Disc Medicine, Inc. · NASDAQ:IRON
Disc Medicine — Investor Presentation (June 2026)
41 slides from Disc Medicine's investor presentation, Jun 15, 2026
Slide 1: Corporate Presentation
Corporate Presentation June 15, 2026
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 its preclinical studies, clinical trials a
Slide 3: Bitopertin, DISC-0974, and DISC-3405 are investigational agents
Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide 3
Slide 4: Agenda
Agenda 1. Introduction 2. Bitopertin in EPP • Type A meeting summary and next steps • EPP data update: HELIOS open label extension 3. Selcodebart (DISC-0974) • RALLY-MF update • MF opportunity and HJV franchise next steps 4. DISC-3405 5. Closing Remarks and Q&A Session
Slide 5: Disc's hematology-focused pipeline
Disc’s hematology-focused pipeline Key programs driving upcoming catalysts PROGRAM Preclinical Phase 1 Phase 2 Phase 3 Heme Bitopertin HEME Synthesis GlyT1 Inhibitor Erythropoietic porphyrias (EPP and XLP) Modulation Oral, once daily Anemia of myelofibrosis (MF) Selcodebart (DISC-0974) Anti-HJV mono
Slide 6: Summary of Updates
Summary of Updates Bitopertin Selcodebart (DISC-0974) Update on Type A Meeting with FDA: Updated data from RALLY-MF Phase 2 trial in anemia of MF: • Aligned with the FDA that the Phase 3 APOLLO study can serve as the basis for CRL response • Meaningful, durable benefits on hemoglobin and transfusion
Slide 7: Agenda
Agenda 1. Introduction 2. Bitopertin in EPP • Type A meeting summary and next steps • EPP data update: HELIOS open label extension 3. Selcodebart (DISC-0974) • RALLY-MF update • MF opportunity and HJV franchise next steps 4. DISC-3405 5. Closing Remarks and Q&A Session
Slide 8: Bitopertin in EPP: Progressing toward CRL response by end of 2026
Bitopertin in EPP: Progressing toward CRL response by end of 2026 In Type A meeting, aligned with the FDA that the Phase 3 APOLLO study can serve as the basis for CRL response and, if successful, support a traditional approval APOLLO trial fully enrolled in March 2026 and topline data expected in Q4
Slide 9: HELIOS: EPP Open-Label Extension
HELIOS: EPP Open-Label Extension Study overview – data as of November 1, 2025 20 mg or 60 mg QD Bitopertin 60 mg QD PBO, 20 mg or 60 mg QD EOS/D1 W8 W16 W24 W52 Q24W PBO Bitopertin Bitopertin Bitopertin Total (n=21) (n=65) (n=86) Enrolled 21 65 86 Discontinued treatment 1 (5%) 3 (5%) 4 (5%) Comp
Slide 10: HELIOS Update: PPIX
HELIOS Update: PPIX Sustained reductions in PPIX for >1 year with continued bitopertin treatment, with greater reductions in participants who received continuous treatment with 60 mg of bitopertin PPIX Change from Baseline Bitopertin–Bitopertin: participants initially randomized to bitopertin (20 or
Slide 11: HELIOS Update: Light Tolerance
HELIOS Update: Light Tolerance Sustained, significant improvements in average light tolerance Mean Change in Light Tolerance Mean Change in Light Tolerance (hr) 10 Overall (n=85) Placebo-Bitopertin (n=20) Bitopertin-Bitopertin (n=65) 8 Placebo Bitopertin Overall Week 24 Bitopertin Bitopertin (FASa)
Slide 12: HELIOS Update: Safety
HELIOS Update: Safety Favorable longer-term safety profile (up to 2.5+ years exposure) • Median exposure: 25.3 months (range: 13.8 to 32.7 months) • 1 participant reported SAEs (unrelated) due to motor vehicle accident • 3 Grade 3 TEAE (unrelated): hypertransaminasaemia, tendon rupture, squamous cel
Slide 13: APOLLO trial overview
APOLLO trial overview Enrollment completed in March 2026; topline data expected Q4 2026 183 patients across sites in the US, Canada, N Size Europe, and Australia Robust Endpoint Trial Longitudinal analysis leverages robust model that 6-month treatment period; Fully enrolled March 2026 Duration demon
Slide 14: APOLLO Baseline Characteristics
APOLLO Baseline Characteristics Adolescent (n=34) Adult (n=149) Overall (n=183) Age (years), Mean 14 42 37 Adolescent, n (%) 34 0 34 (19%) Sex (female), n (%) 20 (59%) 87 (58%) 107 (59%) Diagnosis EPP 31 (91%) 140 (94%) 171 (93%) XLP 3 (9%) 9 (6%) 12 (7%) Prior EPP Treatment Afamelanotide 2 (6%) 50
Slide 15: Agenda
Agenda 1. Introduction 2. Bitopertin in EPP • Type A meeting summary and next steps • EPP data update: HELIOS open label extension 3. Selcodebart (DISC-0974) • RALLY-MF update • MF opportunity and HJV franchise next steps 4. DISC-3405 5. Closing Remarks and Q&A Session
