Atrium Therapeutics (RNA) Investor presentation summary

Vision and strategy

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

• Utilizes a proprietary RNA delivery platform designed for targeted, efficient delivery to heart tissue.

• Pipeline includes two lead programs (ATR 1072 for PRKAG2 syndrome, ATR 1086 for PLN cardiomyopathy) and two undisclosed targets.

Pipeline and clinical progress

• Achieved IND clearance for ATR 1072 and announced the Corventis Phase 1/2 trial for PRKAG2 syndrome.

• ATR 1072 targets PRKAG2 syndrome, a rare, progressive disease with no approved therapies, aiming to normalize AMPK activity and attenuate disease progression.

• ATR 1086 targets PLN cardiomyopathy, designed to silence mutant PLN and prevent protein aggregates, addressing the root cause.

• Both programs demonstrated functional improvement and safety in preclinical models.

• First patient in Corventis Phase 1/2 trial expected by end of 2026, with proof-of-concept data anticipated in 2H 2027.

Market opportunity and patient identification

• Millions with genetic cardiomyopathies lack disease-modifying treatments; only ~1% receive genetic testing.

• PRKAG2 syndrome prevalence likely exceeds initial estimates, with early efforts identifying close to 2,000 individuals.

• Focused patient identification strategy includes partnerships with genetic testing labs and patient advocacy.