IRON News & Events

"We continue to make strong progress across the portfolio, completing enrollment in our Phase 3 trial of bitopertin in EPP and remaining on track to report topline data in the fourth quarter of 2026. We were also pleased to have our Phase 2 data for DISC-0974 in MF anemia selected for oral presentation at ASCO, while continuing to advance multiple additional clinical trials across our pipeline," said John Quisel, Chief Executive Officer and President of Disc. "Together, these milestones reflect the strength of our execution and position us to deliver multiple important catalysts in the second half of this year."

Disc Medicine announced that the last participant has been randomized and dosed in the pivotal Phase 3 APOLLO trial of bitopertin in EPP. Originally planned as a 150-person study, the study was expanded to 183 participants due to patient and physician demand.

The Food and Drug Administration review process for accelerated approvals was meant to drugs for rare diseases to pass the approval process, clearing the drugs based on early biological signs instead of waiting years for definitive proof of real-world outcomes, David Wainer of The Wall Street Journal. Now, however, the accelerated approval pathway isn't closed but has become much narrower and harder to predict. Recently, the FDA issued a string of rejections, including Regenxbio's (RGNX) gene therapy for Hunter Syndrome and Disc Medicine's (IRON) treatment for a blood disorder. In some cases, the agency reversed its own guidelines provided to the companies. In 2025, the FDA greenlighted only nine accelerated approvals, down from 20 in 2024. Other companies in the space include: uniQure (QURE), Moderna (MRNA), Replimune (REPL), and Capricor (CAPR).