We are advancing a diverse pipeline of precision medicines for the treatment of blood cancers and solid tumors.
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.
(KO-539)
Investigator-sponsored studies
Investigator-sponsored studies
Investigator-sponsored studies
Investigator-sponsored studies
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.
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.)
Next-Generation FTI
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.
* 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)
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 become a healthy cell? That’s the mechanism underlying an emerging class of targeted therapies called menin inhibitors.
Our menin inhibitor, ziftomenib, prevents 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 blocked, cancerous juvenile cells can mature into healthy white blood cells.
We believe ziftomenib has potential to address approximately 35% of acute myeloid leukemia cases, including NPM1-mutant AML and KMT2A-rearranged AML. Importantly, we believe it can be combined with other cancer therapies to minimize or prevent treatment resistance and foster enduring patient outcomes.
Imagine if a single key could unlock multiple targeted treatment paths for a range of difficult cancers. A class of precision medicines known as farnesyl transferase inhibitors may hold that power.
Farnesyltransferase is an enzyme that plays a foundational role in many cellular pathways that drive the development and growth of different types of tumors. By inhibiting the enzyme’s function through various mechanisms of action, we believe we can reshape treatment options for many cancers.
Our investigational drug tipifarnib is a farnesyl transferase inhibitor, or FTI, that has demonstrated encouraging clinical activity in a genetically defined subset of head and neck squamous cell carcinoma (HNSCC) — specifically in tumors that express a mutation in the HRAS protooncogene. It is also promising for subsets of additional types of cancer, including breast cancer, renal cell carcinoma and others. Tipifarnib also can be combined with certain approved cancer therapies, which we believe will help address treatment resistance and potentially lead to long-term remission for patients.