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ARTICLE | Clinical News

Patisiran: Extension study additional data

April 27, 2015 7:00 AM UTC

Additional data from 20 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 2.5 point reduction in mNIS+7 score at 12 months vs. a mean 13-18 point increase for historical controls of untreated FAP patients with similar baseline characteristics. Additionally, patisiran led to a mean 0.4 point increase in NIS score at 12 months vs. a mean 10-14 point increase for historical controls. Patients in the extension achieved sustained mean serum TTR knockdown at the 80% target level for about 16 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 Academy of Neurology meeting in Washington, D.C. ...