Our programs comprise 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.

Candidate

Development Approach

Study Startup

Phase 1

Registration Directed

Trial

Menin Inhibitor

Ziftomenib

(KO-539)

Monotherapy

Phase 1

NPM1-mutant Acute Myeloid Leukemia (AML)

Monotherapy

Study Startup

Non-NPM1-m / KMT2A-r AML

Monotherapy

Study Startup

KMT2A-rearranged ALL

Combination with venetoclax + azacitidine

Study Startup

NPM1-mutant AML

Combination with venetoclax + azacitidine

Study Startup

KMT2A-rearranged AML

Combination with cytarabine + daunorubicin (7+3)

Study Startup

NPM1-mutant AML

Combination with cytarabine + daunorubicin (7+3)

Study Startup

KMT2A-rearranged AML

Combination with gilteritinib

Study Startup

NPM1-mutant AML

Combination with FLAG-IDA

Study Startup

NPM1-mutant AML

Combination with FLAG-IDA

Study Startup

KMT2A-rearranged AML

Combination with IDAC/LDAC

Study Startup

NPM1-mutant AML

Combination with IDAC/LDAC

Study Startup

KMT2A-rearranged AML

Post-Transplant Maintenance

Study Startup

NPM1-mutant AML

Post-Transplant Maintenance

Study Startup

KMT2A-rearranged AML

Combination with FLA

Study Startup

Pediatric AML & ALL

Combination with BV-DAM

Study Startup

Pediatric ALL

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 currently being evaluated in the KOMET-001 Trial.

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Farnesyl Transferase Inhibitors (FTI)

Tipifarnib

Monotherapy

Registration Directed

HRAS-mutant Head and Neck Squamous Cell Carcinoma (HNSCC)*

Combination with alpelisib

Phase 1

PIK3CA-dependent HNSCC

Combination with alpelisib

Phase 1

HRAS-dependent HNSCC

Combination with osimertinib

Study Startup

EGFR-mutant Non-Small
Cell Lung Cancer (NSCLC)

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 three clinical trials: HRAS-mutant Head and Neck Squamous Cell Carcinoma (HNSCC) (enrollment ongoing in AIM-HN Trial,) HRAS-dependent and/or PIK3CA-dependent HNSCC (enrollment ongoing in KURRENT-HN Trial,) and EGFR-Mutant NSCLC (KURRENT-LUNG Trial.)

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Farnesyl Transferase Inhibitors

KO-2806

Next-Generation FTI

Combination with targeted therapies

Study Startup

Solid tumors

KO-2806

KO-2806 is a development candidate designed to target innovative biology and address large oncology indications of high unmet need through rational combinations. Multiple advanced lead compounds were identified during a discovery-stage program aimed at developing a next-generation farnesyl transferase inhibitor with improved potency, pharmacokinetic and physicochemical properties relative to tipifarnib. KO-2806 was nominated as our lead development candidate and is currently undergoing investigational new drug (IND)-enabling studies.

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* Trial closed to further enrollment

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

FLA: fludarabine and high dose cytarabine; BV-DAM: bortezomib, vorinostat, dexamethasone, PEG-asparaginase and mitoxantrone

XOSPATA® (gilteritinib); PIQRAY® (alpelisib); TAGRISSO® (osimertinib)