Apremilast

Phase 3 Study Results Demonstrated Three Year, Disease-Free Survival of 96% THOUSAND OAKS, Calif. , Dec. 7, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced new data demonstrating that adding BLINCYTO ® (blinatumomab) to chemotherapy significantly improves disease-free survival (DFS) in newly diagnosed pediatric patients with National Cancer Institute (NCI) standard risk (SR) B-cell acute lymphoblastic leukemia (B-ALL) of average or higher risk of relapse. The data are from a Phase 3 study (AALL1731) conducted by the Children's Oncology Group . The results were simultaneously published in the New England Journal of Medicine and will be presented during the plenary session on Sunday, Dec. 8 , at 2 p.m. PT at the 66 th American Society of Hematology (ASH) Annual Meeting & Exposition in San Diego . "Over the last decade, BLINCYTO has reshaped the treatment landscape for B-ALL, offering a critical lifeline for thousands of adult and pediatric patients," said Jay Bradner , M.D., executive vice president of Research and Development and chief scientific officer at Amgen . "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. We are grateful to the Children's Oncology Group , along with the patients, families and clinical teams, for their dedication and partnership in advancing this critical study to improve the lives of children with cancer." Based on the results of the first pre-specified interim analysis for efficacy, the study met its primary endpoint of DFS and study randomization was terminated early based on the recommendation from the data and safety monitoring committee due to the benefit observed in the BLINCYTO arm compared to the chemotherapy-only arm. Overall, the 3-year DFS 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 0.39 [95% confidence interval (CI) 0.24-0.64], indicating a 61% reduction in the risk of disease relapse, secondary malignant neoplasm or remission death with BLINCYTO. At 3 years, more patients remained alive and cancer free when treated with BLINCYTO plus chemotherapy compared to chemotherapy alone. "The AALL1731 study results are truly practice-changing, further solidifying blinatumomab's role as the standard of care for a large number of children with B-ALL," said Sumit Gupta , M.D., Ph.D., FRCPC, co-chair of the Children's Oncology Group AALL1731 study and oncologist and clinician investigator, Division of Haematology/Oncology at The Hospital for Sick Children ( SickKids ) and associate professor of pediatrics at the University of Toronto . "These breakthrough data showing a significant improvement in disease-free survival are poised to bring substantial clinical value to children with newly diagnosed B-ALL." The addition of BLINCYTO to chemotherapy in standard risk patients resulted in outcomes similar to those previously achieved in only the most favorable pediatric risk subsets. Among SR-Average patients, 3-year DFS was 97.5% for patients treated with BLINCYTO compared to 90.2% for those treated with only chemotherapy (HR 0.33, CI 0.15-0.69). For SR-High patients, 3-year DFS was 94.1% for those treated with BLINCYTO compared to 84.8% for those treated with only chemotherapy (HR 0.45, 95% CI 0.24-0.85). "Relapsed ALL remains a major cause of pediatric cancer mortality, with nearly half of the relapses occurring in children with standard-risk B-ALL," said Rachel E. Rau , M.D., co-chair of the Children's Oncology Group AALL1731 study, pediatric hematologist-oncologist at Seattle Children's Hospital and associate professor of pediatrics at the University of Washington . "These findings underscore the progress made with blinatumomab in preventing relapse and support its role as a critical addition to current therapeutic strategies." Safety results are consistent with the known safety profile of BLINCYTO. BLINCYTO has demonstrated a positive balance of benefits and risks, with only 0.3% of first courses associated with Grade 3+ cytokine release syndrome (CRS) and 0.7% with seizures. A higher risk of infections was observed in the BLINCYTO arm. These results provide the first evidence supporting BLINCYTO for use in the consolidation phase in newly diagnosed pediatric Philadelphia chromosome-negative (Ph-) B-ALL patients. This groundbreaking first-in-class Bispecific T-cell Engager (BiTE ® ) therapy is now backed by additional evidence reinforcing its role in redefining a standard of care for both adult and pediatric patients, starting from one month old, regardless of measurable residual disease (MRD) status. The findings further establish BLINCYTO as a versatile first-line consolidation therapy across all ages and treatment backbones. 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 relating to the trial. About The Children's Oncology Group The Children's Oncology Group ( childrensoncologygroup.org ), a member of the NCI National Clinical Trials Network (NCTN), is the world's largest organization devoted exclusively to childhood and adolescent cancer research. The Children's Oncology 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. Today, more than 80% of the 15,000 children and adolescents diagnosed with cancer each year in the United States are cared for at Children's Oncology Group member institutions. Research performed by Children's Oncology Group institutions over the past 50 years has transformed childhood cancer from a virtually incurable disease to one with a combined 5-year survival rate of 86%. The Children's Oncology Group's mission is to improve the cure rate and outcomes for all children with cancer. About AALL1731 (NCT03914625) The AALL1731 study was a Phase 3 randomized trial to determine if two non-sequential cycles of BLINCYTO added to chemotherapy improved disease-free survival (DFS) in