Disc Medicine (IRON) Stifel 2024 Healthcare Conference summary

Pipeline overview and clinical progress

• Three clinical programs target heme synthesis and iron homeostasis, addressing severe hematologic disorders.

• Lead program bitopertin targets EPP, showing strong phase II results and a path to accelerated approval.

• DISC-0974 manipulates hepcidin for anemia of inflammation, with promising mid-year data and phase II plans.

• DISC-3405 increases hepcidin for iron-loading diseases, with phase II in PV planned for next year.

• Expansion into Europe and focus on centers of excellence for trial recruitment.

Bitopertin and EPP program

• Bitopertin reduces PPIX, improving photosensitivity and quality of life in EPP patients.

• Phase II studies (BEACON, AURORA) showed patients spent more time in sunlight and had fewer attacks.

• FDA supports accelerated approval using PPIX as a surrogate endpoint; confirmatory trial (APOLLO) planned.

• Adolescents included in APOLLO to address early intervention and quality of life.

• HELIOS extension study allows continued treatment; over 80% of eligible patients enrolled.

Market opportunity and patient engagement

• U.S. EPP prevalence estimated at 14,000 diagnosed, with 3,000–6,000 highly engaged patients.

• Patient advocacy groups and centers of excellence facilitate recruitment and engagement.

• European patient group structure mirrors U.S. organization.