Calaspargase Pegol-MKNL

Adolescent and young adult patients treated with asparaginase containing pediatric-inspired regimens showed a 69% reduction in risk of death (HR: 0.31; 95% CI, 0.18-0.55) and higher overall survival compared to matched patients treated with non-pediatric-inspired regimens The two-year rate of stem cell transplant was lower in patients treated with pediatric-inspired regimens compared to patients treated with non-pediatric-inspired regimens BOSTON, Dec. 12, 2022 /PRNewswire/ -- Servier, a leader in oncology committed to bringing the promise of tomorrow to the patients we serve, today announced findings from a retrospective study observing higher overall survival (OS) in adolescent and young adult (AYA) newly-diagnosed acute lymphoblastic leukemia (ALL) patients treated with asparaginase containing pediatric-inspired regimens (PIR) versus non-pediatric-inspired regimens. Detailed results from the new real-world analysis were presented today in an oral session at the 64th Annual Meeting of the American Society of Hematology (ASH). "Pediatric oncologists have long understood the effectiveness of pediatric-inspired regimens for treating children and teenagers with most types of acute lymphoblastic leukemia, but this analysis indicates those benefits extend to a broader population of older adolescents and young adults," said David R. Freyer, DO, MS, study co-author, Co-Director of the AYA Cancer Program at the USC Norris Comprehensive Care Center, and member of the Leukemia Lymphoma Program at Children's Hospital Los Angeles. "Although the use of pediatric-inspired regimens in young adults is inconsistent across adult centers, we believe this analysis supports their use in this patient population and hope it promotes further investigation to better understand their impact on overall survival and long-term outcomes." Study Background: This real-world analysis initially looked at the administrative health claim records of 11,650 newly-diagnosed ALL patients on separate days between 2007-2020. Of those, 2,312 were aged 15-39, and 1,313 had at least one record of a PIR or HyperCVAD or ALL DRG code or other chemotherapy within six months of diagnosis. Patient records were then examined to ensure no evidence of ALL remission or relapse prior to the index date, no evidence of stem cell transplant prior to the index date and at least six months of continuous health plan enrollment before the index date, leaving a total of 596 patients in the analysis. Among the 596 patient records remaining in the analysis, 303 (51%) were confirmed to have not been treated with a PIR, while 187 (31%) were confirmed to have been treated with a PIR. An additional 15% received unknown therapy (due to limited available data) and 3% lacked sufficient information but received treatment that suggests a PIR (and were included in the sensitivity analysis). Non-PIR patients were matched to PIR patients based on propensity score matching to balance the baseline characteristics. A total of 374 matched patients (187 PIR vs. 187 non-PIR) were used in the primary analyses. This is a retrospective study not powered or adjusted for multiplicity to assess OS. P-values are for descriptive purposes only. Findings of sensitivity analyses were consistent with the primary analyses. Results: Overall, this real-world analysis suggests treatment with a PIR leads to higher OS in AYA patients with newly-diagnosed ALL. Patients treated with a PIR (n=187) had a 69% lower risk of death compared to matched patients who received non-PIR (n=187) (hazard ratio [HR]=0.31; 95% confidence interval [CI], 0.18-0.55; log-rank test p-value<0.0001). In addition, the two-year rate of allogeneic stem-cell transplant (SCT) was lower among AYA patients with newly-diagnosed ALL treated with a PIR (16.5%) versus non-PIR (33.4%). "While additional studies are needed, these real-world findings provide important insights into the benefits of pediatric-inspired regimens containing asparaginase in adolescent and young adult patients with newly-diagnosed acute lymphoblastic leukemia, who have a historically poorer survival rate in comparison to pediatric patients," said Susan Pandya, MD, Vice President Clinical Development and Head of Cancer Metabolism Global Development Oncology & Immuno-Oncology, Servier. "We look forward to continuing our research efforts for ASPARLAS, one of our approved asparaginase products, and exploring the potential application in other indications including solid tumors where there is emerging preclinical rationale." The full abstract is available for viewing on the ASH 2022 website. About Servier Pharmaceuticals Servier Pharmaceuticals LLC is a commercial-stage company with a passion for innovation and improving the lives of patients, their families and caregivers. As a privately held company, Servier has the unique freedom to devote all of its time and energy towards patients who require our treatments, care and innovation in areas of unmet medical need. As