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DISC-3405

Phase 2

Polycythemia Vera (PV) | Small molecule | Hematology |Disc Medicine, Inc.|Trials Updated: Oct 5, 2026

DISC-3405 development status

Highest phase Phase 2
Registered trials 3 across 1 sponsor since Sep 2023

DISC-3405 target and mechanism

Molecular targetTMPRSS6
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials2
Total Enrollment84

FDA Designations

ORPHAN_DRUG

DISC-3405 clinical trials

DISC-3405 · 2 trials · 2 indications

Phase 2 1Phase 1 1
NCT06985147A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV)Polycythemia Vera (PV)
ACTIVE NOT_RECRUITING60 Analytics
PHASE2ACTIVE NOT_RECRUITING
A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV)
Polycythemia Vera (PV)Unlock trial analytics

Study Endpoints

Primary Endpoints

Number of participants with treatment-related adverse events as assessed by CTCAE
Up to 365 days

Proportion of participants with treatment-emergent adverse events

Incidence of clinically abnormal vital signs
Up to 365 days

Proportion of participants with changes in vital signs

Incidence of clinically abnormal physical exam
Up to 365 days

Proportion of participants with changes in physical examinations

Incidence of clinically abnormal electrocardiograms
Up to 365 days

Proportion of participants with changes in electrocardiograms (ECGs)

Incidence of abnormal laboratory test results
Up to 365 days

Proportion of participants with changes in clinical laboratory results

Safety and tolerability of DISC-3405 administration in participants with SCD
Up to 36 weeks

Proportion of participants with treatment-emergent adverse events (TEAEs), changes in vital signs, changes in physical examinations, changes in electrocardiograms (ECGs), and changes in clinical laboratory results

Secondary Endpoints

Proportion of participants achieving therapeutic response, defined as absence of phlebotomy eligibility, during the maintenance period
Up to 365 days
Number of phlebotomies during the maintenance and optimization periods
Up to 365 days
Proportion of participants achieving therapeutic response, defined as absence of phlebotomy eligibility, during the optimization period
Up to 365 days
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Within-participant dose escalationEXPERIMENTALThis is an open-label, multicenter, within-participant dose escalation study examining up to 2 dose levels of DISC-3405.

Interventions

NameTypeDescription
DISC-3405DRUGDISC-3405 is administered subcutaneously.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites15

Inclusion Criteria: 1. Aged 18 years or older at the time of signing the informed consent form (ICF). 2. Meet revised 2022 World Health Organization (WHO) criteria for the diagnosis of PV. 3. Complete blood count values at Screening of HCT \<45% or HCT \<48% if followed by a phlebotomy within 2 wee...

Countries:United States
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Competitive Landscape -Polycythemia Vera 19 trials (matched to "Polycythemia Vera (PV)")

Recent Changes (Last 90 Days)

LOWOct 5, 2026NCT07187973lastUpdatePostDate: changed
LOWOct 5, 2026NCT07187973lastUpdatePostDate: changed
LOWSep 23, 2026NCT07187973lastUpdatePostDate: changed
LOWSep 23, 2026NCT07187973lastUpdatePostDate: changed
LOWSep 16, 2026NCT07187973lastUpdatePostDate: changed
LOWSep 16, 2026NCT07187973lastUpdatePostDate: changed
LOWAug 20, 2026NCT07187973lastUpdatePostDate: changed
LOWAug 20, 2026NCT07187973lastUpdatePostDate: changed
LOWAug 20, 2026NCT07187973lastUpdatePostDate: changed
LOWAug 20, 2026NCT07187973lastUpdatePostDate: changed
MEDIUMJul 31, 2026NCT06985147Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMJul 31, 2026NCT06985147Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWJul 30, 2026NCT07187973lastUpdatePostDate: changed
LOWJul 30, 2026NCT07187973lastUpdatePostDate: changed

Frequently asked questions about DISC-3405

What is DISC-3405?

DISC-3405 is an investigational small molecule being developed by Disc Medicine, Inc. for hematologic conditions. It is being studied in polycythemia vera (PV) and sickle cell disease (SCD). DISC-3405 has received an orphan drug designation from the FDA and remains in clinical development.

What is DISC-3405 used for in polycythemia vera?

DISC-3405 is being studied as a treatment for polycythemia vera, a blood disorder characterized by excess red blood cell production. A Phase 2 open-label study is evaluating the drug in adults with PV. It is investigational and has not been approved for any use.

What does DISC-3405 target?

DISC-3405 targets TMPRSS6, a transmembrane serine protease. By acting on TMPRSS6, the small molecule is designed to influence iron regulation pathways relevant to red blood cell production. This mechanism is being explored in polycythemia vera and sickle cell disease.

Who is developing DISC-3405?

DISC-3405 is being developed by Disc Medicine, Inc., a hematology-focused biopharmaceutical company. Disc Medicine trades on the Nasdaq under the ticker symbol IRON. The company is running clinical studies of DISC-3405 in polycythemia vera and sickle cell disease.

What phase is DISC-3405 in?

DISC-3405 is in Phase 2 clinical development. A Phase 2 open-label study in polycythemia vera is active but not recruiting, and a Phase 1b open-label study in sickle cell disease is recruiting. The drug is investigational and has not been approved by the FDA.

What clinical trials is DISC-3405 in?

DISC-3405 is being evaluated in two registered studies. NCT06985147 is a Phase 2 open-label trial in polycythemia vera with 60 participants, currently active but not recruiting. NCT07187973 is a Phase 1b open-label trial in sickle cell disease with 24 participants, currently recruiting in the United States.