Arimoclomol shows promise in protein misfolding disease

Arimoclomol shows promise in protein misfolding disease; preclinical and Phase 1/2 clinical data published in Science Translational Medicine

Orphazyme ApS today announced preclinical and clinical trial phase 1/2 data demonstrating that arimoclomol, an inducer of heat shock proteins, can ameliorate disease and improve muscle function in inclusion body myositis. The exciting findings are the result of a collaboration between researchers from University College London and University of Kansas and are being published this week in Science Translational Medicine.

Corresponding author Professor Linda Greensmith, University College London, says: "Through this collaboration and the support of the MRC Centre we have been able to take our fundamental observations in cell and animal models right through to a proof-of-concept trial in patients. We are actively pursuing this promising approach of manipulation of the heat shock response in IBM as well as other neurodegenerative diseases such as motor neurone disease, in collaboration with the MRC Centre and Danish biotech company Orphazyme ApS."

For more information about the researchers, see their press release here.

Anders Hinsby, Orphazyme CEO, says: "I want to congratulate the researchers from University College London and University of Kansas with this thorough and successful collaboration. The impressive work shows convincingly the potential of arimoclomol as a treatment for diseases of protein misfolding."

Orphazyme CSO, Thomas Kirkegaard adds: "The investigators have been able to conduct a small placebo-controlled, randomized Phase 1/2 study in patients, which showed clear trends of disease amelioration consistent with their preclinical work. These clinical trends of efficacy were surprising and very encouraging given the small number of patients and short duration of treatment but is another pillar to support the continued work with arimoclomol in protein misfolding diseases such as inclusion body myositis and Niemann-Pick disease type C."

Further information: 
Anders Hinsby, CEO of Orphazyme

About Orphazyme

Orphazyme ApS is a Danish biopharmaceutical company, which develops paradigm-changing medicines for the treatment of genetic and degenerative diseases. The company is a global leader in the development of heat shock protein-based therapies. The lead programme is an orally available small molecule in Phase 2/3 clinical development as a treatment for Niemann-Pick disease type C (NP-C). For more information, please visit