IONIS PHARMACEUTICALS, INC. Q1:24 Business Update and Financial Results
May 7, 2024
Nasdaq: IONS
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 | |
Executive Vice President, Research | Chief Commercial Officer |
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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, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward- looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on our Form 10-K for the year ended December 31, 2023, and our most recent Form 10-Q quarterly filing, which are on file with the SEC. Copies of these and other documents are available at www.ionispharma.com.
In this presentation, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and
its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics® is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI® is a trademark of Akcea Therapeutics, Inc. WAYLIVRA® is a registered trademark of Akcea Therapeutics, Inc. QALSODYTM is a trademark of Biogen. SPINRAZA® is a registered trademark of Biogen. WAINUATM is a registered trademark of the AstraZeneca group of companies.
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Introduction
Brett Monia, Ph.D.
Chief Executive Officer
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2024 Off To Strong Start with Several Important Achievements
WAINUA Launched in U.S. for ATTRv-PN1
2 2 2
Positive | Phase 3 Studies | Additional Pipeline | |
Fully Enrolled3 | Achievements4 | ||
Phase 3 Readouts | 2 | ||
ION224 | |
Positive Phase 2 | ION356 Study |
Data (MASH) | |
Start (PMD) | |
1. WAINUA: www.wainua.com. 2. Balance (olezarsen for FCS), OASIS (donidalorsen for HAE). 3. CORE and Essence (olezarsen for sHTG). 4. Phase 2 readout of ION224 for MASH; Started Orbit study in 2024 (ION356 for PMD).
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WAINUA for ATTR-CM: Global Phase 3 Development Program Designed to Deliver Robust Results
Robust
Development
Program
Most comprehensive study to date in ATTR-CM, a fatal
disease
Positioned to deliver most robust data in broad patient
population
Largest study conducted in ATTR-CM now fully enrolled
with >1,400 patients
MRI and scintigraphy sub-studies underway to assess
the effects on cardiac structure and function
Data as Next early as 20251 Steps
1. Timing expectations based on current assumptions and subject to change.
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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, TGs, marked acute pancreatitis reductions, substantial reduction in hospitalizations and favorable safety and tolerability2
- Positive data presented at ACC, published in NEJM3
- NDA submitted; EU filing on track this year
- EAP in U.S. for FCS now open, OLE progressing well
- U.S. Breakthrough Therapy and Orphan Drug designations
- Prepared to launch in advance of anticipated approval4
- Phase 2b study in patients with TG ≥150 mg/dL (HTG) and TG ≥500 mg/dL (sHTG)
- Supportive exposure study
- Statistically significant reductions in apoC-III and TGs (including nearly all HTG patients achieving normal TGs)
- Meaningful reductions in apoB, non-HDL-C, markers of CV risk
- Favorable safety and tolerability
- Positive data presented in late-breaker presentation at ACC, published in NEJM5
- Timing expectations are based on current assumptions and are subject to change. 2. Due to statistical hierarchy, reductions in apoC-III and acute pancreatitis are considered exploratory. 3. Stroes E, et al.N Engl J Med. 2024.
- Assuming priority review and approval. 5.Bergmark, B, et al.N Engl J Med. 2024.
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Olezarsen is Delivering Robust Data Supporting its Potential as a Breakthrough Treatment for FCS and sHTG1
Severe Hypertriglyceridemia (sHTG)
- Pivotal study in patients w/ TG ≥500 mg/dL (sHTG)
- Registrational study
- >600 patients
- Enrollment complete
• Pivotal study in patients w/ | • Supportive Ph3 study in | ||
TG ≥500 mg/dL (sHTG) | patients w/ TG ≥200-500 | ||
• | Confirmatory registrational | mg/dL (HTG) | |
study | • | Supportive exposure study | |
• | 390 patients | • | >1,400 patients |
• | Full enrollment expected | • | Enrollment complete |
mid-year |
-----------------------Data expected in 2025 ------------------------
On Track for Data From All Three Studies by Mid-2025
1. Timing expectations are based on current assumptions and are subject to change.
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Donidalorsen is Delivering Robust Data Supporting its Potential to Advance Prophylactic HAE Treatment1,2
Hereditary Angioedema
Phase 2
- Positive Phase 2 data published in New England Journal of Medicine
- Positive Phase 2 1-year and 2- year OLE data, including positive QoL data reported
- Presenting Phase 2 3-year OLE data in H2:24
- Positive Phase 3 topline data, including achieving:
- Statistically significant reduction in HAE attack rates in patients treated every 4 weeks or 8 weeks
- EAP in U.S. now open
- Presenting data at EAACI, May 31st
-
Phase 3 OLE study in patients who have completed OASIS-
HAE - Expanded enrollment
- Switch cohort in patients previously treated with other prophylactic therapies
- Presenting data at EAACI, May 31st
Preparing to Submit NDA with US FDA; Otsuka Preparing to Submit MAA in EU3
1. Based on Phase 3 data, double blind Phase 2 study data published in NEJM in 2022 and Phase 2 OLE data. 2. Timing expectations based on current assumptions and subject to change. 3. Licensed European commercialization rights to Otsuka in 2023.
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Leading and Validated Neurology Franchise
3 | SPINRAZA | QALSODY | |
SMA (SMN2) | SOD1-ALS (SOD1) | ||
Approved | Zilganersen | ||
Alexander disease | |||
Medicines1 | 12 | (GFAP) | |
ION717 | |||
Prion disease | |||
(PRNP) | |||
Medicines | Ulefnersen | ||
in Clinical | |||
FUS-ALS | |||
Development | (FUS) | ||
6 | |||
ION541 | |||
ALS (ATXN2) | |||
Wholly Owned | |||
Medicines | ION356 | ||
in Clinical | Pelizaeus-Merzbacher | ||
Disease (PLP1) | |||
Development by | |||
ION582 | ION306 | ||
YE:20242 | Angelman syndrome | ||
(UBE3A-ATS) | SMA (SMN2) |
WAINUA
ATTRv-PN (TTR)
Tofersen
Presymptomatic SOD1-ALS
(SOD1)
IONIS-MAPTRx/BIIB080
Alzheimer's disease
(Tau)
ION859
Parkinson's disease
(LRRK2)
Tominersen
Huntington's disease
(HTT)
ION464
Parkinson's disease and
Multiple System Atrophy
(alpha-synuclein)
1. SPINRAZA: www.spinraza.com; QALSODY: www.qalsody.com; Biogen is responsible for commercializing SPINRAZA and QALSODY; WAINUA: www.wainua.com. 2. Timing based on current estimates and subject to change.
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Ionis Pharmaceuticals Inc. published this content on 07 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 07 May 2024 16:04:08 UTC.
