Chi-Med Announces Surufatinib Granted FDA Orphan Drug Designation for Pancreatic Neuroendocrine Tumors

和黄医药宣布索凡替尼获FDA孤儿药资格认定

2019-11-29 13:00:15 Drugs

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Hutchison China MediTech Limited (“Chi-Med”) (AIM/Nasdaq: HCM) today announces that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug designation to surufatinib for the treatment of pancreatic neuroendocrine tumors (“NET”). “NET is an area of significant unmet medical need. The current treatment options are very limited,” said Christian Hogg, CEO of Chi-Med. “The FDA granting Orphan designation is a positive step and continues to reinforce the importance of our research and development in bringing surufatinib to more patients in need.”  If approved by the FDA as an orphan treatment, surufatinib will be entitled to seven years of market exclusivity for the approved indication. Orphan Drug designation also affords certain development cost benefits in the U.S. Surufatinib is under investigation in multiple solid tumors in China and the U.S., both as a monotherapy and in combination with immunotherapies. Surufatinib is the second novel oncology drug discovered by Chi-Med to successfully complete a Phase III trial in China. A New Drug Application (“NDA”) for surufatinib for the treatment of patients with advanced non-pancreatic NET was accepted for review by the China National Medical Products Administration (NMPA) on November 11, 2019. About FDA Orphan Drug Designation The FDA Orphan Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rarer diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.  About Surufatinib Surufatinib (previously known as HMPL-012 or sulfatinib) is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptor (“VEGFR”) and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells.  Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies.  Surufatinib is in several late-stage and proof-of-concept clinical trials in China and proof-of-concept clinical trials in the U.S. According to Frost & Sullivan, the market for anti-angiogenesis VEGF/VEGFR inhibitors in China has grown from US$500 million in 2015 to over US$1.5 billion in 2019 and is expected to reach US$5 billion by 2026. Chi-Med currently retains all rights to surufatinib worldwide. Non-Pancreatic neuroendocrine tumors in China: In 2015, we initiated the SANET-ep study, a Phase III study of surufatinib in advanced neuroendocrine tumors – extra-pancreatic patients in China for whom there is no effective therapy. In June 2019, a 198 patient interim analysis was conducted, leading the independent data monitoring committee to determine that the study met the pre-defined primary endpoint of progression-free survival (“PFS”) and should be stopped early.  Pancreatic neuroendocrine tumors in China: In 2016, we initiated the SANET-p study, which is a pivotal Phase III study in patients with low- or intermediate-grade, advanced pancreatic neuroendocrine tumors in China.  The primary endpoint is PFS.  We expect an interim analysis in the first half of 2020 and enrollment to complete in 2020 (clinicaltrials.gov identifier: NCT02589821).  Neuroendocrine tumors in the U.S. and Europe: We are planning a U.S. registration study in neuroendocrine tumors patients based on the encouraging data from the Phase II and Phase III studies of surufatinib in neuroendocrine tumors in China (clinicaltrials.gov identifier: NCT02267967), and the ongoing Phase Ib study in the U.S. (clinicaltrials.gov identifier: NCT02549937). Biliary tract cancer in China: In March 2019, we initiated a Phase IIb/III study comparing surufatinib with capecitabine in patients with advanced biliary tract cancer whose disease progressed on first-line chemotherapy.  