OPKO Health, Inc. announced the submission of an Investigational New Drug Application (IND) to the U.S. Food and Drug Administration (FDA) to conduct a Phase 2a study of 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. Factor VIIa-CTP is a novel, long-acting recombinant Factor VIIa utilizing OPKO's proprietary technology to extend its circulatory half-life without the use of polymers, encapsulation techniques, or nanoparticles. The technology is based on a naturally occurring peptide, the C-terminal peptide (CTP) of the beta chain of human chorionic gonadotropin.

The CTP technology is also used in OPKO's hGH-CTP, its long-acting recombinant human growth hormone product which is being evaluated in Phase 3 clinical trials for adults and Phase 2 trials for children with growth hormone deficiencies. OPKO recently announced a global agreement with Pfizer for the development and commercialization of hGH-CTP. 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 addition, frequent infusions are onerous when used as preventative prophylactic therapy, especially for children. Pre-clinical studies of intravenous and subcutaneous formulations of its product in hemophilic animal models demonstrated its duration of action and significantly increased survival.