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Imetelstat

Alternative Names: imetelstat, GRN163L, GRN-163L, RYTELO
Clinical Status: Active
Latest Update: 2025-12-08
Latest Update Note: News Article

Product Description

Imetelstat is a first-in-class telomerase inhibitor that could change the course of blood cancers—changing lives for the better. The development of imetelstat is grounded on Nobel Prize-winning scientific discoveries by early Geron collaborators that expanded the understanding of telomeres and telomerase and their role in the proliferation of cancer cells. Using this knowledge, Geron scientists pioneered a new approach in which oligonucleotides (short single strands of synthetic DNA or RNA) inhibit the activity of telomerase, an enzyme that helps maintain telomeres and enables the continued proliferation of malignant cells. (Sourced from: https://www.geron.com/research-and-development/imetelstat/)

Mechanisms of Action: telomerase Inhibitor

Novel Mechanism: No

Modality: Nucleic Acid

Route of Administration: Intravenous

FDA Designation: *

Approval Status: Approved

Approved Countries: None

Approved Indications: None

Known Adverse Events: None

Company: Geron
Company Location:
Company CEO:
Additional Commercial Interests: None

Clinical Description

Map of Global Clinical Trials for Imetelstat

Countries in Clinic: Argentina, Australia, Austria, Belgium, Brazil, Bulgaria, Colombia, Czech Republic, Denmark, France, Georgia, Germany, Hungary, India, Israel, Italy, Malaysia, Netherlands, Poland, Portugal, Russia, Singapore, South Korea, Spain, Taiwan, United Kingdom, United States, Unknown Location

Active Clinical Trial Count: 8

Recent & Upcoming Milestones

  • Clinical Outcomes Reported - Geron presented P3 Myelodysplastic Syndrome results on 2025-12-06 for Imetelstat
  • Clinical Outcomes Reported - Geron presented P1 Myelofibrosis results on 2025-05-30 for Imetelstat
  • Clinical Outcomes Reported - Geron presented P1 Myelofibrosis results on 2024-12-10 for Imetelstat

Highest Development Phases

Phase 3: Myelodysplastic Syndrome|Myelofibrosis|Myeloproliferative Disorders|Polycythemia|Thrombocytosis

Phase 2: Acute Myeloid Leukemia|Preleukemia

Phase 1: Acute Monocytic Leukemia|Acute Myelomonocytic Leukemia|Chronic Myeloid Leukemia|Chronic Myelomonocytic Leukemia|Juvenile Myelomonocytic Leukemia,

Trial ID

Trial

Phase

Trial Status

Disease

Primary Completion Date

Probability of Success

Latest Trial Update Date

Data Updated

NCT05371964

MYF1001

P1

Recruiting

Myelofibrosis

2027-02-01

12%

2025-05-20

Patient Enrollment|Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments

NCT06247787

PEPN2312

P1

Recruiting

Chronic Myeloid Leukemia|Chronic Myelomonocytic Leukemia|Acute Monocytic Leukemia|Acute Myeloid Leukemia|Juvenile Myelomonocytic Leukemia,|Preleukemia|Myelodysplastic Syndrome|Acute Myelomonocytic Leukemia

2026-06-30

50%

2025-02-07

Primary Endpoints|Treatments

NCT05583552

IMpress

P2

Active, not recruiting

Myelodysplastic Syndrome|Preleukemia|Acute Myeloid Leukemia

2025-09-30

12%

2025-03-18

Primary Completion Date|Primary Endpoints|Treatments

NCT04576156

MYF3001

P3

Active, not recruiting

Myelofibrosis|Polycythemia|Thrombocytosis|Myeloproliferative Disorders

2028-06-30

35%

2025-12-05

Patient Enrollment|Primary Endpoints|Treatments

2020-003288-24

2020-003288-24

P3

Active, not recruiting

Myelofibrosis

2026-06-10

35%

2025-05-06

Primary Completion Date|Study Completion Date|Treatments

2023-509120-17-00

GRN163LMYF3001

P3

Recruiting

Myelofibrosis

2027-12-31

35%

2025-05-02

Treatments

2024-511348-25-00

63935937MDS3001

P3

Active, not recruiting

Myelodysplastic Syndrome

2026-10-13

2025-05-02

2022-500721-32-01

IMpress_001

P2

Active, not recruiting

Myelodysplastic Syndrome

2026-06-30

2025-05-02

Treatments