fye2015 analyst meeting acucela inc. · 3/29/2016 · company that specializes in identifying and...

Acucela 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.

FYE2015 Analyst Meeting

Acucela Inc.

Tokyo, Japan

March 29, 2016

DISCLAIMER AND ADDITIONAL INFORMATION

Certain statements contained in this presentation and made in connection therewith are forward-looking statements within the meaning of

Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation

Reform Act of 1995. Any such statements that are not statements of historical fact may be deemed to be forward-looking statements. These

forward-looking statements include statements regarding the ability of the Company to consummate the inversion transaction (the

“Inversion”); the ability of Acucela Japan KK to obtain approval for listing on the Mothers market of the Tokyo Stock Exchange; the ability to

realize the expected benefits of the Inversion; the expected costs relating to the Inversion, which could be greater than expected; the ability

of the Company and Acucela Japan KK to meet the conditions to closing of the Inversion; interpretations of tax law, tax treaties or tax

regulations or enforcements thereof; expectations regarding corporate development activities and the ultimate success of the enterprise;

the Company’s development plans and ability to successfully commercialize its product candidates; the timing of and results from the

Company’s and its collaborators’ ongoing clinical trials and pre-clinical development activities; the potential efficacy, future development

plans and commercial potential of the Company’s and its collaborators’ product candidates and the progress and potential of ongoing

development programs.

These statements are based on current assumptions that involve risks, uncertainties and other factors that could cause the actual results,

events or developments to differ materially from those expressed or implied by such forward-looking statements. These risks and

uncertainties, many of which are beyond our control, include, but are not limited to: the Company may be unable to obtain the shareholder

approval required for the Inversion; the Company may abandon the Inversion; conditions to the closing of the Inversion may not be

satisfied; problems may arise in connection with the relocation of the Company’s headquarters to Japan, which may result in less effective

or efficient operations; the Inversion may involve unexpected costs, unexpected liabilities or unexpected delays; the Company’s business

may suffer as a result of uncertainty surrounding the Inversion; the Company may not realize the anticipated benefits of the Inversion; the

Inversion may negatively impact the Company's relationships, including with employees, suppliers, collaborators, competitors and

investors; the Inversion may result in negative publicity affecting the Company’s business and the price of the Company’s common stock;

the Inversion may have tax consequences for holders of the Company’s common stock; the Company may be adversely affected by other

economic, business, and/or competitive factors; the Company’s investigational product candidates may not demonstrate the expected safety

and efficacy; the Company’s pre-clinical development efforts may not yield additional product candidates; any of the Company’s or its

collaborators' product candidates may fail in development, may not receive required regulatory approvals, or may be delayed to a point

where they are not commercially viable; the success of the Company’s investigational product candidate, emixustat hydrochloride, depends

heavily on the willingness of its collaboration partner to continue to co-develop the investigational product candidate;

2016 March Analyst Meeting 2

the Company’s clinical trials could be delayed; new developments in the intensely competitive ophthalmic pharmaceutical market may

require changes in the Company’s clinical trial plans or limit the potential benefits of its investigational product candidates; the impact of

expanded product development and clinical activities on operating expenses; adverse conditions in the general domestic and global

economic markets; as well as the other risks identified in the Company’s filings with the Securities and Exchange Commission. These

forward-looking statements speak only as of the date hereof and the Company assumes no obligation to update these forward-looking

statements, and readers are cautioned not to place undue reliance on such forward-looking statements. For a detailed discussion of the

foregoing risks 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).

This communication does not constitute an offer to sell or the solicitation of an offer to buy any securities, or a solicitation of any vote or

approval, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to

registration or qualification under the securities laws of any such jurisdiction. The Inversion will be submitted to the shareholders of

Acucela Inc. for their consideration. Acucela Japan KK intends to file a Registration Statement on Form S-4 (the “Form S-4”) with the SEC

that will include a preliminary prospectus of Acucela Japan KK and a preliminary proxy statement of Acucela Inc., and each of Acucela

Japan KK and Acucela Inc. also plan to file other relevant documents with the SEC regarding the Inversion. A definitive proxy

statement/prospectus will be mailed to the shareholders of Acucela Inc. once the Form S-4 has been declared effective by the SEC.

