Ionis Pharmaceuticals, Inc. · NASDAQ:IONS
Ionis Pharmaceuticals — JP Morgan Healthcare Conference 2024
28 slides from Ionis Pharmaceuticals's conference presentation, January 2024
Slide 1: Unlocking Better Futures, Delivering Next-Level Value
Unlocking Better Futures, Delivering Next-Level Value Brett P. Monia, Ph.D., Chief Executive Officer JP Morgan Healthcare Conference | January 2024 | 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: Executing on a Clear Vision
Executing on a Clear Vision Next-Level Value for Patients and All Stakeholders Delivering a Steady Cadence of Potentially Transformational Medicines Prioritizing and Delivering Ionis Medicines Technology Expanding the Ionis Directly to Patients Leadership Wholly Owned Pipeline Financial Strength and
Slide 4: Key 2023 Achievements
Key 2023 Achievements 2 FDA Approvals1 Positive 2 Phase 3 Readouts2 3 Phase 3 Study Starts 4 Key Studies Fully Enrolled3 5 Bepirovirsen (HBV) Additional Positive Clinical Data IONIS-FB-LRx (IgAN) Readouts Zilganersen (Alexander disease) 1. WAINUA: www.wainua.com; QALSODY: www.qalsody.com; Biogen is
Slide 5: Well Positioned to Achieve Even Greater Success in 2024
Well Positioned to Achieve Even Greater Success in 20241 New Commercial 2 2 2 Phase 3 Readouts NDA Filings Launches2 FCS | HAE Including 1st Independent Launch 5 Additional Regulatory Filings Mid-Stage and Data Readouts Approvals2 1. Timing expectations based on current assumptions and subject to ch
Slide 6: WAINUA Approved for ATTRv-PN
WAINUA Approved for ATTRv-PN: Delivering the First Ionis Co-Commercialized Medicine to Patients This Month1,2 FDA Approved on December 21 For ATTR Polyneuropathy, a systemic, progressive and fatal neurological disease 1. WAINUA: www.wainua.com; co-developing and commercializing in the US with AstraZ
Slide 7: WAINUA: Potential to be the Preferred Treatment Option for Patients with ATTR
WAINUA: Potential to be the Preferred Treatment Option for Patients with ATTR1,2 Strong Clinical Profile3 Halted Targeted Sustained Disease Knockdown Benefit Progression Targeted TTR Demonstrated Significant knockdown at halting of improvements in the source with neuropathy measures of consistent an
Slide 8: WAINUA: Potential to be the Preferred Treatment Option for Patients with ATTR
WAINUA: Potential to be the Preferred Treatment Option for Patients with ATTR1,2 Strong Clinical Profile3 Significant Commercial Reach Halted Targeted Sustained Global Patient Administration Disease Knockdown Benefit Partnership Support Profile Progression Targeted TTR Demonstrated Significant Allia
Slide 9: Eplontersen for ATTR-CM: Global Phase 3 Development Program
Eplontersen for ATTR-CM: Global Phase 3 Development Program Designed to Deliver Robust Results Most comprehensive study to date in ATTR-CM, a fatal disease of the heart muscle Positioned to deliver most robust data in broad patient Robust population Development Program Largest study conducted in ATT
Slide 10: Realizing the Promise of our Innovative Medicines
Realizing the Promise of our Innovative Medicines1 First Ionis- Commercialized Medicine First Ionis Independent Next Wave of Positioned to Reach Launches1,4 Wholly Owned Medicines Patients2 Olezarsen Leading Neurology Pipeline FCS & SHTG ATTRv-Polyneuropathy Donidalorsen Proven track record of deliv
Slide 11: Positive Olezarsen Phase 3 Results in FCS Patients
Positive Olezarsen Phase 3 Results in FCS Patients1,2 Reduction in Serum APOCIII Levels Olezarsen treatment resulted in: —Robust and significant reduction in serum APOCIII levels at 6 and 12 months —Statistically significant reductions in triglycerides at 80mg dose —Substantial reductions in acute p
Slide 12: Olezarsen: Familial Chylomicronemia Syndrome
Familial Chylomicronemia Syndrome Regulatory filings planned and potential FDA approval in 2024 based on positive Phase 3 results3 Olezarsen: 1st independent launch3 A Potential New Severe Hypertriglyceridemia Standard-of-Care Treatment for Patients with Severely Elevated Significant opportunity wit
