With SPA in hand, Athersys eyes approval
- Athersys reached an agreement with the U.S. Food and Drug Administration regarding a special protocol assessment (SPA) for its Phase 3 trial of its MultiStem therapy in ischemic stroke.
- The SPA provides a foundation for regulatory approval of the cell therapy, should the trial be positive. The Cleveland-based biotech expects to start the trial in 2017.
- SPAs were a process established by the regulatory agency in 2002 and updated in 2016 that give companies more guidance on areas like trial design and endpoints. An SPA agreement does not guarantee approval, but helps a company develop a drug more in line with FDA expectations.
Under the SPA, Athersys will conduct a late-stage trial that includes 300 patients at sites in North America and Europe that have suffered moderate-to-severe ischemic stroke. Patients will receive a single intravenous dose of either MultiStem or a placebo within 18 to 36 hours after the initial stroke. Patients will be evaluated at three months for their level of disability.
"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," said Chairman and CEO Gil Van Bokkelen.
While the executive was optimistic, investors should be skeptical. The company has been trying to get the allogeneic — or off-the-shelf — stem cell therapy across the approval goal line for the better part of the last five years.
While the therapy showed encouraging signs in early-stage trials, results in Phase 2 were not as positive. While the drug couldn't achieve its primary endpoint in the study, Athersys used a post-hoc analysis to show that the therapy did at least show some signs of improvement at one year — an endpoint that is unlikely to garner a regulatory greenlight.
The drug was previously partnered with Angiotech Pharmaceuticals, but the beleaguered biotech handed back the rights in 2011, forcing Athersys to go it alone. The company also partnered the drug in Japan, but original partner Chugai pulled out. Earlier this year, Healios picked up the Japanese rights.
MultiStem is the biotech's only clinical stage product.
- Athersys Statement
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