Disc Medicine (IRON) TD Cowen 45th Annual Healthcare Conference summary

Company overview and strategy

• Focuses on red blood cell biology, targeting iron and heme metabolism to address rare and common anemias.

• Pipeline includes bitopertin (lead, small molecule), DISC-0974 (antibody), and DISC-3405 (antibody), with additional preclinical assets.

• Programs span rare diseases like erythropoietic protoporphyria (EPP) to large markets such as anemia of chronic kidney disease.

• Strong financial position with runway into 2028, enabling execution across the portfolio.

• Demonstrated rapid progress from preclinical to late-stage clinical development in five years.

Bitopertin program and regulatory progress

• Bitopertin targets EPP by reducing toxic protoporphyrin IX (PPIX) levels, addressing the root cause of the disease.

• Clinical trials show significant reduction in PPIX, increased sunlight tolerance, and elimination of phototoxic reactions at top dose.

• Confirmatory APOLLO trial to start mid-year, with NDA submission planned for the second half.

• FDA alignment on surrogate endpoint (PPIX reduction) and co-primary endpoint (time in light) for approval pathway.

• Target launch population is 6,000 highly engaged EPP patients, with 14,000 diagnosed in total.

Pipeline progress and clinical data

• DISC-0974 in phase II for anemia of myelofibrosis, showing 50%-60% response rates across patient subgroups.

• Plans for pivotal trial design to focus on transfusion burden and hemoglobin improvement.

• Early data in chronic kidney disease show hemoglobin increases; further dose escalation and data expected in the second half.

• DISC-3405 (anti-TMPRSS6) advancing to phase II in polycythemia vera, aiming for monthly dosing and improved patient experience.

• Preclinical data support broader applications, including inflammatory bowel disease and sickle cell disease models.