Ionis Pharmaceuticals, Inc. · NASDAQ:IONS
Ionis Pharmaceuticals — Q1 2024 Earnings Presentation
30 slides from Ionis Pharmaceuticals's earnings presentation, May 7, 2024
Slide 1: Q1:24 Business Update and Financial Results
Q1:24 Business Update and Financial Results May 7, 2024 Nasdaq: IONS
Slide 2: On Today's Earnings Call
On Today’s Earnings Call Brett Monia, Ph.D. Kyle Jenne Beth Hougen Chief Executive Officer Chief Global Product Strategy Officer Chief Financial Officer Richard Geary, Ph.D. Eugene Schneider, M.D. Chief Development Officer Chief Clinical Development Officer Eric Swayze, Ph.D. Jonathan Birchall 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: 2024 Off To Strong Start with Several Important Achievements
2024 Off To Strong Start with Several Important Achievements WAINUA Launched in U.S. for ATTRv-PN1 Positive 2 2 Phase 3 Studies 2 Additional Pipeline Phase 3 Readouts2 Fully Enrolled3 Achievements4 ION224 Positive Phase 2 ION356 Study Data (MASH) Start (PMD) 1. WAINUA: www.wainua.com. 2. Balance (ol
Slide 6: 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 most robust data in broad patient Robust population Development Program Largest study conducted in ATTR-CM now fully enrolled w
Slide 7: Olezarsen Delivering Robust Data for FCS and sHTG
Olezarsen is Delivering Robust Data Supporting its Potential as a Breakthrough Treatment for FCS and sHTG1 Familial Chylomicronemia Syndrome (FCS) • Demonstrated substantial reductions in apoC-III, • Phase 2b study in patients with TG ≥150 mg/dL TGs, marked acute pancreatitis reductions, (HTG) and T
Slide 8: Olezarsen Delivering Robust Data for FCS and sHTG (sHTG studies)
Olezarsen is Delivering Robust 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/d
Slide 9: Donidalorsen Delivering Robust Data for Prophylactic HAE Treatment
Donidalorsen is Delivering Robust Data Supporting its Potential to Advance Prophylactic HAE Treatment1,2 Hereditary Angioedema Phase 2 • Positive Phase 2 data published • Positive Phase 3 topline data, • Phase 3 OLE study in patients in New England Journal of including achieving: who have completed
Slide 10: Leading and Validated Neurology Franchise
Leading and Validated Neurology Franchise SPINRAZA QALSODY WAINUA 3 SMA (SMN2) SOD1-ALS (SOD1) ATTRv-PN (TTR) Zilganersen Tofersen Alexander disease Approved (GFAP) Presymptomatic SOD1-ALS (SOD1) Medicines1 12 ION717 Prion disease IONIS-MAPTRx/BIIB080 (PRNP) Alzheimer’s disease (Tau) Medicines Ulefn
Slide 11: 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 approval OASIS-HAE topline 3-year OLE, HAE decisions, ATTRv-PN data WAINUA OUS filings, ATTRv-PN ATTRv-PN4 IONIS-FB-L
Slide 12: 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 12
Slide 13: WAINUA Approved for ATTRv-PN: Launch Underway
WAINUA Approved for ATTRv-PN: Launch Underway for the First Ionis Co-Commercialized Medicine1 For ATTR Polyneuropathy, a systemic, progressive and fatal neurological disease 1. WAINUA: www.wainua.com; co-developing and commercializing in the U.S. with AstraZeneca. 13
Slide 14: WAINUA: Positioned to Address the High Unmet Need in ATTR
WAINUA: Positioned to Address the High Unmet Need in ATTR1,2,3,4 Ocular Manifestation Cardiovascular Potential to be the treatment of choice for Lumbar Spinal Manifestations Stenosis the global ATTR population with strong clinical profile and monthly self- GI administered auto-injector dosing Manife
Slide 15: Olezarsen: Potential Standard-of-Care for Severely Elevated Triglycerides
Substantial unmet need Positive Balance study results3: • Robust reductions in apoC-III, TGs & favorable Olezarsen: safety and tolerability • Markedly lower rate of acute pancreatitis vs. placebo Potential to become the NDA submitted for FCS, potential FDA Standard-of-Care for approval in 2024; EU f
Slide 16: 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 17: Donidalorsen: A Potential First-in-Class RNA-Targeted Medicine for HAE
Substantial unmet need remains • Potentially fatal breakthrough attacks • Desire for greater treatment simplicity and tolerability Donidalorsen profile1,2: • Significant and sustained reductions in HAE Donidalorsen: attacks (near elimination) • Attractive tolerability and safety • Simplicity of mont
Slide 18: Commercial Infrastructure in Place
Commercial Infrastructure in Place: Ready to Deliver Medicines to People in Need Omnichannel Market Access New Product Market Research Commercial Strategy and Analytics Patients Health Economics and Patient Services Outcomes Research Commercial Marketing Operations Field Team to be Added For Launche
Slide 19: Ionis' Q1 2024 Financial Performance & Clear Path to Positive Cash Flow
Ionis’ Q1 2024 Financial Performance & Clear Path to Positive Cash Flow Beth Hougen Chief Financial Officer 19
Slide 20: Q1:2024 Financial Highlights
Q1:2024 Financial Highlights1 On Track to Achieve 2024 Guidance Commercial Revenue: $59M R&D Revenue: $60M $119M Revenue • SPINRAZA comprised largest component • Reflects the value Ionis’ pipeline • New stream of royalty revenue with and technology create as programs WAINUA launch advance R&D Expens
Slide 21: 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 22: 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’ large Phase 3 studies are at or near full enrollment Medicines
Slide 23: Steady Cadence of Medicines to Power Revenue Growth
Positioned to Deliver Steady Cadence of IONIS-FB-LRx IgA Nephropathy Medicines to Power Revenue Growth1 Bepirovirsen (HBV) Hepatitis B Infection Pelacarsen Lp(a) CVD WAINUA (TTR) ATTR Cardiomyopathy WAINUA (TTR) ATTRv Polyneuropathy QALSODY (SOD1) Pelacarsen SOD1-ALS Lp(a) CVD SPINRAZA (SMN) WAINUA
Slide 24: 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 25: Conclusion
Conclusion Brett Monia, Ph.D. Chief Executive Officer 25
Slide 26: 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 27: Q&A
Q&A 27
Slide 28: Donidalorsen Phase 3 Results
Donidalorsen Phase 3 Results OASIS-HAE and OASIS-Plus: OLE + Switch Webcast May 31st 28
Slide 29: Appendix
Appendix OOOO Ul IONIS)
Slide 30: 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 Additional WAINUA ATTRv-PN OUS submissions (2024) (eplontersen) ATTR-CM Ph3 data (2025)4 FCS OUS submissions (2024)5 Olezarsen sHTG Ph3 data (2025) Donida
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