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ACE-536

Phase 3

Myeloproliferative Disorders | Small molecule | Hematology |Bristol-Myers Squibb Company|Last Updated: Jul 10, 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment313
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04717414An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell TransfusionsPHASE3 ACTIVE NOT_RECRUITING 313Feb 25, 2021Aug 18, 2032Jul 10, 2025185 United States, Argentina +24
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Study Endpoints
Primary Endpoints
Red blood cell-transfusion independence (RBC-TI) ≥ 12 weeks (RBC-TI 12)
Up to 24 weeks

Proportion of subjects who become RBC-transfusion free over any consecutive 12-week period starting within the first 24 weeks.

Secondary Endpoints
Red blood cell-transfusion independence ≥ 16 weeks (RBC-TI 16)
Up to 24 weeks
Duration of Red blood cell-transfusion independence (RBC-TI 12)
Up to end of treatment, approximately 3 years
Reduction of transfusion burden by ≥ 50% and by ≥ 4 units/12 weeks from baseline over any consecutive 12-week period
Up to 24 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Experimental Arm: Luspatercept (ACE-536)EXPERIMENTALLuspatercept will be given to participants via subcutaneous injection (administered on Day 1 of each 21-day treatment cycle)
Control Arm: PlaceboPLACEBO_COMPARATORPlacebo starting dose with volume equivalent to experimental arm subcutaneous injection every 3 weeks (administered on Day 1 of each 21-day treatment cycle)
Interventions
NameTypeDescription
ACE-536DRUGSubcutaneous Injection
PlaceboOTHERSubcutaneous Injection
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites185

Subjects must satisfy the following criteria to be randomized in the study: Inclusion Criteria \- Subject is ≥18 years of age at the time of signing the ICF. * Subject has a diagnosis of PMF according to the 2016 World Health Organization (WHO) criteria or diagnosis of post-ET or post-PV MF accor...

Countries:United StatesArgentinaAustraliaAustriaBelgiumCanadaChileChinaColombiaCzechiaFranceGermanyGreeceHong KongHungaryIrelandIsraelItalyJapanLebanonPolandRomaniaRussiaSouth KoreaSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04717414primaryCompletionDate: changed
LOWMay 24, 2026NCT04717414studyFirstPostDate: changed