ATHERSYS CORPORATE FACT SHEET WINTER 2018 NASDAQ: ATHX

Transcription

1 ATHERSYS CORPORATE FACT SHEET WINTER 2018 Athersys () is an international biotechnology company that is focused in the field of regenerative medicine. We are committed to the development of innovative and best-in-class therapies designed to extend and enhance the quality of human life. We have established a portfolio of therapeutic product development programs to address significant unmet medical need. Our MultiStem cell therapy, a patented and proprietary, off-the-shelf stem cell product, is our lead platform product and is currently in latestage clinical development. Our programs are focused on treating neurological conditions, cardiovascular disease, inflammatory and immune disorders, and other conditions where the current standard of care is limited or inadequate for many patients.

2 CORPORATE FACT SHEET WINTER 2017 KEY PROGRAMS Ischemic Stroke & Other Neurological Programs Our technology has shown promise in treating acute ischemic stroke and other indications where there is neurological damage or injury. In 2016, we completed a Phase 2 study of MultiStem cell therapy to treat patients suffering a moderate to severe ischemic stroke (referred to as the MASTERS study) and announced the final results from the study and positive oneyear follow-up data at the International Stroke Conference. Since that time, we have been actively preparing for the MASTERS-2 trial, a pivotal Phase 3 clinical trial in the U.S and Europe, which we expect to launch in the second quarter of 2018 beginning with specific high-enrolling sites. The design for the MASTERS-2 trial and planned analysis have been agreed upon with the FDA, and the study was granted a Special Protocol Assessment (SPA). The SPA provides agreement from the FDA that the protocol design, clinical endpoints, planned conduct and statistical analyses encompassed in Athersys planned Phase 3 study can address objectives in support of a regulatory submission for approval of the MultiStem product for treating ischemic stroke patients if the trial is successful. Furthermore, in May 2017, we announced that the FDA granted this program Fast Track designation, making the program eligible for rolling submission, accelerated approval and priority review of the biologics license application, facilitating a timely regulatory review. Also, in August 2017, we announced that the European Medicines Device Agency (EMA) had granted the program a Final Scientific Advice positive opinion, establishing alignment between European and U.S. regulators about the potential of approval based on the success of the planned MASTERS-2 study, further expediting development. In October 2017, we announced that we received the Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for this program, which was established under the landmark 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 address unmet medical needs for a serious or life-threatening disease or condition. The designation enables sponsors to interact with the FDA regarding multidisciplinary strategic development plans, including expediting manufacturing development plans for commercialization to support priority review and accelerated approval. In addition to the upcoming launch of the MASTERS-2 trial, MultiStem is being evaluated in ischemic stroke patients in the TREASURE trial in Japan, a confirmatory clinical trial being conducted by our partner, HEALIOS K.K. (Healios). This double-blind, randomized, placebocontrolled study is enrolling approximately 220 patients at leading stroke centers in Japan and is being conducted in accordance with the regulatory framework for regenerative medicine therapies recently implemented by the PMDA and MHLW. This framework is designed to expedite the development and commercialization of promising and innovative regenerative medicines that are shown to exhibit safety and demonstrate potential effectiveness. This program has also received Priority Review designation status in Japan under the Sakigake regulatory framework for innovative therapies. In November 2017, Healios announced that the first patient was enrolled in this trial, and enrollment continues to progress. In March 2018, we announced that we and Healios entered a letter of intent (LOI) to expand our collaboration. In doing so, Healios will commit capital in the amount of $56.1 million in the form of an initial equity investment and license fee payments, of which $31.1 million has already been committed or received. As part of the expansion, Healios would receive a license to MultiStem products for acute respiratory distress syndrome (ARDS) and trauma in Japan, Healios organ bud technology in Japan, and certain ophthalmological indications globally. Also, Healios would receive an exclusive option to license MultiStem products for ischemic stroke, ARDS and trauma in China, and Athersys would be entitled to license fees, milestone payments and escalating royalties for the licensed indications. In addition to our stroke clinical program, we and our collaborators have been actively engaged in evaluating the administration of MultiStem for various other areas of neurological injury and disease, including in preclinical models for the treatment of traumatic brain injury, acute spinal cord injury, neonatal hypoxic ischemia, multiple sclerosis and other indications. Much of this work has been published in leading peer review journals.

3 CORPORATE FACT SHEET WINTER 2017 KEY PROGRAMS Acute Myocardial Infarction (AMI) and Cardiovascular Disease MultiStem cell therapy has also shown promise in treating acute and chronic cardiovascular disease. We have an ongoing Phase 2 clinical study for the administration of MultiStem cell therapy to patients that have suffered significant damage from a heart attack (AMI), and we received grant proceeds from the NIH to help support this clinical study. Our completed Phase 1 clinical trial demonstrated consistent safety and encouraging evidence of therapeutic benefit among AMI patients with severely compromised heart function, and one-year followup data suggested that the benefit observed was sustained over time. We are currently enrolling patients in our Phase 2 study at leading cardiovascular clinical centers in the U.S. Transplantation Support & Other Programs We believe our technology has broad potential application in many areas, including transplantation support, complications from trauma and certain orthopedic applications. We previously completed a clinical study evaluating administration of MultiStem cell therapy to patients at high risk of GvHD (Graft-versus-Host Disease). Data from the study demonstrated safety and also suggested that treatment may reduce the occurrence or severity of GvHD and provide other benefits. We have received orphan drug status from the FDA and EMA, as well as Fast Track status for this program. Acute Respiratory Distress Syndrome (ARDS) MultiStem has also shown promise in treating certain inflammatory and immune conditions, include acute pulmonary inflammation. We are conducting a proofof-concept trial to treat patients suffering from ARDS, a serious immunological and inflammatory condition characterized by widespread inflammation in the lungs and compromised pulmonary function. ARDS can be triggered by pneumonia, sepsis, or trauma and represents a major cause of morbidity and mortality in the critical care setting and an area of substantial unmet medical need. The trial is currently enrolling patients in the U.K. and the U.S. As part of the LOI we entered in with Healios in March 2018, Healios intends to expand its license to develop MultiStem in Japan for the ARDS indication, as well as other indications, such a trauma and ophthalmological conditions. As part of the expansion, Healios would also have an option to obtain a license to develop MultiStem products for ARDS, ischemic stroke and trauma in China.

