Atrium Therapeutics Cleared to Begin Human Trials for PRKAG2 Syndrome

The Corventis™ trial aims to enroll approximately 37 participants to evaluate the safety, tolerability, and efficacy of the drug. The study is divided into two segments: an initial phase to establish safety and optimal dosing, followed by an expansion cohort to monitor changes in cardiac structure and function. Atrium anticipates beginning patient enrollment by the end of 2026, with preliminary proof-of-concept data expected in the second half of 2027.

PRKAG2 syndrome is a rare, life-threatening genetic cardiomyopathy that causes abnormal glycogen accumulation in the heart, leading to thickened muscle walls and severe arrhythmias. Currently, patients have no approved therapies to address the genetic source of the disease, leaving them with only symptomatic treatment options. ATR 1072 uses small interfering RNA to silence mutant mRNA, potentially normalizing protein kinase activity and preventing disease progression. Kathleen Gallagher, CEO of Atrium Therapeutics, noted that the clearance allows the company to accelerate development for a condition characterized by significant unmet medical needs.