The World’s First AAV-Delivered Bispecific Antibody — NovaSight Biotherapeutics’ XMVA09 Injection Successfully Initiates Phase II Clinical Trial

Recently, NovaSight Biotherapeutics successfully held the Phase II clinical trial initiation meeting for its self-developed XMVA09 Injection for the treatment of wet age-related macular degeneration (wAMD). This marks XMVA09 Injection as the world’s first AAV-delivered bispecific antibody to enter Phase II clinical development, as well as the first ophthalmic AAV therapy based on a non-natural capsid in China to enter Phase II clinical development, opening a new chapter in ophthalmic gene therapy both in China and globally.

wAMD is a leading cause of irreversible vision impairment among people over the age of 50, affecting the quality of life of approximately 200 million people worldwide. Under current standard-of-care treatment regimens, patients need to receive intraocular injections of anti-VEGF drugs every one to two months. Treatment interruption or insufficient treatment may lead to reduced long-term efficacy and irreversible vision loss, while gene therapy holds the promise of achieving “one-time administration with lifelong efficacy.” As the world’s only gene therapy with both bispecific antibody targets and intravitreal administration, XMVA09 Injection uses an AAV gene vector carrying a bispecific antibody gene targeting VEGF-A and Ang-2. Through intravitreal injection, it acts directly on RPE cells to express therapeutic proteins, aiming to achieve long-term treatment of wAMD.

XMVA09 Injection was approved by the NMPA to enter clinical development in March 2024. In early April this year, enrollment and treatment of all subjects in its Phase I clinical trial were successfully completed. Preliminary results demonstrated favorable safety and tolerability, while efficacy data showed significant market advantages. The Phase II initiation meeting for XMVA09 Injection in the treatment of wAMD, held in Tianjin, brought together leading expert teams in the field of wAMD treatment in China, NovaSight Biotherapeutics’ core management team, and representatives of clinical research partners to jointly discuss the Phase II clinical study protocol and execution plan for XMVA09 Injection.

At the beginning of the meeting, Professor Hua Yan of Tianjin Medical University General Hospital delivered opening remarks, fully recognizing the efficient completion of the Phase I trial. He emphasized that this achievement was the result of multi-party collaboration and encouraged the team to advance the Phase II study with the same level of execution. In his speech, Dr. Cai Yuan, Chief Executive Officer of NovaSight Biotherapeutics, first expressed sincere gratitude to the teams at all centers involved in the Phase I clinical trial for their hard work. He also extended his appreciation in advance to the teams about to join the Phase II clinical study and expressed his expectation for close collaboration among all parties. The meeting focused on the scientific rationale of the Phase II clinical protocol, laying a solid foundation for project advancement and jointly opening a new clinical stage.

Subsequently, Director Zhou Wei of Tianjin Medical University General Hospital shared data from the Phase I and investigator-initiated trials (IIT), systematically analyzing the efficacy and safety of the drug. He also presented efficacy data and imaging results from representative cases. The data showed significant improvements in patients’ visual acuity and anatomical indicators. Together with long-term IIT data, the preliminary assessment indicated that XMVA09 Injection is generally effective and demonstrates long-term safety. Liu Mingyue, Medical Manager at ClinChoice, reported the Phase I clinical study results. By comparing data from similar products in China and abroad, she noted a clear trend of efficacy improvement in the medium- and high-dose groups of XMVA09, and introduced the Phase II clinical trial protocol, providing an in-depth interpretation of key indicators such as inclusion and exclusion criteria and endpoint assessments. The meeting was chaired by Deng Yiming, Project Management Director at Keying Pharma.

During the expert discussion session, expert teams from multiple clinical centers, including Professor Hua Yan’s team from Tianjin Medical University General Hospital, Professor Gu Yonghao’s team from the First Affiliated Hospital of University of Science and Technology of China, Professor Zhong Jie’s team from Sichuan Provincial People’s Hospital, Professor Han Mei’s team from Tianjin Eye Hospital, Professor Shen Lijun’s team from Zhejiang Provincial People’s Hospital, Professor She Haicheng’s team from Beijing Tongren Hospital, Capital Medical University, Professor Yuan Lili’s team from Shanxi Eye Hospital, and Professor Lei Chunling’s team from Xi’an People’s Hospital, held in-depth discussions on key topics such as improving efficacy evaluation indicators and refining patient enrollment criteria. Drawing on their extensive clinical research experience, experts from each center put forward multiple constructive suggestions for the Phase II clinical trial protocol. Finally, Professor Hua Yan stated in his concluding remarks that the meeting had reached consensus on key issues through practical and efficient discussions, laying a solid foundation for the smooth implementation of the Phase II clinical trial. Moving forward, the team will accelerate the efficient execution of the Phase II study while ensuring research quality.

Dr. Miao Bolong, Chief Medical Officer of NovaSight Biotherapeutics, said: “As the first innovative bispecific antibody therapy administered via intravitreal injection, XMVA09 Injection is expected to significantly reduce the injection frequency for patients with wAMD and become a future first-line maintenance therapy for wAMD. We firmly believe that, driven by the rich clinical experience of the expert teams and close collaboration among all parties, the clinical development of XMVA09 Injection will progress steadily and bring benefits to many patients as soon as possible.”

Dr. Cai Yuan, Chief Executive Officer of NovaSight Biotherapeutics, said: “The initiation of this pivotal Phase II clinical trial represents a key recent milestone for NovaSight Biotherapeutics and an essential step in validating the company’s technological advantages. XMVA09 Injection has advanced smoothly to the next stage with outstanding clinical benefits, which would not have been possible without the support of all our partners, the trust of all shareholders, and the dedication and close collaboration of our team. NovaSight Biotherapeutics will continue to focus on R&D, leverage cutting-edge gene therapy technologies, develop leading innovative products, and benefit the broadest possible patient population.”

About NovaSight Biotherapeutics

NovaSight Biotherapeutics is a high-tech enterprise dedicated to the development of ophthalmic gene therapy products based on cutting-edge gene editing technologies. The company focuses on the development of gene therapies for ophthalmic diseases. NovaSight Biotherapeutics’ core team is a doctoral entrepreneurial team led by a recipient of the National Science Fund for Distinguished Young Scholars. On the R&D side, the company has established two core technology platforms: an AAV-antibody gene therapy platform, including a bispecific antibody optimization platform and a capsid screening platform, and a gene editing platform.

The company is committed to developing ophthalmic gene therapy drugs that are independently accessible to Chinese patients and actively advancing gene therapy into major ophthalmic indications. Using ophthalmology as its breakthrough area, especially common ophthalmic diseases represented by age-related macular degeneration, NovaSight Biotherapeutics aims to help people in China preserve their vision for longer and see better.