CARLSBAD, Calif. and
CAMBRIDGE, Mass., Feb. 16, 2017 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) and Akcea Therapeutics, a
wholly-owned subsidiary of Ionis Pharmaceuticals, Inc., announced
today they have closed on their exclusive, worldwide option and
collaboration agreement with Novartis to develop and commercialize
AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx
following clearance under the Hart-Scott-Rodino Antitrust
Improvements Act. Following this approval and closing of the
transaction, Novartis will pay Ionis and Akcea a $75 million up-front payment and make a
$100 million equity investment in
Ionis, which equates to 1,631,435 shares at $61.30 per share. Novartis has an
obligation to make a further equity investment of $50 million in the next 18 months in either Ionis
at the same premium as the initial investment or in Akcea.
Ionis and Akcea are also eligible to receive a license fee as well
as development, regulatory and commercial milestone payments for
each drug as it advances. In addition, Ionis and Akcea are
eligible to receive tiered royalties in the mid-teens to low twenty
percent range on net sales of each drug.
ABOUT Lp(a)
Lp(a) is considered a key driver for
cardiovascular disease due to its association with an increased
risk of coronary heart disease. Lp(a) is a lipoprotein
particle that is assembled in the liver and consists of the
apolipoprotein(a) protein covalently linked to
LDL-cholesterol. Diet and lifestyle changes have little
impact on Lp(a) levels and current therapies are not able to
adequately reduce elevated levels of Lp(a) to acceptable levels in
patients who have severely elevated Lp(a). Additional
information is available through Lipoprotein (a) Foundation at
www.lipoproteinafoundation.org.
In a Phase 1/2a study in healthy volunteers with elevated
Lipoprotein(a), AKCEA-APO(a)-LRx produced significant
and sustained reductions in Lp(a) of up to 97% with mean reduction
of 79% after only a single, small volume dose and with multiple
doses, observed reductions of Lp(a) of up to 99% with a mean
reduction of 92%.
ABOUT APOCIII AND TRIGLYCERIDES
ApoC-III is a protein
produced in the liver that plays a central role in the regulation
of serum triglycerides. Humans who do not produce ApoC-III
have lower levels of triglycerides and lower instances of
cardiovascular disease. Humans with elevated levels of
ApoC-III have high triglycerides associated with multiple metabolic
abnormalities, such as insulin resistance and/or metabolic
syndrome. In addition, the prevalence of type 2 diabetes is
increased in patients with elevated triglycerides.
AKCEA-APOCIII-LRx is currently being evaluated in a
Phase 1/2a study in healthy volunteers with elevated
triglycerides.
ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics is focused
on developing and commercializing drugs for patients with serious
cardiometabolic diseases caused by lipid disorders.
Established as a wholly owned subsidiary of Ionis Pharmaceuticals,
Inc., Akcea has a robust portfolio of development-stage drugs
covering multiple targets and disease states. The drugs in
Akcea's pipeline are designed using Ionis' advanced RNA-targeted
antisense technology to address a number of lipid risk factors,
including, ApoC-III, triglycerides, Lp(a) and
LDL-cholesterol. Akcea's most advanced program, volanesorsen,
is in Phase 3 development to treat patients with either familial
chylomicronemia syndrome (FCS) or familial partial lipodystrophy
(FPL), two orphan lipid disorders that are characterized by
extremely high triglycerides and ApoC-III. Akcea is located
in Cambridge, Massachusetts.
Additional information about Akcea is available at
http://akceatx.com.
ABOUT IONIS PHARMACEUTICALS, INC.
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) is a drug
that has been approved in the U.S. for the treatment of spinal
muscular atrophy (SMA) in pediatric and adult patients.
Biogen is responsible for commercialization of SPINRAZA.
Drugs currently in Phase 3 development include volanesorsen,
a drug Ionis is developing and plans to commercialize through its
wholly owned subsidiary, Akcea Therapeutics, to treat patients with
either familial chylomicronemia syndrome or familial partial
lipodystrophy; and IONIS-TTRRx, a drug Ionis is
developing with GSK to treat patients with TTR amyloidosis.
Ionis' patents provide strong and extensive protection for
its drugs and technology. Additional information about Ionis
is available at www.ionispharma.com.
IONIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENT
This
press release includes forward-looking statements regarding Ionis
Pharmaceuticals' business, its agreement with Novartis, and the
development, activity, therapeutic and commercial potential and
safety of IONIS-APO(a)-LRx and
IONIS-APOCIII-LRx. 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,
2015, 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," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Ionis
Pharmaceuticals, Inc. SPINRAZA™ is a trademark of Biogen.
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/ionis-and-akcea-close-on-strategic-collaboration-with-global-pharmaceutical-company-to-develop-and-commercialize-akcea-apoa-lrx-and-akcea-apociii-lrx-300408349.html
SOURCE Ionis Pharmaceuticals, Inc.