ORPHA

Orphazyme

Market Cap 1,286.1M

Orphazyme is a Copenhagen-based biotech company with a late-stage clinical pipeline. The company was founded in 2009 based on a scientific discovery of the function of heat-shock proteins and their role in protein-misfolding diseases. The lead clinical asset arimoclomol was acquired from US biotech CytRx in 2011, based on its unique profile and effect on the heat-shock proteins. The drug came with a range of pre-clinical and clinical data in ALS to back up the hypothesis, and Orphazyme is now developing the drug more broadly to explore the full potential. With this, arimoclomol has been moved into three potential registration studies and one Phase II in orphan indications, and they could be granted marketing approval for all four indications by 2023. Orphazyme has a founder-led management team with expertise in both the science and medical aspects. They have approximately 50 employees that back them up, mainly focusing on R&D, but the company’s long-term strategy is to build a commercial infrastructure and become a fully-integrated biopharma, and a subsidiary is already set up in the US for those purposes. The main shareholders are Novo Holdings, LSP V Coöperative, Sunstone Life Science Ventures, and Coöperative Aescap Venture, as well as Danish institutional investors representing a solid ownership foundation. 

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