treatment of medical
conditions where there is significant clinical need, and we are particularly focused on developing therapies
in the regenerative medicine area.
®
We are developing MultiStem
, a patented, adult-derived “off-the-shelf” stem cell therapy platform, for
disease indications in areas of neurological, inflammatory & immune, and cardiovascular disease areas, as
well as other critical care indications where there is substantial unmet medical need due to the limitations
in standard of care.
Developmental Status of Regenerative Medicine Programs
Inflammatory, Immune & RelatedHSC Transplant / GvHDAcute Respiratory Distress Syndrome Trauma Solid Organ Transplant SupportMACOVIA (Athersys) CardiovascularAcute Myocardial Infarction PVD/PAD/CLI Congestive Heart Failure NeurologicalIschemic Stroke Hemorrhagic Stroke Traumatic Brain Injury Multiple Sclerosis Spinal Cord Injury TREASURE (Healios) MASTERS-2 (Athersys) PreclinicalIND/CTAPhase 1Phase 2Phase 3NDA/BLACommercialONE-BRIDGE (HEALIOS) MATRICS-1 (Athersys) MESSAGE FROM THE CHAIRMAN AND CEO
thousand deaths having occurred globally. We
went from a situation where few people had ever
heard the term “coronavirus”, to having it become
part of our daily vocabulary, as we witness and
experience a global pandemic that is impacting all
of us. Although mitigation strategies are helping,
the number of people infected with the virus is
projected to grow in the weeks and months ahead
as the pandemic continues to spread. Irrespective
of what the actual number eventually becomes,
the economic effects and clinical devastation
have already been profound. Epidemiologists
and global health authorities agree that as of
late January 2020, only a few thousand people
were known to be infected, and the virus was
essentially limited to China. In a few short weeks,
the rapid emergence and spread of this pathogen
changed things in ways that seemed unimaginable.
Not only has COVID-19 threatened the health and
well-being of many people and families around
the world, but it has also had a massive toll on
the global economy, strained the capabilities
of healthcare systems across the globe, and
disrupted daily life for almost everyone.
We truly live in unprecedented times.
2019 Annual Report - 1
Gil Van Bokkelen, Ph.D.
Chairman and Chief Executive Officer
To My Fellow Shareholders,
Our company has been built by a dedicated
team of people that is committed to developing
innovative medicines that have the potential to
help patients in meaningful ways. We intend to
do this by overcoming the limitations of current
treatment approaches in areas where there is a
substantial unmet medical need. In our ongoing
journey, we and our many collaborators have
come to understand and appreciate how our
technology appears to have special relevance in
critical care – where patients have experienced
an event that results in significant disability,
hospitalization, and frequently means the patient
is in the intensive care unit. These types of events
can be devastating for both the patient and their
families.
Limitations of current medical care in some
areas have taken on heightened importance due
to recent events. At the time of this writing, we
have exceeded three million confirmed cases
worldwide of patients that have been infected
with the COVID-19 virus, with over two hundred
“In our view, the unprecedented aspect of
what is going on is the rapid, coordinated
global response to this pandemic – and
the role that science and technological
innovation, expedited regulatory
oversight and policymaking are likely
to have on the accelerated development,
validation and delivery of technologies
that will help us effectively address the
challenges we are facing together.”
critically ill (See Figure 1). By ventilating the
patient, oxygen is forced into the lungs at a higher
concentration than normal to keep the patient
alive – but ventilation also creates stress on the
lung tissue that can result in scarring and fibrosis,
causing long-term or permanent structural
damage.
Though many individuals exposed to COVID-19
will experience only mild to moderate symptoms,
medical experts and health authorities now
recognize that the respiratory virus can induce
intense inflammation in the lungs of many infected
patients. When this occurs, it can cause patients
to become seriously or critically ill, and the most
common cause of death among COVID-19 patients
is ARDS.
Figure 1. Infection in the lungs may lead to fluid build up in the
alveoli of the the lungs.
But the emergence of a viral pathogen that has
caused a terrifying and unexpected pandemic
that has the capacity to reach every corner of
the globe is not the unprecedented phenomenon
that I’m referring to – because history teaches
us that this type of event has happened before,
and it will almost certainly happen again. In our
view, the unprecedented aspect of what is going
on is the rapid, coordinated global response to
this pandemic – and the role that science and
technological innovation, expedited regulatory
oversight and policymaking are likely to have
on the accelerated development, validation
and delivery of technologies that will help us
effectively address the challenges we are facing
together.
When we started doing work in the pulmonary
critical care area several years ago, focused
on treating patients with acute respiratory
distress syndrome (ARDS), we recognized
it is an extremely challenging area clinically.
Unfortunately, despite many prior efforts, ARDS
is a condition where there is no FDA approved
effective medicine, and the only real treatment
option currently available is for the patient to
be ventilated. When the lungs become highly
inflamed, the tiny air sacs in the lungs (called
alveoli) become filled with fluid. This process is
referred to clinically as “edema”, and it prevents
the alveoli from absorbing oxygen normally,
2 2019 Annual Report
and subsequently causes the patient to become
Over several years, we conducted studies
evaluating the potential effects of administering
®
MultiStem
cell therapy in animal models of acute
lung injury and inflammation. We evaluated the
effects of MultiStem administration to human
lungs that had been obtained from organ donors,
which had become highly inflamed and were no
longer eligible for lung transplantation. Each of
these studies showed that MultiStem cell therapy
could help reverse inflammation in the lungs and
help restore proper lung function.
We conducted the MUST-ARDS clinical trial,
which was a randomized, double-blind, placebo-
controlled study designed to evaluate the safety
and potential effectiveness for treating patients
with ARDS. As we now know all too well, this
condition can be caused by viral pathogens like
COVID-19, SARS, MERS, H1N1 or other virulent
forms of influenza, as well as trauma or other
events. As we have previously described, the
results of the trial were exciting – both to us,
clinical collaborators and patient advocacy
groups for ARDS. Not only did the trial meet its
primary safety endpoint, with no evidence of
any treatment-related Serious Adverse Events,
but study investigators observed a consistent
pattern of evidence in key clinical parameters that
suggested benefit among the MultiStem treated
patients, including reduced mortality, rapid
improvement in pulmonary function, meaningful
differences in ventilator-free days and ICU-free
days, and corresponding differences in clinical
biomarkers that provide direct support for the
therapeutic hypothesis. This subsequently led to
our third Fast Track designation from the FDA.
Importantly, the study was also designed to follow
patients out for one year, so that their longer-term
improvement and recovery could be evaluated.
We were focused on more than just assessing
the near-term clinical effects – we wanted to
know whether we could help patients get their
quality of life back. The one-year follow up data
was promising, with MultiStem treated patients
reporting substantial improvement in patient self-
care and independence, and the patient’s quality
of life assessment, compared to placebo patients.
Our primary strategic objective, to develop safe
and more effective treatments for patients where
the standard of care is limited or ineffective for
many, remains intact. We continue to advance
our portfolio of critical care programs across
multiple areas, as we also continue to build and
refine our capabilities. However, the recognition
of the potential relevance of our technology as a
treatment option for COVID-19 induced ARDS,
means some of our objectives have evolved. In
April, our board of directors and the leadership
team made the strategic decision to raise more
capital so we could advance some very important
initiatives, including preparing for and initiating a
large, robustly designed trial to evaluate patients
“Not only did the trial meet its primary safety
endpoint, with no evidence of any treatment-related
Serious Adverse Events, but study investigators
observed a consistent pattern of evidence in key
clinical parameters which suggested benefit among
the MultiStem treated patients, including reduced
mortality, rapid improvement in pulmonary
function, meaningful differences in ventilator-
free days and ICU-free days, and corresponding
differences in clinical biomarkers that provide
direct support for the therapeutic hypothesis.”
2019 Annual Report 3
with COVID-19 induced ARDS. This effort has
become a priority for us, public health authorities,
regulators, and our clinical collaborators. Our
team is working hard to expedite the initiation
of the first clinical sites for this important trial,
which we call the MACOVIA study, and we
continue to pursue support from key institutions
to help us conduct it.
Our partnered programs also continue to progress.
As we were completing our MUST-ARDS study,
our partner in Japan, HEALIOS K.K. (Healios) was
preparing to launch their ARDS trial, ONE BRIDGE,
which they initiated in early 2019. They have
publicly announced that they expect to complete
enrollment for the study this year and in April,
they announced the addition of a small cohort to
examine the treatment of a handful of COVID-19
induced ARDS patients. They are also continuing
to advance their registrational TREASURE trial
for ischemic stroke, which they have stated they
expect to fully enroll in 2020. Both of these trials
are expected to produce results in the coming
months and could put Healios on a path to
accelerated approval in Japan. This would mark a
major milestone for both companies, as we work
toward advancing into commercialization.
4 2019 Annual Report
In the meantime, we continue to advance our lead
program, our ongoing Phase 3 trial for ischemic
stroke, the MASTERS-2 study. We have also
received FDA authorization to initiate our Phase 2
trauma clinical trial, which is another important
step forward. We believe our programs in critical
care have tremendous potential for improving the
standard of clinical care, and we look forward to
the continued advancement of these programs, as
well as the completion of these and other studies.
While the COVID-19 induced chaos introduces
some risks and the protective measures that have
been implemented may slow things down a bit,
we will not allow that to stop us. We remain fully
committed to achieving our goals and delivering
on the objectives we have defined for our
shareholders, and we appreciate your continued
faith and support.
Dr. Gil Van Bokkelen
Chairman & CEO
ANNUAL REPORT
2019
Advancing Critical Care Medicine and Helping Patients
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
_____________________________________________
FORM 10-K
_____________________________________________
(Mark one)
ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES
EXCHANGE ACT OF 1934
For the fiscal year ended December 31, 2019
OR
☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES
EXCHANGE ACT OF 1934
For the transition period from to
Commission file number 001-33876
_____________________________________________
Athersys, Inc.
(Exact name of registrant as specified in its charter)
_____________________________________________
Delaware
(State or other jurisdiction of
incorporation or organization)
3201 Carnegie Avenue,
Cleveland, Ohio
(Address of principal executive offices)
20-4864095
(I.R.S. Employer
Identification No.)
44115-2634
(Zip Code)
Registrant’s telephone number, including area code (216) 431-9900
Securities registered pursuant to Section 12(b) of the Act:
Title of each class
Common Stock, par value $0.001 per share
Trading
Symbol
ATHX
Name of each exchange on which registered
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 ☐ No
Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Securities Exchange Act of
1934. Yes ☐ No
Indicate 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 during
the preceding 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 No ☐
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted 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 such
files). Yes No ☐
Indicate 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 ☐
Non-accelerated filer ☐
Accelerated filer
Smaller reporting company
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 financial accounting 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 Rule 12b-2 of the Act). Yes ☐ No
The aggregate market value at June 28, 2019, the last business day of the registrant’s most recently completed second fiscal quarter, of shares of the registrant’s
common stock (based upon the closing price per share of $1.68 of such stock as quoted on the NASDAQ Capital Market on such date) held by non-affiliates of
the registrant was approximately $218.6 million.
The registrant had 164,146,585 shares of common stock outstanding on March 13, 2020.
Documents Incorporated By Reference.
Part III of this Annual Report on Form 10-K incorporates by reference certain information from the registrant’s definitive proxy statement with respect to the
2020 annual meeting of stockholders.
ATHERSYS, INC.
Unless otherwise stated or the context otherwise indicates, all references in this Annual Report on Form 10-K to “Athersys,”
“us,” “our,” “we” or “the Company” mean Athersys, Inc. and its subsidiaries.
TABLE OF CONTENTS
PART I
Item 1. Business
Item 1A. Risk Factors
Item 1B. Unresolved Staff Comments
Item 2. Properties
Item 3. Legal Proceedings
Item 3A. Information About Our Executive Officers
Item 4. Mine Safety Disclosures
PART II
Item 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity
Securities
Item 6. Selected Financial Data
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
Item 7A. Quantitative and Qualitative Disclosures About Market Risk
Item 8. Financial Statements and Supplementary Data
Item 9. Changes in and Disagreements with Accountants on Accounting and Financial Disclosures
Item 9A. Controls and Procedures
Item 9B. Other Information
Item 10. Directors, Executive Officers and Corporate Governance
Item 11. Executive Compensation
PART III
Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters
Item 13. Certain Relationships and Related Transactions, and Director Independence
Item 14. Principal Accounting Fees and Services
PART IV
Item 15. Exhibits and Financial Statement Schedules
Item 16. Form 10-K Summary
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PART I
ITEM 1.
BUSINESS
We are an international biotechnology company that is focused primarily in the field of regenerative medicine. We are
committed to the discovery and development of best-in-class therapies designed to extend and enhance the quality of human
life and have established a portfolio of therapeutic product development programs to address significant unmet medical needs
in multiple disease areas. Our MultiStem® cell therapy, a patented and proprietary allogeneic stem cell product, is our lead
platform product and is currently in clinical development. Our most advanced program is focused on the treatment of ischemic
stroke, which is currently being evaluated in a registrational trial in Japan, and an ongoing Phase 3 clinical trial in North
America under a Special Protocol Assessment, or SPA, and Europe. Our current clinical development programs are focused on
treating critical care and other conditions where current standard of care is limited or inadequate for many patients. These
represent major areas of clinical need, as well as substantial commercial opportunities.
We believe our MultiStem cell therapy represents a potential breakthrough in the field of regenerative medicine and stem cell
therapy and could be used to treat a range of disease indications. MultiStem treatment has shown the potential to enhance tissue
repair and healing in multiple ways, including reducing inflammatory damage, protecting tissue that is at risk following acute
or ischemic injury, and promoting formation of new blood vessels in regions of ischemic injury. These cells appear to be
responsive to the environment in which they are administered, by homing to sites of injury and/or organs involved in injury
response and providing active disease response, while producing proteins that may provide benefit in both acute and chronic
conditions and regulate other cell types. In contrast to traditional pharmaceutical products or biologics that generally act
through a single biological mechanism of action, MultiStem cell therapy may enhance healing and tissue repair through several
distinct mechanisms acting in parallel, resulting in a more effective therapeutic response.
We believe the therapeutic and commercial potential for MultiStem cell therapy to be very broad, applying to multiple areas of
significant unmet medical need, and we are pursuing opportunities in several potential multi-billion dollar markets. While
traditional pharmaceuticals and biologic therapies typically may be used to treat only a single disease or a narrowly defined set
of related conditions, MultiStem cell therapy may have far broader potential and could be developed in different formulations
and with different delivery approaches to effectively treat a wide range of disease indications.
The MultiStem product under development has the potential to be unique among regenerative medicine approaches because it
has the potential to be manufactured on a large scale, may be administered in an “off-the-shelf” manner with minimal
processing, and has the potential to augment healing by providing biological potency and therapeutic effects that other cell
therapy approaches may not be able to achieve. Additionally, MultiStem treatment has consistently demonstrated good
tolerability in both preclinical and clinical studies. Like conventional drugs and biologics, the product is cleared from the body
over time, which we believe may enhance product safety relative to other types of stem cell therapy. While the product does not
permanently engraft in the patient, the therapeutic effects of treatment with MultiStem cells appear to be durable based on both
clinical and preclinical results.
We have evaluated the use of MultiStem cell therapy as a potential treatment in several disease areas. Working with an
international network of leading investigators and prominent research and clinical institutions, and through our own internal
efforts, we have explored the potential for MultiStem cell therapy to be used as a treatment of acute and chronic forms of
neurological conditions or injury, inflammatory and immune disorders, certain pulmonary conditions, and cardiovascular
disease. At present, we have advanced six MultiStem programs into clinical development, targeting areas of significant medical
need and major commercial market opportunities.
As part of the U.S. Government’s response to the outbreak of the 2019 novel coronavirus, COVID-19, we have held
discussions with and made presentations under the Medical Countermeasures TechWatch program to the Biomedical Advanced
Research and Development Authority, or BARDA, and to the U.S. government interagency COVID-19 Medical
Countermeasures task force led by BARDA that also included other relevant governmental agencies and public health
institutions. As a result of this review, our program involving administration of MultiStem for the treatment of Acute
Respiratory Distress Syndrome, or ARDS, was designated as highly relevant by the Medical Countermeasures TechWatch
3
program. Following infection with COVID-19, or other viruses or pathogens that trigger severe pulmonary inflammation,
ARDS can occur, resulting in significant morbidity or death. Discussions between Athersys and BARDA are continuing
regarding the finalization and implementation of a potential collaboration.
Our lead program in the critical care area is our ongoing Phase 3 clinical trial to evaluate the potential for MultiStem treatment
of patients who have suffered neurological damage from an ischemic stroke. The results from our completed Phase 2 study
demonstrated favorable tolerability for MultiStem, consistent with the results from prior studies. Though the Phase 2 study did
not achieve the primary endpoints for the intent-to-treat population, MultiStem treatment was associated with lower rates of
mortality and life-threatening adverse events, infections and pulmonary events, and a reduction in hospitalization and time in
the intensive care unit, or ICU. In addition, analyses show that patients who received MultiStem treatment earlier in the study’s
treatment window (24 to 36 hours post-stroke, in accordance with the original study protocol) had better recovery in
comparison to placebo. Furthermore, analysis of biomarker data obtained from samples of study subjects indicated that
MultiStem treatment reduced post-stroke inflammation compared to placebo, and the results suggest that this effect was more
pronounced for subjects who received MultiStem earlier within the treatment window. This effect is consistent with our
hypothesis regarding mechanisms of action and related preclinical data, and with the clinical data suggesting faster and
improved recovery for MultiStem-treated patients relative to current standard of care.
The one-year follow-up data from the Phase 2 trial demonstrated that MultiStem-treated subjects on average continued to
improve through one year and had a significantly higher rate of “Excellent Outcome,” as defined below, compared to placebo
subjects at one year when evaluating all of the intent-to-treat subjects enrolled in the study. Achievement of an Excellent
Outcome is important because it means that a patient has substantially improved (i.e., receiving an “Excellent” score in each of
the three clinical rating scales used to assess patient improvement) and has regained the ability to live and function
independently with a high quality of life. The relative improvement in Excellent Outcome was even more pronounced in the
study subjects who received MultiStem treatment within 36 hours of the stroke. If MultiStem cell therapy is proven effective in
our ongoing Phase 3 registrational study and if it receives a marketing authorization from the United States Food and Drug
Administration, or FDA, this treatment window and its favorable administration profile would make this therapy available to
most ischemic stroke patients, in contrast to other therapies (e.g., tissue plasminogen activator, or tPA, or mechanical
thrombectomy), which have shorter treatment windows or are limited to certain patients.
We have a collaboration with HEALIOS K.K., or Healios, to develop and commercialize MultiStem for the treatment of certain
indications in Japan. Healios has a license to our technology and is responsible for the development and commercialization of
MultiStem for ischemic stroke and ARDS in Japan on an exclusive basis, and we are responsible for the supply of clinical
product to Healios. An expansion of our Healios collaboration in June 2018 included, among other things, an exclusive license
to our technology for the development and commercialization of additional indications, including (i) ARDS in Japan as noted
above, (ii) certain ophthalmological indications worldwide, (iii) the treatment of diseases of the liver, kidney, pancreas and
intestinal tissue through administration of our products in combination with cells derived from induced pluripotent stem cells,
or iPSC, in Japan, and (iv) the use of MultiStem cells as part of Healios' organ bud technology on a global basis. In addition to
up-front license fees received under our arrangement with Healios, we receive payments for product supply and other
manufacturing services provided, as well as potential milestone achievement and royalties on net sales that vary among the
licensed indications.
In March 2018, Healios purchased 12,000,000 shares of our common stock and a warrant to purchase additional shares of
common stock for $21.1 million. The warrant for up to 4,000,000 shares is exercisable until September 2020.
We have worked closely with Healios to support their development efforts in Japan. In 2016, the Pharmaceuticals and Medical
Devices Agency, or PMDA, authorized the Clinical Trial Notification, for Healios’ Phase 2/3 trial of MultiStem (HLCM051)
entitled, “Treatment Evaluation of Acute Stroke for Using in Regenerative Cell Elements,” or TREASURE. This clinical trial,
which could lead to registration of the product candidate, is currently ongoing and enrolling patients in Japan. Japan’s
Regenerative Medicine regulatory framework is designed to enable rapid development of qualified regenerative medicine
therapies by providing either conditional or full approval of qualified therapies. Under the new framework, Healios' ischemic
4
stroke program has been awarded the SAKIGAKE designation by the PMDA, which is designed to expedite regulatory review
and development and is analogous to Fast Track designation from the FDA.
While Healios is conducting its TREASURE study, our Phase 3 trial is ongoing in the United States, with plans to expand the
study internationally. Following the completion of our Phase 2 trial, or MASTERS-1, we advanced our development efforts in
North America and Europe by engaging in discussions with the FDA, the European Medicines Agency, or EMA, and other
regulators. We received agreement from the FDA under a SPA for the design and planned analysis of our pivotal Phase 3
clinical study of MultiStem for ischemic stroke entitled, “MultiStem Administration for Stroke Treatment and Enhanced
Recovery Study-2,” or MASTERS-2, meaning that the trial is adequately designed to support a Biologic License Application,
or BLA, submission for registration if it is successful. The FDA also granted us Fast Track designation for our clinical product
for the treatment of ischemic stroke. Such designation for a new biologic product means that the FDA will take such actions as
are appropriate to expedite the development and review of our application to approve the product, and specifically, under Fast
Track designation, the program becomes eligible for rolling submission, accelerated approval and priority review of the BLA,
facilitating a timely regulatory review. This program subsequently received the Regenerative Medicine Advanced Therapy, or
RMAT, designation from the FDA, which was established under the 21st Century Cures Legislation. The RMAT designation
may be obtained for eligible cell therapy and other regenerative medicine and advanced therapies when the FDA agrees that
preliminary clinical evidence indicates that the therapy has demonstrated the potential to effectively address unmet medical
needs for a serious or life-threatening disease or condition. The RMAT designation is the equivalent of the non-regenerative
medicine product's Breakthrough Therapy designation, and designated products benefit from all Breakthrough Therapy
features. The designation enables sponsors to discuss with the FDA multidisciplinary strategic development plans, including
expediting manufacturing development plans for commercialization to support priority review and accelerated approval. The
design of MASTERS-2 has also received a Final Scientific Advice positive opinion from the EMA, representing the EMA’s
agreement that successful results from the trial could result in registration and marketing approval of the MultiStem cell
therapy. This positive opinion provides further alignment among the key regulators regarding potential commercialization of
the MultiStem product upon success of this single pivotal trial.
We believe these designations could accelerate the development, regulatory review and subsequent commercialization of
products like MultiStem cell therapy for ischemic stroke, if future clinical evaluation demonstrates appropriate safety and
therapeutic effectiveness.
In January 2019 and January 2020, we announced summary results and one-year follow-up results, respectively, from our
exploratory clinical study of the intravenous administration of MultiStem cell therapy to treat patients who are suffering from
ARDS. The study results provide further confirmation that the MultiStem treatment was well-tolerated and importantly, there
were lower mortality and a greater number of ventilator-free and ICU-free days in the MultiStem-treated patient group
compared to the placebo group. Average quality-of-life outcomes were higher in the MultiStem group compared to placebo
through one year.
In 2019, Healios initiated a clinical trial in Japan for patients with pneumonia-induced ARDS, which is referred to as the ONE-
BRIDGE study, which is actively enrolling patients. ARDS is a serious immunological and inflammatory condition
characterized by widespread inflammation in the lungs. ARDS can be triggered by pneumonia, sepsis, trauma or other events,
and represents a major cause of morbidity and mortality in the critical care setting. It has significant implications, as it prolongs
ICU and hospital stays and requires convalescence in the hospital and rehabilitation. There are limited interventions and no
effective drug treatments for ARDS, making it an area of high unmet clinical need with high treatment costs. Given the high
treatment costs of ARDS, a successful cell therapy could be expected to generate significant savings for the healthcare system
by reducing days on a ventilator and in the ICU and importantly, could reduce mortality and improve quality of life for those
suffering from the condition. ARDS affects annually approximately 400,000 - 500,000 patients collectively in Europe, the
United States and Japan.
We are preparing with the University of Texas Health Science Center at Houston, or UTHealth, to conduct a clinical trial
evaluating MultiStem cell therapy for early treatment and prevention of complications after severe traumatic injury. This first-
ever study of a cell therapy for treatment of a variety of traumatic injuries is intended to be conducted at Memorial Hermann-
5
Texas Medical Center, one of the busiest Level 1 trauma centers in the United States. The study has grant support from the
Medical Technology Enterprise Consortium and the Memorial Hermann Foundation. We intend to provide the clinical product
for the conduct of the trial, as well as regulatory and operational support. We and UTHealth are working to finalize a plan for
the study and this patient population that is acceptable to the FDA.
In addition to these programs, we had been conducting a Phase 2 clinical study for the administration of MultiStem cell therapy
to patients that have suffered an acute myocardial infarction, or AMI, more commonly referred to as a heart attack. This study
was based in part on the favorable results of our previously completed Phase 1 clinical trial that demonstrated tolerability and
encouraging signs of improvement in heart function among patients that exhibited severely compromised heart function prior to
treatment. However, enrollment in the Phase 2 clinical study was very challenging due in part to changes in the standard of care
for the patients, and which limited the time window to obtain informed consent from eligible patients. Due to these challenges
and the difficulties in addressing them, and priorities in other clinical areas, we elected in 2019 to suspend the study and
determine what has been learned. We will evaluate our development strategy before proceeding further.
Additionally, in a completed Phase 1 clinical study, we evaluated the safety, efficacy and potential for MultiStem cell therapy to
prevent or reduce graft-versus-host disease, or GvHD, and other complications, and to provide supportive care to patients
undergoing a hematopoietic stem cell, or HSC, transplant to treat leukemia or certain other blood-borne cancers. Our
MultiStem cell therapy for GvHD has been designated an orphan drug by both the FDA and the EMA for the prevention of
GvHD, which may provide market exclusivity and other substantial potential incentives and benefits. Also, the MultiStem
product was granted Fast Track designation by the FDA for prophylactic administration to prevent or minimize GvHD
following HSC transplantation. Subsequently, our registration study design received a positive opinion from the EMA through
the Protocol Assessment/Scientific Advice procedure. Furthermore, the proposed Phase 2/3 registration study received a SPA
from the FDA. Based on the results of this study and data from other studies that we and our collaborator have conducted, we
believe that MultiStem has therapeutic relevance in patients that have undergone radiation therapy and hematopoietic
transplantation for the treatment of cancer, or that may have been exposed to radiation from other sources. Given advances in
the treatment of patients with cancer, we have not moved forward with this study. Initiation of this trial would depend on the
progress in other clinical trials, the achievement of certain business development and financial objectives, and the development
and success of alternative treatment options for GvHD that would reduce the need for transplant procedures.
MultiStem cell therapy has been evaluated in other disease areas, such as inflammatory bowel disease with a collaborator, solid
organ transplant in an investigator-sponsored study, and a limited number of compassionate use cases. Our current policy
precludes the administration of MultiStem to patients on a compassionate use basis, primarily for financial and logistical
reasons, although we reserve the right to amend this policy in the future if circumstances warrant.
While development of our clinical programs for human health indications remains our priority, based on our research to date
and work performed at our Belgian subsidiary, ReGenesys BV, or ReGenesys, we are evaluating our cell therapy for use in
treating disease and conditions in the animal health segment. We are actively pursuing partnership opportunities to further
develop this program.
Our development approach has historically involved establishing collaborative relationships with leading research and clinical
centers in the United States and internationally. This has enabled us to advance multiple programs in areas of defined unmet
medical need in a resource efficient manner. Furthermore, by emphasizing the potential application of our technologies in areas
of significant clinical need, we believe we are well positioned to utilize recent regulatory initiatives that are designed to
promote the rapid and cost-effective development of innovative new therapies, and are actively pursuing such initiatives. These
include recent programs in the United States and Europe being implemented by the FDA and the EMA involving existing and
potentially broadened application of accelerated review and approval pathways, as well as the accelerated Regenerative
Medicine regulatory framework in Japan that is designed to enable rapid conditional authorization of qualified regenerative
medicine therapies. We believe such initiatives could accelerate the development and commercialization of products like
MultiStem cell therapy, if clinical results demonstrate appropriate safety and therapeutic effectiveness, thereby increasing
shareholder value. Japan’s Regenerative Medicine regulatory framework enacted in 2014 has already resulted in the
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commercial approval of multiple cell therapy products developed by other companies that we are aware of, along with coverage
and reimbursement of those products, and we and Healios intend to utilize this framework.
In addition to our MultiStem clinical programs, we have other earlier-stage programs targeted at indications with significant
unmet medical needs. We may elect to enter into partnerships to advance the development of these programs, as well as certain
new programs involving MultiStem cell therapy, and continue to evaluate partnering opportunities related to certain programs.
For some programs we may elect to fund further development in specific markets in order to maximize value for our
shareholders.
We were incorporated in Delaware on October 24, 1995. On June 8, 2007, we merged with a wholly owned subsidiary of
BTHC VI, Inc., a Delaware corporation, and on August 31, 2007, BTHC VI, Inc. changed its name to Athersys, Inc.
Business Strategy
Our principal business objective is to discover, develop and commercialize novel therapeutic products for disease indications
that represent significant areas of clinical need and where we believe there is a substantial commercial opportunity. The key
elements of our strategy are outlined below:
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Advance our Lead Programs through Clinical Development to Registration and Commercialization. We are
focused on the design and execution of clinical studies, e.g., ischemic stroke, intended to enable product
registration in major markets. We are also engaged in activities intended to enable effective commercialization,
e.g., preparation for scaled, commercial manufacturing. We may partner with other companies to complete such
development and preparation activities, and to market the product upon regulatory approval.
Efficiently Conduct Clinical Development to Establish Clinical Proof-of-Concept and Biological Activity for
Other Product Candidates. We conduct our clinical studies with the intent to establish safety and efficacy
proof-of-concept and/or evidence of biological activity in a number of important disease areas where our cell
therapies are expected to have benefit, such as we have done with ARDS. Our strategy is to conduct well-
designed studies beginning early in the clinical development process, thus establishing a robust foundation for
later-stage development, partnering activity and expansion into complementary areas. We are committed to a
rigorous clinical and regulatory approach, which we believe has helped us to advance our programs efficiently,
providing high quality, transparent communications and regulatory submissions. Our discussions with the FDA,
EMA and PMDA have resulted in productive interactions and important designations that have helped to
advance our programs efficiently.
Continue to Refine and Improve our Manufacturing and Related Processes and Deepen our Understanding of
Therapeutic Mechanisms of Action. A key aspect of MultiStem cell therapy is the ex vivo expansion capacity of
the cells that comprise the product. This enables large-scale production of the clinical product, which is
associated with greater consistency, specificity and cost of goods advantages over other cell therapies. We are
building on this intrinsic biological advantage by advancing and optimizing our production and process
development approaches, through our internal capabilities and efforts, and working with contract
manufacturers. We are focused on development and optimization of new and proprietary manufacturing
techniques and the pharmacy-to-bedside approach to support late-stage development and commercialization of
the MultiStem product. Additionally, we will continue to refine our understanding of our products’ activities
and mechanisms of action to prepare the foundation for product enhancements and next generation
opportunities.
Enter into Arrangements with Business Partners to Accelerate Development and Create Value. In addition to
our internal development efforts, an important part of our strategy is to work with collaborators and partners to
accelerate product development, reduce our development costs and broaden our commercial access. We have
entered into licensing and collaborative arrangements with qualified partners to achieve these objectives. We
anticipate that this strategy will help us to develop a portfolio of high-quality product development
opportunities, enhance our clinical development and commercialization capabilities and increase our ability to
generate value from our proprietary technologies. Historically, we have entered into licensing arrangements
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with companies such as Healios, Chugai Pharmaceutical Co., Ltd., Pfizer Inc., or Pfizer, Bristol-Myers Squibb
Company, or Bristol-Myers Squibb, Johnson & Johnson, Wyeth Pharmaceuticals Inc. (now part of Pfizer), RTI
Surgical, Inc., or RTI, and others. Licensing partnerships generate revenue and provide capital that helps enable
us to advance our programs further in development.
Efficiently Explore New High Potential Therapeutic Applications, Leveraging Third-Party Research
Collaborations and our Results from Related Areas. Our MultiStem cell therapy has shown promise in many
disease areas, including in treating neurological conditions, cardiovascular disease, inflammatory and immune
disorders, certain pulmonary conditions and other conditions where the current standard of care is limited or
inadequate for many patients. We are committed to exploring potential clinical indications where our therapies
may achieve best-in-class profile and where we believe we can effectively address significant unmet medical
needs. In order to achieve this goal, we established collaborative research relationships with investigators from
many leading research and clinical institutions across the United States and Europe, including the Cleveland
Clinic, Case Western Reserve University, University of Minnesota, the Medical College of Georgia at Augusta
University, the University of Oregon Health Sciences Center, UTHealth, the University of Pittsburgh Medical
Center, the Katholieke Universiteit Leuven, University of Regensburg, and other institutions. Through this
network of collaborations, we have evaluated MultiStem cell therapy in a range of preclinical models that
reflect various types of human disease or injury. These collaborative relationships have enabled us to cost
effectively explore where MultiStem cell therapy may have relevance and how it may be utilized to advance
treatment over current standard of care. Additionally, we have shown that we can leverage clinical safety data
and preclinical results from some programs to support accelerated clinical development efforts in other areas,
saving substantial development time and resources compared to traditional drug development where each
program is separately developed.
Continue to Expand our Intellectual Property Portfolio. We have a broad intellectual property estate that covers
our proprietary products and technologies, as well as methods of production and methods of use. Our
intellectual property is important to our business and we take significant steps to protect its value. We have
ongoing research and development efforts, both through internal activities and through collaborative research
activities with others, which aim to develop new technologies, applications and intellectual property and enable
us to file patent applications that cover new applications of our existing technologies or product candidates,
including MultiStem cell therapy and other opportunities. We currently have over 350 patents related to our
technologies, providing protection in the United States, Europe, Japan and other areas.
Our Current Programs
By applying our proprietary MultiStem cell therapy product, we established therapeutic product development programs treating
neurological conditions, cardiovascular disease, inflammatory and immune disorders, certain pulmonary conditions,
cardiovascular disease, and other conditions where the current standard of care is limited or inadequate for many patients. Our
lead programs are focused in the critical care area, with treatment provided in hospitals often in intensive care situations. Our
programs in the clinical development stage include the following:
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Ischemic Stroke: We are conducting a pivotal Phase 3 clinical trial of MultiStem cell therapy for the treatment of
ischemic stroke, referred to as MASTERS-2. We initiated the study with a small number of high-enrolling sites and
are bringing on additional sites over time in line with clinical product supply and clinical operations objectives. The
MASTERS-2 study has received several regulatory distinctions including SPA, Fast Track designation and RMAT
from the FDA, as well as a Final Scientific Advice positive opinion from the EMA, described further below. We
believe these designations could accelerate the development, regulatory review and subsequent commercialization of
products like MultiStem cell therapy for ischemic stroke, if future clinical evaluation demonstrates appropriate safety
and therapeutic effectiveness.
