taspitimagene advec (RZ-001) / Rznomics - LARVOL DELTA

Rznomics…announced that the Phase 1/2a interim clinical results for 'RZ-001 (Taspitimagene advec)', its RNA editing-based anticancer gene therapy for recurrent glioblastoma (rGBM), were presented on June 13th at the Asian Society for Neuro-Oncology Annual Meeting (ASNO 2026) (PRNewswire) - "According to the presentation, 20 patients have completed screening to date, with 10 patients officially enrolled and treated in the trial. The interim analysis revealed that no new or unexpected treatment-related safety concerns have been identified. No dose-limiting toxicity (DLT) was reported, and no treatment-related Grade 4 or higher adverse events were observed. Most reported adverse events were deemed to be disease-related or associated with underlying conditions typical of glioblastoma patients. Furthermore, several patients demonstrated prolonged tumor recurrence inhibition and disease control exceeding six months."

P1/2 data • Glioblastoma

Rznomics, a Korean RNA-based gene therapy developer, said Wednesday that the World Health Organization (WHO) has assigned the International Nonproprietary Name (INN) “Taspitimagene advec” to its RNA-based anticancer gene therapy candidate RZ-001. (Korea Biomedical Review) - "The company said it received official notification from the WHO INN Expert Advisory Panel that the name 'Taspitimagene advec' had been selected in accordance with the latest naming conventions for gene therapies. The designation is expected to be listed as a Proposed INN in the upcoming 'WHO Drug Information List 136,' scheduled for publication in 2026."

Commercial • Hepatocellular Cancer

Rznomics’ Liver Cancer Drug ’RZ-001’ Receives FDA Regenerative Medicine Advanced Therapy Designation (The Asia Business Daily) - "RZ-001 is a treatment for primary hepatocellular carcinoma (HCC) developed using Rznomics' proprietary ribozyme-based RNA editing platform. The company confirmed meaningful early signals in interim results of its Phase 1b/2a clinical trial presented at the American Association for Cancer Research (AACR 2026) in April, and believes the FDA recognized the clinical potential based on these results."

FDA event • Hepatocellular Cancer

A phase 1b/2a, open-label, multicenter, randomized, dose escalation study evaluating the safety, tolerability and efficacy of RZ-001 in combination with valganciclovir (VGCV) and atezolizumab/bevacizumab in subjects with hepatocellular carcinoma (AACR 2026) - "RZ-001 combined with VGCV, atezolizumab and bevacizumab was well tolerated and showed promising early efficacy in hTERT-positive HCC, suggesting a synergistic effect that may enhance antitumor responses when combined with a first-line standard therapy of Atezolizumab and Bevacizumab. Further enrollment is ongoing."

Clinical • Combination therapy • P1/2 data • Hepatocellular Cancer • Oncology • Solid Tumor • TERT

Rznomics said on the 19th it will unveil interim clinical results for its liver cancer drug candidate 'RZ-001' at the American Association for Cancer Research (AACR) (Chosun Biz) - "The clinical trial was conducted in patients with hepatocellular carcinoma, among others, who had no prior systemic therapy. The liver cancer drug candidate was administered in combination with the immune-oncology drugs atezolizumab and bevacizumab. As a result, tumor-shrinking responses were observed in patients. About 23% experienced complete disappearance of cancer cells."

P1/2 data • Hepatocellular Cancer

Rznomics to present RNA-editing cancer therapy data at AACR 2026 (Korea Biomedical Review) - "Rznomics will present interim results of its gene therapy candidate RZ-001 for liver cancer."

Preclinical • Hepatocellular Cancer

Rznomics asks WHO to assign a global drug name for its cancer gene therapy RZ-001 (Korea Biomedical Review) - "The company said it plans to share interim results from its phase 1b/2a hepatocellular carcinoma (HCC) trial in the first half of next year."

Commercial • P1/2 data • Hepatocellular Cancer

A phase I/IIa, open-label, multicentre study evaluating the safety, tolerability, and efficacy of RZ-001 in combination with valganciclovir (VGCV) in patients (pts) with human telomerase reverse transcriptase (hTERT)-positive glioblastoma multiforme (GBM) (ESMO Asia 2025) - "RZ-001 was well tolerated with an acceptable preliminary safety profile and encouraging efficacy results."

Clinical • Combination therapy • P1/2 data • Brain Cancer • Glioblastoma • Oncology • Solid Tumor • TERT

Korean gene therapy RZ-001 reports encouraging phase 1 data in brain cancer (Korea Biomedical Review) - "According to Rznomics, dosing has been completed through the third of five total dose levels, with the fourth currently underway. To date, favorable safety results have been confirmed, and early signals suggesting promising efficacy have also been detected."

