Sobi™ and Dova Pharmaceuticals, will present new data from studies with avatrombopag for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) as well as patients with chronic immune thrombocytopenia (ITP) at the 61st Annual Meeting of the American Society of Hematology (ASH) taking place in Orlando, 7-10 December. In addition, final data from the phase 1/2 repeat dose study with BIVV001 (rFVIIIFc-VWF-XTEN) in severe haemophilia A will be highlighted in a presentation as well as the adaptive design of a study evaluating emapalumab in adults with secondary haemophagocytic lymphohistiocytosis (HLH). Results from a study to evaluate the relative cost-effectiveness of avatrombopag in CLD compared with platelet transfusion or treatment with lusutrombopag, showed that the use of avatrombopag is a practical strategy compared with the cost of both platelet transfusion and lusutrombopag, as it saves cost and reduces the need for prophylactic platelet transfusions.

Results from an online survey highlighting disease burden and impact for patients with immune thrombocytopenia demonstrated the complexity of ITP and the effects of fatigue, bleeding and treatment on the lives of the patients. Additional analyses from the phase 3 study - core and extension phase - with avatrombopag for the treatment of ITP confirm the long-term response rates and efficacy of treatment with avatrombopag in this indication. Final data from a phase 1/2 study of BIVV001 evaluating the safety and pharmacokinetics of repeated dosing in people with severe Haemophilia A will be shared in an oral presentation.

BIVV001 is the first investigational von Willebrand (VWF)-independent factor VIII therapy that is designed to provide high sustained factor activity and extend protection from bleeds with once weekly dosing for people with haemophilia A. BIVV001 is being developed in collaboration with Sanofi. The adaptive design of a study evaluating the efficacy, safety and pharmacokinetics of emapalumab in adult patients with secondary HLH will be presented. The primary efficacy outcome is to evaluate overall response after 4 weeks.

The initial phase will enrol 10 adult patients and the study is expected to open later this year.