BrainStorm Cell Therapeutics Submits IND Amendment to FDA, Paving the Way for Phase 3b Trial of NurOwn® in ALS

Core Viewpoint - BrainStorm Cell Therapeutics Inc. has submitted an Investigational New Drug (IND) amendment for NurOwn®, an autologous mesenchymal stem cell therapy for amyotrophic lateral sclerosis (ALS), paving the way for a Phase 3b clinical trial in collaboration with the FDA under a Special Protocol Assessment (SPA) [1][2] Group 1: IND Submission and Trial Design - The IND amendment submission and FDA's agreement on the trial design under the SPA represent a significant advancement towards making NurOwn available to ALS patients [2] - The Phase 3b trial will enroll approximately 200 ALS participants and will consist of two parts: a 24-week double-blind period followed by an open-label extension [2] - The primary efficacy endpoint will evaluate changes in ALSFRS-R scores from baseline to week 24, comparing the disease progression between NurOwn and placebo groups [2] Group 2: NurOwn Technology and Company Background - NurOwn® technology involves autologous MSC-NTF cells derived from bone marrow, designed to deliver neurotrophic factors and immunomodulatory cytokines to slow or stabilize disease progression in neurodegenerative disorders [4] - BrainStorm Cell Therapeutics is a leading developer of autologous adult stem cell therapeutics for neurodegenerative diseases and holds exclusive rights to the NurOwn® technology platform [5] - The company has received Orphan Drug designation from the FDA and EMA for ALS treatment and has completed a Phase 3 pivotal trial investigating the safety and efficacy of autologous MSC-NTF cells [5]