BrainStorm Secures FDA Approval for Phase 3b ALS Trial Protocol

BrainStorm Cell Therapeutics, a prominent firm specializing in adult stem cell treatments for neurodegenerative disorders, has secured a significant endorsement from the U.S. Food and Drug Administration (FDA). This endorsement comes in the form of a Special Protocol Assessment (SPA) for the company's Phase 3b clinical trial of its flagship product, NurOwn®, intended for the treatment of amyotrophic lateral sclerosis (ALS).

The SPA serves as a validation of the clinical trial's protocol and statistical analysis, ensuring that they meet the necessary standards to support a future Biologics License Application (BLA) for ALS. Chaim Lebovits, BrainStorm's President and CEO, expressed enthusiasm about the agreement, highlighting its role in mitigating regulatory risks associated with the NurOwn program. He emphasized the company's mission to introduce a novel treatment for ALS patients and the SPA's contribution towards realizing that goal.

The Phase 3b study, anticipated to begin in 2024, involves a meticulous review of the protocol, Institutional Review Board approvals, and active engagement with the ALS community. Stacy Lindborg, Co-CEO of BrainStorm, detailed the study's design, which incorporates insights from previous research and focuses on ALS patients in earlier stages of the disease. The study is expected to enroll approximately 200 participants who will be randomly assigned to either the NurOwn or placebo group.

The trial is divided into two parts: Part-A, a double-blind, placebo-controlled phase lasting 24 weeks, and Part-B, an open-label extension phase offering an additional 24 weeks of NurOwn treatment to all participants. The trial's primary goal is to measure changes in the ALS Functional Rating Scale (ALSFRS-R) from the start to the 24th week. The study will also analyze biomarkers related to neuroinflammation, neurodegeneration, and neuroprotection, with an independent Data Monitoring Committee overseeing participant safety.

An SPA is a pivotal process where developers discuss and reach a consensus with the FDA on the design and scope of clinical trials to ensure they meet scientific and regulatory criteria for potential marketing approval. While an SPA agreement confirms the FDA's approval of critical elements of the trial design, it does not extend to every detail of the protocol.

BrainStorm Cell Therapeutics, through its NurOwn® technology, is committed to advancing autologous adult stem cell therapies for ALS and other debilitating conditions. The company's work has garnered support from various institutions, reflecting its innovative approach to addressing significant unmet medical needs.