Cleveland, OHIO, October 2, 2012 – Athersys, Inc. (NASDAQ: ATHX) recently completed enrollment of the first patient cohorts of its Phase 2 study of MultiStem®, a novel adult stem cell therapy being developed by Athersys, administered to patients within approximately 1 to 2 days after they have experienced an ischemic stroke. The independent safety committee reviewed data from these patients, finding that both of the doses evaluated were safe and well tolerated, and therefore, recommended proceeding with high dose administration to patients for the remainder of the trial.
The Phase 2 study is a double blind, placebo-controlled study evaluating the safety and efficacy of MultiStem when administered to patients who have suffered a moderate to moderately severe stroke, as defined by a National Institutes of Health Stroke Scale (NIHSS) score of 8 to 20. Patients enrolled in the study receive a single intravenous dose of MultiStem therapy or placebo in the 24 to 36 hours following the stroke, which is a significant extension of the current treatment window over existing standard of care. The study is currently being conducted at multiple centers throughout the United States.
“We are very excited about the initial results from this program and believe that it could represent a major advance in clinical care for ischemic stroke patients. We believe MultiStem has the potential to significantly enhance patient recovery, as well as meaningfully extend the treatment window over the current standard of care for stroke victims, enabling many more patients to receive treatment,” said Dr. Gil Van Bokkelen, Chairman and Chief Executive Officer of Athersys. “We are pleased with the results from the initial phase of the study, including the additional evidence of a consistent safety profile for MultiStem, and we intend to move the program forward aggressively to completion.”
The first part of the Phase 2 study included two cohorts, with each cohort including a placebo group and a treatment group – a low dose of MultiStem in the first cohort and a higher dose in the second cohort. The third cohort has a placebo group and treatment group, randomized 1 to 1. The study is expected to enroll approximately 136 patients in total.
Primary safety endpoints for the trial include measuring acute infusional reactions over the first 7 days following treatment. Primary efficacy measures include determining the proportion of patients with a modified Rankin Scale of 0 to 2 (which represents patients capable of independent living) at day 90 in the MultiStem treatment group compared to subjects in the placebo treatment group. Secondary endpoints include functional outcome as determined by NIHSS score and Barthel Index. The study includes additional exploratory endpoints, such as measuring stroke infarct size and blood marker changes between the MultiStem and placebo treatment groups.
“We believe that development of a safe and effective therapy for ischemic stroke represents both a major clinical and commercial opportunity, and one that could drive value for our shareholders,” added Dr. Van Bokkelen. “We believe that this Phase 2 study will provide compelling evidence of the potential of the program.”
According to the American Heart Association, approximately 800,000 individuals suffer a stroke each year in the United States, and an estimated 2 million individuals suffer a stroke each year in the United States, Japan and major European countries, combined. Given the current lack of effective therapies, many patients who suffer a stroke suffer long term disability and require extensive physical therapy or experience significant or permanent disability, and as a result, must receive long-term institutional care or be cared for by a family member.
Approximately 85% of strokes are ischemic, meaning they are caused by a blockage of blood flow in the brain, which occurs as a result of a clot or "thrombus". Currently, there is only one FDA-approved drug therapy for the treatment of ischemic stroke, the thrombolytic tPA, which helps to dissolve the blood clot that impedes blood flow in the brain. However, tPA must be administered within three to four hours from when the stroke has occurred in order to be effective. Due to its limited window, only a small percentage of all patients who could potentially benefit from therapy with tPA actually receive treatment.
As a consequence of an aging population, recent forecasts from the American Heart Association project that the prevalence of stroke will increase by 25% in the next twenty years, and the total estimated annual cost for treating and caring for stroke survivors in the United States will skyrocket from $64 billion in 2010 to $140 billion in 2030, representing a substantial increase in costs to the healthcare system. The company believes that the market for a safe and effective therapy for stroke that could be administered within a clinically reasonable time frame could represent a $15 to 20 billion annual market opportunity.
MultiStem® cell therapy is a patented product that has shown the ability to promote tissue repair and healing in a variety of ways, such as through the production of multiple therapeutic factors produced in response to signals of inflammation and tissue damage. MultiStem has demonstrated therapeutic potential for the treatment of inflammatory and immune disorders, neurological conditions, and cardiovascular disease, as well as other areas, and represents a unique "off-the-shelf" stem cell product that can be manufactured in a scalable manner, may be stored for years in frozen form, and is administered without tissue matching or the need for immune suppression. The product is extensively characterized for safety, consistency and potency. Athersys has forged strategic partnerships with Pfizer Inc. to develop MultiStem for inflammatory bowel disease and with RTI Biologics, Inc. to develop cell therapy for use with a bone allograft product in the orthopedic market.
Athersys is a clinical stage biotechnology company engaged in the discovery and development of therapeutic product candidates designed to extend and enhance the quality of human life. The Company is developing its MultiStem® cell therapy product, a patented, adult-derived "off-the-shelf" stem cell product platform for disease indications in the cardiovascular, neurological, inflammatory and immune disease areas. The Company currently has several clinical stage programs involving MultiStem, including for treating inflammatory bowel disease, ischemic stroke, damage caused by myocardial infarction, and for the prevention of graft versus host disease. Athersys has also developed a diverse portfolio that includes other technologies and product development opportunities, and has forged strategic partnerships and collaborations with leading pharmaceutical and biotechnology companies, as well as world-renowned research institutions in the United States and Europe to further develop its platform and products. More information is available at www.athersys.com.
This press release 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. 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 that could cause actual results to differ materially from those implied by forward-looking statements are the risks and uncertainties inherent in the process of discovering, developing, and commercializing products that are safe and effective for use as human therapeutics, such as the uncertainty regarding market acceptance of our product candidates and our ability to generate revenues, including MultiStem for the treatment of inflammatory bowel disease, acute myocardial infarction, stroke and other disease indications, including lysosomal storage disorders, and the prevention of graft-versus-host disease. These risks 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. Other important factors to consider in evaluating our forward-looking statements include: our ability to raise additional capital; final results from our MultiStem clinical trials, including for ischemic stroke; the possibility of delays in, adverse results of, and excessive costs of the development process; our ability to successfully initiate and complete clinical trials; changes in external market factors; changes in our industry's overall performance; changes in our business strategy; our ability to protect our intellectual property portfolio; our possible inability to realize commercially valuable discoveries in our collaborations with pharmaceutical and other biotechnology companies; our ability to meet milestones under our collaboration agreements; our collaborators' ability to continue to fulfill their obligations under the terms of our collaboration agreements; our possible inability to execute our strategy due to changes in our industry or the economy generally; changes in productivity and reliability of suppliers; and the success of our competitors and the emergence of new competitors. You should not place undue reliance on forward-looking statements contained in this press release, and we undertake no obligation to publicly update forward-looking statements, whether as a result of new information, future events or otherwise.
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