Orphazyme phase 2 study of arimoclomol in Gaucher disease demonstrates marked improvements in key clinical markers

by | Jun 29, 2020

Orphazyme A/S (ORPHA.CO), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces topline results from a 6-month phase 2 dose-finding study in Gaucher disease with arimoclomol, an investigational Heat-Shock Protein (HSP) amplifier. The data show a dose-dependent effect of arimoclomol on certain disease-relevant clinical secondary endpoints, such as liver and spleen size. Furthermore, the data demonstrate sustained levels of arimoclomol in the cerebrospinal fluid (CSF), providing further evidence of arimoclomol’s ability to cross the blood-brain barrier.  

Press inquiries should be adressed to:

Sten Verland, PhD MSc

General Partner