10-Q

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 10-Q

(Mark one)

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

For the quarterly period ended September 30, 2017

OR

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

For the transition period from to

Commission File Number 001-36177

GlycoMimetics, Inc.

(Exact name of registrant as specified in its charter)

Delaware	06-1686563
(State or Other Jurisdiction of Incorporation or Organization)	(I.R.S. Employer Identification No.)
9708 Medical Center Drive Rockville, Maryland	20850
(Address of principal executive offices)	(Zip Code)

(240) 243-1201

(Registrant’s telephone number, including area code)

N/A

(Former name, former address and former fiscal year, if changed since last report)

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 Website, 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, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

Large accelerated filer ◻                  Accelerated filer ☒Smaller reporting company ◻

Non-accelerated filer   ◻       (Do not check if a smaller reporting company)

                                                                Emerging growth company ☒

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☒



Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Securities Exchange Act of 1934).    Yes      No   ☒ 

The number of outstanding shares of the registrant’s common stock, par value $0.001 per share, as of the close of business on November 7, 2017 was 34,337,799.

GLYCOMIMETICS, INC.

INDEX TO FORM 10-Q

	PAGE
PART I. FINANCIAL INFORMATION
Item 1. Financial Statements	3
Balance Sheets as of September 30, 2017 (unaudited) and December 31, 2016	3
Unaudited Statements of Operations and Comprehensive Loss for the three and nine months ended September 30, 2017 and 2016	4
Unaudited Statements of Cash Flows for the nine months ended September 30, 2017 and 2016	5
Notes to Unaudited Financial Statements	6
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations	16
Item 3. Quantitative and Qualitative Disclosures about Market Risk	26
Item 4. Controls and Procedures	26
PART II. OTHER INFORMATION
Item 1. Legal Proceedings	27
Item 1A. Risk Factors	27
Item 2. Unregistered Sales of Equity Securities and Use of Proceeds	27
Item 6. Exhibits	28
Signatures	29

Part I. FINANCIAL INFORMATION

Item 1. Financial Statements

GLYCOMIMETICS, INC.

Balance Sheets

	September 30,		December 31,
	2017		2016
Assets	(Unaudited)
Current assets:
Cash and cash equivalents	$	112,872,534	$	40,041,641
Prepaid expenses and other current assets		1,015,747		478,503
Total current assets		113,888,281		40,520,144
Property and equipment, net		1,122,216		1,056,332
Prepaid research and development expenses		759,531		759,531
Deposits		52,320		52,320
Total assets	$	115,822,348	$	42,388,327
Liabilities & stockholders’ equity
Current liabilities:
Accounts payable	$	1,631,722	$	1,565,210
Accrued bonuses		1,146,497		1,432,485
Accrued expenses		3,678,609		3,267,371
Deferred rent		74,072		68,551
Total current liabilities		6,530,900		6,333,617
Deferred rent, net of current portion		726,986		753,579
Total liabilities		7,257,886		7,087,196
Stockholders’ equity:
Preferred stock; $0.001 par value; 5,000,000 shares authorized, no shares issued and outstanding at September 30, 2017 and December 31, 2016		—		—
Common stock; $0.001 par value; 100,000,000 shares authorized, 32,737,799 shares issued and outstanding at September 30, 2017; 100,000,000 shares authorized, 23,250,023 shares issued and outstanding at December 31, 2016		32,736		23,249
Additional paid-in capital		251,550,974		154,254,193
Accumulated deficit		(143,019,248)		(118,976,311)
Total stockholders’ equity		108,564,462		35,301,131
Total liabilities and stockholders’ equity	$	115,822,348	$	42,388,327

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

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GLYCOMIMETICS, INC.

Unaudited Statements of Operations and Comprehensive Loss

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Revenue	$	—	$	18,500	$	—	$	18,500
Costs and expenses:
Research and development expense		5,779,759		5,921,461		17,380,337		17,221,360
General and administrative expense		2,401,976		1,983,795		7,016,081		6,352,355
Total costs and expenses		8,181,735		7,905,256		24,396,418		23,573,715
Loss from operations		(8,181,735)		(7,886,756)		(24,396,418)		(23,555,215)
Other income		231,634		32,063		373,208		73,974
Net loss and comprehensive loss	$	(7,950,101)	$	(7,854,693)	$	(24,023,210)	$	(23,481,241)
Basic and diluted net loss per common share	$	(0.24)	$	(0.34)	$	(0.86)	$	(1.14)
Basic and diluted weighted average number of common shares		32,724,010		23,049,347		27,814,781		20,638,129

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

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GLYCOMIMETICS, INC.

Unaudited Statements of Cash Flows

	Nine Months Ended September 30,
	2017		2016
Operating activities
Net loss	$	(24,023,210)	$	(23,481,241)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation		193,976		139,749
Stock-based compensation expense		2,796,301		2,238,647
Changes in assets and liabilities:
Prepaid expenses and other current assets		(537,244)		(797,906)
Prepaid research and development expenses		—		(63,000)
Deposits		—		(52,320)
Accounts payable		66,512		682,504
Accrued expenses and bonuses		125,250		(3,122,258)
Deferred rent		(21,072)		679,177
Net cash used in operating activities		(21,399,487)		(23,776,648)
Investing activities
Purchases of property and equipment		(259,860)		(265,635)
Net cash used in investing activities		(259,860)		(265,635)
Financing activities
Proceeds from issuance of common stock, net of issuance costs		94,269,213		22,453,785
Proceeds from exercise of stock options		221,027		68,488
Net cash provided by financing activities		94,490,240		22,522,273
Net change in cash and cash equivalents		72,830,893		(1,520,010)
Cash and cash equivalents, beginning of period		40,041,641		46,802,560
Cash and cash equivalents, end of period	$	112,872,534	$	45,282,550
Non-cash investing activities
Property acquisition costs included in accounts payable and accrued expenses	$	—	$	29,329

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

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GLYCOMIMETICS, INC.

Notes to Unaudited Financial Statements

1. Description of the Business

GlycoMimetics, Inc. (the Company), a Delaware corporation headquartered in Rockville, Maryland, was incorporated on April 4, 2003 and commenced operations on May 21, 2003. The Company is a clinical stage biotechnology company focused on the discovery and development of novel glycomimetic drugs to address unmet medical needs resulting from diseases in which carbohydrate biology plays a key role. Glycomimetics are molecules that mimic the structure of carbohydrates involved in important biological processes. The Company’s proprietary glycomimetics platform is based on its expertise in carbohydrate chemistry and its understanding of the role carbohydrates play in key biological processes. Using this expertise and understanding, the Company is developing a pipeline of proprietary glycomimetics designed to inhibit disease-related functions of carbohydrates, such as the roles they play in inflammation, cancer and infection.

The Company’s executive personnel have devoted substantially all of their time to date to the planning and organization of the Company, the process of hiring scientists, initiating research and development programs and securing adequate capital for anticipated growth and operations. The Company has not commercialized any of its drug candidates or commenced commercial operations. The Company is subject to a number of risks similar to those of other companies in similar development stages, including dependence on key individuals, the need to develop commercially viable drugs, competition from other companies, many of whom are larger and better capitalized, and the need to obtain adequate additional financing to fund the development of its drug candidates. The Company has incurred significant operating losses since inception and has relied on its ability to fund its operations through private and public equity financings, and management expects operating losses and negative operating cash flows to continue for the foreseeable future. As the Company continues to incur losses, profitability will be dependent upon the successful development, approval, and commercialization of its drug candidates and achieving a level of revenues adequate to support the Company’s cost structure. The Company may never achieve profitability, and unless and until it does, the Company will continue to need to raise additional capital. Management intends to fund future operations through additional public or private equity or debt offerings and may seek additional capital through arrangements with strategic partners or from other sources.

2. Significant Accounting Policies

Basis of Accounting

The accompanying financial statements were prepared based on the accrual method of accounting in accordance with U.S. generally accepted accounting principles (GAAP).

Unaudited Financial Statements

The accompanying balance sheet as of September 30, 2017 and statements of operations and comprehensive loss for the three and nine months ended September 30, 2017 and 2016 and cash flows for the nine months ended September 30, 2017 and 2016 are unaudited. These unaudited financial statements have been prepared in accordance with the rules and regulations of the United States Securities and Exchange Commission (the SEC) for interim financial information. Accordingly, they do not include all of the information and footnotes required by GAAP for complete annual financial statements. These financial statements should be read in conjunction with the audited financial statements and the accompanying notes for the year ended December 31, 2016 contained in the Company’s Annual Report on Form 10-K filed with the SEC on March 1, 2017. The unaudited interim financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments (consisting of normal recurring adjustments) necessary to state fairly the Company’s financial position as of September 30, 2017, the results of operations for the three and nine months ended September 30, 2017 and 2016 and cash flows for the nine months ended September 30, 2017 and 2016. The December 31, 2016 balance sheet included herein was derived from audited financial statements, but does not include all disclosures including notes required by GAAP for complete annual financial statements. The financial data and other information disclosed in these notes to the financial statements related to the three and nine months ended September 30, 2017 and 2016 are unaudited. Interim results are not necessarily indicative of results for an entire year.

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Use of Estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting periods. Although actual results could differ from those estimates, management does not believe that such differences would be material.

