Kura Oncology Reports Positive Preliminary Data from “Most Exciting Investigational Agent” for Leukemia

Kura Oncology, Inc. (Nasdaq: KURA) has reported preliminary clinical data from the first 20 patients in a Phase 1 dose-escalation trial of the company’s potent and selective menin inhibitor, ziftomenib, in combination with standards of care, in patients with NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML).

The study has demonstrated that ziftomenib at 200 mg QD has been well tolerated and the safety profile consistent with features of underlying disease and backbone therapies.

Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology, said, “We are highly encouraged by these preliminary combination data for ziftomenib and believe they support advancement into the frontline AML population. Given that ziftomenib targets foundational mutations at the core of up to 50% of AML cases, we are encouraged by its potential to transform treatment outcomes across the continuum of care. With the recently announced financing, we remain in a strong financial position with a cash runway expected into 2027, which enables us to invest aggressively in research, development and pre-commercial activities to maximize value of ziftomenib and other pipeline assets.”

Amer Zeidan, MBBS, MHS, interim chief of the Division of Hematologic Malignancies, Director of Hematology Early Therapeutics Research at Yale Cancer Center and lead investigator of the KOMET-007 trial, said, “Ziftomenib is one of the most exciting investigational agents being studied in AML, and I am thrilled to see the rapid pace of accrual into this first-in-human combinational study.”

Highlights 

Kura’s Phase 1 dose-escalation trial showed: 100% CR rate with 7+3 in newly diagnosed NPM1-m and KMT2A-r AML and 56% CR/CRh with ven/aza in menin inhibitor naïve patients with R/R AML.

Kura expects to complete enrollment of 85 patients in KOMET-001, a Phase 2 registration-directed trial of ziftomenib in patients with R/R NPM1-m AML, by the middle of this year.

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. Ziftomenib has received Orphan Drug Designation from the FDA for the treatment of AML.

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 and a high unmet need remains.

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 that target cancer signaling pathways. Ziftomenib is a once-daily, oral drug candidate targeting the menin-KMT2A protein-protein interaction for the treatment of genetically defined AML patients with high unmet need. Kura is currently enrolling patients in a Phase 2 registration-directed trial of ziftomenib in NPM1-m R/R AML (KOMET-001). The Company is also conducting a series of studies to evaluate ziftomenib in combination with current standards of care, beginning with ven/aza and 7+3 in NPM1-m and KMT2A-r newly diagnosed and R/R AML (KOMET-007). Tipifarnib, a potent and selective farnesyl transferase inhibitor (FTI), is currently in a Phase 1/2 trial in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma (KURRENT-HN). Kura is also evaluating KO-2806, a next-generation FTI, in a Phase 1 dose-escalation trial as a monotherapy and in combination with cabozantinib in clear cell renal cell carcinoma and adagrasib in KRASG12C-mutated non-small cell lung cancer (FIT-001). For additional information, please visit Kura’s website at www.kuraoncology.com and follow us on X and LinkedIn.