At the beginning of the New Year 2024, Neurophth Biotechnology Co., Ltd. and Sinopharm Holding jointly announced the successful completion of the winter transportation verification of Newvision® (Elpidaudogene Ocular Injection), an innovative gene therapy drug for Leber hereditary optic neuropathy. In this snowy winter, the transportation vehicle and test samples for the first winter transportation verification of Newvision® arrived as scheduled and successfully reached the First Affiliated Eye Hospital of Harbin Medical University.

Leber hereditary optic neuropathy (LHON) is a mitochondrial disease that mostly affects young men and can lead to visual impairment and blindness in both eyes of patients. Currently, there is no effective clinical treatment or cure for it. The safety and efficacy of Newvision® (NFS-01) have been verified multiple times. It will be the first AAV gene therapy product in China to complete a Phase III clinical study and is expected to become a first-in-class new drug for LHON in the global market. To ensure the smooth launch of the product, Neurophth has actively carried out the transportation verification of Newvision®. Since gene therapy drugs require ultra-low temperature transportation and storage, this winter transportation verification aims to ensure that Newvision® can maintain stable temperature conditions throughout the entire transportation process, whether at the origin, transfer stations or the destination, to guarantee the quality of the drug.

This winter transportation verification took Harbin as the destination. With the support and cooperation of its strategic partner Sinopharm Holding, from January 23 to January 30, 2024, which lasted for 8 days, the transportation vehicle of Newvision® and the test samples set off from the Suzhou factory, arrived in Harbin and then safely returned to the Suzhou factory. The verification covered a maximum transportation distance of approximately 2,500 kilometers, the coldest ambient temperature was as low as minus 20°C, and the longest transportation time exceeded 96 hours. The quality of the drug was guaranteed throughout the transportation process. The design of the transportation conditions fully considered the possible "most extreme conditions" in actual shipments, making full preparations for the commercialization of Newvision®.

There is currently no commercial standard for the transportation and storage of gene therapy drugs in China, and there are still technical gaps that urgently need to be overcome. The key issue is how to ensure both the safety and efficiency of drug transportation. The strong cooperation between Neurophth and Sinopharm Holding, with the careful design of solutions such as ultra-low temperature storage, transportation, and real-time monitoring, and the final successful completion of the verification, undoubtedly sets an industry benchmark for the logistics management of ophthalmic gene therapy drugs.

Dr. Cao Yang, Vice President of the Quality Department of Neurophth, said: "Quality is the lifeline of a product. Especially for products like AAV gene therapy with high technical content and high requirements for transportation and storage, quality assurance is of utmost importance. In the winter transportation verification, our quality team and our partner Sinopharm Holding have demonstrated a rigorous and meticulous work attitude, ensuring the stability and safety of the product under extreme conditions. Such achievements not only reflect the perfection of our quality management system but also show the company's high sense of responsibility towards customers, the market, and life and health. I hope the team will continue to adhere to the principle of 'quality first', continuously optimize the process, and strictly control every link to add more luster to the company's quality reputation."

Dr. Li Zhengbin, Vice President of the Commercial Department of Neurophth, said: "Newvision® is the first AAV gene therapy drug in China that is about to complete the Phase III registration clinical trial. This time, Neurophth Biotechnology and Sinopharm Holding have fully collaborated to make sufficient preparations for the 'last mile' of Newvision®, ensuring that the product can be safely delivered to doctors and patients."

With the rapid development of the gene therapy field in China, more gene therapy products will gradually enter the commercialization stage. The winter verification of Newvision® this time has pioneered the commercial transportation and storage of AAV gene therapy drugs in China, playing a leading role and guiding the gene therapy industry in China to develop towards standardization and normalization.

About Neurophth

Neurophth is a leader in the gene therapy industry for ophthalmic diseases in China. It has subsidiaries in Wuhan, Suzhou, Shanghai and San Diego, the United States, and is committed to developing gene therapies for genetic diseases for patients worldwide. The trial data of the investigator-initiated retinal gene therapy study has successfully verified the AAV platform we use, and the research results have been published in Nature-Scientific Report, Ophthalmology and EbioMedicine. Our core product NR082, which is designed to treat ND4-mediated Leber hereditary optic neuropathy (ND4-LHON), has been granted the Orphan Drug Designation (ODD) by the U.S. FDA and the European Medicines Agency (EMA). It is the first new gene therapy drug of Chinese origin to receive both the IND approval for clinical trials from the NMPA in China and the U.S. FDA. Currently, all patients have been enrolled and administered in the Phase III clinical trial in China, and the first patient has been enrolled and administered in the Phase I/II clinical trial in the United States. The first patient has been enrolled and administered in the international multi-center Phase I/II clinical trial in China and the United States for NFS-02, the second new drug of Neurophth granted the ODD in the United States. NFS-05, the third new gene therapy drug of the company targeting ADOA, has been approved for clinical trials in Australia. The company's pipeline also includes preclinical candidate drugs for optic nerve protection and vascular retinopathy. To learn more about us and our expanding pipeline, please visit www.neurophth.com.

About the Global Procurement and Supply Chain Service Center of Sinopharm Holding

The Global Procurement and Supply Chain Service Center of Sinopharm Holding is the core business management and operation platform of Sinopharm Holding. It undertakes the national import, procurement and distribution tasks of most of the strategic varieties of Sinopharm Holding, as well as the national unified service function. According to the development requirements of the 14th Five-Year Strategic Plan of Sinopharm Holding, the Global Procurement Center continuously integrates procurement resources, explores internal potential, strengthens operation management, and makes efforts to expand new business forms such as in-depth marketing and new business development, constantly increasing the precision, breadth and depth of its operations. It further builds five functional platforms, including the import and distribution platform, the supply chain platform, the marketing platform, the innovation platform, and the medical aesthetics platform.