Kura Oncology Reports Positive Preliminary Ziftomenib Combination Data in Acute Myeloid Leukemia
Kura Oncology Reports Positive Preliminary Ziftomenib Combination Data in Acute Myeloid Leukemia
01-30-2024
– No differentiation syndrome events reported –
– 100% CR rate with 7+3 in newly diagnosed NPM1-m and KMT2A-r AML –
– 56% CR/CRh with ven/aza in menin inhibitor naïve patients with R/R AML –
– 80% of patients remain on trial as of data cutoff, including all NPM1-m patients –
– 200 mg dose of ziftomenib cleared in ven/aza cohorts, enrollment at 400 mg dose ongoing –
– Management to host virtual investor event today at
The first 20 patients were enrolled in KOMET-007 between
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
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/CRh2 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
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
Virtual Investor Event
Kura will host a virtual investor event featuring company management and investigators from the KOMET-007 trial of ziftomenib today at
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 and a high unmet need remains. The menin pathway is considered a driver for multiple genetic alterations of the disease, of which NPM1-mutations are among the most common, representing approximately 30% of AML cases and KMT2A-rearrangements represent approximately 5-10% of AML cases. While patients with NPM1-m 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 R/R 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. Adult patients with NPM1-m AML and select co-mutations and/or R/R disease have a poor prognosis, with median overall survival of only approximately 7.8 months in 2nd line, 5 months in 3rd line and 3.5 months following the 4th line3. Adult patients with KMT2A-r AML have a poor prognosis with high rates of resistance and relapse following standard of care, with median overall survival for this patient population of only 6 months following 2nd line and 2.4 months following 3rd line4. No FDA-approved therapies targeting NPM1-m and KMT2A-r AML currently exist.
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
About
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 efficacy, safety and therapeutic potential of ziftomenib, potential benefits of combining ziftomenib with appropriate standards of care, plans regarding future clinical trials in the ziftomenib program, plans and expected timing for enrollment in the Phase 2 registration-directed trial of ziftomenib, and the Company’s cash runway. 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, risks associated with Kura’s cash needs, 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
Contacts
Investors:
Executive Vice President, Investor Relations &
Corporate Communications
(858) 500-8833
pete@kuraoncology.com
Media:
Alexandra Weingarten
Associate Director, Investor Relations &
Corporate Communications
(858) 500-8822
alexandra@kuraoncology.com
1 Age > 60 years and/or treatment-related AML and/or adverse risk cytogenetics per European LeukemiaNet (ELN)
2 CR with partial hematologic recovery
3 Issa G, et al. Blood Adv 2023;7(6):933-42.
4 Issa GC, et al. Blood Cancer J. 2021;11(9):162.
Source: Kura Oncology, Inc.