Also known as: Ara-C, busulfan
Cytarabine (cytosine arabinoside, ara-C) is an antimetabolite nucleoside-analog chemotherapy used in leukemias and lymphomas. It is administered via injectable routes, including intravenous and intrathecal use. It is a long-standing oncology medicine and is listed as prescription-only.
Recent studies using FDA Adverse Event Reporting System data have identified drug-associated cardiovascular risks, pediatric dry eye, and venlafaxine adverse effects. Findings highlight the importance of pharmacovigilance monitoring.
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.
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.
Moleculin Biotech announced a preliminary blinded composite complete remission rate of 40% in the first 30 subjects of its Phase 2B/3 MIRACLE trial testing Annamycin plus cytarabine for relapsed or refractory acute myeloid leukemia.
The FDA has accepted a new drug application for tirabrutinib, a BTK inhibitor, for relapsed/refractory primary central nervous system lymphoma under the accelerated approval pathway. The regulatory agency set a PDUFA target action date of December 18, 2026.
Moleculin Biotech announced the Japan Patent Office has issued a notice of allowance for a patent covering proprietary methods for reconstituting liposomal Annamycin, strengthening the company's international intellectual property position.
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 |
|---|---|---|---|
| NCT07604233 |
Phase 1/2 FLAG-IDA, VEN and Asciminib in CML and Ph+ AML |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT07537257 |
VEN+IDAC vs IDAC in AML |
ENROLLING_BY_INVITATION | PHASE2/PHASE3 |
| NCT07507760 |
Oral Decitabine Plus Ivosidenib as First Line for Older/Unfit Adult AML Patients |
NOT_YET_RECRUITING | PHASE2 |
| NCT07498465 |
A Study to Find the Highest Dose of SNDX-5613 (Revumenib) as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, and Mixed Phenotype Acute Leukemia |
WITHDRAWN | PHASE1 |
| NCT07490288 |
Venetoclax, Azacitidine and Liposomal Mitoxantrone for Newly Diagnosed AML |
NOT_YET_RECRUITING | NA |
| NCT07486479 |
Venetoclax, Azacitidine, and Mitoxantrone Hydrochloride Liposome Versus Idarubicin and Cytarabine in Newly Diagnosed AML |
NOT_YET_RECRUITING | PHASE3 |
| NCT07444710 |
Testing the Addition of an Anti-Cancer Drug, Glofitamab, to the Usual Chemotherapy Treatment (Alternating R-CHOP/R-DHAP) for Previously Untreated Mantle Cell Lymphoma |
NOT_YET_RECRUITING | PHASE1 |
| NCT07441967 |
The Safety and Efficacy of the Regimen of Thiotepa + Fludarabine +Granulocyte Colony-Stimulating Factor+ Cytarabine + Busulfan in Single Unrelated Umbilical Cord Blood Transplantation |
NOT_YET_RECRUITING | PHASE2 |
| NCT07428486 |
A Phase 1 Study Of FLAG Chemotherapy In Combination With Lisaftoclax And Pelcitoclax In Patients With Relapsed/Refractory T-Cell Acute Lymphoblastic Leukemia |
WITHDRAWN | PHASE1 |
| NCT07301138 |
BEBT-908 Plus Chemotherapy Treatment for CR MRD-Positive MEF2D-Rearranged and Pre-B Acute Lymphoblastic Leukemia Patients |
NOT_YET_RECRUITING | PHASE2 |
