Isis Pharmaceuticals Earns $1.5M from the Advancement of the Phase 2 Study of ISIS-SMN Rx in Infants with Spinal Muscular

Isis Pharmaceuticals Earns $1.5M from the Advancement of the Phase 2 Study of
             ISIS-SMN Rx in Infants with Spinal Muscular Atrophy

PR Newswire

CARLSBAD, Calif., Nov. 12, 2013

CARLSBAD, Calif., Nov. 12, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc.
(NASDAQ: ISIS) announced today that it has earned a $1.5 million milestone
payment from Biogen Idec related to the advancement of the ongoing Phase 2
study of ISIS-SMN[Rx] in infants with spinal muscular atrophy (SMA).


The Phase 2 study of ISIS-SMN[Rx] is an open-label, multiple-dose,
dose-escalation pilot study in infants who have been diagnosed with Type I
SMA.To meet enrollment criteria, infants must be between the ages of three
weeks and seven months, live in close proximity to a study site and pass
screening evaluations conducted at study sites.The study is being conducted
at centers in the United States and Canada.For further study information,
please visit and search for ISIS-SMN[Rx].

]ISIS-SMN[Rx] is an investigational compound designed to alter the splicing of
a closely related gene (SMN2) to increase production of fully functional SMN
protein.The United States Food and Drug Administration granted orphan drug
status and fast track designation to ISIS-SMN[Rx] for the treatment of
patients with SMA.Isis is currently in collaboration with Biogen Idec to
develop and potentially commercialize the ISIS-SMN[Rx] to treat all types of
SMA. Under the terms of the January 2012 agreement, Isis is responsible for
global development and Biogen Idec has the option to license the compound
until completion of the first successful Phase 2/3 study. ISIS-SMN[Rx] is
currently being evaluated in two Phase 1b/2a multiple-dose, dose-escalation
studies. The first is in children with Type II or Type III SMA. The second is
in infants with Type I SMA.

Isis acknowledges support from the following organizations for ISIS-SMN[Rx]:
Muscular Dystrophy Association, SMA Foundation, Families of SMA and
intellectual property licensed from Cold Spring Harbor Laboratory and the
University of Massachusetts Medical School.

SMA is a severe genetic disease that affects approximately 30,000-35,000
patients in the United States, Europe and Japan. SMA is caused by a loss of,
or defect in, the survival motor neuron 1 (SMN1) gene leading to a decrease in
the survival motor neuron (SMN) protein. SMN is critical to the health and
survival of nerve cells in the spinal cord responsible for neuromuscular
growth and function. One in 50 people, the equivalent of about 6 million
people in the United States, are carriers of a defective SMN1 gene, which is
unable to produce fully functional SMN protein. Carriers experience no
symptoms and do not develop the disease. However, when both parents are
carriers, there is a one in four chance that their child will have SMA. The
severity of SMA correlates with the amount of SMN protein. Infants with Type I
SMA, the most severe form of the disease, produce very little SMN protein and
have a life expectancy of less than two years. Children with Type II have
greater amounts of SMN protein but still have a shortened lifespan and are
never able to stand independently. Children with Type III have a normal
lifespan but accumulate life-long physical disabilities as they grow.

Isis is exploiting its leadership position in antisense technology to discover
and develop novel drugs for its product pipeline and for its partners.Isis'
broad pipeline consists of 30 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 for the treatment of
patients with HoFH. Isis' patents provide strong and extensive protection for
its drugs and technology. Additional information about Isis is available at

This press release includes forward-looking statements regarding Isis'
alliance with Biogen Idec, the discovery, development, activity, therapeutic
potential, safety and commercialization of ISIS-SMN[Rx] and the discovery,
development and therapeutic potential of an antisense drug for the treatment
of spinal muscular atrophy. 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, 2012, 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

Isis Pharmaceuticals® is a registered trademark of Isis Pharmaceuticals, Inc.
KYNAMRO™ is a trademark of Genzyme Corporation.

SOURCE Isis Pharmaceuticals, Inc.

Contact: D. Wade Walke, Ph.D., Vice President, Corporate Communications and
Investor, Relations, 760-603-2741; Amy Blackley, Ph.D., Associate Director,
Corporate Communications, 760-603-2772
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