Lumos Pharma (LUMO) Corporate Presentation summary

investment thesis and market opportunity

• Oral LUM-201 targets the $4.7B global growth hormone disorder market, with pediatric growth hormone deficiency (PGHD) as a $1.5B lead indication. Market research indicates rapid conversion to oral therapy and expansion potential.

• LUM-201 features a novel mechanism of action (MOA) leveraging natural physiology, with orphan drug designation in the US/EU and patent protection through 2042.

• The predictive enrichment marker (PEM) strategy de-risks patient selection, identifying likely responders. Phase 2 trials met all primary and secondary endpoints, showing significant increases in annualized height velocity (AHV) and a favorable safety profile in over 1,300 subjects.

• Positive end-of-Phase 2 FDA meeting in Q2 2024 supports a clear regulatory path, with Phase 3 initiation anticipated in Q4 2024.

• LUM-201 could be the first oral therapeutic to disrupt the injectable growth hormone market.

clinical program and data highlights

• LUM-201’s Phase 2 trials (OraGrowtH210 and OraGrowtH212) demonstrated significant growth rate increases from baseline, normalization of IGF-1, and durable effects up to 24 months.

• The PEM test is highly reproducible, enriching for responders and ensuring optimal patient selection.

• LUM-201 achieved growth rates comparable to historical rhGH datasets, with a more moderate year 2 AHV decline than rhGH, likely due to restoration of natural GH pulsatility.

• Safety profile is favorable, with no meaningful treatment-related serious adverse events or dropouts due to adverse events.

• LUM-201’s oral administration, natural MOA, and low cost of goods offer advantages over current standard of care.

regulatory and phase 3 plans

• FDA recognized LUM-201 as a novel growth promoter and supported a placebo-controlled Phase 3 trial design.

• Proposed Phase 3: 12-month, double-blind, placebo-controlled trial in PEM+ PGHD patients, with all placebo subjects crossing over to LUM-201 at 6 months; ~150 subjects across 80-100 sites.

• Co-primary endpoints: superiority of LUM-201 AHV over placebo at 12 months and lower bound of 95% CI above 6.7 cm/year.

• Phase 3 initiation expected by year-end 2024, with the design expected to reduce regulatory risk and improve likelihood of success.