Factor VIIa marzeptacog alfa (activated) – Phase 3 ready

Our most advanced product candidate - MarzAA, a potent, subcutaneously administered, next-generation Factor VIIa variant, is ‘Phase 3 ready’.

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. Our preclinical data has shown that SQ injections of MarzAA given one minute after injury to mirror on-demand therapy significantly reduced bleeding in a mouse model of Hemophilia A. In this setting, reduction of bleeding after SQ administration of MarzAA was as efficient as NovoSeven RT administered IV. Moreover, bleeding was reduced in a dose-dependent manner when MarzAA was given 15 minutes prior to the injury. These data suggest that MarzAA has the potential to be used on-demand for treatment of acute bleeding episodes and supports further clinical testing for on-demand treatment of bleeds in individuals with hemophilia or Factor VII deficiency.

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) Phase 1 trial - completed

We recently completed our Phase 1 MAA-102 study for Marzeptacog alfa (activated) (MarzAA). The results support the use of SQ MarzAA to treat episodic bleeding. We are on track to enroll the first patient in its Phase 3 registration trial evaluating the efficacy of SQ MarzAA to treat bleeding events in individuals with hemophilia A or B with inhibitors in 2020. The Phase 1 study was designed to evaluate the safety, pharmacokinetics and pharmacodynamics of single ascending dose levels of MarzAA and twice and thrice dosing of 60 μg/kg at 3-hourly intervals in individuals with Hemophilia A or B with or without inhibitors. Catalyst reported positive interim data at the 13th Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD) on February 5, 2020.