Lumos Pharma Reaches 50% Randomization Milestone in Phase 2 OraGrowtH210 Trial Evaluating Oral LUM-201 in PGHD

• Interim Data Anticipated by End of 2022   -
• Interim Data from PK/PD OraGrowtH212 Trial also Anticipated by End of 2022 -

Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, announced this morning achievement of the 50% randomization milestone for our Phase 2 OraGrowtH210 Trial evaluating orally administered LUM-201 in pediatric growth hormone deficiency (PGHD). Interim data from both the OraGrowtH210 and OraGrowtH212 Trials are anticipated by the end of 2022.

“We are pleased to have reached an important milestone in our trials evaluating oral LUM-201 in PGHD despite the severe challenges posed by conditions from the Covid-19 pandemic worldwide,” commented Rick Hawkins, CEO and Chairman of Lumos Pharma. “The pace of screening and enrollment for these trials in recent months has been encouraging, and we look forward to the release of interim data for both trials by the end of 2022. We believe the interim data should provide an early indication of efficacy and safety of oral LUM-201 versus standard of care in PGHD.”

The OraGrowtH210 Trial is a multi-site, global trial evaluating orally administered LUM-201 at three dose levels (0.8, 1.6, 3.2 mg/kg/day) against a standard dose of injectable rhGH in approximately 80 subjects diagnosed with idiopathic PGHD, which is less severe than organic PGHD. The objective of this trial is to identify the optimal dose of LUM-201 to be used in a Phase 3 registration trial, based on annualized height velocity from a 6-month dataset, and to prospectively confirm the preliminary validation of our Predictive Enrichment Marker (PEM) strategy. The interim analysis will evaluate the safety and annualized height velocity of the three dose levels of LUM-201 against a standard dose of injectable recombinant human growth hormone (rhGH) in 40 subjects at six months on therapy. The complete set of 6-month, primary outcome data for OraGrowtH210 Trial is anticipated in the second half of 2023.

The OraGrowtH212 Trial is a single site, open-label trial evaluating the pharmacokinetic (PK) and pharmacodynamic (PD) effects of oral LUM-201 in up to 24 PGHD subjects at two dose levels, 1.6 and 3.2 mg/kg/day. The objective of the OraGrowtH212 Trial is to confirm prior clinical data demonstrating the amplified pulsatile release of endogenous growth hormone unique to LUM-201 and its potential for this mechanism of action to contribute to its efficacy in PGHD. The primary endpoint for this trial is six months of PK/PD and height velocity data in up to 24 subjects. Interim data on a minimum of 10 subjects is anticipated by the end of 2022.

Lumos Pharma intends to analyze these interim datasets separately and also perform a combined analysis to increase the number of subjects in the top two LUM-201 dose cohorts. This combined interim analysis of approximately 50 subjects from both the OraGrowtH210 and OraGrowtH212 Trials should provide a robust data package from which to obtain an early indication of the potential of oral LUM-201 in PGHD.

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 2 clinical trial, the OraGrowtH210 Trial, and a PK/PD trial, the OraGrowtH212 Trial, for the treatment of Pediatric Growth Hormone Deficiency (PGHD). If approved by the FDA, LUM-201 would provide an orally administered alternative to recombinant growth hormone injections that PGHD patients otherwise endure for many years of treatment. LUM-201 has received Orphan Drug Designation in both the US and EU.