Applied Therapeutics Reports Additional Pediatric Biomarker Data from ACTION-Galactosemia Kids | Your Money

Significant and Statistically Significant 40% Reduction in Plasma Galactitol with New Weight-Based Dosing Parameters

Baseline galactitol levels clearly correlated with baseline clinical functional outcomes

NEW YORK, Oct. 18, 2021 (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 reported additional biomarker data from the pediatric ACTION-Galactosemia Kids study.

As previously announced, a pharmacokinetic analysis of AT-007 plasma drug levels at Day 30 revealed that dosage in children could be further optimized by adjusting the dose based on weight rather than age. As such, weight-based dosing brackets were implemented and a subset of pediatric patients was dose adjusted for an additional 30 days.

A new data analysis was performed on galactitol reduction across the study, including data from children who received dose adjustment. AT-007 significantly reduced plasma galactitol by approximately 40%, which was statistically significant (p<0.001) versus placebo. The decrease in plasma galactitol was rapid and sustained, with no effect on galactose or Gal-1p levels, and was comparable in all dose groups. AT-007 was safe and well tolerated in children of all ages (2-17). The company plans to file a New Drug Application (NDA) with the US Food and Drug Administration (FDA) in the fourth quarter of this year.

As previously announced, an analysis of the 47 children in the ACTION-Galactosemia Kids study showed a clear correlation between baseline galactitol level and baseline clinical functional outcomes. Children with higher plasma galactitol levels showed greater disease severity than children with lower plasma galactitol levels at baseline. This data will be presented as a recent summary at the 14th International Congress on Inborn Errors of Metabolism (ICIEM) November 21-24, 2021.

“We have shown that toxic galactitol stimulates disease progression and has a profound impact on the quality of life of galactosemia patients,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “Here we showed that AT-007 significantly lowers galactitol levels, which we believe could change the lives of children and adults living with this disease. We are all families participating in the ACTION-Galactosemia clinical program, who have made this study possible, are sincerely grateful.”

“The commercial team has made great progress in preparing for a potential launch of AT-007 in Galactosemia. Our ‘Galactosemia Together’ disease awareness campaign has generated tremendous interest from the physicians and patient community, and our website galactosemia.com has won numerous industry awards. We look forward to potentially bringing AT-007 to these patients who urgently need a treatment option,” said Adam Hansard, Chief Commercial Officer of Applied Therapeutics.

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 significant 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 rare metabolic diseases of the central nervous system, including galactosemia, SORD deficiency and PMM2-CDG. The company is also developing AT-001, a new 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 pass 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 .

For more information, visit www.appliedtherapeutics.com and follow the company on Twitter @Applied—Tx.

Forward-Looking Statements

This press release contains “forward-looking statements” that involve significant risks and uncertainties for the purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical fact, contained 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 referring to future events, circumstances or circumstances) are forward-looking statements. These include, without limitation, statements regarding (i) the timing of our NDA filing for potential approval of AT-007, which will include data from the ACTION-Galactosemia Kids trial and the 90-day safety data in adults with galactosemia , (ii) the timing of our franchise expansion programs for rare diseases in SORD deficiency, (iii) the timing of the initiation and completion of our clinical trials, including ARISE-HF, (iv) the likelihood that data from our clinical trials the future development of our product candidates and (v) the likelihood of obtaining regulatory approval from our product candidates. Forward-looking statements in this press release involve significant risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we therefore make no representation that our plans, intentions, expectations or strategies will will be reached or reached.

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Contacts

Investors: Maghan Meyers (212) 600-1902 or Appliedtherapeutics@argotpartners.com

Media: media@appliedtherapeutics.com

Applied Therapeutics, Inc.

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