SAN DIEGO, Calif. — Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today reported preliminary clinical data from the first 20 patients in KOMET-007, a Phase 1 dose-escalation trial of the Company’s potent and selective menin inhibitor, ziftomenib, in combination with standards of care, including cytarabine/daunorubicin (7+3) and venetoclax/azacitidine (ven/aza), in patients with NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML).
The first 20 patients were enrolled in KOMET-007 between July 2023 and November 2023, including five newly diagnosed patients with adverse risk NPM1-m or KMT2A-r AML and 15 patients with refractory/relapsed (R/R) NPM1-m or KMT2A-r AML.
Continuous daily dosing of ziftomenib at 200 mg QD has been well tolerated and the safety profile consistent with features of underlying disease and backbone therapies. No differentiation syndrome events of any grade were reported, and no dose-limiting toxicities, evidence of QTc prolongation, drug-drug interactions or additive myelosuppression were observed.
As of the data cutoff on January 11, 2024, all newly diagnosed patients treated with ziftomenib and 7+3 achieved a complete remission (CR) with full count recovery, for a CR rate of 100% (5/5), including four patients with NPM1-m AML and one patient with KMT2A-r AML.
The overall response rate (ORR) among R/R patients treated with ziftomenib and ven/aza was 53% (8/15). Among all patients treated with ziftomenib and ven/aza, 40% (6/15) received prior treatment with a menin inhibitor. The CR/CRh rate in patients who were menin inhibitor naïve was 56% (5/9), including 60% (3/5) in patients with NPM1-m AML and 50% (2/4) in patients with KMT2A-r AML. The ORR in patients who received prior venetoclax was 40% (4/10), including 60% (3/5) in patients with NPM1-m AML.
As of the data cutoff, 80% (16/20) of patients remain on trial, including 100% (11/11) of all NPM1-m patients.
“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,” said 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. “In this first public release of early data from the KOMET-007 trial, ziftomenib demonstrates an encouraging safety and tolerability profile in combination with 7+3 and ven/aza, enabling continuous administration while mitigating the risk of differentiation syndrome. The combinations demonstrate encouraging preliminary evidence of clinical activity in patients with refractory/relapsed disease after failure of other agents, including venetoclax, a setting with very limited effective treatment options. Further, the fact that most patients remain on study as of the data cutoff is notable in such difficult-to-treat patient populations.”
The 200 mg dose of ziftomenib has been cleared in the R/R ven/aza cohorts and enrollment at the 400 mg dose is ongoing. Upon determination of a recommended Phase 2 dose, Kura plans to initiate a Phase 1b dose validation/expansion in combination with ven/aza in newly diagnosed patients with NPM1-m (without adverse risk) or KMT2A-r AML.
“We are highly encouraged by these preliminary combination data for ziftomenib and believe they support advancement into the frontline AML population,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “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. We continue to see strong investigator enthusiasm as evidenced by rapid enrollment across studies, and we expect to complete enrollment of all 85 patients in KOMET-001, our Phase 2 registration-directed trial of ziftomenib in patients with R/R NPM1-m AML, by the middle of this year. With the recently announced financing, we remain in a strong financial position with 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.”
About Ziftomenib
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 U.S. Food and Drug Administration for the treatment of AML.
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).
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