Applied Therapeutics to Present at the 2022 Annual Clinical Genetics Meeting of the American College of Molecular Genetics and Genomics
Applied Therapeutics (Nasdaq: APLT) announced the acceptance of their abstract #OP005 for an oral presentation at the 2022 Annual Clinical Genetics Meeting, scheduled for March 22-26 in Nashville. This presentation will focus on the efficacy of AT-007 in reducing toxic galactitol levels in pediatric patients with Classic Galactosemia. Dr. Riccardo Perfetti highlighted the significant correlation between galactitol levels and CNS deficit severity in patients. Approximately 3,000 individuals are affected by Galactosemia in the U.S., emphasizing the importance of ongoing clinical research and development.
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NEW YORK, March 16, 2022 (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, today announced their abstract #OP005 was accepted as an oral platform presentation at the 2022 Annual Clinical Genetics Meeting of the American College of Molecular Genetics and Genomics, March 22 – 26, 2022 in Nashville, Tennessee.
“We are pleased to share this important data with the medical community,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “We demonstrated that galactitol levels correlated with the severity of the CNS deficits in pediatric patients with Classic Galactosemia and that treatment with AT-007 reduced toxic galactitol safely and effectively.”
Presentation Details
Oral Platform Presentation: AT-007 Significantly Reduces Toxic Galactitol in ACTION-Galactosemia Kids - the 1st Therapeutic Interventional Clinical Trial in Children with Classic Galactosemia
- Date and Time: March 23, 12:15 – 12:30 PM CST
- Speaker: Evan Bailey, MD, Executive Clinical Medical Director at Applied Therapeutics
Abstract #OP005: AT-007 Significantly Reduces Toxic Galactitol in ACTION-Galactosemia Kids - the 1st Therapeutic Interventional Clinical Trial in Children with Classic Galactosemia
Evan Bailey, MD, Applied Therapeutics, Stella Wang, BSPharm, MS, MPH, Laura Saltonstall, MD, MBA, Riccardo Perfetti, MD, PhD, and Shoshana Shendelman, PhD.
About Galactosemia
Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. Galactose is found in foods, but is also produced endogenously by the body. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women). There are approximately 3,000 patients with Galactosemia in the US and 80 new births per year.
About AT-007
AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. In an animal model of Galactosemia, AT-007 reduced toxic galactitol levels and prevented disease complications. In clinical trials, AT-007 significantly reduced plasma galactitol levels vs. placebo in adults and children with Galactosemia. AT-007 is currently being studied in a Phase 3 clinical outcomes trial (ACTION-Galactosemia Kids) in children ages 2-17 with Galactosemia, as well as a long-term open label study in adults with Galactosemia. AT-007 has received both Orphan Drug and Pediatric Rare Disease designations from the U.S. Food and Drug Administration (FDA) for the treatment of Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia.
About Applied Therapeutics
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-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. 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.
Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, included in this press release regarding strategy, future operations, prospects, plans and objectives of management, including words such as “may,” “will,” “expect,” “anticipate,” “plan,” “intend,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. Forward-looking statements in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations, or strategies will be attained or achieved.
Such risks and uncertainties include, without limitation, factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in our filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” contained therein. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.
Contacts
Investors:
Maghan Meyers
(212) 600-1902
appliedtherapeutics@argotpartners.com
Media:
media@appliedtherapeutics.com
Applied Therapeutics, Inc.
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