Acucela Announces Publication of Pre-clinical Data for Emixustat Hydrochloride

SEATTLE

Acucela Inc. (TOKYO:4589), a clinical-stage biotechnology company that specializes in discovering and developing novel drug candidates to potentially treat and slow the progression of sight-threatening ophthalmic diseases, today announced the publication of pre-clinical data related to the company’s investigational drug candidate, emixustat hydrochloride (emixustat), in the scientific journal PLOS ONE.

The published article, “Visual Cycle Modulation as an Approach Toward Preservation of Retinal Integrity,” can be accessed at: http://dx.plos.org/10.1371/journal.pone.0124940. For additional information about PLOS ONE, a journal published by the Public Library of Science, please visit: http://www.plosone.org/.

Authors of the paper provide biochemical and electrophysiological data in mouse models of retinal disease to show that emixustat produces a dose-dependent, reversible suppression of both visual chromophore biosynthesis and rod photoreceptor activity. In these models, emixustat treatment was shown to protect photoreceptors from light-mediated damage, reduce levels of toxic retinal fluorophores, such as A2E, and preserve the retinal vasculature during periods of hypoxia. The authors note that these findings could be relevant for the development of potential treatments for certain degenerative retinal diseases.

Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela Inc., said, “I am gratified that our pre-clinical data are now published in the journal PLOS ONE. We are fully committed to visual cycle modulation as an important part of our research.”

Emixustat has been formulated for oral administration and is currently being investigated in a multi-center Phase 2b/3 clinical trial to evaluate safety and efficacy in patients with geographic atrophy (GA) associated with dry age-related macular degeneration (AMD) (Clinicaltrials.gov identifier: NCT01802866).

About Acucela Inc.

Acucela Inc. (www.acucela.com or www.acucela.jp) is a clinical-stage biotechnology company that specializes in discovering and developing novel drug candidates to potentially treat and slow the progression of sight-threatening ophthalmic diseases, which could impact millions of individuals worldwide. Acucela currently has the following investigational candidates in development in collaboration with Otsuka Pharmaceutical Co., Ltd.: emixustat hydrochloride for geographic atrophy associated with dry age-related macular degeneration based on Acucela’s proprietary visual cycle modulation technology; and OPA-6566 for ocular hypertension and glaucoma.

Cautionary Statements

Certain statements contained in this press release are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “will” as well as similar expressions are intended to identify forward-looking statements. These forward-looking statements include Acucela’s expectations regarding corporate development activities and the ultimate success of the enterprise. These statements involve risks and uncertainties that could cause the Company’s actual results to differ materially from those projected in forward-looking statements, including, but not limited to the risk that our investigational product candidates will not demonstrate the expected benefits, the success of our investigational product candidates depends heavily on the willingness of our collaboration partner to continue to co-develop our investigational product candidates, the risk of delays in our expected clinical trials, the risk that new developments in the intensely competitive ophthalmic pharmaceutical market require changes in our clinical trial plans or limit the potential benefits of our investigational product candidates, and other risks and uncertainties inherent in the process of discovering and developing therapeutics that demonstrate safety and efficacy. For a detailed discussion of these and other risk factors, please refer to the Company’s filings with the Securities and Exchange Commission, which are available on the Company’s investor relations Web site (http://ir.acucela.com/) and on the SEC’s Web site (http://www.sec.gov).