BridgeBio Pharma Presents Additional Data and Analyses from its Phase 3 ATTRibute-CM Study in Transthyretin Amyloid Cardiomyopathy (ATTR-CM) at ESC-HF, Including That Acoramidis Treatment Significantly Reduced All-Cause Mortality in a Pre-specified Sensit - Seite 2

“ATTRibute-CM breaks new ground studying the efficacy and safety of acoramidis, a next-generation TTR stabilizer, as the only ATTR-CM outcomes trial to include patients with eGFR <25 mL/min/1.73m²,” said Dr. Poulsen. “We recruited patients with Stage 4 CKD (eGFR between 15 and 30 mL/min/1.73m²) to explore the safety of acoramidis in this very high-risk population; in a pre-specified all-cause mortality (ACM) efficacy analysis of the total ITT population including these patients, acoramidis demonstrated a statistically significant benefit within 30 months. Even in this very high-risk Stage 4 CKD group, ACM favored active treatment with no safety signals of clinical concern.”

Additional acoramidis presentations at ESC Heart Failure included:

• Health-related quality of life in patients with transthyretin amyloid cardiomyopathy treated with acoramidis: an EQ-5D-5L analysis from the ATTRibute-CM study, presented by Peter Van Der Meer, M.D., Ph.D., cardiologist, chair of the Department of Experimental Cardiology and professor of Heart Failure and Translational Cardiology at University Medical Centre Groningen, NL
  • Acoramidis is the only ATTR-CM product candidate assessed with the EuroQoL Health Outcomes Assessment tool EQ-5D-5L and demonstrated a statistically significant and clinically important reduction in the progressive decline in health-related quality of life
• Improved health-related quality of life in acoramidis-treated patients with ATTR-CM, demonstrated by improvements in KCCQ scores, presented by Marianna Fontana, M.D., Ph.D., Professor of Cardiology and Honorary Consultant Cardiologist at the National Amyloidosis Centre, Division of Medicine, University College London, UK
  • Acoramidis treatment reduced the decline in health status and quality of life as shown by statistically significant and clinically meaningful benefits in the KCCQ overall summary score, and supported by numerical and consistent benefits in individual KCCQ domains
• Acoramidis significantly improves NT-proBNP indices that indicate ATTR-CM disease progression and predict subsequent mortality: insights from the ATTRibute-CM study, presented by Pablo Garcia-Pavia, M.D., Ph.D., Iron Gate Majadahonda University Hospital, ES
  • In separate publications, increase in NT-proBNP has been proposed as a metric signaling disease progression (Progressor Index 1) and has been shown to predict mortality in wild-type ATTR-CM at 12 months after diagnosis (Progressor Index 2). Acoramidis slows disease progression as measured by significant improvement in these Progressor Indices.

Based on the positive results from ATTRibute-CM, BridgeBio submitted a New Drug Application to the U.S. Food and Drug Administration, which has been accepted with a PDUFA action date of November 29, 2024, and a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025. BridgeBio has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in Europe.