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BrainStorm Cell Therapeutics Pushes Ahead With NurOwn in ALS, Requests FDA Feedback on New Trial

NEW YORK – BrainStorm Cell Therapeutics on Friday announced plans to continue advancing NurOwn (debamestrocel), a controversial stem cell therapy the company has been developing for amyotrophic lateral sclerosis (ALS) but for which it withdrew a biologics license application last year.

New York-based BrainStorm said it has submitted a special protocol assessment (SPA) request with the US Food and Drug Administration, which, if granted, would allow the company to meet with the regulator to discuss the possibility of a new registrational trial for the treatment. Specifically, Brainstorm wants the agency's feedback on the design and size of a Phase IIIb trial that could support a future marketing application for NurOwn.

"Having an SPA in place is an important next step in advancing our development program for NurOwn," Chaim Lebovits, BrainStorm's president and co-CEO, said in a statement. "We believe that the SPA will potentially de-risk certain regulatory aspects of the program."

BrainStorm in October withdrew its BLA for NurOwn after discussing registrational data on the drug with the FDA and after the agency's Cellular, Tissue, and Gene Therapies Advisory Committee provided feedback on the evidence submitted by the firm in September. At the advisory committee meeting, members nearly unanimously voted that the experimental treatment doesn't appear to be effective in treating ALS based on data BrainStorm provided.

NurOwn comprises autologous multipotent stem cells derived from bone marrow, which are induced to secrete high levels of neurotrophic factors that support neuronal growth and survival.

BrainStorm initially submitted a BLA to the FDA in 2022, and the agency in November 2022 said it refused to accept the submission, determining that the data did not demonstrate substantial evidence of effectiveness for NurOwn and that manufacturing information did not ensure product quality. However, BrainStorm requested that the agency accept the BLA and provided additional retrospective analyses and biomarker analysis, leading the FDA to resume its review of the BLA in February 2023.

"We continue to believe that NurOwn, if approved, has the potential to make a substantial difference in the lives of those affected by this devastating disease," BrainStorm co-CEO Stacy Lindborg said in a statement. "We are grateful for the FDA's ongoing guidance and look forward to finalizing the SPA of this registration-enabling trial."