2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo...

96
2013 ANNUAL REPORT TO STOCKHOLDERS www.acucela.com

Transcript of 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo...

Page 1: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

2013

ANNUAL REPORT TO STOCKHOLDERS

www.acucela.com

Page 2: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh
Page 3: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Dear Shareholders,

Acucela has a clear and precise corporate definition - we are a clinical-stage biotechnology company that specializes in discovering and developing novel therapeutics to treat and slow the progression of sight-threatening ophthalmic diseases impacting millions of individuals worldwide.

Acucela’s mission is:

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

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:

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 under development;

Robust research and development infrastructure: Demonstrated capabilities in ophthalmic research and clinical development; and

Long-standing strategic partnership: Partnered multiple programs with Otsuka Pharmaceutical, a global diversified healthcare company based in Japan.

The Pursuit of Innovation – A Pioneer in Visual Cycle Modulation (VCM) At the heart of our innovative culture is the development and advancement of our proprietary visual cycle modulation (VCM) compounds. The visual cycle is the biological conversion of a photon into an electrical signal in the retina. During exposure to bright light, the visual cycle is extremely active and can produce toxic by-products which cannot be eliminated. Over time, accumulation of these toxic by-products can compromise function of the visual cycle and damage the retina.

Acucela’s VCM compounds are designed to reduce the effects of toxic by-products and oxidative damage as well as protect the retina from light damage. Based on preclinical data, we believe that our VCM-based drug candidates have the potential to demonstrate significant advantages over other therapies.

An Unmet Need in Dry Age-related Macular Degeneration (dry AMD) Age-related macular degeneration is associated with irreversible vision loss in severe cases. More than 10 million people in the United States and more than 120 million people worldwide are affected by AMD.1 Geographic atrophy (GA) is an advanced stage of dry AMD which can lead to legal blindness. There is currently no therapy approved by the Food and Drug Administration to treat dry AMD.

Page 4: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Emixustat hydrochloride is the first internally developed compound by Acucela and our lead VCM product candidate in development to treat GA associated with dry AMD. Emixustat is orally dosed, and has been demonstrated to be generally safe and well-tolerated in completed clinical studies. Emixustat is currently being studied in a Phase 2b/3 clinical trial for the treatment of GA associated with dry AMD. We look forward to continuing to advance this clinical trial in 2014.

2013/early 2014 Company Highlights

We were pleased to announce in 2013/early 2014 that we: - Completed enrollment in the emixustat Phase 2b/3 clinical trial (March 2014); - Appointed Brian O’Callaghan to our Board of Directors (September 2013); - Reached 50% enrollment in the emixustat Phase 2b/3 clinical trial (August 2013);- Announced with our partner, Otsuka Pharmaceutical, the presentation of Phase 2a clinical

results of emixustat in patients with GA associated with dry AMD at the ARVO 2013 Annual Meeting (May 2013);

- Initiated a Phase 2b/3 clinical trial of emixustat in subjects with GA associated with dry AMD (April 2013); and

- Appointed Hans Peter Pfleger, PhD as Vice President Commercial Operations & Strategic Marketing (January 2013).

And, of course, we completed our initial public offering (IPO) in February 2014, raising a net of just over $140M and becoming the first US company to solely list on the Mothers Board of the Tokyo Stock Exchange (ticker symbol: 4589).

Thank you for your continued support as we strive to advance Acucela to its next stage as a company.

Sincerely,

Ryo Kubota, MD, PhD Chairman, President & CEO Acucela Inc.

(1) 2012 Comprehensive Report on The Global Retinal Pharmaceuticals & Biologics Market, Market Scope.

