According to Hemophilia News Today, data collected from a Phase 2b trial highlights that the subcutaneously administered DalcA is effective for the treatment of hemophilia B. Find the full press release from Catalyst Biosciences, who developed DalcA, on their website.
Hemophilia is a rare bleeding disorder in which the blood doesn’t clot properly, causing issues with wound healing. People with hemophilia may bleed longer than others after an injury. This is because their blood does not have enough clotting factors, or proteins needed to control bleeding and coagulation.
People with hemophilia B have a deficient factor IX gene. So, many treatments try to replenish the levels of factor IX within the blood. Some people with hemophilia B will experience less bleeding episodes as they age. Symptoms include nosebleeds, bruising, unprovoked bleeding, urinary and gastrointestinal bleeding, and continued bleeding after injuries or surgeries. Find out more about hemophilia B.
DalcA Trial Results
DalcA stands for dalcinonacog alfa. It is lab-developed factor IX, which can be injected under the skin as a preventative treatment for severe bleeding. Many other hemophilia treatments require intravenous injections. As a result, DalcA is less intrusive and time-consuming for patients.
Previously, a Phase 1/2 study found DalcA to be longer lasting and 22x more potent than other factor IX treatments. In particular, this study compared DalcA to BeneFIX, a factor IX replacement therapy from Pfizer.
This most recent open-label Phase 2b trial followed 6 patients (all male) with acute hemophilia B. Patients received an initial dose of 50 IU/kg DalcA intravenously. Next, patients received 100 IU/kg of DalcA subcutaneously for 28 days.
Final results will be published in June. However, data collected thus far highlights that DalcA raised factor IX in the blood by up to 27% within a 2-week period. The treatment’s half-life was between 70-112 hours. If compared to alternative therapies, researchers could see if this would allow for patients to need less frequent prophylactic treatment.
Finally, researchers found DalcA to be fairly safe and effective. Although two patients experienced side effects, neither developed any additional complications.