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Ionis-2026_01_14_JPMorgan_Presentation.pdf

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1、44thAnnual J.P.Morgan Healthcare Conference 2026Brett Monia,Ph.D.Chief Executive OfficerBreakthrough Therapies Driving Accelerating GrowthJanuary 2026|Nasdaq:IONSForward-Looking StatementsThis presentation includes forward-looking statements regarding our business,financial guidance and the therapeu

2、tic and commercial potential of our commercial medicines,additional medicines in development and technologies and our expectations regarding development and regulatory milestones.Any statement describing Ionis goals,expectations,financial or other projections or guidance,intentions or beliefs is a f

3、orward-looking statement and should be considered an at-risk statement.Such statements are subject to certain risks and uncertainties including but not limited to those related to our commercial products and the medicines in our pipeline,and particularly those inherent in the process of discovering,

4、developing and commercializing medicines that are safe and effective for use as human therapeutics,and in the endeavor of building a business around such medicines.Ionis forward-looking statements also involve assumptions that,if they never materialize or prove correct,could cause its results to dif

5、fer materially from those expressed or implied by such forward-looking statements.Although Ionis forward-looking statements reflect the good faith judgment of its management,these statements are based only on facts and factors currently known by Ionis.Except as required by law,we undertake no obliga

6、tion to update any forward-looking statements for any reason.As a result,you are cautioned not to rely on these forward-looking statements.These and other risks concerning Ionis programsare described in additional detail in Ionis annual report on our Form 10-K for the year ended December 31,2024,and

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1. **公司定位与增长路径**:Ionis为全整合商业化生物科技公司,通过突破性技术驱动高价值管线,加速收入增长,目标2028年实现现金流盈亏平衡。 2. **核心产品进展**: - **Olezarsen**:针对严重高甘油三酯血症(sHTG),III期显著降低甘油三酯(最高72%)及急性胰腺炎事件(NNT=5),2026年美国上市,潜在峰值年销售额超$20亿。 - **Zilganersen**:治疗亚历山大病,NDA计划2026年Q1提交,突破性疗法认定。 - **TRYNGOLZA**:2025年美国销售额$1.05亿,欧盟已启动。 3. **管线布局**: - 心血管领域:6款临床阶段药物(如Pelacarsen针对Lp(a))。 - 神经领域:12款临床阶段药物(如ION582治疗Angelman综合征)。 4. **2026年关键事件**:5项III期数据读出、3款产品上市(包括Bepirovirsen慢性乙肝)、4项NDA提交。
**Ionis新药前景?** **HAE治疗突破?** **神经疾病进展?**
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