Arimoclomol Fails to Show Efficacy in Phase 3 Trial, Topline Data Show

A Phase 3 clinical trial evaluating Orphazyme’s investigational oral therapy arimoclomol for amyotrophic lateral sclerosis (ALS) has failed to meet its primary and key secondary goals. While no safety issues were reported in the trial (NCT03491462), arimoclomol did not significantly extend patients’ lives nor…

Arimoclomol Earns FDA’s Fast Track Status for ALS

The U.S. Food and Drug Administration (FDA) has given fast track designation to Orphazyme’s investigational therapy arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS). A therapy candidate is put on the FDA’s fast…

Orphazyme Partnership to Run Through Extension of Phase 3 Arimoclomol Study

Orphazyme announced that it and Worldwide Clinical Trials will continue their collaboration through the long-term extension of a Phase 3 study into arimoclomol, a potential oral treatment for amyotrophic lateral sclerosis (ALS). ORARIALS-01 is a randomized, placebo-controlled and double-blind trial (NCT03491462) underway at 30 centers across North America…

Enrollment Complete for ORARIALS-01 Trial of Arimoclomol in ALS Patients

Patient enrollment is now complete, ahead of schedule, for the Phase 3 ORARIALS-01 clinical trial assessing the effectiveness of Orphazyme’s investigational therapy arimoclomol in the treatment of amyotrophic lateral sclerosis (ALS). “The completion of enrollment for Orphazyme’s pivotal ALS trial is another great milestone for our clinical development program. We are confident that…

Orphazyme Doses First ALS Patient in Phase 3 Trial of Arimoclomol

The first patient has been dosed in a Phase 3 clinical trial (NCT03491462) evaluating the effectiveness of Orphazyme’s investigational therapy arimoclomol in the treatment of amyotrophic lateral sclerosis (ALS). One of the disease mechanisms known to be involved in the development of ALS is protein misfolding and aggregation…