Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key …

CAMBRIDGE, Mass.--(BUSINESS WIRE)--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 Sareptas 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 Sareptas 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, Flagships 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. Its 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, Flagships quantitative tissue-based diagnostics development expertise will play a key role in accelerating our clinical success. The sophistication and rigor of Flagships 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, Sareptas 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 Sareptas 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. Sareptas exon-skipping technology has the potential to treat a large majority of the DMD population.

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Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key ...