Ionis Pharmaceuticals (NasdaqGS:IONS) Q3 2025 Earnings Call October 29, 2025 11:30 AM ET Company ParticipantsKyle Jenne - Chief Global Product Strategy OfficerRichard Geary - Chief Development OfficerBeth Hougen - CFOWade Walke - SVPGary Nachman - Managing Director of BioPharma/Biotech Equity ResearchMike Ulz - Executive Director of Biotechnology Equity ResearchGena Wang - Managing Director and Biotech Equity ResearchBrett Monia - CEOConference Call ParticipantsMyles Minter - Biotech Equity Research Analyst ...

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]

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 ...

Core Insights - Ionis Pharmaceuticals released topline results from a pivotal study of zilganersen for Alexander disease, a rare neurological condition [1] - Zilganersen showed statistically significant stabilization in gait speed, with a mean difference of 33.3% compared to control at week 61 [2] - The drug demonstrated a positive disease-modifying impact, marking a first in AxD treatment [3] Efficacy and Safety - Zilganersen exhibited consistent benefits across key secondary endpoints, indicating slowed disease progression and stabilization [3] - Key secondary endpoints included various patient and clinician global impression scores, showing favorable trends [4] - The safety profile of zilganersen was favorable, with most adverse events being mild or moderate, and serious adverse events were numerically lower in the treatment group [4] Regulatory and Market Impact - Ionis plans to submit a new drug application to the FDA in Q1 2026 and is considering an Expanded Access Program in the U.S. [5] - Following the news, Ionis stock rose by 4.88% to $63.99 in premarket trading [5]

Q2:2025 Business Update and Financial Results July 30, 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 Brett Monia, Ph.D. Chief Executive Officer Kyle Jenne Chief Global Product Strategy Officer In this presentation, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals® is a registered trademark ...