10-Q 1 wve-10q_20160930.htm 10-Q wve-10q_20160930.htm

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

FORM 10-Q

 

(Mark One)

QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended September 30, 2016

TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from ______ to ______

Commission File No. 001-37627

 

WAVE LIFE SCIENCES LTD.

(Exact name of registrant as specified in its charter)

 

 

Singapore

State or other jurisdiction of

incorporation or organization)

 

Not applicable

(I.R.S. Employer

Identification No.)

 

 

 

8 Cross Street #10-00, PWC Building

Singapore 048424

(Address of principal executive offices)

 

+65 6236 3388

(Registrant’s telephone number)

 

Indicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.    Yes      No   

Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Web site, if any, every Interactive Data File required to be submitted and posted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit and post such files).    Yes      No  

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reporting company. See the definitions of “large accelerated filer,” “accelerated filer” and “smaller reporting company” in Rule 12b-2 of the Exchange Act.

 

Large accelerated filer

 

Accelerated filer

 

 

 

 

 

Non-accelerated filer

(Do not check if a smaller reporting company)

Smaller reporting company

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act).    Yes      No  

The number of outstanding ordinary shares of the registrant as of November 1, 2016 was 23,475,469.

 

 

 

 

 


WAVE LIFE SCIENCES LTD.

QUARTERLY REPORT ON FORM 10-Q

TABLE OF CONTENTS

 

 

 

Page

PART I - FINANCIAL INFORMATION

 

3

Item 1. Financial Statements

 

3

Unaudited Condensed Consolidated Balance Sheets

 

3

Unaudited Condensed Consolidated Statements of Operations

 

4

Unaudited Condensed Consolidated Statements of Comprehensive Loss

 

5

Unaudited Condensed Consolidated Statements of Cash Flows

 

6

Notes to Unaudited Condensed Consolidated Financial Statements

 

7

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

 

16

Item 3. Quantitative and Qualitative Disclosures about Market Risk

 

26

Item 4. Controls and Procedures

 

27

PART II - OTHER INFORMATION

 

28

Item 1. Legal Proceedings

 

28

Item 1A. Risk Factors

 

28

Item 2. Unregistered Sales of Equity Securities and Use of Proceeds

 

28

Item 3. Defaults Upon Senior Securities

 

28

Item 4. Mine Safety Disclosures

 

28

Item 5. Other Information

 

28

Item 6. Exhibits

 

28

 

 

2

 


PART I - FINANCIAL INFORMATION

Item 1. Financial Statements.

WAVE LIFE SCIENCES LTD.

UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS

(In thousands, except share amounts)

 

 

 

September 30, 2016

 

 

December 31, 2015

 

Assets

 

 

 

 

 

 

 

 

Current assets:

 

 

 

 

 

 

 

 

Cash and cash equivalents

 

$

169,015

 

 

$

161,220

 

Accounts receivable

 

 

2,500

 

 

 

 

Prepaid expenses and other current assets

 

 

625

 

 

 

146

 

Deferred tax assets

 

 

 

 

 

18

 

Total current assets

 

 

172,140

 

 

 

161,384

 

Property and equipment, net

 

 

6,693

 

 

 

2,789

 

Deferred tax assets

 

 

32

 

 

 

192

 

Restricted cash

 

 

1,055

 

 

 

1,055

 

Other assets

 

 

53

 

 

 

4

 

Total assets

 

$

179,973

 

 

$

165,424

 

Liabilities, Series A preferred shares and shareholders’ equity

 

 

 

 

 

 

 

 

Current liabilities:

 

 

 

 

 

 

 

 

Accounts payable

 

$

3,825

 

 

$

2,811

 

Accrued expenses and other current liabilities

 

 

4,833

 

 

 

945

 

Current portion of capital lease obligation

 

 

62

 

 

 

62

 

Deferred tax liabilities

 

 

 

 

 

 

Current portion of deferred revenue

 

 

2,705

 

 

 

 

Total current liabilities

 

 

11,425

 

 

 

3,818

 

Long-term liabilities:

 

 

 

 

 

 

 

 

Capital lease obligation, net of current portion

 

 

31

 

 

 

78

 

Deferred revenue, net of current portion

 

 

8,987

 

 

 

 

Other liabilities

 

 

522

 

 

 

163

 

Total long-term liabilities

 

 

9,540

 

 

 

241

 

Total liabilities

 

$

20,965

 

 

$

4,059

 

Series A preferred shares, no par value; 3,901,348 shares issued and outstanding

 

 

7,874

 

 

 

7,874

 

Shareholders’ equity:

 

 

 

 

 

 

 

 

Ordinary shares, no par value; 23,474,369 and 21,551,423 shares issued and outstanding

   at September 30, 2016 and December 31, 2015, respectively

 

 

215,505

 

 

 

185,344

 

Additional paid-in capital

 

 

7,568

 

 

 

3,182

 

Accumulated other comprehensive income

 

 

84

 

 

 

41

 

Accumulated deficit

 

 

(72,023

)

 

 

(35,076

)

Total shareholders’ equity

 

 

151,134

 

 

 

153,491

 

Total liabilities, Series A preferred shares and shareholders’ equity

 

$

179,973

 

 

$

165,424

 

 

The accompanying notes are an integral part of the unaudited condensed consolidated financial statements.

 

 

3

 


WAVE LIFE SCIENCES LTD.

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

(In thousands, except share and per share amounts)

 

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

 

2016

 

 

2015

 

 

2016

 

 

2015

 

Revenue

 

$

392

 

 

$

 

 

$

809

 

 

$

152

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

 

13,686

 

 

 

2,132

 

 

 

26,823

 

 

 

5,589

 

General and administrative

 

 

3,939

 

 

 

2,858

 

 

 

10,809

 

 

 

6,647

 

Total operating expenses

 

 

17,625

 

 

 

4,990

 

 

 

37,632

 

 

 

12,236

 

Loss from operations

 

 

(17,233

)

 

 

(4,990

)

 

 

(36,823

)

 

 

(12,084

)

Other income (expense):

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Interest income (expense), net

 

 

118

 

 

 

25

 

 

 

328

 

 

 

10

 

Other income (expense), net

 

 

(36

)

 

 

112

 

 

 

(25

)

 

 

155

 

Total other income (expense), net

 

 

82

 

 

 

137

 

 

 

303

 

 

 

165

 

Loss before income tax provision

 

 

(17,151

)

 

 

(4,853

)

 

 

(36,520

)

 

 

(11,919

)

Income tax provision

 

 

(384

)

 

 

(83

)

 

 

(427

)

 

 

(182

)

Net loss

 

$

(17,535

)

 

$

(4,936

)

 

$

(36,947

)

 

$

(12,101

)

Net loss per share attributable to ordinary shareholders—basic

   and diluted

 

$

(0.75

)

 

$

(0.54

)

 

$

(1.64

)

 

$

(1.36

)

Weighted-average ordinary shares used in computing net loss

   per share attributable to ordinary shareholders—basic

   and diluted

 

 

23,445,673

 

 

 

9,223,405

 

 

 

22,571,575

 

 

 

8,895,660

 

 

The accompanying notes are an integral part of the unaudited condensed consolidated financial statements.

 

 

4

 


WAVE LIFE SCIENCES LTD.

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS

(In thousands)

 

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

 

2016

 

 

2015

 

 

2016

 

 

2015

 

Net loss

 

$

(17,535

)

 

$

(4,936

)

 

$

(36,947

)

 

$

(12,101

)

Other comprehensive income (loss):

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Foreign currency translation

 

 

8

 

 

 

9

 

 

 

43

 

 

 

(13

)

Comprehensive loss

 

$

(17,527

)

 

$

(4,927

)

 

$

(36,904

)

 

$

(12,114

)

 

The accompanying notes are an integral part of the unaudited condensed consolidated financial statements.

 

 

5

 


WAVE LIFE SCIENCES LTD.

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

(In thousands)

 

 

 

Nine Months Ended September 30,

 

 

 

2016

 

 

2015

 

Cash flows from operating activities

 

 

 

 

 

 

 

 

Net loss

 

$

(36,947

)

 

$

(12,101

)

Adjustments to reconcile net loss to net cash flows used in operating activities:

 

 

 

 

 

 

 

 

Depreciation and amortization

 

 

525

 

 

 

462

 

Share-based compensation expense

 

 

4,319

 

 

 

3,210

 

Deferred rent

 

 

371

 

 

 

(4

)

Deferred income taxes

 

 

178

 

 

 

182

 

Tax benefit related to share-based compensation

 

 

(67

)

 

 

 

Changes in operating assets and liabilities:

 

 

 

 

 

 

 

 

Accounts receivable

 

 

(2,500

)

 

 

193

 

Prepaid expenses and other current assets

 

 

(466

)

 

 

(40

)

Other non-current assets

 

 

(53

)

 

 

 

Accounts payable

 

 

1,899

 

 

 

2,464

 

Accrued expenses and other current liabilities

 

 

2,484

 

 

 

(73

)

Deferred revenue

 

 

11,692

 

 

 

(152

)

Other non-current liabilities

 

 

(13

)

 

 

 

Net cash used in operating activities

 

 

(18,578

)

 

 

(5,859

)

Cash flows from investing activities

 

 

 

 

 

 

 

 

Increase in restricted cash

 

 

 

 

 

(1,055

)

Proceeds from government grant reimbursements for property and equipment

 

 

 

 

 

3

 

Proceeds from the sale of property and equipment

 

 

4

 

 

 

 

Purchases of property and equipment

 

 

(2,838

)

 

 

(887

)

Net cash used in investing activities

 

 

(2,834

)

 

 

(1,939

)

Cash flows from financing activities

 

 

 

 

 

 

 

 

Proceeds from issuance of ordinary shares, net of offering costs

 

 

30,000

 

 

 

11,631

 

Proceeds from issuance of Series B preferred shares, net of offering costs

 

 

 

 

 

62,643

 

Proceeds from government grant

 

 

 

 

 

112

 

Costs associated with initial public offering

 

 

(1,075

)

 

 

(1,417

)

Payments on capital lease obligation

 

 

(47

)

 

 

(112

)

Proceeds from the exercise of share options

 

 

161

 

 

 

 

Tax benefit related to share-based compensation

 

 

67

 

 

 

 

Net cash provided by financing activities

 

 

29,106

 

 

 

72,857

 

Effect of foreign exchange rates on cash

 

 

101

 

 

 

(67

)

Net increase in cash and cash equivalents

 

 

7,795

 

 

 

64,992

 

Cash and cash equivalents at beginning of period

 

 

161,220

 

 

 

1,048

 

Cash and cash equivalents at end of period

 

$

169,015

 

 

$

66,040

 

Supplemental disclosure of cash flow information:

 

 

 

 

 

 

 

 

Reclassification of Series A preferred shares from permanent equity to temporary equity

 

$

 

 

$

7,874

 

Equipment acquired for capital lease obligation

 

$

 

 

$

268

 

Increase in accounts payable for initial public offering costs

 

$

 

 

$

319

 

Property and equipment purchases in accounts payable and accrued expenses at period end

 

$

1,595

 

 

$

 

 

The accompanying notes are an integral part of the unaudited condensed consolidated financial statements.