INVESTORS AND SECURITY HOLDERS OF ACUCELA INC. ARE URGED TO READ THE PROXY STATEMENT/PROSPECTUS (INCLUDING ALL

AMENDMENTS AND SUPPLEMENTS THERETO) CAREFULLY AS WELL AS ANY OTHER RELEVANT DOCUMENTS THAT WILL BE FILED WITH

THE SEC WHEN THEY BECOME AVAILABLE, BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT ACUCELA JAPAN KK,

ACUCELA INC. AND THE INVERSION. Investors and security holders may obtain a free copy of the proxy statement/prospectus and other

relevant documents (when available) filed and to be filed with the SEC from the SEC's web site at www.sec.gov or at the Company’s web site

at ir.acucela.com. Investors and security holders may also read and copy any reports, statements and other information filed by Acucela Inc.

or Acucela Japan KK, with the SEC, at the SEC public reference room at 100 F Street, N.E., Washington, D.C. 20549. Please call the SEC at 1-

800-SEC-0330 or visit the SEC’s website for further information on its public reference room. Investors and security holders may also obtain,

without charge, a copy of the proxy statement/prospectus and other relevant documents (when available) by directing a request by mail or

telephone to Investor Relations, Acucela Inc., 1301 Second Avenue, Suite 4200, Seattle, WA 98101, telephone (206) 805-8300.



Acucela Inc. and its directors and executive officers may be deemed to be participants in the solicitation of proxies from the Company’s

shareholders in connection with the Inversion. Information about these persons is set forth in the Company’s Annual Report on Form 10-K

filed by the Company with the SEC on March 11, 2016, and will be included in the Form S-4 and in any documents subsequently filed by its

directors and officers under the Securities Exchange Act of 1934, as amended. These documents can be obtained free of charge from the

sources indicated above. Investors and security holders may obtain additional information regarding the interests of such persons, which

may be different from those of the Company’s shareholders generally, by reading the proxy statement/prospectus and other relevant

documents regarding the Inversion to be filed with the SEC when they become available.



Ryo Kubota, MD, PhD Profile

• Scientific Research and Ophthalmology

− Performed over 1,000 ocular surgeries to treat cataract, retinal detachment,

diabetic retinopathy, glaucoma, strabismus, and other conditions

− Discovered a glaucoma gene, myocilin; earned the Suda Award

− Established Acucela in 2002; research helped advance the theory that toxic

by-products of the visual cycle could be a causative factor in AMD

• Finance and Business

− Raised over $40M in venture capital rounds A, B and C

− Negotiated and signed a co-development and co-commercialization

partnership for visual cycle modulation candidates with Otsuka

Pharmaceutical (Potential milestone payments of $258M total)

− Raised $163M (gross) in a successful IPO in Japan

(completed in February 2014)


Company Overview


An ophthalmology-focused, science-driven company

People and Strategy • Executive leadership with experience in health care

management, life science administration and technology

• Broad-skilled employee base in research, development and

operations

• Strategic plan to develop an innovative portfolio of

ophthalmology products

Partnership • Long-time partnership with Otsuka Pharmaceutical with

potential high-reward alliance

• Acucela has rights for emixustat hydrochloride in Europe,

South and Central America and most of Africa

• Lanosterol technology partnership with YouHealth and

University of California, San Diego

Technology • Unique mechanism of action in visual cycle modulation

(VCM)

• Lead clinical trial program in geographic atrophy (GA)

associated with dry age-related macular degeneration

(AMD); no treatments currently available

• 112 granted patents; 174 pending patents (as of 12/31/15)

Financials • Successful 2014 IPO; $163M (gross) raised

• Cash, short-term and long-term investments for the three

months ended 12/31/15 was $166M ready to invest in

ongoing programs, business development and internal

research and development

Recent Highlights


Date Highlights

May 13 ‘15 Acucela Announces Publication of Pre-clinical Data for Emixustat Hydrochloride (PLOS ONE)

