Haemophilia A and B
Development and Regulatory status
Aug 14: Orphan drug designation in the EU. 
Trial or other data
Aug 14: Top-line data from Part A of Phase 1 study, in adult healthy volunteers (n=4, 3:1 drug:placebo). Initial results showed that a single, low subcutaneous ALN-AT3 dose of 0.03 mg/kg resulted in an up to 28-32% knockdown of antithrombin (AT) at nadir (p<0.01). This resulted in a statistically significant (p<0.01) increase in peak thrombin generation, that was temporally associated and consistent with the degree of AT knockdown. ALN-AT3 was found to be well tolerated with no significant adverse events reported. Part B of the study to be initiated: open-label, multi-dose, dose-escalation study in up to 18 people with moderate to severe hemophilia A or B. The primary objective of this part of the study is to evaluate the safety and tolerability of multiple doses, specifically three weekly doses, of subcutaneously administered ALN-AT3 in hemophilia subjects.