10 Game-changing Companies in Acute Myeloid Leukemia Drug Development in 2026

Acute myeloid leukemia (AML) is a serious blood cancer that mostly affects adults. In the United States alone, there are an estimated 22,010 new AML cases and about 11,090 deaths in 2025. (Source: www.cancer.gov)

Globally, the burden of AML has grown: incident cases rose from around 79,372 in 1990 to 144,646 in 2021, an increase of over 80% (Source: pubmed.ncbi.nlm.nih.gov). However, even with progress, survival remains poor: in the U.S., the 5-year survival rate is only about 31.9 percent for AML. (Source: www.cancer.gov)

AML is also strongly age-dependent; the median age of diagnosis is about 68 years. (Source: pubmed.ncbi.nlm.nih.gov)

These numbers highlight why developing effective AML drugs is so urgent. Many patients have limited treatment options, especially older adults or those with aggressive or relapsed disease. In response, a large and growing number of biopharma companies are investing in innovative therapies, from targeted small molecules to immunotherapies, to improve survival and quality of life for AML patients.

Projections indicate the global acute myeloid leukemia treatment market to cross a value of 5 billion by 2030. In this blog, we will explore ten companies leading the charge in AML drug development and their groundbreaking contributions to treatment.

Top 10 Companies Driving Innovation in the Acute Myeloid Leukemia Treatment Market

1. Amgen Inc.

Amgen Inc., a biotechnology giant, has made significant strides in the treatment of acute myeloid leukemia. One of its key drugs is Blincyto (blinatumomab), a bispecific T-cell engager (BiTE) designed to help the immune system target leukemia cells. Initially approved for acute lymphoblastic leukemia (ALL), Amgen is now exploring its potential in AML. The company is also investing in next-generation immunotherapies and antibody-drug conjugates for AML. It enters into collaborations with smaller biotech firms to enhance its oncology pipeline, particularly focusing on therapies that exploit genetic vulnerabilities in AML cells.

2. Novartis AG

Novartis AG has been a pioneer in precision medicine for hematological cancers. Its flagship AML drug, Rydapt (midostaurin), is approved for FLT3-mutated AML, a subset of the disease with poor prognosis. Rydapt marked a breakthrough as the first targeted therapy for FLT3 mutations. The company is also involved in developing combination therapies involving Rydapt and other agents to improve survival rates. Additionally, the company is exploring CAR-T cell therapies as a treatment option for AML.

3. Jazz Pharmaceuticals plc

Jazz Pharmaceuticals focuses on niche oncology treatments, including AML. Its key product is Vyxeos (CPX-351), a drug designed specifically for older adults with high-risk AML. Vyxeos has demonstrated superior efficacy compared to standard chemotherapy regimens. In a pivotal study, Vyxeos demonstrated a median overall survival of approximately 9.5 months compared to about 5.9 months for the standard chemotherapy regimen. Recently, Jazz has expanded clinical trials to evaluate Vyxeos in combination with other targeted agents, aiming to broaden its application across different AML subtypes.

In March 2021, Jazz Pharmaceuticals received U.S. Food and Drug Administration (FDA) approval for Vyxeos (daunorubicin + cytarabine) to treat newly diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in pediatric patients aged one year and older.

4. Ryvu Therapeutics

Ryvu Therapeutics specializes in innovative small-molecule therapies targeting cancer pathways. Its lead candidate for AML is RVU120, a selective CDK8/19 inhibitor that modulates transcriptional regulation in leukemia cells. The company has reported promising preclinical data showing RVU120’s potential to overcome resistance mechanisms associated with current treatments. Ryvu is also investigating novel biomarkers to optimize patient selection for its therapies.

In September 2023, Ryvu Therapeutics announced that its global licensee, Menarini Group, will launch a Phase II trial of the investigational dual PIM/FLT3 kinase inhibitor dapolsertib (also known as MEN1703/SEL24) in patients with relapsed or refractory Diffuse Large B‑Cell Lymphoma (DLBCL). The study will evaluate dapolsertib both as a monotherapy and in combination with standard-of-care therapy.

5. AbbVie Inc.

AbbVie Inc. developed Venclexta (venetoclax), a BCL-2 inhibitor that promotes apoptosis in leukemia cells. Venclexta is approved in combination with azacitidine or decitabine for newly diagnosed AML patients who are not eligible for intensive chemotherapy. The company is also exploring the use of Venclexta in combination with other targeted agents to improve outcomes for patients with relapsed AML.

In April 2025, AbbVie announced the introduction of its oral BCL-2 inhibitor Venetoclax into the Indian oncology market, targeting treatment for Acute Myeloid Leukemia (AML) and Chronic Lymphocytic Leukemia (CLL). The launch aims to provide a new option for patients ineligible for intensive chemotherapy.

6. Johnson & Johnson Services, Inc.

Johnson & Johnson’s Janssen division develops novel therapies for hematologic malignancies, including AML. The company does not have an approved drug specifically for AML; however, it conducts research into bispecific antibodies and CAR-T cell therapies targeting antigens expressed on AML cells. Janssen adopts cutting-edge technologies like gene editing and immune modulation for AML treatment.

7. F. Hoffmann-La Roche Ltd

Hoffmann-La Roche Ltd provides groundbreaking drugs and diagnostics that have reshaped cancer care globally. It has developed diagnostic tools that identify genetic mutations driving the disease. The company focuses on advancing personalized medicine approaches using biomarkers to tailor treatments for AML more effectively.

8. Otsuka Pharmaceutical Co., Ltd.

Otsuka Pharmaceutical has made significant contributions through its development of Dacogen (decitabine), a hypomethylating agent used primarily in older adults with AML who cannot tolerate intensive chemotherapy. Otsuka continues to explore combination regimens involving Dacogen and targeted therapies to enhance efficacy while minimizing toxicity.

9. Bristol-Myers Squibb Company

Bristol-Myers Squibb (BMS) is renowned for its immuno-oncology expertise. The company is actively researching immune checkpoint inhibitors like anti-PD-1 and anti-CTLA-4 antibodies for use in AML treatment. It focuses on combining immune checkpoint inhibitors with chemotherapy or targeted agents to overcome immune evasion mechanisms employed by leukemia cells.

10. Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a biopharmaceutical company that develops radiopharmaceuticals targeting leukemia cells. It is currently developing a product called Actimab-Athat treats patients with relapsed AML. Actimab-A is being explored in combination with other therapies.

Conclusion

In sum, AML remains a high-need area in oncology, given its growing incidence, poor long-term survival, and biological complexity. To tackle this, companies across the pharmaceutical and biotech spectrum are making strong commitments. They are developing therapies that target specific mutations (like IDH, FLT3, or NPM1), harness the immune system, or improve standard chemotherapy regimens. Many are conducting clinical trials, forming partnerships, and driving research into novel mechanisms of action. These collective efforts are essential to expanding treatment options, increasing remission rates, and ultimately working toward longer, healthier lives for people with AML.