BridgeBio launches landmark ATTR prevention study

PALO ALTO, Calif. - BridgeBio Pharma, Inc. (NASDAQ:BBIO), a biopharmaceutical company specializing in genetic diseases with a market capitalization of $6.77 billion, has announced the dosing of the first asymptomatic participant in the ACT-EARLY clinical trial. According to InvestingPro data, the company maintains an impressive gross profit margin of 95.35%, though it remains in its growth phase and is not yet profitable. This groundbreaking study tests acoramidis, a transthyretin (TTR) stabilizer, as a potential preventative treatment for transthyretin amyloid disease (ATTR).

ATTR is a progressive and often fatal disease caused by the accumulation of misfolded TTR protein in the body’s tissues and organs. Acoramidis, already approved for treating diagnosed ATTR-CM under the names Attruby™ and BEYONTTRA®, aims to stabilize TTR and thereby delay or prevent the onset of the disease in individuals carrying a pathogenic TTR variant.

The ACT-EARLY study is the first of its kind, aiming to enroll approximately 600 asymptomatic carriers of the TTR variant. Its primary efficacy endpoint is the time to development of ATTR-related cardiomyopathy (ATTR-CM) and/or polyneuropathy (ATTR-PN). With a strong current ratio of 4.57, InvestingPro analysis shows BridgeBio is well-positioned financially to support this extensive clinical program. Additional endpoints include the drug’s safety, tolerability, and effects on cardiac imaging parameters, plasma TTR concentration, nerve conduction, and neurofilament light chain.

This initiative follows the ATTRibute-CM Phase 3 trial, where acoramidis showed a significant reduction in all-cause mortality or cardiovascular-related hospitalization in ATTRv-CM patients. The ACT-EARLY trial aims to establish a new prevention paradigm for the disease in collaboration with physicians and patient advocacy organizations.

Dr. Adam Castaño, Vice President of Global Clinical Development at BridgeBio Cardiorenal, expressed optimism that the study could profoundly impact patients and caregivers by establishing a new standard of preventive care. Dr. Ahmad Masri from Oregon Health & Science University echoed this sentiment, underscoring the need for early intervention in asymptomatic carriers.

The ACT-EARLY trial is currently enrolling participants, with more information available at ACTEARLY.com. BridgeBio is committed to addressing the significant unmet need in the treatment and prevention of genetic diseases, including ATTR. For investors seeking deeper insights, InvestingPro offers comprehensive analysis with additional ProTips and detailed financial metrics in its Pro Research Report, available for over 1,400 US stocks including BBIO.

This news is based on a press release statement from BridgeBio Pharma, Inc.

In other recent news, BridgeBio Pharma has reported impressive financial results for the first quarter of 2025, significantly surpassing market expectations. The company achieved total revenues of $116.6 million, with its key product, Atruvy, generating $36.7 million in net revenue. This strong performance was attributed to successful product launches and strategic milestones, including a $75 million regulatory milestone contributing to the license and services revenue. Scotiabank has responded to this performance by raising the price target for BridgeBio shares to $55, noting the company’s successful commercial performance following the launch of Atruvy for transthyretin amyloid cardiomyopathy (ATTR-CM).

Analyst Greg Harrison from Scotiabank maintained a Sector Outperform rating, emphasizing the strong sales and competitive advantages of Atruvy despite competition from other drugs like tafamidis and Amvuttra. BridgeBio’s stock also saw a positive market reaction, reflecting investor confidence in the company’s performance and future prospects. The company ended the quarter with $540.6 million in cash and cash equivalents and anticipates continued growth in the ATTR Cardiomyopathy market. Looking ahead, BridgeBio is preparing for three additional commercial launches in 2026-2027 and expects a significant increase in future revenues.

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