BrainStorm Cell Therapeutics (BCLI, Financial) has moved forward in the regulatory process with the U.S. Food and Drug Administration (FDA) by submitting an amendment to its Investigational New Drug (IND) application for NurOwn, its innovative therapy for amyotrophic lateral sclerosis (ALS). This development is pivotal as it paves the way for the commencement of a Phase 3b clinical trial.
The upcoming trial for NurOwn is crafted under a Special Protocol Assessment (SPA), a collaborative effort with the FDA that ensures the trial design and its statistical methods are robust enough to support a future Biologics License Application (BLA). This SPA agreement indicates the trial plan aligns with the regulatory standards expected by the FDA.
BrainStorm eagerly awaits a prompt review of the IND amendment by the FDA, which would facilitate the initiation of the clinical trial. The primary goal of the trial is to evaluate the impact of NurOwn on ALS progression by comparing changes in ALSFRS-R scores from the start of the trial to the 24-week mark, contrasting results between those receiving NurOwn and a placebo group.
Completion of the trial's initial phase is crucial, as it will be instrumental for BrainStorm's BLA submission. This step is critical in accelerating NurOwn's journey toward potential approval, offering new hope for ALS patients awaiting advanced treatment options.
