Shares of BrainStorm Cell Therapeutics Inc. (NASDAQ:BCLI) fell almost 40% on Monday morning after Food and Drug Administration (FDA) staff reviewers expressed doubts over the safety and efficacy of the biotech’s therapy NurOwn for neurogenerative disease amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
Brainstorm’s NurOwn is being reviewed by the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee on September 27.
FDA staff reviewers said in briefing documents ahead of the meeting that the agency has “potential issues with the quality and safety of this product.”
They pointed to the company’s Phase 3 study which showed the survival of patients with ALS was worse for those treated with NurOwn than the group treated with the placebo, indicating a lack of survival benefit.
In addition to its clinical and statistical concerns, the FDA reviewers flagged issues with product manufacturing and a lack of data around this.
Examples of excluded key information included missing or inadequate control of materials, validation of methods missing or incomplete, lack of data demonstrating manufacturing consistency, control strategy for prefilled syringes not provided, inadequate manufacturing and testing facility information, and facilities not ready for inspection, they said.
BrainStorm stock shed 37.5% to US$0.51 in early trade on Monday.
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