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WUHAN and SAN DIEGO, Jul. 15, 2022 – Neurophth Therapeutics, Inc., (“Neurophth”) today announced that the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has granted a Breakthrough Therapy Designation (BTD) to the Company’s leading gene therapy drug candidate, NR082 (rAAV2-ND4), for the treatment of Leber’s hereditary optic neuropathy (LHON) associated with mtND4 mutation.
NR082 (rAAV2-ND4), a recombinant adeno-associated viral vector, serotype 2, containing human ND4 codon-optimized 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. Phase I/II/III clinical trials are currently underway after the IND clearance by the China NMPA. Leveraging gene therapy strategy, NR082 viral vector is used through intravitreal injection to deliver the corrective genes to the patients' damaged retinal ganglion cell (RGC), therefore to repair the mitochondrial respiratory chain, improve the sensory function of the RGC and restore the visual function in LHON patients.
A breakthrough therapy must provide effective treatment or prevention for a seriously debilitating or life-threatening condition that has no effective therapy or demonstrate substantial improvement over available therapies. Breakthrough Therapy Designation instituted in China is designed to expedite the development and review of innovative drugs presenting significant clinical advantages. According to the CDE, BTD provides opportunities for more intensive CDE guidance and discussion with respect to clinical trials and development strategy, and for priority review later.
“The BTD is a great milestone for Neurophth and NR082. It’s also an important advancement toward addressing the unmet medical need in LHON patients with a China gene therapy.” said Prof. Bin Li, Founder, Chairman and CEO of Neurophth, “Further support from the CDE will help us accelerate the clinical development and commercialization of NR082. Neurophth will advance the Phase III clinical trial in China, as well as clinical trials in the U.S. Our team is fully committed to bringing NR082 to the market as soon as possible, alleviating patients from ophthalmic diseases with a transforming gene therapy.”
Ms. Yiyuan Chen, Head of Global Regulatory Affairs of Neurophth, commented, “We are very pleased to receive CDE’s BTD, which is another good news following the orphan drug designation (ODD) by the U.S. FDA and EMA, and the IND clearance by both China NMPA and U.S. FDA. This reinforces our confidence in advancing NR082 to be the first marketed gene therapy for ophthalmic diseases in China. Neurophth will continue our work in hopes that this new gene therapy will soon benefit patients around the world.”
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, Neurophth has successfully completed an EOP2 meeting with CDE and reached an agreement on the design of the Phase III clinical trial. 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 www.neurophth.com.