We are working towards improving the quality of life for Haemophilia B patients by evaluating the possibility of extending the dosing intervals from 14 to 21 days. A Phase 3 extension study (PROLONG-9FP) is underway and global regulatory submissions to gain approval for the extended dosing regimen are planned for 2019.
Our commitment to remain at the forefront of innovation is stronger than ever. The acquisition of Calimmune Inc., introduced a new ex vivo haematopoietic stem cell gene therapy (CAL-H/CSL200) for the treatment of sickle cell disease (SCD) into our haematology and thrombosis pipeline. Calimmune’s proprietary platform technologies also have the potential to develop new medicines for a wide range of other rare diseases that complement CSL’s product portfolio and expertise. Complementing the CSL200 program is another exciting program in SCD, CSL889. The CSL889 is plasma derived Hemopexin, an important endogenous molecule whose levels are decreased in patients with sickle cell disease, for treatment of acute vaso-occlusive crisis, a severe and painful complication in SCD patients. Clinical trials using CSL200 and CSL889 are anticipated to start in 2019 and 2020 respectively.