XPO1 Inhibitors
1 drugsAbout XPO1
XPO1 (exportin 1) mediates the transport of proteins and RNA out of the cell nucleus, a process essential for cellular function. By regulating the location of key proteins, XPO1 plays a critical role in maintaining cellular homeostasis.
XPO1 is a compelling oncology target because tumor suppressor proteins are often exported from the nucleus in cancer cells. Inhibiting XPO1 retains these proteins in the nucleus, potentially restoring their tumor-suppressing activity. There is currently no genetic evidence directly linking XPO1 to specific diseases.
XPO1 is targeted by one FDA-approved drug, XPOVIO, a small molecule developed by Karyopharm Therapeutics. XPOVIO was first approved in 2019 for oncology indications.
Top XPO1 Drugs
Karyopharm Therapeutics is the only company with an approved drug targeting XPO1.
High market concentration suggests high entry barriers or limited interest from other companies.
XPO1 Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets XPO1, using small molecule modality.
The exclusive use of small molecules indicates a whitespace opportunity for alternative modalities like antibodies or PROTACs.
XPO1 Clinical Trials 156 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on XPO1. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 66 | 29 | 15 | 22 | 66% |
| Phase 2 | 75 | 12 | 18 | 45 | 40% |
| Phase 3 | 15 | 3 | 1 | 11 | 75% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Phase 3 Readout Calendar Pro
3 Phase 3 trials testing approved XPO1 drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting XPO1. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
XPO1 Drug Approval Timeline (2019 - 2019)
XPOVIO was the first and only drug approved for XPO1 in 2019.
The single approval suggests potential for further drug development or market saturation.
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Competitive Landscape
- • 1 companies competing
- • Market share by company
Full Drug Portfolio
- • All 1 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 1-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 88 (9 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on XPO1. A trial counts if an approved drug acting on XPO1 is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.