CSL Behring Agrees to Acquire Novel Late-Stage Gene Therapy Candidate for Hemophilia B Patients from uniQure
CSL Behring Agrees to Acquire Novel Late-Stage Gene Therapy Candidate for Hemophilia B Patients from uniQure
-- Unique gene therapy has the potential to be one of the first to market treatments to provide potentially long-term benefits with only one dose
-獨特的基因療法有可能成為首批上市的治療方法之一，只需一劑就能提供潛在的長期益處
-- CSL Behring builds on legacy of delivering innovative treatment options for people with Hemophilia B
-CSL Behring在為血友病B患者提供創新治療選擇的遺產基礎上再接再厲
KING OF PRUSSIA, Pa.,June 24, 2020/PRNewswire/ -- Global biotherapeutics leader CSL Behring announced today that it has agreed to acquire exclusive global license rights to commercialize an adeno-associated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec), for the treatment of hemophilia B from uniQure (NASDAQ: QURE), a leading gene therapy company. The AMT-061 program, currently in Phase 3 clinical trials, could be one of the first gene therapies to provide potentially long-term benefits to patients with hemophilia B.
賓夕法尼亞州普魯士國王，2020年6月24日/美通社/--全球生物治療領先者CSL Behring今天宣佈，它已同意獲得獨家全球許可權，將腺相關病毒(AAV)基因治療計劃AMT-061(Etranacogene Dezparvovec)商業化，用於治療血友病B。UniQure(納斯達克市場代碼：Qure)，一家領先的基因治療公司。AMT-061計劃目前處於第三階段臨牀試驗，可能是首批為血友病B患者提供潛在長期益處的基因療法之一。
One dose of AMT-061 has shown to increase Factor IX (FIX) plasma levels – the blood clotting protein lacking in people with hemophilia B – to a degree that reduces or eliminates the tendency for bleeding for many years. Should AMT-061 be successful, appropriate candidate hemophilia B patients would be able to have a one-time treatment to restore FIX activity to functional levels capable of eliminating the need for frequent and ongoing replacement therapies.
一劑AMT-061已經證明可以提高凝血因子IX(FIX)的血漿水平-血友病B患者缺乏的凝血蛋白-達到一定程度，從而減少或消除多年出血的傾向。如果AMT-061成功，合適的候選血友病B患者將能夠進行一次性治療，將FIX活性恢復到功能水平，從而消除對頻繁和持續的替代治療的需要。
"Our vision with hemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease," said CSL's CEO and Managing DirectorPaul Perreault. "With more than three decades of providing lifesaving innovations for the global bleeding disorders community, we are well positioned to maximize the potential benefit of this therapy."
CSL的首席執行官兼總經理Paul Perreault説：“我們對血友病B患者的願景是提供變革性的治療範例，幫助他們擺脱這種疾病的終生負擔。”30多年來，我們一直在為全球出血性疾病社區提供挽救生命的創新，我們處於有利地位，能夠最大限度地發揮這種療法的潛在效益。“
Under the agreement with uniQure, upon closing the transaction CSL Behring will have the exclusive global right to commercialize AMT-061. uniQure will receive an upfront cash payment ofUS$450 millionfollowed by regulatory and commercial sales milestone payments and royalties. Under the terms of the agreement, uniQure will complete the Phase 3 trial and scale up manufacture for early commercial supply under an agreed plan with CSL Behring. The transaction is subject to customary regulatory clearances before closing.
根據與uniQure達成的協議，交易完成後，CSL Behring將擁有將AMT-061商業化的全球獨家權利。UnQure將獲得4.5億美元的預付現金，隨後是監管和商業銷售里程碑付款和特許權使用費。根據協議條款，uniQure將根據與CSL Behring達成的協議計劃，完成第三階段的試驗，並擴大生產規模，以便早期商業供應。這筆交易在完成之前需要接受慣常的監管審批。
"We are thrilled to enter into this commercialization and license agreement with CSL Behring, an ideal commercial partner with global reach and decades of expertise in hemophilia," statedMatt Kapusta, chief executive officer of uniQure. "We believe that through this arrangement, we are ideally positioned to deliver our innovative gene therapy to the largest number of hemophilia B patients as quickly as possible. The transaction represents a major milestone in the development of etranacogene dezaparvovec and, when closed, we expect that it will provide uniQure with significant financial resources to advance and expand our pipeline of gene therapy candidates, anchored by AMT-130 in Huntington's disease, as well as to invest further in our leading manufacturing and technology platform."
UniQure首席執行官馬特·卡普斯塔説：“我們很高興能與CSL Behring達成這項商業化和許可協議。CSL Behring是一個在血友病方面擁有全球業務和數十年專業知識的理想商業夥伴。我們相信，通過這一安排，我們處於最理想的地位，能夠儘快向最多的血友病B患者提供我們的創新基因療法。這項交易代表着etranacogene dezparvovec開發過程中的一個重要里程碑，當交易完成後，我們預計它將為UnQure提供大量的財政資源，以推進和擴大我們以AMT-130為基礎的亨廷頓病基因治療候選產品的流水線，並進一步投資於我們領先的製造和技術平台。“
InDecember 2019, uniQure announced that data from its Phase2bdose-confirmation study of AMT-061 showed that all patients stabilized and sustained FIX activity at functionally high levels one year after a single dose – with increases in FIX activity of up to 50% of normal and a mean of 41%. This exceeds the levels considered sufficient to eliminate or significantly reduce the risk of bleeding events.
