Applied Therapeutics meets with FDA on potential CMT-SORD drug

NEW YORK - Applied Therapeutics, Inc. (NASDAQ:APLT) announced Monday it has completed a meeting with the U.S. Food and Drug Administration regarding a potential new drug application for govorestat to treat Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD). The news has sparked investor interest, with the stock surging over 160% in the past week, according to InvestingPro data.

The clinical-stage biopharmaceutical company, currently valued at $178.58 million, is awaiting official meeting minutes from the FDA before determining next steps. According to the company’s press release statement, a submission strategy for regulatory approval, including under the accelerated approval pathway, has not yet been determined. InvestingPro analysis shows the company maintains a strong balance sheet with more cash than debt, though it remains unprofitable.

CMT-SORD is described as a rare, progressive hereditary neuropathy affecting peripheral nerves and motor neurons. The condition impacts approximately 3,300 patients in the U.S. and 4,000 in the EU. It occurs due to a lack of the enzyme sorbitol dehydrogenase, which leads to sorbitol accumulation in blood and tissues.

Govorestat, a central nervous system penetrant Aldose Reductase Inhibitor, has received Orphan Drug Designation from the FDA for CMT-SORD treatment, along with Orphan Medicinal Product Designation from the European Medicines Agency.

The drug candidate is also being developed for other rare diseases including Classic Galactosemia and phosphomannomutase 2 congenital disorder of glycosylation.

Applied Therapeutics focuses on developing novel drug candidates for rare diseases. The company maintains it remains committed to addressing unmet needs of patients living with CMT-SORD.

In other recent news, Applied Therapeutics, Inc. has entered into a Controlled Equity Offering Sales Agreement with Cantor Fitzgerald & Co. This agreement allows Applied Therapeutics to offer and sell shares of its common stock, with an aggregate offering price of up to $75 million. The sales will be conducted as "at the market" offerings, adhering to SEC regulations. These offerings will be made under the company’s existing shelf registration statement on Form S-3, which became effective earlier this year in May. The prospectus supplement was filed recently to facilitate these transactions. This development marks a significant move for Applied Therapeutics as it seeks to raise capital through equity sales.

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