Disc Medicine (IRON) Wells Fargo 2024 Healthcare Conference summary

Pipeline overview and clinical development

• Three mid-stage clinical assets target heme synthesis and iron metabolism to address rare and common hematologic diseases.

• Lead program, bitopertin, is a heme synthesis inhibitor wrapping up phase II and preparing for phase III in erythropoietic protoporphyria (EPP) next year.

• DISC-0974 targets iron homeostasis, in phase 1b/2 for anemia in myelofibrosis and chronic kidney disease (CKD), with data updates expected this year.

• DISC-3405 increases hepcidin to restrict iron, with polycythemia vera as the lead indication.

• All programs have shown clinical activity and are advancing to next development phases.

Bitopertin in EPP: Mechanism, efficacy, and regulatory path

• Bitopertin inhibits glycine uptake, slowing heme synthesis and reducing toxic protoporphyrin IX (PPIX), the causative agent in EPP.

• Phase II trials showed 40%-50% reduction in PPIX, dose-dependent, with meaningful improvements in photosensitivity.

• Longitudinal analysis revealed patients on bitopertin doubled time in sunlight and had fewer pain attacks compared to placebo.

• Regulatory discussions with FDA are ongoing; phase III is planned for the first half of next year, with Q4 feedback expected on endpoints.

• Addressable EPP market estimated at 14,000 diagnosed and up to 20,000 genetically, with strong patient advocacy and ICD-10 code support.

Additional pipeline programs and market opportunities

• DISC-0974 demonstrated 60%-70% hematologic response in myelofibrosis, with robust safety and compatibility with background therapies.

• CKD anemia program is progressing, with updates on higher dose cohorts and focus on mechanistic translation to erythropoiesis.

• DISC-3405 (anti-TMPRSS6 antibody) showed strong hepcidin induction and iron restriction in healthy volunteers, with plans to enter polycythemia vera.

• DBA (Diamond-Blackfan anemia) is being explored via NIH collaboration, with potential for future development if proof of concept is achieved.

• Company is well-capitalized with $501M in cash, funding operations into 2027 and supporting aggressive advancement of all lead programs.