7 December 2021 07:00 GMT
肝靶向反义疗法用于甲状腺转蛋白淀粉样变性的III期研究, a systemic, progressive and fatal condition
澳门在线赌城娱乐与Ionis制药公司签署了一项新的全球开发和商业化协议, Inc. (Ionis) for eplontersen, formerly known as IONIS-TTR-LRX. Eplontersen是一种配体偶联的反义研究性药物，目前正处于治疗淀粉样转甲状腺素心肌病(atr - cm)和淀粉样转甲状腺素多神经病(atr - pn)的III期临床试验中。. 它旨在减少甲状腺转蛋白(TTR蛋白)的产生，以治疗遗传性和非遗传性TTR淀粉样变(ATTR)。.
两家公司将在美国共同开发和商业化这种新型电池, 而澳门在线赌城娱乐将在世界其他地区进行开发和商业化, except in Latin America.
ATTR-CM is a systemic, 进行性和致命性疾病，可导致进行性心力衰竭并在诊断后4年内死亡.1 该病仍未得到充分诊断，由于缺乏对该病的认识和症状的异质性，该病的患病率被认为被低估.2 遗传性atr - pn是一种使人衰弱的疾病，诊断后5年内可导致周围神经损伤并伴有运动障碍, without treatment, is generally fatal within a decade.3
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, 他说:“Eplontersen有可能阻止trr介导的淀粉样变的进展, irrespective of whether it’s caused by genetic mutations or aging. Thanks to its precise liver-targeting properties, 对于患有这种毁灭性疾病、目前选择有限的患者来说，它也有可能成为一种一流的治疗方法.”
遗传性atr - pn预计将成为两家公司寻求监管机构批准eplontersen的第一个指标, 有可能在2022年底之前向美国食品和药物管理局提交新药申请.
Financial considerations
澳门在线赌城娱乐将向Ionis支付2亿美元的预付款，并在获得监管机构批准后再支付最高4.85亿美元的附加条件付款. It will also pay up to $2.根据5亿至60亿美元的销售门槛，估算90亿美元与销售相关的里程碑, 此外，根据不同地区，版税的比例在两位数到20%之间. The collaboration includes territory-specific development, commercial and medical affairs cost-sharing provisions.
该交易将以现金支付，预计2021年对核心收益中性. It will be accounted for as an intangible asset acquisition, recognised initially at the upfront amount, 任何潜在的未来里程碑付款在确认时资本化为无形资产.
Ionis将继续为现有的临床研究和工艺认证生产和供应epontersen. AstraZeneca will be responsible for commercial supply, with transition timing to be agreed by both parties. AstraZeneca will book all sales generated under the agreement.
The transaction is expected to close in the fourth quarter of 2021, subject to customary closing conditions and regulatory clearances. 该交易不会影响澳门在线赌城娱乐2021年的财务指导.
Notes
Eplontersen
Eplontersen是一种配体共轭反义(LICA)研究药物，旨在减少甲状腺转甲状腺素的产生, or TTR protein, to treat all types of ATTR, a systemic, progressive and fatal disease.
TTR Amyloidosis (ATTR)
ATTR引起的心肌病和多发性神经病是由于衰老或基因突变导致TTR蛋白错误折叠，并在心肌和周围神经中以淀粉样原纤维的形式积累而引起的, respectively. In patients with ATTR, 突变型和野生型的TTR蛋白都以原纤维的形式在组织中形成, such as the peripheral nerves, heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow. TTR原纤维的存在干扰了这些组织的正常功能. As the TTR protein fibrils enlarge, more tissue damage occurs and the disease worsens, resulting in poor quality of life and eventually death. 在世界范围内，估计有30万至50万atr - cm患者4,5 and 10,000 - 40,000 patients with ATTR-PN2.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, 澳门在线赌城娱乐在100多个国家开展业务，其创新药物被全球数百万患者使用. Please visit doobale.com and follow the Company on Twitter @AstraZeneca.
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References
1. Lauppe RE, et al. 瑞典甲状腺转蛋白淀粉样心肌病的全国患病率和特点. Open Heart. 2021 Oct;8(2):e001755. doi: 10.1136/openhrt-2021-001755.
2. González-Duarte A, et al. 非心脏临床病理特征对甲状腺素淀粉样蛋白多发性神经病患者生存的影响. Neurol Ther. 2020;9(1):135-149. doi:10.1007/s40120-020-00183-7.
3. Cortese A, et al. 遗传性转甲状腺蛋白淀粉样变合并多发性神经病的诊断挑战:避免误诊可治疗的遗传性神经病. J Neurol Neurosurg Psychiatry. 2017 May;88(5):457-458. doi: 10.1136/jnnp-2016-315262.
4. Mohamed-Salem L, et al. 老年人群骨扫描心肌摄取评估野生型ATTR的患病率. Int J Cardiol. 2018 Nov 1;270:192-196. doi: 10.1016/j.ijcard.2018.06.006.
5. Cuscaden C, et al. 利用超声心动图和临床相关性的骨扫描估计澳大利亚亚群中甲状腺素转换(ATTR)型心脏淀粉样变的患病率. J Nucl Cardiol. 2020 May 8. doi: 10.1007/s12350-020-02152-x.
Adrian Kemp
Company Secretary
AstraZeneca PLC