CYTR - Orphazyme's arimoclomol shows meaningful treatment effect in late-stage NPC study

CytRx (OTCQB:CYTR) highlights that Orphazyme (NASDAQ:ORPH) has published the results from a Phase 2/3 trial of arimoclomol in Niemann-Pick disease type C (NPC) in the peer-reviewed Journal of Inherited Metabolic Disease. ORPH shares up 4.1% premarket at $5.54. NPC is a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. The trial enrolled 50 patients aged 2–18 years. The primary endpoint was change in 5-domain NPC Clinical Severity Scale (NPCCSS) score from baseline to 12 months. At 12-months, a significant treatment effect in favor of arimoclomol of ?1.40 points (95% CI: ?2.76, ?0.03; p = 0.046) was observed, corresponding to a 65% relative reduction in annual disease progression. In the prespecified subgroup of patients receiving miglustat as routine care, arimoclomol resulted in stabilization of disease severity with a treatment difference of ?2.06 in favor of arimoclomol (p = 0.006). In the

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Orphazyme’s arimoclomol shows meaningful treatment effect in late-stage NPC study