Gilva Therapeutics Co., Ltd.
No. 18, Siyuan St., Zhongzheng Dist., Taipei City 100047 ,
Taiwan (R.O.C.) (Room 909 of Complex for Research Excellence)
Registration No.：89114272
TEL：+886 2 33661914
FAX：+886 2 23652804
GV-100
GV-100 is an oral selective HDAC6 inhibitor. HDAC6 interacts with various substrate proteins, regulating numerous cell physiological or pathological mechanisms. It exerts functions such as anti-inflammatory, anti-fibrotic, and cytoprotective effects by modulating post-translational modifications of crucial proteins within cells. HDAC6 is also overexpressed in ADPKD, fibrotic diseases, neurodegenerative disorders, and cancers, highlighting its significant role in various diseases.
A highly selective HDAC6 inhibitor, GV-100, retains the pharmacological properties of HDAC inhibitors while offering superior safety compared to non-selective HDAC inhibitors. GV-100 has demonstrated anti-inflammatory and anti-fibrotic properties in various cellular and animal studies. Additionally, GV-100 can ameliorating kidney cyst in ADPKD disease animal model. GV-100 can promise more safe and effective treatment options for ADPKD patients in the future.
Autosomal dominant polycystic kidney disease (ADPKD)
Autosomal dominant polycystic kidney disease (ADPKD), is a rare genetic disorder characterized by slowly progressive, bilateral kidney enlargement due to numerous fluid-filled cysts. Clinical symptoms of ADPKD include hematuria, abdominal pain (due to enlarged kidneys), and kidney stones. Cysts progressively replace and damage normal kidney tissue, leading to kidney fibrosis, structural abnormalities, and eventually end-stage renal disease, posing significant clinical and economic burdens on healthcare systems worldwide.
Approximately 85% of cases are caused by mutations in the pkd1 gene (encoding PC1 protein). Severity of renal cyst and renal function correlated with the expression level of PKD1 expression. Therefore, PC1 has proven to be an important target for the treatment of ADPKD.
JYNARQUE (Tolvaptan) is the first and currently the only drug approved by the U.S. Food and Drug Administration (FDA) for ADPKD treatment. It is a selective vasopressin V2-receptor antagonist that slows cyst growth by increasing renal fluid excretion. However, it does not address the epithelial cell proliferation issue caused by the loss function of PC1. Despite its approval, JYNARQUE carries a black box warning due to serious and potentially fatal liver injury. Much unmet need remains in ADPKD.