P1 data • Glioblastoma

Rznomics Inc. Liver Cancer Drug Candidate Secures Fast Track Designation (PRNewswire) - "Rznomics announced on the 14th that its anticancer drug, RZ-001, has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Hepatocellular Carcinoma...It has received Investigational New Drug (IND) approval from both South Korea's Ministry of Food and Drug Safety (MFDS) and the U.S. FDA. Currently, it is in Phase 1b/2a clinical trials for Hepatocellular Carcinoma (HCC)..."

Fast track • Trial status • Hepatocellular Cancer

A Study to Evaluate the Safety, Tolerability and Efficacy of RZ-001 in Combination with Valganciclovir (VGCV) and Atezolizumab/Bevacizumab in Subjects with Hepatocellular Carcinoma (clinicaltrials.gov) - P1/2 | N=45 | Recruiting | Sponsor: Rznomics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Hepatocellular Cancer • Oncology • Solid Tumor

A Study to Evaluate the Safety, Tolerability and Efficacy of RZ-001 with Valganciclovir (VGCV) in Subjects with Hepatocellular Carcinoma (clinicaltrials.gov) - P1/2 | N=42 | Active, not recruiting | Sponsor: Rznomics, Inc. | Recruiting ➔ Active, not recruiting

Combination therapy • Enrollment closed • Gastrointestinal Cancer • Hepatocellular Cancer • Oncology • Solid Tumor • TERT

A Study to Evaluate the Safety, Tolerability and Efficacy of RZ-001 in Combination with Valganciclovir (VGCV) and Atezolizumab/Bevacizumab in Subjects with Hepatocellular Carcinoma (clinicaltrials.gov) - P1/2 | N=45 | Not yet recruiting | Sponsor: Rznomics, Inc.

Combination therapy • New P1/2 trial • Hepatocellular Cancer • Oncology • Solid Tumor

Expanded Access Program for GBM Subjects (clinicaltrials.gov) - P=N/A | N=0 | Temporarily Not Available | Sponsor: Rznomics, Inc.

Combination therapy • New trial • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor

Rznomics announces to secure the Expanded Access Program for patients with Glioblastoma (GBM) (PRNewswire) - "Rznomics, Inc. announced to secure its expanded access program (EAP) from the United States Food and Drug Administration (FDA) for RZ-001, RNA editing gene therapy product for the treatment of patients aged 18 and older with Glioblastoma (GBM)."

FDA event • Glioblastoma

Rznomics' Glioblastoma Treatment Candidate Receives FDA Compassionate Use Approval [Google translation] (Hankyung) - "Rznomics announced on the 14th that its candidate substance 'RZ-001' received compassionate use approval (EAP) from the U.S. Food and Drug Administration (FDA)....With this approval, Rznomics can now use RZ-001 for critically ill glioblastoma patients. Rznomics is conducting a phase 1/2a clinical trial targeting glioblastoma..."

FDA event • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

'10.8 billion legacy' Rznomics accelerates clinical and indication expansion of 'RZ-001' [Google translation] (Medipana) - "RNA-based new drug development company Rznomics is aggressively investing in clinical trials for its new drug candidate 'RZ-001'...According to the Financial Supervisory Service's electronic disclosure system on the 30th, Rznomics will invest approximately 4.2 billion won by next year to conduct clinical trials for the combination therapy of its core pipeline RZ-001 hepatocellular carcinoma treatment with immune-oncology drugs, as well as clinical trials for RZ-001 glioblastoma treatment. The funds for this will be raised through a third-party allocation paid-in capital increase worth 10.8 billion won scheduled for subscription on October 30...Rznomics stated regarding RZ-001 hepatocellular carcinoma treatment and glioblastoma treatment, 'We expect to complete the phase 2a clinical trial after 2026...'"

Financing • Trial status • Brain Cancer • CNS Tumor • Gastrointestinal Cancer • Glioblastoma • Glioma • Hepatocellular Cancer • Liver Cancer • Oncology • Solid Tumor

Rznomics, FDA Approves Clinical Trial Plan for Immunotherapy for Liver Cancer [Google translation] (Nate) - "Rznomics announced on the 19th that it had received approval from the US FDA, following the Ministry of Food and Drug Safety, for a phase 1b and 2a clinical trial using the anticancer drug 'RZ-001' under development in combination with an immune anticancer drug. The plan is to evaluate the efficacy and safety together by administering the drug in combination with the first-line standard treatment (atezolizumab + bevacizumab) to approximately 50 patients diagnosed with hepatocellular carcinoma."

