Applied Therapeutics Announces Acceptance of Late-Breaking Abstract for Oral Presentation at the Peripheral Nerve Society 2025 Annual Meeting

Core Insights - Applied Therapeutics, Inc. announced the presentation of full 12-month clinical results from the INSPIRE Phase 3 trial of govorestat (AT-007) at the Peripheral Nerve Society 2025 Annual Meeting, along with new topline 18-month and 24-month data [1] - The INSPIRE Phase 3 trial is a double-blind, placebo-controlled study evaluating govorestat's effect on patients with Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease [1] - Govorestat is a central nervous system penetrant Aldose Reductase Inhibitor being developed for multiple rare diseases, including CMT-SORD, Classic Galactosemia, and PMM2-CDG [2][4] Presentation Details - The oral presentation titled "INSPIRE CMT-SORD Clinical Trial: Month 18 and Month 24 INSPIRE Trial Results and Designing the Next Randomized Study" will be presented by Dr. Evan Bailey on May 18, 2025 [2] - The presentation is scheduled for 12:25 - 12:40 p.m. BST [2] Regulatory Designations - Govorestat has received Orphan Medicinal Product Designation from the European Medicines Agency for both Galactosemia and CMT disease [3] - It has also received Orphan Drug Designation from the U.S. FDA for Galactosemia, PMM2 deficiency, and SORD Deficiency, along with Rare Pediatric Disease designation for Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia [3] Company Overview - Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel drug candidates targeting rare diseases [4] - The lead drug candidate, govorestat, is aimed at treating CNS rare metabolic diseases, including Classic Galactosemia, CMT-SORD, and PMM2-CDG [4]