Ionis Pharmaceuticals, Inc. · NASDAQ:IONS
Ionis Pharmaceuticals — JP Morgan Healthcare Conference 2025
17 slides from Ionis Pharmaceuticals's conference presentation, January 2025
Slide 1: The Next Chapter: Accelerating Value Through Innovation
The Next Chapter: Accelerating Value Through Innovation Brett P. Monia, Ph.D., Chief Executive Officer JP Morgan Healthcare Conference | January 2025 | 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. Any statement describing Ionis’ goals, expectati
Slide 3: Ionis: Pioneered the Field of Oligonucleotide Therapeutics
Ionis: Pioneered the Field of Oligonucleotide Therapeutics A Rich History Created Industry Leading Medicinal Discovering Chemistry and Manufacturing Capabilities and Developing Transformational RNA-Targeted Optimized and Validated Delivery to Liver Medicines and CNS for Human Therapeutics Optimized
Slide 4: 2020: Charted Transformation to Unlock Substantial Value
2020: Charted Transformation to Unlock Substantial Value 2020 Goals Prioritize and advance wholly owned pipeline for commercial success Build commercial capabilities Expand and diversify our technology Independently bring medicines to patients 4
Slide 5: Strong Execution Positions Ionis to Unlock Substantial Value
Strong Execution Positions Ionis to Unlock Substantial Value 2020 Goals Today: Delivered on Our Goals Prioritize and advance Advanced 10 wholly owned medicines with wholly owned pipeline for transformational potential into mid- and late-stage commercial success development Build commercial Innovativ
Slide 6: Innovative, Scalable Commercial Organization for Global Launches
Innovative, Scalable Commercial Organization for Global Launches Customer Facing Market Access Teams Marketing Market Research and Analytics Health Economics and Patient Services Outcomes Research Omnichannel Commercial Operations New Product Commercial Strategy 6
Slide 7: Ionis Today: Fully Integrated, Commercial-Stage Biotechnology Company
Ionis Today: Fully Integrated, Commercial-Stage Biotechnology Company More to Come … Rare and prevalent opportunities in First Independent focused disease Ionis Launch areas BB First FDA-approved treatment for adults 〉 Neurology First Ionis- with familial branded chylomicronemia 〉 Cardiology Medicin
Slide 8: Inflection 2025: Poised to Deliver a Steady Cadence of New Medicines
Inflection 2025: Poised to Deliver a Steady Cadence of New Medicines Numerous Near-Term Growth Drivers1,2 4 ION582 (Angelman syndrome)4 ION582 Additional medicines (Angelman syndrome) IONIS-FB-LRx Independent Key partner (IgAN) launches over launches over Bepirovirsen next 3 years next 3 years (HBV)
Slide 9: Bringing Benefit to Millions of Patients Through Ionis Innovation
Bringing Benefit to Millions of Patients Through Ionis Innovation 1 RARE AND PREVALENT OPPORTUNITIES IN DISEASES WITH HIGH NEED FCS sHTG HAE ATTRv-PN SMA Lp(a)-CVD ATTR-CM SOD1-ALS HBV Angelman syndrome IgAN FUS-ALS Alexander disease 9 1. Market data on file, assuming approval.
Slide 10: Steady Cadence of Expected Launches to Power Revenue Growth
Steady Cadence of Expected Launches to Power Revenue Growth1 IONIS-MAPTRx (AD) Partnered Bepirovirsen (HBV) Pelacarsen (Lp(a)-CVD) Bepirovirsen (HBV) Pelacarsen (Lp(a)-CVD) >$2B WAINUA (ATTR-CM) WAINUA (ATTR-CM) in Potential Annual Peak IONIS-FB-LRx (IgAN) IONIS-FB-LRx (IgAN) Royalties2 SPINRAZA (SM
Slide 11: Steady Cadence of Expected Launches to Power Revenue Growth
Steady Cadence of Expected Launches to Power Revenue Growth1 Sapablursen (Polycythemia Vera) ION269 (AD) ION440 (MECP2) ION356 (PMD) >$3B ION717 (Prion disease) in Potential ION582 (Angelman Syndrome) Annual Peak Zilganersen (AxD) Product Zilganersen (AxD) Revenue2 TRYNGOLZA (sHTG) TRYNGOLZA (sHTG)
Slide 12: Steady Cadence of Expected Launches to Power Revenue Growth
Steady Cadence of Expected Launches to Power Revenue Growth1 Sapablursen (Polycythemia Vera) ION269 (AD) >$5B ION440 (MECP2) in Potential ION356 (PMD) >$3B Annual Peak ION717 (Prion disease) in Potential Product Revenue ION582 (Angelman Syndrome) Annual Peak + Zilganersen (AxD) Product Revenue2 Roya
Slide 13: Clear Path to Positive Cash Flow Powered by Accelerating Revenue Growth
Clear Path to Positive Cash Flow Powered by Accelerating Revenue Growth1 Revenue Sources Product Revenue Royalties + Sales Milestones from Partners R&D Revenue from Partners $0 Today YE:2030 13 1. Based on current estimates, subject to change.
Slide 14: Innovative Scientific Platform for Future Medicines
Innovative Scientific Platform for Future Medicines Expanding Technology Expanding Optimizing Therapeutic Advances Technology Platform Delivery Opportunities Expected in 2025 Broad Range of • Achieve clinical POC Targeted Delivery Established for Ionis MsPA & siRNA Technologies (e.g., LICA) Franchis
Slide 15: 2025 Key Value-Driving Events
2025 Key Value-Driving Events1 Product Launches Regulatory Actions Phase 2 Phase 3 Donidalorsen Clinical Data Clinical EventsU.S. Events Phase 2 Donidalorsen (HAE) Clinical Data FDA approval Phase 2 Olezarsen Events (HAE) Clinical Data Phase 2 sHTG data TRYNGOLZA Events Clinical Data U.S. & EU Zilga
Slide 16: Accelerating Value Through Innovation
Accelerating Value Through Innovation Proven and prolific discovery and development engine Pipeline delivering at all stages Scalable innovative commercial organization Clear path to positive cash flow1 Improving the lives of millions of patients with transformational medicines2 1. Based on current
Slide 17: Delivering Accelerated Value
Delivering Accelerated Value
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