On January 14, 2026, Blood Cancer United (formerly The Leukemia & Lymphoma Society) announced that the first patient has been treated in a new sub-study of its landmark Beat AML® Master Clinical Trial. In collaboration with AVEO Oncology (an LG Chem company), the trial is evaluating ficlatuzumab — a monoclonal antibody targeting hepatocyte growth factor (HGF) — in combination with the current standard of care (venetoclax + azacitidine) in adults aged 60 years and older with newly diagnosed, untreated acute myeloid leukemia (AML).
The sub-study is currently open at two leading U.S. centers:
- University of California San Francisco (UCSF)
- The University of Kansas Medical Center
Why This Matters – The Unmet Need in Older AML Patients
AML remains one of the most aggressive and deadly blood cancers, especially in patients over 60, who represent the majority of new cases. The introduction of venetoclax + azacitidine in 2020 dramatically improved outcomes compared with traditional intensive chemotherapy, raising median overall survival from ~8–10 months to ~14–15 months in fit older patients. Despite this major advance, most patients still relapse within 12–18 months, and long-term survival rates remain poor (often <20 % at 5 years).
Lore Gruenbaum, Ph.D., Chief Scientific Officer of Blood Cancer United, summarized the challenge:
“The shift to venetoclax + azacitidine five years ago was practice-changing, but most patients still relapse and long-term outcomes remain poor. That’s why we are actively investigating new triplet therapies like this one. These combinations have the potential to improve survival and quality of life.”
Ficlatuzumab blocks HGF, a growth factor frequently overexpressed in AML that promotes leukemia cell survival, proliferation, and resistance to therapy. Preclinical data suggest that adding HGF inhibition to ven/aza may deepen responses, delay relapse, and overcome resistance mechanisms.
The Beat AML Master Trial – A Precision-Medicine Powerhouse
Launched in 2016, Beat AML is the world’s first collaborative precision-medicine master trial in a blood cancer. Instead of running separate studies for each drug or mutation, Beat AML uses one overarching protocol with multiple parallel sub-studies. Patients receive rapid genomic testing (within 7 days of diagnosis) and are matched to the most promising targeted therapy based on their individual molecular profile.
Key milestones to date (as of January 2026):
- >1,850 patients have received genomic testing within 7 days
- >650 patients enrolled in one of the precision-treatment sub-studies
- 14 active U.S. clinical sites (recently expanded to include University of Pittsburgh Medical Center and West Virginia University)
Recent sub-study highlights include:
- Revumenib-based triplet (menin inhibitor + ven/aza) in NPM1-mutated or KMT2A-rearranged AML ? very high complete remission rates and a median overall survival of 15.5 months in the KMT2A subgroup (historically ~2.5 months with ven/aza alone)
- Opti-AML (launched 2023, enrollment completed September 2025): testing whether shortening venetoclax duration from 28 to 14 days (combined with azacitidine) maintains efficacy while reducing toxicity, hospitalizations, infections, and transfusions in older patients
Results from Opti-AML are expected in 2026.
Chief Medical Officer Perspective
John Byrd, M.D., Beat AML Chief Medical Officer and Director of UPMC Hillman Cancer Center, commented:
“Beat AML continues to exceed expectations and has evolved into an extensive network of innovative therapeutic trials. Patients enrolled in a Beat AML treatment trial have achieved improved survival and better quality of life compared with standard-of-care chemotherapy. As we approach 10 years, the master trial continues to advance the frontier of precision medicine in AML.”
Bottom Line – Why This Announcement Is Significant
The ficlatuzumab + ven/aza sub-study represents another bold step in Beat AML’s mission to systematically replace “one-size-fits-all” chemotherapy with biology-driven, personalized regimens. If successful, this triplet could become a new standard backbone for older adults with newly diagnosed AML — a population that historically had very limited options and dismal long-term outcomes.
The trial also exemplifies the power of master-protocol designs: multiple promising drugs can be tested simultaneously, patients are rapidly matched to the best available option, and the entire ecosystem (academic centers, pharma partners, genomic labs, regulators) works together under one umbrella.
For patients and physicians, Beat AML remains one of the most important ongoing initiatives in hematologic oncology — and the new ficlatuzumab arm adds another layer of hope for older adults facing this aggressive disease.
