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NCT06079281 (efzimfotase alfa in hypophosphatasia): BVCT ex-ante prediction vs. reported outcome

On 11 Apr 2025, BioinvestGPT's BVCT predicted technical success and commercial success for this trial. The 11 Oct 2026 readout is classified on the dashboard as False Positive (FP), a missed prediction. Recorded clinical-benefit conclusion: statistically insignificant and commercially probably insufficient.

Clinical Trial

NCTID
NCT06079281
Acronym
HICKORY
Drug Name
efzimfotase alfa
Drug MoA
recombinant human alkaline phosphatase enzyme
Drug Modality
protein biologic
Drug Class
Best-In-Class
Therapeutic Area
Congenital; Hereditary; and Neonatal Diseases and Abnormalities
Indication
hypophosphatasia
Human Patients
124
Sponsor
AstraZeneca
Phase
Phase 3

Prediction of , index 1969

Prospective Prediction
Prediction Index
1969
Prediction Date
Commercial Prediction
SUCCESS (commercially sufficient clinical benefit in 6MWT and RSS moderately superior to asfotase alfa)
Technical Prediction
SUCCESS (statistically significant clinical benefit in 6MWT and RSS compared with placebo with slightly-superior-to-asfotase-alfa tolerability)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
548 days in advance
Readout Data Interpretation
Efzimfotase alfa did not meet primary endpoint of placebo-adjusted improvement in 6MWT (17.05 meters p=0.1675);well tolerated;
Press Release
Prediction Result
Prediction Accuracy
Missed Prediction
Due to
due to suboptimal drug modeling of the clinical impact of one of the three amino acid substitutions that have been permanently improved
Clinical Benefit Conclusion
statistically insignificant and commercially probably insufficient
Prediction Classification
False Positive (FP)

ClinicalTrials.gov record

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

Official title
A Phase 3, Randomized, Double-blinded, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of ALXN1850 (Recombinant Alkaline Phosphatase) Administered Subcutaneously in Adolescent (12 to < 18 Years of Age) and Adult Participants With Hypophosphatasia Who Have Not Previously Been Treated With Asfotase Alfa
Phase
Phase 3
Status
Active not recruiting
Enrollment
124
Lead sponsor
Alexion Pharmaceuticals, Inc.
Conditions
Hypophosphatasia
Interventions
DRUG: ALXN1850; DRUG: Placebo
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2024-01-03
Primary completion
2025-07-09
Completion
2028-03-29
Primary outcomes
  • Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169) (Baseline, Day 169)

NCT06079281 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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