imetelstat

Imetelstat, marketed under the brand name Rytelo, is a first-in-class oligonucleotide telomerase inhibitor. It is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndrome (MDS) who have transfusion-dependent anemia and are unresponsive to or ineligible for erythropoiesis-stimulating agents (ESAs). Administered intravenously, imetelstat functions by binding with high affinity to the template region of the RNA component of human telomerase (hTR), thereby directly and competitively inhibiting telomerase enzymatic activity. This inhibition results in the progressive shortening of telomeres in malignant cells, subsequently reducing their proliferation and inducing apoptotic cell death. The drug exhibits a degree of selectivity for malignant stem and progenitor cells due to their typically higher telomerase activity compared to normal cells. Imetelstat received FDA approval in June 2024 for its MDS indication. It is also currently being investigated for potential use in treating other cancer types.

Brand names

Rytelo

Other names

imetelstat sodiumGRN163LGRN-163LGRN 163L