2025-06-05-Jefferies-迪斯科医药（IRON）_IRON_JEF_2025年医疗保健会议_Bito很快将在红细胞生成性原卟啉症（EPP）提交新药申请_骨髓纤维化（MF）_慢性肾脏病（CKD）贫血为下一个重点_7页_109kb

Disc Medicine (IRON) Equity Research Summary

Disc Medicine is a clinical-stage biopharmaceutical company focusing on treatments for serious hematologic diseases. The company maintains a BUY rating with a price target of $111.00, representing a 131% upside from the prior trading day's close of $48.01.

Bitopertin in Erythropoiesis Progenitor Cells (EPP)

• Disc Medicine is on track to submit a New Drug Application (NDA) for bitopertin in the second half of 2025.
• The NDA will be based on clinical data from the BEACON and AURORA Phase 2 trials, using PPIX reduction as a surrogate endpoint.
• A Phase 3 trial, APOLLO, is ongoing and aims to confirm efficacy for full US approval. It is a double-blind, placebo-controlled study with co-primary endpoints including PPIX reduction and time in sunlight without pain.
• The company reiterated a large commercial opportunity in EPP, with approximately 20,000 potential patients in the U.S., and strong engagement in the target population of about 6,000.
• Regulatory interactions with the FDA have been positive, aligning with accelerated approval goals.

Pipeline Product DISC-0974

• Development is focused on anemia in Myelofibrosis (MF) and Non-Diabetic Nephropathy-CKD (NDD-CKD).
• Phase 2 trial RALLY-MF for anemia of MF is ongoing, with initial data expected in the second half of 2025.
• The large unmet need in MF anemia (no FDA-approved treatments) positions DISC-0974 as a potential first-line therapy, capable of working across anemia severity levels and in combination with JAK inhibitors.
• Phase 1 study for NDD-CKD is expected to release data in the second half of 2025, with two positioning strategies being considered: targeting all patients or specific subsets.
• Analysts highlighted the potential market capture in MF anemia and the competitive landscape, noting Gondola's EPP asset as a potential but far behind competitor.

Key Insights and Analyst Views

• Analyst Roger Song noted encouraging regulator interactions and strong pipeline advancements.
• Financials show a cash runway extending to 2028, supporting ongoing trials.
• Analyst certification confirms personal views and favorable risk/reward, but acknowledges commercial and developmental risks.

For detailed disclosures and valuation, refer to pages 2–7 of the full report.