财务数据 - 2023年第四季度和全年总收入分别为3.25亿美元和7.88亿美元[2] - 2023年全年营运费用为11.41亿美元，非GAAP基础下为10.35亿美元[2] - 2023年全年现金及短期投资达到23亿美元，支持未来投资[8] 版权费和研发 - 2023年第四季度，Ionis Pharmaceuticals的SPINRAZA版税为62百万美元，较上年同期下降了7%[23] - 研发合作协议收入从2022年的51百万美元增长至2023年的179百万美元，增长幅度达250%[23] - Ionis Pharmaceuticals的非GAAP研发和专利费用从2022年的289百万美元降至2023年的237百万美元，降幅为18%[24] 现金及投资 - 公司现金、现金等价物和短期投资从2022年的198.7亿美元增长至2023年的233.1亿美元，增长幅度为17.5%[26]

Ionis公司新闻 - Ionis最近宣布，FDA已授予其在家族性乳糜微粒血症（FCS）治疗方面的候选药物Olezarsen孤儿药物认定[1] - FCS是一种罕见的遗传疾病，其特征是极高的甘油三酯水平和反复发作的急性胰腺炎[3] - Ionis计划基于BALANCE研究的结果向FDA提交Olezarsen在FCS的新药申请，如果获批，将是Ionis首个独立推出的药物[5] - Ionis的股价在过去一年中上涨了18.8%，而行业整体下跌了5.2%[5] Ionis与合作伙伴 - Ionis拥有与Biogen、AstraZeneca、诺华和GSK等大型制药公司合作的广泛项目管线[7] - Ionis已将Spinraza授权给Biogen，后者负责在全球范围内商业化这种用于治疗脊髓性肌萎缩症（SMA）的药物[8] - Ionis与合作伙伴AstraZeneca的Wainua（eplontersen）已获得FDA批准，用于治疗遗传性甲状腺素转运蛋白介导的淀粉样多神经病（hATTR-PN）[9] - AstraZeneca和Ionis将在美国市场推广Wainua用于ATTRv-PN，而AstraZeneca则拥有在美国以外市场商业化Wainua的独家权利[10] - AstraZeneca和Ionis还在为另一种称为由遗传性TTR淀粉样蛋白病引起的心肌病（ATTR-CM）开发eplontersen[11]

Analysts on Wall Street project that Ionis Pharmaceuticals (IONS) will announce quarterly loss of $0.78 per share in its forthcoming report, representing a decline of 110.8% year over year. Revenues are projected to reach $183.6 million, increasing 20.8% from the same quarter last year.The consensus EPS estimate for the quarter has been revised 12.6% lower over the last 30 days to the current level. This reflects how the analysts covering the stock have collectively reevaluated their initial estimates durin ...

CARLSBAD, Calif., Feb. 15, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational medicine olezarsen for the treatment of familial chylomicronemia syndrome (FCS), a rare, genetic disease characterized by extremely elevated triglyceride levels and recurrent acute pancreatitis (AP). The FDA grants Orphan Drug designation to therapies intended for treatment, prevention or diagnos ...

Wall Street expects a year-over-year decline in earnings on higher revenues when Ionis Pharmaceuticals (IONS) reports results for the quarter ended December 2023. While this widely-known consensus outlook is important in gauging the company's earnings picture, a powerful factor that could impact its near-term stock price is how the actual results compare to these estimates.The stock might move higher if these key numbers top expectations in the upcoming earnings report, which is expected to be released on F ...

Ionis Pharmaceuticals与阿斯利康合作 - Ionis Pharmaceuticals宣布FDA已授予eplontersen用于治疗转甲状腺素介导的淀粉样心肌病（ATTR-CM）的快速通道指定[1] - Ionis与英国制药公司阿斯利康合作开发eplontersen[2] CARDIO-TTRansform研究 - Ionis正在进行的CARDIO-TTRansform研究是目前在给定患者群体中进行的最大规模研究，预计2025年公布数据[3] FDA的快速通道指定 - FDA的快速通道指定有助于快速开发和加快审查治疗严重疾病的候选药物，以满足未满足的医疗需求[4]

Ionis Pharmaceuticals与AstraZeneca合作 - Ionis Pharmaceuticals宣布与AstraZeneca合作的eplontersen获得FDA的快速通道指定，用于治疗成人的ATTR-CM[1] - eplontersen被认为有潜力成为ATTR-CM患者的革命性治疗方法，该疾病尽管有治疗选择仍然是一种进行性致命疾病[2] eplontersen的批准和用途 - eplontersen已在美国获得批准用于治疗成人的遗传性transthyretin介导的淀粉样蛋白病的多发性神经病变，品牌名为WAINUA™[3]

公司介绍 - Ionis Pharmaceuticals, Inc.将于2月21日上午11:30在东部时间举行网络直播[1] - 可以在https://ir.ionispharma.com/events-and-presentations/upcoming-events上访问直播[2] - Ionis Pharmaceuticals, Inc.是一家专注于发明治疗严重疾病药物的公司[3]

Ionis Pharmaceuticals (IONS) announced positive top-line results from the phase III OASIS-HAE study on its investigational drug donidalorsen in patients aged 12 years and older with hereditary angioedema (HAE).The OASIS-HAE study randomized study participants into three equal groups over a 24-week treatment period — two groups evaluating an 80mg dose of donidalorsen administered once every four weeks (Q4W) and another group wherein the drug was administered once every eight weeks (Q8W). The third group admi ...

Ionis制药公司新药donidalorsen研究结果 - donidalorsen在每4周或每8周治疗的患者中，HAE发作率显著降低，与安慰剂相比具有统计学意义[2] - donidalorsen在研究中表现出良好的安全性和耐受性，且治疗患者中没有发生严重不良事件[2] - Ionis准备向美国FDA提交新药申请，Otsuka在欧洲提交营销授权申请[3] donidalorsen的孤儿药品认定 - donidalorsen在美国获得了孤儿药品认定，欧盟的孤儿药品认定程序正在进行中[3] Ionis制药公司展示计划 - Ionis计划在即将举行的医学大会上展示第三期OASIS-HAE研究结果[6]