On December 14, 2017, Orphazyme A/S, University of Kansas Medical Center, and UCL announced that Orphazyme formally assumes the sponsorship of Phase II/III arimoclomol trial for the rare neuromuscular disease sporadic Inclusion Body Myositis (sIBM).
The arimoclomol 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.
Principal Investigator of the study and Director of the Neuromuscular Division at KU Medical Center is Professor Mazen M. Dimachkie 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.”
We will continue to provide you with updates as we continue to learn more about this trial and work together with Orphazyme.
Arimoclomol is a new chemical entity with a very favorable safety and tolerability record in humans: Seven Phase I clinical studies have been conducted in healthy volunteers. Arimoclomol is administered orally, three times daily, and can be easily dissolved in liquids or food for best possible patient comfort and compliance. Orphazyme has obtained orphan drug designation for arimoclomol in NPC, ALS, and sIBM from both the FDA and EMA.
About Orphazyme A/S
Orphazyme is a Danish biotech company listed on Nasdaq Copenhagen with a late-stage drug pipeline, developing new treatment options for orphan protein-misfolding diseases. The company was founded in 2009 based on early scientific discovery in heat-shock proteins. The company is headquartered in Copenhagen and currently has 30 employees. The lead candidate arimoclomol is in development as a potential treatment for four orphan diseases; two neuromuscular diseases, sporadic Inclusion Body Myositis (“sIBM”) and Amyotrophic Lateral Sclerosis (“ALS”), and two lysosomal storage diseases, Niemann Pick Type C (“NPC”) and Gaucher disease. For more information, please visit www.orphazyme.com.clinical trialsinclusion body myositis