We received agreement from the FDA under a SPA for the design and planned analysis of our MASTERS-2
pivotal Phase 3 trial. The SPA provides agreement from the FDA that the protocol design, clinical endpoints, planned
conduct and statistical analyses encompassed in MASTERS-2 are sufficient to meet the objectives in support of a
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regulatory submission for approval of the MultiStem product for treating ischemic stroke patients if the trial is
successful. The FDA has also granted us Fast Track designation for our clinical product for the treatment of ischemic
stroke. Such designation for a new biologic product means that the FDA will take such actions as are appropriate to
expedite the development and review of our application to approve the product, and specifically, under Fast Track
designation, the program becomes eligible for rolling submission, accelerated approval and priority review of the BLA
facilitating a timely regulatory review. The design of MASTERS-2 has also received a Final Scientific Advice positive
opinion from the EMA, representing the EMA’s agreement that successful results from the trial could result in
registration and marketing approval of the MultiStem cell therapy. This positive opinion provides further alignment
among the key regulators regarding potential commercialization of the MultiStem product upon success of this single
pivotal trial. We subsequently received RMAT designation from the FDA, which was established under the 21st
Century Cures Act. The RMAT designation may be obtained for eligible cell therapy and other regenerative medicine
and advanced therapies when the FDA agrees that preliminary clinical evidence indicates that the therapy has
demonstrated the potential to effectively address unmet medical needs for a serious or life-threatening disease or
condition. The RMAT designation is the equivalent of the non-regenerative medicine product's Breakthrough Therapy
designation, and designated products benefit from all Breakthrough Therapy features. The designation enables
sponsors to discuss with the FDA multidisciplinary strategic development plans, including expediting manufacturing
development plans for commercialization to support priority review and accelerated approval.
Our MASTERS-2 clinical trial is a randomized, double-blind, placebo-controlled clinical trial designed to
enroll 300 patients in North America, Europe and certain other international locations who have suffered moderate to
moderate-severe ischemic stroke. The enrolled subjects are receiving either a single intravenous dose of MultiStem
cell therapy or placebo, administered within 18-36 hours of the occurrence of the stroke, in addition to the standard of
care. The primary endpoint will evaluate disability using modified Rankin Scale, or mRS, scores at three months,
comparing the distribution, or the “shift,” between the MultiStem treatment and placebo groups. The mRS shift
analyzes patient improvement across the full disability spectrum, enabling recognition of improvements in disability
and differences in mortality and other serious outcomes among strokes of different severities. The study will also
assess Excellent Outcome (the achievement of mRS ≤1, NIHSS ≤1, and Barthel Index ≥95) at three months and one
year as key secondary endpoints. Additionally, the study will consider other measures of functional recovery,
biomarker data and clinical outcomes, including hospitalization, mortality and life-threatening adverse events, and
post-stroke complications such as infection.
Healios’ ongoing TREASURE study in Japan is being conducted at hospitals in Japan that have extensive
experience in providing care for stroke victims. Enrolled subjects are receiving either a single dose of MultiStem or
placebo, administered within 18-36 hours of the occurrence of the stroke, in addition to standard of care in these 220
patients, randomized, double-blind, placebo-controlled trial. The study will evaluate patient recovery through
approximately 90 days and at one year following initial treatment based on Excellent Outcome and other neurological,
functional and clinical endpoints. The trial could lead to registration under Japan’s Regenerative Medicine regulatory
framework, which is designed to enable rapid development of qualified regenerative medicine therapies by providing
either conditional or full approval of qualified therapies. Under the new framework, Healios' ischemic stroke program
has been awarded the SAKIGAKE designation by the PMDA, which is designed to expedite regulatory review and
approval, and is analogous to Fast Track designation from the FDA. We look forward to completing both the
MASTERS-2 and TREASURE trials and using the accelerated pathway afforded to us by the regulators in the United
States, Europe and Japan upon study completion.
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ARDS: In January 2019 and January 2020, we announced summary results and one-year follow-up results,
respectively, from our exploratory clinical study of the intravenous administration of MultiStem cell therapy to treat
patients who are suffering from ARDS. The study results provide further confirmation of tolerability and importantly,
there were lower mortality and a greater number of ventilator-free and ICU-free days in the MultiStem-treated patient
group compared to the placebo group. Average quality-of-life outcomes were higher in the MultiStem group compared
to placebo through one year. Furthermore, analysis of initial biomarker data reflects lower levels of inflammatory
markers/cytokines following MultiStem treatment, an expected mechanism of action in this patient population. Based
on the promising results, Athersys is planning for a registrational trial in this indication. In 2019, Healios initiated a
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clinical trial in Japan for patients with pneumonia-induced ARDS, referred to as the ONE-BRIDGE study, which is
actively enrolling patients. We look forward to the results of this study.
Trauma: We are preparing with UTHealth to conduct a clinical trial evaluating MultiStem cell therapy for early
treatment and prevention of complications after severe traumatic injury. This first-ever study of a cell therapy for
treatment of a wide range of traumatic injuries is intended to be conducted at Memorial Hermann-Texas Medical
Center, one of the busiest Level 1 trauma centers in the United States. The study has grant support from the Medical
Technology Enterprise Consortium and the Memorial Hermann Foundation. We intend to provide the clinical product
for the conduct of the trial, as well as regulatory and operational support. We and UTHealth are working to finalize a
plan for the study and this patient population that is acceptable to the FDA.
AMI: We were conducting a Phase 2 clinical study for the administration of MultiStem cell therapy to patients that
have suffered a heart attack. This study was based in part on the favor results of our previously completed Phase 1
clinical study that demonstrated that the MultiStem therapy was well tolerated and showed encouraging signs of
improvement in heart function among patients that exhibited severely compromised heart function prior to treatment.
This data was published in a leading peer reviewed scientific journal, and one-year follow-up data suggested that the
benefit observed was sustained over time. However, enrollment in the Phase 2 clinical study was very challenging due
in part to changes in the standard of care for the patients. Due to these challenges and the difficulties in addressing
them, and priorities in other clinical areas, we elected in 2019 to suspend the study and determine what has been
learned. We will evaluate our development strategy before proceeding further.
HSC Transplant / GvHD: Currently, this program is staged for future registration-directed development, which
depends on the success and impact of potential alternative therapies for treating the underlying conditions leading to
transplant, as well as other business and financial considerations. Following our completed Phase 1 clinical study of
the administration of MultiStem cell therapy to patients suffering from leukemia or certain other blood-borne cancers,
in which patients undergo radiation therapy and then receive a HSC transplant, we were granted orphan drug
designation by the FDA and the EMA for MultiStem treatment in the prevention of GvHD, and the MultiStem product
was granted Fast Track designation by the FDA for prophylaxis therapy against GvHD following HSC transplantation.
Subsequently, our registration study design received a positive Scientific Advice opinion from the EMA and a SPA
designation from the FDA, and we may pursue this study at the appropriate time.
While development of our clinical programs for human health indications remains our priority, based on our research to date
and work performed at our wholly-owned subsidiary, ReGenesys, we are also evaluating our cell therapy for use in treating
diseases and conditions in the animal health area. We have demonstrated in preclinical animal health models that our cell
therapy can promote tissue repair and healing that could provide meaningful benefits to animal patients, including those
suffering from conditions with unmet medical need. We are actively pursuing partnership opportunities to further develop this
program.
We are engaged in preclinical development and evaluation of MultiStem cell therapy in other indications for human health, as
well as certain indications in the animal health field, and we conduct such work both through our own internal research efforts
and through a broad global network of collaborators. We also engage in discussions with third parties about collaborating in the
development of MultiStem cell therapy for various programs and/or various geographic territories and may enter into one or
more business partnerships to advance these programs over time. We may also elect to develop certain programs independently.
While the MultiStem product platform continues to advance, we are engaged in process development initiatives intended to
increase manufacturing scale, reduce production costs, and enhance process controls and product quality, among other things.
These initiatives are being conducted both internally and outsourced to select contractors, and the related investments are meant
to enable us to meet potential commercial demand in the event of eventual regulatory approval. Until such time as we can
manufacture products ourselves in accordance with good manufacturing practices, we will continue to rely on third-party
manufacturers to make our MultiStem product for clinical trials and eventual commercial sales.
We have a collaboration with Healios that covers MultiStem cell therapy for ischemic stroke and ARDS in Japan and the use of
our technology for Healios’ organ bud program targeted to liver disease and other indications, as well as certain other rights,
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including a license for the use of our MultiStem product to treat certain ophthalmological indications and a license to treat
diseases of the liver, kidney, pancreas and intestinal tissue through administration of our products in combination with iPSC-
derived cells. We provide manufacturing services and supply Healios with clinical product for the licensed indications, and in
the event that we fail to perform our responsibilities to supply product to Healios, then under certain circumstances, we may be
required to grant Healios a license to make the product solely for use in its licensed fields and territories.
Regenerative Medicine Programs
MultiStem — A Novel Therapeutic Modality
We are developing our MultiStem cell therapy, a proprietary non-embryonic, allogeneic stem cell product candidate, that we
believe has potential utility for treating a broad range of diseases and could have widespread application in the field of
regenerative medicine. Unlike traditional bone marrow transplants or other stem cell therapies, MultiStem cells may be
manufactured on a large scale and may be administered without tissue matching or the need for immune suppression, analogous
to type O blood. Potential applications of MultiStem cell therapy include the treatment of critical care indications, neurological
conditions, cardiovascular disease, inflammatory and immune disorders, certain pulmonary conditions and other conditions
where the current standard of care is limited or inadequate for many patients. We believe that MultiStem cell therapy represents
a significant advancement in the field of stem cell therapy. We currently have open Investigational New Drug Applications, or
INDs, for the study of MultiStem administration in distinct clinical indications, and multiple clinical trials are ongoing.
MultiStem cell therapy is a patented biologic product that is manufactured from human stem cells obtained from adult bone
marrow, although these cells may alternatively be obtained from other tissue sources. The product consists of a special class of
human stem cells that have the ability to express a range of therapeutically relevant proteins and other factors, as well as form
multiple cell types. Factors expressed by the cells have the potential to deliver a therapeutic benefit in several ways, such as the
reduction of inflammation, regulation of immune system function, protection of damaged or injured tissue, the formation of
new blood vessels in regions of ischemic injury and augmentation of tissue repair and healing in other ways. Stability studies
have demonstrated that these cells may be stored for an extended period of time in frozen form and are straightforward to
prepare and administer, resulting in an "off-the-shelf" profile. Following administration, the cells have been shown to express
multiple therapeutically relevant proteins, but unlike a traditional transplant, are subsequently cleared from the body over time,
analogous to a drug or other biologics.
We believe that MultiStem represents a potential best-in-class stem cell therapy because it exhibits each of the following
characteristics based on research and development conducted to date:
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Broad plasticity and multiple potential mechanisms of action. MultiStem cells have a demonstrated ability in
animal models to deliver therapeutic benefit by producing factors that protect tissues against damage and
inflammation, as well as enhancing or playing a direct role in revascularization or tissue regeneration, and have
also shown the capacity to form a range of other cell types.
Large-scale production. Unlike conventional stem cells, such as blood-forming or HSCs, mesenchymal stem
cells or other cell types, MultiStem cells have the potential to be produced on a large scale, processed, and
cryogenically preserved, and then used clinically in a rapid and efficient manner. Material obtained from a
single donor may be used to produce hundreds of thousands, or even millions, of individual doses, representing
a yield far greater than we believe other stem cell technologies have been able to achieve.
“Off-the-shelf” utility. Unlike traditional bone marrow or HSC transplants that require extensive genetic
matching between donor and recipient, MultiStem administration does not require tissue matching or
administration of immune suppressive drugs. The MultiStem product is administered as a cryogenically
preserved allogeneic product, meaning that these cells are not genetically matched between donor and recipient.
This feature, combined with the ability to establish large MultiStem banks, could make it practical for clinicians
to efficiently administer this cell therapy to a large number of patients.
Safety. Certain other stem cell types, such as undifferentiated embryonic stem cells or induced pluripotent stem
cells have shown the capacity to form ectopic tissue or teratomas, which are tumor-like growths. These could
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pose serious safety risks to patients. In contrast, MultiStem cells have shown a consistent and favorable
tolerability profile that has been compiled over many years of preclinical study in a range of animal models by
a variety of investigators and that is supported by clinical data from multiple studies to date.
At each step of the MultiStem production process, cells are analyzed according to pre-established criteria to ensure that a
consistent, well-characterized product candidate is produced. Cells are harvested from a prequalified, healthy, consenting
donor, and these cells are then expanded to form a master cell bank from which we subsequently produce clinical grade
material. We have demonstrated the ability to harvest cells that meet our rigorous criteria from healthy donors with a high
degree of consistency. Furthermore, in multiple animal models, MultiStem has been shown to be nonimmunogenic and is
administered without the genetic matching that is typically required for conventional bone marrow or stem cell transplantation.
The distinctive profile of the MultiStem product allows us to pursue multiple high value commercial opportunities from a
single product platform. Based upon work that we and independent collaborators have conducted over the years, we believe
that MultiStem cell therapy has the potential to treat a range of distinct disease indications. As a result, we believe we will be
able to leverage our foundation of a consistent tolerability profile and efficacy data to add clinical indications efficiently,
enabling us to reduce development costs and timelines substantially.
Healthcare represents a significant part of the global economy. In the United States, it represented approximately 17.7% of all
economic activity in 2018, or about $3.6 trillion dollars, annually according to the National Health Expenditure Accounts and
the Centers for Medicaid and Medicare Services. However, the United States, along with many other nations, is experiencing
an unprecedented demographic shift that is resulting in a significantly expanded population of older individuals. According to
United States Census data, in the next few years there will be a dramatic increase in the number of individuals over the age of
65, as this segment of the population increases from 40.2 million individuals in 2010 to more than 72 million people in 2030,
representing an increase of approximately 80%. The aging of the population will create enormous financial and operational
pressure on the healthcare system in the United States and other countries around the world, resulting in significant clinical
challenges, but also resulting in substantial commercial opportunities.
Data from the National Center for Health Statistics shows that as people get older, they are more susceptible to a variety of age
related conditions, including heart disease, stroke, certain forms of cancer, diabetes, progressive neurological disorders, various
chronic inflammatory and immune conditions, renal disease and a range of others. As a consequence, as people get older they
spend far more on healthcare. On average, they spend four to ten times more on healthcare annually at age 65 or beyond than
when they were younger and healthier. According to the Alliance for Aging Research, 83% of healthcare spending is associated
with chronic conditions, and other research from the Department of Health and Human Services shows that 71% of healthcare
spending is associated with multiple chronic conditions. Traditional medical approaches have failed to adequately address this
problem.
We have worked with independent investigators at many leading institutions to study the impact of MultiStem cell therapy in a
range of preclinical models that reflect various types of human disease or injury. To date, we and our collaborators have
published research results illustrating the potential benefits of MultiStem cell therapy in a range of indications including
ischemic stroke, traumatic brain injury, or TBI, brain damage due to restricted blood flow in newborns, spinal cord injury,
myocardial infarction, vascular disease, acute pulmonary distress, bone marrow transplant support/GvHD, and other
indications.
MultiStem for Treating Neurological Conditions, Cardiovascular Disease, Inflammatory and Immune Disorders, Certain
Pulmonary Conditions and Other Conditions
Based on preclinical results, we have advanced MultiStem cell therapy to clinical development stage in several clinical
indications or disease areas, including treatment of ischemic stroke caused by a blockage of blood flow in the brain; ARDS;
complications from trauma; damage caused by myocardial infarction; certain complications associated with traditional bone
marrow or HSC transplantation; inflammatory bowel disease, initially focused on patients suffering from severe, treatment
refractory ulcerative colitis; and to treat or prevent certain complications associated with solid organ transplant. We may expand
to other clinical indication areas as results warrant and resources permit.
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Neurological Injury and Disease — MultiStem for Ischemic Stroke
Another focus of our regenerative medicine program is MultiStem administration for the treatment of neurological injury as a
result of acute or chronic conditions. Neurological injury and disease represent an area of significant unmet medical need, a
major burden on the healthcare system, and also represents a substantial commercial opportunity.
Many neurological conditions require extensive long-term therapy, and many require extended hospitalization and/or
institutional care, creating an enormous quality of life and cost burden. Stroke represents an area where the clinical need is
particularly significant, since it represents a leading cause of death and significant long-term disability. We have published
research with independent collaborating investigators that demonstrates that MultiStem administration conveys biological
benefits in preclinical models of ischemic stroke, as well as other models of neurological damage and injury, including TBI,
neonatal hypoxic ischemia (a cause of neurological damage in infants), and spinal cord injury. We also conducted preclinical
work in other neurological areas and have been awarded grants from time-to-time in support of this work, including the
potential of MultiStem cells to address chronic conditions such as Multiple Sclerosis, or MS, or Parkinson’s disease. Our
research has shown that MultiStem cells convey benefits through distinct mechanisms, including reducing inflammatory
damage, protecting at risk tissue at the site of injury, and through direct neurotrophic effects that stimulate the recovery of
damaged neurons. As a result, we believe that MultiStem cell therapy may have relevance to multiple forms of neurological
injury and disease.
Our initial clinical focus in the neurological area involves evaluating MultiStem administration to treat ischemic stroke.
According to the 2018 American Heart Association, or AHA, statistical update, there are approximately 800,000 individuals in
the United States that suffer a stroke each year, more than two million stroke victims in the United States, Europe and Japan
combined and more than 16.9 million people that suffer a stroke each year globally. The vast majority of these (approximately
85% to 90%) are ischemic strokes, that are caused by a blockage of blood flow in the brain, that cuts off the supply of oxygen
and nutrients, and can result in tissue loss and neurological damage, as well as long-term or permanent disability. The
remaining 10% to 15% are hemorrhagic strokes, which occur when a blood vessel bursts and bleeding into the brain ensues.
Even though ischemic stroke is one of the leading causes of death and disability in the United States, there has been limited
progress toward the development of new treatments that improve the prognosis for stroke victims. The only FDA-approved
drug currently available for treating ischemic stroke is the anti-clotting factor, tPA. According to current clinical guidelines, tPA
must be administered to stroke patients within several hours after the occurrence of the ischemic stroke to dissolve the clot.
Administration of tPA beyond the early treatment window is not recommended, since it can cause cerebral bleeding or even
death. Recent advancements in the development of mechanical clot retrievers and extraction devices may help additional
patients, but such treatments are limited to certain types of strokes and to a limited time window as well. Because of these
limitations, only a small percentage of stroke victims are treated successfully with the currently available therapies—most
simply receive supportive or “palliative” care. The long-term costs of stroke are substantial, with many patients requiring
extended hospitalization, extended physical therapy or rehabilitation for those patients that are capable of entering such
programs, and many require long-term institutional or family care.
In preclinical studies, significant functional improvements have been observed in rodents that have undergone an
experimentally-induced stroke, or that have incurred significant neurological damage due to similar types of ischemic events or
acute injury, such as a result of neonatal hypoxic ischemia or TBI, and then received MultiStem treatment. Published research
has demonstrated that MultiStem administration even one week after a surgically induced stroke results in substantial long-term
therapeutic benefit, as evidenced by the improvement of treated animals compared with controls in a battery of tests examining
mobility, strength, fine motor skills, and other aspects of neurological functional improvement. We believe MultiStem treatment
conveys significant benefits through several mechanisms, including reduction of inflammation and immune system modulation
in the ischemic area, and the protection and rescue of damaged or injured cells, including neuronal tissue. Preclinical research
results demonstrated that MultiStem administration 24 hours following a stroke reduced inflammatory damage in the brain and
resulted in significant functional improvement, and that some of these results were achieved by reducing the inflammatory
response emanating from the spleen in animal models. These results confirmed that MultiStem treatment is well tolerated, does
not require immunosuppression and results in a robust and durable therapeutic benefit, and these results are consistent with
prior results that show MultiStem can provide significant benefits even when administered up to one week after the initial
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stroke event, although earlier treatment (e.g., within 24 hours post-stroke) provided more substantial benefits in these
preclinical studies.
We completed our first clinical study in ischemic stroke, MASTERS-1, which was a randomized, placebo-controlled Phase 2
clinical trial exploring the administration of MultiStem to patients that have suffered an ischemic stroke in the United States
and Europe. The results of this study demonstrated favorable tolerability for MultiStem, consistent with prior clinical studies in
other indications. While the study did not achieve the primary and component secondary endpoints for the intent-to-treat
population, the MultiStem treatment was associated with lower rates of mortality and life-threatening adverse events, infections
and pulmonary events, and also a reduction in hospitalization. In addition, analyses show that patients who received MultiStem
treatment earlier in the study’s treatment window (i.e., 24 to 36 hours post-stroke, as specified in the original study protocol)
had better recovery in comparison to placebo, and this treatment effect appeared to be more pronounced the earlier the
MultiStem administration occurred within this timeframe. Analysis of biomarker data obtained from samples of study subjects
indicated that MultiStem treatment reduces post-stroke inflammation compared to placebo. Furthermore, it appears that this
effect is more pronounced for subjects receiving MultiStem earlier than 36 hours post-stroke. This effect is consistent with our
hypothesis regarding mechanisms of action and related preclinical data, and with the clinical data suggesting faster recovery for
MultiStem-treated patients. One-year follow-up data demonstrated that MultiStem-treated subjects on average continued to
improve through one-year post-treatment and achieved a significantly higher rate of Excellent Outcome compared to placebo
subjects in the intent-to-treat population. We have an ongoing pivotal Phase 3 clinical trial, referred to as MASTERS-2, which
if successful and if the product is approved for commercialization, could make therapy available to most stroke patients in
contrast to other therapies (e.g., tPA), which have substantially shorter treatment windows.
We are also interested in the application of MultiStem for other neurological indications that represent areas of significant
unmet medical need, such as TBI, which represents the leading cause of disability among children and young adults, and a
leading cause of death. Approximately 2.8 million cases of TBI are seen in the United States each year. The United States
Center for Disease Control and Prevention, or CDC, estimates that more than 5.3 million individuals are living with a disability
and have a long-term or lifelong need for help to perform activities of daily living as a result of a TBI. The CDC also estimates
the annual direct and indirect costs for TBI are approximately $76.5 billion a year. In preclinical studies of TBI, administration
of MultiStem dramatically reduced the extent of damage caused by a TBI and promoted accelerated healing of the blood-brain
barrier. With grant funding from the National Institutes of Health, or NIH, we further advanced our MultiStem programs and
cell therapy platform, including further development of MultiStem cell therapy for the treatment of TBI and further
development of our cell therapy formulations and manufacturing capabilities.
We are also conducting preclinical work exploring the application of MultiStem treatment in other neurological indications and
have presented data at leading scientific conferences that demonstrated that intravenous MultiStem administration one day after
spinal cord injury, or SCI, results in statistically significant and sustained improvements in gross locomotor function, fine
locomotor function and bladder control compared to control treated animals. We have published findings that showed that
MultiStem cell therapy was effective in improving the health and recovery of animals following an acute SCI. Intravenous
administration of our cells one day after injury prevented loss of spinal cord tissue, resulting in significant improvement of
walking function and urinary control. Further, we also published an article that provides further evidence that our cell therapy
has the potential to provide benefit following hypoxic ischemia, an injury caused by oxygen deprivation to the brain before or
during birth and a leading cause of cerebral palsy. The article also describes the biological mechanisms through which this cell
therapy delivers benefit. These findings are consistent with previous findings in related areas, such as ischemic stroke, and add
to the scientific foundation supporting MultiStem cell therapy for the treatment of acute neurological injuries.
Over the past several years, we have been utilizing grant funding to investigate the potential for MultiStem treatment for
chronic progressive MS based on initial results in preclinical models. Our previous work, supported by Fast Forward and the
National Multiple Sclerosis Society, demonstrated the potential benefits of MultiStem cell therapy for treating MS. Using
several preclinical models of MS, researchers observed that MultiStem cell administration results in sustained behavioral
improvements, arrests the demyelination process and supports remyelination of affected axons. More recently, we have focused
on the mechanism of action underlying the enhanced remyelination in vivo and shown that MultiStem cells and secreted factors
increase differentiation of oligodendrocytes.
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Inflammatory and Immunological Disorders — MultiStem for Acute Respiratory Distress, Trauma Complications, HSC
Transplant Support and other indications
Inflammatory and immune disorders represent a significant burden to society. There are over 80 recognized autoimmune
disorders, which are conditions caused by an acute or chronic imbalance in the immune system. In these conditions, cells of the
immune system begin to attack certain tissues or organs in the body, resulting in tissue damage and loss of function. Some
inflammatory and immune conditions are associated with age-related conditions (e.g., rheumatoid arthritis), but some are due to
other causes that may be genetic, environmental or a combination of both (e.g., Type 1 diabetes, Inflammatory Bowel Disease).
Still other conditions may reflect complications associated with other diseases or trauma or the treatment of other conditions
(e.g., GvHD, a frequent complication associated with transplant procedures used to treat leukemia or related blood-borne
cancers). Each of these conditions shares certain biological characteristics, in that the immune system imbalance results from
the inappropriate activation of certain populations of immune cells that subsequently results in significant tissue damage and
destruction. This immune imbalance may result in a complex cascade of inflammation that can result in pain, progressive tissue
deterioration and loss of function. While currently available immunomodulatory drugs have proven to be effective for some
patients, they have failed to adequately address the needs of many other patients that suffer from inflammatory and immune
disorders.
In both preclinical and clinical studies, MultiStem cells have shown potent immunomodulatory properties, including the ability
to reduce active inflammation through various modes of action, stimulate tissue repair and restore immune system balance.
Accordingly, we believe that MultiStem cell therapy could have broad application in the area of treating immune system
disorders, including certain acute inflammatory conditions, autoimmune diseases and other conditions.
In animal models, MultiStem cells have demonstrated an ability to reduce the severity of pulmonary distress, reduce alveolar
edema and return lung endothelial permeability to normal. Intravenous MultiStem treatment early following the onset of the
condition may ameliorate the initial hyper-inflammation and reduce the fibrotic activity that follows, thereby speeding the
return to and improving the likelihood of more normal lung function and helping patient recovery.
ARDS is a serious immunological and inflammatory condition characterized by widespread inflammation in the lungs. ARDS
can be triggered by pneumonia, sepsis, or other trauma and represents a major cause of morbidity and mortality in the critical
care setting. It has significant implications, as it prolongs ICU and hospital stays, and requires convalescence in the hospital
and rehabilitation. There are limited interventions and no effective drug treatments for ARDS, making it an area of high unmet
clinical need with high treatment costs. Given the high treatment costs of ARDS, a successful cell therapy could be expected to
generate significant savings for the healthcare system by reducing days on a ventilator, days in the intensive care unit and total
days in the hospital, and could reduce mortality and morbidity, as well as improve quality of life for those suffering from the
condition. The medical need for a safe and effective treatment of ARDS is significant due to its high mortality rate, and it
affects annually approximately 33,000 patients in the UK and 400,000 - 500,000 patients in Europe, the United States and
Japan, alone.
In January 2019 and January 2020, we announced summary results and one-year follow up results, respectively from our
exploratory clinical study of the intravenous administration of MultiStem cell therapy to treat patients who are suffering from
ARDS. The study results provide further confirmation of tolerability associated with MultiStem treatment. Importantly,
MultiStem subjects had lower mortality and a greater number of ventilator-free and ICU-free days compared to patients
receiving placebo. Furthermore, analysis of initial biomarker data reflects lower levels of inflammatory markers/cytokines
following MultiStem treatment, an expected mechanism of action in this patient population.
Our research and others' research suggest that the activation of an acute hyperinflammatory response involving the peripheral
immune system is a conserved biological response that occurs across multiple forms of trauma. For example, a common
complication among trauma victims is Systemic Inflammatory Response Syndrome, which can contribute to or play a causative
role in impaired organ system function, organ failure, or even multi-organ failure. We believe MultiStem can help address this
systemic inflammatory response and its complications, and promote better recovery following trauma. In 2018, we announced
that the Department of Defense, through the Medical Technology Enterprise Consortium, plans to provide funding for a Phase 2
clinical trial to evaluate the administration of MultiStem cell therapy for early treatment and prevention of complications after
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severe traumatic injury, in collaboration with UTHealth. This first-ever study of a cell therapy for treatment of a wide range of
traumatic injuries will be conducted at Memorial Hermann-Texas Medical Center, one of the busiest Level 1 trauma centers
in the United States. We will provide the investigational clinical product for the conduct of the trial, as well as regulatory and
operational support, as our contribution to the trial. The objective of the clinical study is to evaluate the safety and effectiveness
of MultiStem for the treatment of severely injured patients for the prevention and early treatment of complications after severe
traumatic injury. The proposed study is anticipated to be a randomized, double-blind, placebo-controlled Phase 2 clinical trial
estimated to enroll approximately 150 severely injured trauma patients within hours of hospitalization who have survived initial
treatment and are admitted to the ICU. We and UTHealth are working to finalize a plan for the study and this patient population
that is acceptable to the FDA.
Another area of focus is the use of MultiStem cell therapy as adjunctive treatment for HSC/bone marrow transplant used as
therapy in hematologic malignancy. For many types of cancer, such as leukemia or other blood-borne cancers, treatment
typically involves radiation therapy or chemotherapy, alone or in combination. Such treatment can substantially deplete the
cells of the blood and immune system, by reducing the number of stem cells in the bone marrow from which they arise. The
more intense the radiation treatment or chemotherapy, the more severe the resulting depletion is of the bone marrow, blood and
immune system. Other tissues may also be affected, such as cells in the digestive tract and in the pulmonary system. The result
may be severe anemia, immunodeficiency, substantial reduction in digestive capacity, and other problems that may result in
significant disability or death.
One strategy for treating the depletion of bone marrow is to perform a peripheral blood stem cell transplant or a bone marrow
transplant. This approach may augment the patient’s ability to form new blood and immune cells and provide a significant
survival advantage. However, finding a closely matched donor is frequently difficult or even impossible. Even when such a
donor is found, in many cases there are immunological complications, such as GvHD, which may result in serious disability or
death.
Working with leading experts in the stem cell and bone marrow transplantation field, we studied MultiStem in animal models
of radiation therapy and GvHD. In multiple animal models, MultiStem cells have been shown to be non-immunogenic, even
when administered without the genetic matching that is typically required for conventional bone marrow or stem cell
transplantation. Furthermore, in animal model systems testing immune reactivity of T-cells against unrelated donor tissue,
MultiStem has been shown to suppress the T-cell-mediated immune responses that are an important factor in causing GvHD.
MultiStem-treated animals also displayed a significant increase in survival relative to controls. As a result, we believe that
MultiStem administration in conjunction with or following standard HSC transplantation may have the potential to reduce the
incidence or severity of complications and may enhance gastrointestinal function, which is frequently compromised as a result
of radiation treatment or chemotherapy.
We completed a Phase 1 clinical trial examining the safety and tolerability of a single dose or repeat dosing of MultiStem cells
administered intravenously to patients receiving a bone marrow or HSC transplant as part of their treatment of leukemia or
other hematological condition. The trial was an open-label, multicenter trial that involved leading experts in the field of bone
marrow transplantation. We observed a consistent favorable tolerability profile in both the single and multiple dose arms of the
study, and at all dose levels tested. Although the trial was not specifically designed to demonstrate efficacy, we also observed
clinically meaningful improvement in medically important parameters relative to historical clinical experience, including
reduced incidence and severity of acute GvHD, improved relapse free survival, no graft failures and enhanced engraftment
rates relative to other forms of treatment.
We were granted orphan drug designation by the FDA and the EMA for MultiStem treatment in the prevention of GvHD, and
the MultiStem product was granted Fast Track designation by the FDA for prophylaxis therapy against GvHD following HSC
transplantation. Subsequently, our registration study design received a positive opinion from the EMA through the Protocol
Assessment/Scientific Advice procedure. Furthermore, the proposed registration study received SPA designation from the FDA,
meaning that the trial is adequately designed to support a BLA submission for registration if it is successful.
Cardiovascular Disease — Evaluating MultiStem for Treating Damage from a Heart Attack
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Cardiovascular disease is an area of significant clinical need and its prevalence is expected to grow in the years ahead. Despite
treatment advances in recent years, cardiovascular disease remains the leading cause of death and represents one of the leading
causes of disability around the world. In the United States, over one million people suffer a heart attack each year, according to
the AHA 2018 Statistical Update. There were approximately 836,500 deaths (or approximately 1 of every 3 deaths in the
United States) that occurred from all forms of cardiovascular disease, including approximately 366,800 individuals that died as
a result of coronary heart disease. Heart disease remains the leading cause of death in the United States.
In a Phase 1 clinical trial we conducted previously, we explored MultiStem treatment for damage caused by AMI. Myocardial
infarction, more commonly referred to as a heart attack, is caused by the blockage of one or more arteries that supply blood to
the heart. Such blockages can be caused, for example, by the rupture of an atherosclerotic plaque deposit. A variety of risk
factors are associated with an elevated risk of myocardial infarction or atherosclerosis, including age, high blood pressure,
smoking, sedentary lifestyle and genetics. While advances in the diagnosis, prevention and treatment of heart disease have had
a positive impact, there is clearly room for improvement—myocardial infarction remains a leading cause of death and disability
in the United States and the rest of the world.
MultiStem treatment has been studied in validated animal models of AMI, where investigators demonstrated that the
administration of allogeneic MultiStem cells into the hearts of animals damaged by experimentally induced heart attacks
resulted in significant functional improvement in cardiac output and other functional parameters compared with animals that
received placebo or no treatment. Furthermore, the administration of an immunosuppressive drug was not required and
provided no additional benefit in this study and thereby supporting the concept of using MultiStem cells as an allogeneic
product. We completed additional preclinical studies in established pig models of AMI using catheter delivery and examining
various factors such as the route and method of MultiStem administration, dose ranging and timing of treatment.
We conducted a multicenter, open-label Phase 1 clinical trial in this indication and the results showed that MultiStem treatment
was well-tolerated at all dose levels and that patients who received MultiStem treatment exhibited meaningful improvements in
cardiovascular function, including left ventricular ejection fraction, wall motion scores, and other parameters. The Phase 1 data
was published in a leading peer reviewed scientific journal, and one-year follow-up data suggested that the benefit observed
was sustained over time. We were conducting a Phase 2 clinical study for the administration of MultiStem cell therapy to
patients that have suffered a heart attack. This study was based in part on the favorable results of our previously completed
Phase 1 clinical study that demonstrated a favorable tolerability profile and encouraging signs of improvement in heart function
among patients that exhibited severely compromised heart function prior to treatment. However, enrollment in the Phase 2
clinical study was very challenging due in part to changes in standard of care. Due to these challenges and the difficulties in
addressing them, and priorities in other clinical areas, we elected in 2019 to suspend the study and determine what has been
learned. We will evaluate our development strategy before proceeding further.
Other Programs
Animal Health Care
While development of our clinical programs for human health indications remains our priority, based on our research to date
and work performed at our Belgian subsidiary, ReGenesys, we have demonstrated in preclinical animal health models that
MultiStem cell therapy can promote tissue repair and healing that could provide meaningful benefits to animal patients,
including those suffering from serious conditions with unmet medical need. According to Future Market Insights and other
analysts, the global animal healthcare market for 2017 to 2027 was estimated to be valued at approximately $55 billion and is
expected to grow at a compound annual growth rate of more than 4.3% during this period. The companion animal segment is a
particularly fast-growing area, projected to exceed more than $15 billion by 2020.
