Athersys announces ‘disappointing’ results from MASTERS-2 interim analysis


Athersys has announced that the independent data safety monitoring board (DSMB) has completed a preplanned interim analysis of the company’s ongoing, phase three MASTERS-2 pivotal clinical trial evaluating MultiStem (invimestrocel) for the treatment of moderate-to-severe acute ischaemic stroke. The DSMB concluded that the current sample size of 300 patients is insufficiently powered to achieve the primary endpoint analysis of modified Rankin scale (mRS) shift at day 365.

There were no safety issues identified, according to Athersys, and—because the sample size required to achieve statistical significance is considerably larger—the company is intending to conduct additional data analysis with independent statisticians. A press release states that Athersys plans to pause enrolment of new patients while this analysis is being conducted.

Separately, Athersys has announced that it has entered into a memorandum of understanding (MOU) granting Healios global rights to develop and commercialise MultiStem for the treatment of acute respiratory distress syndrome (ARDS). Under the terms of the MOU, Athersys will receive between US$1.5 million and US$4.5 million in near-term payments, with up to US$150 million in potential development and sales milestones, and additional royalties.

Athersys also expects to receive revenue from the sale of existing clinical doses of MultiStem for Healios to use in its phase three clinical trial in ARDS. These doses were manufactured in accordance with the company’s 3D bioreactor process that, earlier this year, received approval from Japan’s Pharmaceuticals and Medical Devices Agency (PMDA).

Athersys says it intends to “continue exploring available strategic options”. However, the release details, in the event Athersys is unable to enter into a strategic transaction or obtain adequate financing in the near-term, it expects to have to file for protection under the bankruptcy laws to allow the company to “conduct an orderly wind down of operations”. In the interim, Athersys claims it is streamlining its operations to preserve its capital and cash resources.

“I would like to thank the many patients, clinicians and vendors that have supported this pivotal phase three trial since its start in 2018,” said Athersys CEO Dan Camardo. “We are disappointed with the results of the unblinded interim analysis indicating a large sample size adjustment would be required to achieve our primary endpoint. We intend to conduct further analysis to better understand these results. The new MOU we have signed with Healios for ARDS provides the company near-term capital and the potential for meaningful milestone payments as we continue to pursue various strategic solutions.”


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