Disc Medicine (IRON) investor relations material

Strategic Overview and Vision

• Transitioning from a science-focused to a commercial-stage company, targeting fundamental red blood cell pathways with genetically validated mechanisms for a spectrum of hematologic diseases, from rare (EPP) to common (anemia of chronic kidney disease, sickle cell, and anemias of inflammation).

• Commercialization strategy focuses on rare disease launch, expanding into hematology-oncology and broader markets, aiming for sustainable standalone growth.

• Three lead programs (bitopertin, DISC-0974, DISC-3405) address US markets exceeding $13B.

• Efficient commercial infrastructure established, including 24 sales reps, 6 medical liaisons, and exclusive specialty pharmacy distribution.

• Company is well-capitalized with $791 million in cash, providing runway into 2029, not assuming any product revenue.

Bitopertin for EPP: Clinical and Commercial Highlights

• Bitopertin NDA submitted and under accelerated FDA review, with anticipated approval by end of January or early February and potential US launch in early to mid-2026.

• Clinical trials (AURORA, BEACON) showed 50–60% reduction in toxic PPIX, 2–3x improvement in sunlight tolerance, and up to 92% reduction in phototoxic reactions.

• Long-term extension data show sustained efficacy and safety, with over 90% of patients reporting significant improvement.

• Launch readiness includes drug supply, access/reimbursement team, 24 sales reps, initial marketing using the label, and comprehensive patient support programs.

• Targeting 14,000 diagnosed US patients (age 12+), with launch focused on top 120 sites and broader outreach to 8,000 additional patients.

Market Opportunity and Launch Dynamics

• EPP market estimated at $2 billion+ if all 14,000 patients are reached; payer mix is favorable with commercial, Medicare, and Medicaid coverage.

• Launch strategy includes patient advocacy, digital tools, direct outreach to physicians, and robust real-world validation.

• Ex-US strategy leverages APOLLO trial as phase III for Europe and Japan, with filings expected to begin in 2027.

• Commercial and clinical expansion supported by strong financial position and scalable infrastructure.