OPKO Health Inc. (NASDAQ: OPK) and Pfizer, Inc. (NYSE:
PFE) announced today that the global Phase 3 trial evaluating
somatrogon dosed once-weekly 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
two-sided 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.
