Disc Medicine Receives FDA CRL, Eyes Q4 2026 APOLLO Phase 3 Topline
Disc Medicine received an FDA Complete Response Letter for bitopertin in protoporphyria, recognizing PPIX reductions in AURORA and BEACON but requiring Phase 3 APOLLO topline results expected Q4 2026. The company has $791 million cash runway into 2029, completed APOLLO enrollment early and plans a Type A meeting.
1. FDA Complete Response Letter Details
The FDA issued a Complete Response Letter for bitopertin’s New Drug Application, agreeing that AURORA and BEACON trials showed significant whole blood metal-free PPIX reductions but did not demonstrate correlation with sunlight exposure endpoints. The agency indicated that APOLLO Phase 3 topline results will be required to support traditional approval.
2. APOLLO Phase 3 Study Status and Timeline
Disc Medicine completed enrollment of the APOLLO trial in March 2026, several months ahead of target without any sample size adjustments. Topline data are expected in Q4 2026 and will form the basis of the company’s response to the CRL.
3. Financial Position and Cash Runway
As of December 31, 2025, Disc Medicine held approximately $791 million in cash, cash equivalents, and marketable securities, delivering financial runway into 2029. This strong balance sheet underpins ongoing development and regulatory efforts for bitopertin.
4. Next Steps and Regulatory Path
Disc plans to request a Type A meeting with the FDA to address the CRL’s findings and will file a formal response once APOLLO data are available. The company anticipates an updated regulatory decision by mid-2027 as it pursues traditional approval.