BridgeBio Pharma, Inc. (Nasdaq: BBIO), a biopharmaceutical company focused on genetic diseases, has announced positive interim results from the ATTRibute-CM open-label extension (OLE) study of acoramidis, presented at the American Heart Association (AHA) Scientific Sessions. Acoramidis, an investigational small molecule designed to stabilize transthyretin (TTR), is being evaluated for its efficacy and safety in treating transthyretin amyloid cardiomyopathy (ATTR-CM). The initial findings were also published in Circulation. The OLE study includes 389 participants who completed the 30-month Phase 3 ATTRibute-CM trial.
Key Findings
Dr. Daniel Judge, professor of medicine and cardiology at the Medical University of South Carolina, highlighted the potential of acoramidis:
Key results from the OLE study presented at AHA include:
- Sustained Clinical Benefit: A confirmed improvement in time to first cardiovascular hospitalization (CVH) or all-cause mortality (ACM) relative to placebo starting at Month 3 in ATTRibute-CM.
- Mortality Reduction: A statistically significant reduction in ACM by 36% at Month 36 (p=0.009) and 34% at Month 42 (p=0.006), according to the Stratified Cox proportional hazards model.
- Composite Outcome Reduction: A reduction in combined ACM and CVH of 46% at Month 36 (p<0.0001) and 48% at Month 42 (p<0.0001), as measured by negative binomial regression.
- Early Benefits: Patients who transitioned from placebo to acoramidis after Month 30 showed improvements compared to projected outcomes for placebo, reinforcing the drug’s early efficacy.
- Safety Profile: Acoramidis remained well-tolerated, with no new clinically significant safety concerns identified during long-term follow-up.
These findings build on the ATTRibute-CM Phase 3 results, where acoramidis demonstrated substantial benefits, including a 50% reduction in the cumulative frequency of CVH relative to placebo at Month 30.
Expert Insights
Jonathan Fox, M.D., Ph.D., Chief Medical Officer of BridgeBio Cardiorenal, emphasized:
Additional Insights from AHA
BridgeBio also presented three posters at AHA highlighting related topics:
- Healthcare Costs and Resource Utilization: ATTR-CM imposes greater costs and healthcare burdens compared to generalized heart failure.
- Evolving Patient Risk Profiles: A systematic review of clinical trials in ATTR-CM patients.
- Survival Outcomes: Acoramidis improved survival irrespective of prior CVH history.
Regulatory Progress
Based on these results, BridgeBio has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration, with a Prescription Drug User Fee Act (PDUFA) target action date of November 29, 2024. A Marketing Authorization Application (MAA) has also been filed with the European Medicines Agency, with a decision anticipated in 2025. BridgeBio has partnered with Bayer to commercialize acoramidis in Europe.
