The landscape of acute myeloid leukemia (AML) treatment is undergoing a significant transformation, with over 120 pipeline therapies currently in development by more than 110 companies worldwide, according to a comprehensive report by DelveInsight.
AML, a fast-growing cancer of the blood and bone marrow, primarily affects adults but can also occur in children. The disease develops when immature blood cells called myeloblasts multiply uncontrollably, leading to symptoms such as fatigue, frequent infections, easy bruising, bleeding, bone pain, and weight loss.
Pipeline Overview and Key Players
The robust pipeline reflects the urgent need for more effective treatments for this aggressive form of leukemia. Leading pharmaceutical and biotechnology companies at the forefront of AML research include GlycoMimetics, BioSight, Merck Sharp & Dohme LLC, Kura Oncology, Senti Biosciences, Chimerix, Takeda, and Gilead Sciences, among others.
"The diversity of approaches being explored is remarkable," said a spokesperson from DelveInsight. "We're seeing everything from targeted small molecules to advanced cell therapies, all aimed at improving outcomes for AML patients."
Promising Therapies in Late-Stage Development
Several promising candidates are advancing through clinical trials:
Uproleselan (GlycoMimetics) - Currently in Phase III, this E-selectin antagonist is designed to be used in combination with chemotherapy. By binding to E-selectin, uproleselan pushes AML cells out of their protective niche, blocks cellular communication signals that promote survival, and sensitizes cancer cells to the toxic effects of chemotherapy.
In May 2024, GlycoMimetics released key findings from its Phase III clinical trial evaluating uproleselan in combination with either mitoxantrone, etoposide, and cytarabine (MEC) or fludarabine, cytarabine, and idarubicin (FAI) chemotherapy regimens for relapsed/refractory AML.
Aspacytarabine (BST-236) (BioSight) - In Phase II development, this novel anti-metabolite consists of cytarabine covalently bound to asparagine. Acting as a pro-drug of cytarabine, it enables delivery of high cytarabine doses to leukemia patients with lower systemic exposure to the free drug.
Ziftomenib (Kura Oncology) - This oral investigational drug candidate targets the menin-KMT2A/MLL protein-protein interaction for treatment of genetically defined AML patients. Currently in Phase I/II trials, ziftomenib has received Orphan Drug Designation from the FDA.
Innovative Approaches and Recent Developments
The pipeline includes several innovative approaches to treating AML:
SENTI-202 (Senti Biosciences) - This first-in-class Logic-gated CAR-NK product is engineered with OR and NOT Logic Gate gene circuits to enhance therapeutic efficacy and safety. It features a dual targeting activating CAR that recognizes both CD33 and FLT3 tumor antigens to improve anti-tumor activity.
Recent developments in the AML treatment landscape include:
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In February 2025, Moleculin Biotech received approval from Ukraine's Ministry of Health to initiate patient enrollment for its Phase III MIRACLE trial, evaluating annamycin with cytarabine for AML treatment.
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In October 2024, Qurient Co. Ltd. received FDA approval for its IND application for adrixetinib (formerly Q702), an oral selective inhibitor targeting AXL/MER/CSF1R kinases. The Phase 1 study will evaluate adrixetinib as monotherapy and in combination with venetoclax and azacitidine for relapsed or refractory AML.
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In September 2024, Rigel Pharmaceuticals announced the enrollment of the first participant in a Phase Ib/II clinical trial evaluating REZLIDHIA (olutasidenib) in combination with decitabine and venetoclax for AML patients with mutated isocitrate dehydrogenase-1 (mIDH1).
Therapeutic Approaches and Administration Routes
The AML pipeline includes diverse therapeutic approaches, with drugs categorized by various routes of administration:
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
Molecule types in development include:
- Recombinant fusion proteins
- Small molecules
- Monoclonal antibodies
- Peptides
- Polymers
- Gene therapies
Market Drivers and Barriers
Several factors are driving growth in the AML treatment market, including rising incidence of the disease, advancements in pharmacology and molecular biology promoting drug development, and an aging global population.
However, challenges remain, including the high cost associated with AML management, side effects of treatments, and other factors that could impede market growth.
Looking Ahead
As research continues to advance, the future of AML treatment looks increasingly promising. The diverse pipeline of therapies targeting various aspects of the disease's biology offers hope for improved outcomes for patients with this challenging form of leukemia.
"The level of innovation we're seeing in AML research is unprecedented," noted a clinical researcher involved in several AML trials. "With so many approaches being explored simultaneously, we're optimistic that the next few years will bring significant improvements in how we treat this disease."
For patients with AML and their families, the robust pipeline represents hope for more effective, less toxic treatments that could potentially transform this life-threatening disease into a more manageable condition.
