Study summary

Arimoclomol did not slow ALS

ORARIALS-01 trial · 239 people, phase 3 · Published May 2024, Lancet Neurology

What was tested

Arimoclomol, a drug meant to help cells clear misfolded proteins, against placebo, in early ALS.

What they found

• Combined function and survival scores over 76 weeks were no better than placebo.
• More people stopped arimoclomol because of side effects (16%).

What it means

One of several broad-acting drugs that failed, pushing research toward gene-specific and platform trials.