Acute Myeloid Leukemia Pipeline Insight 2025: Akeso's FDA ODD for Ligufalimab, 100+ Companies Advancing Targeted, Immune, and Cell-Based Therapies | DelveInsight

The acute myeloid leukemia (AML) treatment pipeline is undergoing a profound transformation, with innovative therapies advancing beyond traditional chemotherapy toward precision, immune-based, and regenerative approaches. AML, an aggressive hematologic malignancy, continues to present high relapse rates and poor long-term survival despite decades of therapeutic progress, underscoring the urgent unmet need.
DelveInsight's "Acute Myeloid Leukemia - Pipeline Insight, 2025 [https://www.delveinsight.com/report-store/acute-myeloid-leukemia-aml-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=jpr]" provides a comprehensive analysis of a rapidly expanding portfolio of over 100+ companies actively developing therapies for AML. Targeted therapies such as FLT3, IDH1/2, and menin inhibitors are delivering more precise, mutation-driven treatment strategies with improved tolerability. Immuno-oncology approaches-including monoclonal antibodies, bispecific T-cell engagers, and antibody-drug conjugates-are emerging as cornerstones of therapy, aiming to enhance durable remission and overcome resistance. Meanwhile, cell-based therapies, including CAR-T and allogeneic NK-cell products, are being explored to harness the immune system against refractory AML.

In addition, novel small molecules modulating apoptosis pathways, epigenetic regulators, and next-generation hypomethylating agents are showing promise in early and mid-stage trials. Digital health platforms and real-world evidence integration are also increasingly shaping AML trial designs and treatment personalization.

In September 2025, Akeso Inc. (9926.HK) announced that its proprietary next-generation humanized IgG4 monoclonal antibody targeting CD47, ligufalimab (AK117), received Orphan Drug Designation (ODD) from the FDA for the treatment of AML, underscoring the growing momentum of immune checkpoint-based strategies in the disease.

With rising research investments, regulatory incentives such as ODD and Breakthrough Therapy designations, and expanding biomarker-driven approaches, the 2025 AML pipeline reflects a paradigm shift-moving from broad cytotoxic regimens toward curative, precision-driven, and combination strategies designed to improve survival outcomes and quality of life.

Interested in learning more about the current treatment landscape and the key drivers shaping the Acute Myeloid Leukemia pipeline? Click here [https://www.delveinsight.com/report-store/acute-myeloid-leukemia-aml-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=jpr]

Key Takeaways from the Acute Myeloid Leukemia Pipeline Report

- DelveInsight's acute myeloid leukemia pipeline analysis depicts a strong space with 100+ active players working to develop 110+ pipeline drugs for acute myeloid leukemia treatment.

- The leading Acute Myeloid Leukemia companies include GlycoMimetics, BioSight, Merck Sharp & Dohme LLC, Kura Oncology, Senti Biosciences, Chimerix, Takeda, Apollo Therapeutics, Immune-Onc Therapeutics, AbbVie/Genentech, Altor BioScience Corporation, Merck & Co, Astex Pharmaceuticals, Karyopharm Therapeutics, Mesoblast, SELLAS Life Sciences Group, Molecular Partners, Kling Biotherapeutics, HitGen, Chordia Therapeutics, Inc, Cullinan Therapeutics Inc., Aptose Biosciences Inc., Celgene, and others are evaluating their lead assets to improve the Acute Myeloid Leukemia treatment landscape.

- Key Acute Myeloid Leukemia pipeline therapies in various stages of development include Uproleselan, BST-236, MK-0482, Ziftomenib, SENTI 202, Dordaviprone, ADCLEC.syn1 CAR-T cell therapy, APL 4098, IO 202, Venetoclax, Nogapendekin alfa inbakicept, Pembrolizumab, Azacitidine/cedazuridine, Selinexor, Rexlemestrocel L - Mesoblast/Teva Pharmaceutical Industries, Galinpepimut S, cKITxCD16axCD47 fusion protein therapeutic, KBA 1331, HGP 2514, CTX-712, CLN-049, CG-806, CC-96191, and others.

- In September 2025, Akeso Inc. (9926.HK) announced that its CD47-targeting antibody, ligufalimab (AK117), received Orphan Drug Designation from the U.S. FDA for treating acute myeloid leukemia (AML).

- In July 2025, ImCheck Therapeutics announced that the FDA granted Orphan Drug Designation to ICT01, their humanized anti-BTN3A monoclonal antibody targeting 92 T cells, for treating acute myeloid leukemia (AML). AML is especially challenging to treat in older or unfit patients ineligible for intensive chemotherapy.

- In July 2025, the FDA accepted the supplemental new drug application for INQOVI (decitabine and cedazuridine) plus venetoclax to treat adults with newly diagnosed acute myeloid leukemia (AML) who cannot undergo intensive chemotherapy. The FDA set a standard review with a decision expected by February 25, 2026. This is based on Phase 2b study results from ASCERTAIN-V.

- In July 2025, Syndax Pharmaceuticals announced that the FDA granted Priority Review for its supplemental New Drug Application (sNDA) for Revuforj (revumenib), targeting relapsed or refractory mutant NPM1 acute myeloid leukemia (AML). The FDA's Real-Time Oncology Review program is expediting the process, with a PDUFA action date set for October 25. Revuforj is an oral menin inhibitor initially approved in 2024 for acute leukemia with KMT2A translocation.

- In June 2025, Moleculin Biotech received FDA agreement on a single pediatric approval study to evaluate Annamycin combined with Cytarabine as a second-line treatment for pediatric relapsed/refractory acute myeloid leukemia (R/R AML).

