CARLSBAD, Calif., Dec. 8, 2020 /PRNewswire/ -- At its Investor Day
yesterday, Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) highlighted
the company's significant achievements in 2020 and outlined its
strategy to realize the substantial opportunity of its
pipeline.
Ionis has been preparing and prioritizing its growing wholly
owned pipeline for commercialization in line with its commercial
strategy. The company's commercial priorities are three-fold: (1)
Initially focusing its commercial efforts on rare diseases within
its prolific neurology and cardiology franchises (2) pioneer new
markets where there are no available treatments (3) create new
standards of care where there has been a lack of innovation to
optimize patient care.
Delivering on these three priorities will have meaningful impact
to patients, their families, and healthcare providers all while
reducing the burden on healthcare systems and driving value for all
Ionis' stakeholders including patients and shareholders.
Ionis also has plans to expand opportunistically to new products
in additional treatment areas such as hematology, endocrinology and
pulmonology.
Ionis projects having the opportunity to launch six or more new
products through 2026, with each being ready for launch in a close
window, ranging from 18 to 24 months between each new product
launch. The expectation is that the implementation of this strategy
will drive double-digit revenue growth and substantial earnings
growth.
Brett P. Monia, Ph.D., chief
executive officer at Ionis, said, "In 2020, we pursued an
aggressive agenda focused on building our commercial plans and
capabilities, progressing the Ionis-owned pipeline, advancing our
technology and growing our leadership position in RNA-targeted
therapeutics. We are pleased to say that we delivered against all
these objectives. We invested in building our commercial plans and
capabilities and began implementation. These actions were
accelerated through the acquisition of our commercial affiliate
Akcea. We have also progressed and substantially expanded the
Ionis-owned pipeline. In addition, we have six Phase 3 trials
underway, initiated 13 Phase 2 trials, achieved multiple, positive
clinical proof-of-concept readouts, and advanced new delivery
platforms."
Dr. Monia continued, "For years we have been recognized for our
excellence in research, early drug development and scientific
innovation. We will now add to this by building a strong and
efficient commercial organization of equal excellence. All of which
will provide substantial benefit to patients and shareholders for
years to come."
Highlights of Investor Day
- Ionis estimates the total market opportunity for the
indications targeted by its pipeline is well in excess of
$15 billion, with a significant
portion from its wholly owned medicines.
- Ionis' cardiovascular franchise includes many potential
first-in-class and/or best-in-class medicines targeting a full
spectrum of cardiovascular disease risk factors. The company
is positioned to potentially launch multiple Ionis-owned
cardiovascular medicines through 2026, including:
-
- AKCEA-APOCIII-LRx: One product with the
potential for addressing multiple indications targeting elevated
triglycerides and the opportunity to set a new standard of care for
triglyceride management
-
- 91% of patients achieved normal serum triglycerides levels with
favorable safety and tolerability in Phase 2
- As announced on Dec. 1,
2020, a Phase 3 study in patients with familial
chylomicronemia syndrome (FCS) is now underway
- Evaluating additional indications with plans to initiate an
additional Phase 3 study in 2021
- AKCEA-TTR-LRx: Opportunity to significantly
expand ATTR franchise
-
- Robust target reductions of more than 90% and favorable safety
and tolerability demonstrated in Phase 1
- Flexibility of at-home monthly self-administration
- Two Phase 3 studies underway – CARDIO-TTRransform for patients
with hereditary or wild type TTR cardiomyopathy and
NEURO-TTRansform for patients with hereditary TTR
polyneuropathy.
