Athersys has announced that the first patient has been enrolled its Phase 3 study entitled, MultiStem Administration for Stroke Treatment and Enhanced Recovery Study-2 (MASTERS-2) to evaluate MultiStem cell therapy treatment of patients who have suffered an ischaemic stroke.
The MASTERS-2 clinical trial is a randomised, double-blind, placebo-controlled clinical trial designed to enrol 300 patients in North America and Europe who have suffered moderate to moderate-severe ischaemic stroke. The enrolled subjects, the press release says, 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.
“We are pleased to have initiated the MASTERS-2 trial, and to have reached this important milestone regarding enrolment of the first patient into the trial,” commented Robert William Mays, vice president of Regenerative Medicine and Head of Neuroscience programs at Athersys. “We believe that MultiStem cell therapy has the potential to improve standard of care for patients that have suffered a debilitating stroke and can also meaningfully extend the treatment window, providing a potential treatment option for many more patients. The commencement of this trial is an important goal that we have been working toward while we continue to support the ongoing TREASURE trial in Japan that is being conducted by our partner, Healios.”
The MASTERS-2 study has received authorisation from the US 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 ischaemic 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.
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 under the 21st Century Cures Legislation passed by Congress in December 2016. 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 commercialisation to support priority review and accelerated approval.