Key points from article :
Athersys faced a significant setback as its stem cell therapy, MultiStem, failed to meet the primary endpoint in a Japanese trial, causing its stock to plummet by over 60%. The TREASURE trial, conducted by Athersys’ Japanese partner Healios, tested the therapy on stroke patients but did not achieve a statistically significant improvement in the primary endpoint of an "excellent outcome" at 90 days. This outcome has cast doubt on the therapy’s immediate viability, despite its advanced designations from the FDA.
MultiStem, designed as an off-the-shelf cell therapy to promote healing and reduce inflammation, was tested in a randomized, double-blind trial involving 206 patients with ischemic stroke. Participants had an average age of 78, substantially older than those in earlier studies, which Athersys argued contributed to slower recovery and less favorable results on the primary endpoint. However, secondary measures, such as the modified Rankin Score (mRS) and Barthel Index, showed some potential for the therapy’s effectiveness in improving functional recovery.
Although the trial failed to meet its primary goal, safety outcomes showed no significant differences between the treatment and placebo groups, maintaining the therapy's safety profile. Athersys remains optimistic, with CEO Dan Camardo expressing confidence in the ongoing MASTERS-2 phase 3 trial, which targets a younger and less severely affected stroke population across the U.S., Europe, and Asia-Pacific. The company believes the TREASURE trial's data may still support further development and eventual market approval in Japan.
