Adaptimmune LLC

On August 2, 2024, the Food and Drug Administration granted accelerated approval to afamitresgene autoleucel (TECELRA, Adaptimmune, LLC), a melanoma-associated antigen A4 (MAGE-A4)-directed genetically modified autologous T cell immunotherapy, for adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and whose tumor expresses the MAGE-A4 antigen as determined by FDA-approved or cleared companion diagnostic devices. Full prescribing information for TECELRA will be posted at 2024 Biological License Application Approvals. Efficacy was evaluated in SPEARHEAD-1, Cohort 1, a multicenter, single-arm, open-label clinical trial that enrolled HLA-A*02:01-03 and 06 allele positive patients with inoperable or metastatic synovial sarcoma who had received prior systemic therapy with either doxorubicin and/or ifosfamide and whose tumor expressed the MAGE-A4 tumor antigen. Patients received lymphodepleting chemotherapy with fludarabine and cyclophosphamide. Fifty-two patients with synovial sarcoma were enrolled and underwent leukapheresis, eight of whom did not receive afamitresgene autoleucel due to death (n=3), loss of eligibility prior to lymphodepleting chemotherapy (n=3), withdrawal by patient (n=1), and investigator decision (n=1). Forty-five patients received lymphodepletion and one patient withdrew consent before treatment, for a total of 44 patients who received a single infusion of afamitresgene autoleucel. The main efficacy outcome measure was overall response rate (ORR) according to RECIST v1.1 evaluated by independent review, supported by duration of response (DOR). ORR was 43.2% (95% Confidence Interval [CI]: 28.4, 59.0). The median time to response was 4.9 weeks (95% CI: 4.4 weeks, 8 weeks). The median DOR was 6 months (95% CI: 4.6, not reached). Among patients who were responsive to the treatment, 45.6% and 39.0% had a duration of response greater than or equal to 6 months and 12 months, respectively. The prescribing information includes a Boxed Warning for serious or fatal cytokine release syndrome (CRS), which may be severe or life-threatening. The most common nonlaboratory adverse reactions (≥ 20%) were cytokine release syndrome, nausea, vomiting, fatigue, infections, pyrexia, constipation, dyspnea, abdominal pain, non-cardiac chest pain, decreased appetite, tachycardia, back pain, hypotension, diarrhea, and edema. The most common grade 3 or 4 laboratory abnormalities (≥ 20%) were lymphocyte count decreased, neutrophil count decreased, white cell blood count decreased, red blood cell decreased, and platelet count decreased. The recommended afamitresgene autoleucel dose is between 2.68 x 109 to 10 x 109 MAGE-A4 T cell receptor (TCR) positive T cells, administered in one or more infusion bags. Do not use a leukodepleting filter or prophylactic systemic corticosteroids. This application is approved under the accelerated approval pathway. To verify the clinical benefit of afamitresgene autoleucel, the FDA and the sponsor agreed upon a postmarketing requirement for an ongoing study in adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and whose tumor expresses the MAGE-A4 antigen. This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. This application was granted Regenerative Medicine Advanced Therapy, priority review, and orphan drug designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088. For assistance with single-patient INDs for investigational oncology products regulated by the Center for Biologics Evaluation and Research, healthcare professionals may email OTPRPMS@fda.hhs.gov. Follow the Oncology Center of Excellence on X: @FDAOncologyExternal Link Disclaimer. Content current as of: 08/02/2024 Regulated Product(s) Drugs 08/02/2024 Drugs Resources for Information | Approved Drugs Oncology (Cancer)/Hematologic Malignancies Approval Notifications Ongoing | Cancer Accelerated Approvals Verified Clinical Benefit | Cancer Accelerated Approvals Withdrawn | Cancer Accelerated Approvals Other | Cancer Accelerated Approvals Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) Short Description Oncology (Cancer)/Hematologic Malignancies Approval Notifications Ongoing | Cancer Accelerated Approvals Verified Clinical Benefit | Cancer Accelerated Approvals Withdrawn | Cancer Accelerated Approvals Other | Cancer Accelerated Approvals Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) Short Description

SILVER SPRING, Md., Aug. 2, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Tecelra (afamitresgene autoleucel), a gene therapy indicated for the treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA antigen(s) A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive, and whose tumor expresses the MAGE-A4 antigen as determined by FDA authorized companion diagnostic devices. Synovial sarcoma is a rare form of cancer in which malignant cells develop and form a tumor in soft tissues of the body. This type of cancer can occur in many parts of the body, most commonly developing in the extremities. The cancerous cells may also spread to other parts of the body. Each year, synovial sarcoma impacts about 1,000 people in the U.S. and most often occurs in adult males in their 30s or younger. Treatment typically involves surgery to remove the tumor and may also include radiotherapy and/or chemotherapy if the tumor is larger, returns after being removed or has spread beyond its original location. "Potentially life-threatening cancers such as synovial sarcoma continue to have a devastating impact on individuals, especially those for whom standard treatments have limited efficacy due to tumor growth and progression," said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research (CBER). "The approval of this state-of-the-art immunotherapy technology provides a critical new option for a patient population in need and demonstrates the FDA's dedication to the advancement of beneficial cancer treatments." Tecelra is also the first FDA-approved T cell receptor (TCR) gene therapy. The product is an autologous T cell immunotherapy composed of a patient's own T cells. T cells in Tecelra are modified to express a TCR that targets MAGE-A4, an antigen (substance that normally triggers your immune system) expressed by cancer cells in synovial sarcoma. The product is administered as a single intravenous dose. Tecelra was approved using the Accelerated Approval pathway, under which the FDA may approve drugs for serious or life-threatening diseases or conditions where there is an unmet medical need and the drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients (improving how patients feel or function, or whether they survive longer). This pathway