Reblozyl (luspatercept-aamt) / BMS, Merck (MSD) - LARVOL DELTA

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 (clinicaltrials.gov) - P4 | N=60 | Active, not recruiting | Sponsor: Bristol-Myers Squibb | Recruiting ➔ Active, not recruiting

Enrollment closed • Anemia • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • SF3B1

AZA+Lus VS AZA Monotherapy in HR-MDS (clinicaltrials.gov) - P3 | N=86 | Not yet recruiting | Sponsor: Peking Union Medical College Hospital

Monotherapy • New P3 trial • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Managing Myelofibrosis: Matching Advances in Treatments With Clinical Unmet Needs. (PubMed, Hematol Oncol) - "The janus kinase inhibitor (JAKi) ruxolitinib has been the mainstay of treatment for over a decade...Other JAKi (pacritinib, momelotinib, jaktinib) address treatment-related cytopenia, expanding the therapeutic utility of this class of agents to patients with baseline anemia or thrombocytopenia. Novel candidates exploit multiple molecular pathways, and offer the potential to improve the management of MF-associated cytopenia (imetelstat, pelabresib, navitoclax, selinexor, luspatercept, sotatercept, elritercept, LCL161, bomedemstat) and recover bone marrow fibrosis (imetelstat, pelabresib, navitoclax and bomedemstat). It remains to be seen if these newer agents can induce any remission in MF and enable patients to come off therapy, but the future is beginning to look much brighter."

Journal • Review • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Thrombocytopenia

Imatinib-Induced Clinical Response in ETV6::ACSL6 Myeloid Neoplasm with Eosinophilic Pneumonitis: A Case Report. (PubMed, Am J Case Rep) - "CASE REPORT We report a rare case of a 71-year-old man with a history of myelodysplastic syndrome/neoplasms (MDS) with mutated [em]SF3B1[/em] and multilineage dysplasia treated with luspatercept followed by azacitidine. CONCLUSIONS Our findings suggest that imatinib should be considered for patients with a myeloid neoplasm with an [em]ETV6::ACSL6[/em] fusion who are refractory to corticosteroids. Further molecular investigations are needed to elucidate the underlying mechanism of imatinib sensitivity in [em]ETV6::ASCL6[/em]-associated disease, given the absence of genetic involvement of a tyrosine kinase."

Journal • Eosinophilia • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Pneumonia • Respiratory Diseases • Transplantation • ETV6 • FIP1L1 • PDGFRB • SF3B1

LUSPA001: 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 (clinicaltrials.gov) - P=N/A | N=140 | Active, not recruiting | Sponsor: Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia

New trial • Beta-Thalassemia • Genetic Disorders

STIMULATED INTO A HEART FAILURE EXACERBATION - Drew Faturos (ACC 2025) - "She continued to take her daily furosemide with no missed doses. A recent safety analysis of luspatercept from Italy shows hypertension, acute heart failure, and atrial fibrillation are the most serious adverse effects of therapy. Our case builds on this data and is the first to describe the development of heart failure exacerbation after initiation of luspatercept with a clear dose response of blood volume expansion and symptom onset. As a result, caution is needed when luspatercept is used in patients with heart failure who are exquisitely sensitive to volume changes."

Atrial Fibrillation • Cardiovascular • Congestive Heart Failure • Fatigue • Heart Failure • Hematological Disorders • Hematological Malignancies • Hypertension • Musculoskeletal Diseases • Myelodysplastic Syndrome • Oncology • Orthopedics • Pulmonary Disease

Oral Arsenic (ATO) in Low-risk Myelodysplastic Syndromes (MDS) (clinicaltrials.gov) - P1 | N=24 | Not yet recruiting | Sponsor: Groupe Francophone des Myelodysplasies | Trial completion date: Mar 2027 ➔ Jun 2027 | Trial primary completion date: Mar 2026 ➔ Jun 2026

Trial completion date • Trial primary completion date • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

LUSPATERCEPT TREATMENT FOR ERYTHROID POOR GRAFT FUNCTION OCCURRING POST HEMATOPOIETIC STEM CELL TRANSPLANTATION (EBMT 2025) - "Luspatercept can effectively increase hemoglobin levels in patients with erythroid poor graft function after HSCT, and further prospective randomized controlled studies is needed."

Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Myeloid Leukemia • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Leukemia • Lymphoma • Myelodysplastic Syndrome • Myelofibrosis • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • Transplantation

LUSPATERCEPT TREATMENT FOR ERYTHROID POOR GRAFT FUNCTION OCCURRING POST HEMATOPOIETIC STEM CELL TRANSPLANTATION (EBMT 2025) - "Luspatercept can effectively increase hemoglobin levels in patients with erythroid poor graft function after HSCT, and further prospective randomized controlled studies is needed."

Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Myeloid Leukemia • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Leukemia • Lymphoma • Myelodysplastic Syndrome • Myelofibrosis • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • Transplantation

EFFECT OF LUSPATERCEPT ON THE TREATMENT OF ANEMIA IN ALLO-HSCT FOR PATIENTS WITH HEMATOLOGICAL DISEASES (EBMT 2025) - "Luspatercept is an efficacious and safe treatment option for patients with anemia after allo-HSCT."

Clinical • Anemia • Bone Marrow Transplantation • Hematological Disorders

EFFECT OF LUSPATERCEPT ON THE TREATMENT OF ANEMIA IN ALLO-HSCT FOR PATIENTS WITH HEMATOLOGICAL DISEASES (EBMT 2025) - "Luspatercept is an efficacious and safe treatment option for patients with anemia after allo-HSCT."

Clinical • Anemia • Bone Marrow Transplantation • Hematological Disorders

A challenging convergence of conditions in a patient with thalassemia major presenting with thymoma and lymphangioleiomyomatosis: a case report. (PubMed, J Med Case Rep) - "This is the first reported case of the coincidental discovery of thymoma and lymphangioleiomyomatosis in a patient with β-thalassemia major. This case emphasizes the importance of thorough radiologic evaluations in patients with β-thalassemia to detect potential neoplastic conditions early. Enhanced awareness among clinicians and radiologists is crucial for the timely diagnosis and management of these patients."

Journal • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Interstitial Lung Disease • Oncology • Pulmonary Disease • Respiratory Diseases • Solid Tumor • Thymoma • Thymus Cancer

ODYSSEY: Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis (clinicaltrials.gov) - P1/2 | N=56 | Recruiting | Sponsor: GlaxoSmithKline | Not yet recruiting ➔ Recruiting | Phase classification: P2 ➔ P1/2

Enrollment open • Phase classification • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytosis

Low-risk MDS-A spotlight on precision medicine for SF3B1-mutated patients. (PubMed, Hemasphere) - "Novel agents such as Luspatercept and Imetelstat have shown promise, but their availability remains restricted and their long-term efficacy is to be investigated. Emerging strategies targeting metabolic mis-splicing (e.g., COASY) with vitamin B5, pyruvate kinase activators, and inhibitors of oncogenic pathways like MYC and BCL-2 represent potential future avenues for treatment, but their clinical utility remains to be fully explored. The current limitations in treatment underscore the urgency of developing novel, more effective therapies for patients with SF3B1-mutated MDS."

IO biomarker • Journal • Review • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • BCL2 • SF3B1

INDEPENDENCE: 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 (clinicaltrials.gov) - P3 | N=309 | Active, not recruiting | Sponsor: Celgene | Trial completion date: Feb 2029 ➔ Aug 2032

Trial completion date • Anemia • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology

LUSPAREAL001: To Evaluate Long- Term Safety and Efficacy of Luspatercept (clinicaltrials.gov) - P=N/A | N=350 | Active, not recruiting | Sponsor: Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia

