Rznomics has presented interim clinical data from its ongoing study of RZ-001, an RNA editing-based investigational anticancer therapy, in patients with hepatocellular carcinoma (HCC) during an oral presentation at the AACR 2026 (American Association for Cancer Research Annual Meeting) in San Diego, California.
The presentation was delivered by Professor Yoon-Jun Kim of the Department of Gastroenterology at Seoul National University Hospital.
The study includes patients with HCC who are refractory to or ineligible for transarterial chemoembolisation (TACE) and have not received prior systemic therapy.
According to the data presented, combination treatment of RZ-001 with atezolizumab and bevacizumab demonstrated an objective response rate (ORR) of 38.5% (confirmed) and 46.2% (unconfirmed) based on recist v1.1 criteria.
Under mrecist criteria, the ORR reached 61.5%, with a complete response (CR) rate of 23%, suggesting deep tumour responses. Given that mrecist reflects tumour necrosis, these findings may indicate meaningful therapeutic impact in HCC.
At interim analysis, responses are typically categorized as ‘confirmed’ or ‘unconfirmed’. Confirmed responses are those validated through subsequent assessments, while unconfirmed responses represent initial observations. In this study, patients classified as having ‘unconfirmed PR’ under recist criteria had achieved an initial tumour size reduction of at least 30%.
In terms of safety, a total of five grade 3 or higher adverse events were reported as related to combination agents, including hypertension (two cases), proteinuria, hyperglycaemia, and gastrointestinal bleeding. Notably, no grade 3 or higher adverse events were attributed to RZ-001.
The company believes these results demonstrate a favourable safety profile for RZ-001, with no treatment-related safety concerns identified to date, along with encouraging signals of antitumour activity in terms of both response depth and overall response rate in combination with standard immunotherapy.
