US-based biopharmaceutical company BridgeBio Pharma has dosed the first subject in the ACT-EARLY study of acoramidis, a drug intended to prevent transthyretin amyloidosis (ATTR).
The subject is asymptomatic and carries a known pathogenic transthyretin (TTR) variant, which is understood to cause ATTR.
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Acoramidis is an oral small-molecule TTR stabiliser whose development is more than 90% complete.
The ACT-EARLY trial aims to test whether prophylactic treatment of asymptomatic carriers of the variant using this TTR stabiliser can delay or prevent the emergence of variant ATTR (ATTRv), also referred to as hereditary ATTR (hATTR).
The study is planning to randomise around 600 asymptomatic carriers of a pathogenic TTR variant.
Its primary efficacy goal is the time to ATTR-CM and/or ATTR-PN development, while further endpoints include the therapy’s tolerability, safety and its impacts on parameters of cardiac imaging, nerve conduction, neurofilament light chain, and plasma TTR concentration.
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By GlobalDataATTRv usually presents earlier and advances more aggressively than the wild-type form of ATTR, resulting in a worse prognosis.
BridgeBio Cardiorenal Global Clinical Development vice-president Adam Castaño said: “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 organisations to establish a new prevention paradigm in an area where there is serious unmet need.”
Based in California, BridgeBio Pharma develops ‘transformative’ medicines for genetic diseases and cancers with obvious genetic drivers.
The company previously carried out the Phase III ATTRibute-CM trial, in which acoramidis decreased the all-cause death risk or first cardiovascular-related hospitalisation in ATTRv-CM subjects by 59.1%.
This finding supports the hypothesis that stabilising TTR with the therapy may delay or prevent variant ATTR-CM.
