We are disappointed to share that PepGen Inc. announced the company’s decision to end development of PGN-EDO51, its investigational therapy for the treatment of people with Duchenne amenable to exon 51 skipping. PepGen indicated that the CONNECT1-EDO51 trial will stop dosing, and the CONNECT2-EDO51 did not dose any participants and will now be closed. Clinical study sites are working closely with study participants and families to support them through the study wind down.
According to PepGen, the company does not believe that it can dose at a high enough level to produce an amount of dystrophin to provide meaningful benefit to people with Duchenne. Based on current data, PepGen feels it is important not to further expose people to a therapy that is unlikely to provide an acceptable benefit/risk profile in the long term.
PPMD is in touch with PepGen and will share any additional information possible with the community. We are grateful to all the families who have participated in clinical trials with PGN-EDO51, and to the PepGen team for their work to advance therapy development in Duchenne. The discontinuation of a program is always difficult for our community, but we remain hopeful that the data from this trial will inform other potential therapeutic options and that these experiences will lead us to the day that we end Duchenne.
Read PepGen’s community letter:
May 28 2025
Dear Duchenne Community:
We are writing to share the news that we have decided to end development of PGN-EDO51, our investigational therapy to treat people with Duchenne muscular dystrophy amenable to exon 51 skipping. This means that the CONNECT1-EDO51 trial, which has been dosing in Canada, will stop dosing. CONNECT2-EDO51 (proposed for other countries and previously open in the UK) did not dose any participants and will not enroll any participants. This study will now be closed
At PepGen, our goal has always been to change the lives of people with DMD. Unfortunately, we believe that the amount of dystrophin produced by people in the trial is not enough to provide meaningful benefit to people with DMD. We do not believe we can dose at a high enough level to produce an amount of dystrophin that would provide genuine benefit to people with DMD.
We are deeply disappointed by this result, but we feel it is important to make this decision based on current data, and not to further expose people to a therapy that is unlikely to provide an acceptable benefit/risk profile in the long term. Our clinical study sites are working closely with study participants and families to support them through the study wind down.
We understand that this may be very disappointing for you, especially if you were a part of our DMD trials, were considering joining our DMD trials, or are one of the many community members who offered us advice on this program. We know that participation in any clinical trial is a huge commitment on the part of the participants and their families, and we appreciate the time and effort that have been put into helping us find out if this investigational therapy might work. We recognize that the discontinuation of any clinical program may feel like a loss, but please remember each study completed contributes to our understanding of DMD. There are now more therapies in development for DMD than at any point in past history, and many are looking very promising. Please keep in touch with your doctors and local advocacy groups to learn more about other therapies in development.
Most importantly, we want to say THANK YOU for your willingness to be involved in the development of PGN-EDO51 as a potential treatment for DMD. Our commitment to neuromuscular diseases is not diminished by this setback.
With the sincere regards of the entire PepGen team,
Paul Streck, MD
Head of Research & Development, PepGen
Jane Larkindale, DPhil
VP Clinical Science and Head of Advocacy
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