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
that all of its marketing applications for volanesorsen have been
accepted for review in the U.S., EU and Canada for the treatment of
patients with familial chylomicronemia syndrome (FCS).
If approved, volanesorsen would be the first therapy indicated
for people with FCS. The U.S. and EU regulatory agencies have
granted Orphan Drug Designation to volanesorsen for this
indication.
FCS is a severe, rare disorder characterized by extremely high
levels of triglycerides, symptoms such as extreme abdominal pain
that affect daily living, and the risk of recurrent, potentially
fatal, acute pancreatitis. FCS impacts people across the globe.
“We are committed to seeking global approvals for volanesorsen
at the outset. We are driven by the stories of people with
FCS worldwide who persevere daily with a debilitating condition
without an effective therapy. The acceptances of our regulatory
filings in the U.S., EU and Canada are important steps forward in
the global regulatory review process for volanesorsen, which brings
us closer to potentially providing the first approved therapy for
the treatment of people with FCS,” said Paula Soteropoulos, chief
executive officer of Akcea Therapeutics. "The entire Akcea team is
dedicated to working closely with each regulatory agency to support
the review of volanesorsen.”
In the U.S., the Food and Drug Administration (FDA) assigned a
Prescription Drug User Fee Act (PDUFA) goal date of August 30,
2018. The New Drug Submission (NDS) has passed screening by Health
Canada and was granted Priority Review. The EMA has validated the
volanesorsen Marketing Authorization Application (MAA).
Volanesorsen has been granted a Promising Innovative Medicine (PIM)
Designation by the United Kingdom’s Medicines and Healthcare
products Regulatory Agency (MHRA) for the treatment of people
with FCS.
ABOUT THE VOLANESORSEN CLINICAL PROGRAM IN
FCSThe applications for volanesorsen for the treatment of
people with FCS are based on data from the Phase 3 APPROACH and
COMPASS studies. The pivotal APPROACH study was the largest study
ever conducted in patients with FCS. APPROACH, a one-year,
randomized, placebo-controlled study in 66 patients with FCS
(average baseline triglycerides of 2,209 mg/dL, or 25.0 mmol/L),
achieved its primary endpoint of reduction in triglycerides at
three months, with a 77% mean reduction in triglycerides, which
translated into a 1,712 mg/dL (19.3 mmol/L) mean absolute
triglyceride reduction in volanesorsen-treated patients. The
treatment difference seen in the study was 77% compared to an 18%
increase for placebo.
In addition, in the APPROACH study, treatment with volanesorsen
was associated with a statistically significant reduced rate of
on-study pancreatitis attacks in the group of patients who had
multiple pancreatitis events during the 5 years prior to screening
and reduced abdominal pain in patients reporting pain during the
screening period.
COMPASS is a study which supports volanesorsen’s regulatory
filings in FCS. COMPASS, a six-month randomized placebo-controlled
study in 113 patients with very high triglycerides (>500 mg/dL),
also achieved its primary endpoint of reduction in triglycerides at
three months, with a 71% mean reduction in triglycerides. In the
COMPASS study, treatment with volanesorsen was associated with a
statistically significant reduction in on-study pancreatitis
attacks.
The most common adverse event in the studies was injection site
reactions, which were mostly mild. Platelet count reductions were
observed in many patients. These platelet declines were not
clinically significant in most patients and were generally well
managed with monitoring and dose adjustment. Five patients
discontinued participation in the APPROACH study due to platelet
count reductions, two of which were severe; four patients
discontinued due to other nonserious adverse events.
Akcea is currently enrolling patients with FCS in the APPROACH
Open Label Extension study. For more information, please
visit www.clinicaltrials.gov.
ABOUT VOLANESORSEN, FCS AND FPL Volanesorsen, a
product of Ionis’ proprietary antisense technology, is in
development for two rare metabolic disorders: FCS and FPL.
Volanesorsen is designed to reduce the production of ApoC-III, a
protein produced in the liver that plays a central role in the
regulation of plasma triglycerides and may also affect other
metabolic parameters. Volanesorsen is under regulatory review in
the U.S., EU and Canada. The U.S. and EU regulatory agencies have
granted Orphan Drug Designation to volanesorsen for the treatment
of patients with FCS.
FCS is a severe, rare disorder characterized by extremely high
levels of triglycerides, daily symptoms such as abdominal pain, and
the risk of recurrent, potentially fatal, acute pancreatitis.
People with FCS are unable to effectively metabolize large,
triglyceride-rich lipid particles called chylomicrons due to a
deficiency in lipoprotein lipase, an enzyme that helps to break
down triglycerides. There is no effective therapy available.
Additional information on FCS is available at www.fcsfocus.com. For
a full list of organizations supporting the FCS community
worldwide, please click here.
FPL is a severe, rare genetic metabolic disorder characterized
by an inability of the body to store fat in normal locations. This
results in high levels of triglycerides in the bloodstream,
abnormal fat distribution around and within organs, such as the
liver and heart, and a range of metabolic abnormalities, including
severe insulin resistance. People with FPL are at increased risk of
acute pancreatitis in addition to other long-term, progressive
manifestations, such as premature cardiomyopathy, atherosclerosis,
and liver disease. Additional information on FPL is available
through Lipodystrophy United at
http://www.lipodystrophyunited.org.
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 patients
with familial chylomicronemia syndrome, or FCS, and is currently in
Phase 3 clinical development for the treatment of patients with
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’S FORWARD-LOOKING STATEMENTThis press
release includes forward-looking statements regarding the business
of Akcea Therapeutics, Inc. and the therapeutic and commercial
potential of volanesorsen 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 STATEMENTThis press
release includes forward-looking statements regarding the business
of Akcea Therapeutics, Inc. and the therapeutic and commercial
potential of volanesorsen 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 GallagherExecutive Director, Corporate
Communications and Investor Relations617-207-8509
kgallagher@akceatx.com
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