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Incyte Corporation

Drugmaker with rare disease work · based in Wilmington, United States · founded 1991 · ticker INCY · incyte.com

Incyte Corporation works on 19 rare diseases we track, including Acute Myeloid Leukemia, Aplastic Anemia and 17 more. It has 45 active clinical trials, and 36 are looking for volunteers now. It also has 4 FDA-approved rare disease drugs.

Incyte Corporation develops targeted therapies for rare blood cancers, graft-versus-host disease, and other rare hematologic and immunologic conditions. Key products include Jakafi (ruxolitinib), a JAK inhibitor approved for myelofibrosis, polycythemia vera, acute graft-versus-host disease, and chronic graft-versus-host disease; Pemazyre (pemigatinib) for cholangiocarcinoma and myeloid/lymphoid neoplasms with an FGFR1 rearrangement; Zynyz (retifanlimab-dlwr) for Merkel cell carcinoma; and Niktimvo (axatilimab), an anti-CSF1R antibody approved in 2024 for chronic graft-versus-host disease.

Incyte Corporation pipeline

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

Approved
Drugs the FDA has approved.
Phase 3
Large studies, usually the last step before a company asks the FDA for approval.
Phase 2
Mid-size studies that check whether a drug works and at what dose.

Chronic Graft-versus-Host Disease 5 trials recruiting · Myelofibrosis 4 trials recruiting · Cholangiocarcinoma 3 trials recruiting · Acute Myeloid Leukemia 2 trials recruiting · Myelodysplastic Syndromes 2 trials recruiting · Non-Hodgkin Lymphoma 2 trials recruiting · Hodgkin Lymphoma 2 trials recruiting · Neurofibromatosis Type 1 recruiting · Immune Thrombocytopenia recruiting · Grade 3b Follicular Lymphoma recruiting · and 9 more

Phase 1
First studies in people, mostly checking safety.

Acute Myeloid Leukemia 2 trials recruiting · Multiple Myeloma 2 trials recruiting · Cholangiocarcinoma recruiting · Non Hodgkin Lymphoma recruiting · Polycythemia Vera recruiting · Chronic Graft-versus-Host Disease recruiting · Myelodysplastic Syndromes

Other studies
Studies with no phase listed, such as registries and natural history studies.

Incyte Corporation clinical trials

These are Incyte Corporation 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.

36 trials looking for volunteers now

Looking for volunteersPhase 1/2Recently updatedNCT03934372

Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors

Tests Ponatinib

For Acute Myeloid Leukemia, Accelerated Phase Chronic Myeloid Leukemia, Blast Phase Chronic Myeloid Leukemia, Chronic Phase Chronic Myeloid Leukemia and Acute Lymphoblastic Leukemia

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ponatinib in children aged 1 to \< 18 years with advanced leukemias, lymphomas, and solid tumors.

Ages 1 Year - 17 Years23 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT07124078

A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)

Tests INCA034176, Best available Treatment (BAT)

For Chronic Graft-versus-host-disease

This study will be conducted to compare Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy.

Ages 2 Years - 17 Years42 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07623200

A Phase 3 Study of INCA033989 Versus Best Available Therapy in Participants With Essential Thrombocythemia

Tests INCA033989, Best Available Treatment

For Essential Thrombocythemia

This study is being conducted to evaluate INCA033989 versus best available therapy in participants with essential thrombocythemia and a CALR mutation previously treated with cytoreductive therapy.

Ages 18 Years+196 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT04374305

Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)

Tests Brigatinib, Neratinib, Retifanlimab, Bevacizumab

For Neurofibromatosis Type 2, Vestibular Schwannoma, Non-vestibular Schwannoma, Meningioma and Ependymoma

This is a multi-arm phase II platform-basket screening study designed to test multiple experimental therapies simultaneously in patients with NF2-related schwannomatosis (NF2-SWN, formerly known as neurofibromatosis type 2) with associated progressive tumors of vestibular schwannomas (VS), non-vestibular schwannomas (non-VS), meningiomas, and ependymomas. This Master Study is being conducted as a "basket" study that may allow people with multiple tumor types associated with NF2-SWN to receive new drugs throughout this study. Embedded within the Master Study are individual drug substudies. * Investigational Drug Sub-study A: Brigatinib * Investigational Drug Sub-study B: Neratinib * Investigational Drug Sub-study C: Retifanlimab plus bevacizumab

Ages 12 Years+6 locations
View full study
Looking for volunteersPhase 1/2Recently updatedNCT03681561

Nivolumab With Ruxolitinib in Relapsed or Refractory Classical Hodgkin Lymphoma

Tests Ruxolitinib, Nivolumab

For Hodgkin Lymphoma

This is a Phase I/II, multicenter, open-label, dose escalation/dose-expansion study to evaluate the tolerability, safety, and the maximum tolerated dose (MTD) of ruxolitinib when given with fixed dose nivolumab in patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

Ages 18 Years+6 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT07104565

Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders

Tests INCA000585

For Immune Thrombocytopenia

This study will evaluate the safety and efficacy of tafasitamab in adult participants with primary autoimmune blood cell disorders.

