Tipifarnib
Tipifarnib is an oral, investigational drug candidate that we are evaluating in multiple Phase 2 clinical trials.
Tipifarnib blocks the activity of an enzyme called farnesyl transferase. Protein farnesylation is a key cell signaling process implicated in various cancer processes, including survival, growth and proliferation of tumor cells.
Tipifarnib has been studied in more than 5,000 oncology patients and was observed to have a manageable side effect profile. Clinical and preclinical data suggest that, in certain selected patient populations, tipifarnib has the potential to provide clinical benefit to cancer patients with limited treatment options.
At Kura we are using a precision medicine approach to develop tipifarnib for the treatment of patients with selected types of cancer, including conducting the following Phase 2 clinical trials in patients with:
- HRAS Mutant Squamous Head and Neck Cancer
- Peripheral T-Cell Lymphomas
- Myelodysplastic Syndromes
- Chronic Myelomonocytic Leukemia
Tipifarnib in Patients with Head and Neck Cancer
Tipifarnib is an oral, investigational drug candidate we are evaluating in patients with certain head and neck cancers that carry HRAS mutations.
HRAS is a proto-oncogene, which is a normal gene that can become an oncogene due to mutations or increased expression. Proto-oncogenes code for proteins that help to regulate cell growth and differentiation and HRAS is mutated and/or expressed in certain head and neck squamous cell carcinoma (HNSCC) tumors.
Although HRAS was discovered more than 40 years ago, no specific therapies have been developed that target tumors with the mutated form of the protein. Tipifarnib is a potent and highly selective inhibitor of farnesyltransferase, a critical enzyme for the activity of HRAS.
The relapsed and/or refractory HNSCC patient population has an overall survival of approximately six to eight months and existing therapeutic options, including new therapies such as immune therapy, typically show a response rate in the range of 10-20 percent.
Encouraging clinical activity of tipifarnib has been observed in patients with HRAS mutant head and neck squamous cell carcinoma (HNSCC) tumors. We are conducting a Phase 2 clinical trial to evaluate the safety and activity of tipifarnib in patients with relapsed and/or refractory HRAS mutant HNSCC.
Tipifarnib in Patients with Peripheral T-Cell Lymphoma (PTCL)
Tipifarnib is an oral, investigational drug candidate being studied for safety and clinical activity in patients with relapsed or refractory Peripheral T-Cell Lymphomas.
We are currently evaluating tipifarnib in a Phase 2 clinical trial in patients with relapsed and/or refractory PTCL.
Encouraging preliminary data from the trial suggest that tipifarnib has anti-tumor activity in certain patients with:
- Angioimmunoblastic T-cell lymphomas (AITL)
- Peripheral T-cell Lymphomas Not Otherwise Specified (PTCL-NOS) with elevated levels of CXCL12 expression
View research on tipifarnib in PTCL.
Tipifarnib in Patients with Myelodysplastic Syndromes (MDS)
Tipifarnib is an oral, investigational drug candidate being studied for safety and antitumor activity in patients with Myelodysplastic Syndromes (MDS).
Encouraging activity of tipifarnib in patients with lower-risk MDS has been observed, including signs of hematologic improvement. In addition, we have identified potential biomarkers, which appear to correlate with the activity of tipifarnib in this patient population.
We are conducting a Phase 2 clinical trial to evaluate the safety and activity of tipifarnib in eligible patients with MDS who have no known curative treatment.
View research on tipifarnib in MDS.
Tipifarnib in Patients with Chronic Myelomonocytic Leukemia (CMML)
Tipifarnib is an oral, investigational drug candidate being studied for safety and antitumor activity in patients with Chronic Myelomonocytic Leukemia (CMML).
Our ongoing Phase 2 study is investigating the antitumor activity in terms of the objective response rate of tipifarnib, and validating potential biomarkers, in eligible patients with CMML.
Tipifarnib and KO-947 are investigational drug candidates and have not yet been approved for use by the U.S. Food and Drug Administration (FDA) or other regulatory authorities.
View research on tipifarnib in CMML.
KO-947
KO-947 is an investigative drug candidate that we are currently evaluating in a Phase 1 clinical trial.
KO-947 blocks the activity of an enzyme called extracellular signal-regulated kinase or ERK. ERK is a critical component of a signaling pathway that has been implicated in various cancer processes, including survival, growth and proliferation of tumor cells.
We have conducted extensive profiling of KO-947 using a broad panel of preclinical models of cancer. Robust and durable activity, including tumor regressions, was observed in a number of disease indications of high unmet need.
Tipifarnib and KO-947 are investigational drug candidates and have not yet been approved for use by the U.S. Food and Drug Administration (FDA) or other regulatory authorities.
In addition, we have also identified potential biomarkers associated with the anti-tumor activity of KO-947, which may enable patient selection strategies for clinical development. Our goal is to identify indications with the potential for single agent activity.
We are currently conducting a Phase 1 clinical trial of KO-947 in patients with locally advanced unresectable or metastatic, relapsed and/or refractory non-hematological malignancies.
View research on KO-947 (ERK Inhibitor).
KO-539
KO-539 is a drug candidate that we are currently evaluating in preclinical testing for treatment of certain kinds of leukemia.
KO-539 blocks the interaction of two proteins called menin and MLL that, together, are critically important for survival, growth and proliferation of certain kinds of leukemia cells.
We have conducted extensive pre-clinical profiling of KO-539. Robust and durable activity, including tumor regressions, was observed in multiple disease models of leukemia that represent high unmet need.
In addition, we have also identified potential biomarkers associated with the anti-tumor activity of KO-539, which may enable patient selection strategies for clinical development.
Our goal is to identify indications with the potential for single agent activity, which may enable accelerated development.
We are currently conducting preclinical development activities for KO-539 with a goal to file an IND and advance this drug candidate into Phase 1 human clinical testing.
View research on KO-539 (Menin-MLL Inhibitor).