Sarepta Therapeutics and University of Western Australia Announce Exclusive Worldwide Licensing Agreement for Exon-Skipping

Sarepta Therapeutics and University of Western Australia Announce Exclusive 
Worldwide Licensing Agreement for Exon-Skipping Program in
Duchenne Muscular Dystrophy 
Deal Enables Development of New Exon-Skipping Drug Candidates in DMD 
CAMBRIDGE, MA and PERTH, AUSTRALIA -- (Marketwired) -- 04/11/13 -- 
Sarepta Therapeutics, Inc. (NASDAQ: SRPT) and the University of
Western Australia (UWA) today announced that they have entered into
an exclusive, worldwide licensing agreement for intellectual property
rights to support the development of exon-skipping drug candidates
for the treatment of Duchenne muscular dystrophy (DMD). The agreement
grants Sarepta rights to UWA's extensive patent portfolio in DMD and
enables the Company to build out its exon-skipping pipeline with new
candidates based on its proprietary phosphorodiamidate morpholino
oligomer (PMO) technology to address the majority of patients with
the disorder worldwide. The deal expands an agreement first signed in
2008, which supported the development of several exon-skipping drugs
including eteplirsen, Sarepta's lead clinical candidate for the
treatment of patients with DMD who have a genotype amenable to
skipping of exon 51. 
"This agreement underscores our commitment to pursue treatments for
all DMD patients who can benefit from our exon-skipping technology,
even those with rare genetic mutations," said Chris Garabedian,
President and Chief Executive Officer of Sarepta Therapeutics. "We
believe this collaboration allows us to apply our unique morpholino
chemistry with UWA's groundbreaking research on the dystrophin gene
to develop potentially best-in-class drugs for this disease across
the globe." 
"We are very encouraged by the recent progress of Sarepta's lead
clinical program eteplirsen, which we believe demonstrates the
strength of the underlying platform technology as well as our
partner's drug development expertise in DMD," said Andy Sierakowski,
Ph.D., Director of the Office of Industry and Innovation at UWA.
"This expanded collaboration with Sarepta enables us to translate our
understanding of the dystrophin gene into additional potential
exon-skipping therapeutics that address a majority of patients with
DMD, representing a major contribution by the University of Western
Australia to the field of gen
etic medicine." 
DMD is a rare and severe genetic disorder that affects boys and young
men. It is associated with errors in the gene for dystrophin, a
protein that plays a key structural role in muscle fibers. Patients
with DMD lack functional dystrophin, and regular activity causes
progressive muscle damage leading to weakness, loss of ambulation,
respiratory and cardiac dysfunction, and eventually premature death.
There are no approved treatments. 
Exon-skipping is an innovative disease-modifying treatment approach
designed to skip an exon in the dystrophin gene, thereby enabling the
repair of specific genetic mutations and the production of a
functional, but shorter, form of dystrophin. Sarepta currently has
four exon-skipping programs in DMD addressing patients with genotypes
amenable to skipping of exons 51, 45, 50 and 53.  
Under the terms of the agreement, UWA is eligible to receive up to
$7.1 million in upfront and development milestone payments, as well
as a low single-digit royalty on net sales of all approved medicines
under the collaboration. Additional financial terms were not
disclosed.  
About Duchenne Muscular Dystrophy 
DMD is an X-linked rare, degenerative neuromuscular disorder causing
severe progressive muscle loss and premature death. One of the most
common fatal genetic disorders, DMD affects approximately one in
every 3,500 boys worldwide. A devastating and incurable
muscle-wasting disease, DMD is associated with specific errors in the
gene that codes for dystrophin, a protein that plays a key structural
role in muscle fiber function. Progressive muscle weakness in the
lower limbs eventually spreads to the arms, neck and other areas.
Eventually, increasing difficulty in breathing due to respiratory
muscle dysfunction requires ventilation support, and cardiac
dysfunction can lead to heart failure. The condition is universally
fatal, and death usually occurs before the age of 30. 
About Sarepta Therapeutics 
Sarepta Therapeutics is focused on developing first-in-class
RNA-based therapeutics to improve and save the lives of people
affected by serious and life-threatening rare and infectious
diseases. The Company's diverse pipeline includes its lead program
eteplirsen, for Duchenne muscular dystrophy, as well as potential
treatments for some of the world's most lethal infectious diseases.
Sarepta aims to build a leading, independent biotech company
dedicated to translating its RNA-based science into transformational
therapeutics for patients who face significant unmet medical needs.
For more information, please visit us at www.sareptatherapeutics.com. 
About the University of Western Australia 
The University of Western Australia is a leading Australian
research-based institution that ranks internationally among the
world's top 100 universities. UWA is a member of Australia's Group of
Eight top research universities, and one of only two Australian
members of the Worldwide Universities Network, a partnership of 18
research-led universities from Europe, Africa, the Americas and the
Asia-Pacific. The University is also a foundation member of the
Matariki Network of high-quality, research-intensive universities
with a particular focus on student experience. The campus is based in
Perth, capital city of Western Australia. 
Forward Looking Statement 
This press release contains forward-looking statements. These
forward-looking statements generally can be identified by use of
words such as "believes or belief," "anticipates," "plans,"
"expects," "will," "intends," "potential," "possible," "advance" and
similar expressions. These forward-looking statements include
statements about the development of eteplirsen and its potential
commercialization as well as statements regarding the potential of
Sarepta's other pipeline candidates. 
Each forward-looking statement contained in this press release is
subject to risks and uncertainties that could cause actual results to
differ materially from those expressed or implied by such statement.
Applicable risks and uncertainties include, among others: subsequent
clinical trials may fail to demonstrate the safety and efficacy of
eteplirsen or replicate results; treatment of patients with DMD using
eteplirsen over a longer duration may not lead to significant
clinical benefit; any of Sarepta's drug candidates, including
eteplirsen, may fail in development, may not receive required
regulatory approvals, or may not become commercially viable due to
delays or other reasons; our enhanced intellectual property rights
may provide us neither the ability to exclude competitors nor freedom
to commercialize our product candidates; and those identified under
the heading "Risk Factors" in Sarepta's Annual Report on Form 10-K
for the year ended December 31, 2012, and filed with the Securities
and Exchange Commission.  
Any of the foregoing risks could materially and adversely affect
Sarepta's business, results of operations and the trading price of
Sarepta's common stock. For a detailed description of risks and
uncertainties Sarepta faces, you are encouraged to review the
Company's filings with the Securities and Exchange Commission. We
caution investors not to place considerable reliance on the
forward-looking statements contained in thi
s press release. Sarepta
does not undertake any obligation to publicly update its
forward-looking statements based on events or circumstances after the
date hereof.  
"Safe Harbor" Statement under the Private Securities Litigation
Reform Act of 1995: The statements that are not historical facts
contained in this release are forward-looking statements that involve
risks and uncertainties, including, but not limited to, the results
of research and development efforts, the results of preclinical and
clinical testing, the effect of regulation by the FDA and other
agencies, the impact of competitive products, product development,
commercialization and technological difficulties, and other risks
detailed in the company's Securities and Exchange Commission filings. 
Sarepta Investor Contact: 
Erin Cox 
+1.857.242.3714 
ecox@sareptatherapeutics.com 
Sarepta Media Contact:
Jim Baker
+1.857.242.3710
jbaker@sareptatherapeutics.com 
UWA Media Contact:
Simon Handford
+61.8.6488.8187
simon.handford@uwa.edu.au 
 
 
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