BrainStorm Receives FDA Clearance to Initiate Phase 3b Trial of NurOwn® for ALS

Core Insights - The U.S. FDA has cleared BrainStorm Cell Therapeutics Inc. to initiate a Phase 3b clinical trial of NurOwn® for ALS treatment, marking a significant milestone for the company and the ALS community [1][3] Company Overview - BrainStorm Cell Therapeutics Inc. specializes in developing adult stem cell therapies for neurodegenerative diseases, with its lead investigational therapy being NurOwn® [5][6] - The NurOwn® platform utilizes autologous mesenchymal stem cells to create neurotrophic factor-secreting cells aimed at modulating neuroinflammation and promoting neuroprotection [5] Clinical Trial Details - The Phase 3b trial will enroll approximately 200 participants at leading academic medical centers, featuring a 24-week randomized, double-blind, placebo-controlled phase followed by a 24-week open-label extension [3] - The primary endpoint of the trial is the change from baseline to week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R) [3][6] - The trial design has been agreed upon with the FDA under a Special Protocol Assessment (SPA), ensuring the study's endpoints and statistical methodology are suitable for a future Biologics License Application (BLA) submission [2][6] Funding and Development Strategy - The company is actively seeking funding through various channels, including non-dilutive grants, to support the timely launch of the clinical study [3] - BrainStorm has previously completed a Phase 3 trial in ALS and is also advancing a proprietary exosome-based platform for delivering therapeutic proteins and nucleic acids [6]