Neurophth Biotechnology Ltd., a fully-integrated genetic medicines company developing AAV-delivered gene therapies for the treatment of ocular diseases, announced the closing of a RMB 400 million ($61.9 million USD) Series-B financing with a premier syndicate of investors, co-led by Guofang Capital and InnoVision Capital. Additional new investors include funds and accounts managed by Oriza Holdings, Harvest Capital and Grand Mount Capital with participating from existing investors Sequoia Capital China and Northern Light Venture Capital.  Proceeds from the Series-B financing will support the anticipated IND submissions, completion of the commercially scalable GMP manufacturing facility in accordance with international quality standards, initiation of establishing the translational research centers and development of the company's extensive portfolio of potentially curative gene therapies for the treatment of ocular genetic diseases.

"The support of our investors not only speaks to the immense potential of our development portfolio of transformative treatment options for patients with ocular diseases but is also a reflection of their confidence in the expertise of the Neurophth team," said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Chief Executive Officer at Neurophth. "As we continue to work toward advancing our lead candidate, NR082 (recombinant adeno-associated viral serotype 2 carrying ND4 gene; rAAV2-ND4), into the clinic for Leber hereditary optic neuropathy (LHON) associated with ND4 mutations over the coming year, we are focused on our mission to serve patients by offering best-in-class, life-transforming therapies." Neurophth expects to initiate clinical trials for its lead program in ND4-mediated LHON in the first half of 2021, followed by the completion of the Suzhou commercial manufacturing facility and initiation of translational R&D centers. Neurophth is currently developing a deep and sustainable pipeline of 10 gene therapy product candidates in both rare and common ocular diseases.

"We have brought together experts in gene therapy with overseas experience to create a company that is uniquely positioned to advance the development and manufacturing of potentially curative gene therapies for ocular diseases, particularly glaucoma and diabetic macular edema with large patient populations," said Bin Li, M.D. Ph.D., Founder and Chairman of the Board of Neurophth. "We believe this financing provides significant validation of our corporate strategy and will enable us to continue to rapidly translate programs from preclinical development into the clinic."

Mr. Yaguang Wei, Partner at Guofang Capital said, "Gene therapy is one of the most promising directions of life science, in which AAV-based gene therapy shows both safety and great clinical potential in the treatment of diseases like ophthalmology. Neurophth has an international top-notch management team and a wealth of ophthalmology gene therapy pipeline, the clinical progress and efficacy of its core pipeline are leading the track in China. We're honored to be part of Neurophth's future success by connecting high-quality industrial resources, facilitating vertical collaboration, and promoting smooth technological transfer for the benefit of patients suffering from eye disorders worldwide."

Mr. Lane Zhao, Founding Partner of InnoVision Capital, said: "Neurophth has achieved significant research and clinical progress since its establishment, showcasing its strong R&D capabilities led by Prof. LI and the world-class seasoned management teams. We are delighted to lead this round, and this investment underscores our long-term commitment to strategically investing in biotech and innovative technologies improving human health and life quality. We look forward to working alongside the Neurophth team to bring high-quality, worldwide competitive, significant gene therapy products to doctors and patients, globally." 

"In the past year, Neurophth has made significant progress in product pipeline and team building, which shows the strong executive power of the management. Sequoia Capital China will continue to support the company and believe that Neurophth can make gene therapy products benefit patients all over the world to meet the unmet clinical needs," said Ms. Trency Gu, Managing Director of Sequoia Capital China.

Neurophth closed its Series-A financing in early 2020 to initiate the construction of the commercially scalable GMP manufacturing facility, establish an international leadership team, and accelerate nonclinical studies of its lead candidate, NR082, which was granted an orphan disease designation (ODD) by the U.S. FDA for the treatment of LHON associated with ND4 mutations in Q3-2020, in addition to advancing other gene therapy programs.

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 of ND4-mediated LHON, has received orphan designations in the U.S. The pipeline also includes ND1-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. 

About Guofang Capital

Guofang Capital was initiated by Shanghai International Group in 2017. Guofang Capital is a professional market-based equity investment platform with an asset under management (AUM) of over 10 billion RMB. Focused on healthcare, AI and IoT industries, the Yangtze River Delta Synergy Industry Investment Fund managed by Guofang Capital aims to play a leading role in guiding private capitals, establishing industrial investment fund ecosystem, and promoting the overall development of China's strategic industries through fund-of-fund (FOF) and direct investment.