Jun 3 ‘15 Acucela Announces Publication of Phase 2a Clinical Trial Results for Emixustat Hydrochloride (June

edition of RETINA)

Jul 2 ‘15 Acucela Hires Roger Girard as Chief Strategy Officer

Aug 5 ‘15 Acucela Hires Dr. Lukas Scheibler as Executive Vice President of Translational Medicine

Aug 25 ‘15 Acucela Hires Dr. George Lasezkay as Executive Vice President of Legal Affairs

Nov 4‘15 Acucela Validates Sampling Method That Could Pave Way for More Efficient Clinical Tests

Jan 11 ‘16 Acucela to Present at the Biotech Showcase™ 2016 in San Francisco

Mar 17 ‘16 Acucela Secures Option to Exclusively License Novel Cataract Treatment

Mar 29 ‘16

Announcement on Proposed Conversion of Japanese Subsidiary into the Holding Company of

Acucela Inc. through Triangular Merger, Application for Listing of Shares as a Domestic Company,

and Partial Amendment to the Bylaws of Acucela Inc.

Treating Sight-Threatening Diseases

Vision R&D New

Business

Current

Pipeline

• Expanding internal

research capabilities

• Emixustat and OPA-6566

• Optimizing asset value

• Seeking innovative

approaches to treat

sight-threatening

diseases


Alliance &

In-Licensing

Corporate Strategy


Continuous development of the current pipeline and increase in

business development activities to expand the portfolio

Current

Pipeline

Emixustat for GA secondary to dry AMD

− June 2016 disclosure on top-line data results of Phase 2b/3 trial

− Obtained feedback from European regulatory agencies regarding registration pathway

for Emixustat in Europe

New Pipeline

Emixustat for Additional Indications

− Evaluating the potential to develop Emixustat for additional indications including

diabetic retinopathy and Stargardt’s disease

Lanosterol technology

− Plans to initiate development of pharmacological treatment candidate for cataract

Increase in internal research and in-licensing

− Expanding internal research capabilities

− Pursuing partnering and in-licensing opportunities focused on innovative ophthalmic

technologies

Organization

Inversion, to become a Japanese company

− Change in technical status to list as a Japanese entity on the TSE Mothers Board,

allowing an increase in information and investment opportunities for both retail and

institutional investors

− Anticipated inversion in September 2016

FYE2015 Financials

Overview of FY2015: P/L

FY2014 FY2015 FY2014 FY2015 Reasons for change

P/L Statement USD USD JPY JPY

Revenues from collaboration 35,396 24,067 4,269,112 2,902,721 Due to fewer billable activities related to Emixustat.

Expenses

R&D 25,582 22,636 3,085,445 2,730,128

Emixustat 24,509 21,060 2,956,005 2,540,027 Due to fewer clinical activities as the clinical trial reaches the final phase.

In-licensed 23 1 2,794 139 Due to termination of the Rebamipide Agreement and the results of the

Phase 1/2 clinical study evaluating OPA-6566 in 2012.

Internal research 1,050 1,575 126,646 189,962 Due to increased internal research activities related to our VCM

compounds.

General and administrative 10,002 27,987 1,206,341 3,375,512

General and administrative 10,002 14,087 1,206,341 1,699,033

Include accounting and compliance services related of $1.0mn, internal

audit and enterprise risk management system of $1.3mn and additional

costs on the new office lease of $0.8mn.

May 1st Special

Shareholder Meeting and

management change

13,900 1,676,479

Include former CEO and management severance of $10.5mn, legal and

consulting fees of $2.3 and costs associated with hiring of new

management and retaining of employees of $1.1mn.

Income (loss) from operations (188) (26,556) (22,674) (3,202,919)

Net income (2,006) (25,509) (241,942) (3,076,641)

Note: ¥120.61= US$1 as of December 30, 2015


(US$ and JP¥ in thousands)

Overview of FY2015: Cash Flow


Cash flow Statement USD USD JPY JPY

Cash flows from operating activities 9,442 (16,871) 1,138,798 (2,034,811)

Due to a net loss of $25.5 mn, a decrease in deferred revenue of

$3.8 mn and an increase in accounts receivables from

collaborations of $0.9 mn, partially offset by $8.9 mn in stock-based

compensation and a $1.1 mn increase in deferred rent and lease

incentives related to the lease on our new corporate headquarters,

and $2.3 mn of amortization of premiums on marketable securities.

