药明生物祝贺合作伙伴Amicus新一代庞贝氏症疗法获FDA突破性疗法认定

WuXi Biologics Congratulates Amicus on Receiving FDA Breakthrough Therapy Designation for AT-GAA 

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上海，2019年2月28日

——药明生物赋能合作伙伴开发的产品再次获FDA突破性疗法认定

全球领先的开放式生物制药技术平台公司药明生物（WuXi Biologics, 2269.HK）的合作伙伴Amicus（纳斯达克股票代码：FOLD）近日宣布，公司用于治疗晚发型庞贝氏症的新一代疗法AT-GAA（ATB200/AT2221）获美国FDA授予突破性疗法认定。药明生物对此表示热烈祝贺！

庞贝氏症是由酸性α-葡萄糖苷酶（GAA）缺乏而引起的一种遗传性溶酶体贮积症（LSD）。AT-GAA是首个获得突破性疗法认定的庞贝氏症在研疗法。

AT-GAA项目于2012年在药明生物平台上从一个“主意”诞生，在公司全球领先生物制药技术及强劲产能实力的支持下，目前已顺利进入三期临床试验阶段。不久前，药明生物与Amicus达成独家商业化生产战略合作，成为ATB200项目商业化阶段的独家原液生产合作伙伴和主要制剂供应合作伙伴，通过全球双重供应策略为其提供高质量生产服务。

“我们对Amicus取得这项激动人心的里程碑表示祝贺，”药明生物CEO陈智胜博士表示，“我们很荣幸通过药明生物世界一流技术能力和强劲产能实力赋能Amicus这样的创新合作伙伴。公司对确保ATB200在全球市场的供应充满信心，衷心祝愿此项目尽早获批上市，造福全球病患。”

值得一提的是，这是药明生物赋能合作伙伴开发的第二个收获突破性疗法认定的产品。艾滋病治疗抗体Trogarzo™是药明生物赋能合作伙伴开发的首个获得突破性疗法认定的产品，于2018年3月获美国FDA批准上市，也是药明生物首个商业化生产项目。

关于AT-GAA

AT-GAA由一种碳水化合物结构优化的独特重组人酸α-葡糖苷酶（rhGAA）ATB200和药物伴侣AT2221组成。临床前研究显示，ATB200与组织酶水平升高，肌肉糖原水平降低以及肌肉力量增强相关。一项全球临床Ⅱ/Ⅲ期研究（ATB200-02）正在评估AT-GAA的安全性、耐受性及药代动力学和药效学情况。

Amicus在晚发型庞贝氏症成人患者中开展的另一项为期52周、双盲、随机全球Ⅲ期临床研究PROPEL（ATB200-03）旨在对比AT-GAA与目前标准疗法酶替代疗法（ERT）阿葡糖苷酶的疗效、安全性和耐受性。更多信息请查询www.clinicaltrials.gov: NCT03729362。

关于Amicus

Amicus Therapeutics（纳斯达克股票代码：FOLD）是一家致力于为罕见代谢疾病患者发现、开发和提供高质量创新药的全球性生物技术公司。公司立足于“以患者为中心”，针对罕见代谢疾病打造了强大的先进在研产品线。更多信息请访问www.amicusrx.com，以及Twitter和LinkedIn。

关于药明生物

药明生物（股票代码：2269.HK）作为一家香港上市公司，是全球领先的开放式、一体化生物制药能力和技术赋能平台。公司为全球生物制药公司和生物技术公司提供全方位的端到端研发服务，帮助任何人、任何公司发现、开发及生产生物药，实现从概念到商业化生产的全过程，加速全球生物药研发进程，降低研发成本，造福病患。截至2018年6月30日，在药明生物平台上研发的综合项目达187个，包括98个处于临床前研究阶段，78个在临床早期（I期，II期）阶段，10个在后期临床（III期）以及1个在商业化生产阶段。预计到2021年，公司在中国、爱尔兰、新加坡、美国规划的生物制药生产基地合计产能约22万升，这将有力确保公司通过健全强大的全球供应链网络为客户提供符合全球质量标准的生物药。如需更多信息，请访问：www.wuxibiologics.com.cn。

相关阅读

WuXi Biologics Congratulates Amicus on Receiving FDA Breakthrough Therapy Designation for AT-GAA 

SHANGHAI

February 28, 2019 

——The second product successfully receiving Breakthrough Therapy Designation that WuXi Biologics has enabled its partner to develop

WuXi Biologics ("WuXi Bio") (Stock code: 2269.HK), a leading global open-access biologics technology platform company offering end-to-end solutions for biologics discovery, development and manufacturing, congratulates its strategic partner Amicus Therapeutics ("Amicus") (Nasdaq: FOLD) on receiving FDA Breakthrough Therapy Designation for AT-GAA (ATB200/AT2221) in late onset Pompe disease, an inherited lysosomal storage disorder caused by the deficiency of an enzyme known as acid alpha-glucosidase (GAA). AT-GAA is the first ever investigational product for Pompe disease to receive breakthrough designation.

The AT-GAA program was initiated at WuXi Biologics in 2012 with just an initial concept and now progresses through a pivotal study enabled by the global leading technology platform and unparalleled manufacturing capacity at WuXi Biologics. Just this month the two companies signed an exclusive manufacturing partnership, through which WuXi Biologics will provide dual commercial sources for both drug substance and drug product of ATB200 and will be the exclusive commercial drug substance partner and key commercial drug product supplier. 

"We congratulate Amicus on achieving this exciting milestone," said Dr. Chris Chen, CEO of WuXi Biologics. "It is a great honor to enable global innovative partners like Amicus through our world-class development and manufacturing capacities and capabilities. We are confident of supplying this critical product to the global market and wish the program a great success to benefit patients worldwide."

This is the second product successfully receiving Breakthrough Therapy Designation that WuXi Biologics has enabled its partner to develop. Trogarzo™, the first to do so, has been approved by the U.S. FDA in March 2018, marking the first commercial product manufactured by WuXi Biologics for the U.S. market.

About AT-GAA

AT-GAA is an investigational therapy that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In preclinical studies, AT-GAA was associated with increased tissue enzyme levels, reduced glycogen levels in muscle, and improvements in muscle strength. A global Phase 1/2 study (ATB200-02) is ongoing to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of AT-GAA.

Amicus has also initiated PROPEL, a global Phase 3 clinical study (also known as ATB200-03) of AT-GAA in adult patients with late onset Pompe disease. PROPEL is a 52-week, double-blind randomized study designed to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa, an enzyme replacement therapy (ERT). More information, including a list of participating sites, is available at www.clinicaltrials.gov: NCT03729362.

About Amicus

Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company's website at www.amicusrx.com, and follow on Twitter and LinkedIn.

About WuXi Biologics

WuXi Biologics (stock code: 2269.HK), a Hong Kong-listed company, is the only open-access biologics technology platform in the world offering end-to-end solutions to empower organizations to discover, develop and manufacture biologics from concept to commercial manufacturing. Our company history and achievements demonstrate our commitment to providing a truly ONE-stop service offering and value proposition to our global clients. As of June 30, 2018, there were a total of 187 integrated projects, including 98 projects in pre-clinical development stage, 78 projects in early-phase (phase I and II) clinical development, 10 projects in late-phase (phase III) development and 1 project in commercial manufacturing. With total estimated capacity of biopharmaceutical production planned in China, Ireland, Singapore and US reaching 220,000 liters by 2021, we will provide our biomanufacturing partners with a robust and premier-quality global supply chain network. For more information on WuXi Biologics, please visit www.wuxibiologics.com.

注：本信息不构成药明生物的信息披露或投资建议