Announcement • Jul 14

Atrium Therapeutics Announces FDA Clearance Of Investigational New Drug Application For ATR 1072 For Treatment Of PRKAG2 Syndrome

Atrium Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application allowing the Company to move forward with its Corventis Phase 1/2 clinical trial designed to evaluate ATR 1072 for the treatment of Protein Kinase AMP-activated non-catalytic subunit Gamma 2 (PRKAG2) syndrome. Corventis is a Phase 1/2 open-label, multicenter clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of ATR 1072. The study will enroll approximately 37 participants across two parts: Part A, multiple ascending dose cohorts to characterize safety and support dose selection, and Part B, a single-arm expansion cohort at the recommended Phase 2 dose to further evaluate safety and efficacy trends in cardiac structure and function. Clinical site initiation activities are currently underway, and Atrium expects the first participant to be enrolled by the end of 2026. Initial trial data demonstrating proof of concept is anticipated in the second half of 2027. ATR 1072, the company's lead product candidate, is a potentially disease-modifying treatment for PRKAG2 syndrome. Using Atrium's precision RNA delivery technology, ATR 1072 uses small interfering RNA (siRNA) to silence mutant PRKAG2 messenger RNA (mRNA), normalize AMP-activated protein kinase (AMPK) activity and reduce pathogenic glycogen accumulation, potentially leading to improved heart function. PRKAG2 syndrome is a rare, autosomal dominant, early-onset cardiomyopathy caused by mutations in the PRKAG2 gene, which encodes the Gamma 2 regulatory subunit of AMPK. Mutations enhance AMPK activity leading to abnormal glycogen accumulation in the heart, thickened heart muscles, electrical conduction problems, and arrhythmias. Based on current scientific literature estimates, there are at least 1,000 – 2,000 people with PRKAG2 syndrome in the US. Current management is limited to symptomatic treatment; no approved therapies exist to address the underlying genetic driver of disease. Corventis is a Phase 1/2 open-label, multicenter clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of ATR 1072 in participants living with PRKAG2 syndrome. The study will enroll approximately 37 participants across two parts: Part A, multiple ascending dose cohorts to characterize safety and support dose selection, and Part B, a single-arm expansion cohort at the recommended Phase 2 dose to further evaluate efficacy trends in cardiac structure and function.

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