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NCT06667453 (PGN-EDODM1 in Myotonic Dystrophy 1): BVCT ex-ante prediction vs. reported outcome

On 14 Nov 2025, BioinvestGPT's BVCT predicted technical success and commercial partial success for this trial. The 30 Mar 2026 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially TBD.

Clinical Trial

NCTID
NCT06667453
Acronym
FREEDOM2-DM1
Drug Name
PGN-EDODM1
Drug MoA
EDO cell-penetrating peptide conjugated with DMPK-CUG-repeats-specific oligonucleotide
Drug Modality
oligonucleotide
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Myotonic Dystrophy 1
Human Patients
24
Sponsor
PepGen
Phase
Phase 2

Prediction of , index 2089

Prospective Prediction
Prediction Index
2089
Prediction Date
Commercial Prediction
partial SUCCESS (commercially maybe sufficient yet weak clinical benefit with superior-to-AOC1001 efficacy in terms of vHOT and muscle strength despite non-superior-to-AOC1001 toxicity)
Technical Prediction
SUCCESS (statistically significant clinical benefit in spliceopathy)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
136 days in advance
Readout Data Interpretation
PGN-EDODM1 5 mg/kg lowest-dose MAD cohort (n=6) demonstrated 7.3% mean splicing correction vs placebo (22.9% excluding outlier); positive trend in vHOT; favorable safety profile in FREEDOM2 study
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially TBD
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

ClinicalTrials.gov record, retrieved 2026-09-24. Third-party data.

Official title
A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
Phase
Phase 2
Status
Recruiting
Enrollment
24
Lead sponsor
PepGen Inc
Conditions
Myotonic Dystrophy 1
Interventions
DRUG: PGN-EDODM1; OTHER: Placebo
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2024-12-10
Primary completion
2027-03
Completion
2027-03
Primary outcomes
  • Safety and tolerability as assessed by number of participants with Adverse Events (AEs) (Baseline through Day 112)

NCT06667453 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

How to read these records: evidence · methodology · all trials