XPO1 exportin 1
ENSG00000082898 · protein coding · Open Targets profile
Mediates the nuclear export of cellular proteins (cargos) bearing a leucine-rich nuclear export signal (NES) and of RNAs. In the nucleus, in association with RANBP3, binds cooperatively to the NES on its target protein and to the GTPase RAN in its active GTP-bound form (Ran-GTP). Docking of this complex to the nuclear pore complex (NPC) is mediated through binding to nucleoporins. Upon transit of a nuclear export complex into the cytoplasm, disassembling of the complex and hydrolysis of Ran-GTP to Ran-GDP (induced by RANBP1 and RANGAP1, respectively) cause release of the cargo from the export receptor. The directionality of nuclear export is thought to be conferred by an asymmetric distribution of the GTP- and GDP-bound forms of Ran between the cytoplasm and nucleus. Involved in U3 snoRNA transport from Cajal bodies to nucleoli. Binds to late precursor U3 snoRNA bearing a TMG cap. {ECO:0000269|PubMed:15574332, ECO:0000269|PubMed:20921223, ECO:0000269|PubMed:9311922, ECO:0000269|PubMed:9323133}.
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, Med-Quality Pocket
- PROTAC
- Database Ubiquitination, Half-life Data, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.03751799836754799 (upper bound 0.0788860023021698). 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 121 registry trials, updated in the last three years, of the most advanced drugs against XPO1 (selinexor), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Jin LuPeking University People's Hospital · 2 of 2 trials Phase 2+
- Liang WangBeijing Tongren Hospital · 2 of 2 trials Phase 2+
- Javier Martín BrotoHospital Universitario Fundación Jiménez Díaz · 1 of 2 trials Phase 2+
- A P ChenNational Cancer Institute LAO · 1 of 1 trials Phase 2+
- Abdulraheem YacoubUniversity of Kansas School of Medicine · 1 of 1 trials Phase 2+
- Adam L GreenChildren's Oncology Group · 1 of 1 trials Phase 2+
- Andrew B LassmanColumbia University · 1 of 1 trials Phase 2+
- Bingshan LiuMedical Monitor · 1 of 1 trials Phase 2+
- Bingzong LiSecond Affiliated Hospital of Soochow University · 1 of 1 trials Phase 2+
- Daniel SherbenouUniversity of Colorado, Denver · 1 of 1 trials Phase 2+
- Depei WuThe First Affiliated Hospital of Soochow University · 1 of 1 trials Phase 2+
- Dickran KazandjianUniversity of Miami · 1 of 1 trials Phase 2+
- Dipenkumar ModiBarbara Ann Karmanos Cancer Institute · 1 of 1 trials Phase 2+
- Francis AyukUniversity Medical Center Hamburg-Eppendorf, Department of Stem Cell Transplantation · 1 of 1 trials Phase 2+
- Hanne NorsethOslo University Hospital · 1 of 1 trials Phase 2+
Registry trials of selinexor 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; 95 of 111 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. 10 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
37 trials of drugs against XPO1 were stopped early; 3 give no reason
Terminated, withdrawn and suspended interventional trials of selinexor, 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 37.
- terminated · 2026-10-26 · University of Utah · Selinexor“PI and Sponsor decision. Accrual was low and there was sufficient data to detect a response.”
- terminated · 2026-01-29 · Mamta Parikh · Selinexor“Poor accrual.”
- terminated · 2026-01-05 · Antengene Corporation · matched by the registry's drug synonyms“Based on the adjustment of clinical research and development strategy,sponsor decided to terminate the study”
- terminated · 2026-01-05 · Universitätsklinikum Hamburg-Eppendorf · selinexor/bortezomib/dexamethasone (SVD)“The reason for the end of recruitment is that the recruitment target could not be achieved despite all efforts and the G-BA (financing party) does not consider a continuation of the AlloRelapseMM study to be expedient.”
