BridgeBio Pharma presented data from a subgroup analysis of its Phase 3 ATTRibute-CM trial, demonstrating acoramidis significantly improved clinical outcomes for both variant (ATTRv) and wild-type (ATTRwt) transthyretin amyloid cardiomyopathy (ATTR-CM) patients. The analysis showed a 59% risk reduction for all-cause mortality or first cardiovascular-related hospitalization in ATTRv-CM patients and a 31% risk reduction in ATTRwt-CM patients. Acoramidis, an orally administered transthyretin stabilizer, achieved near-complete (≥90%) TTR stabilization in both patient subgroups.
This news is particularly important for ATTRv-CM patients, who typically experience earlier onset and more rapid disease progression compared to ATTRwt-CM patients. The significant risk reduction observed with acoramidis represents a substantial advancement in treatment options for this patient population with previously limited therapeutic choices. The rapid onset of benefit, observed within three months, further underscores the potential of acoramidis to meaningfully alter the course of the disease.
The data presented highlighted the impact of acoramidis on several key clinical measures. The drug showed a 42% reduction in the composite endpoint of all-cause mortality and recurrent cardiovascular hospitalizations at 30 months. Additionally, a 50% reduction in cumulative cardiovascular hospitalization frequency was observed at 30 months. Further analyses demonstrated improved NYHA class, indicating better heart failure symptom stabilization, and confirmed that the addition of tafamidis to acoramidis provided no additional benefit. These data solidify the efficacy of acoramidis as a monotherapy for ATTR-CM.
The positive results from the ATTRibute-CM subgroup analysis, coupled with existing approvals in the US, Europe, and Japan, position acoramidis as a leading treatment option for ATTR-CM. This advancement in TTR stabilization therapy offers new hope for patients, particularly those with ATTRv-CM, and paves the way for further research into its long-term benefits and potential use in earlier stages of the disease. The results also underscore the importance of continued research and development of targeted therapies for genetic diseases.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
