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EX-32.2 - EX-32.2 - IDERA PHARMACEUTICALS, INC.idra-20160930ex322fcc215.htm
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EX-31.2 - EX-31.2 - IDERA PHARMACEUTICALS, INC.idra-20160930ex312241f17.htm
EX-31.1 - EX-31.1 - IDERA PHARMACEUTICALS, INC.idra-20160930ex311c31620.htm

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 


 

FORM 10-Q

 


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

 

For the quarterly period ended September 30, 2016

 

or

 

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

 

For transition period from                      to                     .  

 

Commission File Number: 001-31918

 


 

IDERA PHARMACEUTICALS, INC.

(Exact name of registrant as specified in its charter)

 


 

 

 

 

Delaware

    

04-3072298

(State or other jurisdiction of

incorporation or organization)

 

(I.R.S. Employer

Identification No.)

 

 

 

167 Sidney Street

Cambridge, Massachusetts

(Address of principal executive offices)

 

02139

(Zip code)

 

(617) 679-5500

(Registrant’s telephone number, including area code)

 


 

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. (Check one):

 

 

 

 

 

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  ☒

 

 

 

 

Common Stock, par value $.001 per share

    

147,653,120

Class

 

Outstanding as of October 28, 2016

 

 

 


 

IDERA PHARMACEUTICALS, INC.

FORM 10-Q

INDEX

 

 

 

 

 

    

Page

PART I — FINANCIAL INFORMATION 

 

 

Item 1 — Financial Statements — Unaudited 

 

Condensed Balance Sheets as of September 30, 2016 and December 31, 2015 

 

Condensed Statements of Operations and Comprehensive Loss for the Three and Nine Months Ended September 30, 2016 and 2015 

 

Condensed Statements of Cash Flows for the Nine Months Ended September 30, 2016 and 2015 

 

Notes to Condensed Financial Statements 

 

 

 

 

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

 

14 

Item 3 — Quantitative and Qualitative Disclosures About Market Risk 

 

25 

Item 4 — Controls and Procedures 

 

25 

 

 

 

PART II — OTHER INFORMATION 

 

 

Item 1A — Risk Factors 

 

26 

Item 6 — Exhibits 

 

50 

Signatures 

 

51 

 

IMO® and Idera® are our trademarks. All other trademarks and service marks appearing in this Quarterly Report on Form 10-Q are the property of their respective owners.

 


 

FORWARD-LOOKING STATEMENTS

 

This Quarterly Report on Form 10-Q contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, or the Exchange Act. All statements, other than statements of historical fact, included or incorporated in this report regarding our strategy, future operations, clinical trials, collaborations, intellectual property, cash resources, financial position, future revenues, projected costs, prospects, plans, and objectives of management are forward-looking statements. The words “believes,” “anticipates,” “estimates,” “plans,” “expects,” “intends,” “may,” “could,” “should,” “potential,” “likely,” “projects,” “continue,” “will,” and “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We cannot guarantee that we actually will achieve the plans, intentions or expectations disclosed in our forward-looking statements and you should not place undue reliance on our forward-looking statements. There are a number of important factors that could cause our actual results to differ materially from those indicated or implied by forward-looking statements. These important factors include those set forth below under Part II, Item 1A “Risk Factors.” These factors and the other cautionary statements made in this Quarterly Report on Form 10-Q should be read as being applicable to all related forward-looking statements whenever they appear in this Quarterly Report on Form 10-Q. In addition, any forward-looking statements represent our estimates only as of the date that this Quarterly Report on Form 10-Q is filed with the Securities and Exchange Commission, or the SEC, and should not be relied upon as representing our estimates as of any subsequent date. We do not assume any obligation to update any forward-looking statements. We disclaim any intention or obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

 

 

 

iii


 

PART I — FINANCIAL INFORMATION

 

ITEM 1. FINANCIAL STATEMENTS.

 

IDERA PHARMACEUTICALS, INC.

 

CONDENSED BALANCE SHEETS

(UNAUDITED)

 

 

 

 

 

 

 

 

 

 

 

    

September 30, 

    

December 31, 

 

(In thousands, except per share amounts)

 

2016

 

2015

 

ASSETS

 

 

 

 

 

 

 

Current assets:

 

 

 

 

 

 

 

Cash and cash equivalents

 

$

22,154

 

$

26,586

 

Short-term investments

 

 

29,856

 

 

33,574

 

Prepaid expenses and other current assets

 

 

3,455

 

 

3,082

 

Total current assets

 

 

55,465

 

 

63,242

 

Long-term investments

 

 

1,408

 

 

26,997

 

Property and equipment, net

 

 

1,579

 

 

1,692

 

Restricted cash and other assets

 

 

26

 

 

345

 

Total assets

 

$

58,478

 

$

92,276

 

 

 

 

 

 

 

 

 

LIABILITIES AND STOCKHOLDERS’ EQUITY

 

 

 

 

 

 

 

Current liabilities:

 

 

 

 

 

 

 

Accounts payable

 

$

344

 

$

1,169

 

Accrued expenses

 

 

6,118

 

 

4,274

 

Current portion of note payable

 

 

284

 

 

261

 

Current portion of deferred revenue

 

 

1,111

 

 

1,111

 

Total current liabilities

 

 

7,857

 

 

6,815

 

Deferred revenue, net of current portion

 

 

429

 

 

1,262

 

Note payable, net of current portion

 

 

285

 

 

501

 

Other liabilities

 

 

35

 

 

116

 

Total liabilities

 

 

8,606

 

 

8,694

 

Commitments and contingencies

 

 

 

 

 

 

 

Stockholders’ equity:

 

 

 

 

 

 

 

Preferred stock, $0.01 par value, Authorized — 5,000 shares:

 

 

 

 

 

 

 

  Series A convertible preferred stock; Designated — 1,500 shares, Issued and outstanding — 1 share

 

 

 —

 

 

 —

 

Common stock, $0.001 par value, Authorized — 280,000 shares; Issued and outstanding — 121,411 and 121,265 shares at September 30, 2016 and December 31, 2015, respectively

 

 

121

 

 

121

 

Additional paid-in capital

 

 

589,044

 

 

583,676

 

Accumulated deficit

 

 

(539,292)

 

 

(500,081)

 

Accumulated other comprehensive income (loss)

 

 

(1)

 

 

(134)

 

Total stockholders’ equity

 

 

49,872

 

 

83,582

 

Total liabilities and stockholders’ equity

 

$

58,478

 

$

92,276

 

 

The accompanying notes are an integral part of these financial statements.

1


 

IDERA PHARMACEUTICALS, INC.

 

CONDENSED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(UNAUDITED)

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Three Months Ended

 

Nine Months Ended

 

 

 

September 30, 

 

September 30, 

 

(In thousands, except per share amounts)

    

2016

    

2015

    

2016

    

2015

 

Alliance revenue

 

$

323

 

$

20

 

$

918

 

$

59

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

 

9,393

 

 

7,454

 

 

28,817

 

 

25,134

 

General and administrative

 

 

3,907

 

 

4,030

 

 

11,601

 

 

11,688

 

Total operating expenses

 

 

13,300

 

 

11,484

 

 

40,418

 

 

36,822

 

Loss from operations

 

 

(12,977)

 

 

(11,464)

 

 

(39,500)

 

 

(36,763)

 

Other income (expense):

 

 

 

 

 

 

 

 

 

 

 

 

 

Interest income

 

 

90

 

 

123

 

 

320

 

 

239

 

Interest expense

 

 

(19)

 

 

(27)

 

 

(63)

 

 

(81)

 

Foreign currency exchange gain (loss)

 

 

3

 

 

3

 

 

32

 

 

40

 

Net loss

 

$

(12,903)

 

 

(11,365)

 

 

(39,211)

 

 

(36,565)

 

Basic and diluted net loss per common share (Note 13)

 

$

(0.10)

 

$

(0.10)

 

$

(0.32)

 

$

(0.32)

 

Shares used in computing basic and diluted net loss per common share

 

 

121,389

 

 

118,248

 

 

121,332

 

 

113,821

 

Net loss

 

$

(12,903)

 

$

(11,365)

 

$

(39,211)

 

$

(36,565)

 

Other comprehensive gain (loss):

 

 

 

 

 

 

 

 

 

 

 

 

 

Unrealized gain (loss) on available-for-sale securities

 

 

13

 

 

50

 

 

133

 

 

(10)

 

Comprehensive loss

 

$

(12,890)

 

$

(11,315)

 

$

(39,078)

 

$

(36,575)

 

 

The accompanying notes are an integral part of these financial statements.

 

2


 

IDERA PHARMACEUTICALS, INC.  

 

CONDENSED STATEMENTS OF CASH FLOWS

(UNAUDITED)

 

 

 

 

 

 

 

 

 

 

 

 

Nine Months Ended

 

 

 

September 30, 

 

(In thousands)

    

2016

    

2015

 

Cash Flows from Operating Activities:

 

 

 

 

 

 

 

Net loss

 

$

(39,211)

 

$

(36,565)

 

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

 

 

 

 

 

 

 

Non-employee stock option expense

 

 

 —

 

 

142

 

Stock-based compensation

 

 

5,127

 

 

4,013

 

Issuance of common stock for services rendered

 

 

129

 

 

90

 

Accretion of premiums and discounts on investments

 

 

466

 

 

397

 

Depreciation and amortization expense

 

 

484

 

 

346

 

Changes in operating assets and liabilities:

 

 

 

 

 

 

 

Prepaid expenses and other current assets

 

 

(48)

 

 

(1,163)

 

Accounts payable, accrued expenses, and other liabilities

 

 

937

 

 

(1,590)

 

Deferred revenue

 

 

(833)

 

 

 

Net cash used in operating activities

 

 

(32,949)

 

 

(34,330)

 

Cash Flows from Investing Activities:

 

 

 

 

 

 

 

Purchases of available-for-sale securities

 

 

(2,946)

 

 

(63,106)

 

Proceeds from maturity of available-for-sale securities

 

 

29,946

 

 

23,602

 

Proceeds from sale of available-for-sale securities

 

 

1,974

 

 

999

 

Purchases of property and equipment

 

 

(369)

 

 

(659)

 

Net cash provided by (used in) investing activities

 

 

28,605

 

 

(39,164)

 

Cash Flows from Financing Activities:

 

 

 

 

 

 

 

Proceeds from equity financings, net of issuance costs

 

 

 —

 

 

80,599

 

Proceeds from exercise of common stock warrants and options and employee stock purchases

 

 

111

 

 

987

 

Payments on note payable

 

 

(193)

 

 

(59)

 

Payments on capital lease

 

 

(6)

 

 

(8)

 

Net cash (used in) provided by financing activities

 

 

(88)

 

 

81,519

 

Net (decrease) increase in cash and cash equivalents

 

 

(4,432)

 

 

8,025

 

Cash and cash equivalents, beginning of period

 

 

26,586

 

 

19,971

 

Cash and cash equivalents, end of period

 

$

22,154

 

$

27,996

 

 

The accompanying notes are an integral part of these financial statements.

 

3


 

IDERA PHARMACEUTICALS, INC.

