AT-007 demonstrated a robust and sustained reduction in galactitol vs placebo; significant plasma galactitol reduction of ~50% (p<0.01)
AT-007 was well-tolerated; no drug-related adverse events reported at any dose to date
Applied Therapeutics expects to file for regulatory approval of AT-007 in the second half of 2020
Company to host conference call and webcast today at 8:30 a.m. ET
NEW YORK, Jan. 08, 2020 (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 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.