Reblozyl

(Luspatercept)

Phase 3Protein / enzyme

Luspatercept is used to treat anemia (low red blood cell counts) in patients with certain blood disorders, including myelodysplastic syndromes and beta-thalassemia, by helping the body produce more red blood cells.[1] Reblozyl is currently being investigated across 15 indications[3], with its most advanced program in Phase 3.[1]

Reblozyl is one of 339 small molecule programs targeting Ovarian Granulosa Cell Tumor. It is currently in Phase 3.

Indications

Total Trials

Active Trials

338

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[3][1]

Indication

Ovarian Granulosa Cell TumorActive, not recruiting

Maligant Granulosa Cell Tumor of OvaryActive, not recruiting

Myelodysplastic/myeloproliferative NeoplasmActive, not recruiting

Anemia (phenotype)Unknown

AnemiaRecruiting

MyelofibrosisActive, not recruiting

Myelodysplastic SyndromeUnknown

Severe Aplastic AnemiaActive, not recruiting

Acute Myeloid LeukemiaRecruiting

Myelodysplastic SyndromesActive, not recruiting

Ovarian Granulosa Cell TumorPreclinical

Active, not recruiting

Maligant Granulosa Cell Tumor of OvaryPreclinical

Active, not recruiting

Myelodysplastic/myeloproliferative NeoplasmPhase 2

Active, not recruiting

Anemia (phenotype)Preclinical

Unknown

AnemiaPhase 2

Recruiting

MyelofibrosisPhase 2

Active, not recruiting

Myelodysplastic SyndromePreclinical

Unknown

Severe Aplastic AnemiaPhase 2

Active, not recruiting

Acute Myeloid LeukemiaPhase 2

Recruiting

Myelodysplastic SyndromesPhase 3

Active, not recruiting

Showing 10 of 15 indications

Competition

Competitive Landscape

804 competitors in Acute Myeloid Leukemia

#DrugCompanyPhaseTrials

Neupogen

EEastern Cooperative Oncology Group

Phase 3

31 2

Epogen

Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

Phase 3

1 3

Decadron

AAspen Global Incorporated

Phase 3

5 4

Retin-a

VValeant Pharmaceuticals

Phase 3

2 5

Reblozyl

Bristol-Myers Squibb

Phase 3

1 6

Lenograstim

Sanofi

Phase 3

1 7

Experimental

Biogen

Phase 3

1 8

Oblimersen Sodium

NNational Cancer Institute (NCI)

Phase 3

3 9

Valspodar

EEastern Cooperative Oncology Group

Phase 3

3 10

Iomab-B

AActinium Pharmaceuticals

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2026

Phase 2Active, not recruiting

Thalassemia

NCT05567458

Phase 2Recruiting

Anemia

NCT05732961

Phase 2Recruiting

Myelodysplastic Syndromes

NCT05925504

Q4 2026

Phase 2Recruiting

Myelofibrosis

NCT06517875

Active, not recruiting

Thalassemia

NCT06596642

Q1 2027

Phase 3Not yet recruiting

Monotherapy

NCT06927232

Clinical

Trials by Indication[1]

Myelodysplastic Syndromes15 trials

The Tapering Dose of Luspatercept in Patients With Lower-risk Myelodysplastic Syndromes

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT05925504·36 enrolled

A Study to Evaluate Luspatercept Treatment Patterns and Outcomes in Erythropoiesis-Stimulating Agents-Naïve Patients With Lower-Risk Myelodysplastic Syndromes in the United States

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT06851065·418 enrolled

Extension Study to Evaluate Long-Term Effects of Luspatercept in Patients With Myelodysplastic Syndromes (MDS) (A536-05/MK-6143-003)

Completed· Merck & Co. logo

Merck & Co.·Phase 2·NCT02268383·75 enrolled

Show 12 more trials

A Retrospective Study to Describe Real-World Treatment Patterns and Clinical Outcomes Among Patients With Myelodysplastic Syndromes

Withdrawn· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT06581055

A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT02631070·229 enrolled

A Study of Incidence, Treatment Patterns, and Outcomes in Transfusion-dependent Lower-risk Myelodysplastic Syndromes in Spain

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07465029·1,300 enrolled

Treatment Patterns and Outcomes in Patients With Lower-risk Myelodysplastic Syndromes Treated With Luspatercept in China

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07073690·80 enrolled

A Study to Evaluate Treatment Patterns, Outcomes, and Healthcare Resource Utilization in Patients With Lower-Risk Myelodysplastic Syndromes Treated With Luspatercept

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07075406·250 enrolled

A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 4·NCT05891249·85 enrolled

A Study to Evaluate the Efficacy, Drug Levels and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Chinese and Japanese Participants With Ring Sideroblasts Who Require Red Blood Cell Transfusions