 

NOTES TO CONDENSED FINANCIAL STATEMENTS

September 30, 2016

 

(UNAUDITED)

 

(1) Organization

 

Idera Pharmaceuticals, Inc. (“Idera” or the “Company”) is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel oligonucleotide therapeutics for oncology and rare diseases. The Company uses two distinct proprietary drug discovery technology platforms to design and develop drug candidates: its Toll-like receptor (“TLR”) targeting technology and its third-generation antisense (“3GA”) technology. The Company developed these platforms based on its scientific expertise and pioneering work with synthetic oligonucleotides as therapeutic agents. Using its TLR targeting technology, the Company designs synthetic oligonucleotide-based drug candidates to modulate the activity of specific TLRs. Using its 3GA technology, the Company is developing drug candidates to turn off the messenger RNA (“mRNA”) associated with disease causing genes. The Company believes that its 3GA technology may potentially reduce the immunotoxicity and increase the potency of earlier generation antisense and RNA interference (“RNAi”) technologies.

 

The Company’s business strategy is focused on the clinical development of drug candidates for oncology and rare diseases characterized by small, well-defined patient populations with serious unmet medical needs. The Company believes it can develop and commercialize these targeted therapies on its own.  To the extent the Company seeks to develop drug candidates for broader disease indications, it plans to execute early-stage development through proof-of-concept clinical trials and explore potential collaborative alliances to support late-stage development and commercialization.

 

The Company’s TLR-targeted clinical-stage drug candidates are IMO-2125 and IMO-8400.  IMO-2125 is an agonist of TLR9 and IMO-8400 is an antagonist of TLR7, TLR8 and TLR9.  The Company has also created compounds that are agonists of TLR3, TLR7, TLR8 and TLR9, as well as additional antagonist candidates.

 

At September 30, 2016, the Company had an accumulated deficit of $539,292,000. The Company expects to incur substantial operating losses in future periods. The Company does not expect to generate significant product revenue, sales-based milestones or royalties until the Company successfully completes development and obtains marketing approval for drug candidates, either alone or in collaborations with third parties, which the Company expects will take a number of years. In order to commercialize its drug candidates, the Company needs to complete clinical development and comply with comprehensive regulatory requirements.

 

The Company is subject to a number of risks and uncertainties similar to those of other companies of the same size within the biotechnology industry, such as uncertainty of clinical trial outcomes, uncertainty of additional funding, and history of operating losses.

 

(2) New Accounting Pronouncements - Recently Issued

 

In May 2014, the Financial Accounting Standards Board (the “FASB”) issued Accounting Standards Update (“ASU”) No. 2014-09, Revenue from Contracts with Customers (Topic 606), which was amended by ASU No. 2015-14. ASU No. 2014-09, as amended by ASU No. 2015-14, requires an entity to recognize revenue from the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. In particular, this ASU addresses contracts with more than one performance obligation, as well as the accounting for some costs to obtain or fulfill a contract with a customer, and provides for additional disclosures with respect to revenues and cash flows arising from contracts with customers. This ASU will be effective for fiscal years beginning after December 15, 2017, including interim periods within that fiscal year. Early adoption of this ASU is permitted only for fiscal years beginning after December 15, 2016, including interim periods within that fiscal year. The Company is currently evaluating the effect that the adoption of this ASU will have on its financial statements.

 

4


 

In August 2014, the FASB issued ASU No. 2014-15, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern. ASU 2014-15 amends FASB Accounting Standards Codification (“ASC”) 205-40, Presentation of Financial Statements — Going Concern, by providing guidance on determining when and how reporting entities must disclose going-concern uncertainties in their financial statements, including requiring management to perform interim and annual assessments of an entity’s ability to continue as a going concern within one year of the date of issuance of the entity’s financial statements and providing certain disclosures if there is substantial doubt about the entity’s ability to continue as a going concern. ASU 2014-15 will be effective for fiscal years ending after December 15, 2016 and for interim periods thereafter. Early adoption of ASU 2014-15 is permitted. The Company is currently evaluating ASU 2014-15 and has not yet adopted it. The Company believes that, based on its current operating plan, its existing cash, cash equivalents and investments, including the estimated $48.9 million of net proceeds raised in its October 2016 offering, including the partial exercise by the underwriters of their option to purchase additional shares of the offering and after deducting underwriters’ discounts and commissions and estimated offering expenses (see Note 18, “Subsequent Events”), will enable the Company to fund its operations into the first quarter of 2018. The Company has and will continue to evaluate available alternatives to extend its operations beyond the first quarter of 2018.

 

In January 2016, the FASB issued ASU No. 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities. The amendments in ASU 2016-01 address certain aspects of recognition, measurement, presentation and disclosure of financial instruments. ASU 2016-01 will be effective for fiscal years beginning after December 15, 2017, including interim periods within those fiscal years. Early adoption of some of the amendments included in ASU 2016-01 for financial statements of fiscal years or interim periods that have not yet been issued is permitted as of the beginning of the fiscal year of adoption. The Company is currently evaluating the effect that the adoption of ASU 2016-01 will have on its financial statements.

 

In February 2016, the FASB issued ASU No. 2016-02, Leases. The amendments in ASU 2016-02 will require organizations that lease assets, with lease terms of more than 12 months, to recognize on the balance sheet the assets and liabilities for the rights and obligations created by those leases. Consistent with current U.S. Generally Accepted Accounting Principles (“U.S. GAAP”), the recognition, measurement, and presentation of expenses and cash flows arising from a lease by a lessee primarily will depend on its classification as a finance or operating lease. However, unlike current U.S. GAAP which requires only capital leases to be recognized on the balance sheet, ASU No. 2016-02 will require both types of leases to be recognized on the balance sheet. ASU 2016-02 will be effective for fiscal years beginning after December 15, 2018, including interim periods within those fiscal years. Early adoption is permitted. The Company is currently evaluating the effect that the adoption of ASU 2016-02 will have on its financial statements.

 

In March 2016, the FASB issued ASU No. 2016-06, Derivatives and Hedging (Topic 815) Contingent Put and Call Options in Debt Instruments. ASU 2016-06 amends FASB ASC 815-15 to clarify what steps are required when assessing whether the economic characteristics and risks of call (put) options are clearly and closely related to the economic characteristics and risks of their debt hosts, which is one of the criteria for bifurcating an embedded derivative. ASU 2016-06 will be effective for fiscal years beginning after December 15, 2016, including interim periods within those fiscal years. An entity should apply the amendments in ASU No. 2016-06 on a modified retrospective basis to existing debt instruments as of the beginning of the fiscal year for which the amendments are effective. Early adoption is permitted, including adoption in an interim period. The Company is currently evaluating the effect that the adoption of ASU 2016-06 will have on its financial statements.

 

In March 2016, the FASB issued ASU No. 2016-09, Compensation – Stock Compensation (Topic 718). ASU 2016-09 will require organizations to recognize all income tax effects of awards in the statement of operations when the awards vest or are settled. ASU 2016-09 will also allow organizations to repurchase more shares from employees than they could previously purchase for tax withholding purposes without triggering liability accounting and to make a policy election to account for forfeitures as they occur. ASU 2016-09 will be effective for fiscal years beginning after December 15, 2016, and interim periods within those fiscal years. Early adoption is permitted in any interim or annual period. The Company is currently evaluating the effect that the adoption of ASU 2016-09 will have on its financial statements.

 

In April 2016, the FASB issued ASU No. 2016-10, Revenue from Contracts with Customers (Topic 606). ASU 2016-10 amends ASC 606, Revenue from Contracts with Customers, to clarify two aspects of ASC 606, identifying performance obligations and the licensing implementation guidance, while retaining the related principles of those areas. The amendments in ASU 2016-10 do not change the core principle of the guidance in ASC 606. The amendments in ASU No. 2016-10 affect the guidance in ASU 2014-09, Revenue from Contracts with Customers (Topic 606),  which is not yet

5


 

effective. The effective date and transition requirements for the amendments in ASU No. 2016-10 are the same as the effective date and transition requirements in ASC 606 and any other Topic amended by ASU 2014-09. ASU 2015-14, Revenue from Contracts with Customers (Topic 606): Deferral of the Effective Date, defers the effective date of ASU 2014-09 by one year to annual reporting periods beginning after December 15, 2017, including interim reporting periods within that reporting period. The Company is currently evaluating the effect that the adoption of ASU 2016-10 will have on its financial statements.

 

In May 2016, the FASB issued ASU No. 2016-12, Revenue from Contracts with Customers (Topic 606). ASU 2016-12 amends ASC 606 to address certain issues in the guidance on assessing collectability, presentation of sales taxes, noncash consideration, and completed contracts and contract modifications at transition. The amendments in ASU 2016-12 do not change the core principle of the guidance in ASC 606. The amendments in ASU No. 2016-12 affect the guidance in ASU 2014-09, Revenue from Contracts with Customers (Topic 606),  which is not yet effective. The effective date and transition requirements for the amendments in ASU No. 2016-12 are the same as the effective date and transition requirements in ASC 606 and any other Topic amended by ASU 2014-09. ASU 2015-14, Revenue from Contracts with Customers (Topic 606): Deferral of the Effective Date, defers the effective date of ASU 2014-09 by one year to annual reporting periods beginning after December 15, 2017, including interim reporting periods within that reporting period. The Company is currently evaluating the effect that the adoption of ASU 2016-12 will have on its financial statements.

 

(3) Unaudited Interim Financial Statements

 

The accompanying unaudited financial statements included herein have been prepared by the Company in accordance with U.S. GAAP for interim financial information and pursuant to the rules and regulations of the Securities and Exchange Commission (the “SEC”). Accordingly, certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been condensed or omitted pursuant to such rules and regulations. In the opinion of management, all adjustments, consisting of normal recurring adjustments, and disclosures considered necessary for a fair presentation of interim period results have been included. Interim results for the three and nine months ended September 30, 2016 are not necessarily indicative of results that may be expected for the year ending December 31, 2016. For further information, refer to the financial statements and footnotes thereto included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2015, which was filed with the SEC on March 10, 2016.

 

(4) Financial Instruments

 

The fair value of the Company’s financial instruments is determined and disclosed in accordance with the three-tier fair value hierarchy specified in Note 6, “Fair Value of Assets and Liabilities.” The Company is required to disclose the estimated fair values of its financial instruments. The Company’s financial instruments consist of cash, cash equivalents, available-for-sale investments, receivables and a note payable. The estimated fair values of these financial instruments approximate their carrying values as of September 30, 2016 and December 31, 2015. As of September 30, 2016 and December 31, 2015, the Company did not have any derivatives, hedging instruments or other similar financial instruments except for the note issued under the Company’s loan and security agreement, which is discussed in Note 5(a) to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015, including put and call features which the Company determined are clearly and closely associated with the debt host and do not require bifurcation as a derivative liability, or the fair value of the feature is immaterial.

 

(5) Cash and Cash Equivalents

 

The Company considers all highly liquid investments with maturities of 90 days or less when purchased to be cash equivalents. Cash and cash equivalents at September 30, 2016 and December 31, 2015 consisted of cash and money market funds.

 

(6) Fair Value of Assets and Liabilities

 

The Company measures fair value at the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date using assumptions that market participants would use in pricing the asset or liability (the “inputs”) into a three-tier fair value hierarchy. This fair value hierarchy gives the highest priority (Level 1) to quoted prices in active markets for identical assets or liabilities and the lowest priority (Level 3) to unobservable inputs in which little or no market data exists, requiring companies to develop their own

6


 

assumptions. Observable inputs that do not meet the criteria of Level 1, and include quoted prices for similar assets or liabilities in active markets or quoted prices for identical assets and liabilities in markets that are not active, are categorized as Level 2. Level 3 inputs are those that reflect the Company’s estimates about the assumptions market participants would use in pricing the asset or liability, based on the best information available in the circumstances. Valuation techniques for assets and liabilities measured using Level 3 inputs may include unobservable inputs such as projections, estimates and management’s interpretation of current market data. These unobservable Level 3 inputs are only utilized to the extent that observable inputs are not available or cost-effective to obtain. The Company applies ASU No. 2011-04, Fair Value Measurement (Topic 820), in its fair value measurements and disclosures.