Collaborations and Partnerships
Healios
We have entered into a series of agreements with Healios, our collaborator in Japan and currently our largest stockholder.
Under the collaboration that began in 2016, Healios is responsible for the development and commercialization of the
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MultiStem product for the licensed fields in the licensed territories, and we provide manufacturing services to Healios,
currently comprising the supply of product for its clinical trials and preparations for commercial supply in Japan including
transfer of technology to a Japanese contract manufacturer, and we have been compensated for these services.
In 2016, we entered into a license agreement, or First License Agreement, with Healios to develop and commercialize
MultiStem cell therapy for ischemic stroke in Japan and to provide Healios with access to our proprietary Multipotent Adult
Progenitor Cell, or MAPC, technology for use in Healios’ organ bud program worldwide, initially for transplantation to treat
liver disease or dysfunction. Under the First License Agreement, Healios also obtained a right to expand the scope of the
collaboration, and Healios exercised this right in June 2018. Upon the expansion in June 2018, which was broader than that
contemplated in the First License Agreement, we entered into the Collaboration Expansion Agreement, or CEA. Through the
CEA, Healios (i) expanded its First License Agreement to include ARDS in Japan and expanded the organ bud license to
include additional transplantation indications covered under Healios organ bud technology; (ii) obtained a worldwide exclusive
license, or the Ophthalmology License Agreement, for use of MultiStem product to treat certain ophthalmological indications;
(iii) obtained an exclusive license in Japan, or the Combination Product License Agreement, for use of the MultiStem product
to treat diseases of the liver, kidney, pancreas and intestinal tissue through local administration of MultiStem cell therapy in
combination with iPSC-derived cells; (iv) obtained an exclusive, time-limited right of first negotiation, or ROFN Period, to
enter into an option for a license to develop and commercialize certain MultiStem treatments in China, which expired in June
2019; and (v) certain other rights, including an option for an additional non-therapeutic technology license, which also expired.
For all indications, Healios is responsible for the costs of clinical development in its licensed territories, and we provide
manufacturing services to Healios.
Each license agreement with Healios has defined economic terms. Under the First License Agreement that related primarily to
the license to ischemic stroke in Japan, we received a nonrefundable, up-front cash payment of $15 million, and upon the
inclusion of the ARDS field in Japan, we received a nonrefundable, up-front cash payment of $10 million. For the additional
rights granted to Healios under the CEA, including the Ophthalmology License Agreement and the Combination Product
License Agreement, Healios paid us an additional nonrefundable, up-front payment of $10 million, which was paid in four
quarterly installment payments of $2.5 million. Healios may elect to credit up to $10 million against milestone payments that
may become due under the First License Agreement, as expanded to include ARDS, with limitations on amounts that may be
credited to earlier milestone payments versus later milestone payments.
For each of the ischemic stroke indication and the ARDS indication, we may receive aggregate success-based regulatory filing
and approval milestones up to $50 million and potential sales milestones up to $175 million, amounting to $225 million for
each indication (or $450 million in aggregate), subject to potential milestone credits. Milestone payments for all indications
under the collaboration are non-refundable and non-creditable towards future royalties or any other payment due from Healios.
For each of the ischemic stroke indication and the ARDS indication, we are entitled to receive tiered royalties on product sales,
starting in the low double digits and increasing incrementally into the high teens or potentially higher depending on net sales
levels and other factors.
The Ophthalmology License Agreement granted Healios worldwide, exclusive rights to treat certain ophthalmological diseases,
by using either MultiStem cell therapy on a standalone basis or MultiStem in combination with retinal pigment epithelium cells
derived from either iPSC or embryonic stem cells. For the standalone products, we will be entitled to receive success-based
regulatory filing and approval milestones aggregating up to $48.1 million, potential sales milestones of up to $87.5 million, and
tiered royalties on product sales in the single digits depending on net sales levels. For the combination ophthalmology products,
we are entitled to receive a low single-digit royalty, but no milestone payments.
The Combination Product License Agreement granted Healios exclusive rights in Japan to treat diseases of the liver, kidney,
pancreas and intestinal tissue through local administration of MultiStem cell therapy in combination with iPSC-derived cells
through certain delivery methods. We are entitled to receive a low single-digit royalty on net sales of the combination product
treatments, but no milestone payments.
For the organ bud product, we are entitled to receive a fractional royalty on net sales of the organ bud products. For all
indications covered by the Healios organ bud technology that utilize our technology, we may receive payments for
manufactured product supplied to Healios under a manufacturing supply agreement. Additionally, we have a right of first
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negotiation for commercialization of an organ bud product in North America, with such right expiring on the later of (i) the date
five years from the effective date of the First License Agreement and (ii) 30 days after authorization to initiate clinical studies
on an organ bud product under the first investigational new drug application or equivalent in Japan, North America or the
European Union, or EU.
Under the CEA, the ROFN Period with respect to the option for a license in China was extended to June 30, 2019 in exchange
for a $2.0 million payment from Healios that we received in December 2018. The ROFN Period expired on June 30, 2019.
In March 2018, Healios purchased 12,000,000 shares of our common stock and a warrant, or the Healios Warrant, to purchase
up to 20,000,000 additional shares of common stock for $21.1 million, or approximately $1.76 per share. Based upon the
expiration of the ROFN Period at June 30, 2019, the warrant is no longer exercisable for up to 16,000,000 warrant shares. The
Healios Warrant is exercisable for up to 4,000,000 shares at an exercise price equal to a reference price (which is generally
110% of the average closing price per share of our common stock for the ten trading days ending on the trading day
immediately preceding the date the Healios Warrant is exercised), but no less than $1.76 per share. The Healios Warrant expires
in its entirety in September 2020 and may be terminated by us under certain conditions.
In 2017, we signed a clinical trial supply agreement for delivering the planned manufacturing services for Healios’ clinical trial
in Japan treating ischemic stroke patients, which was amended in 2018 to also include the clinical trial supply for Healios'
clinical trial treating ARDS patients. The agreement includes a cost-sharing arrangement associated with our supply of clinical
product for Healios’ TREASURE study in Japan, including Healios’ right to apply cost-share payments as a credit against
certain milestone payments that may become due for the stroke indication under the First License Agreement, and if so applied,
a stroke sales milestone would be increased, as defined. Alternatively, such cost-share payments may be repaid by us at our
election. We use commercially reasonable efforts to supply manufactured product and successfully delivered all product
required by Healios to complete the TREASURE and ONE-BRIDGE studies in 2019. In the event that we are unable to supply
product to Healios, we may notify Healios and grant it a license to make the product solely for use in the licensed areas.
Also in 2017, we entered into a technology transfer services agreement with Healios, in which Healios provides financial
support to establish a contract manufacturer in Japan to manufacture product for Healios. At that time, we also amended the
First License Agreement to confer to Healios a limited license to manufacture MultiStem in the event that we are acquired by a
third-party. Technology transfer services are ongoing.
The First License Agreement will expire automatically when there are no remaining intellectual property rights subject to the
license. Additionally, Healios may terminate the First License Agreement under certain circumstances, including for material
breach and without cause upon advance written notice. We may terminate the First License Agreement if there is an uncured
material breach of the agreement by Healios. Following the expiration or termination of the First License Agreement, Healios
shall pay reduced royalties for continued use of our trademarks.
Following termination of the First License Agreement, the licenses granted to Healios to develop and commercialize MultiStem
in Japan for ischemic stroke and for ARDS will terminate. Healios will transfer ownership to us of its documents related to the
product, the field and the Japan territory, such as regulatory filings, correspondence, approvals and documents; investigator
brochures clinical data; and information related to the product. Further, the nonexclusive license to intellectual property
developed by Healios during the collaboration shall survive termination and become our confidential information.
The Ophthalmology License Agreement and Combination Product License Agreement will expire with respect to each licensed
product in each country upon the latest of four events: (i) expiration of our applicable pre-existing patents, (ii) expiration of our
applicable patents filed after the effective date, (iii) loss of all data or other regulatory exclusivity, and (iv) 10 years after first
commercial sale. Each agreement may expire earlier for products in territories upon certain defined conditions related to the
availability of alternative products. Each agreement would terminate in its entirety when all such product terms for each
territory have expired. After expiration of a product in a territory, or the agreement as a whole, Healios’ licenses remain in
effect and Healios remains obligated to pay royalties at a reduced rate, and for a limited time, at which time the exclusive
nature of the licenses convert to non-exclusive. Additionally, Healios may terminate the agreements under certain
circumstances, including for material breach and without cause upon advance written notice (in which case Healios’ licenses do
not survive). We may terminate either of these agreements if there is an uncured material breach of an agreement by Healios (in
which case Healios’ licenses would not survive).
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University of Minnesota
In 2003, we acquired the exclusive rights to the MAPC technology originally developed at the University of Minnesota
pursuant to a license agreement with the University. We subsequently further developed this technology, including refining and
establishing proprietary methods related to the manufacturing of the cells, creating new intellectual property and patents outside
of the license. We are obligated to pay the University of Minnesota a royalty based on worldwide commercial sales of licensed
products if covered by a valid licensed patent, as well as sublicensing fees and fees related to manufactured product proceeds,
as defined. The low single-digit royalty and sublicense fee rate may be reduced if third-party payments for intellectual property
rights are necessary or commercially desirable to permit the manufacture or sale of the product. The royalty payment obligation
and the term of the license agreement expire upon the last to expire licensed patent. Based on our current patent portfolio, and
absent any continuations, renewals or extensions of existing patents, the last licensed patent to expire under this license
agreement is currently expected to expire in 2029. The license agreement does not have a specific termination date, but the
University of Minnesota can terminate the license agreement for an uncured event of default, as defined, or upon our
bankruptcy and we can terminate the license agreement at any time.
Manufacturing
We work with third parties to manufacture our MultiStem product candidates in accordance with good manufacturing practices,
or GMP, and until such time as we are able to manufacture products ourselves in accordance with GMP, we will rely on such
third-party manufacturers to make our MultiStem product for clinical trials and eventual commercial sales. These third parties
may not deliver sufficient quantities of our MultiStem product, manufacture MultiStem product in accordance with
specifications, or maintain compliance with applicable government regulations. From time to time, such third-party
manufacturers, or their material suppliers, may be subject to inspection by the FDA or other regulators, which under certain
circumstances could result in production stoppages and interruptions in supply, affecting the initiation, execution and timing of
completion of clinical trials and commercial activities. Furthermore, material supply constraints could result in production
delays. We attempt to mitigate risk to our product supply by careful planning of our production and raw material requirements
with sufficient lead times for ramp-up by third-party manufacturers. Additionally, we work with and qualify other third-party
manufacturers to provide alternative manufacturing capacity, if needed, due to delays or interruptions in supply, but such
alternative manufacturers may be subject to similar constraints or issues.
Importantly, we are engaged in process development initiatives intended to increase manufacturing scale, reduce production
costs, and enhance process controls and product quality, among other things. These initiatives are being conducted both
internally and outsourced to select contractors, and the related investments are meant to enable us to meet potential commercial
demand in the event of potential regulatory approval.
Competition
We face significant competition with respect to the various dimensions of our business. With regard to our efforts to develop
MultiStem as a novel stem cell therapy, currently, there are a number of companies that are actively developing stem cell
products, which encompass a range of different cell types, including embryonic stem cells, umbilical cord stem cells, adult-
derived stem cells and processed bone marrow derived cells.
Mesoblast Limited, or Mesoblast, is currently engaged in clinical trials evaluating the safety and efficacy of Revascor, an
allogeneic stem cell product based on mesenchymal stem cell precursors that are obtained from healthy consenting donors.
These cells also appear to display limited expansion potential and biological plasticity. Additionally, Mesoblast is developing
Prochymal, a mesenchymal stem cell product candidate that it acquired from Osiris Therapeutics, Inc.
Other public companies are or may be developing stem-related therapies, including SanBio, Vericel Corporation, Tigenix NV
(acquired by Takeda), Caladrius Biosciences, Inc., Johnson & Johnson, Celgene Corporation, or Celgene, CRYO-CELL
International, Inc., Pluristem Therapeutics, Inc., or Pluristem, and Cytori Therapeutics, Inc., or Cytori. In addition, private
companies, such as Gamida Cell Ltd., Ocata Therapeutics Inc., Plureon Corporation, and others, are also developing cell
therapy related products or capabilities. Given the magnitude of the potential opportunity for stem cell therapy, we expect
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competition in this area to intensify in the coming years. In addition, our other earlier-stage programs may face competition,
including from larger pharmaceutical and biotechnology companies.
Many of our competitors may have substantially greater financial, technical, sales, marketing, and human resources than we do.
These companies may succeed in obtaining regulatory approval for competitive products more rapidly than we can for our
products. In addition, our competitors may develop technologies and products that are cheaper, safer or more effective than
those being developed by us or that would render our technology obsolete. Furthermore, some of these companies may feel
threatened by our activities and attempt to delay or impede our efforts to develop our products or apply our technologies.
Intellectual Property
We rely on a combination of patent applications, patents, trademarks, and contractual provisions to protect our proprietary
rights. We believe that to have a competitive advantage, we must develop and maintain the proprietary aspects of our
technologies. Currently, we require our officers, employees, consultants, contractors, manufacturers, outside scientific
collaborators and sponsored researchers, and other advisors to execute confidentiality agreements in connection with their
employment, consulting, or advisory relationships with us, where appropriate. We also require our employees, consultants, and
advisors that we expect to work on our products to agree to disclose and assign to us all inventions conceived during the
workday, developed using our property, or which relate to our business. We currently have over 350 patents for our
technologies.
We have a broad patent estate with claims directed to compositions, methods of production, and methods of use of certain non-
embryonic stem cells and related technologies. We developed, acquired and exclusively licensed intellectual property covering
our cell therapy product candidates and other applications in the field. Our broad intellectual property portfolio consists of over
330 issued patents (of which 37 are United States patents) and more than 140 global patent applications around our stem cell
technology and MultiStem product platform. This includes 36 United States patents and more than 280 international patents
that apply to MAPC and related products, such as MultiStem. The current intellectual property estate, which incorporates
additional filings and may broaden over time, could provide coverage for our stem cell product candidates, manufacturing
processes and methods of use through 2040 and beyond. Furthermore, an extended period of market exclusivity may apply for
certain products (e.g., exclusivity periods for orphan drug designation or biologics).
We also have established a broad intellectual property portfolio related to our small molecule product candidates, functional
genomics, and other technologies, with over 25 global patents with claims directed to compositions, methods of making, and
methods of using our candidates and technologies, among other claims.
We have been active in the development, improvement and protection of our intellectual property portfolio through our
prosecution efforts, collaborative research efforts, and in-licensing, among other things. From time-to-time, we will also engage
in adversarial processes, such as interference or litigation, to protect or advance certain patents or applications. These activities
represent an important cost of doing business and can result in successes and setbacks due to the nature of the processes. For
example, several years ago, we were involved in several proceedings in the United States and Europe involving a third-party’s
technology developed after the MAPC technology, which ultimately resulted in a license agreement favorable to the Company.
Over time, we expect to be involved in similar proceedings with the objective of developing the portfolio to support and protect
development and commercialization of our or our licensees’ cell therapy products.
We believe that we have broad freedom to use and commercially develop our technologies and product candidates. However, in
the event that we or our collaborators are developing, manufacturing, or selling potential products that are claimed to infringe a
third-party’s intellectual property, a loss in litigation may prevent us from commercializing our products, unless that party
grants us rights to use its intellectual property. Further, we may not be able to obtain any licenses required under any patents or
proprietary rights of third parties on acceptable terms, or at all. Even if we were able to obtain rights to the third-party’s
intellectual property, these rights may be non-exclusive, thereby giving our competitors access to the same intellectual property.
Ultimately, we may be unable to commercialize some of our potential products or may have to cease some of our business
operations as a result of patent infringement claims, which could severely harm our business.
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Research and Development
Our research and development costs, which consist primarily of costs associated with clinical trials, preclinical research,
product manufacturing and process development for manufacturing, salaries and related personnel costs, legal expenses
resulting from intellectual property application and maintenance processes, and laboratory supply and reagent costs, were $39.0
million in 2019, $38.7 million in 2018 and $27.8 million in 2017.
Government Regulation
Our research and development activities, and any products we may develop, are subject to stringent government regulation in
the United States by the FDA and, in many instances, by corresponding foreign and state regulatory agencies. The European
Union, or EU, has vested centralized authority in the EMA and Committee on Proprietary Medicinal Products, or CPMP, to
standardize review and approval across EU member nations. In Japan, PDMA, a division of the Ministry of Health, Labour and
Welfare, or MHLW, regulates the development and commercialization of medical therapies. Recently, Japan’s parliament
enacted new legislation to promote the safe and accelerated development of treatments using stem cells. The new regenerative
medicine law and revised pharmaceutical affairs law define products containing stem cells as regenerative medicine products
and allow for the conditional approval of such products if safety has been confirmed in clinical trials, even if their efficacy has
not been fully demonstrated. The legislation creates a new, faster pathway for cell therapy product approval, and offers the
potential to enable more rapid entry in the Japanese market. The MHLW has been directed to develop and adopt new rules and
procedures to implement this legislation.
These regulatory agencies enforce comprehensive statutes, regulations and guidelines governing the drug development process.
This process involves several steps. Initially, a company must generate preclinical data to show safety before human testing
may be initiated. In the United States, for example, a drug company must submit an IND to the FDA prior to securing
authorization for human testing. The IND must contain adequate data on product candidate chemistry, toxicology and
metabolism and, where appropriate, animal research testing to support initial safety.
Any of our product candidates will require regulatory approval and compliance with regulations made by United States and
foreign government agencies prior to commercialization in such countries. The process of obtaining FDA or foreign regulatory
agency approval has historically been extremely costly and time consuming. The FDA and equivalent foreign regulatory
authorities (such as EMA or PMDA) regulate, among other things, the development, testing, manufacture, safety, efficacy,
record keeping, labeling, storage, approval, advertising, promotion, sale, and distribution of biologics and new drugs.
The standard process required by the FDA before a pharmaceutical agent may be marketed in the United States includes:
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preclinical tests in animals that demonstrate a reasonable likelihood of safety and effectiveness (if possible) in
human patients;
submission to the FDA of an IND, which must become effective before clinical trials in humans can commence.
If Phase 1 clinical trials are to be conducted initially outside the United States, a different regulatory filing is
required, depending on the location of the trial;
adequate and well controlled human clinical trials to establish the safety and efficacy of the drug or biologic
product for the intended disease indication;
for drugs (including biologics), submission of a New Drug Application, or NDA, or a BLA with the FDA; and
FDA approval of the NDA or BLA before any commercial sale or shipment of the drug.
Preclinical studies can take several years to complete, and there is no guarantee that an IND based on those studies will become
effective to permit clinical trials to begin. The clinical development phase generally takes ten to fifteen years, or longer, to
complete (i.e., from the initiation of Phase 1 through completion of Phase 3 studies), and such sequential studies may overlap
or be combined. After successful completion of clinical trials for a new drug or biologic product, FDA approval of the NDA or
BLA must be obtained. This process requires substantial time and effort and there is no assurance that the FDA will accept the
NDA or BLA for filing and, even if filed, that the FDA will grant approval. In the past, the FDA’s approval of an NDA or BLA
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has taken, on average, one to two years, but in some instances may take substantially longer. If questions regarding safety or
efficacy arise, additional studies may be required, followed by a resubmission of the NDA or BLA. Review and approval of an
NDA or BLA can take up to several years. The FDA and other Regulatory agencies such as the EMA and the PMDA have
regulations that allow for faster approval paths and review cycles that may reduce clinical development phase completion to
between five and seven years to commercialization. Such regulations include but are not limited to accelerated/conditional
approval paths and review cycles of between six to ten months (priority/accelerated review cycles). However, there are specific
criteria that must be met to qualify for these paths, such as high unmet medical need, orphan designation, fast track, exceptional
circumstances and breakthrough designation.
In addition to obtaining FDA approval for each product being sold in the United States, each drug manufacturing facility must
be inspected and approved by the FDA. All manufacturing establishments are subject to inspections by the FDA and by other
federal, state, and local agencies, and must comply with GMP requirements. We do not currently have any GMP manufacturing
capabilities and will rely on contract manufacturers to produce material for any clinical trials that we conduct.
We must also obtain regulatory approval in other countries in which we intend to market any drug. The requirements governing
conduct of clinical trials, product licensing, pricing, and reimbursement vary widely from country to country. FDA approval
does not ensure regulatory approval in other countries. The current approval process varies from country to country, and the
time spent in gaining approval varies from that required for FDA approval. In some countries, the sale price of the drug must
also be approved. The pricing review period often begins after market approval is granted. Even if a foreign regulatory
authority approves a drug product, it may not approve satisfactory prices for the product.
In addition to regulations enforced by the FDA and international regulatory agencies, we are also subject to regulation under
the Occupational Safety and Health Act, the Environmental Protection Act, the Toxic Substances Control Act, the Resource
Conservation and Recovery Act, and other present and potential future federal, state, or local regulations. Our research and
development activities involve the controlled use of hazardous materials, chemicals, biological materials, and various
radioactive compounds. Although we believe that our safety procedures for handling and disposing of such materials currently
comply in all material respects with the standards prescribed by state and federal regulations, the risk of accidental
contamination or injury from these materials cannot be completely eliminated. In the event of such an accident, we could be
held liable for any damages that result and any such liability could exceed our available resources.
Employees
We believe that our success will be based on, among other things, the quality of our clinical programs, our ability to invent and
develop superior and innovative technologies and products, and our ability to attract and retain capable management and other
personnel. We have assembled a high-quality team of scientists, clinical development managers, and executives with significant
experience in the biotechnology and pharmaceutical industries. As of December 31, 2019, we employed 83 full-time
employees, including 18 with Ph.D. degrees. In addition to our employees, we also use the service and support of outside
consultants and advisors. None of our employees is represented by a union, and we believe relationships with our employees
are good.
Available Information
We use the Investors section of our website, www.athersys.com, as a channel for routine distribution of important information,
including news releases, analyst presentations and financial information. We post filings as soon as reasonably practicable after
they are electronically filed with, or furnished to, the Securities and Exchange Commission, or SEC, including our annual,
quarterly, and current reports on Forms 10-K, 10-Q, and 8-K; our proxy statements; and any amendments to those reports or
statements. All such postings and filings are available on the Investors section of our website free of charge. In addition, this
website allows investors and other interested persons to sign up to automatically receive e-mail alerts when we post news
releases and financial information on our website. The SEC also maintains a website, www.sec.gov, which contains reports,
proxy and information statements, and other information regarding issuers that file electronically with the SEC. The content on
any website referred to in this annual report on Form 10-K is not incorporated by reference into this annual report unless
expressly noted.
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ITEM 1A.
RISK FACTORS
The statements in this section, as well as statements described elsewhere in this annual report, or in other SEC filings, describe
risks that could materially and adversely affect our business, financial condition and results of operations, which could also
cause the trading price of our equity securities to decline. These risks are not the only risks that we face. Our business, financial
condition and results of operations could also be affected by additional factors that are not presently known to us or that we
currently consider to be immaterial to our operations.
We have incurred losses since inception, and we expect to incur significant net losses in the foreseeable future and may
never become profitable.
Since our inception in 1995, we incurred significant losses and negative cash flows from operations. We incurred net losses of
$44.6 million in 2019, $24.3 million in 2018 and $32.2 million in 2017. As of December 31, 2019, we had an accumulated
deficit of $417.6 million, and we will not commence sales of our clinical product candidates until they receive regulatory
approval for commercialization. We expect to spend significant resources over the next several years to continue our research
and product development programs, including clinical trials of our product candidates and process development and
manufacturing projects, and to prepare for possible regulatory approval and commercial activities. We expect to continue to
incur substantial losses through at least the next several years and may incur losses in subsequent periods, and our ability to
commercialize our product candidates is uncertain. To date, substantially all of our revenue has been derived from corporate
collaborations, license agreements and government grants. In order to achieve profitability, we must develop products and
technologies that can be commercialized by us or through our existing or future collaborations. Our ability to generate revenues
and become profitable will depend on our ability, alone or with potential collaborators, to timely, efficiently and successfully
complete the development of our product candidates. We have never earned revenue from selling a product and we may never
do so, as none of our product candidates have been approved for sale, since they are currently being tested in human and animal
studies. We cannot assure you that we will ever earn sales revenue or that we will ever become profitable. If we sustain losses
over an extended period, we may be unable to continue our business.
We will need substantial additional funding to develop our products and for our future operations. If we are unable to
obtain the funds necessary to do so, we may be required to delay, scale back or eliminate our product development activities
or may be unable to continue our business.
The development of our product candidates will require a commitment of substantial funds to conduct the costly and time-
consuming research, which may include preclinical and clinical testing, necessary to obtain regulatory approvals and bring our
products to market. Net cash used in our operations was $35.3 million in 2019, $13.4 million in 2018 and $24.0 million in
2017.
At December 31, 2019, we had $35.0 million of cash and cash equivalents. However, we will need substantially more funding
to advance our product candidates through development and into commercialization, including to put in place manufacturing
capacity to support such commercial activity. Our future capital requirements will depend on many factors, including:
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our ability to raise capital to fund our operations;
the progress, scope, costs and results of our clinical and preclinical testing of any current or future product
candidates;
the possibility of delays in, adverse events of and excessive costs of the development process;
the cost of manufacturing our product candidates;
the cost of prosecuting, defending and enforcing patent claims and other intellectual property rights;
the time and cost involved in obtaining regulatory approvals;
expenses related to complying with GMP of therapeutic product candidates;
costs of financing or acquiring additional capital equipment and development technologies;
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competing technological and market developments;
our ability to establish and maintain collaborative and other arrangements with third parties to assist in bringing
our products to market and the cost of such arrangements;
the amount and timing of payments or equity investments that we receive from collaborators or changes in or
terminations of future or existing collaboration and licensing arrangements and the timing and amount of
expenses we incur to support these collaborations and license agreements;
costs associated with the integration of any new operation, including costs relating to future mergers and
acquisitions with companies that have complementary capabilities;
expenses related to the establishment of sales and marketing capabilities for products awaiting approval or
products that have been approved;
expenses related to establishing manufacturing capabilities;
the level of our sales and marketing expenses; and
our ability to introduce and sell new products.
The extent to which we utilize our existing equity purchase arrangement with Aspire Capital Fund LLC, or Aspire Capital, as a
source of funding will depend on a number of factors, including the prevailing market price of our common stock, the volume
of trading in our common stock and the extent to which we are able to secure funds from other sources. The number of shares
that we may sell to Aspire Capital under the purchase agreement on any given day and during the term of the agreement is
limited. Additionally, we and Aspire Capital may not affect any sales of shares of our common stock under the purchase
agreement during the continuance of an event of default. Even if we are able to access the over $166.9 million available under
the arrangement as of March 13, 2020, we will still need additional capital to fully implement our business, operating and
development plans.
We have secured capital historically from grant revenues, collaboration proceeds and debt and equity offerings. We will need to
secure substantial additional capital to fund our future operations. We cannot be certain that additional capital will be available
on acceptable terms or at all. To the extent we raise additional capital through the sale of equity securities, including to Aspire
Capital, the ownership position of our existing stockholders could be substantially diluted. If additional funds are raised
through the issuance of preferred stock or debt securities, these securities are likely to have rights, preferences and privileges
senior to our common stock. Fluctuating interest rates could also increase the costs of any debt financing we may obtain.
Importantly, we are approaching near-term milestones, including the results of Healios’ clinical trials, followed by the results of
our MASTERS-2 clinical trial, which we would expect to have a significant impact, favorable or unfavorable, on our ability to
access capital from potential third-party commercial partners or the equity capital markets, for example. Depending on the
outcome of these milestones, we may accelerate or may delay certain programs. In the longer term, we will have to continue to
generate additional capital to meet our needs until we would become cash flow positive as a result of the sales of our clinical
products, if they are approved for marketing.
Failure to successfully address ongoing liquidity requirements will have a material adverse effect on our business. If we are
unable to obtain additional capital on acceptable terms when needed, we may be required to take actions that harm our business
and our ability to achieve cash flow in the future, including possibly the surrender of our rights to some technologies or product
opportunities, delaying our clinical trials or curtailing or ceasing operations.
We are heavily dependent on the successful development and commercialization of MultiStem products, and if we encounter
delays or difficulties in the development of these product candidates, our business could be harmed.
Our success is heavily dependent upon the successful development of MultiStem products for certain diseases and conditions
involving acute or ischemic injury or immune system dysfunction. Our business could be materially harmed if we encounter
difficulties in the development of this product candidate, such as:
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delays in the ability to manufacture the product in quantities or in a form that is suitable for any required
preclinical studies or clinical trials;
an inability to produce the product at an appropriate cost or to scale for commercialization;
delays in the design, enrollment, implementation or completion of required preclinical studies and clinical
trials;
an inability to follow our current development strategy for obtaining regulatory approval from regulatory
authorities because of changes in the regulatory approval process;
less than desired or complete lack of efficacy or safety in preclinical studies or clinical trials; and
intellectual property constraints that prevent us from making, using or commercializing the product candidate.
Our product candidates are currently in the development stage and we have no therapeutic products approved for sale. If we
are unable to develop, obtain regulatory approval or market any of our product candidates, our financial condition will be
negatively affected, and we may have to curtail or cease our operations.
Many factors, known and unknown, can adversely affect clinical trials and the ability to evaluate a product’s efficacy. During
the course of treatment, patients can die or suffer other adverse events for reasons that may or may not be related to the
proposed product being tested. Even if unrelated to our product, certain events can nevertheless adversely impact our clinical
trials. As a result, our ability to ultimately develop and market the products and obtain revenues would suffer.
Even promising results in preclinical studies and initial clinical trials do not ensure successful results in later clinical trials,
which test broader human use of our products. Many companies in our industry have suffered significant setbacks in advanced
clinical trials, despite promising results in earlier trials.
We are in the early stage of product development, and we are dependent on the application of our technologies to discover or
develop therapeutic product candidates. We currently do not sell any approved therapeutic products and do not expect to have
any products commercially available for several years, if at all. You must evaluate us in light of the uncertainties and
complexities affecting an early stage biotechnology company. Our product candidates require additional research and
development, preclinical testing, clinical testing and regulatory review and/or approvals or clearances before marketing. To
date, no one to our knowledge has commercialized any therapeutic products using our technologies and we might never
commercialize any product using our technologies and strategy. In addition, we may not succeed in developing new product
candidates as an alternative to our existing portfolio of product candidates. If our current product candidates are delayed or fail,
or we fail to successfully develop and commercialize new product candidates, our financial condition may be negatively
affected, and we may have to curtail or cease our operations.
We may not successfully maintain our existing collaborative and licensing arrangements, or establish new ones, which
could adversely affect our ability to develop and commercialize our product candidates.
A key element of our business strategy is to commercialize some of our product candidates through collaborations with other
companies. Our strategy includes establishing collaborations and licensing agreements with one or more pharmaceutical,
biotechnology or device companies, preferably after we have advanced product candidates through the initial stages of clinical
development. However, we may not be able to establish or maintain such licensing and collaboration arrangements necessary to
develop and commercialize our product candidates. Even if we are able to maintain or establish licensing or collaboration
arrangements, these arrangements may not be on favorable terms and may contain provisions that will restrict our ability to
develop, test and market our product candidates. Any failure to maintain or establish licensing or collaboration arrangements on
favorable terms could adversely affect our business prospects, financial condition or ability to develop and commercialize our
product candidates.
Our agreements with our collaborators and licensees may have provisions that give rise to disputes regarding the rights and
obligations of the parties. These and other possible disagreements could lead to termination of the agreement or delays in
collaborative research, development, supply, or commercialization of certain product candidates, or could require or result in
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litigation or arbitration. Moreover, disagreements could arise with our collaborators over rights to intellectual property or our
rights to share in any of the future revenues of products developed by our collaborators. These kinds of disagreements could
result in costly and time-consuming litigation. Any such conflicts with our collaborators could reduce our ability to obtain
future collaboration agreements and could have a negative impact on our relationship with existing collaborators.
Currently, our material collaboration and licensing arrangement is with Healios to develop and commercialize MultiStem cell
therapy for the treatment of ischemic stroke and ARDS in Japan, among other things, and we also have license agreements with
third parties pursuant to which we in-license certain aspects of our technologies. These arrangements may not have specific
termination dates; rather, each arrangement terminates upon the occurrence of certain events.
If our collaborators do not devote sufficient time and resources to successfully carry out their contracted duties or meet
expected deadlines, we may not be able to advance our product candidates in a timely manner or at all.
Our success depends on the performance by our collaborators of their responsibilities under our collaboration arrangements.
Some potential collaborators may not perform their obligations in a timely fashion or in a manner satisfactory to us. Typically,
we cannot control the amount of resources or time our collaborators may devote to our programs or potential products that may
be developed in collaboration with us. We are currently involved in multiple research and development collaborations with
academic and research institutions. These collaborators frequently depend on outside sources of funding to conduct or complete
research and development, such as grants or other awards. In addition, our academic collaborators may depend on graduate
students, medical students, or research assistants to conduct certain work, and such individuals may not be fully trained or
experienced in certain areas, or they may elect to discontinue their participation in a particular research program, creating an
inability to complete ongoing research in a timely and efficient manner. As a result of these uncertainties, we are unable to
control the precise timing and execution of any experiments that may be conducted.
Additionally, our current or future corporate collaborators will retain the ability to pursue other research, product development
or commercial opportunities that may be directly competitive with our programs. If these collaborators elect to prioritize or
pursue other programs in lieu of ours, we may not be able to advance product development programs in an efficient or effective
manner, if at all. If a collaborator is pursuing a competitive program and encounters unexpected financial or capability
limitations, they may be motivated to reduce the priority placed on our programs or delay certain activities related to our
programs or be unwilling to properly fund their share of the development expenses for our programs. Any of these
developments could harm our product and technology development efforts, which could seriously harm our business.
We may experience delays in clinical trials and regulatory approval relating to our products that could adversely affect our
financial results and our commercial prospects for our pharmaceutical or stem cell products.
In addition to the regulatory requirements for our pharmaceutical programs, we will also require regulatory approvals for each
distinct application of our MultiStem product. In each case, we will be required to conduct clinical trials to demonstrate safety
and efficacy of MultiStem, or various products that incorporate or use MultiStem. For product candidates that advance to
clinical testing, we cannot be certain that we or a collaborator will successfully complete the clinical trials necessary to receive
regulatory product approvals. This process is lengthy and expensive.
We intend to seek approval for our product candidates through the FDA approval process in the United States, and through
other regulatory agencies outside the United States. To obtain regulatory approvals, we must, among other requirements,
complete clinical trials showing that our products are safe and effective for a particular indication. Under the approval process,
we must submit clinical and non-clinical data to demonstrate the product is safe and effective. For example, we must be able to
provide data and information, which may include extended pharmacology, toxicology, reproductive toxicology, bioavailability
and genotoxicity studies, to establish suitability for late stage clinical trials.