Fair Value Measurements

The Company had no assets or liabilities that were measured using quoted prices for similar assets and liabilities or significant unobservable inputs (Level 2 and Level 3 assets and liabilities, respectively) as of September 30, 2017 and December 31, 2016. The carrying value of cash held in money market funds of approximately $110.9 million and $38.0 million as of September 30, 2017 and December 31, 2016, respectively, is included in cash and cash equivalents and approximates market values based on quoted market prices (Level 1 inputs).

Concentration of Credit Risk

Credit risk represents the risk that the Company would incur a loss if counterparties failed to perform pursuant to the terms of their agreements. Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash and cash equivalents. Cash and cash equivalents consist of money market funds with major financial institutions in the United States. These funds may be redeemed upon demand and, therefore, bear minimal risk. The Company does not anticipate any losses on such balances.

Revenue Recognition

From time to time, the Company is awarded reimbursement contracts for services and development grant contracts with government and non-government entities and philanthropic organizations. Under these contracts, the Company typically is reimbursed for the costs in connection with specific development activities. The Company recognizes revenue to the extent of costs incurred in connection with performance under such grant arrangements.

The Company has entered into a collaborative research and development agreement with Pfizer Inc. (Pfizer). The agreement is in the form of a license agreement (the Pfizer Agreement). The Pfizer Agreement calls for a nonrefundable up-front payment and milestone payments upon achieving significant milestone events. The Pfizer Agreement also contemplates royalty payments on future sales of an approved product. There are no performance, cancellation, termination, or refund provisions in the Pfizer Agreement that contain material financial consequences to the Company.

The primary deliverable under this arrangement is an exclusive worldwide license to the Company’s rivipansel compound, but the arrangement also includes deliverables related to research and preclinical development activities to be performed by the Company on Pfizer’s behalf.

Collaborative research and development agreements can provide for one or more of up-front license fees, research payments, and milestone payments. Agreements with multiple components (deliverables or items) are evaluated according to the provisions of Accounting Standards Codification (ASC) 605-25, Revenue Recognition—Multiple-Element Arrangements, to determine whether the deliverables can be separated into more than one unit of accounting. An item can generally be considered a separate unit of accounting if all of the following criteria are met: (1) the delivered item(s) has value to the customer on a stand-alone basis and (2) if the arrangement includes a general right of return relative to the delivered item(s) then delivery or performance of the undelivered item(s) is considered probable and substantially in control of the Company. Items that cannot be divided into separate units are combined with other units of accounting, as appropriate. Consideration received is allocated among the separate units based on selling price hierarchy. The selling price hierarchy for each deliverable is based on (i) vendor-specific objective evidence (VSOE), if available; (ii) third-party evidence (TPE) of selling price if VSOE is not available; or (iii) an estimated selling price, if neither VSOE nor TPE is available. Management was not able to establish VSOE or TPE for separate unit deliverables, as the Company does not have a history of entering into such arrangements or selling the individual deliverables within such arrangements separately. In addition, there may be significant differentiation in these arrangements, which indicates that comparable third-party pricing may not be available. Management determined that the selling price for the

7

deliverables within the Pfizer Agreement should be determined using its best estimate of selling price. The process of determining the best estimate of selling price involved significant judgment on the Company’s part and included consideration of multiple factors such as estimated direct expenses, other costs, and available clinical development data.

Pursuant to ASC 605-25, each required deliverable under the Pfizer Agreement is evaluated to determine whether it qualifies as a separate unit of accounting. Factors considered in this determination include the research capabilities of Pfizer, the proprietary nature of the license and know-how, and the availability of the Company’s glycomimetics technology research expertise in the general marketplace. Based on all relevant facts and circumstances and, most significantly, on the proprietary nature of the Company’s technology and the related proprietary nature of the Company’s research services, management concluded that stand-alone value does not exist for the license, and therefore, the license is not a separate unit of accounting under the Pfizer Agreement and will be combined with the research and development services (including participation on a joint steering committee). The Company has satisfied the deliverables under the Pfizer Agreement.

Pursuant to ASC 605-28, Revenue Recognition—Milestone Method, at the inception of agreements that include milestone payments, the Company evaluates whether each milestone is substantive and at risk to both parties on the basis of the contingent nature of the milestone. This evaluation includes an assessment of whether (a) the consideration is commensurate with either (1) the entity’s performance to achieve the milestone, or (2) the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the entity’s performance to achieve the milestone, (b) the consideration relates solely to past performance and (c) the consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. In making this assessment, the Company evaluates factors such as scientific, regulatory, commercial and other risks that must be overcome to achieve the respective milestone, the level of effort and investment required to achieve the respective milestone and whether the milestone consideration is reasonable relative to all deliverables and payment terms in the agreement.

Non-refundable development and regulatory milestones that are expected to be achieved as a result of the Company’s efforts during the period of substantial involvement are recognized as revenue upon the achievement of the milestone, assuming all other revenue recognition criteria are met. Milestones that are not considered substantive because the Company does not contribute effort to the achievement of such milestones are generally achieved after the period of substantial involvement and are recognized as revenue upon achievement of the milestone, as there are no undelivered elements remaining and no continuing performance obligation, assuming all other revenue recognition criteria are met. In May 2014, the Company recognized $15.0 million in revenue as a result of the first non-refundable milestone payment received from Pfizer. In June 2015, the Company recognized $20.0 million in revenue as a result of Pfizer dosing the first patient in the Phase 3 clinical trial of rivipansel, which triggered the second non-refundable milestone payment.

Accrued Liabilities

The Company is required to estimate accrued liabilities as part of the process of preparing its financial statements. The estimation of accrued liabilities involves identifying services that have been performed on the Company’s behalf, and then estimating the level of service performed and the associated cost incurred for such services as of each balance sheet date. Accrued liabilities include professional service fees, such as for lawyers and accountants, contract service fees, such as those under contracts with clinical monitors, data management organizations and investigators in conjunction with clinical trials, and fees to contract manufacturers in conjunction with the production of clinical materials. Pursuant to the Company’s assessment of the services that have been performed, the Company recognizes these expenses as the services are provided. Such assessments include: (i) an evaluation by the project manager of the work that has been completed during the period; (ii) measurement of progress prepared internally and/or provided by the third-party service provider; (iii) analyses of data that justify the progress; and (iv) the Company’s judgment.

Research and Development Costs

Except for payments made in advance of services, research and development costs are expensed as incurred. For payments made in advance, the Company recognizes research and development expense as the services are rendered. Research and development costs primarily consist of salaries and related expenses for personnel, laboratory supplies and raw materials, sponsored research, depreciation of laboratory facilities and leasehold improvements, and utilities costs

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related to research space. Other research and development expenses include fees paid to consultants and outside service providers including clinical research organizations and clinical manufacturing organizations.

Stock-Based Compensation

Stock-based payments are accounted for in accordance with the provisions of ASC 718, Compensation—Stock Compensation. The fair value of stock-based payments is estimated, on the date of grant, using the Black-Scholes-Merton model. The resulting fair value is recognized ratably over the requisite service period, which is generally the vesting period of the option.

The Company has elected to use the Black-Scholes-Merton option pricing model to value any options granted. The Company will reconsider use of the Black-Scholes-Merton model if additional information becomes available in the future that indicates another model would be more appropriate or if grants issued in future periods have characteristics that prevent their value from being reasonably estimated using this model.

A discussion of management’s methodology for developing some of the assumptions used in the valuation model follows:

Expected Dividend Yield—The Company has never declared or paid dividends and has no plans to do so in the foreseeable future.

Expected Volatility—Volatility is a measure of the amount by which a financial variable such as share price has fluctuated (historical volatility) or is expected to fluctuate (expected volatility) during a period. Prior to the Company’s initial public offering, there was not a market for the Company’s shares. The Company utilizes the historical volatilities of a peer group (e.g., several public entities of similar size, complexity, and stage of development), along with the Company’s historical volatility since its initial public offering, to determine its expected volatility.

Risk-Free Interest Rate—This is the U.S. Treasury rate for the week of each option grant during the year, having a term that most closely resembles the expected life of the option.

Expected Term—This is a period of time that the options granted are expected to remain unexercised. Options granted have a maximum term of 10 years. The Company estimates the expected life of the option term to be 6.25 years. The Company uses a simplified method to calculate the average expected term.

Expected Forfeiture Rate—Effective on January 1, 2017 with the adoption of ASU 2016-09, Compensation – Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting, the Company elected to account for forfeitures as they occur.

Net Loss Per Common Share

Basic net loss per common share is determined by dividing net loss by the weighted-average number of common shares outstanding during the period, without consideration of common stock equivalents. Diluted net loss per share is computed by dividing net loss by the weighted-average number of common stock equivalents outstanding for the period. The treasury stock method is used to determine the dilutive effect of the Company’s stock options, restricted stock units and warrants.

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Basic and diluted net loss per common share is computed as follows:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Net loss	$	(7,950,101)	$	(7,854,693)	$	(24,023,210)	$	(23,481,241)
Basic and diluted net loss per common share	$	(0.24)	$	(0.34)	$	(0.86)	$	(1.14)
Basic and diluted weighted average common shares outstanding		32,724,010		23,049,347		27,814,781		20,638,129

The following potentially dilutive securities outstanding have been excluded from the computation of diluted weighted average common shares outstanding, as they would be anti-dilutive:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Warrants		553,868		555,412		553,868		555,412
Stock options and restricted stock units		3,421,124		2,815,166		3,421,124		2,815,166

Comprehensive Loss

Comprehensive loss comprises net loss and other changes in equity that are excluded from net loss. For the three and nine months ended September 30, 2017 and 2016, the Company’s net loss equaled comprehensive net loss and, accordingly, no additional disclosure is presented.

