Metabolic and Safety Parameters of Once-Weekly, CTP-Modified Human Growth Hormone (MOD-4023): Results of a Phase 2 Dose and Frequency Finding Study

Program: Abstracts - Orals, Featured Poster Presentations, and Posters
Session: SAT 109-133-GHRH, GH & IGF Biology & Signaling
Bench to Bedside
Saturday, June 15, 2013: 1:45 PM-3:45 PM
Expo Halls ABC (Moscone Center)

Poster Board SAT-120
Gili Hart*1, Vera Popovic2, Miklos I Goth3, Peter Vanuga4, Juraj Payer5, Marija Pfeifer6, Martin Bidlingmaier7, Leanne Amitzi8 and Eyal Fima1
1Prolor Biotech, Nes Ziona, Israel, 2Clinical Centre of Serbia, Belgrade, Serbia, 3MIlitary Hospital - State Health, Budapest, Hungary, 4National Endocrinology and Diabetology Institute, Lubochna, Slovakia, 5University Hospital Ruzinov BA, Bratislava, 6University Medical Center, Ljubljana, Slovenia, 7Ludwig-Maximilians University, Munich, Germany, 8PROLOR BIOTECH, Israel
Objective: Growth Hormone (GH) replacement therapy currently requires daily injections, which may cause poor compliance, inconvenience and distress for patients. CTP-modified hGH (MOD-4023) is being developed for once-weekly administration in Growth Hormone Deficient (GHD) adults and children and IGF-1 results have been previously reported (1). The present data summarize the metabolic and safety parameters of MOD-4023 following 16 weeks of treatment in GHD adults.

Design and methods: 54 GHDA patients (46 males and 8 females) who had IGF-1 levels within ±2 SDS during daily hGH replacement were switched to MOD-4023 administered as once-weekly or every-other-week subcutaneous injections to evaluate safety, tolerability and PK/PD profile. The study was comprised of two stages. Stage I was a dose finding period in which the patients were administered MOD-4023 weekly (or every-other-weekly) for 4 weeks. Stage II was a 16 week extension period of once weekly MOD-4023 administration to collect further safety information and confirm the results obtained in Stage I. The results of Stage II are presented here.

Results: During the 16-week treatment period, most of the patients maintained IGF-1 levels within the normal range (±2 SDS). No accumulation of IGF-I was observed over the studied period. Few AEs were attributed to MOD-4023 overall, and were expected following any r-hGH treatment. Laboratory assessments, vital sign assessments, and physical examinations were unremarkable and revealed no unexpected safety concerns. Fasting glucose, fasting insulin and HbA1c were unchanged. Lipid parameters (including Lp(a) were maintained throughout the treatment period.

Conclusions: MOD-4023 once-weekly treatment was shown to be tolerable and safe in GHD adults. The data derived from this study supports long-term once-weekly dosing with this product and will be further confirmed during the pivotal phase 3 study.

(1) Endocr Rev, Vol. 33 (03_MeetingAbstracts): OR29-5

Nothing to Disclose: GH, VP, MIG, PV, JP, MP, MB, LA, EF

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