 

The table below presents the assets and liabilities measured and recorded in the financial statements at fair value on a recurring basis at September 30, 2016 and December 31, 2015 categorized by the level of inputs used in the valuation of each asset and liability.

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

    

 

 

    

Quoted Prices

    

 

 

    

 

 

 

 

 

 

 

 

in Active

 

 

 

 

 

 

 

 

 

 

 

 

Markets

 

Significant

 

 

 

 

 

 

 

 

 

for Identical

 

Other

 

Significant

 

 

 

 

 

 

Assets or

 

Observable

 

Unobservable

 

 

 

 

 

 

Liabilities

 

Inputs

 

Inputs

 

(In thousands)

 

Total

 

(Level 1)

 

(Level 2)

 

(Level 3)

 

September 30, 2016

 

 

 

 

 

 

 

 

 

 

 

 

 

Assets

 

 

 

 

 

 

 

 

 

 

 

 

 

Money market funds

 

$

21,647

 

$

21,647

 

$

 

$

 

Short-term investments – corporate bonds

 

 

22,591

 

 

 

 

22,591

 

 

 

Short-term investments – municipal bonds

 

 

7,265

 

 

 

 

7,265

 

 

 

Long-term investments – municipal bonds

 

 

1,408

 

 

 

 

1,408

 

 

 

Total Assets

 

$

52,911

 

$

21,647

 

$

31,264

 

$

 —

 

Total Liabilities

 

$

 

$

 

$

 

$

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

December 31, 2015

 

 

 

 

 

 

 

 

 

 

 

 

 

Assets

 

 

 

 

 

 

 

 

 

 

 

 

 

Money market funds

 

$

26,056

 

$

26,056

 

$

 

$

 

Short-term investments – commercial paper

 

 

3,974

 

 

 

 

3,974

 

 

 

Short-term investments –  corporate bonds

 

 

24,575

 

 

 

 

24,575

 

 

 

Short-term investments – municipal bonds

 

 

5,025

 

 

 

 

5,025

 

 

 

Long-term investments – corporate bonds

 

 

21,186

 

 

 

 

21,186

 

 

 

Long-term investments – municipal bonds

 

 

5,811

 

 

 

 

5,811

 

 

 

Total Assets

 

$

86,627

 

$

26,056

 

$

60,571

 

$

 —

 

Total Liabilities

 

$

 

$

 

$

 

$

 

 

The Level 1 assets consist of money market funds, which are actively traded daily. The Level 2 assets consist of corporate bond, commercial paper and municipal bond investments the fair value of which may not represent actual transactions of identical securities. The fair value of corporate and municipal bonds is generally determined from quoted market prices received from pricing services based upon quoted prices from active markets and/or other significant observable market transactions at fair value. The fair value of commercial paper is generally determined based on the relationship between the investment’s discount rate and the discount rates of the same issuer’s commercial paper available in the market which may not be actively traded daily. Since these fair values may not be based upon actual transactions of identical securities, they are classified as Level 2. Since all investments are classified as available-for-sale securities, any unrealized gains or losses are recorded in accumulated other comprehensive income or loss within stockholders’ equity on the balance sheet. The Company did not elect to measure any other financial assets or liabilities at fair value at September 30, 2016 or December 31, 2015.

 

7


 

(7) Investments

 

The Company’s available-for-sale investments at fair value consisted of the following at September 30, 2016 and December 31, 2015:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

September 30, 2016

 

 

    

 

    

Gross

    

Gross

    

Estimated

 

 

 

 

 

Unrealized

 

Unrealized

 

Fair

 

 

 

Cost

 

(Losses)

 

Gain

 

Value

 

 

 

(In thousands)

 

Short-term investments – corporate bonds

 

 

22,603

 

 

(13)

 

 

1

 

 

22,591

 

Short-term investments – municipal bonds

 

 

7,257

 

 

 

 

8

 

 

7,265

 

Total short-term investments

 

 

29,860

 

 

(13)

 

 

9

 

 

29,856

 

Long-term investments – municipal bonds

 

 

1,405

 

 

 —

 

 

3

 

 

1,408

 

Total long-term investments

 

 

1,405

 

 

 —

 

 

3

 

 

1,408

 

Total investments

 

$

31,265

 

$

(13)

 

$

12

 

$

31,264

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

December 31, 2015

 

 

 

 

 

 

Gross

 

Gross

 

Estimated

 

 

 

 

 

 

Unrealized

 

Unrealized

 

Fair

 

 

    

Cost

    

(Losses)

    

Gains

    

Value

 

 

 

(In thousands)

 

Short-term investments – commercial paper

 

$

3,973

 

$

 —

 

$

1

 

$

3,974

 

Short-term investments – corporate bonds

 

 

24,600

 

 

(25)

 

 

 

 

24,575

 

Short-term investments – municipal bonds

 

 

5,025

 

 

 —

 

 

 

 

5,025

 

Total short-term investments

 

 

33,598

 

 

(25)

 

 

1

 

 

33,574

 

Long-term investments – corporate bonds

 

 

21,289

 

 

(103)

 

 

 

 

21,186

 

Long-term investments – municipal bonds

 

 

5,818

 

 

(9)

 

 

2

 

 

5,811

 

Total long-term investments

 

 

27,107

 

 

(112)

 

 

2

 

 

26,997

 

Total investments

 

$

60,705

 

$

(137)

 

$

3

 

$

60,571

 

 

The Company had no realized gains or losses from available-for-sale securities in the nine months ended September 30, 2016 and 2015. There were no losses or other-than-temporary declines in value included in “Interest income” on the Company’s condensed statements of operations and comprehensive loss for any securities for the nine months ended September 30, 2016 and 2015. The Company had no auction rate securities as of September 30, 2016 and December 31, 2015. See Note 4, “Financial Instruments,” and Note 6, “Fair Value of Assets and Liabilities” for additional information related to the Company’s investments.

 

(8) Property and Equipment

 

At September 30, 2016 and December 31, 2015, net property and equipment at cost consisted of the following:

 

 

 

 

 

 

 

 

 

 

 

September 30, 

 

December 31, 

 

(In thousands)

    

2016

    

2015

 

Leasehold improvements

 

$

671

 

$

603

 

Laboratory equipment and other

 

 

4,786

 

 

4,543

 

Total property and equipment, at cost

 

 

5,457

 

 

5,146

 

Less: Accumulated depreciation and amortization

 

 

3,878

 

 

3,454

 

Property and equipment, net

 

$

1,579

 

$

1,692

 

 

Depreciation and amortization expense on property and equipment was approximately $162,000 and $126,000 in the three months ended September 30, 2016 and 2015, respectively, and $469,000 and $329,000 in the nine months ended September 30, 2016 and 2015, respectively. There were $21,000 and $48,000 in non-cash property returns and additions, respectively, in the nine months ended September 30, 2016 and 2015, respectively.

 

8


 

(9) Restricted Cash

 

As part of the Company’s lease arrangement for its office and laboratory facility in Cambridge, Massachusetts, the Company is required to restrict cash held in a certificate of deposit securing a line of credit for the lessor. As of September 30, 2016 and December 31, 2015, the restricted cash amounted to $311,000 held in certificates of deposit securing a line of credit for the lessor.  The lease expires August 2017.  As such, this amount is reported in Prepaid expenses and other current assets as of September 30, 2016 and in Restricted cash and other assets as of December 31, 2015.

 

(10) Comprehensive Loss 

 

Comprehensive income (loss) is defined as the change in equity of a business enterprise during a period from transactions and other events and circumstances from non-owner sources. Comprehensive loss for the nine months ended September 30, 2016 and 2015 is comprised of reported net loss and any change in net unrealized gains and losses on investments during each period, which is included in accumulated other comprehensive income (loss) on the accompanying balance sheets. The Company applies ASU No. 2011-05, Comprehensive Income, by presenting the components of net income and other comprehensive income as one continuous statement.

 

The following table includes the changes in the accumulated balance of the component of other comprehensive income (loss) for the nine months ended September 30, 2016 and 2015:  

 

 

 

 

 

 

 

 

 

 

 

 

Nine Months Ended

 

Nine Months Ended

 

 

 

September 30, 

 

September 30, 

 

(In thousands)

    

2016

    

2015

 

Accumulated unrealized loss on available-for-sale securities at beginning of period

 

$

(134)

 

$

(17)

 

Change during the period

 

 

133

 

 

(10)

 

Accumulated unrealized loss on available-for-sale securities at end of period

 

$

(1)

 

$

(27)

 

 

 

(11) Collaboration with GlaxoSmithKline Intellectual Property Development Limited

 

In November 2015, the Company entered into a collaboration and license agreement with GlaxoSmithKline Intellectual Property Development Limited (“GSK”) to license, research, develop and commercialize pharmaceutical compounds from the Company’s 3GA technology for the treatment of selected targets in renal disease (the “GSK Agreement”). The initial collaboration term is currently anticipated to last between two and four years. In connection with the GSK Agreement, GSK identified an initial target for the Company to attempt to identify a potential population of development candidates to address such target under a mutually agreed upon research plan, currently estimated to take 27 months to complete. From the population of identified development candidates, GSK may designate one development candidate in its sole discretion to move forward into clinical development. Once GSK designates a development candidate, GSK would be solely responsible for the development and commercialization activities for that designated development candidate.

 

At any time during the first two years of the GSK Agreement, GSK has the option to select up to two additional targets, for further research under mutually agreed upon research plans. GSK may then designate one development candidate for each additional target, at which time GSK would have sole responsibility to develop and commercialize each such designated development candidate.

 

In accordance with the GSK Agreement, a Joint Steering Committee (“JSC”) was formed with equal representation from Idera and GSK. The responsibilities of the JSC, include, but are not limited to monitoring the progress of the collaboration, reviewing research plans and dealing with disputes that may arise between the parties. If a dispute cannot be resolved by the JSC, GSK has final decision making authority.

 

Under the terms of the GSK Agreement, the Company received a $2,500,000 upfront, non-refundable, non-creditable cash payment upon the execution of the GSK Agreement.  The Company is eligible to receive up to approximately $100,000,000 in license, research, clinical development and commercialization milestone payments.  Approximately $9,000,000 of these milestone payments are payable by GSK upon the identification of the additional

9


 

targets, the completion of current and future research plans and the designation of development candidates. Approximately $89,000,000 is payable by GSK upon the achievement of clinical milestones and commercial milestones. In addition, the Company is eligible to receive royalty payments on sales upon commercialization at varying rates of up to five percent on annual net sales, as defined in the GSK Agreement.

 

Accounting Analysis

 

The Company evaluated the GSK Agreement in accordance with the provisions of ASC 605-25. The GSK Agreement contains the following initial deliverables: (i) a collaboration license for Idera’s proprietary technology related to the initial target (the “Collaboration License”), (ii) research services (the “Research Services”), and (iii) participation in the JSC (the “JSC Deliverable”).

