CARLSBAD, Calif., and
CAMBRIDGE, Mass., Nov. 5, 2015 /PRNewswire/ -- Isis
Pharmaceuticals, Inc. (NASDAQ: ISIS) and its wholly owned
subsidiary, Akcea Therapeutics, today announced the start of a
Phase 3 study to evaluate the efficacy and safety of volanesorsen
in patients with familial partial lipodystrophy (FPL). Akcea
is currently conducting an international, multi-center Phase 3
study of volanesorsen in patients with Familial Chylomicronemia
Syndrome (FCS). Akcea is developing and plans to
commercialize volanesorsen for two ultra-rare diseases, FCS and
FPL.
Familial partial lipodystrophy is a rare, genetic disorder
characterized by metabolic abnormalities, including
hypertriglyceridemia and extreme insulin resistance, and
abnormalities in the distribution of body fat. Patients with
FPL have a high risk at an early age of diabetes, liver disease,
and cardiovascular disease. In addition, extreme
hypertriglyceridemia puts them at risk for potentially
life-threatening pancreatitis. Since it is most often
an inherited, autosomal dominant disorder, FPL can be passed down
from one generation to the next in affected families.
"Every day, people with FPL struggle to manage the burden this
disease brings across virtually every major organ system in their
body," describes Dr. Elif Oral of
the University of Michigan. "FPL takes an enormous toll on
those affected – both physically and psychologically. There
is still no pharmacologic therapy developed specifically for this
patient population. For these patients in particular, the
potential benefit of inhibiting the synthesis of apoC-III is very
promising."
According to Paula Soteropoulos,
CEO of Akcea Therapeutics: "The late-stage development of
volanesorsen for people with FPL perfectly reflects Akcea's vision,
which is to bring transformative therapies to patients and address
the root causes of serious, underserved cardiometabolic disorders.
Akcea's initiation of this second Phase 3 program reflects our
focus and expertise at developing and commercializing medicines for
complex disorders that are not addressable through other
therapeutic modalities."
Volanesorsen is a Gen. 2.0+ antisense drug designed to reduce
the production of apoC-III, a protein that acts as a key regulator
of triglyceride levels in the blood. Patients with elevated
triglyceride levels are at significant risk for coronary artery
disease and diabetes. Extremely high triglyceride levels put
patients at risk of pancreatitis, a serious and potentially
life-threating illness. In addition, elevated levels of
apoC-III are recognized as an independent contributor to
cardiovascular disease.
The Phase 3 study announced today is a randomized, double-blind,
placebo-controlled, multi-center, international study in
approximately 50 patients with FPL. The study's primary
objective is to evaluate the efficacy and safety of a 300 mg once
weekly dose of volanesorsen given over 12 months. The primary
endpoint of the study is percent change in fasting triglycerides
from baseline after three months of dosing.
ABOUT VOLANESORSEN, FCS and FPL
Volanesorsen (formerly ISIS-APOCIIIRx) is an
antisense drug in development intended to treat patients with
severely high triglycerides either as a single agent or in
combination with other triglyceride-lowering agents.
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.
Volanesorsen is currently being evaluated in a Phase 3 study in
patients with FCS. Patients with FCS are unable to
effectively clear lipid particles called chylomicrons, and as a
result, have extremely high levels of triglycerides, putting them
at risk of potentially life-threatening pancreatitis. FCS is a
rare, genetic disorder and may also be called familial
chylomicronemia or Fredrickson Type 1 hyperlipoproteinemia, or
familial lipoprotein lipase deficiency.
A second Phase 3 study of volanesorsen has begun in patients
with FPL, a rare lipid disorder characterized by abnormal fat
distribution across the body and a range of metabolic
abnormalities, including severe insulin resistance, dyslipidemia
and hypertriglyceridemia, hepatic steatosis and, in affected women,
features of hyperandrogenism. Patients with FPL are at increased
risk of pancreatitis, hepatic steatosis and NASH, enlarged livers,
polycystic ovarian syndrome and premature cardiovascular
disease. Patients with FPL are unable to store fat or
triglycerides in normal fat stores so excess triglycerides are
stored in the liver and muscle and accumulate at high levels in the
bloodstream. Additional information is available through
Lipodystrophy United at http://www.lipodystrophyunited.org/
.
For more information about this clinical trial program for
volanesorsen, please visit www.apociii.com.
ABOUT ISIS PHARMACEUTICALS, INC.
Isis 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, Isis has created a
large pipeline of first-in-class or best-in-class drugs, with over
a dozen drugs in mid- to late-stage development. Drugs
currently in Phase 3 development include volanesorsen, a drug Isis
is developing and plans to commercialize through its wholly owned
subsidiary, Akcea Therapeutics, to treat patients with familial
chylomicronemia syndrome and familial partial lipodystrophy;
ISIS-TTRRx, a drug Isis is developing with GSK to treat
patients with all forms of TTR amyloidosis; and
ISIS-SMNRx, a drug Isis is developing with Biogen to
treat infants and children with spinal muscular atrophy.
Isis' patents provide strong and extensive protection for its drugs
and technology. Additional information about Isis is
available at www.isispharm.com.
ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics is a
development and commercialization company focused on transforming
the lives of patients with serious cardiometabolic lipid disorders.
Established as a wholly-owned subsidiary of Isis Pharmaceuticals,
Inc., Akcea has a robust portfolio of development-stage drugs
covering multiple targets and disease states using advanced
RNA-targeted antisense therapeutics. Akcea's drug pipeline
includes novel antisense drugs designed to address a number of
lipid risk factors, including LDL-Cholesterol, apoC-III,
triglycerides and Lp(a). Akcea's most advanced program,
volanesorsen, is in Phase 3 development to treat patients with
ultra-orphan lipid disorders that are characterized by extremely
high triglycerides and apoC-III, including familial chylomicronemia
syndrome (FCS) and familial partial lipodystrophy (FPL).
Akcea is located in Cambridge,
Massachusetts. Additional information about Akcea is
available at www.akceatx.com.
ISIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENT
This
press release includes forward-looking statements regarding Isis'
business, the business of Akcea Therapeutics, the development,
activity, therapeutic and commercial potential and safety of
volanesorsen for the treatment of patients with FCS and FPL. Any
statement describing Isis' 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. Isis' 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 Isis' forward-looking statements reflect the good faith
judgment of its management, these statements are based only on
facts and factors currently known by Isis. As a result, you
are cautioned not to rely on these forward-looking
statements. These and other risks concerning Isis' programs
are described in additional detail in Isis' annual report on Form
10-K for the year ended December 31,
2014, 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.
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SOURCE Isis Pharmaceuticals, Inc.