can allow earlier approval while the company conducts further trials to verify the predicted clinical benefit. A confirmatory trial is ongoing to verify Tecelra's clinical benefit. "Adults with metastatic synovial sarcoma, a life-threatening form of cancer, often face limited treatment options in addition to the risk of cancer spread or recurrence," said Nicole Verdun, M.D., director of the Office of Therapeutic Products in CBER. "Today's approval represents a significant milestone in the development of an innovative, safe and effective therapy for patients with this rare but potentially fatal disease." The safety and effectiveness of Tecelra was evaluated in a multicenter, open-label clinical trial including patients with inoperable and metastatic synovial sarcoma who had received prior systemic therapy and whose tumor expressed the MAGE-A4 tumor antigen. Effectiveness was evaluated based on overall response rate and the duration of response to treatment with Tecelra. Among the 44 patients in the trial who received Tecelra, the overall response rate was 43.2% and the median duration of response was six months. The most common adverse reactions associated with Tecelra included nausea, vomiting, fatigue, infections, fever, constipation, dyspnea (shortness of breath), abdominal pain, non-cardiac chest pain, decreased appetite, tachycardia (abnormally fast heart rate), back pain, hypotension, diarrhea and edema (swelling due to buildup of fluid in body tissues). Patients treated with Tecelra may experience cytokine release syndrome (CRS), which is a dangerous type of aggressive immune system response, including potentially life-threatening reactions. CRS was observed following administration of Tecelra during clinical trials. A Boxed Warning is included in the label containing information about this risk. Patients may also exhibit Immune Effector Cell-associated Neurotoxicity Syndrome (ICANS), an immune system-related syndrome that can occur following some immunotherapies, infections, secondary malignancies, or hypersensitivity reactions, and severe cytopenia (an abnormally low level of blood cells) for several weeks following lymphodepleting chemotherapy and Tecelra infusion. Patients receiving this product should be monitored for signs and symptoms of infection and are advised not to drive or engage in hazardous occupations or activities for at least four weeks after receiving Tecelra. The FDA granted Tecelra Orphan Drug, Regenerative Medicine Advanced Therapy and Priority Review designations for this indication. This application was reviewed using a coordinated, cross-agency approach, including CBER, the FDA's Oncology Center of Excellence and the Center for Devices and Radiological Health. The FDA granted the approval of Tecelra to Adaptimmune, LLC. Media Contact: Carly (Kempler) Pflaum, (240) 672-8872 Consumer Inquiries: Email or 888-INFO-FDA The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products. SOURCE U.S. Food and Drug Administration

