Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis
Pharmaceuticals, Inc. (NASDAQ:IONS), focused on developing and
commercializing drugs to treat patients with serious
cardiometabolic diseases caused by lipid disorders, today announced
the initiation of an exploratory Phase 2 program of
AKCEA-ANGPTL3-LRx in patients with rare hyperlipidemias.
AKCEA-ANGPTL3-LRx is designed to reduce the production of
angiopoietin-like 3, or ANGPTL3. The absence of ANGPTL3 has been
shown to be cardioprotective and associated with reduced risk of
insulin resistance and diabetes mellitus.
The AKCEA-ANGPTL3-LRx Phase 2 program is designed to include
three clinical studies in patients with one of three rare
hyperlipidemias, including familial chylomicronemia syndrome (FCS),
familial partial lipodystrophy (FPL), and homozygous familial
hypercholesterolemia (HoFH). The objectives of these studies are to
determine the pharmacodynamics, pharmacokinetics, safety and
tolerability of AKCEA-ANGPTL3-LRx, and to further characterize the
drug’s potential in these patient populations.
“ANGPTL3 is a key regulator of a number of lipid and metabolic
pathways and as such is an important target for the therapeutic
management of rare hyperlipidemias, such as FCS, FPL and
HoFH. In FCS, severe elevation of triglycerides results in a
high risk of acute and chronic pancreatitis and other metabolic
complications. Patients with HoFH are subject to early
cardiovascular disease and premature death due to high LDL
cholesterol levels. Patients with FPL often have both high
triglycerides and high LDL cholesterol levels as well as other
complications related to insulin resistance and fatty liver, which
can lead to accelerated atherogenesis and liver failure,” said
Louis O’Dea, chief medical officer at Akcea Therapeutics. “We are
committed to developing additional therapeutic options for these
patient populations whose significant medical needs are currently
greatly underserved.”
Consistent with Akcea’s commitment to the FCS community, the
first study is designed to evaluate the pharmacodynamics,
pharmacokinetics, safety and tolerability of AKCEA-ANGPTL3-LRx in
patients with FCS. Study participants will receive
AKCEA-ANGPTL3-LRx by subcutaneous injection once weekly for 13
weeks. Akcea plans to report top-line data from this study in
2018. Akcea plans to initiate similar pilot studies shortly
in patients with FPL and patients with HoFH. For further
study information, please visit www.clinicaltrials.gov and search
for AKCEA-ANGPTL3-LRx.
ABOUT AKCEA-ANGPTL3-LRx
AKCEA-ANGPTL3-LRx is a ligand conjugated antisense (LICA) drug
designed to reduce angiopoietin-like 3 protein, or ANGPTL3. ANGPTL3
is a key regulator of triglycerides, cholesterol, glucose and
energy metabolism. People with lower levels of ANGPTL3 have lower
LDL-C and triglyceride levels and lower risk of heart attacks.
Ionis and Akcea are developing AKCEA-ANGPTL3-LRx to treat
multiple lipid disorders including rare hyperlipidemias and NAFLD
with metabolic complications.
In a Phase 1/2 clinical study in volunteers with elevated
triglycerides, published in The New England Journal of Medicine,
treatment with multiple doses of AKCEA-ANGPTL3-LRx resulted in
dose-dependent reductions in ANGPTL3 protein of up to 85% after six
weeks of treatment. Treatment with AKCEA-ANGPTL3-LRx also
resulted in substantial and dose-dependent reductions in
triglycerides, LDL cholesterol, VLDL cholesterol, non-HDL
cholesterol, apolipoprotein B and apolipoprotein C-III protein.
AKCEA-ANGPTL3-LRx was well tolerated in the study. No serious
adverse events, including platelet count reductions and injection
site reactions, were reported. Further, there were no
discontinuations during the treatment period. Phase 1 studies
of all three of Akcea’s LICA drugs have shown that doses up to 30
fold lower than non-LICA drugs result in consistent target
reductions and a favorable safety and tolerability profile.
