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Luspatercept

Phase 2

Anemia | Monoclonal antibody | Hematology |Bristol-Myers Squibb Company|Last Updated: Mar 24, 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment189
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05664737A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-ThalassemiaPHASE2 RECRUITING 189 — — Dec 9, 2022Aug 14, 2034Mar 24, 202636 Canada, China +9
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Study Endpoints
Primary Endpoints
Number of participants with ≥ 50% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units during any continuous 12 weeks during Week 13-48 compared to 12-week interval immediately prior to date of first dose
Up to Week 48

Adult TD Cohort

Number of participants with an increase from baseline of ≥ 1.0 grams (g)/decilitre (dL) in mean hemoglobin (Hb) values over the continuous 12-week interval from Week 13 to Week 24 in the absence of RBC transfusion
Up to Week 24

Adult NTD Cohort

Dose-limiting toxicities (DLTs) defined as observance of ≥ Grade 3-related hemolytic crises or ≥ Grade 3-related event outside of the known safety profile occurring within the 21 days from their first dose of study therapy
Up to Week 3

Adolescent TD and NTD Cohorts

Number of participants with adverse events (AEs)
Up to 8.5 years

Adolescent TD and NTD Cohorts

Pharmacokinetics (PK): Serum concentration of Luspatercept
Up to Week 102

Adolescent TD and NTD Cohorts

Secondary Endpoints
Number of participants with ≥ 33% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units during any continuous 24-week interval on treatment compared to 24-week interval immediately prior to date of first dose
Up to Week 108
The longest duration with ≥ 50% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units
Up to Week 108
Number of RBC transfusion units from week 1 to week 48
Up to Week 48
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Transfusion Dependent (TD): Luspatercept + Best supportive care (BSC)EXPERIMENTAL -
Adult TD Cohort: Placebo + BSCPLACEBO_COMPARATOR -
Non-transfusion Dependent (NTD): Luspatercept + BSCEXPERIMENTAL -
Adult NTD Cohort: Placebo + BSCPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
LuspaterceptBIOLOGICALSpecified dose on specified days
PlaceboDRUGSpecified dose on specified days
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites36

Key Inclusion Criteria: * Adult participant≥ 18 years with documented diagnosis of A-Thal HbH disease with Transfusion dependence defined as:. 1. TD participant: ≥ 6 RBC units during the 24 weeks prior to randomization. 2. NTD participant:\< 6 RBC units during the 24 weeks prior to randomizati...

Countries:CanadaChinaGreeceHong KongItalyMalaysiaSaudi ArabiaSingaporeTaiwanThailandTurkey (Türkiye)
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Competitive Landscape -Anemia 17 trials
CompanyTickerTrialsLead PhaseDrugs
Bristol-Myers Squibb CompanyBMY2PHASE3ACE-536, Luspatercept
Akebia Therapeutics, Inc.AKBA3PHASE3Erythropoiesis-Stimulating Agent, Vadadustat, Erythropoiesis Stimulating Agent, Epoetin alga /vadadustat
Takeda Pharmaceutical Co. Ltd. Sponsored ADRTAK1PHASE3Elritercept, Epoetin Alfa
Cerus CorporationCERS1PHASE3Undisclosed
Disc Medicine, Inc.IRON2PHASE2DISC-0974
Incyte CorporationINCY1PHASE1INCB000928, ruxolitinib
Ascentage Pharma Group International Unsponsored ADRAAPG1PHASE1APG-5918
Harvard Bioscience, Inc.HBIO1NAUndisclosed
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Recent Changes (Last 90 Days)
HIGHMay 26, 2026NCT05664737Enrollment: 249 → 189
LOWMay 24, 2026NCT05664737studyFirstPostDate: changed