RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union)

Structure/Sequence

Molecular FormulaC33H42F3N9O2S2

InChIKeyBGGALFIXXQOTPY-NRFANRHFSA-N

CAS Registry2134675-36-6

Clinical Trials associated with Ziftomenib

NCT07007312

/ Not yet recruitingPhase 3

Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination With Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia

Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with eligible genetic alterations. Ziftomenib is a type of therapy known to target the menin pathway in cancer cells.
This protocol has 2 separate studies that will investigate the benefits and risks of adding ziftomenib to standard-of-care (SOC) AML treatments in patients with certain genetic mutations who have not received any treatment for their AML. In the first study, the Nonintensive Therapy Study, older patients or those with serious medical problems will receive the SOC therapies venetoclax (ven) and azacitidine (aza), plus either ziftomenib or a placebo. In the second study, the Intensive Therapy Study, medically fit patients will receive (a) the SOC therapies cytarabine and daunorubicin, plus either ziftomenib or a placebo during a first treatment phase called induction, (b) cytarabine plus either ziftomenib or a placebo during a second treatment phase called consolidation, and (c) ziftomenib or a placebo during a third treatment phase called maintenance.
The physician will determine which study is the appropriate treatment for the patient, but neither the patient nor their physician will know whether the patient has been assigned to receive ziftomenib or a placebo. This design is called "double-blinded".

Start Date01 Sep 2025

Sponsor / Collaborator

Kura Oncology, Inc.

NCT06769490

/ Not yet recruitingPhase 1

Phase 1 Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia

The goal of this all-oral combination is to deliver safe and effective therapy for the largest portion of AML subtypes (NPM1mt, KMT2Ar, NUP98r
40-45%).

Start Date30 Jun 2025

Sponsor / Collaborator-

NCT06930352

/ Not yet recruitingPhase 2IIT

Frontline Ziftomenib in NPM1-Mutated or KMT2A-Rearranged Acute Myeloid Leukemia in Patients Not Eligible for Intensive Induction or Other Therapy

This phase II trial tests how well ziftomenib works in treating patients with NPM1 mutated or KMT2A rearranged acute myeloid leukemia (AML) and are not eligible to receive standard therapy. AML is often due to genetic changes in the cancer cells, including mutations in the NPM1 gene and rearrangements involving the KMT2A gene. These mutations result in activation of the menin pathway. Menin is a type of protein in the body that helps to regulate some of the naturally occurring processes in the body, but can also be involved in some types of cancers. Ziftomenib blocks this menin pathway and may prevent the cancer cells from continuing to grow. Giving ziftomenib may kill more cancer cells in patients with NPM1 mutated or KMT2A rearranged AML that are not eligible to receive standard therapy.

Start Date01 Jun 2025

Sponsor / Collaborator

Ohio State University Comprehensive Cancer Center

[+1]

100 Clinical Results associated with Ziftomenib

100 Translational Medicine associated with Ziftomenib

100 Patents (Medical) associated with Ziftomenib

Literatures (Medical) associated with Ziftomenib

01 Sep 2025·CRITICAL REVIEWS IN ONCOLOGY HEMATOLOGY

Menin inhibitors in KMT2A-rearranged and NPM1-mutated acute leukemia: A scoping review of safety and efficacy

Review

Author: Kalfah, Anas ; Dali, Sara Al ; Mohamed, Shehab F ; Al-Mashdali, Abdulrahman F

BACKGROUND:

Menin inhibitors represent a novel therapeutic approach targeting the Menin-KMT2A interaction in acute leukemias with KMT2A rearrangements or NPM1 mutations. This scoping review synthesizes current clinical evidence for emerging Menin inhibitors in development.

METHODS:

We systematically analyzed clinical trials, conference proceedings, and regulatory documents regarding Menin inhibitors in clinical development through December 2024. Primary outcomes included response rates, minimal residual disease (MRD) status, and safety profiles.

