Sarepta has provided another quarterly update to the community, including information on their PPMO (Peptide Conjugated Phosphorodiamidate Morpholino Oligomer), an exon skipping technology designed to efficiently get inside cells, with the goal of producing more dystrophin in the muscle groups affected in Duchenne.

From Sarepta:

Hello Duchenne Advocacy Community,

This quarter, Sarepta had the great opportunity to meet and learn from Alan Chaulet, Vice President of All Wheels Up, an organization working to promote wheelchair accessible airplane travel. Their work has resonance. Challenging boundaries, thinking expansively, and creating meaningful change is something that we strive to do in our work for the Duchenne Community every day.

To that end, we have been moving our drug development programs forward as quickly and safely as possible. Our first PPMO drug candidate for individuals amenable to exon 51 skipping is enrolling (NCT03375255), preparation of the golodirsen new drug application is underway with the aim of completing submission in late 2018, and our DMD gene therapy partnerships are advancing with multiple individuals dosed. Unfortunately, our plan to bring eteplirsen to patients in Europe has met a hurdle in the regulatory process. As stated in our last newsletter, drug development is not linear or highly predictable as evidenced by the negative CHMP opinion. Our thoughts are with the families impacted by this decision, and we are working with the regulatory bodies in Europe to determine a path forward.

In other news, we recently announced a new gene therapy collaboration focused on Limb Girdle Muscular Dystrophy (LGMD), marking our first venture outside Duchenne. The company is excited to learn more about LGMD and to support the progress made by Myonexus, our partner. Importantly, this expansion does not change our commitment to Duchenne. As Doug, our CEO, often says – we are a Duchenne-focused company, and this will not change.

For the first time, Sarepta will be hosting an R+D day for investor audiences on June 19, 2018. This is another platform to bring awareness to DMD and to highlight Sarepta’s approach for creating meaningful change in the lives of those impacted. The R+D day will be live-streamed, and we will share the link with you in advance.

Additionally, we would like to invite Advocacy leaders to join us for a town hall teleconference following R+D day. Your thoughts and questions are important to us as we shape the future as a company and further partner with the Duchenne community.

Thank you for your continued partnership!

US Clinical Trial Updates

PPMO (Peptide Conjugated Phosphorodiamidate Morpholino Oligomer) (SRP-5051)

• PPMO is an exon skipping technology designed to efficiently get inside cells, with the goal of producing more dystrophin in the muscle groups affected in DMD patients.
• Doug, our CEO, presented at a PPMD webinar on May 9th to provide additional education on the PPMO technology. You can find a link to the presentation on PPMD’s website, or via this link: https://bit.ly/2J8uJdz.
• Update: The details of the open-label extension study (5051-102) will be posted on clinicaltrials.gov soon, and will be open to all individuals who complete our currently enrolling Phase 1 (5051-101) study for males with DMD amenable to Exon 51 skipping. Please reach out to a site investigator for more information. Visit clinicaltrials.gov (NCT03375255) for more.

PMO (Phosphorodiamidate Morpholino Oligomer)

• ESSENCE: The global Phase 3 clinical trial is ongoing, examining two distinct drug candidates: golodirsen, for the potential treatment of individuals with DMD who are amenable to Exon 53 skipping; and casimersen, to potentially treat those amenable to Exon 45 skipping. Enrollment at US ESSENCE sites is closed. Visit clinicaltrials.gov (NCT02500381) to learn more.
• Preparation of the golodirsen New Drug Application (NDA) to the FDA is underway, with the aim of completing submission in late 2018.

Click here to download Sarepta’s full quarterly update.