University of Miami and Orphazyme ApS Announce Successful Phase II Trial of Arimoclomol in ALS Patients

Data presented today at the International ALS/MND Symposium demonstrate safety and preliminary evidence of efficacy of Heat Shock Protein-based therapy in SOD1 ALS

December 9, 2016 – Arimoclomol, a heat shock protein-based therapy, shows promise for the treatment of ALS. Based on the results of a phase II trial, initiated and led by Michael Benatar, MD, PhD, of the University of Miami Miller School of Medicine, the drug is safe and well tolerated. Excitingly, all efficacy measures also show consistent and clinically meaningful trends favoring arimoclomol. Dr. Benatar presented the results of this innovative study today during a late-breaking news session at the 27th International Symposium on ALS/MND in Dublin.

This was a phase II randomized, double-blind, placebo-controlled trial that included 36 patients with rapidly progressive ALS due to mutations in the SOD1 gene. Patients were treated with Arimoclomol at a dose of 200mg three times per day for up to 12 months. The primary outcome was safety and tolerability; secondary outcome was efficacy, measured in terms of survival and functional decline.

“Our ability to detect a potential efficacy signal in such a small study is likely the result of one of the most innovative aspects of this trial – that it specifically enrolled patients who share both a common cause for their disease and a similar clinical phenotype”, says Dr. Benatar, Professor of Neurology, Walter Bradley Chair in ALS Research, and Executive Director of the Kessenich Family ALS Center at the University of Miami. “Another highly innovative aspect of the study was the use of remote assessments, in which most study visits took place in patients’ homes. This was a necessary design element of the study given that patients, recruited from across the United States and Canada, were expected to develop disability very quickly, which would limit their ability to travel to one of the two study centers for trial participation.”

The trial was funded by the ALS Association and the U.S. Food and Drug Administration’s Orphan Products Development Program. Orphazyme ApS, a company developing heat shock protein-based therapies for protein misfolding diseases, provided the study medication. The trial was conducted in collaboration with the NEALS Consortium under the leadership of Dr. Merit Cudkowciz at the Massachusetts General Hospital. The research group of Professor Peter M Andersen of Umea University, Sweden, performed SOD1 genetic testing. Professor Andersen is funded by the Nordic ALS patient organizations, the Swedish Brain Research Foundation, and the Knut and Alice Wallenberg Foundation (KAW).

Lucie Bruijn, PhD, MBA, Chief Science Officer of the ALS Association, added that “this was one of the first trials that the ALS Association funded which was focused on a specific ALS population, those people carrying an SOD1 mutation. Although logistically challenging, it has set the stage for future studies in a sub population of ALS patients, and it made use of the TREAT ALS NEALS Clinical Trials Network. I am very pleased that Dr. Benatar will be working with Orphazyme to move this study as rapidly as possible to the next phase.”

Arimoclomol is a small molecule that is taken orally and distributes throughout the body, including the brain. It acts in stressed cells by stimulating the cells’ own heat shock response, a cell-protective system involved in maintaining proper protein folding. Arimoclomol is beneficial in the SOD1 mouse model of ALS, even when administered after symptom onset. In all phase I and phase II clinical trials to date, arimoclomol has been found to be safe and well tolerated.

“We are very encouraged by the results. We now look forward to meet with the regulatory authorities to agree on next steps for the development of arimoclomol for SOD1-ALS. Moreover, preclinical and clinical data, as well as the mechanism of its action suggest that treatment with arimoclomol may convey benefit to a broader ALS population,“ says Thomas Blaettler, MD, Chief Medical Officer of Orphazyme ApS. “We are deeply grateful to the patients who participated in this study, to Dr. Benatar and all of the investigators, as well as the NEALS Consortium. We look forward to working with Dr. Benatar and his team, to build on these results with a view to developing a new therapeutic option for patients suffering from this devastating disease.”

About the ALS Research Program at the University of Miami Miller School of Medicine

Led by Dr. Michael Benatar and Joanne Wuu, the ALS Research Program at the University of Miami is broadly based, with both pre-clinical and clinical-translational components focused on understanding the early natural history of ALS, including its pre-symptomatic stage; developing and validating biomarkers; and studying the complex relationship between underlying genotype and disease phenotype. Therapy development motivates and guides all aspects of the Research Program. The program is partially supported by ALS Recovery Fund and the Eloise Kimmelman Foundation. For more information, please visit

About Orphazyme ApS

Orphazyme ApS is a Danish biopharmaceutical company, that develops paradigm-changing medicines for the treatment of genetic diseases and neurodegenerative diseases with well characterized mechanism of disease. The lead program is in development as a treatment for lysosomal storage diseases. This family of genetic disorders consists of more than 45 diseases and includes Niemann-Pick disease type C. Lysosomal storage diseases often affect children, most of whom are currently untreatable. Orphazyme is backed by leading European VCs. The strong investor syndicate includes Novo A/S, Sunstone Capital, Aescap Venture, Kurma Partners and Idinvest Partners. For more information, please visit

Further information concerning this SOD1 ALS clinical trial: :
Kai Hill, Communications Manager, The University of Miami Health System and Miller School of Medicine
Tel. 305-243-3249/305-332-3189

Further information concerning arimoclomol and development of arimoclomol for ALS and other diseases:
Helen Frost, Communication & Engagement Manager at Orphazyme ApS