BrainStorm Cell Therapeutics Presented Positive Survival Data from NurOwn® Expanded Access Program at 2024 Annual NEALS Meeting

Core Insights - BrainStorm Cell Therapeutics Inc. presented promising data on NurOwn® at the 2024 NEALS Meeting, indicating potential long-term benefits for ALS patients from extended treatment with debamestrocel [1][3] Group 1: NurOwn and ALS Treatment - The Expanded Access Program (EAP) for NurOwn involved 10 ALS participants, with 9 out of 10 remaining alive at the last visit, showing a median survival time of 46.6 months for the debamestrocel group compared to 41.1 months for the matched control [2][3] - A significant reduction in neurofilament light (NfL), a biomarker for neurodegeneration, was observed in participants who received debamestrocel, while those on placebo showed a 37% worsening in NfL levels [2][3] Group 2: Upcoming Phase 3b Trial - The upcoming Phase 3b trial aims to enroll approximately 200 ALS participants, with a two-part design involving 3 doses of either debamestrocel or placebo over 24 weeks, followed by an open-label period [4][6] - Key entry criteria for the trial include ages 18 to 75, ALS diagnosis within 24 months of screening, and specific ALSFRS-R scores [4][6] Group 3: Efficacy and Regulatory Aspects - The primary efficacy endpoint will compare changes in ALSFRS-R scores from baseline to week 24 between the debamestrocel and placebo groups [4] - The company has secured a Special Protocol Assessment (SPA) agreement with the FDA, which reduces regulatory risks associated with the trial [6] Group 4: NurOwn Technology Overview - NurOwn technology utilizes autologous MSC-NTF cells, which are designed to secrete neurotrophic factors targeting neurodegenerative diseases, with a focus on ALS [7][9] - The clinical program has provided insights into ALS pathology and treatment, including pharmacogenomic analysis of clinical outcomes [8]