近年来，随着制药行业竞争的加剧和全球经济环境的波动，医疗健康领域正在经历着快速的变革。医药企业为了适应这一变化，不断调整其战略方向和组织架构。2024年至今，无论是国际还是国内的医药巨头，都经历了一系列战略调整和高层人事变动。 进入6月，这一趋势并未有所减缓。跨国医药巨头如诺华、礼来、赛诺菲、GSK、拜耳、辉瑞等，以及本土企业如再鼎医药、和黄医药、华润医药等，都陆续宣布了重要的人事变动。这些变动反映了医药行业在不断适应市场变化和寻求新的增长机会。 6月份部分药企主要人事变动情况 图片来源：根据公开信息整理 诺华中国区换帅 “女将”张颖升迁 诺华公司宣布，自2024年6月1日起，张颖女士将由诺华中国区总裁的职位晋升为诺华国际业务部首席商务官（CCO），并负责领导公司的商业上市战略（CLS）团队。 张颖女士拥有在阿斯利康和辉瑞等知名药企的丰富工作经验。自2011年加入诺华以来，她曾担任诺华制药（中国）首席运营官、诺华波兰的制药业务负责人及总裁等关键职位。自2017年4月起，张颖女士开始领导诺华在中国的制药业务。 2022年4月，诺华进行了全球性的组织架构调整，将制药和肿瘤两大业务部门合并为创新药物部门。在中国区，也同步实施了这一合并策略。随后，张颖女士被任命为诺华创新药物中国区的总裁兼董事总经理。自2023年10月起，她开始担任诺华中国区的总裁兼董事总经理，在她的领导下，诺华中国区的业绩持续增长。 根据诺华发布的财务报告，2023年公司总营收达到454.4亿美元，同比增长10%；其中，中国市场的营收为33亿美元，同比增长17%。在2024年第一季度，中国区的业绩收入更是达到了10亿美元，同比增长31%。 随着张颖女士的晋升，Leo Lee先生，目前担任诺华日本区总裁，将接替她成为新的诺华中国区总裁。在过渡期间，Keizo Miyazawa先生将临时担任日本区总裁，负责监督日本区的业务运营。 默沙东中国疫苗事业部负责人 跳槽加入赛诺菲  在6月初，默沙东中国宣布了公司疫苗事业部负责人及助理副总裁唐晓春的离职消息，其在默沙东的最后工作日定于6月17日。 紧接着，在6月18日的公开报道中，唐晓春已经加入了赛诺菲，担任赛诺菲疫苗大中华区总经理，并将成为疫苗事业部管理委员会和中国战略领导团队的一员。 唐晓春曾在2018年离开阿斯利康，转而加入默沙东中国，担任肿瘤医学事务部执行总监，期间成功推动了可瑞达等产品的多个适应证上市。2021年1月6日，默沙东宣布了中国区组织架构的高层调整，原疫苗事业部总经理彭莱莉将于同年4月结束任期离职，随后默沙东中国任命唐晓春接任疫苗事业部负责人，全面负责中国区疫苗业务的商业战略，涵盖销售、市场、政府事务、市场准入和供应链管理等关键领域。 进入2024年以来，赛诺菲响应其“Play to Win”战略计划，陆续放弃了与Denali、Kymab、Amunix等公司在肿瘤学领域的合作项目，通过削减合作和简化研发结构来优化公司运营。在这样的背景下，赛诺菲高层的架构调整也显得顺理成章。 辉瑞引进华尔街分析师 担任首席战略和创新官 在2024年6月3日，华尔街资深分析师Andrew Baum正式加入辉瑞公司，接替前任美国首席商务官兼执行副总裁Aamir Malik，担任首席战略和创新官兼执行副总裁的新职位。 作为辉瑞的新任高管，Baum先生将肩负起推动公司长期企业战略规划的重任，致力于实现患者和股东价值的最大化。他还将主导商务拓展工作，加强与生物科技公司的合作伙伴关系，并对研发管线的商业潜力进行评估。 此外，Baum先生将担任辉瑞投资组合管理团队的主席。该团队是公司最高级别的管理机构，负责对研发管线进行投资组合管理和资本配置，以确保辉瑞能够开发出能够满足患者未被满足需求的疫苗和药物，同时实现商业成功和提高药品的可及性。 在加入辉瑞之前，Andrew Baum在花旗银行担任全球医疗保健部主管及股票研究董事总经理，拥有丰富的行业经验和深厚的专业知识。 拜耳全球生物技术负责人换将 在6月11日，拜耳公司宣布了一个重要的人事变动：高级副总裁兼全球生物技术负责人Jens Vogel博士决定离开他服务了四年的岗位，以追求新的职业发展机会。 Jens Vogel博士自2020年3月加入拜耳，期间负责领导公司全球生物技术网络的整合工作，这包括生物制品的开发、运营、产品供应、技术开发，以及在美国和欧洲的战略功能职能。在加入拜耳之前，Vogel博士在勃林格殷格翰公司担任了近11年的多个领导职务。 尽管Vogel博士未明确说明辞职的具体原因，但他的离职正值拜耳进行重大组织重组的时期。今年1月，拜耳宣布了一项全面的业务改革计划。在2024年的第一季度，公司已经裁减了超过1500个职位，其中大约三分之二是管理职位。截至3月底，拜耳的员工总数降至不足98200人，与去年同期相比下降了3.5%。预计到2024年底，拜耳的各个部门都将在"动态共享所有权"的模式下运作，公司将继续进行人员调整。 与此同时，拜耳任命了新的全球生物制品生产负责人Tina Self女士，她将于6月1日正式上任。Self女士最近的职位是拜耳伯克利公司的副总裁兼工厂经理，该工厂位于加利福尼亚州，并且她也是伯克利园区的负责人。值得一提的是，Self女士是拜耳历史上第一位担任这一职务的女性领导者。 礼来功勋老将辞任CFO  6月5日，礼来公司宣布了一个关键的人事变动：Anat Ashkenazi决定辞去首席财务官（CFO）的职位，以探索制药行业之外的职业道路。 自2021年加入礼来以来，Anat Ashkenazi凭借她在财务领域的深厚经验，担任了多个关键的财务和战略职位。尽管她即将离开CFO的岗位，但Ashkenazi女士将继续作为礼来执行委员会的成员，直至2024年7月。 在Ashkenazi女士担任CFO的三年多时间里，她对公司的财务健康和战略发展做出了巨大贡献，期间礼来公司的股价实现了约300%的增长，市值达到了7910亿美元。在加入礼来之前，她曾担任礼来研究实验室的高级副总裁和财务总监，负责监管公司的商业运营以及研发、制造、质量和财务行政等关键部门。她还曾领导礼来的战略团队和业务转型部门，并在礼来的肿瘤学和糖尿病等全球业务领域担任过财务主管。 随着Ashkenazi女士的离职，礼来公司表示，目前正在积极寻找合适的继任者，既考虑内部候选人，也不排除从外部招募合适的人选。 再鼎医药元老宣布退休  6月11日，再鼎医药宣布了一项重要的人事变动：Harald Reinhart博士，在完成他卓越的职业生涯后，将于本月底退休，并继续作为公司的高级顾问。Reinhart博士曾担任总裁，负责中枢神经系统、自身免疫及感染性疾病领域（NSAilD）的全球开发工作。 随着Reinhart博士的退休，Rafael Amado博士的职责将得到扩展。他将继续担任再鼎医药的总裁，全球肿瘤研发负责人，并全面负责公司的全球研发工作。 Rafael G. Amado博士自2022年12月起加入再鼎医药，领导公司的全球肿瘤研发工作。在加入再鼎医药之前，Amado博士曾在Adaptimmune, LLC担任首席医学官。 在加入Adaptimmune之前，Amado博士在葛兰素史克有着丰富的工作经验，从2008年至2015年，他担任了多项重要职务，并最终成为高级副总裁，负责全球肿瘤研发工作。在2003年至2008年期间，Amado博士在安进公司担任肿瘤领域临床研究和全球开发的执行总监。 中概股Biotech康乃德管理层大换血 6月12日，康乃德公司宣布了一项重大的高层管理变动：首席执行官郑伟和总裁兼董事会主席潘武宾已于6月10日提交辞呈，且该辞职于6月12日正式生效。在公布两位创始人离开的消息的同时，康乃德也宣布了新的领导团队，Kleanthis G. Xanthopoulos被任命为新的董事会主席；Barry Quart被任命为首席执行官兼董事会成员；David Szekeres担任总裁一职。 新任首席执行官Barry Quart博士，拥有超过30年在生物技术和制药行业的丰富经验。在加入康乃德之前，他在辉瑞公司担任全球研发高级副总裁。Quart博士在生物技术和制药公司中担任过多个领导职务，专注于创新医药产品的研发。他曾亲自领导数家早期生物技术公司完成后期临床开发，并制定监管策略，推动产品成功上市。 结   语 在全球医药行业不断变革的背景下，人才流动映射着跨国及本土药企不断调整战略以应对行业发展的新态势。 未来，整个医药行业人员、组织架构调整还将继续。 参考资料：各渠道公开信息 识别微信二维码，添加生物制品圈小编，符合条件者即可加入 生物制品微信群！ 请注明：姓名+研究方向！ 版 权 声 明 本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。