Applied Therapeutics to Present Cross-Sectional Analysis of Galactosemia Disease Progression at 2021 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics
NEW YORK, April 07, 2021 (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 a poster presentation at the upcoming 2021 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics (ACMG) taking place April 13-16, 2021. The abstract and presentation include a cross-sectional analysis of nineteen pediatric patients with Classic Galactosemia, characterizing progressive worsening of the central nervous system (CNS) phenotype with age.
“We are pleased to share this important data with the medical community,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “The severity and significant decline in CNS outcomes with age underscores the urgency for a disease-modifying therapy to halt or prevent damage in Galactosemia. ”
Abstract eP011: Progressive Worsening of Central Nervous System Phenotype in Children with Classic Galactosemia: a Cross-Sectional Analysis
The poster presentation will be available on the ACMG website.
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 Galactosemia, a rare pediatric metabolic disease. The Company initiated a pivotal Phase 1/2 clinical trial in June 2019, read out positive top-line biomarker data in adult Galactosemia patients in January 2020 and announced full data from the trial in April 2020. A pediatric Galactosemia study commenced in June 2020. 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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