Acorda Therapeutics
Annual Report 2014

Plain-text annual report

Spinal Cord Injury S p a s t i c i t y M u l t i p l e S c l e r o s i s Stroke Parkinson’s Disease Ep ilepsy Heart Failure Migraine 2014 ANNUAL REPORT Acorda’s mission is to develop therapies that restore function and improve the lives of people with neurological disorders. Rick DIAGNOSED WITH MS IN 1994 Dear Members of the Acorda Community: In 2014, we made significant strides in our mission to develop therapies that restore function and improve the lives of people with neurological disorders. Our progress was enabled by our grounding in scientific, medical and business excellence, and our focus on three core value drivers: AMPYRA® commercial performance; clinical pipeline; and business development. We responded effectively to these challenges, initiating our Phase 3 post-stroke study with our existing twice-daily formulation of dalfampridine and also launching work on a new QD formulation with several independent collaborators. We have also engaged with FDA regarding PLUMIAZ and expect to provide a detailed update in the second quarter of 2015. As anticipated, a number of parties have filed challenges to our AMPYRA patents. Our legal team is prepared for these challenges. We have five Orange Book listed patents on AMPYRA that extend through 2027, and will vigorously defend our intellectual property. Looking ahead, we are focused on execution in the areas that are critical to growing our business in the coming years: 1 2 3 Continue to build value in the AMPYRA brand in MS. Execute successfully on our high-value pipeline programs. Identify additional business development opportunities that will build on Acorda’s leading capabilities in neurology development and specialty pharmaceutical commercialization. I want to take this opportunity to recognize Acorda’s associates for their dedication and the urgency they bring to developing cutting edge therapies. I’m especially pleased that our newest colleagues from Civitas share this commitment; the integration of our two companies has resulted in a stronger Acorda with a substantially enhanced product pipeline and capabilities. On behalf of our management team and Board of Directors, thank you to our shareholders for your continued support. We are excited about our opportunities to build value in 2015 and beyond, and I look forward to updating you on our progress. Ron Cohen President and CEO Highlights in 2014 included: AMPYRA net revenue increased 21% in 2014 to $366 million. Due to the efforts and expertise of our commercial and medical education teams, AMPYRA is increasingly considered a standard of care among neurologists and the multiple sclerosis (MS) community for improving walking in MS. More than 100,000 people with MS in the U.S. have tried this important therapy since its launch in 2010, and we have been gratified to hear frequently from people who have benefited from AMPYRA about how much this has meant to them and their families. We are projecting continued growth of AMPYRA in 2015, with anticipated net sales between $405-$420 million. We acquired Civitas Therapeutics, adding global rights for two promising compounds to our pipeline, as well as the ARCUS technology platform, which has potential applications in multiple disease states. We also acquired a commercial-scale manufacturing facility for ARCUS-based products. We advanced two therapies into Phase 3 clinical trials: dalfampridine for chronic post-stroke walking deficits (PSWD) and CVT-301 for OFF episodes in Parkinson’s disease. Both of these compounds would address large patient populations with significant unmet medical needs. We completed our first Phase 1 study of rHIgM22 for remyelination in MS. The safety and tolerability findings support continued development of this therapy, which has a novel mechanism. We expect to initiate a second Phase 1 trial in the second quarter of 2015. Remyelination is widely considered to be one of the next frontiers in the treatment of MS. We are excited to be advancing this therapy in clinical trials. We continued to enroll the second Phase 1 study of cimaglermin alfa (also known as GGF2) in heart failure, and expect it to be completed in the second half of 2015. Cimaglermin represents a novel approach to improving heart function; our first Phase 1 trial showed additive improvements in cardiac ejection fraction over optimized therapy in people with Class 2 and 3 heart failure. We ended 2014 in a strong financial position, with over $300 million in cash and projecting a cash flow positive year in 2015. We also experienced two disappointments in 2014: a proposed once-daily (QD) formulation of dalfampridine proved inadequate for inclusion in our post-stroke Phase 3 program, and we received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding our New Drug Application (NDA) for approval of PLUMIAZ to treat seizure clusters. However, the mark of success is not the absence of disappointments, but in how one responds to them. 2 ADVANCED PIPELINE AMPYRA® (DALFAMPRIDINE) $366.2 