Drug intelligence / Profile preview

imetelstat + ruxolitinib

Development stage
Unknown
Lead developer
Geron
Modality
Small Molecules, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous (imetelstat), Oral (ruxolitinib)
01

Overview

**Imetelstat + ruxolitinib** is a combination regimen currently under investigation for the treatment of myelofibrosis. Imetelstat is a first-in-class, direct, and competitive inhibitor of telomerase, acting by binding to the RNA template component of telomerase and blocking its enzymatic activity, leading to telomere shortening, apoptotic cell death, and selective inhibition of malignant stem and progenitor cells[2][4][6]. Ruxolitinib is a small molecule inhibitor of Janus kinase 1 and Janus kinase 2, reducing inflammatory cytokine signaling and splenomegaly in myelofibrosis. Combination therapy seeks to leverage ruxolitinib’s symptom control and imetelstat’s potential disease modification against malignant clonal stem cells[1][3][5]. This combination is being evaluated in the IMproveMF Phase 1/1b clinical trial for patients with intermediate-1, intermediate-2, or high-risk myelofibrosis[3][5].

Other names
imetelstat + ruxolitinibimetelstat plus ruxolitinib
02

Targets

JAK1 (Janus kinase 1)JAK2 (Janus kinase 2)TERC (Telomerase RNA component)

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