Ionis Pharmaceuticals, Inc. · NASDAQ:IONS
Ionis Pharmaceuticals — Shareholder Meeting (June 2024)
32 slides from Ionis Pharmaceuticals's shareholder meeting presentation, Jun 6, 2024
Slide 1: 2024 Annual Shareholders Meeting Corporate Update
2024 Annual Shareholders Meeting Corporate Update June 6, 2024
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. Any statement describing Ionis’ goals, expectati
Slide 3: Executing on a Clear Vision
Executing on a Clear Vision Extending Leadership in RNA-Targeted Therapeutics Delivering a Steady Cadence of Potentially Transformational Medicines Prioritizing and Expanding Delivering Ionis Medicines Leading the Ionis Wholly Owned Directly to Patients Technology Pipeline while Supporting our Partn
Slide 4: 2024 Off To Strong Start with Several Important Achievements
2024 Off To Strong Start with Several Important Achievements New Product Launches 2 U.S launch (ATTRv-PN1) EU launch SOD1-ALS2 Positive 3 4 Phase 3 Studies Fully Enrolled4 Positive 2 Phase 3 Readouts3 Phase 2 Readouts5 ION582 in ION224 in Hereditary Angioedema Severe hypertriglyceridemia sHTG-suppor
Slide 5: Delivering Steady Cadence of Potentially Transformational Medicines
Delivering Steady Cadence of Potentially Transformational Medicines1 9 Medicines in Phase 3 for 11 indications Indication Prevalence2 Next Event3 WAINUA ATTRv-PN OUS approvals (2024) (eplontersen) ATTR-CM Ph3 data (2026)4 FCS FDA approval (2024)5 Olezarsen sHTG Ph3 data (2025) Donidalorsen HAE NDA &
Slide 6: Realizing the Promise of our Innovative Medicines
Realizing the Promise of our Innovative Medicines1 First Ionis-Branded First Ionis Independent Next Wave of Medicine2 Launches1,4 Wholly Owned Medicines Olezarsen Leading Neurology Launch in FCS expected by Pipeline YE:20244 Proven track record of delivering Launched in ATTRv- first-in-class Polyneu
Slide 7: WAINUA Approved for ATTRv-PN: Launch Underway
WAINUA Approved for ATTRv-PN: Launch Underway for the First Ionis Co-Commercialized Medicine1 For Hereditary ATTR Polyneuropathy, a systemic, progressive and fatal neurological disease 1. WAINUA: www.wainua.com; co-developing and commercializing in the U.S. with AstraZeneca. 7
Slide 8: WAINUA for ATTR-CM: Global Phase 3 Development Program
WAINUA for ATTR-CM: Global Phase 3 Development Program Designed to Deliver Robust Results Most comprehensive study to date in ATTR-CM, a fatal disease Positioned to deliver the richest data in broad patient Robust population Development Program Largest study conducted in ATTR-CM now fully enrolled w
Slide 9: Olezarsen: Blockbuster opportunity in FCS and sHTG
Two planned indications: • Starting with rare disease opportunity in FCS • Expanding to broader sHTG population Substantial unmet need Olezarsen: Positive Balance (FCS) study results3: • Robust reductions in apoC-III, TGs & favorable safety and tolerability Blockbuster opportunity • Markedly lower r
Slide 10: Olezarsen is Delivering Positive Data for FCS
Olezarsen is Delivering Positive Data Supporting its Potential as a Breakthrough Treatment for FCS1 Familial Chylomicronemia Syndrome (FCS) • Demonstrated substantial reductions in: • NDA submitted; EU filing on track this year apoC-III, TGs, acute pancreatitis, • EAP in U.S. for FCS now open, hospi
Slide 11: Positive Olezarsen Phase 3 Results in FCS Patients
Positive Olezarsen Phase 3 Results in FCS Patients1,2 Olezarsen treatment resulted in: Reduction in Serum apoC-III Levels — Robust and significant reduction in serum apoC-III levels at 6 and 12 months — Statistically significant reductions in triglycerides at 80mg dose — Substantial reductions in ac
