Dynacure Announces Strategic Collaboration and Option Agreement with Nippon Shinyaku
Dynacure Announces Strategic Collaboration and Option Agreement with Nippon Shinyaku
STRASBOURG, France and PHILADELPHIA and KYOTO, Nov. 10, 2021 /PRNewswire/ -- Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today that Dynacure and Nippon Shinyaku Co., Ltd. (HQs: Kyoto, President: Toru Nakai) have entered a long-term strategic development and commercialization agreement. Nippon Shinyaku has obtained an exclusive option to acquire development and commercial rights from Dynacure to develop DYN101, an investigational antisense oligonucleotide product candidate designed to be a disease-modifying medicine for treatment of the majority of Myotubular and Centronuclear Myopathies (CNM) in both adult and pediatric patients living with this rare life-threatening disorder in Japan. DYN101 is currently being evaluated in a Phase 1/2 clinical trial, UNITE-CNM (DYN101-C101), at multiple clinical sites in Europe. DYN101 has been granted orphan drug designation by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) and rare pediatric disease designation by the FDA. There is currently no FDA-, EMA- or Pharmaceuticals and Medical Devices Agency (PMDA)- approved therapeutic treatment for CNM.
法國斯特拉斯堡和費城京都，2021年11月10日/美通社/--Dynacure是一家臨牀階段公司，專注於開發和商業化新療法，以改變患有罕見疾病的患者的生活，這些患者的治療選擇有限或沒有治療選擇。該公司今天宣佈，Dynacure和日本新亞株式會社(HQS：京都、總裁：Toru Nakai)達成了一項長期戰略開發和商業化協議。日本新屋公司獲得了從Dynacure公司獲得開發和商業權的獨家選擇權，以開發DYN101。DYN101是一種研究用反義寡核苷酸候選產品，旨在成為一種治療日本大多數患有這種罕見的危及生命的疾病的成人和兒童患者的肌管性和中心核肌病(CNM)的藥物。在日本，DYN101是一種試驗性反義寡核苷酸產品候選產品，旨在治療日本大多數患有這種罕見的危及生命的疾病的成人和兒童患者的肌小管和中央核肌病(CNM)。DYN101目前正在進行1/2期臨牀試驗，Unite-CNM(DYN101-C101)，在中國的多個臨牀地點進行評估歐洲。DYN101已獲得美國食品和藥物管理局(FDA)和歐洲藥品管理局(EMA)的孤兒藥物稱號，並獲得FDA的罕見兒科疾病稱號。目前還沒有FDA、EMA或藥品和醫療器械管理局(PMDA)批准的CNM治療方法。
Under the terms of the agreement, Nippon Shinyaku has secured opt-in rights to pursue the development and commercialization of DYN101 in Japan upon analysis of interim data from the Phase 1/2 study, UNITE-CNM. These interim data are anticipated during the second half of 2022. Dynacure will retain rights to rest of world development and commercialization. Dynacure will receive an initial payment of $5 million. The agreement also includes option exercise fee as well as clinical, regulatory and commercial milestone payments of up to $82 million at maximum, in addition to a supply price based on net sales.
根據協議條款，日本新屋株式會社通過對第1/2階段研究Unite-CNM的中期數據進行分析，獲得了在日本進行DYN101開發和商業化的選擇權。這些中期數據預計將在2022年下半年公佈。Dynacure將保留世界其他地區的開發和商業化權利。Dynacure將收到首期付款500萬美元。該協議還包括期權行使費以及臨牀、監管和商業里程碑付款，最高可達8200萬美元，此外還包括基於淨銷售額的供應價格。
"As we look ahead toward commencing a pediatric study based upon interim data expected in 2022 and a pivotal study after final data in 2023, Nippon Shinyaku brings expertise in rare and musculoskeletal disease drug development, a strong network of physician specialists throughout Japan, and a shared urgency to help those affected with CNM," said Frédéric Legros, Chief Operating Officer of Dynacure. "We look forward to leveraging the skills and network Nippon Shinyaku brings and advancing this important clinical program toward commercialization."
