FDA Approves BrainStorm's Phase 3b Trial of NurOwn® for ALS

BrainStorm Cell Therapeutics Inc., a prominent figure in the development of adult stem cell therapies for neurodegenerative diseases, has received authorization from the U.S. Food and Drug Administration (FDA) to commence its Phase 3b clinical trial of NurOwn®. This investigational therapy focuses on treating amyotrophic lateral sclerosis (ALS) using autologous MSC-NTF cells. The green light from the FDA marks an important milestone for both BrainStorm and the broader ALS community, enabling the company to begin recruiting patients for the trial.

The Phase 3b trial, which was planned in coordination with the FDA under a Special Protocol Assessment (SPA), confirms that the study's endpoints and statistical methods are suitable for supporting a potential Biologics License Application (BLA) in the future. This step forward allows BrainStorm to initiate the enrollment of patients, which is anticipated to be around 200 individuals, at various eminent academic medical centers. The trial itself will have a 24-week randomized, double-blind, placebo-controlled phase, followed by a 24-week open-label extension where all participants will be administered NurOwn®.

BrainStorm's President and CEO, Chaim Lebovits, expressed the significance of this development, highlighting the company's readiness to activate clinical sites in stages and prepare for the first patient's enrollment. The company is also engaged in securing funding from various sources, including non-dilutive grants, to ensure the trial's successful and timely execution.

The primary endpoint of this trial is the change observed from the baseline to week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R). For further information regarding the specifics of the trial, interested parties can refer to ClinicalTrials.gov with the identifier NCT06973629.

To discuss these developments, BrainStorm's management will hold a corporate update conference call and webcast scheduled for today at 8:30 a.m. ET. The company plans to continue providing updates as the trial makes progress and achieves key milestones.

BrainStorm Cell Therapeutics Inc. is renowned for its pioneering work in autologous adult stem cell therapies targeting severe neurodegenerative disorders. Their NurOwn® platform, which leverages autologous mesenchymal stem cells (MSCs), generates neurotrophic factor-secreting cells (MSC-NTF) designed to send specific biological signals that manage neuroinflammation and enhance neuroprotection.

NurOwn® is the leading investigational therapy of BrainStorm for ALS. It holds the Orphan Drug designation from both the U.S. FDA and the European Medicines Agency (EMA). Having completed a Phase 3 trial in ALS, the upcoming Phase 3b trial is set to launch under the SPA agreement with the FDA. The NurOwn clinical program has offered significant insights into ALS disease biology, including pharmacogenomic responses related to the UNC13A genotype, biomarker data from multiple time points, and an in-depth analysis of the "Floor Effect," a notable challenge in assessing clinical outcomes in advanced ALS. BrainStorm has published its research and findings extensively in peer-reviewed journals.

Beyond ALS, BrainStorm has completed a Phase 2 open-label trial for progressive multiple sclerosis (MS) with support from the National MS Society. The company is also advancing its proprietary, allogeneic exosome-based platform for delivering therapeutic proteins and nucleic acids. Recently, BrainStorm secured a Notice of Allowance from the U.S. Patent and Trademark Office for its foundational exosome technology patent, strengthening its intellectual property portfolio in the promising field of regenerative medicine.