 

 

6

 


WAVE Life Sciences Ltd.

Notes to Unaudited Condensed Consolidated Financial Statements

 

1. THE COMPANY

Organization

WAVE Life Sciences Ltd. (together with its subsidiaries, “WAVE” or the “Company”) is a preclinical biotechnology company with an innovative and proprietary synthetic chemistry drug development platform that the Company is using to design, develop and commercialize a broad pipeline of first-in-class or best-in-class nucleic acid therapeutic candidates. The Company is initially developing nucleic acid therapeutics that target genetic defects to either reduce the expression of disease-promoting proteins or transform the production of dysfunctional mutant proteins into the production of functional proteins.

The Company was incorporated in Singapore on July 23, 2012 and has its principal office in Cambridge, Massachusetts. The Company was incorporated with the purpose of combining two commonly held companies, WAVE Life Sciences USA, Inc. (“WAVE USA”), a Delaware corporation (formerly Ontorii, Inc.), and WAVE Life Sciences Japan, Inc. (“WAVE Japan”), a company organized under the laws of Japan (formerly Chiralgen., Ltd.), which occurred on September 12, 2012. On May 31, 2016, WAVE Life Sciences Ireland Limited (“WAVE Ireland”) was formed as a wholly-owned subsidiary of WAVE Life Sciences Ltd.  It was formed as a private company limited by shares and the company number is 583482. Its registered office is One Spencer Dock, North Wall Quay, Dublin 1, Ireland.

The Company’s primary activities since inception have been developing a synthetic chemistry drug development platform to design, develop and commercialize nucleic acid therapeutic programs, advancing the Company’s neurology franchise, expanding the Company’s research and development activities to enter the clinic, building the Company’s intellectual property, recruiting personnel and raising capital to support these activities.

Risks and Uncertainties

The Company is subject to risks common to companies in the biotechnology industry including, but not limited to, new technological innovations, protection of proprietary technology, dependence on key personnel, compliance with government regulations and the need to obtain additional financing. The Company’s therapeutic programs will require significant additional research and development efforts, including extensive pre-clinical and clinical testing and regulatory approval, prior to commercialization of any product candidates. These efforts require significant amounts of additional capital, adequate personnel infrastructure and extensive compliance-reporting capabilities. The Company’s therapeutic programs are currently in the development or discovery stage. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained, that any products developed will obtain necessary government regulatory approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees and consultants.

Basis of Presentation

The Company has prepared the accompanying condensed consolidated financial statements in conformity with generally accepted accounting principles in the United States of America (“U.S. GAAP”) and in U.S. dollars.

 

 

2. SIGNIFICANT ACCOUNTING POLICIES

The significant accounting policies described in the Company’s audited financial statements as of and for the year ended December 31, 2015, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”) on March 30, 2016, have had no material changes during the three and nine months ended September 30, 2016, except for the two items discussed below.

Cash and Cash Equivalents

The Company considers all highly liquid securities with original final maturities of three months or less from the date of purchase to be cash equivalents. Cash equivalents are comprised of funds in money market accounts.

7

 


Revenue Recognition

To date, the Company’s only significant source of revenue is derived from the Pfizer Collaboration Agreement (as defined in Note 4), pursuant to which the Company and Pfizer (as defined in Note 4) have agreed to collaborate on the discovery, development and commercialization of stereopure oligonucleotide therapeutics for the Pfizer Programs (as defined in Note 4), each directed at a genetically-defined hepatic target selected by Pfizer. We entered into the Pfizer Collaboration Agreement in May 2016.

The Company presents revenue from the Pfizer Collaboration Agreement under Financial Accounting Standards Board (“FASB”), Accounting Standards Codification (“ASC”) Topic 808, Collaborative Arrangements (“ASC 808”).  In addition, the Company recognizes revenue in accordance with ASC Topic 605, Revenue Recognition (“ASC 605”). Accordingly, revenue is recognized for each unit of accounting when all of the following criteria are met:

 

persuasive evidence of an arrangement exists;

 

delivery has occurred or services have been rendered;

 

the seller’s price to the buyer is fixed or determinable; and

 

collectability is reasonably assured.

Amounts received prior to satisfying the revenue recognition criteria are recorded as deferred revenue. Amounts expected to be recognized as revenue within the 12 months following the balance sheet date are classified as current portion of deferred revenue in the accompanying condensed consolidated balance sheets. Amounts not expected to be recognized as revenue within the 12 months following the balance sheet date are classified as deferred revenue, net of current portion.

The Company evaluates multiple‑element arrangements based on the guidance in ASC Topic 605‑25, Revenue Recognition Multiple‑Element Arrangements (“ASC 605‑25”). Pursuant to the guidance in ASC 605‑25, the Company evaluates multiple‑element arrangements to determine (1) the deliverables included in the arrangement and (2) whether the individual deliverables represent separate units of accounting or whether they must be accounted for as a combined unit of accounting. This evaluation involves subjective determinations and requires the Company to make judgments about the individual deliverables and whether such deliverables are separable from the other aspects of the contractual relationship. Deliverables are considered separate units of accounting provided that the delivered item has value to the customer on a standalone basis and, if the arrangement includes a general right of return relative to the delivered item, delivery or performance of the undelivered item is considered probable and substantially in the Company’s control. In assessing whether an item has standalone value, the Company considers factors such as the research, development, manufacturing and commercialization capabilities of the collaboration partner and the availability of the associated expertise in the general marketplace. In addition, the Company considers whether the collaboration partner can use a deliverable for its intended purpose without the receipt of the remaining deliverable, whether the value of the deliverable is dependent on the undelivered item and whether there are other vendors that can provide the undelivered items.

The Pfizer Collaboration Agreement provides Pfizer with certain options and the Company is required to consider whether such options are substantive.  Options are considered substantive if, at the inception of the arrangement, the Company is at risk as to whether the collaboration partner will choose to exercise the option. Factors that the Company considers in evaluating whether an option is substantive include the cost to exercise the option, the overall objective of the arrangement, the benefit the collaborator might obtain from the arrangement without exercising the option and the likelihood the option will be exercised.

When an option is considered substantive, the Company does not consider the option or item underlying the option to be a deliverable at the inception of the arrangement and the associated option fees are not included in allocable consideration, assuming the option is not priced at a significant and incremental discount. Conversely, when an option is not considered substantive, the Company would consider the option, including other deliverables contingent upon the exercise of the option, to be a deliverable at the inception of the arrangement and a corresponding amount would be included in allocable arrangement consideration. In addition, if the price of the option includes a significant incremental discount, the discount would be included as a deliverable at the inception of the arrangement.

The Company has determined that the options held by Pfizer under the Pfizer Collaboration Agreement are not substantive, mainly because they are deemed to be priced at a significant incremental discount as compared to the upfront license fee. Accordingly, the options are considered deliverables at the inception of the arrangement and, therefore, the Company has included any related amounts in the allocable consideration at the outset of the arrangement, which totals $17.5 million.  

At the inception of an arrangement that includes milestone payments, the Company evaluates whether each milestone is substantive and at risk to both parties on the basis of the contingent nature of the milestone. This evaluation includes an assessment of whether: (1) the consideration is commensurate with either the Company’s performance to achieve the milestone or the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from its performance to achieve the milestone, (2) the

8

 


consideration relates solely to past performance and (3) the consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. The Company evaluates factors such as the scientific, clinical, regulatory, commercial, and other risks that must be overcome to achieve the particular milestone and the level of effort and investment required to achieve the particular milestone in making this assessment. There is considerable judgment involved in determining whether a milestone satisfies all of the criteria required to conclude that a milestone is substantive. Milestones that are not considered substantive are recognized as earned if there are no remaining performance obligations over the remaining period of performance, assuming all other revenue recognition criteria are met.

Aside from the program nomination milestone payments, which relate to the options described above, the remaining milestone payments required under the Pfizer Collaboration Agreement are contingent upon the Company’s performance under the Pfizer Collaboration Agreement, including in certain instances, regulatory approval.  The Company views the milestones as substantive and has excluded the amounts as allocable consideration at the outset of the arrangement.

During the nine months ended September 30, 2016, the Company received a non-refundable upfront payment of $40.0 million under the Pfizer Collaboration Agreement (as defined in Note 4), which included payment for 1,875,000 shares of the Company’s ordinary shares, which were valued at $30.0 million based on the purchase price of $16.00 per share.  During the three months ended September 30, 2016, the Company recorded a receivable of $2.5 million related to a milestone payment that is due from Pfizer under the Pfizer Collaboration Agreement.  

The Company recognizes arrangement consideration allocated to each unit of accounting when all of the revenue recognition criteria in ASC 605 are satisfied for that particular unit of accounting. In the event that a deliverable does not represent a separate unit of accounting, the Company recognizes revenue from the combined unit of accounting over the Company’s contractual or estimated performance period for the undelivered elements, which is typically the term of the Company’s research and development obligations. If there is no discernible pattern of performance or objectively measurable performance measures do not exist, then the Company recognizes revenue under the arrangement on a straight‑line basis over the period the Company is expected to complete its performance obligations. Conversely, if the pattern of performance in which the service is provided to the customer can be determined and objectively measurable performance measures exist, then the Company recognizes revenue under the arrangement using the proportional performance method. Revenue recognized is limited to the lesser of the cumulative amount of payments received or the cumulative amount of revenue earned, as determined using the straight‑line method or proportional performance method, as applicable, as of the period ending date.

The Company has concluded that the deliverables under the Pfizer Collaboration Agreement relate primarily to the research and development required by the Company for each of the programs nominated by Pfizer.  The remaining deliverables, including sample supplies provided by each party to fulfill its obligation as a licensee, participation on a joint steering committee to oversee the research and development activities, and regulatory responsibilities related to filings and obtaining approvals related to the products that may result from each program do not represent separate units of accounting based on their dependence on the research and development efforts.