Recently Issued Accounting Standards

Adopted Accounting Standards

In March 2016, the Financial Accounting Standards Board (FASB) issued ASU No. 2016-09, Compensation – Stock Compensation  (Topic 718): Improvements to Employee Share-Based Payment Accounting. This ASU includes provisions intended to simplify various aspects related to how share-based payments are accounted for and presented in financial statements including the income tax effects of share-based payments, minimum statutory withholding requirements and forfeitures. The new guidance requires all income tax effects of awards to be recognized in the income statement when the awards vest or are settled. It also allows an employer to repurchase more of an employee’s shares than the current standard for tax withholding purposes without triggering liability accounting and to make a policy election to account for forfeitures as they occur. The Company adopted the provisions of ASU 2016-09 on January 1, 2017. The Company has elected to account for forfeitures as they occur. The Company has applied this change using a modified retrospective method through a cumulative-effect adjustment of $19,727 to accumulated deficit. Additionally, the Company recognized deferred tax assets of $98,767 for the excess tax benefits that arose directly from tax deductions related to equity compensation greater than the amounts recognized for financial reporting and also recognized an increase of an equal amount in the valuation allowance against those deferred tax assets. The Company has adopted the additional provisions in the standard and has determined these provisions do not have a material impact on the financial statements.

Accounting Standards Not Yet Adopted

On May 28, 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers  (Topic 606), requiring an entity to recognize the amount of revenue to which it expects to be entitled for the transfer of promised goods or services to customers. The updated standard will replace most existing revenue recognition guidance in GAAP when it becomes effective and permits the use of either the retrospective or cumulative effect transition method. In August 2015, the FASB issued ASU No. 2015-14, Revenue from Contracts with Customers: Deferral of the Effective Date, which deferred the effective date of the new revenue standard for periods beginning after December 15, 2016 to December 15, 2017, with early adoption permitted but not earlier than the original effective date. Accordingly, the updated standard is effective for the Company in the first quarter of fiscal 2018. The Company has evaluated the Pfizer Agreement to determine the impact of the new revenue standard on the upfront and milestone payments within the Pfizer Agreement and has determined that the transition to the new revenue standard will have no material impact on the

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financial statements. The Company expects to adopt the new standard on January 1, 2018 using the full retrospective transition method.

In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842), which supersedes existing guidance on accounting for leases in Leases (Topic 840) and generally requires all leases, including operating leases, to be recognized in the statement of financial position as right-of-use assets and lease liabilities by lessees. The provisions of ASU 2016-02 are to be applied using a modified retrospective approach and are effective for reporting periods beginning after December 15, 2018; early adoption is permitted. The Company is currently evaluating the effect that this ASU will have on the financial statements.

With the exception of the new standards discussed above, there have been no new accounting pronouncements that have significance, or potential significance, to the Company’s financial statements.

3. Prepaid Expenses and Other Current Assets

The following is a summary of the Company’s prepaid expenses and other current assets:

	September 30,		December 31,
	2017		2016
Prepaid expenses	$	940,099	$	199,195
Other receivables		75,648		268,659
Deposits		—		10,649
Prepaid expenses and other current assets	$	1,015,747	$	478,503

4. Property and Equipment

Property and equipment, net consists of the following:

	September 30,		December 31,
	2017		2016
Furniture and fixtures	$	314,024	$	262,135
Laboratory equipment		1,282,655		1,130,180
Office equipment		11,084		6,610
Computer equipment		191,825		169,423
Leasehold improvements		573,165		36,128
Construction in progress		—		508,417
Property and equipment		2,372,753		2,112,893
Less accumulated depreciation		(1,250,537)		(1,056,561)
Property and equipment, net	$	1,122,216	$	1,056,332

Depreciation expense was $70,060 and $45,808 for the three months ended September 30, 2017 and 2016, respectively, and $193,976 and $139,749 for the nine months ended September 30, 2017 and 2016, respectively.

5. Accrued Expenses

The following is a summary of the Company’s accrued expenses:

	September 30,		December 31,
	2017		2016
Accrued research and development expenses	$	2,727,309	$	2,513,243
Accrued consulting and other professional fees		371,595		148,579
Other accrued expenses		187,067		315,002
Accrued employee benefits		392,638		290,547
Accrued expenses	$	3,678,609	$	3,267,371

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6. Operating Leases

The Company leases office and research space in Rockville, Maryland under an operating lease with a term through October 31, 2023 (as amended to date, the Lease) that is subject to annual rent increases. The Company has the right to sublease or assign all or a portion of the premises, subject to the conditions set forth in the Lease. The Lease may be terminated early by either the landlord or the Company in certain circumstances. In connection with the Lease, the Company received rent abatement as a lease incentive. The annual rent increases and rent abatement have been recognized as deferred rent that is being adjusted on a straight-line basis over the term of the Lease.

In March 2016, the Company amended the Lease (the Lease Amendment) to lease additional space as of June 1, 2016. In addition to the other terms of the Lease, the Lease Amendment provided for a tenant improvement allowance reflected in the Company’s financial statements as an increase in capitalized leasehold improvements as incurred and an increase in deferred rent. In May 2016, the Company also paid a security deposit of $52,320 to be held until the expiration or termination of the Company’s obligations under the Lease. The term of the Lease Amendment for the additional space continues through October 31, 2023, the same date as for the premises originally leased under the Lease, subject to the Company’s renewal option set forth in the Lease. The Company’s one-time option to terminate the Lease effective as of October 31, 2020 also applies to the additional space.

Deferred rent related to the Lease was $801,058 and $822,130 at September 30, 2017 and December 31, 2016, respectively. Total rent expense under the Company’s operating leases was $223,337 and $219,983 for the three months ended September 30, 2017 and 2016, respectively, and $667,507 and $557,262 for the nine months ended September 30, 2017 and 2016, respectively.

7. Stockholders’ Equity

At-The-Market Equity Offering

On March 1, 2016, the Company entered into an at-the-market sales agreement with Cowen and Company, LLC to sell the Company’s securities under a shelf registration statement filed in March 2015. The at-the market sales agreement was terminated on May 23, 2017. During the nine months ended September 30, 2017, the Company issued and sold 1,388,647 shares of common stock under the at-the-market sales agreement. The shares were sold at a weighted average price per share of $5.55, for aggregate net proceeds of $7.4 million, after deducting commissions and offering expenses.

On September 28, 2017, the Company entered into a new at-the-market sales agreement with Cowen and Company, LLC to sell the Company’s securities under a shelf registration statement filed in September 2017. Subsequent to September 30, 2017, the Company issued and sold 1,600,000 shares of common stock under the at-the-market sales agreement. The shares were sold at a weighted average price per share of $12.50, for aggregate net proceeds of $19.2 million, after deducting commissions and offering expenses.

Equity Offering

In May 2017, the Company completed a public offering in which the Company sold 8,050,000 shares of its common stock at a price to the public of $11.50 per share. The Company received net proceeds of $86.8 million from this offering, after deducting underwriting discounts, commissions and other offering expenses.

2003 Stock Incentive Plan

The 2003 Stock Incentive Plan (the 2003 Plan) provided for the grant of incentives and nonqualified stock options and restricted stock awards. The exercise price for incentive stock options must be at least equal to the fair value of the common stock on the grant date. Unless otherwise stated in a stock option agreement, 25% of the shares subject to an option grant will vest upon the first anniversary of the vesting start date and thereafter at the rate of one forty-eighth of the option shares per month as of the first day of each month after the first anniversary. Upon termination of employment by reasons other than death, cause, or disability, any vested options shall terminate 60 days after the termination date. Stock options terminate 10 years from the date of grant. The 2003 Plan expired on May 21, 2013.

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A summary of the Company’s stock option activity under the 2003 Plan for the nine months ended September 30, 2017 is as follows:

				Weighted-Average
				Remaining	Aggregate
	Outstanding	Weighted-Average		Contractual Term	Intrinsic Value
	Options	Exercise Price		(Years)	(In thousands)
Outstanding as of December 31, 2016	729,819	$	1.26	3.2
Options exercised	(16,608)		1.65
Options forfeited	—		—
Outstanding as of September 30, 2017	713,211		1.25	2.4	$	9,089
Vested as of September 30, 2017	713,211		1.25	2.4	$	9,089
Exercisable as of September 30, 2017	713,211		1.25	2.4	$	9,089

As of September 30, 2017, the options under the 2003 Plan were fully expensed. Total intrinsic value of the options exercised during the nine months ended September 30, 2017 and 2016 was $103,638 and $95,221, respectively, and total cash received for options exercised was $27,358 and $40,668 during the nine months ended September 30, 2017 and 2016, respectively. The total fair value of shares underlying options which vested in the nine months ended September 30, 2017 and 2016 was $1,573 and $14,450, respectively.

2013 Equity Incentive Plan

The Company’s board of directors adopted, and its stockholders approved, its 2013 Equity Incentive Plan (the 2013 Plan) effective on January 9, 2014. The 2013 Plan provides for the grant of incentive stock options within the meaning of Section 422 of the Internal Revenue Code to the Company’s employees and its parent and subsidiary corporations’ employees, and for the grant of nonstatutory stock options, restricted stock awards, restricted stock unit awards, stock appreciation rights, performance stock awards and other forms of stock compensation to its employees, including officers, consultants and directors. The 2013 Plan also provides for the grant of performance cash awards to the Company’s employees, consultants and directors. Unless otherwise stated in a stock option agreement, 25% of the shares subject to an option grant will typically vest upon the first anniversary of the vesting start date and thereafter at the rate of one forty-eighth of the option shares per month as of the first day of each month after the first anniversary. Upon termination of employment by reasons other than death, cause, or disability, any vested options will terminate 90 days after the termination date, unless otherwise set forth in a stock option agreement. Stock options generally terminate 10 years from the date of grant.

