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BOSTON and WUHAN, China, May 11, 2020 /PRNewswire/ -- Neurophth Therapeutics, Inc., a clinical-stage biotechnology company discovering and developing innovative therapies for ocular and other genetic disease, today announced that the results of two clinical studies utilizing NFS-01 (rAAV2-ND4) in the treatment of Leber's Hereditary Optic Neuropathy (LHON) will be presented in May 2020 at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting and the Association for Vision and Ophthalmology (ARVO) Annual Meeting.
Founder Bin Li, M.D., Ph.D., professor of ophthalmology at the Tongji Medical College of Huazhong University of Science and Technology (HUST), initiated the first China gene therapy clinical trial in 2011, evaluating an adeno-associated virus serotype 2 (AAV2)-based gene therapy in nine Leber's Hereditary Optic Neuropathy (LHON) patients with the G11778A (ND4) mutation. Patients have been followed for over 7 years, several demonstrating significant visual acuity improvement and none reporting severe adverse events.
"The exciting results of the first study (NCT01267422) have been instrumental in developing the largest international clinical study (NCT03153293) in 159 LHON patients, who have been followed for 12 months," said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Chief Executive Officer of Neurophth. "Our results from NFS-01 demonstrate the potential of gene therapy to be both effective and safe for the treatment of LHON and further validate Neurophth's platform which is being applied across a growing pipeline of pre-clinical and clinical programs in gene therapy to treat a wide range of inherited diseases."
ASGCT: American Society of Gene and Cell Therapy Virtual Annual Meeting 2020
Presentations can be accessed through ASGCT's website at www.asgct.org on May 12, 2020.
First China International Gene Therapy Study in Leber's Hereditary Optic Neuropathy (Abstract #1307)
Oral presentation by Alvin Luk, Ph.D., M.B.A., C.C.R.A., Chief Executive Officer, Neurophth
Session Date/Time: Friday, May 15, 10:45AM EDT
Session Title: AAV Vectors - Clinical Studies I
Multiyear Follow-up of AAV2-ND4 Gene Therapy for Leber's Hereditary Optic Neuropathy (Abstract #621)
Poster presentation by Su Xiao, Ph.D., Co-Founder and Chief Technical Operations Officer, Neurophth
Session Date/Time: Wednesday, May 13, 5:30PM EDT
Session Title: AAV Vectors - Clinical Studies
ARVO: Association for Vision and Ophthalmology
The ARVO 2020 Annual Meeting has been modified to a virtual format and presentations have been published online on May 6, 2020.
First China International Gene Therapy Study in Leber's Hereditary Optic Neuropathy (Abstract #5182)
Oral Presentation by Alvin Luk, Ph.D., M.B.A., C.C.R.A., Chief Executive Officer, Neurophth
Narrated presentation will be available online
Scientific Section: Eye Movements/Strabismus/Amblyopia/Neuro-Ophthalmology
Neurophth Therapeutics, Inc. ("Neurophth") is China's first gene therapy company for ophthalmic diseases. Founded in 2016 by Professor Bin Li, M.D., Ph.D., the clinical-stage biotechnology company is dedicated to discovering and developing gene therapies for ocular and other genetic diseases. Currently, Neurophth is advancing several clinical-stage and pre-clinical programs in ophthalmology. In April 2020, Neurophth successfully raised $18.4 million USD in a Series A round led by Fosun's InnoStar Venture and Sequoia Capital China. The company plans to bring the lead assets to patients globally and build a GMP gene therapy manufacturing facility for global supply.
NFS-01 is an AAV construct comprising with promoter, mitochondrial targeting sequence (MTS), correct ND4 gene sequence, and an untranslated region flanked by two inverted terminal repeats (ITRs). In 2011, Neurophth initiated the world's first investigational NFS-01 (AAV2-ND4) gene therapy study (NCT01267422) of a single intravitreal injection into one of the eyes of patient with ND4 mutation associated with Leber's Hereditary Optic Neuropathy (LHON). The corrected ND4 gene is transferred into the cell to be expressed and eventually produces the missing functional protein to restore mitochondrial function of the respiratory chain. After nearly 8 years of long-term follow-up, the treatment displayed good safety profiles and patients have maintained significant visual acuity improvement to-date. These results demonstrated long-term safety and durability of clinical application of gene therapy technology and were published in Ophthalmology 2020. Encouraged by these results, Professor Li's team has completed an international gene therapy clinical trial (NCT03153293) during 2017-2018 in 159 LHON patients in China and Argentina, which is the world's largest clinical trial for LHON gene therapy.
About Leber's Hereditary Optic Neuropathy (LHON)
Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited mitochondrial disorder which often manifests as bilateral visual loss. The most common mutation is the mitochondrial DNA at nucleotide position of 11778G>A, which is located within the NADH ubiquinone oxidoreductase subunit-4 (ND4) gene. This mutation causes impairment of the respiratory chain, adenosine triphosphate deficiency, and oxidative stress in retinal ganglion cells, leading to apoptosis. There is currently no effective treatment or cure for LHON.