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Enasidenib

Phase 2

Clonal Cytopenia of Undetermined Significance | Small molecule | Other |Bristol-Myers Squibb Company|Last Updated: Feb 11, 2026

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment15
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06240754Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2A Decentralized TrialPHASE2 RECRUITING 15Oct 10, 2024Feb 28, 2029Feb 11, 20261 United States
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Study Endpoints
Primary Endpoints
Best hematologic response
Up to 18 cycles (each cycle is 28 days) of treatment (up to approximately 17 months)

Hematologic response to enasidenib will be evaluated according to a modified version of the IWG 2006 Criteria for Hematologic Improvement for patients with MDS on clinical trials

Secondary Endpoints
Toxicity as measured by the number of adverse events experienced by participant
From start of treatment through 30 days after the last day of treatment (up to approximately 18 months)
Change in mutant IDH2 variant allele fraction
Baseline, day 1 of cycles 3/6/9/12/15 (each cycle is 28 days), and end of treatment (up to approximately 17 months)
Duration of hematologic improvement
From start of treatment through completion of treatment (estimated to be 17 months)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
EnasidenibEXPERIMENTALParticipants will receive enasidenib 100 mg daily for 18 cycles (each cycle is 28 days). Participants will continue treatment with enasidenib until confirmed progression to AML or MDS, development of unacceptable toxicity, or suspicion of disease progression, provided the patient is deriving clinical benefit, which will be determined at the discretion of the principal investigator.
Interventions
NameTypeDescription
EnasidenibDRUGProvided by BMS.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Unexplained cytopenia for at least 6 months. Cytopenia is defined as the presence of ≥1 blood count indexes below the following thresholds: * Hgb \<10 g/dL * ANC \<1.8 × 109/L * Platelets \<100 × 109/L * IDH2 gene mutation (R140 or R172), performed locally, at a frequen...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT06240754studyFirstPostDate: changed