Factor VIIa marzeptacog alfa (activated)

Marzeptacog alfa (activated) is a potent, next-generation Factor VIIa that was created to allow for the effective, long-term, prophylaxis in individuals with hemophilia with inhibitors. It was designed to combine higher clot-generating activity and longer activity at the site of bleeding and therefore improved efficacy. Catalyst anticipates that marzeptacog alfa (activated) could be used for subcutaneous prophylactic treatment, and intends to initiate a subcutaneous efficacy trial in the fourth quarter of 2017.

The subcutaneous efficacy trial will be an open-label, dose escalation study in individuals with hemophilia A or B with inhibitors. The objective of the trial will be to inhibit, or minimize, spontaneous bleeding episodes by using individualized dose escalation, if required. The primary endpoint will be a reduction in annualized bleed rate. Each individual’s historical annualized bleed rate will serve as the control. The trial will enroll up to 12 individuals with hemophilia across approximately 10 clinical trial sites globally. Catalyst plans to announce interim data in the first half of 2018.

Factor VIIa marzeptacog alfa (activated) efficacy trial - planned

Factor VIIa marzeptacog alfa (activated) Phase 1 trial - completed

Marzeptacog alfa (activated) has successfully completed a Phase 1 intravenous clinical trial in individuals with severe hemophilia A and B with and without inhibitors. Positive results were reported from this open label, multicenter Phase 1 clinical trial of marzeptacog alfa (activated) at the International Society on Thrombosis and Haemostasis (ISTH) meeting in 2015. The study showed that single intravenous doses of marzeptacog alfa (activated) were well tolerated when administered to individuals with hemophilia A and B, with and without inhibitors, and there were no instances of antibody response or thrombosis. Marzeptacog alfa (activated) demonstrated pharmacological efficacy as measured by significant shortening of aPTT (activated partial thromboplastin time) and PT (prothrombin time) for up to 24 hours post-dosing without inducing markers of thrombosis.

Marzeptacog alfa (activated) has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of individuals with hemophilia A and B.