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AG-221

Phase 3

Leukemia, Myeloid | Small molecule | Oncology |Bristol-Myers Squibb Company|Last Updated: May 18, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLEDDMC
Total Trials1
Total Enrollment319

FDA Designations

No designations recorded

Clinical trial landscape

AG-221 · 3 trials · 4 indications

Phase 3 1Phase 1 2
NCT02577406An Efficacy and Safety Study of AG-221 (CC-90007) Versus Conventional Care Regimens in Older Subjects With Late Stage Acute Myeloid Leukemia Harboring an Isocitrate Dehydrogenase 2 MutationLeukemia, Myeloid
COMPLETED319 Analytics
PHASE3COMPLETED
An Efficacy and Safety Study of AG-221 (CC-90007) Versus Conventional Care Regimens in Older Subjects With Late Stage Acute Myeloid Leukemia Harboring an Isocitrate Dehydrogenase 2 Mutation
Leukemia, MyeloidUnlock trial analytics

Study Endpoints

Primary Endpoints

Overall Survival (OS)
From randomization to death due to any cause (up to approximately 49 months)

The time between randomization and death from any cause. Participants who drop-out or are alive at the end of trial will have their OS times censored at the time of last contact, as appropriate.

Pharmacokinetics - Total [14C] Whole blood and plasma
Approximately 3 weeks

Total \[14C\]-radioactivity in whole blood and plasma

Pharmacokinetics - Total [14C] urine and feces
approximately 3 weeks

Total \[14C\]-radioactivity in urine, and feces (and vomitus, if applicable).

Pharmacokinetics - Total [14C] Cumulative excretion
approximately 3 weeks

Cumulative excretion of total \[14C\]-radioactivity (as a fraction of the radioactive dose) in urine and feces (and vomitus, if applicable)

Pharmacokinetics - Total [14C] radioactivity
approximately 4 weeks

Total \[14C\]-radioactivity whole blood-to-plasma ratios

Pharmacokinetics - Total [14C] metabolites
approximately 3 weeks

Metabolite profiling / characterization in select plasma, urine, and fecal samples

Pharmacokinetics: Metabolite profiling and characterization in select biological matrices (Part 1)
approximately 4 weeks

The analysis for metabolite profiles and characterization are qualitative and there are no units for the analysis.

Pharmacokinetics - Cmax
approximately 4 weeks

Maximum observed plasma concentration

Pharmacokinetics - AUC
approximately 4 weeks

Area under the plasma concentration-time curve

Pharmacokinetics - Tmax
approximately 4 weeks

Time to maximum observed plasma concentration

Pharmacokinetics -T1/2
approximately 4 weeks

Estimate of the terminal elimination half-life

Pharmacokinetics -AUC 0-t
Up to 3 days

Area under the plasma concentration-time curve from time zero to time t

Pharmacokinetics - AUC ∞
Up to 3 days

Area under the plasma concentration-time curve from time zero extrapolated to infinity

Pharmacokinetics -CL/F
Up to 3 days

Apparent total plasma clearance when dosed orally

Pharmacokinetics -Vz/f
Up to 3 days

Apparent volume of distribution during terminal phase

Secondary Endpoints

Overall Response Rate
From randomization up to study completion (approximately 78 months)
Event-Free Survival
From randomization up to study completion (approximately 78 months)
Duration of Response
From randomization up to study completion (approximately 78 months)
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AG-221 plus Best supportive care (BSC)EXPERIMENTALContinuous 28-day cycles of AG 221 100 mg orally (PO) once a day (QD) for 28 days, plus BSC.
Conventional care regimen (CCR)ACTIVE_COMPARATORContinuous 28-day cycles of BSC only, azacitidine subcutaneously (SC) plus BSC, low-dose cytarabine (LDAC) SC plus BSC, or intermediate-dose cytarabine (IDAC) intravenously (IV) plus BSC. Subjects will be assigned by the investigator to one of the CCR treatment options based on the investigator's assessment of subjects' eligibility.
100-mg AG-221 oral solution + a microtracer of [14C]-AG- 221EXPERIMENTALSubjects will receive a 100 mg AG-221 to be swallowed with 240 mL of room-temperature, non-carbonated water.
100-mg AG-221 tablet + 100 micrograms [14C] AG-221EXPERIMENTALFormulated tablet containing 100 mg AG-221 + IV solution containing 100 micrograms \[14C\] AG-221
50 mg AG-221 tabletEXPERIMENTAL50 mg AG-221 tablet given by mouth with 240 mL of non-carbonated, room temperature water, under fasted conditions
100 mg AG-221 tabletEXPERIMENTAL100 mg AG-221 tablet given by mouth with 240 mL of non-carbonated, room temperature water, under fasted conditions
300 mg AG-221 tabletEXPERIMENTAL300 mg AG-221 tablet given by mouth with 240 mL of non-carbonated, room temperature water, under fasted conditions

