Ionis Pharmaceuticals, Inc. · NASDAQ:IONS
Ionis Pharmaceuticals — Q2 2024 Earnings Presentation
35 slides from Ionis Pharmaceuticals's earnings presentation, Aug 1, 2024
Slide 1: Q2:24 Business Update and Financial Results
Q2:24 Business Update and Financial Results August 1, 2024 Nasdaq: IONS
Slide 2: On Today's Earnings Call
On Today’s Earnings Call Brett Monia, Ph.D. Richard Geary, Ph.D. Kyle Jenne Beth Hougen Chief Executive Officer Chief Development Officer Chief Global Chief Financial Officer Product Strategy Officer Eric Swayze, Ph.D. Eugene Schneider, M.D. Jonathan Birchall Executive Vice President, Research Chief
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. Any statement describing Ionis’ goals, expectati
Slide 4: Introduction
Introduction Brett Monia, Ph.D. Chief Executive Officer 4
Slide 5: Numerous Important Achievements in 2024 To Date
2 Numerous Important Achievements in 2024 To Date New Product Launches 3 6 2 U.S launch (ATTRv-PN)1 EU launch (SOD1-ALS)2 Positive Phase 3 Studies Fully Positive Phase 3 Readouts3 Enrolled4 Phase 2 Readouts5 Severe Severe sHTG-supporting ION224 in Hereditary Angioedema hypertriglyceridemia hypertrig
Slide 6: WAINUA: Poised to deliver benefit to underserved patient population
Poised to deliver benefit to underserved patient population ATTRv-PN: Approved with strong clinical profile1,2 • U.S. launch progressing well for the first WAINUA: Ionis co-commercialized medicine • EU regulatory decision expected this year3 Potential to be the ATTR-CM: Global Phase 3 Preferred Trea
Slide 7: Olezarsen: Blockbuster Opportunity
Two planned indications2: • 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
Slide 8: Donidalorsen: A Potential Preferred Treatment for HAE
New prophylactic treatments needed3 Donidalorsen’s clinical results include1: • Substantial and sustained reductions in HAE attacks Donidalorsen: • >80% preference for donidalorsen over other prophylactic treatments4 • Favorable safety and tolerability A Potential • Patient-friendly monthly or every
Slide 9: ION582 for Angelman Syndrome: Positive Early Results
Positive Early Results Seen in the HALOS Study1 • Consistent and meaningful improvements in key areas of clinical function, including communication, cognition ION582 for and motor function • Evidence of consistent improvements across age Angelman groups and genotypes Syndrome: • Favorable safety and
Slide 10: Delivering Important Pipeline Achievements
Delivering Important Pipeline Achievements Richard Geary, Ph.D. Executive Vice President, Development 10
Slide 11: 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 12: Olezarsen Delivered Robust Data for FCS
Olezarsen Delivered Robust Data Supporting its Potential as a Breakthrough Treatment for FCS1 • NDA accepted for Priority Review, December 19, 2024 PDUFA; EU filing on track for this year2 • Positive data presented at ACC, published in NEJM1 • Olezarsen demonstrated substantial reductions in apoC-II
Slide 13: Olezarsen sHTG Development Program
Olezarsen sHTG Development Program Designed to Support Blockbuster Market Opportunity1 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/dL (HTG) • Registrati
Slide 14: Donidalorsen: Robust Data Supports Potential Preferred Treatment for HAE Prophylaxis
Donidalorsen: Robust Data Supports Potential Preferred Treatment for HAE Prophylaxis1,2 Hereditary Angioedema Phase 2 • Positive Phase 2 data • Substantial reductions in HAE • OLE cohort demonstrated that long-term published in New England attack rates + favorable safety and treatment continued to i
Slide 15: Positioned to Deliver Steady Cadence of Potentially Transformational Medicines
Positioned to Deliver Steady Cadence of Potentially Transformational Medicines1 9 Medicines in Phase 3 for 11 indications Indication Prevalence2 Anticipated 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)
Slide 16: Leading Neurology Franchise
Leading Neurology Franchise SPINRAZA 3 SMA (SMN2) 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 ION859 in Clinical Angelman syndrome Parkinson’s disease Deve
Slide 17: ION582: Important Wholly Owned Program
A severe neurodevelopmental disorder of significant unmet need • Estimated 1 in 21,000 people with Angelman syndrome worldwide2 • >100,000 people in major geographies2 ION582: Positive early results seen in the HALOS study1: • Consistent and meaningful improvements in key areas of clinical Important
Slide 18: 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 Donidalorsen Eplontersen OASIS-HAE topline Donidalorsen OUS approvals, data 3-year OLE, HAE ATTRv-PN OASIS-HAE full data WAINUA OUS filings, ATTRv-PN U.S. ATTR
