Applied Therapeutics to Present Data Highlighting AT-007 for the Treatment of Galactosemia at the American Society of Human Genetics (ASHG) 2019 Annual Meeting
Details on the Oral Presentation and Educational Symposium are below:
Title: AT-007, a Novel CNS Penetrant Aldose Reductase Inhibitor Prevents the Metabolic and Tissue Specific Abnormalities of Galactosemia, in a GALT Deficient Rat Model of Disease
Date and Time:
Location: Room 370A – Level 3/Convention Center
The presentation will be available on the ASHG conference website as well as the
Galactosemia Educational Symposium
Title: Development of an Oral Treatment for Galactosemia
Date and Time:
Location: Marriott Marquis Houston,
- Clinical presentation of Classic Galactosemia
Jerry Vockley, MD, PhD, UMPC Children’s Hospital, Pittsburgh University
- Biology and biochemistry of Classic Galactosemia
Gerard Berry, MD, Boston Children’s Hospital and Harvard Medical School
- Preclinical evidence and clinical development for a novel oral compound to prevent complications of Classic Galactosemia
Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics
Additional details for the event can be found here.
Galactosemia is a rare metabolic disease that affects how the body processes a simple sugar called galactose, and for which there is no known cure or approved treatment available. Galactose is found in foods, but the human body also naturally produces galactose on its own, so dietary restriction can’t prevent complications of disease. It is estimated that the U.S. Galactosemia population is approximately 2,800 patients, based on newborn screening data identifying 2,500 infants through 2014, and the estimated birth rate of 80 patients per year. High levels of galactose circulating in the blood and tissues of Galactosemia patients enables Aldose Reductase to convert galactose to a toxic metabolite, called galactitol, which causes long-term complications ranging from CNS dysfunction to cataracts.
AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in Phase 1/2 development for treatment of Galactosemia. AT-007 has been studied in 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 draft 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.
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-001, is a novel aldose reductase inhibitor (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. Applied Therapeutics is also developing AT-007, a central nervous system penetrant ARI, for the treatment of Galactosemia, a rare pediatric metabolic disease, and initiated a Phase 1/2 clinical trial in June 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, expected to advance into a Phase 1 study in 2020.
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Source: Applied Therapeutics