OPKO Submits Investigational New Drug Application to Initiate a Phase 2a Trial for its Long-Acting Coagulation Factor VIIa-CTP to Treat Hemophilia

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 our product in hemophilic animal models demonstrated its duration of action and significantly increased survival.

“We look forward to beginning clinical trials of Factor VIIa-CTP,” stated Phillip Frost, M.D., Chairman and CEO of OPKO Health. “A longer acting Factor VII administered either by IV or subcutaneous administration, could change the Factor VIIa market by permitting children and adults to easily self-administer at home on a prophylactic basis,” continued Dr. Frost.

Factor VIIa-CTP has been granted orphan drug designation in the U.S. and Europe.