Neurophth Secures Australian Approval for Ophthalmic Gene Therapy
Source:   Release Date: 2023-09-05

WUHAN, China and SAN DIEGO, Sept. 6, 2023 -- On September 6, 2023, Neurophth Therapeutics (hereinafter referred to as "Neurophth"), a leader in in-vivo gene therapy for ophthalmic diseases in China, announced the Australian Therapeutic Goods Administration (TGA) has registered and approved its candidate drug, NFS-05, for clinical trials targeting Autosomal Dominant Optic Atrophy (ADOA).


Autosomal Dominant Optic Atrophy (ADOA) is an autosomal dominant inherited optic neuropathy. About 80% of ADOA is caused by mutations in the OPA1 gene. The reduction in OPA1 protein function leads to mitochondrial fragmentation, and increased instability of the mitochondrial respiratory chain complex, which damages mitochondrial function and ultimately results in RGC cell death and optic nerve atrophy. Clinically, patients present with bilateral, slowly progressing visual impairment, temporal pallor of the optic disc, central visual field defects, and abnormalities in color vision.


Currently, no effective treatments are available for ADOA in clinical practice. Neurophth's proprietary ophthalmic injection, NFS-05, utilizes a gene therapy approach. It delivers an AAV vector containing the OPA1 gene into the vitreous cavity. This vector then targets RGC cells, leading to the expression of the OPA1 protein and subsequent restoration of mitochondrial function.


"Neurophth's NFS-05 marks our third drug to receive clinical trial approval and our inaugural approval in Australia," stated Professor Li Bin, Founder, Chairman, and CEO of Neurophth. "This achievement underscores Neurophth's robust R&D capabilities and our unwavering commitment to global outreach. We remain dedicated to harnessing our technical platform, broadening our product pipeline, and diligently expanding beyond China to deliver innovative medical solutions to patients globally."


Dr. Xiaoning Guo, Chief Medical Officer at Neurophth, commented, "Existing clinical interventions for ADOA, including measures to enhance circulation and support nerve health, offer limited benefit. With NFS-05, Neurophth endeavors to target the underlying cause of the disease using gene therapy, aiming for enhanced visual outcomes for patients. We are committed to advancing international multi-center clinical trials for NFS-05, with the goal of rapidly delivering a safe and efficacious treatment to patients."


About Neurophth

Neurophth is China's leading in-vivo gene therapy company for ophthalmic diseases. With subsidiaries in China (Wuhan, Shanghai and Suzhou) and the US (San Diego, CA), Neurophth is dedicated to developing genomic medicines for patients suffering from genetic diseases globally. Our AAV platform has been successfully validated through data from an investigator-initiated retinal gene therapy study. The significant findings from the research have been published in Nature-Scientific Reports, Ophthalmology and EBioMedicine. Our most advanced investigational gene therapy drug candidate, NR082, used for the treatment of Leber's hereditary optic neuropathy (LHON) associated with mtND4 mutation (ND4-LHON), has been granted orphan drug designation (ODD) by the U.S. FDA and the European Medicines Agency (EMA). It is the first Chinese gene therapy drug to receive Investigational New Drug (IND) approval for clinical trials from both China's NMPA and the FDA. Neurophth has completed the enrollment and administration of medication for all patients in the Phase III clinical trial in China as well as for the first patient in the Phase I/II clinical trial in the US. The company's second ODD drug in the US, NFS-02, has enrolled and administered medication to the first patient in its international multi-center Phase I/II clinical trials in both the US and China. The company's third gene therapy drug, NFS-05 for ADOA, has been approved for clinical trials in Australia. The company's pipeline also includes autosomal dominant optic atrophy, optic neuroprotection, vascular retinopathy, and other preclinical candidates. To learn more about us and our growing pipeline, please visit