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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 small molecule being developed for the treatment of Myotonic Dystrophy Type 1 (DM1). It is currently in Phase 2 clinical development and has not been approved by regulatory authorities. The drug is being studied in patients with DM1 to evaluate its safety and efficacy.
PGN-EDODM1 targets the DMPK CUG repeat expansion, which is the underlying genetic cause of Myotonic Dystrophy Type 1. By targeting this specific molecular abnormality, the drug aims to address the root cause of the disease rather than just managing symptoms.
PGN-EDODM1 is being developed by PepGen Inc., a biopharmaceutical company publicly traded under the ticker symbol PEPG. The company is conducting clinical trials to evaluate the drug's potential as a treatment for Myotonic Dystrophy Type 1.
PGN-EDODM1 is currently in Phase 2 clinical development. It is an investigational drug and has not received regulatory approval. The drug has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations by the FDA for the treatment of Myotonic Dystrophy Type 1.
PGN-EDODM1 is being evaluated in two Phase 2 clinical trials. NCT06667453 is a randomized, double-blind, placebo-controlled study enrolling 24 participants in Canada, New Zealand, South Korea, and the United Kingdom. NCT07220603 is an open-label extension study enrolling 48 participants in Canada. Both trials are currently recruiting.
PGN-EDODM1 is not the same as FREEDOM. FREEDOM refers to the clinical trial program for PGN-EDODM1. Specifically, NCT07220603 is titled 'An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)', indicating that FREEDOM is the name of the study, not an alternative name for the drug itself.