Because there is no discernible pattern of performance given the nature of the research and development efforts, the Company recognizes the allocated revenue for each deliverable under the Pfizer Collaboration Agreement on a straightline basis over the period the Company is expected to complete its performance obligations for each deliverable, or unit of accounting. For the first two Pfizer Programs, this period is expected to be from the initiation date of the Pfizer Collaboration Agreement, which was May 5, 2016, and for the other Pfizer Programs, the period is expected to be from the date that work commences on those programs through the earlier of (a) the termination of the research and development performance obligations under the Pfizer Collaboration Agreement, which is May 5, 2020 (the “Research Term”), or (b) the estimated date the Company expects to meet its research and development performance obligations under the Pfizer Collaboration Agreement. Given the uncertainty as to when the research and development performance obligations will be completed, the Company has used the Research Term, for purposes of applying the straight-line method for revenue recognition for the three and nine months ended September 30, 2016.

Unaudited Interim Financial Data

The accompanying interim condensed consolidated balance sheet as of September 30, 2016, the related interim condensed consolidated statements of operations and comprehensive loss for the three and nine months ended September 30, 2016 and 2015, and cash flows for the nine months ended September 30, 2016 and 2015, and the related interim information contained within the notes to the condensed consolidated financial statements have been prepared in accordance with the rules and regulations of the SEC for interim financial information. Accordingly, they do not include all of the information and the notes required by U.S. GAAP for complete financial statements. The financial data and other information disclosed in these notes related to the three and nine months ended September 30, 2016 and 2015 are unaudited. In the opinion of management, the unaudited interim condensed consolidated financial statements reflect all adjustments, consisting of normal and recurring adjustments, necessary for the fair presentation of the

9

 


Company’s financial position and results of operations for the interim periods ended September 30, 2016 and 2015. The results of operations for the interim periods are not necessarily indicative of the results to be expected for the year ending December 31, 2016 or any other interim period or future year or period.

Principles of Consolidation

The Company’s condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All significant intercompany balances and transactions have been eliminated in consolidation.

Recently Issued Accounting Pronouncements

In May 2014, the FASB issued ASU 2014-09, Revenue from Contracts with Customers (Topic 606) (“ASU 2014-09”), which supersedes all existing revenue recognition requirements, including most industry-specific guidance. The new standard requires a company to recognize revenue when it transfers goods or services to customers in an amount that reflects the consideration that the company expects to receive for those goods or services. The new standard will be effective on January 1, 2018 and earlier application is permitted only for annual reporting periods beginning after December 15, 2016, including interim reporting periods within that reporting period. The Company is currently evaluating the potential impact that ASU 2014-09 may have on its consolidated financial statements.

In February 2015, the FASB issued ASU 2015-02, Consolidation (Topic 810) (“ASU 2015-02”), to address financial reporting considerations for the evaluation as to the requirement to consolidate certain legal entities. ASU 2015-02 is effective for fiscal years and for interim periods within those fiscal years beginning after December 15, 2015.  The Company has evaluated the impact of ASU 2015-02 and has concluded that it has no effect on the consolidated financial statements.

In April 2015, the FASB issued ASU 2015-03, Interest—Imputation of Interest (Subtopic 835-30) (“ASU 2015-03”), as part of the initiative to reduce complexity in accounting standards. The update requires that debt issuance costs related to a recognized debt liability be presented in the balance sheet as a direct deduction from the carrying amount of that debt liability, consistent with debt discounts. The Company has evaluated the impact of ASU 2015-03 and has concluded that it has no effect on the consolidated financial statements.

In November 2015, the FASB issued ASU 2015-17, Balance Sheet Classification of Deferred Taxes (“ASU 2015-17”), which requires entities to present deferred tax assets and deferred tax liabilities as noncurrent in a classified balance sheet. The ASU simplifies the current guidance in ASC Topic 740, Income Taxes, which requires entities to separately present deferred tax assets and liabilities as current and noncurrent in a classified balance sheet. ASU 2015-17 is effective for fiscal years beginning after December 31, 2016, and interim periods within those annual periods. Early adoption is permitted for all entities as of the beginning of an interim or annual reporting period. The Company does not expect the impact of ASU 2015-17 to be material to its consolidated financial statements.

In February 2016, the FASB issued ASU 2016-02, Leases (“ASU 2016-02”), which requires a lessee to recognize assets and liabilities on the balance sheet for operating leases and changes many key definitions, including the definition of a lease. The update includes a short-term lease exception for leases with a term of 12 months or less, in which a lessee can make an accounting policy election not to recognize lease assets and lease liabilities. Lessees will continue to differentiate between finance leases (previously referred to as capital leases) and operating leases, using classification criteria that are substantially similar to the previous guidance. For lessees, the recognition, measurement, and presentation of expenses and cash flows arising from a lease have not significantly changed from previous U.S. GAAP. Lessees and lessors are required to recognize and measure leases at the beginning of the earliest period presented using a modified retrospective approach. The modified retrospective approach includes a number of optional practical expedients that entities may elect to apply as well as transition guidance specific to nonstandard leasing transactions. ASU 2016-02 is effective for fiscal years beginning after December 15, 2018, and interim periods within those fiscal years. The Company is currently evaluating the potential impact that ASU 2016-02 may have on its consolidated financial statements.

In March 2016, the FASB issued ASU 2016-09, Improvements to Employee Share-Based Payment Accounting (“ASU 2016-09”), which simplifies share-based payment accounting through a variety of amendments. The standard will be effective for annual reporting periods and interim periods within those annual periods, beginning after December 31, 2016, and early adoption is permitted. The Company does not expect the impact of ASU 2016-09 to be material to its consolidated financial statements. 

In October 2016, the FASB issued ASU 2016-16, Income Taxes (Topic 740): Intra-Entity Transfers of Assets Other Than Inventory (“ASU 2016-16”).  The ASU requires companies to recognize the income tax consequences of an intra-entity transfer of an asset other than inventory when the transfer occurs. Prior to the issuance of this ASU, existing guidance prohibited the recognition of current and deferred income taxes for an intra-entity asset transfer until the asset has been sold to an outside party. ASU 2016-16 will be effective

10

 


for the Company in the first quarter of 2018 with early adoption permitted. The Company is currently evaluating the impact that the adoption of ASU 2016-16 will have on the Company’s consolidated financial statements or related disclosures.

Other accounting standards that have been issued or proposed by the FASB or other standards-setting bodies that do not require adoption until a future date, are not expected to have a material impact on the Company’s consolidated financial statements upon adoption.

 

 

3. SHARE-BASED COMPENSATION

The WAVE Life Sciences Ltd. 2014 Equity Incentive Plan (the “2014 Plan”) authorizes the board of directors or a committee of the board to grant incentive share options (“ISOs”), non-qualified share options (“NQSOs”), share appreciation rights and restricted share awards to eligible employees, outside directors and consultants of the Company. Options generally vest over a period of three or four years, and options that lapse or are forfeited are available to be granted again. The contractual life of all options is ten years from the grant date.

As of September 30, 2016, 1,293,750 ordinary shares remained available for future grant under the 2014 Plan.

The Company measures and records the value of options granted to non-employees over the period of time that services are provided and, as such, unvested portions are subject to re-measurement at subsequent reporting periods.

Share Options

Share option activity under the 2014 Plan for the nine months ended September 30, 2016 is summarized as follows:

 

 

 

Number of

Shares

 

 

Weighted-

Average

Exercise Price

 

Options outstanding as of January 1, 2016

 

 

2,215,342

 

 

$

3.88

 

Granted

 

 

1,359,300

 

 

$

19.65

 

Exercised

 

 

(47,946

)

 

$

3.38

 

Cancelled or forfeited

 

 

(17,454

)

 

$

22.91

 

Outstanding as of September 30, 2016

 

 

3,509,242

 

 

$

9.98

 

Options exercisable as of September 30, 2016

 

 

1,077,232

 

 

$

2.54

 

Options vested and expected to vest as of September 30, 2016

 

 

3,387,639

 

 

$

9.87

 

 

The Company recorded share-based compensation expense related to options granted to non-employees in the amount of $0.9 million and $0.3 million for the three months ended September 30, 2016 and 2015, respectively. During the nine months ended September 30, 2016 and 2015, the Company recorded share-based compensation expense related to options granted to non-employees in the amount of $1.9 million and $1.2 million, respectively. Share-based compensation expense related to non-employees is recorded in research and development expenses.

 

Restricted Share Units

 

Restricted share unit activity for the nine months ended September 30, 2016 is summarized as follows:

 

 

 

RSUs

 

 

Average Grant Date Fair Value (in dollars per share)

 

RSUs Outstanding as of January 1, 2016

 

 

 

 

$

 

Granted

 

 

22,750

 

 

$

21.69

 

Vested

 

 

 

 

$

 

Forfeited

 

 

 

 

$

 

RSUs Outstanding at September 30, 2016

 

 

22,750

 

 

$

21.69

 

 

11

 


In July 2016, the Company granted 22,750 restricted share units with a grant date fair value of $21.69 per unit. The restricted share units fully vest upon the first anniversary of the grant date.  Share-based compensation expense related to the restricted share units is recorded in research and development expenses.

 

Share-based compensation expense for the three and nine months ended September 30, 2016 and 2015 was classified in the condensed consolidated statements of operations as follows:

 

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

 

2016

 

 

2015

 

 

2016

 

 

2015

 

 

 

(in thousands)

 

Research and development expenses

 

$

1,695

 

 

$

476

 

 

$

3,207

 

 

$

1,657

 

General and administrative expenses

 

 

486

 

 

 

242

 

 

 

1,112

 

 

 

1,553

 

Total share-based compensation

 

$

2,181

 

 

$

718

 

 

$

4,319

 

 

$

3,210

 

 

 

4. PFIZER COLLABORATION AND SHARE PURCHASE AGREEMENT

On May 5, 2016, the Company entered into a Research, License and Option Agreement (the “Pfizer Collaboration Agreement”) with Pfizer Inc. (“Pfizer”). Pursuant to the terms of the Pfizer Collaboration Agreement, the Company and Pfizer agreed to collaborate on the discovery, development and commercialization of stereopure oligonucleotide therapeutics for up to five programs (the “Pfizer Programs”), each directed at a genetically-defined hepatic target selected by Pfizer (the “Collaboration”).  The Company received $10.0 million as an upfront license fee under the Pfizer Collaboration Agreement.  Subject to option exercises by Pfizer, assuming five potential products are successfully developed and commercialized, the Company may earn up to $871.0 million in potential research, development and commercial milestone payments, plus royalties, tiered up to low double-digits, on sales of any products that may result from the Collaboration. None of the payments under the Pfizer Collaboration Agreement are refundable.

