Disc Medicine (IRON) H.C. Wainwright 27th Annual Global Investment Conference summary

Lead program and regulatory progress

• Lead asset bitopertin targets red blood cell disorders by manipulating iron and heme metabolism, with phase 2 data completed and an NDA filing planned for October, aiming for a PDUFA date in the second half of next year.

• Confirmatory Apollo trial is underway and enrolling, supporting the accelerated approval pathway.

• Long-term extension and mouse studies suggest significant liver protection by reducing Protoporphyrin IX, though label claims for liver protection will require further studies.

• KOLs strongly support the mechanism of action, expecting broad disease benefit and a competitive advantage over existing therapies due to oral dosing and potential liver protection.

Pediatric and adolescent development plans

• Four adolescents were included in phase 2, showing similar responses to adults; aim to include adolescents on the initial label, but more data may be needed.

• Additional pediatric studies for younger children are planned post-approval, with dosing expected to be weight-based and straightforward.

Commercial readiness and patient identification

• Commercial supply and CMC challenges have been addressed, with a full commercial and medical affairs team in place.

• Claims data identified 14,000 diagnosed US patients, with 6,000–7,000 highly engaged; field teams are validating these numbers and preparing for launch.

• Outreach to less-engaged patients will leverage advocacy and social media, while active patients are being confirmed through direct engagement.