Disc Medicine, Inc.NASDAQ: IRON

2025 ASH Management Call FINAL

· MarketScreener
2025 ASH Management Call

December 7, 2025

1Confidential





Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide


Agenda

01

Introduction and Summary

John Quisel, JD, PhD, Chief Executive Officer

02

Bitopertin Updates

John Quisel, JD, PhD, Chief Executive Officer

DISC-0974

03

  • Updated Data in Anemia of MF

    Will Savage, MD, PhD, Chief Medical Officer

  • MF Anemia Market Opportunity

    Jonathan Yu, Chief Operating Officer

    04

    DISC-3405 Updates

    Jonathan Yu, Chief Operating Officer

    Will Savage, MD, PhD, Chief Medical Officer

    05

    Closing Remarks

    John Quisel, JD, PhD, Chief Executive Officer



    Summary of Updates

    Bitopertin

    Heme Synthesis Modulator

    • NDA accepted on

      November 28

    • Expecting to launch by end of January 2026, if approved

    • Commercialization planning is well underway

    DISC-0974

    Hepcidin Suppression

    • 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 in fatigue

    DISC-3405

    Hepcidin Induction

    • Phase 2 study ongoing in

      polycythemia vera

      - Rapid enrollment has led to study expansion

    • Phase 1b study initiated

      in sickle cell disease

    • Initial data from both studies expected by the end of 2026





    Agenda

    01

    Introduction and Summary

    John Quisel, JD, PhD, Chief Executive Officer

    02

    Bitopertin Updates

    John Quisel, JD, PhD, Chief Executive Officer

    DISC-0974

    03

  • Updated Data in Anemia of MF

    Will Savage, MD, PhD, Chief Medical Officer

  • MF Anemia Market Opportunity

Jonathan Yu, Chief Operating Officer

04

DISC-3405 Updates

Jonathan Yu, Chief Operating Officer

Will Savage, MD, PhD, Chief Medical Officer

05

Closing Remarks

John Quisel, JD, PhD, Chief Executive Officer



Bitopertin NDA Process

Expected review period of 1-2 months under CNPV program



Bitopertin selected for the FDA's Commissioner's

National Priority Voucher (CNPV) Pilot Program

NDA Submitted

September 29

Key Program Benefits

  • Faster review times (expected 1-2 months)

  • Enhanced communication throughout the process

  • Multidisciplinary team-based evaluation

  • Potential for accelerated approval

  • Maintains FDA's rigorous safety and efficacy standards

    NDA Accepted

    November 28

    Anticipated Action Date

    By end of Jan. '26

    Anticipated Drug Supply Availability

    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

    By end of Jan. '26



    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 commercialization and launch planning to be shared at the JP Morgan Healthcare Conference in January 2026



    Agenda

    01

    Introduction and Summary

    John Quisel, JD, PhD, Chief Executive Officer

    02

    Bitopertin Updates

    John Quisel, JD, PhD, Chief Executive Officer

    DISC-0974

    03

    • Updated Data in Anemia of MF

      Will Savage, MD, PhD, Chief Medical Officer

    • MF Anemia Market Opportunity

Jonathan Yu, Chief Operating Officer

04

DISC-3405 Updates

Jonathan Yu, Chief Operating Officer

Will Savage, MD, PhD, Chief Medical Officer

05

Closing Remarks

06

Q&A Session



John Quisel, JD, PhD, Chief Executive Officer



DISC-0974: Novel Anti-HJV mAb to Suppress Hepcidin Designed to enhance iron availability to address a wide range of hematologic disorders

Reduces Hepcidin

Increases Iron

Enables

RBC

Production



Inhibits endogenous

production of hepcidin

Releases iron stores

Enables GI absorption

Potential to treat

wide range of anemias

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