PML-RAR-alpha Inhibitors
1 drugsAbout PML-RAR-alpha
PML-RAR-alpha is a fusion protein resulting from a chromosomal translocation, commonly t(15;17), driving acute promyelocytic leukemia (APL). This aberrant protein disrupts normal myeloid differentiation, leading to accumulation of immature promyelocytes. It acts as an aberrant transcriptional repressor, blocking the expression of genes for normal myeloid maturation.
PML-RAR-alpha is a crucial drug target due to its central role in APL pathogenesis. There is currently no genetic evidence data available linking this target to other diseases.
PML-RAR-alpha is targeted by one FDA-approved drug, TRISENOX (arsenic trioxide), a small molecule. TRISENOX leads to PML-RAR-alpha degradation and differentiation of leukemic cells; CEPHALON is the company marketing this drug.
Top PML-RAR-alpha Drugs
CEPHALON is the only company with an approved drug targeting PML-RAR-alpha.
Low competition suggests high barriers to entry or limited commercial interest, requiring careful market assessment.
PML-RAR-alpha Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets PML-RAR-alpha, using small molecule modality.
The lack of diverse modalities represents a whitespace opportunity for novel therapeutic approaches like antibodies or PROTACs.
PML-RAR-alpha Clinical Trials 37 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on PML-RAR-alpha. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 14 | 4 | 4 | 5 | 50% |
| Phase 2 | 17 | 5 | 4 | 8 | 56% |
| Phase 3 | 6 | 2 | 1 | 3 | 67% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
PML-RAR-alpha Drug Approval Timeline (2000 - 2000)
The first and most recent drug approval was TRISENOX in 2000.
The lack of recent approvals indicates potential saturation or challenges in developing new therapies for this target.
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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: 31 (4 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 PML-RAR-alpha. A trial counts if an approved drug acting on PML-RAR-alpha 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.