- withdrawn · 2025-10-03 · Karyopharm Therapeutics Inc · Selinexor“Sponsor decision”
- terminated · 2025-06-09 · Antengene Corporation · matched by the registry's drug synonyms“The adjustment of the sponsor's research and development strategy”
- terminated · 2025-06-04 · M.D. Anderson Cancer Center · Selinexor“Administratively Complete”
- terminated · 2025-05-30 · Institute of Hematology & Blood Diseases Hospital, China · Selinexor“The number of participants in the study did not meet expectations”
- terminated · 2024-12-16 · Karyopharm Therapeutics Inc · Selinexor“Upon further consideration of the existing data and the competitive landscape, Karyopharm Therapeutics Inc has decided not to pursue the ongoing development of Selinexor in GBM at this time.”
- terminated · 2024-08-23 · Karyopharm Therapeutics Inc · Selinexor“The trial was terminated due to a lack of sufficient anti-melanoma tumor signal for the combination of selinexor + pembrolizumab.”
- withdrawn · 2024-07-10 · Washington University School of Medicine · Selinexor“Study sponsor decided to close study.”
- terminated · 2024-06-13 · University of Texas Southwestern Medical Center · Selinexor“Funding Sponsor no longer supporting study”
- withdrawn · 2024-03-27 · St. Jude Children's Research Hospital · Selinexor“Drug withdrawn by company.”
- terminated · 2023-11-03 · Karyopharm Therapeutics Inc · Selinexor“Sponsor decision”
- terminated · 2023-10-25 · Weill Medical College of Cornell University · Selinexor“low enrollment”
- terminated · 2023-02-16 · Georgetown University · Open Label Selinexor“Slow Accrual”
- terminated · 2023-01-26 · Karyopharm Therapeutics Inc · selinexor“Lack of efficacy at time of interim analysis”
- terminated · 2023-01-26 · Karyopharm Therapeutics Inc · Selinexor“Study was terminated due to Sponsor decision (all except 1 patient were off-treatment and 2 patients were in survival follow-up)”
- terminated · 2023-01-26 · Karyopharm Therapeutics Inc · Selinexor“Due to enrollment challenges. The termination is not a consequence of any safety concern.”
- withdrawn · 2023-01-26 · Karyopharm Therapeutics Inc · Selinexor“Trial never opened.”
- terminated · 2023-01-26 · Karyopharm Therapeutics Inc · Selinexor“Due to enrollment challenges”
- withdrawn · 2023-01-26 · Karyopharm Therapeutics Inc · Selinexor, Placebo (for selinexor)No reason given
- withdrawn · 2023-01-26 · Karyopharm Therapeutics Inc · Selinexor“Terminated this trial and added a Lenalidomide arm to KCP-330-017”
- terminated · 2023-01-26 · Karyopharm Therapeutics Inc · Selinexor (KPT-330)“Due to enrollment challenges and availability of other options for lung cancer patients. The termination is not a consequence of any safety concern.”
- withdrawn · 2023-01-20 · Karyopharm Therapeutics Inc · Selinexor“Due to enrollment challenges \& improved SOC during run-in phase; no patients received KPT-330. Withdrawal is not a consequence of any safety concern.”
Paediatric incentives and obligations for XPO1's drugs
For selinexor: 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 · XPO1 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-002387-PIP02-21 (selinexor, Treatment of endometrial carcinoma): W: decision granting a waiver in all age groups for all conditions or indications, 8 Sept 2021; EMEA-002387-PIP01-18 (selinexor, Treatment of Multiple Myeloma): W: decision granting a waiver in all age groups for all conditions or indications, 6 Dec 2018Rule: 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/3/14/1354 positive 19 Nov 2014: Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma; EU/3/22/2705 positive 11 Oct 2022: Treatment of myelofibrosis; EU/3/21/2509 positive 15 Oct 2021: Treatment of glioma; EU/3/14/1355 positive 19 Nov 2014: Treatment of plasma cell myelomaRule: 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 · Nexpovio: authorised since 26 Mar 2021Rule: 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 Nexpovio, 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 selinexorRule: 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 · 27 interventional studies of selinexor include patients under 18; 18 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 1 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.
| Drug | Type | Highest stage | Indications | Source records |
|---|---|---|---|---|
| SELINEXOR | Small molecule | approval | plasma cell myeloma, diffuse large B-cell lymphoma, neoplasm, liposarcoma, neuroendocrine carcinoma, endometrial cancer, myelofibrosis, endometrial carcinoma, +83 more | Open Targets · ChEMBL |