Neurophth Completes U.S. Patient Enrollment in Opvika® Phase I/II Trial

Neurophth Therapeutics has successfully enrolled the final participant in its Phase I/II clinical study of Opvika® (Esonadogene Imvoparvovec), a treatment for Leber hereditary optic neuropathy (LHON) caused by the ND4 gene mutation. The company is conducting this single-arm, multi-center trial in the United States to assess the safety and efficacy of NR082, a gene therapy for LHON patients with ND4 mutations. The U.S. Food and Drug Administration (FDA) approved the Investigational New Drug (IND) application for NR082 on January 18, 2022, and the first patient was treated in June 2023.
Professor Li Bin, the founder and CEO of Neurophth, expressed gratitude to the patients, their families, and the researchers for their contributions to the clinical trial's progress. He emphasized the company's commitment to advancing global health through Chinese medical innovation. Principal Investigator, Prof. Yaping Joyce Liao of Stanford Health Care's Byers Eye Institute, shared her enthusiasm for the clinical trial's progress and the potential of Opvika as a new therapy for LHON patients.
Neurophth, a leading Chinese gene therapy company specializing in ophthalmic diseases, has subsidiaries in Wuhan, Shanghai, Suzhou, and San Diego, California. The company is dedicated to developing genomic medicines for genetic diseases on a global scale. Their AAV platform has been recognized in several scientific publications and has demonstrated successful trial data for 186 subjects with retinal gene therapies. Opvika, the company's leading gene therapy candidate, has received orphan drug status from both the U.S. FDA and the European Medicines Agency (EMA). Following IND approvals from China's National Medical Products Administration (NMPA) in March 2021 and the U.S. FDA in January 2022, Neurophth has completed enrollment for a Phase III trial in China and a Phase I/II trial in the U.S. The company's second gene therapy, NFS-02, has initiated international multi-center Phase I/II trials in the U.S. and China, and NFS-05, a therapy for ADOA, has received approval for clinical trials in Australia. Neurophth's pipeline also encompasses optic neuroprotection, vascular retinopathy, and other preclinical candidates.

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