Interventions

NameTypeDescription
AG-221DRUGContinuous 28-day cycles of AG 221 100 mg orally (PO) once a day (QD) for 28 days
BSCOTHERBest supportive care includes, hydroxyurea for leukocytosis and/or differentiation-like syndrome, anti-infectives, analgesics, antiemetics, antipyretics, transfusions and nutritional support
AzacitidineDRUGcontinuous 28-day cycles of azacitidine 75 mg/m2/day SC for 7 days, plus BSC
Low-dose cytarabine (LDAC)DRUGcontinuous 28-day cycles of cytarabine 20 mg SC twice a day (BID) for 10 days, plus BSC
Intermediate-dose cytarabine (IDAC)DRUG28-day cycles of cytarabine 0.5 to 1.5 g/m2/day IV for 3 to 6 days, per standard institutional practice, plus BSC; only BSC given after IDAC therapy concludes per standard institutional practice
14C AG-221RADIATION5 mL of 100 micrograms \[14C\] AG-221 given intravenously 4 hours after swallowing the formulated tablet.
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Eligibility Criteria

Age Range60 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites93

Inclusion Criteria: Subjects must satisfy the following criteria to be enrolled in the study: 1. Subject is ≥ 60 years of age at the time of signing the ICF 2. Subject has primary (ie, de novo) or secondary (progression of MDS or myeloproliferative neoplasms (\[MPN\], or therapy-related) AML accor...

Countries:United StatesAustraliaAustriaBelgiumBrazilCanadaChinaCzechiaDenmarkFranceGermanyItalyRussiaSouth KoreaSpainTaiwanTurkey (Türkiye)United Kingdom
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Frequently asked questions about AG-221

What is AG-221 used for?

AG-221 is an investigational small molecule being studied for the treatment of late stage acute myeloid leukemia in older subjects who harbor an isocitrate dehydrogenase 2 mutation. It has also been studied in healthy volunteers for pharmacokinetic and safety purposes. It is not approved and remains in clinical development.

Who makes AG-221?

AG-221 is being developed by Bristol-Myers Squibb Company, traded on the New York Stock Exchange under the ticker BMY. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with acute myeloid leukemia.

What phase is AG-221 in?

AG-221 has completed a Phase 3 clinical trial for late stage acute myeloid leukemia in older subjects with an isocitrate dehydrogenase 2 mutation. It has also completed Phase 1 studies in healthy volunteers. The drug is investigational and not yet approved.

What clinical trials is AG-221 in?

AG-221 has been studied in three completed trials. NCT02577406 is a Phase 3 efficacy and safety study in older subjects with late stage acute myeloid leukemia harboring an IDH2 mutation. NCT02387866 and NCT02443168 are Phase 1 pharmacokinetic and safety studies in healthy male subjects.

What does AG-221 target?

AG-221 targets the isocitrate dehydrogenase 2 (IDH2) enzyme. The Phase 3 trial specifically enrolled patients with acute myeloid leukemia harboring an IDH2 mutation, indicating the drug is designed to act on this molecular target.

Is AG-221 the same as CC-90007?

Yes, AG-221 is also known as CC-90007. The Phase 3 clinical trial NCT02577406 is titled 'An Efficacy and Safety Study of AG-221 (CC-90007) Versus Conventional Care Regimens in Older Subjects With Late Stage Acute Myeloid Leukemia Harboring an Isocitrate Dehydrogenase 2 Mutation.'