Alnylam Pharmaceuticals announced this week that it has begun dosing patients in a phase I trial of its transthyretin-mediated amyloidosis drug ALN-TTR01.
The study is being conducted in Portugal, Sweden, and the UK and will enroll about 28 patients with the disease.
The primary objective of the trial is to evaluate the safety and tolerability of a single dose of intravenous ALN-TTR01, with patients being enrolled into five sequential cohorts of increasing doses ranging from 0.01 to 0.4 mg/kg, Alnylam said. Secondary objectives include characterization of plasma and urine pharmacokinetics of ALN-TTR01, and assessment of pharmacodynamic activity based on measurements of circulating TTR serum levels.
TTR amyloidosis is a hereditary, systemic disease caused by a mutation in the transthyretin gene. ALN-TTR01 is a systemically delivered siRNA designed to inhibit both mutant and wild-type TTR. It is delivered using partner Tekmira Pharmaceutical's stable nucleic acid-lipid particle technology.