Tofersen in SOD1 ALS (VALOR)

Phase 3 Completed

SOD1-mutant ALS

NCT ID NCT02623699 ↗

Phase Phase 3

Status Completed

Sponsor Biogen

Enrolment n = 108

Last updated 2026-04-30

Registered 2026-04-30

Target intervention

Tofersen (antisense oligonucleotide targeting SOD1)

Primary endpoint

ALSFRS-R change at week 28

Summary

Failed primary in VALOR; FDA accelerated approval April 2023 based on plasma neurofilament-light reduction. Surrogate-endpoint case study.

Protocol timeline

Phase I

Phase II

Phase III

Phase IV

Current status Completed

Enrolled n = 108

View on ClinicalTrials.gov ↗

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