Arimoclomol (Orph-001) is a therapy that Orphazyme and the University of Miami are developing for amyotrophic lateral sclerosis.

In addition to ALS clinical trials, Orphazyme is also conducting trials of arimoclomol as a treatment for sporadic inclusion body myositisNiemann-Pick type C, and Gaucher disease.

How arimoclomol works

ALS is a fatal neurodegenerative disease. The death of motor neurons, or nerve cells that control muscle movement, leads to the paralysis that is the hallmark of the disease.

Scientists don’t know what causes all cases of ALS. They do know some forms are inherited, resulting from a mutation of the superoxide dismutase 1, or SOD1, gene. The gene generates the SOD1 protein, and the mutation changes the way the protein functions. It becomes toxic, leading to rapid loss of motor neurons.

Arimoclomol triggers an increase in the production of heat shock proteins, or HSPs, which are involved in the body’s response to stress. HSPs bind to faulty proteins. They also regulate the programmed cell death pathway, the mechanism by which the body gets rid of damaged cells that need to be disposed of.

In ALS, one of the HDPs — HSP70 — can bind to faulty SOD1 protein and remove it. By increasing the production of HSP7, arimoclomol may reduce the rate of motor neuron death and slow the progression of ALS.

A preclinical-trial study of arimoclomol’s ability to treat mice with ALS appeared in the journal Nature Medicine. The mice were able to move much better than untreated mice when they became older, and lived 22 percent longer. The findings suggested that arimoclomol could be an effective ALS treatment.

Arimoclomol in clinical trials

A Phase 2 clinical trial showed that arimoclomol was safe, and that patients tolerated it well. Patients who took arimolomal lived longer than those who received a placebo. In addition, the arimolomal patients did better on a measure of ALS progression than the placebo patients.

Orphazyme enrolled 36 patients with SOD1-related ALS in the trial NCT00706147). They received either arimoclomol or a placebo three times a day for up to 12 months.

The measure of ALS progression that researchers used to assess arimoclomal’s effectiveness during the treatment period and for a month afterward was patients’ rate of decline in the ALS functional rating scale-revised, or ALSFRS-R. The research team also checked for adverse effects of treatment.

Orphazyme presented the results at the 2016 International Symposium on ALS/MND. The positive findings prompted the company to meet with regulators about testing arimoclomol in further clinical trials.

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