Substantial and statistically significant reduction in plasma galactitol

Baseline analysis demonstrates clear relationship between plasma galactitol level and severity of disease in pediatric Galactosemia patients

NEW YORK, Sept. 09, 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 initial biomarker data from the pediatric ACTION-Galactosemia Kids study, as well as baseline analysis of the 47 children enrolled in the study.

The results demonstrate a substantial reduction in plasma galactitol of approximately 35% after 30 days of treatment, which was statistically significant (p<0.001) vs. placebo.  AT-007 was safe and well tolerated in children of all ages (2-17).  A pharmacokinetic analysis of AT-007 plasma drug levels at day 30 revealed that dosing could be further optimized by adjusting dose based on weight rather than age.  As such, weight-based dosing brackets have been implemented for the remainder of the study, and a subset of patients have been dose-adjusted. The Company expects to present additional biomarker data at the optimized drug levels pending analysis at day 30.  Due to the time required for these study changes, the Company now plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in Q4 of this year, and has adjusted projected timelines accordingly.

Additionally, analysis of the 47 children in the ACTION-Galactosemia Kids study demonstrated a clear correlation between baseline galactitol level and baseline clinical functional outcomes. Children with higher plasma galactitol levels displayed greater disease severity vs. children with lower plasma galactitol levels at baseline. This data is the first demonstration of correlation of a biochemical biomarker with severity of disease in Galactosemia patients. Full data will be presented at a future medical conference.

“Reduction in toxic galactitol is critically important to patients with Galactosemia,” said Shoshana Shendelman, PhD, CEO, Founder and Chair of the Board of Applied Therapeutics. “AT-007 has previously demonstrated significant reduction in galactitol in adults with Galactosemia. Now, we have demonstrated a significant reduction in children with this devastating rare disease. As the first interventional clinical study in pediatric Galactosemia patients, ACTION-Galactosemia Kids has already provided important insight into disease severity and progression. This new data demonstrating correlation of galactitol level with severity of disease underscores the importance of reducing this toxic metabolite in patients with 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. These include, without limitation, statements regarding (i) the timing of our NDA submission 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 rare disease franchise expansion programs 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 will support future development of our product candidates and (v) the likelihood of obtaining regulatory approval of our product candidates. 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.

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Contacts

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

Media:
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Applied Therapeutics, Inc.