GC Biopharma Presents Updates on its LSD Treatments at the WORLDSymposium 2024

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  • Recent updates on its development of LSD treatments were presented

YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ — GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.

WorldSymposium 2024 is an international forum for Lysosomal Diseases experts to share and exchange insights for researching better treatment of the disease.

At this year’s symposium, GC Biopharma made an oral presentation on the Phase III clinical trial result of “Hunterase” its Hunter syndrome treatment and released posters on the non-clinical results of ‘GC1130A’, a treatment for Sanfilippo syndrome type A (MPS IIIA), and GC1134A/HM15421, a treatment for Fabry disease.

Hunter syndrome, also known as Mucopolysaccharidosis type II, is a rare disorder with a prevalence of one in 100,000 to 150,000 male live births. Various unpredictable symptoms, such as skeletal abnormalities and intelligence decline, may develop. In severe cases, this leads to deaths before the age of 15. Currently, 70 to 80 patients are reported to have this disorder in Korea.

In 2012, GC Biopharma succeeded in developing the world’s second treatment for Hunter syndrome, “Hunterase” solely using domestic technology. By improving utilization of genetic recombinant technology and purified IDS enzyme, administered intravenously, GC Biopharma has provided a better treatment option for patients with Hunter syndrome, who previously solely depended on a single therapy option.

This Phase III trial was conducted on Hunter syndrome patients aged 5 years or older without previous treatment experience in enzyme replacement therapy. By comparing the patient group that received ‘Hunterase’ intravenously once a week for 52 weeks with the placebo control group, the clinical trial proved the superiority of the treatment with a statistically significant difference in 6-Minute Walk Test [6-MWT] and urinary glycosaminoglycan (urine GAG) results.

The company’s presentation also included posters on non-clinical results of ‘GC1130A’, a treatment for Sanfilippo syndrome type A (MPS IIIA), and GC1134A/ HM15421, a treatment for Fabry disease.

Sanfilippo syndrome (type A) is a recessive genetic disease in which heparan sulfate accumulates in the body due to a genetic defect, causing gradual and severe damage mainly in the brain. This rare disease leads most patients to death at around the age of 15. With no approved treatment available thus far, there exists significant unmet medical needs among patients.

GC Biopharma, in collaboration with Nobel Pharma, is developing an enzyme replacement therapy (ERT) for the disorder, administering intracerebroventricular injection (ICV) of the enzyme (heparan N sulfatase) that is not expressed in the patient’s body to treat brain lesions in MPS IIIA patients.

GC1130A has received Orphan Drug Designation (ODD) from the US FDA and the European EMA based on the efficacy and safety proven in the non-clinical stage. In the poster presentation, the company presented the significant reduction in Heparan sulfate and improvement in brain lesion demonstrated in mice with MPS III type A disease.

Fabry disease involves a deficiency of alpha-galactosidase that causes the body to accumulate glycolipids, leading to damages mainly in heart and kidney. While an approved enzyme replacement therapy for the disorder already exists, there are still unmet medical needs for kidney disease. GC Biopharma, together with Hanmi Pharmaceutical, is developing GC1134A/HM15421, a long-acting alpha-galactosidase that can be administered subcutaneously once a month to improve patient convenience.

The poster presentation by Hanmi Pharmaceutical included the result of animal model evaluation showing continued distribution, kidney function improvement, and fibrosis inhibition efficacy of GC1134A/HM15421 compared to existing treatments.

GC Biopharma commented, “We will continue to build on our experience, knowledge, and expertise in developing treatments for lysosomal storage diseases, and expand our reach to other rare diseases to provide new treatment options to the patients.”

About GC Biopharma

GC Biopharma (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century.

This press release may contain biopharmaceuticals in forward-looking statements, which express the current beliefs and expectations of GC Biopharma’s management. Such statements do not represent any guarantee by GC Biopharma or its management of future performance and involve known and unknown risks, uncertainties and other factors. GC Biopharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule.

GC Biopharma Contacts (Media)

Seunghyun Baek

Sohee Kim

Yelin Jun

Rachel Kim


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