Niemann-Pick Disease is a group lysosomal storage diseases that affect
metabolism. In Niemann-Pick Disease Type C (NPC), patients are not able to metabolize
cholesterol and other lipids properly within the cell. As a consequence,
excessive amounts of cholesterol and other fats accumulate within cells of the brain,
and other organs such as the liver and spleen. This causes a wide range of
symptoms, including behavioral problems, learning disabilities and difficulty
moving and speaking.
It is estimated that there are about 500 cases of NPC worldwide, but due to difficulties in diagnosis it is believed that the number is much higher. However, current treatment options for paediatric patients are only directed towards the symptoms and the majority of patients die before the age of 20. The only authorized product by the European Medicines Agency (EMA) for NPC, miglustat (Zavesca®), has been shown to delay the progression of the disease in some patients.
Orphanzyme, a Danish pharmaceutical startup, aim to “develop heat shock protein based therapy for the treatment of diseases caused by defects in the function and/or metabolism of proteins.” Their lead program, OR-01, recently started a phase II/III trial of patients with NPC to evaluate the efficacy of the drug arimoclomol, and whether it improves quality of life and progression of the disease. For further information on the trial, please visit its website.
Arimoclomol has been granted orphan designations by both the EMA and FDA, and the company has raised over $40 milion to develop arimoclomol and two other compounds that are still in the preclinical stages. Their investor base include Novo A/S, Sunstone Capital, Aescap Venture, Kurma Partners and Idinvest Partners, who must be intrigued about the company’s vision of pioneering heat shock protein based therapies. The startup might be one of the most exciting companies to follow in Scandinavia at the moment!