Revusiran: Phase II data

An open-label, U.S. and U.K. Phase II trial in 26 patients with TTR cardiac amyloidosis showed that once-daily subcutaneous revusiran for 5 days followed by once-weekly 5 or 7.5 mg/kg revusiran for 5 weeks led to knockdown of serum TTR protein levels of up to 98.2%, with a mean maximum knockdown of 87.2%. Additionally, there were no significant changes in exploratory clinical measurements of 6-minute walk distance (6MWD), modified body mass index (BMI), echocardiogram and cardiac MRI, circulating cardiac biomarkers and questionnaires to assess cardiomyopathy symptoms and quality of life (QOL). Revusiran was generally well tolerated with injection-site reactions and transient mild liver function test changes reported as the most common adverse events. The trial enrolled 14 patients with familial amyloidotic cardiomyopathy (FAC), a predominant clinical manifestation of TTR-mediated amyloidosis, and 12 patients with senile systemic amyloidosis (SSA), a non-hereditary form of TTR cardiac amyloidosis. All patients in the trial are eligible to enroll in an open-label extension trial. Data were presented at the American Heart Association meeting in Chicago. ...