What drug is Imetelstat?

Imetelstat, sold under the trade name Rytelo, is an anticancer drug used to treat myelodysplastic syndrome with transfusion-dependent anemia. Imetelstat is an oligonucleotide telomerase inhibitor.

Imetelstat is the first telomerase inhibitor to enter clinical trials. Chemically, imetelstat is a synthetic conjugate consisting of three parts: GRN163, a thiophosphoramide oligonucleotide, and a palmitoyl lipid group. GRN163 is a pharmacological component with telomerase inhibitory effect. The palmitic acid moiety binds to the backbone of the antisense oligonucleotide through a phosphorothioate bond. After inhibition of telomerase activity in vitro, telomere shortening and reduced cell survival were observed. IC50 values u200bu200bfor 10 different pancreatic cell lines ranged from 50 to 200 nM.

The efficacy of imetelstat was evaluated in IMerge (NCT02598661), a randomized (2:1), double-blind, placebo-controlled, multicenter trial involving 178 patients with myelodysplastic syndromes. Participants received 7.1 mg/kg of imetelstat or placebo intravenously during 28-day treatment cycles until disease progression or unacceptable toxicity. Randomization was stratified by previous red blood cell transfusion volume and International Prognostic Scoring System (IPSS) risk group. All participants received supportive care, including red blood cell transfusions.

To summarize,Imetelstat (Rytelo) is an innovative drug targeting patients with a specific type of myelodysplastic syndrome. It works by inhibiting telomerase activity and has shown significant therapeutic effects in clinical trials.