BrainStorm Cell Therapeutics Cleared by FDA to Launch Phase 3b Trial of NurOwn® for ALS
A pioneer in adult stem cell therapies for neurodegenerative diseases BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), has received clearance from the U.S. Food and Drug Administration (FDA) to begin a Phase 3b clinical trial of its investigational therapy NurOwn® for the treatment of amyotrophic lateral sclerosis (ALS).
The green light follows a Special Protocol Assessment (SPA) agreement with the FDA, which confirms that the trial’s design, including its endpoints and statistical analysis, meets the agency’s requirements to support a future Biologics License Application (BLA). With the clearance secured, BrainStorm is now preparing to initiate patient enrollment across clinical sites.
“This FDA clearance is a defining milestone for BrainStorm and the ALS community,” said Chaim Lebovits, President and Chief Executive Officer of BrainStorm. “We are now positioned to swiftly activate clinical sites in a phased manner, diligently preparing to enroll the first patient in the Phase 3b trial. At the same time, we are actively working to secure funding through multiple avenues, including non-dilutive grants, to ensure the timely and successful launch of this critical study.”
The Phase 3b trial aims to enroll around 200 patients at top academic medical centers. It will begin with a 24-week randomized, double-blind, placebo-controlled phase, followed by a 24-week open-label extension in which all participants will receive NurOwn®. The study’s primary endpoint is the change from baseline to week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R).
BrainStorm’s management discussed the trial and provided additional corporate updates during a conference call and webcast this morning. During this call, BrainStorm shared key updates following FDA clearance to initiate its pivotal Phase 3b trial, dubbed ENDURANCE, for its NurOwn® technology platform targeting ALS. The trial, developed under a Special Protocol Assessment (SPA) agreement with the FDA, reflects design improvements based on insights from the previous Phase 3 study, including the elimination of the run-in period and a shortened screening timeline. BrainStorm is making strategic progress in trial readiness, negotiating clinical trial agreements with approximately 15 leading ALS centers across the U.S. and securing a top U.S. clinical site that has already passed FDA inspection. Additionally, the company plans to transfer manufacturing technology to Pluri to expand clean room capacity. At the recent ALS Drug Development Summit, BrainStorm highlighted NurOwn’s promising multi-modal mechanism of action—demonstrating immunomodulatory, neuroprotective, and neurogenic properties in preclinical models. To support the trial’s launch and completion, the company is actively exploring a mix of non-dilutive funding opportunities and strategic partnerships.
