CAMBRIDGE, Mass. and
CARLSBAD, Calif., July 27, 2017 /PRNewswire/ -- Akcea Therapeutics,
Inc. (NASDAQ: AKCA) and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS)
announced today that a marketing authorization application (MAA)
has been submitted to the European Medicines Agency (EMA) for
volanesorsen for the treatment of patients with familial
chylomicronemia syndrome (FCS). Volanesorsen, a product of Ionis'
proprietary antisense technology, was discovered by Ionis and
co-developed by Ionis and Akcea and, if approved, will be
commercialized by Akcea.
"The submission of the volanesorsen MAA to the EMA represents a
critical milestone for Akcea. This filing brings us one step closer
to our goal of providing physicians and patients with a treatment
for a disease with multiple, severe, daily and chronic
manifestations," said Paula
Soteropoulos, president and chief executive officer of
Akcea. "We have had productive communications with regulatory
agencies in the EU, the U.S., and Canada, and are on track to also complete
regulatory submissions in the U.S. and Canada in September. Further, Akcea is
building the organization and infrastructure to commercialize
volanesorsen globally."
FCS is a severe, rare disorder characterized by extremely high
levels of triglycerides and the risk of recurrent, potentially
fatal 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 need new therapies that can meaningfully
lower their severely elevated triglycerides. Because of their very
high triglycerides, these patients suffer from daily and chronic
symptoms and are at significantly increased risk for morbidity
and mortality, primarily due to recurrent attacks of
pancreatitis," said Dr. Marcello
Arca, Head of the Lipid and Atherosclerosis Unit of the
University Hospital Policlinico Umberto I. "Based on the positive
results from the Phase 3 program with volanesorsen, I'm encouraged
that for the first time, these patients may have a new therapy that
can help them achieve the triglyceride reductions they need to
lessen the burden of their disease and decrease their risk for
pancreatitis."
"Akcea has shown great commitment in supporting patients and
working to find a treatment for this debilitating condition. Their
work means that the European FCS community of patients, families,
and physicians are one step closer to having an effective treatment
that will make the life-long struggle of living with the burden of
FCS much more manageable. It is great to be working with a
supportive partner like Akcea, and we also appreciate the
perseverance of our community in the quest to find an effective
treatment," said Jill Prawer,
Founder and Chair, LPLD Foundation.
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. This compared to an 18% increase for placebo, or a -94%
treatment effect. Further, 50% of treated patients achieved
triglyceride levels below 500 mg/dL (5.6 mmol/L), a recognized
threshold for enhanced pancreatitis risk. 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 a higher incidence of
pre-study pancreatitis and reduced abdominal pain in patients
reporting pain as a prominent pre-treatment symptom.
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 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 familial
partial lipodystrophy (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 and the risk of recurrent, potentially
fatal 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 THERAPEUTICS
Akcea 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 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 EU 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
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 and Canada 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 with
GSK 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. Both inotersen and volanesorsen are 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 STATEMENT
This 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, which is on
file with the SEC.
IONIS FORWARD-LOOKING STATEMENT
This press release
includes forward-looking statements regarding the business of Ionis
Pharmaceuticals, 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," "we," "our," and "us" refers to Ionis
Pharmaceuticals or Akcea Therapeutics.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc. SPINRAZA® is a registered trademark of Biogen.
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SOURCE Akcea Therapeutics, Inc.; Ionis Pharmaceuticals, Inc.