Applied Therapeutics Stock Plummets 55% on FDA Uncertainty
Regulatory path for nerve disorder drug govorestat unclear after meeting with agency, delaying a potential New Drug Application.
Shares of Applied Therapeutics (NASDAQ: APLT) plunged more than 55% in pre-market trading after the company revealed that the regulatory path forward for its nerve-disorder drug, govorestat, remains uncertain following a pivotal meeting with the U.S. Food and Drug Administration (FDA).
The clinical-stage biopharmaceutical company announced that discussions with the agency about its New Drug Application (NDA) for the treatment of Charcot-Marie-Tooth Sorbitol Dehydrogenase (CMT-SORD) Deficiency did not yield a clear strategy for submission. This development has , injecting significant uncertainty into the drug's future and erasing gains that had seen the stock rally more than 160% in the prior week.
Investors had been optimistic heading into the meeting, anticipating a potential accelerated approval pathway for govorestat. The drug, a central nervous system penetrant Aldose Reductase Inhibitor, is designed to treat CMT-SORD, a rare and progressive hereditary neuropathy affecting an estimated 3,300 patients in the U.S. The condition, which results from a sorbitol dehydrogenase enzyme deficiency, leads to the accumulation of sorbitol in tissues and can cause significant disability. Govorestat has already received .
The company is now awaiting official meeting minutes from the FDA to determine the next steps. The INSPIRE Phase 2/3 trial data showed that govorestat led to a significant reduction in blood sorbitol levels, a key biomarker of the disease. However, the trial's primary endpoint, a measure of physical function known as the 10-meter walk-run test, , a factor that likely contributed to the FDA's cautious stance.
The sharp sell-off reflects investor disappointment over the lack of a clear and immediate route to market. The pre-meeting surge in share price was fueled by hopes that the confirmatory nature of the recent trial would be sufficient for a swift regulatory review. However, the has tempered those expectations, highlighting the inherent risks in biotech drug development, particularly for rare diseases with complex clinical endpoints. Applied Therapeutics has stated it remains committed to seeking regulatory approval for govorestat, but the timeline for that approval is now considerably more ambiguous.