Rytelo

(Imetelstat)

ApprovedRNA therapy

Rytelo is a rna therapy that works as a Telomerase Inhibitor.[2] It is being used across 3 indications, with its most advanced program in Approved.[1]

Rytelo is one of 82 aso programs targeting Myelodysplastic Syndromes. It is currently in Approved.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Myelodysplastic SyndromesActive, not recruiting

MyelofibrosisActive, not recruiting

Recurrent Childhood Acute Myeloid LeukemiaRecruiting

Myelodysplastic SyndromesApproved

Active, not recruiting

MyelofibrosisPhase 3

Active, not recruiting

Recurrent Childhood Acute Myeloid LeukemiaPhase 1

Recruiting

Competition

Competitive Landscape

51 competitors in Myelodysplastic Syndromes

#DrugCompanyPhaseTrials

Dacogen

Sun Pharmaceutical Industries, Inc.

Approved

18 2

Vidaza

KKaruna Therapeutics, Inc., a Bristol Myers Squibb company

Approved

36 3

Cytosar

Teva

Approved

13 4

Idamycin

Pfizer

Approved

6 5

Reblozyl

Bristol-Myers Squibb

Phase 3

15 6

Rytelo

GGeron Corporation

Approved

11 7

Evrenzo

Astellas

Phase 3

2 8

Xospata

Astellas

Phase 3

1 9

Fludara

Sanofi

Phase 2

5 10

Venclexta

AbbVie

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2028

Phase 1Recruiting

Recurrent Childhood Acute Myeloid Leukemia

NCT06247787

Q2 2028

Phase 3Active, not recruiting

Myelofibrosis

NCT04576156

Q2 2035

Phase 1Recruiting

Relapsed AML

NCT07320235

Clinical

Trials by Indication[1]

Myelofibrosis3 trials

Study to Evaluate Activity of 2 Dose Levels of Imetelstat in Participants With Intermediate-2 or High-Risk Myelofibrosis (MF) Previously Treated With Janus Kinase (JAK) Inhibitor

Completed·GGeron Corporation·Phase 2·NCT02426086·107 enrolled

Imetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis

Completed·GGeron Corporation·Phase 2·NCT01731951·80 enrolled

A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat (GRN163L) Versus Best Available Therapy (BAT) in Patients With Intermediate-2 or High-risk Myelofibrosis (MF) Relapsed / Refractory (R/R) to Janus Kinase (JAK) Inhibitor

Active, not recruiting·GGeron Corporation·Phase 3·NCT04576156·327 enrolled

Multiple Myeloma1 trial

Open Label Study With Imetelstat to Determine Effect of Imetelstat in Patients w/ Previously Treated Multiple Myeloma

Completed·GGeron Corporation·Phase 2·NCT01242930·13 enrolled

Neuroblastoma1 trial

Imetelstat Given Intravenously Alone and With Standard 13-Cis-Retinoic Acid in Children With Neuroblastoma

Withdrawn·NNCIC Clinical Trials Group·Phase 1·NCT01916187

Non-Small Cell Lung Cancer1 trial

Imetelstat as Maintenance Therapy After Initial Induction Chemotherapy in Non-small Cell Lung Cancer (NSCLC)

Completed·GGeron Corporation·Phase 2·NCT01137968·116 enrolled

Lymphoma1 trial

Imetelstat for Children With Refractory or Recurrent Solid Tumors and Lymphoma

Withdrawn·NNational Cancer Institute (NCI)·Phase 1·NCT01568632

4 more indications

Essential Thrombocythemia1 trial

Open Label Study to Evaluate the Activity of Imetelstat in Patients With Essential Thrombocythemia or Polycythemia Vera

Completed·GGeron Corporation·Phase 2·NCT01243073·20 enrolled

Imetelstat1 trial

Expanded Access for Treatment With Imetelstat

Approved for marketing·GGeron Corporation··NCT05937568

Relapsed AML1 trial

Imetelstat Combinations in Relapsed AML

Recruiting·UUnknown Sponsor·Phase 1·NCT07320235·36 enrolled

Recurrent Childhood Acute Myeloid Leukemia1 trial

A Phase 1 Study of GRN163L (Imetelstat) in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia That is in Second or Greater Relapse or That is Refractory to Relapse Therapy; Myelodysplastic Syndrome or Juvenile Myelomonocytic Leukemia in First or Greater Relapse or is Refractory to Relapse Therapy

Recruiting·CChildren's Oncology Group·Phase 1·NCT06247787·36 enrolled

Regulatory

FDA Approval Timeline[4]

Jun 6, 2024

RYTELO

NDA NDA217779

Jun 6, 2024

RYTELO

NDA NDA217779

Apr 14, 2026

RYTELO

NDA NDA217779

Sources

References

[1]
ClinicalTrials.gov — 11 trial records for Imetelstat. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Imetelstat compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Imetelstat. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA217779). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Rytelo. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Geron Corporation company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Telomerase Inhibitor[2]
Modality: RNA therapy[2]
Highest Phase: Approved[1]
Company: GGeron Corporation

Patent Landscape[5]

Patent Cliff

Dec 27, 2026

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9388416Sep 9, 2026

9388415Sep 9, 2026

7494982Dec 27, 2026

12442000Mar 15, 2033

9375485Mar 15, 2033

1 more patents

12171778Jun 16, 2039

Rytelo

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2028

Q2 2028

Q2 2035

Trials by Indication[1]

Myelofibrosis3 trials

Multiple Myeloma1 trial

Neuroblastoma1 trial

Non-Small Cell Lung Cancer1 trial

Lymphoma1 trial

Essential Thrombocythemia1 trial

Imetelstat1 trial

Relapsed AML1 trial

Recurrent Childhood Acute Myeloid Leukemia1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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