Core Insights - Ionis Pharmaceuticals announced positive top-line results from the phase III ESSENCE study for its drug Tryngolza, targeting moderate hypertriglyceridemia in patients at risk for atherosclerotic cardiovascular disease (ASCVD) [1][2] - The study achieved its primary endpoint, showing a statistically significant reduction in triglyceride (TG) levels, with reductions of 61% and 58% for 80 mg and 50 mg monthly doses, respectively [2] - A majority of participants in the study had their TG levels fall within the normal range (<150 mg/dL) post-treatment, and the drug met all key secondary endpoints [3] Recent Developments - Tryngolza received FDA approval for treating familial chylomicronemia syndrome (FCS), marking it as the first approved treatment for this rare genetic condition in the U.S. and Ionis' first independent commercial launch [4] - Ionis out-licensed ex-U.S. rights for Tryngolza to Sweden-based Sobi, which will handle future regulatory filings and commercialization outside the U.S. [5] - The drug is also being evaluated for severe hypertriglyceridemia (sHTG) in two late-stage studies, with data expected in Q3 2025 [6][7] Financial and Revenue Streams - Ionis has a diverse revenue stream through collaborations with major pharmaceutical companies like AstraZeneca, Biogen, GSK, and Novartis, providing funds for its development pipeline [10] - The company earns commercial revenues from royalties on Spinraza, which treats spinal muscular atrophy, and is also involved in marketing Qalsody for amyotrophic lateral sclerosis [11] - Recent approvals for drugs like Wainua and ongoing studies for other candidates could further enhance Ionis' revenue and reduce reliance on collaboration partners [12][14]

节前，阿斯利康在Q1财报会上高调宣布，将全面退出CNS（中枢神经）领域，终止多个管线项目。 这是继阿斯利康提出"800亿美元"收入目标后的第一次大的管线战略抉择。 消息一出，也引起全行业的巨震。毕竟，同时停掉一整个治疗领域是需要相当大的决心的，这意味着大 量的研发投入打水漂，钱是小事，重要的是巨大的时间成本。 阿斯利康的选择很好理解：CNS领域产品开发难度比想象中要高，而为了尽快实现800亿目标，公司只 能在短期去找确定高的大品种去卷执行能力（比如减肥药和ADC），短中期的高KPI压力，逼着公司很 难去从长期考虑CNS这种"潜在回报巨大，但投入周期漫长"的方向。 CNS领域这几年发展可谓是"喜忧参半"。喜在以礼来、渤健以及福泰等公司在机理和产品获批上均实现 部分突破。"忧"这一块，除了阿斯利康，安进、辉瑞、赛诺菲等在此前均放弃该领域产品开发。 而前年艾伯维87亿天价收购的Cerevel Therapeutics约莫重新燃起了CNS领域的热度，如今，其核心用于 治疗精神分裂症的产品Emraclidine，也在两项二期临床中宣告失败。 大厂们的选择是一个"超级风向标"。那么对于国内这些本土CNS龙头，其中不乏恩华 ...

Biogen (BIIB) reported first-quarter 2025 adjusted earnings per share (EPS) of $3.02, which missed the Zacks Consensus Estimate of $3.32.Earnings declined 18% year over year on a reported basis, owing to costs related to an upfront payment of $165 million made to Stoke Therapeutics (STOK) . BIIB entered into a collaboration agreement with STOK for the development and commercialization of the latter’s pipeline candidate, zorevunersen for treating Dravet syndrome in February. (Find the latest EPS estimates an ...

Core Viewpoint - Ionis Pharmaceuticals reported a narrower adjusted loss in Q1 2025 compared to the previous year and exceeded revenue expectations, driven by strong sales from its licensed products and new drug approvals [1][2][6]. Financial Performance - The adjusted loss per share for Q1 2025 was 75 cents, better than the Zacks Consensus Estimate of a loss of $1.11 and an adjusted loss of 77 cents in the same quarter last year [1]. - Total revenues reached $132 million, surpassing the Zacks Consensus Estimate of $120 million, marking an 11% increase year over year [2]. - Commercial revenues amounted to $76 million, a 29% increase year over year, exceeding the Zacks Consensus Estimate of $67 million [6]. - R&D revenues declined 7% year over year to $56 million, but still beat the Zacks Consensus Estimate of $50 million [10]. Revenue Streams - Ionis earns royalties from Biogen on the sales of Spinraza and Qalsody, with Spinraza royalties totaling $48 million, up 26% year over year [7]. - Wainua generated $9 million in royalty revenues, with sales recorded at $39 million by AstraZeneca [7]. - Tryngolza contributed $6 million in product sales during its first quarter of recognition [6]. Cost Structure - Adjusted operating costs rose 5% year over year to $249 million, with SG&A costs increasing by 52% to support commercialization efforts [11]. - R&D costs decreased by 6% as several late-stage studies concluded [11]. Guidance and Future Outlook - Ionis raised its 2025 revenue guidance to between $725 million and $750 million, up from over $600 million, reflecting new licensing deals [12]. - The adjusted operating loss is now expected to be less than $375 million, improved from the previous guidance of less than $495 million [15]. - The company anticipates ending 2025 with approximately $1.9 billion in cash, up from a prior projection of $1.7 billion [16]. Drug Development Updates - Tryngolza is under evaluation in three late-stage studies for severe hypertriglyceridemia, with data expected in 2025 [18]. - Donidalorsen is awaiting FDA approval for hereditary angioedema, with a decision expected by August 21, 2025 [19]. - Zilganersen is in a phase III study for Alexander disease, with data expected in 2025 [20]. Partnerships and Collaborations - AstraZeneca and Ionis are co-marketing Wainua for hereditary transthyretin-mediated amyloid polyneuropathy in the U.S. and have plans for further development in other forms of amyloidosis [21]. - Ionis has out-licensed rights for a rare blood cancer drug to Ono Pharmaceutical, receiving an upfront payment of $280 million and potential milestone payments [23].