Acute leukemia is a rapidly progressing cancer that starts in blood-forming tissue such as bone marrow and leads to large numbers of abnormal white blood cells entering the bloodstream. It generally includes acute lymphoblastic and acute myeloid leukemia subtypes and requires urgent treatment.
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.
ALX Oncology has multiple 2026 catalysts for Evorpacept and ALX2004. Interim ASPEN-09 data are expected in Q3 2026, while an initial safety update from the phase 1 ALX2004 trial is expected in 1H 2026.
Two Nature Chemical Biology studies described new routes to molecular glue discovery. One identified ZZ1 and YPEL5-linked CTLH recruitment, while another found an ENL degrader in leukemia cells.
Syndax Pharmaceuticals announced a collaboration to expand global access to Revuforj while reporting strong commercial performance for its two marketed products, with Revuforj generating $125 million and Niktimvo $152 million in the first 11 months of sales.
Researchers unveil tumor-intrinsic role of PD-L1 in lung cancer progression and develop systematic method for discovering molecular glues that selectively degrade disease proteins, while advances in immunology research aim to improve care for immune-mediated inflammatory diseases.
Researchers developed a high-throughput method to systematically discover molecular glues that trigger degradation of disease-causing proteins, demonstrating efficacy against a leukemia-associated protein and transforming drug discovery from chance to scalable workflow.
Researchers have identified how the MYC protein helps pancreatic cancer evade immune detection, while a separate team discovered methods to permanently switch off cancer genes in leukemia through epigenetic therapy targeting Menin and DOT1L proteins.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| 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 |
| NCT07565220 |
Thiotepa-based Conditioning Regimen With De-escalated Post-graft Cyclophosphamide for Allogeneic Stem Cell Transplantation in Hematologic Malignancies |
NOT_YET_RECRUITING | PHASE1 |
| NCT07559695 |
A Real-world Study on the Efficacy and Safety of Menin Inhibitors as Maintenance After Allo-HSCT |
RECRUITING | |
| NCT07499765 |
Benefits of Formalized Care Support Program Following Acute Leukemia Diagnosis |
RECRUITING | NA |
| NCT07493538 |
MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases |
RECRUITING | PHASE2 |
| NCT07445984 |
Chemogenomic Profiling in Hematological Malignancies (HEM-Profiling 2021) |
RECRUITING | NA |
| NCT07445438 |
Feasibility of a Multi-omics Platform for Hematological Malignancies |
RECRUITING | NA |
| NCT07403630 |
INTEGRATIVE "MULTI-OMICS" AND FUNCTIONAL PLATFORM FOR THE COMPLETE DIAGNOSTIC CHARACTERIZATION OF TUMORS: THE ITALIAN TUMOR CHEMOGENOMIC PROFILER (IT-TCP) |
RECRUITING | NA |
| NCT07383233 |
CD19/BAFF-R in Vivo CAR-T Cell Therapy Targeting Relapsed/Refractory B Cell Acute Leukemia/Malignant Lymphoma |
NOT_YET_RECRUITING | PHASE1 |
| NCT07362095 |
Luspatercept for the Treatment of Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation(Allo-HSCT) |
RECRUITING | PHASE1/PHASE2 |
