2021年12月7日07:00 GMT
Liver-targeted antisense therapy in Phase III 发展 for the treatment of transthyretin amyloidosis, 一个系统, 进行性和致命性疾病
澳门在线赌城娱乐 has entered into a new global 发展 and commercialisation agreement with Ionis Pharmaceuticals, 公司. (Ionis)，以前称为Ionis - ttr - lRX. Eplontersen is a ligand-conjugated antisense investigational medicine currently in Phase III clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN). It is designed to reduce the production of transthyretin (TTR protein) to treat both 在这里ditary and non-在这里ditary forms of TTR amyloidosis (ATTR).
The companies will jointly develop and commercialise eplontersen in the US, while 澳门在线赌城娱乐 will develop and commercialise it in the rest of the world, 除了拉丁美洲.
atr - cm是系统性的, 进行性和致命性疾病 that leads to progressive 心 failure and death within four years from diagnosis.1 It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms.2 Hereditary ATTR-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, 如果不治疗, 通常在十年内死亡.3
Mene Pangalos，澳门第一赌城在线娱乐R的执行副总裁&D, 澳门在线赌城娱乐, said: “Eplontersen has the potential to halt the progression of TTR-mediated amyloidosis, 不管它是由基因突变还是衰老引起的. 由于其精确的肝脏靶向特性, it also has the potential to be a best-in-class treatment for patients suffering from this devastating disease and who currently have limited options.”
Hereditary ATTR-PN is expected to be the first indication for which the companies will seek regulatory approval for eplontersen, with the potential to file a new drug application with the US Food and Drug Administration by the end of 2022.
金融方面的考虑
澳门在线赌城娱乐 will pay Ionis an upfront payment of $200m and additional conditional payments of up to $485m following regulatory approvals. 它还将支付高达2美元的费用.9bn of sales-related milestones based on sales thresholds between $500m and $6bn, plus royalties in the range of low double-digit to mid-twenties percentage depending on the region. 合作包括按地区发展, 商业和医疗事务费用分摊条款.
The transaction will be funded with cash and is expected to be neutral to Core earnings in 2021. 它将作为一项无形资产收购入账, 以预付金额初始确认, with any potential future milestone payments capitalised into the intangible asset as they are recognised.
Ionis will continue to manufacture and supply eplontersen for the existing clinical studies and process qualification. 澳门在线赌城娱乐将负责商业供应, 过渡时间由双方商定. 澳门在线赌城娱乐将记录该协议下产生的所有销售额.
该交易预计将于2021年第四季度完成, 根据惯例成交条件和监管许可. The transaction does not impact the 澳门在线赌城娱乐’s financial guidance for 2021.
笔记
Eplontersen
Eplontersen is a ligand-conjugated antisense (LICA) investigational medicine designed to reduce the production of transthyretin, 或TTR蛋白, 治疗所有类型的ATTR, 一个系统, 进行性和致命性疾病.
淀粉样变(ATTR)
Cardiomyopathy and polyneuropathy due to ATTR are caused by aging or genetic mutations resulting in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium and peripheral nerves, 分别. 在ATTR患者中, both the mutant and wild type TTR protein builds up as fibrils in tissues, 比如周围神经, 心, 消化系统, 眼睛, 肾脏, 中枢神经系统, 甲状腺和骨髓. The presence of TTR fibrils interferes with the normal functions of these tissues. 随着TTR蛋白原纤维的扩大, 更多的组织损伤发生，疾病恶化, 导致生活质量下降，最终死亡. Worldwide, t在这里 are an estimated 300,000 - 500,000 patients with ATTR-CM4,5 1万- 4万名atr - pn患者2.
澳门在线赌城娱乐
澳门在线赌城娱乐(LSE/STO/Nasdaq: AZN)是一家全球性制药公司, 以科学为主导的澳门第一赌城在线娱乐公司，专注于发现, 发展, 以及肿瘤学处方药的商业化, 罕见疾病, 和澳门第一赌城在线娱乐, 包括心血管, 肾 & 新陈代谢和呼吸 & 免疫学. 总部设在剑桥, UK, 澳门在线赌城娱乐 operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. 请访问 澳门在线赌城娱乐.com 并在推特上关注公司 @澳门在线赌城娱乐.
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参考文献
1. Lauppe再保险公司 等. Nationwide prevalence and characteristics of transthyretin amyloid cardiomyopathy in Sweden. 开放的心. 2021年10月,8 (2):e001755. doi: 10.1136 / openhrt - 2021 - 001755.
2. Gonzalez-Duarte, 等. Impact of non-cardiac clinicopathologic characteristics on survival in transthyretin amyloid polyneuropathy. 神经其他计划. 2020;9(1):135-149. doi: 10.1007/s40120-020-00183-7.
3. Cortese, 等. Diagnostic challenges in 在这里ditary transthyretin amyloidosis with polyneuropathy: avoiding misdiagnosis of a treatable 在这里ditary neuropathy. [神经外科精神病学]. 88年5月,2017 (5):457 - 458. doi: 10.1136 / jnnp - 2016 - 315262.
4. Mohamed-Salem L, 等. Prevalence of wild type ATTR assessed as myocardial uptake in bone scan in the elderly population. 心脏内科. 2018年11月1日;270:192-196. doi: 10.1016/j.ijcard.2018.06.006.
5. Cuscaden C, 等. Estimation of prevalence of transthyretin (ATTR) cardiac amyloidosis in an Australian subpopulation using bone scans with echocardiography and clinical correlation. J核心醇. 2020年5月8日. doi: 10.1007/s12350-020-02152-x.
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