Alnylam Pharmaceuticals this week announced that it has completed enrollment in a phase II trial of its lipid nanoparticle-formulated TTR-mediated amyloidosis treatment ALN-TTR02.
The phase II open-label trial is evaluating two doses of ALN-TTR02, ranging from 0.01 mg/kg to 0.30 mg/kg, in 29 patients with ATTR polyneuropathy. Patients are treated either once every four weeks or once every three weeks.
This summer, the company reported positive interim data from the trial showing that its drug was able to knock down levels of the protein that causes the disease by as much as 93 percent (GSN 7/3/2013).
Alnylam said that it remains on track to begin a phase III trial of ALN-TTR02 in ATTR patients with a manifestation of the condition called familial amyloidotic polyneuropathy by the end of this year.