Ferrier science embedded in biotech drug development

A new drug to treat a rare muscle-wasting disease, and manufactured using Ferrier Research Institute technology, has been acquired for commercial development.

A new drug developed to treat a debilitating rare muscle-wasting disease and various kidney diseases, and manufactured using Ferrier Research Institute technology, has been acquired by an American company for commercial development.

New Zealand Pharmaceuticals Limited (NZP) announced last week that the Patent License and related Agreements for DEX-M74 has transferred to Altamira Bio, a subsidiary of NASDAQ-listed Fortress Biotech, who will run the clinical development and commercialisation.

DEX-M74, a natural carbohydrate, is currently in a Phase 2 clinical trial for treatment of the rare genetic disorder GNE myopathy, and a Phase 1 trial in patients with kidney disease is scheduled to begin in September.

GNE myopathy, formerly known as Hereditary Inclusion Body Myopathy, is a severe debilitating muscle wasting disease afflicting approximately 2,000 people worldwide. Most patients develop weakened arm, hand and leg muscles in their early 20s and eventually require a wheelchair.

“This is a significant milestone. NZP manufactures DEX-M74 using technology developed and licensed from Ferrier”, says Director of the Ferrier Research Institute Professor Richard Furneaux.

“In fact, the late Professor Robin Ferrier, after whom our Institute is named, was instrumental in developing the manufacturing process when he worked with us in his retirement.”

DEX-M74 has the potential to supplement the patient’s genetic insufficiency by restoring the sialic acid content of their diseased tissues to natural levels.

“Low sialic acid levels are also a feature of many major kidney diseases, so there is cautious optimism that it will have broader applications”, says Professor Furneaux.

Professor Furneaux says Palmerston North-based NZP is going from strength to strength. “We have had a long and productive relationship with NZP. Their Business Development Manager Dr Selwyn Yorke, who has championed the development of DEX-M74, was member of our research team at one point.”