BrainStorm to Continue with Planned Phase 3b Trial and Remains Committed to Advancing Access for People Living with ALS
BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today acknowledged that the U.S. Food and Drug Administration’s (FDA) consideration of a Citizen Petition requesting a new review of the data supporting NurOwn will provide a critical opportunity to reaffirm its potential as therapy for amyotrophic lateral sclerosis (ALS). A Citizen Petition, submitted to the FDA under the Federal Food, Drug, and Cosmetic Act (21 CFR § 10.30), is a regulatory process that allows any interested party to formally request the Agency to take action on specific matters, such as reviewing data, issuing new guidance, or taking enforcement action. In this case, the Citizen Petition is requesting that the FDA review the data supporting NurOwn.
BrainStorm has consistently supported the integrity and scientific validity of its data and believes a comprehensive review of all evidence is essential, especially as the regulatory landscape for rare diseases with unmet needs continues to evolve. Although BrainStorm was not involved in drafting or submitting this petition or its contents, the company welcomes the FDA’s willingness to reevaluate existing data. It endorses any process that thoroughly assesses the scientific evidence related to potential treatment options for people living with ALS.
“We respect the FDA’s independent review process and welcome its consideration of this request,” said Chaim Lebovits, President and Chief Executive Officer of BrainStorm. “We continue to stand firmly behind the scientific integrity of BrainStorm’s data and remain committed to working collaboratively with the FDA and the ALS community to advance the development of safe and effective therapies.”
BrainStorm is committed to continuing alignment with FDA and will proceed with its Phase 3b ENDURANCE trial of NurOwn under an FDA Special Protocol Assessment (SPA), designed to generate additional robust data and confirm the product’s efficacy and safety in early stage ALS patients. In parallel, the company supports exploration of potential regulatory pathways that may allow appropriate access to NurOwn for individuals living with ALS today.
Bob Dagher, M.D., Chief Medical Officer at BrainStorm, stated, “A new review of the NurOwn data offers an important chance to ensure all available evidence is fully considered. We remain dedicated to rigorous science and to increasing access for ALS patients in need.”
Mary Kay Turner, Senior Vice President, Advocacy and Public Affairs at BrainStorm, added, “The ALS community has been a powerful voice in advocating for new approaches to treatment. We stand with advocates in supporting efforts that prioritize both data-driven decision-making and urgency for patients facing this devastating disease.”
ALS is a rapidly progressive neurodegenerative disease that leads to the loss of motor neurons, severely impairing voluntary muscle movement. Despite recent advances in care, effective disease-modifying treatment options remain limited.
Peer-reviewed analyses have demonstrated clinically meaningful benefits in individuals with less-advanced ALS:
- In the subgroup of participants with baseline ALSFRS-R scores of 35 and above, NurOwn showed statistically significant functional improvement versus placebo.
- Post-hoc analyses revealed NurOwn-treated participants retained an average of two more ALSFRS-R points than placebo recipients – representing meaningful functional preservation.
BrainStorm recently announced new survival data from 10 participants who had previously completed the Phase 3 clinical trial and subsequently entered its Expanded Access Program (EAP). Notably, participants survived more than five years from ALS symptom onset – well above published estimates suggesting that only 10% of people diagnosed with ALS reach that benchmark. The median survival in the group was 6.8 years.
These results strongly suggest that the observed survival outcomes in the EAP patients are unlikely to be due to chance alone and further underscore the need for continued scientific exploration.
BrainStorm will continue to engage transparently with the FDA, clinicians, advocacy groups, and the broader ALS community, and will provide updates as appropriate.