 

The Company has determined that GSK’s options to choose up to two additional targets and to purchase additional collaboration licenses for the Company’s proprietary technology related to each additional target are substantive options. GSK is not contractually obligated to exercise the options. Moreover, as a result of the uncertain outcome of the research activities, there is significant uncertainty as to whether GSK will decide to exercise its options for any additional targets. Consequently, the Company is at risk with regard to whether GSK will exercise the options. The Company has determined that GSK’s options to choose up to two additional targets and to purchase additional collaboration licenses for the Company’s proprietary technology related to each additional target are not priced at a significant and incremental discount.

 

The Company has concluded that the Collaboration License does not qualify for separation from the Research Services. As it relates to the assessment of standalone value, the Company has determined that GSK cannot fully exploit the value of the Collaboration License without receipt of the Research Services from the Company. The Research Services involve unique skills and specialized expertise, particularly as it relates to the Company’s proprietary technology, which is not available in the marketplace. Accordingly, GSK must obtain the Research Services from the Company which significantly limits the ability for GSK to utilize the Collaboration License for its intended purpose on a standalone basis. Therefore, the Collaboration License does not have standalone value from the Research Services. As a result, the Collaboration License and the Research Services have been combined as a single unit of accounting (the R&D Services Unit of Accounting). The Company has concluded that the JSC Deliverable identified at the inception of the arrangement has standalone value from the other deliverables noted based on its nature. Factors considered in this determination included, among other things, the capabilities of the collaboration partner, whether any other vendor sells the item separately, whether the value of the deliverable is dependent on the other elements in the arrangement, whether there are other vendors that can provide the items and if the customer could use the item for its intended purpose without the other deliverables in the arrangement.

 

Therefore, the Company has identified two units of accounting in connection with its initial deliverables under the GSK Agreement as follows: (i) R&D Services Unit of Accounting, and (ii) JSC Deliverable.

 

Allocable arrangement consideration at inception of the GSK Agreement is comprised of the up-front payment of $2,500,000, which was allocated to the R&D Services Unit of Accounting. No amount was allocated to the JSC Deliverable because the related best estimate of selling price was determined to be de minimus. The $2,500,000 was recorded as deferred revenue in the Company’s balance sheet and is being recognized as revenue on a straight line basis as the Research Services are delivered over the estimated 27 month research plan period.

 

Payments to be received in connection with GSK’s identification of additional targets and designation of development candidates are considered substantive options as a result of the uncertainties related to the research, development and commercialization activities, and the uncertainty as to whether GSK will exercise the options.  The substantive options are not priced at a significant incremental discount.  Accordingly, the substantive options are not considered deliverables at the inception of the arrangement and the associated option exercise payments are not accounted for at inception of the agreement. 

The clinical and commercial milestones provided for in the GSK Agreement are all performance obligations of GSK occurring after the Company has completed its obligations.  As a result, they represent contingent revenue to the Company and will be accounted for at the time the contingencies are resolved.

 

10


 

The Company will recognize royalty revenue in the period of sale of the related product(s), based on the underlying contract terms, provided that the reported sales are reliably measurable and the Company has no remaining performance obligations, assuming all other revenue recognition criteria are met.

 

The Company recognized as revenue approximately $278,000 and $833,000 of deferred revenue related to the GSK Agreement during the three and nine months ended September 30, 2016, respectively. This revenue is classified as alliance revenue in the accompanying statements of operations and comprehensive loss. There was approximately $1,540,000 of deferred revenue related to the GSK Agreement at September 30, 2016, including approximately $1,111,000 classified as current portion of deferred revenue in the accompanying balance sheet.

 

(12) Stock-Based Compensation 

 

The Company recognizes all stock-based payments to employees and directors as expense in the statements of operations and comprehensive loss based on their fair values. The Company records compensation expense over an award’s requisite service period, or vesting period, based on the award’s fair value at the date of grant. The Company’s policy is to charge the fair value of stock options as an expense, adjusted for forfeitures, on a straight-line basis over the vesting period, which is generally four years for employees and three years for directors.

 

The Company recorded charges in Total operating expense of $1,630,000 and $1,144,000 in its statements of operations and comprehensive loss for the three months ended September 30, 2016 and 2015, respectively, and $5,127,000 and $4,013,000 in its statements of operations and comprehensive loss for the nine months ended September 30, 2016 and 2015, respectively, for stock-based compensation expense attributable to stock-based payments made to employees and directors. The fair value of each option award is estimated on the date of grant using the Black-Scholes option-pricing model. The following weighted average assumptions apply to the options to purchase 3,281,000 and 2,228,000 shares of common stock granted to employees and directors during the nine months ended September 30, 2016 and 2015, respectively:

 

 

 

 

 

 

 

 

 

 

 

 

Nine Months Ended

 

 

 

September 30, 

 

 

    

2016

    

2015

 

Average risk free interest rate

 

 

1.4%

 

 

1.3%

 

Expected dividend yield

 

 

 

 

 

Expected lives (years)

 

 

4.2

 

 

4.3

 

Expected volatility

 

 

92.8%

 

 

92.0%

 

Weighted average grant date fair value of options granted during the period (per share)

 

$

1.77

 

$

2.57

 

Weighted average exercise price of options granted during the period (per share)

 

$

2.65

 

$

3.85

 

 

The expected lives and the expected volatility of the options granted during the nine months ended September 30, 2016 and 2015 are based on historical experience. All options granted during the nine months ended September 30, 2016 and 2015 were granted at exercise prices equal to the fair market value of the common stock on the dates of grant. 

 

(13) Net Loss per Common Share

 

For the three and nine months ended September 30, 2016 and 2015, basic and diluted net loss per common share is computed using the weighted average number of shares of common stock outstanding during the period. Diluted net loss per common share is the same as basic net loss per common share as the effects of the Company’s potential common stock equivalents are antidilutive. Total antidilutive securities were 73,115,102 and 73,444,753 for the nine months ended September 30, 2016 and 2015, respectively, and consist of stock options, preferred stock and warrants.

 

11


 

(14) Common Stock Warrant Exercises, Stock Option Exercises and Employee Stock Purchases

 

The Company issued common stock as a result of warrant exercises, stock option exercises and employee stock purchases as follows during the nine months ended September 30, 2016 and 2015:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Nine Months Ended

    

Nine Months Ended

 

 

 

September 30, 2016

 

September 30, 2015

 

(In thousands)

    

Shares

    

Proceeds

    

Shares

    

Proceeds

 

Warrant exercises

 

 —

 

$

 —

 

136

 

$

503

 

Stock option exercises

 

 —

 

 

 —

 

332

 

 

431

 

Employee stock purchases

 

79

 

 

111

 

19

 

 

53

 

Total

 

79

 

$

111

 

487

 

$

987

 

 

 

(15) Related Party Transactions

 

The Company issued 66,915 and 23,689 shares of common stock in lieu of director board and committee fees of approximately $129,000 and $90,000 pursuant to the Company’s director compensation program during the nine months ended September 30, 2016 and 2015, respectively. The number of shares issued was calculated based on the market closing price of the Company’s common stock on the issuance date.

See also Note 17, “Financing” and Note 18, “Subsequent Events” for additional information on related party transactions.

 

(16) Deferred Tax Assets

The Company’s deferred tax assets are determined based on temporary differences between the financial reporting and tax bases of assets and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse.  A valuation allowance is recorded against deferred tax assets if it is more likely than not that some portion or all of the deferred tax assets will not be realized. For the nine months ended September 30, 2016 and 2015, the Company did not record any current or deferred income tax provisions or benefits.  Due to the uncertainty surrounding the future realization of the deferred tax assets, the Company has recorded full valuation allowances against its otherwise recognizable deferred tax assets at September 30, 2016 and December 31, 2015.

(17) Financing

On February 19, 2015, the Company closed a follow-on underwritten public offering, in which it sold 23,000,000 shares of common stock at a price to the public of $3.75 per share for aggregate gross proceeds of $86.3 million. The net proceeds to the Company from the offering, after deducting underwriters’ discounts and commissions and other offering costs and expenses, were $80.6 million. Investment funds affiliated with Baker Bros. Advisors LP, one of the Company’s principal stockholders, and two members of the Company’s board of directors purchased 5,333,333 shares in this offering at the $3.75 per share purchase price.

(18) Subsequent Events

 

On October 13, 2016, the Company closed a follow-on underwritten public offering, in which it sold 25,000,000 shares of common stock at a price to the public of $2.00 per share for aggregate gross proceeds of $50.0 million. On October 28, 2016, the Company sold an additional 1,225,243 shares of common stock pursuant to the underwriters’ 30-day option to purchase additional shares at the public offering price less the underwriting discount.  The estimated net proceeds to the Company from the offering, including the exercise by the underwriters of their option to purchase additional shares and after deducting underwriters’ discounts and commissions and other offering costs and expenses, were approximately $48.9 million. Investment funds affiliated with Baker Bros. Advisors LP and Pillar Invest Corporation, two of the Company’s principal stockholders, and certain members of the Company’s board of directors, purchased 5,125,000 shares in this offering at the $2.00 per share purchase price.

 

As of October 13, 2016, Baker Bros. Advisors LP, and certain of its affiliated funds held 10,272,314 shares of the Company’s common stock, warrants to purchase up to 20,316,327 shares of the Company’s common stock at an

12


 

exercise price of $0.47 per share and pre-funded warrants to purchase up to 22,151,052 shares of the Company’s common stock at an exercise price of $0.01 per share.

As of October 13, 2016, entities affiliated with Pillar Invest Corporation held 20,346,942 shares of the Company’s common stock and warrants to purchase up to 11,962,731 shares of the Company’s common stock at exercise prices ranging from $0.47 per share to $1.46 per share.

 

 

 

13


 

ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS.

 

Overview

We are a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel oligonucleotide therapeutics for oncology and rare diseases. We use two distinct proprietary drug discovery technology platforms to design and develop drug candidates: our Toll-like receptor, or TLR, targeting technology and our third-generation antisense, or 3GA, technology. We developed these platforms based on our scientific expertise and pioneering work with synthetic oligonucleotides as therapeutic agents. Using our TLR targeting technology, we design synthetic oligonucleotide-based drug candidates to modulate the activity of specific TLRs. Using our 3GA technology, we are developing drug candidates to turn off the messenger RNA, or mRNA, associated with disease causing genes. We believe that our 3GA technology may potentially reduce the immunotoxicity and increase the potency of earlier generation antisense and RNA interference, or RNAi, technologies.

 

Our business strategy focuses on the clinical development of drug candidates for oncology and rare diseases characterized by small, well-defined patient populations with serious unmet medical needs. We believe we can develop and commercialize these targeted therapies on our own. To the extent we seek to develop drug candidates for broader disease indications, we plan to execute early-stage development through proof-of-concept clinical trials and explore potential collaborative alliances to support late-stage development and commercialization.

 

Our TLR-targeted clinical-stage drug candidates are IMO-2125 and IMO-8400. IMO-2125 is an agonist of TLR9 and IMO-8400 is an antagonist of TLR7, TLR8 and TLR9.

 

TLR Modulation Technology Platform

 

TLRs are key receptors of the immune system and play a role in innate and adaptive immunity. As a result, we believe TLRs are potential therapeutic targets for the treatment of a broad range of diseases. Using our chemistry-based platform, we have designed TLR agonists and antagonists to act by modulating the activity of targeted TLRs. A TLR agonist is a compound that stimulates an immune response through the targeted TLR. A TLR antagonist is a compound that inhibits an immune response by blocking the targeted TLR.

