Applied Therapeutics Reports Third Quarter 2025 Financial Results

Core Insights - The company is making significant progress in its regulatory strategy for govorestat, particularly for the treatment of CMT-SORD, with a meeting scheduled with the FDA in Q4 2025 to discuss the potential NDA submission [1][3][7] - New data on govorestat for PMM2-CDG has been published, showing promising results that support further clinical development [2][4][9] - A leadership transition has occurred within the Board of Directors, with John Johnson stepping down as Executive Chairman [10] Regulatory Strategy - The company received Type C meeting minutes from the FDA regarding govorestat for CMT-SORD, which included constructive feedback on the data submitted and requirements for a potential NDA submission [7] - An additional Type C meeting is planned to discuss the design of a potential Phase 3 trial for CMT-SORD [3][7] - A meeting is also scheduled with the FDA to review govorestat for Classic Galactosemia, following a Complete Response Letter received in November 2024 [6][8] Clinical Development - New data from a single-patient trial for PMM2-CDG was published in JIMD and presented at the 2025 ASHG Annual Meeting, indicating a dose-dependent decrease in whole blood sorbitol and a 46% improvement in the Nijmegen Pediatric CDG Rating Scale [9] - Govorestat has received Orphan Drug Designation and Rare Pediatric Disease designation from the FDA for PMM2-CDG [9] Financial Performance - As of September 30, 2025, the company reported cash and cash equivalents of $11.9 million, a decrease from $79.4 million at the end of 2024 [13] - Research and development expenses for Q3 2025 were $9.6 million, down from $14.8 million in Q3 2024, primarily due to reduced clinical and preclinical expenses [13] - The net loss for Q3 2025 was $19.0 million, or $0.13 per share, compared to a net loss of $68.6 million, or $0.48 per share, for the same period in 2024 [13][21]