Atrium Therapeutics (RNA) Investor presentation summary

Vision and strategy

• Aims to pioneer precision RNA medicines for cardiac diseases, focusing on genetically driven cardiomyopathies.

• Leverages a proprietary RNA delivery platform designed at Avidity Biosciences for targeted therapies.

• Focused pipeline includes two lead programs, PRKAG2 and PLN, with near-term clinical milestones.

• Addresses significant unmet medical need, as millions with genetic cardiomyopathies lack disease-modifying treatments.

• Platform is scalable to broader cardiac diseases beyond rare genetic cardiomyopathies.

Technology and platform

• Utilizes Antibody Oligonucleotide Conjugate (AOC) technology for efficient RNA delivery to heart tissue.

• Designed to directly target pathogenic RNA, potentially modifying disease progression.

• Platform can be expanded to multiple genetically defined cardiomyopathies and additional indications.

Lead programs and pipeline

• ATR 1072 targets PRKAG2 syndrome, a rare, progressive disease leading to sudden cardiac death; no current therapies address the root cause.

• ATR 1072 silences mutant PRKAG2 to normalize AMPK activity, showing normalization of cardiac function in preclinical studies.

• IND filing for ATR 1072 planned for 2H 2026, with Phase 1 trial initiation to follow.

• ATR 1086 targets PLN cardiomyopathy, another rare disease with high risk of sudden death and no approved therapies.

• ATR 1086 silences mutant PLN to prevent protein aggregates, demonstrating improved survival and cardiac function in preclinical models.

• IND-enabling studies for ATR 1086 to begin in 2026, with IND filing anticipated in 2027.