Fitusiran is an investigational, once-monthly and subcutaneously administered RNAi therapeutic targeting antithrombin (AT) to treat hemophilia A and B with and without inhibitors.
The investigational treatment is designed to reduce levels of AT and promote sufficient thrombin generation to restore hemostasis and prevent bleeding.
The three separate trials include Atlas- INH, which a nine-month, open-label, randomized and active-controlled trial that will recruit up to 50 patients with hemophilia A or B with inhibitors securing prior on-demand therapy.
Its primary endpoint is the annualized bleeding rate (ABR), while secondary endpoints include the annualized spontaneous bleeding rate, annualized joint bleeding rate, and quality of life as measured by the Haem-A-QOL score.
Atlas S-A/B is a nine-month, open-label, randomized and active-controlled trial, which will enroll around 100 patients with hemophilia A or B without inhibitors receiving prior on-demand therapy.
ATLAS-PPX is an open-label and one-way crossover study, which will enroll up to 100 patients with hemophilia A or B with or without inhibitors receiving prophylaxis therapy as prior standard of care.
In this trial, patients will be provided with standard of care prophylaxis for six months and then transition to fitusiran treatment for seven months.
In January 2014, Alnylam and Sanofi Genzyme announced partnership to advance the development of RNAi therapeutics to treat patients with rare genetic diseases.
Sanofi's senior global project head for Alnylam portfolio Dr Baisong Mei said: "The Atlas studies, which are designed to evaluate the safety and efficacy of fitusiran across a spectrum of patients living with hemophilia, are expected to enroll approximately 250 patients across three separate trials conducted at over 100 clinical centers around the world."
Image: Alnylam and Sanofi Genzyme have started clinical trial program to assess hemophilia treatment fitusiran. Photo: courtesy of jk1991 / FreeDigitalPhotos.net.