Zacks Small Cap Research – BCLI: Unveils Plans for Phase 3b Trial of NurOwn® in ALS… – Technologist

By David Bautz, PhD

NASDAQ:BCLI

READ THE FULL BCLI RESEARCH REPORT

Business Update

Phase 3b Trial Design Announced; SPA Submitted to the FDA

On March 4, 2024, BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI) presented a poster at the MDA Clinical and Scientific Conference with an outline of the proposed design for a Phase 3b trial of NurOwn® (debamestrocel) in amyotrophic lateral sclerosis (ALS).

The following figure gives an overview of the proposed trial design, which is now under review by the U.S. Food and Drug Administration (FDA). Up to approximately 200 patients with mild-to-moderate ALS will be enrolled into the two-part study: Part A will be a 24-week, randomized, double blind period while Part B will be a 24-week open-label extension period. The primary endpoint will be based on the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R). Patients will be treated at Weeks 0, 8, and 16 during Part A and then Weeks 24, 32, and 40 in Part B.

In February 2024, BrainStorm announced it submitted a Special Protocol Assessment (SPA) to the U.S. FDA regarding the design of the Phase 3b trial. Participating in the SPA program will allow BrainStorm to reach a consensus with the FDA on the Phase 3b trial design and ensure that the study is considered adequate by the FDA to support a future marketing evaluation. A response from the FDA is anticipated within 45 days of submission and the company will then take any feedback into consideration before finalizing the design of the trial.

As a reminder, when examining patients with a baseline ALSFRS-R ≥ 35 in the Phase 3 trial, patients treated with NurOwn exhibited a clinical response 2x higher than placebo and had a significantly slower rate of ALS progression, as shown in the following figure. This supports the company’s decision to include patients with mild-to-moderate ALS in the Phase 3b trial.

Conclusion

We look forward to an update regarding the FDA’s feedback on the SPA and the finalized design for the Phase 3b trial along with guidance on when the trial is expected to initiate. Participating in the SPA program is a smart strategic move as agreement with the FDA on the design for a Phase 3 program will help to de-risk certain regulatory aspects of the program. With the SPA being submitted and a response expected soon from the FDA, we have increased our valuation to $1.00 per share.

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