OPKO Health Completes Enrollment in Global Phase 3 Study of Somatrogon (hGH-CTP) in Growth Hormone Deficient Children

MIAMI, FL, USA I August 03, 2018 I OPKO Health, Inc. (NASDAQ: OPK) has completed enrollment in the Company’s global pivotal Phase 3 study in growth hormone deficient children, evaluating a single weekly injection of its investigational long-acting human growth hormone product, somatrogon, delivered in a multi-dose disposable pen. 

The somatrogon Phase 3 trial is a randomized, open-label, active-controlled study taking place in over 30 countries.  This study enrolled approximately 225 treatment-naïve children with growth hormone deficiency (GHD) who were randomized 1:1 into two arms: once-weekly somatrogon vs once-daily Genotropin®.  The primary endpoint of the trial is height velocity at 52 weeks. Secondary endpoints are safety and pharmacodynamic endpoints.  Children completing this study may enroll in an open-label long-term extension, in which they will receive somatrogon.

Somatrogon (hGH-CTP) is a new molecular entity that maintains the natural sequence of growth hormone, fused with a C-terminus peptide to extend its half-life.  In a Phase 2 pediatric trial in which growth hormone deficient children were treated with once-weekly injections of somatrogon, somatrogon produced pharmacodynamic effects similar to daily growth hormone replacement therapy with comparable efficacy and safety.  The long-term safety of somatrogon and absence of neutralizing antibodies is confirmed in the ongoing extension, as 43 of the Phase 2 pediatric patients are in their fourth and fifth year of treatment. Somatrogon has received Orphan Drug designation in the U.S. and the EU for the treatment of children and adults with GHD. 

“We are pleased to have completed enrollment for this study, as the pediatric segment represents approximately 80% of the market for the treatment of GHD,” stated Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO Health.  “We are hopeful that the outcome of this study will support a dosing change from daily to weekly administration and positively impact the quality of life for children with GHD.”

OPKO has a worldwide collaboration and license agreement with Pfizer Inc. for the development and commercialization of somatrogon.  Under the agreement, OPKO is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing the product.

About OPKO Health, Inc.
OPKO Health is a diversified healthcare company. In diagnostics, its BioReference Laboratories is the nation's third largest clinical laboratory; GeneDx is a rapidly growing genetic testing business; the 4Kscore® prostate cancer test is used to confirm an elevated PSA to help decide about next steps such as prostate biopsy; Claros® 1 is a point of care diagnostics platform with PSA and testosterone as the most advanced in development.  In our pharmaceutical pipeline, RAYALDEE is our first pharmaceutical product to be marketed.  OPK88003, a once weekly oxyntomodulin for type 2 diabetes and obesity in Phase 2 clinical trials, is among a new class of GLP-1 – glucagon receptor dual agonists.  OPK88004, a SARM (Selective Androgen Receptor Modulator) for treating BPH (Benign Prostatic Hypertrophy), urinary incontinence, and other conditions, is in clinical trials.  The Company’s most advanced product utilizing its CTP technology, a once weekly human growth hormone for injection, is in Phase 3 trials, and is partnered with Pfizer.  OPKO has research, development, production and distribution facilities abroad.  More information is available at www.opko.com.


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