Phase 3 clinical trial for arimoclomol does not show benefit to people living with ALS

May 7, 2021 – Biopharmaceutical company Orphazyme A/S announced today that the ORARIALS-01 pivotal trial of the drug arimoclomol did not meet its primary and secondary endpoints to show benefit in people living with ALS. No important safety signals were reported in the trial.

The randomized, placebo-controlled Phase 3 trial was conducted among 245 patients at 29 sites in 12 countries in North America and Europe. The primary endpoint was to determine the efficacy of chronic treatment with arimoclomol compared to placebo in participants with ALS as assessed by function and survival.

“With over 18 months of evaluation, this trial represents one of the longest running clinical studies in this category. While unsuccessful, the data generated will contribute meaningfully to the scientific dialogue on this challenging disease. We will apply the invaluable insights from this and other studies to further our pipeline as we continue to pursue the full potential of the heat shock protein response,” said Thomas Blaettler, MD, Chief Medical Officer, Orphazyme.

We know this therapy is of interest to the ALS community and share Orphazyme’s disappointment in the results. You can read more about the results here.