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news.Alnylam Pharmaceuticals has started the ENDEAVOUR Phase III clinical trial of revusiran in patients with transthyretin (TTR)-mediated familial amyloidotic cardiomyopathy (FAC).
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The trial is designed to evaluate the efficacy and safety of revusiran in patients with FAC, which is one of the predominant clinical manifestations of TTR-mediated amyloidosis (ATTR).
Co-primary endpoints of the randomized, double-blind, placebo-controlled, global trial are the change compared to baseline in 6-minute walk distance (6-MWD) and the percent reduction in TTR burden between placebo- and revusiran-treated patients at 18 months.
Following dosing of first patient in the trial, Alnylam will be eligible to receive a $25m milestone from Genzyme.
The trial’s secondary endpoints include a composite endpoint of cardiovascular mortality and cardiovascular hospitalisation, New York Heart Association (NYHA) class, Kansas City Cardiomyopathy Questionnaire (KCCQ), CV mortality, CV hospitalization and all-cause mortality.
Alnylam executive vice-president and chief medical officer Akshay Vaishnaw said: "Based on our discussions with regulatory authorities in the US and EU, we are aligned on the use of co-primary endpoints of 6-MWD and TTR knockdown at 18 months as measures of revusiran’s efficacy.
"We will now focus on enrollment of FAC patients in ENDEAVOUR as a high priority for Alnylam, advancing our commitment to bring this potential novel therapy to patients and their caregivers."
The trial will include around FAC patients and they will be randomized 2:1, revusiran:placebo, with revusiran administered subcutaneously at 500mg daily for five days then weekly for 18 months.