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- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Retrospective data, Review, Journal: Myelodysplastic Neoplasms (MDS) with Ring Sideroblasts or SF3B1 Mutations: The Improved Clinical Utility of World Health Organization and International Consensus Classification 2022 Definitions, a Single-Centre Retrospective Chart Review. (Pubmed Central) - Apr 26, 2024
In the MEDALIST trial and in an interim analysis of the COMMANDS trial, lower-risk MDS-RS patients had decreased transfusion dependency with luspatercept treatment...There were 29 (74.4%) patients with ?15% RS, 6 (15.4%) with 5 to 14% RS, one (2.6%) with 1% RS, and 3 (7.7%) with no RS. Our study suggests that a quarter of patients would be missed based on the morphologic criterion of only using RS greater than 15% and supports the revised 2022 definitions of the World Health Organization (WHO) and International Consensus Classification (ICC), which shift toward molecularly defined subtypes of MDS and appropriate testing.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Clinical benefit of luspatercept treatment (tx) in transfusion-dependent (TD), erythropoiesis-stimulating agent (ESA) (Hall A; Poster Bd #: 124) - Apr 24, 2024 - Abstract #ASCO2024ASCO_1201;
P3
Our study suggests that a quarter of patients would be missed based on the morphologic criterion of only using RS greater than 15% and supports the revised 2022 definitions of the World Health Organization (WHO) and International Consensus Classification (ICC), which shift toward molecularly defined subtypes of MDS and appropriate testing. Pts were randomized 1:1 to subcutaneous administration of luspatercept (1.0
- Onureg (azacitidine oral) / BMS
Preliminary safety and efficacy of oral azacitidine (Oral-AZA) in patients (pts) with low-/Intermediate (Int)-risk myelodysplastic syndromes (MDS): Phase 2 results from the ASTREON trial. (E450b) - Apr 24, 2024 - Abstract #ASCO2024ASCO_1146;
P2/3
The safety of Oral-AZA 200 mg and 300 mg was consistent with the known Oral-AZA safety profile. Preliminary efficacy data support continued evaluation of Oral-AZA in LR-MDS.
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Preclinical, Journal: Bone marrow Tfr2 deletion improves the therapeutic efficacy of the activin-receptor ligand trap RAP-536 in ?-thalassemic mice. (Pubmed Central) - Apr 17, 2024
Notably, the lack of Tfr2, but not RAP-536, can also attenuate iron-overload and related complications. Overall, our results shed further light on the mechanism of action of Luspatercept and suggest that strategies aimed at inhibiting hematopoietic TFR2 might improve the therapeutic efficacy of activin receptor-ligand traps.
- |||||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Enrollment open: A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or ?-thalassemia (clinicaltrials.gov) - Apr 9, 2024
P=N/A, N=104, Recruiting, Sponsor: Bristol-Myers Squibb
Overall, our results shed further light on the mechanism of action of Luspatercept and suggest that strategies aimed at inhibiting hematopoietic TFR2 might improve the therapeutic efficacy of activin receptor-ligand traps. Not yet recruiting --> Recruiting
- Ojjaara (momelotinib) / GSK
Estimating the financial impact of introducing momelotinib as a treatment option for adult patients with intermediate- or high-risk myelofibrosis with anemia from a US commercial and Medicare payer perspective () - Apr 9, 2024 - Abstract #AMCP2024AMCP_62;
The introduction of MMB resulted in a small positive budget impact. The higher acquisition cost of MMB that drives the budget impact was partially offset by savings due to reduced transfusion-related costs.
- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Preclinical, Review, Journal: The evolution of preclinical models for myelodysplastic neoplasms. (Pubmed Central) - Apr 8, 2024
Genetically engineered mice utilize specific mutations and may not represent the entire array of human MDS; however, genetically engineered mice provided in vivo proof of principle for novel agents such as luspatercept, demonstrating the clinical utility of this approach. This review offers an overview of available preclinical MDS models and potential approaches to accelerate accurate clinical translation.
- | Talzenna (talazoparib) / Pfizer, Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Japanese regulatory, Journal: PMDA regulatory update on approval of new drugs and revisions of precautions; approval of talazoparib tosilate for prostate and breast cancer, and luspatercept for myelodysplastic syndrome in Japan. (Pubmed Central) - Mar 29, 2024
This review offers an overview of available preclinical MDS models and potential approaches to accelerate accurate clinical translation. No abstract available
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Enrollment open: The Tapering Dose of Luspatercept in Patients With Lower-risk Myelodysplastic Syndromes (clinicaltrials.gov) - Mar 12, 2024
P2, N=36, Recruiting, Sponsor: Institute of Hematology & Blood Diseases Hospital, China
No abstract available Not yet recruiting --> Recruiting
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Extramedullary Hematopoiesis Leading to Spinal Cord Compression (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_2227;
Cord compression from EMH has been reported in patients with thalassemia, but should be considered in other patients with myelopathic presentations who are at risk for EMH. Luspatercept could also be a contributing to EMH, and this is the first report leading to spinal cord compression to our knowledge.
