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
shkim20@gccorp.com
Yelin Jun
yelin@gccorp.com
Rachel Kim
rachel.kim@gccorp.com
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SOURCE GC Biopharma