Neurophth announces first patient dosed in Phase III clinical trial for the gene therapy treatment of LHON

Published: Sep. 27, 2022 at 7:30 AM CDT

WUHAN, China and SAN DIEGO, Sept. 27, 2022 /PRNewswire/ -- Neurophth Therapeutics, Inc. ("Neurophth") announced today that the first patient has been dosed in Phase III clinical trial for the treatment of Leber hereditary optic neuropathy (LHON).


Neurophth is conducting a 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. The Investigational New Drug (IND) application of NR082 has been approved by CDE on March 29, 2021. Wenbin Wei, vice president of Beijing Tongren hospital affiliated to Capital Medical University, completed the first dose in the Phase III clinical trial.

Prof. Wenbin Wei, vice president of Beijing Tongren hospital, CMU, commented, "As a clinician, I share the same expectation of LHON patients for effective and affordable treatments. NR082 has shown promising safety and efficacy data in all clinical work to date and is officially advanced into a Phase III trial. Ophthalmic gene therapy is coming of age. We hope that NR082 will be available soon, making a life-changing difference to patients by technological innovation."

Professor Bin Li, Founder, Chairman and CEO of Neurophth, said, " Dosing the first participants in Phase III trial is a significant milestone of Neurophth and a positive sign of the booming of ophthalmic gene therapy industry. Our team will continue to make every effort to accelerate the clinical trials and commercialization process of China's first ophthalmic gene therapy. We believe that NR082 will have positive results of Phase III clinical trial, delivering a solution that can transform the lives of LHON patients in return for their years of waiting."

"There is currently no approved effective treatment for LHON. NR082 leverage gene therapy strategy and use recombinant adeno-associated virus as the vector to deliver the correct genes to the patients' damaged optic ganglion cells, thus restore the vitality and visual function of optic ganglion cells." Dr. Xiaoning Guo, Chief Medical Officer of Neurophth, said, "The Phase III clinical trial is a breakthrough in the ophthalmic gene therapy industry. Neurophth will advance the Phase III clinical trial in hope that safe and effective therapy can soon liberate patients from the darkness."

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. The U.S. Food and Drug Administration (FDA) granted orphan-disease designation to NR082 in September 2020. Safety and efficacy of mtND4 gene therapy have been evaluated in three investigator-initiated trials (IITs) with clinical durability up to 90 months in the first IIT. The results of these three IITs of 186 LHON patients demonstrated that an intravitreal injection of rAAV2-ND4 in subjects with LHON is well tolerated and can be effective at improving visual acuity.

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, the first patient has been dosed in the Phase III clinical trial in September 2022. The pipeline also includes mtND1-mediated LHON (the Company's 2nd US ODD), 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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