• Dr. Chae-Yong Kim of Seoul National University Bundang Hospital delivers oral presentation on interim data from 10 enrolled patients
• A manageable safety profile, with prolonged disease control and tumor recurrence inhibition observed in several patients

SEONGNAM and SEOUL, South Korea, June 15, 2026 /PRNewswire/ -- Rznomics Inc., a biopharmaceutical company specializing in the development of RNA-based gene therapies, 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) held in Kanazawa, Japan.

The presentation was delivered by Dr. Chae-Yong Kim, a professor of the Department of Neurosurgery at Seoul National University Bundang Hospital, highlighting the safety and preliminary efficacy data from the ongoing clinical trial.

RZ-001 is an innovative anticancer gene therapy utilizing Rznomics' proprietary RNA trans-splicing ribozyme platform, engineered to express a therapeutic gene specifically inside tumor cells to induce cancer cell death. 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.

Recurrent glioblastoma is one of the most aggressive and intractable malignant brain tumors, notorious for high recurrence rates and an extremely poor prognosis even after standard-of-care treatments. With currently approved treatment options showing limited efficacy and conventional immuno-oncology therapies repeatedly facing clinical failures, rGBM represents an area with immense unmet medical needs for novel therapeutic mechanisms.

"The secured data demonstrate a manageable safety profile for RZ-001 alongside encouraging preliminary signs of clinical activity," explained Dr. Chae-Yong Kim. "Given that recurrent glioblastoma typically recurs within two to four months, the data from patients showing long-term recurrence inhibition—particularly that of extending past nine months—is highly encouraging. We look forward to further clarifying its full clinical value through additional patient enrollment and long-term follow-up observations."

Meanwhile, RZ-001 is currently undergoing clinical development for both recurrent glioblastoma and hepatocellular carcinoma (HCC). The HCC program recently achieved a significant milestone by receiving Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA this past May.

About Rznomics Inc.
Rznomics is a clinical-stage biopharmaceutical company based in South Korea focused on developing RNA-based gene therapies. The company's proprietary trans-splicing ribozyme platform enables precise RNA editing and has broad applicability across multiple indications. The company signed a research collaboration and license agreement with global pharmaceutical company Eli Lilly in May 2025 for the development of a novel RNA editing therapeutic and listed on the KOSDAQ market in December 2025. (KOSDAQ 476830)

For more information, please visit  www.rznomics.com

RMAT designation based on promising Phase 1b/2a clinical data, including safety profile and preliminary response rates, with trans-splicing ribozyme-based RNA editing platform

SEOUL, South Korea, May 8, 2026 /PRNewswire/ -- Rznomics a biopharmaceutical company specializing in RNA-based gene therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation to RZ-001, its lead investigational candidate for the treatment of hepatocellular carcinoma (HCC).

RMAT designation is a specialized FDA program created to accelerate the development and review of promising new therapies, including gene therapies, intended to treat serious or life-threatening conditions. Applicant is required to submit preliminary clinical evidence suggesting the potential to address unmet medical needs. This designation provides important opportunities during the drug development process, including increased FDA guidance and eligibility for priority and rolling reviews, as well as accelerated approval pathways. By streamlining these regulatory milestones, the program aims to bring transformative innovations to patients more quickly.

RZ-001 is the next-generation oncology therapeutics based on Rznomics' proprietary trans-splicing ribozyme technology platform. By replacing cancer-specific RNA with therapeutic RNA, RZ-001 offers a novel mechanism of action designed to overcome the limitations of conventional therapies. The platform's dual-action approach—enhancing both tumor selectivity and safety—presents a promising new option for HCC patients with limited treatment alternatives. RZ-001 previously received Orphan Drug Designation (ODD) in 2024 and Fast Track Designation (FTD) in 2025 for the treatment of HCC.

The FDA's decision to grant RMAT status highlights the clinical potential and innovativeness of RZ-001, particularly following the meaningful interim signals from the Phase 1b/2a clinical trial presented at the American Association for Cancer Research (AACR) Annual Meeting in April 2026.

"With the RMAT designation, we plan to accelerate our U.S. development and partnership initiatives by initiating formal discussions with the FDA regarding clinical trial design, Chemistry, Manufacturing, and Controls (CMC), and commercialization strategies," said Sung-woo Hong, Vice President of Rznomics.

Seong-Wook Lee, CEO of Rznomics, added, "Receiving RMAT designation for RZ-001 is a profound validation of the innovation and competitiveness of our RNA editing platform by the FDA. We will concentrate our resources on global development and commercialization to provide a breakthrough therapeutic option in the field of HCC, where unmet medical needs remain exceptionally high."

About RMAT Designation

Introduced under the 21st Century Cures Act in 2016, the RMAT designation was established to foster the development of innovative regenerative medicine therapies and expand patient access. The program encompasses cell and gene therapies, therapeutic tissue engineering products, and combination products. To be eligible, a drug must be a regenerative medicine therapy intended to treat serious conditions, with preliminary clinical evidence indicating that the drug has the potential to address unmet medical needs for such a condition.

About Rznomics Inc.