Authorized Shares

The maximum number of shares of common stock that initially could be issued under the 2013 Plan was  1,000,000 shares, plus any shares subject to stock options or similar awards granted under the 2003 Plan that expire or terminate without having been exercised in full or are forfeited to or repurchased by the Company. The number of shares of common stock reserved for issuance under the 2013 Plan automatically increases on January 1 of each year until January 1, 2023, by 3% of the total number of shares of common stock outstanding on December 31 of the preceding calendar year, or a lesser number of shares as may be determined by the Company’s board of directors. The maximum number of shares that may be issued pursuant to exercise of incentive stock options under the 2013 Plan is 20,000,000 shares. As of January 1, 2017, the number of shares of common stock that may be issued under the 2013 Plan was automatically increased by 697,500 shares, representing 3% of the total number of shares of common stock outstanding on December 31, 2016, increasing the number of shares of common stock available for issuance under the 2013 Plan to 2,837,201 shares.

Shares issued under the 2013 Plan may be authorized but unissued or reacquired shares of common stock. Shares subject to stock awards granted under the 2013 Plan that expire or terminate without being exercised in full, or that are paid out in cash rather than in shares, will not reduce the number of shares available for issuance under the 2013 Plan. Additionally, shares issued pursuant to stock awards under the 2013 Plan that the Company repurchases or that are forfeited, as well as shares reacquired by the Company as consideration for the exercise or purchase price of a stock

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award or to satisfy tax withholding obligations related to a stock award, will become available for future grant under the 2013 Plan.

A summary of the Company’s stock option activity under the 2013 Plan for the nine months ended September 30, 2017 is as follows:

				Weighted-Average
				Remaining	Aggregate
	Outstanding	Weighted-Average		Contractual Term	Intrinsic Value
	Options	Exercise Price		(Years)	(In thousands)
Outstanding as of December 31, 2016	2,066,105	$	7.41	7.9
Options granted	674,738		7.03
Options exercised	(32,521)		5.96
Options forfeited	(22,159)		7.35
Outstanding as of September 30, 2017	2,686,163		7.33	7.6	$	17,884
Vested or expected to vest as of September 30, 2017	2,686,163		7.33	7.6	$	17,884
Exercisable as of September 30, 2017	1,453,937		7.84	6.7	$	9,003

The weighted-average fair value of the options granted during the nine months ended September 30, 2017 and 2016 was $4.76 per share and $3.38 per share, respectively, applying the Black-Scholes-Merton option pricing model utilizing the following weighted-average assumptions:

	Nine Months Ended	Nine Months Ended
	September 30, 2017	September 30, 2016
Expected term	6.25 years	6.25 years
Expected volatility	75.20%	68.86%
Risk-free interest rate	2.08%	1.69%
Expected dividend yield	0%	0%

As of September 30, 2017, there was $4,854,266 of total unrecognized compensation expense related to unvested options under the 2013 Plan that will be recognized over a weighted-average period of approximately 2.2 years. Total intrinsic value of the options exercised during the nine months ended September 30, 2017 and 2016 was $228,291 and $2,275, respectively, and total cash received for options exercised was $193,669 and $27,825 during the nine months ended September 30, 2017 and 2016, respectively. The total fair value of shares underlying options which vested in the nine months ended September 30, 2017 and 2016 was $2,872,004 and $2,364,683, respectively.

A restricted stock unit (RSU) is a stock award that entitles the holder to receive shares of the Company’s common stock as the award vests. The fair value of each RSU is based on the closing price of the Company’s stock on the date of grant. The Company has granted RSUs with service conditions (service RSUs) that vest in three equal annual installments provided that the employee remains employed with the Company. As of September 30, 2017, there was $30,763 of unrecognized compensation costs related to unvested service RSUs.

The following is a summary of RSU activity under the 2013 Plan for the nine months ended September 30, 2017:

			Weighted-Average
		Number	Grant Date
	of Shares			Fair Value
Unvested at December 31, 2016		16,916	$	5.04
Granted		—		—
Forfeited		—		—
Vested		4,833		4.61
Unvested at September 30, 2017		12,083		5.21

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Stock-based compensation expense was classified on the statement of operations as follows for the three and nine months ended September 30, 2017 and 2016:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Research and development expense	$	327,133	$	260,882	$	953,821	$	772,163
General and administrative expense		635,212		478,531		1,842,480		1,466,484
Total stock-based compensation expense	$	962,345	$	739,413	$	2,796,301	$	2,238,647

8. Income Taxes

The Company has not recorded any tax provision or benefit for the nine months ended September 30, 2017 and 2016. The Company has provided a valuation allowance for the full amount of its net deferred tax assets since realization of any future benefit from deductible temporary differences, net operating loss carryforwards and research and development credits is not more-likely-than-not to be realized at September 30, 2017 and December 31, 2016.

9. Research and License Agreements

In October 2011, the Company and Pfizer entered into the Pfizer Agreement that provides Pfizer an exclusive worldwide license to rivipansel for vaso-occlusive crisis associated with sickle cell disease and for other diseases for which the drug candidate may be developed. The Company was responsible for completion of the Phase 2 clinical trial, after which Pfizer assumed all further development and commercialization responsibilities. Upon execution of the Pfizer Agreement, the Company received an up-front payment of $22.5 million. The Pfizer Agreement also provides for potential milestone payments of up to $115.0 million upon the achievement of specified development milestones, including the dosing of the first patients in Phase 3 clinical trials for up to two indications and the first commercial sale of a licensed product in the United States and selected European countries for up to two indications; potential milestone payments of up to $70.0 million upon the achievement of specified regulatory milestones, including the acceptance of our filings for regulatory approval by regulatory authorities in the United States and Europe for up to two indications; and potential milestone payments of up to $135.0 million upon the achievement of specified levels of annual net sales of licensed products. Pfizer has the right to terminate the Pfizer Agreement by giving prior written notice.

The Company has determined that each potential future clinical, development and regulatory milestone is substantive. Although sales-based milestones are not considered substantive, they are still recognized upon achievement of the milestone (assuming all other revenue recognition criteria have been met) because there are no undelivered elements that would preclude revenue recognition at that time. The Company is also eligible to receive royalties on future sales contingent upon annual net sales thresholds. In addition, the Company and Pfizer have formed a joint steering committee that will oversee and coordinate activities as set forth in the research program. The $22.5 million up-front payment was recognized over a period of 1.5 years. In May 2014, Pfizer made a $15.0 million non-refundable milestone payment to the Company, which was recognized as revenue by the Company in May 2014 when earned.  In June 2015, Pfizer dosed the first patient in the Phase 3 clinical trial of rivipansel, which triggered a non-refundable milestone payment to the Company of $20.0 million, which the Company recognized as revenue in June 2015. The Company did not recognize any revenue under the Pfizer Agreement during the three and nine months ended September 30, 2017 or 2016.

In February 2004, the Company entered into a research services agreement (the Research Agreement) with the University of Basel (the University) for biological evaluation of selectin antagonists. Certain patents covering the rivipansel compound are subject to provisions of the Research Agreement. Under the terms of the Research Agreement, the Company will owe to the University 10% of all future milestone and royalty payments received from Pfizer with respect to rivipansel. There were no milestone payments due to the University for the three and nine months ended September 30, 2017 or 2016.

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

Certain statements contained in this Quarterly Report on Form 10-Q may constitute 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. The words or phrases “would be,” “will allow,” “intends to,” “will likely result,” “are expected to,” “will continue,” “is anticipated,” “estimate,” “project,” or similar expressions, or the negative of such words or phrases, are intended to identify “forward-looking statements.” We have based these forward-looking statements on our current expectations and projections about future events. Because such statements include risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that could cause or contribute to these differences include those below and elsewhere in this Quarterly Report on Form 10-Q, our Annual Report on Form 10-K, particularly in Part I – Item 1A, “Risk Factors,” and our other filings with the Securities and Exchange Commission. Statements made herein are as of the date of the filing of this Form 10-Q with the Securities and Exchange Commission and should not be relied upon as of any subsequent date. Unless otherwise required by applicable law, we do not undertake, and we specifically disclaim, any obligation to update any forward-looking statements to reflect occurrences, developments, unanticipated events or circumstances after the date of such statement.

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our unaudited financial statements and related notes that appear in Item 1 of this Quarterly Report on Form 10-Q and with our audited financial statements and related notes for the year ended December 31, 2016, which are included in our Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 1, 2017.

Overview

We are a clinical-stage biotechnology company focused on the discovery and development of novel glycomimetic drugs to address unmet medical needs resulting from diseases in which carbohydrate biology plays a key role. Glycomimetics are molecules that mimic the structure of carbohydrates involved in important biological processes. Our proprietary glycomimetics platform is based on our expertise in carbohydrate chemistry and our understanding of the role carbohydrates play in key biological processes. Using this expertise and understanding, we are developing a pipeline of proprietary glycomimetics designed to inhibit disease-related functions of carbohydrates, such as the roles they play in inflammation, cancer and infection. We believe this represents an innovative approach to drug discovery to treat a wide range of diseases.

