FDA Review of Citizen Petition Offers a Fresh Look at NurOwn®'s Evidence of Treatment Effectiveness

Rhea-AI Summary

BrainStorm Cell Therapeutics (NASDAQ: BCLI) announced that the FDA will review a Citizen Petition requesting a new evaluation of data supporting NurOwn®, their ALS treatment therapy. The company, while not involved in the petition's submission, welcomes this development as an opportunity to reaffirm NurOwn's potential effectiveness.

Key clinical findings include: statistically significant functional improvement in patients with baseline ALSFRS-R scores ≥35, preservation of two more ALSFRS-R points compared to placebo, and remarkable survival data showing median survival of 6.8 years in Expanded Access Program participants.

BrainStorm will proceed with its planned Phase 3b ENDURANCE trial under FDA Special Protocol Assessment while exploring potential regulatory pathways for NurOwn access.

Positive

• Significant survival benefit demonstrated with median survival of 6.8 years in EAP participants
• Statistically significant functional improvements shown in patients with ALSFRS-R scores ≥35
• NurOwn-treated participants retained two more ALSFRS-R points versus placebo
• FDA Special Protocol Assessment (SPA) agreement secured for Phase 3b trial

Negative

• Previous FDA concerns about NurOwn's effectiveness necessitated new data review
• Additional Phase 3b trial required before potential approval
• Current limited access to treatment for ALS patients

Insights

Biotech Regulatory Analyst

FDA reviewing NurOwn data via Citizen Petition offers potential regulatory pathway advancement for BrainStorm's ALS therapy amid continuing Phase 3b trial.

The FDA's consideration of a Citizen Petition requesting review of NurOwn's data represents a critical regulatory development for BrainStorm. This regulatory mechanism, while not initiated by BrainStorm, creates a formal pathway for the FDA to reassess previously submitted evidence on NurOwn's efficacy in treating ALS.

The timing is significant as BrainStorm continues with its planned Phase 3b ENDURANCE trial under an FDA Special Protocol Assessment (SPA). This dual-track approach - proceeding with additional confirmatory studies while potentially reopening regulatory consideration of existing data - reflects a sophisticated regulatory strategy.

The press release strategically highlights post-hoc analyses suggesting statistically significant functional improvements in less-advanced ALS patients (ALSFRS-R scores ≥35) and retention of approximately two more ALSFRS-R points versus placebo - a meaningful functional preservation metric in ALS clinical research. Additionally, BrainStorm references survival data showing participants living more than five years from symptom onset (median 6.8 years), exceeding the typical 10% survival benchmark.

From a regulatory perspective, this petition creates potential optionality for BrainStorm. Should the FDA review favor NurOwn's existing data, it could potentially modify requirements for approval or create alternative pathways for certain patient populations. Meanwhile, the ongoing Phase 3b trial provides risk mitigation by generating additional confirming data.

The regulatory landscape for ALS therapies has evolved significantly, with the FDA showing increased flexibility for treatments addressing this high-unmet-need condition. This petition process leverages that evolving framework while BrainStorm maintains traditional development pathways in parallel.

BrainStorm to Continue with Planned Phase 3b Trial and Remains Committed to Advancing Access for People Living with ALS

NEW YORK, July 8, 2025 /PRNewswire/ -- 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.

About NurOwn^®

The NurOwn^® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About BrainStorm Cell Therapeutics Inc.
BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn^® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection.          

NurOwn^® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a second Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit www.brainstorm-cell.com.

Notice Regarding Forward-Looking Statements
This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements.

CONTACTS
 Investors:
 Michael Wood
 Phone: +1 646-597-6983     
 mwood@lifesciadvisors.com 

Media:
 Uri Yablonka, Chief Business Officer
 Phone: +1 917-284-2911     
 uri@brainstorm-cell.com

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SOURCE BrainStorm Cell Therapeutics Inc.

FAQ

What is the status of BrainStorm's NurOwn treatment for ALS in 2025?

NurOwn is undergoing FDA review through a Citizen Petition while BrainStorm proceeds with a Phase 3b ENDURANCE trial under Special Protocol Assessment. The treatment has shown significant survival benefits with median survival of 6.8 years in EAP participants.

What clinical benefits has NurOwn (BCLI) demonstrated in ALS patients?

NurOwn has shown statistically significant functional improvements in patients with ALSFRS-R scores ≥35, and treated participants retained two more ALSFRS-R points compared to placebo. EAP participants achieved a median survival of 6.8 years.

How does the FDA Citizen Petition affect BrainStorm's NurOwn development?

The Citizen Petition provides an opportunity for FDA to conduct a fresh review of NurOwn's effectiveness data, while BrainStorm continues its planned Phase 3b ENDURANCE trial under FDA Special Protocol Assessment.

What is the survival rate for NurOwn-treated ALS patients?

In the Expanded Access Program, participants achieved a median survival of 6.8 years from symptom onset, significantly exceeding the typical benchmark where only 10% of ALS patients survive beyond 5 years.

What are the next steps for BrainStorm's NurOwn ALS treatment?

BrainStorm is proceeding with its Phase 3b ENDURANCE trial under FDA Special Protocol Assessment while exploring regulatory pathways for treatment access and awaiting FDA's review of the Citizen Petition.