OPKO Health Announces Positive Top-Line Results of Phase 3 Efficacy and Safety Study of Somatrogon for the Treatment of Japanese Children with Growth Hormone Deficiency

MIAMI, June 09, 2020 (GLOBE NEWSWIRE) -- Following last October’s announcement of successful results of the global Phase 3 study of once-weekly somatrogon, an investigational agent for the treatment of pediatric growth hormone deficiency (pGHD), OPKO Health Inc. (NASDAQ: OPK) announced today that the Japan Phase 3 clinical trial met its primary and secondary objectives, and demonstrated that the efficacy and safety of somatrogon administered weekly was comparable to GENOTROPIN (somatropin) for injection administered once-daily as measured by annual height velocity after 12 months of treatment in treatment-naïve Japanese pre-pubertal children with GHD. The findings were consistent with the results previously reported in the Phase 3 global study.

Using the Full Analysis Set, the least squared means for the annual height velocity was higher in the somatrogon group (9.65 cm/year) than in the GENOTROPIN group (7.87 cm/year). The point estimate of the treatment difference was 1.79 cm/year with the two-sided 95% confidence interval of (0.97, 2.60). Similarly, greater change in height standard deviation score from baseline at 12 months was observed for the somatrogon group (0.94) compared to the GENOTROPIN group (0.52), indicating that both somatrogon and GENOTROPIN were associated with catch-up growth in treated patients.

Most adverse events were mild to moderate in severity with no notable differences between the treatment groups, and weekly somatrogon administration was generally well-tolerated. A total of six serious adverse events were reported in four patients (two in the somatrogon arm and two in the GENOTROPIN arm). One patient in the GENOTROPIN group discontinued treatment in the study due to adverse events.

“We are pleased to announce positive top-line results from the Japanese Phase 3 clinical study of somatrogon. We have reached an additional clinical development milestone of somatrogon with the demonstration of comparable efficacy and safety of somatrogon administered once-weekly to daily administration of GENOTROPIN in pediatric patients with growth hormone deficiency,” said Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO. “Positive results in both the Japanese and pivotal global Phase 3 pediatric trials are promising for children with pGHD. Treatment with once-weekly somatrogon represents a significant advance, which has the potential to enhance patient adherence and quality of life.”