Neurophth gets Australian approval for ophthalmic gene therapy

(Image Credit: AdobeStock/Radomir Jovanovic)

Neurophth Therapeutics today 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).

According to the company, ADOA is an autosomal dominant inherited optic neuropathy, and 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.

The company also noted that patients diagnosed with the disease present with bilateral, slowly progressing visual impairment, temporal pallor of the optic disc, central visual field defects, and abnormalities in color vision.

Moreover, the company noted that there are no effective treatments on the market for ADOA in clinical practice and its proprietary ophthalmic injection, NFS-05, utilizes a gene therapy approach that delivers an AAV vector containing the OPA1 gene into the vitreous cavity. The company noted this vector then targets RGC cells, leading to the expression of the OPA1 protein and subsequent restoration of mitochondrial function.

Professor Li Bin, founder, chairman, and CEO of Neurophth, pointed out in the news release the significance of the approval

"Neurophth's NFS-05 marks our third drug to receive clinical trial approval and our inaugural approval in Australia," Bin said in the news release. "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."

Xiaoning Guo, PhD, chief medical officer at Neurophth, pointed out in the news release that current treatment otions for ADOA, including options that 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,” Guo said in the news release. “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."

According to news release, Neurophth is an in-vivo gene therapy company for ophthalmic diseases with subsidiaries in China (Wuhan, Shanghai and Suzhou) and the United States (San Diego, California). The company noted that it 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 company said in the release.“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).”