FDA Places Clinical Hold on PepGen’s Duchenne Muscular Dystrophy Trial

Clinical Hold:
The FDA has placed a clinical hold on PepGen's IND application to initiate the CONNECT2-EDO51 Phase 2 study of PGN-EDO51 for Duchenne muscular dystrophy5.

Reason for Hold:
The FDA will provide an official clinical hold letter detailing the reasons within 30 days5.

Impact:
This hold affects the planned Phase 2 study in the U.S. but does not impact ongoing studies in Canada, such as the CONNECT1-EDO51 study45.

Previous Holds:
PepGen previously faced a clinical hold on its Phase 1 trial for PGN-EDODM1, an experimental treatment for myotonic dystrophy type 1 (DM1), in 202314.

Company Response:
PepGen will work closely with the FDA to lift the hold as quickly as possible and remains committed to the development of PGN-EDO515.

Sources:

1. https://www.biopharmadive.com/news/pepgen-fda-clinical-hold-myotonic-dystrophy/651628/

4. https://musculardystrophynews.com/news/phase-1-trial-pepgen-pgn-edodm1-dm1-delayed-fda-clinical-hold/

5. https://www.morningstar.com/news/business-wire/20241216162092/pepgen-announces-clinical-hold-in-the-us-on-ind-application-to-initiate-connect2-edo51-phase-2-study-of-pgn-edo51-for-duchenne-muscular-dystrophy