GC Biopharma/Novel Pharma's Sanfilippo Syndrome Treatment Obtains FDA IND Clearance

2024-05-21

孤儿药临床申请疫苗临床研究

YONGIN, South Korea, May 21, 2024 /PRNewswire/ -- GC Biopharma (006280. KS) and Novel Pharma have announced that the U.S. FDA has cleared the investigational new drug (IND) application for their jointly developed MPSIIIA (Sanfilippo Syndrome Type A) treatment, GC1130A.

With this FDA clearance, the development of GC1130A is set to accelerate, with multinational clinical trials expected to commence this year. Clinical trial sites have been established in the United States, South Korea, and Japan, focusing on demonstrating the drug's safety and tolerability.

GC Biopharma and Novel Pharma have been co-developing this intracerebroventricular enzyme replacement therapy (ICVa ERT) for treatment for MPSIIIA patients who lack Heparan N Sulfatase.

GC1130A has received Orphan Drug Designation (ODD) from both FDA and EMA based on the robust safety and efficacy profile observed during preclinical studies. It also received Rare Pediatric Disease Designation (RPDD) from the FDA.

Sanfilippo Syndrome Type A is a genetic disorder that causes central nervous system damage through the accumulation of heparan sulfate, leading to progressive neurological decline. Without treatment, patients often face life-threatening complications by the age of 15. GC1130A represents a potential breakthrough as the first and only treatment option specifically targeting MPSIIIA.

GC Biopharma's representative further emphasized, "The IND clearance for GC1130A marks a significant milestone in our mission to bring hope to patients and families affected by Sanfilippo Syndrome. We are committed to advancing to phase 1 clinical trials and are dedicated to the development of new therapies, that address unmet medical needs.

About GC Biopharma

Corp.

GC Biopharma Corp. (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 Corp. 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.

About Novel Pharma Inc.

Novel Pharma is an emerging biotechnology company that focuses on developing first-in-class treatments for rare pediatric diseases (MPS/LSD) utilizing ICV (intracerebral ventricular) administration. Headquartered in Seoul, South Korea, it is currently engaged in the development of (i) MPSIIIA, (ii) GM1, (iii) MPSIVB and (iv) Krabbe disease.

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 Corp. Contacts (Media)

Sohee Kim

[email protected]

Yelin Jun

[email protected]

Rachel Kim

[email protected]

SOURCE GC Biopharma

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