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).
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