AXL AXL receptor tyrosine kinase
ENSG00000167601 · protein coding · Open Targets profile
Receptor tyrosine kinase that transduces signals from the extracellular matrix into the cytoplasm by binding growth factor GAS6 and which is thus regulating many physiological processes including cell survival, cell proliferation, migration and differentiation. Ligand binding at the cell surface induces dimerization and autophosphorylation of AXL. Following activation by ligand, AXL binds and induces tyrosine phosphorylation of PI3-kinase subunits PIK3R1, PIK3R2 and PIK3R3; but also GRB2, PLCG1, LCK and PTPN11. Other downstream substrate candidates for AXL are CBL, NCK2, SOCS1 and TNS2. Recruitment of GRB2 and phosphatidylinositol 3 kinase regulatory subunits by AXL leads to the downstream activation of the AKT kinase. GAS6/AXL signaling plays a role in various processes such as endothelial cell survival during acidification by preventing apoptosis, optimal cytokine signaling during human natural killer cell development, hepatic regeneration, gonadotropin-releasing hormone neuron survival and migration, platelet activation, or regulation of thrombotic responses. Also plays an important role in inhibition of Toll-like receptors (TLRs)-mediated innate immune response. {ECO:0000269|PubMed:10403904, ECO:0000269|PubMed:11484958, ECO:0000269|PubMed:12364394, ECO:0000269|PubMed:12490074, ECO:0000269|PubMed:15507525, ECO:0000269|PubMed:15733062, ECO:0000269|PubMed:1656220, ECO:0000269|PubMed:18840707}.
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 Withdrawn means the drug was pulled from the market or from development.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, Druggable Family
- Antibody
- UniProt loc high conf, GO CC high conf, UniProt SigP or TMHMM, Human Protein Atlas loc
- PROTAC
- UniProt Ubiquitination, Database Ubiquitination, Small Molecule Binder
- Other modalities
- Advanced Clinical
gnomAD loss of function constraint: o/e 0.49764999747276306 (upper bound 0.6269999742507935). 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 79 registry trials, updated in the last three years, of the most advanced drugs against AXL (gilteritinib, bemcentinib, ningetinib, mecbotamab vedotin), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Li ZhangSun Yat-sen University · 2 of 2 trials Phase 2+
- Nicholas ShortMD Anderson Center · 2 of 2 trials Phase 2+
- Tapan M KadiaM.D. Anderson Cancer Center · 1 of 2 trials Phase 2+
- Alessandro RambaldiASST Papa Giovanni XXIII · 1 of 1 trials Phase 2+
- Alice MimsBeat AML · 1 of 1 trials Phase 2+
- Christoph RummeltUniversity Hospital Freiburg · 1 of 1 trials Phase 2+
- Courtney DiNardoM.D. Anderson Cancer Center · 1 of 1 trials Phase 2+
- Dean FennellUniversity of Leicester · 1 of 1 trials Phase 2+
- Fabio GuoloIRCCS Policlinico San Martino, UO Clinica Ematologica - Genova · 1 of 1 trials Phase 2+
- Farhad Ravandi-KashaniM.D. Anderson Cancer Center · 1 of 1 trials Phase 2+
- Giovanni MartinelliUniversity of Bologna · 1 of 1 trials Phase 2+
- Guopan YuNanfang Hospital, Southern Medical University · 1 of 1 trials Phase 2+
- Hani GabraBerGenBio ASA · 1 of 1 trials Phase 2+
- Hiroto InabaSt. Jude Children's Research Hospital · 1 of 1 trials Phase 2+
- Hui WeiBlood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College · 1 of 1 trials Phase 2+
Registry trials of gilteritinib, bemcentinib, ningetinib, mecbotamab vedotin 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; 49 of 74 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. 5 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
19 trials of drugs against AXL were stopped early
Terminated, withdrawn and suspended interventional trials of gilteritinib, bemcentinib, ningetinib, mecbotamab vedotin, enapotamab vedotin, BPI-9016, 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 19 most recent of 19.
