Lumos Pharma (LUMO) H.C. Wainwright 26th Annual Global Investment Conference summary

Market overview and opportunity

• Growth hormone market is mature, valued at nearly $5 billion in 2022, projected to exceed $8 billion by 2030, with pediatric growth hormone deficiency (PGHD) representing a $1.5 billion global opportunity.

• Long-acting injectables have expanded the market, but compliance issues persist, especially in children, with only 50-60% adherence to daily injections.

• Oral growth hormone secretagogue is positioned as a disruptive, first-in-class therapy with potential for rapid market conversion and expansion.

Product innovation and differentiation

• The oral secretagogue is a small molecule with a unique mechanism, restoring natural pulsatile growth hormone release and normalizing IGF-1 levels.

• Unlike recombinant growth hormone, which requires supraphysiologic doses, the oral agent achieves growth by restoring endogenous hormone levels, minimizing IGF-1 excursions.

• Orphan drug status has been secured in the US and EU, with IP protection until 2042.

• Cost of goods is expected to be lower than injectables, supporting competitive margins.

Clinical development and regulatory strategy

• Phase II studies met all primary and secondary endpoints, showing significant increases in annualized height velocity and a robust safety profile in over 1,300 patients.

• PEM (Predictive Enrichment Marker) strategy enables selection of moderate PGHD patients most likely to respond, de-risking trials and targeting the majority of the patient population.

• Durability of response is strong, with only a 10% drop-off in year two versus 20% for injectables.

• End-of-phase II FDA meeting was positive; phase III placebo-controlled, randomized, crossover trial in moderate PGHD is set to begin in the second half of 2025.