Unknown status· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT04477850·30 enrolled

An Efficacy and Safety Study of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in Japanese Subjects Who Are Not Requiring Red Blood Cell Transfusion

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT03900715·21 enrolled

A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT03682536·363 enrolled

Real World Use of Luspatercept in Lower Risk Myelodysplastic Syndromes and Beta-thalassemia Patients

Terminated· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07681440·30 enrolled

A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) vs Epoetin Alfa for the Treatment of Anemia Due to Revised International Prognostic Scoring System (IPSS-R) Very Low, Low, or Intermediate-Risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-Stimulating Agent (ESA)-Naive Participants Who Are Non-Transfusion Dependent (NTD): The "ELEMENT-MDS" Trial

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT05949684·402 enrolled

A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)

Active, not recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT06113302·10 enrolled

Anemia7 trials

Luspatercept for the Treatment of Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation(Allo-HSCT)

Recruiting·ZZhujiang Hospital·Phase 1/2·NCT07362095·46 enrolled

Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to MDS With del5q, Refractory/Resistant/Intolerant to Prior Treatments, RBC-TD

Recruiting·AAssociazione Qol-one·Phase 2·NCT05924100·22 enrolled

Luspatercept for Anemia in Lower Risk MDS or Non-proliferative MDS/MPN Neoplasms

Recruiting·HH. Lee Moffitt Cancer Center and Research Institute·Phase 2·NCT05732961·70 enrolled

Show 4 more trials

The Efficacy and Safety of Luspatercept in Improving Early Anemia After HSCT

Not yet recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT07450313·20 enrolled

A Trial Comparing Three Different Treatment Options for Adults With Low-Risk Myelodysplasia and Anemia (A MyeloMATCH Treatment Trial)

Not yet recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT07463820·270 enrolled

A Phase 2, Study for the Treatment of Anemia With Alpha (α)-Thalassemia to Determine the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) in Adults and Evaluate the Safety and Pharmacokinetics in Adolescents

Recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT05664737·189 enrolled

Efficacy and Safety of Luspatercept in Treating Chemotherapy-inducing Anemia in Acute Myeloid Leukemia: a Multicenter, Prospective, Single-arm Study

Recruiting·GGuangdong Second Provincial General Hospital·Preclinical·NCT07663864·40 enrolled

Thalassemia7 trials

A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT03342404·145 enrolled

The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study

Unknown status·SSun Yat-sen University·Phase 4·NCT05462548·20 enrolled

A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT06596642·61 enrolled

Show 4 more trials

Clinical Observation of Luspatercept in Treatment of Chinese Adult β-thalassaemia Patients With TD β-thalassemia

Unknown status·HHematology department of the 920th hospital·N/A·NCT06164821·10 enrolled

A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT05567458·94 enrolled

Extension Study to Evaluate the Safety and Efficacy of Luspatercept in Participants With β-Thalassemia Previously Enrolled in A536-04 (A536-06/MK-6143-004)

Completed· Merck & Co. logo

Merck & Co.·Phase 2·NCT02268409·51 enrolled

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT02604433·336 enrolled

Myelodysplastic Syndrome4 trials

A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia

Recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT06073860·104 enrolled

A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT06045689·106 enrolled

Study of Luspatercept for the Treatment of Anemia in Patients With Myelodysplastic Syndrome (MDS) (MK-6143-001)

Completed· Merck & Co. logo

Merck & Co.·Phase 2·NCT01749514·116 enrolled

Show 1 more trial

Assessment of Effectiveness and Safety of Luspatercept in Patients Suffering From Lower-risk Myelodysplastic Syndrome.

Active, not recruiting·GGWT-TUD GmbH·Phase 3·NCT05181592·70 enrolled

Myelofibrosis2 trials

A Safety and Efficacy Study to Evaluate Luspatercept in Subjects With Myeloproliferative Neoplasm-associated Myelofibrosis Who Have Anemia With and Without Red Blood Cell-transfusion Dependence

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT03194542·95 enrolled

Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis

Recruiting· GSK logo

GSK·Phase 2·NCT06517875·56 enrolled

17 more indications

MDS2 trials

Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA

Recruiting·GGroupe Francophone des Myelodysplasies·Phase 1/2·NCT05181735·150 enrolled

Luspatercept + Darbepoetin in MDS

Recruiting·YYale University·Phase 2·NCT07096297·60 enrolled

Monotherapy2 trials

AZA+Lus VS AZA Monotherapy in HR-MDS

Not yet recruiting·PPeking Union Medical College Hospital·Phase 3·NCT06927232·86 enrolled

Roxadustat Combined With Luspatercept Versus Luspatercept Monotherapy in the Treatment of Refractory MDS-RS

