BridgeBio’s heart drug falls short in pivotal study

Dec. 28, 2021

BridgeBio Pharma announced that a phase 3 study investigating its drug candidate, acoramidis, for the treatment of a rare heart disease failed to meet its primary endpoint at month 12.

California-based BridgeBio revealed topline results from month 12 (part A) of ATTRibute-CM, an ongoing global phase 3 study investigating acoramidis for the treatment of symptomatic transthyretin amyloid cardiomyopathy, a cause of heart failure in older adults. Acoramidis (AG10) is an orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting the series of molecular events that give rise to TTR amyloidosis.

The drugmaker sets its sights on acoramidis besting Pfizer’s similarly acting blockbuster, Vyndaqel, approved by the FDA in 2019.

CEO and founder Neil Kumar called the trial results "disappointing and baffling." The drugmaker attributed the failure to a surprisingly strong placebo response.

All is not lost though — BridgeBio says there is still potential for acoramidis to demonstrate benefit at the month 30 endpoint, which includes all-cause mortality and cardiovascular hospitalizations.