Cash flows from investing activities (152,932) 4,341 (18,445,129) 523,583

Primarily a result of the maturity of marketable securities held as

available for sale being partially offset by the purchase of

marketable securities available for sale.

Cash flows from financing activities 148,274 (1,160) 17,883,328 (139,908) Primarily a result of the repurchase of restricted stock units

withheld for employee taxes.

Cash, cash equivalents and short and

long-term investments - end of period 187,819 166,525 22,652,848 20,084,594




Overview of FY2015: Balance Sheet




Balance sheet statement USD USD JPY JPY

Current Assets 111,714 120,201 13,473,823 14,497,458

Cash, cash equivalents and short-

term investments 103,786 112,010 12,517,628 13,509,540

Non-current Assets 85,252 55,749 10,282,242 6,723,886

Long-term investments 84,033 54,515 10,135,220 6,575,054

Due to increase in costs related to corporate

restructuring and legal and consulting expenses

connected with our May 1, 2015 special shareholders

meeting.

Total Assets 196,966 175,950 23,756,065 21,221,344

Current Liabilities 12,556 8,412 1,514,376 1,014,584 Decrease in deferred revenue from collaborations.

Long-term Liabilities 47 1,104 5,668 133,154 Increase in long-term portion of deferred office rent.

Shareholders‘ Equity 184,363 166,434 22,236,021 20,073,606


3,869 7,033 6,994

(188)

(26,556)

(37,440) (36,940)

(40,000)

(30,000)

(20,000)

(10,000)

-

10,000

FY11 FY12 FY13 FY14 FY15 FY16 Est

Operating Profit (Loss)

Financial Overview


(US$ in thousands)

34,809 41,495 47,024 54,048

196,966 175,950

-

50,000

100,000

150,000

200,000

250,000

FY10 FY11 FY12 FY13 FY14 FY15

Total Assets

14,101 20,840 25,607 31,124

184,363 166,434

-

50,000

100,000

150,000

200,000

FY10 FY11 FY12 FY13 FY14 FY15

Total Shareholder's Equity

34,226

46,424

52,947

35,396

24,067 25,000 27,500

-

10,000

20,000

30,000

40,000

50,000

60,000

FY11 FY12 FY13 FY14 FY15 FY16 Est

Revenues from Collaboration

FY2016 Forecast (as of March 9th 2016)


25,000

(37,440) (36,940)

27,500

(36,940) (35,740)

(50,000)

(40,000)

(30,000)

(20,000)

(10,000)

0

10,000

20,000

30,000

40,000

Revenue fromcollaborations

Low Case High Case

FY 2016 Forecast


• Revenue from collaborations

− Revenue is associated directly with reimbursable costs reflecting

varying level of activities in relation to the completion of Phase 2b/3

clinical trial in the United States and moving forward with Phase 3

clinical trial for Emixustat.

• Loss from operations

− Operational costs are associated with reimbursable R&D costs as well

as non-reimbursable internal R&D and general and administrative

costs.

− Range is due to R&D costs directly associated with the Phase 3

clinical costs.

− Non-reimbursable costs include internal R&D cost (USD $22.2 mn),

general and administrative cost (USD $17.8 mn). Internal R&D cost

include business and development cost (USD $15.0 mn) which

involves in-licensing costs and internal research and development

(USD $3.5 mn) including Diabetic Retinopathy, Stargardt's and other

pre-clinical studies.

