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GC Biopharma and Novel Pharma Initiate Phase I Trial of GC1130A for Sanfilippo Syndrome Type A

• GC Biopharma and Novel Pharma have dosed the first patient in a multinational Phase I clinical trial of GC1130A for Sanfilippo syndrome type A (MPS IIIA). • The Phase I trial will assess the safety, tolerability, and efficacy of GC1130A in children aged 2-6 years with MPS IIIA over a two-year period. • GC1130A is a first-in-class treatment using GC Biopharma’s platform to deliver concentrated recombinant protein via intracerebroventricular (ICV) injection. • The trial will be conducted across multiple sites in the US, Korea, and Japan, addressing a critical unmet need for this devastating genetic disorder.

GC Biopharma and Novel Pharma have announced the dosing of the first patient in a multinational Phase I clinical trial of ‘GC1130A’, a novel drug for Sanfilippo syndrome type A (MPS IIIA). The trial, which has received IND clearance in the US, Korea, and Japan, aims to evaluate the safety, tolerability, and efficacy of GC1130A in children aged between two and six years diagnosed with MPS IIIA over a two-year period.
This Phase I clinical trial will be conducted at multiple institutions, 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. Eligible patients will undergo surgery to implant an intracerebroventricular (ICV) access device and will receive ‘GC1130A’ once every two weeks.

Addressing Unmet Needs in MPS IIIA

Sanfilippo syndrome type A (MPS IIIA) is an autosomal recessive genetic disorder characterized by the accumulation of heparan sulfate, leading to damage in the central nervous system. The primary clinical manifestation is brain deficit, with patients typically succumbing to the disease around the age of 15. Currently, there are no effective treatment options available, highlighting a significant unmet medical need.

GC1130A: A Novel Approach

‘GC1130A’ represents a first-in-class treatment utilizing GC Biopharma’s proprietary platform to produce concentrated, high-quality recombinant protein. This protein is administered via ICV injection to bypass the blood-brain barrier, a method GC Biopharma has experience with, having developed the first ICV administered enzyme replacement therapy (ERT) for Hunter syndrome, marketed in Japan as Hunterase ICV. Preclinical studies have demonstrated that ICV injection is up to 47 times more effective than intrathecal (IT) administration.
SooKyung Shin, Head of Medical Division at GC Biopharma, stated, “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.”

Trial Design and Objectives

The Phase I clinical trial will primarily focus on assessing the safety and tolerability of GC1130A. Secondary endpoints will include evaluating the drug's efficacy in reducing heparan sulfate accumulation and improving neurocognitive outcomes in MPS IIIA patients. The trial's two-year duration will allow for a comprehensive evaluation of the long-term effects of GC1130A.
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Reference News

[1]
GC Biopharma and Novel Pharma First Patient Dosed in Global Phase I Clinical Trial for ...
biospace.com · Nov 25, 2024

GC Biopharma and Novel Pharma dosed the first US patient in a multinational Phase I clinical trial for ‘GC1130A’, an inn...

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