Disc Medicine (IRON) investor relations material

Company overview and philosophy

• Focuses on controlling red blood cell biology by targeting iron and heme metabolism, with three main clinical programs.

• Lead asset, bitopertin, targets heme biosynthesis and is being developed for erythropoietic protoporphyria (EPP).

• Pipeline includes DISC-0974 for anemia in myelofibrosis and a program for polycythemia vera, both in phase 2.

• Underlying philosophy is to address rare hematologic diseases with high unmet need.

Bitopertin (EPP program) progress and launch plans

• NDA for bitopertin submitted in late September; potential approval and launch expected late this year or early next year.

• Received a national priority voucher, accelerating the FDA review timeline by about six months.

• Launch preparations are underway, with logistics for drug supply ready, but some commercial aspects like sales force and payer engagement will lag slightly.

• Initial launch will leverage 100-150 patients from open-label extension studies and strong relationships with top treatment centers.

• About 6,000 highly engaged EPP patients identified in the US, with a sales force targeting their physicians.

Clinical trial and patient access strategy

• Confirmatory Apollo trial for bitopertin is enrolling, with completion expected by May next year; about 150 patients targeted.

• US and ex-US regulatory strategies are aligned, with European and Japanese approvals dependent on Apollo results.

• Patients in trials will transition to commercial product over about a year as reimbursement pathways are established.