All of our product candidates are in clinical development. As these programs progress through clinical development, or
complete additional non-clinical testing, an indication of a lack of safety or lack of efficacy may result in the early termination
of an ongoing study, or may cause us or any of our collaborators to forego further development of a particular product
candidate or program. The FDA or other regulatory agencies may require extensive clinical trials or other testing prior to
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granting approval, which could be costly and time consuming to conduct. Any of these developments could hinder, and
potentially prohibit, our ability to commercialize our product candidates. We cannot assure you that clinical trials will
demonstrate that our products are safe and effective.
Additionally, we may not be able to find acceptable patients or may experience delays in enrolling patients for our currently
planned or any future clinical trials. The FDA, international regulatory agencies or we may suspend our clinical trials at any
time if it is believed that we are exposing the subjects participating in the trials to unacceptable health risks. The regulatory
authorities or institutional review boards and/or institutional biosafety committees at the medical institutions and healthcare
facilities where we seek to sponsor clinical trials may not permit a trial to proceed or may suspend any trial indefinitely if they
find deficiencies in the conduct of the trials.
Product development costs to us and our potential collaborators will increase if we have delays in testing or approvals or if we
need to perform more or larger clinical trials than planned. We expect to continue to rely on third-party clinical investigators at
medical institutions and healthcare facilities to conduct our clinical trials, and, as a result, we may face additional delaying
factors outside our control. Significant delays may adversely affect our financial results and the commercial prospects for our
product candidates and delay our ability to become profitable.
The results seen in animal testing of our product candidates may not be replicated in humans.
Safety and efficacy seen in preclinical testing of our product candidates in animals may not be seen when our product
candidates undergo clinical testing in humans. Preclinical studies and Phase 1 clinical trials are not primarily designed to test
the efficacy of a product candidate in humans, but rather to:
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test short-term safety and tolerability;
study the absorption, distribution, metabolism and elimination of the product candidate;
study the biochemical and physiological effects of the product candidate and the mechanisms of the drug action
and the relationship between drug levels and effect; and
understand the product candidate’s side effects at various doses and schedules.
Success in preclinical studies or completed clinical trials does not ensure that later studies or trials, including continuing non-
clinical studies and large-scale clinical trials, will be successful nor does it necessarily predict future results. The rate of failure
in drug development is quite high, and many companies in the biotechnology and pharmaceutical industries have suffered
significant setbacks in advanced clinical trials, even after promising results in earlier trials. Product candidates may fail to show
desired safety and efficacy in larger and more diverse patient populations in later stage clinical trials, despite having progressed
through early stage trials. Negative or inconclusive results from any of our ongoing preclinical studies or clinical trials could
result in delays, modifications, or abandonment of ongoing or future clinical trials and the termination of our development of a
product candidate. Additionally, even if we are able to successfully complete late stage clinical trials, the regulatory authorities
still may not approve our product candidates.
Even if we obtain regulatory approval of any of our product candidates, the approved products may be subject to post-
approval studies and will remain subject to ongoing regulatory requirements. If we fail to comply, or if concerns are
identified in subsequent studies, our approval could be withdrawn, and our product sales could be suspended.
If we are successful at obtaining regulatory approval for MultiStem or any of our other product candidates, regulatory agencies
in the United States and other countries where a product will be sold may require extensive additional clinical trials or post-
approval clinical studies that are expensive and time consuming to conduct. In particular, therapeutic products administered for
the treatment of persistent or chronic conditions, are likely to require extensive follow-up studies and close monitoring of
patients after regulatory approval has been granted, for any signs of adverse effects that occur over a long period of time. These
studies may be expensive and time consuming to conduct and may reveal side effects or other harmful effects in patients that
use our therapeutic products after they are on the market, which may result in the limitation or withdrawal of our drugs from
the market. Alternatively, we may not be able to conduct such additional trials, which might force us to abandon our efforts to
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develop or commercialize certain product candidates. Even if post-approval studies are not requested or required, after our
products are approved and on the market, there might be safety issues that emerge over time that require a change in product
labeling or that require withdrawal of the product from the market, which would cause our revenue to decline.
Additionally, any products that we may successfully develop will be subject to ongoing regulatory requirements after they are
approved. These requirements will govern the manufacturing, packaging, marketing, distribution, and use of our products. If we
fail to comply with such regulatory requirements, approval for our products may be withdrawn, and product sales may be
suspended. We may not be able to regain compliance, or we may only be able to regain compliance after a lengthy delay,
significant expense, lost revenues and damage to our reputation.
If we inadvertently violate the guidelines pertaining to promotion and advertising of our clinical candidates or approved
products, we may be subject to disciplinary action by the FDA’s Division of Drug Marketing, Advertising, and
Communications or other regulatory bodies.
The FDA’s Division of Drug Marketing, Advertising, and Communications, or DDMAC, is responsible for reviewing
prescription drug advertising and promotional labeling to ensure that the information contained in these materials is not false or
misleading. There are specific disclosure requirements and the applicable regulations mandate that advertisements cannot be
false or misleading or omit material facts about the product. Prescription drug promotional materials must present a fair balance
between the drug’s effectiveness and the risks associated with its use. Most warning letters from DDMAC cite inadequate
disclosure of risk information.
DDMAC prioritizes its actions based on the degree of risk to the public health, and often focuses on newly introduced drugs
and those associated with significant health risks. There are two types of letters that DDMAC typically sends to companies that
violate its drug advertising and promotional guidelines: notice of violation letters, or untitled letters, and warning letters. In the
case of an untitled letter, DDMAC typically alerts the drug company of the violation and issues a directive to refrain from
future violations but does not typically demand other corrective action. A warning letter is typically issued in cases that are
more serious or where the company is a repeat offender. Although we have not received any such letters from DDMAC, we
may inadvertently violate DDMAC’s guidelines in the future and be subject to a DDMAC untitled letter or warning letter,
which may have a negative impact on our business. Similarly, we and our collaborators may inadvertently violate the guidelines
of the foreign equivalent of the FDA's DDMAC, e.g., in Europe or Japan.
We rely on third parties to manufacture our MultiStem product candidate.
Our current business strategy relies on third parties to manufacture our MultiStem product candidates in accordance with GMP
established by the FDA or similar regulations in other countries. Reliance on third-party manufacturers entails risks to which
we would not be subject if we manufactured MultiStem ourselves. Although we are primarily responsible for regulatory
compliance with respect to the manufacture of MultiStem product, we rely on third parties to manufacture the product as cost
effectively as possible and to ensure product quality. Additionally, the production of our MultiStem product requires the
availability of raw materials that are sourced through a limited number of suppliers. The failure of third-party manufacturers or
suppliers to perform adequately or the termination of our arrangements with any of them may adversely affect our business.
These third parties may not deliver sufficient quantities of our MultiStem product, manufacture MultiStem product in
accordance with specifications and cost expectations or comply with applicable government regulations. From time to time,
such third-party manufacturers, or their material suppliers, may experience production delays, stoppages or interruptions in
supply, which may affect the initiation, execution and timing of completion of clinical trials and commercial
activities. Furthermore, our third-party manufacturers may have disruptions in their business operations as a result of business
or strategic decisions or due to economic difficulties facing their businesses, cybersecurity incidents, terrorist activity, public
health crises (such as coronavirus), fires or other natural disasters and could cease operations entirely. The number of third-
party manufacturers with the necessary manufacturing and regulatory expertise and facilities is limited, and it could be
expensive and take a significant amount of time to arrange for alternative manufacturing arrangements.
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If and until we can manufacture our products ourselves, we expect to enter into additional manufacturing agreements for the
production of our products. If any manufacturing agreement is terminated or any third-party collaborator fails to meet our
product specifications or experiences a significant problem that could result in a delay or interruption in the supply of product
materials to us, our clinical trials, business and reputation could be severely impacted. We cannot assure you that manufacturers
on whom we will depend will be able to successfully produce our MultiStem product on commercially acceptable terms, or on
a timely or cost-effective basis. We cannot assure you that manufacturers will be able to manufacture our products in
accordance with our product specifications or will meet regulatory or other requirements. We must have sufficient and
acceptable quantities of our product materials to conduct our clinical trials and ultimately to market our products, if and when
such products have been approved for marketing. If we are unable to obtain sufficient and acceptable quantities of our product,
we may be required to delay the clinical testing and marketing of our products.
If we do not comply with applicable regulatory requirements in the manufacture and distribution of our product candidates,
we may incur penalties that may inhibit our ability to commercialize our products and adversely affect our revenue.
Our failure or the failure of our potential collaborators or third-party manufacturers to comply with applicable FDA or other
regulatory requirements including manufacturing, quality control, labeling, safety surveillance, promoting and reporting may
result in criminal prosecution, civil penalties, recall or seizure of our products, total or partial suspension of production or an
injunction, as well as other regulatory action against our product candidates or us. Discovery of previously unknown problems
with a product, supplier, manufacturer or facility may result in restrictions on the sale of our products, including a withdrawal
of such products from the market. The occurrence of any of these events would negatively impact our business and results of
operations.
If we are unable to attract and retain key personnel and advisors, it may adversely affect our ability to obtain financing,
pursue collaborations or develop our product candidates.
We are highly dependent on our senior executives such as Gil Van Bokkelen, Ph.D., our Chief Executive Officer and Chairman,
William Lehmann, J.D., M.B.A., President and Chief Operating Officer, John Harrington, Ph.D., Executive Vice President and
Chief Scientific Officer, Ivor Macleod, M.B.A, CPA, Chief Financial Officer, and Laura Campbell, CPA, Senior Vice President
of Finance, as well as other personnel.
These individuals are integral to the development and integration of our technologies and to our present and future scientific
collaborations, including managing the complex research processes and the product development and potential
commercialization processes. Given their leadership, extensive technical, scientific and financial expertise and management
and operational experience, these individuals would be difficult to replace. Consequently, the loss of services of one or more of
these named individuals could result in product development delays or the failure of our collaborations with current and future
collaborators, which, in turn, may hurt our ability to develop and commercialize products and generate revenues.
Our future success depends on our ability to attract, retain and motivate highly qualified management and scientific,
development and commercial personnel and advisors. If we are unable to attract and retain key personnel and advisors, it may
negatively affect our ability to successfully develop, test and commercialize our product candidates.
Our ability to compete may decline if we are not successful in adequately protecting our patented and other proprietary
technologies.
Our success depends in part on our ability to obtain and maintain intellectual property that protects our technologies and our
products. Patent positions may be highly uncertain and may involve complex legal and factual questions, including the ability
to establish patentability of compounds and methods for using them for which we seek patent protection. We cannot predict the
breadth of claims that will ultimately be allowed in our patent applications, if any, including those we have in-licensed or the
extent to which we may enforce these claims against our competitors. We have filed multiple patent applications that seek to
protect the composition of matter and method of use related to our programs. In addition, we are prosecuting numerous distinct
patent families directed to composition, methods of production and methods of use of MultiStem and related technologies. If
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we are unsuccessful in obtaining and maintaining these patents related to products and technologies, we may ultimately be
unable to commercialize products that we are developing or may elect to develop in the future.
The degree of future protection for our proprietary rights is therefore highly uncertain and we cannot assure you that:
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we were the first to file patent applications or to invent the subject matter claimed in patent applications relating
to the technologies or product candidates upon which we rely;
others will not independently develop similar or alternative technologies or duplicate any of our technologies;
others did not publicly disclose our claimed technology before we conceived the subject matter included in any
of our patent applications;
any of our pending or future patent applications will result in issued patents;
any of our patent applications will not result in interferences or disputes with third parties regarding priority of
invention;
any patents that may be issued to us, our collaborators or our licensors will provide a basis for commercially
viable products or will provide us with any competitive advantages or will not be challenged by third parties;
we will develop additional proprietary technologies that are patentable;
the patents of others will not have an adverse effect on our ability to do business; or
new proprietary technologies from third parties, including existing licensors, will be available for licensing to
us on reasonable commercial terms, if at all.
In addition, patent law outside the United States is uncertain and, in many countries, intellectual property laws are undergoing
review and revision. The laws of some countries do not protect intellectual property rights to the same extent as domestic laws.
It may be necessary or useful for us to participate in opposition proceedings to determine the validity of our competitors’
patents or to defend the validity of any of our or our licensor’s future patents, which could result in substantial costs and would
divert our efforts and attention from other aspects of our business. With respect to certain of our inventions, we decided not to
pursue patent protection outside the United States, both because we do not believe it is cost effective and because of
confidentiality concerns. Accordingly, our international competitors could develop and receive foreign patent protection for
gene sequences and functions for which we are seeking United States patent protection, enabling them to sell products that we
developed.
Technologies licensed to us by others, or in-licensed technologies, are important to our business. The scope of our rights under
our licenses may be subject to dispute by our licensors or third parties. Our rights to use these technologies and to practice the
inventions claimed in the licensed patents are subject to our licensors abiding by the terms of those licenses and not terminating
them. In particular, we depend on certain technologies relating to our MultiStem technology licensed from the University of
Minnesota, and the termination of this license could result in our loss of some of the rights that enable us to utilize this
technology, and our ability to develop products based on MultiStem could be seriously hampered.
In addition, we may in the future acquire rights to additional technologies by licensing such rights from existing licensors or
from third parties. Such in-licenses may be costly. Also, we generally do not control the patent prosecution, maintenance or
enforcement of in-licensed technologies. Accordingly, we are unable to exercise the same degree of control over this
intellectual property as we do over our internally developed technologies. Moreover, some of our academic institution
licensors, collaborators and scientific advisors have rights to publish data and information to which we have rights. If we
cannot maintain the confidentiality of our technologies and other confidential information in connection with our
collaborations, our ability to protect our proprietary information or obtain patent protection in the future may be impaired,
which could have a significant adverse effect on our business, financial condition and results of operations.
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We may not have adequate protection for our unpatented proprietary information, which could adversely affect our
competitive position.
In addition to patents, we will substantially rely on trade secrets, know-how, continuing technological innovations and licensing
opportunities to develop and maintain our competitive position. However, others may independently develop substantially
equivalent proprietary information and techniques or otherwise gain access to our trade secrets or disclose our technology. To
protect our trade secrets, we may enter into confidentiality agreements with employees, consultants and potential collaborators.
However, these agreements may not provide meaningful protection of our trade secrets or adequate remedies in the event of
unauthorized use or disclosure of such information. Likewise, our trade secrets or know-how may become known through other
means or be independently discovered by our competitors. Any of these events could prevent us from developing or
commercializing our product candidates.
We may be sued for product liability, which could adversely affect our business.
Because our business strategy involves the development and sale by either us or our collaborators of commercial products, we
may be sued for product liability. We may be held liable if any product we develop and commercialize, or any product our
collaborators commercialize that incorporates any of our technology, causes injury or is found otherwise unsuitable during
product testing, manufacturing, marketing, sale or consumer use. In addition, the safety studies we must perform, and the
regulatory approvals required to commercialize our pharmaceutical products, will not protect us from any such liability.
We carry product liability insurance that includes coverage for human clinical trials. Currently, we insure a total limit of
$15 million per occurrence, $15 million annual aggregate coverage for both our products liability policy and our clinical trials
protection. This limit is comprised of both primary and excess coverage. We also intend to seek product liability insurance for
any approved products that we may develop or acquire. However, in the event there are product liability claims against us, our
insurance may be insufficient to cover the expense of defending against such claims or may be insufficient to pay or settle such
claims. Furthermore, we may be unable to obtain adequate product liability insurance coverage for commercial sales of any of
our approved products. If such insurance is insufficient to protect us, our results of operations will suffer. If any product
liability claim is made against us, our reputation and future sales will be damaged, even if we have adequate insurance
coverage.
Many potential competitors, including those who have greater resources and experience than we do, may develop products
or technologies that make ours obsolete or noncompetitive.
We face significant competition with respect to our product candidates. With regard to our efforts to develop MultiStem as a
novel stem cell therapy, currently, there are a number of companies that are actively developing stem cell products, which
encompass a range of different cell types, including embryonic stem cells, adult-derived stem cells, and processed bone marrow
derived cells. Our future success will depend on our ability to maintain a competitive position with respect to technological
advances. Technological developments by others may result in our MultiStem product platform and technologies, as well as our
pharmaceutical formulations, becoming obsolete.
We are subject to significant competition from pharmaceutical, biotechnology and diagnostic companies, academic and
research institutions, and government or other publicly funded agencies that are pursuing or may pursue the development of
therapeutic products and technologies that are substantially similar to our proposed therapeutic products and technologies, or
that otherwise address the indications we are pursuing. Our most significant competitors include major pharmaceutical
companies such as Pfizer, Roche Holding AG, Johnson & Johnson, Sanofi S.A. and GlaxoSmithKline plc, as well as smaller
biotechnology or biopharmaceutical companies such as Celgene, Mesoblast, SanBio, Cytori and Pluristem. Most of our current
and potential competitors have substantially greater research and development capabilities and financial, scientific, regulatory,
manufacturing, marketing, sales, human resources and experience than we do. Many of our competitors have several
therapeutic products that have already been developed, approved and successfully commercialized, or are in the process of
obtaining regulatory approval for their therapeutic products in the United States and internationally.
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Many of these companies have substantially greater capital resources, research and development resources and experience,
manufacturing capabilities, regulatory expertise, sales and marketing resources, established relationships with consumer
products companies and production facilities.
Universities and public and private research institutions are also potential competitors. While these organizations primarily
have educational objectives, they may develop proprietary technologies related to stem cells or secure patent protection that we
may need for the development of our technologies and products. We may attempt to license these proprietary technologies, but
these licenses may not be available to us on acceptable terms, if at all. Our competitors, either alone or with their collaborative
partners, may succeed in developing technologies or products that are more effective, safer, more affordable or more easily
commercialized than ours, and our competitors may obtain intellectual property protection or commercialize products sooner
than we do. Developments by others may render our product candidates or our technologies obsolete.
Our current product discovery and development collaborators are not prohibited from entering into research and development
collaboration agreements with third parties in any product field. Our failure to compete effectively would have a significant
adverse effect on our business, financial condition and results of operations.
The availability, manner, and amount of reimbursement for our product candidates from government and private payers are
uncertain, and our inability to obtain adequate reimbursement for any products could severely limit our product sales.
We expect that many of the patients who seek treatment with any of our products that are approved for marketing will be
eligible for Medicare benefits. Other patients may be covered by private health plans. If we are unable to obtain or retain
adequate levels of reimbursement from Medicare or from private health plans, our ability to sell our products will be severely
limited. The application of existing Medicare regulations and interpretive coverage and payment determinations to newly
approved products is uncertain and those regulations and interpretive determinations are subject to change. Medicare may
change its reimbursement methodology that reduces the Medicare reimbursement rates for many drugs, which may adversely
affect reimbursement for any products we may develop. Medicare regulations and interpretive determinations also may
determine who may be reimbursed for certain services and may limit the pool of patients our product candidates are being
developed to serve.
Our industry is highly regulated and changes in law may adversely impact our business, operations or financial results. We
anticipate continuing debate in the foreseeable future over the research and development, marketing, pricing and
reimbursement for health care products and services, including those that would affect our current product candidates. For
example, federal, state and foreign governments continue to propose legislation designed to contain or reduce health care costs.
Legislation and regulations affecting the pricing of products like our potential products may change further or be adopted
before any of our potential products are approved for marketing. Cost control initiatives by governments or third-party payers
could decrease the price that we receive for any one or all of our potential products or increase patient coinsurance to a level
that make our products under development become unaffordable. In addition, government and private health plans persistently
challenge the price and cost-effectiveness of therapeutic products. Accordingly, these third parties may ultimately not consider
any or all of our products under development to be cost effective, which could result in products not being covered under their
health plans or covered only at a lower price. Any of these initiatives or developments could prevent us from successfully
marketing and selling any of our products that are approved for commercialization.
Public perception of ethical and social issues surrounding the use of adult-derived stem cell technology may limit or
discourage the use of our technologies, which may reduce the demand for our therapeutic products and technologies and
reduce our revenues.
Our success will depend in part upon our ability to develop therapeutic products incorporating or discovered through our adult-
derived stem cell technology. For social, ethical, or other reasons, governmental authorities in the United States and other
countries may call for limits on, or regulation of the use of, adult-derived stem cell technologies. Although we do not use the
more controversial stem cells derived from embryos or fetuses, claims that adult-derived stem cell technologies are ineffective,
unethical or pose a danger to the environment may influence public attitudes. The subject of stem cell technologies in general
has received negative publicity and aroused public debate in the United States and some other countries. Ethical and other
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concerns about our adult-derived stem cell technology could materially hurt the market acceptance of our therapeutic products
and technologies, resulting in diminished sales and use of any products we are able to develop using adult-derived stem cells.
Even if we or our collaborators receive regulatory approval for our products, those products may never be commercially
successful.
Even if we develop pharmaceuticals or MultiStem-related products that obtain the necessary regulatory approval, and we have
access to the necessary manufacturing, sales, marketing and distribution capabilities that we need, our success depends to a
significant degree upon the commercial success of those products. If these products fail to achieve or subsequently maintain
market acceptance or commercial viability, our business would be significantly harmed because our future royalty revenue or
other revenue would be dependent upon sales of these products. Many factors may affect the market acceptance and
commercial success of any potential products that we may discover, including:
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health concerns, whether actual or perceived, or unfavorable publicity regarding our stem cell products or those
of our competitors;
the timing of market entry as compared to competitive products;
the rate of adoption of products by our collaborators and other companies in the industry;
any product labeling that may be required by the FDA or other United States or foreign regulatory agencies for
our products or competing or comparable products;
convenience and ease of administration;
pricing;
perceived efficacy and side effects;
marketing;
availability of alternative treatments;
levels of reimbursement and insurance coverage; and
activities by our competitors.
If we are unable to create and maintain sales, marketing and distribution capabilities or enter into agreements with third
parties to perform those functions, we will not be able to commercialize our product candidates.
We currently have no sales, marketing or distribution capabilities. Therefore, to commercialize our product candidates, if and
when such products have been approved and are ready for marketing, we expect to collaborate with third parties to perform
these functions. We will either need to share the value generated from the sale of any products and/or pay a fee to the contract
sales organization. If we establish any such relationships, we will be dependent upon the capabilities of our collaborators or
contract service providers to effectively market, sell, and distribute our product. If they are ineffective at selling and distributing
our product, or if they choose to emphasize other products over ours, we may not achieve the level of product sales revenues
that we would like. If conflicts arise, we may not be able to resolve them easily or effectively, and we may suffer financially as
a result. If we cannot rely on the sales, marketing and distribution capabilities of our collaborators or of contract service
providers, we may be forced to establish our own capabilities. We have no experience in developing, training or managing a
sales force and will incur substantial additional expenses if we decide to market any of our future products directly. Developing
a marketing and sales force is also time consuming and could delay launch of our future products. In addition, we will compete
with many companies that currently have extensive and well-funded marketing and sales operations. Our marketing and sales
efforts may be unable to compete successfully against these companies.
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We will use hazardous and biological materials in our business. Any claims relating to improper handling, storage or
disposal of these materials could be time consuming and costly.
Our products and processes will involve the controlled storage, use and disposal of certain hazardous and biological materials
and waste products. We and our suppliers and other collaborators are subject to federal, state and local regulations governing
the use, manufacture, storage, handling and disposal of materials and waste products. Even if we and these suppliers and
collaborators comply with the standards prescribed by law and regulation, the risk of accidental contamination or injury from
hazardous materials cannot be completely eliminated. In the event of an accident, we could be held liable for any damages that
result, and any liability could exceed the limits or fall outside the coverage of any insurance we may obtain and exceed our
financial resources. We may not be able to maintain insurance on acceptable terms, or at all. We may incur significant costs to
comply with current or future environmental laws and regulations.
Disputes concerning the infringement or misappropriation of our proprietary rights or the proprietary rights of others could
be time consuming and extremely costly and could delay our research and development efforts.
Our commercial success, if any, will be significantly harmed if we infringe the patent rights of third parties or if we breach any
license or other agreements that we entered into with regard to our technology or business.
We are aware of other companies and academic institutions that have been performing research in the areas of adult-derived
stem cells. In particular, other companies and academic institutions have announced that they have identified nonembryonic
stem cells isolated from bone marrow or other tissues that have the ability to form a range of cell types or display the property
of pluripotency. To the extent any of these companies or academic institutions currently have, or obtain in the future, broad
patent claims, such patents could block our ability to use various aspects of our discovery and development process and might
prevent us from developing or commercializing newly discovered applications of our MultiStem technology, or otherwise
conducting our business. In addition, it is possible that some of the pharmaceutical product candidates we are developing may
not be patentable or may be covered by intellectual property of third parties. For example, over the past several years, we were
involved in proceedings in the United States and Europe with a third-party focused on a technology developed after the MAPC
technology. Ultimately, we reached a settlement agreement with and obtained a license from this third-party, positioning us
advantageously with respect to the achievement of our business objectives. Over time, we expect to be involved in similar
proceedings with the objective of developing the portfolio to support and protect development and commercialization of our or
our licensees’ cell therapy products.
We are not currently a party to any litigation with regard to our patent or trademark positions. However, the life sciences and
other technology industries are characterized by extensive litigation regarding patents and other intellectual property rights.
Many life sciences and other technology companies have employed intellectual property litigation as a way to gain a
competitive advantage. To the extent we are involved in litigation, interference proceedings, oppositions, reexamination, protest
or other potentially adverse intellectual property proceedings as a result of alleged infringement by us of the rights of others or
as a result of priority of invention disputes with third parties, we might have to spend significant amounts of money, time and
effort defending our position and we may not be successful. In addition, any claims relating to the infringement of third-party
proprietary rights or proprietary determinations, even if not meritorious, could result in costly litigation, lengthy governmental
proceedings, divert management’s attention and resources, or require us to enter into royalty or license agreements that are not
advantageous to us. If we do not have the financial resources to support such litigation or appeals, we may forfeit or lose
certain commercial rights. Even if we have the financial resources to continue such litigation or appeals, we may lose. In the
event that we lose, we may be forced to pay very substantial damages; we may have to obtain costly license rights, which may
not be available to us on acceptable terms, if at all; or we may be prohibited from selling products that are found to infringe the
patent rights of others.
Should any person have filed patent applications or obtained patents that claim inventions also claimed by us, we may have to
participate in an interference proceeding declared by the relevant patent regulatory agency to determine priority of invention
and, thus, the right to a patent for these inventions in the United States. Such a proceeding could result in substantial cost to us
even if the outcome is favorable. Even if successful on priority grounds, an interference action may result in loss of claims
based on patentability grounds raised in the interference action. Litigation, interference proceedings or other proceedings could
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divert management’s time and efforts. Even unsuccessful claims could result in significant legal fees and other expenses,
diversion of management’s time and disruption in our business. Uncertainties resulting from initiation and continuation of any
patent proceeding or related litigation could harm our ability to compete and could have a significant adverse effect on our
business, financial condition and results of operations.
An adverse ruling arising out of any intellectual property dispute, including an adverse decision as to the priority of our
inventions, could undercut or invalidate our intellectual property position. An adverse ruling could also subject us to significant
liability for damages, including possible treble damages, prevent us from using technologies or developing products, or require
us to negotiate licenses to disputed rights from third parties. Although patent and intellectual property disputes in the
technology area are often settled through licensing or similar arrangements, costs associated with these arrangements may be
substantial and could include license fees and ongoing royalties. Furthermore, necessary licenses may not be available to us on
satisfactory terms, if at all. Failure to obtain a license in such a case could have a significant adverse effect on our business,
financial condition and results of operations.
To the extent we enter markets outside of the United States, our business will be subject to political, economic, legal and
social risks in those markets, which could adversely affect our business.
There are significant regulatory and legal barriers in markets outside the United States that we must overcome to the extent we
enter or attempt to enter markets in countries other than the United States. We will be subject to the burden of complying with a
wide variety of national and local laws, including multiple and possibly overlapping and conflicting laws. We also may
experience difficulties adapting to new cultures, business customs and legal systems. Any sales and operations outside the
United States would be subject to political, economic and social uncertainties including, among others:
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changes and limits in import and export controls;
increases in custom duties and tariffs;
changes in currency exchange rates;
economic and political instability;
changes in government regulations and laws;
absence in some jurisdictions of effective laws to protect our intellectual property rights; and
currency transfer and other restrictions and regulations that may limit our ability to sell certain products or
repatriate profits to the United States.
Any changes related to these and other factors could adversely affect our business to the extent we enter markets outside the
United States.
Foreign governments often impose strict price controls on approved products, which may adversely affect our future
profitability in those countries, and the re-importation of drugs to the United States from foreign countries that impose price
controls may adversely affect our future profitability.
Frequently foreign governments impose strict price controls on newly approved therapeutic products. If we obtain regulatory
approval to sell products in foreign countries, we may be unable to obtain a price that provides an adequate financial return on
our investment. Furthermore, legislation in the United States may permit re-importation of drugs from foreign countries into the
United States, including re-importation from foreign countries where the drugs are sold at lower prices than in the United States
due to foreign government-mandated price controls. Such a practice, especially if it is conducted on a widespread basis, may
significantly reduce our potential United States revenues from any drugs that we are able to develop.
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If we elect not to sell our products in foreign countries that impose government mandated price controls because we decide
it is uneconomical to do so, a foreign government or patent office may attempt to terminate our intellectual property rights
in that country, enabling competitors to make and sell our products.
In some cases, we may choose not to sell a product in a foreign country because it is uneconomical to do so under a system of
government-imposed price controls, or because it could severely limit our profitability in the United States or other markets. In
such cases, a foreign government or patent office may terminate any intellectual property rights we may obtain with respect to
that product. Such a termination could enable competitors to produce and sell our product in that market. Furthermore, such
products may be exported into the United States through legislation that authorizes the importation of drugs from outside the
United States. In such an event, we may have to reduce our prices, or we may be unable to compete with low-cost providers of
our drugs, and we could be financially harmed as a result.
We may encounter difficulties managing our growth, which could adversely affect our business.
At various times we have experienced periods of rapid growth in our employee numbers as a result of a dramatic increase in
activity in technology programs, genomics programs, collaborative research programs, discovery programs, and scope of
operations. At other times, we had to reduce staff in order to bring our expenses in line with our financial resources. Our
success will also depend on the ability of our officers and key employees to continue to improve our operational capabilities
and our management information and financial control systems, and to expand, train and manage our work force.
If we acquire products, technologies or other businesses, we will incur a variety of costs, may have integration difficulties
and may experience numerous other risks that could adversely affect our business.
To remain competitive, we may decide to acquire additional businesses, products and technologies. We currently have no
commitments or agreements with respect to, and are not actively seeking, any material acquisitions. We have limited
experience in identifying acquisition targets, successfully acquiring them and integrating them into our current infrastructure.
We may not be able to successfully integrate any businesses, products, technologies or personnel that we might acquire in the
future without a significant expenditure of operating, financial and management resources, if at all. In addition, future
acquisitions could require significant capital infusions and could involve many risks, including, but not limited to the
following:
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we may have to issue convertible debt or equity securities to complete an acquisition, which would dilute our
stockholders and could adversely affect the market price of our common stock;
an acquisition may negatively impact our results of operations because it may require us to incur large one-time
charges to earnings, amortize or write down amounts related to goodwill and other intangible assets, or incur or
assume substantial debt or liabilities, or it may cause adverse tax consequences, substantial depreciation or
deferred compensation charges;
we may encounter difficulties in assimilating and integrating the business, technologies, products, personnel or
operations of companies that we acquire;
certain acquisitions may disrupt our relationship with existing collaborators who are competitive to the acquired
business;
acquisitions may require significant capital infusions and the acquired businesses, products or technologies may
not generate sufficient revenue to offset acquisition costs;
an acquisition may disrupt our ongoing business, divert resources, increase our expenses and distract our
management;
acquisitions may involve the entry into a geographic or business market in which we have little or no prior
experience; and
key personnel of an acquired company may decide not to work for us.
Any of the foregoing risks could have a significant adverse effect on our business, financial condition and results of operations.
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Increased information technology security threats and more sophisticated and targeted computer crime could pose a risk to
our systems, networks, and products.
Increased global information technology security threats and more sophisticated and targeted computer crime pose a risk to the
security of our systems and networks and the confidentiality, availability and integrity of our data and communications. While
we attempt to mitigate these risks by employing a number of measures, including employee refreshers, monitoring of our
networks and systems, and maintenance of backup and protective systems, our systems, networks and products remain
potentially vulnerable to advanced persistent threats. Depending on their nature and scope, such threats could potentially lead to
the compromising of confidential information and communications, improper use of our systems and networks, manipulation
and destruction of data, defective products, production downtimes and operational disruptions, which in turn could adversely
affect our reputation, competitiveness and results of operations. Furthermore, we are subject to an increasing number of data
privacy and data protection laws in both the United States and abroad, including the EU's General Data Protection Regulation.
Failure to comply with these regulations could result in fines, penalties or significant legal liability.
We may not be able to utilize a significant portion of our net operating loss or research tax credit carryforwards or other tax
attributes, which could harm our profitability.
At December 31, 2019, we had U.S. federal net operating loss and research and development tax credit carryforwards of
approximately $193.2 million and $12.8 million, respectively. Such operating losses and tax credits may be used to reduce
future taxable income and tax liabilities and will expire at various dates between 2032 and 2039. Additionally, as of
December 31, 2019, we had federal net operating loss carryforwards generated after 2017 of $56.6 million that have an
indefinite life, but with usage limited to 80% of taxable income in any given year. We also had foreign net operating loss
carryforwards of approximately $24.2 million. Such foreign net operating loss carryforwards do not expire. We also had state
and city net operating loss carryforwards aggregating approximately $63.5 million. Such state and city net operating loss
carryforwards may be used to reduce future taxable income and tax liabilities and will expire at various dates between 2020 and
2037. Certain state net operating losses do not expire.
Our ability to utilize our U.S. federal net operating loss and tax credit carryforwards generated prior to October 2012 (the
“Section 382 Limited Attributes”) is substantially limited under Section 382 of the Internal Revenue Code of 1986, as
amended, or the Code, as a result of our equity offering that occurred in October 2012. Similar limitations may apply for state
and local tax purposes. We generated U.S. federal net operating loss carryforwards of $156.5 million, research and
development tax credits of $12.8 million, and state and local net operating loss carryforwards of $63.3 million since 2012
through December 31, 2019.
Our ability to utilize tax attributes, including those that are not part of the Section 382 Limited Attributes may also be limited if
we experience an “ownership change,” for purposes of Section 382 of the Code. A Section 382 “ownership change” generally
occurs if one or more stockholders or groups of stockholders who own at least 5% of our stock increase their ownership by
more than 50 percentage points over their lowest ownership percentage within a rolling three-year period. Similar rules may
apply under state tax laws. Sales of our common stock to Healios, Aspire Capital pursuant to our equity purchase arrangement,
in combination with other issuances or sales of our common stock (including any sales of common stock by Aspire Capital and
certain transactions involving our common stock that are outside of our control) could cause an “ownership change.” If an
“ownership change” occurs, Section 382 of the Code would impose an annual limit on the amount of pre-ownership change net
operating loss carryforwards and other tax attributes we can use to reduce our taxable income, potentially increasing and
accelerating our liability for income taxes, and also potentially causing those tax attributes to expire unused. It is possible that
such an ownership change could materially reduce our ability to use our net operating loss carryforwards or other tax attributes
to offset taxable income, which could harm our profitability. We will update our analysis under Section 382 of the Code prior to
using our tax attributes.
