BrainStorm Cell Therapeutics Granted FDA In-Person Meeting to Discuss Phase 3 Trial for ALS Treatment

BrainStorm Cell Therapeutics Inc. (Nasdaq: BCLI) will meet with the FDA to discuss the regulatory path forward for NurOwn® in amyotrophic lateral sclerosis (ALS) on December 6, 2023. At the meeting, BrainStorm will discuss plans for a Special Protocol Assessment (SPA) with the FDA to agree on the overall protocol design for a confirmatory Phase 3 trial in ALS. The company’s shares surged 22% following the news.

Chaim Lebovits, President and Chief Executive Officer of BrainStorm, said, “We are pleased that the FDA has granted this expedited in-person meeting to discuss the best path forward for NurOwn for ALS. Our proposed plan is to conduct a confirmatory Phase 3b trial and it is important that we are aligned with the Agency on the expected requirements for re-submitting a Biologics License Application. We believe that reaching an agreement through a SPA on the overall protocol design and the adequacy to address the requirements for marketing approval will be a key step to position the company for success and to potentially de-risk the program.”

Highlights

The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders.

BrainStorm’s lead program for NurOwn is for the treatment of ALS.

MSC-NTF cells are harvested from each person with ALS and are manufactured using an innovative and proprietary process to secrete neurotrophic factors to target specific neurodegenerative diseases.

BrainStorm’s NurOwn clinical program has generated valuable insights into the pathology of ALS, as well as disease progression and treatment.

About BrainStorm Cell Therapeutics Inc. 

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. BrainStorm holds the rights to clinical development and commercialization of the NurOwn® technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989), and another grant from the ALS Association and I AM ALS. BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive MS and was supported by a grant from the National MS Society (NMSS).