Key Takeaways:
BridgeBio's acoramidis showed potential reversal of cardiac disease progression in ATTR-CM, with patients gaining 65 extra days alive and out of hospital.
"The clinical community is excited about the potential to restore heart health found in these data," Marianna Fontana, M.D. of University College London, said. "For a long time, patients living with ATTR-CM could only hope for a stop to the otherwise relentless progression of disease."
The Phase 3 ATTRibute-CM cardiac magnetic resonance imaging substudy and its open-label extension provided the first evidence from serial CMR that a therapy can reverse disease progression through Month 42. Roughly half of patients showed improved left ventricular systolic function in the completer analysis, more than double the proportion seen in a National Amyloidosis Centre natural-history cohort or in placebo-treated participants. A post-hoc analysis found acoramidis preserved significantly more time alive outside the hospital, with patients gaining 65 additional days by Month 36 versus baseline placebo patients.
The findings were simultaneously published in the European Journal of Heart Failure. Long-term data through Month 54 showed continued benefit across 56 variant ATTR-CM patients, including 35 with the p.Val142Ile variant, the most common ATTR-CM genetic variant globally, which carries a 3-4 percent carrier frequency in the U.S. Black population. Safety was consistent with prior studies: diarrhea occurred in 11.6 percent of treated patients versus 7.6 percent on placebo, and discontinuation rates were similar at 9.3 percent and 8.5 percent.
Acoramidis, a near-complete (at least 90 percent) transthyretin stabilizer, is approved as Attruby by the U.S. FDA and as BEYONTTRA by the European Medicines Agency, Japan's Pharmaceuticals and Medical Devices Agency, Swissmedic, the UK Medicines and Healthcare Products Regulatory Agency, and Brazil's ANVISA. The data strengthen BridgeBio's position against Pfizer's tafamidis and Alnylam's vutrisiran in the ATTR-CM market, where the treatment approach has long centered on slowing decline rather than restoring heart function.
BridgeBio has also begun dosing patients in ASCEND-ATTR, a Phase 3b/4 study designed to characterize long-term effects of acoramidis on cardiac structure, function, and amyloid burden in about 150 participants. Separately, the company is working with Yale's Cardiovascular Data Science Lab on AI networks for earlier ATTR-CM detection.
Wall Street analysts have set a median price target of $100 on BridgeBio shares, with one buy rating in recent months. The company plans to present additional Attruby data at the Heart Failure Society of America Annual Scientific Meeting in Phoenix on Oct. 9-12, 2026.
This article is for informational purposes only and does not constitute investment advice.
