BBIO News & Events

BridgeBio presented long-term efficacy and safety data from the ATTRibute-CM open-label extension trial, demonstrating sustained clinical benefit of acoramidis through Month 54 in patients with ATTR-CM. These data were presented at the American College of Cardiology, or ACC, annual scientific sessions and expo in a late-breaking oral presentation. These data are now simultaneously published in JAMA Cardiology. At Month 54, continuous acoramidis treatment led to a statistically significant risk reduction of 44.7% in all-cause mortality and 49.3% in cardiovascular mortality versus placebo-to-acoramidis, which is the earliest timepoint in an open-label extension with this magnitude of risk reduction in these events. Through Month 54, acoramidis mitigated the rise in NT-proBNP versus placebo-to-acoramidis to an extent not seen in the era of disease modifying treatments. Early and continuous acoramidis treatment stabilized and maintained all measures of heart failure-related QOL scores, which demonstrates that the improvement in duration and quality of life in patients with ATTR-CM by acoramidis is sustained. Acoramidis continued to be well tolerated with no long-term safety concerns.

BridgeBio announced the submission of its new drug application to the FDA for oral BBP-418 for the treatment of individuals living with limb-girdle muscular dystrophy type 2I/R9. "This NDA submission brings us one step closer to delivering the first approved therapy to individuals and families affected by LGMD2I/R9, a severe, progressive neuromuscular disease," said Christine Siu, CEO of BridgeBio Neuromuscular. "This achievement not only reflects the strength of the data, but also our dedicated focus on addressing the urgency of a community that has long been waiting for meaningful treatment options. We are committed to working closely with the FDA to make this potential disease-modifying therapy available as quickly as possible."