CARLSBAD, Calif., May 15, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will conduct its 2024 virtual Annual Meeting of Stockholders followed by a general corporate update on Thursday, June 6, 2024. The agenda for the event is as follows: About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, c ...

Ionis Pharmaceuticals (IONS) reported a loss of 98 cents per share for first-quarter 2024, which was narrower than the Zacks Consensus Estimate of a loss of $1.10 per share.The bottom line includes compensation expenses related to equity awards. Excluding these special items, adjusted loss per share was 77 cents against a loss of 68 cents per share in the year-ago quarter.Total revenues were $119 million in the first quarter, missing the Zacks Consensus Estimate of $131.5 million. Revenues declined 1.2% yea ...

财务数据和关键指标变化 - 公司在第一季度实现收入1.19亿美元,其中一半来自商业产品,主要包括SPINRAZA的大量版税收入以及WAINUA在美国上市后的版税收入 [18][19] - 非GAAP经营费用适度增加,主要是由于WAINUA在美国的上市推广以及为即将上市的olezarsen和donidalorsen做准备性投入 [18][19] - 公司预计未来几个季度的收入将略有增长,主要来自监管里程碑、许可费和合作伙伴的研发资金 [19][20] - 公司预计2024年全年经营费用将同比增长中高个位数,主要是销售和市场推广费用的增加 [19][20] - 公司预计2024年底现金和投资余额将达到17亿美元 [20][21] 各条业务线数据和关键指标变化 - WAINUA在美国的上市进展良好,与AstraZeneca的合作团队正在有效执行上市计划 [11][12] - olezarsen在FCS和SHTG两个适应症的III期临床试验进展顺利,FCS适应症已提交NDA申请,预计今年底前获批 [5][13][14] - donidalorsen在预防性治疗遗传性血管性水肿的III期OASIS-HAE试验取得积极结果,公司正准备提交监管申请 [6][7][16][17] 各个市场数据和关键指标变化 - WAINUA在美国的上市进展良好,获得主要支付方的覆盖,患者使用体验也很积极 [29][30] - olezarsen在FCS和SHTG两个市场的商业化准备工作正在进行,包括建立销售团队、医疗教育、市场准入等 [33][34][35] - donidalorsen在HAE市场的商业化准备也在进行,这是一个集中的患者群体,公司将建立适当规模的销售团队 [36][37] 公司战略和发展方向及行业竞争 - 公司正在执行战略,通过自主研发和商业化,持续推出创新疗法,为患者带来更好的未来 [3][4][10] - 公司正在加大对神经系统、心血管和罕见疾病等重点领域的投入,并利用现有商业化能力为即将上市的产品做好准备 [8][21][22][23] - 公司正在与合作伙伴共同推进WAINUA在更多市场的上市,并准备独立推出olezarsen和donidalorsen [4][5][6][7] - 公司正在评估将ION224授权给合作伙伴,以最大化该计划的价值 [72][73][76] 管理层对经营环境和未来前景的评论 - 管理层对公司2024年开局良好,有信心通过创新科技和坚持不懈的努力,为患者带来更多转型性疗法 [3][4] - 管理层对WAINUA、olezarsen和donidalorsen的上市前景表示乐观,认为这些产品有望成为各自领域的标准治疗 [4][5][6][7] - 管理层对公司未来几年的投资计划和收入增长前景表示信心,认为这将推动公司实现持续正现金流 [20][21][22][23] 问答环节重要的提问和回答 问题1 **Debjit Chattopadhyay 提问** 询问olezarsen和donidalorsen的扩展使用计划和开放标签研究中的患者数量,这些患者可能成为商业化的低垂果 [26][27] **Brett Monia 回答** 对于olezarsen,几乎所有III期试验的患者都进入了开放标签延长研究;对于donidalorsen,也有绝大部分III期试验患者进入了开放标签研究。这些患者为商业化奠定了基础 [26][27] 问题2 **Gary Nachman 提问** 询问olezarsen FCS适应症的审评时间线,以及公司在商业化方面的具体计划和与WAINUA的协同 [32][33][34] **Brett Monia、Kyle Jenne 和 Jonathan Birchall 回答** 预计在提交NDA后60天内获得FDA的反馈,包括是否获得优先审评资格。公司将利用WAINUA上市时建立的市场准入、医学教育等能力,结合针对罕见病的精准销售团队,为olezarsen的商业化做好充分准备 [32][33][34] 问题3 **Jessica Fye 提问** 询问公司实现持续正现金流的时间线,以及ION541 ALS项目III期试验的关键观察指标和时间线 [40][41][42][43] **Beth Hougen 和 Eugene Schneider 回答** 公司预计未来几年通过多个收入来源,包括现有产品、即将上市产品以及合作伙伴项目,将实现持续正现金流。对于ION541,III期试验主要关注安全性,同时也会观察一些探索性指标如神经元标志物,预计今年中期公布结果 [40][41][42][43]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Commission file number 000-19125 Ionis Pharmaceuticals, Inc. (Exact name of Registrant as specified in its charter) Delaware 33-0336973 (State or oth ...

