市场表现： 2025 年5 月22 日，医药板块涨跌幅-0.73%，跑输沪深300 指数0.67pct，涨跌幅居申万31 个子行业第14 名。各医药子行业中，医疗研发外包(+0.04%)、血液制品(-0.37%)、其他生物制品（-0.51%）表现居 前，医院（-1.83%)、线下药店(-1.65%)、医药流通（-1.37%)表现居后。个股方面，日涨幅榜前3 位分别 为三生国健(+20.00%)、亚虹医药(+12.26%)、迈威生物(+11.71%)；跌幅榜前3 位为赛升药业 （-11.63%）、拓新药业(-10.93%)、拱东医疗（-9.99%)。 行业要闻： 近日，Ionis 宣布，公司开发的反义寡核苷酸（ASO）疗法Tryngolza（Olezarsen）在Essence 临床3 期研 究中取得积极的顶线结果。数据显示，该药在治疗6 个月后显著降低了患者的甘油三酯水平，达到主要 终点，且在所有关键次要终点上也取得统计学显著改善。Olezarsen 是一种反义寡核苷酸疗法，旨在抑 制机体产生载脂蛋白C-III（apoC-III，一种在肝脏中产生的调节血液中TG 代谢的蛋白）。 风险提示：新药研发及上市不及预 ...

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]

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].

Core Insights - Ionis Pharmaceuticals reported a first-quarter EPS loss of 93 cents, an improvement from a loss of 98 cents, and better than the consensus estimate of a $1.12 loss [1] - Revenue for the first quarter of 2025 increased by 10% to $132 million, surpassing the consensus of $125.32 million, driven by higher commercial revenue [1] Financial Guidance - The company raised its 2025 revenue guidance by over 20% to a range of $725-$750 million, compared to previous guidance of over $600 million and the consensus of $659.37 million [4] - Ionis expects a 2025 adjusted operating loss of less than $375 million, improved from prior guidance of less than $495 million, with cash and equivalents projected at approximately $1.9 billion [4] Product Performance - The drug Tryngolza (olezarsen) generated over $6 million in net product sales in its first full quarter post-approval in the U.S. [6] - Wainua (eplontersen) for polyneuropathy generated $39 million in sales, resulting in $9 million in royalty revenue for the first quarter of 2025 [6] - Spinraza (nusinersen) for spinal muscular atrophy achieved global sales of $424 million, leading to $48 million in royalty revenue in the first quarter of 2025 [6]