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NCT05670730 (AOC1044 in Duchenne Muscular Dystrophy): BVCT ex-ante prediction vs. reported outcome

On 7 Jul 2024, BioinvestGPT's BVCT predicted technical success and commercial failure for this trial. The 17 Mar 2025 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant (dystrophin) and commercially TBD (6MWD).

Clinical Trial

NCTID
NCT05670730
Acronym
EXPLORE44
Drug Name
AOC1044 (del-zota)
Drug MoA
antibody–oligonucleotide conjugate that binds the transferrin receptor 1 (TfR1) to deliver a PMO into muscle cells; inducing exon 44 skipping and restoring functional dystrophin synthesis
Drug Modality
antibody-conjugated antisense oligonucleotide ASO
Drug Class
Best-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Duchenne Muscular Dystrophy (DMD)
Human Patients
70
Sponsor
Avidity Biosciences
Phase
Phase 1 Phase 2

Prediction of , index 1000

Prospective Prediction
Prediction Index
1000
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in the registrational endpoint 6MWD non-superior and non-inferior to DYNE251 or SRP5051)
Technical Prediction
SUCCESS (statistically significant clinical benefit in dystrophin protein level)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
253 days in advance
Readout Data Interpretation
del-zota achieved a statistically significant increase of 25% dystrophin production and 40% in exon 44 skipping; 6MWD data immature
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant (dystrophin) and commercially TBD (6MWD)
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants With DMD Mutations Amenable to Exon 44 Skipping
Phase
Phase 1, Phase 2
Status
Completed
Enrollment
70
Lead sponsor
Avidity Biosciences, Inc.
Conditions
Duchenne Muscular Dystrophy; Exon 44
Interventions
DRUG: AOC 1044; DRUG: Placebo
Design
Randomized · Sequential · Quadruple
Primary purpose
Treatment
Start
2022-11-09
Primary completion
2024-11-25
Completion
2024-11-25
Primary outcomes
  • Incidence of treatment-emergent adverse events (TEAEs) (Through study completion, up to Day 85 (Part A) or Day 169 (Part B))

NCT05670730 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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