BridgeBio Shows 42% Reduction in Mortality and Cardiovascular Hospitalizations with Acoramidis in ATTRibute-CM Study

BridgeBio Pharma, Inc., a company specializing in biopharmaceutical treatments for genetic diseases, recently shared a post-hoc analysis from its Phase 3 ATTRibute-CM study at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2024. This study evaluated the effects of acoramidis on patients with transthyretin amyloid cardiomyopathy (ATTR-CM).

Acoramidis, an investigational, orally-administered small molecule, has shown promising results in reducing all-cause mortality (ACM) and recurrent cardiovascular-related hospitalizations (CVH). The analysis revealed a 42% reduction in composite ACM and recurrent CVH events over 30 months compared to a placebo. This significant improvement was noted by using a negative binomial regression model, highlighting the potential of acoramidis to positively impact patient outcomes.

Jonathan Fox, M.D., Ph.D., Chief Medical Officer of BridgeBio Cardiorenal, emphasized the importance of the findings, noting that the data transparency and the continued communication of these results are crucial for assisting healthcare providers in selecting appropriate treatments for their patients. The study aims not only to enhance the clinical science surrounding ATTR-CM but also to underscore the potential of acoramidis as a meaningful treatment option.

The oral presentation by Dr. Daniel Judge of the Medical University of South Carolina shed light on the detailed findings of the analysis. The study found a 42% reduction in the total number of ACM and recurrent CVH events per patient when treated with acoramidis compared to a placebo. Additionally, a 30.5% hazard reduction in these events was observed, reinforcing the benefits of acoramidis in stabilizing transthyretin (TTR), a protein associated with improved clinical outcomes for ATTR-CM patients.

Dr. Judge highlighted that the effects of acoramidis — particularly its impact on reducing hospitalizations and mortality — make a compelling case for its potential as a first-line therapy. The possibility of acoramidis improving the quality of life for patients with ATTR-CM is a significant consideration for healthcare providers.

Furthermore, during the Japanese College of Cardiology's Annual Scientific Session, positive results from a Phase 3 trial conducted in Japan were presented. These results, shared by Dr. Jin Endo of Keio University School of Medicine, showed no mortality over a 30-month treatment period with acoramidis. The findings were consistent with the ATTRibute-CM study results, including improvements in survival, cardiovascular health, physical function, and quality of life.

Following these promising outcomes, BridgeBio has submitted a New Drug Application to the U.S. Food and Drug Administration (FDA), with a decision expected by November 29, 2024. A Marketing Authorization Application has also been submitted to the European Medicines Agency, with an anticipated decision in 2025.

BridgeBio Pharma, Inc. is committed to discovering, creating, and delivering transformative medications for genetic diseases. Founded in 2015, the company is dedicated to advancing genetic medicine to benefit patients as swiftly as possible. Their development pipeline spans from early research to advanced clinical trials, emphasizing the company's focus on innovation and patient care.