Opko and Pfizer Announce Positive Phase 3 Top-line Results For Somatrogon, an Investigational Long-acting Human Growth Hormone

(STL.News) – OPKO Health Inc. (NASDAQ: OPK) and Pfizer Inc. (NYSE: PFE) announced today that the global Phase 3 trial evaluating somatrogon dosed onceweekly in pre-pubertal children with growth hormone deficiency (GHD) met its primary endpoint of non-inferiority to daily GENOTROPIN® (somatropin) for injection, as measured by annual height velocity at 12 months.

Top-line results from the study demonstrated that treatment with somatrogon dosed once-weekly in pre-pubertal children with GHD was non-inferior to somatropin dosed once-daily with respect to height velocity at 12 months of treatment (the primary endpoint); the least square mean was higher in the somatrogon group (10.12 cm/year) than in the somatropin group (9.78 cm/year); the treatment difference (somatrogon – somatropin) in height velocity (cm/year) was 0.33 with a twosided 95% confidence interval of the difference of (-0.39, 1.05).  In addition, change in height standard deviation scores at six and 12 months, key secondary endpoints, were higher in the somatrogon dosed once-weekly cohort in comparison to the somatropin dosed once-daily cohort.  Moreover, at six months, change in height velocity, another key secondary endpoint, was higher in the somatrogon dosed once-weekly cohort in comparison to the somatropin dosed once-daily cohort.  These common measures of growth are employed in the clinical setting to measure the potential level of catch-up growth that subjects may experience relative to heights of age and gender matched peers.

Somatrogon was generally well tolerated in the study and comparable to that of somatropin dosed once-daily with respect to the types, numbers and severity of the adverse events observed between the treatment arms.  Immunogenicity testing and analysis of additional data are ongoing, and full results of the study will be submitted for presentation at a future scientific meeting.

“We are very pleased to announce these favorable top-line results demonstrating the efficacy of somatrogon dosed once-weekly in the pediatric patient population with GHD,” said Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO.  “Somatrogon is a new molecular entity incorporating OPKO’s proprietary long-acting technology.  We believe somatrogon represents a significant advance in the treatment of children with GHD compared to the current standard of one injection per day that could enhance a patient’s adherence to treatment and quality of life.”

“We’re encouraged by these data and look forward to the possibility of bringing this longer-acting therapy to children.  If approved, somatrogon could reduce the daily disease burden on children and their caregivers, potentially increasing treatment adherence,” said Brenda Cooperstone, M.D., Chief Development Officer, Rare Disease, Pfizer Global Product Development.  “At Pfizer, we strive each and every day to advance treatment options that better serve the needs of patients with rare diseases.”

In 2014, Pfizer and OPKO entered into a worldwide agreement for the development and commercialization of somatrogon for the treatment of GHD.  Under the agreement, OPKO is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing the product.