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ARTICLE | Clinical News

Orphazyme seeking path forward for arimoclomol in NPC after Phase II/III miss

October 19, 2018 5:16 PM UTC

Orphazyme A/S (CSE:ORPHA) said arimoclomol missed the primary endpoints in a Phase II/III trial to treat Niemann-Pick disease type C (NPC). The company plans to meet with FDA and EMA to determine next steps for the small molecule inducer of heat shock protein 70 (Hsp70).

On the first primary endpoint, arimoclomol led to a 74% reduction in disease progression on the 5-domain NPC Clinical Severity Scale (NPC-CSS) from baseline to 12 months vs. placebo (p=0.07). The company also said a positive effect with arimoclomol on the second primary endpoint of Clinical Global Impression of Improvement (CGI-I) at 12 months could not be shown due to lower than expected placebo progression rates...

BCIQ Company Profiles

Orphazyme A/S

BCIQ Target Profiles

Heat shock protein 70 (Hsp70)