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Alnylam Completes Enrollment in Phase II Trial of ATTR Drug


NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals announced this week that it has completed enrollment in a Phase II trial of its subcutaneously administered TTR-mediated amyloidosis (ATTR) drug revusiran, formerly known as ALN-TTRsc.

ATTR is a condition characterized by the accumulation of amyloid deposits in tissues due to mutations in the TTR gene. Revusarin, like its intravenously administered Phase III counterpart patisiran, is designed to silence both mutant and wild-type TTR.

The open-label study enrolled 26 patients with manifestations of the disease called familial cardiac amyloidosis (FAC), which primarily affects heart tissue, and senile systemic amyloidosis (SSA), wherein wild-type TTR protein accumulates in the hearts of elderly patients.

The drug was administered initially as daily subcutaneous doses for five days and then once weekly at doses of 5 mg/kg or 7.5 mg/kg over a period of five weeks for a total of 10 doses, according to Alnylam.

The trial's primary endpoints were safety and tolerability, while secondary endpoints included pharmacodynamics and pharmacokinetics. Exploratory clinical endpoint data were also collected at baseline and days 42 and 90 after start of dosing.

Data from this trial are expected to be released later this month.

Alnylam also said that it has kicked off an open-label extension study open to all patients in the Phase II trial. Results from this study are expected to be presented once annually beginning next year.

Completion of the revusiran trial comes just weeks after Alnylam released positive data from a Phase II open-label extension study of patisiran.