Benzinga | Oct 18, 2021 07:14AM EDT

Applied Therapeutics Reports Additional Pediatric Biomarker Data From ACTION-Galactosemia Kids

Substantial and statistically significant reduction in plasma galactitol of 40% 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 pediatric dosing could be further optimized by adjusting dose based on weight rather than age. As such, weight-based dosing brackets were implemented, and a subset of pediatric patients were 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 a dose adjustment. AT-007 substantially reduced plasma galactitol by approximately 40%, which was statistically significant (p<0.001) vs. placebo. Reduction in plasma galactitol was rapid and sustained, with no impact on levels of galactose or Gal-1p, and was similar across dose groups. AT-007 was safe and well tolerated in children of all ages (2-17). The Company plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in Q4 of this year.

As previously announced, an 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 will be presented as a late-breaking abstract at the 14th International Congress on Inborn Errors of Metabolism (ICIEM) November 21-24, 2021.

"We have demonstrated that toxic galactitol drives disease progression and greatly impacts quality of life in Galactosemia patients," said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. "Here we have shown that AT-007 significantly reduces galactitol levels, which we believe could transform the lives of children and adults living with this disease. We are sincerely thankful to all of the families participating in the ACTION-Galactosemia clinical program, who have made this study possible."

"The Commercial team has made great progress in preparing for a possible launch of AT-007 in Galactosemia. Our 'Galactosemia Together' disease state education campaign has generated tremendous interest from the physician 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 are in desperate need of a treatment option," said Adam Hansard, Chief Commercial Officer of Applied Therapeutics.