BrainStorm Cell Therapeutics Gearing Up for Phase 3b Trial of NurOwn, Its ALS Stem Cell Therapy, Submits IND Amendment

BrainStorm Cell Therapeutics has submitted an amendment to its Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for NurOwn®, the company’s autologous mesenchymal stem cell therapy (stem cells derived from a patient’s own bone marrow) for amyotrophic lateral sclerosis (ALS).

NurOwn® is an autologous, bone marrow-derived mesenchymal stem cell therapy for neurodegenerative disorders. Bone marrow is aspirated from the patient, the stem cells are expanded and differentiated ex vivo, then grown under patented conditions to induce secretion of high levels of neurotrophic factors (NTFs).

The end product is what they call “MSC-NTF cells”, which they say may deliver multiple NTFs and immunomodulatory cytokines directly to sites of neurological damage, potentially slowing or stabilizing disease progression.

The amendment precedes the beginning of their Phase 3b trial, which has reportedly been designed under a [Special Protocol Assessment](https://www.fda.gov/regulatory-information/search-fda-guidance-documents/special-protocol-assessment-guidance-industry) (SPA) with the FDA. SPA is a process where the FDA evaluates a trial’s protocol to ensure they are scientifically sound and likely meets regulatory requirements for a future Biologics License Application (BLA).

BrainStorm expects the regulatory review of the IND amendment to proceed quickly, allowing the trial to begin shortly thereafter.

“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.”

The Phase 3b trial aims to enroll approximately 200 ALS participants and will be conducted in two parts:

In Part A, patients will receive three doses of either NurOwn or placebo over a 24-week double-blind period. Following this, all participants—including those initially in the placebo group—will be eligible to transition into an open-label extension (Part B), where they will receive three additional doses of NurOwn over another 24 weeks.

The primary efficacy endpoint will assess changes in ALS Functional Rating Scale-Revised (ALSFRS-R) scores from baseline to week 24, comparing disease progression between the NurOwn and placebo groups. BrainStorm reports that the successful completion of Part A will allow the company to submit a BLA, potentially accelerating the therapy’s path toward regulatory approval.

ALS remains a disease with limited therapeutic options, and BrainStorm states that it is committed to working closely with regulatory agencies, clinical investigators, and the ALS community to ensure timely and rigorous evaluation of NurOwn. The company will provide further updates as the FDA review progresses.

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