ALN-TTR02: Completed Phase II enrollment

Alnylam completed enrollment of 29 patients with TTR-mediated amyloidosis in an open-label, dose-escalation, international Phase II trial evaluating 0.01-0.3 mg/kg IV ALN-TTR02 every 3 or 4 weeks for 2 cycles. Alnylam also began an open-label extension study to the Phase II trial to evaluate 0.3 mg/kg ALN-TTR02 every 3 weeks for up to 2 years. In July, the company reported interim data from 19 patients showing that multiple doses of IV ALN-TTR02 led to rapid, dose-dependent and significant knockdown of serum TTR protein levels of up to 92.8% (see BioCentury, July 15). Additional data from the trial will be presented at the Familial Amyloidotic Polyneuropathy meeting in Rio de Janeiro in November. ...