BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) has submitted an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration (FDA) for its NurOwn therapy, positioning the company to launch a critical Phase 3b clinical trial for amyotrophic lateral sclerosis (ALS) patients.
The amendment represents a significant milestone in the development pathway for NurOwn, an autologous mesenchymal stem cell therapy designed to deliver neurotrophic factors directly to damaged areas in patients with neurodegenerative diseases. This submission follows close collaboration with the FDA under a Special Protocol Assessment (SPA), which validates that the trial design meets regulatory expectations for a potential future Biologics License Application (BLA).
"The submission of this IND amendment, together with the FDA's agreement on our trial design under the SPA, represents a major step toward bringing NurOwn to ALS patients," said Chaim Lebovits, CEO of BrainStorm Cell Therapeutics. "We are optimistic about a rapid review and are eager to initiate our Phase 3b study, which we are undertaking to support regulatory approval."
Trial Design and Endpoints
The Phase 3b trial will employ a rigorous two-part structure involving approximately 200 ALS participants. During Part A, patients will receive three doses of either NurOwn or placebo over a 24-week double-blind period. The trial's primary efficacy endpoint will assess changes in ALS Functional Rating Scale-Revised (ALSFRS-R) scores from baseline to week 24, comparing disease progression between treatment and placebo groups.
Following the double-blind phase, all participants—including those initially receiving placebo—will have the opportunity to transition to an open-label extension (Part B). During this second 24-week period, participants will receive three additional doses of NurOwn, providing valuable long-term safety and efficacy data.
The successful completion of Part A is expected to position BrainStorm for a BLA submission, potentially accelerating NurOwn's path to regulatory approval.
The NurOwn Technology Platform
NurOwn represents an innovative approach to treating neurodegenerative diseases by leveraging the patient's own biological resources. The technology involves harvesting mesenchymal stem cells (MSCs) from a patient's bone marrow, which are then expanded and differentiated under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs).
These autologous MSC-NTF cells are designed to deliver multiple NTFs and immunomodulatory cytokines directly to sites of neurological damage, potentially slowing or stabilizing disease progression. The therapy has received Orphan Drug designation from both the FDA and the European Medicines Agency (EMA) for ALS treatment.
Addressing an Urgent Unmet Need
ALS remains a devastating neurodegenerative disease characterized by progressive loss of motor neurons, leading to muscle weakness, paralysis, and ultimately respiratory failure. With limited therapeutic options currently available, the disease typically results in death within 2-5 years of diagnosis for most patients.
The current treatment landscape for ALS includes only a handful of FDA-approved medications, none of which significantly alter the disease course. Riluzole, the first approved ALS drug, typically extends survival by only 2-3 months, while newer therapies like edaravone and the recently approved AMX0035 (Relyvrio) offer modest benefits.
If successful, NurOwn could represent a meaningful advance in ALS treatment by potentially addressing the underlying neurodegenerative processes rather than merely managing symptoms.
Regulatory Pathway and Next Steps
The SPA agreement with the FDA provides BrainStorm with increased certainty regarding the acceptability of the trial design for regulatory purposes. This agreement indicates that if the study meets its predefined endpoints, the results could support a BLA submission.
BrainStorm anticipates a swift regulatory review of the IND amendment, which would clear the way for trial initiation. The company has completed previous clinical investigations of NurOwn, including a Phase 3 pivotal trial in ALS (NCT03280056) and a Phase 2 open-label multicenter trial in progressive multiple sclerosis (NCT03799718).
"We are committed to working closely with regulatory agencies, clinical investigators, and the ALS community to ensure the rapid and rigorous evaluation of NurOwn," said Lebovits. The company has indicated it will provide updates as the FDA review progresses.
For ALS patients and their families, who face a devastating diagnosis with few treatment options, the advancement of NurOwn into this FDA-aligned Phase 3b trial represents a potential source of hope in an area of significant unmet medical need.
