Disc Medicine (IRON) 3rd Annual Guggenheim Healthcare Innovation Conference summary

APOLLO and launch

• Phase III APOLLO met all primary and secondary endpoints; reducing the toxic metabolite translated into broad clinical improvement, supporting a first therapy aimed at the underlying disease cause.

• Patients typically tolerate sunlight for only 30–40 minutes before severe pain attacks, with the disease shaping daily activities, careers and family life.

• Claims analysis identified 14,000 diagnosed patients: ~6,000 more engaged in care and ~8,000 disengaged; the initial launch focus is the engaged group, supported by ~24 reps.

• The commercial team has identified ~1,600 patients at the top 100 sites; access efforts are ramping fastest there, more gradually across the next ~4,000 patients, and slowest for the 8,000 disengaged patients.

• A U.S. launch was framed for 12–16 months; Europe is the primary ex-U.S. market, with a lean access model planned. Porphyria pricing benchmarks cited were ~$300,000 and ~$600,000 per year for existing therapies.

Selcodebart and myelofibrosis-associated anemia

• Phase II response rates were 70%–80% by clinician assessment and ~50% using the most stringent regulatory calculation; an FDA end-of-phase II meeting is underway to define pivotal trials across patient groups.

• ~22,000 U.S. patients with myelofibrosis have anemia; the pivotal design discussion with FDA will address endpoints and treatment duration, with the goal of reaching the full patient population.

• The planned ASH update will cover the FDA meeting; the resulting alignment is intended to inform next year’s trial-start timing and estimated enrollment duration.

• For transfused patients, a reduction in transfusion burden is a precedent; for non-transfused patients, a hemoglobin increase paired with a quality-of-life measure may be appropriate. A 1.5 g/dL rise is accepted clinically, while FDA assessment may require symptom measures.

• The ASH data cut is expected to include ~60 evaluable patients from a trial expected to reach ~90; management cited ~30% as a meaningful response benchmark and reported ~50% overall in current data.

Polycythemia vera program

• Phlebotomy controls red-cell production by depleting iron but can cause burdensome symptoms; iron restriction may control red-cell production while avoiding nutritional iron deficiency. An estimated 50,000–100,000 U.S. patients are managed with phlebotomy.

• The TMPRSS6-targeting antibody aims for 70%–80% of patients to become phlebotomy-free while maintaining hematocrit at or below 45%; goals include home auto-injector use and fewer side effects.

• ASH will include Q4-week dosing data, following earlier Q2-week data; management views monthly dosing as practical with an auto-injector and potentially easier to manage if anemia occurs than longer dosing intervals.

• A pivotal study is expected to start next year; management sees the recent approval precedent as relevant but expects to confirm the approach with FDA at an end-of-phase II meeting.