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Incyte Corporation

Incyte Corporation works on 19 rare diseases tracked on Trial Friend, including Aplastic Anemia, Cholangiocarcinoma, Chronic Graft-versus-Host Disease and 16 more, with 41 active clinical trials (30 recruiting) and 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 NIKTIMVO (axatilimab), an anti-CSF1R antibody approved in 2024 for chronic graft-versus-host disease.

Type
Diversified Pharma
Ticker
INCY
Headquarters
Wilmington, United States
Founded
1991
Website
incyte.com
41
Active Rare Disease Trials
4
Approved Rare Disease Drugs
19
Rare Diseases in Portfolio
35
Years Active

Focus areas at Incyte Corporation

Within its broader pharmaceutical portfolio, Incyte Corporation has active clinical trial programs and drug development efforts across 19 rare diseases, including Aplastic Anemia, Cholangiocarcinoma, Chronic Graft-versus-Host Disease, Essential Thrombocythemia, Fibrodysplasia Ossificans Progressiva, and 14 additional rare conditions. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.

The clinical trials section below shows active and recruiting rare disease studies sponsored by Incyte Corporation, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.

Incyte Corporation is headquartered in Wilmington, United States, founded in 1991, publicly traded under the ticker symbol INCY. The company maintains a dedicated rare disease division alongside its broader therapeutic portfolio, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.

Incyte Corporation Drug Pipeline

Incyte Corporation has 41 active clinical trials across 4 development stages, with 30 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.

Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.

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8
Early Phase / Phase 18 trials
Recruiting
Acute Myeloid Leukemia+3 more
Recruiting
29
Phase 229 trials
3
Phase 33 trials
1
Other1 trial
Recruiting

Incyte Corporation Clinical Trials (41)

Active and recruiting clinical trials sponsored by Incyte Corporation, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.

Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.

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RECRUITINGPHASE2Recently 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)

Intervention: INCA034176, Best available Treatment (BAT)

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 Years41 locations
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RECRUITINGPHASE3Recently updatedNCT06585774

A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease

Intervention: INCA034176, Placebo, Corticosteroids

This study will be conducted to compare the efficacy of axatilimab versus placebo in combination with corticosteroids as initial treatment for moderate or severe chronic graft-versus-host disease (cGVHD).

Ages 12 Years+131 locations
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RECRUITINGPHASE3Recently updatedNCT07623200

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

Intervention: INCA033989, Best Available Treatment

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+178 locations
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RECRUITINGRecently updatedNCT05919511

Observational Study for Patients at Risk for Chronic Graft-Versus-Host Disease

Intervention: Non-Interventional

The purpose of this prospective observational study is to collect data from participants who have recently had an allogenic Stem Cell Transplant(alloSCT) and are at risk of Chronic Graft Versus Host Disease(cGVHD)

Ages 18 Years+37 locations
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RECRUITINGPHASE2Recently updatedNCT06728410

A Phase II Study of Pemigatinib Plus Durvalumab in Previously Treated Advanced Intrahepatic Cholangiocarcinoma Patients With FGFR-2 Fusion or Rearrangement

Intervention: Pemigatinib, Durvalumab

Intrahepatic CholangiocarcinomaFGFR2 Gene RearrangementFGFR2 Gene Mutation

This is a single arm phase II study of pemigatinib and durvalumab combination in patients with FGFR-2 fusion or rearrangement positive intrahepatic cholangiocarcinoma. Each cycle will be 3 weeks. Pemigatinib is administered at 13.5 mg orally daily 2 weeks on and 1 week off. Durvalumab is administered at 1500 mg intravenously once every 3 weeks. Subjects will require a visit with appropriate laboratory work prior to the start of each cycle. Disease assessment will occur every 9 weeks. Subjects will continue treatment until progression per RECIST 1.1, toxicity or subject/physician decision. A maximum of 24 months (about 35 cycles) of pemigatinib and durvalumab treatment from Cycle 1 Day 1 is allowed.

Ages 18 Years+3 locations
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RECRUITINGPHASE2Recently updatedNCT07011810

Axatilimab for Sclerotic Chronic Graft-versus-Host Disease

Intervention: Axatilimab, Biospecimen Collection, Questionnaire Administration, Skin Biopsy, Skin Measurement

This phase II trial tests how well axatilimab works in treating patients with thickening or hardening (sclerosis) of the skin related to chronic graft-versus-host disease after a donor stem cell transplant. Chronic graft-versus-host disease (cGVHD) remains a major complication of donor stem cell transplants. Sclerosis, while not associated with a higher risk of death, can lead to serious disabilities. Usual treatments for cGVHD can be associated with significant side effects and unsatisfactory outcomes. A monoclonal antibody, like axatilimab, is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Axatilimab blocks a receptor and depletes cells that may be involved in the development of inflammation and fibrosis in cGVHD. Giving axatilimab may improve or prevent worsening of sclerosis related to cGVHD in patients after a donor stem cell transplant.

Ages 18 Years+3 locations
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RECRUITINGPHASE2Recently updatedNCT06345495

High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly

Intervention: Ruxolitinib, Allogeneic Stem Cell Transplantation, Levetiracetam, Eltrombopag, Busulfan, Romiplostim, Fludarabine phosphate, Cyclophosphamide, Mesna, Tacrolimus

SplenomegalyMyelofibrosis

To learn if giving ruxolitinib and busulfan before a stem cell transplant can help to reduce spleen size and help the transplant to succeed.

Ages 18 Years - 75 Years1 location
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Incyte Corporation FDA-Approved Drugs (4)

Medications developed or marketed by Incyte Corporation that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.

Incyte Corporation Trial Locations

Incyte Corporation clinical trials are running at 703 sites in 28 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.

United States
290▼
Italy
61▼
Spain
54▼
Japan
50▼
Germany
47▼
United Kingdom
39▼
France
27▼
Canada
23▼
Australia
21▼
China
17▼
Belgium
15▼
Netherlands
12▼

Rare Disease Focus Areas (19)

Diseases targeted by Incyte Corporation's clinical trial and drug development programs

Aplastic AnemiaBlood & Immune

Aplastic anemia is a rare bone marrow failure syndrome characterized by pancytopenia resulting from absent or severely reduced hematopoiesis. Approximately half are immune-mediated, while others resul...

Prevalence: 1-2 cases per 1 million people per year
CholangiocarcinomaRare Cancers

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. It can occur inside the liver (...

Prevalence: About 8,000 new cases per year in the U.S.; rising incidence worldwide
Chronic Graft-versus-Host DiseaseBlood & Immune

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. It can affect the...

Prevalence: Affects 30 to 70% of patients who receive allogeneic hematopoietic stem cell transplant; approximately 14,000 new cases annually in the U.S.
Essential ThrombocythemiaBlood & Immune

Essential Thrombocythemia is a myeloproliferative neoplasm characterized by overproduction of platelets by bone marrow, resulting in very high platelet counts. Patients are at increased risk for blood...

Prevalence: 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 ProgressivaConnective Tissue & Musculoskeletal

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

Prevalence: Extremely rare, about 1 in 1 million to 1 in 2 million people worldwide
Gastrointestinal Stromal TumorRare Cancers

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

Prevalence: 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

Patient Resources

Organizations and resources related to Incyte Corporation's rare disease focus areas

Frequently Asked Questions About Incyte Corporation

Common questions about Incyte Corporation's rare disease programs, clinical trials, and treatments.