Alznomics, a South Korean biotechnology company focused on RNA-based therapeutics, has reported interim results from its clinical trial of RZ-001 in patients with hepatocellular carcinoma (HCC), showing an overall response rate (ORR) of 38.5% when used in combination with atezolizumab and bevacizumab. The data were presented as an abstract at the American Association for Cancer Research (AACR) Annual Meeting 2026 in San Diego, with a formal oral presentation scheduled for April 19, 2026.
The trial, which is a Phase 1b/2a study, enrolled patients with HCC who were either unresponsive to or ineligible for transarterial chemoembolization (TACE) and had not received prior systemic therapy. According to the company, RZ-001 demonstrated an ORR of 38.5% based on RECIST v1.1 criteria and 53.8% using modified RECIST (mRECIST) criteria, which better captures changes in viable tumor tissue often seen in liver cancer treatments. A complete response (CR) was observed in 23% of patients under mRECIST assessment, with two patients achieving CR as early as six weeks into treatment, one of whom maintained this status through week 30.
Alznomics noted that these results compare favorably to the IMbrave150 trial, the pivotal study that established atezolizumab plus bevacizumab as first-line standard of care for unresectable HCC. The company stated that the ORR with RZ-001 combination therapy exceeded that of IMbrave150 by approximately 10 percentage points and the complete response rate was more than double.
The investigational agent RZ-001 is built on Alznomics’ RNA trans-splicing ribozyme platform, a technology designed to target specific disease-causing RNA sequences, cleave them, and insert therapeutic RNA in their place. In the case of RZ-001, the therapy targets telomerase reverse transcriptase (TERT) mRNA, which is overexpressed in over 80% of hepatocellular carcinomas and several other cancer types. By disrupting TERT expression, RZ-001 aims to induce cancer cell death, promote immune cell infiltration into the tumor microenvironment, and enhance the efficacy of co-administered immune checkpoint inhibitors.
Telomerase, the enzyme encoded by TERT, maintains telomere length at the ends of chromosomes, allowing cells to divide indefinitely—a hallmark of cancer. Targeting TERT has long been considered a promising strategy in oncology due to its near-universal expression in malignant cells and minimal activity in most healthy somatic tissues. However, previous attempts to inhibit telomerase therapeutically have faced challenges related to delivery, specificity, and durability of effect. Alznomics’ approach leverages RNA editing to achieve precise, transient modulation of TERT mRNA without altering the genome, potentially reducing risks associated with permanent genetic modification.
The company highlighted that the observed tumor responses were not solely dependent on measurable shrinkage in tumor diameter. Instead, they emphasized that internal tumor necrosis—visible through imaging techniques used in mRECIST—often correlates with loss of viable tumor activity even when size reduction appears limited. This distinction is particularly relevant in liver cancer, where treatment-induced fibrosis or necrotic debris can obscure true biologic response on conventional imaging.
Alznomics was founded in 2017 by its current CEO, Dr. Sung-Wook Lee, who holds a bachelor’s degree in microbiology from Seoul National University, a master’s in the same field from Seoul National University Graduate School, and a Ph.D. In molecular biology from Cornell University. Prior to establishing Alznomics, Dr. Lee conducted research at Memorial Sloan Kettering Cancer Center and Duke University Medical Center, and served as a professor of molecular biology at Dankook University, where he directed the Nanosensor BioTech Research Center and the Next-Gen Omics Research Institute.
Under Dr. Lee’s leadership, Alznomics has pursued partnerships to advance its RNA platform beyond oncology. In 2023, the company entered into a collaboration and technology licensing agreement with Eli Lilly and Company valued at up to $1.3 billion, focused on developing genetic therapies for hereditary hearing loss using its RNA trans-splicing technology. Dr. Lee has also served as president of both the Korean Nucleic Acid Society and the Korean Society of Gene and Cell Therapy.
The RZ-001 program has received regulatory recognition from the U.S. Food and Drug Administration (FDA), including Fast Track designation and Orphan Drug status for hepatocellular carcinoma. These designations are intended to expedite development and review of therapies for serious conditions with unmet medical demand, particularly those affecting compact patient populations.
As of April 2026, Alznomics continues to enroll patients in the global Phase 1b/2a trial of RZ-001 for HCC and glioblastoma, another malignancy in which TERT promoter mutations are highly prevalent. The company plans to present updated clinical data from the ongoing trial during the oral session at AACR 2026 on April 20.
The findings contribute to a growing body of evidence supporting RNA-targeted therapies as a viable modality in oncology, particularly when combined with immunotherapy. While checkpoint inhibitors like atezolizumab have transformed outcomes for many cancer patients, a significant proportion remain unresponsive. Strategies that modulate the tumor microenvironment—such as reducing immunosuppressive signaling or increasing antigen presentation—may aid overcome resistance, and preliminary data suggest RZ-001 may exert such effects through TERT downregulation and subsequent immune activation.
Experts caution that interim results from early-phase trials require validation in larger, randomized studies before clinical efficacy can be confirmed. The upcoming oral presentation at AACR 2026 will provide an opportunity for independent scrutiny of the data, including safety profiles, duration of response, and biomarker correlations.
For patients and caregivers navigating advanced liver cancer, developments like those from Alznomics represent incremental progress in expanding therapeutic options beyond current standards. Reliable updates on the RZ-001 program can be found through the company’s official communications and peer-reviewed conference proceedings from major oncology gatherings such as AACR, ASCO, and ESMO.
As the scientific community awaits further details from the San Diego meeting, the intersection of RNA biology and immuno-oncology remains a fertile ground for innovation—one where precise molecular interventions may one day complement or enhance the body’s own defenses against cancer.
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