Alnylam reports Phase II data for amyloidosis compound

Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) presented interim data from an open-label Phase II trial evaluating ALN-TTR02 to treat transthyretin (TTR)-mediated amyloidosis. In 19 evaluable patients, multiple doses of IV ALN-TTR02 led to rapid, dose-dependent and significant knockdown of serum TTR protein levels of up to 92.8%. Similar knockdown was observed in patients with both wild-type and mutant TTR protein levels. Data were presented at the Peripheral Nerve Society meeting in St. Malo. Alnylam plans to present full data in November.

The company plans to start a Phase III trial of ALN-TTR02 to treat familial amyloidotic polyneuropathy (FAP), a predominant clinical manifestation of TTR-mediated amyloidosis, by year end. ALN-TTR02 is an RNAi therapeutic targeting the TTR gene using second-generation lipid nanoparticle technology. ...