NZP to produce drug for NIH trial

New Zealand Pharmaceuticals (NZP) has announced it will be involved in a phase I trial treating rare genetic disease Hereditary Inclusion Body Myopathy (HIBM) launched by the US National Institutes of Health (NIH).

NZP, a contract manufacturing pharmaceutical company, will produce the compound, DEX-M74, for the trial.

NZP holds a license to develop DEX-M74 when clinical data is available supporting the drug's safety and efficiency, both in HIBM and in glomerular kidney diseases.

Patients with HIBM suffer from progressive muscle weakness, usually beginning from early adulthood, and eventually require a wheelchair as their leg and arm muscles weaken.

A suspected cause is a low level of sialic acid in muscle proteins due to a mutation in the GNE gene, which normally produces the acid. Sialic acid is important to muscle development, as well as kidney function.

DEX-M74 is a sugar converted by the body into sialic acid. It was found to be effective in controlling muscle weakness in mice with a specific GNE mutation during preparatory animal trials.

The research into the use of the compound to treat HIBM is being led by NIH's National Center for Advancing Translational Sciences (NCATS) and National Human Genome Research Institute (NHGRI).

The phase I trial will focus on safety and tolerability and involve a single dose. But researchers plan to follow up with a multi-dose phase IIa trial.