RZ-001 / Rznomics - LARVOL DELTA

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

RZNOMICS Inc. Announces Clinical Trial Collaboration and Supply Agreement to Evaluate RZ-001 in Combination with anti-PDL1 in liver cancer patients (PRNewswire) - "Rznomics Inc...announced today that it has entered into a clinical collaboration agreement with F. Hoffmann-La Roche Ltd (Roche) to study RZ-001....in combination with Roche's atezolizumab, in patients with hepatocellular carcinoma (HCC). Under the terms of the agreement, Roche will provide atezolizumab to be used in combination with RZ-001 for the planned Phase 1b/2a study."

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

Rznomics Inc. Secures Fast Track Designation from the U.S. FDA for RZ-001 (PRNewswire) - "Rznomics Inc...announced today that it has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Glioblastoma (GBM)."

Fast track designation • Brain Cancer • CNS Tumor • Glioblastoma • Oncology

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 | Not yet recruiting | Sponsor: Rznomics, Inc.

Combination therapy • New P1/2 trial • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • TERT

RZNOMICS Inc. received FDA approval to initiate clinical development of trans-splicing ribozyme-based RNA editing technology in Glioblastoma Multiforme Patients (PRNewswire) - "Rznomics...recently received Phase 1/2a IND approval from the U.S FDA on May 6th for its Glioblastoma Multiforme (GBM) treatment called RZ-001 and thus has achieved an important milestone for the company and the RNA editing field....Being the first U.S. FDA-approved ribozyme-based RNA reprogramming approach to be evaluated in patients, RZ-001, a gene therapy approach utilizing the company's proprietary trans-splicing ribozyme-based RNA reprogramming and editing technology, is a replication-incompetent adenoviral vector that expresses an hTERT targeting ribozyme with multiple additional MoA to treat GBM patients."

IND • New P1/2 trial • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

RNA Reprogramming Therapeutics Based on Tetrahymena Group I Intron for Hepatocellular Carcinoma (ASGCT 2023) - "Based on these results, RZ-001 recently received IND approval for first-in-human clinical 1/2a trials of group I intron based trans-splicing ribozyme from the Korean Ministry of Food and Drug Safety and the United States FDA, and was administered to the first HCC patient. Reprogramming of hTERT mRNA by post-transcriptionally regulated RNA replacement strategy mediated by a cancer-specific ribozyme would provide a clinically relevant, safe, and efficient strategy for HCC treatment."

IO biomarker • Gastrointestinal Cancer • Hepatocellular Cancer • Hepatology • Immune Modulation • Oncology • Solid Tumor • MIR122

Charles River and Rznomics Announce RNA-based Anticancer Gene Therapy Manufacturing Alliance (Businesswire) - "Charles River Laboratories International, Inc....and Rznomics Inc....announced a viral vector contract development and manufacturing organization (CDMO) partnership. Rznomics will leverage Charles River’s viral vector CDMO experience to initiate clinical development of its RNA-based anticancer gene therapy in liver cancer patients....Rznomics received Phase I/IIa IND approval for RZ-001 from the FDA in October, allowing the initiation of an international clinical study in HCC patients. Early-phase trials have also commenced in Korea following IND approval for RZ-001 from the South Korean Ministry of Food and Drug Safety (MFDS), formerly the Korea Food & Drug Administration (KFDA) in June."

Licensing / partnership • 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 Hepatocellular Carcinoma (clinicaltrials.gov) - P1/2 | N=42 | Recruiting | Sponsor: Rznomics, Inc.

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

RZNOMICS Inc. received FDA approval to initiate clinical development of trans-splicing ribozyme-based RNA editing technology in liver cancer patients (PRNewswire) - "Rznomics Inc...recently received Phase 1/2a IND approval from the U.S FDA in October 10^th for its hepatocellular carcinoma (HCC) treatment called RZ-001 and thus has achieved an important milestone for the company and the RNA editing field....The Phase 1/2a clinical trial will be a dose escalation/expansion study to assess the safety and tolerability of RZ-001 and to determine the most effective dose with the least toxicities of RZ-001 in HCC patients with no extrahepatic metastasis....In addition to the HCC, Rznomics is expanding the indication of RZ-001 to glioblastoma multiforme and planning to submit the IND this year."

IND • Brain Cancer • CNS Tumor • Gastrointestinal Cancer • Glioblastoma • Hepatocellular Cancer • Liver Cancer • Oncology • Solid Tumor