Disc Medicine (IRON) Jefferies Global Healthcare Conference 2026 summary

Program updates and clinical progress

• Lead program bitopertin is set for a phase II data readout by year-end, with two phase II programs ongoing and a third in polycythemia vera expecting proof-of-concept data by year-end.

• Myelofibrosis anemia program showed strong interim data at ASCO, with consistent response rates (50%-60%) across all transfusion subgroups, including those on JAK inhibitors.

• End of phase II meeting with FDA is planned for late year, aiming to align on phase III trial design and potentially start pivotal studies in multiple subpopulations next year.

• Expansion into other indications like IBD is underway, with initial IBD data expected early next year and potential for basket trials in broader anemia of inflammation.

Regulatory and pivotal trial strategy

• For transfused myelofibrosis patients, transfusion independence is a likely approval endpoint; for non-transfused, hemoglobin and fatigue scores are key, following recent regulatory precedents.

• Aim to secure FDA alignment on pivotal trial protocols for all subgroups in a single end of phase II meeting, but may stagger studies if needed.

• Each pivotal study could support a label for its population independently due to rare disease status.

Bitopertin in EPP: phase III and commercial readiness

• Bitopertin’s phase III APOLLO trial is fully enrolled, with data readout expected in Q4 and submission planned for next year.

• Phase III design was optimized based on phase II learnings, including endpoint refinement and increased sample size for robust powering.

• Bitopertin targets the root cause of EPP by reducing protoporphyrin IX in whole blood, potentially offering broader disease modification than competitors.

• Pre-commercial activities include mapping patient care centers and maintaining a small sales and MSL team, with plans to scale up for launch upon approval.