Health Canada grants priority review for
volanesorsen NDS filing
Akcea Therapeutics Canada, the Canadian subsidiary of Akcea
Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc.,
focused on developing and commercializing drugs to treat patients
with serious cardiometabolic diseases caused by lipid disorders,
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 applaud Health Canada for granting priority review for the
NDS filing for volanesorsen. This decision underscores the
significant medical need that exists for Canadians suffering with
FCS, a disease characterized by severe chronic and daily symptoms,
including the risk of potentially fatal episodes of acute
pancreatitis. The evidence of the potential for volanesorsen to
benefit individuals with FCS began with a Phase 2 study in Canada,
and the overall clinical program has included six trial sites in
Canada. In fact, many of the patients enrolled in the landmark,
multi-national, Phase 3 APPROACH study in FCS were from Canada,”
said Jared Rhines, general manager, Akcea Therapeutics, Canada. “We
are on track for a potential launch of volanesorsen in 2018, so
that, if approved, we can get this important medicine to patients
as quickly as possible.”
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.
FORWARD-LOOKING STATEMENTThis press release
includes forward-looking statements regarding the business of Akcea
Therapeutics, Inc. and its affiliates 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.
In this press release, unless the context requires otherwise,
“Akcea,” “Company,” “we,” “our,” and “us” refers to Akcea
Therapeutics.
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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