Atrium Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its investigational new drug (IND) application to begin the Corventis Phase I/II clinical trial of ATR 1072 for Protein Kinase AMP-activated non-catalytic subunit Gamma 2 (PRKAG2) syndrome.
The rare autosomal dominant, early-onset cardiomyopathy is caused due to the PRKAG2 gene mutations.
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The open-label, multi-centre trial will enrol about 37 participants in the US.
Part A of the trial will involve multiple-ascending dose cohorts to assess the safety and determine dosing while Part B will be a single-arm expansion cohort at the selected Phase II dose to evaluate the safety and efficacy trends in cardiac function and structure.
Clinical sites are in the process of being initiated, and the company expects to enrol the first participant by the end of 2026. Initial data demonstrating proof of concept are anticipated in the second half of 2027.
Atrium Therapeutics president and CEO Kathleen Gallagher said: “PRKAG2 syndrome and other rare genetic cardiomyopathies represent a profound unmet need — these are progressive, life-altering and life-threatening diseases that often strike early, affect multiple members of the same family, and have no approved therapy to address their root cause.
“FDA clearance of our IND and the launch of the Corventis Phase I/II trial reinforce our team’s ability to move with speed on behalf of patients with the goal of delivering potential disease-modifying treatments.”
ATR 1072 is Atrium Therapeutics’ lead product candidate and uses small interfering RNA aimed at silencing mutant PRKAG2 messenger RNA to normalise protein kinase activity and reduce harmful glycogen build-up in the heart.
The company’s pipeline also consists of ATR 1086 for PLN (phospholamban) cardiomyopathy, and two research targets in rare cardiomyopathies.
