South Korean biopharmaceutical company GC Biopharma Corp (KRX:006280) and emerging biotechnology company Novel Pharma Inc announced on Monday that the first patient in the United States has been dosed in a multinational clinical trial with GC1130A, a new drug for Sanfillippo syndrome type A (MPS IIIA).

MPS IIIA is an autosomal recessive genetic disorder that damages the central nervous system due to the accumulation of heparan sulphate.

GC Biopharma and Novel Pharma have received IND approval for a Phase 1 clinical trial of GC1130A in the US, Korea and Japan. They plan to assess the safety, tolerability and efficacy of GC1130A over a period of two years for children aged between two and six diagnosed with MPS IIIA.

Patients who meet eligibility criteria after screening will undergo surgery to implant an intracerebroventricular (ICV) access device and will receive GC1130A once every two weeks.

A first-in-class treatment, GC1130A uses GC Biopharma's proprietary platform to produce concentrated, high quality recombinant protein which is administered through ICV injection to bypass the blood brain barrier. GC Biopharma produced the first ICV administered enzyme replacement therapy (ERT) for patients with Hunter syndrome, and marketed it in Japan under the brand name Hunterase ICV.