RESULTS:

Thirteen clinical trials investigating six Menin inhibitors (Revumenib, Ziftomenib, Bleximenib, BMF-219, DS-1594, and Enzomenib) were identified. Revumenib demonstrated consistent efficacy across five pivotal trials, achieving MRD-negative rates of 70-90% in both KMT2A-rearranged and NPM1-mutated leukemias, leading to FDA approval in November 2024. Ziftomenib showed particular efficacy in NPM1-mutated cases but exhibited a higher incidence of differentiation syndrome (30%) in KMT2A-rearranged patients. Bleximenib reported a 93% overall response rate, pending MRD validation. Early resistance emergence, primarily through MEN1 mutations, was observed across trials, emerging as early as two treatment cycles.

CONCLUSIONS:

Menin inhibitors demonstrate promising clinical activity in molecularly defined leukemias, with Revumenib establishing proof-of-concept for this therapeutic approach. However, challenges remain, including resistance development, optimal timing of therapy initiation, and determination of effective combination strategies. Larger randomized trials with extended follow-up are needed to establish long-term efficacy and safety profiles. The rapid clinical development of multiple agents in this class suggests an expanding role for Menin inhibitors in leukemia treatment paradigms.

01 Feb 2025·CTS-Clinical and Translational Science

Pharmacokinetics and ADME Characterization After Oral and Intravenous Administration of [¹⁴C]‐Ziftomenib in Healthy Male Participants

Article

Author: Mitra, Amitava ; El‐Shahat, Taha ; Leoni, Mollie ; Dale, Stephen ; Tabachri, Marilyn ; Ahsan, Julie Mackey

ABSTRACT:

Ziftomenib, a potent, selective inhibitor that binds menin at the lysine methyltransferase 2 interaction site, has demonstrated promising clinical activity with manageable toxicity in heavily pretreated patients with acute myeloid leukemia (AML) and nucleophosmin 1 mutations. This phase 1, open‐label study characterized the absorption, metabolism, excretion, and bioavailability of ziftomenib in healthy men and comprised two parts. In part A, a single oral dose of ziftomenib 400 mg (containing 250 μCi [14C]‐ziftomenib) was given to evaluate routes and rates of elimination, total radioactivity, and other pharmacokinetic parameters. In part B, a single oral dose of ziftomenib 400 mg followed by an intravenous dose of ziftomenib < 100 μg (containing 1 μCi [14C]‐ziftomenib) was administered to evaluate absolute bioavailability (both n = 8 patients). A median tmax of 3.5 h and an elimination t1/2 of 61.5 h demonstrated rapid ziftomenib absorption and enabled once‐daily dosing. Total radioactivity recovery was 89.7% in feces and 0.5% in urine over 480 h. Absolute bioavailability of 12.9% was observed. Ziftomenib was primarily metabolized by oxidation, N‐demethylation, and N‐dealkylation, with 19 metabolites recovered in plasma. All metabolites were considered minor (< 10% of total drug‐related exposure). Ziftomenib was the most abundant plasma component (> 10% of total drug‐related exposure). In conclusion, ziftomenib underwent limited metabolism following absorption and was primarily excreted as unchanged parent drug in feces. Ziftomenib was well tolerated with no new safety concerns in healthy men. Considering the pharmacokinetic profile and manageable safety outcomes, these findings support further clinical investigation of ziftomenib as treatment for AML.

01 Oct 2024·LANCET ONCOLOGY

Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial

Article

Author: Nie, Kun ; Patnaik, Mrinal M ; Pettit, Kristen ; Peterlin, Pierre ; Issa, Ghayas C ; Savona, Michael R ; Erba, Harry P ; Salamero, Olga ; Baer, Maria R ; Soifer, Harris S ; Shah, Mithun Vinod ; Walter, Roland B ; Kremyanskaya, Marina ; Rodríguez-Arbolí, Eduardo ; Berthon, Celine ; Adès, Lionel ; Grembecka, Jolanta ; Tabachri, Marilyn ; DeBotton, Stephane ; Leoni, Mollie ; Linhares, Brian M ; Cierpicki, Tomasz ; Altman, Jessica K ; Ray, Joshua ; Wang, Eunice S ; Mitra, Amitava ; Montesinos, Pau ; Foran, James M ; Corum, Daniel ; Heiblig, Mael ; Clegg, Bradley ; Fathi, Amir T ; Schiller, Gary ; Dale, Stephen ; Ahsan, Julie Mackey ; Papayannidis, Cristina

