The USA-based company’s lead drug candidate, AT-007 (govorestat), is a novel central nervous system penetrant aldose reductase inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including galactosemia, SORD deficiency, and PMM2-CDG.

Applied is also developing AT-001, a novel potent ARI, for the treatment of diabetic cardiomyopathy, a fatal fibrosis of the heart. 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.