(US$ in thousands)

Loss from operations Net loss

Emixustat Hydrochloride

Emixustat

Hydrochloride

Emixustat Overview:

Lead Investigational Product Candidate


DISCLAIMER Emixustat Hydrochloride Overview: Lead Investigational Product Candidate

• Oral administration

• Small molecule

• Hypothesized to reduce toxins in the

retina

• Targets RPE65–

a key rate-limiting enzyme

• Measureable effect in the retina

Mechanism of Action:

Visual Cycle Modulation Technology

Visual Cycle Modulation is the use of non-retinoid, small molecule compounds which target

specific proteins of the visual cycle. The intended effect of VCM compounds is to reduce retinal

toxins and preserve the integrity of retinal tissue. Acucela has established a leadership position

in this area as a result of our pioneering efforts to research, discover and develop proprietary

VCM compounds.

ACU-4429 Emixustat


Retinal Pigment Epithelium

11-cis-retinal

11-cis-retinol

all-trans-retinyl ester

all-trans-retinol

Rod Photoreceptor

11-cis-retinal + Opsin

(Rhodopsin)

RPE65

all-trans-retinal

Precursors

of vitamin A

toxins

all-trans-retinol

Emixustat

Formation of Vitamin A Toxins in the Visual Cycle

19 2016 March Analyst Meeting

• In the mammalian visual cycle, the Retinal Pigment Epithelium protein 65 (RPE65) plays a crucial role in the

conversion of dietary vitamin A to visual chromophore, 11-cis-retinal.

• The visual chromophore combines with opsin in rod photoreceptors to form rhodopsin.

• Light activation of rhodopsin triggers an electro-chemical cascade which results in visual sensation.

• 11-cis-retinal and the product of light-activated rhodopsin (all-trans-retinal) have the potential to spontaneously

react with membrane lipids and form vitamin A toxins, such as A2E.

19

-0.05

0

0.05

0.1

0.15

0.2

0.25

Change in GA Lesion Area at Day 90 in Phase 2a

20 2016 March Analyst Meeting

Mea

n c

han

ge

in le

sio

n a

rea

fro

m b

asel

ine

to

mo

nth

3 (

mm

2 )

Pooled Emixustat Placebo

N = 23 N = 8

Numerical

difference of

0.23 mm2,

p > 0.05

Pooled data from all Emixustat treated patients vs. placebo

for change in lesion area from baseline to month 3 (FAF)

Full Analysis Population – Acucela data on file

Source: Dugel P, Novack R, Csaky K, Richmond P, Birch D, Kubota R. Phase II, randomized, placebo-controlled, 90-day study of Emixustat hydrochloride in geographic atrophy associated with dry age-related macular

degeneration. Retina. June 2015; 35(6): 1173–1183

Phase 2b/3 “SEATTLE” Study

• Design

− Two year, randomized, double-masked, dose-ranging study comparing safety and efficacy of

Emixustat with placebo in patients with GA associated with dry AMD

− A total of 508 patients with GA associated with dry AMD were enrolled

• Objectives

− Primary: Determine if Emixustat reduces lesion growth rate compared to placebo

− Secondary:

– Evaluate safety and tolerability

– Assess changes in best-corrected visual acuity

– Evaluate the effect on development of wet AMD

− Top-line 2-year trial results anticipated in June 2016


Drug Development Path Emixustat for GA associated with dry AMD:

Investigational compound based on Acucela’s proprietary VCM

2005 2007 2009 2012 2013

» Phase 2b/3 clinical trial commences

» Two-year treatment

» Toxicology

» Proof-of-concept in

preclinical model

» Medicinal chemistry

» Five phase 1 clinical trials completed

» One phase 2a clinical trial completed

(GA subjects)

» 179 total subjects exposed to Emixustat

» Fast track designation granted

» IND filed

» First human trial

2008

22

2014

» Phase 2b/3 clinical

trial enrollment

completed in 508

subjects

2016

» Phase 2b/3 clinical

trial top line results

expected


Lanosterol

A Pharmacological Treatment Candidate for Cataract

Protein Aggregation in Crystalline Lens

of the Eye Leads to Cataract

Source: Zhao L, Chen XJ, Zhu J, et al. Nature. 2015 Jul 30;523(7562):607-11.

