Athersys (ATHX) Receives FDA Okay Under a SPA for Phase 3 Study of MultiStem

September 28, 2016 6:31 AM EDT

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Athersys, Inc. (NASDAQ: ATHX) announced today that it has received agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the design and planned analysis of a Phase 3 clinical trial of Athersys’ novel MultiStem® cell therapy product for the treatment of ischemic stroke. 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 are acceptable to support a regulatory submission for approval of the MultiStem product for treating ischemic stroke patients. The results from the Phase 3 trial entitled, “MultiStem Administration for Stroke Treatment and Enhanced Recovery Study-2” (MASTERS-2), together with other available clinical data, would provide the foundation of the regulatory package to be submitted for marketing approval.

“This is a major accomplishment for Athersys, as it clearly defines the development and regulatory pathway for the approval of MultiStem cell therapy for the treatment of ischemic stroke,” stated Dr. Gil Van Bokkelen, Chairman and Chief Executive Officer of Athersys. “We would like to thank the FDA for its engagement and guidance in this process, and the clinical investigators who have been critical to our development of this potential treatment for stroke.

“The SPA is important in clarifying and de-risking an accelerated development pathway for us because it means that the successful completion of the MASTERS-2 trial, together with other available clinical data, could enable us to apply for marketing approval in the United States,” continued Dr. Van Bokkelen. “With this goal now achieved, we will continue the process of engagement with the FDA, European and Canadian regulators, as well as the many sites that have expressed an interest in participating in the study, to complete other necessary activities prior to trial initiation. We intend to be prepared to launch the trial in 2017 and will update our stockholders as we move forward with these plans.”

The MASTERS-2 clinical trial will be a randomized, double-blind, placebo-controlled clinical trial designed to enroll 300 patients in North America and Europe who have suffered moderate to moderate-severe ischemic stroke. The enrolled subjects will receive 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 (mRS) scores at three months, comparing the distribution, or the “shift,” between the MultiStem treatment and placebo groups. The mRS shift analysis considers disability across the full spectrum, enabling recognition of large and small improvements in disability and differences in mortality and other serious outcomes, among strokes of different severities. The study will also assess Excellent Outcome (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. Recently, Athersys' partner, HEALIOS KK, successfully completed a review from Japan’s Pharmaceutical and Medical Devices Agency of its Clinical Trial Notification, allowing it to commence a clinical trial evaluating the safety and efficacy of the administration of Athersys’ MultiStem cell therapy for the treatment of ischemic stroke in Japan.

Dr. David Hess, a lead clinical investigator in the planned MASTERS-2 trial, stroke specialist and Professor & Chairman of the Department of Neurology at the Medical College of Georgia at Augusta University, commented, “We were very encouraged by the results from the Phase 2 study, and we believe that MultiStem therapy has the potential to help many stroke patients who do not have access to or did not benefit from other therapies, such as tPA or mechanical thrombectomy. We are excited to be a lead site in the MASTERS-2 trial and look forward to getting started. If the trial is successful, then MultiStem could represent a major advancement in stroke clinical care.”

About Special Protocol Assessments

The Special Protocol Assessment (SPA) process is a procedure by which the FDA provides official evaluation and written guidance on the design and planned analysis of proposed study that are intended to form the basis of efficacy claims for a new drug application or biologic license application. Final marketing approval depends on the results of the efficacy, the adverse event profile and an evaluation of the benefit/risk of treatment demonstrated in the Phase 3 clinical program. The SPA agreement may only be modified through a written agreement between the sponsor and the FDA, or if the FDA becomes aware of a substantial scientific issue essential to product efficacy or safety. For more information on the SPA process, please visit:

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