BACKGROUND:

Ziftomenib (KO-539) is an oral selective menin inhibitor with known preclinical activity in menin-dependent acute myeloid leukaemia models. The primary objective of this study was to determine the recommended phase 2 dose in patients with relapsed or refractory acute myeloid leukaemia based on safety, pharmacokinetics, pharmacodynamics, and preliminary activity.

METHODS:

KOMET-001 is a multicentre, open-label, multi-cohort, phase 1/2 clinical trial of ziftomenib in adults with relapsed or refractory acute myeloid leukaemia. Results of the phase 1 study, conducted at 22 hospitals in France, Italy, Spain, and the USA, are presented here and comprise the dose-escalation (phase 1a) and dose-validation and expansion (phase 1b) phases. Eligible patients were aged 18 years or older, had relapsed or refractory acute myeloid leukaemia, and had an Eastern Cooperative Oncology Group performance status of 2 or less. For phase 1a, patients (all molecular subtypes) received ziftomenib (50-1000 mg) orally once daily in 28-day cycles. For phase 1b, patients with NPM1 mutations or with KMT2A rearrangements were randomly assigned (1:1) using third-party interactive response technology to two parallel dose cohorts (200 mg and 600 mg ziftomenib). Primary endpoints were maximum tolerated dose or recommended phase 2 dose in phase 1a, and safety, remission rates, and pharmacokinetics supporting recommended phase 2 dose determination in phase 1b. Analyses were performed in all patients who received at least one dose of ziftomenib (modified intention-to-treat population). Phase 1a/1b is complete. This trial is registered with ClinicalTrials.gov, NCT04067336, and the EU Clinical Trials register, EudraCT 2019-001545-41.

FINDINGS:

From Sept 12, 2019, to Aug 19, 2022, 83 patients received 50-1000 mg ziftomenib (39 [47%] were male and 44 [53%] were female). Median follow-up was 22·3 months (IQR 15·4-30·2). Of 83 patients, the most common grade 3 or worse treatment-emergent adverse events were anaemia (20 [24%]), febrile neutropenia (18 [22%]), pneumonia (16 [19%]), differentiation syndrome (12 [15%]), thrombocytopenia (11 [13%]), and sepsis (ten [12%]). Overall, 68 of 83 patients had serious adverse events, with two reported treatment-related deaths (one differentiation syndrome and one cardiac arrest). Differentiation syndrome rate and severity influenced the decision to halt enrolment of patients with KMT2A rearrangements. In Phase 1b, no responses were reported in patients treated at the 200 mg dose level. At the recommended phase 2 dose of 600 mg, nine (25%) of 36 patients with KMT2A rearrangement or NPM1 mutation had complete remission or complete remission with partial haematologic recovery. Seven (35%) of 20 patients with NPM1 mutation treated at the recommended phase 2 dose had a complete remission.

INTERPRETATION:

Ziftomenib showed promising clinical activity with manageable toxicity in heavily pretreated patients with relapsed or refractory acute myeloid leukaemia. Phase 2 assessment of ziftomenib combination therapy in the upfront and relapsed or refractory setting is ongoing.

FUNDING:

Kura Oncology.

138

News (Medical) associated with Ziftomenib

03 Jun 2025

·www.biospace.com

Kura Posts New Data, Secures NDA Acceptance in Acute Myeloid Leukemia Race With Syndax

