Rare Disease Report

Catalyst Biosciences Initiates Phase 2 of Potential Hemophilia A and B Therapy

JANUARY 04, 2018
Mathew Shanley
This morning, Catalyst Biosciences announced the initiation and open enrollment of the Phase 2 part of its Phase 2/3 program of marzeptacog alfa (activated) (MarzAA), a highly potent, subcutaneously administered Factor VIIa therapy in development for the treatment of hemophilia A and B with inhibitors.

This Phase 2 open-label, subcutaneous efficacy trial in individuals with hemophilia A or B with inhibitors is intended to evaluate the ability of MarzAA to eliminate – or minimize – spontaneous bleeding episodes. The study has a primary endpoint of reduction in annualized bleed rate that will be compared with each individual’s historical annualized bleed rate as the control.

MarzAA is being developed for prophylaxis in hemophilia A or B with inhibitors. Data from the Phase 1 in 25 individuals with severe hemophilia, with and without inhibitors, showed that MarzAA demonstrated exceptional safety and tolerability.

“For individuals with hemophilia B with inhibitors there are no approved subcutaneous therapies, and a recently approved subcutaneous treatment for hemophilia A with inhibitors has safety concerns,” said Nassim Usman, Ph.D., president and chief executive officer of Catalyst in a press release. “With the initiation of this Phase 2 MarzAA trial, Catalyst is now conducting two clinical studies with subcutaneous candidates for individuals with hemophilia, and we are one step closer to potentially providing safe and convenient treatment options using high potency replacement coagulation factors to restore coagulation.”

Additionally, safety and tolerability of daily subcutaneous dosing and potential inhibitor formation will be monitored, and the trial will enroll up to 12 individuals with hemophilia and an inhibitor across approximately 10 clinical trial sites globally.

MarzAA 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.

Interim data is expected to be announced in the first half of 2018.

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