Poster presentation at WORLDSymposium™ 2018 – arimoclomol as clinical candidate for treatment of Gaucher disease

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Investor news
Orphazyme A/S

No. 02/2018
Copenhagen, February 6, 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 poster on arimoclomol as a clinical candidate for the treatment of neuronopathic Gaucher disease will be presented at WORLDSymposium™ 2018 in San Diego, CA, USA.
The data, which will be presented today at 4.30 PM PST by Orphazyme’s Chief Scientific Officer, Thomas Kirkegaard Jensen, highlights the mechanism of action and efficacy of arimoclomol in preclinical models of Gaucher disease. A Phase II clinical trial of arimoclomol in Gaucher disease is planned to commence in Q2 2018.There are currently no treatments available for the neurological symptoms of Gaucher disease. For an abstract of the poster, please follow this link:
For additional information, please contactOrphazymeAnders Hinsby, CEO                                                             +45 31 44 31 39
About Gaucher disease
Gaucher disease is a rare, inherited, progressive, and debilitating lysosomal storage disorder. Gaucher disease is caused by genetic mutations often resulting in misfolded variants and deficient activity of the enzyme glucocerebrosidase. As a consequence, lipid waste materials build up in the lysosomes of cells throughout the body. Accumulation of lipid waste material in the bone marrow, liver, spleen, and, in some cases, the brain, leads to serious bone complications, enlarged liver and spleen, anemia, low platelets, and sometimes progressive neurological damage. The estimated number of people affected with Gaucher disease in the US and Europe combined is 10,000-15,000. There are no treatments available for the neurological symptoms of Gaucher disease where Orphazyme is focused.
About arimoclomol
Arimoclomol is in development for four severe orphan diseases: sporadic Inclusion Body Myositis (sIBM) and Amyotrophic Lateral Sclerosis (ALS), and two lysosomal storage diseases, Niemann-Pick disease Type C (NP-C) and Gaucher disease. Arimoclomol has been carefully studied in seven Phase I and three Phase II trials and is currently being studied in two Phase II/III trials in NP-C and sIBM.
Arimoclomol works by amplifying the production of a group of rescue proteins, called heat-shock proteins. Heat-shock proteins help restore balance to the cell by improving the folding of dysfunctional misfolded proteins and clear disease-related protein aggregates or improve the function of lysosomes, which are critical cellular processes involved in many diseases.Arimoclomol is a small molecule that has been shown to readily cross the blood brain barrier, access the central nervous system and penetrate the brain which is key to treatment for many neurodegenerative diseases. Arimoclomol is administered orally and can be easily dissolved in liquids or sprinkled on food for convenience and for patients who have difficulty swallowing. Orphazyme has obtained Orphan Drug Designation for arimoclomol in NP-C, Amyotrophic Lateral Sclerosis (ALS), and sIBM from both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). About Orphazyme A/S
Orphazyme is a Danish biopharmaceutical company focused on the development of treatments for patients living with life-threatening or debilitating rare diseases. Our research is concentrated on the cell-protective properties of the body’s own rescue system for diseases caused by misfolded proteins or lysosomal dysfunction.  The company’s lead candidate, arimoclomol, is in development for four severe orphan diseases; two neuromuscular diseases, sporadic Inclusion Body Myositis (sIBM) and Amyotrophic Lateral Sclerosis (ALS), and two lysosomal storage diseases, Niemann-Pick disease Type C (NP-C) and Gaucher disease. We are listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit

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Jasmine Petters

Jasmine Petters

Jasmine hails from the city of Nizams, Hyderabad, and is currently pursuing her M.D. from there only. She is an internet wizard and has keen passion in All Things tech. She is a regular Comment contributor for The Daily Telescope and writes a Web column, in which she covers war, sports, and everything in between.
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