Slide 12: Olezarsen Positive Data for FCS and sHTG
Olezarsen is Delivering Positive Data Supporting its Potential as a Breakthrough Treatment for FCS and sHTG1 Severe Hypertriglyceridemia (sHTG) • Pivotal study in patients w/ • Pivotal study in patients w/ • Supportive Ph3 study in TG ≥500 mg/dL (sHTG) TG ≥500 mg/dL (sHTG) patients w/ TG ≥200-500 mg
Slide 13: Poised to Deliver Olezarsen to the Market
Poised to Deliver Olezarsen to the Market… Focused on the unique needs of patients, caregivers, physicians and payers Building launch momentum through disease awareness and patient identification campaign Market research to identify physicians most likely to prescribe olezarsen Focus: Patients with
Slide 14: Olezarsen Launch is Designed for Commercial Success
Olezarsen Launch is Designed for Commercial Success • Establish olezarsen commercial presence Launch in FCS • Deploy and optimize Ionis’ commercial capabilities ahead of broader sHTG launch Launch in sHTG • Leverage existing treatment Priority practices of priority treaters • Expand physician Growth
Slide 15: Donidalorsen: A Potential Preferred Treatment for People with HAE
New prophylactic treatments needed3 Donidalorsen profile1: • Substantial and sustained reductions in HAE attacks Donidalorsen: • >80% preference for donidalorsen over other prophylactic treatments • Favorable safety and tolerability A Potential • Patient-friendly monthly or every two-month Preferred
Slide 16: Donidalorsen: Robust Data Supports Potential Preferred Treatment for HAE Prophylaxis
Donidalorsen: Robust Data Supports Potential Preferred Treatment for HAE Prophylaxis1 • OLE cohort demonstrated that long-term treatment • Substantial reductions in HAE attack rates + continued to improve HAE attack rates and QoL favorable safety and tolerability measures • Improved QoL measures • P
Slide 17: OLE: Further Reduction in HAE Attacks with Extended Donidalorsen Treatment
OLE: Further Reduction in HAE Attacks with Extended Donidalorsen Treatment1,2 • Q4W substantially reduced mean HAE All patients on donidalorsen attack rates: • 93% improvement from baseline at the start of OASIS-HAE3 • Q8W had a similar effect as Q4W dosing • 92% improvement from baseline at the sta
Slide 18: Donidalorsen Substantially Reduced HAE Attack Rates After Switching
Donidalorsen Substantially Reduced HAE Attack Rates After Switching1-3 % Reduction in HAE Attack Rate4 n= 31 11 22 Baseline, mean ± SD = 0.69 ± 1.09 1.80 ± 1.92 0.61 ± 0.96 Donidalorsen Q4W, mean ± SD = 0.24 ± 0.33 0.48 ± 0.53 0.36 ± 0.69 1. As of February 28, 2024 for Weeks 1-17. 2. Mean (SD). 3. B
Slide 19: >80% of Switch Patients Preferred Donidalorsen
>80% of Switch Patients Preferred Donidalorsen1,2 Data generated from independently administered survey 5% 11% 84% Prefer Donidalorsen Prefer Donidalorsen No Preference Prefer Previous Treatment All Prior Treatments Combined Lanadelumab Berotralstat C1-INH Total (n=25) (n=10) (n=20) (n=55) % of Pati
Slide 20: Donidalorsen: A Potential Preferred Choice for People with HAE
Donidalorsen: A Potential Preferred Choice for People with HAE1,2 Donidalorsen robust data demonstrated: Substantial and sustained reduction in HAE attacks HAE is a severe, rare, genetic Improvement in QoL measures and ≥90% were well-controlled3 disease Extended dosing to monthly and every two-month
Slide 21: Efficient and Targeted Approach to Reach People with HAE and HCPs
