OPKO Biologics to Host Symposium to Present Clinical Data on its Long-acting Human Growth Hormone at the 49th Annual Meeting of the Japanese Society for Pediatric Endocrinology

OPKO Biologics will host a symposium at the 49th Annual Meeting of the Japanese Society for Pediatric Endocrinology and will present the complete 12-month pharmacokinetic, pharmacodynamic, safety and efficacy data of OPKO’s weekly long-acting growth hormone (hGH-CTP) in naïve growth hormone deficient children. The 49th Annual Meeting of the Japanese Society for Pediatric Endocrinology is taking place in Tokyo, Japan from October 8-10.

The data to be presented include results for 53 naïve growth hormone deficient children completing twelve months of weekly treatment with hGH-CTP. This phase 2 study was a one year, dose finding study administering hGH-CTP to growth hormone deficient children once a week using Genotropin® as a daily comparator arm. The twelve months data confirm comparable response of hGH-CTP to daily Genotropin® as reflected by the twelve months IGF-1SDS profile, annual HV, HV SDS and safety profile.

OPKO’s symposium will be held on Friday, Oct. 10, 2015 at 1.30 pm.

OPKO Biologics Presents Clinical Data on its Long-acting Human Growth Hormone (hGH-CTP) in Two Oral Presentations at the 54th Annual Meeting of the European Society for Pediatric Endocrinology (ESPE)

OPKO Biologics presented clinical data from its completed 12-month hGH-CTP Phase 2 pediatric growth hormone deficiency clinical study in two oral presentations at the 54th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE) on October 3, 2015, in Barcelona, Spain. The presentations included pharmacokinetic, pharmacodynamic, safety and efficacy data of OPKO’s hGH-CTP in comparison to a daily Genotropin® arm.

This phase 2 trial was a one-year dose-finding study in which 53 naïve growth hormone deficient children received one of three doses of hGH-CTP once weekly (0.25, 0.48 and 0.66 mg/kg/week) or daily Genotropin® as a comparator arm (34 µg/kg/day). The twelve month data confirmed comparable response of hGH-CTP to daily Genotropin® as reflected by the twelve month pharmacodynamic, efficacy and safety profile.

Following hGH-CTP administration, IGF-1 SDS profile was maintained within the normal range with no accumulation or excessive levels ( > +2 SDS) during the 12 months of treatment.

Among patients treated with hGH-CTP, the average annual height velocity (HV) ranged between 10.5 to 12.5 cm, a comparable response to the daily hGH arm.

In this study, the safety profile of hGH-CTP was also shown to be comparable to daily hGH.

Highlights from the top-line analysis include:

  • There were no patient withdrawals from the study;
  • There were no reports of drug-related serious or unexpected adverse events;
  • There were no clinically significant local tolerability issues; and
  • There were no hGH-CTP neutralizing antibodies.

For all subjects postprandial glucose, insulin and HbA1c (%) levels remained within the normal range throughout the study.

OPKO has a world-wide collaboration agreement with Pfizer Inc. for the development and commercialization of hGH-CTP. Based on the promising phase 2 clinical data, the company confirms its plan to initiate a global pivotal phase 3 study in pre-pubertal GHD children next year following supply of the product by Pfizer in a pen device evaluating a single dose of hGH-CTP versus daily injections of growth hormone.

The presentation are available in the following links:

Presentation #1 – MOD-4023 Safety and Efficacy

Presentation #2 – MOD-4023 PK/PD Modeling 

OPKO Announces Completion of Enrollment in Phase 3 Trial of hGH-CTP in Growth Hormone Deficient Adults

OPKO Health, Inc. has completed enrollment in the single pivotal Phase 3 trial of its long acting human growth hormone (hGH-CTP) in growth hormone deficient (GHD) adults.

The trial is designed to evaluate the safety and efficacy of hGH-CTP with a primary endpoint of superiority compared to placebo in decreasing fat mass in adults with GHD. The trial is a randomized, double-blind, placebo-controlled, multi-center, global study in adults with GHD. The study is divided into two treatment periods: a 26-week, double-blind, placebo-controlled period, followed by a 26-week, open-label extension. For further detail on the Phase 3 study, visit https://clinicaltrials.gov/ct2/show/NCT01909479.

The study is expected to end toward the second half of 2016; regulatory submission will follow study completion.