Unknown status·PPeking Union Medical College Hospital·Phase 4·NCT06006949·62 enrolled

Beta-Thalassemia2 trials

Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)

Completed· Merck & Co. logo

Merck & Co.·Phase 2·NCT01749540·64 enrolled

A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East

Recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT07215975·200 enrolled

Safety and Efficacy2 trials

To Evaluate Long- Term Safety and Efficacy of Luspatercept

Active, not recruiting·FFondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia··NCT06875219·350 enrolled

Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase

Active, not recruiting·FFondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia··NCT06913634·140 enrolled

Severe Aplastic Anemia2 trials

Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia

Completed·BBing Han·Phase 2·NCT05399732·58 enrolled

Treatment of Transfusion-dependent Nonsevere Aplastic Anemia With Luspatercept: a Multicenter Prospective Clinical Study

Not yet recruiting·TThe First Affiliated Hospital of Zhejiang Chinese Medical University·N/A·NCT06964971·90 enrolled

AIN1 trial

Luspatercept for Clonal Cytopenias of Uncertain Significance

Recruiting·WWeill Medical College of Cornell University·Phase 2·NCT06788691·50 enrolled

Myelodysplastic/Myeloproliferative Neoplasm1 trial

Luspatercept With or Without Hydroxyurea for the Treatment of Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis or Unclassifiable With Ring Sideroblasts

Terminated·MMayo Clinic·Phase 2·NCT05005182·3 enrolled

Effectiveness1 trial

A Study to Evaluate Treatment Patterns and Effectiveness of Luspatercept

Active, not recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT06971185·430 enrolled

Immune Thrombocytopenia1 trial

Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia

Recruiting·FFondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico··NCT05931718·200 enrolled

Efficacy1 trial

Efficacy of Luspatercept in ESA-naive LR-MDS Patients With or Without Ring Sideroblasts Who do Not Require Transfusions

Recruiting·UUniversity of Leipzig·Phase 2·NCT05384691·213 enrolled

Myeloid1 trial

Connect® Myeloid Disease Registry

Terminated· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT01688011·2,013 enrolled

Efficacy and Safety1 trial

Efficacy and Safety of Luspatercept: a Study by Fondazione Italiana Sindromi Mielodisplastiche

Completed·FFondazione Italiana Sindromi Mielodisplastiche-ETS··NCT05520749·215 enrolled

Metastatic1 trial

Luspatercept in Metastatic AGCT of the Ovary

Completed·UUniversity Health Network, Toronto·N/A·NCT06254781·1 enrolled

Newly Diagnosed1 trial

Luspatercept Plus CsA vs CsA for the Treatment of Newly Diagnosed Non-Transfusion-Dependent NSAA

Not yet recruiting·PPeking Union Medical College Hospital·Phase 4·NCT06424639·58 enrolled

Lenalidomide1 trial

Luspatercept and Lenalidomide (L2) in Lower-risk, Non-del(5q) MDS Patients

Suspended·—·Phase 1/2·NCT04539236·50 enrolled

Acute Myeloid Leukemia1 trial

Master Screening and Reassessment Protocol (MSRP) for the NCI MyeloMATCH Clinical Trials

Recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT05564390·2,000 enrolled

Myelodysplastic Syndromes (MDS)1 trial

A Phase 3b, Open-label, Single-arm, Rollover Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Recruiting· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT04064060·665 enrolled

Regulatory

FDA Approval Timeline[4]

Nov 8, 2019

REBLOZYL

BLA BLA761136

Sources

References

[1]
ClinicalTrials.gov — 57 trial records for Luspatercept. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Luspatercept compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Luspatercept. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (BLA BLA761136). U.S. Food & Drug Administration.
[5]
SEC EDGAR — Bristol-Myers Squibb company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Transforming Growth Factor Beta Inhibitor[2]
Modality: Protein / enzyme[2]
Highest Phase: Phase 3[1]
Company: Bristol-Myers Squibb

Reblozyl

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2026

Q4 2026

Q1 2027

Trials by Indication[1]

Myelodysplastic Syndromes15 trials

Anemia7 trials

Thalassemia7 trials

Myelodysplastic Syndrome4 trials

Myelofibrosis2 trials

MDS2 trials

Monotherapy2 trials

Beta-Thalassemia2 trials

Safety and Efficacy2 trials

Severe Aplastic Anemia2 trials

AIN1 trial

Myelodysplastic/Myeloproliferative Neoplasm1 trial

Effectiveness1 trial

Immune Thrombocytopenia1 trial

Efficacy1 trial

Myeloid1 trial

Efficacy and Safety1 trial

Metastatic1 trial

Newly Diagnosed1 trial

Lenalidomide1 trial

Acute Myeloid Leukemia1 trial

Myelodysplastic Syndromes (MDS)1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

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