- Orphazyme A/S ORPH announced that results from a Phase 2/3 trial of arimoclomol in Niemann-Pick disease type C (NPC) had been published in the peer-reviewed Journal of Inherited Metabolic Disease (JIMD).
- Arimoclomol is an investigational heat-shock protein amplifier.
- NPC is a rare, inherited progressive neurodegenerative disorder with a high unmet medical need for disease-modifying treatment options.
- The trial demonstrated a statistically significant and clinically meaningful treatment effect supported by substantial and consistent effects across several disease and pharmacodynamic biomarkers.
- See: Orphazyme To Cut Two-Third Workforce Under Restructuring
- At 12-months, a significant treatment effect in favor of arimoclomol of −1.40 points was observed, corresponding to a 65% relative reduction in annual disease progression.
- In the prespecified subgroup of patients receiving miglustat as routine care, arimoclomol stabilized disease severity with a treatment difference of −2.06 in favor of arimoclomol.
- Arimoclomol was well-tolerated, with adverse events occurring in 88.2% of patients receiving arimoclomol and 75.0% of patients receiving placebo.
- Fewer patients had serious adverse events with arimoclomol (14.7%) versus placebo (31.3%).
- Arimoclomol is under regulatory review in Europe, with an anticipated CHMP opinion in Q4 2021.
- Related: FDA Turned Down Arimoclomol Application Asking For Additional Evidence.
- Price Action: ORPH shares are up 12.40% at $5.63 during the premarket session on the last check Monday.
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