Benzinga | Oct 21, 2020 07:08AM EDT

Applied Therapeutics To Present Data On AT-007 For Treatment Of Galactosemia At American Society Of Human Genetics (ASHG) 2020 Annual Meeting

NEW YORK, Oct. 21, 2020 (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, announced today that it will present two poster presentations covering AT-007 in Galactosemia at the upcoming American Society of Human Genetics (ASHG) 2020 Annual Meeting. The presentations include animal model efficacy data and adult clinical data on the safety and biomarker efficacy of Applied Therapeutics' investigational candidate for Galactosemia, AT-007, a Central Nervous System (CNS) penetrant Aldose Reductase Inhibitor (ARI).



"We are pleased to present data on our Galactosemia program at the ASHG conference," said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. "Our preclinical data demonstrates that reduction in galactitol, a toxic metabolite of galactose, prevents long-term CNS complications in an animal model of Galactosemia. In parallel, our clinical data from the ACTION-Galactosemia study demonstrates rapid and sustained reduction in galactitol with once-daily AT-007 treatment. Together, this data represents an important advancement in our understanding of the disease and a potential therapeutic intervention to halt disease progression."

Presentation Details

Poster #1881 (Abstract #3646) Positive Biomarker Efficacy Results from the ACTION-Galactosemia Study

Presenter Riccardo Perfetti, M.D., Ph.D., Chief Medical Officer of Applied Therapeutics

Time Monday, October 26, 6:00 a.m. -- 11:59 p.m. EDT

Poster #1958 (Abstract #3647) Post-Natal Galactitol Reduction is Associated with Normalization of CNS Phenotype in Animal Model of Galactosemia

Presenter Riccardo Perfetti, M.D., Ph.D., Chief Medical Officer of Applied Therapeutics

Time Monday, October 26, 6:00 a.m. -- 11:59 p.m. EDT

Slides will be available on the Presentations and Publications section of the Applied Therapeutics website following the conference.