IND • New P1/2 trial • Gastrointestinal Cancer • Hepatocellular Cancer • Liver Cancer • Oncology • Solid Tumor

A Study to Evaluate the Safety, Tolerability and Efficacy of RZ-001 With Valganciclovir (VGCV) in Subjects With Glioblastoma (clinicaltrials.gov) - P1/2 | N=43 | Recruiting | Sponsor: Rznomics, Inc. | Not yet recruiting ➔ Recruiting

Combination therapy • Enrollment open • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • CD4 • IFNG • IL10 • IL6 • TERT • TNFA

Rznomics, Celltrion’s ‘bevacizumab similar’ “supplied free of charge” [Google translation] (Biospectator) - "Rznomics and Celltrion are using a biosimilar of the VEGF antibody 'bevacizumab (product name Avastin)' in phase 1/2 liver cancer clinical trials of 'RZ-001', an RNA substitution enzyme gene therapy candidate. It was announced on the 14th that a clinical cooperation agreement was signed for evaluation....Under this partnership agreement, Celltrion will provide bevacimab biosimilar 'Vegzelma' to RZ-001, and Celltrion will receive priority approval for local licensing in Korea after Phase 1b/2a clinical trials of RZ-001 from RZ-001. The right to negotiate was secured. Rznomics is planning a phase 1b/2a clinical trial to evaluate the efficacy and safety by adding RZ-001 to the combination therapy of PD-L1 'atezolizumab (product name Tecentriq)' and bevacizumab, the first standard treatment for liver cancer."

Licensing / partnership • Gastrointestinal Cancer • Liver Cancer • Oncology • Solid Tumor

Anti-Cancer Efficacy and Safety of hTERT Splicing Ribozyme-based RNA Editing Therapy for Glioblastoma Multiforme (ASGCT 2024) - "RZ-001 treatment combined with ganciclovir (GCV) induced cytotoxicity in human GBM cells in a dose-dependent manner...Importantly, RZ-001 administration followed by GCV treatment efficiently improved median survival rate in PDX model established with tumor tissues from recurrent and temozolomide (TMZ)-resistant GBM patient, compared with groups treated with control or TMZ...Recently, RZ-001 received Fast Track designation from US FDA for GBM patients. This study and recent recognitions by the US FDA may raise the potential of splicing ribozyme-based RNA editing approaches as a safe and effective therapeutic option for patients with highly unmet medical needs."

Clinical • Late-breaking abstract • Brain Cancer • CNS Tumor • Gastrointestinal Cancer • Glioblastoma • Hepatocellular Cancer • Oncology • Solid Tumor • TERT

Rznomics, new anticancer drug manufacturing method approved for clinical use by the U.S. FDA [Google translation] (HIT News) - "Rznomics announced on the 6th that it has received approval for clinical use of a new manufacturing method for the anticancer drug 'RZ-001 (development code name)' under development from the U.S. Food and Drug Administration (FDA)...According to the company, compared to existing manufacturing methods, higher concentration test drugs can be produced, which not only makes it possible to conduct clinical trials with high concentration doses, but also enables cost-effective supply of test drugs....In addition, the company added that this new manufacturing method development process did not rely on a contract development and manufacturing (CDMO) company, and a large portion of the technology transfer took place at the Algnomics Research Institute."

FDA event • Brain Cancer • CNS Tumor • Gastrointestinal Cancer • Liver Cancer • Oncology • Solid Tumor

Rznomics Inc. Secures Orphan Drug Designation from FDA for RZ-001 in Hepatocellular Carcinoma (PRNewswire) - "Rznomics....has received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Hepatocellular carcinoma (HCC)."

Orphan drug • Hepatocellular Cancer

RZ-001, a treatment for hepatocellular carcinoma, is designated as an orphan drug by the FDA. [Google translation] (HIT News) - "RZ-001 (development code name), which is being developed as a treatment for hepatocellular carcinoma, has been designated as an orphan drug (Orphan Drug Designation) by the U.S. Food and Drug Administration (FDA)."

Orphan drug • Gastrointestinal Cancer • Hepatocellular Cancer • Liver Cancer • Oncology • Solid Tumor

RZ-001, first-line liver cancer treatment combination therapy clinical plan approved [Google translation] (MedigateNews) - "Rznomics announced on the 2nd that 'RZ-001' has received approval from the Ministry of Food and Drug Safety for a clinical trial plan to evaluate combination therapy with first-line standard treatment for liver cancer....In the clinical trial approved this time, RZ-001 will be additionally administered to the atezolizumab/bevacizumab combination therapy, the first standard treatment for liver cancer, with an emphasis on exploring effectiveness as a follow-up."

New trial • Gastrointestinal Cancer • Liver Cancer • Oncology • Solid Tumor