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Applied Therapeutics Tanks As FDA Path For Lead Rare-Disease Drug Turns Uncertain
Benzinga· 2025-11-13 18:05
Core Viewpoint - Applied Therapeutics, Inc. is experiencing a significant decline in stock price while providing updates on its regulatory strategy for Govorestat related to Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD) following a Type C meeting with the FDA [1][4]. Regulatory Strategy - The company received official meeting minutes from the FDA regarding Govorestat for CMT-SORD, which is a genetic defect that leads to toxic sorbitol buildup in cells, damaging peripheral nerves [2]. - Key open issues discussed in the meeting included the need for conclusive evidence on the pathophysiology of CMT-SORD, the use of sorbitol levels as a surrogate endpoint, selection of a primary endpoint for a potential Phase 3 trial, and carcinogenicity testing [3]. - The company plans to request an additional Type C meeting with the FDA to further discuss the design of a potential Phase 3 trial [3]. Financial Position - As of September 30, 2025, the company reported cash and cash equivalents totaling $11.9 million [4]. - The stock price of APLT has decreased by 58.8%, trading at $0.37 during the latest check [4].