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Monday, April 1, 2019

Catalyst Biosciences receives orphan drug desgination for MarzAA in EU

Catalyst Biosciences announced that the European Commission has awarded orphan designation of its Factor VIIa variant marzeptacog alfa, or MarzAA, for the treatment of haemophilia B. Catalyst has also completed dosing in the Phase 2 portion of the Phase 2/3 subcutaneous trial of MarzAA for the treatment of hemophilia A or B with inhibitors. Nine subjects successfully completed dosing and top-line results will be presented in the third quarter of 2019. “Orphan designation is another important acknowledgement of the significant benefits of subcutaneous MarzAA and will complement our orphan drug designation already granted in the U.S. by the Food and Drug Administration,” said Nassim Usman, Ph.D., CEO of Catalyst. “We have completed dosing in the Phase 2 portion of the Phase 2/3 trial for the treatment of hemophilia A or B with inhibitors and have clearly demonstrated efficacy as measured by greater than 90% reduction in annualized bleed rate, as well as bleeding density. We expect to present top-line results from the study in the third quarter of 2019. We also plan to initiate a Phase 3 MarzAA registrational study in 2020 and believe that subcutaneous MarzAA has significant commercial potential in the $2.2B hemophilia inhibitor market.”

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