iStock, Deagreez Kura Oncology won FDA priority review for its drug the day before announcing new data at ASCO 2025 showing remission in about one-quarter of patients. But Syndax’s Revuforj also has priority review in this indication, with a PDUFA date two months earlier. Kura Oncology showed off new Phase II data for its acute myeloid leukemia drug and, at about the same time, got its New Drug Application accepted at the FDA with a PDUFA date set for late this year. That puts Kura in a head-to-head race with Syndax, which submitted an NDA to the agency in April in the same AML subtype for its drug Revuforj, which last fall earned approval for a different type of leukemia. On Sunday, the San Diego–based Kura and its development partner Kyowa Kirin announced that their NDA for ziftomenib, for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with specific mutations, had been accepted by the FDA with priority review. The PDUFA date is set for Nov. 30—just over two months after Syndax’s Sept. 26 PDUFA. Revuforj has also been granted priority review. Whichever drug gets approved first would be the first for patients with AML with this specific mutational background. Kura’s stock jumped about 17% early Monday after the news of the NDA acceptance. Later that day at ASCO 2025, the company showcased the data behind that acceptance. In 92 patients who had been pretreated for R/R NPM1-mutant AML, 21 achieved full remission or remission with some blood cell recovery, good for a complete remission or complete remission with partial hematologic recovery rate of 23%. “Overall, results were in line with expectations,” Mizuho analysts wrote in a note to investors early Tuesday, adding that the presented results lined up with previously released info in the conference’s abstract. Both ziftomenib and Syndax’s Revuforj are oral menin inhibitors, a new class of anti-cancer drugs. Revuforj’s approval for leukemia last November represented the first FDA nod for the class. A week after Revuforj’s FDA approval, Kura signed a co-development and commercialization deal for ziftomenib with Kyowa Kirin, wherein the latter paid Kura $330 million upfront and pledged up to $1.16 billion total in milestone payments. That deal gave Kyowa Kirin commercialization rights for the drug outside the U.S., with potential royalties due to Kura.

Priority ReviewLicense out/inDrug ApprovalNDAPhase 2

02 Jun 2025

·www.fiercebiotech.com

ASCO: Kura, Kyowa reveal 23% remission rate behind oral leukemia drug\'s phase 2 win

“Beyond ziftomenib’s clinical activity, we are highly encouraged by its consistent safety and tolerability profile,” Kura Oncology Chief Medical Officer Mollie Leoni, M.D., said in a release.\n Kura Oncology and Kyowa Kirin have shared details of the phase 2 win for their oral leukemia treatment ziftomenib as the FDA mulls whether to greenlight the selective menin inhibitor.The trial of ziftomenib enrolled 92 heavily pretreated patients with relapsed or refractory (R/R) NPM1-mutant acute myeloid leukemia (AML). Kura and Kyowa announced back in February that the midstage study had hit its primary endpoint of demonstrating a statistically significant improvement in complete response (CR) rate as well as partial hematological recovery (CRh) but held back the data until today.In a session at the American Society of Clinical Oncology (ASCO) annual meeting in Chicago, the companies revealed that 21 patients had either achieved full remission or a remission with some blood cell recovery, equivalent to a CR/CRh rate of 23%.The trial was powered to detect a CR/CRh rate of 20% to 30%, and today’s data seem to fall just within the ballpark expectation of TD Cowen analysts, who predicted at the start of the year that the trial would generate a CR/CRh rate “in the mid-20%s.”A total of 13 patients had a CR, and the remaining eight had a CRh, Kura and Kyowa explained in an accompanying June 2 release. The median duration of these responses was 3.7 months, with 12 of the patients free of any residual cancer cells.A median OS rate of six months was seen across all patients in the study, rising to 16.4 months among those who responded to treatment.The companies submitted an approval request to the FDA in April for ziftomenib in R/R NPM1-mutant AML and have plans to launch two phase 3 trials later this year in AML patients with NPM1 or KMT2A gene mutations and in NPM1 patients who cannot receive intensive chemotherapy. “Beyond ziftomenib’s clinical activity, we are highly encouraged by its consistent safety and tolerability profile,” Kura Chief Medical Officer Mollie Leoni, M.D., said in the release.“Notably, the low rate of myelosuppression, 3% discontinuation rate due to ziftomenib [treatment-related adverse events], lack of clinically significant QTc prolongation, absence of drug-drug interactions and effective management of differentiation syndrome through protocol-specified mitigation strategies underscore ziftomenib’s favorable benefit-risk profile for patients with relapsed or refractory NPM1-mutated AML,” Leoni added.Japan-based Kyowa fronted $330 million last year and pledged up to $1.1 billion in milestones to get in on the ziftomenib action. The two companies are sharing responsibility for launching various trials in AML and other blood cancers “over the next several years,” with Kura funding the development of ex-U.S. trials until the end of 2028, after which point the biopharmas will split the costs.

