BrainStorm submits IND amendment to FDA for Phase 3b trial of NurOwn in ALS

BrainStorm (BCLI) submitted an Investigational New Drug, or IND, amendment to the FDA for NurOwn, the company’s autologous mesenchymal stem cell therapy for amyotrophic lateral sclerosis, or ALS. This milestone sets the stage for the initiation of BrainStorm’s Phase 3b clinical trial, designed in collaboration with the FDA under a Special Protocol Assessment. The SPA agreement confirms that the Phase 3b trial design and statistical analysis plan is in line with the FDA’s expectations, validating its potential to support a future Biologics License Application. The company now anticipates a swift regulatory review of the IND, clearing the way for the trial’s launch. The trial’s primary efficacy endpoint will assess changes in ALSFRS-R scores from baseline to week 24, comparing disease progression between the NurOwn and placebo groups. The successful completion of Part A will position BrainStorm for a BLA submission, accelerating NurOwn’s path to potential approval.