Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, today announced financial results for the second quarter ended June 30, 2024 and provided an update on the development of its lead product candidate, LUM-201.

Financials

For the full year 2023, Lumos Pharma reported annual revenue of $2,051,000 and a net loss of $34,034,000. The company's annual operating cash flow and free cash flow were both -$31,095,000.

In the second quarter of 2024, the company generated $488,000 in royalty revenue, compared to $527,000 in the same period of 2023. Research and development expenses decreased by $1.4 million to $4.6 million, primarily due to decreases in contract manufacturing, personnel-related, and clinical trial expenses. General and administrative expenses also decreased by $0.5 million to $3.7 million, driven by reductions in personnel, travel, consulting, and other costs. The net loss for Q2 2024 was $7.6 million, compared to a net loss of $8.9 million in the prior year period.

As of June 30, 2024, Lumos Pharma had $16.8 million in cash, cash equivalents, and short-term investments, compared to $36.0 million as of December 31, 2023. The company stated that its current cash resources are expected to support operations into the first quarter of 2025.

Business Overview

Lumos Pharma is a clinical-stage biopharmaceutical company focused on the development and commercialization of therapeutics for rare diseases. The company's lead product candidate is LUM-201, a novel, oral growth hormone (GH) secretagogue being evaluated for the treatment of pediatric growth hormone deficiency (PGHD).

LUM-201 is designed to stimulate the natural release of growth hormone from the pituitary gland, in contrast to current standard-of-care injectable recombinant human growth hormone (rhGH) therapies. The company believes LUM-201's unique mechanism of action has the potential to provide an effective, convenient, and more physiologic approach to treating PGHD compared to daily injections.

Lumos Pharma has completed two Phase 2 clinical trials, OraGrowtH210 and OraGrowtH212, evaluating LUM-201 in patients with moderate PGHD. The trials met their primary and secondary endpoints, demonstrating that LUM-201 was able to achieve annualized height velocity (AHV) comparable to daily injectable rhGH, with a favorable safety profile.

Planned Phase 3 Trial

Following a productive end-of-Phase 2 meeting with the FDA, Lumos Pharma has finalized the design of a Phase 3 placebo-controlled clinical trial for LUM-201 in moderate PGHD. The trial will enroll approximately 150 patients across over 80 global sites, with a 2:1 randomization of LUM-201 to placebo.

The trial will have two co-primary endpoints: 1) demonstrating the superiority of LUM-201 in AHV compared to placebo, and 2) ensuring the lower bound of the 95% confidence interval for LUM-201's AHV is above 6.7 cm/year, the minimal clinically meaningful threshold agreed upon with the FDA.

Patients randomized to placebo will cross over to receive LUM-201 after the initial 6-month period, allowing all participants to ultimately receive the active treatment. This design is intended to enhance the trial's appeal to both patients and physicians, facilitating enrollment.

The company expects to finalize the Phase 3 trial design with the FDA in the fourth quarter of 2024 and initiate the study in the second quarter of 2025. This timeline extension from previous guidance allows Lumos Pharma to complete the manufacturing of the LUM-201-matched placebo capsules and establish the necessary stability data.

Strategic Initiatives

In addition to advancing the LUM-201 program, Lumos Pharma has engaged Piper Sandler & Co. to assist the Board of Directors in evaluating strategic opportunities to maximize shareholder value. The company's ongoing business development efforts have generated significant interest in the global potential for LUM-201, and the Board believes it is the right time to formally explore all available options.

Risks and Outlook

Lumos Pharma faces several key risks, including the successful execution of its planned Phase 3 trial for LUM-201, obtaining regulatory approval, and securing the necessary funding to complete development and commercialization. The company will need to raise substantial additional capital to fund the Phase 3 trial and beyond.

Despite these challenges, the company remains confident in LUM-201's potential to transform the $5 billion global growth hormone market from injectable to oral therapy. The positive data from the Phase 2 trials, the FDA's support for a placebo-controlled study design, and the growing enthusiasm from the endocrinology community provide a strong foundation for Lumos Pharma's future development efforts.

Conclusion

Lumos Pharma has made significant progress in advancing its lead product candidate, LUM-201, for the treatment of moderate PGHD. The company's planned Phase 3 trial, designed in collaboration with the FDA, represents a critical next step in potentially bringing the first oral GH secretagogue to market. With a strong scientific rationale, favorable clinical data, and strategic initiatives underway, Lumos Pharma is well-positioned to continue its mission of developing innovative therapies for rare diseases.