Arctic Vision Announces Development and Commercialization of Ophthalmic Cell Therapy AE-101, a Potential Alternative to Corneal Endothelial Transplantation

- Arctic Vison and ActualEyes will co-develop a cell therapy targeting corneal endothelial dysfunction using cultured corneal endothelial cells. This therapy is expected to replace corneal endothelial transplantation.

- Under the terms of the agreement, total potential collaboration payments could exceed $35 million, including an upfront payment, R&D milestone payments, and tiered sales royalties.

May 14, 2021 - Arctic Vision, a clinical-stage ophthalmic biotechnology company focused on innovative therapies for the full spectrum of ocular diseases, and ActualEyes, a clinical-stage Japanese biotechnology company dedicated to the research and development of cell therapies for corneal endothelial diseases, today jointly announced the signing of an exclusive`License`Under the agreement, Arctic Vison will be responsible for the development and commercialization of ActualEyes' core product AE-101 in Greater China (Mainland China, Hong Kong, Macau, and Taiwan) and South Korea, forZhi YuTreatment of corneal endothelial dysfunction.

Under the terms of the agreement, ActualEyes is eligible to receive aggregate payments of up to more than USD 35 million, including an upfront payment, R&D milestone payments, and tiered sales royalties.

Corneal endothelial dysfunction results from damage to the corneal endothelium, which can be caused by various factors, including Fuchs endothelial corneal dystrophy, bullous keratopathy, trauma, glaucoma, and endothelial keratitis. It is estimated that there are over one million patients with corneal endothelial dysfunction in China. For patients with severe disease, the most effective current treatment is corneal transplantation (penetrating keratoplasty and endothelial keratoplasty). However, due to a severe shortage of donor corneas in clinical practice, fewer than 10% of patients in China are able to successfully undergo transplantation surgery. Meanwhile, transplant procedures carry potential risks such as postoperative graft rejection, infection, graft detachment, and unpredictable refractive outcomes. Furthermore, the surgery itself is highly technically demanding and requires surgeons with stringent qualifications and specialized expertise.

AE-101 is a novel cell injection therapy developed using cultured human corneal endothelial cells (hCECs) in combination with a Rho kinase (ROCK) inhibitor. Previous studies[i],[ii] have demonstrated that the injection of human corneal endothelial cells combined with a ROCK inhibitor exhibits favorable efficacy and safety in patients with corneal endothelial dysfunction. Breakthrough clinical research findings on AE-101 by ActualEyes’ scientific founders, Dr. Noriko Koizumi and Dr. Naoki Okumura, and colleagues, have been published in *The New England Journal of Medicine* and *Ophthalmology*. The results indicate that AE-101 effectively restores corneal endothelial function and vision in patients with corneal endothelial dysfunction, with no serious treatment-related adverse events observed within 5 years post-treatment. These findings represent a paradigm shift in corneal regenerative medicine research.

Dr. Eddy Wu, Founder, Chief Executive Officer, and Director of Arctic Vision, stated: "We are delighted to enter into this collaboration with ActualEyes to further advance the development of AE-101, a highly promising cell therapy for the treatment of corneal endothelial dysfunction. China is a region with a high prevalence of corneal diseases; however, due to a long-standing shortage of donor tissue, many patients have been unable to access timely treatment. The introduction of AE-101 will actively address the clinical need arising from the shortage of corneal transplant donors, providing patients with a safer, more effective, and more accessible innovative therapy. As the first cell therapy acquired by Arctic Vision, the introduction of AE-101 is not only a significant milestone in our pipeline expansion but also embodies our overarching strategy to steadfastly deliver breakthrough ophthalmic therapies to patients in China and across Asia, bringing hope for restored vision to more patients!"

ActualEyes President and Chief Executive Officer Iku Sugioka stated: "Cell therapy holds significant positive implications for patients with visual impairment or blindness caused by corneal endothelial dysfunction. Based on prior research findings, AE-101 has demonstrated exciting potential. Compared to traditional surgical interventions, it offers a less invasive and more efficacious therapeutic option in clinical practice. We look forward to partnering with Arctic Vision to advance the development of our core clinical candidate, AE-101. The Arctic Vision management team's outstanding track record in R&D and commercialization gives us confidence that this partnership will mark a promising start to a long-term collaboration. We will work together to bring this innovative cell therapy to broader markets across Asia."

***

About ActualEyes

ActualEyes is a clinical-stage biotechnology company headquartered in Japan, dedicated to the research and development of cell-based and drug therapies for corneal endothelial dysfunction, including Fuchs endothelial corneal dystrophy (FECD) and bullous keratopathy. The company was founded based on the research achievements of Professor Noriko Koizumi and Professor Naoki Okumura at Doshisha University in Kyoto, Japan. For more information, please visit www.actualeyes.co.jp.

About Arctic Vison

Arctic Vision is a clinical-stage ophthalmic biotechnology company with a leading portfolio of breakthrough ophthalmic therapeutic technologies. The Company’s vision is to address unmet medical needs in ophthalmology by delivering innovative therapies in China, across Asia, and globally. Founded with the backing of top-tier life science investors, Arctic Vision is led by an elite industry team with extensive and distinguished experience in the research, development, and commercialization of ophthalmic products both globally and in China. For more information, please visit https://arcticvision.com.

[i]  Kinoshita, Shigeru & Koizumi, Noriko et al. (2018). Injection of Cultured Cells with a ROCK Inhibitor for Bullous Keratopathy. The New England journal of medicine. 378. 995-1003. 10.1056/NEJMoa1712770.

[ii]Numa, Kohsaku & Imai, Kojiro et al. (2020). Five-Year Follow-up of First Eleven Cases Undergoing Injection of Cultured Corneal Endothelial Cells for Corneal Endothelial Failure. Ophthalmology. 128. 10.1016/j.ophtha.2020.09.002.