GC Biopharma and Novel Pharma Begin Global Phase I Trial for MPS IIIA Treatment

In a significant development in the field of biotechnology, GC Biopharma and Novel Pharma announced that the first patient in the United States has been dosed with 'GC1130A', a groundbreaking new drug intended to treat Sanfillippo syndrome type A (MPS IIIA). This marks the start of a multinational clinical trial that has received Investigational New Drug (IND) clearance for Phase I trials in the US, Korea, and Japan.

The clinical trial is designed to evaluate the safety, tolerability, and efficacy of 'GC1130A' over a two-year period in children aged two to six who have been diagnosed with Sanfillippo syndrome type A. This rare genetic disorder, also known as MPS IIIA, is characterized by the accumulation of heparan sulfate in the central nervous system, leading to severe neurological deficits and typically resulting in death by the age of 15. As of now, there is no available treatment for this devastating condition.

'GC1130A' is a pioneering treatment developed using GC Biopharma's proprietary technology, which produces a highly concentrated and high-quality recombinant protein. This protein is administered directly into the brain through an intracerebroventricular (ICV) injection, a method designed to bypass the blood-brain barrier and deliver the treatment more effectively to the brain. The innovation behind 'GC1130A' builds on GC Biopharma's earlier success with Hunterase ICV, the first enzyme replacement therapy (ERT) administered via ICV for patients with Hunter syndrome, which is currently marketed in Japan.

The Phase I clinical trial will be conducted at multiple sites, including two to three institutions in the US, such as the University of California San Francisco (UCSF) Benioff Children's Hospitals, and institutions in Korea, including Samsung Medical Center and Ajou University Hospital, as well as one institution in Japan. Eligible patients will undergo surgery to implant an ICV access device and will receive doses of 'GC1130A' every two weeks.

Non-clinical studies have demonstrated that ICV injections can be up to 47 times more effective than intrathecal (IT) injections, another method of delivering medication to the central nervous system. This significant effectiveness highlights the potential of 'GC1130A' to provide a much-needed treatment option for patients with MPS IIIA.

SooKyung Shin, the Head of the Medical Division at GC Biopharma, expressed optimism about this milestone, stating, "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."

GC Biopharma, formerly known as Green Cross Corporation, is a prominent biopharmaceutical company based in Yongin, South Korea. The company has a long history of providing life-saving protein therapeutics and vaccines and is recognized as a global leader in plasma protein and vaccine product manufacturing.

Novel Pharma, headquartered in Seoul, South Korea, is a biotechnology company focused on developing innovative treatments for rare pediatric diseases, particularly those involving the central nervous system. Their work includes addressing conditions such as MPSIIIA, GM1, MPSIVB, and Krabbe disease, using intracerebral ventricular (ICV) administration to deliver treatments directly to the brain.

The collaborative efforts of GC Biopharma and Novel Pharma represent a promising step forward in the quest to find effective treatments for rare genetic disorders, offering hope to patients and families affected by these challenging conditions.