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NCT05456880 (BEAM-101 in Sickle Cell Disease): BVCT ex-ante prediction vs. reported outcome

On 20 Jan 2024, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 5 Nov 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically significant yet overall weak due to better efficacy and worse safety and commercially TBD.

Clinical Trial

NCTID
NCT05456880
Acronym
BEACON
Drug Name
BEAM-101
Drug MoA
autologous CD34+ A-to-G HBG1/HBG2 promoter base edited hematopoietic stem cells
Drug Modality
base editing gene therapy
Drug Class
First-In-Class
Therapeutic Area
Hemic and Lymphatic Diseases
Indication
Sickle Cell Disease
Human Patients
15
Sponsor
Beam Therapeutics
Phase
Phase 1 Phase 2

Prediction of , index 1434

Prospective Prediction
Prediction Index
1434
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit inferior to Casgevy)
Technical Prediction
partial SUCCESS (statistically maybe significant yet weak clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
290 days in advance
Readout Data Interpretation
BEAM101 achieved ~66% and ~11.8 g/dL HbF response at month 6; which are superior to Casgevy's 45% and 5.8 g/dL HbF response at month 6 (10.1056/NEJMoa2309676); yet a patient died from the necessary busulfan conditioning for BEAM101; inferior to Casgevy's safety; VOC data are premature
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant yet overall weak due to better efficacy and worse safety and commercially TBD
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients With Sickle Cell Disease and Severe Vaso-Occlusive Crises (Beacon Trial)
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
15
Lead sponsor
Beam Therapeutics Inc.
Conditions
Sickle Cell Disease
Interventions
BIOLOGICAL: BEAM-101
Design
Single group · None
Primary purpose
Treatment
Start
2022-08-30
Primary completion
2028-02
Completion
2028-02
Primary outcomes
  • Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline (6 months to time of analysis as compared to baseline)
  • Proportion of patients with successful neutrophil engraftment (BEAM-101 administration to month 24)
  • Time to neutrophil engraftment (BEAM-101 administration to month 24)
  • Time to platelet engraftment (BEAM-101 administration to month 24)
  • Transplant-related mortality within 100 days after beam-101 treatment (BEAM-101 administration to day 100)

NCT05456880 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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