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9.11.2018

Phase III Trial of Arimoclomol for ALS Starts at the University of Miami Health System

Physician-researchers with the University of Miami Health System and the Miller School Medicine recently enrolled the first patient in a phase III trial of the drug Arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS). The randomized patient received the drug on August 10.

ALS, also known as Lou Gehrig’s disease, is a rapidly progressive, fatal neurological disease that attacks the nerve cells responsible for controlling voluntary muscles. Arimoclomol is a heat shock protein-based therapy that shows promise for the treatment of ALS, based on the results of the phase II trial. The Danish biopharmaceutical company Orphazyme A/S, is sponsoring and running the phase III trial.

The U.S. Food and Drug Administration and ALS Association-funded phase II trial, upon which this phase III trial is predicated, was designed and led by Michael Benatar, M.D., Ph.D., professor of neurology, the Walter Bradley Chair in ALS Research and executive director of the ALS Center at the University of Miami. Benatar also assisted in the design and serves as the Lead International Coordinating Investigator of the current phase III trial.

“I am delighted to see Arimoclomol advance into phase III testing and that the eligible population has been broadened to include all patients with ALS,” said Dr. Benatar. “Based on the mechanism of action of the drug and our understanding of the underlying biology of ALS, there is good reason to believe that all patients with ALS might benefit from this therapeutic approach.”

Orphazyme A/S is dedicated to developing treatments for patients living with rare diseases. Arimoclomol acts in stressed cells by stimulating the cells’ own heat shock response, a cell-protective system involved in maintaining proper protein folding.

“Arimoclomol has shown promising results in previous trials and has a favorable safety and tolerability profile,” said Thomas Blaettler, chief medical officer of Orphazyme. “ALS is a truly devastating disease, and we will work to expeditiously advance the phase III trial with the prospect of making a new therapeutic option available to patients.”

The primary objective of the phase III trial is to determine the efficacy of Arimoclomol compared to a placebo. The trial, which will be conducted in North America and Europe, will enroll 231 patients to be randomized in a 2:1 ratio, receiving either Arimoclomol or a placebo for up to 76 weeks. Patients completing the trial will be offered participation in an open-label extension trial.

Patients with ALS interested in learning more about the study and eligibility criteria, and locating participating trial centers can visit the following website: https://orphazyme.longboat.com/.

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