Disc Medicine (IRON) investor relations material

Pipeline overview and upcoming catalysts

• Three clinical-stage molecules focus on red blood cell biology via heme and iron metabolism, with the lead program, bitopertin, set for NDA filing in October and potential launch next year.

• Myeloproliferative disorder programs are in phase II, with data expected late this year and next year.

• The company leverages unique biology, faces little direct competition, and benefits from collaborations, including with a Chinese biotech.

• AI is being explored for automating clinical and regulatory documentation to free up team resources.

• Favorable FDA interactions and rare disease focus support efficient regulatory pathways.

Bitopertin commercial strategy and market opportunity

• NDA submission for erythropoietic protoporphyria (EPP) is imminent, with commercial launch preparations underway and a specialized team in place.

• Patient population is estimated at 14,000 in the U.S., with 6,000 actively engaged and targeted for initial launch; expansion to the full group will rely on advocacy and outreach.

• Dosing is 60 mg once daily, aiming for a 50% reduction in toxic metabolite PPIX, with pricing expected in the $300,000–$600,000/year rare disease range.

• Long-term extension data show sustained PPIX reduction and improved patient-reported outcomes.

• The confirmatory APOLLO trial is enrolling well, expected to complete by next May, with strong confidence in meeting co-primary endpoints.

Clinical insights and competitive landscape

• EPP is a severe disease with debilitating light sensitivity and risk of liver complications; current therapies are limited and often ineffective.

• In myelofibrosis, 0974 shows ~50% response rates for anemia, with efficacy regardless of JAK inhibitor use, and the RALLY-MF trial protocol was updated to include more patients on momelotinib.

• No approved therapies exist for anemia in myelofibrosis; recent competitor trial failures strengthen positioning.

• In CKD, 0974 targets high hepcidin levels to mobilize iron, differentiating from IV iron and EPO therapies; biomarker strategies focus on ferritin and EPO levels.

• Combination approaches with EPO are promising, and subgroup identification may be key in CKD.