Simultaneously with the entry into the Pfizer Collaboration Agreement, the Company entered into a Share Purchase Agreement (the “Pfizer Equity Agreement,” and together with the Pfizer Collaboration Agreement, the “Pfizer Agreements”) with C.P. Pharmaceuticals International C.V., an affiliate of Pfizer (the “Pfizer Affiliate”). Pursuant to the terms of the Pfizer Equity Agreement, the Pfizer Affiliate purchased 1,875,000 of the Company’s ordinary shares (the “Shares”) at a purchase price of $16.00 per share, for an aggregate purchase price of $30.0 million. The Company did not incur any material costs in connection with the issuance of the Shares.

Under the Pfizer Collaboration Agreement, the parties agreed to collaborate during the four-year Research Term. During the Research Term, the Company is responsible to use its commercially reasonable efforts to advance up to five programs through to the selection of clinical candidates. At that stage, Pfizer may elect to license any of these Pfizer Programs exclusively and to have exclusive rights to undertake the clinical development of the resulting clinical candidates into products and the potential commercialization of any such products thereafter. In addition, the Company receives a non-exclusive, royalty-bearing sublicenseable license to use Pfizer’s hepatic targeting technology in any of the Company’s own hepatic programs that are outside the scope of the Collaboration (the “WAVE Programs”). If the Company uses this technology on the WAVE Programs, Pfizer is eligible to receive potential development and commercial milestone payments from the Company. Pfizer is also eligible to receive tiered royalties on sales of any products that include Pfizer’s hepatic targeting technology.

Pfizer nominated two hepatic targets upon entry into the Collaboration in May 2016. In August 2016, Pfizer nominated the third hepatic target under the Collaboration and has the option to nominate two additional targets by November 5, 2017. The Collaboration is managed by a joint steering committee in which both parties are represented equally, which will oversee the scientific progression of each Pfizer Program up to the clinical candidate stage. During the four-year Research Term and for a period of two years thereafter, the Company has agreed to work exclusively with Pfizer with respect to using any of the Company’s stereopure oligonucleotide technology that is specific for the applicable hepatic target which is the basis of any Pfizer Program.  

The stated term of the Pfizer Collaboration Agreement commenced on May 5, 2016 and terminates on the date of the last to expire payment obligations with respect to each Pfizer Program and with respect to each WAVE Program, expires on a program-by-program basis accordingly.  Pfizer may terminate its rights related to a Pfizer Program under the Pfizer Collaboration Agreement at its own convenience upon 90 days’ notice to the Company. The Company may also terminate its rights related to a WAVE Program at its own convenience upon 90 days’ notice to Pfizer.  The Pfizer Collaboration Agreement may also be terminated by either party in the event of an uncured material breach of the Collaboration Agreement by the other party.

12

 


During the three and nine months ended September 30, 2016, the Company recognized revenue of $0.4 million and $0.8 million, respectively, under the Pfizer Collaboration Agreement. Deferred revenue amounted to $11.7 million at September 30, 2016, of which $2.7 million is included in current liabilities.  

 

 

5. NET LOSS PER ORDINARY SHARE

The Company applies the two-class method to calculate its basic and diluted net loss per share attributable to ordinary shareholders, as its Series A preferred shares are participating securities. The two-class method is an earnings allocation formula that treats a participating security as having rights to earnings that otherwise would have been available to ordinary shareholders. However, for the periods presented, the two-class method does not impact the net loss per ordinary share as the Company was in a net loss position for each of the periods presented and holders of Series A preferred shares do not participate in losses.

Basic loss per share is computed by dividing net loss attributable to ordinary shareholders by the weighted-average number of ordinary shares used in computing net loss per share attributable to ordinary shareholders.

The Company’s potentially dilutive shares, which include outstanding share options to purchase ordinary shares and Series A preferred shares, are considered to be ordinary share equivalents and are only included in the calculation of diluted net loss per share when their effect is dilutive.

The following ordinary share equivalents, presented based on amounts outstanding at each period end, were excluded from the calculation of diluted net loss per share attributable to ordinary shareholders for the periods indicated because including them would have had an anti-dilutive effect:

 

 

 

As of September 30,

 

 

 

2016

 

 

2015

 

Options to purchase ordinary shares

 

 

3,509,242

 

 

 

1,943,381

 

Restricted share units

 

 

22,750

 

 

 

 

Series A preferred shares

 

 

3,901,348

 

 

 

3,901,348

 

Series B preferred shares

 

 

 

 

 

5,334,892

 

 

6. LEASES

On September 26, 2016, WAVE Life Sciences USA, Inc. (the “Subsidiary”), a wholly-owned subsidiary of WAVE Life Sciences Ltd. (and together with the Subsidiary for purposes of this Note 6, the “Company”) entered into a Lease Agreement (the “Lease”) with King 115 Hartwell LLC, an affiliate of King Street Properties Investments, LLC (the “Landlord”), primarily to build out and conduct certain of the Company’s Good Manufacturing Practice (“GMP”) manufacturing activities, as well as to provide additional laboratory and office space to support the Company’s growth (the “Manufacturing Facility”). The Manufacturing Facility will supplement the Company’s existing laboratory and office facility (the “Cambridge Facility”) at 733 Concord Avenue, Cambridge, Massachusetts, which serves as the Company’s U.S. headquarters. The Cambridge Facility is also leased to the Company by affiliates of the Landlord. The Manufacturing Facility is located at 115 Hartwell Avenue, Lexington, Massachusetts 02421, which is part of The Hartwell Innovation Campus.

Under the terms of the Lease, the Company will lease 57,561 square feet (the “Initial Manufacturing Space”) for which the Company shall pay an average base rent of approximately $2.8 million per year during the initial term. The initial term of the Lease commences upon the Company’s occupancy of the Manufacturing Facility, which is anticipated under the Lease to be March 21, 2017, and continues for 10 years and nine months from the actual commencement date. The Lease provides the Company with options to renew for two successive terms of five years each, subject to the terms of the Lease.

In addition to the Initial Manufacturing Space, the Company has the option, on or before December 31, 2016, to expand the Initial Manufacturing Space to include the remainder of the building, which is an additional 33,650 square feet (the “Expansion Option”). If the Company exercises the Expansion Option, the Company would be required to pay an additional average base rent of approximately $1.8 million per year during the initial term upon occupancy.

Throughout the term of the Lease, the Company is responsible for paying certain costs and expenses, in addition to the rent, as specified in the Lease, including a proportionate share of applicable taxes, operating expenses, and utilities. The Lease includes various covenants, indemnities, defaults, termination rights, and other provisions customary for lease transactions of this nature, including the posting by the Company of an $2.6 million letter of credit as a security deposit which was not posted prior to September 30, 2016.

 

13

 


 

7. INCOME TAXES

The Company is a multi-national company subject to taxation in the United States, Japan and Singapore. During the nine months ended September 30, 2016 and 2015, the Company recorded a tax provision of $0.4 million and $0.2 million, respectively, each of which are a result of income generated in the United States for each respective period. During the three and nine months ended September 30, 2016 and 2015, the Company recorded no income tax benefits for the net operating losses incurred in Japan and Singapore, due to its uncertainty of realizing a benefit from those items. In May 2016, the Company established a wholly-owned subsidiary in Ireland, however no income tax expense or benefit has been recorded.

The Company’s reserves related to taxes and its accounting for uncertain tax positions are based on a determination of whether and how much of a tax benefit taken by the Company in its tax filings or positions is more-likely-than-not to be realized following resolution of any potential contingencies present related to the tax benefit.

During the nine months ended September 30, 2016, the Company amended its tax filings for transfer pricing adjustments in prior years, resulting in an approximately $1.0 million decrease in the U.S. net operating loss carryforwards and a corresponding decrease in the unrecognized tax benefits. This adjustment has no financial statement impact as historically the net operating losses were netted against the unrecognized tax benefits.

The Company has $0.2 million of federal research and development credit carryforwards as a result of excess tax deductions related to share-based compensation. The Company will realize the benefit of these excess tax deductions through increases to shareholders’ equity in the periods in which these carryforward credits are utilized to reduce cash tax payments.

 

 

8. RELATED PARTIES

The Company had the following related party transactions for the periods presented in the accompanying condensed consolidated financial statements, which have not otherwise been discussed in these notes to the condensed consolidated financial statements:

 

The Company had cash of $128 thousand and $115 thousand at September 30, 2016 and December 31, 2015, respectively, in depository accounts with Kagoshima Bank, Ltd., an affiliate of one of our shareholders, Kagoshima Shinsangyo Sousei Investment Limited Partnership.

 

The Company made payments for lease rentals and other related expenses in the amount of $4 thousand and $55 thousand to Shin Nippon Biomedical Laboratories Ltd. (“SNBL”) for the three months ended September 30, 2016 and 2015, respectively. For the nine months ended September 30, 2016 and 2015 the Company paid SNBL $55 thousand, and $144 thousand, respectively, for lease rentals and other related expenses.  

 

Pursuant to the terms of a service agreement held with SNBL, the Company paid SNBL $0 and $3 thousand for the three months ended September 30, 2016 and 2015, respectively, for accounting and administrative services provided to the Company and its affiliates. For the nine months ended September 30, 2016 and 2015 the Company paid SNBL $3 thousand and $12 thousand, respectively for accounting and administrative services provided to the Company and its affiliates.

 

Pursuant to the terms of a service agreement with SNBL, which the Company entered into in the third quarter 2015, the Company paid SNBL $4 thousand and $329 thousand for the three and nine months ended September 30, 2016, respectively, for contract research services provided to the Company and its affiliates. Although the agreement was entered into in the third quarter 2015, there were no payments made related to this agreement for the three and nine months ended September 30, 2015.

 

In 2012, the Company entered into a consulting agreement with Dr. Gregory L. Verdine for services in the capacity as a scientific advisor. The consulting agreement does not have a specific term and may be terminated by either party upon 14 days’ prior written notice. Pursuant to the consulting agreement, the Company pays Dr. Verdine $13 thousand per month, plus reimbursement for certain expenses.

 

The Company also has an informal consulting arrangement with Dr. Takeshi Wada for scientific advisory services in the amount of 250 thousand Japanese yen, or approximately $2 thousand, per month, plus reimbursement of certain expenses.

