Ionis Pharmaceuticals, Inc. · NASDAQ:IONS
Ionis Pharmaceuticals — 44th Annual J.P. Morgan Healthcare Conference 2026
25 slides from Ionis Pharmaceuticals's conference presentation, January 2026
Slide 1: Breakthrough Therapies Driving Accelerating Growth
44th Annual J.P. Morgan Healthcare Conference 2026 Breakthrough Therapies Driving Accelerating Growth Brett Monia, Ph.D. Chief Executive Officer January 2026 | Nasdaq: IONS
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: 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 4: 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 5: DAWNZERA: A Novel Therapeutic for HAE Prophylaxis
DAWNZERA: A Novel Therapeutic for HAE Prophylaxis1 Delivering on What HAE Patients Need Most First and Only RNA-Targeted Treatment to Prevent HAE Attacks Encouraging Early Launch Indicated for prophylaxis Momentum to prevent attacks of HAE in adult and Prescriptions written for all patient pediatric
Slide 6: 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 7: Olezarsen: A Transformational Medicine for All sHTG Patients with Urgent Unmet Need
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 8: TRYNGOLZA Outperforms Expectations in First Year of Launch
TRYNGOLZA Outperforms Expectations in First Year of Launch as the First FDA-Approved Treatment for FCS1 Generated $105 million in 20253 50%3 $483 $32 Robust efficacy and safety • Significant and sustained triglyceride reductions $19 • Substantial reduction in acute pancreatitis events Convenience of
Slide 9: Olezarsen Phase 3 Program Designed to Support Opportunity in sHTG
Olezarsen Phase 3 Program Designed to Support Opportunity in sHTG Pivotal Studies in People with sHTG (fasting TG ≥500 mg/dL) Primary Endpoint Percent change in fasting triglycerides from baseline to month 6 Acute Pancreatitis Secondary Endpoint Adjudicated event rate between pooled olezarsen compar
Slide 10: Vast Majority of Patients Achieved Significant Reductions in Fasting Triglycerides
Achieved Highly Statistically Significant Reductions in Fasting Triglycerides at 6 Months Vast Majority of Patients Treated with Olezarsen Up to a 72% placebo-adjusted mean reduction in fasting triglycerides1 Achieved (p<0.0001) Triglyceride Levels Below Risk Threshold Up to for Acute 86% achieved T
Slide 11: Olezarsen: First Investigational Treatment to Reduce Acute Pancreatitis Events
Achieved Highly Statistically Significant Reduction in Adjudicated Acute Pancreatitis Events 85% Olezarsen: The First & Only Investigational Reduction in acute pancreatitis events Treatment to compared to placebo at 12 months1 Significantly (p=0.0002) Reduce Acute Number Needed to Treat (NNT) over J
Slide 12: Olezarsen: Poised to Become Ionis' First Blockbuster Medicine
Olezarsen: Poised to Become Ionis’ First Blockbuster Medicine Highly statistically significant and clinically meaningful reductions in fasting triglycerides with favorable safety and Annual Peak Product tolerability1 Revenue Opportunity2 First and only investigational treatment to significantly Incr
Slide 13: Building a Leading Cardiometabolic Pipeline
Building a Leading Wholly Owned Medicines Cardiometabolic Pipeline 1,2 Indication Preclinical Ph1 Ph2 Ph3 Olezarsen sHTG sNDA Submitted (ApoC-III) 2 4 ION775 (ApoC-III) ION501 sHTG Myocardial disease (TfR1-Targeting) Wholly Owned Partnered (undisclosed) Medicines Medicines ION924 Cardiovascular dise
Slide 14: Proven Platform and Capabilities for Delivering Medicines for Neurological Diseases
Proven Platform and Capabilities for Delivering Groundbreaking Medicines for Neurological Diseases Approved Medicines1-3 14 1. SPINRAZA.com 2. QALSODY.com. 3. WAINUA.com.
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 (GFAP) Alexander disease NDA submission planned for Q1:2026 Neurological Diseases ION582 (UBE3A-ATS) Angelman syndrome ION464 Multiple System Atrophy (SNCA) 6 6 ION717 (PRNP) ION356 Prion diseas
Slide 16: Zilganersen: First Anticipated Launch from Wholly Owned Neurology Portfolio
Zilganersen: First Anticipated Launch from Wholly Owned Neurology Portfolio1-3 • ~1 in 1-3 million people with Alexander disease (AxD), The • Progressive and often fatal condition Opportunity • Accounts for ~2-8% of leukodystrophies, although likely underreported4,5 • First and only investigational
Slide 17: ION582: A Promising Investigational Medicine for Angelman Syndrome
ION582: 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 study
Slide 18: Delivering a Steady Cadence of New Medicines
Delivering a Steady Cadence of New Medicines1,2 ION582 (Angelman syndrome) 4 Sefaxersen Independent (IgAN) launches in 2025-2026 Eplontersen (ATTR-CM) Pelacarsen 4 (Lp(a)-CVD) Key partner launches by end of 2027 Bepirovirsen (CHB) Zilganersen (Alexander disease) Olezarsen (sHTG) (HAE) (FCS) 2026 Lau
Slide 19: >$4B Marketed Products and Planned Launches
Ionis-Owned Medicines >$4B in Potential Annual Peak Product Revenue2 Marketed Products and Planned Launches Provide Substantial Revenue Growth >$6B Opportunity1 Partner Medicines >$2B in Potential Annual Peak Royalties2 1. Assumes additional approvals through 2028. Estimated timing of potential U.S.
Slide 20: Clear Path to Sustained Positive Cash Flow
Clear Path to Sustained Positive Cash Flow1 Key Drivers New product launches Growing royalty revenue Positive Cash Flow Critical investments Strong financial for growth 2028 foundation Cash Flow Breakeven Disciplined expense management 1. Based on current assumptions, subject to change. 20
Slide 21: Well Positioned to Continue Driving Breakout Growth: Key Catalysts in 2026
Well Positioned to Continue Driving Breakout 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 Multiple Alzheimer’s Disease (T
Slide 22: Driving Accelerating Growth
Driving Accelerating Growth1,2 2028 Cash Flow Breakeven Clear Path to Sustained Positive Cash Flow Accelerating Revenue Growth Building a Leading Cardiometabolic Disease Portfolio Leading the Way in the Treatment of Neurological Diseases 1. Based on current estimates, subject to change. 2. Assuming
Slide 23: Breakthrough Therapies Driving Accelerating Growth
Breakthrough Therapies Driving Accelerating Growth 23
Slide 24: 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 Clinical Data Events Lp(a) HORIZON data Clinical Daa Events EU approval U.S. approval U.S. submission & Clinical Data Events B
Slide 25: Focused High-Value Pipeline to Drive Continued Growth
Focused High-Value Pipeline to Drive Continued Growth Indication Preclinical Ph1 Ph2 Ph3 Olezarsen (ApoC-III) Severe hypertriglyceridemia sNDA submitted ION775 (ApoC-III) Severe hypertriglyceridemia ION501 (Undisclosed) Myocardial disease ION924 (Apo(a)) Cardiovascular disease Cardiometabolic ION573
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