Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy

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August 21st, 2014

New technology designed to speed dystrophin measurement while ensuring consistency

Automation of dystrophin measurement will aid development of Sarepta’s Duchenne muscular dystrophy candidates

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.”

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