Applied Therapeutics Inc. announced positive topline results from the Pivotal Phase 2 portion of the ACTION-Galactosemia study of AT-007, a central nervous system (CNS) penetrant Aldose Reductase inhibitor, in adult Galactosemia patients. ACTION-Galactosemia is a double-blind placebo-controlled trial evaluating safety and pharmacokinetics of AT-007 in healthy volunteers, as well as safety, pharmacokinetics, and biomarker effects in adult Galactosemia patients over 28 days of once daily oral dosing. The key biomarker outcome of the study was reduction in galactitol, an aberrant toxic metabolite of galactose, formed by Aldose Reductase in Galactosemia patients. AT-007 treatment resulted in a statistically significant and robust reduction in plasma galactitol vs placebo in adult Galactosemia patients. Reductions in galactitol were dose dependent, with higher concentrations of AT-007 resulting in a greater magnitude of reduction in galactitol. At the higher dose tested (20mg/kg), AT-007 significantly reduced plasma galactitol 45-54% from baseline vs. placebo (p<0.01). Galactitol reduction was rapid and sustained over time. No substantial change from baseline was observed in placebo treated patients. AT-007 was well tolerated, with no drug-related adverse events noted to date in Galactosemia patients or in the 72 healthy volunteers treated in Part 1 of the trial. Applied Therapeutics will continue to characterize AT-007 long-term safety in adult Galactosemia patients and intends to initiate a pediatric study in 2020. The company plans to utilize recent FDA guidance permitting biomarker-based development in low prevalence, slowly progressing rare metabolic diseases, such as Galactosemia, and expects to file for regulatory approval in the second half of 2020.