Patisiran: Extension study interim data

Interim data from 19 evaluable patients with TTR-mediated amyloidosis and familial amyloidotic polyneuropathy (FAP) in an open-label extension trial of an open-label, dose-escalation, international Phase II trial showed that 0.3 mg/kg IV patisiran every 3 weeks led to a mean 0.95 point reduction in mNIS+7 score at 6 months vs. a mean 7-10 point increase for historical controls of untreated FAP patients with similar baseline characteristics. Alnylam also said there was no significant evidence for disease worsening observed in measurements of QOL, 10-meter walk test, modified BMI, level of disability and never fiber density. Additionally, patients in the extension achieved sustained mean serum TTR knockdown at the 80% target level for >9 months. The extension trial has enrolled 27 patients and is evaluating 0.3 mg/kg patisiran every 3 weeks for up to 2 years in patients who received 0.01-0.3 mg/kg IV patisiran every 3 or 4 weeks for 2 cycles in the Phase II trial. Data were presented at the American Neurological Association meeting in Baltimore. Interim data from the Phase II trial were reported last year (see BioCentury, July 15, 2013). ...