Myelodysplastic syndrome is a clonal myeloid neoplasm with ineffective blood cell production, cytopenias, and risk of progression to acute myeloid leukemia. It predominantly affects older adults.
The FDA granted Orphan Drug Designation to Cullinan Therapeutics’ CLN-049 for relapsed/refractory AML. CLN-049 is in Phase 1 studies and had already received Fast Track designation.
Senti Biosciences completed enrolment in its Phase I SENTI-202 trial in relapsed or refractory AML and said recent data showed deep, MRD-negative durable complete remissions. The company plans FDA talks in the first half of 2026 on a pivotal registration programme.
The 2026 Tandem Meetings featured new data on CAR-T, allogeneic transplantation, and supportive care. Highlights included early efficacy signals for EB-103, KITE-753, Orca-T, and NXC-201, plus comparative cardiovascular safety data for lisocabtagene maraleucel.
The clinical trials industry is expected to grow from $61.93 billion in 2025 to $99.25 billion by 2033 at a 6.07% CAGR, driven by demand for innovative therapies and chronic disease prevalence. Clinical trials management systems are projected to reach $9.0 billion by 2035.
Faron Pharmaceuticals announces Phase II BEAM-X trial evaluating bexmarilimab plus azacitidine in MRD-positive AML patients after stem cell transplantation. The Nordic AML Group-led trial will enroll 24 patients with first results expected 12-15 months after enrollment begins in Q3 2026.
The FDA has approved Filkri (filgrastim-laha), a biosimilar to Neupogen, for cancer patients at risk of infection due to chemotherapy and other indications. The approval marks Accord BioPharma's sixth biosimilar product.
The FDA approved 46 novel drugs in 2025, down from 50 in 2024. Small molecules accounted for 31 approvals (67%), with oncology leading at nine approvals. Large molecules contributed 15 approvals (33%), spanning ADCs, bispecifics, and subcutaneous delivery innovations.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07582991 |
Ustekinumab for Intestinal Behçet's Syndrome With Myelodysplastic Syndrome |
COMPLETED | PHASE2 |
| NCT07582172 |
Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome |
NOT_YET_RECRUITING | PHASE2 |
| NCT07579429 |
Research On Nicotinamide Riboside Supplement Support in MDS (ROSS Trial) |
NOT_YET_RECRUITING | PHASE2 |
| NCT07573111 |
Study of QUAIL-100 in Pediatric and Young Adult Participants With High-Risk Hematologic Malignancies Who Have Received a Hematopoietic Stem Cell Transplantation |
NOT_YET_RECRUITING | PHASE1 |
| NCT07463820 |
A Trial Comparing Three Different Treatment Options for Adults With Low-Risk Myelodysplasia and Anemia (A MyeloMATCH Treatment Trial) |
NOT_YET_RECRUITING | PHASE2 |
| NCT07455500 |
Study of an Innovative Therapy Using CAR-T Cells Targeting IL-1RAP in Patients With High-Risk Myelodysplastic Syndromes (MDS |
NOT_YET_RECRUITING | NA |
| NCT07422480 |
A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions |
RECRUITING | PHASE3 |
| NCT07347418 |
CD64 CAR T Cell Therapy in Adults With Relapsed and/or Refractory AML or HR-MDS |
NOT_YET_RECRUITING | PHASE1 |
| NCT07346105 |
Orca-T Expanded Access Program Study for Patients With Advanced Hematologic Malignancies |
TEMPORARILY_NOT_AVAILABLE | |
| NCT07285044 |
The Cancer Connected Access and Remote Expertise Beyond Walls Program to Provide In-Home Cancer Treatment and Improve Treatment Satisfaction in Cancer Patients Living in the Florida Panhandle and Surrounding Areas |
RECRUITING | PHASE2 |
