Luspatercept

Summary

Luspatercept (CHEMBL3039545) is an approved protein (ATC B03XA06); indicated across 7 conditions including beta thalassemia and myelodysplastic syndrome.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Protein
• ATC class: B03XA06
• Indications: 7 conditions
• Clinical trials: 57

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3039545
Name	Luspatercept
Type	Protein
Max phase	4
ATC	B03XA06

Also known as: ACE-536, Luspatercept, Luspatercept aamt, Luspatercept-aamt, Reblozyl, LUSPATERCEPT

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

7 indications (5 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
beta thalassemia	4	MONDO:0019402	Orphanet:848
myelodysplastic syndrome	4	MONDO:0018881	EFO:0000198
anemia	4	MONDO:0002280	EFO:0004272
thalassemia	4	MONDO:0000984	EFO:1001996
aplastic anemia	2	MONDO:0015909	HP:0001915

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 57.

Phase distribution

Phase	Trials
PHASE2	24
Not specified	17
PHASE3	9
PHASE4	3
PHASE1/PHASE2	2
PHASE2/PHASE3	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05891249	PHASE4	ACTIVE_NOT_RECRUITING	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
NCT06424639	PHASE4	NOT_YET_RECRUITING	Luspatercept Plus CsA vs CsA for the Treatment of Newly Diagnosed Non-Transfusion-Dependent NSAA
NCT06006949	PHASE4	UNKNOWN	Roxadustat Combined With Luspatercept Versus Luspatercept Monotherapy in the Treatment of Refractory MDS-RS
NCT03682536	PHASE3	ACTIVE_NOT_RECRUITING	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
NCT04064060	PHASE3	RECRUITING	A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
NCT04717414	PHASE3	ACTIVE_NOT_RECRUITING	An 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 Transfusions
NCT05181592	PHASE3	ACTIVE_NOT_RECRUITING	Assessment of Effectiveness and Safety of Luspatercept in Patients Suffering From Lower-risk Myelodysplastic Syndrome.
NCT05949684	PHASE3	ACTIVE_NOT_RECRUITING	ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions
NCT06045689	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants
NCT06927232	PHASE3	NOT_YET_RECRUITING	AZA+Lus VS AZA Monotherapy in HR-MDS
NCT07539779	PHASE2/PHASE3	NOT_YET_RECRUITING	Luspatercept in Preventing Poor Erythroid Engraftment for Hematological Malignancies With Moderate to Severe Myelofibrosis
NCT02604433	PHASE3	COMPLETED	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
NCT02631070	PHASE3	COMPLETED	A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes
NCT04143724	PHASE2	RECRUITING	Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
NCT05384691	PHASE2	RECRUITING	Efficacy of Luspatercept in ESA-naive LR-MDS Patients With or Without Ring Sideroblasts Who do Not Require Transfusions
NCT05564390	PHASE2	RECRUITING	MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
NCT05567458	PHASE2	ACTIVE_NOT_RECRUITING	A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
NCT05664737	PHASE2	RECRUITING	A 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)-Thalassemia
NCT05732961	PHASE2	ACTIVE_NOT_RECRUITING	Luspatercept for Anemia in Lower Risk MDS or Non-proliferative MDS/MPN Neoplasms
NCT05925504	PHASE2	RECRUITING	The Tapering Dose of Luspatercept in Patients With Lower-risk Myelodysplastic Syndromes
NCT06113302	PHASE2	RECRUITING	A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)
NCT06517875	PHASE2	RECRUITING	Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis
NCT06788691	PHASE2	RECRUITING	Luspatercept for Clonal Cytopenias of Uncertain Significance
NCT07096297	PHASE2	RECRUITING	Luspatercept + Darbepoetin in MDS
NCT07331818	PHASE2	NOT_YET_RECRUITING	Luspatercept in Patients Affected With Rare Inherited Anemias
NCT07362095	PHASE1/PHASE2	RECRUITING	Luspatercept for the Treatment of Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation(Allo-HSCT)
NCT07450313	PHASE2	NOT_YET_RECRUITING	The Efficacy and Safety of Luspatercept in Improving Early Anemia After HSCT
NCT07463820	PHASE2	NOT_YET_RECRUITING	A Trial Comparing Three Different Treatment Options for Adults With Low-Risk Myelodysplasia and Anemia (A MyeloMATCH Treatment Trial)
NCT01749514	PHASE2	COMPLETED	Study of Luspatercept for the Treatment of Anemia in Patients With Myelodysplastic Syndrome (MDS) (MK-6143-001)
NCT01749540	PHASE2	COMPLETED	Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
NCT02268383	PHASE2	COMPLETED	Extension Study to Evaluate Long-Term Effects of Luspatercept in Patients With Myelodysplastic Syndromes (MDS) (A536-05/MK-6143-003)
NCT02268409	PHASE2	COMPLETED	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)
NCT03194542	PHASE2	COMPLETED	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
NCT03342404	PHASE2	COMPLETED	A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia
NCT03900715	PHASE2	COMPLETED	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
NCT04477850	PHASE2	COMPLETED	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
NCT04539236	PHASE1/PHASE2	SUSPENDED	Luspatercept and Lenalidomide (L2) in Lower-risk, Non-del(5q) MDS Patients
NCT05005182	PHASE2	TERMINATED	Luspatercept With or Without Hydroxyurea for the Treatment of Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis or Unclassifiable With Ring Sideroblasts
NCT05399732	PHASE2	COMPLETED	Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia
NCT01432717	PHASE1	COMPLETED	Study of ACE-536 in Healthy Postmenopausal Women

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).