BridgeBio Soars on Landmark Heart Drug Trial Results
FDA & Biotech

BridgeBio Soars on Landmark Heart Drug Trial Results

Acoramidis shows a 59% reduction in mortality for a rare heart disease, positioning it as a major challenger in a multi-billion dollar market.

BridgeBio Pharma (NASDAQ: BBIO) has unveiled compelling new data for its investigational heart disease drug, acoramidis, showing a significant reduction in mortality for patients with a rare and fatal condition. The findings, presented at the American Heart Association (AHA) Scientific Sessions and concurrently published in the prestigious JAMA Cardiology, represent a major clinical victory for the company and position acoramidis as a formidable competitor in a market currently dominated by Pfizer.

The long-term data from the ATTRibute-CM study demonstrated that acoramidis led to a 59% reduction in the risk of all-cause mortality over 42 months in patients with the variant form of transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive and debilitating heart disease. Shares of BridgeBio were active in Friday trading, closing at $61.80, giving the company a market capitalization of approximately $12.1 billion as investors digested the landmark results.

The study's outcomes are particularly significant for a specific high-risk patient group with the V142I genetic variant, which is more common in individuals of African or Caribbean descent. In this population, acoramidis achieved a 69% reduction in all-cause mortality. These results could reshape the standard of care for a disease that has historically been underserved.

“These data represent an important finding for patients with the V142I variant of ATTR-CM, a population that has historically had limited access to early diagnosis and treatment,” said Dr. Kevin Alexander of the Stanford University School of Medicine, the first author of the JAMA Cardiology manuscript. “The 69% reduction in all-cause mortality observed with acoramidis is clinically meaningful, with sustained and statistically significant benefits in survival, functional capacity, and quality of life.”

The clinical success of acoramidis places BridgeBio in direct competition with Pfizer’s blockbuster drugs Vyndaqel and Vyndamax, which are the current standard of care for ATTR-CM. The Vyndaqel franchise generated global revenues of $6.3 billion for Pfizer in 2024, highlighting the substantial commercial opportunity for a new, potentially more effective, treatment. BridgeBio's data, showing a clear mortality benefit, could provide a strong competitive edge as it prepares for a potential market entry.

Beyond the survival benefit, the study also reported significant improvements in patients' daily lives. Participants treated with acoramidis showed an 87-meter improvement in the 6-minute walk distance and a 20-point improvement on a key quality-of-life questionnaire, both of which were statistically significant.

Mike Lane, founder of the patient advocacy group Amyloidosis Army, shared a personal perspective on the data's impact: “Living with ATTR-CM means facing a disease that quietly steals pieces of your life. When I see new results and real advances, especially data like this in the variant population, I feel something I haven’t felt in a long time: hope.”

Wall Street has maintained a bullish outlook on the company. Based on the consensus of 19 analysts, BridgeBio holds a “Strong Buy” rating with an average price target of approximately $83, suggesting significant upside from its current valuation. Analysts point to the drug's potential to capture a meaningful share of the ATTR-CM market as a key driver for future growth.

With these definitive Phase 3 results in hand, BridgeBio is expected to move forward with regulatory submissions to the U.S. Food and Drug Administration (FDA) and other global health authorities. A potential approval would mark the company's transition into a major commercial-stage player in the cardiovascular space, validating its strategy of developing therapies for genetically-driven diseases.