CARLSBAD, Calif., Aug. 3, 2015 /PRNewswire/ -- Isis Pharmaceuticals
Inc. (NASDAQ: ISIS), today announced that the FOCUS FH phase 3
study of KYNAMRO® (mipomersen sodium) in patients with
severe heterozygous familial hypercholesterolemia (severe HeFH) met
its primary efficacy endpoint, a statistically significant
reduction in LDL-cholesterol after 60 weeks of treatment of once
weekly injections of 200 mg of KYNAMRO compared to placebo.
LDL-cholesterol reduction was similar to that observed in previous
phase 3 studies. In addition, based on the data available for
review, the safety profile of KYNAMRO observed in the FOCUS FH
trial was similar to the safety profile reported in previous phase
3 studies. Genzyme will provide a more in depth review of the
safety and efficacy data at a future medical meeting.
"We are pleased with the outcome of this fifth KYNAMRO
randomized placebo controlled phase 3 trial, which shows
statistically significant reductions in LDL-cholesterol in severe
HeFH," said Richard Geary, Ph.D.,
senior vice president of development at Isis Pharmaceuticals. "It
is encouraging to see sustained LDL-C reductions over 60 weeks that
were similar to what we've observed in previous phase 3 studies and
a safety profile consistent with previous phase 3 and long-term
extension studies. We look forward to the full analysis and
presentation of this investigational data at an upcoming scientific
meeting."
"These investigational data provide additional information about
KYNAMRO in the treatment of patients with severely elevated
LDL-cholesterol and we look forward to further analysis of these
data," said Genzyme's President and CEO, David Meeker, M.D.
ABOUT FOCUS FH
FOCUS FH was a multicenter, randomized,
placebo-controlled, double-blind, parallel-group study that
enrolled 310 patients aged 18 and older, followed by an open-label
continuation. Cohort 1 included patients with severe HeFH with
LDL-C ≥ 200 mg/dL plus coronary heart disease or LDL-C ≥ 300 mg/dL.
Cohort 2 included patients with HeFH with LDL-C ≥ 160 mg/dL and
<200 mg/dL plus coronary heart disease. Within each cohort,
patients were randomized 2:1 to either 200 mg once weekly, 70 mg
thrice weekly, or placebo for a 60 week study duration. Upon
completion of the 60 week blinded treatment, patients had the
option to enter the open label continuation period for 26 weeks and
receive the full dose regimen of KYNAMRO according to the dosing
schedule they were randomized to during the blinded treatment
phase. The trial was conducted at 131 sites worldwide. The primary
efficacy endpoint evaluated was the LDL-C percent change from
baseline to week 61 for cohort 1 and each dose regimen.
ABOUT KYNAMRO® (mipomersen sodium)
Injection 200 mg/mL
KYNAMRO is an oligonucleotide inhibitor of apolipoprotein B-100
synthesis indicated as an adjunct to lipid-lowering medications and
diet to reduce low density lipoprotein-cholesterol (LDL-C),
apolipoprotein B (apo B), total cholesterol (TC), and non-high
density lipoprotein-cholesterol (non-HDL-C) in patients with
homozygous familial hypercholesterolemia (HoFH). The safety
and effectiveness of KYNAMRO have not been established in patients
with hypercholesterolemia who do not have HoFH. The effect of
KYNAMRO on cardiovascular morbidity and mortality has not been
determined. The safety and effectiveness of KYNAMRO as an
adjunct to LDL apheresis have not been established; therefore, the
use of KYNAMRO as an adjunct to LDL apheresis is not
recommended
About Homozygous Familial Hypercholesterolemia
(HoFH)
HoFH is a rare genetic disease characterized
by extreme cholesterol levels. People with HoFH have inherited
mutations that limit the body's ability to clear cholesterol.
HoFH is extremely rare. As with other rare diseases, the true
prevalence of HoFH may be underestimated because of inadequate data
and under-diagnosis. Today, it is estimated that HoFH affects about
44,000 people globally. Medical literature includes different
criteria for marking an HoFH diagnosis. HoFH may be diagnosed by
clinical or genetic parameters, and may be considered in cases of
unusually high LDL-C. Because HoFH is genetic, it is important that
all family members of people with HoFH know their cholesterol
levels, regardless of their age.
ABOUT ISIS PHARMACEUTICALS, INC.
Isis is exploiting
its leadership position in RNA-targeted technology to discover and
develop novel drugs for its product pipeline and for its
partners. Isis' broad pipeline consists of 38 drugs to treat
a wide variety of diseases with an emphasis on cardiovascular,
metabolic, severe and rare diseases, including neurological
disorders, and cancer. Isis' partner, Genzyme, is
commercializing Isis' lead product, KYNAMRO®, in
the United States and other
countries for the treatment of patients with homozygous FH.
Isis has numerous drugs in Phase 3 development in severe/rare
diseases and cardiovascular diseases. These 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 the polyneuropathy and
cardiomyopathy forms of TTR amyloidosis; and ISIS-SMNRx,
a drug Isis is developing with Biogen to treat infants and children
with spinal muscular atrophy, a severe and rare neuromuscular
disease. Isis' patents provide strong and extensive
protection for its drugs and technology. Additional
information about Isis is available at www.isispharm.com.
ISIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENT
This
press release includes forward-looking statements regarding Isis'
collaboration with Genzyme, a Sanofi company, and the development,
activity, therapeutic benefit and safety of KYNAMRO in treating
patients with high cholesterol. 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.
In this press release, unless the context requires otherwise,
"Isis," "Company," "we," "our," and "us" refers to Isis
Pharmaceuticals and its subsidiaries.
Isis Pharmaceuticals® is a registered trademark of
Isis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark
of Isis Pharmaceuticals, Inc. KYNAMRO® is a
registered trademark of Genzyme Corporation.
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SOURCE Isis Pharmaceuticals, Inc.