Alnylam Pharmaceuticals this week reported top-line results from an ongoing phase I trial of its subcutaneously administered TTR-mediated amyloidosis treatment ALN-TTRsc, which showed the drug could knock down serum levels of its target protein by more than 80 percent..
The release of the data come about a week after Alnylam presented positive phase II data on an intravenous version of the drug called ALN-TTR02 (GSN 7/3/2013).
ATTR is caused by mutations in the TTR gene, which triggers accumulation of abnormal amyloid proteins in the body. ALN-TTRsc comprises siRNAs designed to inhibit both the wild-type and mutant forms of the protein, and is formulated with Alnylam's proprietary GalNAc conjugate technology.
The ongoing phase I trial is designed to test ALN-TTRsc in up to 40 healthy individuals, who receive single or multiple ascending subcutaneous doses ranging from 1.25 mg/kg to 10 mg/kg. Primary objectives of the study are safety and tolerability, and secondary objectives include assessment of clinical activity as measured by serum TTR levels.
Assuming positive final results from the study, Alnylam anticipates starting a phase II trial in ATTR patients with familial amyloidotic cardiomyopathy – one of the manifestations of the disease – later this year. Phase III testing could begin as soon as 2014.