imetelstat + azacitidine

Rytelo + azacitidine is an investigational combination regimen consisting of imetelstat (Rytelo), a first-in-class telomerase inhibitor, and azacitidine, a hypomethylating agent (nucleoside analog). Imetelstat is a lipidated 13-mer oligonucleotide that binds with high affinity to the RNA template region of the telomerase RNA component (TERC), competitively inhibiting telomerase enzymatic activity. This inhibition leads to progressive telomere shortening and triggers apoptosis in telomerase-overexpressing cancer cells, such as acute myeloid leukemia (AML) blasts and leukemia stem cells (LSCs). Azacitidine is a pyrimidine nucleoside analog that incorporates into DNA and RNA, inhibiting DNA methyltransferases (DNMTs) to cause DNA hypomethylation and direct cytotoxicity. The combination is being evaluated in the Phase 1/1b IMAGINE clinical trial for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML), with or without the addition of the BCL-2 inhibitor venetoclax.

Other names

imetelstat and azacitidineRytelo + azacitidineRytelo and azacitidine