In the intricate dance of scientific exploration, BridgeBio has taken a resolute plunge into the depths of phase 3 acoramidis data, revealing new insights into its potential as a treatment for transthyretin amyloid cardiomyopathy (ATTR-CM). The unveiled results, presented at the European Society of Cardiology, shine a spotlight on acoramidis’ remarkable capacity to curtail cardiovascular-related deaths, marking a significant stride in the realm of cardiovascular care.
The freshly disclosed data, unveiled on a Sunday, radiate a ray of hope. Acoramidis exhibited a striking 30% reduction in relative risk for cardiovascular-related deaths compared to the placebo group. The numbers speak volumes—close to 15% of participants in the treatment cohort experienced cardiovascular-related deaths, while a notable 21.3% of patients in the placebo cohort succumbed to the same fate.
BridgeBio’s revelation also delves into the nuances of mortality patterns. The company reports that a significant 79% of the deaths observed in the study were linked to cardiovascular causes. A broader perspective unfurls through the lens of all-cause mortality, showcasing a noteworthy 25% relative risk reduction for patients administered acoramidis, when contrasted against those on placebo—a facet brought to light in an investor presentation that followed the data.
The unveiling of these insights stands as a defining moment, particularly considering that all-cause mortality, a crucial metric, did not exhibit a statistically significant improvement in BridgeBio’s earlier topline hierarchy of endpoints. While the company refrains from explicitly stating the statistical significance of the relative reduction in cardiovascular-related mortality, the data’s significance remains palpable.
The narrative expands beyond mortality, capturing previously reported hospitalization data. Acoramidis emerges as a beacon of hope, with the average annual hospitalization rate for patients receiving the treatment settling at a promising 0.29—an approximation that mirrors the annual hospitalization rate for Medicare patients across the US. The context is intriguingly contrasted with Pfizer’s Vyndaqel, where the mean annual hospitalization rate stands at 1.00.
Further layers of acoramidis’ impact come to light through the lens of a six-minute walk test—a quintessential measure of functional capacity in cardiovascular conditions. The unveiled data reveal that 40% of patients receiving acoramidis reported improvement after 30 months, in comparison to a mere 22% in the placebo group. While specifics regarding the average test results for each group remain veiled, the pattern of progress is unmistakable.
BridgeBio’s unwavering confidence echoes through these revelations, echoing the momentum since the initial data unveiling in July. The company remains steadfast in its mission, aiming to submit an application for FDA approval before the year concludes, with plans to extend submissions to additional markets in 2024. A future brimming with promise awaits, with plans to launch a primary prevention study in the upcoming year.
In the grand orchestration of the pharmaceutical landscape, BridgeBio’s entrance looms, potentially around a year following Alnylam’s Onpattro. The FDA’s forthcoming decision on expanding Onpattro’s label to encompass ATTR-CM marks a dynamic period of change, where innovation converges with medical need to shape the landscape of cardiovascular treatment.