Esonadogene Imvoparvovec

The groundbreaking ADOA treatment marks a significant breakthrough in the field WUHAN, China and SAN DIEGO, Sept. 6, 2023 /PRNewswire/ -- 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 . View original content to download multimedia: SOURCE Neurophth Therapeutics, Inc.

WUHAN, China and SAN DIEGO, Aug. 17, 2023 /PRNewswire/ -- 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 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. 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 Reports, Ophthalmology, 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 . SOURCE Neurophth Therapeutics, Inc.