2019年12月，uniQure宣佈，AMT-061的2b期劑量確認研究的數據顯示，所有患者在單劑服藥一年後，FIX活動穩定並維持在功能性高水平-FIX活動增加高達正常水平的50%，平均增加41%。這超過了被認為足以消除或顯著降低出血事件風險的水平。
According to CSL Behring's Executive Vice President, Head of Research and Development, and Chief Medical Officer Bill Mezzanotte, "We are exceedingly encouraged by the data we've seen on AMT-061. Not only has the treatment option demonstrated robust clinically meaningful responses in FIX activity, but it has also exhibited excellent safety over multiple years of observation. Expanding our gene therapy portfolio to treat hemophilia B, a disease state well known to CSL Behring, exemplifies how we are strategically aligning our rare and serious disease focus and our targeted therapeutic area focus with our core scientific platforms to transform the lives of patients."
根據CSL Behring執行副總裁、研發主管和首席醫療官Bill Mezzanotte的説法，“我們對我們在AMT-061上看到的數據感到非常鼓舞。這種治療方案不僅在FIX活動中表現出了強大的臨牀意義反應，而且在多年的觀察中也表現出了極好的安全性。擴大我們的基因治療組合以治療血友病B，這是CSL Behring所熟知的一種疾病狀態，體現了我們如何從戰略上協調我們罕見和嚴重的疾病重點和
This acquisition will also enhance CSL Behring's capabilities in its growing gene therapy portfolio. The company is currently developing a stem cell gene therapy (CSL200) for the treatment of sickle cell disease and has recently established an alliance with Seattle Children's Research Institute to develop a stem cell gene therapy for primary immunodeficiency diseases -- another rare disease area where CSL Behring has leading capabilities.
此次收購還將增強CSL Behring在其不斷增長的基因治療產品組合中的能力。該公司目前正在開發一種治療鐮狀細胞疾病的幹細胞基因療法(CSL200)，最近還與西雅圖兒童研究所建立了一個聯盟，以開發一種針對原發性免疫缺陷疾病的幹細胞基因療法，這是CSL Behring擁有領先能力的另一個罕見疾病領域。
Perreault added, "Upon approval, this next-generation therapy will be highly complementary to our existing best-in-class hemophilia B product portfolio with an alternate best-in-class treatment option. With the license to AMT-061, we are building on our legacy of delivering lifesaving innovations in hematology where, today, we offer a market leading product for hemophilia B and we are a leader in therapies for treating hemophilia A, von Willebrand disease, thrombosis, and other life-threatening conditions."
Perreault補充説：“一旦獲得批准，這種下一代療法將與我們現有的一流的血友病B產品組合形成高度互補，提供另一流的治療選項。有了AMT-061的許可，我們正在血液學領域提供挽救生命的創新的遺產，今天，我們為血友病B提供了一種市場領先的產品，我們在治療血友病A、血管性血友病、血栓和其他危及生命的疾病的療法方面處於領先地位。”
CSL Behring has put patients first by addressing the world's most serious, complicated and rare diseases for over 100 years. The company is now bringing that same commitment to gene therapy; its mission is to address unmet patient needs and enable patients to get the very most out of life.
CSL Behring 100多年來一直致力於解決世界上最嚴重、最複雜和最罕見的疾病，將患者放在首位。該公司現在正將同樣的承諾帶到基因治療上；它的使命是解決未得到滿足的患者需求，使患者能夠從生活中獲得最大好處。
About Etranacogene Dezaparvovec (AMT-061)
關於古近紀Dezparvovec(AMT-061)
Etranacogene dezaparvovec, also known as AMT-061, consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). AAV5-based gene therapies have been demonstrated to be safe and well tolerated in many clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with uniQure's AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates.
Etranacogene dezparvovec，也被稱為AMT-061，由一個AAV5病毒載體組成，該載體攜帶一個帶有受專利保護的因子IX的Padua變體(FIX-Padua)的基因盒。基於AAV5的基因療法已經在許多臨牀試驗中被證明是安全和耐受性良好的，包括在25名血友病B患者和其他適應症患者身上進行的4項uniQure試驗。在使用uniQure公司基於AAV5的基因療法進行臨牀試驗的患者中，沒有人經歷過對衣殼的任何細胞毒性T細胞介導的免疫反應。此外，臨牀前和臨牀數據顯示，基於AAV5的基因療法對已有AAV5抗體的患者可能在臨牀上有效，因此與其他候選基因治療產品相比，有可能提高患者接受治療的資格。
About CSL Behring
關於CSL Behring
CSL Behringis a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients' needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn.
CSL Behringe是一家全球生物療法領先者，其承諾是拯救生命。我們專注於利用最新技術滿足患者的需求，開發和提供用於治療凝血障礙、原發性免疫缺陷、遺傳性血管性水腫、呼吸系統疾病和神經疾病的創新療法。該公司的產品還用於心臟手術、燒傷治療和預防新生兒溶血病。
CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company,CSL Limited(ASX:CSL;USOTC:CSLLY), headquartered inMelbourne, Australia, employs more than 26,000 people, and delivers its life-saving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit VitaCSLBehring.com/vitaand follow us onTwitter.com/CSLBehring.
CSL Behring運營着世界上最大的血漿收集網絡之一，CSL等離子體。母公司CSL有限公司(澳大利亞證券交易所代碼：CSL；美國SOTC代碼：CSLY)總部設在澳大利亞墨爾本，擁有2.6萬多名員工，為100多個國家和地區的患者提供救命療法。有關生物技術前景的鼓舞人心的故事，請訪問VitaCSLBehring.com/vita，並在Twitter.com/CSLBehring上關注我們。
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SOURCE CSL Behring
源CSL Behring
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