Rznomics Receives Positive Opinion for Orphan Medicinal Product Designation of RZ-001 for Glioma

Rznomics Receives Positive Opinion for Orphan Medicinal Product Designation of RZ-001 for Glioma

SEONGNAM, South Korea, Sept. 30, 2026 /PRNewswire/ -- Rznomics Inc., a biopharmaceutical company specializing in the development of RNA-based gene therapies, announced that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on the Orphan Medicinal Product Designation (OMPD) for RZ-001 (taspitimagene advec) for the treatment of glioma.

RZ-001 is an adenovirus serotype 5-vectored gene therapy built on Rznomics' proprietary RNA trans-splicing ribozyme platform. It targets human telomerase reverse transcriptase (hTERT) mRNA—overexpressed in 80–90% of human cancers—and replaces the target RNA with a therapeutic RNA encoding the cytotoxic herpes simplex virus thymidine kinase (HSV-TK), thereby selectively inducing apoptosis in cancer cells.

This recommendation was grounded in a Phase 1/2a interim clinical data from patients with recurrent glioblastoma (GBM), a subtype of glioma and a highly aggressive WHO Grade IV brain cancer associated with a 1-year survival rate of less than 25%. Despite existing authorized therapies in Europe, the COMP determined that RZ-001 has sufficient potential to provide a "significant benefit".

RZ-001 is currently being evaluated in a Phase 1/2a clinical trial in South Korea (IND clearance in the U.S.) for patients with hTERT-positive recurrent GBM, and has been administered to 10 patients enrolled in clinical trials and to 3 patients under the U.S. FDA's Expanded Access Program.

"This positive opinion from the EMA COMP shows that RZ-001 goes beyond meeting basic prevalence criteria by delivering meaningful, evidence-backed clinical advantages over current standard-of-care treatments for glioma," said Bomi Kim, Head of the Drug Development Center at Rznomics. "We will continue to advance our global regulatory and clinical development roadmap."

"Following our FDA RMAT designation for hepatocellular carcinoma (HCC) in May 2026, this European regulatory recognition further strengthens the therapeutic versatility and commercial potential of our RNA editing platform," added Dr. Seong-Wook Lee, CEO of Rznomics. "We are committed to expanding RZ-001's target indications and accelerating global business development opportunities."

Following formal designation by the European Commission, Rznomics will be eligible for key regulatory and commercial incentives, including up to 10 years of market exclusivity, EMA protocol assistance, and reductions in regulatory and filing fees associated with European marketing applications.

About Glioblastoma (GBM)
Glioblastoma is the most common and aggressive primary malignant brain tumor in adults, classified as a WHO Grade IV tumor. Characterized by rapid progression, high recurrence rates, and resistance to standard therapies, recurrent glioblastoma remains an area of significant unmet medical need with limited effective therapeutic options available globally.

About Orphan Medicinal Product Designation in the European Union
Orphan Medicinal Product Designation in the European Union is granted to medicinal products for the treatment of rare, serious, or life-threatening conditions and provides regulatory incentives, including ten years of market exclusivity following approval, which may be extended to twelve years in certain circumstances.

How RZ-001 (taspitimagene advec) Works
RZ-001 utilizes Rznomics' proprietary RNA trans-splicing ribozyme technology. The ribozyme embedded in RZ-001 selectively recognizes and cleaves hTERT mRNA expressed in cancer cells, replacing it with an RNA strand containing a suicide gene (HSV-TK) derived from the herpes simplex virus. Subsequently, the orally administered antiviral prodrug valganciclovir, is converted to ganciclovir and further activated by HSV-TK, generating cytotoxic metabolites that inhibit viral and cellular DNA synthesis and selectively induce apoptosis in cancer cells.

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 the 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

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SOURCE Rznomics