Acoramidis Beneficial in Transthyretin Amyloid Cardiomyopathy

THURSDAY, Jan. 11, 2024 (HealthDay News) -- Acoramidis yields a significantly better four-step primary hierarchical outcome, including aspects of mortality and morbidity, than placebo for patients with transthyretin amyloid cardiomyopathy, according to a study published in the Jan. 11 issue of the New England Journal of Medicine.

Julian D. Gillmore, M.D., Ph.D., from University College London, and colleagues conducted a phase 3, double-blind trial involving 632 patients with transthyretin amyloid cardiomyopathy who were randomly assigned in a 2:1 ratio to receive acoramidis hydrochloride (800 mg twice daily) or matching placebo for 30 months. The primary hierarchical analysis included death from any cause, cardiovascular-related hospitalization, and the changes from baseline in the N-terminal pro-B-type natriuretic peptide (NT-proBNP) level and 6-minute walk distance.

The researchers found that acoramidis was favored over placebo in the primary analysis, with a corresponding win ratio of 1.8, and 63.7 and 35.9 percent of pairwise comparisons favoring acoramidis and placebo, respectively. More than half the wins and losses to the win ratio were contributed by death from any cause and cardiovascular-related hospitalization together (58 percent of all pairwise comparisons); the highest ratio of wins to losses was yielded by NT-proBNP pairwise comparisons (23.3 versus 7.0 percent). The acoramidis and placebo groups had a similar overall incidence of adverse events (98.1 and 97.6 percent, respectively); serious adverse events occurred in 54.6 and 64.9 percent, respectively.

"These data support the use of acoramidis as an effective and safe treatment option for patients with transthyretin amyloid cardiomyopathy," the authors write.

The study was funded by BridgeBio Pharma, which is developing acoramidis.

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