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.

140

News (Medical) associated with Ziftomenib

12 Jun 2025

·ir.kuraoncology.com

Kura Oncology and Kyowa Kirin Report Positive Updated Combination Data for Ziftomenib in Newly Diagnosed AML at 2025 European Hematology Association Congress

– Encouraging clinical activity with deep responses demonstrated in the KOMET-007 trial with the combination of 600 mg ziftomenib with 7+3 in newly diagnosed patients with NPM1-m and KMT2A-r AML – – 93% (41/44) and 89% (24/27) CRc observed in NPM1-m and KMT2A-r AML response-evaluable patients, respectively – – 71% (24/34) and 88% (14/16) CR measurable residual disease (MRD)-negativity observed among responding NPM1-m and KMT2A-r AML patients, respectively – – 96% (47/49) of NPM1-m and 88% (29/33) KMT2A-r AML patients remain alive and continue on-study – – Combination was well tolerated with no additive myelosuppression – – KOMET-017-IC (intensive chemotherapy) and NIC (non-intensive chemotherapy) randomized phase 3 studies (NCT07007312) expected to start in 2H 2025 – – Kura Oncology to host virtual investor event to discuss results and broader ziftomenib development plan on June 18, 2025 at 4:30pm ET / 1:30pm PT – SAN DIEGO and TOKYO , June 12, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) today provided positive updated clinical data from KOMET-007, a Phase 1a/1b trial of ziftomenib, a highly selective oral investigational menin inhibitor, in combination with standards of care in patients with newly diagnosed NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML). The data for the combination with cytarabine/daunorubicin (7+3) were presented as an oral presentation at the European Hematology Association 2025 Congress (EHA2025) being held in Milan, Italy from June 12-15, 2025 . “The findings presented at EHA2025 underscore the potential of ziftomenib in combination with 7+3 as an early intervention in the frontline setting of AML and could offer a meaningful opportunity to improve patient outcomes,” said Harry Erba , M.D., Ph.D., Director of the Leukemia Program at the Duke Cancer Institute . “The high rates of complete remission and MRD negativity across the 7+3 cohorts are particularly encouraging. The continued rapid enrollment in the Phase 1b portion of this study underscores the urgency and enthusiasm for further evaluating this combination approach.” “We remain very encouraged by the updated clinical activity, safety and tolerability data from the KOMET-007 study evaluating ziftomenib with 7+3 in newly diagnosed AML patients with NPM1 mutations or KMT2A rearrangements,” said Mollie Leoni , M.D., Chief Medical Officer of Kura Oncology . “These updated data reinforce the combination potential of ziftomenib in the frontline setting, strengthening our confidence in its ability to provide a valuable treatment option for a significant portion of the AML population. We and our partners at Kyowa Kirin are working in earnest to prepare for the KOMET-017-IC and NIC pivotal Phase 3 studies, which will enable us to test ziftomenib-based combinations and their potential, if approved, to transform care for AML patients worldwide.” In the ongoing study, ziftomenib dosed once daily at 600 mg in combination with 7+3 continued to demonstrate robust and evolving clinical activity in patients with newly diagnosed AML. Among 71 response-evaluable patients, 92% (65/71) achieved a composite complete remission (CRc) (93% for NPM1-m, 89% for KMT2A-r patients) and 80% (57/71) achieved a complete remission (CR) (84% for NPM1-m, 74% for KMT2A-r patients) at the time of data cutoff. A rate of CR minimal residual disease (CR-MRD) negativity of 71% for NPM1-m with a median time to MRD negativity of 4.7 weeks and a rate of CR-MRD negativity of 88% for KMT2A-r patients with a median time to MRD negativity of 4.4 weeks were observed. Ziftomenib did not delay time to neutrophil and platelet count recovery, which was comparable to intensive chemotherapy regimens. Median follow-up times for the two populations were 24.9 weeks (range 4.3-47.1) in NPM1-m patients and 15.7 weeks (range 1.1-40.3) in KMT2A-r patients. Among response-evaluable NPM1-m patients, neither a median duration of CR nor a median overall survival (OS) had been reached. Among response-evaluable KMT2A-r patients, a median duration of CR was determined to be 25.6 weeks (95% CI, range 