Neurophth Announces Completion of Patient Enrollment in U.S. Phase I/II Clinical Trial of AAV Gene Therapy NFS-01 (Opvika®) for ND4-LHON

Feb 19, 2024 08:43 CST Updated 08:43

Neurophth

Ophthalmic Gene Drug Developer

On February 19, 2024, Neurophth, a leader in gene therapy for ophthalmic diseases, announced the core product of the company.Neuroptima^®(Opvika, Epepedoxi Ophthalmic Injection), used for treatmentND4Mutation-induced Leber's Hereditary Optic Neuropathy (ND4-LHON), Phase I/II clinical trials completed in the United StatesCompletion of enrollment and dosing for all patients.

Neurophth is conducting a Phase I/II, single-arm, multicenter clinical study in the United States to evaluate the safety and efficacy of gene therapy for ND4 mutation-associated Leber's Hereditary Optic Neuropathy (LHON). The Investigational New Drug (IND) application for this project was approved by the U.S. Food and Drug Administration (FDA) on January 18, 2022, and the first patient was dosed in June 2023.

"We are very grateful for the trust and support of the patients and their families, as well as the participation and dedication of all researchers, who played a key role in the successful completion of the Phase I/II clinical enrollment."Professor Bin Li, Founder, Chairman and Chief Executive Officer of NeurophthStated, "Neurophth has taken a solid step on the path of bringing innovative drugs overseas. In China, For Global, we will strive to bring China's medical research achievements to the world, offering more innovative gene therapies to patients with ophthalmic diseases globally."

The Principal Investigator (PI) of this clinical trial,Professor Yaping Joyce Liao from the Byers Eye Institute at Stanford UniversitySaid: "The significant progress achieved in this Phase I/II clinical trial is truly exciting. LHON patients are eagerly awaiting new therapies like Nuweijia® (Opvika) to bring them the possibility of a cure. I am very much looking forward to witnessing the results of the Phase I/II clinical trial together with Neurophth."

About Neurophth

Neurophth is a leader in the gene therapy industry for ophthalmic diseases in China, with subsidiaries in Wuhan, Suzhou, Shanghai, and San Diego, USA, dedicated to developing gene therapies for hereditary diseases for patients worldwide. The trial data from investigator-initiated retinal gene therapy studies have successfully validated the AAV platform we use, and the research results have been published inNature-Scientific Report、OphthalmologyAndEbioMedicineUp.Our Core ProductsNeuroptima^®, Aimed at TreatingND4Mediated Leber's Hereditary Optic Neuropathy (ND4-LHON), has been granted Orphan Drug Designation (ODD) by the U.S. FDA and the European Medicines Agency (EMA). It is the first China-produced gene therapy new drug to receive clinical trial IND approval from both China's NMPA and the U.S. FDA. The Phase III clinical trial in China has completed patient enrollment and dosing, as well as the enrollment and dosing of all patients in the U.S. Phase I/II trials. The company’s second U.S. ODD new drug, NFS-02, has completed the first patient enrollment and dosing in the China-U.S. international multicenter Phase I/II clinical trial. The company’s third gene therapy new drug for ADOA, NFS-05, has been approved for clinical trials in Australia. The company's pipeline also includes preclinical candidate drugs for optic neuroprotection and vascular retinopathy.

E.N.D

Previous article recommendations:

The Competitive Landscape of TIL Cell Therapy in China

The World's First TIL Therapy Approved for Marketing

Government Invests 6 Billion Yuan to Fund the Establishment of a Life Health Research Institute, Focusing on Cutting-edge Topics such as Cell and Gene Therapy

NVIDIA CEO Jensen Huang: The Era of Having to Learn Computer Science and Programming is Over, the Next Golden Track is Life Science and Engineering

The Fate of CAR-T Cells in the Human Body

EU Commission Approves First Gene-Editing Therapy for Marketing

AstraZeneca Invests $300 Million to Build Cell Therapy Factory

LianBio to Cease Operations

First in China, Universal CAR-NK for Treating Systemic Lupus Erythematosus Approved for Clinical Trials

CGT CDMO Leader Acquired for Billions

World's First AAV Gene Therapy for Autism Approved for Clinical Trials

Beijing to Focus on Cell and Gene Therapy, Synthetic Biology Manufacturing, and Other Cutting-Edge Emerging Fields as Key Tasks for 2024

Leading CDMO Company Discloses Nearly 100 Million Yuan Fraud Process

Bi Jingquan: Research should be conducted on removing cell therapy and gene therapy from the negative list for foreign investment.

FDA Releases Guidelines for the Development of Gene-Edited Therapies and CAR-T Therapies (Full Text of Guidelines Attached)

The 10 Major Fields in Biopharmaceuticals to Focus on in the Next Decade

Shize Bio Clinical-Grade iPSC-Derived Cell Therapy for ALS Clinical Research Officially Launched

Overview of U.S. Biomedical Sanctions Against China

Hengrui Medicine Subsidiary Submits First Gene Therapy Product

CDE Releases "Technical Guiding Principles for Non-Clinical Research of Human Stem Cell Products"

CRISPR/Cas9 Gene Editing Therapy Receives FDA Approval for Treating β-Thalassemia

The Top Ten Advances in China's Medical and Biotechnology in 2023 Revealed

Statement: The content is sourced from Neurophth's official channels.The content of this article is intended for knowledge sharing. All content is for reference only and does not constitute any advice.