$ASCO: Kura, Kyowa reveal 23% remission rate behind oral leukemia drug\'s phase 2 win$

Phase 2ASCOClinical ResultAccelerated ApprovalPhase 3

01 Jun 2025

·ir.kuraoncology.com

Kura Oncology and Kyowa Kirin Announce FDA Acceptance and Priority Review of New Drug Application for Ziftomenib in Adults with Relapsed or Refractory NPM1-Mutant AML

– New Drug Application based on positive results from the Phase 2 KOMET-001 trial – – FDA assigns a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025 – – Potential first approval of a menin inhibitor for the treatment of adult patients with relapsed or refractory AML with an NPM1 mutation – SAN DIEGO and TOKYO , June 01, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) today announced the U.S. Food and Drug Administration (FDA) has accepted Kura’s New Drug Application (NDA) seeking full approval for ziftomenib as a treatment for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025 . “The FDA’s acceptance of our New Drug Application marks a significant milestone for Kura and Kyowa Kirin and, more importantly, for patients living with this genetic subset of AML, who face an aggressive form of the disease with few treatment options,” said Troy Wilson , Ph.D., J.D., President and Chief Executive Officer of Kura Oncology . “This achievement reflects the strength of the clinical data for ziftomenib as well as the incredible commitment of our teams. Along with our partners at Kyowa Kirin , we look forward to continuing to work closely with the FDA throughout the review process and to prepare for the anticipated launch of this treatment, which holds potential to meaningfully impact the lives of patients and their families.” The NDA is based on results from the Phase 2 KOMET-001 registrational trial in R/R NPM1-mutant (NPM1-m) AML (NCT #04067336). The KOMET-001 trial achieved its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. Ziftomenib was well‑tolerated with limited myelosuppression and 3% ziftomenib-related discontinuations. The safety and tolerability of ziftomenib were consistent with previous reports, and the benefit-risk profile for ziftomenib is highly encouraging. “Adult R/R NPM1-m AML patients face a significantly poor prognosis, highlighting the urgent need for innovative treatment options that can improve their outcomes,” said Takeyoshi Yamashita , Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin . “The acceptance of this NDA is a crucial step in our ongoing efforts to explore and evaluate various therapeutic strategies for AML through our comprehensive clinical trials. Our dedicated teams at Kyowa Kirin and Kura are fully committed to working tirelessly to ensure that, once approved, ziftomenib is made available to AML patients as quickly as possible. We recognize the importance of this endeavor and are excited about the possibility of making a meaningful impact on the lives of those affected by this challenging disease.” The KOMET-001 registration-directed trial is designed to assess evidence of clinical activity, safety and tolerability of ziftomenib, the only investigational therapy to receive Breakthrough Therapy Designation (BTD) from the FDA for treatment of R/R NPM1-mutant AML. In addition to BTD, ziftomenib has received Fast Track and Orphan Drug Designations. The full data analyses from the KOMET-001 trial of ziftomenib in R/R NPM1-m AML patients have been selected for oral presentation on Monday, June 2nd at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, and an encore presentation is planned at the 2025 European Hematology Association (EHA) Congress . About NPM1-Mutant 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. 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. 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 presence of such 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, 5.3 months in 3rd line, and 3.5 months following the 4th line1. There are currently no FDA-approved therapies targeting NPM1-m AML. About Ziftomenib Ziftomenib is a potent and selective, oral, investigational menin inhibitor currently in development for the treatment of genetically defined AML patients with high unmet need. In April 2024, ziftomenib received BTD from the FDA for the treatment of adult patients with R/R AML with an NPM1 mutation based on data from Kura’s KOMET-001 clinical trial. Additional information about clinical trials for ziftomenib can be found at www.kuraoncology.com/clinical-trials/#ziftomenib. 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 designed to target cancer signaling pathways. In November 2024, Kura Oncology entered into a global strategic collaboration agreement with Kyowa Kirin to develop and commercialize ziftomenib, a menin inhibitor, for AML and other hematologic malignancies. Enrollment in KOMET-001, a Phase 2 registration-directed trial of ziftomenib in R/R NPM1-m AML, has been completed, and in the second quarter of 2025, the companies announced submission of an NDA for ziftomenib for the treatment of adult patients with R/R NPM1-m AML. Kura and Kyowa Kirin are conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in newly diagnosed and R/R NPM1-m and KMT2A-rearranged AML. KO-2806, a next-generation farnesyl transferase inhibitor (FTI), is being evaluated in a Phase 1 dose-escalation trial (FIT-001) as a monotherapy and in combination with targeted therapies for patients with various solid tumors. Tipifarnib, a potent and selective FTI, is currently in a Phase 1/2 trial (KURRENT-HN) in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma. For additional information, please visit Kura’s website at https://kuraoncology.com/ and follow us on X and LinkedIn. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, Kyowa Kirin has invested in drug discovery and biotechnology innovation for more than 70 years and is currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology and rare diseases. A shared commitment to Kyowa Kirin’s values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com. Kura 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; the potential for ziftomenib to obtain FDA approval for the treatment of patients with NPM1-m AML, and the anticipated timing of such FDA approval; and the potential launch of ziftomenib. Factors that may cause actual results to differ materially from those indicated by these forward-looking statements include the risk that Kura may not be able to successfully demonstrate the safety and/or efficacy of its product candidates, including ziftomenib; the risk that Kura may not obtain approval to market its product candidates, including ziftomenib, or that such approval may be delayed; the risk that the collaboration with Kyowa Kirin is unsuccessful; 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. Kura Contacts Investors: Patti Bank Managing Director(415) 513-1284patti.bank@icrhealthcare.com Media:media@kuraoncology.com Kyowa Kirin Contacts Investors: Ryohei Kawai ir@kyowakirin.com Media, Global: Wataru Suzuki media@kyowakirin.com Source: Kura Oncology, Inc.

