Neurophth Begins Phase I/II Trial for Second Gene Therapy with First Patient Dosed

18 August 2023 | Friday | News

Neurophth Therapeutics, Inc. ("Neurophth") announced 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).
Image Source : Public Domain

Image Source : Public Domain


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 conducting 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.

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