- Acoramidis treatment led to a highly significant reduction in all-cause mortality (ACM) and recurrent cardiovascular-related hospitalizations (CVH) at Month 30 compared with placebo - Additionally, results from a Phase 3 trial in adults with ATTR-CM conducted in Japan were presented, with no ACM events reported over the 30 month treatment period in participants administered acoramidis - Greater transthyretin (TTR) stabilization has been associated with improved clinical outcomes for patients,
- Breakthrough Therapy Designation was granted based on preliminary clinical evidence from the PROPEL 2 clinical trial, meeting the FDA’s requirement of potentially demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoint(s) - BridgeBio will leverage the benefits of Breakthrough Therapy Designation to expedite the development and regulatory review of infigratinib in the United States and if approved, infigratinib could be the first-in-class or
- Increase in endogenous cortisol production achieved in all patients in higher dose cohorts of BBP-631, a result seen for the first time ever in CAH patients - The gene therapy was well tolerated with no treatment-related serious adverse events (SAEs) reported - Despite novel scientific advancements achieved with this program, the data do not warrant additional capital investment at this time and the gene therapy budget is being significantly reduced PALO ALTO, Calif., Sept. 10, 2024 (GLOBE NEW