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PGN-EDODM1 for infusion

Phase 1

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06204809Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1PHASE1 COMPLETED 24 — — Dec 12, 2023Oct 28, 2025Feb 13, 202612 United States, Canada +1
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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 — 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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