GC Biopharma and Novel Pharma announced that the first patient in the US has been dosed in multinational clinical trial with 'GC1130A', an innovative new drug for Sanfillippo syndrome type A (MPS IIIA).

GC Biopharma and Novel Pharma have received IND clearance for Phase I clinical trial of 'GC1130A' in the US, Korea, and Japan and are currently conducting multi-national clinical trial. Through this Phase I clinical trial, they plan to evaluate the safety, tolerability, and efficacy of 'GC1130A' over a two-year period for children aged between two and six diagnosed with Sanfillippo syndrome type A (MPS IIIA).

This Phase I clinical trial will be conducted at two to three institutions in the US including University of California San Francisco (UCSF) Benioff Children's Hospitals, Samsung Medical Center and Ajou University Hospital in Korea, and one institution in Japan. 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.

Sanfillippo syndrome type A (MPS IIIA) is an autosomal recessive genetic disorder that damages the central nervous system due to the accumulation of heparan sulfate. The main clinical manifestation is brain deficit, and it is a devastating disease where patients die around the age of 15. As no treatment option currently exists, there is a huge unmet medical need.

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

In a non-clinical study, GC Biopharma has proven that ICV injection is up to 47 times more effective than that of intrathecal (IT).

SooKyung Shin, Head of Medical Division at GC Biopharma commented, "The initiation of patient dosing in the US is a significant milestone for 'GC1130A' global clinical trials and we will do our best to expedite the clinical development process."