MIAMI, FL, USA I February 23, 2016 I OPKO Health, Inc. (NYSE: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.
About Factor VIIa-CTP
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.
Factor VIIa-CTP has been granted orphan drug designation in the U.S. and Europe.
About OPKO Health, Inc.
OPKO Health, Inc. is a diversified healthcare company that seeks to establish industry-leading positions in large, rapidly growing markets. Our diagnostics business includes Bio-Reference Laboratories, the nation’s third-largest clinical laboratory with a core genetic testing business and a 420-person sales force to drive growth and leverage new products, including the 4Kscore® prostate cancer test and the Claros®1 in-office immunoassay platform. Our pharmaceutical business features Rayaldee™, a treatment for SHPT in stage 3-4 CKD patients with vitamin D insufficiency (March 29, 2016 PDUFA date) and VARUBI™ for chemotherapy-induced nausea and vomiting (oral formulation launched by partner Tesaro, IV formulation in Phase 3). Our biologics business includes hGH-CTP, a once-weekly human growth hormone injection (in Phase 3 and partnered with Pfizer), and a long-acting Factor VIIa drug for hemophilia (entering Phase 2a). We also have production and distribution assets worldwide, multiple strategic investments and an active business development strategy. More information is available at www.opko.com.
SOURCE: OPKO Health
Post Views: 214
MIAMI, FL, USA I February 23, 2016 I OPKO Health, Inc. (NYSE: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.
About Factor VIIa-CTP
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.
Factor VIIa-CTP has been granted orphan drug designation in the U.S. and Europe.
About OPKO Health, Inc.
OPKO Health, Inc. is a diversified healthcare company that seeks to establish industry-leading positions in large, rapidly growing markets. Our diagnostics business includes Bio-Reference Laboratories, the nation’s third-largest clinical laboratory with a core genetic testing business and a 420-person sales force to drive growth and leverage new products, including the 4Kscore® prostate cancer test and the Claros®1 in-office immunoassay platform. Our pharmaceutical business features Rayaldee™, a treatment for SHPT in stage 3-4 CKD patients with vitamin D insufficiency (March 29, 2016 PDUFA date) and VARUBI™ for chemotherapy-induced nausea and vomiting (oral formulation launched by partner Tesaro, IV formulation in Phase 3). Our biologics business includes hGH-CTP, a once-weekly human growth hormone injection (in Phase 3 and partnered with Pfizer), and a long-acting Factor VIIa drug for hemophilia (entering Phase 2a). We also have production and distribution assets worldwide, multiple strategic investments and an active business development strategy. More information is available at www.opko.com.
SOURCE: OPKO Health
Post Views: 214
