San Diego – October 29, 2026 -- Atrium Therapeutics (Nasdaq: RNA) has dosed the first patient in Corventis, a Phase 1/2 trial of ATR 1072 in PRKAG2 syndrome, and expects initial proof-of-concept data in the second half of 2027.
Corventis will enroll about 37 participants in two parts
The open-label, multicenter study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of ATR 1072. Part A uses multiple ascending dose cohorts to characterize safety and support dose selection. Part B is a single-arm expansion cohort at the recommended Phase 2 dose, assessing efficacy trends in cardiac structure and function.
The primary endpoint is the incidence of treatment-emergent adverse events. Secondary and exploratory endpoints include PRKAG2 mRNA reduction, siRNA tissue concentrations and plasma pharmacokinetics.
ATR 1072 uses siRNA to silence mutant PRKAG2 mRNA
ATR 1072 is an antibody oligonucleotide conjugate built on Atrium's targeted RNA delivery platform. It is designed to normalize overactivation of AMP-activated protein kinase (AMPK), reduce pathogenic glycogen accumulation and improve heart function. The platform was designed at Avidity Biosciences and combines the tissue selectivity of monoclonal antibodies with oligonucleotides, using non-viral delivery of siRNA.
PRKAG2 syndrome has no approved therapies and affects at least 1,000 to 2,000 people in the US
The prevalence figure is based on current scientific literature estimates. The rare, autosomal dominant cardiomyopathy stems from PRKAG2 mutations that drive AMPK overactivity. The resulting glycogen buildup in heart muscle may lead to heart failure and sudden cardiac death.
Current care is limited to symptomatic management. Michael Nassif, M.D., a heart failure cardiologist at Saint Luke's Mid America Heart Institute, said clinicians "manage the consequences of the disease, not the disease itself," relying on pacemakers and defibrillators.
CEO Kathleen Gallagher called Corventis the first clinical trial in PRKAG2 syndrome to evaluate a potential treatment for the underlying cause of the disease.
Atrium's pipeline also includes ATR 1086 for PLN (phospholamban) cardiomyopathy and two undisclosed research targets in rare cardiomyopathies.