Alnylam Pharma Begins Pilot Phase II Study With ALN-TTRsc

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY), a RNAi therapeutics company, Monday said it has initiated a pilot Phase II study with ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin or TTR gene in development for the treatment of TTR-mediated amyloidosis or ATTR.

Transthyretin-mediated amyloidosis is an inherited, progressively debilitating, fatal disease caused by mutations in the TTR gene.

The Phase II trial, which is now open for enrollment, is aimed at evaluating the tolerability and preliminary clinical activity of ALN-TTRsc in TTR cardiac amyloidosis patients with familial amyloidotic cardiomyopathy.

The company expects to present data from the Phase II trial in late 2014. Assuming positive results, the firm expects to begin a Phase III trial in TTR cardiac amyloidosis patients by the end of 2014.

The Phase II trial is an open-label, multi-dose study of ALN-TTRsc, designed to enroll approximately 15 TTR cardiac amyloidosis patients with familial amyloidotic cardiomyopathy or senile systemic amyloidosis.

The primary objective of the study is to evaluate the general tolerability of ALN-TTRsc. Patients will receive 5 daily doses followed by 5 weekly doses of 5 mg/kg, with follow-up through Day 90.