Page 5: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

UNITED STATES SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 10-K

Commission File Number 000-55133

ACUCELA INC. (Exact name of registrant as specified in its charter)

Washington 02-0592619

(State or other jurisdiction of incorporation or organization)

(I.R.S. Employer Identification Number)

1301 SECOND AVENUE, SUITE 1900 SEATTLE, WASHINGTON

98101

(Address of principal executive offices) (Zip Code)

(206) 805-8300

(Registrant's telephone number, including area code)

Securities registered pursuant to Section 12(b) of the Act: None

Securities registered pursuant to Section 12(g) of the Act:

Common Stock, No Par Value Per Share

Page 6: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ACUCELA INC. FORM 10-K

For the fiscal year ended December 31, 2013 INDEX

PART I

PART II

PART III

PART IV

Page 7: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

FORWARD-LOOKING STATEMENTS

ITEM 1. BUSINESS

Overview

Page 8: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Product Overview

Overview

Emixustat for Age-related Macular Degeneration

Disease Overview.

Product Candidate.

Development Status

Phase 2b/3 Study

Page 9: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Other Potential Markets for Our VCM-Based and Retinal Product Candidates

Diabetic Retinopathy / Diabetic Macular Edema.

Disease Overview.

Product Candidate.

Development Status.

Other Ophthalmic Diseases.

Stargardt Disease

Retinopathy of Prematurity

Retinitis Pigmentosa

Page 10: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

OPA-6566 for Glaucoma

Disease Overview.

Product Candidate.

Development Status.

Collaborations with Otsuka

Emixustat hydrochloride

Page 11: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh
Page 12: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

OPA-6566

Page 13: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Intellectual Property

Visual Cycle Modulation

OPA-6566

Page 14: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Other Technologies

Competition

Dry AMD

Page 15: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Glaucoma

Sales and Marketing

Financial Information about Segments and Geographic Areas

Research and Development

Manufacturing and Supply

Government and Other Regulation

General Overview

Page 16: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

United States

Page 17: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Phase 1:

Phase 2:

Phase 3:

Phase 4:

Page 18: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Post-Approval Requirements

Page 19: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Fast Track Designation

Other Regulatory Considerations

Patent Term Restoration and Marketing Exclusivity

Page 20: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Third-Party Payor Coverage and Reimbursement

Page 21: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

The Physician Payment Sunshine Act

Foreign Regulations

Employees

Page 22: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ITEM 1A. RISK FACTORS

Risks Related to Our Business and Industry

We do not have any products that are approved for commercial sale.

Revenues from research and development activities in collaboration with Otsuka and Otsuka’s funding of our portion of development costs under the Emixustat Agreement represented all of our revenues during the three years ended December 31, 2013, and the loss of these revenues would adversely affect our business.

Business—Collaborations with Otsuka

Our long-term prospects are dependent on our product candidates and we cannot be certain that they will achieve success in clinical trials, regulatory approval or be successfully commercialized.

Page 23: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Even if clinical trials are successful in the future, our growth prospects could be adversely affected and we may need to curtail or cease operations if we do not pursue the optimal commercialization strategy for us under our collaboration agreements.

Page 24: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

The pharmaceutical market is intensely competitive. Even if we are successful in obtaining approval of any of our product candidates, we may be unable to compete effectively with existing drugs, new treatment methods and new technologies.

Business—Competition

Market acceptance of emixustat and other products we develop in the future may be limited.

Our historical results of operations may not be indicative of our future profitability or growth, and we may not be able to continue to maintain or increase our profitability or growth.

Page 25: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Management’s Discussion and Analysis of Financial Condition and Results of Operations

Our operating results may fluctuate in the future, which could cause our stock price to decline.

Our planned investment in building a sales and marketing capability will require significant resources and may not generate the return we anticipate.

We may not be successful in our efforts to expand our portfolio of product candidates.

Page 26: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Relying on third-party manufacturers may result in delays in our clinical trials and product introductions.

We are dependent on our management team, particularly Ryo Kubota, M.D., Ph.D., our founder and chief executive officer, and if we are unable to retain and motivate our key management and scientific staff, our drug development programs may be delayed and we may be unable to successfully develop or commercialize our product candidates.

