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NCT04906460 (WVE-N531 in Duchenne Muscular Dystrophy): BVCT ex-ante prediction vs. reported outcome

On 12 May 2024, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 24 Sep 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically partially significant (dystrophin expression) and partially TBD (functional endpoints) and commercially TBD.

Clinical Trial

NCTID
NCT04906460
Acronym
FORWARD-53
Drug Name
WVE-N531
Drug MoA
stereopure exon 53 skipping antisense oligonucleotides
Drug Modality
siRNA
Drug Class
Best-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Duchenne Muscular Dystrophy (DMD)
Human Patients
11
Sponsor
Wave Life Sciences
Phase
Phase 1 Phase 2

Prediction of , index 1595

Prospective Prediction
Prediction Index
1595
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in 6MWD non-superior and non-inferior to DYNE251 or SRP5051)
Technical Prediction
partial SUCCESS (statistically significant clinical benefit in dystrophin protein level) partial FAILURE (statistically insignificant clinical benefit in NSAA/10MRW/SV95C)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
135 days in advance
Readout Data Interpretation
a mean absolute dystrophin expression of 5.5%; which is relatively higher than competitor drugs.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically partially significant (dystrophin expression) and partially TBD (functional endpoints) and commercially TBD
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
An Open-label Phase 1b/2 Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Phase
Phase 1, Phase 2
Status
Recruiting
Enrollment
26
Lead sponsor
Wave Life Sciences USA, Inc.
Conditions
Duchenne Muscular Dystrophy
Interventions
DRUG: WVE-N531
Design
Single group · None
Primary purpose
Treatment
Start
2021-09-28
Primary completion
2026-06-27
Completion
2027-04-24
Primary outcomes
  • Part A: Safety: Proportion of patients with adverse events (AEs) (Day 1 (initial dose) up to 24 weeks after the last dose of Part A)
  • Part B: Pharmacodynamics: Dystrophin level (% normal dystrophin) as assessed by Western blot of muscle tissue following multiple doses of WVE-N531 (At Week 26 and at Week 50 of Part B)
  • Part C: Pharmacodynamics: Change from baseline dystrophin level (% normal dystrophin) as assessed by a validated assay analysis in muscle tissue following multiple doses of WVE-N531 (At Baseline and following 24 weeks of treatment in Part C)

NCT04906460 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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