Disc Medicine (IRON) H.C. Wainwright 28th Annual Global Investment Conference summary

Key clinical data and program updates

• Initial data for anti-TMPRSS6 antibody (DISC-3405) in polycythemia vera showed induction of hepcidin, iron restriction, strong hematocrit control, reduced phlebotomy burden, and favorable safety profile.

• Q2W dosing demonstrated efficacy, with expectations that Q4W dosing will also be effective based on PK/PD data and early mechanism engagement.

• Up to 78% of patients were phlebotomy-free between weeks 12–32, indicating strong clinical benefit.

• Sickle cell and beta thalassemia are prioritized as future indications, with initial sickle cell data expected by year-end and a significant opportunity identified in beta thalassemia.

• DISC-0974 showed strong results in RALLY-MF across patient groups, with response rates of 70–80% and learnings from prior phase III studies guiding trial design.

Competitive landscape and dosing strategy

• Antibody modality chosen for its dosing flexibility and ability to titrate iron restriction, aiming for a "Goldilocks" dosing interval (Q2W/Q4W) for optimal patient convenience and safety.

• Competitors include MIMRYLO (weekly peptide with high injection site reactions) and siRNA approaches with less frequent dosing, but monthly or biweekly dosing seen as optimal for adherence.

• Flexibility between Q2W and Q4W dosing is valued, with Q4W as the base assumption but Q2W available as needed.

Study design enhancements and operational learnings

• APOLLO trial for bitopertin incorporates lessons from AURORA, focusing on the last month of the six-month study to minimize placebo effects and increasing sample size to 75 per arm.

• European sites added and stratified by geography to ensure robust data; dropout rates have been minimal, with high patient retention.

• Exploratory liver enzyme measures included, but primary focus remains on approval endpoints; adolescent data in APOLLO may support future pediatric access.