South Korea Approves Catalyst’s IND Application for Hemophilia B Blood-clotting Treatment

South Korea has approved an Investigational New Drug (IND) application for Catalyst Biosciences’ experimental therapy CB 2679d/ISU304, a highly potent next-generation coagulation Factor IX variant for the treatment of hemophilia B.

Catalyst focuses on developing medications for hematology conditions (blood disorders), which include coagulation factors that can prevent spontaneous bleeding in patients with hemophilia, even during surgery.

“We believe that stopping bleeding is good, but preventing bleeding is better,” Dr. Nassim Usman, president and CEO of Catalyst, said in a press release. “CB 2679d/ISU304 has the properties required, including high potency, to allow for daily subcutaneous injection with the potential to achieve stable and normal Factor IX clotting levels.”

In November 2016, Catalyst Biosciences reported positive preclinical results showing that when CB 2679d was administrated subcutaneously, or beneath the skin, in mice with hemophilia B, it led to a dose-dependent increase in the coagulation Factor IX (FIX) antigen, resulting in a coagulation potency 17 times higher when compared to another FIX product (BeneFix).

In a few months, Catalyst and its collaborator ISUS Abxis (a South Korea-based company) will begin a proof-of-concept Phase 1/2 clinical trial to evaluate CB 2679d in patients with hemophilia B using single and multiple subcutaneous injections.

After the Phase 1/2 study, ISU Abxis will have exclusive commercial rights in South Korea and may be eligible for profit sharing on commercialization outside of South Korea. Catalyst will retain full commercial and development rights outside of South Korea for CB 2679d/ISU304.

Currently available coagulation factors are injected directly into a patient’s veins; clotting factors that can be injected just under patient’s skin may improve treatments.

Catalyst is also planning another clinical trial in hemophilia inhibitor patients using its next-generation coagulation Factor VIIa (activated marzeptacog alfa; formerly known as CB 813d). Marzeptacog alfa (activated) has shown promise in a Phase 1 trial in severe hemophilia A and B patients who are or aren’t taking inhibitor medication, according to company presentations in February.

“We are very pleased with the progress we and our collaborator ISU Abxis have made in advancing this Factor IX candidate towards the clinic and we look forward to the initiation of a Phase 1/2 clinical trial next quarter,” Usman said.