Disc Medicine (IRON) Morgan Stanley 24th Annual Global Healthcare Conference summary

Macro environment and strategic considerations

• No significant competition from China-origin innovation in lead indications; pipeline advancing to phase III with commercial transition expected soon.

• AI adoption is in early stages, focusing on patient and physician identification in rare diseases to drive peak revenue.

• MFN pricing policy impacts overseas market strategies and partnership decisions, with a clear commitment to building in the U.S. market.

Clinical development and regulatory updates

• APOLLO phase III trial for bitopertin in EPP is aligned with FDA on endpoints; focus is on delivering strong phase III data.

• Powering assumptions for APOLLO are based on months two to four of phase II data, aiming for robust detection of drug effect.

• Seasonality is well represented in phase II data, with larger drug effect expected in summer; rapid European enrollment may favor detection.

• Success in APOLLO hinges on meeting co-primary endpoints (PP9 and sunlight exposure) with p<0.05.

• Patient advocacy and real-world experience reinforce the clinical impact of bitopertin, with high patient enthusiasm for long-term extension.

Commercial outlook and competition

• U.S. launch will target both SCENESSE users and patients at centers of excellence, validated by field force and claims data.

• Commercial expenses are fully loaded into a cash runway model extending to 2029, with ongoing ramp-up in disease education and physician engagement.

• Field force will be tailored by indication, with separate teams for porphyria and heme/onc populations.

• Competition in EPP includes SCENESSE and dersimelagon, with bitopertin differentiated by targeting the root cause of disease.