IND Amendment for Nurown Submitted

This important milestone sets the stage for the initiation of BrainStorm's highly anticipated Phase IIIb clinical trial, which has been designed in collaboration with the FDA under a Special Protocol Assessment (SPA). The SPA agreement confirms that the Phase IIIb trial design and statistical analysis plan is in line with the FDA's expectations, validating its potential to support a future Biologics License Application (BLA). The company now anticipates a swift regulatory review of the IND, clearing the way for the trial's launch.

"The submission of this IND amendment, together with the FDA's agreement on our trial design under the SPA, represents a major step toward bringing NurOwn to ALS patients," said Chaim Lebovits, CEO of BrainStorm Cell Therapeutics. "We are optimistic about a rapid review and are eager to initiate our Phase IIIb study, which we are undertaking to support regulatory approval."

The Phase IIIb trial is expected to enroll approximately 200 ALS participants and will be conducted in two parts. In Part A, patients will receive three doses of either NurOwn or placebo over a 24-week double-blind period. Following this, all participants - including those initially in the placebo group - will have the opportunity to transition to an open-label extension (Part B), receiving three additional doses of NurOwn over another 24 weeks. The trial's primary efficacy endpoint will assess changes in ALSFRS-R scores from baseline to week 24, comparing disease progression between the NurOwn and placebo groups. The successful completion of Part A will position BrainStorm for a BLA submission, accelerating NurOwn's path to potential approval.

ALS remains a devastating disease with limited therapeutic options, and BrainStorm is committed to working closely with regulatory agencies, clinical investigators, and the ALS community to ensure the rapid and rigorous evaluation of NurOwn. The company will provide updates as the FDA review progresses.