CLEVELAND, Nov. 15, 2017 (GLOBE NEWSWIRE) -- Athersys, Inc. (ATHX) announced today that its partner, HEALIOS K.K. (“Healios”), has enrolled the first patient in its study in Japan evaluating MultiStem® cell therapy treatment of patients who have suffered an ischemic stroke (the “TREASURE” study).
The TREASURE study is designed to enroll a total of 220 patients with moderate to severe strokes and will be randomized 1:1, with patients receiving a single intravenous infusion of MultiStem or placebo within 18 to 36 hours of the onset of the stroke. The primary efficacy outcome is the proportion of subjects achieving an Excellent Outcome at three months following treatment. Excellent Outcome is a commonly used measure of efficacy in stroke treatment, evaluated using three standard clinical rating scales of functional and neurological deficit and recovery following the stroke, including the NIH Stroke Scale, modified Rankin Scale and Barthel Index.
“We are pleased that the Healios TREASURE trial in Japan is now enrolling patients,” stated Dr. Gil Van Bokkelen, Chairman and Chief Executive Officer of Athersys. “With a successful trial outcome, Japan’s progressive regulatory framework for regenerative medicine products provides the potential for either conditional or full marketing approval. Additionally, the priority review designation obtained under the Sakigake framework is designed to speed the review by Japan’s Pharmaceutical and Medical Devices Agency, allowing advancement of this program in a highly efficient manner.”
“This is the largest stem cell trial initiated under the new regenerative medicine regulatory framework established in Japan, and it represents an important milestone,” added Dr. Van Bokkelen. “We believe that MultiStem has the potential to meaningfully enhance recovery following a stroke and substantially extend the treatment window from several hours under current standard of care, which is only relevant to a small percentage of stroke patients, out to 36 hours, enabling treatment of a much larger number of people,” concluded Dr. Van Bokkelen.
In parallel with the TREASURE trial, Athersys has been planning for its Phase 3 study entitled, MultiStem Administration for Stroke Treatment and Enhanced Recovery Study-2 (“MASTERS-2”), to be conducted in North America and Europe. The MASTERS-2 study has received authorization from the U.S. Food and Drug Administration (“FDA”) under a Special Protocol Assessment (“SPA”) for the design and planned analysis of this pivotal clinical trial. The SPA provides formal agreement from the FDA that the protocol design, clinical endpoints, planned conduct and statistical analyses in this Phase 3 study are acceptable to support a regulatory submission for marketing approval of MultiStem cell therapy as a product for treating ischemic stroke patients. Additionally, the MultiStem stroke program has been awarded Fast Track designation by the FDA, meaning that the program is eligible for accelerated approval, priority review and rolling submission of the biologics license application, which facilitates a timely regulatory review.
Recently, in addition to the SPA and Fast Track designations from the FDA, the MASTERS-2 trial also obtained a Final Scientific Advice positive opinion from the European Medicines Agency, and received the Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, which was established this year 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 address unmet medical needs for a serious or life threatening disease or condition. 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 MASTERS-2 clinical trial is planned to 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 to 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, life-threatening adverse events and post-stroke complications, such as infection.