 

Our TLR agonist lead drug candidate IMO-2125 is an agonist of TLR9. Our TLR antagonist lead drug candidate is IMO-8400, which is an antagonist of TLR7, TLR8 and TLR9. We also have created compounds that are agonists of TLR3, TLR7, TLR8 and TLR9 as well as additional antagonist candidates.

 

We are evaluating IMO-2125 for the treatment by intra-tumoral injection of multiple oncology indications both in combination with checkpoint inhibitors and as monotherapy. In addition, we are developing IMO-8400 for the treatment of a rare disease called dermatomyositis.

 

Intra-tumoral IMO-2125 Development Program in Immuno-oncology

 

Recent advancements in cancer immunotherapy have included the approval and late-stage development of multiple checkpoint inhibitors, which are therapies that target mechanisms by which tumor cells evade detection by the immune system. Despite these advancements, many patients fail to respond to these therapies. For instance, approximately fifty percent of patients with melanoma fail to respond to therapy with approved checkpoint inhibitors. Current published data suggests that the lack of response to checkpoint inhibition is related to a non-immunogenic tumor micro environment. Because TLR9 agonists stimulate the immune system, we believe that there is a scientific rationale to evaluate the combination of intra-tumoral injection of our TLR9 agonists with checkpoint inhibitors. Specifically, we believe that intra-tumoral injection of our TLR9 agonists activates a local immune response in the injected tumor, which complements the effect of the systemically administered checkpoint inhibitors. In studies in preclinical cancer models conducted in our laboratories, intra-tumoral injection of TLR9 agonists has potentiated the anti-tumor activity of multiple checkpoint inhibitors in multiple tumor models. These data have been presented at a number of scientific conferences from 2014 through 2016. We believe that these data support evaluation of combination regimens including a TLR9 agonist and a checkpoint inhibitor for the treatment of cancer.

We are initially developing IMO-2125 for use in combination with checkpoint inhibitors for the treatment of anti-PD1 refractory metastatic melanoma. We believe, based on internal commercial research that we conducted, that in the

14


 

United States, by 2025, approximately 20,000 people will have metastatic melanoma and approximately 13,000 of those people will have metastatic melanoma that is anti-PD1 refractory. We also believe that TLR9 agonists may be useful in other tumor types that are unaddressable with current immunotherapy due in part to low mutation load and low dendritic cell infiltration, which include non-small cell lung cancer, head and neck cancer, renal cell cancer and bladder cancer. We believe, based on internal commercial research that we conducted, that in the United States, by 2025, approximately 160,000 people will have tumor types that are addressable with current immunotherapy and approximately 70,000 of those people will have tumor types that are anti-PD1 refractory.

In June 2015, we entered into a strategic research alliance with the University of Texas, MD Anderson Cancer Center, or MD Anderson, to commence clinical development of IMO-2125 in combination with checkpoint inhibitors. In December 2015, we initiated a Phase 1/2 clinical trial to assess the safety and efficacy of IMO-2125, administered intra-tumorally, in combination with ipilimumab, a CTLA4 antibody marketed as Yervoy® by Bristol-Myers Squibb Company, in patients with metastatic melanoma (refractory to treatment with a PD1 inhibitor, also referred to as anti-PD1 refractory). We recently amended the trial protocol to enable an additional arm to study the combination of IMO-2125 with pembrolizumab, an anti-PD1 antibody marketed as Keytruda® by Merck & Co. in the same patient population. In the Phase 1 portion of this clinical trial, escalating doses of IMO-2125 ranging from 4 mg through 32 mg in the ipilimumab arm and ranging from 8 mg through 32 mg in the pembrolizumab arm are being administered intra-tumorally into a selected tumor lesion, together with the standard dosing regimen of ipilimumab or pembrolizumab, administered intravenously. The primary objectives of the Phase 1 portion of the trial include characterizing the safety of the combinations and determining the recommended Phase 2 dose. A secondary objective of the Phase 1 portion of the trial is describing the anti-tumor activity of IMO-2125 when administered intra-tumorally in combination with ipilimumab or pembrolizumab. The primary objectives of the Phase 2 portion of the trial will be to characterize the safety of the combinations and determine the activity of the combinations utilizing immune-related response criteria. Additionally, a secondary objective of the Phase 2 portion of the trial is to assess treatment response using traditional RECIST criteria. Serial biopsies will be taken of selected injected and non-injected tumor lesions to assess immune changes and response assessments. We anticipate that the trial may enroll approximately 60 patients.

 

In September 2016, we disclosed early clinical results from the 4 mg and 8 mg dosing cohorts of the Phase 1 ipilimumab combination portion of the trial in which three of six evaluable patients demonstrated clinical responses (one complete response and two partial responses). We also disclosed that the drug was well tolerated through the initial dosing of the 16 mg dosing cohort. We are currently enrolling the 32 mg dosing cohort in the ipilimumab arm of the trial as well as the 8 mg dosing cohort in the pembrolizumab arm of the trial. We will be presenting available translational, efficacy and safety data findings from the 4 mg, 8 mg and 16 mg dosing cohorts in the ipilimumab arm during an oral presentation at the Society for Immunotherapy of Cancer (SITC) Annual Meeting in November 2016.

 

We plan to transition to the Phase 2 portion of the clinical trial upon completion of both the ipilimumab and pembrolizumab dose finding arms. In the Phase 2 portion of the trial, patients will be randomized to receive intra-tumoral IMO-2125 in combination with either ipilimumab or pembrolizumab at the recommended dose determined by the Phase 1 portion of the trial. The Phase 2 portion of the trial will be conducted at multiple clinical sites.

We expect to have data from each of the cohorts in the ipilimumab arm of the Phase 1 portion of the trial by the end of 2016 and plan to then request an End-of-Phase 1 meeting with the U.S. Food and Drug Administration, or the FDA, to discuss the regulatory pathway for IMO-2125 in the anti-PD1 refractory metastatic melanoma population.

Additionally, we are planning to initiate a Phase 1 trial with IMO-2125 administered as a single agent intra-tumorally in multiple tumor types during the first quarter of 2017. We are also planning to initiate a Phase 2 clinical trial with IMO-2125 administered intra-tumorally together with other checkpoint inhibitors in multiple tumor types in the second half of 2017.

IMO-8400 in Rare Diseases

 

We have initiated clinical development of IMO-8400 for the treatment of rare diseases. We have selected dermatomyositis as the first rare disease for which we are developing IMO-8400. We selected this indication for development based on the reported increase in TLR expression in this disease state, expression of cytokines indicative of key TLR-mediated pathways and the presence of auto-antibodies that can induce TLR-mediated immune responses.

 

15


 

We considered that multiple independent research studies across a broad range of autoimmune diseases, including both dermatomyositis and psoriasis, have demonstrated that the over-activation of TLRs plays a critical role in disease maintenance and progression. In autoimmune diseases, endogenous nucleic acids released from damaged or dying cells initiate signaling cascades through TLRs, leading to the induction of multiple pro-inflammatory cytokines. This inflammation causes further damage to the body's own tissues and organs and the release of more self-nucleic acids, creating a self-sustaining autoinflammatory cycle that contributes to chronic inflammation in the affected tissue, promoting disease progression.

We believe that we demonstrated proof of concept for our approach of using TLRs to inhibit the over-activation of specific TLRs for the treatment of psoriasis and potentially other autoimmune diseases in a randomized, double-blind, placebo-controlled Phase 2 clinical trial of IMO-8400 that we conducted in patients with moderate to severe plaque psoriasis, a well-characterized autoimmune disease. In this trial, we evaluated IMO-8400 at four subcutaneous dose levels of 0.075 mg/kg, 0.15 mg/kg, 0.3 mg/kg, and 0.6 mg/kg, versus placebo, administered once weekly for 12 weeks in 46 patients. The trial met its primary objective as IMO-8400 was well tolerated at all dose levels with no treatment-related discontinuations, treatment-related serious adverse events or dose reductions. The trial also met its secondary objective of demonstrating clinical activity in psoriasis patients, as assessed by the Psoriasis Area Severity Index.

Dermatomyositis is a rare, debilitating, inflammatory muscle and skin disease associated with significant morbidity, decreased quality of life and an increased risk of premature death. While the cause of dermatomyositis is not well understood, the disease process involves immune system attacks against muscle and skin that lead to inflammation and tissue damage. Major symptoms can include progressive muscle weakness, severe skin rash, calcium deposits under the skin (calcinosis), difficulty swallowing (dysphagia) and interstitial lung disease. We believe, based on internal commercial research that we conducted, that dermatomyositis affects approximately 25,000 people in the United States, and is about twice as common in women as men, with a typical age of onset between 45 and 65 years in adults. Dermatomyositis represents one form of myositis, a spectrum of inflammatory muscle diseases that also includes juvenile dermatomyositis, polymyositis and inclusion body myositis.

In August 2014, we initiated a collaboration with The Myositis Association, or TMA, a leading U.S. patient advocacy organization focused on myositis, to advance the clinical development of IMO-8400 for the treatment of dermatomyositis. Under the collaboration, we and TMA agreed to develop educational programs for patients and healthcare providers on TLR antagonism and opportunities to participate in clinical research. In addition, we formed an advisory committee of leading independent experts in the treatment of dermatomyositis to advise us on the development of IMO-8400 in dermatomyositis.

 

In December 2015, we initiated a Phase 2, randomized, double-blind, placebo-controlled clinical trial designed to assess the safety, tolerability and treatment effect of IMO-8400 in adult patients with dermatomyositis. Eligibility criteria include evidence of active skin and muscle involvement. Patients in the trial are randomized to one of three groups to receive once weekly subcutaneous injections of: placebo, 0.6 mg/kg or 1.8 mg/kg of IMO-8400 for a period of 24 weeks. The trial is expected to enroll approximately 36 patients and is being conducted at approximately 22 centers in the United States, the United Kingdom and Sweden. The primary efficacy endpoint is the change from baseline in the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), a validated outcome measure of skin disease. Additional exploratory endpoints include muscle strength and function (which are among the International Myositis Assessment & Clinical Studies Group (IMACS) core set measures), patient-reported quality of life and biochemical markers of disease activity. We expect to complete enrollment of this trial in the second half of 2017 with data available in early 2018.

Third-generation Antisense (3GA)

 

Third-generation Antisense (3GA) Technology to Target mRNA

 

We are developing our 3GA technology to "turn off" the mRNA associated with disease causing genes. We have designed 3GA oligonucleotides to specifically address challenges associated with earlier generation antisense and RNAi technologies.

 

Our focus is on creating 3GA candidates targeted to specific genes to treat cancer and rare diseases. Our key considerations in identifying disease indications and gene targets in our 3GA program include: strong evidence that the disease is caused by a specific protein; clear criteria to identify a target patient population; biomarkers for early assessment of clinical proof of concept; a targeted therapeutic mechanism of action; unmet medical need to allow for a rapid

16


 

development path to approval and commercial opportunity. Based on these criteria, we are developing 3GA compounds against multiple gene targets, including NLRP3 (NOD-like receptor family, pyrin domain containing protein 3) and DUX4 (Double Homeobox 4). Potential disease indications include, but are not limited to, interstitial cystitis, lupus nephritis, uveitis and facioscapulohumeral muscular dystrophy (FSHD).

