FDA Grants AT-007 Pediatric Rare Disease Designation and Orphan Designation for Treatment of PMM2-CDG
AT-007, a novel CNS penetrant Aldose Reductase inhibitor (recently given the INN scientific name gavorestat) has demonstrated biological activity in a validated model of PMM2-CDG. Applied Therapeutics plans to initiate a clinical study in 2021 and is currently working with CDG experts and the FDA to design a robust clinical program.
“PMM2-CDG is a debilitating disease with no drugs available, which is central to Applied Therapeutics’ mission of creating life-changing treatments for patients who desperately need them,” said
About Applied Therapeutics
Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company’s lead drug candidate, AT-007, is a novel central nervous system penetrant aldose reductase inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia and PMM2-CDG. The Company initiated a Galactosemia clinical program in June 2019, read out positive data in adult Galactosemia patients in
The Company is also developing AT-001, a novel potent ARI that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The Company initiated a Phase 3 registrational study in DbCM in September 2019. 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, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications.
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Source: Applied Therapeutics