Disc Medicine, Inc. · NASDAQ:IRON
Disc Medicine — 2025 ASH Management Call
31 slides from Disc Medicine's conference presentation, Dec 8, 2025
Slide 1: 2025 ASH Management Call
2025 ASH Management Call December 7, 2025 Confidential Exhibit 99.1
Slide 2: Disclaimer and FLS
Disclaimer and FLS This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to: (i) the registrational pathway for bitope
Slide 3: Investigational agents not approved for use
Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide CONFIDENTIAL
Slide 4: Agenda
Agenda 03 DISC-0974 Updated Data in Anemia of MF Will Savage, MD, PhD, Chief Medical Officer MF Anemia Market Opportunity Jonathan Yu, Chief Operating Officer Closing Remarks John Quisel, JD, PhD, Chief Executive Officer 05 Q&A Session 04 Bitopertin Updates John Quisel, JD, PhD, Chief Executive Offi
Slide 5: Summary of Updates
Summary of Updates CONFIDENTIAL Consistent and substantial ↓ hepcidin and ↑ iron Positive, durable benefits on hemoglobin and transfusion burden in anemia of MF across a broad range of MF patients Similar response rates regardless of underlying MF-directed therapy Clinically meaningful improvements
Slide 6: Agenda
Agenda 03 DISC-0974 Updated Data in Anemia of MF Will Savage, MD, PhD, Chief Medical Officer MF Anemia Market Opportunity Jonathan Yu, Chief Operating Officer Closing Remarks John Quisel, JD, PhD, Chief Executive Officer 05 Q&A Session 04 Bitopertin Updates John Quisel, JD, PhD, Chief Executive Offi
Slide 7: Commitment to ensuring access to bitopertin for patients quickly
Disc is committed to ensuring access to bitopertin for patients as quickly as possible and is advancing commercial readiness activities to support potential launch of bitopertin on accelerated timeline, if approved NDA=New Drug Application Bitopertin selected for the FDA’s Commissioner’s National Pr
Slide 8: Bitopertin Next Steps
Bitopertin Next Steps Collaborate with FDA throughout their ongoing review Continue accelerated activities to support a potential US approval and launch by end of January 2026 Drive enrollment of ongoing APOLLO confirmatory trial in US, UK, Canada, Australia and Europe Further details on commerciali
Slide 9: Agenda
Agenda 03 DISC-0974 Updated Data in Anemia of MF Will Savage, MD, PhD, Chief Medical Officer MF Anemia Market Opportunity Jonathan Yu, Chief Operating Officer Closing Remarks John Quisel, JD, PhD, Chief Executive Officer 05 Q&A Session 04 Bitopertin Updates John Quisel, JD, PhD, Chief Executive Offi
Slide 10: DISC-0974: Novel Anti-HJV mAb to Suppress Hepcidin
DISC-0974: Novel Anti-HJV mAb to Suppress Hepcidin Designed to enhance iron availability to address a wide range of hematologic disorders Releases iron stores Enables GI absorption Increases Iron Reduces Hepcidin Inhibits endogenous production of hepcidin Potential to treat wide range of anemias Ena
Slide 11: RALLY-MF: Study Overview and Baseline Characteristics
RALLY-MF: Study Overview and Baseline Characteristics Data as of October 16 CONFIDENTIAL nTD (n=30) TD Low (n=10) TD High (n=7) Overall (n=47) Age, median (range), years 69.5 (54, 83) 74.5 (31, 87) 72 (61, 87) 70 (31, 87) Concomitant medication, n (%) JAK inhibitor Ruxolitinib Momelotinib Pacritinib
Slide 12: RALLY-MF: Interim Results Pharmacodynamics
RALLY-MF: Interim Results Pharmacodynamics DISC-0974 demonstrated consistent decreases in hepcidin and increases in serum iron across patients Similar results seen regardless of transfusion status nTD TD Low TD High Hepcidin by Transfusion Cohort Iron by Transfusion Cohort
Slide 13: RALLY-MF: Interim Results Hemoglobin and FACIT-Fatigue
RALLY-MF: Interim Results Hemoglobin and FACIT-Fatigue Overall, nTD and TD Low patients had meaningful responses on hemoglobin and FACIT-Fatigue with greatest improvements seen in those achieving a major hematologic response Hemoglobin FACIT-Fatigue Hemoglobin FACIT-Fatigue Hemoglobin analysis exclu
Slide 14: RALLY-MF: Hematologic Response: nTD and TD Low Patients
RALLY-MF: Interim Results Hematologic Response: nTD and TD Low Patients Hematologic response achieved across transfusion cohorts and regardless of concomitant JAKi use >60% overall response rate and >50% major response rate in both nTD and TD Low cohorts nTD (n=24) TD Low (n=7) Mean Hgb ↑ ≥1.5 g/dL
Slide 15: RALLY-MF: Hematologic Response: TD High
RALLY-MF: Interim Results Hematologic Response: TD High 67% of TD High patients with at least 85 days on study had ≥ 50% reduction in transfusion requirement Initial data for additional N=3 TD High patients trending towards major response of TI >12 weeks Abbreviations: Hgb = hemoglobin; TI = transfu
Slide 16: RALLY-MF: Patients on Concomitant JAK inhibitors
RALLY-MF: Interim Results Patients on Concomitant JAK inhibitors Similar responses on hepcidin, iron, and hematologic parameters regardless of concomitant JAKi use Overall, 50% of participants receiving concomitant JAKi therapy achieved a major hematologic response with similar responses regardless
Slide 17: RALLY-MF: Interim Results Safety