 

We are currently conducting clinical, regulatory and commercial analysis activities of these compounds, including IND-enabling studies of a compound against NLRP3, and plan to submit an investigational new drug application, or IND, for one of these compounds in 2017 and initiate a Phase 1 human clinical proof-of-concept trial in the second half of 2017. We plan to announce the first disease indication for which we plan to develop one of our 3GA compounds in January 2017. During the first half of 2016, we generated 3GA compounds for a series of additional gene targets. We expect that these will enable us to continue to expand our pipeline opportunities for both internal development as well as collaborations in areas outside of our focus. We have recently presented several pre-clinical data updates at significant oligonucleotide medical and scientific conferences.

 

Collaboration with GlaxoSmithKline Intellectual Property Development Limited

 

In November 2015, we entered into a collaboration and license agreement with GlaxoSmithKline Intellectual Property Development Limited, or GSK, to license, research, develop and commercialize pharmaceutical compounds from our 3GA technology for the treatment of selected targets in renal disease, which we refer to as the GSK Agreement. The initial collaboration term is currently anticipated to last between two and four years. In connection with the GSK Agreement, GSK identified an initial target for us to attempt to identify a potential population of development candidates to address such target under a mutually agreed upon research plan, currently estimated to take 27 months to complete. From the population of identified development candidates, GSK may designate one development candidate in its sole discretion to move forward into clinical development. Once GSK designates a development candidate, GSK would be solely responsible for the development and commercialization activities for that designated development candidate.

 

At any time during the first two years of the GSK Agreement, GSK has the option to select up to two additional targets, for further research under mutually agreed upon research plans. GSK may then designate one development candidate for each additional target, at which time GSK would have sole responsibility to develop and commercialize each such designated development candidate.

 

Under the terms of the GSK Agreement, we received a $2,500,000 upfront, non-refundable, non-creditable cash payment upon the execution of the GSK Agreement. We are eligible to receive up to approximately $100,000,000 in license, research, clinical development and commercialization milestone payments, including the $2,500,000 upfront payment. Approximately $9,000,000 of these milestone payments are payable by GSK upon the identification of additional targets, the completion of current and future research plans and the designation of development candidates. Approximately $89,000,000 is payable by GSK upon the achievement of clinical milestones and commercial milestones. In addition, we are eligible to receive royalty payments based on net sales of licensed products following commercialization at varying rates of up to five percent on annual net sales, as defined in the GSK Agreement.

 

Additional Programs

 

IMO-9200 for Autoimmune Disease.    We have developed a second novel synthetic oligonucleotide antagonist of TLR7, TLR8, and TLR9, IMO-9200, as a drug candidate for potential use in selected autoimmune disease indications. In 2015, we completed a Phase 1 clinical trial of IMO-9200 in healthy subjects as well as additional preclinical studies of IMO-9200 for autoimmune diseases. In 2015, we determined not to proceed with the development of IMO-9200 because the large autoimmune disease indications for which IMO-9200 had been developed did not fit within the strategic focus of our company. We continue to explore and pursue strategic alternatives for IMO-9200.

 

IMO-8400 for B-Cell Lymphomas.   In December 2013, we initiated a Phase 1/2 clinical trial of IMO-8400 in patients with Waldenström's macroglobulinemia, and in March 2014, we initiated a Phase 1/2 clinical trial of IMO-8400 in diffuse large B-cell lymphoma, or DLBCL, harboring the MYD88 L265P oncogenic mutation.

In December 2015, we presented interim clinical data from the Phase 1/2 clinical trial of IMO-8400 in Waldenström's macroglobulinemia, which showed signals of positive clinical activity as well as safety in the first three dosing cohorts of the trial. For much of 2016, we continued dose escalation to a higher dose level to determine if stronger activity would be observed.

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In September 2016, we announced that we had suspended the clinical development of IMO-8400 for B-cell lymphomas, including our ongoing trials in Waldenström's macroglobulinemia and DLBCL, and plan to explore strategic alternatives for IMO-8400 in these indications. This decision was based upon our prioritization of the clinical development plans for IMO-2125 and our assessment that the level of clinical activity seen in the Waldenström's macroglobulinemia trial would not support the development of IMO-8400 for these indications as a monotherapy, the very slow enrollment rate in DLBCL and our commercial assessment. No patients are currently enrolled in the trial of IMO-8400 in DLBCL, and we will not enroll any additional patients in that trial. We plan to finish treating patients in the trial of IMO-8400 in Waldenström's macroglobulinemia but enrollment of new patients has been suspended. In these trials under our B-cell lymphoma program, IMO-8400 was generally well tolerated at all dose levels evaluated, with only one treatment-related discontinuation due to adverse events and no dose reductions. The treatment-related discontinuation involved a single patient who experienced a serious adverse event that was possibly related to IMO-8400.

 

Accumulated Deficit

 

As of September 30, 2016, we had an accumulated deficit of $539,292,000. We expect to incur substantial operating losses in future periods. We do not expect to generate significant product revenue, sales-based milestones or royalties from our development programs until we successfully complete development and obtain marketing approval for drug candidates, either alone or in collaborations with third parties, which we expect will take a number of years. In order to commercialize our drug candidates, we need to complete clinical development and comply with comprehensive regulatory requirements.

 

CRITICAL ACCOUNTING POLICIES AND ESTIMATES

 

This management’s discussion and analysis of financial condition and results of operations is based on our financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States. The preparation of these financial statements requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting period. On an ongoing basis, management evaluates its estimates and judgments, including those related to stock-based compensation. Management bases its estimates and judgments on historical experience and on various other factors that are believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.

 

We regard an accounting estimate or assumption underlying our financial statements as a “critical accounting estimate” where:

 

·

the nature of the estimate or assumption is material due to the level of subjectivity and judgment necessary to account for highly uncertain matters or the susceptibility of such matters to change; and

 

·

the impact of the estimates and assumptions on financial condition or operating performance is material.

 

Our significant accounting policies are described in Note 2 of the notes to our financial statements in our Annual Report on Form 10-K for the year ended December 31, 2015. Not all of these significant policies, however, fit the definition of critical accounting policies and estimates. We believe that our accounting policies relating to revenue recognition, stock-based compensation and research and development prepayments, accruals and related expenses, as described under the caption “Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations — Critical Accounting Policies and Estimates” in our Annual Report on Form 10-K for the year ended December 31, 2015, fit the description of critical accounting estimates and judgments. There were no changes in these policies during the nine months ended September 30, 2016.

 

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RESULTS OF OPERATIONS

 

Three and Nine Months Ended September 30, 2016 and 2015

 

Alliance Revenue

 

Alliance revenue increase by approximately $303,000 from $20,000 in the three months ended September 30, 2015 to $323,000 in the three months ended September 30, 2016 and by approximately $859,000 from $59,000 in the nine months ended September 30, 2015 to $918,000 in the nine months ended September 30, 2016 as a result of revenue recognized under the GSK Agreement. In November 2015, in connection with the execution of the GSK Agreement, we received a $2,500,000 upfront payment that we recorded as deferred revenue.  We are recognizing this deferred revenue as revenue on a straight line basis over the anticipated 27-month performance period under the GSK Agreement. Accordingly, we recognized approximately $278,000 and $833,000 of alliance revenue related to the GSK Agreement during the three and nine months ended September 30, 2016, respectively. We also recognized revenue from the reimbursement by licensees of costs associated with patent maintenance as alliance revenue in the three and nine months ended September 30, 2016 and 2015.

 

Research and Development Expenses

 

Research and development expenses increased by $1,939,000, or 26%, from $7,454,000 for the three months ended September 30, 2015, to $9,393,000 for the three months ended September 30, 2016 and by $3,683,000, or 15%, from $25,134,000 for the nine months ended September 30, 2015, to $28,817,000 for the nine months ended September 30, 2016. In the following table, research and development expenses are set forth in the following six categories which are discussed beneath the table:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Three months ended

 

 

 

Nine months ended

 

 

 

 

 

September 30, 

 

Percentage

 

September 30, 

 

Percentage

 

 

 

(in thousands)

 

Increase

 

(in thousands)

 

Increase

 

 

    

2016

    

2015

    

(Decrease)

    

2016

    

2015

    

(Decrease)

 

IMO-8400 external development expense

 

$

2,771

 

$

2,426

 

14%

 

$

8,814

 

$

6,952

 

27%

 

IMO-2125 external development expense

 

 

723

 

 

597

 

21%

 

 

2,330

 

 

597

 

290%

 

IMO-9200 external development expense

 

 

185

 

 

139

 

33%

 

 

471

 

 

2,404

 

(80%)

 

Other drug development expense

 

 

3,246

 

 

1,962

 

65%

 

 

9,668

 

 

8,673

 

11%

 

Basic discovery expense

 

 

2,468

 

 

2,330

 

6%

 

 

7,534

 

 

6,508

 

16%

 

 

 

$

9,393

 

$

7,454

 

26%

 

$

28,817

 

$

25,134

 

15%

 

 

IMO-8400 External Development Expenses.    These expenses include external expenses that we have incurred in connection with IMO-8400 since October 2012, when we commenced clinical development of IMO-8400. These external expenses include payments to independent contractors and vendors for drug development activities conducted after the initiation of IMO-8400 clinical development but exclude internal costs such as payroll and overhead expenses. Since October 2012, we have incurred approximately $31,940,000 in IMO-8400 external development expenses through September 30, 2016, including costs associated with our Phase 1 clinical trial in healthy subjects;  our Phase 2 clinical trial in patients with psoriasis;  preparation for and conduct of our Phase 1/2 clinical trial in patients with Waldenström’s macroglobulinemia and our Phase 1/2 clinical trial in patients with DLBCL harboring the MYD88 L265P oncogenic mutation, which we announced plans to discontinue; the preparation for and conduct of our ongoing Phase 2 clinical trial in patients with dermatomyositis;  the manufacture of additional drug substance for use in our clinical trials;  and expenses associated with our collaboration with Abbott Molecular for the development of a companion diagnostic for identification of patients with B-cell lymphoma harboring the MYD88 L265P oncogenic mutation. 

The increases in our IMO-8400 external development expenses in the three and nine months ended September 30, 2016, as compared to the three and nine months ended September 30, 2015, were primarily due to increases in costs associated with our ongoing Phase 2 clinical trial in patients with dermatomyositis and costs incurred in connection with the manufacture of additional drug substance for use in our clinical trials in the three and nine months ended September 30, 2016. An increase in the cost of conducting our Phase 1/2 clinical trial in patients with DLBCL harboring the MYD88 L265P oncogenic mutation also contributed to the increase in IMO-8400 external development expenses in the three months ended September 30, 2016. These increases were partially offset by a decrease in the cost of developing a companion diagnostic for identification of patients with B-cell lymphoma harboring the MYD88 L265P oncogenic mutation.

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We expect our IMO-8400 external development expenses to increase during 2016, as compared to 2015.  In September 2016, we announced that we had suspended the clinical development of IMO-8400 for B-cell lymphomas, including our trials in Waldenström’s macroglobulinemia and DLBCL, and plan to explore strategic alternatives for IMO-8400 in these indications.  We expect to continue to incur costs associated with IMO-8400 as we continue our ongoing Phase 2 clinical trial of IMO-8400 in patients with dermatomyositis, finish treating enrolled patients in our clinical trial of IMO-8400 in Waldenström’s macroglobulinemia and wind down our clinical development of IMO-8400 in Waldenström’s macroglobulinemia and DLBCL.

