Alnylam Pharma said that the study is expected to enroll approximately 28 ATTR patients. Further, the trial is designed to provide preliminary data on human proof of concept based on measurements of TTR serum levels.
ALN-TTR01 is a systemically delivered RNAi therapeutic being developed for the treatment of ATTR, including familial amyloidotic polyneuropathy (FAP) and familial amyloidotic cardiomyopathy (FAC).
The primary objective of the study 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 0.4mg/kg.
Whereas, the secondary objectives include characterisation of plasma and urine pharmacokinetics of ALN-TTR01 and assessment of pharmacodynamic activity based on measurements of circulating TTR serum levels.
Akshay Vaishnaw, senior vice president of clinical research at Alnylam, said: “We are very excited about the potential for our ALN-TTR program to make a significant impact in the treatment of this disease. Our pre-clinical data in this program are very encouraging and point to the potential for significant clinical impact.”