Akcea Therapeutics, Inc. (NASDAQ:AKCA), 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 initiation of a
Phase 2b clinical study of AKCEA-APOCIII-LRx in patients with
hypertriglyceridemia and established cardiovascular disease (CVD).
The study will evaluate the safety and efficacy of different doses
and dosing frequencies of AKCEA-APOCIII-LRx in this patient
population and provide valuable information for a potential Phase 3
cardiovascular outcomes study.
AKCEA-APOCIII-LRx is part of a strategic collaboration with
Novartis to develop and co-commercialize AKCEA-APO(a)-LRx and
AKCEA-APOCIII-LRx. Under the terms of the collaboration, if
Novartis exercises its option after successful completion of the
Phase 2 study, it will be responsible for a global Phase 3
cardiovascular outcome study as well as worldwide development and,
if approved, global co-commercialization activities.
AKCEA-APOCIII-LRx, an antisense drug that uses Ionis’ advanced
LIgand
Conjugated Antisense (LICA)
technology, is designed to reduce the production of apolipoprotein
C-III, or apoC-III. Elevated levels of apoC-III correlate with high
triglyceride levels and have been associated with metabolic
abnormalities and premature cardiovascular disease.
“The combination of elevated levels of apoC-III and
triglycerides has long been linked with cardiovascular disease,
but, until now, no drug has been capable of addressing both risk
factors. In our Phase 1 study AKCEA-APOCIII-LRx significantly
reduced both apoC-III and triglycerides up to 84% and 71%,
respectively, and demonstrated a good safety and tolerability
profile,” said Louis O’Dea, chief medical officer at Akcea
Therapeutics. “These data strongly support further evaluation
of AKCEA-APOCIII-LRx. We believe positive results from studies in
this broad patient population could lead to wide-ranging advances
in the treatment of people with apoC-III and triglyceride-driven
cardiovascular disease.”
The multicenter, randomized, double-blind, placebo-controlled,
dose-ranging Phase 2b study will evaluate the safety and efficacy
of AKCEA-APOCIII-LRx in approximately 100 patients with
hypertriglyceridemia and established CVD. Akcea anticipates
reporting top-line data from this study in 2019. For further study
information, please visit www.clinicaltrials.gov and search
for AKCEA-APOCIII-LRx.
Akcea recently reported data from a Phase 1/2a clinical study of
AKCEA-APOCIII-LRx in healthy volunteers and patients with elevated
triglyceride levels. Patients in the study were treated with
multiple doses at either weekly or monthly dosing intervals.
Patients treated with AKCEA-APOCIII-LRx demonstrated significant
dose-dependent reductions in apoC-III protein and triglycerides. No
serious adverse events, no platelet count reductions or changes in
liver function, and no adverse events leading to treatment
discontinuation were observed.
ABOUT THE AKCEA AND NOVARTIS COLLABORATION
In January 2017, Akcea and Ionis entered into an exclusive,
worldwide option and collaboration agreement with Novartis to
develop and commercialize AKCEA-APOCIII-LRx and
AKCEA-APO(a)-LRx. Akcea is conducting a Phase 2 dose-ranging study
for AKCEA-APO(a)-LRx in patients with high lipoprotein(a), or
Lp(a), with established cardiovascular disease with data planned in
2018 and a Phase 2b dose-ranging study for AKCEA-APOCIII-LRx in
patients with hypertriglyceridemia and established cardiovascular
disease with data planned in 2019. The goal of both studies is to
choose the optimal dose and evaluate alternative dose schedules,
such as monthly dosing, for Phase 3 cardiovascular outcomes
studies. Novartis has the option to license each drug after
successful completion of the Phase 2 dose-ranging study and
end-of-Phase 2 meeting with FDA. Upon option exercise for each
drug, Novartis will pay Akcea a $150 million license fee
of which 50% will be paid to Ionis. If licensed, Novartis plans to
conduct a global Phase 3 cardiovascular outcome study in high-risk
patients. Novartis will be responsible for worldwide development
and, if approved, co-commercialization activities. Akcea retains
the right to co-commercialize any successful drug through its
specialty sales force focused on lipid specialists on terms and
conditions to be agreed with Novartis.
ABOUT AKCEA-APOCIII-LRx AKCEA-APOCIII-LRx is a
ligand conjugated antisense (LICA) drug designed to reduce the
production of apolipoprotein C-III, or apoC-III. ApoC-III is a
protein produced in the liver that plays a central role in the
regulation of serum triglycerides. Genetically reduced levels
of apoC-III are correlated to lower levels of triglycerides and
lower risk of cardiovascular disease whereas elevated levels of
apoC-III correlate with high triglyceride levels that have been
associated with multiple metabolic abnormalities, such as insulin
resistance and/or metabolic syndrome. Akcea and Ionis are
developing AKCEA-APOCIII-LRx to treat hypertriglyceridemia and
established cardiovascular disease.
In a Phase 1/2a clinical study in healthy volunteers and
patients with elevated triglyceride levels, treatment with multiple
doses of AKCEA-APOCIII-LRx resulted in dose-dependent reductions in
apoC-III protein of up to 84% after six weeks of treatment.
Treatment with AKCEA-APOCIII-LRx also resulted in dose-dependent
reductions in triglycerides of up to 71%. Significant
dose-dependent reductions of up to 30% in apolipoprotein B (apoB)
and increases of up to 100% in high-density lipoprotein cholesterol
(HDL-C), were also observed. Both decreased levels of apoB and
increased levels of HDL-C are associated with decreased
cardiovascular risk. AKCEA-APOCIII-LRx was well tolerated in the
study. No serious adverse events, no platelet count reductions or
changes in liver function, and no adverse events leading to
treatment discontinuation were observed. Phase 1 studies of all
three of Akcea’s LICA drugs have shown that doses up to 30 fold
lower than non-LICA drugs result in consistent target reductions
and a favorable safety and tolerability profile.
ABOUT AKCEA THERAPEUTICSAkcea Therapeutics, an
affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), 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, including volanesorsen, AKCEA-APO(a)-LRx,
AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx, all with the potential to
treat multiple diseases. All four drugs were discovered by and are
being co-developed with Ionis, a leader in antisense therapeutics,
and are 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 the therapeutic and commercial potential of
volanesorsen, AKCEA-APOCIII-LRx 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,
“Ionis”, “Akcea,” “Company,” “Companies” “we,” “our,” and “us”
refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc.
Media and Investor Contact: Kathleen
Gallagher
Head of Communications and Investor Relations, Akcea
Therapeutics (617)-207-8509 kgallagher@akceatx.com
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