Amgen's Blincyto keeps 96% of pediatric leukemia patients alive and disease-free in 'practice changing' 3-year study

Ten years after its first FDA approval, Amgen’s Blincyto continues to make inroads in treating acute lymphoblastic leukemia. Now, the drug has delivered a phase 3 win that further demonstrates its benefits in the most common form of the disease.

In newly diagnosed pediatric patients with B-cell acute lymphoblastic leukemia (B-ALL) that fits into the National Cancer Institute’s definition of standard risk, the three-year disease-free survival rate for those treated with Blincyto and chemotherapy was 96% compared with 87.9% for chemotherapy alone, Amgen reported Saturday.

The result came from a Phase 3 study called AALL1731 conducted by the Children's Oncology Group.

The data indicate a 61% reduction in the risk of disease relapse, remission, death or secondary malignant neoplasm associated with Blincyto's addition to chemo, according to Amgen's press release.

Based on the recommendation of the data and safety monitoring committee, study randomization was ended early due to the benefit seen in the treatment arm. Blincyto’s addition to chemotherapy in standard-risk patients yielded outcomes similar to those previously found in “only the most favorable pediatric risk subsets,” the company noted.

“Over the last decade, Blincyto has reshaped the treatment landscape for B-ALL, offering a critical lifeline for thousands of adult and pediatric patients," Amgen’s chief scientific officer and head of R&D, Jay Bradner, M.D., explained in a company press release. "These powerful new data leave us little doubt about the profound impact of this medicine for a large number of children affected by this disease.”

The new results are “truly practice-changing,” study co-chair Sumit Gupta, M.D., Ph.D., added in the release. B-ALL is the most common cancer in children and the most common type of ALL, representing approximately 88% of childhood ALL cases. An estimated 6,550 new U.S. cases of ALL were diagnosed in 2024 in both children and adults, according to Amgen.

The study was conducted by the Children’s Oncology Group, the world’s largest organization devoted exclusively to childhood cancer research. NCI's Cancer Therapy Evaluation Program, the trial’s sponsor, will share the data with the FDA as part of “ongoing communications” regarding the study, according to the release.

So far, Blincyto has collected approvals across several settings in CD19-positive B-ALL, including for those one month or older with CD19-positive Philadelphia chromosome-negative B-ALL during the consolidation phase of multiphase therapy. 

The drug is also approved to treat CD19-positive B-ALL that's in first or second complete remission with minimal residual disease at or greater than 0.1%, as well as relapsed or refractory CD19-positive B-ALL.

The bispecific T-cell engager remains a growth driver for Amgen, delivering double-digit sales increases a decade into its launch. During the third quarter of this year, the drug’s sales increased 49% year-over-year to $327 million. Last year, Blincyto brought in $861 million in global sales.

Meanwhile, a new competitor may be on the horizon after Merck’s $700 million purchase of a CD3xCD19 bispecific made by Curon Biopharmaceutical. So far, the drug has demonstrated early evidence of efficacy in ALL.