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NCT06204809 (PGN-EDODM1 in Myotonic Dystrophy Type 1): BVCT ex-ante prediction vs. reported outcome

On 24 Apr 2024, BioinvestGPT's BVCT predicted technical success and commercial partial success for this trial. The 24 Feb 2025 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically significant (splicing correction) and commercially maybe sufficient yet weak (functional outcome).

Clinical Trial

NCTID
NCT06204809
Acronym
FREEDOM-DM1
Drug Name
PGN-EDODM1
Drug MoA
anti-DMPK-CUG-repeats-specific oligonucleotide
Drug Modality
peptide-conjugated antisense oligonucleotide ASO
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Myotonic Dystrophy Type 1
Human Patients
32
Sponsor
PepGen
Phase
Phase 1

Prediction of , index 1558

Prospective Prediction
Prediction Index
1558
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
306 days in advance
Readout Data Interpretation
PGN-EDODM1 achieved 29.1% mean splicing correction without improved functional outcomes at day 28 (trends only)
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant (splicing correction) and commercially maybe sufficient yet weak (functional outcome)
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)
Phase
Phase 1
Status
Completed
Enrollment
24
Lead sponsor
PepGen Inc
Conditions
Myotonic Dystrophy 1
Interventions
DRUG: PGN-EDODM1 for infusion; OTHER: Placebo
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2023-12-12
Primary completion
2025-10-28
Completion
2025-10-28
Primary outcomes
  • Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs (Baseline to Week 16)

NCT06204809 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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