Applied Therapeutics announces data from ongoing Phase 3 INSPIRE trial

Applied Therapeutics announced positive sorbitol reduction data from the ongoing global Phase 3 INSPIRE trial. The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily oral AT-007 in approximately 50 patients age 16-55 with SORD Deficiency in the US and Europe. In a pre-specified interim analysis of the ongoing Phase 3 INSPIRE trial, AT-007 reduced sorbitol levels by a mean of approximately 52% over 90 days of treatment in patients with SORD Deficiency. At baseline, the mean blood sorbitol level of SORD patients was approximately 29,000ng/ml, with a range of approximately 22,000ng/ml-38,000ng/ml. In the INSPIRE trial, a baseline cross-sectional analysis of the relationship between sorbitol level, age and clinical outcome measures demonstrated a statistically significant correlation between sorbitol level and key clinical outcome measures, including 10-meter-walk/run speed, 4-stair climb speed, and sit-to-stand test. Taken together with the biology of the disease and strong supportive animal model data, the Company believes that compelling data exists demonstrating that sorbitol reduction is reasonably likely to predict effect on clinical outcomes over time. The Company is working with the FDA to determine the appropriate regulatory path forward, as well as data required for an NDA submission, with the shared goal of bringing a safe and effective treatment to patients with SORD Deficiency as expeditiously as possible.