Slide 13: Donidalorsen: A Potential First-in-Class Silencer for Hereditary Angioedema
Phase 3 data planned for Q1:20241 Substantial unmet need remains — Potentially fatal breakthrough attacks — Desire for greater treatment simplicity Donidalorsen: and tolerability A Potential Donidalorsen anticipated profile2: First-in-Class — Significant, rapid and sustained reductions in HAE attack
Slide 14: Donidalorsen: Potential Preferred Treatment for HAE Prophylaxis
Donidalorsen: Potential Preferred Treatment for HAE Prophylaxis1 • Phase 3 OLE study underway in patients • Phase 3 OASIS-HAE global study in 91 who have completed OASIS-HAE patients • Switch study underway in patients • Monthly and bi-monthly dosing previously treated with other prophylactic • Full
Slide 15: Realizing the Promise of our Innovative Medicines
Realizing the Promise of our Innovative Medicines1 First Ionis- Commercialized Medicine First Ionis Independent Next Wave of Positioned to Reach Launches1,4 Wholly Owned Medicines Patients2 Olezarsen Leading Neurology Pipeline FCS & SHTG ATTRv-Polyneuropathy Donidalorsen Proven track record of deliv
Slide 16: Leading and Validated Neurology Franchise
Leading and Validated Neurology Franchise 3 SPINRAZA QALSODY WAINUA SMA (SMN2) SOD1-ALS (SOD1) ATTRv-PN (TTR) Approved Medicines1 1. SPINRAZA: www.spinraza.com; QALSODY: www.qalsody.com; Biogen is responsible for commercializing SPINRAZA and QALSODY; WAINUA: www.wainua.com 16
Slide 17: Leading and Validated Neurology Franchise
Leading and Validated Neurology Franchise 3 SPINRAZA QALSODY WAINUA SMA (SMN2) SOD1-ALS (SOD1) ATTRv-PN (TTR) Zilganersen Tofersen Approved Alexander disease (GFAP) Presymptomatic SOD1-ALS (SOD1) Medicines1 11 ION717 IONIS-MAPTRx/BIIB080 Prion disease Alzheimer’s disease (PRNP) (Tau) Medicines ION85
Slide 18: Our Next Wave: Wholly Owned Neurology Medicines in Clinical Development by YE:2024
Our Next Wave: 6 Wholly Owned Neurology Medicines in Clinical Development by YE:2024 with More to Follow1 Expand into Next Key Areas of Neurology Expand into Dementia Rare Pediatric Neurology is the Foundation 1. Timing based on current estimates, subject to change. 18
Slide 19: Our Next Wave: Wholly Owned Neurology Medicines in Clinical Development by YE:2024
Our Next Wave: 6 Wholly Owned Neurology Medicines in Clinical Development by YE:2024 with More to Follow1 Rare Pediatric Dementia Future Wave Neurology Zilganersen ION717 Neuromuscular Alexander Disease Prion Disease and Peripheral Pivotal study underway (PRNP) Neuropathies First in patient study un
Slide 20: 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 21: Technology Advancements Powering Future Medicines
Technology Advancements Powering Future Medicines Expanding Optimizing Delivery for Technology Platform New Therapeutic Opportunities MsPA Backbone Ionis siRNA Bicycle-siRNA Bicycle ASO Enables Demonstrates Target Reduction Target Reduction Less Frequent Dosing1,2 Competitive Profile2,3 in Muscle1 i
Slide 22: Key 2023 Achievements
Key 2023 Achievements Positive Clinical Data Events Enrollment Achievements Regulatory Actions Eplontersen: Phase 3, NEURO- Donidalorsen: Phase 3, OASIS-HAE WAINUA: FDA approval, ATTRv-PN1 TTRansform 35, 66 & 85-week data, full enrollment, HAE QALSODY: FDA approval SOD1-ALS ATTRv-PN Eplontersen: Pha
Slide 23: 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 data 3-year OLE, HAE decisions, ATTRv-PN WAINUA OASIS-PLUS OUS filings, ATTRv-PN ATTRv-PN3 OLE ION
Slide 24: 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 NDA filing (2024) Olezarsen SHTG Ph3 data (2025) Donidalorsen
Slide 25: Positioned to Deliver Steady Cadence of Wholly Owned and Partnered Medicines
Positioned to Deliver Steady Cadence of IONIS-FB-LRx Wholly Owned and Partnered Medicines1 IgA Nephropathy 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 C
Slide 26: 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
Slide 27: 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
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