Ionis Pharmaceuticals, Inc. · NASDAQ:IONS
Ionis Pharmaceuticals — Q1 2026 Earnings Presentation
23 slides from Ionis Pharmaceuticals's earnings presentation, Apr 29, 2026
Slide 1: First Quarter 2026 Business Update & Financial Results
First Quarter 2026 Business Update & Financial Results April 29, 2026 Nasdaq: IONS 1
Slide 2: On Today's Earnings Call
On Today’s Earnings Call Kyle Jenne Brett Monia, Ph.D. Chief Global Product Beth Hougen Chief Executive Officer Strategy Officer Chief Financial Officer Holly Kordasiewicz, Ph.D. Eugene Schneider, M.D. Eric Swayze, Ph.D. Chief Development Officer Chief Clinical Executive Vice President, Development
Slide 3: Forward-Looking Statements
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 re
Slide 4: Introduction
Introduction Brett Monia, Ph.D. Chief Executive Officer 4
Slide 5: Well Positioned to Continue Driving Accelerating Growth
Well Positioned to Continue Driving Accelerating Growth Key Catalysts in 20261 5 Phase 3 Bepirovirsen Pelacarsen 4 NDA Zilganersen Bepirovirsen 3 Olezarsen Zilganersen Eplontersen Pelacarsen Launches Bepirovirsen Data Sefaxersen Submissions Eplontersen Readouts Ulefnersen 2 Phase 3 Salanersen Sapabl
Slide 6: Strengthening Our Commercial Foundation
Strengthening Our Commercial Foundation Providing multi-billion-dollar revenue potential for Ionis1 First Independent Preparations Preparations on Launch Generating Second Independent on Track for First Track for First Strong Patient Launch Underway Broad Patient Independent Demand Population Launch
Slide 7: Olezarsen: Poised to Become Ionis' First Multi-Billion Dollar Medicine
Olezarsen: Poised to Become Ionis’ First Multi-Billion Dollar Medicine >3 million people with sHTG in the U.S.1 • Includes >1 million people with high-risk sHTG1 • Early launch focus on high-risk sHTG with >880 mg/dL or ≥500 mg/dL + AP history and/or comorbidities Annual Peak Product Revenue Opportu
Slide 8: Zilganersen: Preparations on Track for First Independent Neurology Launch
Zilganersen: Preparations on Track for First Independent Neurology Launch1 Zilganersen for the Treatment of Alexander Disease First and only investigational medicine to demonstrate clinically meaningful disease-modifying impact Prevalence: ~1 in 1-3 million; accounts for ~2-8% of leukodystrophies, a
Slide 9: Delivering a Steady Cadence of New Medicines
Delivering a Steady Cadence of New Medicines1,2 ION582 (Angelman syndrome) Ulefnersen (FUS-ALS) 4 Independent Sefaxersen (IgAN) launches in 2025-2026 Eplontersen (ATTR-CM) Pelacarsen 5 (Lp(a)-CVD) Partner launches by end of 2027 Bepirovirsen (CHB) Zilganersen (Alexander disease) Olezarsen (sHTG) (HA
Slide 10: Building on our Commercial Success
Building on our Commercial Success Kyle Jenne Chief Global Product Strategy Officer 10
Slide 11: Focused Commercial Execution Building Sustainable TRYNGOLZA Demand
Focused Commercial Execution Building Sustainable TRYNGOLZA Demand1 Generated $27 million in Q1:2026 +350% $27 Robust efficacy and safety • Significant and sustained triglyceride reductions $19 • Substantial reduction in acute pancreatitis events Convenience of once-monthly self-administration $6 wi
Slide 12: Strong Commercial Execution and Compelling Product Profile Driving Increasing TRYNGOLZA Demand
Strong Commercial Execution and Compelling Product Profile Driving Increasing TRYNGOLZA Demand1,2 Strong Robust Physician Broad Patient Uptake Engagement Access Effective patient identification efforts; strong Targeting ~20k physicians with Broad FCS access and coverage patient growth expanded field
Slide 13: Olezarsen in sHTG: Launch Readiness and a Larger Opportunity
Olezarsen in sHTG: Launch Readiness and a Larger Opportunity Groundbreaking Robust Payer Engagement Pivotal sHTG Results1 HCP Demand Educating on clinical and Highly statistically significant and economic burden of disease clinically meaningful reductions Strong enthusiasm for and associated budget
Slide 14: DAWNZERA Launch Gaining Significant Momentum
DAWNZERA Launch Gaining Significant Momentum1 Delivering on What HAE Patients Need Most First and Only RNA-Targeted Treatment to Prevent HAE Attacks Generated $16 million in Q1:2026 Indicated for prophylaxis to prevent attacks of HAE in adult and +128% pediatric patients ≥12 years old $16 Prescripti
Slide 15: Zilganersen: Ionis' First Anticipated Neurology Launch
Substantial Unmet Need Alexander disease is a rare, progressive and often fatal neurological condition No approved disease-modifying treatments Zilganersen: Groundbreaking Phase 3 Data Ionis’ First First time an investigational medicine has shown a positive disease- modifying impact in Alexander dis
Slide 16: Innovative Commercial Organization with Proven Ability to Bring Medicines to People
Innovative Commercial Organization with Proven Ability to Bring Medicines to People with Serious Diseases BB Top-Tier Team Demonstrated Scaling Capabilities Strong Initial for Upcoming Launch Execution Launches 16
Slide 17: Q1:2026 Financial Performance and 2026 Financial Guidance
Q1:2026 Financial Performance and 2026 Financial Guidance Beth Hougen Chief Financial Officer 17
Slide 18: Q1:2026 Financial Highlights
Q1:2026 Financial Highlights1 Commercial Revenue: $108M R&D Revenue: $138M Revenues $27M in TRYNGOLZA product sales Includes approximately $95M of $246M $16M in DAWNZERA product sales milestone payments from multiple Total commercial revenues increased partnerships ~42% YoY R&D Expenses2: $1
Slide 19: Improved 2026 Financial Guidance Reflects Growing Contribution from Ionis Launches
Improved 2026 Financial Guidance Reflects Growing Contribution from Ionis Launches1-3 Revenue Operating Loss Cash $875-$900M $425-475M4 >30% vs 20253 Similar to 20253 >$1.6B Previous: $800-825M Previous: $500-550M No Change Increase of $75M Improved by $75M versus prior guidance versus prior guidanc
Slide 20: Conclusion
Conclusion Brett Monia, Ph.D. Chief Executive Officer 20
Slide 21: 2026 Key Value-Driving Events
2026 Key Value-Driving Events1 Clinical Events Regulatory Actions Product Launches Phase 3 Phase 2 Pelacarsen Phase 2 Phase 2 Bepirovirsen Donidalorsen Olezarsen Zilganersen DAWNZERA Clinical Data Events Lp(a) HORIZON data Clinical Daa Events EU approval U.S. approval U.S. submission ClinicalEU Data
Slide 22: Breakthrough Therapies Driving Accelerating Growth
Breakthrough Therapies Driving Accelerating Growth 22
Slide 23: Q&A
Q&A IONIS
Ionis Pharmaceuticals — Q1 2026 Earnings Presentation on 6ix