BridgeBio Pharma, Inc. (BBIO) presented compelling post-hoc analysis results from its Phase 3 ATTRibute-CM study, highlighting the efficacy of acoramidis in treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The data, shared at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2024, showed a significant reduction in all-cause mortality (ACM) and recurrent cardiovascular-related hospitalizations (CVH) events in patients treated with acoramidis compared to placebo.
The analysis revealed a 42% reduction in the composite endpoint of ACM and recurrent CVH events at 30 months with acoramidis treatment. This reduction was observed using both a negative binomial regression model and a per-patient analysis. Additionally, the Andersen-Gill model demonstrated a 30.5% hazard reduction in ACM and recurrent CVH events at 30 months with acoramidis treatment.
These findings underscore the potential of acoramidis as a first-line therapy for ATTR-CM. Dr. Daniel Judge, a professor of medicine and cardiology at the Medical University of South Carolina, emphasized that “this post-hoc analysis provides further evidence that near-complete TTR stabilization with acoramidis can improve clinical outcomes for patients with ATTR-CM.” He further highlighted the potential of acoramidis to enhance quality of life for patients by reducing hospitalizations and all-cause mortality.
ATTR-CM is a rare and serious heart condition caused by the accumulation of abnormal transthyretin (TTR) proteins in the heart’s main pumping chamber. This accumulation leads to impaired heart function and can significantly affect a patient’s quality of life.
Based on the promising results from the ATTRibute-CM study, BridgeBio submitted an FDA marketing application, which has been accepted with a PDUFA action date of November 29, 2024. The company also submitted a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025.
The positive results from the study have been well-received by investors, with BBIO stock rising 2.39% to $25.54 at last check on Monday. This development could pave the way for acoramidis to become a critical treatment option for patients suffering from ATTR-CM, offering hope for a better future for those affected by this debilitating condition.
