Phase 1/2a clinical trial in patients with liver cancer that does not respond to transarterial chemoembolization

Rznomics (CEO Seong-wook Lee) announced on the 22nd that its developmental cancer treatment, “RZ-001(developmental code name), targeting hepatocellular carcinoma, has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).

With Orphan Drug Designation, the company becomes eligible for benefits such as tax credits for research and development expenses, exemption from approval application fees, and a seven-year market exclusivity period starting from the approval date. The designation is expected to provide favorable conditions during the priority and accelerated review decisions.

RZ-001 is currently undergoing Phase 1/2a clinical trials, having received approval for its clinical trial plan from the Ministry of Food and Drug Safety in Korea and the FDA. The trial focuses on patients with hepatocellular carcinoma who do not respond to transarterial chemoembolization(TACE) and aims to develop a treatment option that can be prioritized before using systemic therapies.

Seong-wook Lee, CEO of Rznomics, said, "Effective treatments for hepatocellular carcinoma are urgently needed at various stages, and we will do our best to make RZ-001 a new therapeutic option."

In addition to the ongoing clinical development, Rznomics has expanded the application of RZ-001 to include glioblastoma, receiving Fast Track designation from the FDA  in November last year. The company is actively engaged in global clinical development through collaborations with pharmaceutical companies, including a clinical partnership with Roche.