 

 

14

 


9. GEOGRAPHIC DATA

The Company’s long-lived assets consist of property and equipment and are located in the following geographical areas:

 

 

 

September 30, 2016

 

 

December 31, 2015

 

 

 

(in thousands)

 

Asia

 

$

476

 

 

$

578

 

United States

 

 

6,217

 

 

 

2,211

 

Total long-lived assets

 

$

6,693

 

 

$

2,789

 

 

15

 


Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations.

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and in our Annual Report on Form 10-K for the year ended December 31, 2015, filed with the Securities and Exchange Commission (“SEC”) on March 30, 2016 (the “2015 Annual Report on Form 10-K”). Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth under the caption “Risk Factors” in our 2015 Annual Report on Form 10-K, our actual results could differ materially from the results described, in or implied, by these forward-looking statements

Special Note Regarding Forward-Looking Statements

This Quarterly Report on Form 10-Q contains forward-looking statements that involve substantial risks and uncertainties. In some cases, forward-looking statements are identified by the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “future,” “goals,” “intend,” “likely,” “may,” “might,” “ongoing,” “objective,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “strategy,” “will” and “would” or the negative of these terms, or other comparable terminology intended to identify statements about the future. Forward-looking statements include statements about the success, cost and timing of our product development activities and future clinical trials; the timing of and our ability to obtain and maintain regulatory approvals for any of our product candidates; our ability to identify and develop new product candidates; our intellectual property position; our manufacturing and commercialization capabilities and strategy; our use of proceeds from our initial public offering; our estimates regarding future expenses and needs for additional financing; our ability to identify, recruit and retain key personnel; our financial performance; our competitive position; our liquidity and working capital requirements; and the expected impact of new accounting standards. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these statements, including the following: the ability of our preclinical studies to produce data sufficient to support the filing of investigational new drug applications and the timing thereof; our ability to continue to build and maintain the company infrastructure and personnel needed to achieve our goals; the clinical results of our programs, which may not support further development of our product candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; our effectiveness in managing future clinical trials and regulatory processes; the success of our platform in identifying viable candidates; the continued development and acceptance of nucleic acid therapeutics as a class of drugs; our ability to demonstrate the therapeutic benefits of our stereopure candidates in clinical trials, including our ability to develop candidates across multiple therapeutic modalities; our ability to obtain, maintain and protect intellectual property; our ability to enforce our patents against infringers and defend our patent portfolio against challenges from third parties; our ability to raise additional capital as needed; and competition from others developing therapies for similar uses, as well as the information under the caption “Risk Factors” contained in the 2015 Annual Report on Form 10-K filed with the SEC and in other filings we make with the SEC. If our forward-looking statements prove to be inaccurate, the inaccuracy may be material. In light of the significant uncertainties in these forward-looking statements, these statements should not be regarded as representations or warranties by us or any other person that we will achieve our objectives and plans in any specified time frame, or at all. We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

As used in this Quarterly Report on Form 10-Q, unless otherwise stated or the context otherwise indicates, references to “WAVE,” the “Company,” “we,” “our,” “us” or similar terms refer to WAVE Life Sciences Ltd. and our wholly-owned subsidiaries.

Overview

We are a preclinical biotechnology company with an innovative and proprietary synthetic chemistry drug development platform that we are using to design, develop and commercialize a broad pipeline of first-in-class or best-in-class nucleic acid therapeutic candidates. Nucleic acid therapeutics have the potential to address diseases that have been difficult to treat with small molecule drugs or biologics and have emerged as a large and promising class of drugs. We are initially developing nucleic acid therapeutics that target genetic defects to either reduce the expression of disease-promoting proteins or transform the production of dysfunctional mutant proteins into the production of functional proteins. Building upon the innovative work of our scientific founders, Gregory L. Verdine, Ph.D. and Takeshi Wada, Ph.D., our preclinical studies have demonstrated that our stereopure nucleic acid therapeutics may achieve superior drug properties as compared to mixture-based nucleic acid therapeutics. Our platform is designed to enable us to rationally design, optimize and manufacture stereopure nucleic acid therapeutics. Further, our platform has the potential to be used to design therapies that utilize any of the major molecular mechanisms employed by nucleic acid therapeutics, including antisense, ribonucleic acid interference (“RNAi”) and exon skipping.

16

 


We are advancing a diverse pipeline of stereopure nucleic acid medicines across a broad spectrum of rare genetic diseases in multiple therapeutic areas, with a focus on treatments for neurological, neuromuscular and neurosensory disorders. Our most advanced therapeutic programs are in Huntington’s disease (“HD”) and Duchenne muscular dystrophy (“DMD”). In Huntington’s disease, we have lead programs targeting HTT SNP-1 and HTT SNP-2 and in DMD, our lead program targets Exon 51. We expect to file investigational new drug applications (“INDs”) with the U.S. Food and Drug Administration (“FDA”) for HTT SNP-1 and HTT SNP-2 in late 2016 and Exon 51 in mid-2017. In addition to our lead programs, we have identified over 20 potential target indications and we are working towards candidate selection for Amyotrophic Lateral Sclerosis (“ALS”), among other indications.

In May 2016, we entered into a collaboration with Pfizer focused on the advancement of genetically defined targets for the treatment of metabolic diseases, combining our platform together with GalNAc and Pfizer's hepatic targeting technology for enhanced delivery to the liver (the “Pfizer Collaboration Agreement”).  We continue to evaluate other partnering arrangements as part of our strategic plan to forge collaborations with leaders in therapeutic areas outside of our neurology franchise to bring the potential benefits of stereopure medicines to as many patients as possible.

As part of our strategy to bring greater control over the advancement of our pipeline and the execution of our clinical development plans, we entered into a lease agreement for a new facility on September 26, 2016.  Our plans for this new facility are to build out and enable our internal GMP manufacturing capabilities as well as to provide additional laboratory and office space to support our deep pipeline and long-term growth.  Since our inception in 2012, we have devoted substantially all of our resources to developing an innovative and proprietary synthetic chemistry drug development platform that we are using to design, develop and commercialize nucleic acid therapeutic programs, advancing our neurology franchise, expanding our research and development activities as we prepare to enter the clinic, building our intellectual property portfolio, developing our supply chain, planning our business strategy, raising capital and providing general and administrative support for these operations. To date, we have not generated any product revenue and we have primarily financed our operations through sales of our securities.

In November 2015, we completed an initial public offering of our ordinary shares, in which we received aggregate net proceeds, inclusive of the partial exercise of the underwriters’ over-allotment option in December 2015, of approximately $100.4 million.

We have never been profitable, and since our inception, we have incurred significant operating losses. Our net loss was $36.9 million and $12.1 million in the nine months ended September 30, 2016 and 2015, respectively. As of September 30, 2016 and December 31, 2015, we had an accumulated deficit of $72.0 million and $35.1 million, respectively. We expect to incur significant expenses and increasing operating losses for the foreseeable future.

Financial Operations Overview

Revenue

We have not generated any product revenue since our inception and do not expect to generate any revenue from the sale of products for the foreseeable future. Our revenue during the three and nine months ended September 30, 2016 represented revenue earned under the Pfizer Collaboration Agreement that we entered into in May 2016. Our revenue during the nine months ended September 30, 2015 was related to research and development services performed under an agreement that was terminated in May 2015.

Operating Expenses

Our operating expenses since inception have consisted primarily of research and development costs and general and administrative costs.

Research and Development Expenses

Research and development expenses consist primarily of costs incurred for our research activities, including our discovery efforts, and the development of our product candidates, which include:

 

expenses incurred under agreements with third parties, including contract research organizations (“CROs”) that conduct research and preclinical studies on our behalf, as well as contract manufacturing organizations (“CMOs”) that manufacture drug products for use in our preclinical studies;

 

employee salaries, bonuses and other related benefits costs, including share-based compensation expense, for personnel in our research and development organization;

 

costs of third-party consultants, including fees, share-based compensation and related travel expenses;

17

 


 

the cost of sponsored research, which includes laboratory supplies and facility-related expenses, including rent, maintenance and other operating costs; and

 

costs related to compliance with regulatory requirements.

We recognize research and development costs as incurred, which are reflected in our financial statements as prepaid or accrued research and development expenses. We recognize external development costs based on an evaluation of the progress to completion of specific tasks using information provided to us by our vendors. Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected in our financial statements as prepaid or accrued research and development expenses.

Our primary research and development focus since inception has been the development of our innovative and proprietary synthetic chemistry drug development platform. We are using our platform to design, develop and commercialize a broad pipeline of nucleic acid therapeutic candidates.

Our direct research and development expenses are tracked on a program-by-program basis and consist primarily of CROs, consultants, and other external costs incurred in connection with our preclinical studies and regulatory fees. However, we do not allocate the cost of sponsored research on a program-by-program basis, because these costs are deployed across multiple product programs under development and, as such, are classified as costs of our research. The cost of sponsored research includes laboratory supplies, equipment repairs and maintenance and facility-related expenses.

The table below summarizes our research and development expenses incurred on our platform and by program:

 

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

 

2016

 

 

2015

 

 

2016

 

 

2015

 

 

 

(in thousands)

 

HD HTT SNP-1 and HD HTT SNP-2 programs (1)

 

$

3,791

 

 

$

54

 

 

$

6,949

 

 

$

268

 

DMD Exon 51 program

 

 

994

 

 

 

81

 

 

 

1,861

 

 

 

268

 

Other discovery programs, platform development

   and identification of potential drug discovery

   candidates

 

 

8,901

 

 

 

1,997

 

 

 

18,013

 

 

 

5,053

 

Total research and development expenses

 

$

13,686

 

 

$

2,132

 

 

$

26,823

 

 

$

5,589

 

 

(1)

Given the nature of program development for the HD HTT SNP-1 and HD HTT SNP-2 programs, the costs incurred in these programs have been common to both programs and therefore are not separable. We expect that upon the filing of an IND with respect to the lead product candidate in each of these programs and the initiation of clinical trials for each such candidate, the costs incurred for each such candidate will be separate and distinct.

Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect that our expenses related to salaries, bonuses and other related benefits costs will increase in the future as we attract and maintain additional personnel. We expect that our research and development expenses will continue to increase in the foreseeable future as we initiate clinical trials for certain of our product candidates, continue to discover and develop additional product candidates, and pursue later stages of clinical development of our product candidates.

General and Administrative Expenses

General and administrative expenses consist primarily of salaries, bonuses and other related benefits costs, including share-based compensation, for personnel in our executive, finance, corporate, business development, legal and administrative functions. General and administrative expenses also include legal fees relating to intellectual property and general corporate matters; expenses associated with being a public company; professional fees for accounting, auditing, tax and consulting services; insurance costs; travel expenses; other operating costs; and facility-related expenses.