a leader in oncology, Servier is committed to finding solutions that will address today's challenges. The company's oncology portfolio includes innovative medicines designed to bring more life-saving treatments to a greater number of patients, across the entire spectrum of disease and in a variety of tumor types. Servier has significantly accelerated its investment in hard-to-treat cancers with more than 50% of its research and development dedicated to delivering significant advances in areas of high unmet need that may truly move the needle for our patients. Servier believes co-creation is fundamental to driving innovation and is actively building alliances, acquisitions, licensing deals and partnerships that bring solutions and accelerate access to therapies. With the company's commercial expertise, global reach, scientific expertise and commitment to clinical excellence, Servier Pharmaceuticals is dedicated to bringing the promise of tomorrow to the patients that we serve. For more information: . About Servier Founded to serve health, Servier is a global group governed by a Foundation that aspires to have a meaningful social impact, both for patients and for a sustainable world. With its unique governance model, it can fully serve its vocation with a long-term vision: being committed to therapeutic progress to serve patient needs. The 21,800 employees of the Group are committed to this shared vocation, source of inspiration every day. As a world leader in cardiology, Servier's ambition is to become a renowned, focused and innovative player in oncology by targeting hard-to-treat cancers. That is why the Group allocates over 50% of its R&D budget to developing targeted and innovative therapies in oncology. Neuroscience and immuno-inflammatory diseases are the future growth drivers. In these areas, Servier is focused on a limited number of diseases in which accurate patient profiling makes it possible to offer a targeted therapeutic response through precision medicine. To promote access to quality care for all at a lower cost, the Group also offers a range of quality generic drugs covering most pathologies, relying on strong brands in France, Eastern Europe, Brazil and Nigeria. In all these areas, the Group includes the patient voice at each stage of the life cycle of a medicine. Headquartered in France, Servier relies on a strong geographical footprint in over 150 countries and achieved a revenue of €4.7 billion in 2021. For more information on the new Group website: servier.com. Press contacts Servier Pharmaceuticals (U.S.) Julia Ferreira [email protected] Servier Group (France and worldwide) Sonia Marques [email protected] +33 (0)1 55 72 40 21 / + 33 (0)7 84 28 76 13 Disclosures This release contains general information about the Servier Group and its entities (hereinafter "Servier and its Affiliates") and is intended for informational purposes only. The information is thought to be reliable; however, Servier and its Affiliates make no representation as to the accuracy or completeness of the information contained herein or otherwise provided and accept no responsibility or liability, in contract, in tort, in negligence, or otherwise, should the information be found to be inaccurate or incomplete in any respect. 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注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）。因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。 关键词：ALL；Tecartus；新疗法 10月1日，吉利德旗下的凯特制药宣布FDA批准其Tecartus（brexucabtagene autoleucel）用于治疗成人复发或难治性B细胞前体急性淋巴细胞白血病（ALL）患者。 Tecartus是一种自体抗CD19 CAR-T细胞疗法，2020年7月被FDA加速批准用于治疗先前接受过2种或多种系统疗法（包括一种BTK抑制剂）的复发或难治性套细胞淋巴瘤（R/R MCL）成人患者。 此次Tecartus获批治疗ALL是基于1/2期研究ZUMA-3的数据。该研究是一项国际多中心、单臂、开放标签注册研究，在接受标准系统疗法或造血干细胞移植后难治或复发的成人ALL患者中展开，主要终点为由独立审查委员会评估的完全缓解（CR）或血液学不完全恢复的完全缓解。 结果显示：中位随访时间12.3个月，在疗效可评估患者中 65%患者达到CR或血液学恢复不完全的 CR，超50%患者的CR持续时间超过12个月，中位缓解持续时间 (DOR) 为 13.6 个月。安全性方面，在接受目标剂量治疗的患者中，3级以上细胞因子释放综合征和神经毒性事件发生率分别为26%和35%，通常可控。   急性淋巴细胞白血病（ALL）是一种常见的恶性血液病，以骨髓和淋巴组织中不成熟淋巴细胞的异常增殖和聚集为特点，可累及淋巴结、脾脏、肝脏、中枢神经系统和其他器官。ALL主要分为T细胞ALL、前体B细胞ALL和成熟B细胞白血病三类，其中前体B细胞ALL是ALL的最常见形式，约占75%。 目前，ALL治疗除传统的化疗和造血干细胞移植，全球已经批准了多款新疗法，其中Gleevec/Glivec、Sprycel和Iclusig均属于酪氨酸激酶抑制剂 (TKI)，Blincyto是全球获批的首个CD19-CD3双特异性T细胞衔接（BiTE）免疫疗法，Besponsa是一款CD22靶向抗体药物偶联物（ADC），而Kymriah是一种CD19靶向的基因修饰自体T细胞免疫细胞疗法（CAR-T疗法）。 而且，近年来FDA还批准另外两款新的ALL药物，即施维雅的Asparlas（calaspargase pegol-mknl，Cal-PEG）和Jazz制药的Rylaze。其中Asparlas是一种长效天冬酰胺特异性酶，2018年12月被FDA批准作为多药化疗方案的一个组成部分，用于1个月至21岁的儿童和青少年ALL患者的治疗。Rylaze 是一种生物制剂重组的菊欧文氏菌天冬酰胺酶，以特殊荧光菌的技术平台研发而来，2021年7月被FDA批准作为化疗方案的一部分，治疗对常见化疗方案中大肠杆菌源性天冬酰胺酶制品过敏的ALL和淋巴母细胞淋巴瘤（LBL）的1个月龄以上的儿童和成人患者。 此外，目前还有其他几款药物被开发用于治疗ALL，如亘喜生物的GC027、Autolus Therapeutics的AUTO3。其中GC027利用亘喜生物独有的TruUCAR专利技术以及无需进行HLA匹配的健康供者T细胞开发的一款即用型CAR-T细胞疗法，通过靶向CD7抗原表达的肿瘤细胞治疗T细胞恶性肿瘤。目前，该药治疗R/R急性T淋巴细胞白血病(T-ALL)的一项I期临床试验取得积极结果。AUTO3是一种自体富集T细胞，经单个逆转录病毒载体修饰表达2个独立的嵌合抗原受体（CAR），分别靶向B淋巴细胞抗原CD19和CD22，并且每个CAR都针对单个靶点活性进行了独立优化。通过同时针对2种B细胞抗原，AUTO3旨在减少B细胞恶性肿瘤患者因单一抗原丢失导致的复发。2019年4月，该药被FDA授予治疗ALL的孤儿药资格（ODD）。 药事纵横投稿须知：稿费已上调，欢迎投稿 各位朋友好，觉得本文对您有帮助，请随手点一下下方的在看，以便让你的朋友也能看到哦。