AT-02 – Targeting Amyloid Deposits in Systemic Amyloidosis

Systemic amyloidosis is characterised by the abnormal accumulation of amyloid deposits in tissues and organs. Cardiac involvement is particularly important because amyloid deposition within the myocardium can lead to increased ventricular wall thickness, impaired cardiac function and progressive heart failure. Current treatments for some forms of amyloidosis primarily reduce the production of amyloid-forming precursor proteins; AT-02 was developed with a different strategy — targeting existing amyloid deposits and promoting their removal.

The AT02-001 (NCT05521022) Phase 1 study was designed primarily to assess the safety, tolerability and pharmacokinetics of AT-02, initially following single ascending doses in healthy volunteers and patients with systemic amyloidosis, followed by multiple doses in patients with systemic amyloidosis. It also evaluated pharmacodynamic effects and preliminary clinical activity. The subsequent AT02-002 (NCT05951049) Phase 2 open-label extension study evaluates the long-term safety, tolerability and clinical activity of AT-02 in patients with systemic amyloidosis. Cardiac assessments include CMR and echocardiography, alongside cardiac biomarkers and functional measures.