Financial Data and Key Metrics Changes - In Q3 2025, the company generated $157 million in revenue, a 17% increase year over year, and $740 million for the first nine months, reflecting a 55% increase compared to the prior year [23][24] - Tringolsa reported $32 million in net product sales, representing a nearly 70% increase quarter over quarter [13][23] - Royalty revenues increased by approximately 13% to $76 million in Q3, driven by contributions from Spinraza and Waylivra [24] - The company raised its 2025 financial guidance, now expecting total revenue between $875 million and $900 million, an increase of $50 million from prior guidance [25][26] Business Line Data and Key Metrics Changes - Tringolsa's launch is gaining momentum, with a significant increase in prescribers and patient identification efforts [15][16] - Donzera, approved for hereditary angioedema, is seeing strong early adoption, with patients switching from prior therapies and treatment-naive patients starting on Donzera [18][19] - Olzarsen showed a significant reduction in triglycerides and acute pancreatitis events, positioning it for a strong launch next year [9][10] - Zilganersen demonstrated a disease-modifying effect in Alexander disease, with plans for an independent launch next year [10][11] Market Data and Key Metrics Changes - The company anticipates targeting approximately 20,000 healthcare providers (HCPs) for Olzarsen, covering around 360,000 patients with severe hypertriglyceridemia (SHTG) [32] - The U.S. prophylactic HAE market is well established, with many patients dissatisfied with current therapies, indicating a significant opportunity for Donzera [19][66] Company Strategy and Development Direction - The company is focused on executing its commercial strategy for Tringolsa and Donzera while preparing for upcoming launches of Olzarsen and Zilganersen [22][28] - The pipeline includes multiple late-stage programs, with expectations for four key launches from the partnered pipeline by the end of 2027 [6][12] - The company aims to achieve cash flow breakeven by 2028, driving long-term value creation [26][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing momentum across the business, with strong performance from independent launches and a robust pipeline [4][28] - The company is optimistic about the potential of Olzarsen and Zilganersen to address significant unmet medical needs [10][11] - Management highlighted the importance of patient identification and education efforts to maximize the impact of new therapies [15][16] Other Important Information - The company plans to submit a new drug application for Zilganersen in Q1 2026 and is initiating an expanded access program in the U.S. [11] - The company has a strong balance sheet, expecting to end the year with over $2.1 billion in cash [26] Q&A Session Summary Question: Thoughts on the launch curve for Olzarsen in SHTG - Management indicated strong interest from HCPs and plans to target approximately 20,000 HCPs covering 360,000 patients, expecting strong uptake based on phase 3 data [32] Question: Concerns regarding acute pancreatitis events in Olzarsen studies - Management reassured that the data is groundbreaking and will be presented at AHA, with no significant concerns regarding acute pancreatitis events [36] Question: Peak revenue potential for Donzera and Olzarsen - Management expects peak sales for Donzera to exceed $500 million and for Olzarsen to exceed $1 billion [38] Question: Pricing strategy for Olzarsen - Management is still working on pricing and expects to provide more clarity next year [40] Question: Early prescriptions for Donzera - Management reported strong early adoption, with switches from existing therapies and newly diagnosed patients [66]

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Business Momentum and Pipeline Achievements - Ionis' first independent launch is exceeding expectations, with a second underway[13] - Olezarsen demonstrated statistically significant reductions of up to 72% in placebo-adjusted fasting triglycerides in sHTG patients[14, 18] - Olezarsen showed a highly statistically significant 85% reduction in adjudicated acute pancreatitis events compared to placebo[14, 21] - Zilganersen demonstrated statistically significant stabilization on the primary endpoint of gait speed in AxD patients[14] - ION582 showed consistent and meaningful improvements in key areas at 6 months in Angelman Syndrome patients[36] Commercial Performance and Strategy - TRYNGOLZA Q3 2025 net sales reached $32 million, with approximately 70% growth[13, 48] - DAWNZERA U S launch is underway with early excitement from physicians and patients[13] - The company is targeting over 3,000 physicians to increase FCS awareness[53] - DAWNZERA has a peak sales potential of over $500 million[65] Financial Highlights and Guidance - Q3 2025 commercial revenue was $116 million and R&D revenue was $41 million, totaling $157 million[75] - The company increased its 2025 revenue guidance to $875-$900 million, including $85-$95 million from TRYNGOLZA net revenues[77]

分组1 - Ionis Pharmaceuticals reported a quarterly loss of $0.61 per share, which was better than the Zacks Consensus Estimate of a loss of $1.15, representing an earnings surprise of +46.96% [1] - The company posted revenues of $157 million for the quarter ended September 2025, exceeding the Zacks Consensus Estimate by 21.23%, and showing an increase from $134 million in the same quarter last year [2] - Ionis Pharmaceuticals has surpassed consensus EPS estimates and revenue estimates for four consecutive quarters [2] 分组2 - The stock has gained approximately 110.4% since the beginning of the year, significantly outperforming the S&P 500's gain of 17.2% [3] - The current consensus EPS estimate for the upcoming quarter is -$1.18 on revenues of $141.77 million, and for the current fiscal year, it is -$2.10 on revenues of $857.58 million [7] - The Medical - Drugs industry, to which Ionis Pharmaceuticals belongs, is currently ranked in the top 37% of over 250 Zacks industries, indicating a favorable outlook compared to lower-ranked industries [8]

- TRYNGOLZA® generated $32 million in net product sales in the third quarter 2025 - - DAWNZERA™ (donidalorsen) launch of to encouraging start - - Olezarsen significantly reduced triglycerides and acute pancreatitis events in severe hypertriglyceridemia (sHTG) in landmark Phase 3 studies; sNDA submission on track by year-end – - Positive pivotal zilganersen results in Alexander disease position Ionis for first independent neurology launch in 2026 - Exhibit 99.1 Ionis reports third quarter 2025 financial resu ...

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Core Insights - Ionis Pharmaceuticals has been recognized as the 2 top employer in the biopharmaceutical industry by Science magazine in its first year of eligibility, highlighting its industry-leading innovation and strong company culture [1][2][3] Company Recognition - The ranking reflects Ionis's commitment to scientific excellence, alignment of company and employee values, and a culture of respect, as indicated by the results of approximately 5,500 completed surveys [2][3] Leadership Commentary - The CEO of Ionis, Brett P. Monia, emphasized that the recognition showcases the caliber of their science and the unique spirit of the company culture, driven by a passionate and inquisitive team [2][3] Company Overview - Ionis Pharmaceuticals has been a pioneer in RNA-targeted medicines for three decades, focusing on innovative therapies in neurology, cardiometabolic diseases, and gene editing [4]

Core Points - Ionis Pharmaceuticals, Inc. will host a live webcast on October 29th at 11:30 a.m. Eastern Time to discuss its Q3 2025 financial results and progress on key programs [1] Company Information - Ionis Pharmaceuticals has been innovating in the field of medicine for three decades [1]

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Core Viewpoint - JP Morgan upgraded Ionis Pharmaceuticals from neutral to overweight, highlighting potential label expansion for Tryngolza and promising candidates in its pipeline [2] Company Summary - The price target for Ionis Pharmaceuticals was raised from $49 to $80, indicating a potential upside of approximately 16% based on the closing price on October 7 [2]