Menin Inhibitors- Target Population, Competitive Landscape, and Market Forecast - 2034

Key Highlights:

• The menin protein is a one-of-its kind, scaffold nuclear protein which is encoded by the tumor suppressor MEN1 (Menin 1) gene. MEN1 mutations are well known to be implicated in the causation of sporadic or autosomal dominant hereditary cancer syndromes which affect the endocrine system, known as the MEN1 syndrome.
• Menin inhibitors are small molecule inhibitors that bind with high affinity to the KMT2A binding pocket of menin. These agents inhibit the interaction between menin and KMT2A which inhibits KMT2A-dependent transcription of downstream target genes.
• Menin inhibitors are primarily used to treat specific subtypes of acute myeloid leukemia characterized by mutations in the KMT2A gene (formerly known as MLL) or the NPM1 gene and also in early research for acute lymphoblastic leukemia, colorectal cancer and other solid tumors.
• Syndax's REVUFORJ (revumenib) is the first and only approved Menin inhibitor for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older. It received regulatory approval from US Food and Drug Administration (FDA) in November 2024.
• Menin Inhibitors has a robust pipeline consisting of products such as ziftomenib, enzomenib (DSP-5336), bleximenib, and others which are in late Phases (III/II).
• Syndax, Kura Oncology, Sumitomo Pharma, Johnson & Johnson, and several other companies are currently engaged in the development and production of Menin inhibitors, which have the potential to significantly impact and enhance the Menin inhibitors market.
• In June 2025, Kura Oncology and Kyowa Kirin announced that the US 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.
• Ziftomenib data in European Hematology Association (EHA) 2025: 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.

DelveInsight's "Menin Inhibitors- Target Population, Competitive Landscape, and Market Forecast - 2034" report delivers an in-depth understanding of the Menin, historical and Competitive Landscape as well as the Menin inhibitor market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The Menin inhibitors market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM Menin inhibitors market size from 2020 to 2034. The report also covers current Menin treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

Menin Inhibitor Understanding

Menin Inhibitor Overview

The menin protein is a one-of-its kind, scaffold nuclear protein which is encoded by the tumor suppressor MEN1 (Menin 1) gene. MEN1 mutations are well known to be implicated in the causation of sporadic or autosomal dominant hereditary cancer syndromes which affect the endocrine system, known as the MEN1 syndrome. This demonstrates the tumor suppressor activity of the gene. While the biological basis of this phenomenon is being actively studied, loss of cell cycle regulation, and disruption of inhibition of transcription factors such as JunD resulting from MEN1 mutation and a truncated menin protein are a few proposed mechanisms. On the contrary, the menin protein exerts a paradoxically different function in the hematopoietic pathological states, where it serves as an essential oncogenic cofactor for the maintenance and propagation of leukemogenesis initiated by the oncoprotein KMT2A, as well as the KMT2A fusion protein complexes.

Menin inhibitors are small molecule inhibitors that bind with high affinity to the KMT2A binding pocket of menin. These agents inhibit the interaction between menin and KMT2A which inhibits KMT2A-dependent transcription of downstream target genes. These agents thereby inhibit an important component of the pathogenesis of KMT2Ar and NPM1m AML. Similarly, disruption and inhibition of the menin and KMT2A interaction is also being studied as a therapeutic strategy in leukemic states driven by other molecular rearrangements which result in increased HOXA gene transcription.

Menin Inhibitor Epidemiology

The Menin inhibitors epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented as total cases of selected indication for Menin inhibitor, total eligible patient pool for Menin inhibitor in selected indication, total treated cases in selected indication for Menin inhibitors in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), and the United Kingdom, and Japan from 2020 to 2034.

• In 2024, the incident population of acute myeloid leukemia in the United States was found to be around 21,250.
• Assessments as per DelveInsight showed that there were more males than females affected by acute myeloid leukemia in the United States in 2024.
• Most cases were from the age above 65 years.
• Among EU4 and the UK, Germany has the highest number of acute myeloid leukemia cases, which were around 4,680 cases in 2024.

Menin Drug Chapters

The drug chapter segment of the Menin inhibitor reports encloses a detailed analysis of late-stage (Phase III and Phase II) pipeline drugs. It also helps understand the Menin inhibitor's clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Marketed Menin Inhibitor

REVUFORJ (revumenib): Syndax

REVUFORJ is a menin inhibitor indicated for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older. It blocks the interaction of both wild-type lysine methyltransferase 2A (KMT2A) and KMT2A fusion proteins with menin. The binding of KMT2A fusion proteins with menin is involved in KMT2A-rearranged (KMT2Ar) acute leukemias through activation of a leukemogenic transcriptional pathway. In nonclinical studies using cells that express KMT2A fusions, inhibition of the menin-KMT2A interaction with revumenib altered the transcription of multiple genes including differentiation markers.

