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Clinical data | |
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Trade names | Rytelo |
Other names | GRN163L |
AHFS/ Drugs.com | Rytelo |
License data |
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Routes of administration | Intravenous |
ATC code | |
Legal status | |
Legal status | |
Identifiers | |
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CAS Number |
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PubChem CID | |
DrugBank | |
ChemSpider | |
UNII |
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KEGG | |
ChEMBL |
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Chemical and physical data | |
Formula | C148H211N68O53P13S13 |
Molar mass | 4610.18 g·mol−1 |
3D model ( JSmol) | |
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Imetelstat, sold under the brand name Rytelo, is an anti-cancer medication used for the treatment of myelodysplastic syndromes with transfusion-dependent anemia. [1] Imetelstat is an oligonucleotide telomerase inhibitor. [1] [2]
The most common adverse reactions include decreased platelets, decreased white blood cells, decreased neutrophils, increased aspartate aminotransferase, increased alkaline phosphatase, increased alanine aminotransferase, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache. [3]
Imetelstat was approved for medical use in the United States in June 2024. [3] [4]
Imetelstat is indicated for the treatment of adults with low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents. [1] [3]
Imetelstat is the first telomerase inhibitor to enter clinical trials. [5]
Chemically, imetelstat is a synthetic conjugate consisting of three parts: GRN163, a thio phosphoramide oligonucleotide, and a palmitoyl lipid group. [5] GRN163 is the pharmacological component with telomerase inhibition based on experiments with poly-G oligonucleotides first conducted at the University of Nebraska Medical Center under contract with Lynx Therapeutics. [6] The palmitic acid moiety is conjugated via a phosphothioate linkage to the backbone of the antisense oligonucleotide.[ medical citation needed] Telomere shortening and lower cell viability are observed after inhibition of telomerase activity in vitro.[ medical citation needed] IC50 values ranged from 50 to 200nM for 10 different pancreatic cell lines. [7]
The efficacy of imetelstat was evaluated in IMerge (NCT02598661), a randomized (2:1), double-blind, placebo-controlled multicenter trial in 178 participants with myelodysplastic syndromes. [3] Participants received an intravenous infusion of imetelstat 7.1 mg/kg or placebo in 28-day treatment cycles until disease progression or unacceptable toxicity. [3] Randomization was stratified by prior red blood cell transfusion burden and by International Prognostic Scoring System (IPSS) risk group. [3] All participants received supportive care, which included red blood cell transfusions. [3]
Imetelstat was approved for medical use in the United States in June 2024. [3] The FDA granted the application for imetelstat orphan drug designation. [3] [8] [9]
Imetelstat is the international nonproprietary name. [10]
{{
cite web}}
: CS1 maint: archived copy as title (
link)
![]() | |
![]() | |
Clinical data | |
---|---|
Trade names | Rytelo |
Other names | GRN163L |
AHFS/ Drugs.com | Rytelo |
License data |
|
Routes of administration | Intravenous |
ATC code | |
Legal status | |
Legal status | |
Identifiers | |
| |
CAS Number |
|
PubChem CID | |
DrugBank | |
ChemSpider | |
UNII |
|
KEGG | |
ChEMBL |
|
Chemical and physical data | |
Formula | C148H211N68O53P13S13 |
Molar mass | 4610.18 g·mol−1 |
3D model ( JSmol) | |
| |
|
Imetelstat, sold under the brand name Rytelo, is an anti-cancer medication used for the treatment of myelodysplastic syndromes with transfusion-dependent anemia. [1] Imetelstat is an oligonucleotide telomerase inhibitor. [1] [2]
The most common adverse reactions include decreased platelets, decreased white blood cells, decreased neutrophils, increased aspartate aminotransferase, increased alkaline phosphatase, increased alanine aminotransferase, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache. [3]
Imetelstat was approved for medical use in the United States in June 2024. [3] [4]
Imetelstat is indicated for the treatment of adults with low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents. [1] [3]
Imetelstat is the first telomerase inhibitor to enter clinical trials. [5]
Chemically, imetelstat is a synthetic conjugate consisting of three parts: GRN163, a thio phosphoramide oligonucleotide, and a palmitoyl lipid group. [5] GRN163 is the pharmacological component with telomerase inhibition based on experiments with poly-G oligonucleotides first conducted at the University of Nebraska Medical Center under contract with Lynx Therapeutics. [6] The palmitic acid moiety is conjugated via a phosphothioate linkage to the backbone of the antisense oligonucleotide.[ medical citation needed] Telomere shortening and lower cell viability are observed after inhibition of telomerase activity in vitro.[ medical citation needed] IC50 values ranged from 50 to 200nM for 10 different pancreatic cell lines. [7]
The efficacy of imetelstat was evaluated in IMerge (NCT02598661), a randomized (2:1), double-blind, placebo-controlled multicenter trial in 178 participants with myelodysplastic syndromes. [3] Participants received an intravenous infusion of imetelstat 7.1 mg/kg or placebo in 28-day treatment cycles until disease progression or unacceptable toxicity. [3] Randomization was stratified by prior red blood cell transfusion burden and by International Prognostic Scoring System (IPSS) risk group. [3] All participants received supportive care, which included red blood cell transfusions. [3]
Imetelstat was approved for medical use in the United States in June 2024. [3] The FDA granted the application for imetelstat orphan drug designation. [3] [8] [9]
Imetelstat is the international nonproprietary name. [10]
{{
cite web}}
: CS1 maint: archived copy as title (
link)