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55th Annual ESPE

Paris, France
10 Sep 2016 - 12 Sep 2016

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Paris, France: 10-12 September 2016 Further information

Rapid Free Communications

Growth: Clinical

hrp0086rfc8.1 | Growth: Clinical | ESPE2016

Somavaratan (VRS-317) Treatment of Children with Growth Hormone Deficiency (GHD): Results at 2 Years (NCT02068521)

Bright George , Moore Wayne V. , Nguyen Huong Jil , Kletter Gad B. , Miller Bradley S. , Fechner Patricia Y. , Ng David , Humphriss Eric , Cleland Jeffrey L.

Background: Somavaratan, a novel long-acting rhGH fusion protein with t1/2>100 h, previously demonstrated clinically meaningful improvements in height velocity (HV) and IGF-I in prepubertal GHD children (Moore JCEM 2016).Objective and hypotheses: To evaluate maintenance of somavaratan treatment effects in the 2nd treatment year.Method: After subcutaneous pediatric doses were evaluated in a single dose PK/PD study (<e...

hrp0086rfc8.2 | Growth: Clinical | ESPE2016

Pharmacokinetic Modelling Predicts Native hGH Levels Following Administration of a Sustained-Release Prodrug, TransCon hGH, to Children with GHD

Sprogoe Kennett , Beckert Michael , Christoffersen Eva Dam , Gilfoyle David , Wegge Thomas

Background: TransCon Growth Hormone is a once-weekly sustained-release prodrug of recombinant human growth hormone (hGH, somatropin). Based on the inert TransCon prodrug technology unmodified hGH is released with a Cmax and AUC comparable to daily therapy. TransCon hGH leverages the known pharmacology of daily hGH and is being developed for the treatment of growth hormone deficiency (GHD).Objective and hypotheses: Both hGH levels and resulting IGF-I leve...

hrp0086rfc8.3 | Growth: Clinical | ESPE2016

Batch-to-Batch Consistency of a Highly O-Glycosylated Long-Acting Human Growth Hormone (MOD-4023)

Moschcovich Laura , Guy Rachel , Felikman Yana , Zakar Miri , Hershkovitz Oren

Background: OPKO Biologics is a clinical-stage public company developing long-acting therapeutic proteins utilizing CTP technology. The technology involves fusion of the C-terminal peptide of human chorionic gonadotropin (hCG), which is highly O-glycosylated, to the target protein. CTP enabled the production of a long-acting human growth hormone (hGH) (MOD-4023), which supports a single weekly injection in growth hormone-deficient patients. MOD-4023 is manufactured as a non-vi...

hrp0086rfc8.4 | Growth: Clinical | ESPE2016

A Hybrid Fc-fused Human Growth Hormone, GX-H9, Shows a Potential for Weekly and Semi-monthly Administration in Clinical Studies

Lee EunJig , Schopohl Jochen , Mykola Aryaev , Kim Tae Kyung , Ahn Young-Joo , Woo Jung-Won , Jang Woo Ick , Sung Young-Chul , Keyoung H. Michael

Background, Objective and hypotheses: GX-H9 is a hybrid Fc-based long-acting recombinant human growth hormone (hGH). The safety, tolerability, and PK/PD of single ascending dose in healthy volunteers were assessed to determine GX-H9 doses capable of normalizing IGF-1 level. The PK/PD, safety, efficacy and tolerability of multiple sequential doses of GX-H9 in adult growth hormone deficiency (AGHD) were compared to that of a daily recombinant hGH in AGHD. A Phase 2 study in pedi...

hrp0086rfc8.5 | Growth: Clinical | ESPE2016

Optimal Sampling of IGF-1 During Weekly Administration of a Long Acting Human Growth Hormone (MOD 4023)

Fisher Dennis M. , Mendelson Michal Jaron , Vander Shelly , Koren Ronit , Hart Gili

Background: OPKO Biologics is developing MOD-4023, a long-acting growth hormone (GH), intended for weekly dosing for the treatment of idiopathic GH deficiency in children. At ESPE2015, we presented pharmacokinetic (PK) and pharmacodynamic (PD, based on IGF-1) models for weekly MOD-4023 administration in children aged 3–11 years. These models confirm that IGF-1 (and IGF-1 SDS) varies during the dosing interval. One critical clinical and research issue is when to optimally ...

hrp0086rfc8.6 | Growth: Clinical | ESPE2016

A Six-Month Safety and Efficacy Study of TransCon hGH Compared to Daily hGH in Pre-Pubertal Children with Growth Hormone Deficiency (GHD)

Chatelain Pierre , Malievsky Oleg , Radziuk Klaudziya , Senatorova Ganna , Beckert Michael

Background: TransCon hGH is a long-acting prodrug of recombinant human growth hormone (hGH) that releases fully active unmodified hGH into the blood compartment. This presentation will detail the final safety and efficacy results of TransCon hGH in a Phase 2 study in children with GHD over a treatment period of six months.Objective and hypotheses: The objective of this Phase 2 study in GHD was to investigate 1) safety and tolerability, 2) pharmacokinetic...

hrp0086rfc8.7 | Growth: Clinical | ESPE2016

Safety and Tolerability of Once-Weekly Administration of CTP-Modified Human Growth Hormone (MOD-4023): 24-month Complete Dataset Results of a Phase 2 Study in Children with Growth Hormone Deficiency

Zelinska Nataliya , Skorodok Julia , Malievsky Oleg , Rosenfeld Ron G. , Zadik Zvi , Koren Ronit , Vander Shelly , Hart Gili , Radziuk Klaudziya

Background: Daily injections are currently required for growth hormone (GH) replacement therapy, which may cause poor compliance, inconvenience and distress for patients. MOD-4023 is a CTP-modified human GH (hGH) developed for once-weekly administration in growth hormone deficient (GHD) adults and children.Objective and hypotheses: In the present Phase 2 study, the safety and tolerability of once-weekly subcutaneous (SC) administration of MOD-4023 were a...

hrp0086rfc8.8 | Growth: Clinical | ESPE2016

Efficacy of Once-Weekly Administration of CTP-Modified Human Growth Hormone (MOD-4023): 24-month Complete Database Results of a Phase 2 Study in Children with Growth Hormone Deficiency

Zelinska Nataliya , Skorodok Julia , Malievsky Oleg , Rosenfeld Ron G. , Zadik Zvi , Koren Ronit , Vander Shelly , Hart Gili , Raduk Dmitri

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