Critical Importance of FDA AdComm Meeting for ALS Therapy NurOwn

NurOwn (BrainStorm Cell Therapeutics) is an investigational therapy made up of autologous mesenchymal stromal cells secreting neurotrophic factors cells (MSC-NTF) for the potential treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The submitted biologics license application (BLA) for the agent was based on a phase 3 study (NCT03280056) in which the agent originally failed to meet its primary end point; however, it continued to have significant effects on less severe forms of ALS. In the original analysis, 33% and 28% of patients on NurOwn and placebo respectively, met the primary end point of change in disease progression of at least 1.25 points on ALS Functional Rating Scale (ALSFRS-R).