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About RNA methyltransferases

RNA methyltransferases catalyze the addition of methyl groups to RNA, regulating splicing, stability, and translation. These enzymes are emerging as drug targets due to their role in critical cellular processes.

1
Approved Drugs
1
Companies
1
Indications
1
Therapeutic Areas
Broadest Approval
ONUREG
Bristol-Myers Squibb
1
approved indications

Top RNA methyltransferases Drugs

ONUREG
Bristol-Myers Squibb
1 indications · 2020
🏢

Bristol-Myers Squibb is the only company with an approved drug targeting RNA methyltransferases.

RNA methyltransferases Drug Modality Landscape

Modalities

Small molecule
1
100%

Routes of Administration

💊 Oral
1
100%
💡

Only one approved drug targets RNA methyltransferases, using small molecule modality.

Explore alternative modalities like oligonucleotides or PROTACs to differentiate from existing approaches and expand therapeutic potential.

Oral option available Small molecules only

RNA methyltransferases Clinical Trials 549 trials

Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on RNA methyltransferases. Source: ClinicalTrials.gov. How we count

549
Total Trials
246
Active
170
Completed
57%
Completion Rate

Completion by Phase

Phase Total Completed Stopped Active Completion
Phase 1 254 81 75 93 52%
Phase 2 229 70 38 120 65%
Phase 3 59 17 13 29 57%
Phase 4 7 2 1 4 67%

Top Sponsors

M.D. Anderson Cancer Center 49 57%
National Cancer Institute (N... 30 67%
Celgene 21 90%
The First Affiliated Hospita... 18 100%
Institute of Hematology & Bl... 15 0%
AbbVie 13 57%
Novartis Pharmaceuticals 11 20%
Groupe Francophone des Myelo... 11 78%

By Modality

Small molecule
549 57%
Source: ClinicalTrials.gov · Completion rate = completed ÷ (completed + terminated + withdrawn)

Phase 3 Readout Calendar Pro

2 Phase 3 trials testing approved RNA methyltransferases drugs across all sponsors.

Full calendar →
Q4 2027
Azacitidine
Taiho Oncology, Inc. · Myelodysplastic Syndromes
Estimated · aging NCT04256317
Q4 2027
BL-M11D1
Sichuan Baili Pharmaceutical Co., Ltd. · Acute Myeloid Leukemia
Estimated · aging NCT07255872

Coverage: trials whose intervention is an approved drug targeting RNA methyltransferases. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.

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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: 236 (84 companies)
  • • Completion rates by condition
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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 RNA methyltransferases. A trial counts if an approved drug acting on RNA methyltransferases 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.