ESPE Abstracts (2016) 86 RFC8.6

ESPE2016 Rapid Free Communications Growth: Clinical (8 abstracts)

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

Pierre Chatelain a , Oleg Malievsky b , Klaudziya Radziuk c , Ganna Senatorova d & Michael Beckert e

aDiv. Endocrinologie Pediatrique - Hopital Mere-Enfant, University Claude Bernard Lyon1, Lyon, France; bBashkir State Medical University, Ufa, Russia; c2nd Children City Clinic, Minsk, Belarus; dKharkiv National Medical University, Kharkiv, Ukraine; eAscendis Pharma A/S, on behalf of the TransCon hGH Study Group, Hellerup, Denmark

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) pharmacokinetics (PK) and pharmacodynamics and 3) efficacy of TransCon hGH in children with Growth Hormone Deficiency (GHD).

Method: Pre-pubertal, treatment naïve GHD children received s.c. injections of one of three once-weekly TransCon hGH doses (0.14, 0.21 and 0.30 mg hGH/kg per week) or daily hGH (0.03 mg hGH/kg per day=0.21 mg/kg per week) over a 6-month treatment period, in a randomized, comparator-controlled Phase 2 study. The patients’ GHD diagnosis was established in accordance with international consensus guidelines, based on auxology (height & height velocity), GH stimulation tests & IGF-I.

Results: Safety and efficacy (annualized height velocity), as well as PK and PD data of 53 GHD patients treated over a 6-month period with TransCon hGH or daily hGH will be presented. All TransCon hGH doses demonstrated an excellent safety/local tolerability profile (comparable to daily hGH) and an excellent growth - mean annualized height velocities ranging between 11.9 cm to 13.9 cm for the different dose levels of TransCon hGH compared to 11.6 cm mean annualized height velocity for daily hGH treatment.

Conclusion: TransCon hGH has demonstrated efficacy (auxology) and safety comparable to that observed with daily hGH. Observed injection site reactions are similar to what is expected with daily hGH injections, with no nodule formation or lipoatrophy noted. The low immunogenicity, comparable to daily hGH treatments was confirmed. Hence, this TransCon hGH Phase 2 study conducted in a pediatric population with GHD confirmed the very good safety and efficacy profile of TransCon hGH, an hGH prodrug, and forms the basis for Phase 3 development.

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