Recent Updates
Recently added Catalysts

PGN-EDODM1

Phase 2

Myotonic Dystrophy 1 | Small molecule | Rare Disease |PepGen Inc.|Last Updated: Jul 29, 2026

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment72

FDA Designations

ORPHAN_DRUGFAST_TRACKRARE_PEDIATRIC_DISEASE

Clinical trial landscape

PGN-EDODM1 · 2 trials · 1 indication

Phase 2 2
NCT07220603An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)Myotonic Dystrophy 1
RECRUITING48 Analytics
NCT06667453A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1Myotonic Dystrophy 1
RECRUITING24 Analytics
PHASE2RECRUITING
An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
Myotonic Dystrophy 1Unlock trial analytics
PHASE2RECRUITING
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Myotonic Dystrophy 1Unlock trial analytics

Study Endpoints

Primary Endpoints

Safety and tolerability as assessed by number of participants with Adverse Events (AEs)
Baseline through Week 108

Secondary Endpoints

Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
Day 1 through Day 84
Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
Day 1 through Day 84
Apparent Terminal Half-Life (t½) of PGN-EDODM1
Day 1 through Day 84
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
PGN-EDODM1EXPERIMENTALParticipants will receive doses of PGN-EDODM1 once every 4 weeks (Q4W)
PlaceboPLACEBO_COMPARATORParticipants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks

Interventions

NameTypeDescription
PGN-EDODM1DRUGAdministered by intravenous (IV) infusion
PlaceboOTHERAdministered by intravenous (IV) infusion
Unlock Study Design Details

Eligibility Criteria

Age Range16 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites3

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...

Countries:CanadaNew ZealandSouth KoreaUnited Kingdom
Unlock Eligibility Criteria

Recent Changes (Last 90 Days)

LOWJul 29, 2026NCT06667453lastUpdatePostDate: changed
LOWJul 29, 2026NCT06667453lastUpdatePostDate: changed
LOWMay 26, 2026NCT07220603primaryCompletionDate: changed
MEDIUMMay 26, 2026NCT06667453primaryCompletionDate: changed
LOWMay 24, 2026NCT07220603studyFirstPostDate: changed
LOWMay 24, 2026NCT06667453studyFirstPostDate: changed

Frequently asked questions about PGN-EDODM1

What is PGN-EDODM1 used for in Myotonic Dystrophy 1?

PGN-EDODM1 is an investigational small molecule being developed for the treatment of Myotonic Dystrophy 1, a rare genetic disorder. It is currently in Phase 2 clinical development and has not been approved by the FDA. The drug is being studied in clinical trials to evaluate its safety and efficacy in people with this condition.

Who makes PGN-EDODM1?

PGN-EDODM1 is being developed by PepGen Inc., a biopharmaceutical company. The company is conducting clinical trials to evaluate the drug for the treatment of Myotonic Dystrophy 1. PepGen Inc. is publicly traded under the ticker symbol PEPG.

What phase is PGN-EDODM1 in?

PGN-EDODM1 is currently in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The drug has received FDA designations including Orphan Drug, Fast Track, and Rare Pediatric Disease, which are intended to facilitate its development and review.

What clinical trials is PGN-EDODM1 in?

PGN-EDODM1 is being studied in two Phase 2 clinical trials. The first trial, NCT06667453, is a randomized, double-blind, placebo-controlled study enrolling 24 participants with Myotonic Dystrophy 1 in Canada, New Zealand, South Korea, and the United Kingdom. The second trial, NCT07220603, is an open-label extension study enrolling 48 participants in Canada.

Is PGN-EDODM1 FDA approved?

No, PGN-EDODM1 is not FDA approved. It is an investigational drug currently in Phase 2 clinical trials for the treatment of Myotonic Dystrophy 1. The drug has received FDA designations including Orphan Drug, Fast Track, and Rare Pediatric Disease, but these designations do not indicate approval.