million in 2014 net sales, a 21% increase over 2013 More than 100,000 people with MS have tried AMPYRA since launch ZANAFLEX® (TIZANIDINE HCI) QUTENZA® (CAPSAICIN) DALFAMPRIDINE Initiated Phase 3 clinical trial for chronic post-stroke walking deficits (PSWD) in December 2014 CVT-301 Initiated Phase 3 clinical trial for OFF episodes in Parkinson’s disease in December 2014 PLUMIAZ™ CIMAGLERMIN ALFA Initiated second Phase 1 heart failure clinical trial in November 2013; results expected in 2015 rHlgM22 Completed first Phase 1 MS clinical trial in 2014 Expect to initiate second Phase 1 study in 2Q 2015 CVT-427 Preparing for first Phase 1 clinical trial 3  We hav e a de e p u nde r s tandi ng o f and e xp e rie nc e i n ne rv o u s s ys t e m re s earch , pro d u ct de v e l o p me nt and c o mme rcializatio n. CIVITAS THERAPEUTICS ACQUISITION In October 2014, we completed the acquisition of Civitas Therapeutics, a privately-held biotechnology company with two novel neurology products in development. There are three aspects that made this transaction very attractive to us, and that have the potential to deliver substantial value over time: PRODUCTS: We obtained global rights to CVT-301 and CVT-427, ARCUS® TECHNOLOGY: ARCUS is a proprietary dry-powder pulmonary delivery in development for Parkinson’s disease and migraine, system that has potential applications in multiple disease respectively. CVT-301 offers a late-stage opportunity that areas. This platform is being studied to allow consistent addresses a clear unmet need for people with Parkinson’s and precise delivery of significantly larger doses of disease—treating OFF episodes, which become more medication than are possible with conventional pulmonary frequent as the disease progresses. OFF episodes delivery systems. The ARCUS inhaler is a passive, breath- are defined by periods of time when background actuated, reusable device operated by the user breathing in levodopa (L-dopa) therapy does not adequately control on the mouthpiece. The ARCUS technology has been used Parkinson’s symptoms, leading to reduced ability to to deliver more than one million doses to patients in clinical move, muscle stiffness and tremor. These episodes can trials of various compounds. be very disruptive to the lives of people with Parkinson’s disease, their families and caregivers. CVT-301 utilizes the ARCUS® technology and is being studied to provide Our acquisition of Civitas included a commercial-scale manufacturing facility for ARCUS-based products, located rapid, reliable delivery of a precise dose of L-dopa in Chelsea, MA. through the lungs to return people with Parkinson’s to an ON state; L-dopa is viewed as the gold standard in the treatment of Parkinson’s disease. We initiated a Phase 3 TEAM: Just as important as the products and technology, the Civitas clinical trial of CVT-301 in December 2014. team joining Acorda has strengthened our organization. In CVT-427 is a triptan-based migraine therapy; we are experience with the development programs for CVT-301 and preparing to initiate the first Phase 1 clinical trial of CVT-427, our organizations were very closely aligned in terms this compound. CVT-427 also utilizes the ARCUS of culture, values and commitment to the patients we serve. addition to their expertise in the ARCUS technology and technology platform. 5 2015 GUIDANCE Our guidance range reflects We’re investing in late-stage trials We place a high priority on continued confidence in the growth for CVT-301, dalfampridine in managing SG&A; our guidance of AMPYRA, increasing from $366.2 chronic post-stroke walking deficits represents a minimal increase over million in net sales from 2014. (PSWD) and PLUMIAZ. Acorda has 5 clinical-stage programs that have potential to improve the lives of millions of patients and drive shareholder value. 2014 despite added infrastructure related to the Civitas acquisition. 2014 HIGHLIGHTS 6 MANAGEMENT TEAM Ron Cohen, M.D. David Lawrence, M.B.A. President & Chief Executive Officer Chief of Business Operations Richard P. Batycky, Ph.D. Michael Rogers Chief Technology Officer Chief Financial Officer and Site Head Andrew R. Blight, Ph.D. Chief Scientific Officer Lauren Sabella Chief Commercial Officer Tierney Saccavino Enrique J. Carrazana, M.D. Executive Vice President, Chief Medical Officer Corporate Communications Denise Duca, Ed. M. Executive Vice President, Human Resources Jane Wasman, J.D. President, International & General Counsel Andrew A. Hindman Chief Business Development Officer BOARD OF DIRECTORS Ron Cohen, M.D. Founder Barry Greene Board Member since 2007 Peder K. Jensen, M.D. Board Member since 2011 John P. Kelley Board Member since 2008 Sandra Panem, Ph.D. Lorin J. Randall Board Member since 1998 Board Member since 2006 Steven M. Rauscher Ian F. Smith Board Member since 2005 Board Member since 2007 8 Kristie DIAGNOSED WITH MS IN 1999 CONTACT: Jeff Macdonald Senior Director, Corporate Communications jmacdonald@acorda.com 914-326-5232 

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