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, acquired hemophilia and other bleeding disorders. 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 both subcutaneous prophylactic treatment and intravenous acute treatment. The Company initiated a Phase 2/3 subcutaneous prophylaxis efficacy trial in the first quarter of 2018.

Factor VIIa marzeptacog alfa (activated) Phase 2/3 efficacy trial - ongoing

The Phase 2 portion of the subcutaneous efficacy trial (NCT03407651) is an open-label, dose escalation study in individuals with hemophilia A or B with inhibitors. The objective of the trial is 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 top-line results by the end of 2018.

Catalyst recently announced positive interim results from this clinical trial of marzeptacog alfa (activated) in individuals with hemophilia A or B with inhibitors. Three subjects have successfully completed long-term daily dosing with marzeptacog alfa (activated). One subject with a historic annualized bleed rate of 26.7, or one bleed approximately every two weeks, completed the trial with no bleeds after 50 days of treatment with 60 µg/kg marzeptacog alfa (activated). A second subject, who had a historic annualized bleed rate of 16.6, had no bleeds during treatment with 30 µg/kg marzeptacog alfa (activated) for 50 days. The last subject, who had a historic annualized bleed rate of 15.9, had no bleeds during treatment with 30 µg/kg marzeptacog alfa (activated) for 44 days. No anti-drug antibodies or injection site reactions have been detected to date, after more than 200 subcutaneous doses of marzeptacog alfa (activated).

Factor VIIa marzeptacog alfa (activated) efficacy trial design

MarzAA Phase 2 image

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, showing favorable safety, tolerability and pharmacological data after single intravenous doses of marzeptacog alfa (activated), were originally reported from this open label, multicenter Phase 1 clinical trial at the International Society on Thrombosis and Haemostasis (ISTH) meeting in 2015 and were recently published in the Journal of Thrombosis and Hemostasis.

Marzeptacog alfa (activated) has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for routine prophylaxis to prevent bleeding episodes in individuals with hemophilia A or B with inhibitors.