ABOUT AKCEA THERAPEUTICSAkcea Therapeutics, an
affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical
company focused on developing and commercializing drugs to treat
patients with serious cardiometabolic diseases caused by lipid
disorders. Akcea is advancing a mature pipeline of four novel
drugs, including volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx
and AKCEA-APOCIII-LRx, all with the potential to treat multiple
diseases. All four drugs were discovered by and are being
co-developed with Ionis, a leader in antisense therapeutics, and
are based on Ionis’ proprietary antisense technology. The most
advanced drug in its pipeline, volanesorsen, is under regulatory
review in the U.S., EU and Canada for the treatment of familial
chylomicronemia syndrome, or FCS, and is currently in Phase 3
clinical development for the treatment of familial partial
lipodystrophy, or FPL. Akcea is building the infrastructure to
commercialize its drugs globally with a focus on lipid specialists
as the primary call point. Akcea is located in Cambridge,
Massachusetts. Additional information about Akcea is available at
www.akceatx.com.
ABOUT IONIS PHARMACEUTICALS, INC.Ionis is the
leading company in RNA-targeted drug discovery and development
focused on developing drugs for patients who have the highest unmet
medical needs, such as those patients with severe and rare
diseases. Using its proprietary antisense technology, Ionis has
created a large pipeline of first-in-class or best-in-class drugs,
with over three dozen drugs in development.
SPINRAZA® (nusinersen) has been approved in global
markets for the treatment of spinal muscular atrophy (SMA).
Biogen is responsible for commercializing SPINRAZA. Drugs that have
successfully completed Phase 3 studies include inotersen, an
antisense drug Ionis is developing to treat patients with
hereditary TTR amyloidosis (hATTR), and volanesorsen, an antisense
drug discovered by Ionis and co-developed by Ionis and Akcea
Therapeutics to treat patients with either familial
chylomicronemia syndrome or familial partial lipodystrophy. Akcea,
an affiliate of Ionis, is a biopharmaceutical company focused on
developing and commercializing drugs to treat patients with serious
cardiometabolic diseases caused by lipid disorders. If approved,
volanesorsen will be commercialized through Ionis' affiliate,
Akcea. Inotersen filings for marketing approval have been submitted
in the U.S. and EU. Volanesorsen filings for marketing
approval have been submitted in the U.S., EU, and Canada. Ionis'
patents provide strong and extensive protection for its drugs and
technology. Additional information about Ionis is available
at www.ionispharma.com.
AKCEA FORWARD-LOOKING STATEMENTThis press
release includes forward-looking statements regarding the business
of Akcea Therapeutics, Inc. and the therapeutic and commercial
potential of volanesorsen, AKCEA-ANGPTL3-LRx and other products in
development. Any statement describing Akcea’s 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, particularly those inherent in the process of
discovering, developing and commercializing drugs that are safe and
effective for use as human therapeutics, and in the endeavor of
building a business around such drugs. Akcea’s 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 Akcea’s forward-looking statements reflect the good faith
judgment of its management, these statements are based only on
facts and factors currently known by Akcea. As a result, you are
cautioned not to rely on these forward-looking statements. These
and other risks concerning Akcea’s programs are described in
additional detail in its final prospectus for its initial public
offering and its most recent quarterly report on Form 10-Q, which
is on file with the SEC.
IONIS FORWARD-LOOKING STATEMENT This press
release includes forward-looking statements regarding the business
of Akcea Therapeutics, Inc. and the therapeutic and commercial
potential of AKCEA-ANGPTL3-LRx 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,
particularly those inherent in the process of discovering,
developing and commercializing drugs that are safe and effective
for use as human therapeutics, and in the endeavor of building a
business around such drugs. 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, 2016, and its most recent quarterly report
on Form 10-Q, which are on file with the SEC. Copies of these
and other documents are available from the Company.
In this press release, unless the context requires otherwise,
“Ionis”, “Akcea,” “Company,” “Companies” “we,” “our,” and “us”
refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc.
Media and Investor Contact: Kathleen
Gallagher
Head of Communications and Investor Relations, Akcea
Therapeutics (617)-207-8509 kgallagher@akceatx.com
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