Baseline demographics of the OraGrowtH210 trial studying LUM-201 in idiopathic pediatric growth hormone deficiency (iPGHD) interim analysis data

Abstract

LUM-201 (ibutamoren), an agonist of the growth hormone (GH) Secretagogue Receptor 1a (GHSR1a), is an investigational oral GH secretagogue currently in three iPGHD Phase 2 trials. A LUM-201 predictive enrichment marker (PEM) may be used to select previously diagnosed iPGHD patients likely to respond to LUM-201. PEM positivity is defined as baseline insulin-like growth factor-1 (IGF-1) level >30 ng/mL and a peak GH of ≥ 5 ng/mL in response to a single 0.8 mg/kg oral dose of LUM-201. LUM-201, potent and durable, acts on the GHSR1a which is expressed in the hypothalamus and anterior pituitary. LUM-201 enhances the amplitude of pulsatile releases of GH over 24 hours, while simultaneously acting as a functional antagonist at the somatostatin receptor. In turn, these effects increase the levels of IGF-1, which together with GH, reach the open growth plates and stimulate growth. The OraGrowtH210 Trial is a multi-center Phase 2 trial randomizing 80 iPGHD PEM+ subjects equally to 3 LUM-201 doses (0.8, 1.6, or 3.2 mg/kg/day) or to recombinant human GH (rhGH) at 34 μg/kg/day dose. This abstract depicts the baseline demographics in the protocol-specified six-month interim analysis (IA) subset. OraGrowtH210 Trial IA Baseline Demographics Shown to Impact Growth Summary: Randomization did not result in a balance of growth predictive factors due to small cohort numbers at the IA. The factors predicting improved first year growth (younger age, shorter stature, greater MPH, and higher BMI) occurred in the rhGH cohort (shown in bold). The full data set of 80 subjects should better balance the factors that are related to growth across all four cohorts. The IA 6-month annualized height velocity will be presented at the meeting. (Table Presented) .