Ionis Pharmaceuticals, Inc. · NASDAQ:IONS
Ionis Pharmaceuticals — Shareholder Meeting (June 2026)
22 slides from Ionis Pharmaceuticals's shareholder meeting presentation, June 2026
Slide 1: 2026 Annual Shareholders Meeting Corporate Update
2026 Annual Shareholders Meeting Corporate Update June 2026 Nasdaq: IONS 1
Slide 2: 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 3: Ionis: Pioneered the Field of Oligonucleotide Therapeutics
Ionis: Pioneered the A Rich History Discovering and Developing Field of Oligonucleotide Transformational RNA-Targeted Medicines Therapeutics Created Industry Optimized and Leading Medicinal Validated Delivery Chemistry and to Liver and CNS Manufacturing for Human Capabilities Therapeutics 4 Partnere
Slide 4: Well Positioned for Accelerating Growth
Well Positioned for Accelerating Growth Fully integrated, commercial-stage biotechnology company Groundbreaking technology fueling high-value innovative pipeline Consistently delivering breakthrough clinical results enabling highly successful commercial launches1,2 Clear path to accelerating revenue
Slide 5: Strong Track Record of Industry-Leading Success
Strong Track Record of Industry-Leading Success1-4 Key Recent Achievements 6 Positive Phase 3 4 Approved Data Readouts Medicines 2 Independent 11Medicines in Launches Late-Stage Development 1. TRYNGOLZA is approved in the U.S. for Familial Chylomicronemia Syndrome (FCS) in adults as an adjunct to di
Slide 6: 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 7: Delivering Transformational Medicines in Focused Therapeutic Areas
Delivering Transformational Medicines in Focused Therapeutic Areas Cardiometabolic Rare and prevalent patient populations Neurology in focused disease First- or best-in-class medicines that areas First- or best-in-class medicines target cardiometabolic diseases, the to address a broad range of leadi
Slide 8: Olezarsen: A Transformational Medicine for All sHTG Patients
Olezarsen: A Transformational Medicine for All sHTG Patients with Urgent Unmet Need1 Familial • Up to ~3,000 people in the U.S.2-6 Chylomicronemia • Rare genetic disorder: Most severe form of sHTG caused by Syndrome loss of LPL activity6,7 (FCS) • Significant risk for acute, potentially fatal pancre
Slide 9: Building Sustainable TRYNGOLZA Demand in FCS
Focused Commercial Execution Building Sustainable TRYNGOLZA Demand in FCS1 Generated $27 million in Q1:2026 +350% $27 Robust efficacy and safety • Substantial reductions in triglycerides and in acute pancreatitis events $19 Convenience of once-monthly self-administration with an autoinjector $6 EU l
Slide 10: 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 11: Building a Leading Cardiometabolic Pipeline
Building a Leading Wholly Owned Medicines Cardiometabolic Pipeline 1,2 Indication Preclinical Ph1 Ph2 Ph3 Olezarsen sHTG PDUFA: June 30, 2026 (ApoC-III) 2 4 ION775 (ApoC-III) ION501 sHTG Myocardial disease (TfR1-Targeting) Wholly Owned Partnered (undisclosed) Medicines Medicines ION924 Cardiovascula
Slide 12: Leading Neurology Portfolio Positioned to Deliver Accelerating Value
Leading Neurology Portfolio Positioned to 3 Approved Medicines1 Deliver Accelerating Value Strong track record in delivering first-in-class neurology medicines Well-positioned to deliver a steady cadence of medicines2 • Strong pipeline of wholly owned and partnered medicines in clinical development
Slide 13: 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, p
Slide 14: Obudanersen: A Promising Investigational Medicine for Angelman Syndrome
Obudanersen: A Promising Investigational Medicine for Angelman Syndrome • >100k people in major geographies with Angelman syndrome, The a severe, rare neurodevelopmental disorder1 Opportunity • Significant unmet need with no approved disease-modifying treatments • Positive results seen in the HALOS
Slide 15: Leading the Way in the Treatment of Neurological Diseases
Wholly Owned Medicines Leading the Way in Indication Preclinical Ph1 Ph2 Ph3 the Treatment of Zilganersen Alexander disease PDUFA: September 22, 2026 Neurological Diseases (GFAP) Obudanersen Angelman syndrome (UBE3A-ATS) ION464 Multiple System Atrophy 7 6 (SNCA) ION717 Prion disease (PRNP) Wholly Ow
Slide 16: 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 17: Delivering a Steady Cadence of New Medicines
Delivering a Steady Cadence of New Medicines1,2 Obudanersen (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
Slide 18: >$5B Marketed Products and Planned Launches
Ionis-Owned Medicines >$5B in Potential Annual Peak Product Revenue2 Marketed Products and Planned Launches Provide Substantial Revenue Growth >$7B Opportunity1 Partner Medicines >$2B in Potential Annual Peak Royalties2 1. Assumes additional approvals through 2028. Estimated timing of potential U.S.
Slide 19: Clear Path to Sustained Positive Cash Flow
Clear Path to Sustained Positive Cash Flow1 Key Drivers New product launches Growing royalty revenue Critical investments for growth Positive Cash Flow 2022 2027 2028 and beyond Strong financial foundation Disciplined expense Today management 1. Based on current assumptions, subject to change. 19
Slide 20: Driving Accelerating Growth
Driving Accelerating Growth1,2 2028 Cash Flow Breakeven Clear Path to Sustained Positive Cash Flow Accelerating Revenue Growth Fully-integrated commercial stage biotechnology company Steady cadence of positive clinical trial results, successful product approvals and launches Established leadership i
Slide 21: Q&A Session
Q&A Session To ask a question, simply type your question in the “Submit a Question” box below and click “Send” 21
Slide 22: Delivering Accelerated Value
Delivering Accelerated Value
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