Catalyst Biosciences announces publication of hemophilia Phase 1 data

Catalyst Biosciences announced the peer-reviewed publication of previously reported data from the Phase 1 trial of marzeptacog alfa in individuals with hemophilia A or B with or without inhibitors in the Journal of Thrombosis and Haemostasis.The paper, entitled: “Phase 1, single-dose escalating study of marzeptacog alfa, a recombinant factor VIIa variant, in patients with severe hemophilia,” details the safety, tolerability, pharmacokinetics and pharmacodynamics of single ascending intravenous bolus doses of MarzAA. Subjects in the international Phase 1 open-label study were assigned to single dose MarzAA cohorts. MarzAA showed linear dose-response PK across the 4.5-30 mug/kg dose range, with a terminal half-life of 3.5 hours. Dose-dependent shortening of activated partial thromboplastin time and prothrombin time, and an increase in peak thrombin, determined with a thrombin generation assay, was also observed. MarzAA was well tolerated at all dose levels and was not associated with dose-limiting toxicity. No treatment-emergent severe or serious adverse events occurred.”MarzAA showed favorable pharmacological data in this first-in-human study and no potential safety concerns were identified. Together, these results supported further examination of MarzAA for the treatment of hemophilia A or B with inhibitors, particularly via subcutaneous administration,” said Ralph Gruppo, M.D., lead study author.
https://thefly.com/landingPageNews.php?id=2785147