To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)

A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients With Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)

The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).

The AG10-504 study is the extension of the Phase 3 AG10-501 study (ACT-EARLY) which was designed to investigate if the use of acoramidis as a preventative intervention in individuals who are carriers of a known pathogenic transthyretin (TTR) variant but with no clinical evidence of ATTR could prevent or delay the onset of clinically detectable ATTR and the considerable morbidity and mortality that result from this devastating, progressive, and ultimately fatal disease. Only participants who have completed the AG10-501 study (ACT-EARLY) with a diagnosis of ATTR-CM may enroll in this AG10-504 Open Label Extension (OLE) study.

The primary objective of the AG10-504 study is to evaluate the long-term safety and tolerability of acoramidis in participants with newly diagnosed ATTR-CM. The duration of this study will be up to 5 years.

Currently, acoramidis is approved for the treatment of ATTR-CM in some regions including the United States, all member states of the European Union (EU), Albania, Bosnia Herzegovina, Brazil, Iceland, Israel, Japan, Liechtenstein, Montenegro, North Macedonia, Norway, Switzerland, and the United Kingdom (UK).

Key Inclusion Criteria:

Participants must have completed the AG10-501 study (ACT-EARLY study) within the past 60 calendar days with a diagnosis of ATTR-CM (based on the AG10-501 protocol definition of ATTR-CM).

Key Exclusion Criteria:

1. Participants who completed the AG10-501 study with a diagnosis of ATTR-PN only or who permanently discontinued study drug prior to diagnosis of ATTR-CM in AG10-501.
2. History of light-chain amyloidosis (AL) or another non-TTR amyloid subtype (eg, ApoA-1, gelsolin).
3. History of a monoclonal paraprotein or abnormal light chains in serum or urine (ie, monoclonal gammopathy of undetermined significance, MGUS) in which AL has not been ruled out.
4. Stage IV or V chronic kidney disease.
5. Active malignancy, except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix that has been successfully treated.
6. History of any organ transplant (with the exception of corneal transplant).
7. Known hypersensitivity to acoramidis or any of its excipients.
8. Major surgery planned during the next 6 months (some exceptions).
9. Treatment of ATTR-CM with any ATTR-oriented on- or off-label or OTC product.