Rznomics is a clinical-stage biopharmaceutical company based in South Korea focused on developing RNA-based gene therapies. The company's proprietary trans-splicing ribozyme platform enables precise RNA editing and has broad applicability across multiple indications. The company signed a research collaboration and license agreement with global pharmaceutical company Eli Lilly in May 2025 for the development of a novel RNA editing therapeutic and listed on the KOSDAQ market in December 2025. (KOSDAQ 476830)

For more information, please visit  www.rznomics.com

– RECIST-based ORR of 38.5% (confirmed) and 46.2% (unconfirmed)

– mRECIST-based ORR of 61.5% with a complete response (CR) rate of 23%

– No Grade 3 or higher adverse events related to RZ-001 observed

– Highlights clinical potential of RNA trans-splicing ribozyme platform technology

SEOUL, South Korea, April 21, 2026 /PRNewswire/ -- Rznomics announced today that it presented interim clinical data from its ongoing study of RZ-001, an RNA editing-based investigational anticancer therapy, in patients with hepatocellular carcinoma (HCC) during an oral presentation at the AACR 2026 (American Association for Cancer Research Annual Meeting), held on April 19, 2026, in San Diego, California.

The presentation was delivered by Professor Yoon-Jun Kim of the Department of Gastroenterology at Seoul National University Hospital.

The study includes patients with HCC who are refractory to or ineligible for transarterial chemoembolization (TACE) and have not received prior systemic therapy.

According to the data presented, combination treatment of RZ-001 with atezolizumab and bevacizumab demonstrated an objective response rate (ORR) of 38.5% (confirmed) and 46.2% (unconfirmed) based on RECIST v1.1 criteria.

Under mRECIST criteria, the ORR reached 61.5%, with a complete response (CR) rate of 23%, suggesting deep tumor responses. Given that mRECIST reflects tumor necrosis, these findings may indicate meaningful therapeutic impact in HCC.

At interim analysis, responses are typically categorized as "confirmed" or "unconfirmed." Confirmed responses are those validated through subsequent assessments, while unconfirmed responses represent initial observations. In this study, patients classified as having "unconfirmed PR" under RECIST criteria had achieved an initial tumor size reduction of at least 30%.

In terms of safety, a total of five Grade 3 or higher adverse events were reported as related to combination agents, including hypertension (2 cases), proteinuria, hyperglycemia, and gastrointestinal bleeding. Notably, no Grade 3 or higher adverse events were attributed to RZ-001.

The company believes these results demonstrate a favorable safety profile for RZ-001, with no treatment-related safety concerns identified to date, along with encouraging signals of antitumor activity in terms of both response depth and overall response rate in combination with standard immunotherapy.

"This oral presentation at AACR represents an important milestone for our lead program RZ-001," said Seong-Wook Lee, Chief Executive Officer of Rznomics. "We are encouraged by the early efficacy and safety signals observed, and we believe these data support the potential of RZ-001 as a novel therapeutic approach for HCC."

He added, "Beyond the RZ-001 program, these findings could further support the clinical applicability of our RNA trans-splicing ribozyme platform technology."

Hepatocellular Carcinoma (HCC)

Hepatocellular Carcinoma (HCC) is the most common type of primary liver cancer, accounting for the vast majority of liver cancer cases worldwide. It originates in the hepatocytes, the main cells of the liver, and is often associated with chronic liver diseases such as cirrhosis or viral hepatitis (B or C). HCC is known for its aggressive nature and high recurrence rates, presenting a significant global health challenge. Because it is often diagnosed at advanced stages, there is a critical unmet medical need for innovative therapies that can provide deeper and more durable responses than current systemic treatments.

Transarterial Chemoembolization (TACE)

Transarterial Chemoembolization (TACE) is a minimally invasive, localized procedure frequently used to treat patients with intermediate-stage liver cancer. The procedure involves delivering a concentrated dose of chemotherapy directly to the tumor through the hepatic artery, while simultaneously blocking (embolizing) the blood vessels that supply the tumor with oxygen and nutrients. While TACE can be effective for localized control, patients are considered "TACE refractory" when the tumor continues to progress or recurs despite multiple sessions, at which point systemic therapy is typically required.

RECIST v1.1 (Response Evaluation Criteria in Solid Tumors)

RECIST v1.1 is the standardized international guideline used to evaluate how solid tumors respond to treatment based on anatomical changes in tumor size. It primarily focuses on the sum of the longest diameters of target lesions, where a significant reduction in physical dimensions is categorized as a "response." While it remains the gold standard for most oncology trials, it mainly tracks physical shrinkage and may not fully capture the immediate biological impact of therapies that induce cell death without an immediate change in the overall tumor mass.

mRECIST (modified RECIST)

mRECIST is a specialized assessment tool developed specifically for Hepatocellular Carcinoma (HCC) to provide a more accurate evaluation of therapeutic efficacy. Unlike traditional criteria that measure the total size of a tumor, mRECIST focuses on the "viable" or living portion by measuring arterial enhancement—the area with active blood flow as seen on imaging. This is particularly critical in HCC because effective targeted or local treatments often cause internal tumor necrosis (cell death). In such cases, the tumor's overall size may remain the same even though the cancer cells are dead, a successful response that mRECIST is uniquely designed to capture.

About Rznomics Inc.

Rznomics is a clinical-stage biopharmaceutical company based in South Korea focused on developing RNA-based gene therapies. The company's proprietary trans-splicing ribozyme platform enables precise RNA editing and has broad applicability across multiple indications. The company signed a research collaboration and license agreement with global pharmaceutical company Eli Lilly in May 2025 for the development of a novel RNA editing therapeutic and listed on the KOSDAQ market in December 2025. (KOSDAQ 476830)

For more information, please visit  www.rznomics.com