More and more, the importance of rare, lysosomal storage diseases (LSDs) is being recognised. The latest Nature | Outlook article, called ‘Drug development: Through the barrier’, highlights the great need for improved treatment, especially for small molecules which can cross the blood-brain barrier. For this reason, Orphazyme’s paradigm-changing technology led by small molecule oral drug arimoclomol, which can reach the central nervous system, has been featured in the story (Nature outlook article).
Arimoclomol works by stimulating stressed cells to produce the stress-response protein Heat Shock Protein 70 (HSP70), which in turn helps cells to correctly process accumulated waste products that have reached toxic levels. It looks promising as a treatment for a number of neurodegenerative disorders, since it can travers the blood-brain barrier. Orphazyme is currently testing arimoclomol in a clinical trial for the treatment of Niemann-Pick type C, an LSD which affects an estimated one in 100,000 newborns (see www.AIDNPC.com), as well as for other neurodegenerative diseases such as sporadic inclusion body myositis (sIBM) and amyotrophic lateral sclerosis (ALS/Lou Gehrig's disease).
Orphazyme’s Chief Scientific Officer Thomas Kirkegaard Jensen said, “The focus in Nature on our technology and lead clinical candidate, arimoclomol, as a novel therapeutic opportunity with wide ranging implications for the lysosomal diseases is a testament to the great work being done by Orphazyme’s scientists and our world-leading academic collaborators”.
Orphazyme ApS is a Danish biopharmaceutical company which develops paradigm-changing medicines for the treatment of genetic diseases. The lead program is in development as a treatment for lysosomal storage diseases. This family of genetic disorders consisting of more than 45 diseases, including Niemann-Pick disease type C, often affect children; most of whom are currently untreatable. Orphazyme’s AIDNPC study is a phase III clinical trial to investigate a novel treatment for Niemann-Pick disease type C, which launched in July 2016 (www.AIDNPC.com). 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, visit www.orphazyme.com.
Helen Frost, Communication & Engagement Manager at Orphazyme ApS
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