RALLY-MF: Interim Results Safety Preferred Term nTD (n=30) TD Low (n=10) TD High (n=7) Overall (n=47) Any TEAE, n (%) 23 (76.7) 7 (70.0) 5 (71.4) 35 (74.5) Related TEAE, n (%) 3 (10.0) 4 (40.0) 4 (57.1) 11 (23.4) SAE, n (%) 7 (23.3) 1 (10.0) 1 (14.3) 9 (19.1) AESIs, n (%) 2 (6.7) 0 0 2 (4.3) ≥ Grade
Slide 18: DISC-0974: Emerging product profile for MF anemia therapy
DISC-0974: Emerging product profile aiming to address key needs for MF anemia therapy CONFIDENTIAL Key Needs for Anemia Therapy Works across anemia severity levels 1 Works as a monotherapy 2 Works with any MF-directed therapy 3 Supports optimization MF-directed therapy regimen 4 Superior response ra
Slide 19: MF Anemia Opportunity
MF Anemia Opportunity Greater than $2B addressable market in the US ~22K US addressable MF patients with anemia Severe Condition Progressive in nature, with significant impact on prognosis and QoL Well Characterized Market MF expert network and treatment pathway are well-established Clear Unmet Need
Slide 20: Next Steps
Next Steps CONFIDENTIAL Other Anemias of Inflammation Phase 2 study in anemia of IBD expected to initiate early 2026 Exploratory work in additional anemia indications IND-enabling activities for long-acting anti-HJV (DISC-0998) Phase 2 data expected H2 2026 EOP2 meeting expected H2 2026 Pivotal tria
Slide 21: Agenda
Agenda 03 DISC-0974 Updated Data in Anemia of MF Will Savage, MD, PhD, Chief Medical Officer MF Anemia Market Opportunity Jonathan Yu, Chief Operating Officer Closing Remarks John Quisel, JD, PhD, Chief Executive Officer 05 Q&A Session 04 Bitopertin Updates John Quisel, JD, PhD, Chief Executive Offi
Slide 22: Anti-TMPRSS6 mAb Induces Hepcidin
Anti-TMPRSS6 mAb Induces Hepcidin Designed to limit iron levels with potential to address a wide range of hematologic disorders Promotes Iron Restriction Decreases GI Absorption Limits Iron Availability Increases Hepcidin Enables Endogenous Production of Hepcidin Erythrocytosis (PV) Ineffective Eryt
Slide 23: DISC-3405 Development Plans
DISC-3405 Development Plans Advancing program into POC studies with an ongoing Phase 2 trial in polycythemia vera and Phase 1b in sickle cell disease Phase 1 SAD/MAD in HV Phase 2 Proof-of-Concept Study in Polycythemia Vera Exploration of Other Indications Demonstrate proof-of-mechanism (hepcidin, i
Slide 24: Initial Market Opportunities
Initial Market Opportunities Offering derisked, differentiated product profiles in high unmet need areas CONFIDENTIAL Polycythemia Vera ~150,000 US Patients ~75k treated patients; significant room for market development; operational synergies with MF treaters Treatments offer suboptimal HCT control,
Slide 25: Polycythemia Vera Phase 2 Study: RESTORE-PV
Polycythemia Vera Phase 2 Study: RESTORE-PV Initiated June 2025; significant interest in the study has led to a protocol amendment to increase the number of patients included Endpoints: Safety, PK, PD (hepcidin, iron, hematocrit), phlebotomy rate Initial Data Expected H2 2026 Design Maintenance 2 (2
Slide 26: Sickle Cell Disease Phase 1b Study
Sickle Cell Disease Phase 1b Study Initiated October 2025; data expected H2 2026 Endpoints: Safety, PK, PD (hepcidin, iron, hematologic parameters, hemolysis markers) Exploratory endpoints: PROs (pain, fatigue), changes in SCD complication rates Design Optional Maintenance Dose escalation Screening
Slide 27: Agenda
Agenda 01 Introduction and Summary John Quisel, JD, PhD, Chief Executive Officer 03 DISC-0974 Updated Data in Anemia of MF Will Savage, MD, PhD, Chief Medical Officer MF Anemia Market Opportunity Jonathan Yu, Chief Operating Officer Closing Remarks John Quisel, JD, PhD, Chief Executive Officer 05 Q&
Slide 28: Summary of Updates
Summary of Updates CONFIDENTIAL Preparing for potential bitopertin approval and launch by end January 2026 Initial RALLY-MF data supports DISC-0974 target profile in anemia of MF; planning to advance to pivotal study following Ph 2 topline and EOP2 meeting with FDA First in-patient studies for DISC-
Slide 29: Disc's Hematology-Focused Pipeline
Disc’s Hematology-Focused Pipeline Key Programs Driving Upcoming Catalysts Preclinical Phase 1 Bitopertin† GlyT1 Inhibitor Oral, once-daily Phase 2 Phase 3 / Confirmatory DISC-0974‡ Anti-HJV monoclonal antibody Subcutaneous, once-monthly DISC-0998‡ Anti-HJV monoclonal antibody Extended half-life Ery
Slide 30: Projected Upcoming Milestones and Events
Projected Upcoming Milestones and Events Multiple additional data catalysts anticipated in the next 18 months Program Indication H2 2025 H1 2026 H2 2026 Erythropoietic Porphyrias (EPP and XLP) NDA Submission CNPV Received NDA Filing Decision Potential Approval (by end of January) Launch and commerci
Slide 31: Agenda
Agenda 01 Introduction and Summary John Quisel, JD, PhD, Chief Executive Officer 03 DISC-0974 Updated Data in Anemia of MF Will Savage, MD, PhD, Chief Medical Officer MF Anemia Market Opportunity Jonathan Yu, Chief Operating Officer 04 Bitopertin Updates John Quisel, JD, PhD, Chief Executive Officer