BridgeBio Pharma Announces Positive Phase 3 Results for Acoramidis in ATTR-CM

BridgeBio Pharma's Phase 3 ATTRibute-CM study shows acoramidis significantly reduces disease progression in patients with transthyretin amyloid cardiomyopathy. The study met its primary endpoint, demonstrating improved outcomes for patients with this rare and fatal disease.

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Bijay Laxmi
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BridgeBio Pharma Announces Positive Phase 3 Results for Acoramidis in ATTR-CM

BridgeBio Pharma Announces Positive Phase 3 Results for Acoramidis in ATTR-CM

BridgeBio Pharma has announced positive results from its Phase 3 ATTRibute-CM study, which evaluated the efficacy and safety of acoramidis in patients with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM). The study met its four-step primary hierarchical endpoint, including mortality, cardiovascular-related hospitalization, change in N-terminal pro-B-type natriuretic peptide (NT-proBNP), and six-minute walk test.

Why this matters: This breakthrough in treating ATTR-CM, a rare and fatal disease, offers new hope for patients and their families. The development of effective treatments for such diseases can also drive innovation and progress in the broader field of cardiology.

The ATTRibute-CM study demonstrated that acoramidis significantly reduced the decline in health-related quality of life and improved NT-proBNP indices in patients with ATTR-CM. The study showed a significant reduction in NT-proBNP progressor indices 1 and 2, which are biomarkers of disease progression and predictors of subsequent mortality in ATTR-CM.

At Month 30, 87.1% of patients in the acoramidis group met the criteria for NT-proBNP progressor index 1 (increase in NT-proBNP ≥30 pg/mL), compared to 73.8% in the placebo group (p=0.0001). For NT-proBNP progressor index 2 (increase in NT-proBNP ≥500 pg/mL), 81.2% of patients in the acoramidis group met the criteria at Month 30, compared to 64.9% in the placebo group (p=0.0001).

The ATTRibute-CM study was a 30-month, Phase 3, randomized, double-blind, placebo-controlled trial that enrolled 421 patients with symptomatic ATTR-CM. The study was conducted at multiple sites worldwide, and the primary endpoint was evaluated in the modified intent-to-treat (mITT) population.

"We are thrilled to announce the positive results from our Phase 3 ATTRibute-CM study, which demonstrate the potential of acoramidis to improve outcomes for patients with ATTR-CM," said [Name], CEO of BridgeBio Pharma. "We look forward to continuing to work with regulatory authorities to bring this important new treatment option to patients as soon as possible." Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare, progressive, and fatal disease characterized by the accumulation of misfolded transthyretin protein in the heart, leading to heart failure. Currently, there are limited treatment options available for patients with ATTR-CM, and the disease is associated with a poor prognosis and reduced quality of life.

The positive results from the ATTRibute-CM study represent a significant milestone in the development of acoramidis as a potential treatment for ATTR-CM. BridgeBio Pharma presented the findings at the European Society of Cardiology Heart Failure Congress 2024 in Palo Alto, California, highlighting the impact of this groundbreaking study on the future management of ATTR-CM patients.

Key Takeaways

  • BridgeBio Pharma's Phase 3 ATTRibute-CM study shows acoramidis reduces mortality and cardiovascular-related hospitalization in ATTR-CM patients.
  • Acoramidis significantly improves health-related quality of life and reduces NT-proBNP indices in ATTR-CM patients.
  • 87.1% of patients in the acoramidis group met NT-proBNP progressor index 1 criteria at Month 30, vs. 73.8% in the placebo group.
  • The 30-month study enrolled 421 patients with symptomatic ATTR-CM at multiple sites worldwide.
  • Acoramidis has potential to improve outcomes for patients with ATTR-CM, a rare and fatal disease with limited treatment options.