Acucela Inc. (Tokyo:4589) (“Acucela”), a clinical-stage ophthalmology company that specializes in identifying and developing novel therapeutics to treat and slow the progression of sight-threatening ophthalmic diseases, and YouHealth Eyetech, Inc. (“YouHealth”), announced today an agreement involving an exclusive option from YouHealth on lanosterol technology licensed from the Regents of the University of California, San Diego for the non-surgical pharmacologic treatment of cataracts.

Cataracts are responsible for 51% of all blindness1 as well as 24 million surgical procedures worldwide in 20152. Cataracts are a pervasive condition characterized by optical opacities that develop as a result of protein aggregation in the crystalline lens of the eye, generally due to the natural aging process. Currently, there is no non-surgical treatment available for cataracts. A non-invasive therapy that improves lens clarity and vision at the early stage of cataract development and may reverse lens opacification at later stages would be considered a significant medical breakthrough.

This therapy, if approved, would be the first of its kind for the pharmacologic treatment of cataracts. Acucela plans to initially evaluate lanosterol as a treatment for mild cataracts with an option to fully develop and commercialize the compound globally with the exception of Greater China including China, Taiwan and Hong Kong. The use of lanosterol in other ophthalmic conditions such as presbyopia may be investigated by Acucela in the future. YouHealth’s parent company, Guangzhou Kang Rui Biological Pharmaceutical Technology Co. Ltd. (“Kang Rui”) will be responsible for lanosterol’s clinical development and commercialization in Greater China including China, Taiwan and Hong Kong.

Lanosterol, a naturally occurring compound found in the human body, has shown promise for this condition in several preclinical studies. It has been identified as a key molecule in the prevention of lens protein aggregation and lens opacification. A scientific collaboration between Dr. Kang Zhang MD, PhD, Dept. of Ophthalmology, University of California, San Diego and scientists associated with Kang Rui identified two genetic mutations that impair key catalytic functions responsible for maintaining the normal crystalline structure of the lens. They then identified and published the therapeutic effect of lanosterol in in-vitro, cell-transfection and in-vivo experiments3.

“The potential for the lanosterol technology to provide an entirely new treatment paradigm for early stage cataracts raises hope for millions who now can only wait for their cataracts to worsen to the point where surgery is warranted. We welcome the opportunity to work with YouHealth to make this therapy a reality,” stated Dr. Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela.

“We are excited to work with Acucela to advance this novel technology into development for the treatment of cataract,” said Rui Hou, CEO of Kang Rui.

1 Visiongain, Ophthalmic Drugs Market Forecast, 2015-2025; World Health Organization
2 Market Scope, The Global IOL Market 2015
3 Nature 523 (2015) 607-614

About Acucela Inc.

Acucela Inc. (http://www.acucela.com/ or http://www.acucela.jp) is a clinical-stage ophthalmology company that specializes in identifying and developing novel therapeutics to treat and slow the progression of sight-threatening ophthalmic diseases affecting millions of people worldwide. Acucela’s lead investigational drug candidate, emixustat hydrochloride, is currently in clinical development in collaboration with Otsuka Pharmaceutical Co., Ltd. for geographic atrophy associated with dry age-related macular degeneration based on Acucela’s proprietary visual cycle modulation technology.

About Guangzhou Kang Rui Biological Pharmaceutical Technology Co. Ltd. and YouHealth Eyetech, Inc.

Guangzhou Kang Rui Biological Pharmaceutical Technology Co. Ltd., the parent company of YouHealth Eyetech, Inc., is a leading R&D company specialized in novel ophthalmic drug and treatment developments in China. It has several first in class drugs under development for cataract, age-related macular degeneration, corneal diseases, glaucoma and dry eye.

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