Applied Therapeutics to Present Cross-Sectional Analysis of Galactosemia Disease Progression at 2021 Annual Clinical Genetics...
07 Aprile 2021 - 1:00PM
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. ”
Presentation
DetailsAbstract 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
TherapeuticsApplied 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.
To learn more, please visit
www.appliedtherapeutics.com and follow the company
on Twitter @Applied_Tx.
Investors:Maeve Conneighton(212) 600-1902
orappliedtherapeutics@argotpartners.com
Media:media@appliedtherapeutics.com
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