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If we do not continue to meet the listing standards established by The NASDAQ Capital Market, the common stock may not
remain listed for trading.
The NASDAQ Capital Market has established certain quantitative criteria and qualitative standards that companies must meet
in order to remain listed for trading on these markets. We cannot guarantee that we will be able to maintain all necessary
requirements for listing; therefore, we cannot guarantee that our common stock will remain listed for trading on The NASDAQ
Capital Market or other similar markets.
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ITEM 1B.
UNRESOLVED STAFF COMMENTS
Not applicable.
ITEM 2.
PROPERTIES
Our principal offices are located at 3201 Carnegie Avenue in Cleveland, Ohio. We currently lease approximately 45,000 square
feet of space for our corporate offices and laboratories, with state-of-the-art laboratory space. The lease began in 2000 and
currently expires in March 2021. Our rent is approximately $267,000 per year and our rental rate has not changed since the
lease inception in 2000. Also, we currently lease office and laboratory space for our Belgian subsidiary. With the extension
signed in March 2020, the lease currently expires in July 2021, and we have an option to renew annually through July 2022.
The annual rent in Belgium is approximately $190,000 and is subject to adjustments based on an inflationary index.
ITEM 3.
LEGAL PROCEEDINGS
From time to time, we may become subject to various legal proceedings that are incidental to the ordinary conduct of our
business. Currently, there are no such proceedings.
ITEM 3A.
INFORMATION ABOUT OUR EXECUTIVE OFFICERS
The information under this Item is furnished pursuant to the instructions to Item 401 of Regulation S-K.
The following sets forth the name, age, current position and principal occupation and employment during the past five years of
our executive officers.
Gil Van Bokkelen, Ph.D.
Age: 59
Dr. Van Bokkelen has served as our Chief Executive Officer and Chairman since August 2000. Dr. Van Bokkelen co-founded
Athersys in 1995 and has served as Chief Executive Officer and Director since the Company’s founding. Prior to May 2006, he
also served as the Company’s President. Dr. Van Bokkelen is also the Chairman of the Board of Governors for the National
Center for Regenerative Medicine. He serves on the board of the Alliance for Regenerative Medicine (and served as chairman
from 2010 through 2012), a Washington D.C. based consortium of companies, patient advocacy groups, disease foundations,
and clinical and research institutions that are committed to the advancement of the field of regenerative medicine, and served ex
officio from 2013 to 2014. He has served on a number of other boards, including the Biotechnology Innovation Organization’s
board of directors (from 2001 to 2004, and from 2008 to present). He received his Ph.D. in Genetics from Stanford University
School of Medicine, his B.A. in Economics from the University of California at Berkeley, and his B.A. in Molecular Biology
from the University of California at Berkeley.
John J. Harrington, Ph.D.
Age: 52
Dr. Harrington co-founded Athersys in 1995 and has served as our Executive Vice President, Chief Scientific Officer and
Director since our founding. Dr. Harrington led the development of the RAGE technology, as well as its application for gene
discovery, drug discovery and commercial protein production applications. He is a listed inventor on over 20 issued or pending
United States patents, has authored numerous scientific publications, and has received numerous awards for his work, including
being named one of the top international young scientists by MIT Technology Review in 2002. Dr. Harrington has overseen the
therapeutic product development programs at Athersys since its inception and is also focused on the clinical development and
manufacturing of MultiStem. During his career, he has also held positions at Amgen and Scripps Clinic. He received his B.A. in
Biochemistry and Cell Biology from the University of California at San Diego and his Ph.D. in Cancer Biology from Stanford
University.
40
William (B.J.) Lehmann, Jr., J.D.
Age: 54
Mr. Lehmann joined Athersys in September 2001 and has served as our President and Chief Operating Officer since June 2006.
Prior to that time, Mr. Lehmann was Athersys’ Executive Vice President of Corporate Development and Finance from August
2002 until June 2006, when he became Athersys’ President and Chief Operating Officer. From 1994 to 2001, Mr. Lehmann was
with McKinsey & Company, Inc., or McKinsey, an international management consulting firm, where he worked extensively
with new technology and service-based businesses in a variety of industries. Prior to joining McKinsey, he worked at Wilson,
Sonsini, Goodrich & Rosati, a Silicon Valley law firm, and worked with First Chicago Corporation, a financial institution.
Mr. Lehmann received his J.D. from Stanford University, his M.B.A. from the University of Chicago, and his B.A. from the
University of Notre Dame.
Ivor Macleod, CPA, MBA
Age:58
Mr. Macleod joined Athersys in January 2020 as our Chief Financial Officer. Previously he served as the Chief Financial
Officer and Chief Compliance Officer of Eisai Inc., the U.S. pharmaceutical subsidiary of Eisai Co., Ltd., a research-based
human health care company that discovers, develops and markets products globally, from 2015 to 2018. Prior to joining Eisai,
Mr. Macleod served as Vice President Finance - Merck Research Labs at Merck & Co., Inc., a global health care company that
delivers innovative health solutions through its prescription medicines, vaccines, biologic therapies and animal health
productions, from 2012 to 2015. Before joining Merck, Mr. Macleod served from 1998 to 2012 at F. Hoffmann-La Roche, Inc.,
a multinational health care company, in various roles, including as North American Chief Financial Officer from 2000 to 2011
and General Manager from 2010 to 2011. Mr. Macleod received his B.S. from St. Andrews University in Scotland and his
M.B.A. from the University of Arizona. Mr. Macleod is a Certified Public Accountant licensed in Virginia.
Laura K. Campbell, CPA
Age: 56
Ms. Campbell joined Athersys in January 1998 and has served as our Senior Vice President of Finance since March 2016.
Ms. Campbell joined us as Controller from January 1998, followed by Director of Finance and Senior Director of Finance, and
then served as our Vice President of Finance from June 2006 until March 2016. Prior to Athersys, she was at Ernst & Young
LLP, a public accounting firm, for eleven years in the firm’s audit practice. During her tenure with Ernst & Young LLP,
Ms. Campbell specialized in entrepreneurial services and the biotechnology industry sector and assisted in several initial public
offerings. Ms. Campbell received her B.S., with distinction, in Business Administration from The Ohio State University and is
a Certified Public Accountant.
ITEM 4.
MINE SAFETY DISCLOSURES
Not applicable.
41
PART II
ITEM 5.
MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER
MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES
Our common stock is traded on the NASDAQ Capital Market under the symbol “ATHX.”
Holders
As of February 29, 2020, there were approximately 516 holders of record of our common stock. Additionally, shares of
common stock are held by financial institutions as nominees for beneficial owners that are deposited into participant accounts
at the Depository Trust Company, which are held of record by Cede & Co. and are included in the holders of record as one
stockholder.
Unregistered Sales
Since 2011, we have had in place equity purchase agreements with Aspire Capital, which provide us the ability to sell shares of
our common stock to Aspire Capital from time-to-time. During the quarter ended December 31, 2019, we sold an aggregate of
4,000,000 shares of common stock to Aspire Capital under our equity purchase agreement, generating aggregate proceeds of
$5.1 million. Each issuance of these unregistered shares qualifies as an exempt transaction pursuant to Section 4(a)(2) of the
Securities Act of 1933. Each issuance qualified for exemption under Section 4(a)(2) of the Securities Act of 1933 because none
involved a public offering. Each offering was not a public offering due to the number of persons involved, the manner of the
issuance and the number of securities issued. In addition, in each case Aspire Capital had the necessary investment intent.
ITEM 6.
SELECTED FINANCIAL DATA
(in thousands, except per share data)
2019
2018
2017
2016
2015
Year Ended December 31,
Consolidated Statement of Operations Data:
Revenues:
Contract revenue (1)
Grant revenue
Total revenues
Costs and expenses:
Research and development
General and administrative
Depreciation
Total costs and expenses
Gain from insurance proceeds, net
Loss from operations
Other income (expense):
Income (expense) from change in fair
value of warrants
Other income (expense), net
Loss before income taxes
Income tax benefit
Net loss
Net loss per share, basic
Weighted average shares outstanding, basic
Net loss per share, diluted
Weighted average shares outstanding, diluted
$
$
$
$
5,517 $
116
5,633
23,737 $
554
24,291
2,843 $
865
3,708
16,238 $
1,109
17,347
39,045
11,378
698
51,121
—
(45,488 )
38,656
10,442
855
49,953
617
(25,045 )
27,841
8,466
684
36,991
—
(33,283 )
—
906
(44,582 )
—
(44,582 ) $
(0.29 ) $
151,696
—
762
(24,283 )
—
(24,283 ) $
(0.18 ) $
136,641
728
314
(32,241 )
—
(32,241 ) $
(0.29 ) $
112,053
(0.29 ) $
(0.18 ) $
(0.29 ) $
151,696
136,641
112,053
24,838
7,835
382
33,055
682
(15,026 )
(557 )
209
(15,374 )
37
(15,337 ) $
(0.18 ) $
84,715
(0.18 ) $
84,715
42
10,298
1,650
11,948
21,316
7,536
267
29,119
—
(17,171 )
772
(61 )
(16,460 )
38
(16,422 )
(0.20 )
82,144
(0.20 )
82,851
2019
2018
2017
2016
2015
December 31,
Consolidated Balance Sheet Data:
Cash and cash equivalents
$
Working capital
Total assets
Warrant liabilities and note payable
Total stockholders’ equity
35,041 $
24,334
41,666
—
23,271
51,059 $
42,365
61,730
—
43,116
29,316 $
21,107
33,593
—
23,376
14,753 $
9,405
19,060
1,004
11,181
23,027
19,251
25,129
839
19,724
(1) We adopted Accounting Standards Update, or ASU, No. 2014-09, Revenue from Contracts with Customers, effective
January 1, 2018 as further described in Note B to the consolidated financial statements. As a result, the recognized revenue
in 2019 and 2018 is not accounted for on the same basis as the prior years and is not comparable largely due to the timing of
revenue recognition.
43
ITEM 7.
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND
RESULTS OF OPERATIONS
You should read the following discussion and analysis in conjunction with “Item 8. Financial Statements and Supplementary
Data” included below in this annual report on Form 10-K.
Overview
We are an international biotechnology company that is focused primarily in the field of regenerative medicine. Our MultiStem
cell therapy, a patented and proprietary allogeneic stem cell product, is our lead platform product and is currently in clinical
development in several therapeutic and geographic areas. Our most advanced program is an ongoing Phase 3 clinical trial for
treatment of ischemic stroke. Our current clinical development programs are focused on treating neurological conditions,
cardiovascular disease, inflammatory and immune disorders, certain pulmonary conditions and other conditions where the
current standard of care is limited or inadequate for many patients, particularly in the critical care segment.
As part of the U.S. Government’s response to the outbreak of the 2019 novel coronavirus, COVID-19, we have held
discussions with and made presentations under the Medical Countermeasures TechWatch program to BARDA and to the U.S.
government interagency COVID-19 Medical Countermeasures task force led by BARDA that also included other relevant
governmental agencies and public health institutions. As a result of this review, our program involving administration of
MultiStem for the treatment of ARDS was designated as highly relevant by the Medical Countermeasures TechWatch program.
Following infection with COVID-19, or other viruses or pathogens that trigger severe pulmonary inflammation, ARDS can
occur, resulting in significant morbidity or death. Discussions between Athersys and BARDA are continuing regarding the
finalization and implementation of a potential collaboration.
Current Programs
Our MultiStem cell therapy product development programs in the clinical development stage include the following:
Ischemic Stroke: We are conducting a pivotal Phase 3 clinical trial of MultiStem cell therapy for the
•
treatment of ischemic stroke, referred to as MASTERS-2. Our MASTERS-2 clinical trial is a randomized, double-
blind, placebo-controlled clinical trial designed to enroll 300 patients in North America, Europe and certain other
international locations, who have suffered moderate to moderate-severe ischemic stroke. We initiated the study with a
small number of high-enrolling sites and are bringing on additional sites over time in line with clinical product supply
and clinical operations objectives. The MASTERS-2 study has received several regulatory distinctions including
Special Protocol Assessment, or SPA, designation, Fast Track designation and Regenerative Medicine Advanced
Therapy designation, which was established under the 21st Century Cures Legislation from the United States Food
and Drug Administration, or FDA, as well as a Final Scientific Advice positive opinion from the European Medicines
Agency, or EMA.
In addition, HEALIOS K.K., or Healios, has an ongoing clinical trial, TREASURE, evaluating the safety and
efficacy of administration of MultiStem cell therapy for the treatment of ischemic stroke in Japan. TREASURE will be
evaluated under the progressive framework for regenerative medicine therapies in Japan. Under the new framework,
Healios' ischemic stroke program has been awarded the SAKIGAKE designation by the Pharmaceuticals and Medical
Devices Agency, which is designed to expedite regulatory review and approval, and is analogous to Fast Track
designation from the FDA.
We look forward to the completion of both the MASTERS-2 and TREASURE trials and using the accelerated
pathways afforded to us by the regulators in the United States, Europe and Japan.
•
ARDS: In January 2019 and January 2020, we announced summary results and one-year follow up results,
respectively, from our exploratory clinical study of the intravenous administration of MultiStem cell therapy to treat
patients who are suffering from ARDS. The study results continue to demonstrate a predictable and favorable
tolerability profile with no new safety signals identified associated with MultiStem treatment. Importantly, there were
lower mortality and a greater number of ventilator-free and ICU-free days in the MultiStem-treated patient group
44
compared to the placebo group. Average quality-of-life outcomes were higher in the MultiStem group compared to
placebo through one year. Furthermore, inflammatory markers/cytokines were lower in the MultiStem treatment group
than the placebo group. Based on the promising results, Athersys is planning for a potential registrational trial in this
indication. In 2019, Healios initiated a clinical trial in Japan for patients with pneumonia-induced ARDS, which is
referred to as the ONE-BRIDGE study, which is actively enrolling patients. We look forward to the results of this
study.
Trauma: We have previously announced with University of Texas Health Science Center at Houston, or
•
UTHealth, our plans to conduct a clinical trial evaluating MultiStem cell therapy for early treatment and prevention of
complications after severe traumatic injury. This first-ever study of a cell therapy for treatment of a wide range of
traumatic injuries is intended to be conducted at Memorial Hermann-Texas Medical Center, one of the busiest Level 1
trauma centers in the United States. The study has grant support from the Medical Technology Enterprise Consortium
and the Memorial Hermann Foundation. We will provide the clinical product for the conduct of the trial, as well as
provide regulatory and operational support. We and UTHealth are working to finalize a plan for the study and this
patient population that is acceptable to the FDA.
•
Acute Myocardial Infarction, or AMI: We were conducting a Phase 2 clinical study for the administration
of MultiStem cell therapy to patients that have suffered a heart attack. This study was based in part on the favorable
results of our previously completed Phase 1 clinical study that demonstrated tolerability and encouraging signs of
improvement in heart function among patients that exhibited severely compromised heart function prior to treatment.
This data was published in a leading peer reviewed scientific journal, and one-year follow-up data suggested that the
benefit observed was sustained over time. However, enrollment in the Phase 2 clinical study was very challenging due
in part to changes in standard of care. Due to these challenges and the difficulties in addressing them and priorities in
other clinical areas, we elected in 2019 to suspend the study and determine what has been learned. We will evaluate
our development strategy before proceeding further.
Hematopoietic Stem Cell, or HSC, Transplant Support / Prevention of Graft-versus-Host Disease, or
•
GvHD: Currently, this program is staged for future registration-directed development, which depends on the success
and impact of potential alternative therapies for treating the underlying conditions leading to transplant, as well as
other business and financial considerations. Following our completed Phase 1 clinical study of the administration of
MultiStem cell therapy to patients suffering from leukemia or certain other blood-borne cancers, in which patients
undergo radiation therapy and then receive a HSC transplant, we were granted orphan drug designation by the FDA
and the EMA for MultiStem treatment in the prevention of GvHD, and the MultiStem product was granted Fast Track
designation by the FDA for prophylaxis therapy for GvHD following HSC transplantation. Subsequently, our
registration study design received a positive Scientific Advice opinion from the EMA and a SPA designation from the
FDA, and we may pursue this study at the appropriate time.
We are engaged in preclinical development and evaluation of MultiStem cell therapy in other indications for human health, as
well as certain indications in the animal health field, and we conduct such work both through our own internal research efforts
and through a broad global network of collaborators. We also engage in discussions with third parties about collaborating in the
development of MultiStem cell therapy for various programs and/or various geographic territories and may enter into one or
more business partnerships to advance these programs over time. We may also elect to develop certain programs independently.
While the MultiStem product platform continues to advance, we are engaged in process development initiatives intended to
increase manufacturing scale, reduce production costs and enhance process controls and product quality, among other things.
These initiatives are being conducted both internally and outsourced to select contractors, and the related investments are meant
to enable us to meet potential commercial demand in the event of eventual regulatory approval. Until such time as we are able
to manufacture products ourselves in accordance with good manufacturing practices, we will continue to rely on third-party
manufacturers to make our MultiStem product for clinical trials and eventual commercial sales. These third parties may not
deliver sufficient quantities of our MultiStem product, manufacture MultiStem product in accordance with specifications, or
comply with applicable government regulations. From time to time, such third-party manufacturers, or their material suppliers,
45
may experience production delays, stoppages or interruptions in supply, which may affect the initiation, execution and timing
of completion of our and our partners' clinical trials or commercial activities.
In addition to our manufacturing efforts, in other areas we are stepping up our planning and preparations for the potential
commercialization of our MultiStem product candidate. We are advancing our strategies for market access and reimbursement,
working with third-party experts to plan and undertake initiatives to position the product appropriately and effectively
communicate to payors its value to them and patients. We are developing our go-to-market strategies, which could include
third-party marketing partners in certain areas and the creation of a commercial sales force in other areas. We are also working
with outside experts to develop proprietary solutions to the unique requirements related to the cell therapy supply chain and
clinical site logistics. For example, working with an outside partner, we have been developing a proprietary cryogenic system
designed to securely store and dispense our product in hospital pharmacies or other suitable clinical locations. Our intention is
to be prepared to enable commercialization as soon as reasonably possible following successful completion of pivotal studies,
application and approval by regulators.
We have a collaboration with Healios that covers MultiStem cell therapy for ischemic stroke and ARDS in Japan. The
collaboration includes the use of our technology for Healios’ organ bud program targeted to liver disease and other indications,
as well as certain other rights, including a license for the use of our MultiStem product to treat certain ophthalmological
indications and a license to treat diseases of the liver, kidney, pancreas and intestinal tissue through administration of our
products in combination with induced pluripotent stem cells, or iPSC-derived cells. We provide product supply and
manufacturing technology transfer services to Healios, and in the event that we fail to perform our responsibilities to supply
clinical trial product to Healios, then under certain circumstances, we may be required to grant Healios a license to make the
product solely for use in its licensed fields and territories.
Financial
We have entered into a series of agreements with Healios, our collaborator in Japan and currently our largest stockholder.
Under the collaboration that began in 2016, Healios is responsible for the development and commercialization of the
MultiStem product for the licensed fields in the licensed territories, and we provide services to Healios for which we are
compensated. Each license agreement with Healios has defined economic terms, and we may receive success-based milestone
payments, some of which may be subject to credits. While there is no assurance that we will receive milestone proceeds under
the Healios collaboration, any milestone payment we receive is non-refundable and non-creditable towards future royalties or
any other payment due from Healios. Also, we are entitled to receive tiered royalties on net product sales, as defined in the
license agreements.
In connection with an equity investment in us made by Healios in March 2018, Healios has a warrant to purchase up to
4,000,000 shares of our common stock at an exercise price equal to a reference price, as defined, but no less than $1.76 per
share, and the warrant generally expires in September 2020. While we may generate proceeds from this warrant in the future, it
has not been exercised as of December 31, 2019.
We have had equity purchase agreements in place since 2011 with Aspire Capital Fund LLC, or Aspire Capital, which provide
us the ability to sell shares to Aspire Capital from time-to-time, as appropriate. The current facility was entered into in February
2018 and includes Aspire Capital's commitment to purchase up to an aggregate of $100 million of shares of common stock over
a three-year period. The terms of this 2018 equity facility are similar to the previous arrangements, and we issued 450,000
shares of our common stock to Aspire Capital as a commitment fee in February 2018 and filed a registration statement for the
resale of 24,700,000 shares of common stock in connection with the equity facility. Also, in connection with this equity facility,
Aspire Capital invested $1.0 million to purchase 500,000 shares of common stock at $2.00 per share. In November 2019, we
entered into a new equity facility to replace the current facility, which will provide us with the ability to sell shares to Aspire
Capital up to $100.0 million in aggregate to support operational and other initiatives over the next several years. The terms of
the 2019 equity facility are similar to the previous equity facilities with Aspire Capital, and we issued 350,000 shares of our
common stock to Aspire Capital as a commitment fee in November 2019. We registered for the resale of 31,000,000 shares of
our common stock in connection with this facility.
46
During the years ended December 31, 2019, 2018 and 2017, we sold 14,475,000, 8,708,582 and 9,400,000 shares, respectively,
to Aspire Capital at average prices of $1.41, $1.78 and $1.75 per share, respectively. In the first quarter of 2020 through March
13, 2020, we generated an additional $5.5 million in proceeds from the use of our equity purchase arrangement.
Results of Operations
Since our inception, our revenues have consisted of license fees, contract revenues, royalties and milestone payments from our
collaborators, and grant proceeds. We have not derived revenue from our commercial sale of therapeutic products to date since
we are in clinical development. Research and development expenses consist primarily of external clinical and preclinical study
fees, manufacturing and process development costs, salaries and related personnel costs, legal expenses resulting from
intellectual property prosecution processes, facility costs, and laboratory supply and reagent costs. We expense research and
development costs as they are incurred. We expect to continue to make significant investments in research and development to
enhance our technologies, advance clinical trials of our product candidates, expand our regulatory affairs and product
development capabilities, conduct preclinical studies of our product, manufacture our product candidates and prepare for
potential commercialization of our MultiStem cell therapy product. General and administrative expenses consist primarily of
salaries and related personnel costs, professional fees and other corporate expenses. We expect to continue to incur substantial
losses through at least the next several years.
Year Ended December 31, 2019 Compared to Year Ended December 31, 2018
Revenues. Revenues decreased to $5.6 million for the year ended December 31, 2019 from $24.3 million in 2018. Contract
revenues from our collaboration with Healios decreased $16.8 million period-over-period, as 2018 included the impact of the
2018 collaboration expansion and 2019 included reductions in variable consideration under our Healios arrangement. Grant
revenue decreased by $0.4 million in the year ended December 31, 2019 compared to the year ended December 31, 2018,
primarily due to the completion of grant-funded projects. Our collaboration revenues fluctuate from period-to-period based on
new licenses conferred and the delivery of goods and services under our arrangement with Healios. We expect our collaboration
revenues to vary over time as we contract with Healios to perform manufacturing services and as we potentially enter into new
collaborations.
Research and Development Expenses. Research and development expenses increased to $39.0 million for the year ended
December 31, 2019 from $38.7 million for the year ended December 31, 2018. The increase in research and development
expenses year-over-year of $0.3 million related primarily to increased personnel costs of $1.5 million, including stock-based
compensation, consulting costs of $0.6 million, and other costs of $0.1 million. These increases were partially offset by
decreases in clinical trial and manufacturing process development costs of $1.1 million and license fees of $0.8 million related
to the 2018 Healios collaboration expansion. Based on our current clinical development, manufacturing, process development
and regulatory affairs plans, we expect our 2020 annual research and development expenses to be higher compared to 2019,
and such costs will vary over time based on clinical manufacturing campaigns, the timing and stage of clinical trials underway,
manufacturing process development projects and regulatory initiatives. These variations in activity level may also impact our
accounts payable, accrued expenses and prepaid expenses balances from period-to-period. Other than external expenses for our
clinical and preclinical programs, we do not track our research expenses by project; rather, we track such expenses by the type
of cost incurred.
General and Administrative Expenses. General and administrative expenses increased to $11.4 million in 2019 from
$10.4 million in 2018. The $1.0 million increase was due primarily to increases in legal and professional services, other outside
services and stock compensation expense. We expect our general and administrative expenses may increase in 2020 primarily
related to personnel costs.
Depreciation. Depreciation expense decreased to $0.7 million in 2019 from $0.9 million in 2018 due to equipment being fully
depreciated.
47
Other Income, net. Other income, net, for the years ended December 31, 2019 and 2018 was $0.9 million and $0.8 million,
respectively, and was comprised of interest income and expense, refundable foreign tax credits and foreign currency gains and
losses.
Comparison of the years ended December 31, 2018 and 2017
See the Management Discussion and Analysis section of our Annual Report on Form 10-K for the year ended December 31,
2018 for a discussion of our results of operations for the year ended December 31, 2018 compared to the year ended December
31, 2017.
Liquidity and Capital Resources
Our sources of liquidity include our cash balances. At December 31, 2019, we had $35.0 million in cash and cash equivalents.
We have primarily financed our operations through business collaborations, grant funding, and equity financings including
through our equity facility. We conduct all of our operations through our subsidiary, ABT Holding Company. Consequently, our
ability to fund our operations depends on ABT Holding Company’s financial condition and its ability to make dividend
payments or other cash distributions to us. There are no restrictions such as government regulations or material contractual
arrangements that restrict the ability of ABT Holding Company to make dividend and other payments to us.
We incurred losses since inception of operations in 1995 and had an accumulated deficit of $417.6 million at December 31,
2019. Our losses have resulted principally from costs incurred in research and development, clinical and preclinical product
development, manufacturing and process development, acquisition and licensing costs, and general and administrative costs
associated with our operations.
We use all of our sources of capital to develop our technologies, to discover and develop therapeutic product candidates, to
prepare for potential commercialization of our product candidates, develop business collaborations and to potentially acquire
certain technologies and assets.
In the first quarter of 2019, we received the final $2.5 million quarterly installment payment in connection with the June 2018
expansion of our collaboration with Healios. We are also entitled to receive potential milestones payments, subject to certain
credits, and royalties from Healios under our licensed programs. We receive payments from Healios for clinical product supply,
other manufacturing-related services, and commercial supply. Certain proceeds from Healios may be used by Healios to offset
milestone payments that may become due in the future.
In connection with an equity investment in us made by Healios in March 2018, Healios has a warrant to purchase up to
4,000,000 shares of our common stock at an exercise price equal to a reference price, as defined, but no less than $1.76 per
share, and the warrant generally expires in September 2020. While we may generate proceeds from this warrant in the future, it
has not been exercised as of December 31, 2019.
We have had an equity purchase arrangement in place with Aspire Capital since 2011, through a series of equity facilities, each
with similar durations and terms. Our current facility with Aspire Capital was entered into in February 2018 and includes
Aspire Capital’s commitment to purchase up to an aggregate of $100.0 million of shares of our common stock over a three-year
period, and an investment in us of $1.0 million at $2.00 per share of common stock. In November 2019, we entered into a new
equity facility to replace the current facility when it concludes, which new equity facility will provide us with the ability to sell
shares to Aspire Capital up to $100.0 million in aggregate to support operational and other initiatives. The terms of the 2019
equity facility are similar to the previous equity facilities with Aspire Capital, and we issued 350,000 shares of our common
stock to Aspire Capital as a commitment fee in November 2019. We registered for the resale of 31,000,000 shares of our
common stock in connection with this facility.
During the years ended December 31, 2019 and 2018, we sold 14,475,000 and 8,708,582 shares, respectively, to Aspire Capital
at average prices of $1.41 and $1.78 per share, respectively.
48
As part of the U.S. Government’s response to the outbreak of the 2019 novel coronavirus, COVID-19, we have held
discussions with and made presentations under the Medical Countermeasures TechWatch program to BARDA and to the U.S.
government interagency COVID-19 Medical Countermeasures task force led by BARDA that also included other relevant
governmental agencies and public health institutions. As a result of this review, our program involving administration of
MultiStem for the treatment of ARDS was designated as highly relevant by the Medical Countermeasures TechWatch program.
Following infection with COVID-19, or other viruses or pathogens that trigger severe pulmonary inflammation, ARDS can
occur, resulting in significant morbidity or death. Discussions between Athersys and BARDA are continuing regarding the
finalization and implementation of a potential collaboration.
We have filed a shelf registration statement on Form S-3 with the Securities and Exchange Commission, or SEC, that was
declared effective on January 29, 2020 and allows for the sale of equity securities up to $100 million over a three-year period,
which is fully available to us. Whether we sell securities under the registration statement will depend on a number of factors,
including the market conditions at that time, our cash position at that time and the available and terms of alternative sources of
capital.
In May 2019, we entered into an open market sale agreement with Jefferies LLC, or Jefferies, as sales agent, pursuant to which
we could offer and sell, from time to time, through Jefferies, shares of our common stock having an aggregate offering price of
up to $50.0 million. The shares could have been offered and sold pursuant to our registration statement on Form S-3 that was
declared effective by the SEC on March 21, 2017 but will no longer be effective on March 21, 2020. Because that registration
statement on Form S-3 will soon expire, the open market sale agreement with Jefferies will not be operable without additional
SEC filings. We did not sell any shares of our common stock under this agreement.
We will require substantial additional funding in order to continue our research and product development programs, including
clinical trials of our product candidates and process development and manufacturing projects, and to prepare for possible
regulatory approval and commercial activities. At December 31, 2019, we had available cash and cash equivalents of $35.0
million, and we intend to meet our short-term liquidity needs with available cash, including expected cash receipts from our
collaboration with Healios, available proceeds from our existing equity facility, potential delays in certain non-core programs,
and our ability to defer certain spending. Furthermore, we are actively pursuing new collaborative opportunities and other
potential sources of funding, which could reduce the current level of usage of our equity facility and potentially accelerate
certain costs. If sufficient capital is available, we would plan to accelerate our clinical activity and preparation for regulatory
application, approval and commercialization, including commercial manufacturing.
Importantly, we are approaching near-term milestones, including the results of Healios’ clinical trials, followed by the results of
our MASTERS-2 clinical trial, which we would expect to have a significant impact, favorable or unfavorable, on our ability to
access capital from potential third-party commercial partners or the equity capital markets, for example. Depending on the
outcome of these milestones, we may accelerate or may delay certain programs. In the longer term, we will have to continue to
generate additional capital to meet our needs until we would become cash flow positive as a result of the sales of our clinical
products, if they are approved for marketing. Such capital would come from new and existing collaborations and the related
license fees, milestones and potential royalties, the sale of equity securities from time to time including through our equity
facility, grant-funding opportunities, deferral of certain discretionary costs and the staging of certain development costs, as
needed.
Additionally, we may raise capital from time to time through our equity purchase arrangement with Aspire, subject to its
volume and price limitations, equity offerings and Healios' potential exercise of its Healios Warrant. In the first quarter of 2020
through March 13, 2020, we generated an additional $5.5 million in proceeds from the use of our equity purchase arrangement.
We also manage our cash by deferring certain discretionary costs and staging certain development costs to extend our
operational runway, as needed. Over time, we may consider borrowing from financing institutions or royalty financing
arrangements.
Our capital requirements over time depend on a number of factors, including progress in our clinical development programs,
preparing for potential commercialization of our product candidates, potential product launch, our clinical and preclinical
pipeline of additional opportunities and their stage of development, additional external costs such as payments to contract
49
research organizations and contract manufacturing organizations, additional personnel costs and the costs in filing and
prosecuting patent applications and enforcing patent claims. Furthermore, delays in product supply for our and Healios' clinical
trials may impact the timing and cost of such studies, and delays in product supply following Healios' potential product launch
may impact the timing of royalties that we may receive. The availability of funds impacts our ability to advance multiple
clinical programs concurrently, and any shortfall in funding could result in our having to delay or curtail research and
development efforts. Further, these requirements may change at any time due to technological advances, business development
activity or competition from other companies. We cannot assure you that adequate funding will be available to us or, if
available, that it will be available on acceptable terms.
We expect to continue to incur substantial losses through at least the next several years and may incur losses in subsequent
periods, and our ability to commercialize our product candidates is uncertain. The amount and timing of our future losses are
highly uncertain. Our ability to achieve and thereafter sustain profitability will be dependent upon, among other things,
successfully developing, commercializing and obtaining regulatory approval or clearances for our technologies and products
resulting from these technologies.
Cash Flow Analysis
Net cash used in operating activities was $35.3 million, $13.4 million and $24.0 million in 2019, 2018 and 2017, respectively,
and represented the use of cash to fund operations, clinical trials, preclinical research and process development activities; net of
receipts from collaborative arrangements such as Healios. Net cash used in operating activities may fluctuate significantly
period-to-period, as it has over the past several years, primarily due to the receipt of collaboration fees and payment of specific
clinical trial costs, such as clinical manufacturing campaigns, contract research organization costs, and manufacturing process
development projects. These variations in activity level may also impact our accounts payable, accrued expenses and prepaid
expenses balances from period-to-period.
Net cash used in investing activities was $0.6 million, $0.9 million and $0.3 million in 2019, 2018 and 2017, respectively,
related to the purchase of equipment for our manufacturing and process development activities, which was partially offset by
insurance proceeds received in 2018 related to the 2016 flood. We expect that our capital equipment expenditures will increase
in 2020 compared to 2019 to support our manufacturing process development needs.
Financing activities provided net cash of $19.9 million in 2019, $36.0 million in 2018, and $38.9 million in 2017. In 2018,
Healios invested $21.1 million in our common stock, and in 2017, we received $20.9 million of net proceeds from a common
stock offering. Additional proceeds relate to the exercise of common stock warrants in 2017 and equity sales to Aspire Capital
in each of the three years, net of shares of common stock retained for withholding taxes on share-based awards.
Off-Balance Sheet Arrangements
We have no off-balance sheet arrangements.
Critical Accounting Policies and Management Estimates
The SEC defines critical accounting policies as those that are, in management’s view, important to the portrayal of our
financial condition and results of operation and demanding of management’s judgment. Our discussion and analysis of
financial condition and results of operations are based on our consolidated financial statements, which have been prepared in
accordance with United States generally accepted accounting principles. The preparation of these financial statements requires
us to make estimates on experience and on various assumptions that we believe are reasonable under the circumstances, the
results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily
apparent from other sources. Actual results may differ from those estimates.
A discussion of the material implications of uncertainties associated with the methods, assumptions and estimates underlying
our critical accounting polices is as follows:
50
Revenue Recognition
Our license and collaboration agreements may contain multiple elements, including license and technology access fees,
research and development funding, product supply revenue, service revenue, cost-sharing, milestones and royalties. The
deliverables under such an arrangement are evaluated under ASU 606, Revenue from Contracts with Customers. Each
deliverable is evaluated to recognize revenue in a manner that depicts the transfer of promised goods or services to customers
in an amount that reflects the consideration to which we expect to be entitled in exchange for those goods or services.
We have license and other agreements with Healios that contain multiple elements and performance obligations. For a
description of the collaboration agreements and the determination of contract revenues, see Note E to our audited consolidated
financial statements.