8.3-NE), and a median OS had not been reached. Notably, 96% (47/49) of NPM1-m patients and 88% (29/33) of KMT2A-r patients remained alive and on study. The safety population included 82 newly diagnosed adult patients with NPM1-m or KMT2A-r AML from the pooled Phase 1a/1b portions of the trial at the 600 mg QD dose of ziftomenib. The safety profile observed with ziftomenib was consistent with previously reported data. Ziftomenib-related adverse events (TRAEs) of ≥ Grade 3 (Gr3), which occurred in more than 10% of patients were febrile neutropenia (15%), decreased platelet count (15%), anemia (11%) and decreased neutrophil count (11%). One case of differentiation syndrome (KMT2A-r, Gr3) was successfully managed by protocol-specified mitigation strategies. Two cases of investigator-assessed QTc prolongation (both KMT2A-r, Gr3) were reported; both patients were on other medications (posaconazole and/or piperacillin/tazobactam), which have been identified as potentially causing QT prolongation at the time of QT assessment. No dose-limiting toxicities, drug-drug interactions, clinically meaningful ziftomenib-associated QTc prolongation or additive myelosuppression were observed. “Despite the availability of approved therapies for AML, up to 70% of patients who initially achieve a complete response relapse within three years – highlighting a substantial unmet need,” said Takeyoshi Yamashita , Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin . “The data presented at EHA2025 suggest a favorable safety, tolerability, and efficacy profile for ziftomenib. We are encouraged by its potential as a future first-line treatment option and are committed to advancing the KOMET-017 Phase 3 trial, expected to begin later this year, to further evaluate its value in AML care.” The EHA2025 oral presentation highlighting ziftomenib combined with 7+3 in newly diagnosed (1L) NPM1-m and KMT2A-r AML, and an encore presentation of results from the KOMET-001 registrational trial of ziftomenib in relapsed/refractory (R/R) NPM1-m AML (also presented during EHA2025) are available in the Posters and Presentations section of the Kura website. The KOMET-017 protocol consists of 2 separate Phase 3 studies, which will investigate the benefits and risks of adding ziftomenib to standards of care treatments in patients newly diagnosed NPM1-m or KMT2A-r AML and which is registered at www.clinicaltrials.gov as NCT07007312. Virtual Investor Event Kura will host a virtual investor event featuring company management and investigators from the KOMET-007 trial of ziftomenib in combination with 7+3 in patients with NPM1-m and KMT2A-r AML at 4:30pm ET / 1:30pm PT on Wednesday, June 18, 2025. Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The event can also be accessed on the Investors section of Kura’s website at www.kuraoncology.com. An archived replay will be available shortly after the conclusion of the live event. 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. Ziftomenib, a once-daily, oral menin inhibitor, is the first and only investigational therapy to receive Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for the treatment of R/R NPM1-m AML. In November 2024, Kura Oncology entered into a global strategic collaboration agreement with Kyowa Kirin to develop and commercialize ziftomenib 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 the FDA’s acceptance of a New Drug Application for ziftomenib for the treatment of adult patients with R/R NPM1-m AML and assignment of a Prescription Drug User Fee Act target action date of November 30, 2025. 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. Ziftomenib is also being evaluated in a Phase 1 dose-escalation trial (KOMET-015) in combination with imatinib for treatment of patients with advanced GIST. 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 the Kura 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; potential benefits of combining ziftomenib with intensive chemotherapy and other standards of care for AML; progress of the ziftomenib program and clinical trials, including the KOMET-017-IC pivotal Phase 3 study; and the potential for ziftomenib to obtain regulatory approval. 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, 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, 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 3Clinical ResultPhase 1Phase 2Breakthrough Therapy

