Blood Cancer United Expands its Beat AML® Master Clinical Trial Advancing Next-Generation Treatment

WASHINGTON, Jan. 14, 2026 /PRNewswire/ -- Blood Cancer United®, formerly The Leukemia & Lymphoma Society, announced today that the first patient has received treatment in a new sub-study of its Beat AML® Master Clinical Trial (Beat AML).

In collaboration with AVEO Oncology, an LG Chem company, ("AVEO") the new sub-study is evaluating ficlatuzumab, a monoclonal antibody targeting hepatocyte growth factor (HGF), in combination with venetoclax and azacitidine in adults 60 years and older with newly diagnosed, untreated acute myeloid leukemia (AML).

The new sub-study is open at the University of California San Francisco and The University of Kansas Medical Center.

"The shift five years ago to venetoclax plus azacitidine as the standard of care for older adults with AML was practice-changing, but most patients still relapse, and long-term outcomes remain poor," said Blood Cancer United Chief Scientific Officer Lore Gruenbaum, Ph.D. "That's why we are actively investigating new triplet therapies like in this new sub-study. These combination therapies have the potential to improve survival and quality of life."

New Triplet Sub-Study Builds on Beat AML Progress

The latest sub-study is one of four that is currently open and is part of the Beat AML Master Clinical Trial, which tests multiple treatments for different subtypes under one master umbrella. Beat AML uses genomic testing to match patients with the most promising targeted treatment based on their unique genetic mutations.

To date, more than 1,850 patients have received genomic testing within seven days of diagnosis, and more than 650 have enrolled in one of the Beat AML precision treatment sub-studies. Fourteen clinical sites are open in the United States, including recent additions at the University of Pittsburgh Medical Center and West Virgina University.

Beat AML unites leading blood cancer experts, pharmaceutical partners, genomic testing firms, data specialists, clinical trial sites and regulators to break down silos and accelerate patient-focused discoveries.

Two other sub-studies have recently achieved key milestones:

• Revumenib-Based Triplet Shows Strong Responses: An early phase Beat AML sub-study found that adding the menin inhibitor revumenib to ven/aza produced high rates of complete remission. The trial enrolled adults 60 and older with NPM1 mutations or KMT2A rearrangements. In the phase 1 trial, patients with KMT2A-rearranged AML who received the triplet combination had a median overall survival of 15.5 months. Historically, treatment with venetoclax and azacitidine alone results in a median overall survival of 2.5 months for these patients.
• Optimizing Shorter Treatment Cycles for Older AML Patients: Launched in June 2023, Opti-AML, a Beat AML sub-study, is studying whether a shorter duration of venetoclax treatment—14 days compared to the FDA-approved 28 days— combined with azacitidine can achieve a similar complete remission rate in newly diagnosed AML patients age 60 and older. The shorter duration is expected to speed blood count recovery, reduce infections, and lower the frequency of hospitalizations and blood transfusions. Patient enrollment in Opti-AML was completed in September 2025 with results expected in 2026.

"Beat AML continues to exceed expectations and has evolved into what it is today, an extensive network of innovative therapeutic trials," says John Byrd, M.D., Beat AML Chief Medical Officer and UPMC Hillman Cancer Center Director. "Patients enrolled in a Beat AML treatment trial have achieved improved survival and better quality of life compared to those who received standard-of-care chemotherapy. As we approach 10 years, the master trial continues to advance the frontier of precision medicine in AML."

About Blood Cancer United^® and the Beat AML^® Master Clinical Trial
Blood Cancer United (formerly The Leukemia & Lymphoma Society) is the largest global nonprofit focused on blood cancer patient support, research, and advocacy. The organization's mission is to cure blood cancer and improve the quality of life of all patients and their families. To achieve it, Blood Cancer United brings together a community of people—patients and their families, volunteers, healthcare providers, scientists, staff, partners, fundraisers, and philanthropists—who believe all blood cancer patients deserve longer, fuller lives. For support and to learn more, visit www.BloodCancerUnited.org.

The Beat AML® Master Clinical Trial (NCT03013998), launched in 2016 by Blood Cancer United, is the first collaborative precision medicine clinical trial in a blood cancer. The trial uses advanced genomic technology to match patients to the most promising targeted treatment based on their unique genetic mutations. The trial tests multiple therapies in multiple sub-studies simultaneously and has already generated strong results, showing superior survival rates and better quality of life. Over the past decade, Beat AML has grown into a powerful ecosystem of pharma companies, genetic testing experts, data specialists, regulatory agencies, and other industry partners working toward better and safer treatments for patients with AML. For more information, visit www.bloodcancerunited.org/beataml

Media Contact:
Ryan McDonald
Blood Cancer United
[email protected]

SOURCE Blood Cancer United® formerly The Leukemia & Lymphoma Society