Atrium Therapeutics (RNA) Investor presentation summary

Strategic positioning and unmet need

• Focused on precision RNA medicines for genetic cardiomyopathies, where disease-modifying therapies are lacking and care is largely symptom-focused.

• ~2 million people in the U.S. are diagnosed with genetic cardiomyopathies; the presentation estimates only ~1% receive genetic testing.

• PRKAG2 and PLN cardiomyopathies have no approved therapies targeting their underlying causes; U.S. patient estimates are 1,000–2,000 for PRKAG2 and 2,000–4,000 for pathogenic PLN variants.

Platform and pipeline

• Antibody oligonucleotide conjugate platform is designed to deliver RNA therapies to heart tissue and directly target pathogenic RNA across genetically defined cardiomyopathies.

• Lead programs are ATR 1072 for PRKAG2 syndrome and ATR 1086 for PLN cardiomyopathy, alongside two undisclosed rare cardiomyopathy targets.

• Potential expansion pathways include AMPK dysregulation across genetic cardiomyopathies and calcium cycling beyond PLN cardiomyopathy.

ATR 1072 and PRKAG2 syndrome

• ATR 1072 is designed to reduce mutant PRKAG2 RNA and normalize AMPK activity; early lab engagement identified close to 2,000 people with a PRKAG2 mutation from a subset of labs.

• In a nonclinical model, a single 3 mg/kg surrogate dose reversed cardiac glycogen accumulation; other reported findings included reduced heart weight and wall thickness and improved ECG conduction measures.

• In cynomolgus monkeys, ATR 1072 was generally well tolerated in a 9-month repeat-dose study, with no dose-limiting toxicity at the highest dose tested.

• Corventis Phase 1/2 is an open-label adaptive trial with ~37 participants planned across ~15 global sites; initial proof-of-concept data are expected in 2H 2027.