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Applied Therapeutics, Inc. is a clinical-stage biopharmaceutical company. It is focused on developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company’s lead drug candidate, govorestat, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of central nervous system (CNS) rare metabolic diseases, including Galactosemia, Sorbitol Dehydrogenase (SORD) Deficiency, and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy (DbCM) a fatal fibrosis of the heart. It has completed a Phase I/II clinical trial evaluating AT-001 in approximately 120 patients with type 2 diabetes, in which no drug-related adverse effects or tolerability issues were observed. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of Diabetic retinopathy.
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