BrainStorm Cell Therapeutics Inc. announced that it has submitted a Special Protocol Assessment (SPA) request to the U.S. Food and Drug Administration (FDA) for a Phase 3b study of NurOwn, its investigational treatment for amyotrophic lateral sclerosis (ALS). The submission follows an in-person meeting that BrainStorm held with the FDA in December 2023. The interactions leading up to this submission have provided guidance on advancing the planned Phase 3b trial.

Participating in an SPA program would allow BrainStorm to reach consensus with the FDA on its clinical trial design, including the proposed statistical analysis, before the study is initiated. This would help ensure that the study would be considered adequate to support a future marketing evaluation. A Special Protocol Assessment (SPA) is a process in which drug developers may ask to meet with the FDA to reach agreement on the design and size of certain clinical trials to determine if they adequately address scientific and regulatory requirements for a study that could support marketing approval.

An SPA agreement indicates concurrence by FDA with the adequacy and acceptability of specific critical elements of overall protocol design for a study intended to support a future marketing application. These elements are critical to ensuring that the trial conducted under the protocol can be considered an adequate and well-controlled study that can support marketing approval. Feedback on these issues provides the greatest benefit to companies in planning late-phase development strategy.

An SPA agreement does not indicate FDA concurrence on every protocol detail. The NurOwn®? technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders.

The NurOwn clinical program has generated valuable insights into the pathology of ALS, as well as disease progression and treatment. Since the initial Phase 3 readout, BrainStorm has shared the full dataset through rigorous peer-reviewed analysis, including: quantification of Floor Effect, which had been noted, but never before explored in depth; evaluation of multiple pre-specified biomarkers, collected at seven different points across 20 weeks during the trial, allowing a longitudinal view; and analysis of genetic data, which represents one of the first ALS trials to prospectively invoke pharmacogenomic analysis of clinical outcome, offering great promise for the development of future treatments for ALS.