Company Profile
Orphazyme US, Inc.
Company Overview
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.
Company History
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.
Notable Accomplishments / Recognition
Orphazyme is developing arimoclomol in four indications. Orphazyme has fully recruited a Phase II/III trial in NPC (trial results expected Q3 2018), initiated a Phase II/III trial in sIBM in August 2017 and plans to start a Phase II/III trial in ALS in Q3 2018.
In addition, a Phase II Proof-of-Concept trial in Gaucher disease will be initiated in Q2 2018. If positive, all three Phase II/III trials are intended to form basis for a single study filing in each respective indication.
The Company expects to have completed all three phase II/III trials by the end of 2020, with the first potential marketing authorization in 2020.