BrainStorm Cell Therapeutics Inc., a leading developer of adult stem cell therapies for neurodegenerative diseases, announced that the US Food and Drug Administration (FDA) has cleared the company to initiate its phase 3b clinical trial of NurOwn (autologous MSC-NTF cells) for the treatment of amyotrophic lateral sclerosis (ALS).
The trial design was previously agreed upon with the FDA under a Special Protocol Assessment (SPA), confirming the study's endpoints and statistical methodology are appropriate to support a future Biologics License Application (BLA) submission. This clearance allows the company to proceed with patient enrollment.
""This FDA clearance is a defining milestone for BrainStorm and the ALS community,"" said Chaim Lebovits, president and chief executive officer of BrainStorm. ""We are now positioned to swiftly activate clinical sites in a phased manner, diligently preparing to enroll the first patient in the phase 3b trial. At the same time, we are actively working to secure funding through multiple avenues, including non-dilutive grants, to ensure the timely and successful launch of this critical study.""
The phase 3b trial will enroll approximately 200 participants at leading academic medical centres and will consist of a 24-week randomized, double-blind, placebo-controlled phase followed by a 24-week open-label extension where all participants will receive NurOwn. The primary endpoint is the change from baseline to week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R).
BrainStorm Cell Therapeutics Inc. is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection.
