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PGN-EDODM1 for infusion · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| PGN-EDODM1 | EXPERIMENTAL | PGN-EDODM1 for infusion |
| Placebo | PLACEBO_COMPARATOR | 0.9% NaCl |
| Name | Type | Description |
|---|---|---|
| PGN-EDODM1 for infusion | DRUG | Single dose of PGN-EDODM1 by intravenous (IV) infusion |
| Placebo | OTHER | Administered by IV infusion |
Inclusion Criteria: * Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats * Medical Research Council (MRC) score of ≥ Grade 4- in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against...
PGN-EDODM1 is an investigational therapy being developed for the treatment of myotonic dystrophy type 1, a rare genetic disorder. It is administered by infusion. The program is in Phase 1 clinical development, and the drug has not been approved for any indication.
PGN-EDODM1 is being developed by PepGen Inc., which trades under the ticker symbol PEPG. PepGen is the sponsor of the clinical program evaluating the drug in myotonic dystrophy type 1.
PGN-EDODM1 is in Phase 1 clinical development. Its single Phase 1 trial, NCT06204809, has been completed. The drug is investigational and has not received FDA approval for any indication.
PGN-EDODM1 has been studied in one Phase 1 trial, NCT06204809, titled a Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1. The trial enrolled 24 participants across the United States, Canada, and the United Kingdom and is now completed.
PGN-EDODM1 has received two FDA designations: Orphan Drug and Fast Track. These designations relate to its development for myotonic dystrophy type 1. The drug remains investigational and has not been approved by the FDA.
The Phase 1 trial NCT06204809 was a randomized, double-blind, placebo-controlled study of PGN-EDODM1 in adults with myotonic dystrophy type 1. It enrolled 24 participants aged 18 years and older, across the United States, Canada, and the United Kingdom, and evaluated safety, tolerability, pharmacokinetics, and pharmacodynamics.