– Numerous value-driving
commercial, regulatory and pipeline milestones anticipated
–
CARLSBAD, Calif., Jan. 8, 2024
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
today announced highlights from the Company's 2023 achievements and
previewed a number of important milestones expected in 2024. Ionis
will provide a business update at the 42nd Annual J.P. Morgan
Healthcare Conference on Wednesday, January
10 at 3pm PT; the presentation
is available today on the Ionis website.
![(PRNewsfoto/Ionis Pharmaceuticals, Inc.) (PRNewsfoto/Ionis Pharmaceuticals, Inc.)](https://mma.prnewswire.com/media/882077/Ionis_Logo.jpg)
In 2023, Ionis made significant progress in advancing a steady
cadence of medicines for people with serious diseases, including
the U.S. approval of WAINUA™ (eplontersen) for the treatment of
polyneuropathy of hereditary transthyretin-mediated amyloidosis
(ATTRv-PN) and the accelerated approval of QALSODY®
(tofersen) for SOD-1 amyotrophic lateral sclerosis (ALS). Ionis
also shared positive Phase 3 trial readouts for WAINUA and for
investigational olezarsen in familial chylomicronemia syndrome
(FCS), a rare, life-threatening disease characterized by severely
elevated triglycerides; olezarsen is also being evaluated in an
ongoing Phase 3 program in severe hypertriglyceridemia. The
late-stage pipeline expanded to nine medicines in Phase 3 trials
for 11 potential indications and Ionis reported a number of
additional positive data readouts. Ionis also continued to make key
advances in its industry-leading technology platform, which now
includes multiple RNA modalities and gene editing to support the
next wave of medicines.
"With the recent approval of our first co-commercialized
medicine, WAINUA for ATTRv-PN, and the potential for our first
independent commercial launch with olezarsen in FCS later this
year, Ionis is delivering on our vision to unlock better futures
for people with serious diseases by building a leading
fully-integrated, science-driven biopharmaceutical company," said
Brett P. Monia, Ph.D., Ionis' chief
executive officer. "We expect 2024 to be another robust year of
execution and achievement, beginning with topline Phase 3 results
expected in the first quarter for donidalorsen in hereditary
angioedema. Ionis also looks forward to many additional regulatory
milestones, important readouts across our mid- and late-stage
pipeline and continued technology advances in our leading RNA
therapeutics platform."
2024 Anticipated Highlights Include:
- Continued progress with WAINUA, Ionis' first
co-commercialized medicine in collaboration with AstraZeneca in the
U.S.:
- U.S. launch for ATTRv-PN in January
- European Medicines Agency (EMA) approval decision; additional
country approvals
- Additional regulatory filings for ATTRv-PN
- Continuing the fully enrolled, landmark CARDIO-TTRansform trial
in ATTR-cardiomyopathy, with data expected as early as 2025
- Advancing olezarsen, our first anticipated independent
launch:
- Detailed Phase 3 FCS results presentation
- U.S. Food and Drug Administration (FDA) regulatory filing in
FCS, potential approval decision (assuming priority review) and
U.S. launch
- EMA filing for FCS
- Completion of enrollment in severe hypertriglyceridemia Phase 3
studies, with data expected in 2025
- Pivotal results and potential regulatory filing for
investigational donidalorsen, our second anticipated independent
launch:
- Phase 3 OASIS-HAE in first quarter and OASIS-Plus switch
results by mid-year
- Potential U.S. regulatory filing in hereditary angioedema
- Advancing additional independent investigational
medicines:
- Ionis expects to have six independent neurology programs in
clinical development by year-end 2024, including new programs in
rare pediatric diseases and severe dementias, which currently have
no approved therapies
- Phase 2 results for ION224 in nonalcoholic steatohepatitis
anticipated
- Continued progress with key partnered programs,
including:
- QALSODY: EU SOD1-ALS CHMP and approval decision (Biogen)
- ION582: Phase 1/2 clinical results in Angelman syndrome
(Biogen)
- ION541: Phase 2 results in ALS (Biogen)
- IONIS-FB-LRx: Phase 2 results in IGA nephropathy and
geographic atrophy, a serious eye disease (Roche)
For more information about QALSODY, visit
https://www.qalsody.com/. This indication is approved under
accelerated approval based on reduction in plasma neurofilament
light chain (NfL) observed in patients treated with QALSODY.
Continued approval for this indication may be contingent upon
verification of clinical benefit in confirmatory trial(s).
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 link to U.S. Full Prescribing Information for
WAINUA.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted
therapy, pioneering new markets and changing standards of care.
Ionis currently has five marketed medicines and a promising
late-stage pipeline highlighted by cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision to
become the leader in genetic medicine, utilizing a multi-platform
approach to discover, develop and deliver life-transforming
therapies.
To learn more about Ionis visit www.ionispharma.com and
follow us on X (Twitter) @ionispharma and LinkedIn.
Ionis' Forward-looking Statements
This press release includes forward-looking statements regarding
Ionis' business, and the therapeutic and commercial potential of
Ionis' 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. 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, 2022, and most recent
Form 10-Q, which are on file with the SEC. Copies of these and
other documents are available at www.ionispharma.com.
Ionis Pharmaceuticals® is a registered trademark of
Ionis Pharmaceuticals, Inc. WAINUA™ is a trademark of AstraZeneca
plc. QALSODY® is a registered trademark of Biogen.
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SOURCE Ionis Pharmaceuticals, Inc.