Tides Express | HaiChang Bio's Antisense Oligonucleotide AKT-1 Inhibitor Receives CDE Approval for Domestic Clinical Trial

20April 2, 2024On the 5th, the official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration announced that the antisense oligonucleotide drug WGI-0301 (alias "HC0301"), developed by Zhejiang Haichang Biomedical Technology Co., Ltd. (hereinafter referred to as "Haichang Biotech"), has received tacit approval from CDE for clinical trials and will carry out its first clinical trial in China.

WGI-0301 is a next-generation AKT-1 inhibitor (serine/threonine protein kinase AKT inhibitor) developed using QTsomeTM nucleic acid delivery technology, which encapsulates the antisense oligonucleotide Archexin within lipid nanoparticles (LNP), for the treatment of advanced hepatocellular carcinoma. The drug specifically binds to AKT-1 mRNA, thereby reducing AKT-1 protein expression. This unique mechanism demonstrates significant potential and value in anti-tumor therapy, offering broad developmental prospects for future research and treatment.

The AKT-1 target acts on the PI3K/AKT/mTOR signaling pathway, playing a crucial role in cancer cell proliferation, survival, angiogenesis, metastasis, and drug resistance. It is overexpressed in various cancers, including liver cancer, kidney cancer, breast cancer, colorectal cancer, gastric cancer, and pancreatic cancer. WGI-0301 exhibits highly selective specificity and low propensity for drug resistance. Utilizing Haichang Biotech's globally intellectual property-protected QTsomeTM nucleic acid delivery technology, it addresses key issues such as low efficiency of nucleic acid uptake and insufficient targeting, effectively meeting the needs for liver cancer treatment.

As early as October 2023, WGI-0301 received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA). This designation was based on a Phase I dose-escalation clinical study (NCT05267899) conducted in the United States for advanced solid tumors, which demonstrated excellent safety and efficacy. In early February this year, WGI-0301 (HC0301) received FDA approval for Phase II clinical trials by default and will initiate an international multicenter clinical study for advanced liver cancer at the Marlene and Stewart Greenebaum Cancer Center at the University of Maryland.