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NCT04601051 (NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With…): BVCT ex-ante prediction vs. reported outcome

On 23 Jul 2023, BioinvestGPT's BVCT predicted technical partial failure and commercial failure for this trial. The 27 Oct 2025 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically insignificant and commercially probably insufficient.

Clinical Trial

NCTID
NCT04601051
Drug Name
NTLA-2001 (nex-z)
Drug MoA
NP-delivered CRISPR-Cas9 reducing TTR production
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
Human Patients
72
Sponsor
Intellia Therapeutics
Phase
Phase 1

Prediction of , index 1116

Prospective Prediction
Prediction Index
1116
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit)
Technical Prediction
partial FAILURE (statistically insignificant clinical benefit in 6MWT for cardiomyopathy or in 10MWT for polyneuropathy)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
827 days in advance
Readout Data Interpretation
nex-z phase 3 clinical trials in ATTR-CM (MAGNITUDE) and ATTR-PN (MAGNITUDE-2) have been paused due to grade 4 hepatotoxicity in a patient
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically insignificant and commercially probably insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
Phase
Phase 1
Status
Completed
Enrollment
72
Lead sponsor
Intellia Therapeutics
Conditions
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy; Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy; Wild-Type Transthyretin Cardiac Amyloidosis
Interventions
BIOLOGICAL: NTLA-2001
Design
Non randomized · Sequential · None
Primary purpose
Treatment
Start
2020-11-05
Primary completion
2025-09-12
Completion
2025-09-12
Primary outcomes
  • Number of Participants with Treatment-Emergent Adverse Events (up to Day 730)
  • Number of Participants with Clinically Significant Clinical Laboratory Test Findings (up to Day 730)
  • Number of Participants with Clinically Significant Safety Measurements (up to Day 730)
  • Percent Change from Baseline in Serum TTR (enzyme-linked immunosorbent assay [ELISA]) (up to Day 730)
  • Percent Change from Baseline in Serum Prealbumin (up to Day 730)

NCT04601051 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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