Orphazyme Forms Scientific Advisory Board and Confirms the Enrolment Initiation for the Clinical Programme

Orphazyme Forms Scientific Advisory Board and Confirms the Enrolment Initiation for the Clinical Programme of Arimoclomol in Niemann-Pick Disease Type C in June 2015

Orphazyme today announced the assembly of its Scientific Advisory Board (SAB) to support the upcoming clinical trial in Niemann-Pick disease type C.

The SAB comprises world-leading medical, clinical and scientific professionals in lysosomal storage diseases, including Niemann-Pick disease type C: Prof. Marc Patterson (USA, Mayo Clinic), Dr. Christine í Dali (Denmark, Rigshospitalet), Prof. Mark Walterfang (Australia, University of Melbourne) and Prof. Frances Platt (UK, University of Oxford).

"It is with great pleasure that we can present this exceptional group from Europe, USA and Australia as our SAB,” Anders Hinsby, Chief Executive Officer of Orphazyme announced. ”They will be able to provide cutting edge expertise and experience to our clinical programme of assessing arimoclomol, an orally available therapy, for Niemann-Pick disease type C.”

Carlos Camozzi, Chief Medical Officer, adds: ”Orphazyme is excited about the continuous collaboration with the SAB members and expert clinicians throughout Europe on our Niemann-Pick disease type C clinical programme. We have planned an investigator meeting in May to effectively start patient recruitment in June.”

Further information: 
Anders Hinsby, CEO of Orphazyme
E-mail: amh@orphazyme.com

Carlos Camozzi, CMO of Orphazyme
E-mail: crc@orphazyme.com

About arimoclomol

Arimoclomol is an orally available small molecule inducer of the heat shock response. The molecule has been well-tolerated in multiple clinical studies and is now entering a phase II/III study in Niemann-Pick disease type C (NP-C).

Arimoclomol works by harnessing the cell protective properties of the heat shock proteins (HSPs). In many inherited diseases, such as Niemann-Pick disease type C, the pathology is caused by the dysfunction of one or more proteins as a consequence of genetic mutations. HSPs are so-called chaperone proteins, which are essential for maintaining and restoring proper protein function and has been shown to alleviate disease in several preclinical studies, including Niemann-Pick disease type C (Kirkegaard et al. manuscript submitted for publication 2015). Moreover, HSP70 has been shown to be effective in correcting conditions of lysosomal deficiency. This latter function is achieved by a mechanism involving binding to the inner lysosomal membranes and working as a co-factor for the proper function of a number of lysosomal enzymes (Kirkegaard et al. Nature 2010, 463:549-53).

Arimoclomol as a treatment for Niemann-Pick disease type C has been granted Orphan Drug Status in the US and EU.

About Orphazyme

Orphazyme ApS is a Danish biopharmaceutical company, which develops paradigm-changing medicines for the treatment of genetic diseases. The lead program is entering clinical development as treatment for lysosomal diseases. This family of genetic disorders includes NP-C and consists of more than 45 diseases, often affecting children, most of which are currently untreatable and often fatal. Orphazyme has raised more than €37M in seed, Series A and Series B financing from a strong investor base, which includes Novo A/S, Sunstone Capital, Aescap Venture, Kurma Partners and Idinvest Partners. For more information, please visit www.orphazyme.com.