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PGN-EDODM1 · 2 trials · 1 indication
| Arm | Type | Description |
|---|---|---|
| PGN-EDODM1 | EXPERIMENTAL | Participants will receive doses of PGN-EDODM1 once every 4 weeks (Q4W) |
| Placebo | PLACEBO_COMPARATOR | Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks |
| Name | Type | Description |
|---|---|---|
| PGN-EDODM1 | DRUG | Administered by intravenous (IV) infusion |
| Placebo | OTHER | Administered by intravenous (IV) infusion |
Inclusion Criteria: * Participant has completed a prior study with PGN-EDODM1 Exclusion Criteria: * Abnormal laboratory tests at screening considered clinically significant by the Investigator * Use of an investigational drug (other than PGN-EDODM1), device, or product, within 30 days or 5 half-l...
PGN-EDODM1 is an investigational therapy being developed for the treatment of myotonic dystrophy type 1, a rare genetic disorder. It is in Phase 2 clinical development and has not been approved by the FDA. The drug has received Orphan Drug and Fast Track designations from the FDA for this indication.
PGN-EDODM1 targets the DMPK CUG repeat expansion, the toxic RNA repeat that underlies myotonic dystrophy type 1. By aiming at this expanded repeat, the therapy is designed to address the root genetic cause of the disease rather than just its symptoms. It is a small molecule therapeutic.
PGN-EDODM1 is being developed by PepGen Inc., which trades on the Nasdaq under the ticker symbol PEPG. PepGen is a clinical-stage biotechnology company focused on rare disease therapies. The company is running two active Phase 2 trials of PGN-EDODM1 in myotonic dystrophy type 1.
PGN-EDODM1 is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. Two Phase 2 trials are currently recruiting participants, and the program has received both Orphan Drug and Fast Track designations from the FDA.
PGN-EDODM1 is being studied in two recruiting Phase 2 trials: NCT06667453, a placebo-controlled study enrolling 24 participants across Canada, New Zealand, South Korea, and the United Kingdom, and NCT07220603, the FREEDOM-OLE open-label extension study enrolling 48 participants in Canada. Together these trials plan to enroll 72 participants.
No, PGN-EDODM1 is not approved by the FDA. It is an investigational drug still in Phase 2 clinical development for myotonic dystrophy type 1. The FDA has granted it Orphan Drug and Fast Track designations, which are intended to support and expedite the development of therapies for serious conditions.