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NCT04849910 (VOR33 in CD33+ acute myeloid leukemia or myelodysplastic…): BVCT ex-ante prediction vs. reported outcome

On 7 Nov 2023, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 8 May 2025 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically probably insignificant and commercially probably insufficient.

Clinical Trial

Drug Name
VOR33
Drug MoA
allogeneic CRISPR/CAS9 genome-edited anti-CD33 HSPC cell therapy VOR33 + anti-CD33 ADC mylotarg
Drug Modality
cell therapy
Drug Class
Best-In-Class
Therapeutic Area
Neoplasms
Indication
CD33+ acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS)
Human Patients
67
Sponsor
Vor Biopharma
Phase
Phase 1 Phase 2

Prediction of , index 1316

Prospective Prediction
Prediction Index
1316
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient additive clinical benefit)
Technical Prediction
partial SUCCESS (statistically maybe significant yet weak additive clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
548 days in advance
Readout Data Interpretation
VOR33 + mylotarg’s further development has been discontinued
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically probably 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
A First-In-Human, Open-Label, Multicenter Study of VOR33 in Patients With Acute Myeloid Leukemia Who Are at High-Risk for Leukemia Relapse Following Hematopoietic Cell Transplantation
Phase
Phase 1, Phase 2
Status
Terminated
Enrollment
67
Lead sponsor
Vor Biopharma
Conditions
Leukemia, Myeloid, Acute; Myelodysplastic Syndromes
Interventions
BIOLOGICAL: VOR33; DRUG: Mylotarg
Design
Randomized · Sequential · None
Primary purpose
Treatment
Start
2021-12-16
Primary completion
2025-05-28
Completion
2025-05-28
Primary outcomes
  • Incidence of neutrophil engraftment (Day 28)

NCT04849910 on ClinicalTrials.gov

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