英国制药商夏尔（Shire）7月23日宣布，与美国生物技术公司ArmaGen就实验性药物AGT-182达成了一项全球许可和合作协议，该药是一种实验性酶替代疗法（ERT），开发用于亨特氏综合征(（Hunter syndrome）中枢神经系统和躯体症状的潜在治疗。此前，FDA和欧盟均已授予AGT-182孤儿药地位。此次合作将加强Shire公司在罕见病领域的创新研发管线，同时也强调了该公司对亨特氏综合征患者群体的长期承诺。
AGT-182被设计为充分利用人体跨血脑屏障（BBB）运输物质的天然系统，利用可递送胰岛素至大脑的相同的受体。AGT-182通过将替代性IDS酶与可结合血脑屏障（BBB）上受体的一种抗体融合而成，旨在通过抗体与受体结合使IDS酶跨越血脑屏障（BBB）。
根据协议条款，Shire将支付ArmaGen公司2.25亿美元，获得AGT-182的全球商业化权利。作为协议的一部分，ArmaGen将负责开展并完成AGT-182的一项I/II期临床研究，之后Shire将负责进一步的临床开发，包括III期临床及商业化。
关于亨特氏综合征，Shire的药物Elaprase于2006年获FDA批准，该药是全球获批的首个亨特氏综合征治疗药物。目前，Shire也正在开发另一种实验性药物SHP-609，该药目前正处于II期临床，开发用于亨特氏综合征患者中枢神经系统症状的治疗。AGT-182有望成为Shire公司亨特氏综合征项目中一个重要的新产品。
同时，Shire计划到2020年，从罕见病业务收入30亿美元。本月中旬，艾伯维（AbbVie）已与Shire达成547亿美元收购协议，也同样看好Shire罕见病业务的未来。该笔收购交易预计将于本年度第四季度完成，届时，合并后的新公司将市值1370亿美元，将在全球拥有9个研发中心和14个生产基地，雇员超过30000人。
英文原文：
Shire Enters Strategic Licensing and Collaboration Agreement with ArmaGen
Strengthens Leadership Position in Treatments for Hunter syndrome and Commitment to MPS II Patient Community
Lexington, Massachusetts, US – July 23, 2014 - Shire plc (LSE: SHP, NASDAQ: SHPG), the global specialty biopharmaceutical company, and ArmaGen, a US privately held biotechnology company, today announced a worldwide licensing and collaboration agreement for AGT-182, an investigational enzyme replacement therapy (ERT) for the potential treatment of both the central nervous system (CNS) and somatic manifestations in patients with Hunter syndrome (MPS II). This collaboration strengthens Shire’s rare disease pipeline of innovative therapies where there is high unmet need, and underscores the company’s long standing commitment to the Hunter syndrome community.
Under the terms of the agreement, Shire will obtain worldwide commercialization rights for AGT-182 in exchange for payments of approximately $225 million to ArmaGen, including an initial upfront payment of $15 million in cash and equity, an additional equity investment, R&D funding, development milestones and sales milestones, in addition to royalty payments. As part of the agreement, ArmaGen will be responsible for conducting and completing the Phase I/II study which it expects to initiate before the end of 2014, after which point Shire will be responsible for further clinical development, including Phase III trials, and commercialization.
Dr. Philip J. Vickers, Global Head of Research and Development at Shire, said, “Our agreement with ArmaGen marks our continued promise to the Hunter syndrome community to bring novel therapies that have the potential to dramatically redefine the treatment paradigm and address the most critical unmet needs. AGT-182 has the potential to be an important new therapy to our portfolio of programs for the treatment of both the CNS and somatic manifestations of Hunter syndrome. We look forward to collaborating with ArmaGen and leveraging our ability to successfully develop medicines to treat this rare, life-threatening disease.”
Shire researched, developed and commercialized the first treatment approved for Hunter syndrome. This agreement with ArmaGen expands Shire’s commitment to finding treatments for Hunter syndrome, which also includes SHP-609, Shire’s product currently being investigated to treat the CNS manifestations associated with Hunter syndrome.
James Callaway, Ph.D., Chief Executive Officer of ArmaGen said, “Shire is the ideal partner for AGT-182, based on the company’s international reach and expertise in serving patients with Hunter syndrome. We look forward to beginning the Phase I/II clinical trial of AGT-182 in collaboration with Shire and leveraging their expertise with these patients.”
About AGT-182
AGT-182, which has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), is designed to take advantage of the body’s natural system for transporting products across the blood brain barrier (BBB) by using the same receptor that delivers insulin to the brain. AGT-182 is engineered by the fusion of the replacement IDS enzyme to an antibody that binds to a receptor on the BBB. The IDS enzyme is designed to cross the BBB attached to that antibody.
About Hunter syndrome (MPS II)
Hunter syndrome or Mucopolysaccharidosis II (MPS II) is a rare, life-threatening genetic disorder that results from the absence or insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. Without this enzyme, cellular waste products called mucopolysaccharides, also known as glycosaminoglycans or GAGs accumulate in tissues and organs, which then begin to malfunction. Possible signs and symptoms include large head, enlarged abdomen, frequent ear infections, difficulty breathing, joint stiffness, and short stature. It is estimated that 2/3 of all MPS II patients will be affected with CNS disease; this translates into a prevalence of around 1,200 patients worldwide.