IMO-2125 External Development Expenses.   These expenses include external expenses that we have incurred in connection with the development of IMO-2125 as part of our immuno-oncology program. These external expenses include payments to independent contractors and vendors for drug development activities conducted after the initiation of IMO-2125 clinical development in immuno-oncology, but exclude internal costs such as payroll and overhead expenses. We commenced clinical development of IMO-2125 as part of our immuno-oncology program in July 2015 and from July 2015 through September 30, 2016 we incurred approximately $3,513,000 in IMO-2125 external development expenses as part of our immuno-oncology program, including costs associated with the preparation for and conduct of the ongoing Phase 1/2 clinical trial being conducted under our research alliance with MD Anderson to assess the safety and efficacy of IMO-2125 in combination with ipilimumab and with pembrolizumab in patients with metastatic melanoma, the manufacture of additional drug substance for use in our clinical trials and additional nonclinical studies. The $3,513,000 in IMO-2125 external development expenses excludes costs incurred prior to July 2015 with respect to IMO-2125, including costs incurred for the development of IMO-2125 for the treatment of patients with chronic hepatitis C virus which we discontinued in the third quarter of 2011.

We expect our IMO-2125 external development expenses to increase during 2016, as compared to 2015, as we plan to continue our Phase 1/2 clinical trial being conducted under our research alliance with MD Anderson to assess the safety and efficacy of IMO-2125 in combination with ipilimumab and with pembrolizumab in patients with metastatic melanoma, work on the design and planning for additional clinical trials of IMO-2125 and develop our strategy to optimize IMO-2125, and continue manufacturing activities and nonclinical studies.

IMO-9200 External Development Expenses.    These expenses include external expenses that we have incurred in connection with IMO-9200 since October 2014, when we commenced clinical development of IMO-9200. These external expenses include payments to independent contractors and vendors for drug development activities conducted after the initiation of IMO-9200 clinical development but exclude internal costs such as payroll and overhead expenses. We have incurred approximately $4,632,000 in IMO-9200 external development expenses from October 2014 through September 30, 2016, including costs associated with our Phase 1 clinical trial in healthy subjects, the manufacture of additional drug substance for use in our clinical and nonclinical trials and additional nonclinical studies. We classified the IMO-9200 external development expenses incurred prior to October 2014 in other drug development expenses.

The decrease in IMO-9200 external development expenses in the nine months ended September 30, 2016, as compared to the nine months ended September 30, 2015, reflects decreases in clinical and nonclinical trial costs, drug manufacturing and nonclinical study costs incurred during the nine months ended September 30, 2016, as compared to the corresponding 2015 period.  The increase in IMO-9200 external development expenses in the three months ended September 30, 2016, as compared to the three months ended September 30, 2015, resulted from the manufacture of drug substance for stability testing.  We expect our IMO-9200 external development expenses to decrease during 2016, as compared to 2015, as we determined not to proceed with the development of IMO-9200 but continue to explore and pursue strategic alternatives for IMO-9200.

Other Drug Development Expenses. These expenses include external expenses associated with preclinical development of identified compounds in anticipation of advancing these compounds into clinical development. In addition, these expenses include internal costs, such as payroll and overhead expenses, associated with preclinical development and products in clinical development. The external expenses associated with preclinical compounds include payments to contract vendors for manufacturing and the related stability studies, preclinical studies, including animal toxicology and pharmacology studies, and professional fees. Other drug development expenses also include costs associated with compounds that were previously being developed but are not currently being developed.

 

The increases in other drug development expenses in the three and nine months ended September 30, 2016, as compared to the three and nine months ended September 30, 2015, were primarily due the costs of additional headcount associated with our expanded drug development programs and costs associated with the manufacture of 3GA drug supplies,

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offset by lower consulting costs during the three and nine months ended September 30, 2016.  In addition, the nine months ended September 30, 2015 included costs associated with the manufacture of IMO-2055 drug supply and other drug development expenses of IMO-2125 development expenses incurred prior to the commencement of its clinical development in our immuno-oncology program in July 2015. Costs associated with the clinical development of IMO-2125 since July 2015 are included in IMO-2125 external development expenses.

 

Basic Discovery Expenses. These expenses include our internal and external expenses relating to our discovery efforts with respect to our TLR-targeted programs, including agonists and antagonists of TLR3, TLR7, TLR8 and TLR9, and our 3GA program. These expenses reflect payments for laboratory supplies, external research, and professional fees, as well as payroll and overhead expenses.  The increases in basic discovery expenses in the three and nine months ended September 30, 2016, as compared to the three and nine months ended September 30, 2015, were primarily due to increases in payroll and stock-based compensation, the costs of laboratory supplies and facilities expenses during the three and nine months ended September 30, 2016. The increases in basic discovery expenses in the three and nine months ended September 30, 2016 were partially offset by decreases in external research and recruiting expenses in the three and nine months ended September 30, 2016.

 

We do not know if we will be successful in developing any drug candidate from our research and development programs. At this time, and without knowing the results from our ongoing clinical trial of IMO-8400, our ongoing clinical trial of IMO-2125, and our ongoing development of compounds in our 3GA program, we cannot reasonably estimate or know the nature, timing, and costs of the efforts that will be necessary to complete the remainder of the development of, or the period, if any, in which material net cash inflows may commence from, any drug candidate from our research and development programs. Moreover, the clinical development of any drug candidate from our research and development programs is subject to numerous risks and uncertainties associated with the duration and cost of clinical trials, which vary significantly over the life of a project as a result of unanticipated events arising during clinical development.

 

General and Administrative Expenses

 

General and administrative expenses decreased by $123,000 from $4,030,000 in the three months ended September 30, 2015, to $3,907,000 in the three months ended September 30, 2016 and decreased by $87,000, from $11,688,000 in the nine months ended September 30, 2015, to $11,601,000 in the nine months ended September 30, 2016.  General and administrative expenses consist primarily of salary expense, stock-based compensation expense, consulting fees and professional legal fees associated with our patent applications and maintenance, our corporate regulatory filing requirements, our corporate legal matters, and our business development initiatives.

 

The changes in general and administrative expenses during the three and nine months ended September 30, 2016 reflect increases in salary expense, legal fees associated with our patent filing and maintenance and accounting and auditing fees, including the cost of Sarbanes-Oxley compliance and the related internal control audit, offset by decreases in corporate legal fees and investor relations expenses as compared to the three and nine months ended September 30, 2015. The changes in general and administrative expenses during the nine months ended September 30, 2016 also reflect an increase in stock-based compensation.

 

Interest Income

 

Interest income decreased by $33,000 from $123,000 in the three months ended September 30, 2015 to $90,000 in the three months ended September 30, 2016 and increased by $81,000 from $239,000 in the nine months ended September 30, 2015 to $320,000 in the nine months ended September 30, 2016. These variances were primarily due to fluctuations of investment balances in the three and nine months ended September 30, 2016 and 2015, which were impacted by the investment of funds obtained from our follow-on underwritten public offering in February 2015 and from warrant and option exercises since June 2015, offset by general spending.  

 

Interest Expense

 

Interest expense decreased during the three and nine months ended September 30, 2016, as compared to the three and nine months ended September 30, 2015, primarily due to a decrease in the outstanding principal amount of our note under our loan and security agreement with Oxford Finance LLC.

 

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Net Loss

As a result of the factors discussed above, our net loss was $12,903,000 for the three months ended September 30, 2016, compared to $11,365,000 for the three months ended September 30, 2015 and $39,211,000 for the nine months ended September 30, 2016, compared to $36,565,000 for the nine months ended September 30, 2015.  Since January 1, 2001, we have primarily been involved in the development of our TLR pipeline. From January 1, 2001 through September 30, 2016, we incurred losses of $279,099,000. We also incurred net losses of $260,193,000 prior to December 31, 2000 during which time we were primarily involved in the development of earlier generation antisense technology. Since our inception, we had an accumulated deficit of $539,292,000 through September 30, 2016. We expect to continue to incur substantial operating losses in the future.

LIQUIDITY AND CAPITAL RESOURCES

 

Sources of Liquidity

 

We require cash to fund our operating expenses and to make capital expenditures. Historically, we have funded our cash requirements primarily through the following:

 

·

sale of common stock, preferred stock and warrants and warrant exercises;

 

·

debt financing, including capital leases;

 

·

license fees, research funding and milestone payments under collaborative and license agreements; and

 

·

interest income.

 

We have an effective shelf registration statement on Form S-3 that permits us to offer and sell, as of October 28, 2016, up to an additional $61,300,000 of securities in one or more offerings.

 

Cash Flows

 

Nine Months Ended September 30, 2016

 

As of September 30, 2016, we had approximately $53,418,000 in cash, cash equivalents and investments, a net decrease of approximately $33,739,000 from December 31, 2015. Net cash used in operating activities totaled $32,949,000 during the nine months ended September 30, 2016, reflecting our $39,211,000 net loss for the period, as adjusted for non-cash income and expenses, including stock-based compensation, depreciation and amortization expense and accretion of investment premiums. Net cash used in operating activities also reflects changes in our prepaid expenses, accounts payable, accrued expenses and other liabilities and the recognition of deferred revenue.

 

The $28,605,000 net cash provided by investing activities during the nine months ended September 30, 2016 reflects proceeds from the maturity of $29,946,000 of available-for-sale securities, which are investments that we do not have the positive intent to hold to maturity at the time of purchase, and proceeds from the sale of $1,974,000 of available-for-sale securities,  partially offset by the purchase of $2,946,000 of available-for-sale securities and payments for the purchase of $369,000 in property and equipment.

 

The $88,000 net cash used in financing activities during the nine months ended September 30, 2016 reflects $193,000 in payments on our note payable, partially offset by $111,000 in net proceeds from employee stock purchases under our 1995 Employee Stock Purchase Plan, or ESPP.

 

Nine Months Ended September 30, 2015

 

As of September 30, 2015, we had approximately $94,694,000 in cash, cash equivalents and investments, a net increase of approximately $46,123,000 from December 31, 2014. Net cash used in operating activities totaled $34,330,000 during the nine months ended September 30, 2015, reflecting our $36,565,000 net loss, as adjusted for non-cash income and expenses, including stock-based compensation, depreciation and amortization. Net cash used in operating activities also reflects changes in our prepaid expenses and accounts payable, accrued expenses and other liabilities.

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The $39,164,000 net cash used in investing activities during the nine months ended September 30, 2015 reflects the purchase of $63,106,000 of available-for-sale securities, which are investments that we do not have the positive intent to hold to maturity at the time of purchase, the maturity of $23,602,000 of available-for-sale securities, the sale of $999,000 of available-for-sale securities, and payments for the purchase of $659,000 in property and equipment.

 

The $81,519,000 net cash provided by financing activities during the nine months ended September 30, 2015 primarily reflects $80,599,000 in net proceeds from our follow-on underwritten public offering of our common stock in February 2015 and $987,000 in net proceeds from employee stock purchases under our ESPP and the exercise of common stock options.

