2025-06-12-Jefferies-迪斯克医药（IRON）_EHA25（+）长期数据为主要管线资产提供进一步支持_14页_4mb

Disc Medicine Inc (IRON) is a clinical-stage biopharmaceutical company focused on hematological diseases targeting heme and iron metabolism.

Company Update

Presented 5 posters at EHA'25 across key pipeline assets (bitopertin, DISC-0974, '3405).

Analyst Ratings

• Recommendation: BUY
• Current Price: $52.71
• Price Target (PT): $111.00 (111% upside)
• 12-Month Views: $111 (Base Case), $143 (+171% Upside), $20 (-62% Downside)

Key Assets & Data

1. Bito (Bitopertin, GlyT1 inhibitor) – Erythropoietic Protoporphyria (EPP):

• Positive longer-term Phase 2 HELIOS trial data: sustained PPIX reduction, improved QoL, and favorable safety profile (no SAEs, median exposure ~18 mo).

2. DISC-0974 (Anti-HJV monoclonal antibody) – Anemia of Myelofibrosis (MF):

• Promising results from Ph1b continuation phase:
  • nTD pts: 50% mean Hgb increase >1.5 g/dL >12 weeks.
  • TD Low pts: 80% transfusion independence >16 weeks.
  • Favorable safety and sustained hepcidin suppression.

3. DISC-0974 Preclinical Data: Synergistic benefits with ESA or luspatercept in mice for enhanced iron mobilization and erythropoiesis.

4. DISC-3405 (Anti-TMPRSS6 monoclonal antibody):

• SAD/MAD Phase 1 data: dose-dependent increases in hepcidin, decreases in iron, and reductions in hemoglobin/reticulocytes.
• IV iron pulse study: 94% reduction in iron absorption.

Investment Thesis

• Targets rare hem diseases (EPP) and anemia (MF, CKD).
• Peak revenue projections: ~$17B for EPP (75%/70%/60% scenario), $600M+ for MF/CKD.
• Value drivers: lead assets advancing to Phase 3, partnerships, and radiological asset potential.

Key Catalysts

• 2H25: Bito NDA submission for EPP, '0974 Ph2 data releases.
• Pipeline: '3405 phase 2 trial in PV expected results 2026.

Top Risks

• Supply chain management dependency on CDMOs (manufacturing, cGMP compliance).
• Employee/contractor engagements with potential conflicts.
• Clinical/regulatory/financial hurdles for pipeline assets.

Market Cap & Analysts

• Market Cap: $18.0B
• Coverage by Jefferies; Analyst Certifications included.

Business Risks

• Reliance on single CDMO for supply chain.
• Employee/consultant engagements may create conflicts.
• Employee engagement and diversity recruitment efforts.

Valuation Summary

• PT: $111 (DCF-based), upside scenarios $143, downside $20.
• DCF Revenue Scenario:
  • Base: $111
  • Upside: $143 (+171%)
  • Downside: $20 (-62%)