We anticipate that our general and administrative expenses will increase in the future, primarily due to additional compensation, including salaries, benefits, incentive arrangements and share-based compensation awards, as we increase our employee headcount to support the expected growth in our research and development activities and the potential commercialization of our product candidates.

18

 


Other Income (Expense), net

Other income consists primarily of interest income earned on cash and cash equivalents balances for the three and nine months ended September 30, 2016. For the nine months ended September 30, 2015, our other income mainly consisted of reimbursement of research and development costs under a research and development grant awarded by the Japanese Ministry of Economy, Trade and Industry.  

Income Taxes

We are a multi-national company subject to taxation in the United States, Japan and Singapore. During the nine months ended September 30, 2016 and 2015, we recorded a tax provision of $0.4 million and $0.2 million, respectively, which is a result of U.S. income generated under research and management services arrangements between our U.S. and Singapore entities.

Critical Accounting Policies and Significant Judgments and Estimates

Our consolidated financial statements are prepared in accordance with generally accepted accounting principles in the United States of America. The preparation of our financial statements and related disclosures requires us to make estimates, assumptions and judgments that affect the reported amount of assets, liabilities, costs and expenses, revenue, and related disclosures. During the three and nine months ended September 30, 2016, there were no material changes to our critical accounting policies, except for our revenue recognition policy as it relates to the Pfizer Agreements, which we entered into in May 2016, discussed above.

Our critical accounting policies are described under the caption “Management’s Discussion and Analysis of Financial Condition and Results of Operations— Critical Accounting Policies and Significant Judgments and Estimates” in our 2015 Annual Report on Form 10-K. We believe that of our critical accounting policies, the accounting policies with respect to income taxes, share-based compensation and revenue recognition involve the most judgment and complexity.

Accordingly, we believe these identified policies are critical to fully understanding and evaluating our financial condition and results of operations. If actual results or events differ materially from the estimates, judgments and assumptions used by us in applying these policies, our reported financial condition and results of operations could be materially affected.

Revenue Recognition

As of September 30, 2016, our only significant source of revenue related to the Pfizer Collaboration Agreement, which we entered into in May 2016, related to the discovery, development and commercialization of stereopure oligonucleotide therapeutics for up to five programs, each directed at a genetically-defined hepatic target selected by Pfizer (see Note 4 of the accompanying Notes to the Unaudited Condensed Consolidated Financial Statements).

We present revenue from the Pfizer Collaboration Agreement under Financial Accounting Standards Board (“FASB”), Accounting Standards Codification (“ASC”), Topic 808, Collaborative Arrangements.  We recognize revenue in accordance with ASC Topic 605, Revenue Recognition (“ASC 605”). Accordingly, revenue is recognized for each unit of accounting when all of the following criteria are met:

 

persuasive evidence of an arrangement exists;

 

delivery has occurred or services have been rendered;

 

the seller’s price to the buyer is fixed or determinable; and

 

collectability is reasonably assured.

Amounts received prior to satisfying the revenue recognition criteria are recorded as deferred revenue in our consolidated balance sheets.

Multiple Element Arrangements

We analyze multiple element arrangements based on the guidance in ASC Topic 605‑25, Revenue Recognition—Multiple Element Arrangements (“ASC 605‑25”). Pursuant to the guidance in ASC 605‑25, we evaluate multiple element arrangements to determine (1) the deliverables included in the arrangement and (2) whether the individual deliverables represent separate units of accounting or whether they must be accounted for as a combined unit of accounting. This evaluation involves subjective determinations and requires management to make judgments about the individual deliverables and whether such deliverables are separable from the other aspects of the contractual relationship. Deliverables are considered separate units of accounting provided that: (i) the delivered item(s) has value to the customer on a standalone basis and (ii) if the arrangement includes a general right of return relative to the delivered

19

 


item(s), delivery or performance of the undelivered item(s) is considered probable and substantially within our control. In assessing whether an item has standalone value, we consider factors such as the research, development, manufacturing and commercialization capabilities of the collaboration partner and the availability of the associated expertise in the general marketplace. We also consider whether our collaboration partner can use a deliverable for its intended purpose without the receipt of the remaining deliverable(s), whether the value of the deliverable is dependent on the undelivered item(s), and whether there are other vendors that can provide the undelivered item(s).

Options under a collaboration agreement are considered substantive if, at the inception of the arrangement, we are at risk as to whether the collaboration partner will choose to exercise the option. Factors that we consider in evaluating whether an option is substantive include the cost to exercise the option, the overall objective of the arrangement, the benefit the collaboration partner might obtain from the arrangement without exercising the option, and the likelihood the option will be exercised. When an option is considered substantive, we would not consider the option or item underlying the option to be a deliverable at the inception of the arrangement and the associated option fees are not included in allocable consideration, assuming the option is not priced at a significant and incremental discount. Conversely, when an option is not considered substantive, we would consider the option, including other deliverables contingent upon the exercise of the option, to be a deliverable at the inception of the arrangement and a corresponding amount would be included in allocable arrangement consideration. In addition, if the price of the option includes a significant incremental discount, the discount would be included as a deliverable at the inception of the arrangement.

Allocation of Arrangement Consideration

Arrangement consideration that is fixed or determinable is allocated among the separate units of accounting using the relative selling price method. The applicable revenue recognition criteria in ASC 605 are applied to each of the separate units of accounting in determining the appropriate period and pattern of recognition.

Pattern of Recognition

We recognize arrangement consideration allocated to each unit of accounting when all of the revenue recognition criteria in ASC 605 are satisfied for that particular unit of accounting. In the event that a deliverable does not represent a separate unit of accounting, we recognize revenue from the combined unit of accounting over our contractual or estimated performance period for the undelivered elements, which is typically the term of our research and development obligations. If there is no discernible pattern of performance or objectively measurable performance measures do not exist, then we recognize revenue under the arrangement on a straight‑line basis over the period we are expected to complete our performance obligations. Conversely, if the pattern of performance in which the service is provided to the customer can be determined and objectively measurable performance measures exist, then we recognize revenue under the arrangement using the proportional performance method. Revenue recognized is limited to the lesser of the cumulative amount of payments received or the cumulative amount of revenue earned, as determined using the straight‑line method or proportional performance method, as applicable, as of the period ending date.

We have concluded that the deliverables under the Pfizer Collaboration Agreement relate primarily to the research and development required by us for the programs nominated by Pfizer.  The remaining deliverables, including sample supplies provided by us to fulfill our obligation as a licensee, participation on a joint steering committee to oversee the research and development activities, and regulatory responsibilities related to filings and obtaining approvals related to the products that may result from each program do not represent separate units of accounting based on their dependence on the research and development efforts.

Because there is no discernible pattern of performance given the nature of the research and development efforts, we recognize the revenue under the Pfizer Collaboration Agreement on a straight‑line basis over the period we are expected to complete our performance obligations.

Recognition of Milestones

At the inception of an arrangement that includes milestone payments, we evaluate whether each milestone is substantive and at risk to both parties on the basis of the contingent nature of the milestone. This evaluation includes an assessment of whether: (1) the consideration is commensurate with either our performance to achieve the milestone or the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from its performance to achieve the milestone, (2) the consideration relates solely to past performance and (3) the consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. We evaluate factors such as the scientific, clinical, regulatory, commercial, and other risks that must be overcome to achieve the particular milestone and the level of effort and investment required to achieve the particular milestone in making this assessment. There is considerable judgment involved in determining whether a milestone satisfies all of the criteria required to conclude that a milestone is substantive. Milestones that are not considered substantive are recognized as earned if there are no remaining performance obligations over the remaining period of performance, assuming all other revenue recognition criteria are met.

20

 


Results of Operations

Comparison of the three months ended September 30, 2016 and 2015:

 

 

 

Three Months Ended September 30,

 

 

 

 

 

 

 

2016

 

 

2015

 

 

Increase

(Decrease)

 

 

 

(in thousands)

 

Revenues

 

$

392

 

 

 

 

 

$

392

 

Operating expenses

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

 

13,686

 

 

 

2,132

 

 

 

11,554

 

General and administrative

 

 

3,939

 

 

 

2,858

 

 

 

1,081

 

Total operating expense

 

 

17,625

 

 

 

4,990

 

 

 

12,635

 

Loss from operations

 

 

(17,233

)

 

 

(4,990

)

 

 

(12,243

)

Other income (expense), net

 

 

82

 

 

 

137

 

 

 

(55

)

Loss before income taxes

 

 

(17,151

)

 

 

(4,853

)

 

 

(12,298

)

Income tax benefit (provision)

 

 

(384

)

 

 

(83

)

 

 

(301

)

Net loss

 

$

(17,535

)

 

$

(4,936

)

 

$

(12,599

)

 

Revenue

There was $0.4 million in revenue for the three months ended September 30, 2016, and there was no revenue for the three months ended September 30, 2015. The $0.4 million of revenue for the three months ended September 30, 2016 was earned under the Pfizer Collaboration Agreement, which was entered into in May 2016.

Research and Development Expenses

 

 

 

Three Months Ended September 30,

 

 

 

 

 

 

 

2016

 

 

2015

 

 

Increase

 

 

 

(in thousands)

 

HD HTT SNP-1 and HD HTT SNP-2 programs (1)

 

$

3,791

 

 

$

54

 

 

$

3,737

 

DMD Exon 51 program

 

 

994

 

 

 

81

 

 

 

913

 

Other discovery programs, platform development and

   identification of potential drug discovery candidates

 

 

8,901

 

 

 

1,997

 

 

 

6,904

 

Total research and development expenses

 

$

13,686

 

 

$

2,132

 

 

$

11,554

 

 

(1)

Given the nature of program development for the HD HTT SNP-1 and HD HTT SNP-2 programs, the costs incurred in these programs have been common to both programs and therefore are not separable. We expect that upon the filing of an IND with respect to the lead product candidate in each of these programs and the initiation of clinical trials for each such candidate, the costs incurred for each such candidate will be separate and distinct.

Research and development expenses were $13.7 million for the three months ended September 30, 2016, compared to $2.1 million for the three months ended September 30, 2015. The increase of $11.6 million was due primarily to the following:

 

an increase of $3.7 million in research and development expenses related to our HD HTT SNP-1 and HD HTT SNP-2 programs under our collaboration with Children’s Hospital of Philadelphia and other organizations for preclinical research studies and development expenses for IND-enabling activities related to these programs;

 

an increase of $0.9 million in research and development expenses related to our DMD Exon 51 program for collaborations with the University of Oxford and other organizations for preclinical research studies; and

 

an increase of $6.9 million in research and development expenses related to other discovery programs, platform development and identification of potential drug discovery candidates, due to an increase of $2.4 million in salary, bonus and related benefits costs and an increase of $1.2 million in share-based compensation expense, both of which are the result of an increase in employee headcount, and an increase of $3.3 million in research and development supplies and services expenses and facility-related expenses.