About InnoVision Capital

InnoVision Capital is a leading private equity firm founded by former KKR senior executives. With best-in-class international vision, outstanding experience, and industry resources, InnoVision Capital primarily focuses on investment opportunities across the Technology, Consumer, and Healthcare sectors. InnoVision is supported by a diverse group of world-class institutional investors, including university foundations and industry giants among others. Since the inception, the firm has successfully invested into Beike, JD Logistics, China Securities, 58.com, AutoHome, ChannelSoft, Wanhua Chemical, HQWX, China UnionPay Merchant Services, Swan Home Services, Butel, Uni-Ubi, Kedu Healthcare, Huanyu Education, Lalami, Lvkon, Qutoutiao, etc.

About Sequoia Capital China

The Sequoia Capital China team helps daring founders build legendary companies. In partnering with Sequoia Capital China, companies benefit from our unmatched community and the lessons we've learned over 49 years. As "The Entrepreneurs Behind The Entrepreneurs", Sequoia Capital China focuses on three sectors: TMT, healthcare and consumer/service. Over the past 16 years we've had the privilege of working with approximately 600 companies in China.

About Suzhou Oriza Holdings

Suzhou Oriza Holdings Corporation is an investment holding company with nearly 100 billion RMB under management. Its businesses cover equity investment, debt financing and investment & financing services. Oriza Holdings has achieved a great success in exploring financial creation, boosting science creation, guiding fund accumulation and upgrading industries through the coordination of the three businesses of capital operation, risk control and operation allocation, including China' s first national fund-of-funds (FOFs) of equity investment etc. Oriza Holdings is committed to becoming the preferred financial partner for entrepreneurs and an investment holding company with international influence.

About Suzhou Harvest Capital

Suzhou Harvest Capital is a fund management company established by the Management Committee of Suzhou Industrial Park (SIP), with a registered capital of 50 million RMB. Currently, the fund management scale is 12 billion RMB, of which 10 billion RMB is a government industry guidance fund and 2 billion RMB of angel funds. The management team members of Harvest Capital are all from well-known domestic investment institutions, with strong industrial background and rich investment experience. Involving in direct investment and fund-of-funds investment, Harvest Capital gives full play to the leading role of industrial guidance fund and fund of angel funds to promote the implementation and development of projects of key industries of SIP, thereby accelerating the construction of SIP into a world-class high-tech industrial park.

About Northern Light Venture Capital

Northern Light Venture Capital (NLVC) is a leading China-focused venture capital firm targeting early stage opportunities of innovation and disruptive technology. Since founded in 2005, Northern Light has backed near 400 ventures in enterprise, healthcare and consumer. Leveraging significant investing and entrepreneurial expertise in both China and the United States, the NLVC team looks to support entrepreneurs with groundbreaking ideas and exceptional vision to develop lasting ventures that positively impact society. 

About Grand Mount Capital

Grand Mount Capital (GMC) is a dedicated healthcare venture capital firm established by state-owned capital and investment professionals in 2019. GMC's investment spans between biopharmaceuticals, medical devices, and diagnostics. Adhering to the strategy of "industry participation+ capital assistance", GMC works closely with multiple healthcare conglomerates to add value to portfolio companies by providing active support in market access, collaborations, regulatory guidance, etc. GMC aims to deliver long-term and sustainable return for investors while becoming a trusted partner to entrepreneurs that aim to build great companies with breakthrough technologies.

Reference

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

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.

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.

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.

Carelli V, Carbonell M, deCoo IF, et al. International consensus statement on the clinical and therapeutic management of Leber hereditary optic neuropathy. Journal of Neuroophthalmology, 2017; 37: 371-381.

Meyerson C, Van Stavern G, Mcclelland C. Leber hereditary optic neuropathy: current prespectives. Clinical Ophthalmology, 2015; 9:1165-1176.

Yu-Wai-Man P, Votruba M, Burte F, et al. A neurodegenerative perspective on mitochondrial optic neuropathies. Acta Neuropathology, 2016; 132: 789-806.

Newman NJ, Lott MT, Wallace DC. The clinical characteristics of pedigrees of Leber's hereditary optic neuropathy with the 11778 mutation. American Journal of Ophthalmology, 1991; 111: 750-762.

Jurkute N, Harvey J, Yu-Wai-Man P. Treatment strategies for Leber hereditary optic neuropathy. Current Opinion of Neurology, 2019; 32: 99-104.