Lumos Pharma Reports Positive End-of-Phase 2 FDA Meeting and Q1 2024 Financial Results

Lumos Pharma, Inc., a clinical-stage biopharmaceutical company focusing on treatments for rare diseases, has revealed positive developments following an End-of-Phase 2 meeting with the FDA, announced updates on its clinical programs, and shared financial results for the quarter ending March 31, 2024.

Rick Hawkins, Chairman and CEO of Lumos Pharma, expressed satisfaction with the FDA meeting, which acknowledged the unique mechanism of LUM-201, a growth hormone secretagogue, and endorsed a placebo-controlled design for the Phase 3 trial. Lumos Pharma plans to propose a single, double-blinded, placebo-controlled Phase 3 study with a 2:1 randomization involving around 150 patients. The study design details are expected to be finalized with the FDA by the third quarter, aiming to initiate the trial by year-end.

Updated data from the Phase 2 OraGrowtH trials demonstrate the significant efficacy of LUM-201 in increasing growth from the baseline in annualized height velocity (AHV) at 6 and 12 months, with durable effects extending to 24 months. These outcomes suggest that LUM-201 could be the first oral therapeutic for moderate pediatric growth hormone deficiency, advancing toward a Phase 3 registrational trial and potential approval.

Key recent highlights include:

1. End-of-Phase 2 Meeting with FDA: 
- The FDA supported a placebo-controlled trial design for the Phase 3 study of LUM-201, reflecting their recognition of its unique qualities as a growth hormone secretagogue. 
- The proposed Phase 3 trial will involve approximately 150 patients over 12 months, featuring a double-blinded, placebo-controlled design with a 2:1 randomization. The placebo-controlled portion will last six months, which is expected to increase the likelihood of success compared to a non-inferiority study.
- The company aims to begin the Phase 3 trial in the fourth quarter of 2024, subject to FDA approval.

2. Updated LUM-201 Data:
- Full 12-month data from the OraGrowtH210 trial showed LUM-201 achieving substantial growth increases from baseline with AHVs of 8.2 cm/year and 7.6 cm/year at 6 and 12 months, respectively, for the 1.6 mg/kg dose, compared to a baseline growth of 4.7 cm/year.
- Combined data from the OraGrowtH210 and OraGrowtH212 trials indicated that LUM-201 maintains a durable treatment effect over 24 months.
- The year-2 decline in AHV for LUM-201 was more moderate (9.9%) compared to a historical decline of 19.7% observed in recombinant human growth hormone (rhGH) benchmarks, likely due to LUM-201’s restoration of GH and IGF-1 levels.

3. Medical Meeting Presentations:
- Data from Phase 2 trials were presented at notable medical conferences such as the Pediatric Endocrinology Society (PES), the 10th International Congress of the Growth Hormone Research Society (GRS), and the European Congress of Endocrinology (ECE). These presentations highlighted that LUM-201 promotes growth comparable to injectable rhGH with significantly less exposure to circulating growth hormone.

4. Upcoming Data Presentations:
- Additional analyses and full 12-month data from the OraGrowtH212 trial, alongside updated 24-month combined data, will be presented in Q2 2024. Two abstracts have been accepted for poster presentation at the Endocrine Society (ENDO) Annual Meeting.

Financially, Lumos Pharma ended the first quarter of 2024 with $23.2 million in cash, cash equivalents, and short-term investments. This is a decrease from $36.1 million at the end of 2023. The company anticipates that its current cash reserves will support operations through the third quarter of 2024, covering Phase 3 trial planning and preparatory activities.

Research and development expenses for the quarter increased to $7.2 million, driven by higher licensing, clinical trial, and consulting expenses. Conversely, general and administrative expenses decreased to $3.8 million due to reduced licensing, travel, and consulting costs. The net loss for the quarter was $10.4 million, compared to $7.3 million in the same period of the previous year.

Lumos Pharma's progress and ongoing efforts highlight their commitment to advancing LUM-201 as a promising oral therapeutic for pediatric growth hormone deficiency.

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