Clear Vision

Highly ordered proteins

Impact on Vision

Cloudy Vision

Denatured proteins

Low protein order /

protein aggregation

Denatured proteins aggregate and obstruct light, causing cloudy vision.


0

10

20

30

40

50

60

70

80

40-49 50-54 55-59 60-64 65-69 70-74 75-79 80+

Prevalence of Cataracts by Age Group in 2015

Cataract Prevalence Increases with Age

By age 80, 70% of

people will have a

cataract.

Per

cen

t

Age Group

903 million people worldwide were affected by cataracts in 2015,

a number which is expected to grow to 1 billion people by 2020

Sources: Prajna NV, Ravilla TD, Srinivasan S. Cataract Surgery. In: Debas HT, Donkor P, Gawande A, Jamison DT, Kruk ME, Mock CN, editors. Essential Surgery: Disease Control Priorities, Third Edition (Volume 1). Washington (DC): The International Bank for Reconstruction and Development / The World Bank; April 2, 2015. Chapter 11.; Market Scope, Global IOL Market 2015.tar Source: https://nei.nih.gov/health/cataract/cataract_facts; : https://nei.nih.gov/health/cataract/cataract_facts


https://nei.nih.gov/health/cataract/cataract_facts

https://nei.nih.gov/health/cataract/cataract_facts

Cataract Surgery Rates Are Projected to Increase

Sources: Market Scope, Global IOL Market 2015; Market Scope, The Global Cataract Surgical Equipment Market, 2015 Abbreviation: CAGR, compound annual growth rate.

4.3M

4.3M

Global Forecast for Cataract Surgery

Num

ber

of S

urge

ries

(mill

ions

)

Year

Japan

Europe

US

Global 3.5%

3.1%

2.0%

2.0%

28.3M

1.6M

CAGR

Global IOL (intraocular lens) market were $3.3 billion in 2015 and estimated to

grow to $4.4 billion by 2020.

Global surgical equipment market were $1.4 billion on 2014 and estimated to

grow to $2.2 billion in 2019.


Cataract Surgery is Invasive and Refractive

Correction Necessary with Many Patients

Multiple incisions are made to

access the lens

High-frequency ultrasound

breaks up the lens into small

pieces, then removed with

suction (phacoemulsification)

A clear intraocular lens is

inserted in the same location

the natural lens occupied

The incisions are closed and

a protective shield is placed

over the eye (in some

procedures)

Source: Prajna NV, Ravilla TD, Srinivasan S. Cataract Surgery. In: Essential Surgery: Disease Control Priorities, Third Edition (Volume 1). Washington (DC): The International Bank for Reconstruction and Development / The World Bank; April 2, 2015. Chapter 11. http://www.allaboutvision.com/conditions/cataract-surgery.htm

Procedure leaves about 40% of patients with need for refractive correction.


Surgery is Not Indicated for Mild Cataracts

Normal Mild Moderate Severe

Currently, no proven treatment to reverse or slow the progression

of cataract

Lens Opacities Classification

System III

Grade 1 2 3 4 5 6

In Japan, Pirenoxine is indicated for the prevention of cataract, but not for treatment.

Sources: Prajna NV, Ravilla TD, Srinivasan S. Cataract Surgery. In: Essential Surgery: Disease Control Priorities, Third Edition (Volume 1). Washington (DC): The International Bank for Reconstruction and Development / The World Bank; 2015 Apr 02. Chapter 11; Optometric Clinical Practice Guideline – Care of the Adult Patient with Cataract. AMERICAN OPTOMETRIC ASSOCIATION, 1995 243 N. Lindbergh Blvd., St. Louis, MO 63141-7881; Chylack LT Jr, Wolfe JK, Singer DM, et al. The Lens Opacities Classification System III. The Longitudinal Study of Cataract Study Group. Arch Ophthalmol. 1993 Jun;111(6):831-6.

For moderate to severe cases of cataracts, surgery is the ONLY option.