Page 27: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Our workforce reduction may impact the performance of our continuing personnel, and make it more difficult to retain the services of key personnel.

If any products we develop become subject to third-party reimbursement practices, unfavorable pricing regulations or healthcare reform initiatives, our business could be harmed.

Page 28: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

In order to achieve our commercialization goals, we will need to grow the size of our organization, and we may experience difficulties in managing this growth.

We face the risk of product liability claims and may not be able to obtain insurance, and we may have exposure to significant contingent liabilities.

We may need additional financing, which may be difficult to obtain. Our failure to obtain necessary financing or doing so on unattractive terms could adversely affect our development programs and other operations.

We are an “emerging growth company,” and any decision on our part to comply only with certain reduced disclosure requirements applicable to emerging growth companies could make our common stock less attractive to investors.

Page 29: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Regulatory Risks

We may not be able to obtain regulatory approval for any of the products resulting from our development efforts, including emixustat and OPA-6566. Failure to obtain these approvals could materially harm our business.

Page 30: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

The “fast track” designation for development of emixustat for dry macular degeneration (geographic atrophy) may not actually lead to a faster regulatory review or approval process.

Our products could be subject to restrictions or withdrawal from the market and we may be subject to penalties if we fail to comply with regulatory requirements, or if we experience problems with our products, when and if any of them are approved.

Failure to obtain regulatory approval in foreign jurisdictions would prevent us from marketing our products outside of the United States.

Risks Relating to Intellectual Property and Other Legal Matters

If our efforts to protect the proprietary nature of the intellectual property related to our products are not adequate, we may not be able to compete effectively in our markets.

Page 31: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Business—Intellectual Property

If we are unable to protect the confidentiality of our proprietary information and know-how, the value of our technology and products could be adversely affected.

Third-party claims of intellectual property infringement may prevent or delay our discovery, development and commercialization efforts with respect to emixustat and our other product candidates.

Page 32: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

We may become involved in lawsuits to protect or enforce our patents or the patents of our licensors, which could be expensive, time consuming and unsuccessful.

If we use hazardous or biological materials in a manner that causes injury or violates applicable law, we may be liable for damages.

Risks Related to Ownership of Our Common Stock

The price of our common stock may be volatile, and you could lose all or part of your investment.

Page 33: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

The price of our stock could decrease as a result of shares being sold in the market.

As a public company, we will be subject to additional financial and other reporting and corporate governance requirements that may be difficult for us to satisfy, will raise our costs and may divert resources and management attention from operating our business.

Any future issuance of equity or debt securities by us may adversely affect the rights or value of our previously issued common stock.

Page 34: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Because the principal trading market for our shares is the Mother’s market of the Tokyo Stock Exchange, the corporate governance rules of the major U.S. stock exchanges will not apply to us. As a result, our governance practices may differ from those of a company listed on such U.S. exchanges.

A limited number of shareholders will have the ability to influence the outcome of director elections and other matters requiring shareholder approval.

Anti-takeover provisions under Washington law could make an acquisition of us more difficult and affect the market price of our common stock.

We do not expect to pay dividends in the foreseeable future. As a result, you must rely on stock appreciation for any return on your investment.

Page 35: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ITEM 1B. UNRESOLVED STAFF COMMENTS

ITEM 2. PROPERTIES

ITEM 3. LEGAL PROCEEDINGS

ITEM 4. MINE SAFETY DISCLOSURES

PART II

ITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES

Market Information

Holders of Common Stock

Dividends

Recent Sales of Unregistered Securities

Page 36: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Use of Proceeds

Share Repurchases

Page 37: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ITEM 6. SELECTED FINANCIAL DATA

Years Ended December 31,

Statements of Income Data: 2013 2012 2011 (in thousands, except per share data)

As of December 31,

Balance Sheet Data: 2013 2012 (in thousands)

