Maltese Patient Received World’s First ND1-LHON Gene Therapy Injection Donated by Neurophth in China

WUHAN, China and SAN DIEGO — Matthias Szabo Zarb, a Maltese college student, was diagnosed with Leber hereditary optic neuropathy caused by ND1 mutation (ND1-LHON) two years ago, which led to a rapid deterioration in his visual acuity and inability to engage in normal activities. Through the National Alliance for Rare Diseases Support, he learned about NFS-02, a gene therapy drug targeting ND1-LHON which is developed by the Chinese ophthalmic gene therapy company, Neurophth. Following a recommendation from the Embassy of China in Malta, Ms. Michelle Muscat, the chairman of the Malta National Alliance for Rare Diseases Support, contacted Neurophth. Neurophth decided to donate NFS-02 and recommended Dr. Yong Zhang, director of ophthalmology center at Taihe Hospital, as the treating physician. Upon arrival, the patient received free expedited genetic tests supported by Neurophth. On March 4th, representatives of Neurophth donated NFS-02 to the Maltese patient at Taihe Hospital. Notably, Professor Zhang Yong performed the surgery for the patient on the same day.

The Embassy of China in Malta expressed their gratitude to Neurophth for their support in treating international patients. They praised the company for donating drug to Mr. Matthias Szabo Zarb and his family, as well as fostering closer ties between China and Malta. The collaboration has received high attention from the Maltese government. The National Alliance for Rare Diseases Support organized a press conference where the Speaker of the Maltese Parliament delivered a speech. Former Maltese Prime Minister Muscat and his wife also conveyed their appreciation to the embassy.

In 2017, Professor Li Bin, the former director of the Ophthalmology Department of Tongji Hospital of Huazhong University of Science and Technology, launched the world’s largest sample size clinical trial for gene therapy for ND4-LHON. The study included 149 patients from China and 10 patients from Argentina. The 12-month follow-up results showed that NFS-01 has remarkable curative effect and no obvious serious adverse events have been found. NFS-02 is Neurophth’s second gene therapy, in development for the treatment of mtND1-mediated LHON. After six years of development, the company’s technology platform has become more advanced. NFS-02 has been used to treat multiple patients in the investigator-initiated clinical trial (IIT) in China and received orphan drug designation and IND approval from the U.S. FDA.