Yahoo Finance

By David Bautz, PhD

NASDAQ:BCLI

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Business Update

Update on Planned Phase 3b Trial of NurOwn^® in ALS

On December 30, 2024, BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI) conducted an investor update call to provide a corporate update. The company is currently preparing for a Phase 3b clinical trial of NurOwn in patients with amyotrophic lateral sclerosis (ALS). During 2024, the company achieved a number of significant milestones in support of the upcoming Phase 3b trial:

• In April 2024, the company announced it had received written agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) on the design of the Phase 3b trial. The SPA agreement indicates that the FDA concurs with the adequacy and acceptability of specific elements of a protocol’s overall design to ensure that the trial conducted under the protocol can be considered an adequate and well-controlled study to support marketing approval.

• In June 2024, the company announced it reached alignment with the FDA on the Chemistry, Manufacturing, and Controls (CMC) aspect of the Phase 3b trial. This means that the company has resolved all previous outstanding CMC issues identified by the FDA. Given the complexities of cell therapy products, reaching alignment with the FDA ahead of the Phase 3b trial is an additional means to lower the regulatory risk for NurOwn.

• In November 2024, the company announced it has entered into a Memorandum of Understanding (MOU) with Pluri Inc. to manufacture NurOwn for use in the planned Phase 3b trial. Pluri will provide GMP-compliant manufacturing of NurOwn for the trial and the companies will explore the potential for manufacturing support for potential future commercial distribution of NurOwn, if approved.

BrainStorm has continued to indicate that the Phase 3b trial will initiate in the first quarter of 2025. However, this will be dependent upon obtaining the necessary funding to conduct the trial. The company is pursuing non-dilutive financing and grants as well as other funding opportunities.

Positive Survival Data Presented at 2024 NEALS Meeting

In October 2024, BrainStorm presented a poster, titled ‘Debamestrocel Long-Term Benefits on Survival and Neurodegeneration in ALS Expanded Access Program’ (Dagher et al., 2024), which discussed the results achieved with ALS patients that participated in the Expanded Access Program (EAP) for NurOwn. Highlights from the poster include:

• A total of ten participants (6 debamestrocel, 4 placebo) from BrainStorm’s prior Phase 3 trial enrolled in the EAP, which took place over two 28-week periods. Participants received debamestrocel every eight weeks for a maximum of six doses over the two periods.

• A comparable cohort was identified from the PRO-ACT database through a comparison of baseline characteristics using propensity score matching (PSM).

• At the final visit in the EAP, 9/10 participants were alive. A comparison of the survival curves showed a statistically significant difference in favor of debamestrocel (P=0.0379) with a median survival time of 46.6 months compared to 41.1 months for the matched control group.

• For the participants that received placebo in the Phase 3 trial, the group median change in neurofilament light (NfL) was 37%, indicating worsening neurodegeneration. However, after those participants received debamestrocel in the EAP, the majority showed a stabilization in NfL levels.