Alnylam gains on ALN-TTRsc data

Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) jumped $5.93 (16%) to $43.53 on Thursday after reporting top-line data from a Phase I trial evaluating subcutaneous ALN-TTRsc to treat transthyretin (TTR)-mediated amyloidosis. ALN-TTRsc led to significant knockdown of serum TTR protein levels of more than 80% vs. placebo (p<0.01), a secondary endpoint. The trial is enrolling up to 40 healthy volunteers to receive single doses of 1.25, 2.5, 5 or 10 mg/kg ALN-TTRsc or multiple doses of 2.5, 5 or 10 mg/kg ALN-TTRsc. Alnylam declined to disclose the number of volunteers or the doses included in the top-line analysis. Final data will be presented at the Heart Failure Society of America meeting in September.

Alnylam plans to start a Phase II trial of ALN-TTRsc to treat familial amyloidotic cardiomyopathy (FAC), a predominant clinical manifestation of TTR-mediated amyloidosis, by year end. Assuming positive data, the company plans to start a Phase III trial in 2014. ALN-TTRsc is a subcutaneous formulation of an RNAi therapeutic targeting the TTR gene using Alnylam's GalNAc-conjugate delivery platform. ...