Ages 18 Years+39 locations
View full study
Looking for volunteersPhase 1/2Recently updatedNCT04061421

Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes Overlap Syndromes (ABNL-MARRO)

Tests ASTX727, Itacitinib, Ruxolitinib

For MDS/MPN

ABNL-MARRO (A Basket study of Novel therapy for untreated MDS/MPN and Relapsed/Refractory Overlap Syndromes) is an international European-American cooperation providing the framework for collaborative studies to advance treatment of myelodysplastic/myeloproliferative neoplasms (MDS/MPN) and explore clinical-pathologic markers of disease severity, prognosis and treatment response. ABNL MARRO 001 (AM-001) is an Open label, phase 1/2 study within the framework of the ABNL-MARRO that will test novel treatment combinations in MDS/MPN. Each Arm of AM-001 will test an active myeloid target compound in combination with ASTX727, an oral drug combining fixed doses of the DNA methyltransferase inhibitor (DNMTi) decitabine and the cytidine deaminase inhibitor E7727, also known as cedazuridine in a single tablet.

Ages 18 Years+4 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT05890352

Study Adding Drugs to Usual Treatment for Large B-Cell Lymphoma That Returned or Did Not Respond to Treatment

Tests Biospecimen Collection, Computed Tomography, Lenalidomide, Magnetic Resonance Imaging, Positron Emission Tomography, Quality-of-Life Assessment, Questionnaire Administration, Tafasitamab, Tazemetostat, Zanubrutinib

For Grade 3b Follicular Lymphoma, High Grade B-Cell Lymphoma, High Grade B-Cell Lymphoma With MYC and BCL2 and/or BCL6 Rearrangements, Recurrent Diffuse Large B-Cell Lymphoma and Refractory Diffuse Large B-Cell Lymphoma

This phase 2 trial studies the side effects and best dose of tazemetostat and zanubrutinib in combination with tafasitamab and lenalidomide, and to see how well these combinations work in treating patients with large B-cell lymphoma that returned or did not respond to earlier treatment. Tazemetostat is in a class of medications called EZH2 inhibitors. It helps to stop the spread of cancer cells. Zanubrutinib is in a class of medications called kinase inhibitors. It works by blocking the action of the abnormal protein that signals cancer cells to multiply. This helps stop the spread of cancer cells. tafasitamab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. Lenalidomide is in a class of medications called immunomodulatory agents. It works by helping the bone marrow to produce normal blood cells and by killing abnormal cells in the bone marrow. The addition of tazemetostat or zanubrutinib to tafasitamab and lenalidomide may be able to shrink the cancer or extend the time without cancer symptoms coming back.

Ages 18 Years+104 locations
View full study
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FDA-approved drugs from Incyte Corporation

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Jakafi ruxolitinib

For Polycythemia Vera, Myelofibrosis and Chronic Graft-versus-Host Disease. Approved Nov 16, 2011. Taken by mouth.

Niktimvo axatilimab-csfr

For Chronic Graft-versus-Host Disease. Approved Aug 14, 2024. Taken by intravenous route.

Pemazyre pemigatinib

For Cholangiocarcinoma. Approved Apr 17, 2020. Taken by mouth.

Zynyz retifanlimab-dlwr

For Merkel Cell Carcinoma. Approved Mar 22, 2023. Taken by intravenous route.

Where Incyte Corporation trials take place

The trials above run at 707 study sites in 28 countries. Tap a country to see its states, cities and hospitals.

United States
366▼
Italy
48▼
Spain
41▼
United Kingdom
36▼
Germany
32▼
Japan
31▼
Australia
21▼
France
20▼
Belgium
16▼
Canada
14▼
Netherlands
10▼
Turkey (Türkiye)
9▼

Rare diseases Incyte Corporation works on

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

Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a fast-growing cancer of the bone marrow in which immature white blood cells called blasts crowd out normal blood cells, causing fatigue, infections and bleeding...