Most human proteins are modified by the addition of complex carbohydrates to the surface of the proteins. The addition of these carbohydrate structures affects the functions of these proteins and their interactions with other molecules. Our initial research and development efforts have focused on drug candidates targeting selectins, which are proteins that serve as adhesion molecules and bind to carbohydrates that are involved in the inflammatory component and progression of a wide range of diseases, including hematologic disorders, cancer and cardiovascular disease. Inhibiting specific carbohydrates from binding to selectins has long been viewed as a potentially attractive approach for therapeutic intervention. The ability to successfully develop drug-like compounds that inhibit binding with selectins, known as selectin antagonists, has been limited by the complexities of carbohydrate chemistry. We believe our expertise in carbohydrate chemistry enables us to design selectin antagonists and other glycomimetics that inhibit the disease-related functions of certain carbohydrates.

We are focusing our initial efforts on drug candidates for rare diseases that we believe will qualify for orphan drug designation. Our first drug candidate, rivipansel, is a pan-selectin antagonist being developed for the treatment of vaso-occlusive crisis, or VOC, a debilitating and painful condition that occurs periodically throughout the life of a person with sickle cell disease. We have entered into an agreement with Pfizer Inc., or Pfizer, for the further development and potential commercialization of rivipansel worldwide. Rivipansel has received fast track designation from the U.S. Food and Drug Administration, or FDA, as well as orphan drug designation from the FDA in the United States and from the European Medicines Agency, or EMA, in the European Union. We believe the clinical progress of rivipansel provides evidence of the significant potential of our lead program and our proprietary glycomimetics platform.

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Building on our experience with rivipansel, we are developing a pipeline of other glycomimetic drug candidates. Our second glycomimetic drug candidate, GMI-1271, is a specific E-selectin inhibitor, which we are developing to be used in combination with chemotherapy to treat patients with either acute myeloid leukemia, or AML, or multiple myeloma, or MM, both of which are life-threatening hematologic cancers, and potentially other hematologic cancers as well. We have completed enrollment in a Phase 1/2 clinical trial of GMI-1271 as an adjunct to standard chemotherapy in patients with AML and are currently conducting a Phase 1 clinical trial of GMI-1271 combined with chemotherapy for the treatment of MM. In the Phase 2 portion of our AML clinical trial, AML patients treated with GMI-1271, combined with chemotherapy, have experienced higher-than-expected remission rates and lower-than-expected induction-related mortality rates. We believe the data generated in this Phase 1/2 clinical trial support conducting one or more additional clinical trials of GMI-1271 in AML, with the ultimate goal of obtaining marketing approval from the FDA.

GMI-1271 received orphan drug designation from the FDA in May 2015 for the treatment of AML. In June 2016, GMI-1271 received fast track designation from the FDA for the treatment of adult patients with relapsed or refractory AML and elderly patients aged 60 years or older with AML. In May 2017, GMI-1271 received Breakthrough Therapy designation from the FDA for the treatment of adult patients with relapsed or refractory AML. In May 2017, the European Commission, based on a favorable recommendation from the EMA Committee for Orphan Medicinal Products, granted orphan designation for GMI-1271 for the treatment of AML.

In May 2015, we commenced our multinational, Phase 1/2, open-label trial of GMI-1271 as an adjunct to standard chemotherapy in patients with AML. This trial in males and females with AML enrolled patients at a number of academic institutions in the United States, Ireland and Australia. The trial consists of two parts. In the Phase 1 portion, escalation testing was performed to determine a recommended GMI-1271 dose in combination with standard chemotherapy to be used in the Phase 2 portion. In the Phase 2 portion of the trial, dose expansion was performed at the recommended dose of 10 mg/kg GMI-1271 in combination with standard chemotherapy.  The primary objective of the trial was to evaluate the safety of GMI-1271 in combination with chemotherapy. Secondary objectives were to characterize pharmacokinetics, or PK, pharmacodynamics, or PD, and to observe anti-leukemic activity. There were a total of 19 patients with relapsed or refractory AML enrolled and dosed with a single cycle of treatment with GMI-1271 and chemotherapy in the Phase 1 portion of the trial.

In the Phase 2 portion, one cohort of 25 patients over 60 years of age with newly diagnosed AML and a second cohort comprised of 47 patients with relapsed or refractory AML were enrolled.  Unlike in the Phase 1 portion, some of the patients in the Phase 2 portion were treated with multiple cycles of GMI-1271. In May 2017, we completed enrollment of 91 patients in the Phase 1/2 trial. We plan to provide additional updates from this clinical trial in the fourth quarter of 2017 and in 2018. We are consulting with the FDA regarding the design of a potential Phase 3 pivotal trial that could support an application for marketing approval for GMI-1271 for the treatment of AML. Based on the feedback from the FDA, we plan to initiate this pivotal trial in mid-2018.

In June 2017, we presented new data from the Phase 2 portion of the trial at the June 2017 annual meetings of the American Society of Clinical Oncology, or ASCO, and the European Hematology Association, or EHA. In the relapsed or refractory disease arm of the trial, 66 patients had been enrolled. Of the 54 relapsed/refractory patients with AML for whom data is available, the CR/CRi rate was 41%. The CR/CRi rate is the percentage of patients who achieved remission, with either full or incomplete blood count recovery. The mortality rate among this group at 60 days was 7%. Median overall survival of the 19 patients enrolled in the Phase 1 portion of the trial was 7.6 months. We believe these results compare favorably to what would be expected in this population, based on published historical controls in similar patients. In the newly-diagnosed, treatment-naïve elderly arm of the trial, 25 patients had been enrolled. Among these 25 patients, the CR/CRi rate was 68%, with a 73% rate for patients with de novo disease and 64% for patients with secondary AML.

Our data from the GMI-1271 program has been selected for two oral presentations at the upcoming annual meeting of the American Society of Hematology, or ASH, to be held in December 2017. One of these presentations will be an update on the current data set from the Phase 1/2 open-label trial of GMI-1271 as an adjunct to standard chemotherapy in patients with AML. The second oral presentation will present data from preclinical studies in which E-selectin was upregulated in a model of AML and increased resistance to chemotherapy, which we believe can be reversed by treatment with GMI-1271.

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In December 2015, at the ASH annual meeting, we presented preclinical data suggesting that GMI-1271 could reverse resistance of certain chemotherapies seen in MM. In September 2016, we dosed the first patient in a Phase 1 multiple dose-escalation clinical trial in defined populations of patients with MM who have not responded optimally to standard chemotherapy. In this trial, we are evaluating the efficacy, safety and PK of GMI-1271, combined with bortezomib- or carfilzomib-based chemotherapy, for the treatment of MM. We are currently enrolling patients at clinical trial sites in Ireland and have initiated the enrollment process at additional sites in Europe.

In addition to GMI-1271, we have designed a family of small molecule drug candidates that simultaneously inhibit both E-selectin and CXCR4. We have selected one of these compounds, GMI-1359, for development as a potential treatment for certain malignancies. Since E-selectin and CXCR4 are both adhesion molecules that keep cancer cells in the bone marrow, we believe that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that affect the bone marrow such as AML and MM, as compared to targeting CXCR4 alone. In December 2016 at the ASH annual meeting, we presented preclinical data suggesting that GMI-1359 has a unique tumor cell mobilization kinetic profile and enhanced the ability of chemotherapy to target and improve survival from a high-risk form of mutated AML.

In 2016, we completed enrollment in a Phase 1 single-dose escalation trial of GMI-1359 in healthy volunteers. In this trial, volunteer participants received a single injection of GMI-1359, after which they were evaluated for safety, tolerability, PK and PD. The randomized, double-blind, placebo-controlled, escalating dose study was conducted at a single site in the United States. We are currently expanding enrollment in this trial with two additional cohorts and anticipate selecting an initial cancer indication for this drug candidate and determining an optimal dose for further clinical evaluation in 2018.

Using our glycomimetics platform, we have also designed inhibitors that specifically block the binding of galectin-3 to carbohydrate structures. Galectin-3 is a protein that is known to play critical roles in many pathological processes, including fibrosis, inflammation, cancer and cardiovascular disease. We plan to optimize these compounds and conduct preclinical experiments in the fourth quarter of 2017 and in 2018 to further characterize the effects of galectin-3 inhibitors on immune processes and anti-fibrotic activity. We are also designing other galectin inhibitors that we believe could be used to treat various diseases.

We commenced operations in 2003, and our operations to date have been limited to organizing and staffing our company, business planning, raising capital, developing our glycomimetics platform, identifying potential drug candidates, undertaking preclinical studies and conducting clinical trials of rivipansel, GMI-1271 and GMI-1359. To date, we have financed our operations primarily through private placements of our securities, upfront and milestone payments under our collaboration with Pfizer and the net proceeds from our IPO in January 2014, at-the-market sales facilities with Cowen and Company LLC, or Cowen, and our public offerings of common stock in June 2016 and May 2017. We have no approved drugs currently available for sale, and substantially all of our revenue to date has been revenue from the upfront and milestone payments from Pfizer, although we have received nominal amounts of revenue under research grants.

Prior to our IPO, we raised an aggregate of $86.6 million to fund our operations, of which $22.5 million was an upfront payment under our collaboration with Pfizer and $64.1 million was from the sale of our convertible promissory notes and convertible preferred stock. The IPO provided us with net proceeds of $57.2 million, and we received a non-refundable milestone payment from Pfizer in May 2014 of $15.0 million. In August 2015, we received another non-refundable milestone payment from Pfizer of $20.0 million following the dosing of the first patient in the Phase 3 clinical trial of rivipansel. We received an additional $19.7 million in net proceeds from our public offering in June 2016 and $86.8 million in net proceeds from our public offering in May 2017. In 2016 and to date in 2017 we have received an aggregate of $30.5 million of net proceeds under the at-the-market facilities with Cowen.