Slide 16: Selcodebart (DISC-0974): Novel anti-HJV mAb to suppress hepcidin
Selcodebart (DISC-0974): Novel anti-HJV mAb to suppress hepcidin Designed to enhance iron availability to address a wide range of hematologic disorders Enables Reduces Increases RBC Hepcidin Iron Production Inhibits endogenous Releases iron stores Potential to treat production of hepcidin Enables GI
Slide 17: RALLY-MF: Study overview and baseline characteristics
RALLY-MF: Study overview and baseline characteristics Data as of April 27, 2026 Screening Treatment Period Follow-Up Optional Continuation (28 Days) (6 cycles, q28 days) (28 Days) (Up to 2 years) Key Study Endpoints Anemia response defined by cohort (TI, transfusion burden reduction, Hgb change); Ir
Slide 18: Updated RALLY-MF phase 2 data
Updated RALLY-MF phase 2 data Positive, durable benefits on hemoglobin and transfusion burden in anemia of MF across a broad range of patients Hepcidin by Transfusion Cohort Iron by Transfusion Cohort Hematologic Response 150 300 nTD Serum Hepcidin, ng/mL 125 TD Low 250 Serum Iron, ng/mL TD High Mea
Slide 19: Updated RALLY-MF data: nTD
Updated RALLY-MF phase 2 data nTD (n=31): Early-acting, meaningful, and durable hemoglobin increases with 55% of patients achieving major response and 68% achieving overall response nTD Hematologic Resposne nTD Hemoglobin Increase from Baseline Evaluable participants (n=31) Treatment period Optional
Slide 20: Updated RALLY-MF data: TD Low
Updated RALLY-MF phase 2 data TD Low (n=11): Meaningful, and durable hemoglobin increases translating to transfusion reduction, with 64% of patients achieving major response and 73% achieving overall response TD Low Hematologic Resposne TD Low Hemoglobin Increase from Baseline Evaluable participants
Slide 21: Updated RALLY-MF data: TD High
Updated RALLY-MF phase 2 data TD High (n=8): Meaningful transfusion reduction, with 50% of patients achieving major response and 88% achieving overall response TD High (n=8) Overall response 88 ≥50% reduction in transfusion requirement Major response 50 TI ≥12 weeks 0 20 40 60 80 100 % of Responders
Slide 22: Patient Reported Outcomes in RALLY-MF
Updated RALLY-MF phase 2 data nTD and TD Low patients experienced clinically significant improvement across multiple Patient Reported Outcomes FACIT-Fatigue (nTD patients) FACIT Fatigue (TD Low patients) 12 nTD - Major responders 12 TD Low - Major responders FACIT-Fatigue improvement 9 and Hgb chang
Slide 23: Efficacy regardless of concomitant JAK inhibitor use
RALLY-MF phase 2 data update Selcodebart has demonstrated efficacy regardless of concomitant JAK inhibitor use, supporting utilization across all anemic MF patients Hepcidin by JAKi Cohort Iron by JAKi Cohort Hemoglobin by JAKi Cohort Response by JAKi Cohort Evaluable participants (n=50) Evaluable p
Slide 24: RALLY-MF Safety
Updated RALLY-MF phase 2 data Safety Preferred Term nTD (n=35) TD Low (n=13) TD High (n=13) Overall (n=61) Any TEAE, n (%) 33 (94.3) 12 (92.3) 11 (84.6) 56 (91.8) Related TEAE, n (%) 6 (17.1) 4 (30.8) 5 (38.5) 15 (24.6) SAE, n (%) 8 (22.9) 1 (7.7) 3 (23.1) 12 (19.7) AESIs, n (%) 2 (5.7) 0 0 2 (3.3)
Slide 25: Myelofibrosis opportunity
Myelofibrosis opportunity Positioned for use across all anemia MF patients, regardless of background MF-directed therapy, setting up for a potential blockbuster opportunity Significant Unmet Need for Anemia-Focused Therapy ~$2.0B Total Est. 2024 JAK Inhibitor US Sales (MF) Anemia is associated with
Slide 26: Selcodebart: Emerging product profile for MF anemia therapy
Selcodebart: Emerging product profile aiming to address key needs for MF anemia therapy Key Needs for Anemia Therapy Selcodebart Emerging Product Profile ✓ Updated Phase 2 data show strong hematologic responses 1 Works across anemia severity levels across NTD, TD Low, and TD High patients 2 Works as
Slide 27: Selcodebart in other anemias of inflammation
Selcodebart in other anemias of inflammation Inflammatory bowel disease mouse model Mouse analog of selcodebart supressed hepcidin, increased serum iron, and increased hemoglobin in anemic IBD mice Treatment also demonstrated disease-modifying and anti-inflammatory effects Target Engagement Hematolo
Slide 28: RALLY IBD: Phase 2 trial design