Lanosterol

A New Treatment Paradigm for Cataracts

• Non-invasive, pharmacological treatment

• Naturally occurring compound in the human body

Lanosterol :

• Dissolves protein aggregates and establishes order

• Has the potential to reverse lens opacification

The graphic portrayal of the treatment candidate Lanosterol is used for illustrative purposes only, and, does not constitute an actual product nor is intended to suggest a particular form of therapy delivery mechanism.


Pre-Clinical: Reduction of Cataract Severity

RE

DU

CE

D C

AT

AR

AC

T S

EV

ER

ITY

LANOSTEROL

reverses cataracts in

rabbit lens

Before

treatment

After treatment

Cat

arac

t Gra

de

Before After

P=.003 3 – 2 – 1 – 0 -

*

*

LANOSTEROL

reverses cataracts in

dogs

Cat

arac

t Gra

de

Before After

P=.009 3 – 2 – 1 – 0 -

*

*

*

*

Before treatment After treatment

Ex-

vivo

In

viv

o

Source: Zhao L, Chen XJ, Zhu J, et al. Nature. 2015 Jul 30;523(7562):607-11.

Lanosterol “dissolves” protein aggregates, establishes protein order,

and reduces opacity.


Lanosterol May Provide

the Missing Treatment Option for Cataract

The graphic portrayal of the treatment candidate Lanosterol is used for illustrative purposes only, and, does not constitute an actual product nor is intended to suggest a particular form of therapy delivery mechanism.

Sources: Prajna NV, Ravilla TD, Srinivasan S. Cataract Surgery. In: Essential Surgery: Disease Control Priorities, Third Edition (Volume 1). Washington (DC): The International Bank for Reconstruction and Development / The World Bank; 2015 Apr 02. Chapter 11; Optometric Clinical Practice Guideline – Care of the Adult Patient with Cataract. AMERICAN OPTOMETRIC ASSOCIATION, 1995 243 N. Lindbergh Blvd., St. Louis, MO 63141-7881; Chylack LT Jr, Wolfe JK, Singer DM, et al. The Lens Opacities Classification System III. The Longitudinal Study of Cataract Study Group. Arch Ophthalmol. 1993 Jun;111(6):831-6.

Lens Opacities Classification

System III

Grade 1 2 3 4 5 6

Normal Mild Moderate Severe

Lanosterol may reverse lens opacification with moderate to severe cases of cataract.


Lanosterol Development Plan

Preclinical development

to initiate in 2016

• Formulation development

• Toxicology study

Proof of Concept / Phase 1

Study to initiate in 2017

• Assess lens opacity in humans


Inversion

Potential Benefits of the Inversion

• Potential benefits that would increase shareholder:

− Increased availability, quantity and prominence of information about the Holding Company for Japanese

investors, which can be made available in the Kaisha Shikiho and Nikkei Kaisha Joho, two publications

frequently used by Japanese investors to find information on listed companies in Japan. This increased

access to information may enhance the understanding of our business and provide more effective

communication to our Japanese investors;

− As a Japanese entity, the Holding Company will be eligible for future consideration to be included in the

Mothers Index of the TSE;

− Institutional investors with a focus on TSE-listed companies, who by mandate or other internal fund

restrictions have not been able to invest in non-Japanese domiciled equities, will have the opportunity to

invest; and

− Increased analyst research coverage, if investor demand for listed securities of the Holding Company

increases following the Triangular Merger.

• Increase its visibility and business presence in Japan:

− Allow for opportunities such as conducting internal research and establishing partnerships for R&D and

drug development through collaborations with Japanese pharmaceutical companies and academic

institutions.


Anticipated Schedule and Proposed Scheme

2016 Events Related to the Inversion

March Board resolution

April

May

June Anticipated execution on the triangle merger

July

Aug Anticipated date for the annual meeting of shareholders

Sep

• Anticipated date of delisting (Acucela Inc.)

• Anticipated effective date of the Triangular Merger

• Anticipated date of listing of the Holding Company

Company’s

Shareholders

Acucela Inc.

Holding Company

U.S. Subsidiary

Japan U.S.

1: Current Status

Company’s

Shareholders

Holding Company

U.S. Subsidiary

Japan U.S.

