Clinical Trial: Orphazyme to Enroll 231 People with ALS in Phase III Arimoclomol Clinical Trial

The ALS Association Supported Research In Arimoclomol Development

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Biopharmaceutical company Orphazyme A/S launched a phase III clinical trial of arimoclomol, giving a dose to a person with ALS for the first time, the company announced recently. The randomized and placebo-controlled trial will enroll 231 people with ALS in North America and Europe to determine efficacy and safety of the oral drug.

Dr. Michael Benatar, executive director of the Kessenich Family ALS Center at the University of Miami and principal investigator of the study, presented data of the successful phase II clinical trial testing arimoclomol in patients carrying SOD-1 mutations at the International ALS MND Symposium in Dublin in December 2016.

The ALS Association, along with the U.S. Food and Drug Administration’s (FDA) Orphan Products Development Program, provided funding for the trial. The study was not powered to determine therapeutic effect; however, the data demonstrated safety and preliminary evidence for efficacy, which led to this new phase III trial. The open access data was later published in Neurology in January 2018.

“On behalf of the patients struck by this devastating disease, I am delighted to see this Phase III trial up and running. The ALS Association invested in the early clinical trials and are pleased to see this moving forward to a Phase III study and to seeing the potential of arimoclomol unfold.” (Dr. Lucie Bruijn, Chief Scientist, The ALS Association)

Arimoclomol is a heat shock, protein-based therapy. The heat shock response in cells is a protective system responsible for handing cell stress and is involved in proper folding of proteins. When proteins become misfolded during cell stress, they are vulnerable to aggregation, meaning they clump together and do not get recycled properly in the cell. This leads to cell toxicity and eventual cell death.

Arimoclomol is designed to produce a heat shock protein (HSP), called HSP-70, during cell stress. This helps clear aggregated proteins from the cell, while also helping prevent newly-built proteins from aggregating, thus preventing cell toxicity and death.

The trial will be a randomized, double-blinded, placebo-controlled phase III study that will assess the safety and efficacy of arimoclomol in the broad ALS population. The drug will be tested in a broad ALS population due to the drug’s broad mechanism of action. The FDA also endorsed a phase III trial in a broad population.

Patients will be randomized in a 2:1 ratio to receive either arimoclomol or placebo over a 76-week period. Primary endpoints include a combined assessment of function and survival (CAFS). Secondary endpoints include survival, changes in ALS Functional Rating Scale-Revised (ALSFRS-R), and slow vital capacity (SVC). Completion is expected in the first half of 2021. An open-label extension study will be offered after trial completion.

Patients with ALS interested to learn more about the study, eligibility, and locating participating trial centers can go to the following websites:

More information about the trial can be found on the NEALS clinical trial site and at, NCT03491462.

Read press release here.

Clinical trial sites:

  • University of Miami – Miami, Fla.

*Currently, only one out of 30 trial sites have been announced. Trial sites will be added to the above websites, once they are approved and open.


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