Corporate Fact Sheet 2ND QUARTER 2015 Acucela Inc. (Code No.: 4589, Tokyo Stock Exchange Mothers) is a clinical-stage biotechnology company that specializes in discovering and developing novel therapeutics to potentially treat and slow the progression of sight-threatening ophthalmic diseases affecting millions of individuals worldwide. Vision Innovative ophthalmology company focused on developing therapies to treat sightthreatening diseases Mission • To address unmet medical needs in sight-threatening diseases through scientific innovations • To build a unique company with continuous value creation for society • To create an excellent working environment and quality of life for employees Business Model Company Highlights Our business model has three strategically important foundational components that we expect will drive the Company’s growth in the large, underserved ophthalmic disease market: • 5/18/2015 Acucela Nominates Shintaro Asako to Its Board of Directors Strong and differentiated science: A franchise opportunity in visual cycle modulation (VCM), with one compound currently in a Phase 2b/3 trial and several other VCM compounds in development; Robust research and development infrastructure: Demonstrated capabilities in ophthalmic research and clinical development; and • 5/13/2015 Acucela Announces Publication of Pre-clinical Data for Emixustat Hydrochloride • 5/1/2015 Acucela Shareholders Elect New Board Members • 3/31/2015 Acucela Granted New Patent Covering Method of Use for Emixustat Hydrochloride Long-standing strategic partnerships: Partnered programs with Otsuka Pharmaceutical Co., Ltd., a global diversified healthcare company based in Japan. Pipeline DRUG VCM Emixustat HCI INDICATION PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3 ORIGINATOR ACUCELA TERRITORY Acucela North America (Joint), EU, South America and Africa Acucela Worldwide Otsuka US (Joint)3 Age-Related Macular Degeneration (AMD) Diabetic Retinopathy (DR)/Diabetic Macular Edema (DME) Stargardt, Retinitis Pigmentosa, Retinopathy of Prematurity1 Other VCM and retinal product candidates Stargardt, Retinitis Pigmentosa, Retinopathy of Prematurity OPA-6566 Glaucoma Under evaluation 2 1 Refers to back-up compounds 2 In 2012, we conducted a Phase 1/2 clinical trial in patients with glaucoma or ocular hypertension in the United States for OPA-6566, an adenosine A2a receptor agonist, under a development and collaboration agreement, or the Glaucoma Agreement, and our co-development partner, Otsuka, is currently evaluating next steps for the program. 3 Following completion of the Phase 2 and Phase 3 clinical trials, subject to our election to co-develop and co-promote OPA-6566. Acucela Corporate Fact Sheet 2ND QUARTER 2015 Investigational Product Candidates in Clinical Development Emixustat Hydrochloride (geographic atrophy associated with dry age-related macular degeneration) Age-related macular degeneration (AMD) is an ocular disease which compromises the retina and can result in vision loss, even blindness. The disease exists in two forms, dry (non-exudative) and wet (exudative). The dry form is characterized by the presence of lesions in the back of the eye which can spread over time. The most advanced stage of dry AMD is known as geographic atrophy (GA). Patients with GA can experience a gradual loss of fine central vision that is needed to see objects clearly and to perform common daily tasks, such as reading and driving. The wet form is characterized by the presence of leaky blood vessels in the back of the eye which deposit fluid and blood beneath the retina and is associated with a rapid loss of vision. Advanced AMD (GA and wet AMD) is the leading cause of vision loss and blindness among persons age 50 years and older in the United States. Emixustat hydrochloride is the first chemical entity in a class of novel, small molecule compounds called visual cycle modulators (VCMs). These compounds have been designed to specifically target enzymes of the visual cycle with the intent of slowing the progression of certain retinal diseases. Emixustat hydrochloride is currently being evaluated in a Phase 2b/3 clinical trial to determine whether it slows the progression of lesions in patients with GA associated with dry AMD (ClinicalTrials.gov identifier: NCT01802866). OPA-6566 (glaucoma) Glaucoma is a progressive optic neuropathy that leads to irreversible damage to retinal ganglion cells. The damage may lead to diminished visual function and blindness, especially when not adequately treated. Globally, glaucoma is one of the leading causes of blindness. Since there is no known cure for chronic glaucoma, the principal goal of therapy is to prevent further progression and preserve visual function once therapy is initiated. Most glaucoma cases are characterized by elevated intraocular pressure, or IOP, that, among other factors, leads to structural abnormalities of the optic disc or retinal nerve fiber layer, resulting in abnormalities of the visual field. These abnormalities are often asymptomatic until they become extensive. OPA-6566, developed by Otsuka Pharmaceutical, is a selective adenosine A2a receptor agonist that is formulated as a topical ophthalmic solution to reduce IOP in patients with ocular hypertension or open-angle glaucoma. OPA-656 employs a mechanism of action that is differentiated from existing therapies. In contrast to prostaglandin analogues, which reduce IOP by enhancing uveoscleral outflow, or ocular beta-blockers, which reduce the formation of aqueous humor, OPA-6566 is designed to enhance the conventional aqueous outflow pathway via the trabecular meshwork/Schlemm’s canal. Because of this novel mechanism of action for reducing IOP, Acucela believes OPA-6566 could be beneficial as a monotherapy treatment or in combination with other therapies. Leadership Ryo Kubota, MD, PhD Chairman, President and Chief Executive Officer John Gebhart Chief Financial Officer Karin Ludwig, MD Vice President Clinical Development Hans-Peter Pfleger, PhD Vice President Commercial Operations and Strategic Marketing Board of Directors Ryo Kubota, MD, PhD Chairman, President and Chief Executive Officer Acucela Inc. Yoshitaka Kitao Chief Executive Officer, SBI Holdings, Inc. Steve Tarr, PhD Chief Operating Officer Shiro Mita, PhD President and Chief Executive Officer M’s Science Corporation Ted Danse Chief Business Officer Eisaku Nakamura Director, Koinobori Associates Inc. Dewey H. Blocker, Jr. Vice President Finance Robert Takeuchi President, RT Consulting, Inc. Statements contained herein are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained herein that are not statements of historical fact may be deemed to be forward-looking statements. These statements involve risks and uncertainties. For a detailed discussion of these and other risk factors, please refer to our filings with the Securities and Exchange Commission, which are available on our investor relations Web site (http://ir.acucela.com/) and on the SEC’s Web site (http://www.sec.gov). Contacts US Headquarters 1301 Second Avenue Suite 4200 Seattle, WA 98101-3805 Phone: 206.805.8300 Fax: 206.805.8301 www.acucela.com Japan Office Level 18 Yebisu Garden Place Tower 4-20-3 Ebisu Shibuya-ku Tokyo 150-6018 Phone: +81.3.5789.5872 Fax: +81.3.5789.5874 www.acucela.jp Business Development Inquiries Ted Danse Chief Business Officer [email protected] Media Inquiries [email protected]