In a significant stride towards combating neurodegenerative diseases, BrainStorm Cell Therapeutics Inc. has announced its submission of a Special Protocol Assessment (SPA) request to the U.S. Food and Drug Administration (FDA) for a Phase 3b study of NurOwn®, its pioneering investigational treatment for amyotrophic lateral sclerosis (ALS).
This move, following a constructive in-person meeting with the FDA in December 2023, marks a pivotal step in the clinical development of NurOwn®.
The SPA program participation is aimed at reaching a consensus with the FDA on the clinical trial design, including the proposed statistical analysis, before the study’s initiation. This consensus is crucial as it ensures the study’s adequacy to support a future marketing evaluation, with a response from the FDA anticipated within 45 days of submission.
Chaim Lebovits, President and CEO of BrainStorm, highlighted the SPA’s importance in advancing the development program for NurOwn®. He expressed optimism about de-risking certain regulatory aspects of the program and the company’s eagerness to work with the FDA to complete the SPA.
NurOwn® leverages the autologous MSC-NTF cells technology platform, representing a promising therapeutic approach to targeting disease pathways in neurodegenerative disorders. These cells are harvested from individuals with ALS and are manufactured using a proprietary process to secrete neurotrophic factors, targeting specific neurodegenerative diseases.
The lead program for NurOwn® is the treatment of ALS, demonstrating BrainStorm’s long-term commitment through preclinical research and a series of clinical studies.
The NurOwn® clinical program has provided valuable insights into ALS pathology, disease progression, and treatment. It includes a comprehensive analysis of the initial Phase 3 readout, offering a promising outlook for the development of future treatments for ALS.
BrainStorm Cell Therapeutics Inc. stands as a leader in developing innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases.
With NurOwn® having received Orphan Drug designation status from both the FDA and the European Medicines Agency (EMA) for the treatment of ALS, the company’s efforts are a beacon of hope for those affected by this devastating disease.
As we await further developments, the ALS community and stakeholders in the neurodegenerative disease treatment landscape watch closely. The potential approval of NurOwn® could herald a new era in ALS treatment, offering substantial improvements in the lives of those battling this condition.
BrainStorm Cell Therapeutics‘ commitment to innovation and its proactive engagement with regulatory bodies underscore the company’s dedication to bringing groundbreaking treatments to patients in need.
The journey of NurOwn® through the clinical trial phases and its eventual market introduction will undoubtedly be a significant milestone in the fight against ALS and other neurodegenerative diseases.