Date: 2016-02-23
Type of information: Treatment of the first patient
phase: 1-2
Announcement: treatment of the first patient
Company: Opko Health (USA - Fl)
Product: MOD-5014 (Factor VIIa-CTP)
Action
mechanism: protein/coagulation factor. 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 .
Disease: hemophilia A, hemophilia B
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Country: USA
Trial
details: This Phase 1/2a single dose, dose-escalating study is evaluating the acute safety, pharmacokinetics (PK) and pharmacodynamics (PD) properties of MOD-5014 in adult subjects with moderate/severe congenital hemophilia A or B. This will be a single-dose, open label, dose-escalating study. Each dose cohort will be concluded by a safety review, following which escalation to the next dose cohort will be approved. (NCT02418793)
Latest
news: * On February 23, 2016, OPKO Health 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 . * On January 30, 2015, OPKO Health announced the submission of an Investigational New Drug Application (IND) to the 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. 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.
