A New Opportunity for ALS Treatment: NurOwn® Gains Attention from FDA Review

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

In a significant development for the ALS community, BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a forefront innovator in adult stem cell therapies targeting neurodegenerative diseases, is embracing the opportunity presented by the U.S. Food and Drug Administration’s (FDA) consideration of a Citizen Petition. This petition calls for a renewed review of the data surrounding BrainStorm's flagship treatment, NurOwn®, which is thought to have substantial potential for patients suffering from amyotrophic lateral sclerosis (ALS).

The Citizen Petition enables any individual or entity to formally request the FDA to reassess existing data, generate new guidelines, or enforce specific regulations, emphasizing a transparent governmental review process. Though BrainStorm did not play a role in writing or submitting this petition, they wholeheartedly support this re-evaluation and firmly believe it is crucial to reassess the scientific evidence related to ALS treatments, especially given the ever-evolving regulatory landscape surrounding rare diseases.

Chaim Lebovits, BrainStorm's President and CEO, indicated, "We respect the FDA's independent review process and welcome its consideration of this request. Our commitment to the integrity of BrainStorm's data remains strong, and we are eager to collaborate with the FDA and the ALS community to develop safe and effective therapies."

BrainStorm is concurrently advancing its Phase 3b ENDURANCE trial of NurOwn, which is conducted under an FDA Special Protocol Assessment (SPA) agreement. This phase is designed to yield additional robust data confirming the efficacy and safety of NurOwn® in early-stage ALS patients. Furthermore, BrainStorm is exploring potential alternative regulatory routes to ensure timely access to NurOwn for individuals currently battling ALS.

Dr. Bob Dagher, Chief Medical Officer of BrainStorm, spotlighted the significance of the FDA's renewed investigation, stating that it offers a vital platform to ensure that all existing evidence is thoroughly scrutinized. "Our relentless pursuit of rigorous science stems from our dedication to enhancing patient access to effective ALS therapies," Dagher stated.

Mary Kay Turner, the Senior Vice President of Advocacy and Public Affairs at BrainStorm, emphasized the powerful influence of the ALS community in pushing for innovative treatment options. She stated, "With their advocacy, we support the urgency of finding solutions that truly prioritize data-driven decisions equivalent to the pressing needs of patients suffering from this devastating disease."

ALS, a rapidly advancing neurodegenerative illness, is notoriously known for causing significant deterioration of motor neuron function, leading to extensive impairment of voluntary muscle control. Despite recent advancements in medical care, viable options for modifying disease progression are notably scarce.

Peer-reviewed studies have highlighted NurOwn's promising effects on patients with earlier stages of ALS. For instance, in a notable subgroup analysis, patients who initially scored high on the ALSFRS-R scale showed statistically significant functional improvement compared to those who received a placebo. Specific post-hoc analysis indicated that participants treated with NurOwn retained an average gain of two ALSFRS-R points over their placebo counterparts, corresponding to significant functional stability.

In a related breakthrough, BrainStorm unveiled new survival data derived from ten participants who initially completed the Phase 3 clinical trials and subsequently benefited from its Expanded Access Program (EAP). Remarkably, these individuals survived for over five years from the onset of ALS symptoms—far surpassing recognized statistical predictions that suggest merely 10% of ALS patients reach this survival milestone. The median survival for this particular group was reported at 6.8 years, further solidifying the prospect that these observed outcomes are more than mere coincidence and showcasing the necessity for ongoing scientific scrutiny.

Keeping health officials, clinicians, and the ALS community in the loop, BrainStorm intends to maintain open dialogues through transparent engagements, ensuring they share any pertinent updates.

About NurOwn®

The NurOwn® technology platform employs a groundbreaking approach utilizing autologous MSC-NTF cells—produced from a patient’s own bone marrow—to generate a therapeutic product rich in neurotrophic factors (NTFs) crucial for addressing nerve damage. This innovative treatment aims to deliver various NTFs while modulating immune response directly to the damage site, thereby presenting a potential route to slow or stabilize ALS advancement.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) stands out as a prominent developer of autologous adult stem cell therapies aimed at alleviating debilitating neurodegenerative disorders. Recognized for its proprietary NurOwn® platform and having garnered Orphan Drug designation from both the FDA and the EMA, BrainStorm is setting the course for future advances in ALS treatment. With engaged clinical trials and peer-reviewed research, BrainStorm continues to pave the path toward breakthroughs in stem cell therapy and neurological care, underscoring the company's commitment to bringing hope to ALS patients worldwide.