November 2016

We are aware of the severe unmet needs in treating Duchenne muscular dystrophy (DMD) in Europe. Sarepta is committed to the global community and to working closely with our partners in Europe.  Our ultimate goal is to develop treatments for all patients affected by DMD. 

Eteplirsen Marketing Authorisation Application (MAA) Update

• We are currently planning to submit the MAA for eteplirsen to the European Medicines Agency (EMA) by the end of 2016. As you may be aware, once a submission is validated by EMA’s Committee for Medicinal Products for Human Use (CHMP), the review period for an application by the CHMP is typically 12-15 months.

Planned and Ongoing Clinical Trials with Eteplirsen

• In order to satisfy the requirements of the Pediatric Investigational Plan (PIP) for eteplirsen with the EMA, Sarepta has built an extensive clinical program to evaluate safety and efficacy. This includes conducting a study of eteplirsen in infants and young boys, ≥ 6 months to 4 years of age, in Europe.  
• The PROMOVI (“Study 301”) clinical sites continue to enroll patients in the United States (U.S.).
• Studies 203 (boys between the ages of 4-6) and 204 (late ambulatory to non-ambulatory) will continue until completion (96 weeks). We believe these data will provide more information to help evaluate the safety and efficacy of eteplirsen in a broad patient population. 
• The approval in the U.S. was under the accelerated approval pathway which requires further studies to confirm clinical benefit. Discussions are currently ongoing with the FDA to agree on protocol design, which is expected to be finalized in Q2 2017. We currently anticipate that this study will have clinical sites in Europe.
• We are currently evaluating the timing for an open-label safety study with eteplirsen for the boys and young men who participated in studies 33 and 28.

Clinical Trials for Skipping Exon 45 and Exon 53

• The Phase 3 study of SRP-4045 and SRP-4053 (ESSENCE) is currently active and enrolling patients in the United States. We continue to work with clinical sites throughout Europe and Canada to ensure patient enrollment can begin as soon as possible. We currently anticipate that enrollment at European and Canadian clinical sites will begin in the coming months.
• Study 4053-101 that is being conducted at clinical sites in the United Kingdom, France and Italy, has completed enrollment and we expect to see some data generated from this trial in 2017.

Expanded Access/Compassionate Use

• We are mindful of the urgency of patient needs for access around the globe. We have determined that at this time and stage of our development, the best path to providing access to patients is through the regulatory approval process. 

Sarepta’s Ongoing Commitment to Furthering Research in DMD

• Sarepta has a pipeline and development plan to further research and development in DMD.  
• Exon-skipping therapeutics for patients amenable to skipping exons 52, 50, and 8 are in, or nearing, preclinical development. 
• Exon-skipping therapeutics for patients amenable to skipping exons 35 and 55 are in an early stage discovery phase before initiating preclinical development. 
• We have a follow-on exon skipping evaluation program in place to identify candidates for other exon skips. We are prioritizing the evaluation of potential exon-skipping therapeutics to move forward, based on mutations that are most common, with the goal of reaching the broadest population as soon as possible.  
• Our team is continually exploring potential research collaborations that aim to advance the development of treatments for DMD, like our recent partnerships with Summit and Catabasis. 
• Sarepta plans to initiate a Duchenne registry program in 2017. Capturing the long-term safety and efficacy of eteplirsen in the real-world setting is of utmost importance in the near and long term.