21

 


General and Administrative Expenses

General and administrative expenses were $3.9 million for the three months ended September 30, 2016, compared to $2.9 million for the three months ended September 30, 2015. The increase of approximately $1.0 million was primarily due to the increase in salary and related benefits and share-based compensation, which are the result of an increase in employee headcount.  

Foreign currency translation did not have a significant impact on changes in our consolidated general and administrative expenses from the three months ended September 30, 2015 to the three months ended September 30, 2016.

Income Tax Benefit (Provision)

During the three months ended September 30, 2016 and 2015, we recorded a tax provision of $0.4 million and $0.1 million, respectively, which is a result of U.S. income generated under research and management services arrangements between our U.S. and Singapore entities. During the three months ended September 30, 2016 and 2015, we recorded no income tax benefits for the net operating losses incurred in Japan and Singapore, due to uncertainty regarding future taxable income in these jurisdictions.

Comparison of the nine months ended September 30, 2016 and 2015:

The following table summarizes our results of operations for the nine months ended September 30, 2016 and 2015:

 

 

 

Nine Months Ended September 30,

 

 

 

 

 

 

 

2016

 

 

2015

 

 

Increase

(Decrease)

 

 

 

(in thousands)

 

Revenues

 

$

809

 

 

 

152

 

 

$

657

 

Operating expenses

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

 

26,823

 

 

 

5,589

 

 

 

21,234

 

General and administrative

 

 

10,809

 

 

 

6,647

 

 

 

4,162

 

Total operating expense

 

 

37,632

 

 

 

12,236

 

 

 

25,396

 

Loss from operations

 

 

(36,823

)

 

 

(12,084

)

 

 

(24,739

)

Other income (expense), net

 

 

303

 

 

 

165

 

 

 

138

 

Loss before income taxes

 

 

(36,520

)

 

 

(11,919

)

 

 

(24,601

)

Income tax benefit (provision)

 

 

(427

)

 

 

(182

)

 

 

(245

)

Net loss

 

$

(36,947

)

 

$

(12,101

)

 

$

(24,846

)

 

Revenue

There was $0.8 million in revenue for the nine months ended September 30, 2016, which represents an increase of approximately $0.6 million in revenue over the $0.2 million of revenue for the nine months ended September 30, 2015. The $0.8 million of revenue for the nine months ended September 30, 2016 was earned under the Pfizer Collaboration Agreement, which we entered into in May 2016. The $0.2 million of revenue for the nine months ended September 30, 2015 was earned for research and development performed under a different collaboration agreement, which was entered into in 2014 and which was terminated in May 2015.

22

 


Research and Development Expenses

The table below summarizes our research and development expenses incurred for the nine months ended September 30, 2016 and 2015:

 

 

 

Nine Months Ended September 30,

 

 

 

 

 

 

 

2016

 

 

2015

 

 

Increase

 

 

 

(in thousands)

 

HD HTT SNP-1 and HD HTT SNP-2 programs (1)

 

$

6,949

 

 

$

268

 

 

$

6,681

 

DMD Exon 51 program

 

 

1,861

 

 

 

268

 

 

 

1,593

 

Other discovery programs, platform development and

   identification of potential drug discovery candidates

 

 

18,013

 

 

 

5,053

 

 

 

12,960

 

Total research and development expenses

 

$

26,823

 

 

$

5,589

 

 

$

21,234

 

 

(1)

Given the nature of program development for the HD HTT SNP-1 and HD HTT SNP-2 programs, the costs incurred in these programs have been common to both programs and therefore are not separable. We expect that upon the filing of an IND with respect to the lead product candidate in each of these programs and the initiation of clinical trials for each such candidate, the costs incurred for each such candidate will be separate and distinct.

Research and development expenses were $26.8 million for the nine months ended September 30, 2016 compared to $5.6 million for the nine months ended September 30, 2015. The increase of $21.2 million was due primarily to the following:  

 

an increase of $6.7 million in research and development expenses related to our HD HTT SNP-1 and HD HTT SNP-2 programs under our collaboration with Children’s Hospital of Philadelphia and other organizations for preclinical research studies and development expenses for IND-enabling activities related to these programs;

 

an increase of $1.6 million in research and development expenses related to our DMD Exon 51 program for collaborations with the University of Oxford and other organizations for preclinical research studies; and

 

an increase of $13.0 million in research and development expenses related to other discovery programs, platform development and identification of potential drug discovery candidates, due to an increase  of $5.3 million in salary, bonus and related benefits costs and an increase of $1.6 million in share-based compensation expense, both of which are the result of an increase in employee headcount, and an increase of $6.1 million in research and development supplies and services expenses and facility-related expenses.

Foreign currency translation did not have a significant impact on changes in our consolidated research and development expenses from the nine months ended September 30, 2015 to the nine months ended September 30, 2016.

General and Administrative Expenses

General and administrative expenses were $10.8 million for the nine months ended September 30, 2016, compared to $6.6 million for the nine months ended September 30, 2015. The increase of approximately $4.2 million was primarily due to the increase in salary, bonus and related benefits costs due to the increase in employee headcount.

Foreign currency translation did not have a significant impact on changes in our consolidated general and administrative expenses from the nine months ended September 30, 2015 to the nine months ended September 30, 2016.

Income Tax Benefit (Provision)

During the nine months ended September 30, 2016 and 2015, we recorded a tax provision of $0.4 million and $0.2 million, respectively, which is a result of U.S. income generated under research and management services arrangements between our U.S. and Singapore entities. During the nine months ended September 30, 2016 and 2015, we recorded no income tax benefits for the net operating losses incurred in Japan and Singapore, due to uncertainty regarding future taxable income in these jurisdictions.

Liquidity and Capital Resources

On November 16, 2015, we completed an initial public offering of our ordinary shares, in which we issued and sold 6,375,000 ordinary shares at a price to the public of $16.00 per share. On December 4, 2015, we issued an additional 618,126 ordinary shares at a price of $16.00 per share pursuant to a partial exercise of the underwriters’ over-allotment option. The aggregate net proceeds to us from our initial public offering, inclusive of the partial over-allotment exercise, were approximately $100.4 million after deducting underwriting discounts and commissions and offering expenses payable by us. Prior to the completion of our initial public offering,

23

 


we financed our operations through private placements of our debt and equity securities, which resulted in net proceeds of $89.3 million from such transactions.

On May 5, 2016, we entered into a Research, License and Option Agreement (the “Pfizer Collaboration Agreement”) with Pfizer Inc. (“Pfizer”) and a Share Purchase Agreement (the “Pfizer Equity Agreement” and together with the Pfizer Collaboration Agreement, the “Pfizer Agreements”) with an affiliate of Pfizer. Pursuant to the Pfizer Agreements, Pfizer paid us $40.0 million upfront, including $10.0 million as an upfront license fee and $30.0 million in the form of an equity investment in which we sold 1,875,000 of our ordinary shares to an affiliate of Pfizer.

Since our inception, we have not generated any product revenue and have incurred recurring net losses.

As of September 30, 2016, we had cash and cash equivalents totaling $169.0 million and an accumulated deficit of $72.0 million and restricted cash of $1.1 million related primarily to a letter of credit for our office and laboratory space in Cambridge, Massachusetts.

We expect that the cash resources we had on hand at September 30, 2016 will fund our operating expenses and capital expenditure requirements into 2019. We have based this estimate on assumptions that may prove to be incorrect, and we may use our available capital resources sooner than we currently expect. In addition, we may elect to raise additional funds before we need them if the conditions for raising capital are favorable due to market conditions or strategic considerations even if we believe we have sufficient funds for our current or future operating plans.

Until we can generate significant revenue from product sales, if ever, we expect to finance our operations through a combination of public or private equity or debt financings or other sources, which may include collaborations with third parties. Adequate additional financing may not be available to us on acceptable terms, or at all. Our inability to raise capital as and when needed would have a negative impact on our financial condition and our ability to pursue our business strategy. We will need to generate significant revenue to achieve profitability, and we may never do so.

Cash Flows

The following table summarizes our sources and uses of cash and cash equivalents for the nine months ended September 30, 2016 and 2015:

 

 

 

Nine Months Ended September 30,

 

 

 

2016

 

 

2015

 

 

 

(in thousands)

 

Cash used in operating activities

 

$

(18,578

)

 

$

(5,859

)

Cash used in investing activities

 

 

(2,834

)

 

 

(1,939

)

Cash provided by financing activities

 

 

29,106

 

 

 

72,857

 

Effect of foreign exchange rates of cash

 

 

101

 

 

 

(67

)

Net increase (decrease) in cash and cash equivalents

 

$

7,795

 

 

$

64,992

 

 

Operating Activities

During the nine months ended September 30, 2016, operating activities used approximately $18.6 million of cash, which was the result of our net loss of $36.9 million, offset by changes in operating assets and liabilities of $13.0 million and non-cash charges of $5.3 million. The non-cash charges were related primarily to share-based compensation of $4.3 million. The cash provided from changes in operating assets and liabilities was driven by the $11.7 million increase in deferred revenue which was the result of the upfront payment received and the receivable recorded related to the Pfizer Collaboration Agreement.

During the nine months ended September 30, 2015, operating activities used $5.9 million of cash, which was the result of our net loss of $12.1 million, offset by non-cash charges of $3.9 million and changes in operating assets and liabilities of $2.4 million. The non-cash charges were related primarily to share-based compensation of $3.2 million. The changes in operating assets and liabilities were mainly driven by the $2.5 million of changes in accounts payable.

Investing Activities

During the nine months ended September 30, 2016, investing activities used $2.8 million of cash, consisting primarily of purchases of property and equipment.  

24

 


During the nine months ended September 30, 2015, investing activities used $1.9 million of cash, consisting of restricted cash of $1.0 million primarily placed in favor of a letter of credit for our office and laboratory space in Cambridge, Massachusetts, along with purchases of property and equipment of $0.9 million.

Financing Activities

During the nine months ended September 30, 2016, net cash provided by financing activities was $29.1 million, which was primarily due to the $30.0 million in proceeds from the issuance of 1,875,000 shares to an affiliate of Pfizer related to the Pfizer Equity Agreement.

