GC1130A / GC Biopharma, Novel Pharma - LARVOL DELTA

GC Pharma…announced on the 5th that its Sanfilippo Syndrome Type A treatment (Project Name: GC1130A), which it is developing in-house, has received orphan drug designation from the Ministry of Food and Drug Safety (MFDS). [Google translation] (BioTimes)

Orphan drug • Lysosomal Storage Diseases

GC service, ’GC2126A’ non-clinical, etc. announced at the Lysosomal Disease WORLD Symposium [Google translation] (HIT News) - "At the symposium, the company presented posters on the non-clinical results of its oral chaperone treatment 'GC2126A' for 'GM1 gangliosidosis (GM1)' and the non-clinical results of its treatment 'GC1130A' for Sanfilippo syndrome type A (MPSIIIA), while also identifying the latest research trends in various rare diseases...In this poster presentation...shared the non-clinical results showing that a new candidate for an oral chaperone treatment for GM1 confirmed a dose-dependent increase in β-galactosidase activity in various tissues, including the brain, in a study of diseased animals, and that oral administration for 7 days showed an effect of reducing GM1-gangliosidosis accumulation in the brain by more than 70%....In the poster presentation of the nonclinical results of GC1130A, a treatment for Sanfilippo syndrome type A (MPSIIIA), the nonclinical results showing that drug delivery via intracerebroventricular (ICV) administration is more effective..."

Preclinical • Lysosomal Storage Diseases

GC Biopharma and Novel Pharma First Patient Dosed in Global Phase I Clinical Trial for MPS IIIA Treatment (PRNewswire) - "GC Biopharma and Novel Pharma today announced that the first patient in the US has been dosed in multinational clinical trial with 'GC1130A', an innovative new drug for Sanfillippo syndrome type A (MPS IIIA).....This Phase I clinical trial will be conducted at two to three institutions in the US 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. Patients who meet eligibility criteria after screening will undergo surgery to implant an intracerebroventricular (ICV) access device and will receive 'GC1130A' once every two weeks."

Trial status • Genetic Disorders • Metabolic Disorders • Rare Diseases

Phase 1 Study of GC1130A in Pediatric Patients With Sanfilippo Syndrome Type A (MPS IIIA) (clinicaltrials.gov) - P1 | N=9 | Recruiting | Sponsor: GC Biopharma Corp | Not yet recruiting ➔ Recruiting

Enrollment open • Lysosomal Storage Diseases • Pediatrics

Phase 1 Study of GC1130A in Pediatric Patients With Sanfilippo Syndrome Type A (MPS IIIA) (clinicaltrials.gov) - P1 | N=9 | Not yet recruiting | Sponsor: GC Biopharma Corp

New P1 trial • Lysosomal Storage Diseases • Pediatrics

GC Biopharma/Novel Pharma’s Sanfilippo Syndrome Treatment Obtains FDA IND Clearance (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...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."

IND • New trial • Orphan drug • Genetic Disorders