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BridgeBio Pharma Has Dosed The First Asymptomatic Participant With A Known Pathogenic Transthyretin Variant, Which May Lead To Transthyretin Amyloid Disease In ACT-EARLY Study With Acoramidis

Benzinga·05/13/2025 11:38:44
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  • 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
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