In November 2024, Syndax Pharmaceuticals announced that the FDA has approved REVUFORJ as the first and only menin inhibitor for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older. The FDA previously granted Breakthrough Therapy and Fast Track designations as well as Priority Review for REVUFORJ. The New Drug Application (NDA) received approval through the FDA's Real Time Oncology Review (RTOR) program.

Completed submission of sNDA for R/R mNPM1 AML in April 2025, based on positive pivotal data reported in 4Q 2024.

Emerging Menin Inhibitors

Ziftomenib: Kura Oncology

Ziftomenib is an investigational drug candidate and oral inhibitor of menin-KMT2A (MLL) for the treatment of Acute Myeloid Leukemia (AML), with the potential to combine with other targeted therapies. Ziftomenib is currently being evaluated as a monotherapy in the KOMET-001 trial and as a combination therapy with certain standards of care across multiple lines of therapy in the KOMET-007, KOMET-008 trials and KOMET-017.

In April 2024, the FDA granted breakthrough therapy designation to ziftomenib as a treatment for those with relapsed/refractory acute myeloid leukemia harboring NPM1 mutations, according to a press release from the developers Kura Oncology.

Menin Inhibitor Market Outlook

Menin inhibitors have emerged as a transformative class of targeted therapies in the treatment of acute leukemias, particularly for genetically defined subtypes such as NPM1-mutant and KMT2A-rearranged acute myeloid leukemia (AML). These genetic alterations are associated with poor prognosis and resistance to standard therapies, creating a high unmet need for innovative, molecularly targeted treatments. Among the front-runners in this class is REVUFORJ (revumenib), developed by Syndax, which received FDA approval in November 2024 for the treatment of relapsed or refractory acute leukemia. The drug has been granted Orphan Drug Designation and Breakthrough Therapy Designation, underscoring its clinical significance and potential to redefine standards of care in select AML subpopulations.

In the emerging pipeline, ziftomenib from Kura Oncology stands out as the most advanced competitor, currently in Phase III trials. It also holds both Breakthrough Therapy and Orphan Drug Designation, reflecting strong early clinical data and regulatory momentum. Ziftomenib is being investigated not only as monotherapy but also in combinations with venetoclax/azacitidine and with standard induction chemotherapy (7+3) for NPM1-mutant and KMT2A-rearranged AML, which could potentially broaden its use in both frontline and relapsed/refractory settings.

Other notable candidates include enzomenib (DSP-5336) from Sumitomo Pharma America, currently in Phase II with Fast Track and Orphan Drug Designations, and bleximenib (JNJ-75276617) from Johnson & Johnson, also in Phase II development for relapsed/refractory AML. These agents reinforce the growing interest among major pharmaceutical players in the menin inhibition space and highlight the potential for diverse therapeutic strategies targeting this pathway.

What makes menin inhibitors especially promising is their ability to directly modulate epigenetic and transcriptional drivers of leukemogenesis. By targeting the menin-KMT2A/MLL1 interaction, these agents can suppress leukemic gene expression programs at the source, offering a precision medicine approach for patients who currently have limited options. Furthermore, the oral route of administration adds convenience and potential quality-of-life benefits for patients undergoing long-term treatment.

Looking ahead, REVUFORJ is expected to maintain leadership in the near term given its first-in-class approval and established efficacy in relapsed/refractory patients. However, as Ziftomenib and other next-generation menin inhibitors progress through pivotal trials, the competitive landscape will likely shift, particularly if these candidates demonstrate superior efficacy in genetically defined subsets or offer enhanced durability in combination regimens. Additionally, regulatory designations and expedited review pathways may further accelerate the time-to-market for pipeline agents.

In conclusion, the menin inhibitor market is poised for rapid growth, with REVUFORJ setting the foundation and a strong pipeline of innovative, genetically targeted therapies on the horizon. With high levels of unmet need, particularly in NPM1-mutant and KMT2A-rearranged AML, this class is expected to play a critical role in the next generation of precision oncology therapies for hematologic malignancies.

Menin Drugs Uptake

This section focuses on the uptake rate of potential emerging Menin expected to be launched in the market during 2025-2034.

Menin Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I. It also analyzes key players involved in developing targeted therapeutics.

The presence of numerous drugs under different stages is expected to generate immense opportunity for Menin inhibitor market growth over the forecasted period.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Menin inhibitor therapies.

KOL Views

To keep up with current and future market trends, we take Industry Experts' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on Menin Inhibitor's evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, drug uptake, along challenges related to accessibility.

