Orphazyme in final preparation stages for a Phase II/III clinical trial

Orphazyme prepares for Phase II/III registration-enabling clinical trial in Niemann-Pick disease type C with Arimoclomol

As a follow-up on the presentation delivered at the Niemann-Pick UK (NP-UK) meeting (26-27 September 2014), it is with great anticipation and pleasure that we announce that Orphazyme is now in the final preparation stages for a Phase II/III registration-enabling clinical trial in Niemann-Pick disease type C with Arimoclomol, a small molecule inducer of heat shock proteins.

As presented at the NP-UK family conference, Orphazyme has obtained very promising data from its preclinical studies with Arimoclomol in cells from patients, as well as in the Niemann-Pick disease type C mouse model.

This work has been done in collaboration with Professor Frances Platt, University of Oxford.

Arimoclomol is orally available and its safety profile has been established through extensive and multiple Phase I clinical trials.

In order to reach a trial design from a clinical and regulatory perspective that allows the appropriate assessment of the efficacy of arimoclomol, Orphazyme has sought the guidance of the regulatory authorities as well as clinicians and other stakeholders.

The clinical programme is composed of an Observational Study, expected to start a staggered enrolment in the first quarter of 2015 (March 2015). After 6 months of follow-up, all the patients that complete the Observational Study will enter (upon consent) into the Interventional Phase II/III trial with Arimoclomol.

This will be a multi-centre trial and work is now underway to establish clinical centres in both Europe and the USA. These will be announced once discussions are completed.

Key proposed inclusion criteria for participation in the Observational Study:

  • NPC diagnosis genetically confirmed
  • Male/female aged 2-18 (+11 months)
  • Ability to walk independently or with assistance
  • Treated or not treated with Miglustat

Key proposed exclusion criteria:

  • Severe liver insufficiency or liver transplant
  • Patients with established epileptic “seizures period” (>3 epileptic seizures that required medication within 6 months prior to Observational Study)
  • Treatment with Cyclodextrin or any other Investigational Medicinal Product (IMP) within 4 weeks prior to study enrolment

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

About Orphazyme

Orphazyme ApS is a Danish biopharmaceutical company, which develops paradigm-changing medicines for the treatment of genetic diseases. The lead program is in preclinical development as treatment for lysosomal storage diseases. This family of genetic disorders consists of more than 45 diseases, most of which are currently untreatable and often fatal. Orphazyme has raised more than 17 million euro in seed and series A financing, from a strong investor base which includes Novo Ventures, Sunstone Capital and Aescap Venture. For more information, please visit www.orphazyme.com.