- Investors

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]