SAN DIEGO, Calif. — Atrium Therapeutics, Inc. (Nasdaq: RNA) (the “Company”), a biopharmaceutical company dedicated to delivering RNA therapeutics to the heart, announced today 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.
“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,” said Kathleen Gallagher, President and Chief Executive Officer, Atrium Therapeutics. “FDA clearance of our IND and the launch of the Corventis™ Phase 1/2 trial reinforce our team’s ability to move with speed on behalf of patients with the goal of delivering potential disease-modifying treatments.”
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.
About ATR 1072
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.
About PRKAG2 Syndrome
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.
About the Corventis™ Trial
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. Additional information about the trial is available at www.corventistrial.com.
About Atrium Therapeutics
Atrium Therapeutics, Inc. (Nasdaq: RNA) is pioneering targeted delivery of ribonucleic acid (RNA) therapeutics to the heart to transform the standard of care for people living with cardiomyopathies. The Company’s proprietary technology – designed at Avidity Biosciences, Inc. – combines the tissue selectivity of monoclonal antibodies (mAbs) and other targeted delivery ligands with the precision of oligonucleotides. Atrium Therapeutics’ platform is designed to selectively target the underlying drivers of genetically driven cardiac diseases through targeted, non-viral delivery of small interfering RNA (siRNA). This approach builds upon learnings from demonstrated delivery to the skeletal muscle and applies it for efficient delivery to the heart with the potential to overcome challenges associated with non-specific tissue delivery. The Company’s pipeline consists of two precision cardiology candidates, ATR 1072 for PRKAG2 (Protein Kinase AMP-activated non-catalytic subunit Gamma 2) syndrome and ATR 1086 for PLN (phospholamban) cardiomyopathy, and two undisclosed research targets in rare cardiomyopathies.
For more information about our RNA delivery platform, development pipeline and people, please visit https://atriumtherapeutics.com/ and engage with us on LinkedIn.
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