Data Supporting Use of Predictive Enrichment Markers for Lumos Pharma’s LUM-201 Therapy in Clinical Trials for Moderate PGHD Published in Journal of the Endocrine Society

Data Analysis Supports Use of Predictive Enrichment Markers in Phase 2b OraGrowtH210 Trial
AUSTIN, Texas – Lumos Pharma, Inc., a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, announced that results of peer-reviewed analyses of data from two prior pediatric growth hormone deficiency (PGHD) studies have been published in the Journal of the Endocrine Society (JES). The retrospective studies provide support for the use of predictive enrichment markers (PEMs) in clinical trials to assess the patient population likely to benefit from treatment with LUM-201.

In a manuscript by George M. Bright, MD, et al, entitled, Development of a Predictive Enrichment Marker for Oral GH Secretagogue LUM-201 in Children with Growth Hormone Deficiency, a peer-reviewed analysis of the data from a clinical trial previously conducted by Merck (the Merck 020 study) was performed. The Merck 020 study was a placebo-controlled trial of LUM-201 and recombinant human growth hormone (rhGH) in which all randomized subjects had pretreatment testing, including IGF-1 and peak GH levels determined after a single dose of LUM-201. In subsequent analysis of this data set, two markers suitable for enriching in LUM-201 responsive patients (termed Predictive Enrichment Markers or PEMs) were identified, baseline IGF-1 cut-off level > 30 ng/ml and peak GH level ≥ 5 ng/mL after a single oral dose of LUM-201. The cut-off values for GH were arrived at using a receiver operator characteristic curve or ROC analysis and the IGF-1 cut-offs were determined using an iterative filtering approach. Additionally, those who did not meet our criteria and did not respond well to LUM-201 treatment grew very well on the standard of care, rhGH. The PEMs identified from the analysis of the Merck 020 study data are being used prospectively to select PGHD patients for enrollment in our ongoing OraGrowtH210 Trial.

In a manuscript by Werner F. Blum, PhD, et al, entitled, Corroboration Between Prediction Enrichment Markers for Height Velocity to rhGH and an Oral GH Secretagogue Treatment in Children with Moderate GHD, researchers conducted a data-mining analysis of children with growth hormone deficiency (GHD) in the GeNeSIS legacy database – a prospective, open-label, observational research program conducted by Eli Lilly between 1999 and 2015. The purpose of the data mining analysis was to evaluate if baseline IGF-1 and stimulated peak growth hormone were independent predictors of 12-month height velocity in children with idiopathic growth hormone deficiency. The analysis corroborated the use of these PEMs as meaningful indicators of the severity of GHD and the response to treatment with rhGH, with researchers concluding that “IGF-1 and stimulated GH alone or together are significant indicators of the degree of pediatric GHD, independent of other markers.” This manuscript’s definition of patients with moderate GHD is aligned with the baseline IGF-1 and peak GH levels noted above. This analysis also illustrated that approximately 60% of the total diagnosed pediatric GHD patient population meet our definition of PEM-positive, i.e. they are moderately GHD deficient and are believed likely to respond to LUM-201. Additionally, these data illustrate the differential first-year response to rhGH between the more severely deficient (PEM-negative) patients with a mean height velocity (HV) of 9.6 cm/yr compared to 8.3 cm/yr in the moderately GH deficient patients (PEM-positive). The distinct response the moderate and more severe patient subsets have to rhGH treatment highlights their differing physiologies and alludes to why the moderate group may be more responsive to LUM-201.

“We believe these data support our ongoing Phase 2b OraGrowtH210 Trial design, where we will use these PEMs to select patients and 0.8, 1.6, and 3.2mg/kg LUM-201 doses to confirm our PEM strategy and determine the optimal dose for a Phase 3 registration trial,” said Rick Hawkins, Chairman, CEO and President of Lumos Pharma. “We are confident that the OraGrowtH210 Trial will provide further evidence that oral LUM-201 can provide results for appropriately selected patients without the daily or weekly injections involved with current therapy.”

About Lumos Pharma

Lumos Pharma, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of therapeutics for rare diseases. Lumos Pharma was founded and is led by a management team with longstanding experience in rare disease drug development and received early funding from leading healthcare investors, including Deerfield Management, a fund managed by Blackstone Life Sciences, Roche Venture Fund, New Enterprise Associates (NEA), Santé Ventures, and UCB. Lumos Pharma’s lead therapeutic candidate is LUM-201, an oral growth hormone stimulating small molecule, currently being evaluated in a Phase 2b clinical trial, the OraGrowtH210 Trial, for the treatment of Pediatric Growth Hormone Deficiency (PGHD). If approved by the FDA, LUM-201 would provide an orally administered alternative to daily injections that current PGHD patients endure for many years of treatment. LUM-201 has received Orphan Drug Designation in both the US and EU. For more information, please visit


Lisa Miller
Lumos Pharma Investor Relations
[email protected]