Applied Therapeutics announces topline results of ARISE-HF Phase 3 of AT-001

Applied Therapeutics announced the topline results of the ARISE-HF Phase 3 trial of AT-001, caficrestat, in patients with Diabetic Cardiomyopathy or DbCM, at high risk of progression to overt heart failure. The primary endpoint of the study was stabilization or improvement in cardiac functional capacity as measured by Peak VO2 in patients treated with AT-001 1500mg twice daily, BID, as compared to placebo. The placebo-treated group declined by a mean of -0.31 ml/kg/min over 15 months of treatment, while the AT-001 1500mg BID group remained primarily stable, with a mean change of -0.01 ml/kg/min over 15 months. While a trend favored active treatment, the difference between active and placebo treated groups was not statistically significant .”AT-001 stabilized cardiac functional capacity as compared to placebo, and prevented clinically significant worsening of disease, an effect which was strengthened in patients not on concomitant treatment with an SGLT2 or GLP-1,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “Given its favorable safety and tolerability profile and oral dosing, we believe that AT-001 represents an important potential tool for physicians in treatment of DbCM patients. We thank the patients and families who participated in the ARISE-HF study and made this important work possible.”Given these encouraging results, the Company plans to focus on identifying an appropriate path forward through partnering in order to bring AT-001 to DbCM patients. Current resources are expected to be focused on the development, regulatory and commercial preparations for the govorestat rare disease program. The Company submitted a New Drug Application NDA to the U.S. Food and Drug Administration FDA in December 2023 for govorestat for the treatment of Classic Galactosemia. The Marketing Authorization Application was validated and accepted for review by the European Medicines Agency in December 2023.