Publication of manuscript related to Phase II trial results for arimoclomol in patients with SOD1 ALS

Investor news
Orphazyme A/S

No. 01/2018                                                                                                                                                           


Copenhagen, January 24, 2018 – Orphazyme A/S, a Danish biotech company listed on Nasdaq Copenhagen (TICKER: ORPHA.CO), with a late-stage, orphan-drug pipeline, today announced that a manuscript reporting the Phase II trial results for arimoclomol in patients with SOD1 Amyotrophic Lateral Sclerosis (ALS) was published in the peer-reviewed clinical journal Neurology®. The manuscript highlights arimoclomol’s favorable safety profile and provides data on efficacy that supports the continued development of arimoclomol in a Phase II/III trial.

The manuscript, which is lead-authored by primary investigator Michael Benatar, MD and PhD from the University of Miami, confirms that arimoclomol is safe and well-tolerated at a dosage of 200mg three times a day for up to 12 months.

Additionally, secondary endpoints looking at the efficacy of arimoclomol all favored arimoclomol. The trial was not powered to demonstrate efficacy, but trends of benefit were consistently observed across the full range of pre-specified efficacy outcome measures, including survival, function (ALSFRS-R), vital capacity, as well as the combined assessment of function and survival (CAFS).

Michael Benatar, MD and PhD, primary investigator says: In addition to providing evidence of the safety and tolerability of arimoclomol 200mg three times daily over a 12-month period, the consistency of the preliminary efficacy data across a range of pre-specified endpoints, and in the only pre-specified subgroup, strongly supports further development of arimoclomol in ALS.

As previously communicated, Orphazyme intends to conduct a Phase II/III trial to support the application for a marketing authorization in ALS. The trial is planned to be initiated in H2 2018.

A link to the article is available on in the Media section.



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Anders Hinsby, CEO                                                             +45 31 44 31 39


About ALS
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive and invariably fatal neurological disease. Despite being classified as a rare disease, ALS is considered one of the more common neuromuscular diseases worldwide. The incidence of ALS is estimated at between 1-3 per 100,000 individuals per year globally. The patient population in Europe and the United States is estimated to be approximately 50,000 patients. Currently, there are only a limited number of available treatments for ALS patients and these only impart a modest effect.

About arimoclomol
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

Pièces jointes

01-2018 Publication of manuscript related to Phase II trial results for arimoclomol in patients with SOD1 ALS