Last update 28 Oct 2026

Imetelstat

Overview

Basic Info

Drug Type
Oligonucleotide
Synonyms
Imetelstat (USAN/INN), Imetelstat sodium, GRN 140719
+ [11]
Action
inhibitors
Mechanism
Telomerase inhibitors
Originator Organization
Drug Highest PhaseApproved
First Approval Date
United States (06 Jun 2024),
RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union)
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Structure/Sequence

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External Link

KEGGWikiATCDrug Bank
D09629Imetelstat

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Transfusion dependent anaemia
European Union
07 Mar 2025
Transfusion dependent anaemia
Iceland
07 Mar 2025
Transfusion dependent anaemia
Liechtenstein
07 Mar 2025
Transfusion dependent anaemia
Norway
07 Mar 2025
myelodysplastic anemia
United States
06 Jun 2024
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Myelodysplastic SyndromesNDA/BLA
United States
12 Jan 2024
MyelofibrosisPhase 3
United States
12 Apr 2021
MyelofibrosisPhase 3
Argentina
12 Apr 2021
MyelofibrosisPhase 3
Australia
12 Apr 2021
MyelofibrosisPhase 3
Austria
12 Apr 2021
MyelofibrosisPhase 3
Belgium
12 Apr 2021
MyelofibrosisPhase 3
Brazil
12 Apr 2021
MyelofibrosisPhase 3
Bulgaria
12 Apr 2021
MyelofibrosisPhase 3
Colombia
12 Apr 2021
MyelofibrosisPhase 3
Denmark
12 Apr 2021
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
468
xqhlyzcanp(vueuodlrsr) = srbykrilof ktkukloacv (lsxdojozyq )
Positive
12 May 2026
xqhlyzcanp(vueuodlrsr) = jygvtsmnbq ktkukloacv (lsxdojozyq )
Not Applicable
40
canhpzbhsn(skpnxaclll) = Among 9 pts with ASXL-1 mutation, 66.7% (6 pts) responded compared to 29% (9/31) WT, p= 0.04 dsjdhjzlwq (wdmlywzqwu )
Positive
12 May 2026
Phase 2
113
ltgydzhnwv(ivtyklpvla) = tgovkylxvr udrkmobifd (ptgvqwyzya, 25.5 - 36.9)
Positive
12 May 2026
Best Available Therapy (BAT)
ltgydzhnwv(ivtyklpvla) = qmjnqrfgok udrkmobifd (ptgvqwyzya, 13.1 - 30.5)
Phase 2
46
(Imetelstat Sodium (1 Treatment / Cycle))
cjdzexalsu = wncgspbvxj scuppsjnwf (tgrwvzdrsf, vlmzxtvjqn - sgsgoehxjk)
-
12 May 2026
(Imetelstat Sodium (2 Treatments / Cycle))
cjdzexalsu = yxxlvexgyd scuppsjnwf (tgrwvzdrsf, yjzfhxbtqm - xyhdwfvfxg)
Phase 3
226
kjvrgsqsdq(zkgvpmdqpz) = lgmotsfbfp mvgviwnucs (zsautiyhmn )
Positive
06 Dec 2025
Phase 2
24
wgshslqotn(zvkhfpequv) = 24 SAEs occurred in 19 patients from August 2024 until 8th May 2025, of which none were deemed related to imetelstat. The most common SAEs were febrile neutropenia (n=6), disease progression/transformation to AML (n=4), sepsis (n=2) and fever (n=2). Overall, 11/24 SAEs resulted in death (n=4 disease progression or AML, n=2 cardiac/cardiorespiratory arrest, n=1 febrile neutropenia, n=1 sepsis, general health alteration and death of unknown reason). 8 of the SAEs resolved without sequelae and 2 remained unresolved. In the first cohort, 30 SAEs occurred in 18 patients, with 3 deemed possibly related to imetelstat (pneumonia [n=2], febrile neutropenia [n=1]). The most common SAEs were disease progression/transformation to AML (n=9), pneumonia (n=3), febrile neutropenia (n=3), and sepsis (n=2). 10/30 SAEs resulted in death. qgnblmetix (cahlriakws )
Negative
06 Dec 2025
Phase 3
178
hifjcnsfye(pewutahqyz) = xydqvidegb epqaskjewr (kwcyixmavw, 38.3 - NE)
Positive
06 Dec 2025
Placebo
hifjcnsfye(pewutahqyz) = qlqiatdios epqaskjewr (kwcyixmavw, 37.4 - NE)
Phase 2
43
iqqrkvclcr(tegyhvcgna) = xhdpjsygii dmablpnycx (crkjwocrsf )
Positive
06 Dec 2025
qxyzuiafcf(gcahgxtnyo) = zdusivptdi wpvabogcae (avlreswfhn )
Not Applicable
Myelofibrosis
JAK2 mutation | calreticulin (CALR) mutation
197
baxfuoczby(bevdvdqssh) = 42% kmubhrhgpx (vdcafjodwz )
Positive
30 May 2025
Phase 2/3
Myelodysplastic Syndromes
ESA | luspatercept | lenalidomide ...
226
taqzpsfazs(xvjojlddpz) = lstrnjophg juahqqlqsx (eexttpyiik )
Positive
30 May 2025
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Approval

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Regulation

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