• Commenced data analysis of the Phase 3 Clinical Study of hGH-CTP in Growth Hormone Deficient Adults
  • Initiated Global Pediatric Phase 3 Clinical Study of hGH-CTP in Growth Hormone Deficient Children

MIAMI, FL, USA I December 30, 2016 I OPKO Health, Inc. (NASDAQ:OPK) announced it has commenced data analysis in the phase 3, double-blind, placebo-controlled study of its investigational long-acting human growth hormone product (hGH-CTP) in adults with growth hormone deficiency (GHD). The multinational, multi-center study, which utilized a 2:1 randomization between hGH-CTP and placebo, enrolled 203 subjects, 198 of whom received at least one dose of study treatment.  Treatment was administered through a weekly injection.

On the primary endpoint of change in trunk fat mass from baseline to 26 weeks, there was no statistical difference between hGH-CTP and placebo.  However, after unblinding the study, OPKO identified one or more outliers that may have affected the primary outcome.  As a result, OPKO is undertaking further review of the study population as promptly as possible.  The safety profile observed in this study was consistent with that known for growth hormone treatments.  A greater percentage of subjects on hGH-CTP normalized serum concentrations of insulin-like growth factor-I compared to placebo.  Additional efficacy and safety data and analyses from the study will be released once available. 

In addition to completing the Phase 3 adult GHD study, OPKO announces that it has initiated a pivotal Phase 3 study this month in pre-pubertal growth hormone deficient children to evaluate weekly treatment with hGH-CTP versus daily injections of Genotropin. The hGH-CTP will be delivered in a pen device in this multi-regional study. 

OPKO has a world-wide collaboration and license agreement with Pfizer Inc. for the development and commercialization of hGH-CTP.  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 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, an FDA-approved treatment for SHPT in stage 3-4 CKD patients with vitamin D insufficiency, VARUBI™ for chemotherapy-induced nausea and vomiting (oral formulation launched by partner TESARO and IV formulation PDUFA date: January 2017), TT401, a once or twice weekly oxyntomodulin for type 2 diabetes and obesity which is a clinically advanced drug candidate among the new class of GLP-1 glucagon receptor dual agonists, and TT701, an androgen receptor modulator for androgen deficiency indications.  Our biologics business includes hGH-CTP, a once-weekly human growth hormone injection (in phase 3 and partnered with Pfizer), a long-acting Factor VIIa drug for hemophilia (in phase 2a) and a long-acting oxyntomodulin for diabetes and obesity (in phase 1). 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.