Athersys, Inc. (NASDAQ:ATHX) announced today that its partner, HEALIOS K.K. (Healios), has provided updates on its two clinical programs evaluating MultiStem® (invimestrocel, HLCM051) cell therapy, for which Healios has a license and is responsible for the development and commercialization of MultiStem for ischemic stroke and acute respiratory distress syndrome (ARDS) in Japan on an exclusive basis.
Healios reported that the last patient in its Phase 2/3 TREASURE study, evaluating MultiStem for the treatment of ischemic stroke, completed the 365-day follow-up visit. Additionally, Healios updated its progress in its discussions with the Pharmaceuticals and Medical Devices Agency (PMDA) regarding its MultiStem ARDS program and results from its ONE-BRIDGE study.
The last patient follow-up visit represents an important milestone in the TREASURE study, as it completes the collection of all patient data and enables the final validation of the data, analyses, and preparation of results by its contract research organization for unblinding and topline disclosures. Healios anticipates that it will receive the final topline results and make disclosures in May 2022.
TREASURE is a randomized, double-blind, placebo-controlled study in Japan evaluating the administration of MultiStem treatment within 18-36 hours after an ischemic stroke in over 200 patients with moderate to moderate-severe strokes. The TREASURE study, like its global counterpart, Athersys’ MASTERS-2 study, was designed based on the promising results from Athersys’ MASTERS-1 study for stroke patients treated with MultiStem therapy within 36 hours of the stroke. In the MASTERS-1 study, stroke patients receiving early MultiStem treatment had better outcomes than placebo patients at 90 days following treatment, and further improved outcomes at one year. Athersys expects that the TREASURE 90-day and one-year results would be strong indicators of the potential results from Athersys’ MASTERS-2 study.
“We are happy to report that Healios has completed the last patient follow-up visit and will soon disclose topline results from this important study,” commented Dr. John Harrington, Chief Scientific Officer of Athersys. “Furthermore, we are optimistic about the results and look forward to evaluating the data and better understanding its implications for our MASTERS-2 study.”
In addition, Healios has announced feedback received from the PMDA after recent consultation meetings to obtain guidance and advice pertaining to its application for MultiStem approval for ARDS, an orphan regenerative medicine program, on the basis of its Phase 2 ONE-BRIDGE study. The ONE-BRIDGE clinical trial was designed as a 30-patient, open label study, and demonstrated promising impact from MultiStem treatment on ventilator free days (VFD) and mortality. While the PMDA did not disagree with the efficacy and safety conclusions of the ONE-BRIDGE study, the PMDA advised Healios that additional supporting data is necessary for application for approval of MultiStem treatment for the ARDS indication. As a result of the guidance from the PMDA, Healios does not currently plan to apply for approval in Q2 2022. Healios will continue discussions with PMDA and work toward early application, and while it is undetermined at this time, it is expected that the application for approval is unlikely to take place in the current fiscal year.
Athersys is currently conducting its Phase 2/3 MACOVIA study, evaluating MultiStem treatment for ARDS patients, which includes patients diagnosed with ARDS due to COVID-19. The Company recently announced FDA approval to use product manufactured with its next generation bioreactor-based platform in the MACOVIA study.
Athersys continues to believe that MultiStem treatment has potential to have a substantial, positive impact on ARDS patients based on favorable data from its own double-blind, placebo-controlled Phase 1/2 MUST-ARDS study and strong supporting data from the Healios ONE-BRIDGE study. For example, on a combined basis, MultiStem-treated subjects had, on average, 5.5 more VFD in the first 28-days following diagnosis than non-treated subjects (with a p-value = 0.07) and, on a median basis, 10.5 more VFD during the 28-day period. Further, there was also strong evidence of a favorable impact from treatment on mortality and quality of life metrics. Athersys plans to work closely with Healios to build the supporting data necessary to apply for approval in Japan, including exploring the possibility of using certain MACOVIA clinical data.
“We congratulate Healios for achieving this important milestone in the TREASURE study and look forward to the topline results in May,” said Mr. Dan Camardo, Chief Executive Officer of Athersys. “We also look forward to continuing our work with Healios to advance the ARDS program in Japan. This is a very exciting time for both companies, and we remain committed to our partnership with Healios to work toward bringing MultiStem to market in Japan and eventually to the U.S. and other regions.”
Please refer to the following Healios disclosures for more information: https://ssl4.eir-parts.net/doc/4593/tdnet/2103272/00.pdf and https://ssl4.eir-parts.net/doc/4593/tdnet/2103271/00.pdf
