Acute myeloid leukemia (AML) is a heterogeneous blood cancer arising from clonal expansion of myeloid precursors in bone marrow, causing marrow failure and circulating blasts. It is also called acute myelogenous leukemia and requires rapid specialist treatment.
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.
Two separate clinical trials have reported advances in cancer care. An oral drug combination of decitabine-cedazuridine and venetoclax showed efficacy in older patients with acute myeloid leukemia, with a 47% complete response rate in a phase 2b trial. A separate randomized controlled trial found that delaying pegfilgrastim administration to 72 hours post-chemotherapy significantly reduced bone pain in women with stage I–III breast cancer.
Moleculin Biotech's Phase 2B/3 MIRACLE trial of Annamycin plus cytarabine in relapsed or refractory acute myeloid leukemia reported a preliminary complete remission rate of approximately 30% and composite remission rate exceeding 40% in the first 45 subjects. The blinded data show a substantial improvement over historical rates with cytarabine alone. The trial is expected to reach its first unblinding milestone in mid-2026.
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 FDA approved oral decitabine and cedazuridine tablets plus venetoclax for newly diagnosed AML in patients 75 and older or those ineligible for intensive induction chemotherapy. The approval was supported by phase 1/2 ASTX727-07 data showing a 41.6% complete response rate.
Moleculin said its MIRACLE Phase 2B/3 AML trial is nearing first unblinding, with blinded CRc above 40% and CR around 30%. The first 45-subject readout is expected before June 30, 2026.
Kyowa Kirin and Kura Oncology started a Japanese Phase II registrational study of ziftomenib in adults with relapsed or refractory NPM1-mutated AML. Kyowa Kirin plans to seek regulatory approval in Japan after the trial.
Omeros reported initial nonhuman primate data for OncotoX-AML in acute myeloid leukemia. One treatment course reduced myeloid progenitor cells by up to 99% and was well tolerated.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07587944 |
Biomarkers in Bone Marrow Supernatant for Predicting AML Chemosensitivity |
RECRUITING | |
| NCT07539818 |
Azacitidine + Venetoclax VS Azacitidineas Maintenance Therapy in AML |
NOT_YET_RECRUITING | PHASE3 |
| NCT07516353 |
my.naviGATE: A Guide to After-Treatment Effects for Adolescents and Young Adults |
NOT_YET_RECRUITING | NA |
| NCT07508982 |
Phase1b/2 Trial Of AZA + APG1252 In Patients With High-Risk AML |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| 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 |
| NCT07490288 |
Venetoclax, Azacitidine and Liposomal Mitoxantrone for Newly Diagnosed AML |
NOT_YET_RECRUITING | NA |
| NCT07347171 |
A Phase 1 Study of CG009301 for Injection in Adult Subjects With Recurrent or Refractory Haematological Malignancies |
RECRUITING | PHASE1 |
| NCT07342244 |
Safety and Efficacy of CLL1 CAR-T Followed by Allogeneic Hematopoietic Stem Cell Transplantation in the Treatment of Relapsed/Refractory Acute Myeloid Leukemia |
NOT_YET_RECRUITING | NA |
| NCT07163728 |
Leptin: A Marker for AML Chemo-Sensitivity |
RECRUITING | |
| NCT07131085 |
QH101 Cell Therapy Relapsed/Refractory(R/R) Acute Myeloid Leukemia(AML) and Myelodysplastic Syndromes(MDS) |
NOT_YET_RECRUITING | PHASE1 |
