Primary Endpoint in Pivotal Acute Ischemic Stroke Trial Will Become mRS Shift Analysis at Day 365
Modifications Reflect Observations from Healios' Recently Completed TREASURE Trial in Japan and the Evolution of Stroke Standard of Care
Athersys, Inc. (NASDAQ: ATHX), a cell therapy and regenerative medicine company developing MultiStem® (invimestrocel) for critical care indications, announced planned amendments to its MASTERS-2 clinical trial protocol following a Type B meeting with the U.S. Food & Drug Administration (FDA). Held on March 21, 2023, the meeting addressed Athersys’ proposed modifications that seek to establish primary and secondary endpoints that it believes best reflect the full potential benefit of MultiStem treatment for patients with acute, moderate-to-severe ischemic stroke as well as the evolving standard of care.
Following a meeting Athersys convened in November 2022 of leading stroke experts, regulatory specialists, and statisticians to discuss potential changes, Athersys proposed four modifications to its ongoing pivotal Phase 3 MASTERS-2 clinical trial protocol, all of which were accepted by the FDA. After finalizing agreement around the statistical approach, Athersys will implement the following amendments to the MASTERS-2 protocol:
- Athersys will change the timing of the primary endpoint assessed by shift analysis in modified Rankin Scale (mRS) score to Day 365, from Day 90 previously.
- Athersys will retain shift analysis in mRS score at Day 90 as a key secondary endpoint, along with other revised secondary endpoints.
- Athersys will remove eligibility caps on concomitant reperfusion therapy (e.g., tPA, MR imaging or tPA+MR imaging) to ensure the final study population is reflective of current standard of care in the population eligible for this therapy.
- Athersys may elect to have an independent statistician conduct an interim analysis to assess potential sample size adjustment. MASTERS-2 currently plans to enroll 300 patients and enrollment, as previously communicated, is >50% complete.
“The MASTERS-2 clinical trial protocol changes agreed to by the FDA reflect what we have learned from the completed MultiStem Phase 2 MASTERS-1 trial and the TREASURE clinical trial run in Japan by our partner Healios, as well as the significant evolution of standard of care in treating acute ischemic stroke. We appreciate the FDA’s guidance, which we believe ultimately will benefit stroke patients worldwide,” stated Dan Camardo, Chief Executive Officer of Athersys. “We view the outcome of our meeting as the best-case scenario. Although changing the primary endpoint to Day 365 extends the duration of MASTERS-2, we believe our accepted modifications enable accelerated patient enrollment and provide a higher conviction for demonstrating treatment potential.”
Athersys was previously granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track designation and Special Protocol Assessment (SPA) agreement for the use of MultiStem in the treatment of ischemic stroke. These designations enable sponsors to work closely with the FDA and receive guidance on expediting advancement of designated programs.
“The proposed changes we submitted to the FDA allow us to thoroughly evaluate the mechanisms through which we hypothesize MultiStem cell treatment can provide benefit to patients suffering an acute ischemic stroke,” commented Dr. Robert W. Mays, Executive Vice President of Regenerative Medicine for Athersys. “This outcome more accurately reflects our belief that MultiStem’s treatment effect extends beyond Day 90 and is better reflected with a Day 365 assessment of functional recovery.”
Additional information regarding the MASTERS-2 clinical trial is available here.
MultiStem (invimestrocel) cell therapy is a patented regenerative medicine product in clinical development that has shown the ability to promote tissue repair and healing in a variety of ways, such as through the production of therapeutic factors in response to signals of inflammation and tissue damage. MultiStem therapy’s potential for multidimensional therapeutic impact distinguishes it from traditional biopharmaceutical therapies focused on a single mechanism of benefit. The therapy 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. Based upon its efficacy profile, its novel mechanisms of action, and a favorable and consistent tolerability demonstrated in clinical studies, we believe that MultiStem therapy could provide a meaningful benefit to patients, including those suffering from serious diseases and conditions with unmet medical need.
Athersys is a 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, initially for disease indications in the neurological, inflammatory and immune, and other critical care indications and has several ongoing clinical trials evaluating this potential regenerative medicine product. Athersys has forged strategic partnerships and a broad network of collaborations to further advance MultiStem cell therapy toward commercialization. Investors and others should note that we may post information about the Company on our website at www.athersys.com and/or on our accounts on Twitter, Facebook, LinkedIn or other social media platforms. It is possible that the postings could include information deemed to be material information. Therefore, we encourage investors, the media and others interested in the Company to review the information we post on our website at www.athersys.com and on our social media accounts. Follow Athersys on Twitter at www.twitter.com/athersys. Information that we may post about the Company on our website and/or on our accounts on Twitter, Facebook, LinkedIn or other social media platforms may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties. You should not place undue reliance on forward-looking statements contained on our website and/or on our accounts on Twitter, Facebook, LinkedIn or other social media platforms, 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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