OPKO Presented New Data at International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress on Its Long-Acting Clotting Factor VIIa

New Preclinical Data further demonstrate the safety and efficacy of MOD-5014 (FVIIa -CTP) for both Intravenous and Subcutaneous Administration

OPKO Health, Inc. presented new data on its long-acting clotting Factor VIIa-CTP (MOD-5014) at the International Society on Thrombosis and Haemostasis Congress (ISTH 2015) held June 20-25, 2015 in Toronto, Ontario, Canada. OPKO’s Factor VIIa-CTP is a next-generation investigational therapy currently in a Phase 2a U.S. based study for the treatment of patients with hemophilia.

Currently, Factor VIIa therapy is available only as an intravenous (IV) formulation, which requires frequent multiple infusions to treat a bleeding episode due to Factor VIIa’s short half-life. In addition, the requirement for multiple weekly infusions can be onerous for patients interested in preventative prophylactic treatment of the disease, especially children.

Previously presented pre-clinical data utilizing both intravenous and subcutaneous administration in hemophilic animal models demonstrated the long acting properties of Factor VIIa-CTP as compared to commercial rFVIIa, with superior half-life, reduced bleeding and increased survival for a significantly longer period.

Five abstracts were accepted for presentation at the ISTH conference. The following abstracts were presented from 5:00-7:00 p.m. local time on June 23-24th.

PO062-TUE: EX-VIVO AND IN-VITRO COMPARATIVE ASSESSMENT OF MOD-5014 (FACTOR VIIA-CTP), A NOVEL LONG-ACTING COAGULATION FACTOR TO RECOMBINANT FVIIA

PO550-TUE: EFFECT OF A CARBOXY-TERMINAL PEPTIDE (CTP) ON FACTOR VIIA ACTIVITY

PO594-WED: COMPREHENSIVE ASSESSMENT OF THE SAFETY AND EFFICACY OF FACTOR VIIA-CTP SUPPORTING PHASE 2A STUDY

PO595-WED: FACTOR VIIA-CTP, A LONG-ACTING COAGULATION FACTOR PROPOSING A NOVEL SUBCUTANEOUS (SC) ROUTE OF ADMINISTRATION FOR PROPHYLACTIC TREATMENT -EFFICACY AND SAFETY IN PREPARATION FOR FIRST IN HUMAN STUDY

PO048-WED: ASSESSMENT OF MOD-5014, A LONG ACTING FVIIA, PHARMACOKINETIC, PHARMACODYNAMICS AND CORRECTION OF HEMOPHILIC COAGULOPATHY IN DOGS WITH HEMOPHILIA A AS PART OF THE PREPARATION FOR FIRST IN HUMAN STUDY

Twelve Months Data Supporting Once-Weekly Dosing of hGH-CTP in Growth Hormone Deficient Pediatric Population was Presented at ENDO 2015 Conference

OPKO Health, Inc. announced the presentation of two posters at the 97th Annual Meeting of the Endocrine Society (ENDO 2015) on March 5th, 2015 in San Diego, California, that included twelve months data from OPKO’s advanced phase 2 trial for its long acting human growth hormone hGH-CTP in growth hormone deficient pediatric subjects.

The presented data included results for 45 patients completing twelve months of treatments. This phase 2 study is a dose finding study administering three different doses of once weekly hGH-CTP comparing to a fixed dose of daily Genotropin® to growth hormone deficient children. The twelve months data confirm comparable response of hGH-CTP to daily Genotropin®, as reflected by the similar twelve months IGF-1SDS profile, annual height velocity (HV), HV SDS and safety profile. The data affirm the selection of an optimal dose that will potentially demonstrate non-inferiority compared to daily hGH in the upcoming phase 3 study in pediatric GHD patients.

OPKO poster presentations included the following:

THR-148: “Top Line Results of 12 Months of Once-Weekly Administration of CTP-Modified Human Growth Hormone (MOD-4023): Phase 2 Dose Finding Study in Children with Growth Hormone Deficiency (GHD)”

THR-151: “Twelve Months Pharmacokinetics and Pharmacodynamics Profile of Once-Weekly, CTP-Modified Human Growth Hormone (MOD-4023): Phase 2 Study in Children with Growth Hormone Deficiency”

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.