Since inception, we have incurred significant operating losses. We have generated cumulative revenue of $58.6 million since our inception through September 30, 2017, primarily consisting of the $22.5 million upfront payment from Pfizer in 2011, the $15.0 million non-refundable milestone payment in May 2014 and the $20.0 million non-refundable milestone payment in August 2015. We had an accumulated deficit of $135.1 million as of September 30, 2017, and we expect to continue to incur significant expenses and operating losses over at least the next several years. Our net losses may fluctuate significantly from quarter to quarter and year to year, depending on the timing of our clinical trials, the

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receipt of milestone payments, if any, under our collaboration with Pfizer, and our expenditures on other research and development activities. We anticipate that our expenses will increase substantially as we:

· initiate and conduct our planned clinical trials of GMI-1271 and GMI-1359;

· continue the research and development of our other drug candidates;

· seek to discover and develop additional drug candidates;

· seek regulatory approvals for any drug candidates other than rivipansel that successfully complete clinical trials;

· ultimately establish a sales, marketing and distribution infrastructure and scale up external manufacturing capabilities to commercialize any drug candidates other than rivipansel for which we may obtain regulatory approval;

· maintain, expand and protect our intellectual property portfolio;

· hire additional clinical, quality control and scientific personnel; and

· add operational, financial and management information systems and personnel, including personnel to support our drug development and potential future commercialization efforts.

To fund further operations, we will need to raise capital. We may obtain additional financing in the future through the issuance of our common stock, through other equity or debt financings or through collaborations or partnerships with other companies. We may not be able to raise additional capital on terms acceptable to us, or at all, and any failure to raise capital as and when needed could compromise our ability to execute on our business plan. Although it is difficult to predict future liquidity requirements, we believe that our existing cash and cash equivalents, together with interest thereon, will be sufficient to fund our operations at least through the second half of 2019. However, our ability to successfully transition to profitability will be dependent upon achieving a level of revenues adequate to support our cost structure. We cannot assure you that we will ever be profitable or generate positive cash flow from operating activities.

Our Collaboration with Pfizer

In October 2011, we entered into the license agreement with Pfizer under which we granted Pfizer an exclusive worldwide license to develop and commercialize products containing rivipansel for all fields and uses. The license also covers specified back-up compounds along with modifications of and improvements to rivipansel that meet defined chemical properties. Pfizer is required to use commercially reasonable efforts, at its expense, to develop, obtain regulatory approval for and commercialize rivipansel for sickle cell disease in the United States. Under the terms of the agreement, we received a $22.5 million upfront payment. We are also eligible to earn potential milestone payments of up to $115.0 million upon the achievement of specified development milestones, including the dosing of the first patients in Phase 3 clinical trials for up to two indications and the first commercial sale of a licensed product in the United States and selected European countries for up to two indications, up to $70.0 million upon the achievement of specified regulatory milestones, including the acceptance of our filings for regulatory approval by regulatory authorities in the United States and Europe for up to two indications, and up to $135.0 million upon the achievement of specified levels of annual net sales of licensed products. We are also eligible to receive tiered royalties for each licensed product, with percentages ranging from the low double digits to the low teens, based on net sales worldwide, subject to reductions in specified circumstances.

The first potential milestone payment under the Pfizer agreement was $35.0 million upon the initiation of dosing of the first patient in a Phase 3 clinical trial of rivipansel by Pfizer. Under the collaboration, Pfizer made a $15.0 million non-refundable milestone payment to us in May 2014, which we recognized as revenue in May 2014, when earned, and the dosing of the first patient in the Phase 3 clinical trial in June 2015 triggered the remaining $20.0 million milestone payment to us.  We recorded the $20.0 million milestone payment as revenue in June 2015. There were no milestone payments received from Pfizer for the three or nine months ended September 30, 2017.

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We entered into a research services agreement with the University of Basel, or the University,  for biological evaluation of selectin antagonists. Certain patents covering the rivipansel compound are subject to provisions of the Research Agreement. Under the terms of the Research Agreement, we will owe to the University 10% of all future milestone and royalty payments received from Pfizer with respect to rivipansel. In February 2016, we paid $2.0 million to the University based upon the non-refundable milestone payments from Pfizer. We recorded these payments during the year ended December 31, 2015, at the time the payments became due to the University. There were no additional payments due to the University for the three months or nine months ended September 30, 2017.

Critical Accounting Policies and Significant Judgments and Estimates

There have been no material changes in our critical accounting policies, estimates and judgments during the nine months ended September 30, 2017 compared to the disclosures in Part II, Item 7 of our Annual Report on Form 10-K for the year ended December 31, 2016.

Components of Operating Results

Revenue

To date, we have not generated any revenue from the sale of our drug candidates and do not expect to generate any revenue from the sale of drugs in the near future. Substantially all of our revenue recognized to date has consisted of the upfront and milestone payments under our agreement with Pfizer.

Since our inception, we have also recognized a nominal amount of revenue under research grant contracts, generally to the extent of our costs incurred in connection with specific research or development activities.

Research and Development

Research and development expenses consist of expenses incurred in performing research and development activities, including compensation and benefits for full-time research and development employees, facilities expenses, overhead expenses, cost of laboratory supplies, clinical trial and related clinical manufacturing expenses, fees paid to contract research organizations and other consultants and other outside expenses. Other preclinical research and platform programs include activities related to exploratory efforts, target validation, lead optimization for our earlier programs and our proprietary glycomimetics platform.

To date, our research and development expenses have related primarily to the development of rivipansel and our other drug candidates. In April 2013, when we completed our Phase 2 clinical trial of rivipansel, all further clinical development obligations associated with rivipansel shifted to Pfizer.

We do not currently utilize a formal time allocation system to capture expenses on a project-by-project basis because we are organized and record expense by functional department and our employees may allocate time to more than one development project. Accordingly, we only allocate a portion of our research and development expenses by functional area and by drug candidate.

Research and development costs are expensed as incurred. Non-refundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized. The capitalized amounts are expensed as the related goods are delivered or the services are performed.

Research and development activities are central to our business model. Drug candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later stage clinical trials. We expect our research and development expenses to increase over the next several years as we seek to progress GMI-1271, GMI-1359 and our other drug candidates through clinical development. For example, as we prepare to potentially submit an application for marketing approval for GMI-1271, we will incur substantial expenses in scaling up the production and manufacturing of GMI-1271. However, it is difficult to determine with certainty the duration and completion costs of our current or future preclinical studies and clinical trials of our drug candidates, or if, when or to what extent we will generate revenues from the commercialization

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and sale of any of our drug candidates that obtain regulatory approval. We may never succeed in achieving regulatory approval for any of our drug candidates.

The duration, costs and timing of clinical trials and development of our drug candidates will depend on a variety of factors that include:

· per patient trial costs;

· the number of patients that participate in the trials;

· the number of sites included in the trials;

· the countries in which the trial is conducted;

· the length of time required to enroll eligible patients;

· the number of doses that patients receive;

· the drop-out or discontinuation rates of patients;

· potential additional safety monitoring or other studies requested by regulatory agencies;

· the duration of patient follow-up; and

· the safety and efficacy profile of the drug candidate.

In addition, the probability of success for each drug candidate will depend on numerous factors, including competition, manufacturing capability and commercial viability. We will determine which programs to pursue and how much to fund each program in response to the scientific and clinical success of each drug candidate, as well as an assessment of each drug candidate’s commercial potential.

General and Administrative

General and administrative expenses consist primarily of salaries and other related costs, including stock-based compensation, for personnel in executive, finance, accounting, business development and human resources functions. Other significant costs include facility costs not otherwise included in research and development expenses, legal fees relating to patent and corporate matters and fees for accounting and consulting services. We anticipate that our general and administrative expenses will increase in the future to support our continued research and development activities.

Other Income

Other income consists of interest income earned on our cash and cash equivalents.