Exhibit 99.1 Ionis reports first quarter 2024 financial results WAINUATM launch on track; EU and Canada approval decisions expected this year Olezarsen NDA submitted to FDA for FCS; preparing EU regulatory submission Positive Phase 3 donidalorsen data for HAE; preparing regulatory submissions On track to achieve 2024 financial guidance CARLSBAD, Calif., May 7, 2024 – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”), today reported financial results for the first quarter ended March 31, 2024. “Ioni ...

Ionis Pharmaceuticals (IONS) came out with a quarterly loss of $0.98 per share versus the Zacks Consensus Estimate of a loss of $1.10. This compares to loss of $0.87 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 10.91%. A quarter ago, it was expected that this drug discovery and development company would post a loss of $0.78 per share when it actually produced a loss of $0.06, delivering a surprise of 92.31%.Over the last fo ...

Wall Street analysts expect Ionis Pharmaceuticals (IONS) to post quarterly loss of $1.10 per share in its upcoming report, which indicates a year-over-year decline of 26.4%. Revenues are expected to be $131.51 million, up 0.4% from the year-ago quarter.Over the past 30 days, the consensus EPS estimate for the quarter has been adjusted downward by 1.2% to its current level. This demonstrates the covering analysts' collective reassessment of their initial projections during this period.Prior to a company's ea ...

The market expects Ionis Pharmaceuticals (IONS) to deliver a year-over-year decline in earnings on higher revenues when it reports results for the quarter ended March 2024. This widely-known consensus outlook is important in assessing the company's earnings picture, but a powerful factor that might influence 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 ...

Webcast scheduled for Tuesday, May 7 at 11:30 a.m. Eastern TimeCARLSBAD, Calif., April 23, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Tuesday, May 7th at 11:30 a.m. Eastern Time to discuss its first quarter 2024 financial results.The webcast may be accessed at https://ir.ionispharma.com/events-and-presentations/upcoming-events. A replay will be available for a limited time at the same address.About Ionis Pharmaceuticals, Inc.For three ...

研究结果 - Ionis Pharmaceuticals宣布了其主导独立调查药物olezarsen的Phase 3 Balance研究的完整结果[1] - Olezarsen在治疗家族性乳糜微粒血症（FCS）成人患者时，80毫克每月的剂量达到了显著降低三酸甘油酯（TGs）的主要终点[1] - Olezarsen在减少急性胰腺炎（AP）事件方面表现出显著效果，与安慰剂相比，患者发生AP事件的次数明显减少[1] - Olezarsen在安全性和耐受性方面表现良好，与安慰剂组相比，没有与olezarsen相关的严重不良事件[1] - Olezarsen的研究结果已在2024年美国心脏病学学会（ACC）年会上展示，并同时发表在《新英格兰医学杂志》上[1] - Olezarsen的研究结果显示，患者在12个月内的全因住院率显著降低[1] - Olezarsen对于FCS患者的治疗可能是一种突破性的新选择，尚未在美国获得批准的治疗方法[1] - Olezarsen在减少TG水平与减少AP事件发生率之间的关联性得到了验证，这为olezarsen成为FCS患者的标准治疗方法提供了支持[1] 会议信息 - Ionis将于4月8日上午10:00举行网络研讨会，讨论Balance研究的详细结果[12]