Page 38: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ITEM 7. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS This Report, including the “Management's Discussion and Analysis of Financial Condition and Results of Operations,” contains forward-looking statements regarding future events and our future results that are based on our current expectations, estimates, forecasts, and projections about our business, our results of operations, the industry in which we operate and the beliefs and assumptions of our management. Words such as “expects,” “anticipates,” “targets,” “goals,” “projects,” “would,” “could,” “intends,” “plans,” “believes,” “seeks” and “estimates,” variations of these words, and similar expressions are intended to identify those forward-looking statements. These forward-looking statements are only predictions and are subject to risks, uncertainties and assumptions that are difficult to predict. Therefore, actual results may differ materially and adversely from those expressed in any forward-looking statements. Factors that might cause or contribute to such differences include, but are not limited to, those discussed in this Report under the section entitled “Risk Factors” in Item 1A of Part I and elsewhere herein, and in other reports we file with the SEC. While forward-looking statements are based on reasonable expectations of our management at the time that they are made, you should not rely on them. We undertake no obligation to revise or update publicly any forward-looking statements for any reason, whether as a result of new information, future events or otherwise, except as may be required by law.

Overview

Page 39: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Critical Accounting Policies and Estimates

Revenue Recognition

Multiple Element Arrangements

Page 40: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Substantive Milestone Payments

Deferred Revenue

Page 41: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Funded Development

Research and Development and Clinical Trial Accounting

Income Taxes

Tax Loss Carryforwards

Uncertain Tax Positions

Page 42: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Stock-Based Compensation

Volatility.

Expected term.

Risk-free interest rate.

Dividend yield.

Fair value of underlying stock.

Valuation of Privately-Held-Company Equity Securities Issued as Compensation

Description of Operating Accounts

Revenue

Research and development

Page 43: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

General and administrative

Interest income

Interest expense

Other income (expense)

Income tax benefit (expense)

Results of Operations

Year Ended December 31, 2012 to 2013 % Change

Year Ended December 31, 2011 to 2012 % Change 2013 2012 2012 2011

Year ended December 31, 2013 compared to the year ended December 31, 2012

Revenues from collaborations with related party

Page 44: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Years Ended December 31, 2012 to 2013 $ Change

2012 to 2013 % Change 2013 2012

Proprietary

In-Licensed

Expenses.

Research and development.

Years Ended December 31, 2012 to 2013 $ Change

2012 to 2013 % Change 2013 2012

Page 45: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Proprietary

In-Licensed

Internal Research

General and administrative

Income tax expense

Year ended December 31, 2012 compared to the year ended December 31, 2011

Revenues from collaborations with related party.

Year Ended December 31, 2011 to 2012 $ Change

2011 to 2012 % Change 2012 2011

Page 46: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Proprietary

In-Licensed

VCM Research

Expenses.

Research and development.

Years Ended December 31, 2011 to 2012 $ Change

2011 to 2012 % Change 2012 2011

Proprietary

In-Licensed

Internal Research

Page 47: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

General and administrative.

Income tax benefit (expense)

Liquidity and Capital Resources

Years Ended December 31, 2013 2012 2011

Page 48: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Cash Flows From Operating Activities

Cash Flows From Investing Activities

Cash Flows From Financing Activities

Contractual Obligations and Commitments

Payments Due by Period

Total Less than

1 Year 1-3 Years 3-5 Years More than

5 Years

Co-Development and Co-Promotion Options

Strategic Restructuring

Page 49: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Contingently Repayable Advances

Off-Balance Sheet Transactions

JOBS Act

Page 50: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ITEM 7A. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK

Interest Rate and Credit Quality Risk

ITEM 8. FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA

ITEM 9. CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE

ITEM 9A. CONTROLS AND PROCEDURES

Evaluation of Disclosure Controls and Procedures

Management’s Report on Internal Control over Financial Reporting

Changes in Internal Control Over Financial Reporting

Page 51: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ITEM 9B. OTHER INFORMATION

ITEM 10. DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE

Executive Officers of the Registrant

Name Age Position(s)

Ryo Kubota, M.D., Ph.D.

