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. At Catalyst, we anticipate that marzeptacog alfa (activated) could be used for both subcutaneous prophylactic treatment and subcutaneous or intravenous acute treatment of bleeds.

Factor VIIa marzeptacog alfa (activated) Phase 2 efficacy trial - completed

Final results from all nine subjects that completed dosing in the Phase 2 open-label trial were presented at the International Society for Thrombosis & Hemostasis (ISTH) meeting in July 2019. The Phase 2 trial of our subcutaneous (SQ) Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA) for prophylaxis met the primary endpoint of significantly reducing the annualize bleed rate (ABR) in patients with hemophilia A or B with inhibitors. The study also met all secondary endpoints of safety, tolerability and lack of anti-drug antibody or inhibitor formation.

The objective of the trial was to eliminate, or minimize, spontaneous bleeding episodes by using individualized dose escalation, if a spontaneous bleeding event occurs. The primary endpoint was a reduction in annualized bleed rate (ABR) at the final dose level. Each individual’s recorded ABR for 6 months prior to treatment served as the control. A secondary endpoint compared the proportion of days with bleeding recorded during the prior 6 months with the proportion of any bleeding days during marzeptacog alfa (activated) (MarzAA) treatment.

Factor VIIa marzeptacog alfa (activated) efficacy trial design

MarzAA Phase 2 image

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

We initiated a phase 1 subcutaneous dose escalation study. This study will evaluate the pharmacokinetics and pharmacodynamics in individuals with hemophilia A or B with or without inhibitors to determine if the timing of the peak levels achieved are sufficient to treat a breakthrough bleed with subcutaneous dosing.