Atrium Therapeutics, Inc. announced on October 27, 2026, that the first patient has been dosed in the Phase 1/2 Corventis clinical trial. The trial is evaluating ATR 1072, the company's lead investigational therapy, for the potential treatment of Protein Kinase AMP-activated non-catalytic subunit Gamma 2 (PRKAG2) syndrome.
PRKAG2 syndrome is a rare, autosomal dominant, early-onset cardiomyopathy caused by genetic mutations in the PRKAG2 gene. These mutations lead to overactivity of AMP-activated protein kinase (AMPK), resulting in glycogen accumulation in heart muscle, thickened heart muscles, and electrical conduction problems. The company estimates there are between 1,000 and 2,000 people with the syndrome in the United States. Currently, there are no approved therapies to address the underlying genetic driver of the disease, and management is limited to symptomatic treatment.
The Corventis trial is an open-label, multicenter study designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ATR 1072. The study will enroll approximately 37 participants across two parts. Part A involves multiple ascending dose cohorts to characterize safety and support dose selection, while Part B is a single-arm expansion cohort at the recommended Phase 2 dose to further evaluate efficacy trends in cardiac structure and function.
ATR 1072 is an antibody oligonucleotide conjugate (AOC) designed to silence mutant PRKAG2 messenger RNA (mRNA) to normalize AMPK overactivation. The company expects to report initial proof-of-concept data from the Corventis trial in the second half of 2027.
