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NCT06079736 (PGN-EDO51 in Duchenne Muscular Dystrophy): BVCT ex-ante prediction vs. reported outcome

On 23 Apr 2024, BioinvestGPT's BVCT predicted technical success and commercial failure for this trial. The 30 Jul 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially TBD (pending functional efficacy data).

Clinical Trial

NCTID
NCT06079736
Acronym
CONNECT1-EDO51
Drug Name
PGN-EDO51
Drug MoA
EDO cell-penetrating peptide conjugated with dystrophin exon 51 skipping oligonucleotide
Drug Modality
peptide-conjugated antisense oligonucleotide ASO
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Duchenne Muscular Dystrophy (DMD)
Human Patients
10
Sponsor
PepGen
Phase
Phase 2

Prediction of , index 1557

Prospective Prediction
Prediction Index
1557
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in the registrational endpoint 6MWD non-superior-to and non-inferior-to SRP-5051)
Technical Prediction
SUCCESS (statistically significant clinical benefit in dystrophin protein level)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
98 days in advance
Readout Data Interpretation
5mg low-dose exon skipping level and dystrophin level comparable to SRP5051 20mg (NCT04004065).
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially TBD (pending functional efficacy data)
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)
Phase
Phase 2
Status
Terminated
Enrollment
7
Lead sponsor
PepGen Inc
Conditions
Duchenne Muscular Dystrophy
Interventions
DRUG: PGN-EDO51
Design
Non randomized · Sequential · None
Primary purpose
Treatment
Start
2024-01-03
Primary completion
2025-08-28
Completion
2025-08-28
Primary outcomes
  • Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period) (Baseline to Week 16)
  • Adverse events and serious adverse events (long-term safety and tolerability of PGN-EDO51 in LTE period) (Baseline to Week 108)

NCT06079736 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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