A scientist handling a rack of blue test tubes.

Pipeline

We are advancing a diverse pipeline of precision medicines for the treatment of blood cancers and solid tumors.

Our programs consist of small molecule drug candidates that target cancer signaling pathways, where each program builds on our expertise in precision medicines.

Program

Development Approach

Research

Dose Escalation

Dose Optimization

Registration-Enabling

Regulatory Submission

Clinical Trial

Menin Inhibitor

Ziftomenib**

Monotherapy

REGISTRATION DIRECTED

NPM1-mutant AML1

Monotherapy

DOSE-ESCALATION

KMT2A-rearranged ALL

Monotherapy

DOSE-ESCALATION

Non-NPM1-mutant / Non-KMT2A-rearranged AML

Combinations with venetoclax/azacitidine, and cytarabine + daunorubicin (7+3)

DOSE-ESCALATION

NPM1-mutant AML

Combinations with venetoclax/azacitidine, and cytarabine + daunorubicin (7+3)

DOSE-ESCALATION

KMT2A-rearranged AML

Combinations with gilteritinib, FLAG-IDA, LDAC

DOSE-ESCALATION

NPM1-mutant AML

Combinations with gilteritinib, FLAG-IDA, LDAC

DOSE-ESCALATION

KMT2A-rearranged AML

Combination with imatinib

DOSE-ESCALATION

Advanced gastrointestinal stromal tumors (GIST)

Combinations with venetoclax/azacitidine, cytarabine + daunorubicin (7+3)

REGISTRATION-ENABLING

NPM1-mutant AML2

Combinations with venetoclax/azacitidine, cytarabine + daunorubicin (7+3)

REGISTRATION-ENABLING

KMT2A-rearranged AML2

Ziftomenib

Ziftomenib is being investigated in up to 50% of AMLs, including NPM1-mutant AML and KMT2A-rearranged AML. It is also being investigated in combination with other cancer therapies.

**In Nov. 2024, Kura entered a global strategic collaboration agreement with Kyowa Kirin Co., Ltd. to develop and commercialize ziftomenib for AML and other hematologic malignancies.

View publications and presentations

Farnesyl Transferase Inhibitors

KO-2806

NEXT-GEN FTI

Monotherapy, combinations with cabozantinib and adagrasib

DOSE-ESCALATION

Solid tumors

Monotherapy, combinations with cabozantinib and adagrasib

DOSE-ESCALATION

Renal cell carcinoma (RCC)

Monotherapy, combinations with cabozantinib and adagrasib

DOSE-ESCALATION

KRASG12C-mutant non-small cell lung cancer (NSCLC), Colorectal cancer (CRC), Pancreatic ductal adenocarcinoma (PDAC)

KO-2806

KO-2806 is an investigational potent next-generation FTI designed to potentially improve upon earlier FTI drug candidates. KO-2806 is not FDA-approved and safety and efficacy have not been established.

View publications and presentations

Farnesyl Transferase Inhibitors

Tipifarnib

Combination with alpelisib

DOSE-VALIDATION

PIK3CA-dependent head and neck squamous cell carcinoma (HNSCC)3

Tipifarnib

Tipifarnib is an oral investigational FTI that is not FDA-approved. Safety and efficacy have not been established.

View publications and presentations

Next-Gen Menin Inhibitor

RESEARCH

Diabetes

The safety and efficacy of investigational agents and/or investigational uses of approved products have not been established.

FLAG-IDA: fludarabine, high-dose cytarabine (Ara-C), granulocyte-colony stimulating factor (G-CSF) and idarubicin; LDAC: Low-dose cytarabine

XOSPATA® (gilteritinib); PIQRAY® (alpelisib)

1 NDA accepted for filing with PDUFA date of November 30, 2025.
2 KOMET-017 program to advance directly to Registration-Enabling trials, leveraging data from Phase 1 combination trials.
3 Enrollment is complete.

ABOUT MENIN INHIBITORS

Most cancer drugs work by killing diseased cells faster than they kill healthy tissue. But what if you didn’t have to kill the cancer cell? What if you could convince it to differentiate? That’s the mechanism underlying an emerging class of targeted therapies called menin inhibitors.

Our menin inhibitor, ziftomenib, inhibits the interaction of two proteins, menin and KMT2A/MLL, that, when fused together, are responsible for survival and proliferation of certain kinds of leukemia cells. When this process is inhibited, cancerous juvenile cells are thought to mature into white blood cells.

ABOUT FARNESYL TRANSFERASE INHIBITORS

Imagine if a single key had the potential to unlock multiple targeted treatment paths for a range of difficult cancers. A class of medicines known as farnesyl transferase inhibitors may hold that ability.

Farnesyltransferase is an enzyme that plays a key regulatory role in cellular pathways that drive resistance to a range of targeted therapies in different types of tumors. By inhibiting the enzyme’s function, we believe we can reshape combination treatment options for many cancers.

Our investigational drug tipifarnib is a farnesyl transferase inhibitor, or FTI, that is being studied in a genetically defined subset of head and neck squamous cell carcinoma (HNSCC) — specifically in tumors that express a mutation in the HRAS proto-oncogene. KO-2806 is an investigational potent next-generation inhibitor of farnesyl transferase designed to potentially improve upon earlier FTI drug candidates.