Our programs consist of small molecule drug candidates that target cancer signaling pathways, where each program builds on our expertise in precision medicines and seeks to drive better outcomes for patients and broaden the eligible patient population through rational combinations.

Program

Development Approach

Research

Dose Escalation

Dose Optimization

Registration-Enabling

Regulatory Submission

Clinical Trial

Menin Inhibitor

Ziftomenib**

Monotherapy

REGISTRATION DIRECTED

NPM1-mutant acute myeloid leukemia (AML)1

Monotherapy

DOSE-ESCALATION

KMT2A-rearranged ALL

Monotherapy

DOSE-ESCALATION

Non-NPM1-m / Non-KMT2A-r 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 an investigational drug candidate and oral inhibitor of menin-KMT2A (MLL) for the treatment of Acute Myeloid Leukemia (AML), with the potential to combine with other targeted therapies. Ziftomenib is currently being evaluated as a monotherapy in the KOMET-001 trial and as a combination therapy with certain standards of care across multiple lines of therapy in the KOMET-007 and KOMET-008 trials. The R/R NPM1-m AML portion of the Phase 2 KOMET-001 trial has completed enrollment and is not currently recruiting.

**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.

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Farnesyl Transferase Inhibitors

KO-2806

Next-Generation 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)

KO-2806

KO-2806 is a potent next-generation FTI designed to improve upon potency, pharmacokinetic and physicochemical properties of earlier FTI drug candidates and address large oncology indications of high unmet need through rational combinations. Preclinical data is supportive of KO-2806 in combination with tyrosine kinase inhibitors (TKIs) and KRAS inhibitors, to drive enhanced antitumor activity and address mechanisms of innate and adapted resistance to targeted therapies. KO-2806 is currently being evaluated as a monotherapy and in combination with cabozantinib in ccRCC and with adagrasib in KRASG12C-mutated NSCLC (FIT-001).

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Farnesyl Transferase Inhibitors

Tipifarnib

Combination with alpelisib

DOSE-VALIDATION

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

Tipifarnib

Tipifarnib is an oral, investigational drug candidate and highly selective inhibitor of farnesyltransferase. Farnesyl transferase inhibitors (FTI) – such as tipifarnib – have multiple potential therapeutic applications, including direct inhibition of oncogenic proteins; overcoming drug resistance; and preventing emergence of resistance. Tipifarnib is currently being evaluated in patients with PIK3CA-dependent HNSCC (enrollment ongoing in KURRENT-HN trial).

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Next-Gen Menin Inhibitor

Monotherapy (Beta Cell Regeneration)

RESEARCH

Diabetes

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 Topline results reported on February 5, 2025; patients remain on study in survival follow-up.
2 KOMET-017 program to advance directly to Registration-Enabling trials, leveraging data from Phase 1 combination trials.