BridgeBio Pharma Inc. has initiated dosing in the ASCEND-ATTR trial, a Phase 3b/4 study evaluating the long-term effects of acoramidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
The study will enroll approximately 150 adults with either wild-type or variant transthyretin-mediated amyloidosis, monitoring cardiac structure, function, and amyloid burden over 36 months. Key assessments include cardiovascular magnetic resonance and cardiac echocardiography at baseline, Month 12, Month 24, and Month 36, with the primary efficacy endpoint defined as responder status at the final interval based on improvements in left ventricular systolic function.
Acoramidis, marketed as Attruby, is being evaluated against a placebo in the trial. Adverse events reported in prior clinical data included diarrhea in 11.6% of acoramidis-treated patients versus 7.6% on placebo, and upper abdominal pain in 5.5% versus 1.4%, respectively. Discontinuation rates due to adverse events were 9.3% for acoramidis and 8.5% for placebo.
The trial follows BridgeBio’s presentation of acoramidis data at the European Society of Cardiology Congress, where the company highlighted the drug’s potential in addressing ATTR-CM, a progressive and fatal condition. BridgeBio’s shares closed at $80.40 on August 25, with pre-market activity showing a 5.41% rise to $84.75.
