CMR Imaging Supports ATTRibute-CM Study's Positive Clinical Outcomes in ATTR-CM Patients

BridgeBio Pharma, a biopharmaceutical firm, has reported positive findings from a substudy of its Phase 3 ATTRibute-CM trial, which evaluated acoramidis, a novel TTR stabilizer. The study, presented at the ACC Annual Scientific Sessions, suggests that acoramidis could lead to structural and functional heart improvements in patients with ATTR-CM. The CMR imaging substudy showed that acoramidis might help in cardiac remodeling and recovery, with key imaging parameters indicating stability or improvement in cardiac structure over 30 months, compared to a deterioration in the placebo group. Additionally, the treatment preserved or showed a trend towards enhancing cardiac function measures.

The results are consistent with the positive outcomes from BridgeBio’s global ATTRibute-CM Phase 3 trial, which met its primary endpoint with high statistical significance. Acoramidis was found to be well-tolerated, with no significant safety concerns. Following these results, BridgeBio has submitted a New Drug Application to the FDA, accepted with a PDUFA action date set for late November 2024, and a Marketing Authorization Application to the EMA, with a decision anticipated in 2025.

Marianna Fontana, a Professor of Cardiology, emphasized the potential of acoramidis to induce cardiac amyloid regression in some patients, a novel observation in controlled clinical trials. The CMR imaging was conducted on 52 participants, with readings taken at the National Amyloidosis Centre, blinded to other clinical information.

BridgeBio, founded in 2015, is dedicated to developing genetic medicine to rapidly assist patients with genetic diseases and cancers. The company's pipeline includes programs from early science to advanced clinical trials.