BridgeBio announces first participant dosed with acoramidis in ACT-EARLY

BridgeBio (BBIO) Pharma announced that the first asymptomatic participant with a known pathogenic transthyretin variant, that may lead to transthyretin amyloid disease, has been dosed in ACT-EARLY with acoramidis. Acoramidis is a selective, small molecule, orally administered near-complete 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 Castano, M.D., Vice President of Global Clinical Development at BridgeBio Cardiorenal and Head of the ACT-EARLY clinical program.