RALLY IBD: Phase 2 trial design Initiated Q1 2026 Study Population Study Schema • N=21 • ≥18 years of age with IBD DISC-0974 60 mg Screen D1 D8 D15 D29 D43 D57 D85/EOS D92 D99 D113 D127 D141 EOS (sc) • Mild disease assessed by baseline R endoscopy at screening Scr Treatment Period Optional OLE for p
Slide 29: Anti-HJV franchise: Next steps and future development
Anti-HJV franchise: Next steps and future development Anemia of Myelofibrosis • Additional RALLY-MF data expected Q4 2026 • End of Phase 2 Meeting with FDA expected by end of year • Phase 3 Pivotal trial initiation expected H1 2027 Other Anemias of Inflammation • RALLY-IBD signal-seeking Phase 2 stu
Slide 30: Agenda
Agenda 1. Introduction 2. Bitopertin in EPP • Type A meeting summary and next steps • EPP data update: HELIOS open label extension 3. Selcodebart (DISC-0974) • RALLY-MF update • MF opportunity and HJV franchise next steps 4. DISC-3405 5. Closing Remarks and Q&A Session
Slide 31: Anti-TMPRSS6 mAb induces hepcidin
Anti-TMPRSS6 mAb induces hepcidin Limits iron levels with potential to address a wide range of hematologic disorders Limits Modulates Increases Iron RBC Hepcidin Availability Production Enables Endogenous Promotes Iron Restriction Erythrocytosis (PV) Production of Hepcidin Decreases GI Absorption In
Slide 32: DISC-3405 development plans
DISC-3405 development plans Advancing program into POC studies with Phase 2 polycythemia vera and planned Phase 1b sickle cell disease initiation anticipated by year end Phase 2 Proof-of-Concept Study in Polycythemia Vera • Strong proof of therapeutic hypothesis; clarity on regulatory development pa
Slide 33: DISC-3405 healthy volunteer data
DISC-3405 healthy volunteer data In healthy volunteers, DISC-3405 significantly increases hepcidin and decreases in iron, leading to reductions in hemoglobin and hematocrit that are expected to be beneficial in PV patients PK/PD: 300 mg SC SAD PK/PD: 150 mg PK/PD SC MAD Profile Hemoglobin Hemoglobin
Slide 34: DISC-3405: Polycythemia vera opportunity
DISC-3405: Polycythemia vera opportunity Multi-billion-dollar market with significant unmet need for an effective, safe, and convenient treatment to maintain HCT <45% Polycythemia Vera DISC-3405 Positioning ~150,000 US Patients Attractive Market Current Treatment Options ~75k treated patients; signi
Slide 35: RESTORE-PV: Polycythemia vera phase 2 trial design
RESTORE-PV: Polycythemia vera phase 2 trial design Initiated June 2025; significant interest in the study has led to a protocol amendment to increase the number of patients included Design Dose escalation Maintenance 1 Maintenance 2 (12 weeks) (20 weeks) (20 weeks) 150 mg x1 300 mg x1 Observation Pe
Slide 36: Iron restriction in sickle cell disease
Iron restriction in sickle cell disease Potential for iron restriction through inhibition of TMPRSS6 to benefit SCD by reducing HbS concentration Growing Body of Evidence for Iron Restriction for Disease Modification in Sickle Cell Disease DISC-3405 in a Townes Model 3 and 10 mg/kg IP weekly for 8 w
Slide 37: Sickle cell disease phase 1b trial design
Sickle cell disease phase 1b trial design Initiated October 2025 Design Dose escalation Optional Maintenance 4 weeks 8 weeks 8 weeks 12 weeks Cohort A 75 mg 150 mg 300 mg 300 mg HbSC or HbSS X1 dose X1 dose X1 dose Q4W (n=12) 4-week Screening safety follow-up Optional Cohort B (n≤12, HbSC or HbSS) D
Slide 38: Agenda
Agenda 1. Introduction 2. Bitopertin in EPP • Type A meeting summary and next steps • EPP data update: HELIOS open label extension 3. Selcodebart (DISC-0974) • RALLY-MF update • MF opportunity and HJV franchise next steps 4. DISC-3405 5. Closing Remarks and Q&A Session
Slide 39: Summary and next steps
Summary and next steps Program Summary Next Steps • Aligned with FDA on approach to CRL response APOLLO Phase 3 topline data expected Q4 2026 Bitopertin • Sustained PPIX reductions and light tolerance CRL response submission expected by end of 2026 Heme Synthesis improvements with bitopertin in HELI
Slide 40: Disc Medicine: Built for Sustainable Growth
Disc Medicine: Built for Sustainable Growth Three programs addressing blockbuster markets with significant potential for expansion Bitopertin Selcodebart (DISC-0974) DISC-3405 Phase 3 Ongoing POC Established POC Study Initiated CRL response expected to be Additional MF data and Phase 3 Initial data
Slide 41: Q&A
@ disc medicine - Q&A