Orphazyme rises on positive trial of arimoclomol in Niemann-Pick disease type C

Shares of Denmark’s biopharma company Orphazyme (CPD: ORPH) rose 12.4% to $5.63 on Monday, as it announced that positive results from a Phase II/III trial of arimoclomol, an investigational heat-shock protein amplifier, in Niemann-Pick disease type C (NPC) have been published in the peer-reviewed Journal of Inherited Metabolic Disease (JIMD). The online publication is available  here.

This marks a positive reversal in the fortunes of Orphazyme, which in June this year had a major setback on the news of a delay for its lead pipeline candidate. Rather than granting approval of Orphazyme’s new drug application for arimoclomol, the US Food and Drug Administration issued a Complete Response Letter (CRL).

“We are pleased to share the data from our Phase II/III trial in JIMD. NPC is a rare, inherited progressive neurodegenerative disorder with a high unmet medical need for disease-modifying treatment options. This trial demonstrated a statistically significant and clinically meaningful treatment effect of arimoclomol in NPC supported by significant and consistent effects across several disease- and pharmacodynamic biomarkers. We believe these data establish the potential of arimoclomol as an efficacious and well-tolerated disease-modifying treatment for NPC,” said Thomas Blaettler, chief medical officer at Orphazyme.