
Core Insights - New pharmacogenomic data on the impact of the UNC13A genotype on treatment response in ALS patients treated with NurOwn® will be presented at the ISCT 2025 Annual Meeting [1][2] - The findings are significant as they may help predict treatment responses based on genetic factors, advancing personalized medicine for ALS [2][3] Company Overview - BrainStorm Cell Therapeutics Inc. is a leading developer of autologous adult stem cell therapies for neurodegenerative diseases, with its proprietary NurOwn® platform utilizing mesenchymal stem cells to produce neurotrophic factor-secreting cells [4][5] - NurOwn® has received Orphan Drug designation from both the FDA and EMA, and a Phase 3 trial in ALS has been completed, with a confirmatory Phase 3b trial set to launch under a Special Protocol Assessment agreement with the FDA [5][6] Clinical Development - The upcoming Phase 3b trial aims to build on existing clinical data and demonstrate NurOwn's potential in early ALS, with positive communication ongoing with the FDA [3][5] - The recent findings provide insights into treatment effects in patient subpopulations and represent a first-of-its-kind pharmacogenomic analysis in ALS, focusing on how genetic differences affect therapy response [3][6] Presentation Details - The oral presentation titled "Debamestrocel Effect on Clinical and Biomarker Endpoints by UNC13A Genotype in Phase 3 ALS Trial" will take place on May 8, 2025, at the ISCT meeting in New Orleans [3]