Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium

Orphazyme US
Investor news                                                                                                         
No. 02/2021                                                                                                                                    

Chicago, USA, February 4, 2021 – Orphazyme US (ORPH), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announced plans to present a series of data on its investigational drug arimoclomol during the 17^th Annual WORLDSymposium Scientific Meeting, to be held virtually on February 8-12, 2021.

“The WORLDSymposium, while virtual this year, offers an important opportunity to present the mechanisms of the Heat-Shock Protein response and how that translates into potential therapeutic utility for lysosomal diseases such as Niemann-Pick disease Type C (NPC),” said Daniel Gallo, Head, U.S. Medical, Orphazyme. “We look forward to convening with the research community during the conference and to showcasing these data in support of arimoclomol.”

Arimoclomol data being presented at the virtual WORLDSymposium are:

• Persistent effect of arimoclomol in patients with Niemann-Pick disease Type C: 12-month results from an open-label extension of a pivotal phase 2/3 study
  • Presented by Marc Patterson, M.D.
  • Wednesday, February 10, Poster 191
     
• Pharmacokinetics properties of arimoclomol in Niemann-Pick disease Type C: Modest and not clinically relevant effect of bodyweight or age
  • Presented by Thomas Anderson
  • Thursday, February 11, Poster 5