Ionis Pharmaceuticals, Inc. · NASDAQ:IONS
Ionis Pharmaceuticals — Q3 2024 Earnings Presentation
31 slides from Ionis Pharmaceuticals's earnings presentation, Nov 6, 2024
Slide 1: Q3:24 Business Update and Financial Results
Q3:24 Business Update and Financial Results November 6, 2024 Nasdaq: IONS
Slide 2: On Today's Earnings Call
On Today’s Earnings Call Brett Monia, Ph.D. Eugene Schneider, M.D. Kyle Jenne Beth Hougen Chief Executive Officer Chief Clinical Development Chief Global Chief Financial Officer Officer Product Strategy Officer Richard Geary, Ph.D. Eric Swayze, Ph.D. Jonathan Birchall Chief Development Officer Execu
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: Realizing the Promise of our Wholly Owned Innovative Medicines
Realizing the Promise of our Wholly Owned Innovative Medicines1,2 Olezarsen Donidalorsen ION582 First-mover Advantage for Potential Preferred Potential Transformational Two Patient Populations: Prophylactic Treatment Medicine for FCS and sHTG for HAE Angelman Syndrome Independent U.S. Launch August
Slide 6: Numerous Important Achievements in 2024 To Date
2 Numerous Important Achievements in 2024 To Date New Product Launches U.S launch EU launch 4 6 4 (ATTRv-PN)1 (SOD1-ALS)2 Positive Phase 3 Studies Positive Phase 3 Readouts3 Fully Enrolled4 Phase 2 Readouts5 Olezarsen Donidalorsen Olezarsen Donidalorsen ION582 (OASIS-HAE & OASISplus (CORE, CORE2 & E
Slide 7: Delivering Important Pipeline Achievements
Delivering Important Pipeline Achievements Eugene Schneider, M.D. Chief Clinical Development Officer 7
Slide 8: Olezarsen: Wholly Owned Blockbuster Opportunity
Two planned indications: • Starting with rare disease opportunity in FCS • Expanding to broader sHTG population Substantial unmet need Olezarsen: Positive Balance (FCS) study results4: • Robust reductions in apoC-III, TGs & favorable safety and tolerability Wholly Owned • Markedly lower rate of acut
Slide 9: Donidalorsen: A Wholly Owned Potential Preferred Treatment for HAE
New prophylactic treatments needed3 Donidalorsen’s clinical results include1: • Substantial and sustained reductions in HAE attacks Donidalorsen: • New positive Phase 2 OLE data in patients treated up to three years • Improved QoL measures A Wholly Owned Potential • High levels of disease control Pr
Slide 10: 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 11: Positioned to Deliver Steady Cadence of Potentially Transformational Medicines
Positioned to Deliver Steady Cadence of Potentially Transformational Medicines1 9 investigational 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
Slide 12: Leading Neurology Franchise
Leading Neurology Franchise SPINRAZA 3 SMA (SMN2) Zilganersen Tofersen Alexander disease Presymptomatic SOD1-ALS (SOD1) Approved (GFAP) Medicines1 13 Ulefnersen FUS-ALS IONIS-MAPTRx/BIIB080 Alzheimer’s disease (Tau) (FUS) ION859 Medicines Parkinson’s disease ION582 in Clinical Angelman syndrome (LRR
Slide 13: ION582: Positive Early Results Seen in the HALOS Study
Positive Early Results Seen in the HALOS Study1 • Consistent and meaningful improvements in key areas of clinical function, including communication, cognition ION582: and motor function • Evidence of consistent improvements across age groups and genotypes A Promising New • Favorable safety and toler
Slide 14: Upcoming Key Value-Driving Events
Upcoming Key Value-Driving Events1 Q4:2024 and 2025 Phase 2 Phase 3 New Product Regulatory Actions Clinical Data Events Clinical Data Events Launches Eplontersen Olezarsen OUS approvals, CORE, CORE2, ATTRv-PN ESSENCE data WAINUA Sapablursen sHTG EU + other countries Olezarsen ATTRv-PN Polycythemia v
Slide 15: 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 15
Slide 16: WAINUA Approved for ATTRv-PN: Launch Progressing Well
WAINUA Approved for ATTRv-PN: Launch Progressing Well for the First Ionis Co-Commercialized Medicine1 Substantial and sustained Q-o-Q growth of 44% driven by strong demand2 Encouraging patient mix and breadth of prescribers Physicians report positive patient experience: • Quality-of-life improvement
Slide 17: Olezarsen: Designed to Address Two Patient Populations
Rare disease opportunity3-5 Familial No approved treatments in the U.S. Chylomicronemia Olezarsen: Syndrome Significant risk for acute, potentially fatal pancreatitis Designed to Planned first indication launch with high margin potential Address Two Patient Populations Large addressable market6-9 wi
Slide 18: Comprehensive Launch Approach Focused on Targeted Education, Engagement and Patient Support
Comprehensive Launch Approach Focused on Targeted Education, Engagement and Patient Support Comprehensive Education Field Medical Patient Access Commercial and Patient and Payer and Support Execution Identification Engagement Programs 18
Slide 19: Our Second Planned Independent Launch: Donidalorsen for HAE
Our Second Planned Independent Launch: Donidalorsen for HAE 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 Neede
Slide 20: 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 21: Delivering Medicines to People in Need
Delivering 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 January
Slide 22: Q3 2024 Financial Performance
Q3 2024 Financial Performance Beth Hougen Chief Financial Officer 22
Slide 23: Q3:2024 YTD Financial Highlights
Q3:2024 YTD Financial Highlights1 On Track to Achieve 2024 P&L Guidance; Increased Cash Guidance to ~$2.2 Billion Commercial Revenue: $207M R&D Revenue: $272M • SPINRAZA comprised largest • Reflects the value Ionis’ $479M Revenue component pipeline and technology • New stream of royalty revenue crea
Slide 24: On Track to Achieve 2024 P&L Financial Guidance
On Track to Achieve 2024 P&L Financial Guidance Increased Cash Guidance to ~$2.2B Reflects Equity Offering Proceeds Revenue Operating Loss Cash >$575 <$475 from $1.7B ~$2.2 from $1.7B million million1 billion Prior: $1.7 billion Expectations for 2024: Revenue: Substantial and sustained • Commercial:
Slide 25: 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 25
Slide 26: Conclusion
Conclusion Brett Monia, Ph.D. Chief Executive Officer 26
Slide 27: 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 28: Q&A
Q&A
Slide 29: Slide 29
Slide 30: Appendix
Appendix OOOO... ae IONIS =
Slide 31: Key Value-Driving Events Planned For 2024
Key Value-Driving Events Planned For 20241 Phase 2 Phase 3 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 OUS filings, ATTRv-PN WAINUA U.S. ATTR
Ionis Pharmaceuticals — Q3 2024 Earnings Presentation on 6ix