Rare Disease Report

Data Supports Safety Profile of ALS Drug

JANUARY 25, 2018
Mathew Shanley
The peer-reviewed clinical journal Neurology has published the Phase 2 trial results for arimoclomol in patients with SOD1 amyotrophic lateral sclerosis (ALS).

The developer of the drug, Danish biotech Orphazyme A/S, announced the news this morning, and is expected to initiate a Phase 2/3 trial to support the application for marketing authorization of the drug in ALS in the second half of 2018.

ALS is characterized by neurological symptoms like seizures, delayed motor development, and cognitive problems that commonly develop from a buildup of cholesterol in the brain. Orphazyme A/S has already received orphan drug designation for arimoclomol, an orally available small molecule, in ALS from both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA).  

Michael Benatar, M.D., Ph.D., lead author and primary investigator on the study, confirms the safety of arimoclomol and that it is well-tolerated at a dosage of 200mg 3 times a day for up to 12 months: “In addition to providing evidence of the safety and tolerability of arimoclomol 200mg three times daily over a 12-month period, the consistency of the preliminary efficacy data across a range of pre-specified endpoints, and in the only pre-specified subgroup, strongly supports further development of arimoclomol in ALS,” he said.

Thirty-eight participants were randomized in the study, 36 of whom were included in the prespecified intent-to-treat analysis. Adverse events (AEs) were typically mild and deemed unlikely or not related to the drug.

Secondary endpoints in the Phase 2 evaluated the efficacy of arimoclomol all favored arimoclomol. The trial was not designed for the drug to exhibit efficacy, but trends of clinical benefit were consistently observed across the full range of pre-specified efficacy outcome measures, including survival, function (ALSFRS-R), vital capacity, and the combined assessment of function and survival (CAFS).

Previously, 7 Phase 1 clinical studies of arimoclomol were conducted in healthy volunteers. The drug can be easily dissolved in liquids or food to achieve the best possible patient comfort and compliance.

Per the ALS Association, between 1-3 per 100,000 individuals are diagnosed annually worldwide.

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