Rznomics Co., Ltd.

Anticancer drug utilizing Trans-splicing Ribozyme technology Second Fast Track Designation following brain cancer approval SEONGNAM, South Korea, Feb. 18, 2025 /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 (HCC). This marks the second Fast Track Designation for RZ-001, following its designation in November 2023 for Glioblastoma (GBM), a hard-to-treat brain cancer. The Fast Track program, established by the FDA, supports the development of drugs for serious or life-threatening diseases by expediting the approval process. Once a drug is designated and if relevant criteria are met, it benefits from accelerated review and approval, as well as more frequent and flexible communication with the FDA. Additionally, during the Biologics License Application review, a rolling review can be conducted for each data section, providing an advantage in the evaluation process. RZ-001 is an anticancer drug developed using Trans-splicing Ribozyme, Rznomics' proprietary RNA editing platform technology. 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) and Phase 1/2a trials for Glioblastoma (GBM). Furthermore, for Glioblastoma (GBM), RZ-001 has been approved under the FDA's Expanded Access Program (EAP), which allows compassionate use of investigational treatments outside clinical trials. The program is currently being conducted at Harvard University Hospital. Dr. Seong-Wook Lee, CEO of Rznomics, stated, "We believe this designation recognizes the potential of RZ-001 as an innovative cancer therapy. We are committed to expediting its clinical development to provide effective treatment options for patients suffering from hard-to-treat cancers." About RZNOMICS As a biopharmaceutical company founded in the laboratory of Professor Seong-Wook Lee, Dankook University Department of Bioconvergence Engineering, Rznomics is researching with the goal of developing new RNA-based gene therapeutic bio-drugs for cancer and incurable diseases. Rznomics' core platform technology is based on an RNA replacement enzyme, known as trans-splicing ribozyme, which can edit target RNA through simultaneous destruction and repair (and/or reprogramming) to yield the desired therapeutic RNA, thus, selectively inducing therapeutic gene activity in cells expressing the target RNA. For more information, please visit Contact Yonsoo Jeong Business Development / Associate Manager Email: [email protected] Logo - SOURCE Rznomics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SEONGNAM, South Korea, Nov. 25, 2024 /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). Generally, the EAP also known as 'compassionate use,' is a pathway provided by the U.S. FDA that allows patients with serious or immediately life-threatening conditions to gain access to investigational medical products (drugs, biologics, or medical devices) outside of clinical trials. GBM is known as the most malignant tumor in Central Nervous system with high mortality rate but lacks effective therapies. TERT promoter mutations, which are associated with TERT upregulation, are found in up to 80% of glioblastoma (GBM) patients. This increased TERT expression is strongly linked to a poor prognosis, reflecting the aggressive nature of the disease. RZ-001, the RNA replacement enzyme-based cancer gene therapy, targets and cleaves hTERT mRNA and replaces the mRNA with the therapeutic gene RNA. This induces anti-cancer activity and cytotoxic effect by reducing hTERT expression and simultaneously trans-ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. "We are excited to offer RZ-001 through this EAP, providing a potential new treatment option for GBM patients with limited alternatives," said Dr. Chiocca, Professor at Harvard Medical School executive Director of the Center for Tumors of the Nervous System at Mass General Brigham Cancer Institute. Seong-Wook Lee, PhD, CEO of Rznomics, stated, "We hope RZ-001 can serve as a good alternative for patients who have had difficulty with existing treatments." He also assured that the Rznomics team is committed to expediting the clinical development process to secure timely market authorization. In previous releases, Rznomics noted that RZ-001 has received Fast Track designations and Phase I/IIa IND approval for RZ-001 from the FDA and the South Korean Ministry of Food and Drug Safety (MFDS) in Glioblastoma and the clinical trial has been investigating the safety, tolerability, and efficacy of RZ-001 in patients with GBM. A clinical trial has recently commenced, marking the start of RZ-001 administration. About Rznomics As a biopharmaceutical company founded in the laboratory of Professor Seong-Wook Lee, Dankook University Department of Bioconvergence Engineering, Rznomics is researching with the goal of developing new RNA-based gene therapeutic bio-drugs for cancer and incurable diseases. Rznomics' core platform technology is based on an RNA replacement enzyme, known as trans-splicing ribozyme, which can edit target RNA through simultaneous destruction and repair (and/or reprogramming) to yield the desired therapeutic RNA, thus, selectively inducing therapeutic gene activity in cells expressing the target RNA. For more information, please visit SOURCE Rznomics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Rznomics gets approves U.S. FDA For anticancer Candidate Based On Gene Therapy Rznomics gets approves U.S. FDA For anticancer Candidate Based On Gene Therapy RZ001+T-Sentric + Avastin Approved Phase 1b/2a clinical trial for hepatocellular carcinoma patients Rznomics, a gene therapy drug-based anticancer drug developer, will start clinical trials in the United States that use its candidate and immune anticancer drugs in combination. Rznomics announced on the 19th that it has received approval from the U.S. Food and Drug Administration (FDA) for its phase 1b/2a clinical trial in which the anticancer drug candidate "RZ-001" and immuno-cancer drugs are combined. The plan is to evaluate both effectiveness and safety by co-administering RZ-001 together with the first standard treatment (Tscentrick + Avastin) to about 50 patients diagnosed with hepatocellular carcinoma. RZ-001 is an anticancer drug candidate that is being developed by applying the RNA editing platform technologies owned by Rznomics. It targets telomerase (hTERT) RNA, which is specifically expressed in cancer cells, by delivering RNA enzymes with adenovirus as a vector. Normal cells become shorter in telomeres, and when there is not much time left, they perceive themselves to be 'old' and die without further dividing. However, in cancer cells, telomerase, an enzyme that increases the length of telomeres that is decreasing again, is overactive, and telomeres may continue to lengthen. This is the cause of cancer cells continuing to divide without dying. RZ-001 removes hTERT RNA from hepatocytes that have become cancerous cells so that telomerase is not expressed. It induces apoptosis by making telomeres shorten normally. HSV-TK is also expressed at the cut hTERT site. Antiviral drugs (balgancyclover) administered with RZ-001 selectively attack only cancerous cells in response to this gene. It is a dual mechanism that removes the telomerase gene that causes infinite proliferation of cancer cells and inserts virus-derived genes to cause antiviral drugs to attack cancer cells. The FDA-approved clinical trial was combined with ticentric + avastin, which is the most widely used first-line treatment for hepatocellular carcinoma. If differentiated safety and efficacy are demonstrated compared to existing treatments, it could open the way for them to enter the largest market of first-line treatments. Rznomics plans to collaborate with large domestic and international pharmaceutical companies for this clinical trial. Among the clinical drugs, atetzolizumab has been contracted to receive supply from Roche and bevacizumab from Celltrion. Seong-wook Lee, CEO of Rznomics, said, "As many researchers and institutions cooperate in RZ-001 clinical development, we will do our best to succeed as an innovative anticancer drug."