Phase 1Priority ReviewBreakthrough TherapyClinical ResultLicense out/in

100 Deals associated with Ziftomenib

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Acute myeloid leukemia with mutated NPM1	NDA/BLA	United States	Kura Oncology, Inc.	08 Apr 2025
Acute Myeloid Leukemia	Phase 3	-	Kura Oncology, Inc.	01 Sep 2025
Mixed phenotype acute leukemia	Phase 2	United States	Kura Oncology, Inc.	12 Sep 2019
Mixed phenotype acute leukemia	Phase 2	Belgium	Kura Oncology, Inc.	12 Sep 2019
Mixed phenotype acute leukemia	Phase 2	Canada	Kura Oncology, Inc.	12 Sep 2019
Mixed phenotype acute leukemia	Phase 2	France	Kura Oncology, Inc.	12 Sep 2019
Mixed phenotype acute leukemia	Phase 2	Germany	Kura Oncology, Inc.	12 Sep 2019
Mixed phenotype acute leukemia	Phase 2	Italy	Kura Oncology, Inc.	12 Sep 2019
Mixed phenotype acute leukemia	Phase 2	Spain	Kura Oncology, Inc.	12 Sep 2019
Mixed phenotype acute leukemia	Phase 2	United Kingdom	Kura Oncology, Inc.	12 Sep 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04067336 (KOMET-001, ASCO2025)	Phase 1/2	Relapsing acute myeloid leukemia NPM1-mutant	112	Ziftomenib 600 mg QD	arzebxvgpr(ftsgdvzefv) = ≤5% anemia fvppztvcyb (adnvvkipia ) View more	Positive	30 May 2025
NCT04067336 (KOMET-001, Company_Website) Manual	Phase 1/2	Acute Myeloid Leukemia NPM1 Mutation	-	Ziftomenib	ipprbkwbnz(dpuimqxdsz) = The KOMET-001 trial achieved its primary endpoint of CR plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. biufslyapp (farspsxcvq ) Met View more	Positive	07 Feb 2025
KOMET-007 (GlobeNewswire) Manual	Phase 1	Acute myeloid leukemia with mutated NPM1 \| Acute Leukemia with a KMT2A Translocation First line NPM1 Mutation \| KMT2A Rearrangement	54	ziftomenib +venetoclax/azacitidine	wpepgsogvy(dyltdtertd) = lrhtvhnzzc pwkdonhpfq (sddazuyyyq ) View more	Positive	09 Dec 2024
KOMET-007 (GlobeNewswire) Manual	Phase 1		54	Ziftomenib+cytarabinecytarabine/daunorubicindaunorubicin	wpepgsogvy(dyltdtertd) = vjuavdorrj pwkdonhpfq (sddazuyyyq ) View more	Positive	09 Dec 2024