Acute Myeloid Leukemia Overview

Acute Myeloid Leukemia (AML) is a fast-growing cancer of the blood and bone marrow characterized by the abnormal proliferation of immature white blood cells (myeloblasts). It interferes with normal blood cell production, leading to symptoms such as fatigue, frequent infections, and easy bruising or bleeding. AML primarily affects older adults but can occur at any age. The disease progresses rapidly and requires prompt treatment, which may include chemotherapy, targeted therapy, or stem cell transplantation. Advances in diagnostics and emerging targeted therapies continue to improve outcomes for AML patients.

Find out more about Acute Myeloid Leukemia medication at https://www.delveinsight.com/report-store/acute-myeloid-leukemia-aml-pipeline-insight [https://www.delveinsight.com/report-store/acute-myeloid-leukemia-aml-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=jpr]

Acute Myeloid Leukemia Treatment Analysis: Drug Profile

Ziftomenib: Kura Oncology

Ziftomenib is a novel, oral, once-daily investigational therapy targeting the menin-KMT2A/MLL protein-protein interaction for genetically defined AML patients with significant unmet needs. In preclinical studies, it demonstrated inhibition of the KMT2A/MLL complex, leading to reduced HOXA9/MEIS1 expression and strong anti-leukemic effects. Ziftomenib has received Orphan Drug Designation from the U.S. FDA and is currently in Phase III clinical trials for the treatment of AML.

Annamycin: Moleculin Biotech, Inc.

Annamycin is a next-generation anthracycline being evaluated in the Phase III MIRACLE trial for relapsed or refractory AML, in combination with cytarabine (AnnAraC regimen). It has shown a lack of cardiotoxicity in multiple clinical trials, potentially allowing for higher cumulative dosing compared to traditional anthracyclines. Annamycin is also under development for soft tissue sarcoma and holds promise as a transformative option for second-line AML treatment.

SNDX-5613 (Revumenib): Syndax Pharmaceuticals

Revumenib is an oral, selective menin-KMT2A (MLL) interaction inhibitor in development for AML with KMT2A rearrangements or NPM1 mutations. It works by disrupting the menin-KMT2A complex, restoring normal gene expression and promoting leukemic cell differentiation. Revumenib has shown encouraging results in early-phase trials for relapsed/refractory AML and has received Breakthrough Therapy and Orphan Drug designations from the FDA. It is currently in Phase II/III development for AML.

BST-236 (Aspacytarabine): BioSight

BST-236 is a novel cytarabine-based prodrug, chemically linked to asparagine to enable high-dose cytarabine delivery with reduced systemic exposure. Designed to improve safety and efficacy in AML patients, it is currently in Phase II clinical trials for the treatment of acute myeloid leukemia.

Learn more about the novel and emerging Acute Myeloid Leukemia pipeline therapies [https://www.delveinsight.com/report-store/acute-myeloid-leukemia-aml-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=jpr].

Acute Myeloid Leukemia Therapeutics Assessment

By Product Type

- Mono

- Combination

- Mono/Combination.

By Stage

- Late-stage products (Phase III)

- Mid-stage products (Phase II)

- Early-stage product (Phase I) along with the details of

- Pre-clinical and Discovery stage candidates

- Discontinued & Inactive candidates

By Route of Administration

- Oral

- Intravenous

- Subcutaneous

- Parenteral

- Topical

By Molecule Type

- Recombinant fusion proteins

- Small molecule

- Monoclonal antibody

- Peptide

- Polymer

- Gene therapy

Scope of the Acute Myeloid Leukemia Pipeline Report

- Coverage: Global

- Key Acute Myeloid Leukemia Companies: GlycoMimetics, BioSight, Merck Sharp & Dohme LLC, Kura Oncology, Senti Biosciences, Chimerix, Takeda, Apollo Therapeutics, Immune-Onc Therapeutics, AbbVie/Genentech, Altor BioScience Corporation, Merck & Co, Astex Pharmaceuticals, Karyopharm Therapeutics, Mesoblast, SELLAS Life Sciences Group, Molecular Partners, Kling Biotherapeutics, HitGen, Chordia Therapeutics, Inc, Cullinan Therapeutics Inc., Aptose Biosciences Inc., Celgene, and others.

- Key Acute Myeloid Leukemia Pipeline Therapies: Uproleselan, BST-236, MK-0482, Ziftomenib, SENTI 202, Dordaviprone, ADCLEC.syn1 CAR-T cell therapy, APL 4098, IO 202, Venetoclax, Nogapendekin alfa inbakicept, Pembrolizumab, Azacitidine/cedazuridine, Selinexor, Rexlemestrocel L - Mesoblast/Teva Pharmaceutical Industries, Galinpepimut S, cKITxCD16axCD47 fusion protein therapeutic, KBA 1331, HGP 2514, CTX-712, CLN-049, CG-806, CC-96191, and others.

Explore detailed insights on drugs used in the treatment of Acute Myeloid Leukemia here [https://www.delveinsight.com/report-store/acute-myeloid-leukemia-aml-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=jpr].

Table of Contents

1. Introduction

2. Executive Summary

3. Acute Myeloid Leukemia Pipeline: Overview

4. Analytical Perspective In-depth Commercial Assessment

5. Acute Myeloid Leukemia Pipeline Therapeutics

6. Acute Myeloid Leukemia Pipeline: Late-Stage Products (Phase III)

7. Acute Myeloid Leukemia Pipeline: Mid-Stage Products (Phase II)

8. Acute Myeloid Leukemia Pipeline: Early Stage Products (Phase I)

9. Therapeutic Assessment

10. Inactive Products

11. Company-University Collaborations (Licensing/Partnering) Analysis

12. Key Companies

13. Key Products

14. Unmet Needs

15. Market Drivers and Barriers

16. Future Perspectives and Conclusion

17. Analyst Views

18. Appendix

About DelveInsight

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