- ATTRibute-CM, BridgeBio’s Phase 3 clinical trial of acoramidis in patients with ATTR-CM, achieved statistical significance in reducing the risk of ACM or first CVH versus placebo in ATTRv-CM patients (59.1% risk reduction), establishing the mechanistic hypothesis that stabilizing TTR may delay or prevent ATTRv-CM
- ACT-EARLY is a registrational, randomized, double blind, placebo controlled, event driven prevention study that will enroll ~600 asymptomatic carriers of a pathogenic TTR variant. Diagnosis of ATTRv disease will be evaluated as the primary analysis of the study
- In the ACT-EARLY study, BridgeBio will partner globally with ATTR amyloidosis treating physicians and patient advocacy organizations with the hope of addressing a serious unmet need and proving that ATTRv can be delayed or prevented
- Acoramidis is approved as Attruby™ by the U.S. FDA and is approved as BEYONTTRA® by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency, and the UK Medicines and Healthcare Products Regulatory Agency
PALO ALTO, Calif., May 13, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, announced today that the first asymptomatic participant with a known pathogenic transthyretin (TTR) variant, that may lead to transthyretin amyloid disease (either cardiomyopathy, ATTR-CM, polyneuropathy, ATTR-PN, or both), has been dosed in ACT-EARLY with acoramidis. Acoramidis is a selective, small molecule, orally administered near-complete (≥90%) TTR stabilizer.
“Launching ACT-EARLY is part of our ongoing commitment to further the genetic understanding of the variants causing ATTR and to ensure patients from around the world have access to optimal care. Our hope is that this study will have profound impact to patients and caregivers, and we look forward to growing our partnership with providers and patient advocacy organizations to establish a new prevention paradigm in an area where there is serious unmet need,” said Adam Castaño, M.D., Vice President of Global Clinical Development at BridgeBio Cardiorenal and Head of the ACT-EARLY clinical program.
ACT-EARLY is the first ever primary prevention study for ATTR, testing the hypothesis that prophylactic treatment of asymptomatic carriers of a pathogenic TTR variant with the near-complete TTR stabilizer, acoramidis, could delay the onset or prevent the development of variant ATTR (ATTRv), also known as hereditary ATTR (hATTR). ATTRv often presents earlier and progresses more aggressively than the wild-type form of ATTR, leading to significantly worse prognosis. The study aims to randomize ~600 asymptomatic carriers of a pathogenic TTR variant. The primary efficacy endpoint is time to development of ATTR-CM and/or ATTR-PN. Additional endpoints include safety and tolerability of acoramidis, and its effects on cardiac imaging parameters, plasma TTR concentration, nerve conduction and neurofilament light chain.
“Current approved therapies for ATTR amyloidosis are only approved to treat diagnosed disease and can only be expected to slow disease progression. There are still many people who carry a genetic variant which puts them at risk of this progressive and fatal disease, and who typically watched other family members suffer through it. Currently, there are no proven prevention treatment options,” said Ahmad Masri, M.D., M.S., Cardiomyopathy Section Head and Director of the Cardiac Amyloidosis Program at Oregon Health & Science University. “By collaborating with BridgeBio on this groundbreaking study, I am hopeful that we can fill the significant gap in care for asymptomatic carriers of a genetic variant by providing potential preventative intervention early with resulting greater clinical benefit than addressing the disease at a later stage.”