Our Science
We are advancing the delivery of targeted RNA therapies to the heart for people with rare, genetically driven cardiomyopathies. Current management is limited to symptomatic therapies, and there are little to no approved therapies to treat the underlying causes of these diseases. We believe there is an opportunity to take a precision medicine approach in cardiology targeting the underlying drivers of genetic cardiomyopathies.
Atrium Therapeutics
Our Pipeline
Program/Indication
Target
Research
Ind Enabling
Clinical
Phase 1
Phase 2
Phase 3
Anticipated upcoming milestone
PRKAG2 syndrome
PRKAG2
IND Filing in 2H'26
PLN cardiomyopathy
PLN
IND Filing in 2H’27
Our pipeline includes two lead development candidates: ATR 1072 for PRKAG2 syndrome and ATR 1086 for PLN cardiomyopathy. Both conditions are severe, life-threatening, rare autosomal dominant progressive cardiomyopathies with no approved treatment options and high unmet need. The company has two additional undisclosed research stage targets in rare cardiomyopathies.
ATR 1072
Our lead product candidate, ATR 1072, is a potentially disease-modifying treatment for PRKAG2 syndrome. siRNA works through a sequence-specific gene silencing mechanism to reduce PRKAG2 mRNA, normalize AMPK activity and reduce the pathogenic glycogen accumulation, potentially leading to improved heart function.
We are initially focused on addressing the unmet need in PRKAG2 syndrome, and believe there is potential to address unmet need in broader HCM and heart failure populations given the role of elevated AMPK activity has been observed in human heart failure and preclinical models of cardiac disease.
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 heart muscle cells leading to thickened heart muscles, electrical conduction problems, and arrhythmias. There are 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.
ATR 1086
ATR 1086 is designed to address the underlying genetic driver of disease by reducing PLN expression in cardiac muscle, rather than treating symptoms alone. We believe it has the potential to address a broader population of heart failure and cardiomyopathy patients, particularly those characterized by calcium handling defects and reduced SERCA2a activity.
By selectively reducing PLN expression, ATR 1086 is expected to relieve SERCA2a inhibition and restore calcium cycling dynamics, which may improve cardiac performance. This therapeutic rationale is further supported by human genetic studies, which have identified a common SNP at the PLN locus that upregulates PLN mRNA and is associated with increased heart failure risk. These findings suggest the therapeutic potential for PLN reduction for calcium-driven cardiomyopathies.
About PLN Cardiomyopathy
PLN (phospholamban) cardiomyopathy is a rare autosomal dominant, progressive cardiac disease caused by mutations in PLN, a key regulator of SERCA2a calcium pump. Pathogenic variants produce protein aggregates that disrupt endoplasmic reticulum processes and lead to dilated, arrhythmogenic, or hypertrophic cardiomyopathies and a significantly increased risk of heart failure and sudden cardiac death. There are 2,000 – 4,000 people with pathogenic PLN variants in the US. No approved therapies target the underlying molecular cause of the disease.