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AG-221

Phase 3

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment319
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
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 MutationPHASE3 COMPLETED 319Dec 30, 2015Mar 25, 2024May 18, 202593 United States, Australia +16
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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.

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.
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
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Eligibility Criteria
Age Range60 Years — 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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