ASH2024	Not Applicable	-	-	Ziftomenib 200 mg	hgafbmmxyy(xajtphxcyr) = zdjektkcin iglqvlenfc (oewalldzcy ) View more	-	08 Dec 2024
ASH2024	Not Applicable	-	-	Ziftomenib 400 mg	hgafbmmxyy(xajtphxcyr) = rxnoqvkdnu iglqvlenfc (oewalldzcy ) View more	-	08 Dec 2024
ASH2024	Not Applicable	-	-	Ziftomenib 200 mg	ppimdykgjo(jkqxaskbpe) = iefsknawgk gtkhyvdkak (hulpgsxujn )	-	07 Dec 2024
ASH2024	Not Applicable	-	-	Ziftomenib 400 mg	ppimdykgjo(jkqxaskbpe) = ungkabugdl gtkhyvdkak (hulpgsxujn )	-	07 Dec 2024
NCT04067336 (KOMET-001, Pubmed \| Lancet Oncol) Manual	Phase 1/2	Acute Myeloid Leukemia NPM1 Mutation \| KMT2A Rearrangement	83	Ziftomenib 50-1000 mg	ztsfrwoaxs(kvpyogbpgj) = the most common grade 3 or worse treatment-emergent adverse events were anaemia (20 [24%]), febrile neutropenia (18 [22%]), pneumonia (16 [19%]), differentiation syndrome (12 [15%]), thrombocytopenia (11 [13%]), and sepsis (ten [12%]). ninbstmetc (nugdmkbspg ) View more	Positive	01 Oct 2024
NCT04067336 (KOMET-001, SOHO2023)	Phase 1/2	Acute myeloid leukemia with mutated NPM1 FLT3 \| IDH1/2 \| NPM1	29	Ziftomenib 600mg	ownlofskcj(allmvgnfnm) = developed in 1 of 29 patients, detected at C4D28; the patient maintained stable disease through cycle 7 hvcdtrmjwa (jmqfpqcvxt ) View more	Positive	01 Sep 2023
NCT04067336 (KOMET-001, EHA2023)	Phase 1/2	leukemia acute ambiguous lineage \| Relapsing acute myeloid leukemia \| Mixed phenotype acute leukemia ... View more	-	Ziftomenib	kyeojuuhnl(hucqipgeys) = jbvcwtpnbu ymrksoower (nsbhfciffn ) View more	-	08 Jun 2023
NCT04067336 (KOMET-001, ASH 12022 \| ASH 22022) Manual	Phase 1/2	Relapsing acute myeloid leukemia	-	Ziftomenib 200 mg	jsujzmyhla(qhgqtlzkfl) = poiyiofjqd znqanpbcbe (jruweqigxe, 0 - 26.5) View more	Positive	15 Nov 2022
NCT04067336 (KOMET-001, ASH 12022 \| ASH 22022) Manual	Phase 1/2	Relapsing acute myeloid leukemia	-	Ziftomenib 600 mg	jsujzmyhla(qhgqtlzkfl) = jjdijeyerj znqanpbcbe (jruweqigxe, 5.5 - 57.2) View more	Positive	15 Nov 2022

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