children with newly diagnosed pediatric National Cancer Institute (NCI) standard risk (SR) B-cell acute lymphoblastic leukemia (B-ALL). The study enrolled 4,264 newly diagnosed NCI SR B-ALL patients, of whom 2,334 were risk stratified at the end of induction therapy as either SR-Average or SR-High. At the first planned interim efficacy analysis (data cutoff June 30, 2024 ), 1,440 of the eligible and evaluable patients had been randomized. The AALL1731 study was designed and conducted independently from industry. The Cancer Therapy Evaluation Program (CTEP) of the NCI sponsored the trial and provided funding to the Children's Oncology Group to conduct the study. NCI is part of the National Institutes of Health (NIH). In addition, Amgen provided BLINCYTO and support through an NCI Cooperative Research and Development Agreement. About Acute Lymphoblastic Leukemia (ALL) ALL, also known as acute lymphoblastic leukemia, is a fast-growing type of blood cancer that develops in the bone marrow and can sometimes spread to other parts of the body, including the lymph nodes, liver, spleen and central nervous system. ALL is a rare disease, with an estimated 6,550 new cases, affecting both children and adults, diagnosed in the U.S. in 2024. 1 B-ALL begins in immature cells that would normally develop into B-cell lymphocytes, which are white blood cells that grow in bone marrow. 2,3 B-ALL is the most common type of ALL, constituting approximately 75% of cases in adults and approximately 88% in children, the most common cancer in children. 4,5 About BLINCYTO ® (blinatumomab) BLINCYTO is the first globally approved Bispecific T-cell Engager (BiTE ® ) immuno-oncology therapy that targets CD19 surface antigens on B cells. BiTE ® molecules fight cancer by helping the body's immune system detect and target malignant cells by engaging T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. By bringing T cells near cancer cells, the T cells can inject toxins and trigger cancer cell death (apoptosis). BiTE ® immuno-oncology therapies are currently being investigated for their potential to treat a wide variety of cancers. BLINCYTO was granted Breakthrough Therapy and Priority Review designations by the U.S. FDA and is approved in the U.S. for the treatment of: In the European Union (EU), BLINCYTO is indicated as monotherapy for the treatment of: BLINCYTO ® IMPORTANT SAFETY INFORMATION WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGICAL TOXICITIES including IMMUNE EFFECTOR CELL-ASSOCIATED NEUROTOXICITY SYNDROME Contraindications BLINCYTO ® is contraindicated in patients with a known hypersensitivity to blinatumomab or to any component of the product formulation. Warnings and Precautions Adverse Reactions Dosage and Administration Guidelines INDICATIONS BLINCYTO ® (blinatumomab) is indicated for the treatment of CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL) in adult and pediatric patients one month and older with: Please see BLINCYTO ® full Prescribing Information , including BOXED WARNINGS. About Bispecific T-Cell Engager (BiTE ® ) Technology BiTE technology is a targeted immuno-oncology platform that is designed to engage a patient's own T cells to any tumor-specific antigen, activating the cytotoxic potential of T cells to eliminate detectable cancer. The BiTE immuno-oncology platform has the potential to treat different cancer types through tumor-specific antigens. The BiTE platform has a goal of leading to off-the-shelf solutions, which have the potential to make innovative T-cell treatment available to all providers when their patients need it. For more than a decade, Amgen has been advancing this innovative technology, which has demonstrated strong efficacy in hematological malignancies and now a solid tumor with the approval of IMDELLTRA. Amgen remains committed to progressing multiple BiTE molecules across a broad range of hematologic and solid tumor malignancies, paving the way for additional applications in more tumor types. Amgen is further investigating BiTE technology with the goal of enhancing patient experience and therapeutic potential. To learn more about BiTE technology, visit BiTE ® Technology 101 . About Amgen Amgen discovers, develops, manufactures and delivers innovative medicines to help millions of patients in their fight against some of the world's toughest diseases. More than 40 years ago, Amgen helped to establish the biotechnology industry and remains on the cutting-edge of innovation, using technology and human genetic data to push beyond what's known today. Amgen is advancing a broad and deep pipeline that builds on its existing portfolio of medicines to treat cancer, heart disease, osteoporosis, inflammatory diseases and rare diseases. In 2024, Amgen was named one of the "World's Most Innovative Companies" by Fast Company and one of "America's Best Large Employers" by Forbes, among other external recognitions . Amgen is one of the 30 companies that comprise the Dow Jones Industrial Average ® , and it is also part of the Nasdaq-100 Index ® , which includes the largest and most innovative non-financial companies listed on the Nasdaq Stock Market based on market capitalization. For more information, visit Amgen.com and follow Amgen on X , LinkedIn , Instagram , TikTok , YouTube and Threads . 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CONTACT: Amgen, Thousand Oaks Elissa Snook , 609-251-1407 (media) Justin Claeys , 805-313-9775 (investors) References View original content to download multimedia: https://www.prnewswire.com/news-releases/blincyto-blinatumomab-added-to-chemotherapy-significantly-improves-survival-in-newly-diagnosed-pediatric-patients-with-b-cell-precursor-acute-lymphoblastic-leukemia-b-all-302325381.html SOURCE Amgen