How common: The National Cancer Institute's SEER program estimates 22,720 new US cases and 11,500 deaths in 2026, about 1.1% of new cancers, and 83,311 people were living with AML in 2023

Aplastic Anemia

Aplastic anemia is a rare bone marrow failure syndrome characterized by pancytopenia resulting from absent or severely reduced hematopoiesis.

How common: 1-2 cases per 1 million people per year

Cholangiocarcinoma

Cholangiocarcinoma is a rare and aggressive cancer that forms in the bile ducts, the thin tubes that carry digestive fluid (bile) from the liver to the small intestine.

How common: About 8,000 new cases per year in the U.S.; rising incidence worldwide

Chronic Graft-versus-Host Disease

Chronic graft-versus-host disease is an immune-mediated complication after allogeneic stem cell or bone marrow transplant, in which donor immune cells attack the recipient's tissues.

How common: Affects 30-70% of patients who receive allogeneic hematopoietic stem cell transplant; about 14,000 Americans were living with chronic GVHD in 2016, according to a claims study funded in part by Kadmon (Bachier et al., Transplantation and Cellular Therapy, 2021)

Essential Thrombocythemia

Essential Thrombocythemia is a myeloproliferative neoplasm characterized by overproduction of platelets by bone marrow, resulting in very high platelet counts.

How common: About 1 new case per 100,000 people each year, with an estimated 30 to 60 per 100,000 living with it (NORD); average age at diagnosis is in the mid-fifties

Fibrodysplasia Ossificans Progressiva

Fibrodysplasia Ossificans Progressiva (FOP) is an extremely rare genetic disorder characterized by progressive heterotopic ossification, where soft tissues (muscles, tendons, ligaments) gradually...

How common: Extremely rare, about 1 in 1 million to 1 in 2 million people worldwide

Gastrointestinal Stromal Tumor

Gastrointestinal stromal tumors (GISTs) are the most common mesenchymal tumors of the gastrointestinal tract, arising from the interstitial cells of Cajal or their precursors.

How common: About 6,000 new cases per year in the U.S. (American Cancer Society); true incidence may be higher as small GISTs are often incidentally discovered

Hodgkin Lymphoma

Hodgkin lymphoma is a cancer of the lymphatic system characterized by the presence of abnormal Reed-Sternberg cells.

How common: About 8,920 new cases expected in the U.S. in 2026 (American Cancer Society); approximately 2.6 per 100,000 people

Immune Thrombocytopenia

Immune thrombocytopenia (ITP) is a rare autoimmune disorder in which the immune system destroys platelets, the blood cells that stop bleeding, and slows their production.

How common: Estimated 9 to 20 per 100,000 people; children often have a temporary form that resolves within months

Mantle Cell Lymphoma

Mantle cell lymphoma is a rare type of non-Hodgkin lymphoma that starts in B cells, a kind of white blood cell.

How common: About 1 in 200,000 people (Cleveland Clinic)

Merkel Cell Carcinoma

Merkel cell carcinoma is a rare, aggressive skin cancer that arises from Merkel cells, specialized touch receptors in the skin.

How common: About 3,000 new cases per year in the U.S.; incidence has tripled over the past 2 decades

Multiple Myeloma

Multiple myeloma is a cancer of plasma cells, the white blood cells that make antibodies.

How common: About 36,000 new US diagnoses expected in 2026 and roughly 203,000 people living with it, which puts myeloma right at the edge of the US rare disease threshold; it is 1.7% of all new cancers (NCI SEER)

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

Primary Central Nervous System Lymphoma

Primary central nervous system lymphoma (PCNSL) is a fast-growing lymphoma that starts in the brain, spinal cord, eyes or the membranes around them and usually stays there.

How common: About 5 new cases per million people a year in the US (Ono Pharmaceutical and Deciphera); more than half of patients are 60 or older

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

Von Willebrand Disease

Von Willebrand disease is the most common inherited bleeding disorder, caused by quantitative or qualitative deficiency of von Willebrand factor.

How common: 1-3% of the general population; 1 in 10,000 with clinically significant disease

Warm Autoimmune Hemolytic Anemia

Warm autoimmune hemolytic anemia (wAIHA) is a rare autoimmune disease in which the immune system makes antibodies, usually of the IgG type, that stick to red blood cells at normal body temperature...

How common: About 1 to 3 new cases per 100,000 people each year, according to the FDA and NORD

Where to get support

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

Questions about Incyte Corporation