BridgeBio Pharma (BBIO) said real-world data showed its heart drug acoramidis was associated with better outcomes than tafamidis, the established treatment for transthyretin amyloid cardiomyopathy, a progressive and often fatal heart condition.
The findings, presented at the Heart Failure Society of America’s annual meeting in Phoenix, drew on Epic COSMOS, the largest US electronic health records database, which covers more than 300mn patients. After matching, the analysis compared 910 patients taking acoramidis with 1,774 taking tafamidis.
Acoramidis was associated with an 18 per cent lower risk of a composite of death, all-cause hospitalization, new-onset atrial fibrillation and diuretic intensification. Risks of cardiovascular and heart failure hospitalization were each 27 per cent lower, and the risk of all-cause hospitalization was 25 per cent lower. Differences in death and atrial fibrillation favored acoramidis but were not statistically significant.
“While electronic health record studies have well-recognized limitations, these data allow us to see how these therapies perform outside of a clinical trial,” said David Lanfear, chief scientific officer and vice-president of research at Henry Ford Health, who led the analysis. He added that further studies were warranted.
BridgeBio said the results echoed an earlier claims-based analysis that found a 34 per cent reduction in clinical worsening events with acoramidis versus tafamidis. The company acknowledged the limits of observational research, including the possibility of residual confounding and short follow-up, which restricted the number of events available to assess outcomes such as mortality. The full results are under peer review.
The company also presented further analyses of its Phase 3 ATTRibute-CM trial. In a post hoc analysis, mortality risk roughly doubled after a single cardiovascular hospitalization and quadrupled after two or more, which BridgeBio said underscored the importance of preventing admissions. Acoramidis reduced the annualized frequency of such hospitalizations by 50 per cent through month 30 compared with placebo.
Other analyses pointed to benefits in patients with the p.Val142Ile gene variant and in women, though both subgroups were small. Acoramidis is sold as Attruby in the US and Beyonttra in Europe.