The Corventis Phase 1/2 study will enroll about 37 participants, with first enrollment expected by the end of 2026 and initial proof-of-concept data anticipated in the second half of 2027.
Atrium Therapeutics said the FDA (U.S. Food and Drug Administration) cleared its Investigational New Drug application for ATR 1072, allowing the company to begin the Corventis Phase 1/2 trial in PRKAG2 syndrome, a rare inherited cardiomyopathy with no approved therapy aimed at the underlying cause. The open-label, multicenter study is designed to assess safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy, and is expected to enroll about 37 participants in two parts, including multiple ascending dose cohorts and a single-arm expansion cohort at the recommended Phase 2 dose. Atrium said site initiation is underway, with the first participant expected by the end of 2026 and initial proof-of-concept data anticipated in the second half of 2027. The company describes ATR 1072 as its first precision cardiology program to enter the clinic and says it uses siRNA (small interfering RNA) to silence mutant PRKAG2 mRNA (messenger RNA), normalize AMPK activity and reduce pathogenic glycogen accumulation in the heart.