- terminated · 2026-05-26 · The University of Texas Health Science Center at San Antonio · bemcentinib“Investigational drug limitations”
- withdrawn · 2026-05-14 · Sunshine Lake Pharma Co., Ltd. · matched by the registry's drug synonyms“Withdrawn proactively due to the company's development strategy adjustment and changes in the drug R\&D direction.”
- terminated · 2026-03-18 · Nerviano Medical Sciences · matched by the registry's drug synonyms“Study stopped due to strategic reasons. The decision is not based on specific safety findings as the safety observed in the phase II part of the study is in line with what was reported in the phase I part.”
- terminated · 2026-01-05 · Astellas Pharma Global Development, Inc. · gilteritinib“The study was terminated due to enrollment challenges.”
- terminated · 2025-10-29 · BerGenBio ASA · Bemcentinib“Lack of efficacy”
- terminated · 2025-03-07 · Aptose Biosciences Inc. · matched by the registry's drug synonyms“Change in corporate strategy”
- terminated · 2024-11-27 · Astellas Pharma Global Development, Inc. · Gilteritinib“Study terminated due to adverse events related to the combination therapy”
- terminated · 2024-08-07 · Kronos Bio · Gilteritinib“Sponsor decision”
- terminated · 2024-05-31 · Celgene · Gilteritinib“Business objectives have changed.”
- terminated · 2024-02-08 · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · matched by the registry's drug synonyms“NCI decided to terminate ABTC Consortium due to NCI moving in different direction for Brain Cancer - official close date is 10/31/23”
- terminated · 2023-11-09 · University of Texas Southwestern Medical Center · Bemcentinib“Funding was terminated early because research interests were going in a different direction.”
- withdrawn · 2023-04-25 · Ayman H Qasrawi · Gilteritinib 40 MG Oral Tablet“Sponsor withdrew funding”
- terminated · 2023-03-02 · Jiangsu Alphamab Biopharmaceuticals Co., Ltd · Ningetinib“Cohort A,B,C end enrollments. Cohort D and E were considered to have no significant clinical benefit at the SMC meeting and decided to terminate enrollment.”
- terminated · 2022-10-28 · Jiangsu Alphamab Biopharmaceuticals Co., Ltd · Ningetinib Tosylate（multi-target TKI）“The sponsor's development strategy adjustment resolution is terminated”
- withdrawn · 2022-06-15 · Erasca, Inc. · Gilteritinib“Sponsor decision.”
- withdrawn · 2022-06-09 · City of Hope Medical Center · Gilteritinib Fumarate“Drug availability”
- terminated · 2021-11-09 · BerGenBio ASA · Bemcentinib; pembrolizumab“Based on planned futility assessment”
- terminated · 2021-03-24 · Sunshine Lake Pharma Co., Ltd. · matched by the registry's drug synonyms“Company strategy adjustment”
- terminated · 2021-03-24 · Sunshine Lake Pharma Co., Ltd. · matched by the registry's drug synonyms“The effect of CT053PTSA in preciously treated patients with advanced and metastatic RCC is not as good as pre-expected”
Paediatric incentives and obligations for AXL's drugs
For gilteritinib, bemcentinib, ningetinib, mecbotamab vedotin, enapotamab vedotin, BPI-9016: 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 · AXL 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 · EMA/PE/0000221199 (bemcentinib, Treatment of non-small cell lung cancer): W: decision granting a waiver in all age groups for all conditions or indications, 6 Dec 2024; EMEA-002064-PIP01-16-M06 (Gilteritinib (as fumarate), Treatment of acute myeloid leukaemia): PM: decision on the application for modification of an agreed PIP, 16 Aug 2024Rule: 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/3/17/1961 positive 17 Jan 2018: Treatment of acute myeloid leukaemiaRule: 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 EU/3/17/1961, 2026-11-11
- Authorised in the EU: yes · Xospata: authorised since 24 Oct 2019, orphan medicineRule: 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 EPAR Xospata, 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: no · FDA's table lists no pediatric approval of gilteritinib, bemcentinib, ningetinib, mecbotamab vedotin, enapotamab vedotin, bpi-9016Rule: 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 · 11 interventional studies of gilteritinib, bemcentinib, ningetinib, mecbotamab vedotin, enapotamab vedotin, bpi-9016 include patients under 18; 9 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 7 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.