- IONIS-AGT-LRx: Large unmet need in
patients with treatment-resistant hypertension (RHTN)
-
- Two Phase 2 clinical studies: Patients with mild HTN and
patients with uncontrolled HTN who are on two (65%) or three (35%)
antihypertensive medications
- Positive Phase 2 study in patients in uncontrolled HTN:
patients achieved mean reductions of 12 mmHg and 6 mmHg in systolic
and diastolic blood pressure from their own baseline, respectively,
after eight weeks of once-weekly 80 mg
IONIS-AGT-LRx
- IONIS-AGT-LRx has demonstrated a favorable safety
and tolerability profile in clinical trials to date
- More detailed results to be presented at an upcoming medical
conference
- Ionis' neurology franchise has the potential to establish
the standard of care for millions of patients and generate
substantial value as it advances its first-in-class medicines to
the market. The neurological disease market is a nascent market
poised for substantial growth. Ionis believes it can be the
catalyst for this growth as it is positioned to launch multiple
Ionis-owned medicines through 2026, including:
-
- ION363: First medicine in development to specifically
target FUS-ALS, a rare, rapidly progressing form of ALS
-
- Pivotal study on track for initiation in 2021
- Potential for a rapid path to the market
- ION716: Potential to be first approved treatment for
prion diseases
-
- Designed to reduce production of prion protein, root cause of
prion disease
- Pursuing pre-symptomatic (genetic carriers) and symptomatic
(genetic and sporadic) indications
- Pivotal study planned for 2021, design should provide a rapid
path to market
Akcea Integration Update
The recently completed acquisition of Akcea has created a
stronger, more efficient company, further bolstering Ionis'
financial strength. The integration of Akcea is ahead of schedule,
delivering cost synergies and efficiencies. It was also announced
that Akcea is going to commercialize TEGSEDI and WAYLIVRA in
Europe through a distribution
agreement with Swedish Orphan Biovitrum AB ("Sobi"), an
international biopharmaceutical company that focuses on rare
diseases. Under the terms of this agreement, Akcea retains the
marketing authorization ("MAH") for both medicines in Europe. Additionally, Akcea will continue to
maintain limited European operations including regulatory,
manufacturing, and the management of relationships with key opinion
leaders. Akcea will continue to lead the TEGSEDI and WAYLIVRA
global commercial strategy. The agreement provides Ionis the
flexibility to reinvest resources to support its other commercial
plans.
Oral Delivery Development Update
Ionis and AstraZeneca are committed to bringing the best
possible PCSK9 antisense treatments to patients and have been
collaborating on both the subcutaneous and oral formulations. The
subcutaneous formulation of ION449 has a potential best-in-class
profile and is advancing rapidly toward Phase 3 development.
Preclinical and early clinical data give Ionis and AstraZeneca
confidence that they can achieve effective oral delivery of ION449
and other ASOs. Based on ongoing research and experience to
date, both companies believe they can improve upon the current oral
formulation. Therefore, Ionis and AstraZeneca have decided to
terminate the Phase 1 PCSK9 oral study. Ionis and AstraZeneca will
continue to broadly work together to further optimize the oral
delivery of ASOs, including ION449.
Ionis is expanding its oral delivery to include medicines from
its pipeline and has increased its internal investment in oral
delivery research. The company plans on initiating one or more
programs from its pipeline within the next year or two. Candidates
for consideration include IONIS-TTR-LRx,
IONIS-PKK-LRx, ION994 (AGT), and ION547. Success would
further enhance the commercial value of Ionis-owned programs.
Webcast
Additional details are available in the replay of the webcast.
It is available here for a limited time.
About Ionis Pharmaceuticals, Inc.
As the leader in RNA-targeted drug discovery and development,
Ionis has created an efficient, broadly applicable, drug discovery
platform called antisense technology that can treat diseases where
no other therapeutic approaches have proven effective. Our drug
discovery platform has served as a springboard for actionable
promise and realized hope for patients with unmet needs. We created
the first and only approved treatment for children and adults with
spinal muscular atrophy as well as the world's first RNA-targeted
therapeutic approved for the treatment of polyneuropathy in adults
with hereditary transthyretin amyloidosis. Our sights are set on
all the patients we have yet to reach with a pipeline of more than
40 novel medicines designed to potentially treat a broad range of
diseases, including neurological, cardio-renal, metabolic,
infectious, and pulmonary diseases.
To learn more about Ionis visit www.ionispharma.com or follow us
on twitter @ionispharma.
Forward-looking Statements
This presentation includes forward-looking statements regarding
our business, financial guidance and the therapeutic and commercial
potential of SPINRAZA® (nusinersen), TEGSEDI® (inotersen),
WAYLIVRA® (volanesorsen) and Ionis' technologies and products in
development, including the business of Akcea Therapeutics, Inc.,
Ionis' wholly owned subsidiary. 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 those related to the impact COVID-19 could
have on our business, and 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, 2019 and our most
recent Form 10-Q quarterly filing, which are on file with the SEC.
Copies of this 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 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. SPINRAZA® is a registered trademark of Biogen.
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SOURCE Ionis Pharmaceuticals, Inc.