21

Results of Operations for the Three and Nine Months Ended September 30, 2017 and 2016

The following tables set forth our results of operations for the three and nine months ended September 30, 2017 and 2016:

	Three Months Ended September 30,				Period-to-Period
(in thousands)	2017		2016		Change
Revenue	$	—	$	18	$	(18)
Costs and expenses:
Research and development expense		5,780		5,921		(141)
General and administrative expense		2,402		1,984		418
Total costs and expenses		8,182		7,905		277
Loss from operations		(8,182)		(7,887)		(295)
Other income		232		32		200
Net loss and comprehensive loss	$	(7,950)	$	(7,855)	$	(95)

	Nine Months Ended September 30,				Period-to-Period
(in thousands)	2017		2016		Change
Revenue	$	—	$	18	$	(18)
Costs and expenses:
Research and development expense		17,380		17,221		159
General and administrative expense		7,016		6,352		664
Total costs and expenses		24,396		23,573		823
Loss from operations		(24,396)		(23,555)		(841)
Other income		373		74		299
Net loss and comprehensive loss	$	(24,023)	$	(23,481)	$	(542)

Research and Development Expense

The following tables summarize our research and development expense by functional area for the three and nine months ended September 30, 2017 and 2016:

	Three Months Ended September 30,				Period-to-Period
(in thousands)	2017		2016		Change
Clinical development	$	1,109	$	2,389	$	(1,280)
Manufacturing and formulation		1,893		893		1,000
Contract research services, consulting and other costs		308		332		(24)
Laboratory costs		499		466		33
Personnel-related		1,644		1,580		64
Stock-based compensation		327		261		66
Research and development expense	$	5,780	$	5,921	$	(141)

	Nine Months Ended September 30,				Period-to-Period
(in thousands)	2017		2016		Change
Clinical development	$	4,392	$	5,403	$	(1,011)
Manufacturing and formulation		4,165		3,780		385
Contract research services, consulting and other costs		1,196		1,354		(158)
Laboratory costs		1,456		1,237		219
Personnel-related		5,217		4,675		542
Stock-based compensation		954		772		182
Research and development expense	$	17,380	$	17,221	$	159

22

During the three months ended September 30, 2017, our research and development expense decreased by $141,000, or 2%, compared to the same period in 2016. The decrease was primarily caused by lower clinical trial expenses related to the Phase 2 clinical trial of GMI-1271 for the treatment of AML due to patient enrollment completion in May 2017 and a decrease in costs for non-clinical toxicology studies and clinical studies for GMI-1359. These decreases were offset in part by additional costs related to the manufacturing of Phase 3 clinical supplies of GMI-1271. Personnel-related and stock-based compensation expenses increased due to annual salary adjustments for research and development personnel and annual stock option awards granted in the first quarter of 2017. During the nine months ended September 30, 2017, our research and development expense increased by $159,000, or 1%, compared to the same period in 2016.  The increase was caused by the ongoing costs associated with the clinical trials for GMI-1271 in AML and MM, partially offset by a decrease in expenses related to non-clinical toxicology and clinical studies and manufacturing and process development for GMI-1359. Personnel-related and stock-based compensation expenses increased due to annual salary adjustments for research and development personnel and annual stock option awards granted in the first quarter of 2017.

The following tables summarize our research and development expense by drug candidate for the three and nine months ended September 30, 2017 and 2016:

	Three Months Ended September 30,				Period-to-Period
(in thousands)	2017		2016		Change
GMI-1271	$	2,921	$	2,799	$	122
GMI-1359		253		715		(462)
Other research and development		635		565		70
Personnel-related and stock-based compensation		1,971		1,842		129
Research and development expense	$	5,780	$	5,921	$	(141)

	Nine Months Ended September 30,				Period-to-Period
(in thousands)	2017		2016		Change
GMI-1271	$	8,760	$	7,619	$	1,141
GMI-1359		562		2,316		(1,754)
Other research and development		1,887		1,838		49
Personnel-related and stock-based compensation		6,171		5,448		723
Research and development expense	$	17,380	$	17,221	$	159

General and Administrative Expense

The following tables summarize the components of our general and administrative expense for the three and nine  months ended September 30, 2017 and 2016:

	Three Months Ended September 30,				Period-to-Period
(in thousands)	2017		2016		Change
Personnel-related	$	729	$	640	$	89
Stock-based compensation		635		479		156
Legal, consulting and other professional expenses		863		719		144
Other		175		146		29
General and administrative expense	$	2,402	$	1,984	$	418

	Nine Months Ended September 30,				Period-to-Period
(in thousands)	2017		2016		Change
Personnel-related	$	2,282	$	2,019	$	263
Stock-based compensation		1,842		1,467		375
Legal, consulting and other professional expenses		2,385		2,412		(27)
Other		507		454		53
General and administrative expense	$	7,016	$	6,352	$	664

23

During the three months ended September 30, 2017, our general and administrative expense increased by $418,000 compared to the same period in 2016, reflecting an increase of 21%. During the nine months ended September 30, 2017, our general and administrative expense increased by $664,000 compared to the same period in 2016, reflecting an increase of 10%. These increases in general and administrative expense were primarily attributable to annual salary adjustments awarded to general and administrative personnel and an increase in stock-based compensation expense caused by the 2017 awards to employees and directors.

Liquidity and Capital Resources

Sources of Liquidity

We have financed our operations primarily through private placements of our capital stock, our IPO, our at-the-market sales facilities with Cowen, our public offerings in June 2016 and May 2017 and upfront and milestone payments from Pfizer. As of September 30, 2017, we had $112.9 million in cash and cash equivalents.

We are potentially eligible to earn a significant amount of milestone payments and royalties under our agreement with Pfizer. Our ability to earn these payments and their timing is dependent upon the outcome of Pfizer’s activities and is uncertain at this time.

On March 1, 2016, we entered into an at-the-market sales agreement with Cowen to sell shares of our common stock having an aggregate offering price of up to $40.0 million through Cowen acting as our sales agent. For the nine months ended September 30, 2017, we sold an aggregate of 1,388,647 shares of our common stock under the at-the-market facility, for net proceeds of $7.4 million. We and Cowen terminated the agreement in May 2017. As of its termination , we had sold an aggregate of 2,057,438 shares for net proceeds of $11.3 million under the at-the-market facility.

In May 2017, we completed a public offering in which we sold 8,050,000 shares of our common stock at a price to the public of $11.50 per share. We received net proceeds of $86.8 million from this offering, after deducting underwriting discounts, commissions and other offering expenses.

On September 28, 2017, we entered into a new at-the-market sales agreement with Cowen, under which we may offer and sell, from time to time at our sole discretion, shares of our common stock having an aggregate offering price of up to $100.0 million through Cowen acting as our sales agent. As of the date of this report, we have sold an aggregate of 1,600,000 shares of our common stock under the new at-the-market facility, for net proceeds of $19.2 million.

Funding Requirements

Our primary uses of capital are, and we expect will continue to be, compensation and related expenses, third-party clinical research and development services, laboratory and related supplies, clinical costs, legal and other regulatory expenses and general overhead costs.

The successful development of any of our drug candidates is highly uncertain. As such, at this time, 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 GMI-1271 or our other drug candidates. We are also unable to predict when, if ever, material net cash inflows will commence from rivipansel or GMI-1271. This is due to the numerous risks and uncertainties associated with developing drugs, including the uncertainty of:

· successful enrollment in, and completion of, clinical trials;

· receipt of marketing approvals from applicable regulatory authorities;

· establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers;

· obtaining and maintaining patent and trade secret protection and regulatory exclusivity for drug candidates;

24

· launching commercial sales of drugs, if and when approved, whether alone or in collaboration with others; and

· obtaining and maintaining healthcare coverage and adequate reimbursement.

A change in the outcome of any of these variables with respect to the development of any of our drug candidates would significantly change the costs and timing associated with the development of that drug candidate. Because our drug candidates are in various stages of clinical and preclinical development and the outcome of these efforts is uncertain, we cannot estimate the actual amounts necessary to successfully complete the development and commercialization of our drug candidates or whether, or when, we may achieve profitability. Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity or debt financings and collaboration arrangements, including our existing collaboration with Pfizer. Except for Pfizer’s obligation to make milestone payments under our agreement with them, we do not have any committed external source of liquidity.

To the extent that we raise additional capital through the future sale of equity or debt, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing common stockholders. If we raise additional funds through the issuance of convertible debt securities, these securities could contain covenants that would restrict our operations.

We may require additional capital beyond our currently anticipated amounts. Additional capital may not be available on reasonable terms, or at all. If we raise additional funds through collaboration arrangements in the future, we may have to relinquish valuable rights to our drug candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our drug development or future commercialization efforts or grant rights to develop and market drug candidates that we would otherwise prefer to develop and market ourselves.

Outlook

Based on our research and development plans and our timing expectations related to the progress of our programs, we expect that our existing cash and cash equivalents as of September 30, 2017 will enable us to fund our operating expenses and capital expenditure requirements at least through the second half of 2019. We have based this estimate on assumptions that may prove to be wrong, and we could use our capital resources sooner than we expect. Additionally, the process of testing drug candidates in clinical trials is costly, and the timing of progress in these trials is uncertain.

Cash Flows

The following is a summary of our cash flows for the nine months ended September 30, 2017 and 2016:

	Nine Months Ended  September 30,
(in thousands)	2017		2016
Net cash provided by (used in):
Operating activities	$	(21,399)	$	(23,777)
Investing activities		(260)		(266)
Financing activities		94,490		22,522
Net change in cash and cash equivalents	$	72,831	$	(1,521)

Operating Activities

Net cash used in operating activities for the nine months ended September 30, 2017 included the ongoing costs associated with the GMI-1271 clinical development programs. Net cash used in operating activities for the nine months ended September 30, 2016 included the costs associated with the advancement of the GMI-1271 and GMI-1359 development programs. In addition, the $2.0 million milestone license fee due to the University of Basel and accrued as a liability as of December 31, 2015 was paid during the nine months ended September 30, 2016.

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Investing Activities

Net cash used in investing activities for the nine months ended September 30, 2017 included capital expenses relating to the additional leasehold improvements and the purchase of laboratory equipment. Net cash used in investing activities for the nine months ended September 30, 2016 included capital expenses relating to the additional leasehold improvements including architect and project management fees.

Financing Activities

Net cash provided by financing activities for the nine months ended September 30, 2017 consisted primarily of the net proceeds of $86.8 million from our public offering in May 2017 and $7.4 million from at-the-market sales under our sales agreement with Cowen. Net cash provided by financing activities during the nine months ended September 30, 2016 comprised primarily of the net proceeds of $19.8 million from our public offering in June 2016 and $2.7 million from at-the-market sales under our sales agreement with Cowen.

