Clinical trial of KH658 evaluates single suprachoroidal space administration of gene-therapy treatment for wet AMD

CHENGDU, China and ROCKAWAY, N.J., May 02, 2025 (GLOBE NEWSWIRE) -- Chengdu Origen Biotechnology Co., Ltd. (“Chengdu Origen”) and Vanotech Ltd. (“Vanotech”) announced today the first patient dosed in the VAN-2401 multi-center Phase 1 clinical trial evaluating treatment with KH658 for patients with wet Age-related Macular Degeneration (wet AMD). This multi-center, open-label, dose-escalation clinical trial in the U.S. will assess the safety, tolerability and efficacy of KH658 as a single suprachoroidal space administration of gene therapy for patients with previously treated wet AMD.

KH658 is a recombinant adeno-associated virus vector that encodes a human VEGF receptor fusion protein. In preclinical studies of wet AMD disease models, suprachoroidal space administration of KH658 resulted in retention of the transgene product in the retina for prolonged periods and prevented the disease progression. These findings may indicate the potential of KH658 to offer a single administration treatment for wet AMD patients.

“Dosing our first patient in the VAN-2401 Phase 1 trial is an important milestone in the advancement of KH658 by exploring the potential of single suprachoroidal space administration of gene therapy as treatment for wet AMD. KH658 is designed to deliver anti-VEGF continuously to the retina, with the potential to provide sustained levels that can control the disease,” said Avner Ingerman, M.D., Chief Medical Officer of Vanotech.

“I am excited to participate as a principal investigator in this important study. Dosing the first patient is an important milestone in the development of the single-administration gene therapy approach, which may hold promise to our patients in need of frequent treatment for their retinal diseases,” said Mark Barakat, M.D., a principal investigator in the VAN-2401 Phase 1 trial, Director of Research at Retina Macula Institute of Arizona in Scottsdale Arizona.

About the VAN-2401 Phase 1 Clinical Trial
VAN-2401 is a Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH658 Gene Therapy in Participants with Neovascular Age-related Macular Degeneration. The study is expected to include approximately nine previously treated wet AMD subjects that are responsive to anti-vascular endothelial growth factor (anti-VEGF) therapy. For more information, please visit https://www.clinicaltrials.gov/study/NCT06825858

About Wet AMD
Wet AMD is the rarer form of AMD, characterized by loss of vision due to abnormal blood vessel formation in and under the retina. These blood vessels tend to leak, and the fluid interferes with the normal retinal structure and function, leading to vision loss. Wet AMD is a leading cause of irreversible vision loss or blindness in the United States and Europe, especially if left untreated. It is estimated that ~1.5 million people living with wet AMD in the United States.

Anti-VEGF therapies have become the standard of care for wet AMD. Anti-VEGF treatments reduce leakage and fluid accumulation, thereby restoring normal retinal architecture and function, in many patients. Anti-VEGF agents often require frequent, repeat injections to maintain efficacy and control the disease.

About Chengdu Origen and Vanotech
Chengdu Origen is a clinical-stage gene therapy company focused on developing gene therapy for unmet medical needs and providing meaningful clinical benefits for patients suffering from genetic and chronic diseases and has comprehensive viral vector manufacturing capabilities and infrastructure.

Vanotech is operating as the sponsor-representative, and is responsible for the clinical development program of KH658 in the United States. Vanotech is also currently conducting VAN-2201, a Phase 1, multi-center, open-label, dose-escalation study in subjects with wet AMD to evaluate safety, tolerability and efficacy of a single administration of KH631. For more information, please visit https://www.clinicaltrials.gov/study/NCT05657301.

Forward Looking Statements
This press release contains “forward-looking statements,” within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, Chengdu Origen’s and Vanotech’s research and development plans in connection with its clinical development program and the gene therapy treatments. Such forward-looking statements include inherent risks and uncertainties, including factors that could cause actual results to differ materially from those projected by such forward-looking statements. All of Chengdu Origen/Vanotech development timelines could be subject to adjustment depending on recruitment rate, regulatory agency review and other factors that could delay the initiation and completion of clinical trials. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of pandemics and other health emergencies, including their impact on the global supply chain; the uncertainty that our product candidates will receive regulatory approval necessary to initiate human clinical studies; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products;

All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. Investors are cautioned not to place undue reliance on these forward-looking statements. These forward-looking statements speak only as of the date of this press release. Chengdu Origen and Vanotech undertake no obligation to update any forward-looking statements, whether as a result of new information, future developments or otherwise.