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Development of a Predictive Enrichment Marker for the Oral GH Secretagogue LUM-201 in Pediatric Growth Hormone
George M Bright1, Minh-Ha T Do1, John C McKew1
1Lumos Pharma, Inc., 4200 Marathon Blvd., Suite 200, Austin, Texas 78756, USA.
Insights
Predictive markers can identify children with growth hormone deficiency (GHD) who will respond to oral growth hormone (GH) secretagogue LUM-201 or recombinant human GH (rhGH). This aids in personalized GHD treatment selection.
Area of Science:
- Pediatric Endocrinology
- Pharmacology
- Biochemistry
Background:
- Growth hormone deficiency (GHD) affects children, with varying responses to treatments.
- Oral growth hormone (GH) secretagogue LUM-201 is a potential treatment option.
- Identifying predictive enrichment markers (PEM) can personalize GHD therapy.
Purpose of the Study:
- To identify pretreatment testing markers for LUM-201 responsiveness in children with GHD.
- To determine if these markers can predict growth responses to LUM-201 and recombinant human GH (rhGH).
Main Methods:
- Analysis of a randomized, placebo-controlled trial involving 68 prepubertal children with GHD.
- Evaluation of pretreatment testing to identify predictive enrichment markers (PEM).
- Assessment of sensitivity, specificity, and predictive accuracy for growth responses to LUM-201 and rhGH.
Main Results:
- Two PEMs identified: baseline insulin-like growth factor I (IGF-I) >30 ng/mL and peak GH response ≥5 ng/mL to LUM-201.
- PEM-positive status predicts better growth response to LUM-201.
- PEM-negative status predicts better growth response to rhGH.
Conclusions:
- Combined baseline IGF-I and peak GH response to LUM-201 are effective PEMs.
- These PEMs predict 6-month growth responses to LUM-201 and rhGH in prepubertal children with GHD.
- Personalized treatment selection for GHD is enhanced by these predictive markers.
Context:
We hypothesize, based on the degree of residual hypothalamic-pituitary function, that some, but not all, children with growth hormone deficiency (GHD) may have beneficial growth responses to the orally administered growth hormone (GH) secretagogue LUM-201.
Objective:
To determine if pretreatment testing can identify predictive enrichment markers (PEM) for subjects with adequate residual function who are responsive to LUM-201.
Methods:
We performed an analysis of a completed, randomized, placebo-controlled trial of LUM-201, a GH secretagogue receptor agonist, in which all randomized subjects had pretreatment testing. This international multicenter study conducted in pediatric endocrinology clinics included 68 naïve-to-treatment, prepubertal children with established diagnoses of GHD. Outcome measures included the sensitivity, specificity, and predictive accuracy of potential markers to predict 6-month growth responses to oral LUM-201 and daily rhGH.
Results:
Two PEM were identified for use in defining PEM-positive status: (1) baseline insulin-like growth factor I (IGF-I) concentration >30 ng/mL and (2) peak GH response of ≥5 ng/mL upon administration of single-dose LUM-201. PEM-positive status enriches a population for better growth responses to LUM-201. PEM-negative status enriches a population for better growth responses to rhGH.
Conclusion:
Combined, the peak GH response to single-dose LUM-201 and the baseline IGF-I concentration are effective PEMs for 6-month growth responses to LUM-201 and rhGH in prepubertal children with GHD.
