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NCT05156320 (Apitegromab in Spinal Muscular Atrophy): BVCT ex-ante prediction vs. reported outcome

On 6 Aug 2024, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 7 Oct 2024 readout is classified on the dashboard as True Negative (TN). Recorded clinical-benefit conclusion: statistically significant (yet weak benefit with negative dose-response relationship) and commercially insufficient (1.8 point is below the 3-point minimal threshold of clinically meaningful improvement in HFMSE score doi.org/10.1007/s40120-024-00653-2).

Clinical Trial

NCTID
NCT05156320
Acronym
SAPPHIRE
Drug Name
Apitegromab
Drug MoA
latent myostatin mAb inhibitor
Drug Modality
monoclonal antibody
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Spinal Muscular Atrophy (SMA)
Human Patients
188
Sponsor
Scholar Rock Holding
Phase
Phase 3

Prediction of , index 528

Prospective Prediction
Prediction Index
528
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient additive clinical benefit in HFMSE total score non-superior to nusinersen/risdiplam monotherapy)
Technical Prediction
FAILURE (statistically maybe significant yet very weak additive clinical benefit in HFMSE total score)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
62 days in advance
Readout Data Interpretation
apitegromab achieved 1.8 point HFMSE score difference at week 52 for the combined 10mg/kg and 20mg/kg populations (p=0.0192) yet only 1.4 point HFMSE score difference at week 52 for the 20mg/kg population (p=0.1149).
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant (yet weak benefit with negative dose-response relationship) and commercially insufficient (1.8 point is below the 3-point minimal threshold of clinically meaningful improvement in HFMSE score doi.org/10.1007/s40120-024-00653-2)
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy
Phase
Phase 3
Status
Completed
Enrollment
188
Lead sponsor
Scholar Rock, Inc.
Conditions
Spinal Muscular Atrophy; Spinal Muscular Atrophy Type 3; Spinal Muscular Atrophy Type 2; SMA; Neuromuscular Diseases; Muscular Atrophy; Atrophy; Muscular Atrophy, Spinal; Neuromuscular Manifestations; Anti-myostatin
Interventions
DRUG: Apitegromab; DRUG: Placebo
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2022-04-14
Primary completion
2024-12-18
Completion
2024-12-18
Primary outcomes
  • Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score. (Baseline up to 12 months.)

NCT05156320 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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