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ACE-536
ACE-536 is an activin receptor ligand trap that inhibits activin signaling to promote erythropoiesis and increase hemoglobin levels.
ACE-536 is an activin receptor ligand trap that inhibits activin signaling to promote erythropoiesis and increase hemoglobin levels. Used for Beta-thalassemia, Myelodysplastic syndromes (MDS).
At a glance
| Generic name | ACE-536 |
|---|---|
| Also known as | Luspatercept, BMS-986346 |
| Sponsor | Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA |
| Drug class | Activin receptor ligand trap |
| Target | Activin type II receptor |
| Modality | Biologic |
| Therapeutic area | Hematology |
| Phase | Phase 3 |
Mechanism of action
ACE-536 binds to activin type II receptors, blocking activin signaling which normally suppresses red blood cell production. By inhibiting this negative regulator of erythropoiesis, the drug stimulates the bone marrow to produce more red blood cells, thereby increasing hemoglobin and reducing anemia. This mechanism is distinct from traditional erythropoiesis-stimulating agents (ESAs) as it works through a different pathway.
Approved indications
- Beta-thalassemia
- Myelodysplastic syndromes (MDS)
Common side effects
- Headache
- Fatigue
- Bone pain
- Thrombotic events
Key clinical trials
- A Trial Comparing Three Different Treatment Options for Adults With Low-Risk Myelodysplasia and Anemia (A MyeloMATCH Treatment Trial) (PHASE2)
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
- Luspatercept for Clonal Cytopenias of Uncertain Significance (PHASE2)
- 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 (PHASE2)
- ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions (PHASE3)
- Luspatercept + Darbepoetin in MDS (PHASE2)
- A Study of Incidence, Treatment Patterns, and Outcomes in Transfusion-dependent Lower-risk Myelodysplastic Syndromes in Spain
- Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
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