Contract Revenue from Healios: At the inception of the Healios arrangement and again each time that the arrangement has
been modified, all material performance obligations are identified, which include (i) licenses to our technology, (ii) product
supply services, and (iii) services to transfer technology to a contract manufacturer on Healios’ behalf. We determine whether
the performance obligations are both capable of being distinct and distinct within the context of the contract. We develop
assumptions that require judgment to determine the standalone selling price in order to account for our collaborative
agreements, as these assumptions typically include probabilities of obtaining marketing approval for the product candidates,
estimated timing of commercialization, estimated future cash flows from potential product sales of our product candidates,
estimating the cost and markup of providing product supply and technical services, and appropriate discount rates.
In order to determine the transaction price, in addition to the fixed payments, we estimate the amount of variable consideration
utilizing the expected value or most likely amount method, depending on the facts and circumstances relative to the contract,
and the estimates for variable consideration are reassessed each reporting period. We constrain, or reduce, the estimates of
variable consideration if it is probable that a significant reversal of previously recognized revenue could occur throughout the
life of the contract, and both the likelihood and magnitude of a potential reversal of revenue are taken into consideration.
At inception and upon each modification date, once the estimated transaction price is established, amounts are allocated to each
separate performance obligation on a relative standalone selling price basis. These performance obligations include any
remaining, undelivered elements at the time of modifications and any new elements from a modification to the arrangement if
the conditions are not met for being treated as a separate agreement.
For performance obligations satisfied over time, we apply an appropriate method of measuring progress each reporting period
and, if necessary, adjust the estimates of performance and the related revenue recognition. Our technology transfer services are
satisfied over time, and we recognize revenue in proportion to the contractual services provided. For performance obligations
satisfied at a point in time (i.e., product supply), we recognize revenue upon delivery.
Clinical Trial Costs
Clinical trial costs are accrued based on work performed by outside contractors that manage and perform the trials, and that
manufacture clinical product. We obtain initial estimates of total costs based on enrollment of subjects, project management
estimates, manufacturing estimates and other activities. Actual costs are typically charged to us and recognized as the tasks are
completed by the contractor, and if we are invoiced based on progress payments as opposed to actual costs, we develop
estimates of work completed to date. Accrued clinical trial costs may be subject to revisions as clinical trials progress, and any
revisions are recorded in the period in which the facts that give rise to the revisions become known.
Pending Adoption of New Accounting Pronouncements
Refer to Note B to the consolidated financial statements for a discussion of recently issued accounting standards.
51
CAUTIONARY NOTE ON FORWARD-LOOKING STATEMENTS
This annual report on Form 10-K contains forward-looking statements within the meaning of the Private Securities Litigation
Reform Act of 1995 that involve risks and uncertainties. These forward-looking statements relate to, among other things, the
expected timetable for development of our product candidates, our growth strategy, and our future financial performance,
including our operations, economic performance, financial condition, prospects, and other future events. We have attempted to
identify forward-looking statements by using such words as “anticipates,” “believes,” “can,” “continue,” “could,” “estimates,”
“expects,” “intends,” “may,” “plans,” “potential,” “should,” “suggest,” “will,” or other similar expressions. These forward-
looking statements are only predictions and are largely based on our current expectations. These forward-looking statements
appear in a number of places in this annual report.
In addition, a number of known and unknown risks, uncertainties, and other factors could affect the accuracy of these
statements. Some of the more significant known risks that we face are the risks and uncertainties inherent in the process of
discovering, developing, and commercializing products that are safe and effective for use as therapeutics, including the
uncertainty regarding market acceptance of our product candidates and our ability to generate revenues. The following risks
and uncertainties may cause our actual results, levels of activity, performance, or achievements to differ materially from any
future results, levels of activity, performance, or achievements expressed or implied by these forward-looking statements:
•
•
•
•
•
•
•
•
•
•
•
•
•
•
•
our ability to raise capital to fund our operations;
the timing and nature of results from MultiStem clinical trials, including the MASTERS-2 Phase 3 clinical trial
and the Healios TREASURE and ONE-BRIDGE clinical trials in Japan;
the possibility of delays in, adverse results of, and excessive costs of the development process;
our ability to successfully initiate and complete clinical trials of our product candidates;
the possibility of delays, work stoppages or interruptions in manufacturing by third parties or us, such as due to
material supply constraints or regulatory issues, which could negatively impact our trials and the trials of our
collaborators;
uncertainty regarding market acceptance of our product candidates and our ability to generate revenues,
including MultiStem cell therapy for the treatment of ischemic stroke, ARDS, AMI and trauma, and the
prevention of GvHD and other disease indications;
changes in external market factors;
changes in our industry’s overall performance;
changes in our business strategy;
our ability to protect and defend our intellectual property and related business operations, including the
successful prosecution of our patent applications and enforcement of our patent rights, and operate our business
in an environment of rapid technology and intellectual property development;
our possible inability to realize commercially valuable discoveries in our collaborations with pharmaceutical
and other biotechnology companies;
our ability to meet milestones and earn royalties under our collaboration agreements, including the success of
our collaboration with Healios;
our collaborators’ ability to continue to fulfill their obligations under the terms of our collaboration agreements
and generate sales related to our technologies;
the success of our efforts to enter into new strategic partnerships and advance our programs, including, without
limitation, in North America, Europe and Japan;
our possible inability to execute our strategy due to changes in our industry or the economy generally;
52
•
•
•
changes in productivity and reliability of suppliers;
the success of our competitors and the emergence of new competitors; and
the risks mentioned elsewhere in this annual report on Form 10-K under Item 1A, “Risk Factors.”
Although we currently believe that the expectations reflected in the forward-looking statements are reasonable, we cannot
guarantee our future results, levels of activity or performance. We undertake no obligation to publicly update forward-looking
statements, whether as a result of new information, future events or otherwise, except as otherwise required by law. You are
advised, however, to consult any further disclosures we make on related subjects in our reports on Forms 10-Q, 8-K and 10-K
furnished to the SEC. You should understand that it is not possible to predict or identify all risk factors. Consequently, you
should not consider any such list to be a complete set of all potential risks or uncertainties.
ITEM 7A.
QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK
Interest Rate Risk
Our exposure to interest rate risk is related to our investment portfolio and our borrowings. Fixed rate investments and
borrowings may have their fair market value adversely impacted from changes in interest rates. Due in part to these factors, our
future investment income may fall short of expectations. Further, we may suffer losses in investment principal if we are forced
to sell securities that have declined in market value due to changes in interest rates. When appropriate based on interest rates,
we invest our excess cash primarily in debt instruments of the United States government and its agencies and corporate debt
securities, and as of December 31, 2019, we had no investments.
We have entered into loan arrangements with financial institutions when needed and when available to us. At December 31,
2019, we had no borrowings outstanding.
53
ITEM 8.
FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA
Athersys, Inc.
Consolidated Financial Statements
Years Ended December 31, 2019, 2018 and 2017
Contents
Reports of Independent Registered Public Accounting Firm
Consolidated Balance Sheets as of December 31, 2019 and 2018
Consolidated Statements of Operations and Comprehensive Loss for each of the years ended December 31,
2019, 2018 and 2017
Consolidated Statements of Stockholders’ Equity for each of the years ended December 31, 2019, 2018 and
2017
Consolidated Statements of Cash Flows for each of the years ended December 31, 2019, 2018 and 2017
Notes to Consolidated Financial Statements
55
57
58
59
60
61
54
Report of Independent Registered Public Accounting Firm
The Board of Directors and Stockholders of Athersys, Inc.
Opinion on the Financial Statements
We have audited the accompanying consolidated balance sheets of Athersys, Inc. (the Company) as of December 31, 2019 and
2018, the related consolidated statements of operations and comprehensive loss, stockholders’ equity and cash flows for each of
the three years in the period ended December 31, 2019, and the related notes and financial statement schedule listed in the
Index at Item 15(a) (2) (collectively referred as the “consolidated financial statements”). In our opinion the consolidated
financial statements present fairly, in all material respects, the financial position of the Company at December 31, 2019 and
2018, and the results of its operations and its cash flows for each of the three years in the period ended December 31, 2019, in
conformity with U.S. generally accepted accounting principles.
We also have audited, in accordance with the standards of the Public Company Accounting Oversight Board (United States)
(PCAOB), the Company’s internal control over financial reporting as of December 31, 2019, based on criteria established in
Internal Control-Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway Commission
(2013 framework), and our report dated March 16, 2020 expressed an unqualified opinion thereon.
Adoption of ASU No. 2014-09
As discussed in Note B to the consolidated financial statements, the Company changed its method of accounting for
recognizing revenue due to the adoption of ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606), effective
January 1, 2018.
Basis for Opinion
The financial statements are the responsibility of the Company’s management. Our responsibility is to express an opinion on
the Company’s financial statements based on our audits. We are a public accounting firm registered with the PCAOB and are
required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the applicable
rules and regulations of the Securities and Exchange Commission and the PCAOB.
We conducted our audits in accordance with the standards of the PCAOB. Those standards require that we plan and perform the
audit to obtain reasonable assurance about whether the financial statements are free of material misstatement, whether due to
error or fraud. Our audits included performing procedures to assess the risks of material misstatement of the 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 disclosures in the 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 financial statements. We believe that our audits provide a reasonable basis for our opinion.
/s/ Ernst & Young LLP
We have served as the Company’s auditor since 1998.
Cleveland, Ohio
March 16, 2020
55
Report of Independent Registered Public Accounting Firm
To Board of Directors and Stockholders of Athersys, Inc.
Opinion on Internal Control over Financial Reporting
We have audited Athersys, Inc.'s internal control over financial reporting as of December 31, 2019, based on criteria established
in Internal Control—Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway
Commission (2013 framework) (the COSO criteria). In our opinion, Athersys, Inc. (the Company) maintained, in all material
respects, effective internal control over financial reporting as of December 31, 2019, based on the COSO criteria.
We also have audited, in accordance with the standards of the Public Company Accounting Oversight Board (United States)
(PCAOB), the consolidated balance sheets of the Company as of December 31, 2019 and December 31, 2018, the related
consolidated statements of operations and comprehensive loss, stockholders’ equity and cash flows for each of the three years
in the period ended December 31, 2019, and the related notes and financial statement schedule listed in the Index at Item
15(a)(2) and our report dated March 16, 2020 expressed an unqualified opinion thereon.
Basis for Opinion
The Company’s management is responsible for maintaining effective internal control over financial reporting and for its
assessment of the effectiveness of internal control over financial reporting included in the accompanying “Management’s
Report on Internal Control over Financial Reporting.” Our responsibility is to express an opinion on the Company’s internal
control over financial reporting based on our audit. We are a public accounting firm registered with the PCAOB and are
required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the applicable
rules and regulations of the Securities and Exchange Commission and the PCAOB.
We conducted our audit in accordance with the standards of the PCAOB. Those standards require that we plan and perform the
audit to obtain reasonable assurance about whether effective internal control over financial reporting was maintained in all
material respects.
Our audit included obtaining an understanding of internal control over financial reporting, assessing the risk that a material
weakness exists, testing and evaluating the design and operating effectiveness of internal control based on the assessed risk, and
performing such other procedures as we considered necessary in the circumstances. We believe that our audit provides a
reasonable basis for our opinion.
Definition and Limitations of Internal Control over Financial Reporting
A company’s internal control over financial reporting is a process designed to provide reasonable assurance regarding the
reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally
accepted accounting principles. A company’s internal control over financial reporting includes those policies and procedures
that (1) pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and
dispositions of the assets of the company; (2) provide reasonable assurance that transactions are recorded as necessary to permit
preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and
expenditures of the company are being made only in accordance with authorizations of management and directors of the
company; and (3) provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use, or
disposition of the company’s 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 any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate
because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.
/s/ Ernst & Young LLP
Cleveland, Ohio
March 16, 2020
56
Athersys, Inc.
Consolidated Balance Sheets
(In Thousands, Except Share and Per Share Amounts)
Assets
Current assets:
Cash and cash equivalents
Accounts receivable
Accounts receivable from Healios
Unbilled accounts receivable from Healios
Prepaid expenses and other
Total current assets
Equipment, net
Deposits and other
Total assets
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
Accounts payable to Healios
Accrued compensation and related benefits
Accrued clinical trial related costs
Accrued expenses
Deposit from Healios
Deferred revenue - Healios
Total current liabilities
Advance from Healios
Other long-term liabilities
Stockholders’ equity:
Preferred stock, at stated value; 10,000,000 shares authorized, and no shares issued and
outstanding at December 31, 2019 and 2018
Common stock, $0.001 par value; 300,000,000 shares authorized; 159,791,585 and
144,292,739 shares issued and outstanding at December 31, 2019 and 2018, respectively
Additional paid-in capital
Accumulated deficit
Total stockholders’ equity
Total liabilities and stockholders’ equity
See accompanying notes.
57
$
$
$
December 31,
2019
2018
35,041 $
17
945
—
1,168
37,171
2,882
1,613
41,666 $
9,048 $
1,068
773
1,160
723
—
65
12,837
5,338
220
51,059
262
1,108
3,620
1,791
57,840
3,002
888
61,730
9,163
—
1,901
1,276
461
2,000
674
15,475
3,139
—
—
—
160
440,735
(417,624 )
23,271
41,666 $
144
416,014
(373,042 )
43,116
61,730
$
Athersys, Inc.
Consolidated Statements of Operations and Comprehensive Loss
(In Thousands, Except Per Share Amounts)
Revenues
Contract revenue from Healios
Royalty and other contract revenue
Grant revenue
Total revenues
Costs and expenses
Research and development (including stock compensation expense of $2,217,
$1,609 and $1,232 in 2019, 2018 and 2017, respectively)
General and administrative (including stock compensation expense of $2,634,
$2,240 and $1,812 in 2019, 2018 and 2017, respectively)
Depreciation
Total costs and expenses
Gain from insurance proceeds, net
Loss from operations
Income from change in fair value of warrants, net
Other income, net
Net loss and comprehensive loss
Net loss per common share, basic and diluted
Weighted average shares outstanding, basic and diluted
See accompanying notes.
Years Ended December 31,
2019
2018
2017
$
5,517 $
—
116
5,633
22,276 $
1,461
554
24,291
918
1,925
865
3,708
39,045
38,656
27,841
11,378
698
51,121
—
(45,488 )
—
906
10,442
855
49,953
617
(25,045 )
—
762
8,466
684
36,991
—
(33,283 )
728
314
$
$
(44,582 ) $
(24,283 ) $
(32,241 )
(0.29 ) $
(0.18 ) $
151,696
136,641
(0.29 )
112,053
58
Balance at January 1, 2017
Cumulative effect of accounting change
Stock-based compensation
Issuance of common stock from warrant
exercises
Issuance of common stock, net of
issuance costs
Issuance of common stock under equity
compensation plans
Net and comprehensive loss
Balance at December 31, 2017
Cumulative effect of accounting change
Stock-based compensation
Issuance of warrant to Healios at fair
value
Issuance of common stock, net of
issuance costs
Issuance of common stock to Healios, net
of issuance costs
Issuance of common stock under equity
compensation plans
Net and comprehensive loss
Balance at December 31, 2018
Stock-based compensation
Issuance of common stock, net of
issuance costs
Issuance of common stock under equity
compensation plan
Net and comprehensive loss
Balance at December 31, 2019
See accompanying notes.
Athersys, Inc.
Consolidated Statements of Stockholders’ Equity
(In Thousands, Except Share Amounts)
Preferred Stock
Common Stock
Number
of Shares
Stated
Value
— $ —
Number
of Shares
86,629,302 $
Par
Value
87 $
Additional
Paid-in
Capital
329,373 $
Accumulated
Deficit
Total
Stockholders’
Equity
(318,279 ) $
(110 )
—
—
—
—
(32,241 )
(350,630 )
1,871
—
—
—
—
—
(24,283 )
(373,042 )
—
—
—
(44,582 )
(417,624 ) $
11,181
(110 )
3,154
1,862
39,813
(283 )
(32,241 )
23,376
1,871
3,849
1,080
16,628
20,995
(400 )
(24,283 )
43,116
4,851
20,284
(398 )
(44,582 )
23,271
—
—
—
—
—
—
—
—
—
—
—
—
—
—
—
—
—
1,843,363
—
33,172,300
—
—
—
—
—
—
—
—
—
—
432,488
—
122,077,453
—
—
9,658,582
12,000,000
556,704
—
144,292,739
—
—
2
33
—
—
122
—
—
9
12
1
—
144
—
3,154
1,860
39,780
(283 )
—
373,884
3,849
1,080
16,619
20,983
(401 )
—
416,014
4,851
—
14,825,000
15
20,269
—
—
—
—
— $ —
673,846
—
159,791,585 $ 160 $
1
—
(399 )
—
440,735 $
59
Athersys, Inc.
Consolidated Statements of Cash Flows
(In Thousands)
Operating activities
Net loss
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation
Stock-based compensation
Discount on revenue from issuance of warrant
Other
Stock–based patent license and settlement expense
Gain from insurance proceeds, net
Change in fair value of warrant liabilities
Changes in operating assets and liabilities:
Accounts receivable
Accounts receivable from Healios - billed and unbilled
Prepaid expenses and other
Accounts payable and accrued expenses
Accounts payable to Healios
Advances and deposits from Healios
Deferred revenue - Healios
Deferred revenue
Net cash used in operating activities
Investing activities
Proceeds from insurance, net
Purchases of equipment
Net cash used in investing activities
Financing activities
Proceeds from issuance of common stock, net
Proceeds from issuance of common stock to Healios, net
Proceeds from exercise of warrants
Shares retained for withholding tax payments on stock-based awards
Net cash provided by financing activities
(Decrease) increase in cash and cash equivalents
Cash and cash equivalents at beginning of year
Cash and cash equivalents at end of year
See accompanying notes.
Years Ended December 31,
2019
2018
2017
$
(44,582 ) $
(24,283 ) $
(32,241 )
698
4,851
—
—
—
—
—
245
3,783
857
(1,836 )
1,068
199
(609 )
—
855
3,849
1,080
—
315
(617 )
—
324
(4,545 )
(1,346 )
4,269
—
4,889
2,110
(250 )
684
3,044
—
(22 )
3,150
—
(728 )
12
(153 )
(206 )
1,537
—
134
—
771
(35,326 )
(13,350 )
(24,018 )
—
(579 )
(579 )
20,311
—
—
(424 )
19,887
(16,018 )
51,059
35,041 $
617
(1,532 )
(915 )
15,415
20,995
—
(402 )
36,008
21,743
29,316
51,059 $
—
(285 )
(285 )
37,287
—
1,862
(283 )
38,866
14,563
14,753
29,316
$
60
Athersys, Inc.
Notes to Consolidated Financial Statements
A. Background
We are an international biotechnology company focused in the field of regenerative medicine and operate in one business
segment. Our operations consist of research, preclinical development and clinical development activities, and our most
advanced program is in Phase 3 clinical development.
We have incurred losses since our inception in 1995 and had an accumulated deficit of $417.6 million at December 31, 2019,
and we will not commence sales of our clinical product candidates until they receive regulatory approval for
commercialization. We will require significant additional capital to continue our research and development programs, including
progressing our clinical product candidates to commercialization and preparing for commercial-scale manufacturing and sales.
At December 31, 2019, we had available cash and cash equivalents of $35.0 million. We believe that these funds, expected cash
receipts primarily attributed to our collaboration with HEALIOS K.K. (“Healios”), available proceeds from our existing equity
facility, potential delays in certain non-core programs, and our ability to defer certain spending will enable us to meet our
obligations as they come due at least for the next year, from the date of the issuance of these consolidated financial statements.
Furthermore, we are actively pursuing new collaborative opportunities and other potential sources of funding, which could
reduce the current level of usage of our equity facility and potentially accelerate certain costs. If sufficient capital is available,
we would plan to accelerate our clinical activity and preparation for regulatory application, approval and commercialization,
including commercial manufacturing.
Importantly, we are approaching near-term milestones, including the results of Healios’ clinical trials, followed by the results of
our MASTERS-2 clinical trial, which we would expect to have a significant impact, favorable or unfavorable, on our ability to
access capital from potential third-party commercial partners or the equity capital markets, for example. Depending on the
outcome of these milestones, we may accelerate or may delay certain programs. In the longer term, we will have to continue to
generate additional capital to meet our needs until we would become cash flow positive as a result of the sales of our clinical
products, if they are approved for marketing. Such capital would come from new and existing collaborations and the related
license fees, milestones and potential royalties, the sale of equity securities from time to time including through our equity
facility and grant-funding opportunities.
B. Accounting Policies
Principles of Consolidation
The consolidated financial statements include our accounts and results of operations and those of our wholly owned
subsidiaries. All intercompany accounts and transactions have been eliminated in consolidation.
Revenue Recognition
Our license and collaboration agreements may contain multiple elements, including license and technology access fees,
research and development funding, product supply revenue, service revenue, cost-sharing, milestones and royalties. As further
described below, on January 1, 2018, we adopted Accounting Standards Update (“ASU”) No. 2014-09, Revenue from Contracts
with Customers (“Topic 606”), to account for revenue. The deliverables under our arrangements are evaluated under Topic 606
which requires an entity to recognize revenue in a manner that depicts the transfer of promised goods or services to customers
in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services.
61
Milestone Payments
Topic 606 does not contain guidance specific to milestone payments, but rather requires potential milestone payments to be
considered in accordance with the overall model of Topic 606. As a result, revenues from contingent milestone payments are
recognized based on an assessment of the probability of milestone achievement and the likelihood of a significant reversal of
such milestone revenue at each reporting date. This assessment may result in recognizing milestone revenue before the
milestone event has been achieved. Since the milestones in the Healios arrangement are generally related to development and
commercial milestone achievement by Healios, we have not included any of the Healios milestones in the estimated transaction
price of the Healios arrangement, since they would be constrained, as a significant reversal of revenue could result in future
periods.
Other than for our collaboration with Healios that has remaining deliverables, as of the date of adoption of Topic 606 on
January 1, 2018, we had recognized the full amount of license fees under our collaboration agreements as contract revenue
under the prior guidance associated with multiple-element arrangements, since the performance periods for our multiple
element arrangements had concluded. The events triggering any future contingent milestone payments from these arrangements
were determined to be non-substantive and revenue will be recognized in the period that the triggering event occurs, and the
remaining potential commercial milestones will be recognized when earned.
Grant Revenue
Grant revenue, which is not within the scope of Topic 606 for our grant arrangements, consists of funding under cost
reimbursement programs primarily from federal and non-profit foundation sources for qualified research and development
activities performed by us, and as such, are not based on estimates that are susceptible to change. Such amounts are invoiced
and recorded as revenue as grant-funded activities are performed, with any advance funding recorded as deferred revenue until
the activities are performed.
Royalty Revenue
We generate royalty revenue from the sale of licensed products by our licensees. Royalty revenue is recognized upon the later
to occur of (i) achievement of the collaborator’s underlying sales and (ii) satisfaction of any performance obligation(s) related
to these sales, in each case assuming the license to our intellectual property is deemed to be the predominant item to which the
sales-based royalties relate.
Unbilled Accounts Receivable
We record amounts that are due to us under contractual arrangements for which invoicing has not yet occurred if our
performance has concluded for the billable activity, and we have the unconditional right to the consideration, but such amounts
have not yet been billed. The remaining transaction price for the performance obligations that were not yet delivered was not
significant at December 31, 2019. At December 31, 2018, unbilled accounts receivable from Healios was $3.6 million, which
includes $2.5 million of license fees that were paid to us by Healios in the first quarter of 2019 related to the expansion of our
arrangement described in Note E. The remainder of the unbilled accounts receivable on the consolidated balance sheets at
December 31, 2018 relates to manufacturing technology transfer services performed that were not yet billed to Healios.
Contractual Right to Consideration and Deferred Revenue
Amounts included in deferred revenue or contract assets are determined at the contract level, and for our Healios arrangement,
such amounts are included in a contract asset or liability depending on the overall status of the arrangement. Amounts received
from customers or collaborators in advance of our performance of services or other deliverables are included in deferred
revenue, while amounts for performance of services or other deliverables before customer payment is received or due are
included in contract assets, with those amounts that are unconditional being included in either accounts receivable or unbilled
accounts receivable. Grant proceeds received in advance of our performance under the grant is included in deferred revenue.
Generally, deferred revenue and contract assets or liabilities are classified as current assets or obligations, as opposed to non-
current.
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Deposit from Healios
Included in the deposit from Healios at December 31, 2018 is a $2.0 million payment received from Healios to extend the
period of its exclusive right to negotiate for an option to expand its license to develop and commercialize certain disease
indications in China. These nonrefundable proceeds will be creditable against potential milestone payments under the existing
Healios licenses. The exclusive right of negotiation period expired on June 30, 2019.
Advances from Healios
The clinical trial supply agreement with Healios was amended in July 2017 to clarify a cost-sharing arrangement associated
with our supply of clinical product for Healios' ischemic stroke trial in Japan. The proceeds from Healios for clinical supply
that relate specifically to the cost-sharing arrangement may (i) result in a decrease in the amount we receive from Healios upon
the achievement of certain milestones and an increase to a commercial milestone or (ii) be repaid at our election. While the
amendment to the supply agreement resulted in a revision to the terms associated with the product supply, namely the cost of
product supply, the revision did not affect any of the performance obligations under the overall arrangement. The proceeds
from Healios that relate specifically to the cost-sharing arrangement for Healios’ ischemic stroke study in Japan are recognized
as a non-current advance from Healios until the related milestones are achieved or such amounts are repaid to Healios at our
election. No revenue has been recognized yet related to this advance.
Recently Adopted Accounting Standards
In May 2014, the Financial Accounting Standards Board ("FASB") issued guidance codified in ASC 606, Revenue Recognition
- Revenue from Contracts with Customers ("Topic 606"), which amends the guidance in former ASC 605, Revenue Recognition.
The core principle of Topic 606 is that an entity should recognize revenue to depict the transfer of promised goods or services
to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those good
or services. We adopted Topic 606 utilizing the modified retrospective transition method applied to contracts that were not
complete as of January 1, 2018. We evaluated all of our arrangements on a contract-by-contract basis, identifying all of the
performance obligations, including those that are contingent. For our contracts with customers that contain multiple
performance obligations, we account for the individual performance obligations separately when they are both capable of being
distinct, whereby the customer can benefit from the service either on its own or together with other resources that are readily
available from third parties or from us, and are distinct in the context of the contract, whereby the transfer of the services is
separately identifiable from other promises in the contract. Under the new standard, we assessed whether licenses granted under
our collaboration and license agreements were distinct in the context of the agreement from other performance obligations and
functional when granted. After considering the relative selling prices of the contract elements and the allocation of revenue
thereto, we recognized a cumulative effect adjustment of $1.9 million as an adjustment to the opening balance of our December
31, 2018 accumulated deficit primarily related to a contract asset since the revenue permitted to be recognized at inception was
not limited to the cash proceeds received as of that time, which was a requirement of the previous guidance. We concluded that
the new guidance resulted in revisions to accounting for our arrangement with Healios, only, since our other collaborations had
no remaining performance obligations and potential contingent receipts would be constrained. Notes E and F further describe
our arrangement with Healios. The comparative information for fiscal year ended December 31, 2017 has not been restated and
continues to be reported under the accounting standards in effect for those periods.
In January 2019 we adopted ASU 2016-02, Leases ("Topic 842"), which requires lessees to put most leases with a term greater
than 12 months on their balance sheets, but recognize expenses on their statement of operations in a manner similar to current
accounting practice. Under the guidance, lessees initially recognize a lease liability for the obligation to make lease payments
and a right-of-use (“ROU”) asset for the right to use the underlying asset for the lease term. The lease liability is measured at
the present value of the lease payments over the lease term. The ROU asset is measured at the lease liability amount, adjusted
for lease prepayments, lease incentives received and the lessee’s initial direct costs. The guidance is effective for the annual and
interim periods beginning after December 15, 2018. The impact of adoption to our consolidated financial statements is further
discussed in Note J.
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Cash and Cash Equivalents
We consider all highly liquid investments with a maturity of three months or less when purchased to be cash equivalents. Cash
equivalents are primarily invested in money market funds. The carrying amount of our cash equivalents approximates fair value
due to the short maturity of the investments. Cash used in investing activities excluded $0.1 million of accrued capital
expenditures in 2018.
Research and Development
Research and development expenditures, which consist primarily of costs associated with clinical trials, preclinical research,
product manufacturing and process development for manufacturing, personnel, legal fees resulting from intellectual property
application and maintenance processes, and laboratory supply and reagent costs, including direct and allocated overhead
expenses, are charged to expense as incurred.
Clinical Trial Costs
Clinical trial costs are accrued based on work performed by outside contractors that manage and perform the trials, and those
that manufacture the investigational product. We obtain initial estimates of total costs based on enrollment of subjects, trial
duration, project management estimates, manufacturing estimates, patient treatment costs and other activities. Actual costs may
be charged to us and recognized as the tasks are completed by the contractor or, alternatively, may be invoiced in accordance
with agreed-upon payment schedules and recognized based on estimates of work completed to date. Accrued clinical trial costs
may be subject to revisions as clinical trials progress, and any revisions are recorded in the period in which the facts that give
rise to the revisions become known.
Royalty Payments and Sublicense Fees
We are required to make royalty payments to certain parties based on our product sales under license agreements. No royalties
were recorded during the three-year period ended December 31, 2019, since we have not yet generated sales revenue. We are
also required to record sublicense fees from time-to-time in connection with license fees from collaborators and clinical and
commercial milestone achievement, of which $0.1 million and $0.6 million were recorded as research and development
expenses in the Consolidated Statements of Operations in the years ended December 31, 2019 and 2018, respectively.
Long-Lived Assets
Equipment is stated at acquired cost less accumulated depreciation. Laboratory and office equipment are depreciated on the
straight-line basis over the estimated useful lives (three to ten years). Leasehold improvements are amortized over the shorter of
the lease term or estimated useful life.
Long-lived assets are evaluated for impairment when events or changes in circumstances indicate that the carrying amount of
the asset or related group of assets may not be recoverable. If the expected future undiscounted cash flows are less than the
carrying amount of the asset, an impairment loss is recognized at that time. Measurement of impairment may be based upon
appraisal, market value of similar assets or discounted cash flows.
Proceeds from Insurance
In 2016, our facility sustained flood damage representing both an unusual and infrequent event, and we recognized a net
insurance recovery gain of $0.7 million in 2016 that was reported as a separate component of our loss from operations. An
additional $0.6 million of insurance proceeds, net of associated expenses, were received in 2018, concluding the insurance
claim. Proceeds from insurance settlements, except for those directly related to investing or financing activities, were
recognized as cash inflows from operating activities. Since the majority of the damage from the flood was to fully depreciated
leasehold improvements, the amount of losses was less than the amount of the insurance proceeds received.
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Patent Costs and Rights
Costs of applying for, prosecuting and maintaining patents and patent rights are expensed as incurred. We have filed for broad
intellectual property protection on our proprietary technologies and have numerous United States and international patents and
patent applications related to our technologies.
Warrants
We account for common stock warrants as either liabilities or as equity instruments depending on the specific terms of the
warrant agreements. Generally, warrants are classified as liabilities, as opposed to equity, if the agreement includes the potential
for a cash settlement or an adjustment to the exercise price, and warrant liabilities are recorded at their fair values at each
balance sheet date. We had no warrant liabilities at December 31, 2019 and 2018. Refer to Note F for a warrant issued in 2018
to Healios (the “Healios Warrant”), which is accounted for as an equity instrument.
Concentration of Credit Risk
Our accounts receivable are generally comprised of amounts due from collaborators and granting authorities and are subject to
concentration of credit risk due to the absence of a large number of customers. At December 31, 2019 and 2018, most of our
accounts receivable are due from Healios. We do not typically require collateral from our customers.
Use of Estimates
The preparation of financial statements in conformity with accounting principles generally accepted in the United States
requires management to make estimates and assumptions that affect the amounts reported in the financial statements and
accompanying notes. Actual results could differ from those estimates.
Stock-Based Compensation
We recognize stock-based compensation expense on the straight-line method and use a Black-Scholes option-pricing model to
estimate the fair value of option awards. The expected term of options granted represent the period of time that option grants
are expected to be outstanding. We use the “simplified” method to calculate the expected life of option grants given our limited
history of exercise activity and determine volatility by using our historical stock volatility. The fair value of our restricted stock
units is equal to the closing price of our common stock on the date of grant and is expensed over the vesting period on a
straight-line basis.
Options may be exercised for cash or by a cashless exercise that is permitted under certain conditions. In the event of a cashless
exercise, we retain the number of shares equivalent to the exercise cost based on the market value at the time of exercise and
issue the net number of shares to the holder.
We recognize income tax benefits and deficiencies as income tax expense or benefit in the consolidated statement of operations
and the tax effects of exercised or vested awards are treated as discrete items in the reporting period in which they occur. We
also recognize excess tax benefits regardless of whether the benefit reduces taxes payable in the current period. Excess tax
benefits are classified along with other income tax cash flows as an operating activity in the consolidated statement of cash
flows. Regarding forfeitures, we account for them when they occur.
All the aforementioned estimates and assumptions are evaluated on a quarterly basis and may change as facts and
circumstances warrant. Changes in these assumptions can materially affect the estimate of the fair value of our share-based
payments and the related amount recognized in our financial statements.
Annual stock-based awards to employees typically vest over a four-year period, although the 2018 awards vest over a three-
year period, have an exercise price equal to the fair market value of a share of common stock on the grant date and have a
contractual term of 10 years. The following weighted-average input assumptions were used in determining the fair value of the
Company’s stock options:
65
Volatility
Risk-free interest rate
Expected life of option
Expected dividend yield
Income Taxes
December 31,
2019
2018
2017
71.1 %
2.0 %
70.8 %
2.8 %
71.2 %
2.0 %
6.2 years
6.0 years
6.2 years
0.0 %
0.0 %
0.0 %
Deferred tax liabilities and assets are determined based on the differences between the financial reporting and tax basis of
assets and liabilities and are measured using the tax rate and laws currently in effect. We evaluate our deferred income taxes to
determine if a valuation allowance should be established against the deferred tax assets or if the valuation allowance should be
reduced based on consideration of all available evidence, both positive and negative, using a “more likely than not” standard.
We had no liability for uncertain income tax positions as of December 31, 2019 and 2018. Our policy is to recognize potential
accrued interest and penalties related to the liability for uncertain tax benefits, if applicable, in income tax expense. Net
operating loss and credit carryforwards since inception remain open to examination by taxing authorities and will for a period
post utilization.
Net Loss per Share
Basic and diluted net loss per share have been computed using the weighted-average number of shares of common stock
outstanding during the period. For each reporting period in which we have outstanding warrants, we evaluate the income from
such warrant liabilities and consider whether it results in a potentially dilutive effect to net loss per share. There were no such
dilutive effects from warrant liabilities for each of the periods ended December 31, 2019, 2018 and 2017.
