Applied Therapeutics is a clinical-stage biopharmaceutical company focused on rare metabolic diseases. The company has no approved products and no product revenue. Applied Therapeutics' entire pipeline is built around inhibiting aldose reductase (AR), an enzyme that converts glucose or galactose into sorbitol under oxidative stress. Excess sorbitol is toxic to cells, causing damage to nerves, the brain, heart, and eyes. Applied Therapeutics argues its AR inhibitors (ARIs) are more potent and selective than earlier-generation ARIs, which failed due to liver toxicity. The lead drug, govorestat (AT-007), is a CNS-penetrant ARI targeting two rare pediatric diseases: Classic Galactosemia (roughly 8,000 patients worldwide, no approved treatment) and SORD Deficiency (roughly 8,400 patients worldwide, no approved treatment). The FDA issued a Complete Response Letter for govorestat in Galactosemia in November 2024, and Applied Therapeutics is working to address the deficiencies. For SORD Deficiency, Applied Therapeutics completed a Phase 2/3 trial and is evaluating whether the data supports an NDA submission. Two other pipeline compounds — one targeting diabetic cardiomyopathy, one targeting diabetic retinopathy — are deprioritized. Applied Therapeutics funds operations through equity offerings and has not yet commercialized any products. If govorestat is approved, Applied Therapeutics plans to sell directly in the U.S. and has licensed European rights to Advanz Pharma in exchange for milestones of up to €134M and a 20% royalty on European net sales.