Biopharmaceutical firm Orphazyme has reported positive interim results from an open-label extension study of a Phase II/III clinical trial of arimoclomol in patients with Niemann-Pick disease Type C (NPC).

Arimoclomol is an investigational candidate that works to increase heat-shock proteins production in cells under stress or toxicity. Heat-shock proteins naturally protect cells in the body.

At 12 months, the open-label extension data demonstrated sustained effect in decreasing disease progression over two years. The Orphazyme said that the results also validated the safety and efficacy profile of the drug.

The 12-month period of the trial was completed by 41 patients who were all treated with arimoclomol.

Patients who switched from placebo to the drug were observed to have a decrease in disease progression similar to those who received arimoclomol in the randomised, placebo-controlled Phase II/III trial.

The disease progression was measured using the 5-domain NPC Clinical Severity Scale (5-domain NPCCSS), which was the primary endpoint of the trial.

The data also showed that patients treated with the drug for a total of two years had greater progress in the open-label extension versus those in the placebo-controlled period.

In subgroups of participants aged four and above, subjects taking miglustat as part of their routine clinical care experienced greater benefit with early arimoclomol treatment compared to delayed therapy.

A genetic sub-group analysis demonstrated that the drug had a statistically significant effect on disease progression compared to placebo in patients with homozygous functional null mutations, which are predictive of rapidly progressive disease.

Orphazyme CEO Kim Stratton said: “These new data confirm our commercial preparations for the launch of arimoclomol in the US as well as in other key markets. We look forward to seeking approval and bringing this innovative treatment to the market to address the significant unmet need in this devastating disease. 

“Arimoclomol is a very promising compound, which has the potential to change lives for the better, and we are continuing its development in three other indications: amyotrophic lateral sclerosis (ALS), sporadic inclusion body myositis (sIBM), and Gaucher disease.”

The company is planning to submit applications seeking approval for the drug in NPC in the US and Europe in the first half and the second half of this year, respectively.