Alnylam and Sanofi commence Phase III programme for haemophilia drug
Alnylam Pharmaceuticals and Sanofi Genzyme have commenced the Phase III clinical programme (ATLAS) of RNAi therapeutic fitusiran in patients with haemophilia A or B, with or without inhibitors.
Fitusiran is an investigational, subcutaneous, once-a-month therapeutic candidate that targets antithrombin (AT).
Designed to assess the safety and efficacy of fitusiran, the ATLAS programme will include around 250 subjects receiving prophylactic therapy in three separate clinical trials at more than 100 sites.
Alnylam Fitusiran vice-president and general manager Akin Akinc said: “ATLAS will include patients with haemophilia A and B with or without inhibitors, and patients previously receiving on-demand or prophylactic therapy with replacement factors or bypassing agents.”
The ATLAS-INH trial will enrol up to 50 patients with inhibitors previously receiving on-demand therapy, while the ATLAS-A/B trial will recruit 100 patients without inhibitors and receiving on-demand therapy.
Both the nine-month, open-label, randomised, active-controlled trials will evaluate annualised bleeding rate (ABR) as the primary endpoint, and annualised spontaneous bleeding rate, quality of life and annualised joint bleeding rate as the key secondary endpoints.
The third trial in the programme is the open-label, one-way crossover ATLAS-PPX study that will involve about 100 patients with or without inhibitors and who are receiving prior standard-of-care prophylaxis therapy.
During the ATLAS-PPX trial, patients will be administered with standard-of-care prophylaxis for initial six months and will later be transitioned to fitusiran for a period of seven months.
The trial will measure the primary endpoint of ABR in both the fitusiran and the factor / bypassing agent prophylaxis period, while its key secondary endpoints are similar to that of the ATLAS-INH and ATLAS-A/B trials.