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

03 Jun 2025

·www.pharmaceutical-technology.com

ASCO25: Kura’s Phase II ziftomenib results trigger NDA acceptance from FDA

The FDA has set a 30 November 2025 PDUFA date for approval of ziftomenib in NMP1-m r/r AML. Photo by SOPA Images/LightRocket via Getty Images A November 2025 PDUFA date has been set for Kura Oncology’s oral acute myeloid leukaemia (AML) drug ziftomenib following the unveiling of positive Phase II data. Results from the pivotal Phase I/II KOMET-001 trial (NCT04067336) were presented at the 2025 American Society of Clinical Oncology (ASCO) conference taking place over 30 May to 3 June in Chicago, Illinois. In a 2 June abstract, ziftomenib demonstrated positive disease response in relapsed or refractory AML patients with NPM1-mutation, regardless of prior venetoclax use or stem cell transplantation. Since the release of positive Phase II KOMET-001 results, the US Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) from Kura and Kyowa Kirin, the company’s partner for non-US development and commercialisation of ziftomenib in relapsed or refractory AML. A PDUFA date is scheduled for 30 November 2025. The NDA acceptance builds on a breakthrough designation the FDA awarded for ziftomenib, a menin inhibitor, in April 2024 for this patient population. KOMET-001 enrolled 112 patients with NPM1-m relapsed or refractory AML, 92 of whom were assessed in its Phase II portion. Patients received 600mg of oral ziftomenib daily having been heavily pretreated previously; 60% had prior venetoclax exposure and 23% had undergone previous stem cell transplant. Phase II KOMET-001 data show ziftomenib led to complete response (CR) or CR with partial haematological recovery (CRh) in 23% of participants with rates of 21% and 24% for venetoclax-naïve and exposed patients, respectively. Among 15 responsive patients, 67% demonstrated minimal residual disease (MRD) negativity at a median follow-up of 4.2 months. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence As per a 2 June release, Kura CEO Troy Wilson highlighted the drug’s potential to occupy a vacant niche in AML treatment. “NPM1 mutations are among the most common in AML, representing approximately 30% of cases, and there are no FDA-approved therapies specifically for this population,” he said. Kura have also begun enrolling patients in its Phase I KOMET-015 trial of ziftomenib to treat advanced gastrointestinal stromal tumours (GIST) as of April 2025. Kyowa Kirin retain the option to collaborate with Kura for ziftomenib development in this and other solid tumour indications in return for payments to Kura of up to $228m. The companies’ current partnership grants Kura a potential $1.16bn from Kyowa Kirin for the rights to ziftomenib for AML outside of the US. GlobalData predicts ziftomenib to generate total sales of $69m in 2026, though this hinges on a 2025 approval. These are expected to increase to $1.88bn annually by 2031. GlobalData is the parent company of Pharmaceutical Technology . Pharmaceutical Technology Excellence Awards - Have you nominated? Nominations are now open for the prestigious Pharmaceutical Technology Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact . This is your chance to showcase your achievements, highlight industry advancements , and gain global recognition . Don't miss the opportunity to be honoured among the best - submit your nomination today! Nominate Now

Clinical ResultPhase 1Phase 2NDABreakthrough Therapy

100 Deals associated with Ziftomenib

R&D Status

10 top R&D records.

