BLINCYTO® Enhances Survival in Newly Diagnosed Pediatric B-ALL Patients

Amgen recently shared promising new data showcasing the benefits of adding BLINCYTO (blinatumomab) to chemotherapy for pediatric patients newly diagnosed with National Cancer Institute (NCI) standard risk (SR) B-cell acute lymphoblastic leukemia (B-ALL). This Phase 3 study, titled AALL1731, was conducted by the Children's Oncology Group and reveals significant improvements in disease-free survival (DFS) for these young patients. The findings were published in the New England Journal of Medicine and presented at the American Society of Hematology (ASH) Annual Meeting & Exposition.

BLINCYTO has been instrumental in transforming the treatment landscape for B-ALL over the past decade, particularly for both adult and pediatric patients. The latest data underscores the drug's profound impact, offering a critical treatment option for a large number of children affected by this disease. Jay Bradner, M.D., executive vice president of Research and Development and chief scientific officer at Amgen, expressed gratitude towards the Children's Oncology Group, patients, families, and clinical teams for their dedication to advancing this important study.

The study met its primary DFS endpoint based on the first pre-specified interim analysis for efficacy. Consequently, the study's randomization was terminated early due to the observed benefits in the BLINCYTO arm compared to the chemotherapy-only arm. The three-year DFS rate was 96.0% for patients treated with chemotherapy plus BLINCYTO, compared to 87.9% for those treated with only chemotherapy. The hazard ratio (HR) was calculated at 0.39, indicating a 61% reduction in the risk of disease relapse, secondary malignant neoplasm, or remission death with BLINCYTO. More patients were alive and cancer-free at three years with the addition of BLINCYTO to chemotherapy.

Sumit Gupta, M.D., Ph.D., FRCPC, co-chair of the AALL1731 study, emphasized that these results are practice-changing, further establishing blinatumomab as the standard of care for many children with B-ALL. The breakthrough data show a significant improvement in disease-free survival, offering substantial clinical benefits to children with newly diagnosed B-ALL.

In standard risk patients, the addition of BLINCYTO resulted in outcomes similar to those previously achieved in the most favorable pediatric risk subsets. For SR-Average patients, the three-year DFS was 97.5% with BLINCYTO compared to 90.2% with only chemotherapy. For SR-High patients, the three-year DFS was 94.1% with BLINCYTO compared to 84.8% with only chemotherapy.

Rachel E. Rau, M.D., co-chair of the AALL1731 study, highlighted the significance of these findings, noting that relapsed ALL remains a major cause of pediatric cancer mortality. The data underscore the progress made with blinatumomab in preventing relapses and support its role as a critical addition to current therapeutic strategies.

The safety profile of BLINCYTO was consistent with known data, showing a positive balance of benefits and risks. Only 0.3% of first courses were associated with Grade 3+ cytokine release syndrome (CRS), and 0.7% with seizures. A higher risk of infections was noted in the BLINCYTO arm.

These results provide the first evidence supporting the use of BLINCYTO in the consolidation phase for newly diagnosed pediatric Philadelphia chromosome-negative (Ph-) B-ALL patients. This innovative Bispecific T-cell Engager (BiTE) therapy is now reinforced by additional evidence, underlining its role in redefining the standard of care for both adult and pediatric patients, regardless of measurable residual disease (MRD) status.

The NCI's Cancer Therapy Evaluation Program (CTEP), which sponsored the study, will share data with the U.S. Food and Drug Administration as part of their ongoing communications related to the trial.

The Children's Oncology Group, a member of the NCI National Clinical Trials Network, is the world's largest organization dedicated exclusively to childhood and adolescent cancer research. This group unites over 10,000 experts in childhood cancer at more than 200 leading children's hospitals, universities, and cancer centers across North America, Australia, New Zealand, and Saudi Arabia in the fight against childhood cancer. Research by the Children's Oncology Group has significantly improved childhood cancer survival rates over the past 50 years.

The AALL1731 study was a Phase 3 randomized trial to determine if the addition of BLINCYTO to chemotherapy could improve disease-free survival in children with newly diagnosed NCI standard risk B-ALL. The study enrolled 4,264 patients, with 2,334 stratified as SR-Average or SR-High at the end of induction therapy. The first interim efficacy analysis included 1,440 randomized patients.

The trial, sponsored by CTEP of the NCI and funded by the Children's Oncology Group, was independently designed and conducted. Amgen provided BLINCYTO and support through an NCI Cooperative Research and Development Agreement.