Applied Therapeutics, Inc.

We are a clinical-stage biopharmaceutical company developing new drug candidates for diseases with significant unmet medical needs. Our strategy involves targeting previously identified disease processes where earlier drugs failed due to poor effectiveness and tolerability. We use advanced technology to design improved drugs, utilize biomarkers early to confirm biological activity, and focus on potentially faster regulatory approval paths. Our primary molecular target is aldose reductase (AR), an enzyme that converts glucose to sorbitol under oxidative stress and is linked to multiple diseases. Previous attempts to inhibit AR were unsuccessful due to nonselective inhibition, leading to limited efficacy and significant off-target side effects. Our AR program includes several potent and selective AR inhibitors, engineered with specific tissue permeability profiles to target various diseases, such as diabetic complications, heart disease, and rare metabolic diseases. Our lead candidate, AT-007 (also known as govorestat), is a central nervous system (CNS) penetrant aldose reductase inhibitor (ARI). We are developing AT-007 to treat rare metabolic diseases, including Galactosemia and CMT-SORD (Charcot-Marie-Tooth-SORD or Sorbitol Dehydrogenase (SORD) Deficiency). Galactosemia is a rare pediatric metabolic disease affecting how the body processes galactose, with no known cure or approved treatment. The U.S. Food and Drug Administration (FDA) has granted AT-007 orphan drug designation and rare pediatric disease designation for Galactosemia. In June 2021, the FDA granted Fast Track Designation to AT-007 for Galactosemia. In February 2024, the FDA accepted a New Drug Application (NDA) for AT-007 for the treatment of Classic Galactosemia.