Company Profile
ORPHAZYME A/S
Company Overview
At Orphazyme our work is focused on the cell-protective properties of the heat-shock response, a natural defence mechanism in all our cells. The heat-shock response protects cells from an accumulation of misfolded proteins or other waste products, which would otherwise lead to toxicity and disease. The heat-shock response is generated through the production of heat-shock proteins, which act as the cells’ lifeguards.
Today, Orphazyme’s technology is based around the drug arimoclomol, which stimulates an increased production of heat-shock proteins in cells experiencing stress or toxicity.
When we founded Orphazyme, our focus was on making a positive difference for patients with rare diseases with a high unmet need. In line with this, we are today working to create new therapeutic approaches for a number of different rare diseases, including sporadic Inclusion Body Myositis (sIBM), Amyotrophic Lateral Sclerosis (ALS), Niemann-Pick disease Type C (NPC), and Gaucher disease. Our aim is to slow or even prevent disease progression and we believe our approach has the potential to dramatically improve the lives of those suffering from a range of degenerative diseases which currently do not have satisfactory treatments.
VISION
To profoundly impact the lives of patients with orphan diseases and their families.
STRATEGY
The strategy is to develop treatments for orphan diseases with protein misfolding where it can apply its specialised know-how in HSPs.
The objective is to successfully conduct and complete the planned and ongoing trials of arimoclomol for the treatment of the neuromuscular diseases, sIBM and ALS, and the lysosomal storage diseases, NPC and Gaucher disease.
As the clinical development programme for arimoclomol progresses, Orphazyme intends to refine and finalise its commercialisation strategy and build its commercial structure and operations. The Company intends to build its own sales force in key markets. In markets outside the United States and Europe, Orphazyme currently intends to partner with local/regional distributors or license partners in certain other geographical areas.
Protein misfolding is the hallmark of a broad range of diseases and Orphazyme’s strategy is to use its expertise, including proprietary know-how to select and develop new leads for suitable diseases. In line with this, the Company is developing a proprietary suite of NMEs with improved characteristics. Orphazyme intends to select diseases suitable for the NMEs based on genetic and mechanistic insights into selected protein misfolding diseases.
Company History
Orphazyme was founded in 2009 with the objective to develop new therapies for patients suffering from protein misfolding diseases with no or limited treatment options available.
At inception, the Company was based on a scientific discovery on the function of HSPs by Thomas Kirkegaard Jensen (current CSO) and Professor Marja Jäättelä that was published in the scientific journal Nature. Since inception, the Company has translated the scientific discovery into a late stage clinical development programme.