- BrainStorm Cell Therapeutics (BCLI, Financial) has submitted an IND amendment to the FDA for its ALS therapy, NurOwn®.
- The upcoming Phase 3b trial, designed under an FDA Special Protocol Assessment (SPA), will enroll about 200 ALS participants.
- NurOwn® has received Orphan Drug designation from both the FDA and EMA for ALS treatment.
BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a biotechnology company focused on developing innovative cell therapies for neurodegenerative diseases, has announced a significant regulatory development. The company has submitted an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration (FDA) for NurOwn®, its autologous mesenchymal stem cell therapy tailored for amyotrophic lateral sclerosis (ALS). This submission marks a crucial step towards launching a Phase 3b clinical trial, which is designed in consultation with the FDA under a Special Protocol Assessment (SPA).
The FDA's agreement to the SPA signifies pre-approval of the trial design, bolstering BrainStorm's future Biologics License Application (BLA) prospects if the trial results are favorable. This Phase 3b trial is structured to include approximately 200 ALS participants in two parts: Part A consists of a 24-week double-blind phase with three doses of NurOwn or placebo, followed by Part B, an open-label extension where all participants receive three additional doses of NurOwn over another 24 weeks. The primary endpoint is the change in ALSFRS-R scores, a critical metric for assessing ALS progression, from baseline to week 24.
With NurOwn®'s technology harnessing autologous MSC-NTF cells from bone marrow stem cells to secrete high levels of neurotrophic factors, BrainStorm aims to address crucial neurodegenerative pathways. The therapy holds Orphan Drug designations from both the FDA and the European Medicines Agency (EMA), further underscoring its potential to significantly impact ALS treatment. As BrainStorm progresses towards trial commencement, updates will be provided regarding FDA reviews and trial developments, reinforcing their commitment to ALS patients and advancing therapeutic solutions.
