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PGN-EDODM1 for infusion

Phase 1

Myotonic Dystrophy 1 | Small molecule | Rare Disease |PepGen Inc.|Last Updated: Feb 13, 2026

Success Probability

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment24

FDA Designations

ORPHAN_DRUGFAST_TRACKRARE_PEDIATRIC_DISEASE

Clinical trial landscape

PGN-EDODM1 for infusion · 1 trial · 1 indication

Phase 1 1
NCT06204809Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1Myotonic Dystrophy 1
COMPLETED24 Analytics
PHASE1COMPLETED
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy 1
NCT0620480924
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Study Endpoints

Primary Endpoints

Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs
Baseline to Week 16

Secondary Endpoints

Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
Baseline up to Day 3
Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
Baseline up to Day 3
Apparent Terminal Half-Life (t½) of PGN-EDODM1
Baseline up to Day 3
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
PGN-EDODM1EXPERIMENTALPGN-EDODM1 for infusion
PlaceboPLACEBO_COMPARATOR0.9% NaCl

Interventions

NameTypeDescription
PGN-EDODM1 for infusionDRUGSingle dose of PGN-EDODM1 by intravenous (IV) infusion
PlaceboOTHERAdministered by IV infusion
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Eligibility Criteria

Age Range18 Years to 60 Years
SexALL
Healthy VolunteersNo
Study Sites12

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...

Countries:United StatesCanadaUnited Kingdom
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Frequently asked questions about PGN-EDODM1 for infusion

What is PGN-EDODM1 used for?

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 1 clinical development and has not been approved by the FDA. The drug is intended for adult patients aged 18 years and older.

Who is developing PGN-EDODM1?

PGN-EDODM1 is being developed by PepGen Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol PEPG. The company is conducting clinical trials for this drug in the United States, Canada, and the United Kingdom.

What phase is PGN-EDODM1 in?

PGN-EDODM1 is in Phase 1 clinical development. It is an investigational drug and has not received FDA approval. The drug has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations by the FDA for the treatment of Myotonic Dystrophy 1.

What clinical trials is PGN-EDODM1 in?

PGN-EDODM1 has one completed Phase 1 clinical trial, identified as NCT06204809. This study evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of PGN-EDODM1 in 24 participants with Myotonic Dystrophy Type 1. The trial was randomized, double-blind, and placebo-controlled.

Is PGN-EDODM1 FDA approved?

No, PGN-EDODM1 is not FDA approved. It is an investigational drug currently in Phase 1 clinical development. The FDA has granted it Orphan Drug, Fast Track, and Rare Pediatric Disease designations, which provide certain development incentives but do not indicate approval.