Ionis Pharmaceuticals Inc. (IONS) announced that the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency has adopted a positive opinion recommending approval of DAWNZERA for the routine prevention of recurrent attacks of hereditary angioedema in adults and adolescents aged 12 years and older.Hereditary angioedema (HAE) is a rare, potentially life-threatening genetic condition characterized by recurrent episodes of severe swelling in various parts of the body, including ...

Financial Data and Key Metrics Changes - In Q3 2025, the company generated $157 million in revenue, a 17% increase year-over-year, and for the first nine months, revenue totaled $740 million, reflecting a 55% increase compared to the prior year [23][24] - TRYNGOLZA product sales reached $32 million, representing a nearly 70% increase over the second quarter [23][24] - Royalty revenues increased by approximately 13% to $76 million in Q3, driven by contributions from SPINRAZA and WAINUA [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 - TRYNGOLZA, the FDA-approved treatment for familial chylomicronemia syndrome, continues to show strong momentum with significant sales growth [5][14] - DAWNZERA, approved for hereditary angioedema, has seen strong early adoption, with initial prescriptions being filled shortly after approval [19][20] - The company anticipates two additional independent launches next year, with olzarsen for severe hypertriglyceridemia and zilganersen for Alexander disease [6][10] Market Data and Key Metrics Changes - The U.S. prophylactic HAE market is well established, with approximately 20% of patients switching treatments annually, indicating a significant opportunity for DAWNZERA [20] - The severe hypertriglyceridemia (sHTG) patient population is estimated to exceed 1 million in the U.S., with many patients struggling to manage triglyceride levels with current treatments [16][17] Company Strategy and Development Direction - The company is focused on executing its commercial strategy for TRYNGOLZA and DAWNZERA while preparing for upcoming launches of olzarsen and zilganersen [22][27] - The company aims to achieve cash flow breakeven by 2028, driving long-term value creation [26][27] - The commercial team is expanding outreach to healthcare providers and enhancing patient identification efforts to maximize the potential of its therapies [15][17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the continued success of TRYNGOLZA and DAWNZERA, highlighting the strong early feedback from physicians and patients [19][20] - The company is optimistic about the upcoming data presentations and regulatory submissions, which are expected to further validate its pipeline and market potential [9][11] - Management emphasized the importance of addressing unmet needs in serious diseases and the potential for multi-billion dollar revenue from its pipeline [7][10] Other Important Information - The company announced the retirement of Richard Geary, Chief Development Officer, recognizing his contributions over the past 30 years [28] - The company is preparing for a significant data presentation at the American Heart Association (AHA) conference, which is expected to highlight the efficacy of olzarsen [38][39] Q&A Session Summary Question: What is the expected launch curve for olzarsen in sHTG? - The company anticipates strong interest from healthcare providers and plans to target approximately 20,000 HCPs covering around 360,000 patients with sHTG [31][33] Question: Any concerns regarding acute pancreatitis events in olzarsen studies? - Management expressed confidence in the groundbreaking results and emphasized that the data will be presented at AHA, with no significant concerns noted [37][38] Question: What are the peak revenue expectations for olzarsen and DAWNZERA? - Peak sales for DAWNZERA are expected to exceed $500 million, while olzarsen is anticipated to surpass $1 billion [39][40] Question: What is the pricing strategy for olzarsen? - The company is still evaluating pricing and will announce final pricing upon approval of the sHTG indication [44][45] Question: How is the early adoption of DAWNZERA progressing? - Early adoption is strong, with both switching patients and newly diagnosed patients starting treatment [72] Question: What is the source of revenue growth for WAINUA? - The revenue growth is primarily driven by new patient identification and the product's performance in improving quality of life [73]

Q3:2025 Business Update and Financial Results October 29, 2025 Nasdaq: IONS 1 On Today's Earnings Call Eugene Schneider, M.D. Chief Clinical Development Officer Eric Swayze, Ph.D. Executive Vice President, Research 2 Richard Geary, Ph.D. Chief Development Officer Kyle Jenne Chief Global Product Strategy Officer Beth Hougen Chief Financial Officer Forward-Looking Statements This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial pote ...

2 Ionis Leadership Here Today Innovation Day 2025: Accelerating Growth through Life-Changing Medicines OCTOBER 7, 2025 | Nasdaq: IONS Forward-Looking Statements This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic 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, fina ...

Company Highlights - Aurinia Pharmaceuticals Inc. (AUPH) has seen solid growth for its product LUPKYNIS, with expected net product sales for 2025 ranging from $250 million to $260 million, up from $216.2 million last year [2][3] - Arrowhead Pharmaceuticals Inc. (ARWR) is preparing for its first commercial launch in 2025, with its lead drug candidate Plozasiran under FDA review, decision expected on November 18, 2025 [4][5] - Beta Bionics Inc. (BBNX) reported a 54% growth in second-quarter net sales, with expectations for full-year 2025 net sales to range between $88 million and $93 million, compared to $65.1 million last year [7][8] - Kiniksa Pharmaceuticals International plc (KNSA) anticipates net product revenue for its drug Arcalyst to be between $625 million and $640 million for 2025, up from $417 million in 2024 [10][11] - Ionis Pharmaceuticals Inc. (IONS) generated net product sales of $19 million in Q2 2025, with a supplemental new drug application expected by year-end [12][14] - Stoke Therapeutics Inc. (STOK) is developing Zorevunersen for Dravet syndrome, with a 52-week high of $24.60 reached recently, representing an 82% gain in less than two months [16][17] - Fortress Biotech Inc. (FBIO) is awaiting an FDA decision on its investigational drug CUTX-101 for Menkes disease, expected on September 30, 2025 [18][21] Stock Performance - AUPH stock increased by 168% over 1.5 years, reaching a 52-week high of $12.87 [3] - ARWR stock gained 65% in less than 9 months, hitting a 52-week high of $31.13 [5] - BBNX stock rose nearly 55% in less than 4 months, reaching a 52-week high of $26.66 [8] - KNSA stock saw a 46% increase in less than four months, touching a 52-week high of $37.34 [11] - IONS stock gained 52% in less than two months, reaching a 52-week high of $64.72 [15] - STOK stock increased by 82% in less than two months, achieving a 52-week high of $24.60 [17] - FBIO stock rose by 61% in less than a month, reaching a 52-week high of $3.97 [21]