We have outstanding options, restricted stock units and outstanding warrants that were not used in the calculation of diluted net
loss per share because to do so would be antidilutive. The following instruments, were excluded from the calculation of diluted
net loss per share because their effects would be antidilutive:
Stock options
Restricted stock units
Warrants
Recently Issued Accounting Standards
Years ended December 31,
2019
13,975,671
2,032,180
4,000,000
20,007,851
2018
10,955,508
1,656,688
18,500,000
31,112,196
2017
8,919,113
1,648,986
—
10,568,099
In December 2019, the FASB issued ASU 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes.
The ASU simplifies the accounting for income taxes, changes the accounting for certain income tax transactions, and other
minor changes. This guidance is effective for fiscal years beginning after December 15, 2020, including interim periods, with
early adoption permitted. We are currently assessing the impact that this ASU will have on our consolidated financial
statements and disclosures.
In November 2018, the FASB issued ASU 2018-18, Collaborative Arrangements (“Topic 808”): Clarifying the Interaction
between Topic 808 and Topic 606. The amendments in this update make targeted improvements to generally accepted
accounting principles (“GAAP”) for collaborative arrangements as follows: clarify that certain transactions between
collaborative arrangement participants should be accounted for as revenue under Topic 606 when the collaborative arrangement
participant is a customer in the context of a unit of account. In those situations, all the guidance in Topic 606 should be applied,
including recognition, measurement, presentation, and disclosure requirements; add unit-of-account guidance in Topic 808 to
align with the guidance in Topic 606 (that is, a distinct good or service) when an entity is assessing whether the collaborative
arrangement or a part of the arrangement is within the scope of Topic 606; and require that in a transaction with a collaborative
arrangement participant that is not directly related to sales to third parties, presenting the transaction together with revenue
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recognized under Topic 606 is precluded if the collaborative arrangement participant is not a customer. The provisions of ASU
2018-18 are effective for years beginning after December 15, 2019. We are currently evaluating the impact of this clarifying
guidance.
In August 2018, the FASB issued ASU 2018-15, Intangibles-Goodwill and Other-Internal-Use Software: Customer’s
Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement that is a Service Contract (“ASU 2018-
15”). ASU 2018-15 requires implementation costs incurred by customers in cloud computing arrangements (i.e., hosting
arrangements) to be capitalized under the same premises of authoritative guidance for internal-use software and deferred over
the noncancellable term of the cloud computing arrangements plus any option renewal periods that are reasonably certain to be
exercised by the customer or for which the exercise is controlled by the service provider. The guidance is effective for the
annual and interim periods beginning after December 15, 2019, with early adoption permitted. Entities can choose to adopt
prospectively or retrospectively. We will adopt the standard effective January 1, 2020 prospectively and are currently evaluating
the potential impact on our consolidated financial statements.
In June 2016, the FASB issued ASU 2016-13, Measurement of Credit Losses on Financial Instruments (Topic 326). This ASU
replaces the incurred loss impairment methodology in current GAAP with a methodology that reflects expected credit losses
and requires consideration of a broader range of reasonable and supportable information to inform credit loss estimates.
Subsequent to issuing ASU 2016-13, the FASB issued ASU 2019-10, Financial Instruments - Credit Losses (Topic 326):
Effective Dates, delaying the effective date for smaller reporting companies until January 2023. We are currently evaluating the
potential impact of adoption of this standard on our consolidated financial statements and disclosures, and we do not intend to
early adopt.
C. Equipment
Equipment consists of (in thousands):
Laboratory equipment
Office equipment and leasehold improvements
Process development equipment not yet in service
Accumulated depreciation
December 31,
2019
2018
8,008 $
3,191
574
11,773
(8,891 )
2,882 $
7,444
3,043
822
11,309
(8,307 )
3,002
$
$
During 2019, we disposed of approximately $0.1 million of obsolete laboratory equipment, office equipment and leasehold
improvements, all of which were fully depreciated.
D. Financial Instruments
Fair Value Measurements
We classify the inputs used to measure fair value into the following hierarchy:
Level 1
Level 2
Unadjusted quoted prices in active markets for identical assets or liabilities.
Unadjusted quoted prices in active markets for similar assets or liabilities, or unadjusted quoted prices for
identical or similar assets or liabilities in markets that are not active, or inputs other than quoted prices that are
observable for the asset or liability.
Level 3
Unobservable inputs for the asset or liability.
At December 31, 2019, we had no financial assets or liabilities measured at fair value on a recurring basis.
The Healios Warrant that was issued in March 2018 was measured at fair value on a nonrecurring basis and is a Level 3 equity
instrument under the hierarchy. Refer to Note F regarding its valuation.
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Financing Arrangements
We lease office and laboratory space under operating leases. The lease for our corporate offices and laboratories began in 2000
and currently expires in March 2021, with options to renew annually through March 2024. Our rent is approximately $267,000
per year and our rental rate has not changed since the lease inception in 2000. We also lease office and laboratory space for our
Belgian subsidiary, which expires in July 2021. Annual rent expense is approximately $190,000, subject to adjustments based
on an inflationary index.
Aggregate rent expense for the years ended December 31, 2018 and 2017 recognized prior to the adoption of Topic 842 was
approximately $493,000 and $477,000, respectively. Refer to Note J regarding our adoption of Topic 842 in January 2019 to
account for our leases.
E. Collaborative Arrangements and Revenue Recognition
Healios Collaboration
In 2016, we entered into a license agreement (the “First License Agreement”) with Healios to develop and commercialize
MultiStem cell therapy for ischemic stroke in Japan and to provide Healios with access to our proprietary MAPC technology
for use in Healios' organ bud program, initially for transplantation to treat liver disease or dysfunction. Under the terms of the
First License Agreement, Healios also obtained a right to expand the scope of the collaboration to include the exclusive rights
to develop and commercialize MultiStem for the treatment of two additional indications in Japan, which at that time included
acute respiratory distress syndrome (“ARDS”) and another indication in the orthopedic area, and all indications for the organ
bud program. In accordance with the First License Agreement, in addition to potential royalties and milestones, we received a
nonrefundable up-front cash payment of $15 million, and if expanded at Healios’ election, Healios would pay an additional $10
million cash payment. Healios exercised its option to expand the collaboration in June 2018, as described below.
Under the collaboration, Healios is responsible for the development and commercialization of the MultiStem product in the
licensed territories, and we provide manufacturing services to Healios, currently comprising the supply of product for its
clinical trials and the transfer of technology to a contract manufacturer in Japan to produce product for Healios. We receive
payments for these products and services provided to Healios.
In 2017, we signed a clinical trial supply agreement for delivering the planned manufacturing services for Healios’ clinical trial
in Japan treating ischemic stroke patients. The clinical trial supply agreement was amended later that year to clarify the
operational elements, terms and cost-sharing arrangement associated with our supply of clinical material and certain
adjustments to potential milestone payments related to the clinical product supply for Healios’ TREASURE study in Japan.
Healios’ cost-share payments may be creditable against milestone payments that may become due under the First License
Agreement and a sales milestone would be increased, or such payments may be repaid by us at our election. Services to Healios
under the clinical trial supply agreement are ongoing.
Also, in 2017, we entered into a technology transfer services agreement with Healios, in which Healios provides financial
support to establish a contract manufacturer in Japan to produce product for Healios. At that time, we also amended the First
License Agreement to confer to Healios a limited license to manufacture MultiStem if we are acquired by a third-party.
Services to Healios under the technology transfer services agreement are ongoing.
In March 2018, we entered into a letter of intent (“LOI”), with Healios outlining the terms for a potential expansion of the
relationship with Healios beyond that contemplated by the First License Agreement, to include, among other things, the
exercise of its option to license the ARDS field in Japan and the organ bud program on a global basis, a worldwide exclusive
license for use of MultiStem product to treat certain ophthalmological indications, and an exclusive option to a license to
develop and commercialize certain MultiStem treatments in China. In connection with the LOI, in March 2018, Healios
purchased 12,000,000 shares of our common stock and the Healios Warrant for $21.1 million, or approximately $1.76 per
share.
In June 2018, Healios exercised its option to expand the collaboration to include ARDS and expand organ bud as contemplated
by the First License Agreement, and entered into the Collaboration Expansion Agreement (“CEA”) that included new license
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agreements and rights that further broadened the collaboration. Under the CEA, Healios (i) expanded its First License
Agreement to include ARDS in Japan, expanded the organ bud license to include all transplantation indications, and terminated
Healios’ right to include a designated orthopedic indication per the First License Agreement; (ii) obtained a worldwide
exclusive license, or the Ophthalmology License Agreement, for use of MultiStem product to treat certain ophthalmological
indications; (iii) obtained an exclusive license in Japan (the "Combination Product License Agreement”), for use of the
MultiStem product to treat diseases of the liver, kidney, pancreas and intestinal tissue through local administration of
MultiStem in combination with iPSC-derived cells; (iv) obtained an exclusive, time-limited right of first negotiation (“ROFN
Period”) to enter into an option for a license to develop and commercialize certain MultiStem treatments in China, which has
since expired; and (v) an option for an additional non-therapeutic technology license. which has also expired. For all
indications, Healios is responsible for the costs of clinical development in its licensed territories, and we provide manufacturing
services to Healios.
For the rights granted to Healios under the CEA, Healios paid us a nonrefundable, up-front cash payment of $10.0 million to
exercise its option to license ARDS and expand its license for organ bud, as contemplated by the First License Agreement, and
paid an additional $10.0 million for the new license rights, which has been paid in full in four quarterly installment payments of
$2.5 million. The payments were received in the second, third and fourth quarters of 2018 with the final payment received in
the first quarter of 2019. Healios may elect to credit up to $10.0 million against milestone payments that may become due
under the First License Agreement, as expanded to include ARDS, with limitations on amounts that may be credited to earlier
milestone payments versus later milestone payments.
For each of the ischemic stroke indication and the ARDS indication, we may receive success-based regulatory filing and
approval and sales milestones aggregating up to $225 million in aggregate for each indication, subject to potential milestone
credits. Milestone payments are non-refundable and non-creditable towards future royalties or any other payment due from
Healios. We may also receive tiered royalties on net product sales, starting in the low double digits and increasing
incrementally into the high teens depending on net sales levels.
For standalone products sold by Healios under the Ophthalmology License Agreement, we are entitled to receive success-based
regulatory filing and approval and sales milestones aggregating up to $135.6 million and tiered royalties on net product sales in
the single digits depending on net sales levels. For the combination products under the Ophthalmology License Agreement, we
will be entitled to receive a low single-digit royalty, but no milestone payments. Under the Combination Product License
Agreement, we are entitled to receive a low single-digit royalty on net sales of the combination product treatments, but no
milestone payments. For the organ bud product, we are entitled to receive a fractional royalty percentage on net sales of the
organ bud products; and we have a time-limited right of first negotiation for commercialization of an organ bud product in
North America.
Under the CEA, the ROFN Period with respect to the option for a license in China was extended to June 30, 2019 in exchange
for a $2.0 million payment from Healios that we received in December 2018. The extension payment will be applied as a credit
against any potential milestone payments under the current licenses, subject to certain limitations. The ROFN Period expired on
June 30, 2019.
In connection with the entry into the CEA, we amended the terms of the Healios Warrant as addressed in Note F.
Healios Revenue Recognition
At the inception of the Healios arrangement and again each time that the arrangement has been modified, all material
performance obligations were identified, which include (i) licenses to our technology, (ii) product supply services, and (iii)
services to transfer technology to a contract manufacturer on Healios’ behalf. It was determined that these performance
obligations were both capable of being distinct and distinct within the context of the contract. We develop assumptions that
require judgment to determine the standalone selling price in order to account for our collaborative agreements, as these
assumptions typically include probabilities of obtaining marketing approval for the product candidates, estimated timing of
commercialization, estimated future cash flows from potential product sales of our product candidates, estimating the cost and
markup of providing product supply and technical services, and appropriate discount rates.
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In order to determine the transaction price, in addition to the fixed payments, we estimate the amount of variable consideration
utilizing the expected value or most likely amount method, depending on the facts and circumstances relative to the contract,
and the estimates for variable consideration are reassessed each reporting period. We constrain, or reduce, the estimates of
variable consideration if it is probable that a significant reversal of previously recognized revenue could occur throughout the
life of the contract, and both the likelihood and magnitude of a potential reversal of revenue are taken into consideration.
The pricing for certain product supply provided to Healios is driven off the underlying cost per dose over the entire life of the
agreement and is subject to variability as those costs change. During our evaluation of variable consideration in the third and
fourth quarters of 2019, we revised our estimated transaction price due to changes in the underlying cost per dose of the
product supply occurring during the respective quarter. We estimate the cost per dose for the life of the contract taking into
consideration historical experience of our contract manufacturers and anticipated changes to production yields and other
factors. During the third quarter of 2019, the price per dose from our contract manufacturers decreased for the first time under
this arrangement. As such, we reduced the expected transaction price to the current estimated value and applied the reduction to
the undelivered elements of the overall arrangement at the time this product supply performance obligation originated.
Furthermore, unrelated to the cost per dose changes, the number of doses of clinical product requested by Healios was amended
in the third quarter of 2019, and our revenues were further reduced during the period. Similarly, during the third and fourth
quarters of 2019, certain services related to technology transfer activities were determined to be constrained since the activity
may no longer be required under the arrangement.
At inception and upon each modification date, once the estimated transaction price is established, amounts are allocated to each
separate performance obligation on a relative standalone selling price basis. These performance obligations include any
remaining, undelivered elements at the time of modifications and any new elements from a modification to the arrangement if
the conditions are not met for being treated as a separate agreement. Following the June 2018 modification, the specific
performance obligations that had been delivered included the licenses, and the performance obligations that were not yet fully
delivered included clinical product manufacturing services and technology transfer services that we provide to a contract
manufacturer in Japan. In the third quarter of 2018, an additional modification was executed to add clinical product
manufacturing services for Healios’ planned ARDS study, which resulted in a new performance obligation, creating a
modification to the arrangement and remeasurement of the transaction and standalone selling prices for the undelivered
elements on the modification date.
For performance obligations satisfied over time, we apply an appropriate method of measuring progress each reporting period
and, if necessary, adjust the estimates of performance and the related revenue recognition. Our technology transfer services are
satisfied over time, and we recognize revenue in proportion to the contractual services provided. For performance obligations
satisfied at a point in time (i.e., product supply), we recognize revenue upon delivery.
The remaining transaction price for the performance obligations that were not yet delivered is not significant at December 31,
2019. At December 31, 2019, the contract liability included in Deferred Revenue - Healios on the consolidated balance sheets,
is properly classified as a current liability since the rights to consideration are expected to be satisfied, in all material respects,
within one year.
We included as a reduction of the transaction price of the licenses granted in the June 2018 expansion, the value of a portion of
the Healios Warrant that was issued in March 2018 in connection with the then-proposed expansion under a letter of intent.
Under the agreements in the June 2018 expansion that included an amendment to the Healios Warrant, 4,000,000 shares
(“Warrant Shares”) became exercisable, and as a result, $1.1 million of the $5.3 million initial warrant valuation was recorded
in June 2018 as a reduction of revenue. In accordance with the June 2018 amendment to the Healios Warrant, the remaining
16,000,000 shares would not be exercisable until the execution of an option for a license in China, and the remaining $4.2
million of the Healios Warrant was reversed against additional paid-in-capital. See Note F.
Also, see Note B regarding our revenue recognition policies.
70
Advance from Healios
In 2017, we amended the clinical trial supply agreement for the manufacturing of investigational product for Healios for its
Japan stroke clinical study to clarify a cost-sharing arrangement. The proceeds from Healios that relate specifically to the cost-
sharing arrangement may either (i) result in a reduction in the proceeds we receive from Healios upon the achievement of two
potential milestones and an increase to a commercial milestone under the First License Agreement for stroke or (ii) be repaid to
Healios at our election, as defined. The cost-sharing proceeds received are recognized on the balance sheet as a non-current
advance from customer until the related milestone is achieved, unless such amounts are repaid to Healios at our election, at
which time, the culmination of the earnings process will be complete and revenue will be recognized.
Disaggregation of Revenues
We recognize license-related amounts, including upfront payments, exclusivity fees, additional disease indication fees, and
development, regulatory and sales-based milestones, at a point in time when earned. Similarly, product supply revenue is
recognized at a point in time, while service revenue is recognized when earned over time. The following table presents our
contract revenues disaggregated by timing of revenue recognition and excludes royalty revenue (in thousands):
Twelve months ended
December 31, 2019
Twelve months ended
December 31, 2018
Point in
Time
Over Time
Point in
Time
Over Time
$
$
1,624 $
2,167
—
—
3,791 $
—
—
1,726
—
1,726
$
$
17,682 $
1,445
—
251
19,378 $
—
—
3,149
—
3,149
Contract revenue from Healios:
License fee revenue
Product supply revenue
Service revenue
Other contract revenue
Total disaggregated revenues
F. Capitalization and Warrant Instruments
Capitalization
At December 31, 2019 and December 31, 2018, we had 300 million shares of common stock and 10.0 million shares of
undesignated preferred stock authorized. No shares of preferred stock have been issued as of December 31, 2019 and 2018.
The following shares, in thousands, of common stock were reserved for future issuance:
Stock-based compensation plans
Healios Warrants to purchase common stock
December 31
2019
2018
30,054
4,000
34,054
16,096
18,500
34,596
71
Equity Issuance - Healios
In March 2018, Healios purchased 12,000,000 shares of our common stock for $21.1 million, or approximately $1.76 per share,
and the Healios Warrant to purchase up to an additional 20,000,000 shares. In connection with this investment, we entered into
an Investor Rights Agreement (the "Investor Rights Agreement") that governs certain rights of Healios and us relating to
Healios’ ownership of our common stock. The Investor Rights Agreement provides for customary standstill and voting
obligations, transfer restrictions and registration rights for Healios. Additionally, we agree to provide notice to Healios of
certain equity issuances and to allow Healios to participate in certain issuances in order maintain its proportionate ownership of
our common stock as of the time of such issuance. We further agreed that during such time as Healios beneficially owns more
than 5.0% but less than 15.0% of our outstanding common stock, our Board of Directors (the “Board”) will nominate a Healios
nominee suitable to us to become a member of the Board, and during such time as Healios beneficially owns 15.0% or more of
our outstanding common stock, our Board will nominate two suitable Healios nominees to become members of the Board, at
each annual election of directors. Healios nominated an individual to the Board, who was elected at the 2018 annual
stockholders’ meeting. As a result of Healios’ investment, Healios became a related party, and the transactions with Healios are
separately identified within these financial statements as related party transactions.
At the time of the investment in March 2018, the 20,000,000 Warrant Shares would not become exercisable until the planned
collaboration expansion was completed, which at the time included an option to commercialize in China. At the time of the
June 2018 expansion, however, the parties had not reached agreement on the option so Athersys agreed to provide Healios with
a right of first negotiation with respect to the option, and therefore, the parties bifurcated the Healios Warrant so that 4,000,000
Warrant Shares became exercisable with the June 2018 expansion and the remaining 16,000,000 Warrant Shares would become
exercisable if Healios agreed to execute an option for a license in China. As of June 30, 2019, 16,000,000 Warrant Shares were
no longer exercisable and expired under the terms of the Healios Warrant, since an option in China was not executed. The
4,000,000 Warrant Shares are exercisable at the greater of $1.76 and the Reference Price (which is generally 110% of the
average closing price per share of the Company's common stock for the ten trading days ending on the trading day immediately
preceding the date the Warrant is exercised).
The value of the Healios Warrant was considered as an element of compensation in the transaction price of the Healios
collaboration expansion. We evaluated the various terms of the Healios Warrant and concluded that it is accounted for as an
equity instrument at inception and $5.3 million was computed as the best estimate of the fair value of the Healios Warrant at the
time of issuance in March 2018. The fair value was computed using a Monte Carlo simulation model that included probability-
weighted estimates of potential milestone points in time that could impact the value of the Healios Warrant during its term. The
fair value was recorded as additional paid-in capital in the first quarter of 2018, with the offset being included in other asset
related to Healios, and the asset would be included as an element of compensation in the transaction price upon the
consummation of the expansion that was proposed in March 2018 under the LOI.
Upon the modification of the Healios Warrant in June 2018 in connection with the expansion of the collaboration that included
the bifurcation of the Healios Warrant due to the change related to China rights, we reassessed the fair value of the Healios
Warrant immediately before and after the modification using the same valuation methodology, which resulted in no incremental
fair value to be recorded. The value of the 4,000,000 Warrant Shares that became exercisable upon the June 2018 expansion of
$1.1 million was recorded as a reduction to the revenue recognized for the delivered licenses in June 2018. See Note E.
However, since the June 2018 expansion agreements made the exercisability of the 16,000,000 shares underlying the Healios
Warrant contingent on entering into an option for a license in China, we considered the ability to apply the $4.2 million value of
such Warrant Shares as an element of compensation to be constrained. Therefore, the remaining asset was reversed against
additional paid-in-capital.
Equity Purchase Agreement
We have had equity purchase agreements in place since 2011 with Aspire Capital Fund LLC (“Aspire Capital”) that provide us
the ability to sell shares to Aspire Capital from time to time, as appropriate. The current agreement with Aspire Capital was
entered into in February 2018 and includes Aspire Capital’s commitment to purchase up to an aggregate of $100.0 million of
shares of our common stock over a three-year period. The terms of the 2018 equity facility are similar to the previous
arrangements, and we issued 450,000 shares of our common stock to Aspire Capital as a commitment fee in February 2018 and
filed a registration statement for the resale of 24,700,000 shares of common stock in connection with the equity facility. Also,
72
in connection with this equity facility, in February 2018, Aspire Capital invested $1.0 million in us at $2.00 per share of
common stock. During the years ended December 31, 2019, 2018 and 2017, we sold 14,475,000, 8,708,582 and 9,400,000
shares, respectively, to Aspire Capital at average prices of $1.41, $1.78 and $1.75 per share, respectively. In the first quarter of
2020 through March 13, 2020, we generated an additional $5.5 million in proceeds from the use of our equity purchase
arrangement.
In November 2019, we entered into a new facility to replace the current facility, which will provide us with the ability to
purchase up to an aggregate of $100.0 million of shares of common stock over a three-year period. The terms of the 2019
equity facility are similar to the previous arrangements, and we issued 350,000 shares of common stock to Aspire Capital as a
commitment fee in November 2019 and filed a registration statement for the resale of 31,000,000 shares of common stock in
connection with the new equity facility.
Open Market Sale Agreement
In May 2019, we entered into an open market sale agreement with Jefferies LLC ("Jefferies"), as sales agent, pursuant to which
we could offer and sell, from time to time, through Jefferies, shares of our common stock having an aggregate offering price of
up to $50.0 million. The shares could have been offered and sold pursuant to our registration statement on Form S-3 that was
declared effective by the SEC on March 21, 2017 but will no longer be effective on March 21, 2020. Because that registration
statement on Form S-3 will soon expire, the open market sale agreement with Jefferies will not be operable without additional
SEC filings. We did not sell any shares of our common stock under this agreement.
License Agreement and Settlement
In October 2017, we entered into an agreement to settle longstanding intellectual property disagreements with a third-party. As
part of the agreement, we were granted a worldwide, non-exclusive license, with the right to sublicense, to the other party’s
patents and applications that were at the core of the intellectual property dispute, for use related to the treatment or prevention
of disease or conditions using cells. In return, we agreed not to enforce our intellectual property rights against the party with
respect to certain patent claims, nor to further challenge the patentability or validity of certain applications or patents. Upon
execution of the license and settlement agreement in 2017, we paid $0.5 million and issued 1,000,000 shares of our common
stock with a fair value of $2.3 million. In 2018, in accordance with the agreement, we paid an additional $1.0 million and we
issued 500,000 additional shares of our common stock related to a patent issuance. This contingent obligation to issue 500,000
shares of common stock was originally recorded in accrued license fee expense on the consolidated balance sheets at December
31, 2017 at a fair value of $0.9 million. The actual issuance of the 500,000 shares in May 2018 was recorded at an actual fair
value of $1.2 million, resulting in $0.3 million of additional paid-in-capital and research and development expense in 2018.
There will be no royalty, milestone or other payments due to the third-party associated with the development and
commercialization of our cell therapy products. Our payment obligations are concluded.
G. Stock-Based Compensation
Our 2019 Equity and Incentive Compensation Plan (the "EICP") authorizes an aggregate of approximately 18,500,000 shares of
common stock for awards to employees, directors and consultants. The EICP was approved in June 2019 and replaced our prior
long-term incentive plans. The EICP authorizes the issuance of stock-based compensation in the form of stock options, stock
appreciation rights, restricted stock, restricted stock units, performance shares and units, and other stock-based awards. As of
December 31, 2019, a total of 6,106,927 shares (including 255,656 shares related to an expired incentive plan) of common
stock have been issued under our equity incentive plans.
As of December 31, 2019, a total of 14,048,065 shares were available for issuance under our EICP, and stock-based awards to
purchase 16,007,851 shares (including 872,187 shares related to an expired incentive plan) of common stock were outstanding.
We recognized $4.9 million, $3.8 million and $3.0 million of stock-based compensation expense in 2019, 2018 and 2017,
respectively.
73
Stock Options
The weighted average fair value of options granted in 2019, 2018 and 2017 was $1.00, $1.46 and $0.95 per share, respectively.
The total fair value of options vested during 2019, 2018 and 2017 was $3.0 million, $2.5 million and $2.0 million, respectively.
The total intrinsic value of options exercised during the years ended December 31, 2019, 2018 and 2017 was not significant. At
December 31, 2019, total unrecognized estimated compensation cost related to unvested stock options was approximately $5.5
million, which is expected to be recognized by the end of 2023 using the straight-line method. The weighted average
contractual life of unvested options at December 31, 2019 was 8.8 years. The aggregate intrinsic value of fully vested option
shares and option shares expected to vest as of December 31, 2019 was not significant.
A summary of our stock option activity and related information is as follows:
Outstanding January 1, 2017
Granted
Exercised
Forfeited / Expired
Outstanding December 31, 2017
Granted
Exercised
Forfeited / Expired
Outstanding December 31, 2018
Granted
Exercised
Forfeited / Expired
Outstanding December 31, 2019
Vested during 2019
Vested and exercisable at December 31, 2019
Number
of Options
9,236,228 $
2,596,480
(136,056 )
(2,777,539 )
8,919,113
2,434,732
(112,484 )
(285,853 )
10,955,508
3,402,608
(48,152 )
(334,293 )
13,975,671 $
2,508,333 $
8,919,741 $
Weighted
Average
Exercise
Price
2.76
1.47
1.50
4.76
1.78
2.26
1.57
2.62
1.87
1.55
1.40
2.71
1.77
1.88
1.81
Exercise Price
$1.01 – $1.55
$1.56 – $1.98
$2.06 – $3.84
Options Outstanding
Options Vested and Exercisable
December 31, 2019
Number
of
Options
6,832,730
2,685,058
4,457,883
13,975,671
Weighted
Average
Remaining
Contractual
Life
8.0 years $
4.7 years $
7.2 years $
Weighted
Average
Exercise
Price
1.47
1.72
2.26
Number
of
Options
3,223,605
2,499,294
3,196,842
8,919,741
Weighted
Average
Remaining
Contractual
Life
3.2 years $
4.1 years $
5.0 years $
Weighted
Average
Exercise
Price
1.42
1.72
2.26
74
Restricted Stock Units
A summary of our restricted stock unit activity and related information is as follows:
Outstanding January 1, 2017
Granted
Vested-common stock issued
Forfeited / Expired
Outstanding December 31, 2017
Granted
Vested-common stock issued
Forfeited / Expired
Outstanding December 31, 2018
Granted
Vested-common stock issued
Forfeited / Expired
Outstanding December 31, 2019
Vested/Issued cumulative at December 31, 2019
Number
of
Restricted
Stock Units
1,201,159 $
1,054,720
(571,118 )
(35,775 )
1,648,986
787,968
(741,424 )
(38,842 )
1,656,688
1,350,150
(938,311 )
(36,347 )
2,032,180 $
5,508,176 $
Weighted
Average
Fair Value
1.92
1.46
1.75
1.82
1.69
2.31
1.81
1.74
1.93
1.55
1.87
1.69
1.71
1.75
The total fair value of restricted stock units vested during 2019, 2018 and 2017 was $1.8 million, $1.3 million and $1.0 million,
respectively. At December 31, 2019, total unrecognized estimated compensation cost related to unvested restricted stock units
was approximately $3.4 million, which is expected to be recognized by the end of 2023 using the straight-line method.
H. Income Taxes
At December 31, 2019, we had U.S. federal net operating loss and research and development tax credit carryforwards of
approximately $193.2 million and $12.8 million, respectively. Such operating losses and tax credits may be used to reduce
future taxable income and tax liabilities and will expire at various dates between 2032 and 2039. Additionally, as of
December 31, 2019, we had federal net operating loss carryforwards generated after 2017 of $56.6 million that have an
indefinite life, but with usage limited to 80% of taxable income in any given year. We also had foreign net operating loss
carryforwards of approximately $24.2 million. Such foreign net operating loss carryforwards do not expire. We also had state
and city net operating loss carryforwards aggregating approximately $63.5 million. Such state and city net operating loss
carryforwards may be used to reduce future taxable income and tax liabilities and will expire at various dates between 2020 and
2037. Certain state net operating losses do not expire.
The utilization of net operating loss and tax credit carryforwards generated prior to October 2012 (the “Section 382 Limited
Attributes”) is substantially limited under Section 382 of the Internal Revenue Code of 1986, as amended, (the “IRC”). We
generated U.S. federal net operating loss carryforwards of $156.5 million, research and development tax credits of $12.8
million, and state and local net operating loss carryforwards of $63.3 million since 2012. We will update our analysis under
Section 382 prior to using these attributes.
75
A reconciliation of the federal statutory income tax rate to our effective tax rate is as follows:
Statutory federal income tax rate
State income taxes - net of federal tax benefit
Other permanent differences
Valuation allowances
Research and development - U.S.
Effective tax rate for the year
Significant components of our deferred tax assets are as follows (in thousands):
Net operating loss carryforwards
Research and development credit carryforwards
Compensation expense
Other
Total deferred tax assets
Valuation allowance for deferred tax assets
Net deferred tax assets
Percent of Income
before Income Taxes
2019
2018
21.0 %
0.9 %
(2.3 )%
(25.9 )%
6.3 %
— %
21.0 %
0.9 %
(3.7 )%
(29.2 )%
11.0 %
— %
December 31,
2019
2018
48,182 $
12,797
1,156
903
63,038
(63,038 )
— $
38,813
9,979
1,552
1,166
51,510
(51,510 )
—
$
$
Because of our cumulative losses, substantially all the deferred tax assets have been fully offset by a valuation allowance. We
have not paid income taxes for the three-year period ended December 31, 2019.
In December 2017, the U.S. federal government enacted legislation commonly referred to as the “Tax Cuts and Jobs Act” (the
“TCJA”). The TCJA made widespread changes to the IRC, including, among other items, a reduction in the federal corporate
tax rate from 35% to 21%, effective January 1, 2018. The TCJA also eliminated alternative minimum tax and the 20-year
carryforward limitation for net operating losses incurred after December 31, 2017 and imposes a limit on the usage of net
operating losses incurred after such date equal to 80% of taxable income in any given year. The 80% usage limit will not have
an economic impact on us until our current net operating losses are either utilized or expire. The carrying value of our deferred
tax assets and liabilities is determined by the enacted U.S. corporate income tax rate. Consequently, any changes in the U.S.
corporate income tax rate impacts the carrying value of our deferred tax assets and liabilities. The Deemed Repatriation
Transition Tax (“Transition Tax”) is a tax on previously untaxed accumulated and current earnings and profit (“E&P”) of
certain of our foreign subsidiaries. To determine the amount of Transition Tax, a company must determine, in addition to other
factors, the amount of post-1986 E&P of the relevant foreign subsidiaries as well as the amount of non-U.S. income tax paid on
such earnings. We have an overall foreign E&P deficit and accordingly have not recorded any Transition Tax obligation.
As of December 31, 2018, we have completed the accounting for all the impacts of the TCJA. We continue to evaluate the impacts
of the TCJA and will consider additional guidance from the U.S. Treasury Department, Internal Revenue Service or other
standard-setting bodies. However, no additional adjustments were recorded by us during the measurement period in 2018 as
permitted by SEC Staff Accounting Bulletin 118, Income Tax Accounting Implications of the Tax Cuts and Jobs Act.
I. Profit Sharing and 401(k) Plan
We have a profit sharing and 401(k) plan that covers substantially all employees and allows for discretionary contributions by
us. We make employer contributions to this plan, and the expense was approximately $0.4 million, $0.5 million and $0.3
million in 2019, 2018 and 2017, respectively.
76
J. Leases
In February 2016, the FASB issued Topic 842, which requires lessees to record most leases with a term greater than 12 months
on their balance sheets, but recognize expenses on their statement of operations in a manner similar to current accounting
practice. Under the guidance, lessees initially recognize a lease liability for the obligation to make lease payments and a ROU
asset for the right to use the underlying asset for the lease term. The lease liability is measured at the present value of the lease
payments over the lease term. The ROU asset is measured at the lease liability amount, adjusted for lease prepayments, lease
incentives received and the lessee’s initial direct costs.
We adopted ASU 2016-02, Leases ("Topic 842") effective January 1, 2019, using the modified retrospective transition option.
The adoption of the standard resulted in the recording of ROU assets, primarily consisting of operating leases of facilities and
minor equipment, and lease liabilities of $1.0 million as of the commencement date. The adoption did not have a material
impact on our consolidated statements of operations and comprehensive loss or cash flows related to existing leases. As a
result, there was no cumulative-effect adjustment.
We elected certain practical expedients as part of the adoption, which allow us to not reassess our prior conclusions about lease
identification, lease classification and initial direct costs. We also elected the short-term lease recognition exemption for all
leases that qualify and will not recognize ROU assets or lease liabilities for those leases. Lastly, we elected to separate lease
and non-lease components only for contract manufacturing assets based on an assessment of the contract terms. We did not
separate lease and non-lease components for all other existing asset classes. Most leases do not contain an implicit discount
rate; therefore, we estimate our incremental borrowing rate to discount the lease payments based on information available at
lease commencement.
Our facilities leases contain one or more options to renew after the noncancellable term. The exercise of lease renewal options
is not reasonably certain upon lease commencement and is at management's sole discretion. Our ROU assets are included
within deposits and other in our consolidated balance sheet at December 31, 2019. Associated lease liabilities are included in
accrued expenses and other, and other long-term liabilities in our consolidated balance sheet at December 31, 2019. Lease
expense for lease payments is recognized on a straight-line basis over the lease term within loss from operations in the
consolidated statements of operations and comprehensive loss. Payments for certain lease agreements are adjusted annually for
changes in an index or rate. We had no finance leases, residual value guarantees, restrictive covenants, subleases or sale
leasebacks at December 31, 2019.
As of December 31, 2019, ROU assets and lease liabilities were each $0.7 million. The weighted-average remaining term for
lease contracts was 1.6 years at December 31, 2019, with maturities ranging from 15 months to 50 months. The weighted-
average discount rate was 5.3% at December 31, 2019. We paid $0.5 million for operating leases included in the measurement
of lease liabilities during the year ended December 31, 2019.
