BrainStorm Seeks FDA Approval for Advanced ALS Trial

BrainStorm Cell Therapeutics, a prominent company specializing in adult stem cell treatments for neurodegenerative conditions, has recently taken a significant step forward in its research endeavors. The firm has put forth a Special Protocol Assessment (SPA) request to the U.S. Food and Drug Administration (FDA) concerning a Phase 3b study for its ALS treatment, NurOwn®. This initiative comes on the heels of a face-to-face meeting with the FDA in December 2023, which provided valuable insights for the progression of the impending trial.

The SPA process is pivotal as it enables BrainStorm to align with the FDA on the clinical trial's structure and statistical methods prior to its commencement. This alignment is expected to ensure the study's adequacy in supporting future marketing efforts. The company anticipates a response from the FDA within 45 days post-submission.

Chaim Lebovits, BrainStorm's President and CEO, emphasized the importance of the SPA for the advancement of NurOwn's development. He expressed the belief that this agreement could mitigate certain regulatory risks associated with the program and showed eagerness to collaborate with the FDA to finalize the SPA.

Stacy Lindborg, Co-CEO of BrainStorm, highlighted the company's unwavering commitment to the ALS community. She stated that NurOwn, if granted approval, could significantly improve the lives of those suffering from ALS and expressed gratitude for the FDA's guidance.

NurOwn® is an innovative therapeutic platform using autologous MSC-NTF cells, which are derived from ALS patients and processed through a unique method to release neurotrophic factors aimed at neurodegenerative diseases. BrainStorm's dedication to ALS research is evident through its extensive preclinical work and clinical trials, all documented in peer-reviewed journals.

The NurOwn clinical program has been instrumental in understanding ALS's pathology, disease progression, and potential treatments. Post the initial Phase 3 results, BrainStorm has shared comprehensive data through peer-reviewed analysis, which includes a detailed examination of the Floor Effect, evaluation of biomarkers, and genetic data analysis, marking a pioneering step in pharmacogenomic studies for ALS.

BrainStorm Cell Therapeutics holds exclusive rights to the NurOwn® platform for clinical development and commercialization. The autologous MSC-NTF cells have received Orphan Drug status from both the FDA and the European Medicines Agency for ALS treatment. The company has completed a Phase 3 ALS trial and a Phase 2 trial for progressive MS, supported by grants from various institutions.

BrainStorm's work in advancing stem cell therapies for ALS underscores the company's commitment to innovation and improving patient outcomes in the face of debilitating diseases.