- With experienced commercial team in place, Ionis poised to
independently bring steady cadence of potentially transformational
RNA-targeted medicines to patients beginning in 2024
- Plan to add four new wholly owned neurology medicines to
clinical pipeline by end of 2024
- Innovative technology advances unlocking new opportunities
in neurology and cardiology as well as in new therapeutic
areas
- With multiple launches anticipated over the coming years,
Ionis positioned for next-level value creation driven by
significant revenue growth and positive cash flow
- Ionis to webcast Innovation Day today at 8:00 a.m. ET
CARLSBAD, Calif., Oct. 4, 2023
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) is
holding an investor and analyst event, Ionis Innovation Day:
Discovering, Developing and Delivering Transformational Medicines,
in New York City today.
Presentations from Ionis leaders and external physicians will
highlight Ionis' comprehensive integrated research, development,
and commercialization capabilities, along with key pipeline and
technology highlights.
"Our leadership in RNA-targeted medicine includes multiple
modalities empowering us to advance the right medicines for the
right targets," said Brett P. Monia,
Ph.D., Ionis' chief executive officer. "Our powerful and prolific
research and development engine has discovered groundbreaking
medicines for people with devastating diseases. We have matched
this engine with a talented commercial organization poised to
deliver a steady flow of wholly owned medicines to patients. With
last week's announcement of positive Phase 3 data for olezarsen in
people with familial chylomicronemia syndrome (FCS), we're moving
ahead with preparations for the first of multiple anticipated
independent commercial launches."
Event location, webcast and replay information
The event will take place at the Westin New York Grand
Central from 8:00 a.m. to 3:00 p.m. Eastern Time and
will also be webcast. Please click here to register
for the event. A webcast replay will be available within 48 hours
after the event
at https://ir.ionispharma.com/events-and-presentations/upcoming-events.
Highlights include:
Near-Term Commercial Medicines
- Ionis has made important progress in building our commercial
and medical affairs organizations led by individuals with deep,
relevant expertise across rare and broad diseases.
- With the eplontersen PDUFA date of Dec. 22, 2023 nearing for ATTR polyneuropathy
(PN), Ionis is ready to bring this important new medicine to
patients along with our partner AstraZeneca, if approved.
- Eplontersen has the potential to be the treatment of choice,
backed by strong efficacy and safety data, a self-administration
profile and a strong global alliance with AstraZeneca.
- Last week, positive Phase 3 NEURO-TTRansform data were
published in The Journal of the American Medical
Association, and regulatory filings are planned in the EU and
other countries this year and next.
- The Phase 3 CARDIO-TTRansform trial, the largest trial
ever conducted in ATTR cardiomyopathy patients, is fully enrolled
and expected to read out as early as 1H 2025.
- Olezarsen is poised to be Ionis' first wholly
owned independent commercial launch.
- Last week, Ionis announced positive Phase 3 olezarsen
Balance results in FCS, showing statistically significant
triglyceride lowering and a substantial reduction in acute
pancreatitis events in addition to a favorable safety and
tolerability profile. Based on these data, Ionis is planning to
file U.S. and EU regulatory applications in early 2024.
- Phase 3 studies continue for severe hypertriglyceridemia
(SHTG), a more prevalent disease affecting more than three million
people in the U.S.
- Henry Ginsburg, M.D., Professor of Medicine, Columbia University, will speak at the event to
share his perspectives on the acute need for new treatments to
treat people with FCS and SHTG.
- Donidalorsen was recently granted U.S. Orphan Drug
Designation.
- Ionis plans to report Phase 3 results with donidalorsen for
prophylactic treatment of hereditary angioedema (HAE) in the first
half of 2024. Ionis also plans to report topline data from a second
Phase 3 study evaluating switching from currently approved HAE
treatments in mid-2024.
- Two-year results from the Phase 2 open-label-extension trial
showed that donidalorsen treatment resulted in a 96% overall
sustained mean reduction from baseline in HAE attack rates.
These data will be presented at a medical congress later this
year.
- Raffi Tachdjian, M.D., MPH,
FAAAAI, FACAAI, Associate Clinical Professor of Medicine and
Pediatrics UCLA School of Medicine; Chief, Division of Allergy and
Immunology Providence St. John Medical Center will discuss the need
for new prophylactic HAE treatments as more than one-third of
patients on prophylactic treatment report more than two attacks per
month.
Next Wave of Wholly Owned Medicines
- Building on Ionis' proven track record in neurology with
two approved medicines, 12 in clinical trials and more than 10 in
lead optimization/preclinical development, Ionis is advancing a
wholly owned neurology pipeline.
- The lead independent program, zilganersen for the
treatment of Alexander disease, a
serious genetic leukodystrophy, is now in Phase 3 development.
- In addition, Ionis will announce today that four new
wholly owned medicines in rare pediatric neurological diseases and
dementias are planned to enter first-in-patient studies by the end
of 2024:
- ION356 (PLP1), for the treatment of children
with Pelizaeus-Merzbacher Disease, a severe
leukodystrophy, which recently received Rare Pediatric Disease
Designation and Orphan Drug Designation from the U.S. FDA;
- ION440 for the treatment of children with MeCP2 duplication
syndrome, which causes significant developmental delays,
impaired motor function and seizures;
- ION717, for the treatment of prion disease, which
causes fatal dementia, is planned to enter a first-in-patient study
in 2023;
- And, an undisclosed genetic dementia program, which
is currently in preclinical development.
Advancing Technologies to Reach More Patients in Need
- Ionis' leading medicinal chemistry platform is enabling
technological advances to enhance existing medicine profiles and
expand into new tissues and/or therapeutic areas.
- Ionis is applying new chemistries such as the Mesyl
Phosphoramidate (MsPA) backbone to improve the duration of
effect and therapeutic index of investigational medicines.
- The company is using multiple drug modalities,
including antisense (ASO) and small interfering RNA (siRNA),
to identify the best potential medicine for a particular target
and/or tissue.
- By leveraging targeted delivery approaches, including
Bicycle® technology via our collaboration with Bicycle
Therapeutics, Ionis is developing medicines that have antibody-like
selectivity with a low total molecular weight, which allows
delivery to skeletal and cardiac muscle. Progress is also
being made in delivering medicines across the blood-brain barrier
with systemic dosing.
Clear Path to Unlocking Next-Level Value
- Ionis has a solid financial foundation with a strong
balance sheet with ~$2B in cash, and
existing substantial and sustained revenue streams.
- Ionis is investing efficiently to bring our near-term
medicines to patients.
- Ionis anticipates that revenue growth from multiple
medicines as well as ongoing partner and royalty revenue
will drive future positive cash flow.
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 four 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 Twitter @ionispharma.
Ionis' Forward-looking Statements
This press release includes forward-looking statements regarding
our business, financial guidance and the therapeutic and commercial
potential of QALSODY™ (tofersen), SPINRAZA® (nusinersen), TEGSEDI®
(inotersen), WAYLIVRA® (volanesorsen), eplontersen, olezarsen,
donidalorsen, ulefnersen, zilganersen, pelacarsen, bepirovirsen,
IONIS-FB-LRx, Ionis' technologies, and Ionis' 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. 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 December 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.
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SOURCE Ionis Pharmaceuticals, Inc.