OPKO Health Announces Dosing of First Patient in Phase 2a Clinical Study of a Long-Acting Factor VIIa for the Treatment of Hemophilia

OPKO Health, Inc. OPK today announced dosing of the first patient in a Phase 2a study evaluating the safety of a long-acting Factor VIIa in hemophilia patients. The Phase 2a study is a dose escalation study to determine safety and explore efficacy endpoints in patients with OPKO's long-acting version of coagulation Factor VIIa (Factor VIIa-CTP) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX. The study is intended to enroll 24 patients in the United States. "This is a significant step in the development of another drug candidate utilizing our CTP technology," commented Phillip Frost, MD, Chairman and Chief Executive Officer of OPKO. "Later this year, we expect to initiate a second Phase 2a trial for Factor VIIa-CTP in a subcutaneous formulation that, if successful, could provide prophylactic therapy to prevent bleeding episodes and thereby change the treatment paradigm for patients with hemophilia." Currently, Factor VIIa therapy is available only as an intravenous (IV) formulation which, due to Factor VIIa's short half-life, requires multiple infusions to treat a bleeding episode in hemophilia A or B patients with inhibitors. In addition, frequent infusions are onerous when used as prophylactic therapy, especially for children. Pre-clinical studies of intravenous and subcutaneous formulations of our product in hemophilic animal models demonstrated its duration of action and significantly increased survival.