BridgeBio Pharma released data from its Phase 3 ATTRibute-CM trial of acoramidis, showing a link between early, sustained increases in serum transthyretin (TTR) levels and improved survival in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The study, published in the •Journal of the American College of Cardiology•, revealed that for every 5 mg/dL increase in serum TTR within 28 days of starting acoramidis, the relative risk of mortality decreased by up to 31.6% over 30 months. This suggests that higher TTR stabilization achieved with acoramidis correlates with better clinical outcomes.
This research is crucial for understanding and treating ATTR-CM, a progressive and often fatal disease. The findings establish a direct correlation between rapid TTR stabilization and improved survival, potentially offering a new prognostic biomarker for the disease. This could lead to more informed treatment decisions, allowing physicians to select therapies that maximize TTR stabilization early in the course of the disease and potentially improving patient outcomes. The identification of an early biomarker is especially valuable in ATTR-CM due to the disease’s progressive nature.
The ATTRibute-CM study showed a rapid and substantial average increase of 9.1 mg/dL in serum TTR levels within 28 days of starting acoramidis, an effect sustained throughout the 30-month trial period. Further analysis demonstrated that this early TTR increase was independently associated with reduced mortality, regardless of other risk factors like TTR variant status or disease severity. 42-month data from the open-label extension study reinforced these findings, demonstrating statistically significant reductions in all-cause mortality and cardiovascular-related hospitalizations.
This research strengthens the case for acoramidis as a treatment for ATTR-CM. The ability to rapidly and sustainably increase TTR levels, coupled with the correlation between these increased levels and survival, positions acoramidis as a potentially impactful therapy. Furthermore, the identification of serum TTR as a prognostic biomarker could shift the paradigm in ATTR-CM treatment, emphasizing early intervention and TTR stabilization as key therapeutic goals. This could stimulate further research into TTR-stabilizing therapies and refine treatment strategies for this serious condition.
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.
