BridgeBio reports results from Phase 3 study of ATTR-CM

BridgeBio Pharma reports positive results from ATTRibute-CM, its Phase 3 study of acoramidis in transthyretin amyloid cardiomyopathy, or ATTR-CM. ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin. Key results from the clinical trial include: A highly statistically significant improvement in the primary endpoint demonstrated by a Win Ratio of 1.8. An 81% on-treatment survival rate, which begins to approach actuarial models of life expectancy absent ATTR-CM. The absolute risk reduction was 6.43% and the relative risk reduction was 25%. A highly statistically significant relative risk reduction of 50% on frequency of cardiovascular-related hospitalization. The impact and marked magnitude of risk reduction was seen across all analytical methods employed. The Company consistently observed a statistically significant treatment effect at 30 months across additional measured markers of morbidity, quality of life, and function: Change from baseline in N-terminal prohormone of brain natriuretic peptide; Change from baseline in Kansas City Cardiomyopathy Questionnaire; Change from baseline in 6-minute walk distance; No safety signals of potential clinical concern were identified. A key objective in the rational drug design of acoramidis was to maximize TTR stabilization at clinically achieved blood concentrations. Several lines of evidence suggest that maximizing stabilization could lead to improved benefits for ATTR patients: Historical ATTR genotype/phenotype data and the disease-protective properties of trans-allelic, trans-suppressor variants relative to pathogenic variants in compound heterozygotes and the general, nonvariant population; The outperformance of 80mg tafamidis vs 20mg tafamidis in the previously published ATTR-ACT trial; Results from ATTR-polyneuropathy clinical trials