Atrium Therapeutics (RNA) Stifel 2026 Healthcare Conference 2026 summary

Vision and market opportunity

• Focus on precision RNA medicines for genetic cardiomyopathies, where RNA delivery to the heart has historically been a challenge.

• U.S. cardiomyopathy affects ~2M people; ~1M cases are estimated to be genetic, but only 1% of patients are genetically tested.

• PRKAG2 symptoms often begin in the late teens or early 20s; phospholamban cardiomyopathy commonly emerges in patients’ 30s and 40s.

• Current care is largely symptomatic; initial programs aim to address disease causes and potentially slow progression or reverse disease effects.

Platform and pipeline

• Platform uses a monoclonal antibody and linker from prior neuromuscular programs, with chemically modified oligos designed for greater potency and durability; preclinical studies showed heart delivery.

• Antibody targets transferrin receptor; programs are administered by IV infusion every six weeks, with siRNA delivered to the heart.

• Pipeline includes ATR-1072 for PRKAG2 syndrome, ATR-1086 for phospholamban cardiomyopathy, and two additional rare cardiomyopathy programs.

• Research is assessing indication expansion across rare cardiomyopathies tied to AMPK and calcium-handling pathways; the first additional program is targeted for development-candidate selection in 2027.

ATR-1072 and Corventis study

• Corventis cleared to proceed over summer 2026 and dosed its first patient in the weeks before the conference; the phase I/II study is evaluating PRKAG2 syndrome.

• Dose escalation uses flat 40 mg, 80 mg and 160 mg doses, followed by a long-term extension to identify a phase II dose and an expansion cohort of 25 participants.

• Primary endpoint is safety; secondary and exploratory measures include cardiac delivery and target knockdown, echocardiograms, ECGs and arrhythmias.

• Initial proof-of-concept data are expected in H2 2027, with focus on heart delivery, target knockdown and safety; enrollment and site activation are ongoing.

• Preclinical mouse data showed cardiac glycogen levels near control, improved PR interval and reduced wall thickness; monkey studies showed knockdown, and chronic toxicology had no dose-limiting toxicity.