Incyte Corporation

Incyte Corporation works on 17 rare diseases tracked on Trial Friend, including Aplastic Anemia, Cholangiocarcinoma, Chronic Graft-versus-Host Disease and 14 more, with 39 recruiting clinical trials 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
39
Active Rare Disease Trials
4
Approved Rare Disease Drugs
17
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 17 rare diseases, including Aplastic Anemia, Cholangiocarcinoma, Chronic Graft-versus-Host Disease, Essential Thrombocythemia, Fibrodysplasia Ossificans Progressiva, and 12 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 all active and recruiting 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 39 active clinical trials across 4 development stages, with 39 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 includes all of Incyte Corporation's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.

Understand Incyte Corporation's pipeline
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8
Early Phase / Phase 18 trials
Recruiting
Myeloproliferative Neoplasms
Recruiting
Acute Myeloid Leukemia+3 more
Recruiting
Graft Vs Host Disease+2 more
Recruiting
Myeloproliferative Neoplasms
Recruiting
26
Phase 226 trials
Locally Advanced Ampulla of Vater Carcinoma+4 more
Recruiting
Leukemia+2 more
Recruiting
4
Phase 34 trials
1
Other1 trial
Recruiting

Incyte Corporation Clinical Trials (39)

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.

Ask about Incyte Corporation's trials
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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 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+128 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+175 locations
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RECRUITINGPHASE2Recently updatedNCT07780838

Alternating Chemo-immunotherapy and FGFR Inhibitor for FGFR-positive Biliary or Cholangiocarcinoma

Intervention: Biospecimen Collection, Cisplatin, Computed Tomography, Durvalumab, Gemcitabine, Magnetic Resonance Imaging, Pemigatinib, Survey Administration

Locally Advanced Ampulla of Vater CarcinomaLocally Advanced Biliary Tract CarcinomaLocally Advanced Extrahepatic CholangiocarcinomaLocally Advanced Gallbladder CarcinomaLocally Advanced Intrahepatic Cholangiocarcinoma

This phase II trial tests how well giving gemcitabine, cisplatin and durvalumab alternating with pemigatinib for the treatment of biliary tract cancer with FGFR2 alterations that cannot be removed by surgery (unresectable), that has spread to nearby tissue or lymph nodes (locally advanced) or that has spread from where it first started (primary site) to other places in the body (metastatic). Gemcitabine is a chemotherapy drug that blocks the cells from making DNA and may kill cancer cells. Cisplatin is in a class of medications known as platinum-containing compounds. It works by killing, stopping or slowing the growth of cancer cells. Immunotherapy with monoclonal antibodies, such as durvalumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Pemigatinib is in a class of medications called kinase inhibitors. It works by blocking the action of an abnormal FGFR protein that signals cancer cells to multiply. This may help keep cancer cells from growing and may kill them. Giving gemcitabine, cisplatin and durvalumab alternating with pemigatinib may work well for the treatment of unresectable, locally advanced or metastatic biliary tract cancer with FGFR2 alterations.

Ages 18 Years+1 location
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RECRUITINGPHASE1Recently updatedNCT07225439

Rituximab (Rtx) + Tafasitamab in Combination With Allogeneic NK Cells for Treatment of Relapsed/Refractory (r/r) B-cell Non-Hodgkin Lymphoma (NHL)

Intervention: Allogeneic NK cells, Rituximab, Tafasitamab, Interleukin-2, Fludarabine/cyclophosphamide

Non Hodgkin LymphomaB-cell Non Hodgkin LymphomaDiffuse Large B Cell LymphomaHigh-grade B-cell LymphomaPrimary Mediastinal Large B Cell Lymphoma

This research study is for people who have relapsed or refractory B-cell non-Hodgkin's lymphoma (NHL) that has not responded to two or more lines of therapy. The purpose of this study is to identify the recommended dose of allogeneic NK cells in combination with IL-2, Tafasitamab and Rituximab for the treatment of relapsed or refractory B-cell non-Hodgkin's lymphoma. NK cells are an investigational (experimental) treatment which means they are not approved by the Food and Drug Administration (FDA). NK cells are a type of lymphocyte that's part of the body's natural immune system, and they can kill cancer cells by creating pores in the cancer cell membranes and inducing apoptosis (programmed cell death). Participants in this study will receive lymphodepleting chemotherapy, as well as Allogeneic NK cells, Tafasitamab and Interleukin-2 (IL-2) by an intravenous (IV) infusion. Participants are expected to complete one cycle, and they may be eligible to complete a second cycle of the same regiment if they have stable disease, partial or complete remission at the end of the first cycle. Participants will be in this study for about 12 months.

Ages 18 Years+2 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+2 locations
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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.

Drug NameBrand NameRare DiseasesApproval Date
RUXOLITINIB
Kinase Inhibitor [EPC]
JAKAFI
oral
Nov 16, 2011
TAFASITAMAB-CXIXMONJUVI
intravenous
—Jul 31, 2020
RUXOLITINIBOPZELURA
topical
Sep 21, 2021
PEMIGATINIB
Kinase Inhibitor [EPC]
PEMAZYRE
oral
Apr 17, 2020

Incyte Corporation Trial Locations

Incyte Corporation clinical trials are running at 819 sites in 25 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
92▼
Spain
74▼
Germany
64▼
Japan
61▼
United Kingdom
46▼
France
39▼
Canada
26▼
Belgium
21▼
Australia
16▼
Austria
15▼
Netherlands
13▼

Rare Disease Focus Areas (17)

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: Approximately 1-2 cases per 100,000 people; median age of diagnosis is 60 years
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, approximately 1 per 2 million people globally; extremely small patient population
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 4,000-6,000 new cases per year in the U.S.; 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.