On November 7, 2023 Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, reported that topline results met primary and secondary endpoints for its Phase 2 Dose-Finding OraGrowtH210 Trial and Phase 2 Pharmacokinetic/Pharmacodynamic (PK/PD) OraGrowtH212 Trial evaluating oral LUM-201 for subjects with moderate pediatric growth hormone deficiency (PGHD) who screened PEM-positive utilizing Lumos Pharma’s predictive enrichment marker (PEM) strategy (Press release, Lumos Pharma, NOV 7, 2023, View Source [SID1234637146]). Lumos also announced its financial results for the quarter ended September 30, 2023.

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"With the end of Phase 2 readout from our OraGrowtH210 and OraGrowtH212 trials announced today, we are thrilled that these data support advancing our clinical program towards a pivotal Phase 3 trial for potentially the first oral therapeutic for moderate PGHD," said Rick Hawkins, Chairman and CEO of Lumos Pharma. "Data confirm the optimal dose is 1.6 mg/kg/day LUM-201 to advance to Phase 3, and preliminary data show durability out to 24 months of treatment. Additionally, the OraGrowtH212 data reaffirms our confidence in our oral compound’s unique mechanism of action, the importance of the natural, pulsatile release of growth hormone, and its impact on restoring growth."

Recent Highlights

•Phase 2 OraGrowtH210 and PK/PD OraGrowtH212 Trials met all primary and secondary endpoints. Data from the topline Phase 2 OraGrowtH210 Trial demonstrated that the 1.6 mg/kg/day LUM-201 dose produced a mean annualized height velocity (AHV) of 8.2 cm/yr at six months on treatment for moderate PGHD subjects, in line with historical data in moderate PGHD patients1,2,3,4. Additionally, at twelve months on treatment, a durable effect was also observed with LUM-201 achieving AHV of 8.0 cm/yr at the 1.6 mg/kg dose, within the targeted 2 cm/yr margin of the comparator injectable rhGH arm. Data also provided preliminary validation of the PEM strategy, with prespecified primary and secondary outcomes met, de-risking our patient selection for our Phase 3 program. Data from the OraGrowtH212 Trial confirmed that LUM-201’s unique pulsatile mechanism produces an increase in the growth rates by restoring growth hormone secretion and IGF-1 to within normal ranges. The safety profile for LUM-201 remained clean throughout both Phase 2 trials, with no safety concerns identified in either of our Phase 2 trials conducted thus far. For a link to the Company’s conference call and presentation of the data refer to the Events & Presentations page in the Investors & Media section of the Company’s website.

•OraGrowtH212 Trial Data Presented at the 2023 annual meeting of the European Society for Paediatric Endocrinology. An oral presentation of the deconvolution analysis of growth hormone (GH) concentration sampled over 12 hours at baseline and after 6 months of therapy with daily oral LUM-201 illustrated how treatment with LUM-201 increases AHV, total GH secretion, and serum IGF-1 and IGFBP3 for individuals with moderate PGHD. Measured at 6 months compared to baseline, data showed a 60% increase in GH secretion to a level comparable to established values in normal healthy children and a 62% increase in AHV.

•Independent Panel of Renowned Pediatric Endocrinologists Discussed PGHD and Therapeutic Landscape. On September 5, 2023, a panel of five pediatric endocrinologists participated in a webinar where they discussed the benefits that the oral therapeutic LUM-201 candidate may provide compared to current injectable therapeutic options.

Upcoming Events

•Virtual KOL Event Planned. The Company plans to host a virtual KOL Event on December 6th to discuss topline results from OraGrowtH210 and OraGrowtH212 trials in greater detail and provide updates on clinical and corporate strategy. Management will be joined by the following three esteemed thought leaders in the field of endocrinology:
•Andrew Dauber, MD, Chief of Endocrinology at Children’s National Medical Center, Washington, D.C.
•Fernando Cassorla, MD, Chief of Pediatric Endocrinology at the Institute of Maternal and Child Research, University of Chile
•Leslie A. Soyka, MD, Chief of Pediatric Endocrinology, UMass Memorial Medical Center; Associate Professor, UMass Chan Medical School, Worcester, MA

Access information regarding the KOL Event will be provided at a later date.

1 Blum et al JES 2021, 2 Lechuga-Sancho et al JPEM 2009, 3 Ranke et al JCEM 2010, 4 For all OraGrowtH Trial AHV values, ANCOVA Model Terms: treatment, Age at dose 1, Sex, Baseline HT SDS, Baseline BMI SDS, Baseline IGF-1 SDS, LUM-201 PEM, Baseline BA Delay

Financial Results for the Quarter Ended September 30, 2023

•Cash Position – Lumos Pharma ended the quarter on September 30, 2023 with cash, cash equivalents and short-term investments totaling $42.7 million compared to $67.4 million on December 31, 2022. The Company expects cash use of approximately $9.0 to $10.0 million in the fourth quarter of 2023. Cash on hand as of September 30, 2023 is expected to support operations through the third quarter of 2024, inclusive of the activities related to planning and initiation of a pivotal Phase 3 clinical trial.
•R&D Expenses – Research and development expenses were $5.0 million for the quarter ended September 30, 2023, compared to $4.1 million for the same period in 2022, primarily due to an increase of $0.9 million in clinical trial expenses and $0.2 million in consulting expenses, offset by a decrease of $0.2 million in personnel-related expenses.

Exhibit 99.1

•G&A Expenses – General and administrative expenses were $3.9 million for the quarter ended September 30, 2023, compared to $3.9 million for the same period in 2022, primarily due to an increase of $0.3 million in personnel-related expenses offset by a $0.3 million decrease in royalty expense.
•Net Loss – The net loss for the quarter ended September 30, 2023 was $8.3 million compared to a net loss of $7.3 million for the same period in 2022.
•Lumos Pharma ended the third quarter 2023 with 7,914,582 shares outstanding.

About Lumos Pharma’s Clinical Trials

Phase 2 OraGrowtH210 Trial Design

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) compared to daily injectable recombinant human growth hormone (rhGH) 34 µg/kg/day in 82 subjects diagnosed with moderate PGHD. The trial population was enriched for subjects responsive to LUM-201 during screening by applying the specific PEM cutoffs of a baseline IGF-1 value > 30 ng/ml and a peak growth hormone value of ≥ 5 ng/ml after administering a single dose of 0.8 mg/kg of LUM-201 to treatment-naïve PGHD patients. The study was not designed to evaluate efficacy and demonstrate non-inferiority to daily GH.

OraGrowtH212 Trial Design

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 treatment-naïve PGHD subjects at two dose levels, 1.6 and 3.2 mg/kg/day. Every subject in the OraGrowtH212 Trial was PEM-positive and, therefore, enriched for responsiveness to LUM-201.

Switch Study, OraGrowtH213 Trial, Evaluating LUM-201 in OraGrowtH210 Subjects Previously on rhGH

The OraGrowtH213 Trial is an open-label, multi-center, Phase 2 trial evaluating the growth effects and safety of LUM-201 following 12 months of daily rhGH in up to 20 idiopathic PGHD subjects who have completed the OraGrowtH210 Trial. Subjects will be administered LUM-201 at a dose level of 3.2 mg/kg/day for up to 12 months.