On December 16, 2023, the 2nd International Forum on LHON was successfully held. Internationally renowned neuro-ophthalmology experts such as Nancy Newman and Patrick Yu-Wai-Man were invited to preside over and give lectures at the conference. At the meeting, Professor Marcela Ciccioli from the University of Buenos Aires in Argentina presented the "Results of the Five-year Follow-up of Argentine Patients with LHON-ND4 Treated with NFS-01 (NR082, Trade Name: Newvision®)".

In 2018, Professor Li Bin, the founder of Neurophth, led the team to complete the IIT2 clinical study of gene therapy for LHON with a global sample size of 159 cases, the largest of its kind. Among them, 10 Argentine patients were recommended by the patient association (Stargardt’s APNES - RETINA ARGENTINA & PANAIRD) led by Professor Marcela Ciccioli. With the help of the Argentine patient association, they traveled half the globe from Argentina to China. Under the leadership of Professor Zhang Yong from Taihe Hospital in Shiyan City, they successfully received the treatment with NFS-01 and completed their journey to pursue light.

During the follow-up of these patients, Professor Ciccioli found that all 10 patients had achieved visual improvement to varying degrees. Among the 9 patients who completed the five-year follow-up, 6 patients showed significant improvement in the visual acuity of the treated eye, all exceeding 0.3 LogMAR, and they have maintained the visual benefits for 5 years, with a fundamental improvement in their learning and quality of life.

Professor Ciccioli introduced: "Five years after the treatment with NFS-01 (NR082), the patients have regained many of their learning and living abilities before the onset of the disease. They have a brand-new perspective to face the future path of their lives."

Professor Li Bin, the founder, Chairman and CEO of Neurophth, said: "We are very glad to see that the Argentine patients who came to China to receive the treatment with NFS-01 five years ago have achieved significant visual improvement and enhancement in their quality of life. We will spare no effort to promote the global commercialization of Newvision® (NFS-01) to help LHON patients around the world regain their sight."

About Neurophth
Neurophth is a leading company in China specializing in gene therapy for ophthalmic diseases. 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 US 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 US 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.