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NCT05027269 (AOC 1001 in Myotonic Dystrophy Type 1): BVCT ex-ante prediction vs. reported outcome

On 22 Apr 2023, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 8 Sep 2026 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically insignificant (in trial) and commercially insufficient.

Clinical Trial

NCTID
NCT05027269
Acronym
MARINA
Drug Name
AOC 1001
Drug MoA
anti-DMPK siRNA
Drug Modality
small interfering RNA (siRNA) antisense oligonucleotide
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Myotonic Dystrophy Type 1 (DM1)
Human Patients
38
Sponsor
Avidity Biosciences
Phase
Phase 1 Phase 2

Prediction of , index 999

Prospective Prediction
Prediction Index
999
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit)
Technical Prediction
FAILURE (statistically insignificant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
1235 days in advance
Readout Data Interpretation
Del-desiran did not meet its primary endpoint of vHOT in phase 3 HARBOR trial
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically insignificant (in trial) and commercially insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
Phase
Phase 1, Phase 2
Status
Completed
Enrollment
39
Lead sponsor
Avidity Biosciences, Inc.
Conditions
DM1; Myotonic Dystrophy 1; Myotonic Dystrophy; Myotonic Dystrophy Type 1 (DM1); Dystrophy Myotonic; Myotonic Disorders; Steinert Disease; Myotonic Muscular Dystrophy
Interventions
DRUG: AOC 1001; DRUG: Placebo
Design
Randomized · Sequential · Quadruple
Primary purpose
Treatment
Start
2021-10-28
Primary completion
2023-02-14
Completion
2023-02-14
Primary outcomes
  • Frequency of treatment emergent adverse events (TEAEs) (Through study completion, up to Day 183)

NCT05027269 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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