Dynacure公司首席運營官弗雷德裏克·萊格羅斯(Frédéric Legros)説：“當我們期待着在2022年開始一項基於中期數據的兒科研究，並在2023年最終數據之後開始一項關鍵研究時，日本新雅庫帶來了罕見和肌肉骨骼疾病藥物開發方面的專業知識，日本各地強大的內科專家網絡，以及幫助那些受CNM影響的人的共同緊迫性，”Dynacure首席運營官弗雷德裏克·萊格羅斯(Frédéric Legros)説。我們期待着利用日本新谷公司帶來的技能和網絡，推動這一重要的臨牀項目走向商業化。“
Masaya Toda, Corporate Officer, Head of Licensing and Alliance Division from Nippon Shinyaku added, "Dynacure has worked tirelessly to advance a potential therapy for a patient community with no treatment options, and the reach and resources Nippon Shinyaku brings throughout Japan are an ideal complement to these efforts. We are committed to do our part to help alleviate the burden on patients living with CNM, and we are looking forward to working with our new partners at Dynacure to advance this potentially life changing therapy in Japan."
來自日本新雅庫的公司官員、許可和聯盟部門負責人Masaya Toda補充説：“Dynacure不知疲倦地為沒有治療選擇的患者羣體推進一種潛在的療法，以及日本新雅庫帶來的覆蓋範圍和資源日本是對這些努力的理想補充。我們致力於儘自己的一份力量，幫助減輕CNM患者的負擔，我們期待着與我們在Dynacure的新合作伙伴合作，在日本推進這種可能改變生活的療法。“
About Myotubular and Centronuclear Myopathies
Myotubular and Centronuclear Myopathies (CNM) are serious, rare, life-threatening disorders that affect skeletal muscles from birth. CNM derives its name based on the central location of the muscle fiber nucleus, which is an abnormal finding observed in muscle biopsies. People with CNM begin experiencing muscle weakness at any time from birth to early adulthood and many patients die within the first 18 months of life. Patients who survive longer require intense medical management and nearly uninterrupted support, including permanent ventilation, brace with head support and feeding tubes. The disease is driven by mutations in multiple genes including MTM1, DNM2 and BIN1 and Dynacure scientists have discovered a link between an increase in DNM2 protein and the direct cause of the disease 4. The three classical forms of CNM are X-linked myotubular myopathy (XLCNM), autosomal dominant CNM (ADCNM), and autosomal recessive CNM (ARCNM), which are all associated with poor prognosis. Myotubular and Centronuclear Myopathies affect an estimated 4,000 to 5,000 patients in the European Union, United States, Japan and Australia1.
關於肌管性肌病和中心核肌病
肌管和中心核肌病(CNM)是一種嚴重的、罕見的、危及生命的疾病，從出生起就會影響骨骼肌。CNM的名字來源於肌肉纖維核的中心位置，這是在肌肉活檢中觀察到的異常發現。CNM患者從出生到成年早期的任何時候都會出現肌肉無力，許多患者在出生後18個月內死亡。存活時間較長的患者需要密集的醫療管理和幾乎不間斷的支持，包括永久通氣、頭部支撐和餵養管。這種疾病是由包括MTM1、DNM2和BIN1在內的多個基因突變引起的，Dynacure的科學家發現DNM2蛋白的增加與疾病的直接原因之間存在聯繫4。X連鎖肌管性肌病(XLCNM)、常染色體顯性遺傳性CNM(ADCNM)和常染色體隱性遺傳性CNM(ARCNM)是CNM的三種典型類型，均與預後不良有關。據估計，在歐盟、美國和美國，約有4000到5000名患者患有肌管性和中心核肌病。日本和澳大利亞1.
