Study summary

An antisense drug for the most common genetic ALS did not work

BIIB078 trial · 106 people, phase 1 · Published July 2024, Lancet Neurology

What was tested

BIIB078, an antisense drug targeting the C9orf72 gene repeat, the most common genetic cause of ALS, in an early safety trial.

What they found

• No reduction in neurofilament and no clinical benefit compared with placebo.
• Development was stopped.

What it means

C9orf72 ALS remains a major unsolved target; other approaches are being tried.