Kura Oncology Announces First Patients Dosed in Phase 2 Registration-Directed Trial of Ziftomenib in NPM1-Mutant Acute Myeloid Leukemia
– Phase 1 trial showed 30% CR rate among 20 NPM1-mutant AML patients treated at recommended Phase 2 dose –
– Phase 2 registration-directed trial expected to enroll 85 patients in the
“Dosing the first patients in our registration-directed trial of ziftomenib marks a significant milestone for our menin inhibitor program,” said
In the Phase 1 clinical trial, ziftomenib showed as of the data cutoff on
The primary endpoint in the Phase 2 registration-directed trial in patients with NPM1-mutant relapsed or refractory AML, is CR or complete response with hematologic recovery (CRh), and key secondary endpoints include clinical benefit as well as safety and tolerability. In addition to continued evaluation of ziftomenib as a monotherapy in NPM1-mutant AML, Kura plans to initiate the KOMET-007 and KOMET-008 trials later this year to evaluate ziftomenib in combination with current standards of care in earlier lines of therapy and across multiple patient populations, including NPM1-mutant and KMT2A-rearranged AML.
About Acute Myeloid Leukemia
AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells or platelets. Despite the many available treatments for AML, prognosis for patients remains poor. NPM1-mutations are among the most common genetic alterations, representing approximately 30% of AML cases. While patients with NPM1-mutant AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the relapsed or refractory setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A and IDH1/2, with prognosis heavily influenced by the occurrence of co-occurring mutations. Median overall survival is only six months following relapse for NPM1-mutant patients. KMT2A-rearrangements are less frequent, representing approximately 5-10% of AML. No FDA-approved therapies targeting NPM1-mutant and KMT2A-rearranged AML currently exist.
Ziftomenib is a novel, once-daily, oral investigational drug candidate targeting the menin-KMT2A/MLL protein-protein interaction for treatment of genetically defined AML patients with high unmet need. In preclinical models, ziftomenib inhibits the KMT2A/MLL protein complex and exhibits downstream effects on HOXA9/MEIS1 expression and potent anti-leukemic activity in genetically defined preclinical models of AML. Ziftomenib has received Orphan Drug Designation from the
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 ziftomenib, potential benefits of combining ziftomenib with appropriate standards of care, and progress and expected timing of the ziftomenib 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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Source: Kura Oncology, Inc.