CARLSBAD, Calif., April 9, 2018 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense
therapeutics, today announced that it has licensed
IONIS-AZ6-2.5-LRx, or AZD2693, to AstraZeneca (NYSE:
AZN) following advancement of the drug into development.
IONIS-AZ6-2.5-LRx is designed to inhibit an undisclosed
target to treat patients with nonalcoholic steatohepatitis (NASH).
In conjunction with this milestone, global, science-led
biopharmaceutical company AstraZeneca will pay a $30 million license fee to Ionis. AstraZeneca
will be responsible for further development and commercialization
of IONIS-AZ6-2.5-LRx.
"This is the third drug to enter development under our strategic
collaboration with AstraZeneca to discover drugs to treat
cardiovascular, renal and metabolic diseases.
IONIS-AZ6-2.5-LRx incorporates many of the advancements
we have made in antisense technology, including our
LIgand-Conjugated Antisense (LICA) and Generation 2.5 chemistry,
and is the second drug in our collaboration to incorporate both
modifications. By combining Generation 2.5 and LICA, we generate
drugs that have the advantages of both higher affinity chemistry
and efficient cell-specific targeting. This combination provides us
with drugs that are substantially more potent than either
Generation 2.5 or LICA alone, and supports administration of
infrequent, very low doses, and even enables the potential for oral
dosing," said Brett P. Monia, chief
operating officer, senior vice president of antisense drug
discovery and translational medicine at Ionis Pharmaceuticals. "We
are pleased with how quickly we moved IONIS-AZ6-2.5-LRx
from target validation into development, exemplifying the
efficiency of our antisense platform. AstraZeneca has played a
strategic role in advancing this program forward by providing both
preclinical and development expertise in NASH that has contributed
to the rapid advance of this drug into development. We look forward
to AstraZeneca moving this program swiftly into clinical testing
and ultimately to the market."
As IONIS-AZ6-2.5-LRx advances in development, Ionis
may also receive up to $300 million
in additional development and regulatory milestone payments, as
well as tiered royalties up to the low teens from sales of the
drug.
ABOUT IONIS AND ASTRAZENECA
AstraZeneca and Ionis have
a strategic alliance focused on leveraging antisense technology to
discover and develop antisense therapies for cardiovascular,
metabolic and renal diseases. The collaboration builds on a broad
existing relationship between the two companies and is supported by
Ionis' expertise in cardiovascular disease and antisense technology
and AstraZeneca's drug development expertise and infrastructure. In
the collaboration, Ionis is primarily responsible for creating
development candidates. AstraZeneca paid Ionis an upfront fee of
$65 million at the initiation of the
collaboration. Beyond AZD2693, AstraZeneca has the option to
license additional antisense drugs upon development candidate
nomination and will pay a license fee for each drug licensed plus
development and regulatory milestones and sales royalties for each
program that AstraZeneca advances. Ionis and AstraZeneca are also
collaborating to discover and develop antisense drugs to treat
cancer.
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 40
drugs in development. SPINRAZA® (nusinersen) has been
approved in global markets for the treatment of spinal muscular
atrophy (SMA). Biogen is responsible for commercializing SPINRAZA.
Drugs that have successfully completed Phase 3 studies include
inotersen, an antisense drug Ionis is developing to treat patients
with hereditary ATTR (hATTR) amyloidosis, and volanesorsen, an
antisense drug discovered by Ionis and co-developed by Ionis and
Akcea Therapeutics, Inc. 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
rare diseases. If approved, inotersen and volanesorsen will be
commercialized through Ionis' affiliate, Akcea. Inotersen is under
regulatory review in the U.S. and EU. Volanesorsen is under
regulatory review in the U.S., EU, and Canada. 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'
alliance with AstraZeneca and the therapeutic and commercial
potential of IONIS-AZ6-2.5-LRx (AZD2693). 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, 2017, which is on file with the SEC.
Copies of this 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 Akcea Therapeutics, Inc.
SPINRAZA® is a registered trademark of Biogen.
View original content with
multimedia:http://www.prnewswire.com/news-releases/ionis-and-astrazeneca-advance-new-drug-for-nash-300625828.html
SOURCE Ionis Pharmaceuticals, Inc.