February 19, 2024 — Neurophth Biotechnology Co., Ltd. (“Neurophth”), a China-based leader in gene therapy for ophthalmic diseases, announced today that patient enrollment and dosing have been completed for the U.S. Phase I/II clinical trial of its core product, Nurofex® (Opvika, Eperadogene Emaparvovec Ophthalmic Injection), designed to treat Leber’s Hereditary Optic Neuropathy (LHON) caused by ND4 mutations (ND4-LHON).

About the U.S. Phase I/II Clinical Trial

Neurophth is conducting a single-arm, multicenter Phase I/II clinical study in the U.S. to evaluate the safety and efficacy of its gene therapy for ND4-associated LHON. The Investigational New Drug (IND) application for this trial was approved by the U.S. Food and Drug Administration (FDA) on January 18, 2022, with the first patient enrolled and dosed in June 2023.

Leadership Perspectives

Professor Bin Li, Founder, Chairman, and CEO of Neurophth, stated:
“We extend our deepest gratitude to the patients, their families, and all investigators for their trust, support, and dedication, which were pivotal in achieving this milestone. Neurophth has taken a solid step forward in bringing innovative therapies from China to the global stage. Guided by our vision—‘In China, For Global’—we will continue striving to advance Chinese medical research and deliver groundbreaking gene therapies to patients worldwide.”

Professor Yaping Joyce Liao, Principal Investigator (PI) of the trial from the Byers Eye Institute at Stanford University, commented:
“The progress in this Phase I/II trial is highly encouraging. LHON patients urgently await novel treatments like Nurofex® (Opvika) that offer hope for recovery. I look forward to collaborating with Neurophth to analyze the trial outcomes.”

About Neurophth

Neurophth is a leader in the in vivo gene therapy industry for ophthalmic diseases in China. Neurophth 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 Newvision® is designed to treat ND4-mediated Leber hereditary optic neuropathy (ND4-LHON) and 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, the enrollment and administration of patients in the Phase III clinical trial in China and the enrollment and administration of all patients in the Phase I/II clinical trial in the United States have been completed. The enrollment and administration of the first patient 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, have been completed. 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.