Neurophth Announces IND Approval by the NMPA for Leber Hereditary Optic Neuropathy Gene Therapy

WUHANand SUZHOU,China,April 1,2021 -- Neurophth Biotechnology Ltd.,a fully-integrated genomic medicines company developing adeno-associated viral (AAV)-delivered gene therapies for the treatment of ocular diseases,recently announced the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has approved the Company's Investigational New Drug (IND) application of NR082 (recombinant adeno-associated virus serotype 2 carrying ND4 gene; rAAV2-ND4) for Leber hereditary optic neuropathy (LHON) patients with ND4 mutations. It marks the first AAV2 gene therapy IND application approval in China. Previously,the company announced NR082 has granted orphan drug designation by the US FDA in September 20201.

The CDE of China NMPA-approved clinical trial is a multicenter,open-labeled,single-arm study investigating the safety and efficacy of NR082 in LHON patients with ND4 mutations. Investigational NR082,a novel recombinant adeno-associated viral serotype 2 (rAAV2) containing a codon-optimized of NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer,is a novel ophthalmic injection that is being developed for the treatment of LHON associated with ND4 mutations.

The team of Professor Bin Li,Founder and Chairman of Neurophth,has started the research and exploration of ophthalmic gene therapy technology since 2008 and launched the world's first investigator-initiated trial (IIT-1) of LHON gene therapy in 2011. Between 2017 and 2018,the team also completed the enrollment of an international multicenter clinical trial (IIT-2) of LHON gene therapy with the largest sample size in the world.Safety,efficacy and clinical durability up to 90 months of LHON gene therapy have been demonstrated in these two investigator-initiated trials (IITs). The results of these two studies of 168 LHON patients demonstrated that an intravitreal injection of rAAV2-ND4 in LHON patients is well tolerated and can be effective at improving visual acuity2,3,4,which provides favorable support for the industrial development of LHON candidate drugs.

"We are extremely pleased that the NMPA has granted permission to proceed with this clinical study. This is a big step toward a potential therapy for the LHON community. It has been a long journey since the team started supporting this promising gene therapy research over a decade ago. We are thrilled that it is now entering the clinic,bringing tremendous hope to families and the entire LHON community. I'd like to thank the team involved for their continued support," said Prof. Bin Li.

Dr. Alvin Luk,CEO of Neurophth,added,"NR082 is the first AAV ocular gene therapy to enter active clinical development in China. The program captures the essence of our mission – to liberate patients from debilitating ophthalmic diseases by rapidly advancing the in-vivo gene therapy programs into clinical development. We look forward to working with our study investigators,regulatory agency,the patient community,and families to get the clinical trial underway,the first of 10 gene therapy products currently under development at Neurophth."

About Neurophth

Neurophth is China's first gene therapy company for ophthalmic diseases. Headquartered in Wuhan with subsidiaries in Shanghai,Suzhou,and US,Neurophth,a fully integrated company,is striving to discover and develop gene therapies for patients suffering from blindness and other eye diseases globally. Our AAV validated platform which has been published in Nature - Scientific Reports,Ophthalmology,and EBioMedicine,successfully has delivered proof-of-concept data with investigational gene therapies in the retina. Our most advanced investigational candidate,NR082 (NFS-01 project,rAAV2-ND4),in development for the treatment ofND4-mediated LHON,has received orphan designation in theU.S. The pipeline also includesND1-mediated LHON,autosomal dominant optic atrophy,glaucoma,diabetic macular edema,and five other preclinical candidates. Neurophth has initiated the scaling up in-house manufacturing process in single-use technologies to support future commercial demand at the Suzhou facility. To learn more about us and our growing pipeline,visitwww.neurophth.com.

Reference

1. Press release from Neurophth. https://www.prnewswire.com/news-releases/neurophth-therapeutics-treatment-of-lebers-hereditary-optic-neuropathy-gene-therapy-nr082-was-granted-orphan-drug-designation-by-us-fda-301138001.html

2. Wan X,Pei H,Zhao MJ,et al. Efficacy and safety of rAAV2-ND4 treatment for Leber's hereditary optic neuropathy. Scientific Report,2016; 6:21587.

3. Yang S,Ma SQ,Wan X,et al. Long-term outcomes of gene therapy for the treatment of Leber's hereditary optic neuropathy. EBioMedicine,2016; 10:258-268.

4.Yuan JJ,Zhang Y,Liu H,et al. Seven-year follow-up of gene therapy for Leber's hereditary optic neuropathy. Ophthalmology,2020; 127(8): 1125-1127.

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