Enrollment completed in Phase III trial of somatrogon to treat growth hormone deficient children.- OPKO Health Inc.

OPKO Health, Inc. has completed enrollment in the Company’s global pivotal Phase III 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 III 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 II 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 II 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.

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.