FDA Approves BrainStorm’s Phase IIIb Trial Design for NurOwn in Early-Stage ALS

1. BrainStorm Cell Therapeutics received approval from the FDA for the design of a Phase IIIb confirmatory trial of its lead candidate, NurOwn (debamestrocel), in early-stage ALS.
2. The FDA's agreement was made under a Special Protocol Assessment (SPA), which validates the clinical trial protocol and statistical analysis.
3. The Phase IIIb trial is expected to begin in 2024 and will consist of two parts: a double-blind, placebo-controlled period of 24 weeks involving approximately 200 patients (Part A), and a 24-week open-label period (Part B).
4. The primary endpoint of the trial is the change in the Revised Amyotrophic Lateral Sclerosis Functional Rating (ALSFRS-R) total score.
5. BrainStorm Cell Therapeutics has faced challenges with NurOwn, including a Biologics License Application (BLA) rejection in October 2023, and a previous Phase III trial that did not meet its primary endpoint.
6. The Phase III trial did show a notable decrease in neurofilament light, which is considered a positive finding.
7. GlobalData predicts global sales of NurOwn to reach $964 million in 2029.
8. ALS is a fatal type of motor neuron disease with a high failure rate in trials due to the disease's unclear etiology and complex pathophysiology, resulting in a persistent unmet need for effective treatments.