– Accelerating value creation with numerous commercial, regulatory and pipeline milestones –– Ionis outlines clear path to sustained positive cash flow –CARLSBAD, Calif., Jan. 13, 2025 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced highlights from the Company's 2024 achievements and previewed important milestones expected in 2025. Ionis will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15 at 10:30am PT; the presentation is a ...

公司进展与未来规划 - 公司预计在2025年将有四个关键临床试验结果公布，并计划在2026年至2028年间推出四款商业化产品 [1] - 公司预计在2025年第一季度完成ulixacaltamide的Essential3项目的中期分析，并计划在2025年提交新药申请（NDA） [1] - 公司预计在2026年提交relutrigine的新药申请，并计划在2025年上半年启动elsunersen的关键试验 [3] - 公司现金及投资总额约为4.7亿美元，足以支持其运营至2028年 [1] 产品管线更新 Ulixacaltamide（用于特发性震颤） - 特发性震颤在美国有700万患者，目前治疗不足且负担沉重 [4] - Essential3项目包括两项III期注册研究，Study 1为平行设计、安慰剂对照研究（N=400），Study 2为随机撤药研究（N=200） [4] - 自2023年11月开始招募以来，已有超过10万名患者表示有兴趣参与研究 [4] - 公司计划在2025年提交ulixacaltamide的新药申请，并在Essential3项目取得积极结果后重新启动其在帕金森病中的研究 [5] Vormatrigine（用于常见癫痫） - 美国约有350万患者患有常见癫痫，钠通道疗法是治疗基石，但现有药物存在安全性和疗效限制 [5] - Vormatrigine是迄今为止设计的最强效钠通道调节剂，精准靶向成人常见癫痫中的钠通道过度兴奋状态 [6] - RADIANT研究预计在2025年上半年公布顶线结果，POWER1研究预计在2025年下半年公布顶线结果 [8] - EMPOWER观察性研究已招募超过2000名患者，旨在更好地描述癫痫负担 [8] Relutrigine（用于发育性和癫痫性脑病） - 发育性和癫痫性脑病在美国影响约20万患者，通常表现为出生时或出生后不久发作 [7] - Relutrigine在EMBOLD研究的队列1中显示出前所未有的疗效，46%的安慰剂调整后月度运动性癫痫发作减少 [9] - 公司计划在2025年上半年启动EMERALD注册研究，并预计在2026年上半年公布EMBOLD研究队列2的顶线结果 [9] Elsunersen（用于早期发作性SCN2A-DEE） - SCN2A-DEE是一种罕见的遗传性癫痫，早期发作且对发育有严重影响 [11] - EMBRAVE研究的第1部分分析显示，四名患者在四个月内癫痫负担减少了43% [11] - 公司计划在2025年第一季度分享elsunersen的注册研究设计和预期时间表 [16] 其他进展 - 公司与UCB达成协议，UCB选择授权KCNT1小分子候选药物的全球开发和商业化权利，公司可获得高达1亿美元的开发和商业化里程碑付款 [16] - 公司计划在2025年提名其早期ASO治疗计划的开发候选药物，包括PRAX-080、PRAX-090和PRAX-100 [16] 公司背景 - 公司是一家临床阶段的生物制药公司，专注于将遗传学见解转化为治疗中枢神经系统（CNS）疾病的疗法，特别是神经元兴奋-抑制失衡相关的疾病 [19] - 公司通过其专有的小分子平台Cerebrum™和ASO平台Solidus™，开发针对罕见和常见神经系统疾病的多样化、多模式CNS产品组合 [19]

CARLSBAD, Calif., Jan. 8, 2025 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Brett P. Monia, Ph.D., chief executive officer, will present a company overview at the 43rd Annual J.P. Morgan Healthcare Conference at 10:30 a.m. PT on Wednesday, January 15, 2025. A live webcast of the presentation can be accessed on the Investors & Media section of the Ionis website at https://ir.ionis.com/events. A replay will be available on the Ionis website within 48 hours and will be archiv ...

Shares of Ionis Pharmaceuticals (IONS) rose nearly 5% in after-market trading on Thursday after the FDA approved its RNA-targeted therapy, olezarsen, in adults with familial chylomicronemia syndrome (FCS). The drug will be marketed under the trade name Tryngolza.The FDA’s nod to Tryngolza makes it the first approved treatment for FCS in the country. The drug’s commercial launch is part of management's broad strategy to deliver a steady flow of wholly-owned medicines to patients. Besides being Ionis’ first w ...

TRYNGOLZA shown to significantly reduce triglycerides and substantially reduce acute pancreatitis events in adults with FCS; a rare, highly debilitating and life-threatening disease Indicated for adults with FCS regardless of genetically or clinically confirmed diagnosis  TRYNGOLZA is the first of four independent launches planned over the next three years, pending approvals Ionis to host webcast today at 6:45pm ET CARLSBAD, Calif., Dec. 19, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) ...

Ionis Pharmaceuticals (IONS) closed the last trading session at $38.26, gaining 1.3% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $59.65 indicates a 55.9% upside potential.The average comprises 20 short-term price targets ranging from a low of $37 to a high of $77, with a standard deviation of $10.76. While the lowest estimate indicates a decline of 3.3% from the current price l ...

Ionis Pharmaceuticals (IONS) could be a solid addition to your portfolio given its recent upgrade to a Zacks Rank #2 (Buy). This rating change essentially reflects an upward trend in earnings estimates -- one of the most powerful forces impacting stock prices.The Zacks rating relies solely on a company's changing earnings picture. It tracks EPS estimates for the current and following years from the sell-side analysts covering the stock through a consensus measure -- the Zacks Consensus Estimate.Since a chan ...

Ionis Pharmaceuticals (IONS) has been beaten down lately with too much selling pressure. While the stock has lost 13.5% over the past four weeks, there is light at the end of the tunnel as it is now in oversold territory and Wall Street analysts expect the company to report better earnings than they predicted earlier.How to Determine if a Stock is OversoldWe use Relative Strength Index (RSI), one of the most commonly used technical indicators, for spotting whether a stock is oversold. This is a momentum osc ...

Ionis Pharmaceuticals (IONS) incurred a loss of 95 cents per share for third-quarter 2024, which was narrower than the Zacks Consensus Estimate of a loss of $1.16 per share.Earnings include compensation expenses related to equity awards. Excluding these special items, adjusted loss per share was 72 cents compared with a loss of 85 cents per share in the year-ago quarter.Total revenues were $134 million in the third quarter, beating the Zacks Consensus Estimate of $129.0 million. Revenues declined 7% year ov ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Title of each class Trading symbol Name of each exchange on which registered Common Stock, $.001 Par Value "IONS" The Nasdaq Stock Market LLC 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 September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Com ...