David L. Lowrance

Hien DeYoung

Directors

Name Age

Page 52: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Executive Officers of the Registrant

Peter A. Kresel

Glen Y. Sato

Michael T. Schutzler

Brian O’Callaghan

Section 16(a) Beneficial Ownership Reporting Compliance

Page 53: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Code of Business Conduct and Ethics

Identification of Audit Committee and Financial Expert

ITEM 11. EXECUTIVE COMPENSATION

Summary Compensation

Summary Compensation Table

Name and principal position Year Salary Bonus Stock

Awards(1) Option Awards

Non-Equity Incentive

Plan Compen- sation(2)

All Other Compen-

sation Total(3)

Page 54: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

2013 Stock Awards

—Executive Employment Arrangements—Ryo Kubota, M.D., Ph.D

Non-Equity Incentive Plan Compensation

Named Executive Officer

Corporate Goals and Objectives

Individual Goals and Objectives

Page 55: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Named Executive Officer Actual Award Amount

Outstanding equity awards at December 31, 2013

Option awards Stock awards

Number of Securities Underlying

Unexercised Options Option Exercise Price(1)

Option Expiration

Date

Number of Shares of Stock

that Have Not Vested(2)

Market Value of Shares of Stock

that Have Not Vested(3) Name Exercisable Unexercisable

Management’s Discussion and Analysis of Financial Condition and Results of Operation—Critical Accounting Policies and Estimates—Stock-Based Compensation

Page 56: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Executive Employment Arrangements

Ryo Kubota, M.D., Ph.D.

2013 Stock Awards

David L. Lowrance

Page 57: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Hien DeYoung

Compensation Committee Interlocks and Insider Participation

ITEM 12. SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS

Security Ownership of Certain Beneficial Owners and Management

Page 58: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Name and Address of Beneficial Owner

Shares Beneficially Owned

Percent 5% Shareholders

Officers and Directors

Equity Compensation Plan Information

Page 59: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Number of Securities to be Issued Upon

Exercise of Outstanding Options

Weighted Average Exercise Price of

Outstanding Options

Number of Securities Remaining Available for Future Issuance

Under Equity Compensation Plans (Excluding Securities Reflected in Column

(a))

Plan Category (a) (b) (c)

ITEM 13. CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS AND DIRECTOR INDEPENDENCE

Transactions with Otsuka

Collaborations with Otsuka.

Item 1. Business-Collaborations with Otsuka

Arrangement with Otsuka.

Page 60: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Contingently Convertible Debt with SBI

Holder Principal

Current Interest

Rate Maturity Date

Interest Paid Since

January 2012

Amount Outstanding

as of December 31,

2013

Total

Consulting Relationship

Loans to Executive Officers

Executive Compensation—Summary Compensation—2013 Stock Awards Executive Compensation—Executive Employment Arrangements—Ryo Kubota, M.D., Ph.D.

Review, Approval or Ratification of Transactions with Related Parties

Page 61: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Independence of Directors

ITEM 14. PRINCIPAL ACCOUNTANT FEES AND SERVICES

Information Regarding our Independent Registered Public Accounting Firm

2013 2012

Audit Fees.

Audit-Related Fees.

Tax Fees.

All Other Fees.

Policy on Audit Committee Pre-Approval of Audit and Permissible Non-Audit Services of Independent Registered Public Accounting Firm

ITEM 15. EXHIBITS AND FINANCIAL STATEMENT SCHEDULES

Page 62: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

SIGNATURES

ACUCELA INC.

Page 63: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh
Page 64: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Incorporated by Reference Exhibit Number

Filed Herewith Form

Period Ending Exhibit Filing Date

Page 65: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ACUCELA INC. INDEX TO FINANCIAL STATEMENTS

Page

Page 66: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

REPORT OF INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRM

Page 67: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ACUCELA INC. BALANCE SHEETS

(in thousands)

December 31, 2013 2012

Assets

Liabilities and shareholders’ equity

Page 68: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ACUCELA INC. STATEMENTS OF INCOME

(in thousands, except per share data)

Year Ended December 31,

2013 2012 2011

Page 69: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ACUCELA INC.

