Riccardo Perfetti, MD, PhD: AT-001 for Diabetic Complications

While the fields of endocrinology and cardiometabolic health have seen incredible strides in therapies and technologies in recent years, the treatment of diabetic cardiomyopathy remains a serious challenge. 


However, results of a new study presented at the American Diabetes Association’s (ADA) 80th Scientific Sessions suggests an aldose reductase inhibitor named AT-001 could one day provide a therapy option to patients with diabetic cardiomyopathy.




“This year at ADA we are pleased to present comprehensive data on our aldose reductase inhibitor program, including technological advancements that enabled us to overcome off-target safety and efficacy hurdles of the past, and mechanistic data on the role of aldose reductase toxicity during hyperglycemia,” said Riccardo Perfetti, MD, PhD, chief medical officer of Applied Therapeutics, in a statement.

In an effort to assess the safety and tolerability of the next generation aldose reductase inhibitor, investigators assessed the agent in a phase 1/2, 3-part, randomized, placebo-controlled trial in 120 adults with type 2 diabetics.

During the 28-day trial, all doses and all dosing regimens were well-tolerated by participants. No treatment-related serious adverse events and no adverse events requiring discontinuation of study treatment were observed during the study. Additionally, no clinically relevant changes in liver or kidney function were recorded.

Based on the results of the study and others, investigators hope to further explore AT-001 in other diabetes complications, including the phase 2/3 ARISE-HF (NCT04083339) study.

To learn more about AT-001 and its potential, HCPLive® invited Perfetti to take part in a special edition ADA 2020 House Call.

This study, “Safety and Tolerability of the Next Generation Aldose Reductase Inhibitor (ARI) AT-001 Supports Initiation of the Pivotal Trial in Diabetic Cardiomyopathy (DbCM),” was presented at ADA 2020.