Defeat Duchenne Canada is saddened to hear the news that PepGen has decided to end development of PGN-EDO51, a medicine which is being tested as a treatment for Duchenne muscular dystrophy (DMD). One of the most common causes of DMD is a change or a missing or extra part of the dystrophin gene, called exons. Some medicines, like PGN-EDO51, are medicines that help make to skip the part of the gene that does not work properly. PGN-ED051 is an exon skipping medicine that targets exon 51. This means that the CONNECT1-EDO51 trial, which has been dosing in Canada, and the CONNECT2-EDO51 Phase 2 study, which was previously active in the United Kingdom (UK) and already on hold in the United States (US), will stop.
PepGen has stated that the decision is based on not a significant enough amount of dystrophin being produced at the 10 mg/kg dose level in the CONNECT1-EDO51 trial to provide genuine benefit to people with DMD.
“We are disappointed by the dystrophin results observed in the 10 mg/kg dose cohort in CONNECT1, as it was our hope that we could improve upon existing therapies for patients in a more profound way.”
James McArthur, PhD, President and CEO of PepGen
Are you or your child participating in the CONNECT1 trial? Contact us to be personally updated when additional information is available at [email protected]
Read the full press release and the Community Letter from PepGen below.
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