CARLSBAD, Calif., Dec. 19, 2016 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has
advanced its lead drug candidate IONIS-AZ4-2.5-LRx,
which has now been renamed AZD8233, into preclinical development.
AZD8233 is designed to inhibit an undisclosed target to treat
cardiovascular disease and is also the first of a new class of
antisense drugs that uses both Ionis' proprietary Generation 2.5
chemistry and its LIgand Conjugated Antisense
or LICA technology. In conjunction with this milestone, Ionis
earned a $25 million milestone
payment from AstraZeneca (NYSE:AZN).
"This is the first drug to enter development under our strategic
collaboration with AstraZeneca in cardiovascular, metabolic and
renal diseases. We are pleased with how quickly this important
program has advanced. AZD8233 incorporates many of the recent
advancements we have made in antisense technology, including LICA
and Generation 2.5 chemistry," said Brett
Monia, Ph.D., senior vice president of drug discovery at
Ionis. "By combining Generation 2.5 and LICA, we have generated a
drug that has the advantages of both higher affinity chemistry and
efficient cell-specific targeting. This combination has allowed us
to produce a drug that is substantially more potent than either
Generation 2.5 or LICA alone, which should support both a very low
dose and infrequent dosing."
As AZD8233 advances in development, Ionis will be eligible to
receive up to $300 million in
additional development and regulatory milestone payments as well as
tiered, low double-digit royalties from sales of the drug.
AstraZeneca will be responsible for further developing and
commercializing AZD8233.
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
AZD8233, 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
a dozen drugs in mid- to late-stage development. 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; IONIS-TTRRx, a drug Ionis is developing
with GSK to treat patients with TTR amyloidosis; and
SPINRAZATM (nusinersen), a drug licensed by Biogen to
treat infantile and later-onset spinal muscular atrophy.
Ionis' patents provide strong and extensive protection for its
drugs and technology. Additional information about Ionis is
available at www.ionispharma.com.
ABOUT ASTRAZENECA
AstraZeneca is a global, science-led
biopharmaceutical company that focuses on the discovery,
development and commercialisation of prescription medicines,
primarily for the treatment of diseases in three main therapy areas
- Oncology, Cardiovascular & Metabolic Diseases and
Respiratory. The Company also is selectively active in the
areas of autoimmunity, neuroscience and infection.
AstraZeneca operates in over 100 countries and its innovative
medicines are used by millions of patients worldwide. For
more information, please visit www.astrazeneca.com.
IONIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENT
This
press release includes forward-looking statements regarding Ionis'
alliance with AstraZeneca. 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. SPINRAZATM is a trademark of
Biogen.
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/ionis-and-astrazeneca-advance-first-generation-25-lica-drug-into-preclinical-development-to-treat-cardiovascular-disease-300381338.html
SOURCE Ionis Pharmaceuticals, Inc.