> Trialight public data. Index of pages: https://trialight.com/llms.txt
> Web page: https://trialight.com/data/asset/ziftomenib-e12216d9-eb96-486f-8b52-ea71142dd851
> Record changed: 2026-10-13

# Ziftomenib

Highest phase reached

Phase 1

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL5095038)

Drug type

Small molecule

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL5095038)

## Development status

**Listing at source**

Listed

**Highest phase reached**

Phase 1

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL5095038)

## Names and chemistry

**Drug type**

Small molecule

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL5095038)

**Drug name**

Ziftomenib

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL5095038)

**Molecular weight**

717.89

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL5095038)

**Structure (SMILES)**

CNc1nc(NC2CCN(Cc3ccc4c(cc(C#N)n4C\[C@H\](C)N4CCN(S(C)(=O)=O)CC4)c3C)CC2)c2cc(CC(F)(F)F)sc2n1

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL5095038)

### Other names (18)

- ko 539
- Ko 539
- Ko-539
- KO-539
- ko539
- KO539
- komzifti
- KOMZIFTI
- menin mixed lineage leukemia protein protein interaction inhibitor ko 539
- Menin-Mixed Lineage Leukemia Protein-Protein Interaction Inhibitor KO 539
- ziftomenib
- Ziftomenib
- Ziftomenib \[INN\]
- Ziftomenib \[USAN\]
- Ziftomenib \[WHO-DD\]
- ziftomenib inn
- ziftomenib usan
- ziftomenib who dd

## Identifiers

**CAS registry numbers**

- 2134675-36-6

**ChEMBL ID**

CHEMBL5095038

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL5095038)

**InChIKey**

BGGALFIXXQOTPY-NRFANRHFSA-N

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL5095038)

**PubChem compound IDs**

- 138497449

**RxNorm IDs (RxCUI)**

- 2725985

**FDA ingredient codes (UNII)**

- 4MOD1F4ENC

## Regulatory information

### Other regulatory facts

**Patents**

8

**Designations**

7

**Earliest recorded loss of exclusivity**

16 Jul 2044

**First in class**

No

**Fast track**

Yes

**Breakthrough designation**

Yes

**Orphan designation**

No

**Priority review**

Yes

**Orphan exclusivity**

No

**Accelerated approval**

No

## Pharmacology

### Drug classification

**Pharmacology name**

ziftomenib

**Type**

Synthetic organic

**Drug name stem**

\-menib

**Approval recorded by Guide to Pharmacology**

Yes

### Investigated indications (4)

- **Condition**  
Leukemia, Myeloid, Acute

**Highest recorded phase**  
1

**Condition identifier**  
EFO:0000222

**Subject identifier**  
D015470
- **Condition**  
Leukemia

**Highest recorded phase**  
1

**Condition identifier**  
EFO:0000565

**Subject identifier**  
D007938
- **Condition**  
Leukemia, Lymphoid

**Highest recorded phase**  
1

**Condition identifier**  
EFO:0004289

**Subject identifier**  
D007945
- **Condition**  
Leukemia, Biphenotypic, Acute

**Highest recorded phase**  
1

**Condition identifier**  
MONDO:0019460

**Subject identifier**  
D015456

### Open Targets indications (10)

- **Condition identifier**  
EFO\_0000222

**Highest recorded phase**  
1
- **Condition identifier**  
MONDO\_0004643

**Highest recorded phase**  
1
- **Condition identifier**  
MONDO\_0004967

**Highest recorded phase**  
1
- **Condition identifier**  
MONDO\_0005059

**Highest recorded phase**  
1
- **Condition identifier**  
MONDO\_0010643

**Highest recorded phase**  
1
- **Condition identifier**  
MONDO\_0011719

**Highest recorded phase**  
1
- **Condition identifier**  
MONDO\_0015667
- **Condition identifier**  
MONDO\_0015667

**Highest recorded phase**  
1
- **Condition identifier**  
MONDO\_0018874

**Highest recorded phase**  
3
- **Condition identifier**  
MONDO\_0020743

**Highest recorded phase**  
1

### Trial references (5)

- NCT04067336  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04067336)
- NCT05848687  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05848687)
- NCT06376162  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06376162)
- NCT06440135  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06440135)
- NCT06448013  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06448013)

## Chemical properties

**Hydrogen bond acceptors**

11

**Hydrogen bond donors**

2

**Polar surface area**

122.42

**Calculated partition coefficient (ALogP)**

5.01

**Calculated partition coefficient (XLogP)**

5.6

**Prodrug**

No

**Chirality**

Single enantiomer

**Inorganic**

No

**Rule-of-five violations**

3

**Rotatable bonds**

10

**Heavy atoms**

49

**Molecular formula**

C33H42F3N9O2S2

**Monoisotopic mass**

717.28549845

**Natural product**

No

## National Drug Codes

- 84696-200

## Evidence counts

**Recorded assays**

60

**Active assays**

4

### Active assay types

- Confirmatory

## Related records

### Trials (16)

- A Phase 2 Clinical Study of Ziftomenib in Patients With Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia
- A Phase I Study Investigating the Combination of the Menin Inhibitor Ziftomenib With Venetoclax and Gemtuzumab in Pediatric Patients With Acute Myeloid Leukemia
- A Phase I Study Investigating the Combination of the Ziftomenib, Venetoclax and Azacitidine in Pediatric Relapsed and Refractory Acute Leukemias
- [A Study of Ziftomenib in Combination With Imatinib in Patients With Advanced Gastrointestinal Stromal Tumors (GIST)](https://trialight.com/data/trial/a-study-of-ziftomenib-in-combination-with-imatinib-in-31470af4-d3ca-43bc-bd8b-eeb7780e380d.md)
- [A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML](https://trialight.com/data/trial/a-study-to-investigate-the-safety-and-tolerability-of-3a4970a8-b886-418f-bda5-cb9655741f9c.md)
- Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia
- Expanded Access to Ziftomenib
- First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia
- Phase 1/1b Trial Of Olutasidenib And Ziftomenib For NPM1 And IDH1 Co-Mutated Acute Myeloid Leukemia
- [Safety and Tolerability of Ziftomenib Combinations in Patients With Relapsed/Refractory Acute Myeloid Leukemia](https://trialight.com/data/trial/safety-and-tolerability-of-ziftomenib-combinations-in-efd67cbe-6049-4b3f-831e-bee7792f10a1.md)
- [Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML](https://trialight.com/data/trial/studies-to-assess-ziftomenib-in-combination-with-ven-aza-or-91559fd5-f7c5-4cc5-b0d6-1628aace2632.md)
- TINI 2: Total Therapy for Infants With Acute Lymphoblastic Leukemia II
- Ziftomenib + Mezigdomide in Adolesc. and Adults w/ R/R AML
- Ziftomenib for the Treatment of Patients With NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia Not Eligible for Standard Therapy
- Ziftomenib in Combination With Chemotherapy for Children With Relapsed/Refractory Acute Leukemia
- Ziftomenib Maintenance Post Allo-HCT

### In development for (4)

- Acute leukemia of ambiguous lineage Phase 1
- [Acute myeloid leukemia](https://trialight.com/data/condition/acute-myeloid-leukemia-b0971a7a-c18d-544e-bd7f-b1a13b9caa30.md) Phase 1
- [Leukemia](https://trialight.com/data/condition/leukemia-519aefcd-8521-5941-851b-5b77ca925ca5.md) Phase 1
- Lymphoid leukemia Phase 1

### Other linked conditions (1)

- Acute myeloid leukemia by FAB classification

---

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