SAN DIEGO, Calif., April 19, 2026

Rznomics Inc. announced encouraging interim clinical results from its ongoing study of RZ-001, an innovative RNA editing-based anticancer therapy, presented during an oral session at the AACR Annual Meeting 2026. The study evaluates RZ-001 in combination with atezolizumab and bevacizumab in patients with hepatocellular carcinoma (HCC), demonstrating strong anti-tumor activity and a favorable safety profile in a population with limited treatment options and high unmet medical need.

Interim Clinical Data Demonstrates Strong Tumor Responses

The study includes patients with advanced HCC who are refractory to or ineligible for transarterial chemoembolization (TACE) and have not received prior systemic therapy. According to the interim analysis, the combination of RZ-001 with immunotherapy achieved an objective response rate (ORR) of 38.5% (confirmed) and 46.2% (unconfirmed) based on RECIST v1.1 criteria, reflecting significant tumor shrinkage in a meaningful proportion of patients.

Importantly, under mRECIST criteria, which better capture tumor necrosis in liver cancer, the ORR increased to 61.5%, including a complete response (CR) rate of 23%, indicating deep and potentially durable therapeutic effects. These findings are particularly notable as mRECIST evaluates viable tumor tissue, suggesting that the therapy is effectively inducing cancer cell death even when tumor size changes are less apparent. The data highlight the potential of RZ-001 to enhance current standard-of-care regimens and deliver improved clinical outcomes in a challenging disease setting.

Favorable Safety Profile Supports Combination Use

Advertisement

cGxPJobs Banner

Safety findings from the interim dataset reinforce the clinical viability of RZ-001, with no Grade 3 or higher adverse events attributed to the investigational therapy. A total of five serious adverse events, including hypertension, proteinuria, hyperglycemia, and gastrointestinal bleeding, were reported; however, these were associated with the combination agents rather than RZ-001 itself.

This distinction underscores the therapy’s clean safety profile and compatibility with existing immunotherapy treatments. The absence of severe toxicity linked to RZ-001 is particularly important in HCC, where patients often have compromised liver function and limited tolerance for aggressive treatments. These results support the continued development of RZ-001 as part of combination strategies aimed at maximizing efficacy while maintaining patient safety.

RNA Editing Platform Drives Next-Generation Oncology Innovation

RZ-001 is developed using Rznomics’ proprietary RNA trans-splicing ribozyme platform, a cutting-edge technology designed to precisely edit RNA and correct disease-causing genetic abnormalities. This approach represents a next-generation advancement in precision oncology, moving beyond conventional therapies toward targeted molecular intervention at the RNA level.

The positive interim data not only validate the potential of RZ-001 but also highlight the broader applicability of the platform across multiple cancer indications. HCC remains one of the most aggressive and difficult-to-treat cancers globally, often diagnosed at advanced stages with high recurrence rates and poor prognosis. By demonstrating strong response rates, deep tumor regression, and a favorable safety profile, RZ-001 positions itself as a promising new therapeutic candidate capable of transforming treatment paradigms.

The company emphasized that this AACR presentation marks a key milestone in the clinical advancement of RNA-based therapies, with early data supporting further development and potential expansion into larger trials. As additional data emerge, RZ-001 could play a critical role in redefining combination therapy strategies for liver cancer, while also advancing the integration of RNA editing technologies into mainstream oncology care. The study’s findings provide renewed optimism for patients with advanced HCC, highlighting the potential for more effective and durable treatment options in the near future.

Source: Rznomics press release