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NCT05337553 (Taldefgrobep alfa in Spinal Muscular Atrophy): BVCT ex-ante prediction vs. reported outcome

On 15 May 2024, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 25 Nov 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically insignificant and commercially insufficient.

Clinical Trial

NCTID
NCT05337553
Acronym
RESILIENT
Drug Name
Taldefgrobep alfa
Drug MoA
selective ACVR2B antagonist
Drug Modality
recombinant protein biologic
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Spinal Muscular Atrophy (SMA)
Human Patients
269
Sponsor
Biohaven
Phase
Phase 3

Prediction of , index 1621

Prospective Prediction
Prediction Index
1621
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient additive clinical benefit in MFM32)
Technical Prediction
partial SUCCESS (statistically maybe significant yet very weak clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
194 days in advance
Readout Data Interpretation
taldefgrobep alfa + SoC failed to achieved the MFM32 primary efficacy endpoint at week 48 compared with SoC alone
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically insignificant and commercially insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension
Phase
Phase 3
Status
Active not recruiting
Enrollment
269
Lead sponsor
Biohaven Pharmaceuticals, Inc.
Conditions
Spinal Muscular Atrophy; Neuromuscular Diseases; SMA
Interventions
DRUG: taldefgrobep alfa; DRUG: Placebo; DRUG: taldefgrobep alfa
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2022-07-06
Primary completion
2024-09-25
Completion
2027-12
Primary outcomes
  • Efficacy of taldefgrobep alfa compared to placebo in change in the 32 item Motor Function Measure (MFM-32) total score (Baseline to Week 48)

NCT05337553 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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