Queen Square Centre for Neuromuscular Diseases


Orphazyme assumes sponsorship of Phase II/III arimoclomol sIBM trial

20 December 2017

On December 14, 2017, Orphazyme A/S, University of Kansas Medical Center, and UCL announced that Orphazyme formally assumes the sponsorship of the Phase II/III arimoclomol trial for the neuromuscular disease sporadic Inclusion Body Myositis (sIBM).

The trial was conceived by expert investigators at KU Medical Center and UCL to assess the efficacy and safety of arimoclomol for the treatment of sIBM. The KU Medical Center and UCL teams bring long-standing knowledge on the basic and clinical science of sIBM.

The multi-centre trial initiated patient enrolment in August 2017 in the USA and Europe and so far, 9 patients have been enrolled. The trial is intended to support a registration of arimoclomol for the treatment of sIBM. Results from the trial are expected in H1 2020.

Anders Hinsby, CEO of Orphazyme said: "With the first dosing of patients in the sIBM study, we mark an important milestone in the development of our pipeline. We hope this trial, conducted with the foremost experts in the field, will lead to the first effective treatment for this devastating disease."

Richard Barohn, Vice Chancellor for Research and Professor of Neurology at the KU Medical Center said: "This trial is the culmination of a sustained, investigator-led effort to move from cells, through animal studies, to patients. We are happy to work with Orphazyme to explore arimoclomol's potential."

Professor Michael Hanna, Director of the MRC Centre for Neuromuscular Diseases and the UCL Institute of Neurology, said: "Arimoclomol has strong potential benefit in patients with this disabling disease. We made the case quite strongly that arimoclomol should be properly tested in a large trial, and it is most exciting that we have now started the next step in the clinical development."

Professor Mazen M. Dimachkie, Principal Investigator of the study and Director of the Neuromuscular Division at KU Medical Center said: "The situation is dire for the patients as they progressively lose dexterity, mobility and independence. We do not have any effective treatment for this major unmet medical need, and I am excited to be leading this important trial."