Catalyst Biosciences recently announced that top-line data from the Phase 1/2 trial of CB 2679d/ISU304 in patients with hemophilia B will be presented at the 11th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), Feb. 7-9 in Madrid, Spain.
The oral presentation, titled “Phase 1/2 Trial of Subcutaneously Administered Factor IX Variant CB2679d/ISU304: Pharmacokinetics and Activity,” will be presented by Howard Levy, Catalyst’s chief medical officer, according to a press release.
Patients with hemophilia B lack the clotting protein factor IX (FIX), leading to severe and spontaneous bleeding episodes. Currently, the approved therapies for this disorder require frequent intravenous infusions and are unable to steadily normalize the levels and activity of FIX.
Preclinical studies showed that daily subcutaneous injection of CB 2679d/ISU304, developed in collaboration with South Korea’s ISU Abxis, could normalize FIX levels and that it was significantly more potent than other FIX products.
Catalyst believes the therapy may allow subcutaneous prophylactic, or preventive, treatment of individuals with this disorder by achieving normal FIX activity levels in the blood.
The open-label phase 1/2 trial (NCT03186677) is currently investigating the safety, overall stability, and response of CB 2679d in patients with severe hemophilia B. The study is being conducted at three clinical centers in South Korea and is expected to include 12 patients with moderate to severe hemophilia B.
The trial includes various patient groups with dose variations of a single intravenous delivery of CB 2679d. Another group received daily subcutaneous injections of CB 2679d.
Early results of the first group of treated patients showed that the response induced by intravenous dosing of CB 2679d was 22 times more potent than a similar dose of BeneFix, Pfizer’s standard-of-care replacement therapy. Additionally, CB 2679d remained longer in blood circulation (36 hours) compared to BeneFix (25 hours).
Further results showed that the duration of action of subcutaneous CD 2679d was almost five times greater than intravenous delivery (98.7 hours vs 27.6 hours). So far, no serious adverse events have been reported.
Results from the group of patients who received daily subcutaneous injections of CD 2679d for six days will be presented at EAHAD.
Complete trial data are expected in the first quarter of 2018. Positive results will put CD 2679d one step closer to becoming an available treatment for hemophilia B patients.