DelveInsight's analysts connected with 15+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as Johns Hopkins Sidney Kimmel Cancer Center, UCSF Health, Memorial Sloan Kettering Cancer Center, , Roswell Park Comprehensive Cancer Center, Edinburgh Cancer Research UK Centre at the University of Edinburgh, Division of Hematology & Oncology, University of Illinois Health, Memorial Sloan Kettering Cancer Center, Oncology Department at San Luigi Hospital Center for Thoracic Cancers at the Massachusetts General Hospital, Dana-Farber Brigham Cancer Center, and others.

Their opinion helps understand and validate current and emerging therapy treatment patterns or Menin market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy. In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in event-free survival, one of the most important primary outcome measures is event-free survival and overall survival.

Further, the therapies' safety is evaluated, wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

Reimbursement may be referred to as the negotiation of a price between a manufacturer and payer that allows the manufacturer access to the market. It is provided to reduce the high costs and make the essential drugs affordable. Health technology assessment (HTA) plays an important role in reimbursement decision-making and recommending the use of a drug. These recommendations vary widely throughout the seven major markets, even for the same drug.

In the US healthcare system, both Public and Private health insurance coverage are included. Also, Medicare and Medicaid are the largest government-funded programs in the US. The major healthcare programs including Medicare, Medicaid, the Children's Health Insurance Program (CHIP), and the state and federal health insurance marketplaces are overseen by the Centers for Medicare & Medicaid Services (CMS). Other than these, Pharmacy Benefit Managers (PBMs), and third-party organizations that provide services and educational programs to aid patients are also present.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Key Updates on Menin Inhibitors

• In June 2025, Johnson & Johnson announced new Phase Ib data showing encouraging antileukemic activity and a promising safety profile for bleximenib (JNJ-75276617) in combination with venetoclax and azacitidine (VEN + AZA) for the treatment of acute myeloid leukemia (AML) harboring KMT2A gene rearrangements (KMT2Ar) or NPM1 gene mutations (NPM1m).
• In June 2025, Syndax presented new REVUFORJ data in relapsed/refractory mNPM1 and NUP98r Acute Leukemia from AUGMENT-101 trial at EHA 2025.
• In June 2025, Kura oncology and kyowa kirin report positive updated combination data for ziftomenib in newly diagnosed AML at 2025 European Hematology Association Congress.
• In July 2024, Sumitomo Pharma announced that the US FDA granted Fast Track designation to DSP-5336 for the treatment of patients with relapsed or refractory AML with a KMT2A rearrangement, also known as, mixed lineage leukemia rearrangement (MLLr) or nucleophosmin mutation (NPM1m).
• The abstract list is not exhaustive, will be provided in the final report

Scope of the Report:

• The report covers a segment of key events, an executive summary, and a descriptive overview of the Menin, explaining its mechanism, and therapies (emerging).
• Comprehensive insight into the Competitive Landscape, and forecasts, the future growth potential of treatment rate, drug uptake, and drug information have been provided.
• Additionally, an all-inclusive account of emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current landscape.
• A detailed review of the Menin market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis, expert insights/KOL views, and treatment preferences that help shape and drive the 7MM Menin market.

Menin Inhibitor Report Insights

• Menin Targeted Patient Pool
• Therapeutic Approaches
• Menin Pipeline Analysis
• Menin Market Size and Trends
• Existing and Future Market Opportunity

Menin Inhibitor Report Key Strengths

• Ten years Forecast
• The 7MM Coverage
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

Menin Inhibitor Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint)

Key Questions:

• What was the Menin inhibitor total market size, the market size by therapies, market share (%) distribution, and what would it look like in 2034? What are the contributing factors for this growth?
• Which drug is going to be the largest contributor in 2034?
• Which is the most lucrative market for Menin Inhibitor?
• What are the risks, burdens, and unmet needs of treatment with Menin -based/targeting therapies? What will be the growth opportunities across the 7MM for the patient population of Menin -based/targeting therapies?
• What are the key factors hampering the growth of the Menin Inhibitor market?
• What are the indications for which recent novel therapies and technologies have been developed to overcome the limitations of existing treatments?
• What key designations have been granted to the therapies for Menin inhibitors?
• What is the cost burden of approved therapies on the patient?
• Patient acceptability in terms of preferred therapy options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies?

Reasons to buy:

• The report will help develop business strategies by understanding the latest trends and changing dynamics driving the Menin Inhibitor Market.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain) the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of indication-wise current and emerging therapies under the conjoint analysis section to provide visibility around leading indications.
• Highlights of Access and Reimbursement policies of approved therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.