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 UpdatesBitopertin
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
Agenda01
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 StepsCollaborate 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
Agenda01
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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