Catalyst Biosciences Inc. (NASDAQ:CBIO) has announced safety and efficacy results from its Phase 2b study of subcutaneously administered Dalcinonacog Alfa (DalcA).
Catalyst presented DalcA study data at the World Foundation of Haemophilia summit
DalcA is a next-generation Factor IX therapy that the company is developing for hemophilia B treatment. The company presented the data in a poster at the Virtual World Foundation of Haemophilia Summit that took place between June 14 and June 19, 2020. The poster was titled, “Phase 2b Trial to evaluate the safety and factor IX levels of a daily subcutaneous prophylaxis treatment regimen of dalcinonacog alfa in Hemophilia B.”
The study’s data indicated that the administration of daily subcutaneous DalcA dosing after 28 days achieved protective Factor IX target levels of more than 12% in all subjects. There were Factor IX levels of around 27% with a half-life of between 2.5 days and 5.1 days without bleeds. This demonstrated effective prophylaxis as well as the potential of less frequent or lower dosing.
No adverse events reported in the study
One participant withdrew from the study after seven days following an injection site reaction from the first three subcutaneous doses. There were no serious adverse effects as well as neutralizing anti-drug antibodies reported. Some of the participants reported mild ISR of redness and pain more so from the first injections. Thrombotic events did not occur; neither did thrombin-antithrombin, blood coagulation fragment 1+2, or fibrinogen show any signs of prothrombotic effects.
The company’s CEO and President Nassim Usman indicated that the study data demonstrated a clean safety and excellent efficacy profile. All the study participants attained steady-state Factor IX activity levels that exceeded the 12% primary endpoint. Usman added that a small, simple volume subcutaneous therapy to offer protective Factor IX levels is a huge step in Haemophilia B management.
The company designed the trial to evaluate the daily subcutaneous dosing of DalcA and its ability to maintain steady-state Factor IX levels in six severe Haemophilia subjects. The participants received an intravenous does and then followed by daily DalcA subcutaneous doses for 28 days.