FDA Approves Phase IIIb Trial Plan for Early-Stage ALS Treatment

BrainStorm Cell Therapeutics has received validation from the FDA for the design of its Phase IIIb trial for the ALS treatment candidate, NurOwn, following a previous rejection. The company had withdrawn its Biologics License Application after an FDA advisory committee voted against it in September 2023. The Special Protocol Assessment (SPA) agreement with the FDA confirms the trial’s methodology and statistical approach for the late-stage study, which BrainStorm CEO Chaim Lebovits views as a step towards regulatory approval.

The SPA agreement does not guarantee approval but is seen as reducing risks associated with the clinical program. The trial, set to begin this year, will consist of a 24-week placebo-controlled phase followed by an open-label extension. The primary goal is to measure changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. The study will also examine biomarkers related to neuroinflammation, neurodegeneration, and neuroprotection.

NurOwn is a stem cell therapy that utilizes a patient’s bone marrow to extract mesenchymal stem cells, which are then prompted to secrete factors that support neuronal health and potentially counteract ALS-related motor neuron degeneration. Despite the FDA’s initial rejection due to conflicting data and perceived lack of efficacy, a subgroup of patients with early-stage ALS showed a significant response rate to NurOwn compared to placebo.

BrainStorm's stock has seen an uptick following the FDA’s agreement on the trial design. The company’s stock price had plummeted but has been on a gradual recovery since the submission of the SPA request in February 2024. The upcoming Phase IIIb trial will focus on patients with early-stage ALS.