3: After the Triangular Merger


Company’s

Shareholders

Acucela Inc.

Holding Company

U.S. Subsidiary

Japan U.S.

2: Triangular Merger

• The Company is the dissolving

company, and the U.S. Subsidiary

is the surviving company.

• As a result, the Company’s

shareholders acquire shares of the

Holding Company in exchange for

the Company’s shares.

Distribution of

Shares of the

Holding Company

Triangular

Merger

Management Team


An experienced management team driving current and future

pipeline development, including device, pharma & biotech assets

George Lasezkay, PharmD, JD

EVP General Counsel

Allergan, Inc.

RetroSense Therapeutics,

Amakem Therapeutics NV,

Novagali Pharma, SA

Neurotech Pharmaceuticals, Inc.

Acuity Pharmaceuticals,

ISTA Pharmaceuticals, Inc.

Lukas Scheibler, PhD

EVP R&D

Alcon

Novartis Pharmaceuticals John Gebhart

Chief Financial Officer Qliance Medical Management Consultant to Remote

Medical International, Ventripoint, and others

Ted Danse, MBA

Chief Business Officer

Neurotech Pharmaceuticals, Inc.

ISTA Pharmaceuticals Inc., Allergan,

Inc., Coopervision, Bausch & Lomb ,

Schering-Plough

Roger Girard

Chief Strategy Officer

Xecutive Advisory Partners LLC

Terra Vista Software

CareCap, Bellevue,

Neuro-ID, IsoRay Medical

Ryo Kubota, MD, PhD

Chairman, President and CEO

Appendix

Board of Directors

Directors Background

Ryo Kubota, MD PhD Chairman, President and Chief Executive Officer and also founder of Acucela Inc.

Shintaro Asako Chief Executive Officer - DeNA West

Previously, Chief Financial Officer - MediciNova, Inc.

Shiro Mita, PhD President and Chief Executive Officer - M’s Science Corporation

Previously: Executive Director of Drug Discovery, Director - Santen Pharmaceuticals Co., Ltd

Eisaku Nakamura

Director - Koinobori Associates Inc.

Previously: Director and General Manager - Bio Sight Capital Co., Ltd , Chief Executive Officer and

President - Berevno Corporation, Board of Directors - CanBas Corporation and Activus Pharma Co. Ltd.

Robert Takeuchi

President - RT Consulting, Inc.

Previously: President - SOFTBANK Finance, America Corporation, Director of International Equity Sales -

Credit Suisse First Boston, Board of Directors SBI Investment Co., Ltd. and Quark Pharmaceuticals, Inc.


Partnership with Otsuka Pharmaceutical


Investigational Product

Candidate Potential Indication License Territory Financial Terms

Emixustat hydrochloride

(developed by Acucela)

Dry AMD and other

ophthalmic indications

Joint (50/50) - North America

Acucela - Europe, South and

Central America and most of

Africa

Otsuka – Asia-Pacific, some

countries in Africa/Middle East

• Otsuka paid $5M cash upfront payment to

Acucela

• Potential milestone payments: $258M total

• Currently Otsuka and Acucela are equally

sharing all development expenses; Otsuka

loans funds to Acucela for the payment of

Acucela’s share of the development expenses

through to product launch

OPA-6566

(developed by Otsuka)

Glaucoma and other

ophthalmic indications

United States • Currently evaluating next steps for the

program

Unchanged stable relationship with Otsuka Pharmaceutical

Acucela’s Intellectual Property Position

Update in VCM and Emixustat

(Dec 31, 2015)

Emixustat related patents VCM related

patents

Other

patents

Total

patents

Region US Non-US US Non-US

Granted patents 3 15 17 50 27 112

Pending patents 2 52 13 106 1 174

Expiry year 2029 2028-2033 2028-2034 2028-2034


Acucela intends to aggressively protect and defend its intellectual

property position in Emixustat and VCM

• Strong patent portfolio is critical to our success

• Patents cover compositions of matter and methods of using the compositions

Acucela 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.

fye2015 analyst meeting acucela inc. · 3/29/2016 · company that specializes in identifying and...

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