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Endocrine Abstracts (2016) 41 GP139 | DOI: 10.1530/endoabs.41.GP139

ECE2016 Guided Posters Paediatric Endocrinology & Development (10 abstracts)

2nd year efficacy results of once-weekly administration of CTP-modified human growth hormone (MOD-4023): a phase 2 study in children with growth hormone deficiency

Nataliya Zelinska 1 , Julia Skorodok 2 , Oleg Malievsky 3 , Ron G Rosenfeld 4 , Zvi Zadik 5 , Ronit Koren 6 , Leanne Amitzi 6 , Oren Hershkovitz 6 , Gili Hart 6 & Klaudziya Radziuk 7

1Ukrainian Children Specialized Clinical Hospital, Kyev, Ukraine; 2St Petersburg State Pediatric Medical Academy, St Petersburg, Russia; 3Bashkir State Medical University, Ufa, Russia; 4Oregon Health and Science University, Oregon, USA; 5Kaplan Medical Center, Rehovot, Israel; 6OPKO Biologics, Nes Ziona, Israel; 72nd Children City Clinic, Minsk, Belarus.

Objective: Growth hormone (GH) replacement therapy currently requires daily injections. This may cause poor compliance, inconvenience and distress for patients. CTP-modified hGH (MOD-4023) has been developed for once-weekly administration in growth hormone deficient (GHD) adults and children. The 18 and 24 month efficacy of once-weekly subcutaneous (SC) administration of MOD-4023 was evaluated in a Phase 2 study in children with GH deficiency.

Design and methods: A one year, randomized, controlled Phase 2 study was conducted in 53 pre-pubertal children with GHD receiving once-weekly SC injections of one of three MOD-4023 doses (0.25, 0.48, and 0.66 mg/kg per week) vs daily hGH (34 μg/kg/day). Forty-six subjects were rolled over to continue with the same MOD-4023 dose in an open-label extension (OLE), which will routinely assess growth until marketing approval. Height velocity (HV) results during the 2nd year of MOD-4023 treatment for 45 patients were compared to historical controls (1). IGF-1 and IGFBP-3 were monitored as well.

Results: The analysis included 2nd year height velocity data for 45 patients. All three doses of MOD-4023 demonstrated promising 2nd year growth, while the two higher doses of MOD-4023 resulted in better growth in comparison to the lower dose of MOD-4023 (0.25 mg/kg per week), and in line with reported age- and GHD severity-matched historical control (1).

Conclusions: The efficacy of single weekly administration of MOD-4023 for the treatment of pediatric GHD patients was further confirmed during the 2nd year of treatment as part of the OLE of a Phase 2 study. This further affirms that a single weekly injection of MOD-4023 has the potential to replace daily hGH injections in children with GHD and provides additional efficacy data to support dose selection for OPKO’s upcoming Phase 3 trial.

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