Alnylam Pharma Starts Phase 1 Clinical Trial For ALN-TTRsc02 - Quick Facts

(RTTNews) - Alnylam Pharmaceuticals Inc. (ALNY) said it has initiated a Phase 1 clinical trial with ALN-TTRsc02, a subcutaneously administered investigational RNAi therapeutic for the treatment of transthyretin or TTR-mediated amyloidosis (ATTR amyloidosis).

The company will conduct the Phase 1 trial in normal healthy volunteers. The Phase 1 trial of ALN-TTRsc02 is a randomized, placebo-controlled, single ascending-dose study designed to enroll up to a total of 100 normal healthy volunteers.

The primary objective of the study is to evaluate safety and tolerability of a single subcutaneous dose of ALN-TTRsc02. Secondary objectives include evaluation of pharmacokinetics and clinical activity for ALN-TTRsc02.

Initiation of this trial is based on encouraging pre-clinical results, including data presented last year at the Oligonucleotide Therapeutics Society or OTS meeting held October 11 to 14, 2015.

The company has guided that it expects to report initial clinical data from this study in late 2016, and if positive, plans to initiate a Phase 3 study in 2017.

Eric Green, Vice President, General Manager of TTR Program said, "We believe ALN-TTRsc02 has the potential to become a best-in-class, once-quarterly, subcutaneous treatment regimen for the treatment of ATTR amyloidosis. With just four doses anticipated per year, ALN-TTRsc02 could offer patients with ATTR amyloidosis an important new therapeutic option to manage their disease."