Health Canada grants priority review for
volanesorsen NDS filing
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, and Ionis,
today announced the filing of a New Drug Submission (NDS) to Health
Canada for volanesorsen, an investigational medicine for the
treatment of familial chylomicronemia syndrome (FCS). Health Canada
has also granted priority review for the volanesorsen NDS. Priority
review provides for the "fast-tracking" of eligible regulatory
filings in Canada intended for the treatment, prevention or
diagnosis of serious, life-threatening or severely debilitating
diseases or conditions.
“We are pleased to have completed our third global regulatory
filing for volanesorsen. This is a tremendous achievement for Akcea
and Ionis, and I would like to express my thanks to all those who
helped us get to this place. We are also pleased that Health Canada
has granted priority review for the volanesorsen NDS for the
treatment of FCS in Canada, which could accelerate access to this
important new medicine for Canadians suffering with FCS,” said
Paula Soteropolous, president and chief executive officer of Akcea.
“With regulatory filings for marketing authorization for
volanesorsen submitted in the U.S., EU and now Canada, we are on
track for a potential global commercial launch of volanesorsen in
2018.”
FCS is a severe, rare disorder characterized by extremely high
levels of triglycerides, symptoms such as abdominal pain that
affect daily living, 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.
“Patients with FCS have triglyceride levels that can reach 20 to
30 times those of healthy individuals. This predisposes them
to episodes of acute pancreatitis, which is potentially fatal,”
said Dr. Robert Hegele, distinguished professor of medicine and
biochemistry, Western University and the director of Lipid Genetics
Clinic and staff endocrinologist at the London Health Sciences
Centre. “Today, there is no effective therapy for FCS patients, so
I’m encouraged that we are now very close to having, for the first
time, a therapeutic option for FCS patients that can substantially
reduce triglycerides to levels that markedly reduce the risk for
pancreatitis and could relieve some of the other symptoms that FCS
patients live with on a daily basis.”
“We are encouraged that Health Canada has granted priority
status to volanesorsen, thereby recognizing the serious, severely
debilitating nature of FCS and the potential role this new therapy
could play,” says Durhane Wong-Rieger, president of the Canadian
Organization of Rare Disorders and chair of the Canadian Heart
Patient Alliance. “I have met several individuals with FCS, and
have heard many of their stories of intense pain, their utter lack
of control in preventing an attack and their fear of the inevitable
consequences of their disease.”
ABOUT THE VOLANESORSEN CLINICAL PROGRAM The
submission of volanesorsen for the treatment of FCS is based on
data from the Phase 3 APPROACH and COMPASS studies. The pivotal
APPROACH study, 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 is 94% 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.
The COMPASS study, 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 and Ionis continue to conduct the BROADEN study, a Phase 3
clinical trial in patients with familial partial lipodystrophy
(FPL), which continues to enroll, with topline data expected in
2019. Akcea plans to file for marketing authorization for
volanesorsen to treat FPL in 2019 if the data from the BROADEN
study are positive.
The U.S. and EU regulatory agencies have granted Orphan Drug
Designation to volanesorsen for the treatment of patients with FCS.
Volanesorsen has also received Orphan Drug Designation in the EU
for the treatment of FPL.
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.
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 and
through the FCS Foundation at http://www.livingwithfcs.org and the
LPLD Alliance at www.lpldalliance.org.
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 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
with the potential to treat multiple diseases, including
volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and
AKCEA-APOCIII-LRx. All four drugs were discovered and are being
co-developed by Ionis, a leader in antisense therapeutics, 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 the U.S., Europe, Japan, Canada and Brazil for
the treatment of spinal muscular atrophy (SMA). Biogen is
responsible for commercializing SPINRAZA. Drugs that have
successfully completed Phase 3 studies include inotersen
(IONIS-TTRRx), an antisense drug Ionis is developing to treat
patients with TTR amyloidosis, 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. Volanesorsen filings for marketing approval have been filed
in the U.S., EU and Canada. Inotersenis progressing toward
regulatory filings for marketing authorization. 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 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,
“Akcea,” “Ionis,” “Company,” “we,” “our,” and “us” refers to Akcea
Therapeutics or Ionis Pharmaceuticals.
Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc. Ionis Pharmaceuticals™ is a trademark of Ionis
Pharmaceuticals, Inc.
Media and Investor Contact:D. Wade Walke,
Ph.D.Vice President, Corporate Communications and Investor
Relations760-603-2741
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