San Francisco-based Catalyst Biosciences has completed toxicology studies of its new drug CB 2679d/ISU304 — a highly potent next-generation coagulation factor to treat hemophilia B. This follows the therapy’s recent Investigational New Drug (IND) approval, a necessary step before starting clinical studies, by the Korean Ministry of Food and Drug Safety.
Patients with hemophilia B lack the clotting protein factor IX. This leads to severe and spontaneous bleeding episodes, as well as longer bleeding times that can become life-threatening.
“Individuals with severe hemophilia B will experience abnormal bleeding, not just after an injury, but spontaneously into potentially any organ, joints or muscles,” Nassim Usman, PhD, president and CEO of Catalyst, said in a press release. “Without preventative treatment or prophylaxis, these individuals may experience several spontaneous bleeding episodes per month.”
Standard treatment requires frequent intravenous infusions of human factor IX. However, adherence and access to peripheral veins can be difficult, often requiring the use of central venous devices that increase the risk of infection and thrombosis. Furthermore, the leading human factor IX on the market has a short half-life and is therefore not ideal for preventative treatment — leading to the need for safer, more efficient therapies.
“Our vision is to prevent bleeding with the stabilization and normalization of clotting levels using more convenient subcutaneous dosing,” Usman said.
Catalyst has been developing CB 2679d/ISU304, a factor IX variant, in collaboration with South Korea’s ISU ABXIS. This project is part of its larger goal of developing novel hematology medicines. Pre-clinical studies in animal models of hemophilia have shown that a daily subcutaneous injection of CB 2679d/ISU304 can normalize human factor IX levels.
ISU ABXIS is now in charge of advancing the factor IX program towards its first human clinical trial. In this regard, it plans to launch a Phase 1/2 study evaluating single and multiple subcutaneous injections of CB 2679d/ISU304 in people with severe hemophilia B in the next few months.