Applied Therapeutics announces results from 12-month interim analysis of AT-007

Applied Therapeutics announced positive interim 12-month results from the ongoing Phase 3 INSPIRE trial, in which the primary endpoints and several key secondary endpoints were achieved. The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily oral govorestat in 56 patients aged 16-55 with SORD Deficiency in the US and Europe. Interim Analysis Results: Demonstrated statistically significant correlation between sorbitol level and the prespecified CMT-FOM composite clinical endpoint. Govorestat treatment provided sustained reduction in sorbitol level in patients with SORD Deficiency over 12 months of treatment, which was statistically significant compared to placebo. Govorestat treatment also resulted in a highly statistically significant effect on the CMT Health Index, an important patient-reported outcome measure of disease severity and well-being, which was a secondary endpoint in the study. Govorestat was safe and well tolerated, with similar incidence of adverse events between active and placebo-treated groups.