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Disc Medicine

Rare disease drugmaker · based in Watertown, United States · founded 2017 · ticker IRON · discmedicine.com

Disc Medicine works on 5 rare diseases we track, including Erythropoietic Protoporphyria, Myelodysplastic Syndromes and 3 more. It has 3 active clinical trials, and 2 are looking for volunteers now.

Disc Medicine is a hematology company developing disease-modifying treatments for erythropoietic porphyrias and other rare hematologic disorders.

Disc Medicine pipeline

Each row lists the diseases Disc Medicine is studying at that stage, from the ones closest to approval down to the earliest work.

Phase 2
Mid-size studies that check whether a drug works and at what dose.
Phase 1
First studies in people, mostly checking safety.

Disc Medicine clinical trials

These are Disc Medicine trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

2 trials looking for volunteers now

Looking for volunteersPhase 1Recently updatedNCT07187973

A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)

Tests DISC-3405

For Sickle Cell Disease

This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.

Ages 18 Years+11 locations
View full study
Looking for volunteersPhase 1/2Recently updatedNCT05320198

Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia

Tests DISC-0974

For Myelofibrosis; Anemia, Anemia, Myelofibrosis, Myelofibrosis Due to and Following Polycythemia Vera and Primary Myelofibrosis

This phase 1b/2a open-label study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of DISC-0974 as well as categorize the effects on hematologic response in participants with myelofibrosis or myelodysplastic syndrome and anemia.

Ages 18 Years+35 locations
View full study

1 trial underway, not taking new volunteers

Underway, not taking new volunteersPhase 2Recently updatedNCT06985147

A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV)

Tests DISC-3405

For Polycythemia Vera (PV)

This open-label, multicenter, within-participant dose escalation study examining up to 2 dose levels of DISC-3405 will assess the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of DISC-3405 in participants with polycythemia vera (PV).

Ages 18 Years+15 locations
View full study
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More about Disc Medicine

The company's lead candidate bitopertin (a GlyT1 inhibitor for erythropoietic protoporphyria) received a Complete Response Letter from the FDA in February 2026; on October 19, 2026 Disc reported that the Phase 3 APOLLO trial met all primary and secondary endpoints and said it plans to resubmit by the end of 2026 with an FDA decision anticipated by mid-2027.

Disc Medicine also develops DISC-0974 for myelofibrosis-associated anemia and DISC-3405, an anti-TMPRSS6 monoclonal antibody in a Phase 2 trial for polycythemia vera and a Phase 1b trial for sickle cell disease.

Where Disc Medicine trials take place

The trials above run at 61 study sites in 2 countries. Tap a country to see its states, cities and hospitals.

United States
57▼
Australia
4▼

Rare diseases Disc Medicine works on

Each disease page lists every open trial, not only Disc Medicine's, plus approved treatments.

Erythropoietic Protoporphyria

Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are rare inherited disorders in which a light-sensitive chemical called protoporphyrin builds up in red blood cells and skin, so...

How common: NORD estimates EPP affects about 1 in 75,000 to 1 in 200,000 people in Europe and says the number in the US is unknown

Myelodysplastic Syndromes

Myelodysplastic syndromes are a group of blood cancers in which the bone marrow fails to produce enough healthy blood cells.

How common: 10,000 to 15,000 new cases annually in the U.S.; estimated 60,000 to 170,000 Americans living with MDS

Myelofibrosis

Myelofibrosis is a myeloproliferative neoplasm in which bone marrow develops scarring (fibrosis), leading to anemia, enlarged spleen, and abnormal blood cell counts.

How common: Approximately 0.5-1.5 cases per 100,000 people; median age of diagnosis is 65 years

Polycythemia Vera

Polycythemia Vera is a myeloproliferative neoplasm in which bone marrow produces too many red blood cells, leading to thick blood that carries oxygen less effectively and increases clot risk.

How common: About 44 to 57 per 100,000 people in the US, from 2 large health plans in 2008 to 2010 (Mehta et al., Leukemia and Lymphoma 2014); median age of diagnosis is 65 years

Sickle Cell Disease

Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion.

How common: About 100,000 Americans with SCD; about 1 in 365 Black or African American births and 1 in 16,300 Hispanic American births

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Disc Medicine's own site is discmedicine.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Questions about Disc Medicine