Neurophth Announces First Patient Dosed in Phase I/II Clinical Trial of Second Gene Therapy
Source:   Release Date: 2023-08-17
WUHAN and SAN DIEGO, August 17th, 2023 – Neurophth Therapeutics, Inc. (“Neurophth”) announced today that the first patient has been dosed in the international multi-region, multi-center Phase I/II clinical trial for the treatment of Leber hereditary optic neuropathy caused by ND1 mutation (ND1-LHON).


Neurophth is conducting a Phase I/II, multi-region, multi-center, single-arm, open-labeled, dose-finding study aimed at evaluating the safety, tolerability, and efficacy of NFS-02 in LHON patients with ND1 mutations in both China and the United States. The Investigational New Drug (IND) application of NFS-02 has been approved by the U.S. FDA in December 2022 and has been approved by China National Medical Products Administration (NMPA) on April 17, 2023. Led by Prof. Jia Qu and Rong Zhou from the Eye Hospital of Wenzhou Medical University, the team successfully completed the first dose in the Phase I/II clinical trial.


“Today’s milestone is a significant step forward. NFS-02 is Neurophth’s second gene therapy to enter clinical trials, demonstrating the company's strong operations.” said Professor Bin Li, Founder, Chairman and CEO of Neurophth, “NFS-02 is currently the only ND1-LHON treatment in development worldwide. In response to patients’ expectations, we are committed to conduct global multi-region, multi-center clinical trials to ensure that it’s on the path towards early drug approval and commercialization in the future.”


“In the coming months, we will continue to accelerate the patient enrollment process of the China-U.S. Phase I/II clinical trial. We anticipate positive results of the clinical trial and hope that it can bring us closer to achieving the goal of building a brighter future for global patients affected by LHON. Furthermore, we are actively exploring other indications where gene therapy may offer significant therapeutic benefit in the hope of meeting more unmet clinical needs.” said Dr. Xiaoning Guo, Chief Medical Officer of Neurophth.


About Neurophth

Neurophth is China’s leading in-vivo gene therapy company for ophthalmic diseases. With subsidiaries in China (Wuhan, Shanghai, and Suzhou) and US (San Diego, California), Neurophth, a fully integrated company, is striving to discover and develop genomic medicines for patients suffering from genetic diseases globally. Our validated AAV platform, which has been published in Nature - Scientific ReportsOphthalmology, and EBioMedicine, has successfully delivered proof-of-concept investigator-initiated trials data of 186 subjects with investigational gene therapies in the retina. Our most advanced investigational gene therapy drug candidate, NR082 (rAAV2-ND4), in development for the treatment of mtND4-mediated LHON, has been granted orphan drug designation (ODD) by the U.S. FDA and EMA. After the IND clearance by the China NMPA in March 2021 and the U.S. IND by FDA in January 2022, Neurophth has completed patient enrollment for Phase III clinical trial in China and dosed the patient in the U.S. Phase I/II clinical trial. Recently, our second gene therapy NFS-02, has dosed the patient in China-U.S. Phase I/II clinical trial. The pipeline also includes autosomal dominant optic atrophy, optic neuroprotection, vascular retinopathy, and five other preclinical candidates. To learn more about us and our growing pipeline, visit