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Biomarker, Trial completion date, Trial primary completion date: AIHA ITP CIN: Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia (clinicaltrials.gov) - Mar 7, 2024
P=N/A, N=200, Recruiting, Sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Luspatercept could also be a contributing to EMH, and this is the first report leading to spinal cord compression to our knowledge. Trial completion date: Jun 2026 --> Jun 2030 | Trial primary completion date: Sep 2023 --> Sep 2026
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Enrollment open: A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS) (clinicaltrials.gov) - Mar 4, 2024
P1, N=40, Recruiting, Sponsor: M.D. Anderson Cancer Center
Trial completion date: Jun 2026 --> Jun 2030 | Trial primary completion date: Sep 2023 --> Sep 2026 Not yet recruiting --> Recruiting
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal: Luspatercept: A peaceful revolution in the standard of care for myelodysplastic neoplasms. (Pubmed Central) - Mar 4, 2024
Not yet recruiting --> Recruiting No abstract available
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Retrospective data, Journal: Treatment patterns and outcomes with luspatercept in patients with lower-risk myelodysplastic syndromes: A retrospective US cohort analysis. (Pubmed Central) - Mar 4, 2024
No abstract available No abstract available
- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Trial completion date, Trial primary completion date: Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (?)-Thalassemia (clinicaltrials.gov) - Mar 2, 2024
P2, N=99, Recruiting, Sponsor: Celgene
No abstract available Trial completion date: Oct 2028 --> Nov 2026 | Trial primary completion date: Apr 2028 --> Jun 2026
- ||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Enrollment change: A Study to Determine the Efficacy and Safety of Luspatercept in Adult and Adolescent Participants With Alpha (?)-Thalassemia (clinicaltrials.gov) - Feb 28, 2024
P2, N=249, Recruiting, Sponsor: Bristol-Myers Squibb
Trial completion date: Oct 2028 --> Nov 2026 | Trial primary completion date: Apr 2028 --> Jun 2026 N=177 --> 249
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Trial completion date, Trial termination, Trial primary completion date: Luspatercept With or Without Hydroxyurea for the Treatment of Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis or Unclassifiable With Ring Sideroblasts (clinicaltrials.gov) - Feb 21, 2024
P2, N=3, Terminated, Sponsor: Mayo Clinic
N=177 --> 249 Trial completion date: May 2025 --> Jun 2023 | Active, not recruiting --> Terminated | Trial primary completion date: May 2024 --> Apr 2023; Slow accrual
- |||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Trial completion date, Trial primary completion date: LTFU: A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials (clinicaltrials.gov) - Feb 15, 2024
P3, N=665, Recruiting, Sponsor: Celgene
Trial completion date: May 2025 --> Jun 2023 | Active, not recruiting --> Terminated | Trial primary completion date: May 2024 --> Apr 2023; Slow accrual Trial completion date: Mar 2030 --> May 2028 | Trial primary completion date: Mar 2030 --> May 2028
- ||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Phase classification, Enrollment change, Trial completion date, Trial primary completion date: Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (?)-Thalassemia (clinicaltrials.gov) - Feb 15, 2024
P2, N=99, Recruiting, Sponsor: Celgene
Trial completion date: Mar 2030 --> May 2028 | Trial primary completion date: Mar 2030 --> May 2028 Phase classification: P2a --> P2 | N=54 --> 99 | Trial completion date: Nov 2026 --> Oct 2028 | Trial primary completion date: Jun 2026 --> Apr 2028
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
ANALYSIS OF THE EFFICACY OF LUSPATERCEPT IN THE TREATMENT OF ANEMIA AFTER ALLOGENEIC HAEMATOPOIETIC STEM CELL TRANSPLANTATION (ePoster area) - Feb 14, 2024 - Abstract #EBMT2024EBMT_949;
The effectiveness of luspatercept in treating anemia after a transplant is promising. However, it is important to note that the small sample size of this study warrants further research with a larger sample size.
- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Review, Journal, HEOR: Health-related quality of life with standard and curative therapies in thalassemia: A narrative literature review. (Pubmed Central) - Jan 25, 2024
Luspatercept showed clinically meaningful improvement in physical functioning among TDT and NTDT. Furthermore, HSCT and gene therapy were associated with better physical, emotional, and mental domains scores.
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Enrollment closed, Enrollment change: Luspatercept With or Without Hydroxyurea for the Treatment of Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis or Unclassifiable With Ring Sideroblasts (clinicaltrials.gov) - Jan 23, 2024
P2, N=3, Active, not recruiting, Sponsor: Mayo Clinic
Furthermore, HSCT and gene therapy were associated with better physical, emotional, and mental domains scores. Recruiting --> Active, not recruiting | N=33 --> 3
- || imetelstat (GRN163L) / Geron, Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Review, Journal: Phase to phase: Navigating drug combinations with hypomethylating agents in higher-risk MDS trials for optimal outcomes. (Pubmed Central) - Jan 22, 2024
Effective therapeutic strategies require accurate reporting of adverse events, highlighting the need for clarity in criteria like the Common Terminology Criteria for Adverse Events (CTCAE). We provide an overview on negative clinical trials in HR MDS, analyze possible reasons and explore possibilities to optimize future clinical trials in this challenging patient population.
- | RLYB331 / Rallybio, Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Preclinical, Journal: A human anti-matriptase-2 antibody limits iron overload, ?-globin aggregates and splenomegaly in ?-thalassemic mice. (Pubmed Central) - Jan 19, 2024
RAP-536L alone did not prevent iron overload but significantly reduced apoptosis in the erythroid populations of the bone marrow, normalized red blood cell counts and improved hemoglobin and hematocrit levels. Interestingly, the association of RLYB331 with RAP-536L entirely reversed the ?-thalassemia phenotype in Hbbth3/+ mice and simultaneously corrected iron overload, ineffective erythropoiesis, splenomegaly and hematological parameters, suggesting that a multi-functional molecule consisting of the fusion of RLYB331 with Luspatercept (human version of RAP-536L) would allow administration of a single medication addressing simultaneously the different pathophysiological aspects of ?-thalassemia.
- || Review, Journal: ACVR1: A Novel Therapeutic Target to Treat Anemia in Myelofibrosis. (Pubmed Central) - Jan 11, 2024
Luspatercept (ACVR2B ligand trap) is assessed in transfusion-dependent MF patients in a registrational phase 3 trial. Approved ACVR1 inhibitors and novel agents in development are poised to improve the outcomes of anemic MF patients.
- || Review, Journal: Treatment of anemia in myelofibrosis: focusing on Novel Therapeutic Options. (Pubmed Central) - Dec 11, 2023
Standard approaches to treat myelofibrosis-related anemia have limited efficacy and are associated with toxicity. New drugs have shown positive results in myelofibrosis-associated anemia when used alone or in combination.
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
New trial: Clinical Observation of Luspatercept in Treatment of Chinese Adult ?-thalassaemia Patients With TD ?-thalassemia (clinicaltrials.gov) - Dec 10, 2023
P=N/A, N=10, Recruiting, Sponsor: Hematology department of the 920th hospital
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal: Next-generation therapy for lower-risk MDS. (Pubmed Central) - Dec 9, 2023
pathway has led to the approval of luspatercept in LR-MDS with ring sideroblasts or SF3B1 mutation, potentially replacing first-line ESAs in this population. Here, we also discuss the evolving standard of care for the treatment of LR-MDS and explore some of the most promising next-generation agents under investigation.
- || vactosertib (TEW-7197) / National OncoVenture, Theragen Etex, Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Review, Journal, Metastases: Targeting transforming growth factor beta signaling in metastatic osteosarcoma. (Pubmed Central) - Nov 29, 2023
For instance, Luspatercept, a TGF-? ligand trap, has been approved by the FDA for the treatment of anemia associated with myeloid dysplastic syndrome (MDS) with ring sideroblasts/mutated
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Phase classification: MAXILUS: A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants (clinicaltrials.gov) - Nov 18, 2023
P3, N=100, Recruiting, Sponsor: Bristol-Myers Squibb
ligand trap, has been approved by the FDA for the treatment of anemia associated with myeloid dysplastic syndrome (MDS) with ring sideroblasts/mutated Phase classification: P3b --> P3
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal: The Evolving Landscape: Exploring the Future of Myelodysplastic Syndrome Treatment with Dr. Rami Komrokji. (Pubmed Central) - Nov 14, 2023
Clinical trials within homogeneous patient groups with similar molecular profiles are becoming more common, enhancing treatment precision. In conclusion, the future of MDS treatment is moving towards personalized medicine, leveraging advanced technologies like NGS and molecular insights to improve outcomes in the realm of hematological malignancies.
- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Phase classification: LTFU: A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials (clinicaltrials.gov) - Nov 14, 2023
P3, N=665, Recruiting, Sponsor: Celgene
In conclusion, the future of MDS treatment is moving towards personalized medicine, leveraging advanced technologies like NGS and molecular insights to improve outcomes in the realm of hematological malignancies. Phase classification: P3b --> P3
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal: Long-term utilization and benefit of luspatercept in transfusion-dependent, erythropoiesis-stimulating agent-refractory or -intolerant patients with lower-risk myelodysplastic syndromes with ring sideroblasts. (Pubmed Central) - Nov 6, 2023
Phase classification: P3b --> P3 No abstract available
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal: Recent findings in myelodysplastic syndrome (Pubmed Central) - Nov 5, 2023
No abstract available The IPSS-M also represents an important extension with regard to prognosis estimation for patients with therapy-related MDS.In 2020 Luspatercept has been approved for transfusion-dependent lower risk MDS patients harboring ring sideroblasts
- Aranesp (darbepoetin alfa) / Amgen, Kyowa Kirin, Ilaris (canakinumab) / Novartis
Phase 1b/2 Study Evaluating the Safety and Efficacy of Canakinumab with Darbepoetin Alfa in Patients with Lower-Risk Myelodysplastic Syndromes (MDS) Who Have Failed Erythropoietin Stimulatin... (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_6068;
Canakinumab inhibited inflammasome activation as evidenced by a reduction of ASC specks although this did not translate to clinical responses. Phase 2 of the study will focus on patients with lower transfusion burden and molecular subsets with highest IL-1
- Epogen (epoetin alfa) / Amgen, Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Patient-Reported Outcomes (PRO) of Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent (ESA)-Na (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_6044;
Luspatercept was also found to be well-tolerated by the majority of patients. All of these PRO findings further support the benefits of luspatercept in ESA-na
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Impact of Mutational Burden and IPSS-M on Response to ESAs in Lower Risk Myelodysplastic Neoplasms (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_6043;
The number of mutations did not correlate with ESA response, while IPSS-M score, which considers the prognostic weight of specific mutations, showed predictive significance. In the multivariate analysis IPSS-M score and TD were strong predictors of ESA response, irrespective of baseline sEPO, potentially defining at diagnosis LR-MDS for whom novel agents like luspatercept could be considered as first line therapeutic option.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Impact of Genomic Landscape and Mutational Burden on Primary Endpoint Responses in the COMMANDS Study (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_6038;
In both ITT and RS+ pts, luspatercept showed either superior or comparable clinical benefit in pts with LR-MDS with various BL MB. Interestingly, both luspatercept and ESAs had similar clinical benefit across various MB in the RS- subgroup.
- IOA-360 / iOnctura, IOA-359 / iOnctura
Preclinical Activity of Novel TGF Beta Receptor I Kinase Inhibitors IOA-359 and IOA-360 for Treatment of Anemia in MDS/AML (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_6036;
Furthermore, in selected samples, addition of Luspatercept led to greater maturation of erythrocytes. Our current results support the preclinical in vitro efficacy of ALK5 inhibitors IOA-359 and IOA-360 alone and in combination with Luspartercept, highlighting their potential for further development and clinical testing in MDS/AML.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Downregulation of Mitochondrial Complex II (MC II) in Myelodysplastic Syndromes (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_6030;
To understand whether SDH dysregulation alone can contribute to MDS pathobiology, we generated a doxycycline-inducible and reversible mouse model (SDH-KD) that expresses shRNAs against the SDH subunits (SDHA, B, and D) in HSCs. In conclusion, our results indicate that pseudohypoxia- and TGF-
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Clinical Parameters, Anemia, and Spleen Response in Patients with MF-Related Anemia Treated with Luspatercept: Efficacy Sub-Analysis from the ACE-536-MF-001 Study (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_6011;
P2
Spleen size stability was observed in all patients; this suggests that LUSPA does not worsen splenomegaly and may support continual use of RUX for MF disease and symptoms. Study support : Bristol Myers Squibb.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Efficacy and Safety of Luspatercept in Patients Enrolled in the BELIEVE Trial: Data from the Phase 3b Long-Term Rollover Study (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_5296;
P3
At data cutoff, treatment was ongoing for 138 patients, 90 in the luspatercept group and 48 in the crossover group. Median (range) total treatment duration over the combined studies was 229.1 (1.7
