The FDA's initial review concluded that current data from BrainStorm Cell Therapeutics' phase 3 trial in amyotrophic lateral sclerosis (ALS) does not sufficiently provide the threshold of substantial evidence to support a marketing application.
The New York-based biotech's NurOwn technology platform (autologous MSC-NTF cells) represents a investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. The phase 3 pivotal trial in ALS, also known as Lou Gehrig’s disease, investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells.
While the FDA concluded from their review that the current level of clinical data does not provide enough evidence to support a Biologics License Application (BLA) for the company's lead asset, this recommendation does not preclude Brainstorm from proceeding with a BLA submission.
"Brainstorm will first consult with principal investigators, ALS experts, expert statisticians, regulatory advisors, and ALS advocacy groups to assess the benefit/risk of a BLA submission before making a final decision," said Chaim Lebovits, CEO Brainstorm Cell Therapeutics.
Despite the lackluster top-line results from the phase 3 ALS study of the NurOwn treatment, Brainstorm expects positive phase 2 data from a study testing its effects on progressive multiple sclerosis.
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