We are sharing an important update with our community regarding PepGen Inc.’s investigational therapy, PGN-EDO51, for Duchenne muscular dystrophy (DMD). We have received news that PepGen has made the decision to end the development of the PGN-EDO51 programme.
This means that the CONNECT2-EDO51 study, which was previously open for recruitment in the UK and proposed for other countries (and did not dose or enrol any participants) and will now be closed. Furthermore, the CONNECT1-EDO51 trial, which had been dosing participants in Canada, will now cease dosing.
This news follows on from our update on the 12th of March regarding the companies decision to pause the CONNECT2-EDO51 study.
PepGen has communicated that, despite their goal to make a meaningful difference for people with DMD, their analysis of the data from the CONNECT1-EDO51 trial indicates that the amount of dystrophin produced by participants in response to treatment was not sufficient to provide a meaningful clinical benefit. They do not believe it is possible to dose the therapy at a level that would achieve the genuine benefit required.
We understand that this news is deeply disappointing, particularly for families who participated in the trials, were considering joining, or have followed this programme with hope. Participation in any clinical trial represents an immense commitment from individuals and their families, and we recognise the significant time and effort that has been invested.
PepGen has stated that this decision was made based on current data, to avoid further exposing individuals to a therapy unlikely to provide an acceptable benefit/risk profile in the long term. Their clinical study sites are now working directly with participants and families to support them through the wind-down process. Action Duchenne appreciates PepGen’s transparency in sharing this challenging news with the wider community.
We remain steadfast in our commitment to supporting individuals and families living with Duchenne. If you have any questions or concerns regarding this news, please do reach out to us at info@actionduchenne.org.
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