Positive late-stage data on Orphazyme's rare disease drug

Danish biotech firm Orphazyme (ORPHA: CPH) today announced encouraging top-line results for its clinical Phase II/III trial with orally administered arimoclomol for the treatment of patients with Niemann-Pick disease Type C (NPC), with the news sending the firm’s shares up 2.27% to 72.00 Danish kroner.

The trial was a multi-center, prospective, double-blinded, placebo-controlled interventional study with a 12-month duration. In total, 50 patients were enrolled in the European Union and USA. The purpose of the trial was to assess the efficacy and safety of arimoclomol, compared to placebo, in the treatment of NPC, administered in addition to the patient's standard-of-care. The primary endpoints, 5-domain NPC-CSS and Clinical Global Impression of Improvement (CGI-I), evaluated the treatment difference between the arimoclomol-treated and the placebo group after 12 months of treatment.

Orphazyme acquired right to arimoclomol from CytRx in 2011 in a deal worth up to $120 million to the latter and included two other compounds, iroxanadine and bimoclomol.