Our pipeline consists of small molecule agents that target cancer signaling pathways, where each program builds on our expertise in precision medicines.

Program

Development Approach

Research

Phase 1 Dose Escalation

Phase 1 Dose Expansion

Registrational

U.S. FDA Approved

Clinical Trial

Menin Inhibitor

KOMZIFTI™

(ziftomenib)*

Monotherapy

U.S. FDA APPROVED

Adults with R/R NPM1-m AML

Menin Inhibitor

Ziftomenib**

Combination with 7+3(IC)

REGISTRATIONAL

Frontline NPM1-m or KMT2A-r AML

Combination with venetoclax/azacitidine (NIC)

REGISTRATIONAL

Frontline NPM1-m

Combination with venetoclax/azacitidine

PHASE 1 DOSE EXPANSION

R/R NPM1-m or KMT2A-r AML

Combination with 7+3 and quizartinib

PHASE 1 DOSE ESCALATION

Frontline NPM1-m / FLT3-m AML

Combination with 7+3 or venetoclax/azacitidine

PHASE 1 DOSE EXPANSION

Frontline NPM1-m or KMT2A-r AML

Combination with gilteritinib

PHASE 1 DOSE EXPANSION

R/R NPM1-m / FLT3-m AML

Combinations with FLAG-IDA and LDAC

PHASE 1 DOSE ESCALATION

R/R NPM1-m or KMT2A-r AML

Monotherapy

PHASE 1 DOSE EXPANSION

R/R Non-NPM1-m / Non-KMT2A-r AML

Monotherapy

PHASE 1 DOSE ESCALATION

R/R KMT2A-r ALL

Combination with imatinib

PHASE 1 DOSE ESCALATION

Advanced gastrointestinal stromal tumors (GIST)

Ziftomenib

Ziftomenib is being investigated in up to 50% of AMLs across NPM1-m, KMT2A-r and FLT3-m AML, and in combination with other cancer therapies.

View publications and presentations

Farnesyl Transferase Inhibitor

Darlifarnib (KO-2806)

NEXT-GEN FTI

Combination with cabozantinib

PHASE 1 DOSE EXPANSION

Renal cell carcinoma (RCC)

Combination with adagrasib

PHASE 1 DOSE ESCALATION

Non-small cell lung cancer (NSCLC), colorectal cancer (CRC), pancreatic ductal adenocarcinoma (PDAC)

Darlifarnib (KO-2806)

Darlifarnib (KO-2806) is an investigational next-generation farnesyl transferase inhibitor (FTI). Combination therapy using FTIs has the potential to address drug resistance and provide deeper and more durable anti-tumor activity. Darlifarnib is not FDA-approved and safety and efficacy have not been established.

View publications and presentations

Farnesyl Transferase Inhibitors

Tipifarnib

Combination with alpelisib

REGISTRATION-ENABLING

PIK3CA-dependent head and neck squamous cell carcinoma (HNSCC)2

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 (KO-7246)

RESEARCH

Diabetes and cardiometabolic disease

The investigational agents and investigational uses of marketed products identified above have not been approved by the US Food and Drug Administration. Safety and efficacy have not been established.

-m, mutated; -r, rearranged; R/R, relapsed/refractory; 7+3, 7 days of cytarabine + 3 days of daunorubicin; IC, intensive chemotherapy; NIC, non-intensive chemotherapy; ALL, acute lymphoblastic leukemia; FLAG-IDA: fludarabine, high-dose cytarabine (Ara-C), granulocyte-colony stimulating factor (G-CSF) and idarubicin; LDAC: Low-dose cytarabine

Progress bars indicate the stage of development based on ongoing or completed activities. A partial bar indicates a phase in progress; a full bar indicates completion of a phase. Bars do not represent scale, duration, or likelihood of success.

*KOMZIFTI (ziftomenib) was approved by the U.S. Food and Drug Administration for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options.

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