Parent Project Muscular Dystrophy and Sarepta Therapeutics Partner to Launch Duchenne Outcomes Research Interchange, a Patient-Data Warehouse Serving the Entire Duchenne Community

HACKENSACK, N.J. and CAMBRIDGE, Mass., Sept. 7, 2018 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced plans today to launch a patient- and physician-reported outcomes data warehouse – the Duchenne Outcomes Research Interchange (the Interchange) – which will combine data from The Duchenne Registry with clinician entered data provided by its inaugural partner, Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases. The Duchenne Outcomes Research Interchange, scheduled to launch in 2019, will aggregate patient and clinician entered data obtained through various means, providing critical information to clinicians, sponsors, payers, regulators, and the patients/families who participate in PPMD's Duchenne Registry.

In parallel to PPMD establishing a new patient engagement platform, Sarepta will initiate their post-approval patient registry which will collect clinician-reported outcomes data from those with Duchenne on EXONDYS 51^® (eteplirsen). The registry will later expand to the broader Duchenne community.  These data will flow into the Interchange, governed by a steering committee and PPMD and become part of a comprehensive data set, which is intended to inform the understanding of the long-term impact of the therapies developed by Sarepta. PPMD plans to invite industry, advocacy, and academic partners to join the Interchange, following the successful integration with Sarepta.

For the last several years, PPMD has been pursuing enhancements of The Duchenne Registry, a patient reported outcomes (PRO) registry. The need to combine patient-reported outcomes data with clinician-reported outcomes data was clear, but with a new therapeutic landscape, the goal is to not only combine these data sets but to also incorporate electronic health record data (EHR) into a combined, centralized data interchange that could benefit the entire community. The Interchange will provide this central repository for all types of Duchenne data and 'real world evidence' to be collected and analyzed in one place.

The Duchenne Registry has been an invaluable resource for all Duchenne stakeholders, since its inception over 10 years ago, thanks to the participation of thousands of families who keep their records up-to-date. The creation of the Interchange, and premier partnership with Sarepta, firmly places PPMD's registry at the center of the Duchenne data world. For patients and families who currently engage with PPMD's Duchenne Registry, the transition will be relatively seamless, providing enhanced access, as well as new and improved features and real life conveniences for a more engaging and valuable user experience.

Knowing the importance of 'real-world evidence' to regulators and payers, PPMD has been collecting patient-reported outcome data for over a decade, informing the natural history of Duchenne. PPMD will now build upon this knowledge by combining it with clinician-reported outcome data to better meet the needs of the patient, research, and industry communities, enhancing the value of patient-reported data in a post-marketing surveillance space.

PPMD and Sarepta are working with preeminent companies involved in data acquisition and data aggregation to ensure that the Interchange partners chosen understand the needs of the Duchenne community and can offer the most advanced technology possible.

"Since a small group of parents and grandparents formed PPMD almost 25 years ago, we have believed in the importance of innovation and patient data in the fight to end Duchenne," said Pat Furlong, Founding President & CEO of PPMD. "The Duchenne Registry, now powered with 10 years of robust data thanks to our incredible community, is ready to expand and evolve. Partnering with Sarepta, the first company in the U.S. to receive an approval, to launch the Duchenne Outcome Research Interchange made perfect sense, and they will contribute their own powerful data set. We look forward to creating a tool that multiple stakeholders can turn to in an effort to accelerate the clinical trial process and bring safe and effective therapies to families faster."

"We could not be more proud to be partnering with PPMD on this important initiative," stated Doug Ingram, Sarepta's president and chief executive officer. "The potential of the Duchenne Outcomes Research Interchange is immense and the impact profound, serving as a trusted resource for patient and clinician reported outcomes. The repository of data culled from the Interchange holds the potential to improve the way in which the science and clinical development programs advance and ultimately how the best therapies reach patients with expediency."

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube.

About Sarepta Therapeutics

Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit www.sarepta.com.

About EXONDYS 51

EXONDYS 51 uses Sarepta's proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. 

Important Safety Information About EXONDYS 51

Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

In the 88 patients who received ≥30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in ≥10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

For further information, please see the full Prescribing Information.

About The Duchenne Registry

The Duchenne Registry (formerly DuchenneConnect) began in 2007, when a group of thought leaders in the Duchenne and Becker muscular dystrophy community began discussing the need for a new kind of resource that would connect and serve the needs of the entire community. What they envisioned was a central hub that would bring together those living with Duchenne or Becker, along with their families and caregivers, to connect them with medical research, clinical care, clinical trials, and each other. At the same time, it would also be a resource for researchers and industries with an interest in Duchenne, allowing access to aggregate, de-identified data provided by patients and their families — information that could prove vital to advances in care and treatment. Today, the result of this endeavor is The Duchenne Registry, the largest, most comprehensive registry for Duchenne and Becker muscular dystrophy, as well as female carriers.

Ann Martin, Director of The Duchenne Registry, adds that the success of this platform is a direct result of community participation: "When families join and update their account in The Duchenne Registry, they are strengthening the power of a 10-year-old network of patient-powered data that will be used to improve care for people living with Duchenne and increase understanding of the disorder. Families become citizen scientists by contributing to real scientific research, without ever leaving their homes."

To learn more about The Duchenne Registry, visit our website.

Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding PPMD's plan to launch the Interchange in 2019; Sarepta's plan to initiate its post-approval patient registry, and the plan to expand the registry to the broader Duchenne community and to flow these data into the Interchange, with the intention to inform the understanding of the long-term impact of the therapies developed by Sarepta; PPMD's plan to invite industry, advocacy, and academic partners to join the Interchange, following the successful integration with Sarepta; the expectation that the transition from Duchenne Registry to the Interchange will be relatively seamless; and the potential and the expected impact of the interchange.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others: the expected benefits and opportunities related to the partnership between PPMD and Sarepta may not be realized or may take longer to realize than expected; and those risks identified under the heading "Risk Factors" in Sarepta's most recent Annual Report on Form 10-K for the year ended December 31, 2017 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

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 Sarepta's 2017 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

SOURCE Parent Project Muscular Dystrophy

Related Links

http://www.parentprojectmd.org