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Efficacy and Safety of Luspatercept in Patients with HbE/ (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_5295;
P3
Median (range) total treatment duration over the combined studies was 229.1 (1.7 The BELIEVE trial enrolled 336 pts, 52 (15.5%) of whom had HbE/
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Luspatercept for the Treatment of Anemia in Non-Transfusion-Dependent ?-Thalassemia: Final Safety and Efficacy Data from the BEYOND Trial (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_5294;
P2, P3
A total of 76/96 (79.2%) pts in the luspatercept arm completed 144 wk of treatment (tx), with 55/96 (57.3%) completing 192 wk of tx; 28/96 (29.2%) pts on luspatercept discontinued tx. The median (range) luspatercept tx duration was 202.8 (15.0
- vamifeport (VIT-2763) / , Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Combination Therapy with Luspatercept and the Ferroportin Inhibitor Vamifeport Is Superior to Either Drug Alone in Improving Anemia and Reducing Myeloid Skewing in MDS (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_5282;
Furthermore, vamifeport improves the myeloid skewing in the MDS model, suggesting disease-modifying activity as single agent as well as combined therapy with luspatercept. Together, these data prove that combo therapies aimed at restricting iron and boosting erythroid maturation may offer additive beneficial effects in MDS and provide pre-clinical evidence for combining iron restriction and TFG-
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Real-World Impact of Luspatercept on Patients with Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions and with Prior Exposure to Erythropoietin-Stimulating Agents: A United Stat... (Marriott Marquis - Marriott Grand) - Nov 3, 2023 - Abstract #ASH2023ASH_5082;
In this study, over half of pts achieved 8-week and 12-week TI status during the follow-up period, and more than a quarter did not require any RBCT over a 24-week period. Further, most pts treated with luspatercept did not require any additional MDS-related treatments during the follow-up period.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Long-Term Evaluation of Luspatercept in Erythropoiesis-Stimulating Agent (ESA)-Intolerant/Refractory Patients (pts) with Lower-Risk Myelodysplastic Syndromes (LR-MDS) in the Phase 3 MEDALIST... (Marriott Marquis - Marriott Grand) - Nov 3, 2023 - Abstract #ASH2023ASH_5081;
The long-term safety profile of luspatercept is consistent with previous shorter-term reports, and AEs were mostly lower grade with rates of fatigue decreasing with increasing luspatercept dose. These data demonstrate the long-term efficacy and safety of luspatercept in pts with LR-MDS intolerant/ineligible for ESA treatment.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Real-World Retrospective Study of Non-Transfusion Dependent Patients with Myelodysplastic Syndromes Treated with Luspatercept: A Healthcare Claims Database Study (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_4769;
Almost all patients with LR-MDS who were NTD at luspatercept initiation maintained 8-week NTD status during the 6 months after luspatercept initiation. The majority of patients did not receive a RBCT for a 24-week period after luspatercept initiation.
- etavopivat (NN7535) / Novo Nordisk
FORTITUDE: A Phase 2 Open-Label Study in Progress to Evaluate Etavopivat for the Treatment of Anemia in Patients with Lower-Risk Myelodysplastic Syndromes (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_4187;
P2
Erythropoiesis-stimulating agents and other drugs, including lenalidomide, luspatercept and imetelstat (an investigational product), can lead to RBC transfusion independence and improve Hb levels in many patients; however, responses are generally transient and novel treatments are needed for this population...Additional exclusion criteria include prior treatment with azacitidine; decitabine; erythropoietin, other hematopoietic growth factor treatment or lenalidomide within 30 days of Day 1 or anticipated to be required during the study; and luspatercept within 30 days of Day 1 for NTD patients and within 16 weeks of Day 1 for TD patients...Summary : Etavopivat is a novel, investigational, once-daily, selective PKR activator with the potential to improve RBC health and lifespan. This phase 2 study will assess the safety of etavopivat and its impact on Hb levels and RBC transfusion burden in patients with LR-MDS and anemia.
- DFV890 / Novartis
Safety and Preliminary Efficacy of DFV890 in Adult Patients with Myeloid Diseases: A Phase 1b Study (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_4186;
P1
This phase 2 study will assess the safety of etavopivat and its impact on Hb levels and RBC transfusion burden in patients with LR-MDS and anemia. Eligible pts must be 18 years of age, with an Eastern Cooperative Oncology Group performance status ?2, candidates for serial bone marrow assessments who are willing to undergo a bone marrow aspirate/biopsy during the trial, and meet one of the following: (a) IPSS-R