About DYN101
DYN101, an investigational antisense oligonucleotide product candidate using Ionis Pharmaceuticals' proprietary antisense technology, is designed to reduce the expression of dynamin 2 protein (DNM2) for the treatment of CNM. Preclinical studies have shown that DYN101 has the potential to be disease modifying in CNM, with preclinical activity observed in animal models of XLCNM and ADCNM 2,3. Prevention and reversion of the disease were observed with a dose-dependent improvement in whole body strength and mice survival. The development plan for DYN101 was designed to be broad and it is the only known program investigating a treatment for the majority of CNM patients, comprised of those who have XLCNM or ADCNM. DYN101 is currently under clinical investigation in several European countries. DYN101 has been granted Orphan Drug designations by the FDA and EMA and rare pediatric disease designation by the FDA.
關於DYN101
DYN101是一種研究中的反義寡核苷酸產品，採用Ionis製藥公司的專有反義技術，旨在減少用於治療CNM的Dynamin 2蛋白(DNM2)的表達。臨牀前研究表明，DYN101在CNM中具有潛在的疾病修飾作用，在XLCNM和ADCNM動物模型中觀察到臨牀前活性。2,3。觀察到疾病的預防和逆轉，全身力量和小鼠存活率都有劑量依賴性的改善。DYN101的開發計劃被設計得很廣泛，它是目前已知的唯一一個研究大多數CNM患者(包括患有XLCNM或ADCNM的患者)的治療方法的計劃。DYN101目前正在幾個歐洲國家進行臨牀研究。DYN101已獲得FDA和EMA的孤兒藥物稱號，並獲得FDA的罕見兒科疾病稱號。
About Dynacure
Dynacure is a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options. The Dynacure team leverages its proven track record in rare disease drug development to build a pipeline of novel drug candidates. Dynacure is developing DYN101, an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein for the treatment of Myotubular and Centronuclear Myopathies, in strategic collaboration with Ionis Pharmaceuticals.
關於Dynacure
Dynacure是一家臨牀階段的公司，專注於開發和商業化新療法，以改變那些治療選擇有限或沒有治療選擇的罕見疾病患者的生活。Dynacure團隊利用其在罕見病藥物開發方面久經考驗的記錄，建立了一條新的候選藥物流水線。Dynacure正在與Ionis製藥公司進行戰略合作，開發DYN101，這是一種研究用反義產品候選產品，旨在減少Dynamin 2蛋白的表達，用於治療肌小管和中心核肌病。
Dynacure is headquartered in Strasbourg, France with a corporate office in Philadelphia, PA, USA.
Dynacure總部設在法國斯特拉斯堡，公司辦事處設在賓夕法尼亞州費城，美國。
For more information, please visit www.dynacure.com.
欲瞭解更多信息，請訪問www.dynacure.com。
About Nippon Shinyaku
Nippon Shinyaku, established in 1919, has a long-history as a research-oriented pharmaceutical company in Japan. It has been dedicated to research and development of innovative drugs. Areas of specialty for both R&D and marketing by Nippon Shinyaku include hematology, intractable and rare diseases, urology and gynecology. By implementing its own drug discovery research, licensing-in from other companies and product lifecycle management (PLCM), Nippon Shinyaku is striving to address unmet medical needs and speedily launch pharmaceuticals to help patients.
關於日本新谷
日本新屋成立於1919年，作為一家以研究為導向的製藥公司，在日本有着悠久的歷史。它一直致力於創新藥物的研究和開發。日本新谷公司研發和營銷的專業領域包括血液學、疑難病和罕見疾病、泌尿科和婦科。通過實施自己的藥物發現研究、從其他公司獲得許可以及產品生命週期管理(PLCM)，日本新屋株式會社正在努力解決未得到滿足的醫療需求，並迅速推出藥物來幫助患者。
For further information, please visit https://www.nippon-shinyaku.co.jp/english/
欲瞭解更多信息，請訪問網址：https://www.nippon-shinyaku.co.jp/english/。
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SOURCE Dynacure
源動態確保
Related Links
相關鏈接
https://www.nippon-shinyaku.co.jp/english/
https://www.dynacure.com/
Https://www.nippon-shinyaku.co.jp/english/Https://www.dynacure.com/
譯文內容由第三人軟體翻譯。