Slide 19: Preparing to Bring Important Ionis Medicines to Patients
Preparing to Bring Important Ionis Medicines to Patients Kyle Jenne Chief Global Product Strategy and Operations Officer 19
Slide 20: WAINUA Approved for ATTRv-PN: Launch Progressing Well
WAINUA Approved for ATTRv-PN: Launch Progressing Well for the First Ionis Co-Commercialized Medicine1 For Hereditary ATTR Polyneuropathy, a systemic, progressive and fatal disease 1. WAINUA: www.wainua.com; co-developing and commercializing in the U.S. with AstraZeneca. 20
Slide 21: Olezarsen: Designed to Address Two Patient Populations
Rare disease opportunity ~1-13 people per million in the U.S.4-6 Familial No approved treatments in the U.S. Chylomicronemia Olezarsen: Syndrome Significant risk for acute, potentially fatal pancreatitis Designed to Potential first indication launch for olezarsen Address Two Patient Populations Larg
Slide 22: 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 Market research to identify physicians most likely to prescribe olezarsen Patients with FCS Patient & ca
Slide 23: HAE Landscape Dynamics Underscore Donidalorsen's Potential
HAE Landscape Dynamics Underscore Donidalorsen’s Potential1,2 Well Defined Growing New People with Concentrated Efficient Population Global Treatment HAE Prescriber Commercial with >20K Market Options Have Shown Base Model People with Needed Willingness in the US HAE to Switch in U.S. & EU 1. Market
Slide 24: Donidalorsen: Clinical Results Support Potential to be a Preferred Choice
Donidalorsen: Clinical Results Support Potential to be a Preferred Choice for People with HAE1,2 Potential first-in-class RNA-targeted medicine Substantial and sustained attack rate reduction with long-term durability and disease control demonstrated in the studies Strong patient preference results
Slide 25: Ready to Deliver Medicines to People in Need
Ready to Deliver Medicines to People in Need Olezarsen Donidalorsen Co-Developing and Independent U.S. Launch in FCS Independent U.S. Launch in HAE Co-Commercializing expected by YE:20242,3 expected in 20252,3 in the U.S. with AstraZeneca Large Patient Population 3 Building on Launched in ATTRv-PN J
Slide 26: Q2 2024 Financial Performance & Clear Path to Positive Cash Flow
Q2 2024 Financial Performance & Clear Path to Positive Cash Flow Beth Hougen Chief Financial Officer 26
Slide 27: H1:2024 Financial Highlights
H1:2024 Financial Highlights1 On Track to Achieve 2024 Guidance Commercial Revenue: $132M R&D Revenue: $213M • SPINRAZA comprised largest • Reflects the value Ionis’ $345M Revenue component pipeline and technology • New stream of royalty create as programs revenue with WAINUA launch advance R&D Expe
Slide 28: On Track to Achieve 2024 Financial Guidance
On Track to Achieve 2024 Financial Guidance Revenue Operating Loss Cash >$575 <$475 ~$1.7 million million1 billion Expectations for 2024: Revenue: Substantial and sustained • Commercial: sustained SPINRAZA royalties; WAINUA royalties • R&D: multiple sources from numerous advancing programs Operating
Slide 29: Investing Efficiently to Drive Positive Cash Flow
Investing Efficiently to Drive Positive Cash Flow Go-to-Market Integrated commercial capabilities in place; Activities right-sizing and scaling for successful launches Modest Expense Growth over the Short- and Mid-Term Late-Stage Ionis’ current large Phase 3 studies are fully enrolled Medicines SG&A
Slide 30: Clear Path to Drive Value Creation
Clear Path to Drive Value Creation Foundation Invest Growth Invest to Bring Important Medicines to Patients Positive Cash Flow Robust Innovative Powered by Pipeline Positions Ionis Advancing Pipeline Substantial Revenue to Drive Value and Technology Growth to Drive Steady Cadence of New Medicines 30
Slide 31: Conclusion
Conclusion Brett Monia, Ph.D. Chief Executive Officer 31
Slide 32: Q2:2024 Achievements Accelerate Positive Momentum
Q2:2024 Achievements Accelerate Positive Momentum New Medicines for • Strong WAINUA launch continued for hereditary ATTR polyneuropathy1 Patients Today • QALSODY approved for SOD1-ALS in the EU2 Olezarsen: • Positive FCS data presented at ACC, published in NEJM • NDA for FCS accepted for Priority Re
Slide 33: Ionis is Well-Positioned for Substantial Growth
Ionis is Well-Positioned for Substantial Growth 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 medicines to the market E
Slide 34: Q&A
Q&A
Slide 35: Slide 35
Ionis Pharmaceuticals — Q2 2024 Earnings Presentation on 6ix