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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
Acute Lymphoblastic Leukemia	Phase 2	United States	Kura Oncology, Inc.	12 Sep 2019
Acute Lymphoblastic Leukemia	Phase 2	Belgium	Kura Oncology, Inc.	12 Sep 2019
Acute Lymphoblastic Leukemia	Phase 2	Canada	Kura Oncology, Inc.	12 Sep 2019
Acute Lymphoblastic Leukemia	Phase 2	France	Kura Oncology, Inc.	12 Sep 2019
Acute Lymphoblastic Leukemia	Phase 2	Germany	Kura Oncology, Inc.	12 Sep 2019
Acute Lymphoblastic Leukemia	Phase 2	Italy	Kura Oncology, Inc.	12 Sep 2019
Acute Lymphoblastic Leukemia	Phase 2	Poland	Kura Oncology, Inc.	12 Sep 2019
Acute Lymphoblastic Leukemia	Phase 2	Spain	Kura Oncology, Inc.	12 Sep 2019

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04067336 (KOMET-001, ASCO2025) Manual	Phase 1/2	Acute myeloid leukemia with mutated NPM1 NPM1 Mutation	112	Ziftomenib 600 mg QDZiftomenib 600 mg QD (Phase 1b/2)	pyzngoyhxa(xcjixdrnlz) = kfltgokokm vjsmoyxoba (uzsjcazglp, 17 - 34) View more	Positive	30 May 2025
				Ziftomenib 600 mg QDZiftomenib 600 mg QD (Phase 2)	pyzngoyhxa(xcjixdrnlz) = btvbnrqcad vjsmoyxoba (uzsjcazglp, 15 - 33)
NCT04067336 (KOMET-001, Company_Website) Manual	Phase 1/2	Acute Myeloid Leukemia NPM1 Mutation	-	Ziftomenib	fuefkritma(pfhrdtjuid) = The KOMET-001 trial achieved its primary endpoint of CR plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. mfpexenzyk (vnvrnxyvwm ) 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	cwqfizdcxj(xrcbzfkfff) = ydihttgjbw pjqaztfwgs (sghzrvtufv ) View more	Positive	09 Dec 2024
				Ziftomenib+cytarabinecytarabine/daunorubicindaunorubicin	cwqfizdcxj(xrcbzfkfff) = ebnsnpvcgd pjqaztfwgs (sghzrvtufv ) View more
ASH2024	Not Applicable	-	-	Ziftomenib 200 mg	mhonozgcib(togjfcigah) = hiqugojlsm iczvrhabyo (sfktfbedzo ) View more	-	08 Dec 2024
				Ziftomenib 400 mg	mhonozgcib(togjfcigah) = apvyfezqqf iczvrhabyo (sfktfbedzo ) View more
ASH2024	Not Applicable	-	-	Ziftomenib 200 mg	jvtezorjup(uxxtmsdkxa) = nncnvlmtwr emkpfahyjg (hyumwpzqhe )	-	07 Dec 2024
				Ziftomenib 400 mg	jvtezorjup(uxxtmsdkxa) = nbevfqruae emkpfahyjg (hyumwpzqhe )
NCT04067336 (KOMET-001, Pubmed \| Lancet Oncol) Manual	Phase 1/2	Acute Myeloid Leukemia NPM1 Mutation \| KMT2A Rearrangement	83	Ziftomenib 50-1000 mg	zzfkdelvzd(czxewndugv) = 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%]). pvzhdqapfc (drhovngwcj ) 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	fklsdhnnpq(ydybyllwbf) = developed in 1 of 29 patients, detected at C4D28; the patient maintained stable disease through cycle 7 kawvwxwjgf (purkxqsbkp ) 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	uaqmpzshhp(uaybytbtug) = zvqjgsrbxi teffyjwmwi (eijwquugnm ) View more	-	08 Jun 2023
NCT04067336 (KOMET-001, ASH 12022 \| ASH 22022) Manual	Phase 1/2	Relapsing acute myeloid leukemia	-	Ziftomenib 200 mg	dxgpfkeyco(vtditynuku) = cwcefxpnvr fxlxboksfz (aeryzobnpi, 0 - 26.5) View more	Positive	15 Nov 2022
				Ziftomenib 600 mg	dxgpfkeyco(vtditynuku) = atkiqftobp fxlxboksfz (aeryzobnpi, 5.5 - 57.2) View more

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