The Src Kinase inhibition platform refers to novel small molecule compounds that have differentiated mechanisms of actions including: (1) the inhibition of the activity of Src Kinase and (2) the inhibition of tubulin polymerization. We believe the combination of the two mechanisms of action provides a broader range of anti- cancer activity compared to either mechanism of action alone. Our three key clinical product candidates in this platform are KX-01 ointment for actinic keratosis (AK), pre-cancerous lesions and psoriasis; KX-01 oral for solid and liquid tumors and potential skin cancer indications and pre-cancerous lesions and KX-02 oral for glioblastoma multiforme (GBM).

We are conducting an approximately 160-patient Phase 2 study of KX-01 ointment for treatment of AK across 16 sites. AK is a common disease, with a prevalence of approximately 58 million patients in the United States. If left untreated, 10-15% of AK lesions will develop into skin cancers. Our Phase 1 clinical study and preliminary data from our Phase 2 clinical study demonstrated a complete response rate of up to 43%, with no severe local skin reactions reported with the dosing regimen studied. Currently available treatments are limited by severe local skin reactions such as vesicultation, pustulation, erosion and ulceration, which compromise patient compliance. We believe physicians and patients have avoided topical treatments because of the pronounced side effects associated with current treatments such as ingenol mebutate and fluorouracil.  A new ointment product with good clinical activity and a favorable side effect profile is expected to provide substantial benefit to a greater number of patients and capture substantial new market share for treatment of this condition. We expect to receive the complete primary endpoint data from this Phase 2 study in the first half of 2017. If we receive confirmatory results, along with regulatory consultation with FDA regarding the design of the Phase 3 program, we anticipate initiating two pivotal Phase 3 studies in the second-half of  2017.

KX01: An Ointment Formulation

The topical ointment formulation of KX01 is expected to be useful in a variety of cancer and pre-cancer related skin ailments. Clinical data in the US indicate our ointment is very likely to be a promising product for the treatment of actinic keratosis as compared to current therapy. Dermatologists have been excited from observations made in the clinic from patients’ response of KX01 ointment treated against actinic keratosis. Clinical evaluation for its utility in treating psoriasis has also begun.

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KX01: An Oral Formulation

KX-01 is a novel orally administered small molecule compound which we discovered and developed, and which demonstrates at least two methods of action: Src tyrosine kinase inhibition (non-ATP competitive); and tubulin polymerization. The combination of two MOAs, provides a broader range of anti-cancer activity in vitro and in animal tumor models than would be the case with either MOA alone. Since KX-01 is readily absorbed orally it allows multi-day dosing regimens to optimize the efficacy vs toxicity that are not practical with IV tubulin targeting drugs such as vincristine and vinblastine. We are currently exploring its best application in multiple tumor types.

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KX02: An Oral Formulation

KX-02 is the second small molecule we developed using our Src Kinase Inhibition platform. Although KX-02 is an analog of KX-01, it has significantly different physical properties. These properties allow KX-02 to freely cross the blood-brain-barrier such that the concentration in the brain is equal to, or somewhat greater than, that in the plasma. This trait is uncommon for oncology drugs and highlights the potential for KX-02 as a novel therapy for unmet medical needs such as brain cancers, including GBM and brain metastases. The FDA has granted Orphan Drug Status to KX-02 for the treatment of gliomas. KX-02 is a non-ATP competitive Src Kinase inhibitor and tubulin polymerization inhibitor.  Studies of KX-02 in preclinical syngeneic mouse GBM models resulted in the complete eradication, without recurrence, of the tumors in an average of approximately 30% of treated mice. KX-02’s multiple mechanisms of action, or MOAs, along with its ability to cross the blood-brain-barrier, make it a novel molecule for the treatment of brain tumors. KX-02 is currently in the early stages of a U.S. Phase 1 clinical trial in solid tumor patients. A Chinese IND has been filed by our development partner in China to start a study in primary brain tumors with KX-02 which we believe will commence in 2017.

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