Drug intelligence / Profile preview

imetelstat + pacritinib

Development stage
Preclinical
Lead developer
Geron
Modality
Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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 Molecules
Administration
Intravenous, Oral
01

Overview

This combination consists of two distinct investigational agents: - **Imetelstat** is a first-in-class oligonucleotide direct and competitive inhibitor of telomerase, targeting malignant hematopoietic stem and progenitor cells by binding the RNA template region of telomerase and inhibiting telomerase activity, leading to telomere shortening and apoptotic cell death of cancer cells. Developed and manufactured by Geron, it is administered intravenously and approved for myelodysplastic syndromes, while under investigation for myelofibrosis[2][4]. - **Pacritinib** is a small-molecule kinase inhibitor that orally inhibits wild-type and mutant JAK2, FMS-like tyrosine kinase 3 (FLT3), and also exerts inhibitory effects on additional kinases (CSF1R, IRAK1). It is mainly approved and used for myelofibrosis in patients with severe thrombocytopenia, developed and manufactured by CTI BioPharma (now part of Sobi)[6][8]. As a combination, imetelstat + pacritinib is being explored as a novel treatment in myelofibrosis, although no published clinical data specifically describes the efficacy, safety, or recommended dose of this particular combination to date. There are ongoing studies with combinations of imetelstat with other JAK inhibitors, but limited or no public clinical data yet on the imetelstat + pacritinib combination in humans[1][5][7][9].

Other names
imetelstat sodium
02

Targets

CSF1R (Macrophage colony-stimulating factor receptor)FLT3 (Fms related receptor tyrosine kinase 3)IRAK1 (Interleukin-1 receptor-associated kinase 1)JAK2 (Janus kinase 2)TERC (Telomerase RNA component)TERT (Telomerase)

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