Kura Oncology Announces Positive Results from Registration-Directed Study of Tipifarnib in Patients with HRAS Mutant HNSCC
– Meaningful antitumor activity observed in recurrent/metastatic HRAS mutant HNSCC, including CR –
– Data validate activity of farnesyltransferase inhibitors and support rapid advancement of KO-2806 into combinations with targeted therapies in large solid tumor indications –
– Results to be presented in late-breaking oral session at 2023
These clinical results are being featured during a late-breaking oral session at the 2023
As of the data cutoff on
Review Facility (n=50)
|Best Overall Response, n (%)|
|Confirmed Complete Response (CR)||1 (2)||1 (2)|
|Confirmed Partial Response (PR)||14 (28)||9 (18)|
|Stable Disease (SD)||17 (34)||14 (28)|
|Progressive Disease (PD)||6 (12)||14 (28)|
|Not Evaluable (NE)||12 (24)||12 (24)|
|ORR, n (%) [95% CI] ||15 (30) [0.18, 0.45]||10 (20) [0.10, 0.34]|
|mDoR, months [95% CI]||5.6 [3.88, 9.23]||6.5 [3.88, -]|
|mPFS, months [95% CI]||3.7 [2.60, 5.55]||2.6 [1.87, 4.40]|
*ORR, objective response rate; -, not calculable; mDoR, median duration of response; mPFS, median progression free survival; CI, confidence interval.
Both assessments by investigators and IRF observed one patient achieving a CR on treatment. Patients had a median of two prior lines of therapy (range 0-6) in the recurrent/metastatic setting and robust activity was seen in second line treatment and beyond with greater activity observed in the second line versus the third line and subsequent treatments. The ORR in second line treatment was 29% [0.13, 0.51] in the IRF assessment. The ORR for three FDA-approved therapies for the treatment of HNSCC in the second line range from 13-16%.
“The results from the AIM-HN study are encouraging as they demonstrate meaningful clinical benefit of tipifarnib in a subset of HNSCC for which there are currently no other targeted therapies in development, and a significant unmet need exists for the population,” said
Patients in the AIM-HN trial received tipifarnib at a dose of 600 mg orally twice daily on days 1-7 and 15-21 of 28-day cycles. Tipifarnib was generally well-tolerated with a manageable safety profile. The most common grade 3 or 4 treatment-related adverse events (TRAEs) seen in at least 10% of patients were cytopenias and TRAEs led to discontinuation of treatment in 7% of patients.
“We continue to be encouraged by the compelling safety profile and activity of tipifarnib as a monotherapy in this difficult-to-treat population of advanced head and neck cancer, supported by our Breakthrough Therapy Designation from the FDA,” said
AIM-HN is a multicenter, open-label, pivotal study evaluating the efficacy of tipifarnib in HRAS mutant HNSCC. Eligibility criteria include patients with recurrent or metastatic HRAS mutant HNSCC at any VAF level. The primary and key secondary endpoints of this trial include ORR and DoR in the high VAF population. The trial was designed to enroll at least 59 patients with HRAS mutant HNSCC who received prior platinum-based therapy and was closed to enrollment in
Head and neck squamous cell carcinoma (HNSCC) is the seventh most common cancer worldwide, accounting for more than 500,000 new cases each year. Despite advances in immunotherapy, the prognosis for advanced HNSCC patients remains poor, with an estimated median overall survival of 13-15 months in patients when stratified by PD-L1 expression. Although the anti-epidermal growth factor receptor (EGFR) antibody, cetuximab, was approved more than a decade ago, development of biomarker-directed therapies in HNSCC has been stymied by the limited number of druggable targets in the genomic landscape and the challenge of managing drug refractory, recurrent/metastatic HNSCC.
Memorial Sloan Kettering (MSK) has institutional financial interests related to
This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the efficacy, safety and therapeutic potential of tipifarnib, potential benefits of combining tipifarnib with appropriate standards of care, and progress and expected timing of the tipifarnib program and clinical trials. Factors that may cause actual results to differ materially include the risk that compounds that appeared promising in early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials, the risk that Kura may not obtain approval to market its product candidates, uncertainties associated with performing clinical trials, regulatory filings, applications and other interactions with regulatory bodies, risks associated with reliance on third parties to successfully conduct clinical trials, the risks associated with reliance on outside financing to meet capital requirements, and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words “may,” “will,” “would,” “could,” “should,” “believes,” “estimates,” “projects,” “promise,” “potential,” “expects,” “plans,” “anticipates,” “intends,” “continues,” “designed,” “goal,” or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties the Company faces, please refer to the Company's periodic and other filings with the
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1 HRAS variant allele frequency ≥ 20% and treated with at least one dose of tipifarnib
2 One low VAF patient (< 20% VAF) was a responder to treatment
Source: Kura Oncology, Inc.