WUHAN, China and SAN DIEGO, Calif. — Neurophth Therapeutics, Inc. announced today that the last patient has been enrolled in Phase I/II clinical trial of Opvika® (Esonadogene Imvoparvovec) for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON).
Neurophth is conducting a Phase I/II, single-arm, multi-center study aimed at evaluating the safety and efficacy of NR082 in LHON patients with ND4 mutations in US. The Investigational New Drug (IND) application of NR082 has been approved by the U.S. Food and Drug Administration on January 18, 2022, and the first patient was dosed in June 2023.
“We are extremely grateful for the trust and support of patients and their families, as well as the dedication of all researchers. They have played a crucial role in the successful completion of patient enrollment for the Phase I/II clinical trial,” said Professor Li Bin, Founder, Chairman, and CEO of Neurophth. “Neurophth has taken a solid step forward in going global. As our motto is “In China, for global”, we will strive to bring China’s medical development and more innovative gene therapies to patients worldwide.”
The principal investigator, Prof. Yaping Joyce Liao from Stanford Health Care (SHC) – Byers Eye Institute, comments, “I am excited about the significant progress in the completion of patient enrollment and dosing in the Phase I/II clinical trial. LHON patients are eagerly waiting for a new therapy like Opvika. I look forward to seeing the results from this Phase I/II clinical trial with 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 Opvika, 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 for the Phase I/II clinical trial in the U.S. Our second gene therapy NFS-02, has dosed 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. Our pipeline also includes optic neuroprotection, vascular retinopathy, and five other preclinical candidates.
Contact
Phone: +86-27-65524119
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