Arrowhead Pharmaceuticals (ARWR) FY Conference Summary Company Overview - **Company**: Arrowhead Pharmaceuticals (ARWR) - **Event**: FY Conference held on June 09, 2025 - **Key Speakers**: James Hamilton (CMO and Head of R&D), Vince Ambulone (VP of Finance and IR) Key Points Industry Context - Arrowhead is positioned in the biotechnology sector, focusing on RNA interference (RNAi) therapeutics, particularly for rare diseases and metabolic disorders [5][6][8]. Financial Health - The company has successfully augmented its balance sheet, securing funding through 2028, which allows for multiple potential independent commercial launches [7][8][66]. Product Pipeline - **Plazasiran**: - First independent commercial launch anticipated with a PDUFA date set for November 18, 2025 [5][6]. - Targeting triglyceride lowering for familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG) [6][20]. - Positive data from the PALISADE Phase III study supports its efficacy [6]. - **Ongoing Studies**: - Full enrollment for Phase III studies expected by mid-2025, with completion and subsequent launch into SHTG anticipated in 2026 [7][20]. - Additional studies targeting rare muscular diseases and obesity assets are also in the pipeline, with readouts expected later in 2025 [8]. Competitive Landscape - Arrowhead acknowledges the competitive market for triglyceride-lowering therapies, particularly with Ionis Pharmaceuticals' product, Olezarsen [19][20]. - The company believes that having multiple players in the market will enhance disease state education and patient access [18][20]. Regulatory and Market Strategy - Arrowhead is confident in its communications with the FDA regarding the approval process for Plazasiran, despite the uncertainties in the biotech sector [11][12]. - The company is preparing for potential pricing strategies, considering market dynamics and the competitive landscape [22][23]. Clinical Trials and Safety - The SHASTA-5 study is designed to assess pancreatitis risk in high-risk patients, which is crucial for payer acceptance, especially in European markets [27][28][36]. - Arrowhead is focused on demonstrating a material benefit in reducing acute pancreatitis events to enhance reimbursement prospects [28][37]. Future Developments - Arrowhead is exploring combination therapies with existing GLP-1 agents to enhance weight loss outcomes and improve patient adherence [60][62]. - The company is also advancing its CNS platform, targeting ATAXN2 and tau expression, with clinical trials expected to commence within the next 6-12 months [65]. Business Development - Arrowhead plans to pursue additional partnerships and collaborations to leverage its discovery engine and expand its pipeline [66][68]. - The company is open to product and discovery partnerships, particularly in the cardiometabolic space, to support its commercialization efforts [68]. Conclusion Arrowhead Pharmaceuticals is strategically positioned for growth with a robust pipeline and a strong financial foundation. The upcoming launch of Plazasiran and ongoing studies in various therapeutic areas highlight the company's commitment to addressing unmet medical needs in the biotechnology sector. The competitive landscape and regulatory environment will be critical factors influencing Arrowhead's market success in the coming years.

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 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]