OPKO Biologics presented clinical data from its completed 12-month hGH-CTP Phase 2 pediatric growth hormone deficiency clinical study in two oral presentations at the 54th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE) on October 3, 2015, in Barcelona, Spain. The presentations included pharmacokinetic, pharmacodynamic, safety and efficacy data of OPKO’s hGH-CTP in comparison to a daily Genotropin® arm.
This phase 2 trial was a one-year dose-finding study in which 53 naïve growth hormone deficient children received one of three doses of hGH-CTP once weekly (0.25, 0.48 and 0.66 mg/kg/week) or daily Genotropin® as a comparator arm (34 µg/kg/day). The twelve month data confirmed comparable response of hGH-CTP to daily Genotropin® as reflected by the twelve month pharmacodynamic, efficacy and safety profile.
Following hGH-CTP administration, IGF-1 SDS profile was maintained within the normal range with no accumulation or excessive levels ( > +2 SDS) during the 12 months of treatment.
Among patients treated with hGH-CTP, the average annual height velocity (HV) ranged between 10.5 to 12.5 cm, a comparable response to the daily hGH arm.
In this study, the safety profile of hGH-CTP was also shown to be comparable to daily hGH.
Highlights from the top-line analysis include:
- There were no patient withdrawals from the study;
- There were no reports of drug-related serious or unexpected adverse events;
- There were no clinically significant local tolerability issues; and
- There were no hGH-CTP neutralizing antibodies.
For all subjects postprandial glucose, insulin and HbA1c (%) levels remained within the normal range throughout the study.
OPKO has a world-wide collaboration agreement with Pfizer Inc. for the development and commercialization of hGH-CTP. Based on the promising phase 2 clinical data, the company confirms its plan to initiate a global pivotal phase 3 study in pre-pubertal GHD children next year following supply of the product by Pfizer in a pen device evaluating a single dose of hGH-CTP versus daily injections of growth hormone.
The presentation are available in the following links: