KIT KIT proto-oncogene, receptor tyrosine kinase
ENSG00000157404 · protein coding · Open Targets profile
Tyrosine-protein kinase that acts as a cell-surface receptor for the cytokine KITLG/SCF and plays an essential role in the regulation of cell survival and proliferation, hematopoiesis, stem cell maintenance, gametogenesis, mast cell development, migration and function, and in melanogenesis. In response to KITLG/SCF binding, KIT can activate several signaling pathways. Phosphorylates PIK3R1, PLCG1, SH2B2/APS and CBL. Activates the AKT1 signaling pathway by phosphorylation of PIK3R1, the regulatory subunit of phosphatidylinositol 3-kinase. Activated KIT also transmits signals via GRB2 and activation of RAS, RAF1 and the MAP kinases MAPK1/ERK2 and/or MAPK3/ERK1. Promotes activation of STAT family members STAT1, STAT3, STAT5A and STAT5B. Activation of PLCG1 leads to the production of the cellular signaling molecules diacylglycerol and inositol 1,4,5- trisphosphate. KIT signaling is modulated by protein phosphatases, and by rapid internalization and degradation of the receptor. Activated KIT promotes phosphorylation of the protein phosphatases PTPN6/SHP-1 and PTPRU, and of the transcription factors STAT1, STAT3, STAT5A and STAT5B. Promotes phosphorylation of PIK3R1, CBL, CRK (isoform Crk-II), LYN, MAPK1/ERK2 and/or MAPK3/ERK1, PLCG1, SRC and SHC1. {ECO:0000269|PubMed:10397721, ECO:0000269|PubMed:12444928, ECO:0000269|PubMed:12511554, ECO:0000269|PubMed:12878163, ECO:0000269|PubMed:17904548, ECO:0000269|PubMed:19265199, ECO:0000269|PubMed:21135090, ECO:0000269|PubMed:21640708, ECO:0000269|PubMed:7520444, ECO:0000269|PubMed:9528781}.
Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.
Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.
Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.
Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.
Tractability
- Small molecule
- Approved Drug, Structure with Ligand, High-Quality Ligand, High-Quality Pocket, Druggable Family
- Antibody
- Phase 1 Clinical, GO CC high conf, UniProt loc med conf, UniProt SigP or TMHMM, Human Protein Atlas loc
- PROTAC
- Literature, UniProt Ubiquitination, Database Ubiquitination, Half-life Data, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.1513800024986267 (upper bound 0.22991999983787537). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.
Who runs trials of these drugs
Principal investigators and study chairs on 400 registry trials, updated in the last three years, of the most advanced drugs against KIT (pexidartinib, sorafenib, ripretinib, tivozanib, midostaurin, pazopanib, masitinib, quizartinib, cediranib, avapritinib, sunitinib, imatinib), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Eric JonaschM.D. Anderson Cancer Center · 3 of 3 trials Phase 2+
- Joyce F LiuNRG Oncology · 3 of 3 trials Phase 2+Ph3NCT02446600 · Testing the Use of A Single Drug (Olaparib) or the Combination of Two Drugs (Cediranib and Olaparib) Compared to the Usual Chemotherapy for Women With Platinum Sensitive Ovarian, Fallopian Tube, or Primary Peritoneal Cancerbrief
- Musa YilmazM.D. Anderson Cancer Center · 3 of 3 trials Phase 2+Ph1/Ph2NCT04128748 · Liposomal Cytarabine and Daunorubicin (CPX-351) and Quizartinib for the Treatment of Acute Myeloid Leukemia and High Risk Myelodysplastic Syndromebrief
- Alice P ChenNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Amado ZuritaM.D. Anderson Cancer Center · 2 of 2 trials Phase 2+
- Arun RajanNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Eric ShermanMemorial Sloan Kettering Cancer Center · 2 of 2 trials Phase 2+
- Geneviève CheneUniversity Hospital, Bordeaux, France · 2 of 2 trials Phase 2+
- Ghassan Abou-AlfaMemorial Sloan Kettering Cancer Center · 2 of 2 trials Phase 2+
- Jean-Yves BLAYCentre Leon Berard · 2 of 2 trials Phase 2+
- Jessica GartrellSt. Jude Children's Research Hospital · 2 of 2 trials Phase 2+
- Joseph W KimYale University Cancer Center LAO · 2 of 2 trials Phase 2+
- Juan BerguaHospital San Pedro Alcántara · 2 of 2 trials Phase 2+
- Jung-min LeeNRG Oncology · 2 of 2 trials Phase 2+
- Laura EssermanUniversity of California, San Francisco · 2 of 2 trials Phase 2+
Registry trials of pexidartinib, sorafenib, ripretinib, tivozanib, midostaurin, pazopanib, masitinib, quizartinib, cediranib, avapritinib, sunitinib, imatinib updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 280 of 389 have all sites in one country.
Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. 11 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
473 trials of drugs against KIT were stopped early; 20 of the 200 most recent give no reason
Terminated, withdrawn and suspended interventional trials of pexidartinib, sorafenib, ripretinib, tivozanib, midostaurin, pazopanib, masitinib, quizartinib, cediranib, avapritinib, sunitinib, imatinib, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 25 most recent of 473.
- withdrawn · 2026-10-01 · City of Hope Medical Center · Tivozanib“Study Abandoned withdrawn”
- terminated · 2026-08-25 · Association of Urologic Oncology (AUO) · SunitinibNo reason given
- terminated · 2026-05-29 · Sarit Assouline · Imatinib“Insufficient patient accrual”
- terminated · 2026-05-20 · Massachusetts General Hospital · Tivozanib“Slow accrual”
- suspended · 2026-05-06 · AB Science · Masitinib (4.5)“The study has been suspended but may start again in the future.”
- terminated · 2026-04-14 · Institut Claudius Regaud · Pazopanib“Slow recruitment rate”
- terminated · 2026-03-25 · University of Maryland, Baltimore · Imatinib“The Principal Investigator left the institution. The study was halted prematurely.”
- terminated · 2026-03-13 · Centre Oscar Lambret · Imatinib“Study stopped at the end of the first step”
- terminated · 2026-01-29 · Indiana University · Imatinib Mesylate“low accrual”
- suspended · 2025-10-23 · Mirror Biologics, Inc. · Sorafenib“Sponsor unable to fund”
- terminated · 2025-08-22 · OHSU Knight Cancer Institute · imatinib mesylateNo reason given
- withdrawn · 2025-08-06 · M.D. Anderson Cancer Center · Sorafenib“0 participants”
- terminated · 2025-06-27 · AHS Cancer Control Alberta · Imatinib Oral Tablet“Lack of Accrual”
- terminated · 2025-06-03 · M.D. Anderson Cancer Center · Imatinib Mesylate“Per PI Request. 2) No additional benefit was noted with the addition of Ruxolitinib.”
- terminated · 2025-05-01 · Institute of Hematology & Blood Diseases Hospital, China · Imatinib“terminated”
- terminated · 2025-04-30 · University of Washington · Pazopanib hydrochloride“Study was terminated at completion of the Phase 1 dose escalation due to loss of financial support.”
- withdrawn · 2025-04-27 · Medical University of South Carolina · matched by the registry's drug synonyms“study is being rewritten for different disease population”
- terminated · 2025-03-21 · Gulam Manji · matched by the registry's drug synonyms“Poor enrollment”
- terminated · 2025-03-07 · Aptose Biosciences Inc. · matched by the registry's drug synonyms“Change in corporate strategy”
- terminated · 2025-02-26 · Cancer Institute and Hospital, Chinese Academy of Medical Sciences · Sorafenib“IDMC recommended to terminate the study since Interim analysis showed that the experimental group had significant superiority over the control group in TTP and OS (p<0.001), meeting the predefined interim analysis endpoints in the study protocol.”
- terminated · 2025-02-25 · Niguarda Hospital · Midostaurin“didn't reach the primary endpoint”
- terminated · 2025-02-14 · AIO-Studien-gGmbH · matched by the registry's drug synonyms“insufficient accrual rate”
- terminated · 2025-02-12 · Georgetown University · matched by the registry's drug synonyms“Lack of efficacy”
- terminated · 2025-02-12 · Georgetown University · sunitinib and capecitabine“Unanticipated side effects and futility”
- terminated · 2025-01-17 · Gruppo Italiano Malattie EMatologiche dell'Adulto · matched by the registry's drug synonyms“low enrollment rate”
Paediatric incentives and obligations for KIT's drugs
For pexidartinib, sorafenib, ripretinib, tivozanib, midostaurin, pazopanib, masitinib, quizartinib, cediranib, avapritinib, sunitinib, imatinib: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.
- FDA pediatric target list: yes · KIT is on the list. Membership alone neither requires nor waives a pediatric study.Rule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
- EU paediatric investigation plan: yes · EMEA-002358-PIP02-18-M04 (avapritinib, Treatment of all conditions included in the category of malignant neoplasms (except haematopoietic and lymphoid tissue neoplasms)): PM: decision on the application for modification of an agreed PIP, 25 Oct 2024; EMEA-000780-PIP01-09-M08 (Midostaurin, Treatment of acute myeloid leukaemia;Treatment of malignant mastocytosis;Treatment of mast cell leukaemia): PM: decision on the application for modification of an agreed PIP, 16 Aug 2024; EMEA-001821-PIP01-15-M08 (quizartinib, Treatment of acute myeloid leukaemia): PM: decision on the application for modification of an agreed PIP, 14 Jun 2024; EMEA-002643-PIP01-19-M01 (imatinib, Treatment of acute lymphoblastic leukaemia;Treatment of chronic myeloid leukaemia): PM: decision on the application for modification of an agreed PIP, 29 Oct 2021; +11 moreRule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
- EU orphan designation: yes · EU/3/16/1722 positive 29 Aug 2016: Treatment of amyotrophic lateral sclerosis; EU/3/13/1200 expired 13 Nov 2013: Treatment of papillary thyroid cancer; EU/3/13/1199 expired 13 Nov 2013: Treatment of follicular thyroid cancer; EU/3/21/2449 positive 21 Jun 2021: Treatment of pulmonary arterial hypertension; +21 moreRule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
- Authorised in the EU: yes · Glivec: authorised since 7 Nov 2001; Ayvakyt: authorised since 24 Sept 2020, orphan medicine, conditional; Sunitinib Accord: authorised since 11 Feb 2021; Qinlock: authorised since 18 Nov 2021, orphan medicine; +22 moreRule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
- FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the linkRule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
- FDA pediatric oncology approval: yes · Imatinib Mesylate [Gleevec] &, 25 Jan 2013: Pediatric patients with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy; Imatinib Mesylate [Gleevec] &, 27 Sept 2006: Newly diagnosed adult and pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phaseRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-11
- Registered studies open to children: yes · 250 interventional studies of pexidartinib, sorafenib, ripretinib, tivozanib, midostaurin, pazopanib, masitinib, quizartinib, cediranib, avapritinib, sunitinib, imatinib include patients under 18; 47 are recruiting, not yet recruiting or activeRule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-11
Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs
All 38 drugs and clinical candidates as a table
Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.