Earlier this month Sarepta announced a change in leadership, with CMO Ed Kaye taking over as interim CEO.

Following that announcement the team at Sarepta organized a call with Duchenne patient organizations and foundations to update them on the transition.

The messages were very clear from the call:

  • Everything is still moving forward - they are all about continuity.
  • There is a renewed passion to get 51 done and to work on the follow on exons.
  • Sarepta has a dedicated team who have redoubled their efforts.

Below is a short update from the team at Sarepta regarding their ongoing Duchenne program.

PPMD remains focused on accelerating Duchenne development pipelines so that safe and effective therapies are available to our community at the earliest moment possible – and stands at the ready, as always, to continue partnering with Sarepta to help in any and all ways.


Sarepta’s Goal for the DMD Community
Sarepta’s goal is to be part of the DMD community and have open lines of communication with patients, care givers, and advocates. We look forward to partnering with the community to make advances in the field of DMD. We remain relentless and committed to developing exon-skipping therapies. We understand and are keenly aware that time is of the essence for DMD families.

Background of Interim CEO and CMO, Edward M. Kaye, M.D.
Dr. Kaye has been Sarepta’s Chief Medical Officer since June of 2011, during which time he has been responsible for the company’s medical and clinical operations. He was previously Group Vice President of Clinical Development at Genzyme Corporation, one of the most successful biotechnology companies in the United States, from April 2007 to June 2011, where he supervised clinical development programs for rare diseases, including lysosomal storage diseases and genetic neurological disorders. While at Genzyme, Dr. Kaye established a track record of leading teams through the regulatory process and brought some of the most successful rare-disease drugs to market, including Myozyme, Lumizyme, and Fabrazyme. Prior to this, Dr. Kaye held various roles at Genzyme Corporation since 2001, including Vice President of Medical Affairs for Lysosomal Storage Diseases, Vice President of Clinical Research and Interim Head of PGH Global Medical Affairs.

Clinical Updates

  • We do not anticipate changes to any of our ongoing or planned clinical programs.
  • Eteplirsen Confirmatory Study (PROMOVI/Study 4658-301): Phase III open-label safety & efficacy study to treat ambulatory boys with DMD
    • Update: Enrolling and dosing at various sites. We plan to initiate an extension phase of this study at its conclusion and are designing a protocol that avoids treatment interruptions when patients roll over into the extension phase.
  • Eteplirsen Safety Study (Study 4658-204): Phase II open-label safety study to treat advanced stage DMD
    • Update: Enrolling and dosing at various sites. Progressing on schedule with completion of enrollment anticipated by mid-year.
  • Eteplirsen Safety Study (Study 4658-203): Phase II open-label safety study to treat early stage DMD
    • Update: We expect to announce enrollment, to begin in the second quarter of 2015.
  • Exon 53 Skipping (SRP-4053) Study (Study 4053-101)
    • Update: First phase of the study has been completed at clinical sites in Europe
  • Exon 45 Skipping (SRP-4045)and Exon 53 Skipping (SRP-4053) Study (Study 4045-301)
    • Update: We expect to initiate the trial in the US by mid-year, with enrollment beginning in Europe late 2015 or early 2016

Regulatory Update

  • We are working diligently to collect and analyze the data that were requested by the FDA for inclusion in our NDA submission for eteplirsen.
  • We continue to work towards a mid-year submission of our eteplirsen NDA.

Contact for Questions

Forward Looking-Statements
This patient community update contains various forward-looking statements regarding regulatory, clinical and other Sarepta matters. Each forward-looking statement contained in this update 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 those identified under the heading “Risk Factors” in Sarepta’s Annual Report on Form 10-K for the year ended December 31, 2014  (http://www.sec.gov/Archives/edgar/data/873303/000119312515063997/d...
and other documents filed by Sarepta with the Securities and Exchange Commission.

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