Cabozantinib (s)-Malate

The FDA has approved Exelixis’ cancer drug Cabometyx to treat advanced neuroendocrine tumors inside and outside of the pancreas, marking two new indications for the company’s primary source of revenue. The new label includes adults and children 12 years and older with previously treated, locally advanced or metastatic tumors that can’t be removed with surgery. The cancer treatment was already approved to treat renal cell carcinoma, hepatocellular carcinoma and thyroid cancer. The approval, announced Wednesday, was based on a double-blind, placebo-controlled study with two separate cohorts for patients with tumors inside and outside of the pancreas, respectively. In the cohort of patients with tumors inside the pancreas, Cabometyx-treated participants notched a median progression-free survival of 13.8 months, compared to 3.3 months for patients given placebo. The overall response rate was 18% and 0%, respectively. In the other cohort, which included 100 additional patients, median PFS was 8.5 months in the treatment arm compared to 4.2 months for placebo. The overall response rate was 5% for patients given Cabometyx. Overall survival data weren’t mature for either cohort. Exelixis and the FDA said that the safety profile was consistent with Cabometyx in other cancer types, though the rate of hypertension, regardless of treatment arm, was higher in patients with neuroendocrine tumors compared to the other cancers that the drug is approved to treat. The company also said that “a majority of patients treated with Cabometyx required dose modifications or reductions to manage adverse events.” The additional indications add fuel to Exelixis’ core revenue engine, after global revenue for the cabozantinib franchise, which Cabometyx is the foundation of, exceeded $2.5 billion in 2024. Further growing the franchise was the core argument of activist investors at Farallon Capital Management, who rebuilt the board almost two years ago. More treatments for patients with neuroendocrine tumors are in the works. Amy Peterson, Exelixis’ chief medical officer and EVP of product development and medical affairs, reaffirmed in a statement the company’s plans to launch a study of its clinical-stage zanzalintinib compared to everolimus in the first half of this year.

– Oral presentation to highlight preclinical data further supporting combination of KO-2806 with TKIs in ccRCC – – Company expects to present data from the Phase 1 FIT-001 trial evaluating KO-2806 and cabozantinib in patients with RCC in 2H 2025 – SAN DIEGO, March 26, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today announced that an abstract containing preclinical data for KO-2806, the Company’s next-generation farnesyl transferase inhibitor (FTI), in combination with cabozantinib, a tyrosine kinase inhibitor (TKI), in clear cell renal cell carcinoma (ccRCC), has been accepted for an oral presentation at the upcoming American Association for Cancer Research (AACR) Annual Meeting in Chicago, IL on April 29, 2025. “The latest findings for our next-generation farnesyl transferase inhibitor, KO-2806, being presented at this year’s AACR Annual Meeting, add to the growing body of data demonstrating the potential of FTIs as companion therapeutic agents to augment the antitumor activities of various targeted therapies and to overcome resistance in combination,” said Francis Burrows, Ph.D., Chief Scientific Officer of Kura Oncology. “We continue to make good progress in our FIT-001 trial evaluating KO-2806 in solid tumor indications where there is unmet medical need, and we look forward to presenting the first clinical data for KO-2806 as monotherapy and in combination with cabozantinib for the treatment of RCC later this year.” The title and session information for the oral presentation are listed below. Full abstract details including title and text are currently available to registrants via the AACR online itinerary planner. Details of the oral presentation, entitled “Farnesyl transferase inhibitor KO-2806 enhances the antitumor activity of cabozantinib in ccRCC tumors that progress on anti-VEGFR agents” (oral 6370), are as follows: Session Date and Time: Monday, April 28, 2025; 2:30 PM - 4:45 PM CTSession Title: Minisymposium Novel Antitumor AgentsPresentation Time: 4:25 PM - 4:40 PM CT A copy of the presentation will be available in the Posters and Presentations section on Kura’s website at the beginning of the presentation session. About Kura Oncology Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company’s pipeline consists of small molecule drug candidates targeting cancer signaling pathways. Ziftomenib, a once-daily, oral menin inhibitor, is the first and only investigational therapy to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of relapsed/refractory (R/R) NPM1-mutant acute myeloid leukemia (AML). In November 2024, Kura Oncology entered into a global strategic collaboration agreement with Kyowa Kirin Co., Ltd. to develop and commercialize ziftomenib for AML and other hematologic malignancies. Enrollment in a Phase 2 registration-directed trial of ziftomenib in R/R NPM1-mutant AML has been completed, and the companies anticipate submission of a New Drug Application (NDA) to the FDA in the second quarter of 2025. Kura Oncology and Kyowa Kirin are also conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in newly diagnosed and R/R NPM1-mutant and KMT2A-rearranged AML. KO-2806, a next-generation farnesyl transferase inhibitor, is being evaluated in a Phase 1 dose-escalation trial as a monotherapy and in combination with targeted therapies. Tipifarnib, a potent and selective farnesyl transferase inhibitor, is currently in a Phase 1/2 trial in combination with alpelisib for patients with PIK3CA-dependent HNSCC. For additional information, please visit Kura’s website at www.kuraoncology.com and follow us on X and LinkedIn. Forward-Looking Statements 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 potential of FTIs to augment the antitumor activities of targeted therapies and to overcome resistance; the progress of Kura’s clinical trials; the efficacy, safety and therapeutic potential of Kura’s product candidates, ziftomenib, KO-2806 and tipifarnib; the expected timing and presentation of results and data from clinical trials; and the anticipated timing of submission of an NDA for ziftomenib. 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 Securities and Exchange Commission, which are available at www.sec.gov. Such forward-looking statements are current only as of the date they are made, and Kura assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Contacts Investors:Patti BankManaging Director(415) 513-1284patti.bank@icrhealthcare.com Media:Alexandra WeingartenAssociate Director, Corporate Communications(858) 500-8822alexandra@kuraoncology.com