Commercialization of Gene Therapy is coming soon: Neurophth Completes Patient Enrollment for Phase III clinical trial for the gene therapy treatment of LHON

    WUHAN, China and SAN DIEGO, Feb. 21, 2023 /PRNewswire/ -- Neurophth Therapeutics, Inc. ("Neurophth") announced today that the last patient has been enrolled in the Phase III clinical trial for the treatment of Leber hereditary optic neuropathy (LHON) in China. It marks the completion of patient enrollment of the Phase I/II/III, multi-center, two-parts study aimed at evaluating the safety, tolerability, and efficacy of NR082 in LHON patients with ND4 mutations.

    Milestones of NR082

    • September 24, 2020 - NR082 (rAAV2-ND4, NFS-01) was granted an orphan drug designation (ODD) by the U.S. FDA for the treatment of Leber's Hereditary Optic Neuropathy (LHON) associated with ND4 mutation. It marks China's first self-developed orphan drug granted by the U.S. FDA.
    • March 30, 2021 - The Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has approved Neurophth's Investigational New Drug (IND) application of NR082. It marks the first AAV2 gene therapy IND application approval in China.
    • June 28, 2021 - The first patient has been dosed in G.O.L.D. clinical trial for the treatment of LHON.
    • January 18, 2022 - Neurophth received the U.S. Food and Drug Administration clearance of its investigational new drug (IND) application on the NR082, the first of its kind in China, for the treatment of LHON associated with ND4 mutation.
    • January 24, 2022 – The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) granted the orphan drug designation for NR082. NR082 is China's first self-developed in vivo gene therapy that has been granted orphan drug designation by EMA.
    • May 25, 2022 - Neurophth has successfully completed an EOP2 meeting with The Center for Drug Evaluation and reached an agreement on the design of the Phase III clinical trial.
    • July 15, 2022 - The Center for Drug Evaluation of China National Medical Products Administration has granted a Breakthrough Therapy Designation (BTD) to NR082.
    • September 27, 2022 – The first patient has been dosed in Phase III clinical trial. Wenbin Wei, vice president of Beijing Tongren hospital affiliated to Capital Medical University, completed the first dose.
    • February 22, 2023 – NR082 has completed the patient enrollment of the Phase III clinical trial.

    "We are extremely excited to announce the completion of the last patient visit in this Phase III study, which marks a significant advancement for NR082 as well as a minor step toward the commercialization of China's self-developed ocular gene therapy. It often takes a decade to develop an innovative drug, and Chinese ophthalmic gene therapy drug costs much more than that." said Professor Bin Li, Founder, Chairman and CEO of Neurophth, "We would like to express our gratitude to all clinical investigators, trial participants and their families for their time and commitment, and also the excellent teamwork of the entire company to complete the Phase III clinical trial within 5 months. We are confident that the company will live up to our promise to commercialize Chinese in vivo gene therapy soon and make it available to patients."

    "The design of the NR082 clinical trial and the selection of the optimal dose were based on multiple successful previous studies and in accordance with the CDE. Neurophth and CRO collaborated closely to overcome the challenges of COVID-19 and complete the enrollment of all patients successfully." said Dr. Xiaoning Guo, Chief Medical Officer of Neurophth, "We are optimistic about the outcome of this clinical trial. If the study meets the pre-defined endpoints, we will submit a New Drug Application as soon as possible for the early launch of NR082 in China and in the meantime, advance the progress of clinical trial in the U.S. to offer a direly needed treatment for LHON patients."

    About Leber's Hereditary Optic Neuropathy (LHON)

    Leber hereditary optic neuropathy (LHON) is a maternally inherited blinding bilateral optic atrophy with a prevalence of around 1 in 31,000 to 1 in 54,000 particularly in young adult males. There are three mitochondrial DNA point mutations account for about over 90% of all LHON cases, namely, G3460A in ND1, G11778A in ND4 and T14484C in ND6, with G11778A mutation in NADH-dehydrogenase subunit 4 (ND4) gene causing a ND4 subunit arginine to be incorrectly replaced by a histidine and reducing the activity of NADH dehydrogenase by 50-80% as being the most common mutation worldwide. These mutations affect complex I subunits of the mitochondrial respiratory chain, impairing mitochondrial function and increasing the production of reactive oxygen species. The retinal ganglion cells (RGCs) appear to be selectively vulnerable to mitochondrial dysfunction resulting in apoptotic cell death, optic nerve degeneration, and the development of optic atrophy. Thus, the pathophysiology of LHON is characterized by selective loss of RGCs and their axons, which leads to rapidly progressive bilateral vision loss. There is currently no approved effective treatment for LHON and the current treatment remains limited.

    About NR082

    Investigational NR082 (rAAV2-ND4), a novel recombinant adeno-associated viral vector, serotype 2, containing a mitochondria codon-optimized NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer, is a novel gene therapy product that is being developed for the treatment of Leber hereditary optic neuropathy (LHON) associated with mtND4 mutations. NR082 can deliver the correct genes to the patients' damaged optic ganglion cells through introvitinreous injection, thus repair the mitochondrial biological respiratory chain, and restore the vitality and visual function of optic ganglion cells.

    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. Recently, our second gene therapy drug candidate NFS-02, has been granted IND clearance from U.S. FDA. The pipeline also includes autosomal dominant optic atrophy, optic neuroprotection, vascular retinopathy, and five other preclinical candidates. Neurophth has scaled up in-house manufacturing capability in Suzhou facility utilizing single-use technologies to support future commercial demand. To learn more about us and our growing pipeline, visit

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