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.
Myelofibrosis recruiting · Polycythemia Vera
Sickle Cell Disease recruiting
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
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
Tests DISC-3405
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
1 trial underway, not taking new volunteers
A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV)
Tests DISC-3405
Pick a condition. One email when a new trial opens, a trial changes or the FDA decides, from any company, not only Disc Medicine. Unsubscribe anytime.
- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
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.
Rare diseases Disc Medicine works on
Each disease page lists every open trial, not only Disc Medicine's, plus approved treatments.
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 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 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 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 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.
- National Organization for Rare Disorders (NORD): Erythropoietic Protoporphyria and X-Linked ProtoporphyriaErythropoietic Protoporphyria
- United Porphyrias Association: EPP and XLPErythropoietic Protoporphyria
- MDS FoundationMyelodysplastic Syndromes
- AAMDS International FoundationMyelodysplastic Syndromes
- National Organization for Rare Disorders (NORD)Myelofibrosis
- Cervantes F et al., New prognostic scoring system for primary myelofibrosis, Blood 2009Myelofibrosis
- National Organization for Rare Disorders (NORD)Polycythemia Vera
- Blood Cancer United (formerly The Leukemia & Lymphoma Society)Polycythemia Vera