Applied Therapeutics eyes galactosemia submission based on biomarker data

Applied Therapeutics believes it can submit an NDA for its lead program to FDA based on new biomarker data from a Phase I/II trial, citing 2018 draft guidance from the agency on slowly progressing metabolic disorders.

The company said Wednesday that AT-007, its CNS-penetrant aldose reductase inhibitor, significantly reduced plasma galactitol vs. placebo in adults with galactosemia. At the highest dose in the trial’s Phase II portion, the reductions were 45-54% from baseline (p<0.01 vs. placebo)...