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NCT05120830 (NTLA-2002 in Hereditary Angioedema): BVCT ex-ante prediction vs. reported outcome

On 3 Mar 2024, BioinvestGPT's BVCT predicted technical success and commercial success for this trial. 2 readouts followed: 2 Jun 2024, True Positive (TP); 27 Apr 2026, True Positive (TP).

Clinical Trial

NCTID
NCT05120830
Acronym
NTLA-2002
Drug Name
NTLA-2002
Drug MoA
LNP-delivered CRISPR/CAS9-based anti-KLKB1
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Immune System Diseases
Indication
Hereditary Angioedema
Human Patients
37
Sponsor
Intellia Therapeutics
Phase
Phase 1 Phase 2

Prediction of , index 1494

Prospective Prediction
Prediction Index
1494
Prediction Date
Commercial Prediction
SUCCESS (commercially sufficient clinical benefit)
Technical Prediction
SUCCESS (statistically significant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
91 days in advance
Readout Data Interpretation
-98% mean monthly HAE attacks vs -85% by PHVS’s deucrictibant.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially sufficient
Prediction Classification
True Positive (TP)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
785 days in advance
Readout Data Interpretation
lonvo-z (NTLA-2002) achieved 87% reduction in HAE attacks vs placebo in Phase 3 HAELO over 6 months (p<0.0001); favorable safety with all AEs mild or moderate; BLA filing planned 2H 2026.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially sufficient (correct efficacy prediction when the asset was still in phase 1/2 stage)
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
37
Lead sponsor
Intellia Therapeutics
Conditions
Hereditary Angioedema
Interventions
BIOLOGICAL: Biological NTLA-2002; OTHER: Normal Saline IV Administration
Design
Randomized · Sequential · Double
Primary purpose
Treatment
Start
2021-12-10
Primary completion
2024-04-04
Completion
2026-07
Primary outcomes
  • Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs) (From NTLA-2002 infusion up to week 104 post-infusion)
  • Number of HAE attacks per month (Weeks 1-16) (From study drug infusion up to week 16 post-infusion)

NCT05120830 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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