PUBLICATION OF MANUSCRIPT ON PRECLINICAL PROOF-OF-CONCEPT OF ARIMOCLOMOL IN GAUCHER DISEASE

Copenhagen, November 27, 2018 – Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced that a manuscript providing preclinical Proof-of-Concept for its lead compound, arimoclomol, in Gaucher disease was published in the peer-reviewed scientific journal EBioMedicine. The manuscript details how the heat-shock amplifier, arimoclomol, improves the refolding, maturation, and lysosomal activity of glucocerebrosidase (GCase), the enzyme deficient in Gaucher disease and linked to Parkinson’s disease.

A range of biological and biochemical assays were used to asses the effect of arimoclomol on GCase activity in ex vivo systems of fibroblasts and neuronal-like cells from Gaucher disease patients. Data showed that arimoclomol induced relevant Heat-Shock Proteins (HSPs), such as HSP70, and enhanced the folding, maturation, activity, and correct cellular localization of mutated GCase across several genotypes, including common mutations in primary cells from Gaucher disease.

Thomas Kirkegaard Jensen, Co-Founder and Chief Scientific Officer, says: “We are excited that the excellent work of our scientists and close collaborators is now available for all to read. The findings provide a cornerstone of our lysosomal disease programs and ambition to provide a treatment for all patients suffering from these devastating diseases”.

To read the full-length article, please visit the Media section on Orphazyme’s website: www.orphazyme.com