NEW YORK, April 10, 2025 /PRNewswire/ — BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a biotechnology company advancing innovative cell therapies for neurodegenerative diseases, announced today the submission of an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration (FDA) for NurOwn®, its autologous mesenchymal stem cell therapy for amyotrophic lateral sclerosis (ALS). This submission paves the way for the initiation of the company’s pivotal Phase 3b clinical trial, designed in collaboration with the FDA under a Special Protocol Assessment (SPA).
The SPA agreement confirms FDA alignment with the trial’s design and statistical analysis plan, bolstering confidence in its potential to support a future Biologics License Application (BLA). “This IND amendment, coupled with the FDA’s SPA agreement, marks significant progress toward delivering NurOwn to ALS patients,” stated Chaim Lebovits, CEO of BrainStorm Cell Therapeutics. “We anticipate a timely review and are eager to commence the Phase 3b trial, a crucial step toward regulatory approval.”
The Phase 3b trial will enroll approximately 200 ALS participants in a two-part study. Part A will assess the efficacy of three NurOwn doses versus placebo over 24 weeks in a double-blind setting. Part B will offer all participants, including those initially receiving placebo, three additional NurOwn doses in an open-label extension over another 24 weeks. The primary endpoint is the change in ALSFRS-R scores from baseline to week 24, comparing NurOwn and placebo groups. Successful completion of Part A is intended to support a BLA submission.
ALS, a devastating disease with limited treatment options, underscores the urgency of this trial. BrainStorm remains dedicated to collaborating with regulatory agencies, clinicians, and the ALS community to expedite the rigorous evaluation of NurOwn. NurOwn utilizes MSC-NTF cells, derived from a patient’s own bone marrow mesenchymal stem cells (MSCs). These MSCs are expanded and differentiated ex vivo under patented conditions to secrete high levels of neurotrophic factors (NTFs) and immunomodulatory cytokines. This targeted delivery aims to slow or stabilize ALS progression.
https://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-submits-ind-amendment-to-fda-paving-the-way-for-phase-3b-trial-of-nurown-in-als-302425558.html
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