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Disc Medicine (IRON) investor relations material

Disc Medicine Morgan Stanley 23rd Annual Global Healthcare Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Morgan Stanley 23rd Annual Global Healthcare Conference summary10 Sep, 2025

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.

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Q3 202511 Nov, 2025
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Frequently asked questions

Disc Medicine Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of innovative treatments for severe hematologic disorders. Founded as Gemini Therapeutics Inc before evolving into its current form, the company has a pipeline that includes therapies aimed at erythropoietic porphyrias (including erythropoietic protoporphyria and X-linked protoporphyria), Diamond-Blackfan Anemia, anemia of myelofibrosis, chronic kidney disease, and polycythemia vera among others. Disc Medicine's preclinical programs also target anemia associated with inflammatory diseases. The company is headquartered in Watertown, Massachusetts, and its shares are listed on the NASDAQ.

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