New trial • Beta-Thalassemia • Genetic Disorders

Emerging Pathogenetic Mechanisms and New Drugs for Anemia in Myelofibrosis and Myelodysplastic Syndromes. (PubMed, Am J Hematol) - "These include TGF-β ligand traps (luspatercept, elritercept), activin A receptor type 1 (ACVR1)/activin receptor-like kinase 2 (ALK2) inhibitors (momelotinib, zilurgisertib), and anti-hemojuvelin antibody-based therapies (DISC-0974). Luspatercept and momelotinib are approved for anemia related to lower-risk MDS and MF, respectively, and represent an important addition to the treatment armamentarium, along with imetelstat, a telomerase inhibitor, recently ratified for anemia in lower-risk MDS. A promising strategy to overcome the limitations of existing anemia-directed therapies includes the use of drug combinations with complementary mechanisms (luspatercept + erythropoiesis stimulating agents, luspatercept + momelotinib, DISC-0974 + momelotinib), and harnessing the erythropoietic potential of sodium-glucose co-transporter-2 inhibitors (SGLT-2I). Future research should address the complex pathophysiology of anemia, standardize definitions for anemia with gender-specified..."

Journal • Review • Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Myelofibrosis • Oncology • ACVR1 • SMAD2 • SMAD4 • TGFB1

Real world efficacy of luspatercept in patients with lower-risk myelodysplastic syndromes/neoplasms (MDS); a single center study in a heavily pretreated cohort. (PubMed, Leuk Lymphoma) - "Eleven percent progressed to HR-MDS or AML. Median overall survival was not reached and did not differ between responders and nonresponders (p=NS)."

Journal • Real-world evidence • Acute Myelogenous Leukemia • Cardiovascular • Fatigue • Hematological Disorders • Hematological Malignancies • Hypertension • Myelodysplastic Syndrome • Oncology

Long-Term Experience with Luspatercept in Relapsed/Refractory Myelodysplastic Neoplasms: A Chinese Real-World Study. (PubMed, Adv Ther) - "Luspatercept is proved efficacious and well tolerated in relapsed/refractory LR-MDS and appears to be beneficial in reducing disease progression and prolonging survival."

Journal • Real-world evidence • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • Respiratory Diseases

A Study to Evaluate Luspatercept Treatment Patterns and Outcomes in Erythropoiesis-Stimulating Agents-Naïve Patients With Lower-Risk Myelodysplastic Syndromes in the United States (clinicaltrials.gov) - P=N/A | N=400 | Active, not recruiting | Sponsor: Bristol-Myers Squibb

New trial • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Safety and efficacy of luspatercept in treating anemia associated with myelodysplastic syndrome with ring sideroblasts in Asian patients who require red blood cell transfusions: a phase II bridging study. (PubMed, Ther Adv Hematol) - P2 | "These results support luspatercept as a well-tolerated, efficacious alternative to transfusions for Asian patients with lower-risk MDS, who tend to have more severe anemia. Trial registration: clinicaltrials.gov, NCT04477850."

Journal • P2 data • Acute Myelogenous Leukemia • Anemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

A treatment with luspatercept for patients with low-risk myelodysplastic syndrome (JSMO 2025) - No abstract available

Clinical • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Bristol Myers Squibb Reports Fourth Quarter and Full-Year Financial Results for 2024 (Businesswire) - "Growth Portfolio worldwide revenues increased to $22.6 billion compared to $19.4 billion in the prior year period, representing growth of 17% on a reported basis, or 19% when adjusted for foreign exchange impacts. Growth Portfolio revenues were primarily driven by higher demand for Reblozyl, Breyanzi, Camzyos and Opdualag."

Commercial • Chronic Lymphocytic Leukemia • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Mantle Cell Lymphoma • Melanoma • Myelodysplastic Syndrome

What we now BELIEVE is achievable with luspatercept in transfusion-dependent β-thalassaemia. (PubMed, Lancet Haematol) - No abstract available

Journal • Beta-Thalassemia

Long-term efficacy and safety of luspatercept for the treatment of anaemia in patients with transfusion-dependent β-thalassaemia (BELIEVE): final results from a phase 3 randomised trial. (PubMed, Lancet Haematol) - P3 | "These long-term results affirm luspatercept's efficacy in addressing key unmet needs of patients with transfusion-dependent β-thalassaemia with a manageable safety profile."

Journal • P3 data • Anemia • Beta-Thalassemia • Hematological Disorders • Musculoskeletal Pain • Oncology • Pain