PepGen announced this morning that the United States FDA has put a “clinical hold” (which means a trial cannot be done now) on the Phase 2 clinical trial of the medicine called 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.
PGN-EDO51 is being tested in a Phase 2 clinical trial in Canada called CONNECT1. In the CONNECT1 trial, the safety and effectiveness of multiple ascending doses (MAD) of PGN-EDO51 are being tested, starting with 5mg/kg. To date, all people in the 5mg/kg dose group have been treated and those who have been or will be treated with a higher 10mg/kg dose have been enrolled. Some have already been dosed and are now in the long-term study of the treatment. To date, adverse events (or side effects) in the CONNECT1 trial of PGN-EDO51 have been mild.
The CONNECT2 trial in the UK right now has the same two, first cohort dosing as CONNECT1. The clinical hold of the CONNECT2 trial in the US does not impact the CONNECT1 trial in Canada, nor the ongoing enrollment of CONNECT2 in the United Kingdom. It only impacts starting the CONNECT2 trial in the US.
There are many reasons why the FDA may call for a clinical hold in general. Some reasons for a clinical hold can be that there is too much risk of illness or injury, or materials needed for the trial are not complete or do not include enough information to assess the risks, and many other reasons. Currently, it has not been said why the FDA has decided the PGN-EDO51 trial cannot begin in the US.
PepGen has informed us that they are waiting for more information from the FDA and will work closely with the agency to resolve the hold on the clinical trial in the US.
Are you or your child participating in the CONNECT1 trial? Contact us to be personally updated when additional information is available at [email protected]
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