GlobeNewswire Inc | Oct 21, 2020 07:00AM EDT

October 21, 2020

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 todaythat 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, saidRiccardo 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 StudyPresenter Riccardo Perfetti, M.D., Ph.D., Chief Medical Officer ofApplied TherapeuticsTime 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 GalactosemiaPresenter Riccardo Perfetti, M.D., Ph.D., Chief Medical Officer ofApplied TherapeuticsTime Monday, October 26, 6:00 a.m. 11:59 p.m. EDT

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

AboutApplied TherapeuticsApplied Therapeuticsis a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Companys 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 inJune 2019, read out positive top-line biomarker data in adult Galactosemia patients inJanuary 2020and announced full data from the trial inApril 2020. A pediatric Galactosemia study commenced inJune 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 inSeptember 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.

About AT-007

AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in clinical development for treatment of Galactosemia. AT-007 has been studied in an animal model of Galactosemia, which demonstrated that AT-007 reduces toxic galactitol levels and prevents disease complications.Applied Therapeuticsis conducting a biomarker based development program in patients with Galactosemia, based on the recently released industry guidance on drug development for low prevalence, slowly progressing rare metabolic diseases. The company received Orphan Designation for AT-007 for Galactosemia inMay 2019 and Pediatric Rare Disease Voucher (PRV) designation in 2020.

Investors:Maeve Conneighton(212) 600-1902 orappliedtherapeutics@argotpartners.com

Media:Gleb Sagitovmedia@appliedtherapeutics.com