STATEMENTS OF COMPREHENSIVE INCOME (in thousands)

Year Ended December 31, 2013 2012 2011

Page 70: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

AC

UC

EL

A IN

C.

STAT

EM

EN

TS

OF

SHA

RE

HO

LD

ER

S’ E

QU

ITY

(in

thou

sand

s)

Con

vert

ible

Pre

ferr

ed S

tock

A

dditi

onal

Pa

id-I

n C

apita

l

Acc

umul

ated

O

ther

C

ompr

ehen

sive

L

oss

Acc

umul

ated

D

efic

it To

tal

Seri

es A

Se

ries

B

Seri

es C

C

omm

on S

tock

Sh

ares

A

mou

nt

Shar

es

Am

ount

Sh

ares

A

mou

nt

Shar

es

Am

ount

2,73

4$

2,05

1 17

,900

$ 13

,387

11

,807

$

12,7

71

11,9

71

$ 3,

654

$ 2,

728

$ (7

) $

(3,4

60 )

$ 31

,124

Page 71: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ACUCELA INC.

STATEMENTS OF CASH FLOWS

(in thousands)

Year Ended December 31,

2013 2012 2011 Cash flows from operating activities

Cash flows from investing activities

Cash flows from financing activities

Supplemental disclosure

Page 72: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

ACUCELA INC.

NOTES TO FINANCIAL STATEMENTS

Note 1. Summary of Business and Significant Accounting Policies

Overview

Use of Estimates

Cash and Cash Equivalents

Investments

Page 73: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Restricted Investments

Accounts Receivable

Property and Equipment

Revenue Recognition

Multiple Element Arrangements

Page 74: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Substantive Milestone Payments

Deferred Revenue

Income Taxes

Stock-Based Compensation

Expected Term

Page 75: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Expected Volatility

Expected Dividend

Risk-Free Interest Rate

Years ended December 31, 2013 2012 2011

Research and Development Costs

Deferred Offering Costs

Note 2. Net Income Per Share

Page 76: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Year Ended December 31, 2013 2012 2011

Note 3. Concentration of Risk

Note 4. Segment Reporting

Note 5. Collaboration and License Agreements

Emixustat Collaboration

Page 77: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh
Page 78: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Revenue Recognition-Milestone Method

OPA-6566 Collaboration

Rebamipide Collaboration

Page 79: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Continued Involvement of the CEO

Note 6. Investments and Cash and Cash Equivalents

December 31, 2013

Amortized Cost

Gross Unrealized Fair

Value Holding Gains

Holding Losses

December 31, 2012

Amortized Cost

Gross Unrealized Fair

Value Holding Gains

Holding Losses

Page 80: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Note 7. Fair Value Measurements

December 31, 2013

Level 1 Level 2 Level 3 Total

Page 81: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

December 31, 2012

Level 1 Level 2 Level 3 Total

Note 8. Property and Equipment

December 31,

2013 2012

Page 82: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Note 9. Contingently Convertible Debt with Related Party

Page 83: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Note 10. Income Taxes

December 31,

2013 2012

Page 84: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Year Ended December 31,

2013 2012 2011

Year Ended December 31,

2013 2012 2011

Note 11. Commitments

Leases

Page 85: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Credit Facility

Note 12. Shareholders’ Equity

Common Stock

Convertible Preferred Stock

Conversion

Liquidation

Page 86: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Dividends

Voting

Changes in Accumulated Other Comprehensive Loss (in thousands):

Year Ended December 31,

2013 2012

Note 13. Stock-Based Compensation

Stock Option Plan

Page 87: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Shares Available for Grant