During the nine months ended September 30, 2015, net cash provided by financing activities was $72.9 million, primarily from the issuance of ordinary shares to investors during the period.

Effect of Foreign Exchange Rates on Cash

During the nine months ended September 30, 2016, the effect of changes in foreign exchange rates on cash was $0.1 million, primarily due to changes in the Japanese yen from December 31, 2015 to September 30, 2016.

During the nine months ended September 30, 2015, the effect of changes in foreign exchange rates on cash was less than $0.1 million, primarily due to changes in the Japanese yen from December 31, 2014 to September 30, 2015.

Funding Requirements

We expect our expenses to increase substantially in connection with our ongoing research and development activities. In addition, we expect to incur additional costs associated with operating as a public company. We anticipate that our expenses will increase substantially if and as we:

 

file INDs and initiate clinical trials for our programs in Huntington’s disease and DMD;

 

conduct research and continue preclinical development of discovery targets and other future potential pipeline candidates;

 

make strategic investments in manufacturing processes and formulations;

 

develop manufacturing capabilities through building out our internal manufacturing capabilities, outsourcing and potentially building a scalable manufacturing facility;

 

maintain our intellectual property portfolio and consider the acquisition of complementary intellectual property; and

 

seek regulatory approvals for our product candidates.

We may experience delays or encounter issues with any of the above, including but not limited to failed studies, complex results, safety issues or other regulatory challenges.

Because of the numerous risks and uncertainties associated with the development of drug candidates or follow-on programs and because the extent to which we may enter into collaborations with third parties for development of product candidates is unknown, we are unable to estimate the amounts of increased capital outlays and operating expenses associated with completing the research and development for our therapeutic programs. Our future capital requirements for our therapeutic programs will depend on many factors, including:

 

the progress and results of conducting research and continued preclinical development within our therapeutic programs and with respect to future potential pipeline candidates;

 

the cost of manufacturing clinical supplies of our product candidates;

 

the costs, timing and outcome of regulatory review of our product candidates;

 

the costs and timing of future commercialization activities, including manufacturing, marketing, sales and distribution, for any of our product candidates for which we receive marketing approval;

 

the revenue, if any, received from commercial sales of our product candidates for which we receive marketing approval;

 

the costs and timing of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending any intellectual property-related claims;

25

 


 

the effect of competing technological and market developments; and

 

the extent to which we acquire or invest in businesses, products and technologies, including entering into licensing or collaboration arrangements for product candidates, including the Pfizer Collaboration Agreement.

Identifying potential product candidates and conducting preclinical studies and clinical trials is a time-consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval and achieve product sales. In addition, our product candidates, if approved, may not achieve commercial success. Our product revenue, if any, will be derived from sales of products that we do not expect to be commercially available for many years, if ever. Accordingly, we will need to obtain substantial additional funds to achieve our business objectives.

Adequate additional funds may not be available to us on acceptable terms, or at all. We do not currently have any committed external source of funds, except for the aforementioned Pfizer Collaboration Agreement which was entered into on May 5, 2016. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing shareholders will be diluted, and the terms may include liquidation or other preferences that adversely affect the rights of our shareholders. Additional debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends and may require the issuance of warrants, which could potentially dilute our shareholders’ ownership interests.

If we raise additional funds through collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development programs or any future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Contractual Obligations and Commitments

There have been no material changes to our contractual obligations and commitments set forth under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations- Contractual Obligations and Commitments” in our 2015 Annual Report on Form 10-K, except as disclosed below.

On September 26, 2016, our wholly-owned subsidiary, WAVE Life Sciences USA, Inc., entered into a Lease Agreement (the “Lease”) with King 115 Hartwell LLC, primarily to build out and conduct certain GMP manufacturing activities, as well as to provide additional laboratory and office space to support our growth.  The Lease obligates us to pay an average base rent of approximately $2.8 million for the 10-year and 9-month initial term.  In addition, throughout the term of the Lease, we are responsible for paying certain costs and expenses, in addition to the rent, as specified in the Lease, including a proportionate share of applicable taxes, operating expenses, and utilities.

Off-Balance Sheet Arrangements

We had no off-balance sheet arrangements (as that term is defined in Item 303(a)(4)(ii) of Regulation S-K) as of  September 30, 2016 that had or were reasonably likely to have a current or future effect on our financial condition, changes in financial condition, revenues or expenses, results of operations, liquidity, capital expenditures or capital resources.

Recently Issued Accounting Pronouncements

For detailed information regarding recently issued accounting pronouncements and the expected impact on our condensed consolidated financial statements, see Note 2, “Significant Accounting Policies” in the notes to the condensed consolidated financial statements appearing elsewhere in this Quarterly Report on Form 10-Q.

Item 3. Quantitative and Qualitative Disclosures about Market Risk.

Market risk represents the risk of loss that may impact our financial position due to adverse changes in financial market prices and rates. Our market risk exposure is primarily the result of fluctuations in interest rates and foreign exchange rates as well as, to a lesser extent, inflation, and capital market risk.

26

 


Interest Rate Risk

We are exposed to interest rate risk in the ordinary course of our business. Our cash and cash equivalents are held in readily available checking and money market accounts.

Foreign Currency Risk

We are exposed to market risk related to changes in the value of the Japanese yen, which is the currency in which our Japanese subsidiary conducts its business. As of September 30, 2016 and December 31, 2015, 0.5% and 0.5% of our assets, respectively, were located in Japan. Additionally, 0.5% and 6.4% of our general and administrative expenses were transacted in Japanese yen during the nine months ended September 30, 2016 and 2015, respectively. Furthermore, 2.1%, and 7.5% of our research and development expenses were transacted in Japanese yen during nine months ended September 30, 2016 and 2015, respectively. When the U.S. dollar strengthens relative to the yen, our U.S. dollar reported revenue and expense from non-U.S. dollar denominated income and operating costs will decrease. Conversely, when the U.S. dollar weakens relative to the yen, our U.S. dollar reported revenue and expenses from non-U.S. dollar denominated income and operating costs will increase. Changes in the relative values of currencies occur regularly and, in some instances, could materially adversely affect our business, results of operations, financial condition or cash flows. Our foreign currency sensitivity is affected by changes in the Japanese yen, which is impacted by economic factors both locally in Japan and worldwide. A hypothetical 10% change in foreign currency rates would not have a material impact on our historical financial position or results of operations.

Inflation Risk

We do not believe that inflation had a material effect on our business, financial condition or results of operations for the three and nine months ended September 30, 2016 and 2015.

Capital Market Risk

We currently have no product revenues and depend on funds raised through other sources. One possible source of funding is through further equity offerings. Our ability to raise funds in this manner depends in part upon capital market forces affecting our share price.

Item 4. Controls and Procedures.

Evaluation of Disclosure Controls and Procedures

Our management, with the participation of our principal executive officer and principal financial officer, evaluated the effectiveness of our disclosure controls and procedures as of September 30, 2016. The term “disclosure controls and procedures,” as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended, or the Exchange Act, means controls and other procedures of a company that are designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is recorded, processed, summarized and reported, within the time periods specified in the SEC’s rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is accumulated and communicated to its management, including its principal executive and principal financial officers, as appropriate to allow timely decisions regarding required disclosure. Management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Based on the evaluation of our disclosure controls and procedures as of September 30, 2016, our principal executive officer and principal financial officer concluded that, as of such date, our disclosure controls and procedures were not effective at the reasonable assurance level, due to the material weakness described below.

Material Weakness and Remediation of Material Weakness

The management of the company is responsible for establishing and maintaining adequate internal control over financial reporting as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act. In connection with the audit of our consolidated financial statements for the years ended December 31, 2015, 2014 and 2013, we and our independent registered public accounting firm identified a material weakness in our internal control over financial reporting. A material weakness is a deficiency, or a combination of deficiencies, in internal control over financial reporting, such that there is a reasonable possibility that a material misstatement of our annual or interim financial statements will not be prevented or detected on a timely basis.

Prior to the completion of our initial public offering, we were a private company and had limited accounting and financial reporting personnel and other resources with which to address our internal controls and procedures. Our lack of adequate accounting personnel resulted in the identification of a material weakness in our internal control over financial reporting. Specifically, we did not

27

 


appropriately design and implement controls over the review and approval of manual journal entries and the related supporting journal entry calculations.

We have made progress toward implementing a remediation plan to address the material weakness described above. We have hired and intend to hire additional accounting and finance personnel and we have implemented an enterprise resource system. Moreover, we have been working with an external consultant to strengthen and better document our internal controls. We expect that these efforts will result in a more robust review process and increase the supervision and monitoring of our financial reporting process.

Changes in Internal Control over Financial Reporting

Other than as described above, there were no changes in our internal control over financial reporting identified in connection with the evaluation required by Rule 13a-15(d) and 15d-15(d) of the Exchange Act that occurred during the fiscal quarter ended September 30, 2016 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

 

 

PART II – OTHER INFORMATION

Item 1. Legal Proceedings.

We are not currently a party to any material legal proceedings.

Item 1A. Risk Factors.

In addition to the other information set forth in this Quarterly Report on Form 10-Q, you should carefully consider the factors discussed under the caption “Risk Factors” in our 2015 Annual Report on Form 10-K, which could materially affect our business, financial condition or results of operations.  There have been no material changes in or additions to the risk factors included in our 2015 Annual Report on Form 10-K.

Item 2. Unregistered Sales of Equity Securities and Use of Proceeds.

(a)

Recent Sales of Unregistered Equity Securities

None.

(b)

Use of Proceeds

On November 10, 2015, the SEC declared our registration statement on Form S-1 (Registration No. 333-207379) effective for our initial public offering. There has been no material change in the planned use of proceeds from our initial public offering as described in our final prospectus filed with the SEC on November 12, 2015 pursuant to Rule 424(b). We have been using and will continue to use the net offering proceeds to advance our product candidates through clinical trial programs and for working capital and general corporate purposes.

Item 3. Defaults Upon Senior Securities.

None.

Item 4. Mine Safety Disclosures.

Not applicable.

Item 5. Other Information.

Not applicable

Item 6. Exhibits.

The exhibits listed in the Exhibit Index to this Quarterly Report on Form 10-Q are incorporated herein by reference.

 

 

28

 


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.

Date: November 9, 2016

 

WAVE LIFE SCIENCES LTD.

 

 

 

By:

 

/s/ Paul B. Bolno, M.D.

 

 

Paul B. Bolno, M.D.

 

 

President and Chief Executive Officer

 

 

(Principal Executive Officer)

 

 

 

By:

 

/s/ Keith C. Regnante

 

 

Keith C. Regnante