The FDA has approved Agios Pharmaceuticals' mitapivat (AQVESME) as the first oral treatment for anemia in thalassemia, reducing transfusion needs by 40-50%. Meanwhile, a clinical trial found a three-times-weekly dosing of vadadustat (Vafseo) is safe for kidney dialysis patients, potentially improving convenience.
Ruxolitinib remains the standard frontline therapy for intermediate-2 and high-risk myelofibrosis, with phase 3 and real-world data showing spleen and symptom benefits. Cytopenias, transfusion needs and resistance after 2 to 3 years continue to drive interest in new combinations.
The myelofibrosis therapeutic pipeline now includes over 40 candidates from more than 35 companies, with recent developments including Orphan Drug Designation for CK0804 and multiple Phase III trial initiations. Key clinical milestones include completed enrollment in Karyopharm's SENTRY trial and upcoming Phase I data from Incyte's combination therapy studies.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07752121 |
First-Line Luspatercept in Transfusion-Dependent Lower-Risk Myelodysplastic Neoplasms |
NOT_YET_RECRUITING | |
| NCT07725497 |
A Study of Luspatercept in People With Anemia |
RECRUITING | PHASE2 |
| NCT07681440 |
A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia |
TERMINATED | |
| NCT07636486 |
Luspatercept vs Epoetin in Treating Poor Erythroid Engraftment for Hematological Malignancies |
NOT_YET_RECRUITING | PHASE2/PHASE3 |
| NCT07215975 |
A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East |
RECRUITING | |
| NCT06424639 |
Luspatercept Plus CsA vs CsA for the Treatment of Newly Diagnosed Non-Transfusion-Dependent NSAA |
WITHDRAWN | PHASE4 |
| NCT06113302 |
A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS) |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT06045689 |
A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT05949684 |
ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT05664737 |
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 |
RECRUITING | PHASE2 |
