Sarepta Therapeutics, Inc., a developer of innovative RNA-based
therapeutics, and Flagship Biosciences LLC, a leading
tissue-based companion diagnostics firm, today announced a multi-year,
multi-product partnership for the development of automated quantitative
endpoint measurements in muscular dystrophy to support the advancement
of Sarepta’s Duchenne muscular dystrophy (DMD) drug pipeline, including
its lead candidate, eteplirsen.
DMD is caused by the absence of functional dystrophin in affected
patients’ muscle tissue. Dystrophin protein level is a fundamental
biomarker used to assess therapies that aim to produce and restore the
expression of dystrophin, such as exon-skipping therapies like
eteplirsen. In order to optimally and efficiently evaluate therapeutic
efficacy in patients, the next generation of protocols are being
developed to digitally automate and standardize dystrophin measurement
in tissue biopsies to speed the process while ensuring consistency. The
establishment of these new standardized methods for automated
quantitation is being enabled though the proprietary image analysis
platform and digital pathology capabilities developed by Flagship.
This newly established collaboration with Flagship demonstrates
Sarepta’s commitment to enhancing the objective measurement of
dystrophin in tissue samples for its growing pipeline of RNA-based
therapeutics to treat DMD. The agreement between the companies also
further strengthens Flagship Biosciences’ leadership role in diagnostic
image analysis and digital pathology to support and optimize targeted
drug development.
Dave Young, Flagship’s Chief Pathologist, commented, “Flagship
Biosciences has developed tools and expertise in quantitative pathology,
image analysis, and tissue-based assays that are well-suited for use in
a regulated environment. It’s exciting to work with a partner like
Sarepta to design and implement an integrated fit-for-purpose assay and
automated quantitative interpretation approach that accelerates the
development of drugs for unmet needs such as eteplirsen for the
treatment of DMD.”
“Sarepta is fully committed to quickly and thoughtfully developing
effective therapies to treat Duchenne muscular dystrophy,” said Ed Kaye,
M.D., Chief Medical Officer of Sarepta. “As our RNA-based therapies
advance in Duchenne and other disease areas, Flagship’s quantitative
tissue-based diagnostics development expertise will play a key role in
accelerating our clinical success. The sophistication and rigor of
Flagship’s image analysis capabilities are exceptional. As we embark
upon several clinical studies within multiple centers both in the US and
Europe, our ability to automate the dystrophin quantification process
while insuring speed, accuracy and consistency is important to our
ongoing and future clinical development efforts.”
Steve Potts, Ph.D., Chief Executive Officer of Flagship Biosciences,
further added, “Sarepta’s novel approaches to therapeutic RNA targeting
such as exon-skipping must be matched with equally ground-breaking
approaches in digital diagnostic laboratory measurements. It is a
pleasure to see the discipline and commitment by both Sarepta and
Flagship to meet the demand for automating the precise evaluation and
standardization of endpoints used in these clinical trials.”
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 born worldwide. A devastating and icurable 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 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’s Proprietary Exon-Skipping Platform Technology
Eteplirsen is Sarepta's lead drug candidate and is designed to address
the underlying cause of DMD by enabling the production of a functional
internally deleted dystrophin protein. Data from clinical studies of
eteplirsen in DMD patients have demonstrated a broadly favorable safety
and tolerability profile and restoration of dystrophin protein
expression. Eteplirsen uses Sarepta's novel phosphorodiamidate
morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping
technology to skip exon 51 of the dystrophin gene enabling the repair of
specific genetic mutations that affect approximately 13 percent of the
total DMD population. By skipping exon 51, eteplirsen may restore the
gene's ability to make a shorter, but still functional, form of
dystrophin from messenger RNA, or mRNA. Promoting the synthesis of an
internally deleted dystrophin protein is intended to stabilize or
significantly slow the disease process and prolong and improve the
quality of life for patients with DMD. Sarepta is also developing other
PMO-based exon-skipping drug candidates intended to treat additional
genetic subpopulations of patients with DMD by restoring dystrophin
production. Sarepta’s exon-skipping technology has the potential to
treat a large majority of the DMD population.
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 and follow-on drug
candidates, 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.sarepta.com.
About Flagship Biosciences
Flagship Biosciences delivers quantitative pathology services for drug
and medical device development. Flagship specializes in the development
of tissue-based
diagnostic assays which support pharmacodynamic, surrogate efficacy,
or patient selection approaches. Flagship Biosciences services are
currently utilized by over 100 pharmaceutical and biotech firms for both
diagnostic and therapeutic programs. Flagship Biosciences is a privately
held company located in Westminster, Colorado, and serves a global base
of clients. For more information, please visit us at www.flagshipbio.com.
Forward Looking Statements
Except for historical information, the matters discussed in this news
release may be considered "forward-looking" statements within the
meaning of Section 27A of the Securities Act of 1933, as amended and
Section 21E of the Securities Exchange Act of 1934, as amended. Such
statements include declarations regarding the intent, belief or current
expectations of the Company and its management, including those related
to cash flow, gross margins, revenues, and expenses are dependent on a
number of factors outside of the control of the company including, inter
alia, the markets for the Company’s products and services, costs of
goods and services, other expenses, government regulations, litigations,
and general business conditions. See Risk Factors in the Company’s Form
10-K for the fiscal year ended July 31, 2013. Investors are cautioned
that any such forward-looking statements are not guarantees of future
performance and involve a number of risks and uncertainties that could
materially affect actual results. The Company disclaims any obligations
to update any forward-looking statement as a result of developments
occurring after the date of this press release.
Copyright Business Wire 2014