Number of Option Shares Outstanding

Weighted Average Exercise Price of

Option Shares Options

Exercisable

December 31, 2013

Exercise Prices

Number of Option Shares

Weighted Average Remaining Contractual Life

(Years) Options Exercisable

Page 88: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

CEO Equity Agreement

Note 14. 401(k) Retirement Plan

Note 15. Related-Party Transactions

Note 16. Strategic Restructuring

Page 89: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Severance and Other Termination Benefits

Note 17. Subsequent Events

Initial Public Offering

December 31, 2013

Pro forma December 31, 2013

Page 90: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

Note 18. Quarterly Information (Unaudited)

March 31 June 30 September 30 December 31

(In thousands, except per share amounts)

2013

March 31 June 30 September 30 December 31

(In thousands, except per share amounts)

2012

Page 91: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh
Page 92: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh
Page 93: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh
Page 94: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh
Page 95: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

EXECUTIVE OFFICERS

Ryo Kubota, MD, PhD Chairman, President and Chief Executive Officer Dave Lowrance, CPA Chief Financial Officer, Corporate Secretary and Treasurer Hien DeYoung, CHIC, SPHR Vice President, Human Resources

BOARD OF DIRECTORS

Ryo Kubota, MD, PhD Chairman, President and Chief Executive Officer Peter Kresel Senior Vice President of Global Regulatory Affairs, Allergan (retired) Brian O’Callaghan Chairman and CEO, Sonrgy Inc. Glen Sato, JD Partner, Cooley LLP Michael Schutzler CEO, The Washington Technology Industry Association (WTIA)

SHAREHOLDERS’ INFORMATION

Corporate Headquarters 1301 Second Avenue, Suite 1900 Seattle, WA 98101-3805 (206) 805-8300 www.acucela.com

Annual Shareholders Meeting 1 p.m. Pacific Time, Thursday, June 5, 2014 Acucela Inc. Corporate Headquarters 1301 Second Avenue, Suite 1900 Seattle, WA 98101-3805

Stock Listing Acucela common stock is listed under the code 4589 on the Mothers Market of the Tokyo Stock Exchange.

Transfer Agent Computershare Shareholder Relations P.O. Box 30170 College Station, TX 77842-3170 877-522-7762 Outside U.S.: 201-680-6578 www.computershare.com/investor

Forward-Looking Statements The statements contained in this report that are not purely historical are forward-looking statements. These statements include, but are not limited to, statements about our plans, objectives, expectations, strategies, intentions or other characterizations of future events or circumstances. These statements are generally identified by the words “expects,” anticipates,” “intends,” “plans,” “believes,” “seeks,” “estimates” and similar expressions. These forward-looking statements are based on current information and expectations and are subject to a number of risks and uncertainties, including, but not limited to the risk that our product candidates will not demonstrate the expected benefits and will not achieve regulatory approval or be successfully commercialized, the success of our product candidates depends heavily on the willingness of our collaboration partner to continue to co-develop our 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 product candidates, our dependence on and our ability to retain and motivate our key management and scientific staff, including Ryo Kubota, M.D., Ph.D., and other risks and uncertainties inherent in the process of discovering and developing therapeutics that demonstrate safety and efficacy. Our actual results could differ materially and adversely from those expressed or implied by these forward-looking statements. For a detailed description of our risks and uncertainties, you are encouraged to review the documents filed with the Securities and Exchange Commission. We assume no obligation to revise or update any such forward-looking statements.

Page 96: 2013 ANNUAL REPORT TO STOCKHOLDERS · dv zhoo dv surwhfwlqj wkh uhwlqd iurp oljkw gdpdjh 7kh ylvxdo f\foh lv wkh elrorjlfdo frqyhuvlrq ri d skrwrq lqwr dq hohfwulfdo vljqdo lq wkh

1301 Second AvenueSuite 1900, 19th Floor

Seattle, WA 98101-3805206.805.8300

www.acucela.com