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 approved Tregzi (Orca-T), a first-of-its-kind regulatory T cell-based immunotherapy, for adults with blood cancers undergoing matched donor stem cell transplants. Approval was based on the PRECISION-T trial, in which 78% of treated patients achieved chronic GVHD-free survival at one year versus 38.4% with standard transplant.
New CAR T-cell data show 100% MRD negativity in high-risk smoldering multiple myeloma, a novel CAR T approach for AML, and a new T-cell pathway, while a case report warns of a rare therapy-related complication.
Research estimates 300 million people worldwide live with rare diseases, yet only 5% of conditions have approved treatments. Patients face diagnostic delays of 4.7 to 8 years, with emerging AI and machine learning tools showing promise in accelerating diagnosis and drug development.
Cantargia reported full year 2025 financial results with net sales of SEK 316.7 million and cash of SEK 281.8 million. The company secured a SEK 124 million financing package to advance clinical development of nadunolimab, including a phase Ib study combining it with a RAS inhibitor in pancreatic cancer.
The FDA has cleared an IND for Amphista's novel BRD9 degrader AMX-883 for AML, approved Lantheus' new prostate cancer imaging agent Pylarify TruVu, and accepted Biofrontera's sNDA for Ameluz PDT in superficial basal cell carcinoma. These regulatory milestones span hematologic and solid tumor areas, addressing significant unmet medical needs.
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.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07780565 |
Study of Monzosertib In Patients With R/R AML Or High-Risk Myelodysplastic Syndrome |
NOT_YET_RECRUITING | PHASE1 |
| NCT07780552 |
CHIP in Health and Disease |
NOT_YET_RECRUITING | |
| NCT07763054 |
Low-dose Liposomal Amphotericin B for Antifungal Prophylaxis in Prolonged Neutropenia Patients |
RECRUITING | |
| NCT07758218 |
Allogeneic iNKT Cell Therapy (agenT-797) After SCT |
NOT_YET_RECRUITING | PHASE1 |
| NCT07737769 |
Arsenic Trioxide to Prevent Relapse in Patients Undergoing Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia or Myelodysplastic Syndrome |
NOT_YET_RECRUITING | PHASE1 |
| 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 |
RECRUITING | PHASE2 |
| NCT07579429 |
Research On Nicotinamide Riboside Supplement Support in MDS (ROSS Trial) |
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 |
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 |
