Disc Medicine (IRON) TD Cowen 46th Annual Health Care Conference summary

Key program updates and clinical progress

• Three clinical-stage programs target iron and heme metabolism to address rare and common red blood cell disorders, with significant progress across the pipeline.

• Lead program bitopertin for EPP is in a phase III trial (APOLLO), with top-line data expected in Q4 and a projected launch by mid-next year, following a recent CRL from the FDA.

• DISC-0974 targets anemia of myelofibrosis, showing strong phase II data and aiming for pivotal trial design by year-end.

• DISC-3405, focused on polycythemia vera, doubled its trial size due to rapid enrollment and expects phase II data by year-end.

• Additional indications under investigation include anemia of inflammatory bowel disease and sickle cell disease, with early data anticipated this year.

Regulatory and commercial strategy

• Bitopertin's regulatory path involves a Type A FDA meeting in Q2 to confirm CRL requirements, with a response and potential approval review by mid-next year.

• Commercial team was reduced due to regulatory delays, but a core team remains to validate patient accounts and prepare for launch.

• Patient community shows strong enthusiasm for bitopertin, reinforcing market potential.

• Market size estimates: EPP ($2B+), myelofibrosis ($4B+), polycythemia vera ($7B+).

• $791 million cash runway extends into 2029, supporting ongoing development and launches.

Clinical trial design and endpoints

• APOLLO phase III trial for EPP is highly powered, with co-primary endpoints of PP9 reduction and increased time in light, both required for success.

• Myelofibrosis program aims to address all anemia subtypes, with endpoints under discussion with FDA; fatigue and prevention of transfusion dependence are key considerations.

• PV program leverages validated iron restriction mechanisms, with phase II data expected to clarify the path to pivotal trials.

• Upcoming data presentations planned at major hematology conferences, including updates from HELIOS, RALLY-MF, and RESTORE-PV trials.