Ziftomenib is currently being evaluated across numerous clinical trials spanning Phase 1 through Phase 3 for acute myeloid leukemia—particularly NPM1-mutated and KMT2A-rearranged subtypes—and gastrointestinal stromal tumors. Encompassing actively recruiting, not yet recruiting, and active non-recruiting protocols, these studies evaluate ziftomenib as both monotherapy and combination therapy in frontline and relapsed/refractory settings. Sponsored by pharmaceutical developers such as Kura Oncology and Kyowa Kirin alongside academic institutions like M.D. Anderson Cancer Center and Massachusetts General Hospital, these trials investigate the drug's safety, pharmacokinetics, and therapeutic efficacy.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT07007312 | RECRUITING | Acute Myeloid Leukemia (AML) |
Kura Oncology, Inc. |
2025-09-26 | PHASE3 |
| NCT05735184 | RECRUITING | Acute Myeloid Leukemia; Mixed Lineage Leukemia Gene Mutation; Refractory AML; AML With Mutated NPM1; Acute Myeloid Leukemia Recurrent; Acute Myeloid Leukemia, in Relapse; NPM1 Mutation; KMT2Ar; Myeloid Sarcoma; Nucleophosmin 1-mutated Acute Myeloid Leukemia |
Kura Oncology, Inc. |
2023-07-18 | PHASE1 |
| NCT06448013 | RECRUITING | Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2025-03-07 | PHASE1 |
| NCT06397027 | RECRUITING | Refractory Acute Leukemia; Pediatric Relapsed |
M.D. Anderson Cancer Center |
2024-12-27 | PHASE1 |
| NCT05848687 | RECRUITING | Lymphoblastic Leukemia |
Tanja Andrea Gruber |
2023-11-03 | PHASE1; PHASE2 |
| NCT04067336 | ACTIVE_NOT_RECRUITING | Advanced Malignant Neoplasm; Acute Myeloid Leukemia; Mixed Lineage Leukemia; Mixed Lineage Acute Leukemia; Acute Leukemia of Ambiguous Lineage; Mixed Phenotype Acute Leukemia; Acute Lymphoblastic Leukemia |
Kura Oncology, Inc. |
2019-09-12 | PHASE1; PHASE2 |
| NCT06769490 | RECRUITING | Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2025-07-10 | PHASE1 |
| NCT06930352 | RECRUITING | Acute Myeloid Leukemia |
Uma Borate |
2026-10-01 | PHASE2 |
| NCT06655246 | RECRUITING | Gastrointestinal Stromal Tumor (GIST); Gastrointestinal Stromal Cancer; Gastrointestinal Stromal Neoplasm; Gastrointestinal Stromal Tumor, Malignant; Gastrointestinal Stromal Cell Tumors |
Kura Oncology, Inc. |
2025-03-27 | PHASE1 |
| NCT07355335 | NOT_YET_RECRUITING | KMT2A-rearranged; NPM1-mutant Refractory or Relapsed AML |
Massachusetts General Hospital |
2026-07-09 | PHASE1 |
| NCT07623616 | RECRUITING | Acute Myeloid Leukemia (AML) |
Kyowa Kirin Co., Ltd. |
2026-04-23 | PHASE2 |
| NCT07411586 | ACTIVE_NOT_RECRUITING | Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2026-05-28 | PHASE1 |
| NCT06376162 | RECRUITING | Relapsed/Refractory KMT2A-r Acute Leukemia; Relapsed/Refractory NUP98-r Acute Leukemia; Relapsed/Refractory NPM1-m Acute Leukemia |
PedAL BCU, LLC |
2025-03-18 | PHASE1 |
| NCT06001788 | RECRUITING | AML; AML With Mutated NPM1; Hematologic Malignancy; KMT2Ar; NPM1 Mutation; MLL Rearrangement; Leukemia; Acute Myeloid Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Acute Leukemia; Neoplasms by Histologic Type |
Kura Oncology, Inc. |
2024-02-22 | PHASE1 |
| NCT06440135 | RECRUITING | Acute Myeloid Leukemia; Acute Myeloid Leukemia in Remission; NPM1 Mutation; KMT2A Rearrangement |
Massachusetts General Hospital |
2024-06-11 | PHASE1 |
(data from https://clinicaltrials.gov, updated on 2026-08-20)
Check the
Ziftomenib (KO-539)
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
Appendix RUO and cGMP Quality Standards
| Quality Dimension | RUO (Research Use Only) | cGMP (Current Good Manufacturing Practice) |
|---|---|---|
| Clinical Applicability | Prohibited in human clinical trials or medical diagnostics. | Mandatory for human clinical trials (Phase I–III) and therapies. |
| Regulatory Status | Non-regulated grade; exempt from drug manufacturing laws. | Legally enforced by health authorities (e.g., FDA, EMA, NMPA). |
| Facility Environment | Unclassified analytical or research laboratories. | Validated Cleanrooms (ISO Class 5–8) with continuous monitoring. |
| Quality Control | Basic purity and activity testing. | Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma). |
| Process Validation | Basic equipment calibration; no process validation required. | Full qualification (IQ/OQ/PQ) and complete batch records. |
| Quality Assurance | Vendor self-declared without required formal QMS. | Mandatory QA/QC unit, Change Control, CAPA, and vendor audits. |
| Regulatory Impact | High risk of IND rejection if used as a critical raw material. | Required for IND/NDA filings, supported by Drug Master Files (DMF). |
Footnotes
Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).
- Ziftomenib (KO-539) Mechanism of Action: Menin Inhibition in Leukemic Blasts Research
- Ziftomenib (KO-539) Solubility in DMSO
- Ziftomenib (KO-539) Stock Solution
- Ziftomenib (KO-539) Storage
- Ziftomenib (KO-539) Stability
- Ziftomenib (KO-539) Molecular Weight
- Ziftomenib (KO-539) CAS Number
- Ziftomenib (KO-539) Chemical Structure (2D and 3D)
- Ziftomenib (KO-539) MedChemExpress (HY-132001)? Try a better option.
- Ziftomenib (KO-539) Sigma (TA9H98DCB3CD)? Try a better option.
- Ziftomenib (KO-539) Cayman (45139)? Try a better option.
- Ziftomenib (KO-539) Abmole (M13430)? Try a better option.
- Ziftomenib (KO-539) SDS｜MSDS
Note: Technical data last updated: Sep 1, 2026.