Concentrated Prescriber Base Efficient and Targeted Majority of People with HAE in the US are Treated by Allergists Approach to Reach People ~1,000 Allergist/Immunologists Manage with HAE and HCPs >70% of HAE Patients1 Efficient Field Team Planning for <100 Person Customer-Facing Team Field Sales Re
Slide 22: Leading and Validated Neurology Franchise
Leading and Validated Neurology Franchise SPINRAZA QALSODY WAINUA 3 SMA (SMN2) SOD1-ALS (SOD1) ATTRv-PN (TTR) Zilganersen Alexander disease Tofersen Approved (GFAP) Presymptomatic SOD1-ALS (SOD1) Medicines1 11 Ulefnersen FUS-ALS IONIS-MAPTRx/BIIB080 (FUS) Alzheimer’s disease (Tau) Medicines ION582 I
Slide 23: Our Next Wave: Wholly Owned Neurology Medicines in Clinical Development
Our Next Wave: 7 Wholly Owned Neurology Medicines in Clinical Development by YE:2024 with More to Follow1 Rare Pediatric Dementia Future Wave Neurology Zilganersen Alexander Disease ION717 Pivotal study underway Prion Disease Neuromuscular (PRNP) and Peripheral First in patient study underway Neurop
Slide 24: Positioned to Deliver Steady Cadence of Medicines to Power Revenue Growth
Positioned to Deliver Steady Cadence of Medicines to Power Revenue Growth1 Bepirovirsen (HBV) Hepatitis B Infection Pelacarsen Lp(a) CVD WAINUA (TTR) ATTR Cardiomyopathy WAINUA (TTR) Pelacarsen ATTRv Polyneuropathy Lp(a) CVD QALSODY (SOD1) WAINUA (TTR) SOD1-ALS ATTR Cardiomyopathy SPINRAZA (SMN) WAI
Slide 25: Clear Path to Drive Value Creation
Clear Path to Drive Value Creation Foundation Invest Growth Solid Invest to Bring Important Financial Foundation Medicines to Patients Positive Cash Flow and Powered by Robust Innovative Advancing Pipeline Substantial Revenue Pipeline Position Ionis and Technology Growth to Drive Value to Drive Stea
Slide 26: Advancing RNA and DNA Technologies for Future Medicines
Advancing RNA and DNA Technologies for Future Medicines Expanding Technology Optimizing Expanding Therapeutic Platform Delivery Opportunities Broad Range of Technologies Targeted Delivery Established Franchises ASO | siRNA | DNA Editing (e.g., LICA) Cardiovascular | Neurology Optimizing Cardiac Musc
Slide 27: Key Value-Driving Events Planned For 2024
Key Value-Driving Events Planned For 20241 Phase 3 Phase 2 New Product Regulatory Actions Clinical Data Events Clinical Data Events Launches Eplontersen Donidalorsen Donidalorsen OUS approvals, OASIS-HAE topline 3-year OLE, HAE ATTRv-PN data WAINUA OUS filings, ATTRv-PN ATTRv-PN4 IONIS-FB-LRx OASIS-
Slide 28: Driving Next-Level Value for Patients and All Ionis Stakeholders
Well-Positioned to Build on Momentum by Executing on Strategic Priorities Integrated Commercial Wholly Owned Pipeline Capabilities in Place 01 Advancing and growing our wholly owned pipeline in focused therapeutic areas (neurology and cardiology) 02 Steady cadence of new potentially transformational
Slide 29: Q&A Session
Q&A Session To ask a question, simply type your question in the “Submit a Question” box below and click “Send” 29
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Slide 31: Appendix
Appendix COO ae IONIS
Slide 32: Responsibility Program Supports Impact & Value
Responsibility Program Supports Impact & Value Ionis Corporate Responsibility Strategic Pillars Innovate to improve the Empower our employees lives of people with and communities serious diseases We are committed to fostering an We innovate across the business inclusive culture that drives and work
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