FDA Grants RMAT Designation to RZ-001 for Hepatocellular Carcinoma

The FDA has granted a regenerative medicine advanced therapy (RMAT) designation to RZ-001 for the treatment of patients with hepatocellular carcinoma (HCC), according to Rznomics, the company developing the investigational targeted therapy. ¹

The RMAT designation, which is designed to accelerate the development and review of regenerative medicine therapies in areas of high unmet medical need, is backed by initial clinical data showing promise for RZ-001 in HCC. The FDA previously granted RZ-001 both orphan drug designation and fast track designation for HCC.

"With the RMAT designation, we plan to accelerate our US development and partnership initiatives by initiating formal discussions with the FDA regarding clinical trial design, Chemistry, Manufacturing, and Controls, and commercialization strategies," Sung-woo Hong, vice president of Rznomics stated in a press release.¹

RZ-001 is designed to selectively target and cleave human telomerase reverse transcriptase (hTERT) mRNA, thereby suppressing hTERT expression in cancer cells. The agent also exerts cytotoxic activity by inserting an HSVtk-encoding sequence into reprogrammed hTERT mRNA through trans-ligation. In addition, RZ-001 has been shown to enhance immune cell infiltration within tumors, decrease VEGF expression, and alter the tumor microenvironment in ways that may improve sensitivity to immunotherapy.¹

Interim phase 1b/2a study data for RZ-001 were presented at the 2026 American Association for Cancer Research (AACR) Annual Meeting by Professor Yoon-Jun Kim of the Department of Gastroenterology at Seoul National University Hospital.² Patients enrolled in the study had HCC and were refractory to or ineligible for transarterial chemoembolization. Study participants had not been treated with prior systemic therapy.

The results showed that among patients treated with RZ-001 in combination with atezolizumab (Tecentriq) and bevacizumab (Avastin), the confirmed objective response rate (ORR) was 38.5% by RECIST v1.1 criteria, with an unconfirmed ORR of 46.2%. At the time of the interim analysis, confirmed responses were defined as those validated through subsequent assessments, while unconfirmed responses reflected initial tumor reductions of at least 30% that had not yet been re-evaluated.

Under mRECIST criteria the ORR reached 61.5%, with a complete response rate of 23%. Because mRECIST captures tumor necrosis that RECIST may miss, this finding suggests RZ-001 may be inducing deep biological responses beyond what standard anatomical measurements reflect. The complete response rate in particular is noteworthy given the historically poor outcomes in this patient population following TACE failure.²

The safety data showed that among all patients enrolled, there were 5 grade 3 or higher adverse events reported and attributed to the combination agents. These included 2 cases of hypertension and 1 case each of proteinuria, hyperglycemia, and gastrointestinal bleeding. This safety profile is consistent with the known toxicities of atezolizumab and bevacizumab. Importantly, there were no grade 3 or higher adverse events reported that were attributed to treatment with RZ-001 itself. The absence of any serious treatment-related toxicities linked to the investigational agent is a meaningful finding given the fragile nature of patients with advanced liver disease and cirrhosis, who often have limited tolerance for additional hepatotoxic insults.

"Receiving RMAT designation for RZ-001 is a profound validation of the innovation and competitiveness of our RNA editing platform by the FDA. We will concentrate our resources on global development and commercialization to provide a breakthrough therapeutic option in the field of HCC, where unmet medical needs remain exceptionally high," Seong-Wook Lee, CEO of Rznomics, stated in the news release.¹

REFERENCES

1. Rznomics Inc. Rznomics announces U.S. FDA Regenerative Medicine Advanced Therapy designation granted to 'RZ-001' for hepatocellular carcinoma. PR Newswire. Published May 8, 2026. https://tinyurl.com/4j2aw7uk

2. Rznomics Inc. Rznomics reports interim clinical data for RZ-001 in hepatocellular carcinoma at AACR 2026 demonstrating encouraging efficacy and favorable safety profile. PR Newswire. Published April 20, 2026. https://tinyurl.com/yhrb9pya