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NCT05030831 (INZ-701 in ABCC6 Deficiency Causing Pseudoxanthoma Elasticum): BVCT ex-ante prediction vs. reported outcome

On 16 Jan 2024, BioinvestGPT's BVCT predicted technical success and commercial success for this trial. The 8 Apr 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant (phase 1/2) and commercially TBD.

Clinical Trial

NCTID
NCT05030831
Drug Name
INZ-701
Drug MoA
soluble recombinant ENPP1 enzyme
Drug Modality
fusion protein biologic
Drug Class
First-In-Class
Therapeutic Area
Cardiovascular Diseases
Indication
ABCC6 Deficiency Causing Pseudoxanthoma Elasticum (PXE)
Human Patients
10
Sponsor
Inozyme Pharma
Phase
Phase 1 Phase 2

Prediction of , index 870

Prospective Prediction
Prediction Index
870
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
83 days in advance
Readout Data Interpretation
safe with clinical improvement in multiple exploratory efficacy endpoints.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant (phase 1/2) and commercially TBD
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults With ABCC6 Deficiency Manifesting as Pseudoxanthoma Elasticum (PXE)
Phase
Phase 1, Phase 2
Status
Completed
Enrollment
10
Lead sponsor
Inozyme Pharma
Conditions
ATP-Binding Cassette Subfamily C Member 6 Deficiency; Pseudoxanthoma Elasticum; Generalized Arterial Calcification of Infancy
Interventions
DRUG: INZ-701
Design
Single group · None
Primary purpose
Treatment
Start
2022-04-11
Primary completion
2024-07-09
Completion
2024-07-09
Primary outcomes
  • Number of Treatment Emergent Adverse Events (TEAEs) (32 days (Dose Evaluation Period))
  • Number of Treatment Emergent Adverse Events (TEAEs) (52 weeks (Day 1 through Safety Follow-up Visit))

NCT05030831 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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