- BrainStorm Cell Therapeutics (BCLI, Financial) to present at 2025 ALS Drug Development Summit.
- The company will share new biomarker insights from their investigational cell therapy, NurOwn.
- BrainStorm is set to launch a Phase 3b trial under a Special Protocol Assessment (SPA) with the FDA.
BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in developing innovative cellular therapies for neurodegenerative diseases, has announced its participation in the upcoming 4th Annual ALS Drug Development Summit, set to take place from May 12-14, 2025, in Boston, MA. The company aims to share crucial insights into its investigational cell therapy, NurOwn®, which holds promise for patients with amyotrophic lateral sclerosis (ALS).
Netta Blondheim-Shraga, PhD, Senior Vice President of Research & Development at BrainStorm, will present novel cerebrospinal fluid (CSF) biomarker findings. These findings suggest a potential multimodal mechanism of action for NurOwn, providing a deeper understanding of its influence on ALS disease pathways and the varied clinical outcomes in ALS patients.
Dr. Bob Dagher, Chief Medical Officer at BrainStorm, will lead a workshop on adaptive and decentralized clinical trial designs. These models are intended to enhance trial enrollment, expand geographic reach, and ease participation burdens for individuals with ALS.
Furthermore, Mary Kay Turner, Senior Vice President of Global Patient Advocacy & Public Affairs, will facilitate a panel discussion on “ALS Clinical Trials and Access to Investigational Therapies.” This panel will explore how patient and caregiver perspectives can shape trial protocols and improve accessibility and trust in clinical research.
The summit marks a pivotal moment for BrainStorm as it gears up to initiate a Phase 3b trial of NurOwn under an SPA agreement with the U.S. Food and Drug Administration (FDA). This agreement acknowledges that the trial's design and statistical evaluation are sufficient to support a potential future marketing application if successful.
BrainStorm's NurOwn has received Orphan Drug status from both the FDA and the European Medicines Agency (EMA), underscoring its potential as a breakthrough therapy for ALS. In addition to ALS, BrainStorm is also exploring its MSC-NTF cells in progressive multiple sclerosis (MS) and developing an exosome-based platform for delivering therapeutic proteins and nucleic acids.