Aside from facilities and minor equipment, we have various supply agreements with third-party manufacturers, which involve
the lease of manufacturing facilities and equipment, as defined in Topic 842. We have elected to separate lease and non-lease
components for these arrangements. These manufacturing agreements have variable lease payments, which typically become
binding once certain manufacturing milestones are achieved, and as such, are not included in ROU assets and liabilities until
such payments are no longer variable.
Lease Costs
The table below presents certain information related to the lease costs (in thousands) for operating leases as of December 31,
2019:
Operating lease cost
Short-term lease cost
Variable lease cost (1)
Total lease cost
(1) Includes lease components from our third-party manufacturing agreements.
77
Twelve months ended
December 31, 2019
487
61
205
753
$
$
Undiscounted Cash Flows
The following table summarizes future maturities (in thousands) for operating lease liabilities as of December 31, 2019:
2020
2021
2022
2023
2024
Total minimum lease payments
Less: amount of lease payments representing interest
Present value of operating lease liabilities
$
$
489
198
15
12
2
716
30
686
K. Quarterly Financial Data (unaudited)
The following table presents quarterly data for the years ended December 31, 2019 and 2018, in thousands, except per share
data:
First
Quarter
Second
Quarter
2019
Third
Quarter
Fourth
Quarter
Full Year
Revenues
Loss from operations
Net loss
Basic and diluted net loss per common share (1) $
$
1,445 $
(13,260 )
(12,956 )
(0.09 ) $
4,262 $
(9,901 )
(9,688 )
(0.06 ) $
(361 ) $
(12,342 )
(12,015 )
(0.08 ) $
287 $
(9,985 )
(9,923 )
(0.06 ) $
5,633
(45,488 )
(44,582 )
(0.29 )
Revenues
Income (loss) from operations
Net income (loss)
Basic and diluted net income (loss) per common
share (1)
$
$
First
Quarter
Second
Quarter
2018
Third
Quarter
Fourth
Quarter
Full Year
1,066 $
(10,262 )
(10,155 )
19,391 $
6,745
6,933
2,321 $
(9,976 )
(9,740 )
1,513 $
(11,552 )
(11,321 )
24,291
(25,045 )
(24,283 )
(0.08 ) $
0.05 $
(0.07 ) $
(0.08 ) $
(0.18 )
(1) Due to the effect of quarterly changes to outstanding shares of common stock and weightings, the annual loss per share will
not necessarily equal the sum of the respective quarters.
78
ITEM 9.
Not applicable.
CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND
FINANCIAL DISCLOSURE
ITEM 9A.
CONTROLS AND PROCEDURES
Evaluation of disclosure controls and procedures: An evaluation was carried out under the supervision and with the
participation of our management, including our principal executive officer and our principal financial officer, of the
effectiveness of our disclosure controls and procedures as of the end of the period covered by this annual report on Form 10-K.
Based on that evaluation, these officers have concluded that as of December 31, 2019, our disclosure controls and procedures
are effective.
Management’s report on internal control over financial reporting: Management is responsible for establishing and
maintaining adequate internal control over financial reporting, as such term is defined in Exchange Act Rule 13a-15(f). Under
the supervision and with the participation of management, including our principal executive officer and principal financial
officer, we conducted an evaluation of the effectiveness of internal control over financial reporting based on the 2013
framework in Internal Control — Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway
Commission. Based on this evaluation under the 2013 framework in Internal Control — Integrated Framework, management
concluded that our internal control over financial reporting was effective as of December 31, 2019. The effectiveness of our
internal control over financial reporting as of December 31, 2019 has been audited by Ernst & Young LLP, an independent
registered public accounting firm, as stated in their report, which is included in Item 8 of this annual report on Form 10-K and
incorporated herein by reference.
Changes in internal control: During the fourth quarter of 2019, there has been no change in our internal control over financial
reporting that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.
ITEM 9B.
OTHER INFORMATION
On January 9, 2020, the Board of Directors of the Company, based upon the recommendation of the Compensation Committee
of the Board of Directors of the Company, approved a cash bonus incentive plan (the “Plan”) for the year ending December 31,
2020 for the named executive officers of the Company. The Plan provides that each participant is eligible to earn a bonus
during the award term of January 1, 2020 through December 31, 2020. The Plan provides for the following target bonus
percentages of the named executive officer’s salary during the award term, weighted as set forth below on the achievement of
specified corporate goals, with the remainder based on individual/functional performance. The corporate goals include
advancing the Company’s clinical programs for MultiStem and manufacturing process development initiatives, executing
against the established operating plan and capital acquisition objectives, and advancement of strategic partnership and program
activities. There is no formally adopted plan document for the Plan.
Title
Chief Executive Officer
President & Chief Operating Officer
Executive Vice President & Chief Scientific Officer
Chief Financial Officer
Senior Vice President of Finance
Target
Bonus
Weighting on
Corporate Goals
60 %
45 %
45 %
40 %
35 %
100 %
80 %
80 %
80 %
60 %
A summary of the plan is attached to this annual report on Form 10-K as Exhibit 10.45 and is hereby incorporated herein by
reference thereto.
79
PART III
ITEM 10.
DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE
The information required by this item is incorporated by reference to the Proxy Statement with respect to the 2020 Annual
Meeting of Stockholders.
ITEM 11.
EXECUTIVE COMPENSATION
The information required by this item is incorporated by reference to the Proxy Statement with respect to the 2020 Annual
Meeting of Stockholders.
ITEM 12.
SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT
AND RELATED SHAREHOLDER MATTERS
EQUITY COMPENSATION PLAN INFORMATION
The following table sets forth certain information regarding the Company’s equity compensation plans as of December 31,
2019, unless otherwise indicated.
Number of
securities
remaining
available for
future
issuance
under equity
compensation
plans
(excluding
securities
reflected
in column
(c) (1)
(a))
14,048,065
—
14,048,065
Number of
securities
to be issued
upon
exercise of
outstanding
awards
(a) (1)
15,135,664 $
872,187 $
16,007,851
Weighted-
average
exercise
price
of
outstanding
awards
(b) (2)
1.79
1.51
Plan Category
Equity compensation plan approved by security holders
Equity compensation plan not approved by security holders (3)
Total
_________________
(1)
(2)
(3)
Included in column (a) and (c) are both stock option and RSU awards under our equity compensation plans.
Reflects the weighted-average exercise price of outstanding stock options only, as opposed to RSUs that do not have
an exercise price. The weighted average exercise price of all outstanding stock option awards under our plans is $1.77
and the weighted average remaining term is 7.11 years.
The shares of common stock included in this plan category are issuable pursuant to outstanding awards under the
Athersys, Inc. Equity Incentive Compensation Plan. This plan expired on June 8, 2017; therefore, no new awards can
be issued under this plan.
ITEM 13.
CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTOR
INDEPENDENCE
The information required by this item is incorporated by reference to the Proxy Statement with respect to the 2020 Annual
Meeting of Stockholders.
ITEM 14.
PRINCIPAL ACCOUNTANT FEES AND SERVICES
The information required by this item is incorporated by reference to the Proxy Statement with respect to the 2020 Annual
Meeting of Stockholders.
80
PART IV
ITEM 15.
EXHIBITS AND FINANCIAL STATEMENT SCHEDULES
(a)(1) Financial Statements:
The following consolidated financial statements of Athersys, Inc. are included in Item 8:
Reports of Independent Registered Public Accounting Firm
Consolidated Balance Sheets as of December 31, 2019 and 2018
Consolidated Statements of Operations and Comprehensive Loss for each of the years ended December 31, 2019,
2018 and 2017
Consolidated Statements of Stockholders’ Equity for each of the years ended December 31, 2019, 2018 and 2017
Consolidated Statements of Cash Flow for each of the years ended December 31, 2019, 2018 and 2017
Notes to Consolidated Financial Statements
(a)(2) Financial Statement Schedules:
The following financial statement schedule of Athersys, Inc. is included:
Schedule II – Valuation and Qualifying Accounts
Balance at
Beginning
of Year
Additions
Deductions
Balance at
End of Year
(In thousands)
Year Ended December 31, 2019
Deducted from asset accounts:
Tax valuation allowances
Total 2019
Year Ended December 31, 2018
Deducted from asset accounts:
Tax valuation allowances
Total 2018
Year Ended December 31, 2017
Deducted from asset accounts:
Allowance for doubtful accounts-note receivable
Tax valuation allowances
Total 2017
$
$
$
$
$
$
51,510 $
51,510 $
11,528 $
11,528 $
— $
— $
63,038 (A)
63,038
44,829 $
44,829 $
6,681 $
6,681 $
— $
— $
51,510 (A)
51,510
376 $
54,772
55,148 $
— $
—
— $
(376 ) $
(9,943 )
(10,319 ) $
— (B)
44,829 (A)
44,829
(A) – Substantially all our deferred tax assets are offset by valuation allowances.
(B) – Reserve on note receivable that was fully-reserved. We wrote-off the note in 2017.
All other schedules for which provision is made in the applicable accounting regulation of the SEC are not required under the
related instructions or are inapplicable and, therefore, omitted.
81
(a)(3) Exhibits.
Exhibit No.
Exhibit Description
3.1
3.2
3.3
4.1
4.2
4.3
10.1*
10.2*
10.3
10.4*
10.5
10.6
10.7†
10.8†
10.9†
Certificate of Incorporation of Athersys, Inc., as amended as of June 20, 2013 (incorporated herein by reference
to Exhibit 3.1 to the registrant’s Quarterly Report on Form 10-Q (Commission No. 001-33876) filed with the
Commission on May 8, 2019)
Certificate of Amendment to Certificate of Incorporation of Athersys, Inc., as amended as of June 7, 2017
(incorporated herein by reference to Exhibit 3.1 to the registrant’s Quarterly Report on Form 10-Q
(Commission No. 001-33876) filed with the Commission on August 9, 2017)
Bylaws of Athersys, Inc., as amended and restated as of March 13, 2019 (incorporated herein by reference to
Exhibit 3.1 to the registrant’s Current Report on Form 8-K (Commission No. 001-33876) filed with the
Commission on March 14, 2019)
Common Stock Purchase Warrant issued to HEALIOS K.K. by Athersys, Inc. dated March 14, 2018
(incorporated herein by reference to Exhibit 4.1 to the registrant's Quarterly Report on Form 10-Q
(Commission 001-33876) filed with the Commission on May 10, 2018)
Amendment No. 1 to Common Stock Purchase Warrant issued to HEALIOS K.K. by Athersys, Inc. dated as of
June 6, 2018 (incorporated herein by reference to Exhibit 4.1 to the registrants Quarterly Report on Form 10-Q
(Commission No. 001-33876) filed with the Commission on August 9, 2018)
Description of the Registrant's Securities Registered Pursuant to Section 12 of the Securities Exchange Act of
1934
Research Collaboration and License Agreement, dated as of December 8, 2000, by and between Athersys, Inc.
and Bristol-Myers Squibb Company (incorporated herein by reference to Exhibit 10.1 to the registrant’s
Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission on June 14, 2007)
Cell Line Collaboration and License Agreement, dated as of July 1, 2002, by and between Athersys, Inc. and
Bristol-Myers Squibb Company (incorporated herein by reference to Exhibit 10.2 to the registrant’s Current
Report on Form 8-K/A (Commission No. 000-52108) filed with the Commission on September 27, 2007)
Amendment No. 1 to Cell Line Collaboration and License Agreement, dated as of January 1, 2006, by and
between Athersys, Inc. and Bristol-Myers Squibb Company (incorporated herein by reference to Exhibit 10.36
to the registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission on
June 14, 2007)
Extended Collaboration and License Agreement, dated as of January 1, 2006, by and between Athersys, Inc.
and Bristol-Myers Squibb Company (incorporated herein by reference to Exhibit 10.3 to the registrant’s
Current Report on Form 8-K/A (Commission No. 000-52108) filed with the Commission on September 27,
2007)
Amendment dated as of March 31, 2009 to the Extended Collaboration and License Agreement, by and
between Athersys, Inc. and Bristol-Myers Squibb Company effective January 1, 2006 (incorporated herein by
reference to Exhibit 10.1 to the registrant’s Current Report on Form 8-K (Commission No. 001-33876) filed
with the Commission on April 9, 2009)
Amendment No. 3 to Extended Collaboration and License Agreement, dated January 31, 2012, by and between
ABT Holding Company and Bristol-Myers Squibb Company (incorporated by reference to Exhibit 10.3 to the
registrant’s Quarterly Report on Form 10-Q (Commission No. 001-33876) filed with the Commission on
May 14, 2012)
Athersys, Inc. Equity Incentive Compensation Plan (incorporated herein by reference to Exhibit 10.11 to the
registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission on June 14,
2007)
Amended and Restated Employment Agreement, dated as of December 1, 1998 but effective as of April 1,
1998, by and between Athersys, Inc. and Dr. Gil Van Bokkelen (incorporated herein by reference to Exhibit
10.14 to the registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission
on June 14, 2007)
Amendment No. 1 to Amended and Restated Employment Agreement, dated as of May 31, 2007, by and
between Advanced Biotherapeutics, Inc. and Gil Van Bokkelen (incorporated herein by reference to Exhibit
10.15 to the registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission
on June 14, 2007)
82
10.10†
10.11†
10.12†
10.13†
10.14†
10.15†
10.16†
10.17†
10.18†
10.19†
10.20†
10.21†
10.22†
10.23*
10.24
Non-Competition and Confidentiality Agreement, dated as of December 1, 1998, by and between Athersys, Inc.
and Dr. Gil Van Bokkelen (incorporated herein by reference to Exhibit 10.16 to the registrant’s Current Report
on Form 8-K (Commission No. 000-52108) filed with the Commission on June 14, 2007)
Amended and Restated Employment Agreement, dated as of December 1, 1998 but effective as of April 1,
1998, by and between Athersys, Inc. and Dr. John J. Harrington (incorporated herein by reference to Exhibit
10.17 to the registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission
on June 14, 2007)
Amendment No. 1 to Amended and Restated Employment Agreement, dated as of May 31, 2007, by and
between Advanced Biotherapeutics, Inc. and John Harrington (incorporated herein by reference to Exhibit
10.18 to the registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission
on June 14, 2007)
Non-Competition and Confidentiality Agreement, dated as of December 1, 1998, by and between Athersys, Inc.
and Dr. John J. Harrington (incorporated herein by reference to Exhibit 10.19 to the registrant’s Current Report
on Form 8-K (Commission No. 000-52108) filed with the Commission on June 14, 2007)
Employment Agreement, dated as of May 22, 1998, by and between Athersys, Inc. and Laura K. Campbell
(incorporated herein by reference to Exhibit 10.20 to the registrant’s Current Report on Form 8-K (Commission
No. 000-52108) filed with the Commission on June 14, 2007)
Amendment No. 1 to Employment Agreement, dated as of May 31, 2007, by and between Advanced
Biotherapeutics, Inc. and Laura Campbell (incorporated herein by reference to Exhibit 10.21 to the registrant’s
Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission on June 14, 2007)
Employment Agreement, dated as of January 1, 2004, by and between Advanced Biotherapeutics, Inc. and
William Lehmann (incorporated herein by reference to Exhibit 10.28 to the registrant’s Current Report on
Form 8-K (Commission No. 000-52108) filed with the Commission on June 14, 2007)
Amendment No. 1 to Employment Agreement, dated as of May 31, 2007, by and between Advanced
Biotherapeutics, Inc. and William Lehmann (incorporated herein by reference to Exhibit 10.29 to the
registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission on June 14,
2007)
Amendment No. 2 to Employment Agreement, dated as of January 24, 2014, by and between Advanced
Biotherapeutics, Inc. and William Lehmann (incorporated herein by reference to Exhibit 10.24 to the
registrant’s Annual Report on Form 10-K for the year ended December 31, 2013 (Commission No. 001-33876)
filed with the Commission on March 13, 2014)
Non-Competition and Confidentiality Agreement, dated as of September 10, 2001, by and among Athersys,
Inc., Advanced Biotherapeutics, Inc. and William Lehmann (incorporated herein by reference to Exhibit 10.30
to the registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission on
June 14, 2007)
Form Incentive Agreement by and between Advanced Biotherapeutics, Inc. and named executive officers, and
acknowledged by Athersys, Inc. and ReGenesys, LLC (incorporated herein by reference to Exhibit 10.31 to the
registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed with the Commission on June 14,
2007)
Form Amendment No. 1 to Incentive Agreement by and between Advanced Biotherapeutics, Inc. and named
executive officers, and acknowledged by Athersys, Inc. and ReGenesys, LLC (incorporated herein by reference
to Exhibit 10.32 to the registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed with the
Commission on June 14, 2007)
Form Amendment No. 2 to Incentive Agreement by and between Advanced Biotherapeutics, Inc. and named
executive officers, and acknowledged by Athersys, Inc. and ReGenesys, LLC (incorporated herein by reference
to Exhibit 10.2 to the registrant’s Current Report on Form 8-K (Commission No. 001-33876) filed with the
Commission on June 20, 2013)
Exclusive License Agreement, dated as of May 17, 2002, by and between Regents of the University of
Minnesota and MCL LLC, assumed by ReGenesys, LLC through operation of merger on November 4, 2003
(incorporated herein by reference to Exhibit 10.34 to the registrant’s Current Report on Form 8-K (Commission
No. 000-52108) filed with the Commission on June 14, 2007)
Form Indemnification Agreement for Directors, Officers and Directors and Officers (incorporated herein by
reference to Exhibit 10.1 to the registrant’s Current Report on Form 8-K (Commission No. 000-52108) filed
with the Commission on August 6, 2007)
83
10.26†
10.27†
10.28†
10.29†
10.30†
10.31†
10.34
10.35
10.36
10.37
10.38
10.40
Form of Incentive Stock Option Agreement (incorporated herein by reference to Exhibit 10.47 to the
registrant’s Annual Report on Form 10-K for the year ended December 31, 2010 (Commission No. 001-33876)
filed with the Commission on March 25, 2011)
Form of Nonqualified Stock Option Agreement for Non-Employee Directors (incorporated herein by reference
to Exhibit 10.48 to the registrant’s Annual Report on Form 10-K for the year ended December 31, 2010
(Commission No. 001-33876) filed with the Commission on March 25, 2011)
Athersys, Inc. Amended and Restated 2007 Long-Term Incentive Plan (incorporated herein by reference to
Exhibit 4.1 to the Company’s Registration Statement on Form S-8 (Registration No. 333-212119) filed with the
Securities and Exchange Commission on June 20, 2016)
Form of Nonqualified Stock Option Agreement for Non-Employee Directors pursuant to the Athersys, Inc.
Amended and Restated 2007 Long-Term Incentive Plan (Amended and Restated Effective June 16, 2011)
(incorporated herein by reference to Exhibit 10.49 to the registrant’s Quarterly Report on Form 10-Q
(Commission No. 001-33876) filed with the Commission on May 6, 2011)
Form of Restricted Stock Unit Agreement (incorporated herein by reference to Exhibit 10.2 to the registrant’s
Quarterly Report on Form 10-Q (Commission No. 001-33876) filed with the Commission on August 10, 2011)
Form of Restricted Stock Unit Agreement (incorporated herein by reference to Exhibit 10.2 to the registrant’s
Current Report on Form 8-K (Commission No. 001-33876) filed with the Commission on June 20, 2013)
License Agreement by and between ABT Holding Company and Healios K.K., dated as of January 8, 2016
(incorporated herein by reference to Exhibit 10.1 to the registrant’s Quarterly Report on Form 10-Q
(Commission No. 001-33876) filed with the Commission on May 5, 2016)
Common Stock Purchase Agreement, dated as of February 1, 2018, by and between Athersys, Inc. and Aspire
Capital Fund, LLC (incorporated herein by reference to Exhibit 10.1 to the registrant’s Current Report on Form
8-K (Commission No. 001-33876) filed with the Commission on February 1, 2018)
Registration Rights Agreement, dated as of February 1, 2018, by and between Athersys, Inc. and Aspire Capital
Fund, LLC (incorporated herein by reference to Exhibit 10.2 to the registrant’s Current Report on Form 8-K
(Commission No. 001-33876) filed with the Commission on February 1, 2018)
First Amendment to License Agreement, dated as of July 21, 2017, by and between ABT Holding Company
and Healios K.K. (incorporated herein by reference to Exhibit 10.1 to the registrant’s Quarterly Report on Form
10-Q (Commission No. 001-33876) filed with the Commission on November 8, 2017)
Second Amendment to License Agreement, dated as of September 19, 2017, by and between ABT Holding
Company and Healios K.K. (incorporated herein by reference to Exhibit 10.2 to the registrant’s Quarterly
Report on Form 10-Q (Commission No. 001-33876) filed with the Commission on November 8, 2017)
Investor Rights Agreement, by and between Athersys, Inc. and HEALIOS K.K., dated as of March 13, 2018
(incorporated herein by reference to Exhibit 10.2 to the registrant's Quarterly Report on Form 10-Q
(Commission No. 001-33876) filed with the Commission on May 10, 2018)
10.41 *
Collaboration Expansion Agreement, by and between Athersys, Inc. and HEALIOS K.K., dated as of June 6,
2018 (incorporated herein by reference to Exhibit 10.1 to the registrant's Quarterly Report on Form 10-Q
(Commission No. 001-33876) filed with the Commission on August 9, 2018)
10.42
10.43
10.44
Amendment No. 1 to Collaboration Expansion Agreement, by and between Athersys, Inc. and HEALIOS K.K.,
dated as of August 31, 2018 (incorporated herein by reference to Exhibit 10.1 to the registrant's Quarterly
Report on Form 10-Q (Commission No. 001-33876) filed with the Commission on November 6, 2018)
Amendment No. 2 to Collaboration Expansion Agreement, by and between Athersys, Inc. and HEALIOS K.K.,
dated as of December 6, 2018 (incorporated herein by reference to Exhibit 10.44 to the registrant's Annual
Report on Form 10-K (Commission No. 001-33876) filed with the Commission on March 15, 2019)
Amendment No. 3 to Collaboration Expansion Agreement, by and between Athersys, Inc. and HEALIOS K.K.,
dated as of December 14, 2018 (incorporated herein by reference to Exhibit 10.45 to the registrant's Annual
Report on Form 10-K (Commission No. 001-33876) filed with the Commission on March 15, 2019)
10.45†
Summary of Athersys, Inc. 2020 Cash Bonus Incentive Plan
10.46†
Athersys, Inc. 2019 Equity and Incentive Compensation Plan (incorporated herein by reference to Exhibit 4.4
to the registrant's Registration Statement on Form S-8 (Registration No. 333-232075) filed with the
Commission on June 12, 2019)
84
10.47†
10.48†
10.49†
10.50†
10.51†
10.52
10.53
21.1
23.1
24.1
31.1
31.2
32.1
Form of Incentive Stock Option Agreement pursuant to the Athersys, Inc. 2019 Equity and Incentive
Compensation Plan (incorporated herein by reference to Exhibit 10.2 to the registrant's Quarterly Report on
Form 10-Q (Commission No. 001-33876) filed with the Commission on August 7, 2019)
Form of Non-Qualified Stock Option Agreement pursuant to the Athersys, Inc. 2019 Equity and Incentive
Compensation Plan (incorporated herein by reference to Exhibit 10.3 to the registrant's Quarterly Report on
Form 10-Q (Commission No. 001-33876) filed with the Commission on August 7, 2019)
Form of Non-Qualified Stock Option Agreement (Directors) pursuant to the Athersys, Inc. 2019 Equity and
Incentive Compensation Plan (incorporated herein by reference to Exhibit 10.4 to the registrant's Quarterly
Report on Form 10-Q (Commission No. 001-33876) filed with the Commission on August 7, 2019)
Form of Restricted Stock Unit Agreement (Executives) pursuant to the Athersys, Inc. 2019 Equity and
Incentive Compensation Plan (incorporated herein by reference to Exhibit 10.5 to the registrant's Quarterly
Report on Form 10-Q (Commission No. 001-33876) filed with the Commission on August 7, 2019)
Form of Restricted Stock Unit Agreement (Non-Executive) pursuant to the Athersys, Inc. 2019 Equity and
Incentive Compensation Plan (incorporated herein by reference to Exhibit 10.6 to the registrant's Quarterly
Report on Form 10-Q (Commission No. 001-33876) filed with the Commission on August 7, 2019)
Common Stock Purchase Agreement, dated as of November 5, 2019 by and between Athersys, Inc. and Aspire
Capital Fund, LLC (incorporated herein by reference to Exhibit 10.1 to the registrant's Quarterly Report on
Form 10-Q (Commission No. 001-33876) filed with the Commission on November 6, 2019)
Registration Rights Agreement, dated as of November 5, 2019, by and between Athersys, Inc. and Aspire
Capital Fund, LLC (incorporated herein by reference to Exhibit 10.2 to the registrant's Quarterly Report on
Form 10-Q (Commission No. 001-33876) filed with the Commission on November 6, 2019)
List of Subsidiaries
Consent of Ernst & Young LLP, Independent Registered Public Accounting Firm
Power of Attorney
Certification of Gil Van Bokkelen, Chairman and Chief Executive Officer, pursuant to SEC Rules 13a-14(a)
and 15d-14(a) adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.
Certification of Laura K. Campbell, Senior Vice President of Finance, pursuant to SEC Rules 13a-14(a) and
15d-14(a) adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.
Certification of Gil Van Bokkelen, Chairman and Chief Executive Officer, and Laura Campbell, Senior Vice
President of Finance, pursuant to 18 U.S.C. Section 1350, adopted pursuant to Section 906 of the Sarbanes-
Oxley Act of 2002.
101.INS
XBRL Instance Document
101.SCH
XBRL Taxonomy Extension Schema Document
101.CAL
XBRL Taxonomy Extension Calculation Linkbase Document
101.DEF
XBRL Taxonomy Extension Definition Linkbase Document
101.LAB
XBRL Taxonomy Extension Label Linkbase Document
101.PRE
XBRL Taxonomy Extension Presentation Linkbase Document
_______________
*
†
Confidential treatment requested as to certain portions, which portions have been filed separately with the SEC
Indicates management contract or compensatory plan, contract or arrangement in which one or more directors or
executive officers of the registrant may be participants
ITEM 16.
None.
FORM 10-K SUMMARY
85
Pursuant 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 signed on its behalf by the undersigned, thereunto duly authorized, in the city of Cleveland, State of Ohio, on
March 16, 2020.
SIGNATURES
ATHERSYS, INC.
By:
/s/ Gil Van Bokkelen
Gil Van Bokkelen
Title: Chief Executive Officer and
Chairman
Pursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed below by the following
persons on behalf of the registrant and in the capacities and on the date indicated.
Signature
/s/ Gil Van Bokkelen
Gil Van Bokkelen
/s/ Laura K. Campbell
Laura K. Campbell
*
John J. Harrington
*
Hardy TS Kagimoto
*
Lorin J. Randall
*
Title
Chief Executive Officer and Chairman of
the Board of Directors (Principal Executive
Officer)
Senior Vice President of Finance (Principal
Financial Officer and Principal Accounting
Officer)
Executive Vice President, Chief Scientific
Officer and Director
Director
Director
Jack L. Wyszomierski
Director
*
Lee E. Babiss
*
Ismail Kola
Director
Director
Date
March 16, 2020
March 16, 2020
March 16, 2020
March 16, 2020
March 16, 2020
March 16, 2020
March 16, 2020
March 16, 2020
*
Gil Van Bokkelen, by signing his name hereto, does hereby sign this Form 10-K on behalf of each of the above named
and designated directors of the Company pursuant to Powers of Attorney executed by such persons and filed with the
Securities and Exchange Commission.
By:
/s/ Gil Van Bokkelen
Gil Van Bokkelen
Attorney-in-fact
86
CERTIFICATIONS
EXHIBIT 31.1
I, Gil Van Bokkelen, certify that:
1.
2.
3.
4.
I have reviewed this annual report on Form 10-K of Athersys, Inc.;
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 the statements made, in light of the circumstances under which such statements were made, not
misleading with respect to the period covered by this report;
Based on my knowledge, the financial statements, and other financial information included in this report, fairly present
in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the
periods presented in this report;
The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and
procedures (as defined in Exchange 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 the registrant and have:
(a)
(b)
(c)
(d)
Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be
designed under our supervision, to ensure 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;
Designed such internal control over financial reporting, or caused such internal control over financial reporting to
be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting
and the preparation of financial statements for external purposes in accordance with generally accepted
accounting principles;
Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our
conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered
by this report based on such evaluation; and
Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during
the registrant’s most recent fiscal 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; and
5.
The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control
over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or
persons performing the equivalent functions):
(a)
(b)
All significant deficiencies and material weaknesses in the design or operation of internal control over financial
reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and
report financial information; and
Any fraud, whether or not material, that involves management or other employees who have a significant role in
the registrant’s internal control over financial reporting.
March 16, 2020
/s/ Gil Van Bokkelen
Gil Van Bokkelen
Chief Executive Officer and
Chairman of the Board of Directors
87
CERTIFICATIONS
EXHIBIT 31.2
I, Laura K. Campbell, certify that:
1.
2.
3.
4.
I have reviewed this annual report on Form 10-K of Athersys, Inc.;
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 the statements made, in light of the circumstances under which such statements were made, not
misleading with respect to the period covered by this report;
Based on my knowledge, the financial statements, and other financial information included in this report, fairly present
in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the
periods presented in this report;
The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and
procedures (as defined in Exchange 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 the registrant and have:
(a)
(b)
(c)
(d)
Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be
designed under our supervision, to ensure 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;
Designed such internal control over financial reporting, or caused such internal control over financial reporting to
be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting
and the preparation of financial statements for external purposes in accordance with generally accepted
accounting principles;
Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our
conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered
by this report based on such evaluation; and
Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during
the registrant’s most recent fiscal 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; and
5.
The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control
over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or
persons performing the equivalent functions):
(a)
(b)
All significant deficiencies and material weaknesses in the design or operation of internal control over financial
reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and
report financial information; and
Any fraud, whether or not material, that involves management or other employees who have a significant role in
the registrant’s internal control over financial reporting.
March 16, 2020
/s/ Laura K. Campbell
Laura K. Campbell
Senior Vice President of Finance
88
EXHIBIT 32.1
CERTIFICATION PURSUANT TO
18 U.S.C. SECTION 1350,
AS ADOPTED PURSUANT TO
SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002
In connection with the Annual Report of Athersys, Inc. (the “Company”) on Form 10-K for the year ended December 31, 2019,
as filed with the Securities and Exchange Commission on the date hereof (the “Report”), each of the undersigned officers of the
Company certifies, pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002,
that, to such officer’s knowledge:
(1) The Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and
(2) The information contained in the Report fairly presents, in all material respects, the financial condition and results of
operations of the Company as of the dates and for the periods expressed in the Report.
Date: March 16, 2020
/s/ Gil Van Bokkelen
Name: Gil Van Bokkelen
Title: Chairman and Chief Executive Officer
/s/ Laura K. Campbell
Name: Laura K. Campbell
Title: Senior Vice President of Finance
The foregoing certification is being furnished solely pursuant to 18 U.S.C. Section 1350 and is not being filed as part of the
Report or as a separate disclosure document.
89
MANAGEMENT
Gil Van Bokkelen, Ph.D.
BOARD OF DIRECTORS
Gil Van Bokkelen, Ph.D.
STOCKHOLDER INFORMATION
Corporate Headquarters
Chairman and Chief Executive Officer
William (B.J.) Lehmann, Jr., J.D.
President and Chief Operating Officer
John Harrington, Ph.D.
Chairman and Chief Executive Officer
of Athersys, Inc. and Chairman of the
Board of Governors of the National
Center for Regenerative Medicine
Lee. E. Babiss, Ph.D.
Chief Scientific Officer, Executive Vice
President
Ivor Macleod, M.B.A, C.P.A.
Chief Financial Officer
Laura Campbell, C.P.A.
Senior Vice President of Finance
Manal Morsy, M.D., Ph.D., MBA
Senior Vice President, Head of Global
Regulatory Affairs
Greg Liposky, M.B.A.
Senior Vice President, Commercial
Manufacturing
Maia Hansen, M.B.A.
Lead Director
Retired; performing consulting
services for drug discovery
companies. Formerly Chief Scientific
Officer and Excecutive Vice President
of Discovery Innovation of PPD, Inc.
John J. Harrington, Ph.D.
Chief Scientific Officer, Executive
Vice President and Director of
Athersys, Inc.
Hardy TS Kagimoto, M.D.
Chairman and Chief Executive Officer
of HEALIOS K.K.
Ismail Kola, Ph.D.
Athersys, Inc.
3201 Carnegie Avenue
Cleveland, OH 44115-2634
T: (216) 431-9900
F: (216) 361-9495
www.athersys.com
ndependent Auditors
I
Ernst & Young LLP
Cleveland, OH 44113
Investor Relations
Karen Hunady
T: (216) 431-9900 ext. 511
khunady@athersys.com
Registrar and Transfer Agen
t
Computershare
PO Box 505000
Louisville, KY 40233-5000
T: (781) 575-2879
www.computershare.com
Stockholder Inquiries
Senior Vice President, Head of
Operations and Supply Chain
Robert (Willie) Mays, Ph.D.
Vice President of Regenerative
Medicine, Head of Neuroscience
Programs
Anthony Ting, Ph.D.
Vice President of Regenerative
Medicine, Head of Cardiopulmonary
Programs
Rakesh Ramachandran, M.S.
Vice President, Head of Information
Technology and Communications
Eric Jenkins, M.D.
Senior Medical Director, Head of
Clinical Operations
Formerly Executive Vice President
of UCB S.A. and President and
Chief Scientific Officer of UCB New
Medicines. In retirement, currently
Senior Partner of Forepont Capital.
Lorin J. Randall
Independent financial consultant;
former Senior Vice President and
Chief Financial Officer of Eximias
Pharmaceutical Corporation
Jack L. Wyszomierski
Questions regarding stock transfer
requirements, lost certificates
and change of address should be
directed to the transfer agent,
Computershare. Other stockholder
or investor inquiries, including
requests for our filings with the U.S.
Securities and Exchange Commission
or other information, should be
directed to ir@athersys.com.
Stock Listing
Retired; former Executive Vice
President and Chief Financial Officer
of VWR International, LLC
The Company’s common stock trades
on the NASDAQ Capital Market under
the symbol “ATHX”.
MEDIA RELATIONS
Russo Partners, LLC
David Schull
T: (212) 845-4271
David.schull@russopartnersllc.com
This annual report contains forward-looking statements. These statements are based on certain assumptions and management’s current knowledge. Accordingly,
we caution you not to unduly rely on forward-looking statements, which speak only as of the date of this annual report. We intend these statements to be covered
by the safe harbor provision of the Private Securities Litigation Reform Act of 1995. The words “expects,” “anticipates,” “believes,” “may,” “should,” “projects,”
“forecasts,” “will,” and similar expressions are intended to identify forward-looking statements. We caution you that forward-looking statements involve risks and
uncertainties that could cause acual results to vary from those statements. For a discussion of these risks see “Item 1A - Risk Factors” in the Company’s Annual
Report on Form 10-K included herein. The Company undertakes no obligation to updated any forward-looking statement.
Athersys.com
3201 Carnegie Avenue
Cleveland, OH 44115-2634