 

Funding Requirements

 

We have incurred operating losses in all fiscal years since our inception except 2002, 2008 and 2009, and we had an accumulated deficit of $539,292,000 at September 30, 2016. We expect to incur substantial operating losses in future periods. These losses, among other things, have had and will continue to have an adverse effect on our stockholders’ equity, total assets and working capital. We have received no revenues from the sale of drugs. As of October 15, 2016, substantially all of our revenues have been from collaboration and license agreements. We have devoted substantially all of our efforts to research and development, including clinical trials, and we have not completed development of any drugs. Because of the numerous risks and uncertainties associated with developing drugs, we are unable to predict the extent of any future losses, whether or when any of our products will become commercially available or when we will become profitable, if at all.

 

We do not expect to generate significant additional funds internally until we successfully complete development and obtain marketing approval for products, either alone or in collaboration with third parties, which we expect will take a number of years. In addition, we have no committed external sources of funds.

 

We had cash, cash equivalents and investments of approximately $53,418,000 at September 30, 2016. In October 2016, we received estimated net proceeds of approximately $48,902,000 from a follow-on public offering, including from the partial exercise by the underwriters of their option to purchase additional shares of the offering, after deducting underwriters’ discounts and commissions and estimated offering expenses.  We believe that, based on our current operating plan, our existing cash, cash equivalents and investments, including the net proceeds from the offering, will enable us to fund our operations into the first quarter of 2018. Specifically, we believe that our available funds will be sufficient to enable us to:

·

participate in an FDA End-of-Phase 1 meeting to obtain FDA feedback on the regulatory pathway for IMO-2125;

·

complete our ongoing Phase 1/2 clinical trial of IMO-2125 in combination with ipilimumab or pembrolizumab in anti-PD1 refractory metastatic melanoma;

·

prepare for the initiation of a pivotal Phase 3 clinical trial of IMO-2125 in combination with a checkpoint inhibitor for the treatment of anti-PD1 refractory metastatic melanoma;

·

initiate a Phase 1 intra-tumoral monotherapy clinical trial of IMO-2125 in multiple refractory tumor types;

·

initiate a Phase 2 multi-arm clinical trial of IMO-2125 in combination with a checkpoint inhibitor in multiple refractory tumor types;

·

complete our ongoing Phase 2 clinical trial of IMO-8400 in patients with dermatomyositis; and

·

submit an IND and initiate a Phase 1 human clinical proof-of-concept trial for one of our 3GA compounds.

We expect that we will need to raise additional funds in order to conduct any other clinical development of our TLR drug candidates or to conduct any other development of our 3GA technology, and to fund our operations. We are seeking and expect to continue to seek additional funding through collaborations, the sale or license of assets or financings of equity or debt securities. We believe that the key factors that will affect our ability to obtain funding are:

23


 

·

the results of our clinical and preclinical development activities in our rare disease program, our immuno-oncology program and our 3GA program, and our ability to advance our drug candidates and 3GA technology on the timelines anticipated;

·

the cost, timing, and outcome of regulatory reviews;

·

competitive and potentially competitive products and technologies and investors' receptivity to our drug candidates and the technology underlying them in light of competitive products and technologies;

·

the receptivity of the capital markets to financings by biotechnology companies generally and companies with drug candidates and technologies such as ours specifically; and

·

our ability to enter into additional collaborations with biotechnology and pharmaceutical companies and the success of such collaborations.

In addition, increases in expenses or delays in clinical development may adversely impact our cash position and require additional funds or cost reductions.

Financing may not be available to us when we need it or may not be available to us on favorable or acceptable terms or at all. We could be required to seek funds through collaborative alliances or through other means that may require us to relinquish rights to some of our technologies, drug candidates or drugs that we would otherwise pursue on our own. In addition, if we raise additional funds by issuing equity securities, our then existing stockholders will experience dilution. The terms of any financing may adversely affect the holdings or the rights of existing stockholders. An equity financing that involves existing stockholders may cause a concentration of ownership. Debt 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 are likely to include rights that are senior to the holders of our common stock. Any additional debt or equity financing may contain terms which are not favorable to us or to our stockholders, such as liquidation and other preferences, or liens or other restrictions on our assets. As discussed in Note 10 to the financial statements appearing in our Annual Report on Form 10-K for the year ended December 31, 2015 that was filed with the SEC, additional equity financings may also result in cumulative changes in ownership over a three-year period in excess of 50% which would limit the amount of net operating loss and tax credit carryforwards that we may utilize in any one year.

If we are unable to obtain adequate funding on a timely basis or at all, we will be required to terminate, modify or delay preclinical or clinical trials of one or more of our drug candidates, significantly curtail or terminate discovery or development programs for new drug candidates or relinquish rights to portions of our technology, drug candidates and/or products.

 

Contractual Obligations

 

During the nine months ended September 30, 2016, there were no material changes outside the ordinary course of our business to our contractual obligations as disclosed in our Annual Report on Form 10-K for the year ended December 31, 2015.

 

As of September 30, 2016, we had no off-balance sheet arrangements.

 

New Accounting Pronouncements

New accounting pronouncements are discussed in Note 2 in the notes to the financial statements in this Quarterly Report on Form 10-Q.

 

 

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ITEM 3. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK.

 

As of September 30, 2016, all material assets and liabilities are in U.S. dollars, which is our functional currency.

 

We maintain investments in accordance with our investment policy. The primary objectives of our investment activities are to preserve principal, maintain proper liquidity to meet operating needs and maximize yields. Although our investments are subject to credit risk, our investment policy specifies credit quality standards for our investments and limits the amount of credit exposure from any single issue, issuer or type of investment. We regularly review our investment holdings in light of the then current economic environment. We do not own auction rate securities or derivative financial investment instruments in our investment portfolio. At September 30, 2016, all of our invested funds were invested in two money market funds, classified in cash and cash equivalents on the accompanying balance sheet, corporate bonds and municipal bonds classified in short-term investments on the accompanying balance sheet and municipal bonds classified in long-term investments on the accompanying balance sheet.

Based on a hypothetical ten percent adverse movement in interest rates, the potential losses in future earnings, fair value of risk sensitive financial instruments, and cash flows are immaterial, although the actual effects may differ materially from the hypothetical analysis. 

ITEM 4. CONTROLS AND PROCEDURES.

 

(a) 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 defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act) as of September 30, 2016. In designing and evaluating our disclosure controls and procedures, management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives and our management necessarily applied its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Based on this evaluation, our principal executive officer and principal financial officer concluded that as of September 30, 2016, our disclosure controls and procedures were (1) designed to ensure that material information relating to us is made known to our principal executive officer and principal financial officer by others, particularly during the period in which this report was prepared, and (2) effective, in that they provide reasonable assurance that information required to be disclosed by us in the reports we file or submit under the Exchange Act is recorded, processed, summarized and reported within the time periods specified in the SEC’s rules and forms.

(b) Changes in Internal Controls. No change in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) occurred during the fiscal quarter ended September 30, 2016 that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.

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PART II — OTHER INFORMATION

ITEM 1A. RISK FACTORS.

RISK FACTORS

Investing in our securities involves a high degree of risk. You should carefully consider the risks and uncertainties described below in addition to the other information included or incorporated by reference in this Quarterly Report on Form 10-Q before purchasing our common stock. Our business, financial condition and results of operations could be materially and adversely affected by any of these and currently unknown risks or uncertainties. In that case, the market price of our common stock could decline, and you may lose all or part of your investment in our securities.

Risks Relating to Our Financial Results and Need for Financing

We will need additional financing, which may be difficult to obtain. Our failure to obtain necessary financing or doing so on unattractive terms could result in the termination of our operations and the sale and license of our assets or otherwise adversely affect our research and development programs and other operations.

We had cash, cash equivalents and investments of approximately $53.4 million at September 30, 2016. In October 2016, we received estimated net proceeds of approximately $48.9 million from a follow-on public offering, including the partial exercise by the underwriters of their option to purchase additional shares of the offering and after deducting underwriters’ discounts and commissions and estimated offering expenses.  We believe that, based on our current operating plan, our existing cash, cash equivalents and investments, including the net proceeds of the offering, will enable us to fund our operations into the first quarter of 2018. Specifically, we believe that our available funds will be sufficient to enable us to:

·

participate in an FDA End-of-Phase 1 meeting to obtain FDA feedback on the regulatory pathway for IMO-2125;

·

complete our ongoing Phase 1/2 clinical trial of IMO-2125 in combination with ipilimumab or pembrolizumab in anti-PD1 refractory metastatic melanoma;

·

prepare for the initiation of a pivotal Phase 3 clinical trial of IMO-2125 in combination with a checkpoint inhibitor for the treatment of anti-PD1 refractory metastatic melanoma;

·

initiate a Phase 1 intra-tumoral monotherapy clinical trial of IMO-2125 in multiple refractory tumor types;

·

initiate a Phase 2 multi-arm clinical trial of IMO-2125 in combination with a checkpoint inhibitor in multiple refractory tumor types;

·

complete our ongoing Phase 2 clinical trial of IMO-8400 in patients with dermatomyositis; and

·

submit an IND and initiate a Phase 1 human clinical proof-of-concept trial for one of our 3GA compounds.

We expect that we will need to raise additional funds in order to conduct any other clinical development of our TLR drug candidates or to conduct any other development of our 3GA technology, and to fund our operations. We are seeking and expect to continue to seek additional funding through collaborations, the sale or license of assets or financings of equity or debt securities. We believe that the key factors that will affect our ability to obtain funding are:

·

the results of our clinical and preclinical development activities in our rare disease program, our immuno-oncology program and our 3GA program, and our ability to advance our drug candidates and 3GA technology on the timelines anticipated;

·

the cost, timing, and outcome of regulatory reviews;

·

competitive and potentially competitive products and technologies and investors' receptivity to our drug candidates and the technology underlying them in light of competitive products and technologies;

·

the receptivity of the capital markets to financings by biotechnology companies generally and companies with drug candidates and technologies such as ours specifically; and

·

our ability to enter into additional collaborations with biotechnology and pharmaceutical companies and the success of such collaborations.

26


 

In addition, increases in expenses or delays in clinical development may adversely impact our cash position and require additional funds or cost reductions.

Financing may not be available to us when we need it or may not be available to us on favorable or acceptable terms or at all. We could be required to seek funds through collaborative alliances or through other means that may require us to relinquish rights to some of our technologies, drug candidates or drugs that we would otherwise pursue on our own. In addition, if we raise additional funds by issuing equity securities, our then existing stockholders will experience dilution. The terms of any financing may adversely affect the holdings or the rights of existing stockholders. An equity financing that involves existing stockholders may cause a concentration of ownership. Debt 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 are likely to include rights that are senior to the holders of our common stock. Any additional debt or equity financing may contain terms which are not favorable to us or to our stockholders, such as liquidation and other preferences, or liens or other restrictions on our assets. As discussed in Note 10 to the financial statements appearing in our Annual Report on Form 10-K for the year ended December 31, 2015 that was filed with the SEC, additional equity financings may also result in cumulative changes in ownership over a three-year period in excess of 50% which would limit the amount of net operating loss and tax credit carryforwards that we may utilize in any one year.

If we are unable to obtain adequate funding on a timely basis or at all, we will be required to terminate, modify or delay preclinical or clinical trials of one or more of our drug candidates, significantly curtail or terminate discovery or development programs for new drug candidates or relinquish rights to portions of our technology, drug candidates and/or products.

We have incurred substantial losses and expect to continue to incur losses. We will not be successful unless we reverse this trend.

We have incurred losses in every year since our inception, except for 2002, 2008, and 2009 when our recog