Applied Therapeutics to Present Data on AT-007 for the Treatment of Galactosemia at the American Society of Human Genetics (A...
21 Ottobre 2020 - 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, 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
StudyPresenter: Riccardo Perfetti, M.D., Ph.D.,
Chief Medical Officer of Applied
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 of Applied
TherapeuticsTime: 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.
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.
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
Therapeutics is 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 in May 2019 and
Pediatric Rare Disease Voucher (PRV) designation in 2020.
Investors:Maeve Conneighton(212) 600-1902
orappliedtherapeutics@argotpartners.com
Media:Gleb
Sagitovmedia@appliedtherapeutics.com
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