Applied Therapeutics to Present at the 2022 Annual Clinical Genetics Meeting of the American College of Molecular Genetics and Genomics
NEW YORK, March 16, 2022 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced their abstract #OP005 was accepted as an oral platform presentation at the 2022 Annual Clinical Genetics Meeting of the American College of Molecular Genetics and Genomics, March 22 – 26, 2022 in Nashville, Tennessee.
“We are pleased to share this important data with the medical community,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “We demonstrated that galactitol levels correlated with the severity of the CNS deficits in pediatric patients with Classic Galactosemia and that treatment with AT-007 reduced toxic galactitol safely and effectively.”
Oral Platform Presentation: AT-007 Significantly Reduces Toxic Galactitol in ACTION-Galactosemia Kids - the 1st Therapeutic Interventional Clinical Trial in Children with Classic Galactosemia
- Date and Time: March 23, 12:15 – 12:30 PM CST
- Speaker: Evan Bailey, MD, Executive Clinical Medical Director at Applied Therapeutics
Abstract #OP005: AT-007 Significantly Reduces Toxic Galactitol in ACTION-Galactosemia Kids - the 1st Therapeutic Interventional Clinical Trial in Children with Classic Galactosemia
Evan Bailey, MD, Applied Therapeutics, Stella Wang, BSPharm, MS, MPH, Laura Saltonstall, MD, MBA, Riccardo Perfetti, MD, PhD, and Shoshana Shendelman, PhD.
Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. Galactose is found in foods, but is also produced endogenously by the body. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women). There are approximately 3,000 patients with Galactosemia in the US and 80 new births per year.
AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. In an animal model of Galactosemia, AT-007 reduced toxic galactitol levels and prevented disease complications. In clinical trials, AT-007 significantly reduced plasma galactitol levels vs. placebo in adults and children with Galactosemia. AT-007 is currently being studied in a Phase 3 clinical outcomes trial (ACTION-Galactosemia Kids) in children ages 2-17 with Galactosemia, as well as a long-term open label study in adults with Galactosemia. AT-007 has received both Orphan Drug and Pediatric Rare Disease designations from the U.S. Food and Drug Administration (FDA) for the treatment of Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia.
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, SORD Deficiency and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, 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, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications.
To learn more, please visit www.appliedtherapeutics.com and follow the company on Twitter @Applied_Tx.
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Applied Therapeutics, Inc.