The primary endpoint is overall survival (OS) (clinicaltrials.gov identifier NCT03873532). Immunotherapy combinations: In November 2018 and September 2019, we entered into collaboration agreements to evaluate the safety, tolerability and efficacy of surufatinib in combination with anti-programmed cell death protein 1 (PD-1) monoclonal antibodies.  This included global collaborations to evaluate the combination of surufatinib with Tuoyi®, approved in China by Shanghai Junshi Biosciences Co. Ltd, and with Tyvyt®, approved in China by Innovent Biologics, Inc. About Neuroendocrine Tumors (NET) Neuroendocrine tumors form in cells that interact with the nervous system or in glands that produce hormones.  They can originate in various parts of the body, most often in the gut or the lungs and can be benign or malignant.  Neuroendocrine tumors are typically classified as pancreatic neuroendocrine tumors or non-pancreatic neuroendocrine tumors.  Approved targeted therapies include Sutent® and Afinitor® for pancreatic neuroendocrine tumors, or well-differentiated, non-functional gastrointestinal or lung neuroendocrine tumors. According to Frost and Sullivan, there were 19,000 newly diagnosed cases of neuroendocrine tumors in the U.S. in 2018.  Importantly, neuroendocrine tumors are associated with a relatively long duration of survival compared to other tumors.  As a result, there were approximately 141,000 estimated patients living with neuroendocrine tumors in the U.S. in 2018 of which over 90%, or approximately 132,000, were non-pancreatic neuroendocrine tumor patients. In China, there were approximately 67,600 newly diagnosed neuroendocrine tumor patients in 2018 and, considering the current incidence to prevalence ratio in China, potentially as many as 300,000 patients living with the disease in the country1.  It is estimated that approximately 80% of the patients living with neuroendocrine tumors in China are non-pancreatic neuroendocrine tumor patients. About Chi-Med Chi-Med (AIM/Nasdaq: HCM) is an innovative biopharmaceutical company which researches, develops, manufactures and markets pharmaceutical products.  Its Innovation Platform, Hutchison MediPharma, has about 470 scientists and staff focusing on discovering, developing and commercializing targeted therapeutics and immunotherapies in oncology and autoimmune diseases.  It has a portfolio of eight cancer drug candidates currently in clinical studies around the world.  Chi-Med’s Commercial Platform manufactures, markets, and distributes prescription drugs and consumer health products, covering an extensive network of hospitals across China. Chi-Med is headquartered in Hong Kong and is dual-listed on the AIM market of the London Stock Exchange and the Nasdaq Global Select Market.  For more information, please visit: www.chi-med.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the U.S. Private Securities Litigation Reform Act of 1995.  These forward-looking statements reflect Chi-Med’s current expectations regarding future events, including its expectations regarding the NDA approval and launch of surufatinib for the treatment of patients with non-pancreatic NET in China, the further clinical development of surufatinib in this and other indications, its expectations as to whether clinical studies of surufatinib would meet their primary or secondary endpoints, and its expectations as to the timing of the completion and the release of results from such studies.  Forward-looking statements involve risks and uncertainties.  Such risks and uncertainties include, among other things, assumptions regarding the sufficiency of its data to support NDA approval of surufatinib for the treatment of patients with non-pancreatic NET in China, its potential to gain expeditious approvals for surufatinib in other jurisdictions such as the U.S. and EU, the safety profile of surufatinib, the potential for surufatinib to become a new standard of care for non-pancreatic NET patients, its ability to implement and complete its further clinical development plans for surufatinib, its potential commercial launch of surufatinib in China and other jurisdictions and the timing of these events.  Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.  For further discussion of these and other risks, see Chi-Med’s filings with the U.S. Securities and Exchange Commission and on AIM.  Chi-Med undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise. Source: Chi-Med   Posted: November 2019
和记中国医药科技有限公司(“ Chi-Med ”)( AIM / Nasdaq : HCM )今天宣布,美国食品药品监督管理局(Food and Drug Administration)(“ FDA ”)已将 Orapha 药物指定用于治疗胰腺神经内分泌肿瘤(“ NET ”)。 “ NET 是一个重要的未满足医疗需求领域。目前的治疗方案非常有限,” Chi-Med 首席执行官 Christian Hogg 说。“ FDA 批准孤儿药物的指定是一个积极的步骤,并且继续加强我们的研究和开发对更多有需要的患者使用 surufatinib 的重要性。”这是一个很好的例子 如果获得 FDA 批准作为孤儿治疗, surufatinib 将有权获得批准适应症的七年市场独占性。孤儿药物指定在美国也提供了一定的开发成本效益。 在中国和美国, Surufatinib 正在研究多种实体肿瘤,既作为单一疗法,也结合免疫疗法。 Surufatinib 是 Chi-Med 为在中国成功完成 III 期试验而发现的第二种新型肿瘤药物。舒非替尼用于治疗晚期非胰网膜癌患者的新药申请(简称“ NDA ”)于2019年11月11日获得中国国家医药产品监督管理总局( NMPA )受理。 关于 FDA 孤儿药物指定 FDA 孤儿药物指定计划为药物和生物制剂提供孤儿身份,这些药物和生物制剂被定义为用于安全有效的治疗、诊断或预防影响美国不到20万人的罕见疾病/疾病的药物和生物制剂。或影响200,000多人但预计无法收回开发和销售治疗药物的费用。 关于 Surufatinib Surufatinib (以前称为 HMPL-012或 Sulatinib )是一种新型的口服血管免疫激酶抑制剂,选择性地抑制与血管内皮生长因子受体( VEGFR )和成纤维细胞生长因子受体( FGFR )相关的酪氨酸激酶活性,两者均抑制血管生成。集落刺激因子-1受体( CSF-1R )调节肿瘤相关巨噬细胞,促进机体对肿瘤细胞的免疫应答。其独特的双重作用机制可能非常适合与其他免疫疗法的可能组合。Surufatinib 在中国处于多个后期和概念验证临床试验阶段,在美国处于概念验证临床试验阶段。 根据 Frost & Sullivan 的数据,中国抗血管生成 VEGF / VEGFR 抑制剂市场规模已从2015年的5亿美元增长到2019年的15亿美元以上,预计到2026年将达到50亿美元。 Chi-Med 目前在全球范围内保留对 Surufatinib 的所有权利。 中国非胰腺神经内分泌肿瘤:2015年,我们启动了 SANET-ep 研究,这是苏菲替尼在晚期神经内分泌肿瘤中的 III 期研究——在中国没有有效治疗的胰外患者。2019年6月进行了198例患者的中期分析,领导独立数据监测委员会确定研究满足了预先定义的无进展生存的主要终点(“ PFS ”),应尽早停止。 中国胰腺神经内分泌肿瘤:2016年,我们启动了 SANET-p 研究,这是中国低度或中度、晚期胰腺神经内分泌肿瘤患者的关键 III 期研究。主要终点是 PFS 。我们预计在2020年上半年进行中期分析,2020年完成注册(临床试验用药.gov 标识符: NCT02588921)。 美国和欧洲的神经内分泌肿瘤:我们正在根据中国神经内分泌肿瘤的 surufatinib II 期和 III 期研究的令人鼓舞数据,规划美国在神经内分泌肿瘤患者中的注册研究(临床 altrials.gov 标识符: NCT02267967)。美国正在进行的 Ib 期研究(临床试验用药.gov 标识符: NCT0254937)。 我国胆道癌:2019年3月,我们启动了一项 IIb / III 期研究,比较 surufatinib 与卡培他滨治疗晚期胆道癌的疗效。主要终点是整体生存( OS )(临床试验用药.gov 标识符 NCT03873532)。 免疫治疗组合:2018年11月和2019年9月,我们签订合作协议,结合抗程序性细胞死亡蛋白1( PD-1)单克隆抗体,评估 surufatinib 的安全性、耐受性和有效性。这包括全球合作评估 surufatinib 与 Tuoyi ®(上海君实生物科技有限公司在中国批准)和 Innovation Biologics , Inc .在中国批准的 Tyvyt ®。 关于神经内分泌肿瘤( NET ) 神经内分泌肿瘤形成于与神经系统相互作用的细胞或产生激素的腺体。它们可以起源于身体的各个部位,最常见的是在肠道或肺部,可能是良性或恶性的。神经内分泌肿瘤一般分为胰腺神经内分泌肿瘤或非胰腺神经内分泌肿瘤。已批准的靶向治疗包括用于胰腺神经内分泌肿瘤的 Su帐篷®和 Afinitor ®或分化良好的非功能性胃肠或肺神经内分泌肿瘤。 根据 Frost 和 Sullivan 的数据,2018年美国有19,000例新诊断的神经内分泌肿瘤。重要的是,与其他肿瘤相比,神经内分泌肿瘤的生存期相对较长。因此,2018年美国约有14.1万名神经内分泌肿瘤患者,其中90%以上(约13.2万)为非胰腺神经内分泌肿瘤患者。 在中国,2018年新诊断的神经内分泌肿瘤患者约为6.76万人,考虑到目前在中国的发病率占比,有可能多达30万人生活在全国1。据估计,我国约80%的神经内分泌肿瘤患者为非胰腺神经内分泌肿瘤患者。 关于 Chi-Med Chi-Med ( AIM / Nasdaq : HCM )是一家研究、开发、制造和销售医药产品的创新生物制药公司。其创新平台 Hutchison MediPharma 拥有约470名科学家和员工,专注于发现、开发和商业化肿瘤和自身免疫性疾病的靶向治疗和免疫疗法。该公司目前在世界各地的临床研究中拥有8个候选癌症药物。Chi-Med 的商业平台制造、营销和分销处方药和消费者健康产品,覆盖中国各地广泛的医院网络。 Chi-Med 总部位于香港,在伦敦证券交易所 AIM 市场和纳斯达克全球精选市场两地上市。详情请访问: www.chi-med 。com 。 前瞻性陈述 本新闻稿包含前瞻性声明,其含义与美国1995年私人证券诉讼改革法案的“安全港”条款一致。这些前瞻性陈述反映了 Chi-Med 目前对未来事件的预期,包括其对 NDA 批准和在中国推出 Surufatinib 治疗非胰腺 NET 患者的预期、在此和其他适应症中 surufatinib 的进一步临床开发。它对 surufatinib 的临床研究是否能达到其主要或次要终点的期望,以及对完成和公布这类研究结果的时间的期望。前瞻性陈述涉及风险和不确定性。这些风险和不确定性包括,除其他外,关于其数据是否足以支持 NDA 批准 surufatinib 用于中国非胰腺 NET 患者的治疗的假设、其在美国和欧盟等其他司法管辖区获得 surufatinib 快速批准的可能性、 surufatinib 的安全状况、surufatinib 有可能成为非胰腺 NET 患者新的护理标准,有能力实施和完成其进一步的临床开发计划,在中国和其他司法管辖区推出 surufatinib 的潜力,以及这些事件的时机。提醒现有和潜在投资者不要过度依赖这些前瞻性陈述,这些陈述仅在本协议签订之日起生效。有关这些风险和其他风险的进一步讨论,请参见 Chi-Med 向美国证券交易委员会和 AIM 提交的文件。Chi-Med 不承担因新信息、未来事件或情况或其他原因而更新或修改本新闻稿所载信息的义务。 资料来源: Chi-Med 张贴日期:2019年11月

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