Disc Medicine (IRON) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Company overview

• Focuses on controlling red blood cell formation by targeting heme biosynthesis and iron metabolism, with three clinical candidates addressing various red blood cell disorders.

Bitopertin program and APOLLO phase III trial

• Phase III data for bitopertin expected next quarter, with primary endpoint based on increased time in sunlight without pain for porphyria patients.

• Phase II AURORA trial showed a 14.9-hour increase in sunlight exposure in the last month, translating to significant life improvements for patients.

• Phase III APOLLO trial powered conservatively using an 11-hour effect size, with robust design to account for seasonality and regional differences.

• European enrollment occurred between November and March, ensuring readouts during optimal months for detecting drug effect.

Liver safety and expanded access

• Liver enzyme assessments are being tracked in APOLLO, but the trial excludes patients with significant liver damage; any liver benefit may require further study.

• Expanded access program (EAP) has high demand, with enrollment criteria similar to clinical trials and plans to transition EAP and extension study patients to commercial drug upon approval.