Rznomics announced on February 14 that its RNA-based anticancer candidate, RZ-001, has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the treatment of hepatocellular carcinoma (HCC). RZ-001 utilizes the company’s proprietary RNA trans-splicing ribozyme platform technology.

This is the second Fast Track designation for RZ-001, following its designation for glioblastoma in November 2023. Rznomics has also received FDA approval for an Expanded Access Program (EAP) for glioblastoma, which is currently underway at Harvard Medical School-affiliated hospitals.

RZ-001 is being developed as a treatment for both liver cancer and glioblastoma, with ongoing Phase 1b/2a trials for HCC and Phase 1/2a trials for glioblastoma, following IND approvals from the Korean Ministry of Food and Drug Safety (MFDS) and the FDA.

The therapy leverages Rznomics' RNA trans-splicing ribozyme platform, delivered via an adenoviral vector to selectively target human telomerase reverse transcriptase (hTERT) RNA expressed in cancer cells. Simultaneously, it induces expression of the HSVtk gene, which triggers anticancer activity.

Seong-Wook Lee, CEO of Rznomics, stated, “We are committed to accelerating the clinical development of RZ-001 to provide effective treatment options for patients suffering from hard-to-treat cancers.”

The FDA’s Fast Track designation is granted to therapies that address serious or life-threatening conditions, facilitating faster development and regulatory review. It allows for more frequent interactions with the FDA, rolling review of the drug’s application, and potentially expedited approval.