– Accelerating value creation with numerous
commercial, regulatory and pipeline milestones –
– Ionis outlines clear path to sustained
positive cash flow –
CARLSBAD, Calif., Jan. 13,
2025 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) today announced highlights from the Company's 2024
achievements and previewed important milestones expected in 2025.
Ionis will provide a business update at the 42nd Annual J.P.
Morgan Healthcare Conference on Wednesday,
January 15 at 10:30am PT; the
presentation is available today on the Ionis website.
"With last month's U.S. approval and launch of TRYNGOLZA™
(olezarsen) as the first-ever therapy for familial chylomicronemia
syndrome and the potential approval of donidalorsen in hereditary
angioedema later this year, Ionis has entered a new chapter as a
fully integrated commercial-stage biotechnology company that
independently brings our innovative medicines to patients," said
Brett P. Monia, Ph.D., chief
executive officer, Ionis. "TRYNGOLZA is the first of four
independent Ionis product launches anticipated over the next three
years. We also expect four key launches from important partnered
programs within this timeframe. Collectively, these medicines stand
to help millions of patients with serious diseases and create
substantial value for all our stakeholders. To that end, we expect
increasing product and royalty revenue to enable Ionis to achieve
our goal of being cash flow positive."
2025 Anticipated Highlights Include:
- Launch TRYNGOLZA, Ionis' first independent product and the
first-ever therapy for U.S. patients with familial chylomicronemia
syndrome (FCS):
- U.S. Food and Drug Administration (FDA) Approval for FCS on
December 19, 2024
- Potential European Medicines Agency (EMA) approval for FCS
- Phase 3 results and U.S. regulatory submission for olezarsen
in severe hypertriglyceridemia, a large patient population with
high unmet need
- Phase 3 topline data for CORE, CORE 2 and ESSENCE Phase 3
trials expected in second half of 2025
- Potential approval of donidalorsen, a first-in-class
RNA-targeted medicine for hereditary angioedema and Ionis' second
anticipated independent launch:
- U.S. FDA action date of August 21,
2025
- U.S. commercial field team scale-up and launch
- Continue progress with WAINUA™ (eplontersen), Ionis'
first U.S. co-commercialized medicine in collaboration with
AstraZeneca:
- Continued positive performance in U.S. launch for
polyneuropathy of hereditary transthyretin-mediated amyloidosis
(ATTRv-PN)
- OUS approvals for ATTRv-PN
- Continuing the fully enrolled, landmark CARDIO-TTRansform trial
in ATTR cardiomyopathy, with data expected in the second half of
2026
- Advance the next wave of wholly owned medicines for serious
neurological diseases, including seven clinical-stage
therapies:
- Phase 3 trial start in first half of 2025 for ION582 for
Angelman syndrome
- Phase 3 data for zilganersen in Alexander disease, a rare
leukodystrophy with no approved disease-modifying therapies
- Phase 2 data for ION464 (SNCA) in multiple system atrophy
- Continue momentum with key partnered programs,
including:
-
- Phase 3 data for pelacarsen, a potentially groundbreaking
medicine for Lp(a)-driven cardiovascular disease (Novartis)
- Continue advancing next-generation technologies for
RNA-targeted medicines
- Achieve clinical proof of concept for Mesyl Phosphoramidate
(MsPA) backbone and siRNA platforms
- Advance first Bicycle-siRNA into clinical development
- Select first clinical candidate engineered to cross the blood
brain barrier
INDICATION for TRYNGOLZA™ (olezarsen)
TRYNGOLZA™
(olezarsen) was approved by the U.S. Food and Drug
Administration as an adjunct to diet to reduce triglycerides
in adults with familial chylomicronemia syndrome (FCS).
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
TRYNGOLZA is contraindicated in patients with a history of serious
hypersensitivity to TRYNGOLZA or any of the excipients in
TRYNGOLZA. Hypersensitivity reactions requiring medical treatment
have occurred.
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions (including symptoms of bronchospasm,
diffuse erythema, facial swelling, urticaria, chills and myalgias)
have been reported in patients treated with TRYNGOLZA. Advise
patients on the signs and symptoms of hypersensitivity reactions
and instruct patients to promptly seek medical attention and
discontinue use of TRYNGOLZA if hypersensitivity reactions
occur.
ADVERSE REACTIONS
The most common adverse reactions (incidence >5% of
TRYNGOLZA-treated patients and >3% higher frequency than
placebo) were injection site reactions, decreased platelet count
and arthralgia.
Please see full Prescribing Information for
TRYNGOLZA.
INDICATION for WAINUA™ (eplontersen)
WAINUA injection,
for subcutaneous use, 45 mg is indicated for the treatment of the
polyneuropathy of hereditary transthyretin-mediated amyloidosis in
adults.
IMPORTANT SAFETY INFORMATION for
WAINUA™ (eplontersen)
WARNINGS AND PRECAUTIONS
Reduced Serum Vitamin A Levels and Recommended
Supplementation WAINUA leads to a decrease in serum vitamin A
levels. Supplement with recommended daily allowance of vitamin A.
Refer patient to an ophthalmologist if ocular symptoms suggestive
of vitamin A deficiency occur.
ADVERSE REACTIONS
Most common adverse reactions (≥9% in WAINUA-treated patients) were
vitamin A decreased (15%) and vomiting (9%).
Please see U.S. Full Prescribing Information for
WAINUA.
About Ionis Pharmaceuticals, Inc.
For three decades,
Ionis has invented medicines that bring better futures to people
with serious diseases. Ionis currently has six marketed medicines
and a leading pipeline in neurology, cardiology, and other areas of
high patient need. As the pioneer in RNA-targeted medicines, Ionis
continues to drive innovation in RNA therapies in addition to
advancing new approaches in gene editing. A deep understanding of
disease biology and industry-leading technology propels our work,
coupled with a passion and urgency to deliver life-changing
advances for patients. To learn more about Ionis,
visit Ionis.com and follow us
on X (Twitter), LinkedIn and Instagram.
Ionis Forward-Looking Statements
This press release
includes forward-looking statements regarding Ionis' business and
the therapeutic and commercial potential of TRYNGOLZA, Ionis'
technologies and other products in development. 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 Form 10-K for the year
ended Dec. 31, 2023, and most recent Form 10-Q, which are on
file with the SEC. Copies of these and other documents are
available at www.Ionis.com.
Ionis Pharmaceuticals® and TRYNGOLZA™ are trademarks
of Ionis Pharmaceuticals, Inc. WAINUA™ is a registered
trademark of the AstraZeneca plc.
Ionis Investor Contact:
D. Wade Walke, Ph.D.
IR@ionis.com
760-603-2331
Ionis Media Contact:
Hayley
Soffer
media@ionis.com
760-603-4679
View original content to download
multimedia:https://www.prnewswire.com/news-releases/ionis-enters-new-chapter-in-2025-as-commercial-stage-biotech-with-multiple-independent-product-launches-anticipated-and-continued-late-stage-pipeline-momentum-302348723.html
SOURCE Ionis Pharmaceuticals, Inc.