BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) experienced a significant drop in premarket trading, with shares falling by 56% on Thursday. This came after advisors to the U.S. Food and Drug Administration (FDA) rejected the company's investigational treatment for amyotrophic lateral sclerosis (ALS).
FDA Advisors Question Effectiveness of NurOwn
The treatment in question, called NurOwn, utilizes mesenchymal stem cell therapy. However, the FDA advisors voted against it, stating that there was insufficient evidence of its effectiveness in treating mild to moderate ALS. In a briefing document released ahead of the meeting, the FDA outlined several objections, including the therapy's unclear and inconsistent mechanism of action across the application.
Co-CEO of BrainStorm, Stacy Lindborg, expressed disappointment in the committee's decision, referring to it as a "sad outcome for the ALS community." Despite this setback, the company remains confident in NurOwn's potential and believes that the data presented during the meeting makes a compelling case for its approval.
As a result of this news, BrainStorm shares have seen a significant decline of 76% year-to-date. In contrast, the broader market, as represented by the S&P 500, has experienced an 11.3% gain during the same period.
The decision by FDA advisors highlights the challenges faced by companies developing treatments for devastating diseases like ALS. BrainStorm will need to address the concerns raised by the FDA before moving forward with its therapeutic candidate.