4 RECENT HIGHLIGHTS Significantly expanded our existing Healios collaboration, including a $21.1 million equity investment and $10.0 million in guaranteed license fees, and, if the expansion is consummated, an additional $25.0 million in license fee payments over time. As part of the expansion, Healios would receive a license to MultiStem products for ARDS, trauma and Healios organ bud technology in Japan, and certain ophthalmological indications globally. Also, Healios would receive an exclusive option to license MultiStem products for ischemic stroke, ARDS and trauma in China, and Athersys would be entitled to license fees, milestone payments and escalating royalties for the licensed indications; Advanced our preparations for the MASTERS-2 Phase 3 registration study for ischemic stroke, to enable initiation of this important study expected in the second quarter of 2018; Entered into a new equity facility in February 2018 as a follow-on to our current facility, with right to sell up to $100 million of common stock to Aspire Capital, LLC over three-year period, providing access to capital as needed to support our operations; Received multiple special designations from regulators for our stroke program this year, including RMAT designation and Fast Track designation from the FDA, as well as a Final Scientific Advice positive opinion from EMA; Expanded manufacturing and process development collaborative relationships, including Nikon CeLL innovation Co., Ltd., and progressed key manufacturing campaigns and process development projects; and Progressed Phase 1/2 study evaluating administration of MultiStem therapy to ARDS patients, and our Phase 2 acute myocardial infarction study. DEVELOPMENT STATUS OF REGENERATIVE MEDICINE PROGRAMS

5 CORPORATE FACT SHEET WINTER 2018 (in thousands, except share and per share data) SELECTED FINANCIAL DATA Year Ended December 31, Consolidated Statement of Operations Data: Revenues: Contract Revenue $ 2,843 $ 16,238 $ 10,298 $ 286 Grant Revenue 865 1,109 1,650 1,337 Total Revenues 3,708 17,347 11,948 1,623 Costs and Expenses: Research and development 27,841 24,838 21,316 23,366 General and administrative 8,466 7,835 7,536 6,909 Depreciation Total costs and expenses 36,991 33,055 29,119 30,635 Gain from insurance proceeds, net Loss from operations (33,283) (15,026) (17,171) (29,012) Other (expense) income: Other income (expense), net and tax benefit (23) 339 Income (expense) from change in fair value of warrants 728 (557) 772 6,591 Net loss $ (32,241) $ (15,337) $ (16,422) $ (22,082) Basic loss per share $ (0.29) (0.18) (0.20) (0.29) Weighted average shares - outstanding, basic 112,053,369 84,715,471 82,143,610 76,954,503 KEY RATIOS (Per Yahoo Finance as of March 12, 2018 ) Price & Volume Balance Sheet Recent Price $ 2.13 Cash ($mil) (MRQ) Week High $ 2.63 Current Ratio (MRQ) Week Low $ 1.02 Debt ($mil) (MRQ) N/A Average Vol (Mil) (3 mos) 0.98 Share Related Items Valuation Ratios Market Cap ($Mil) Price/Sales (TTM) Shares Out (Mil) Price/Book (MRQ) Actual shares outstanding as of Sept. 30th was million 2 Actual cash as of Sept. 30th was $28.32 million Gil Van Bokkelen, Ph.D. Chairman and CEO John Harrington, Ph.D. Executive Vice President & Chief Scientific Officer Manal Morsy, M.D., Ph.D., MBA Senior Vice President, Global Regulatory Affairs Anthony Ting, Ph.D. Vice President of Regenerative Medicine, Head of Cardiopulmonary Programs Raymond Starling, Ph.D. Vice President, Clinical Development & Operations MANAGEMENT William (B.J.) Lehmann, Jr., J.D. President & Chief Operating Officer Laura Campbell, C.P.A. Senior Vice President of Finance Robert (Willie) Mays, Ph.D. Vice President of Regenerative Medicine, Head of Neuroscience Programs Rakesh Ramachandran, M.S. Vice President, Information Technology & Communications ANALYST COVERAGE Katherine Xu - William Blair Jason Kolbert - Maxim Group Chad Messer - Needham & Co, LLC Stephen Brozak - WBB Securities CORPORATE HEADQUARTERS Athersys, Inc Carnegie Avenue Cleveland, OH Telephone: Fax: Corporate Partnerships: corporate@athersys.com Scientific Collaboration: scientific@athersys.com Human Resources: jobs@athersys.com Investor Relations: ir@athersys.com MEDIA RELATIONS Russo Partners, LLC David Schull, (212) or (858) David.schull@russopartnersllc.com CORPORATE COMMUNICATIONS & INVESTOR RELATIONS Karen Hunady, (216) khunady@athersys.com The information contained herein was obtained from the management of Athersys, Inc. and other sources Athersys believes to be reliable. The matters discussed in this document include forward-looking statements, the accuracy of which is subject to risks and uncertainties. Undue reliance should not be placed on these forward-looking statements and the Company undertakes no obligation to update forward-looking statements. Please see Athersys s most recent Annual Report, Form 10-K, Form 10-Q, Form 8-K and other SEC filings for additional information about the Company and related risks. MultiStem is a registered trademark of Athersys, Inc. Athersys.com