Disc Medicine (IRON) H.C. Wainwright 4th Annual BioConnect Investor Conference summary

Clinical development updates

• Enrollment in the APOLLO confirmatory study for bitopertin exceeded targets, reflecting strong demand and unmet need in both the U.S. and Europe.

• Epidemiological and claims data suggest the U.S. EPP patient population may be larger than previously estimated, with up to 14,000 identified in claims databases and a core group of about 6,000 actively engaged in care.

• The APOLLO phase III data is expected in Q4 this year, with a potential approval timeline around mid-next year following a six-month review period.

• Pediatric and adolescent enrollment in trials is ongoing, aiming for a label that includes patients as young as 12, with plans to expand to younger populations.

Regulatory and operational strategy

• The FDA's complete response letter cited insufficient correlation between PPIX levels and symptoms, with the APOLLO study expected to address this.

• A Type A meeting with the FDA will focus on data analysis and NDA resubmission strategy, aiming for efficient re-filing.

• Organizational restructuring followed the accelerated approval setback, retaining a third of the team to maintain physician relationships and market understanding.

Pipeline and market opportunities

• DISC-0974 showed efficacy in anemic myelofibrosis patients, including those on various JAK inhibitors, with consistent response rates across subgroups.

• Interim data for DISC-0974 indicate 50%-60% major response rates, with further data expected at upcoming conferences and study completion by year-end.

• Development in chronic kidney disease was halted due to mixed results, while IBD remains a focus due to promising preclinical and mechanistic rationale.

• DISC-3405 aims to offer a monoclonal antibody for polycythemia vera with improved dosing and safety, and a study in sickle cell disease is planned, leveraging the benefits of iron restriction.