Off-Balance Sheet Arrangements

During the nine months ended September 30, 2017, we did not have, and we do not currently have, any off-balance sheet arrangements, as defined under SEC rules.

JOBS Act

In April 2012, the Jumpstart Our Business Startups Act of 2012, or the JOBS Act, was enacted. Section 107(b) of the JOBS Act provides that an emerging growth company can take advantage of an extended transition period for complying with new or revised accounting standards. Thus, an emerging growth company can delay the adoption of certain accounting standards until those standards would otherwise apply to private companies. We have irrevocably elected not to avail ourselves of this extended transition period, and, as a result, we will adopt new or revised accounting standards on the relevant dates on which adoption of such standards is required for other public companies.

Item 3. Quantitative and Qualitative Disclosures about Market Risk

The market risk inherent in our financial instruments and in our financial position represents the potential loss arising from adverse changes in interest rates. As of September 30, 2017 and December 31, 2016, we had cash and cash equivalents of $112.9 million and $40.0 million, respectively. We generally hold our cash in interest-bearing money market accounts. Our primary exposure to market risk is interest rate sensitivity, which is affected by changes in the general level of U.S. interest rates. Due to the short-term maturities of our cash equivalents and the low risk profile of our investments, an immediate 100 basis point change in interest rates would not have a material effect on the fair market value of our cash equivalents.

Item 4. Controls and Procedures

(a) Evaluation of Disclosure Controls and Procedures

The term “disclosure controls and procedures,” as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended, or the Exchange Act, refers to controls and procedures that are designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is recorded, processed, summarized and reported, within the time periods specified in the Security and Exchange Commission’s rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that such information is accumulated and communicated to a company’s management, including its principal executive and principal financial officers, as appropriate to allow timely decisions regarding required disclosure.

In designing and evaluating our disclosure controls and procedures, management recognizes that disclosure controls and procedures, no matter how well conceived and operated, can provide only reasonable, not absolute, assurance that the objectives of the disclosure controls and procedures are met. Additionally, in designing disclosure

26

controls and procedures, our management necessarily was required to apply its judgment in evaluating the cost-benefit relationship of possible disclosure controls and procedures. The design of any system of controls also is based in part upon certain assumptions about the likelihood of future events, and there can be no assurance that any design will succeed in achieving its stated goals under all potential future conditions; over time, controls may become inadequate because of changes in conditions, or the degree of compliance with policies or procedures may deteriorate. Because of the inherent limitations in a control system, misstatements due to error or fraud may occur and not be detected.

Our management, with the participation of our Chief Executive Officer and our Chief Financial Officer, has evaluated the effectiveness of our disclosure controls and procedures as of September 30, 2017, the end of the period covered by this Quarterly Report on Form 10-Q. Based upon such evaluation, our Chief Executive Officer and our Chief Financial Officer have concluded that our disclosure controls and procedures were effective as of such date at the reasonable assurance level.

(b) Changes in Internal Controls Over Financial Reporting

There have not been any changes in our internal controls over financial reporting during our fiscal quarter ended September 30, 2017 that materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

PART II. OTHER INFORMATION

Item 1. Legal Proceedings

From time to time, we are subject to litigation and claims arising in the ordinary course of business. We are not currently a party to any material legal proceedings and we are not aware of any pending or threatened legal proceeding against us that we believe could have a material adverse effect on our business, operating results, cash flows or financial condition.

Item 1A. Risk Factors

Our business is subject to risks and events that, if they occur, could adversely affect our financial condition and results of operations and the trading price of our securities. Our risk factors as of the date of this quarterly report on Form 10‑Q have not changed materially from those described in “Part I, Item 1A. Risk Factors” of our Annual Report on Form 10‑K for the fiscal year ended December 31, 2016, filed with the Securities and Exchange Commission on March 1, 2017.

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

None.

27

Item 6. Exhibits

Exhibit No.	Document
3.1	Amended and Restated Certificate of Incorporation of the Registrant (incorporated herein by reference to Exhibit 3.1 to the Registrant’s Current Report on Form 8‑K (File No. 001‑36177), filed with the Commission on January 15, 2014).
3.2	Amended and Restated Bylaws of the Registrant (incorporated herein by reference to Exhibit 3.2 to the Registrant’s Current Report on Form 8‑K (File No. 001‑36177), filed with the Commission on January 15, 2014).
4.1	Specimen stock certificate evidencing shares of Common Stock (incorporated herein by reference to Exhibit 4.2 to Amendment No. 2 to the Registrant’s Registration Statement on Form S‑1 (File No. 333‑191567), filed with the Commission on October 31, 2013).
31.1*	Certification of Principal Executive Officer under Section 302 of the Sarbanes-Oxley Act.
31.2*	Certification of Principal Financial Officer under Section 302 of the Sarbanes-Oxley Act.
32.1**	Certifications of Principal Executive Officer and Principal Financial Officer under Section 906 of the Sarbanes-Oxley Act.
101.INS	XBRL Instance Document
101.SCH	XBRL Taxonomy Extension Schema Document
101.CAL	XBRL Taxonomy Extension Calculation Linkbase Document
101.DEF	XBRL Taxonomy Extension Definition Linkbase Document
101.LAB	XBRL Taxonomy Extension Label Linkbase Document
101.PRE	XBRL Taxonomy Extension Presentation Linkbase Document

*     Filed herewith

**   These certifications are being furnished solely to accompany this quarterly report pursuant to 18 U.S.C. Section 1350, and are not being filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, and are not to be incorporated by reference into any filing of the registrant, whether made before or after the date hereof, regardless of any general incorporation language in such filing.

28

SIGNATURES

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

	GLYCOMIMETICS, INC.
Date: November 8, 2017	By:	/s/ Brian M. Hahn
		Brian M. Hahn
		Chief Financial Officer
		(On behalf of the Registrant and as Principal Financial Officer)

29

EX-31.1

Exhibit 31.1

CERTIFICATION OF PRINCIPAL EXECUTIVE OFFICER

PURSUANT TO SECTION 302 OF THE SARBANES-OXLEY ACT OF 2002

I, Rachel K. King, certify that:

1.I have reviewed this Quarterly Report on Form 10-Q for the period ended September 30, 2017 of GlycoMimetics, Inc. (the “registrant”);

2.Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3.Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4.The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;

(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

(c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and

(d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and

5.The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):

(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and

(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.

Date: November 8, 2017

/s/ Rachel K. King

Rachel K. King

Chief Executive Officer

(principal executive officer)

EX-31.2

Exhibit 31.2

CERTIFICATION OF PRINCIPAL FINANCIAL OFFICER

PURSUANT TO SECTION 302 OF THE SARBANES-OXLEY ACT OF 2002

I, Brian M. Hahn, certify that:

1.I have reviewed this Quarterly Report on Form 10-Q for the period ended September 30, 2017 of GlycoMimetics, Inc. (the “registrant”);

2.Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3.Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4.The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;

(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

(c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and

(d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and

5.The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):

(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and

(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.

Date: November 8, 2017

/s/ Brian M. Hahn

Brian M. Hahn

Chief Financial Officer

(principal financial officer)

EX-32.1

Exhibit 32.1

CERTIFICATIONS OF

PRINCIPAL EXECUTIVE OFFICER AND PRINCIPAL FINANCIAL OFFICER

PURSUANT TO 18 U.S.C. SECTION 1350,

AS ADOPTED PURSUANT TO

SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002

Pursuant to the requirement set forth in Rule 13a-14(b) of the Securities Exchange Act of 1934, as amended, (the “Exchange Act”) and Section 1350 of Chapter 63 of Title 18 of the United States Code (18 U.S.C. §1350), Rachel K. King, Chief Executive Officer of GlycoMimetics, Inc. (the “Company”), and Brian M. Hahn, Chief Financial Officer of the Company, each hereby certifies that, to the best of his or her knowledge:

1. The Company’s Quarterly Report on Form 10-Q for the period ended September 30, 2017, to which this Certification is attached as Exhibit 32.1 (the “Periodic Report”), fully complies with the requirements of Section 13(a) or Section 15(d) of the Exchange Act; and

2. The information contained in the Periodic Report fairly presents, in all material respects, the financial condition and results of operations of the Company.

IN WITNESS WHEREOF, the undersigned have set their hands hereto as of the 8th day of November, 2017.

/s/ Rachel K. King	/s/ Brian M. Hahn
Rachel K. King	Brian M. Hahn
Chief Executive Officer	Chief Financial Officer

*This certification accompanies the Periodic Report to which it relates, is not deemed filed with the Securities and Exchange Commission and is not to be incorporated by reference into any filing of GlycoMimetics, Inc. under the Securities Act of 1933, as amended, or the Exchange Act (whether made before or after the date of the Periodic Report), irrespective of any general incorporation language contained in such filing.

XML

Document and Entity Information - shares	9 Months Ended
	Sep. 30, 2017	Nov. 07, 2017
Document And Entity Information [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Sep. 30, 2017
Document Fiscal Year Focus	2017
Document Fiscal Period Focus	Q3
Entity Registrant Name	GLYCOMIMETICS INC
Entity Central Index Key	0001253689
Current Fiscal Year End Date	--12-31
Entity Current Reporting Status	Yes
Entity Filer Category	Accelerated Filer
Entity Common Stock, Shares Outstanding		34,337,799

XML

Balance Sheets - USD ($)	Sep. 30, 2017	Dec. 31, 2016
Current Assets:
Cash and cash equivalents	$ 112,872,534	$ 40,041,641
Prepaid expenses and other current assets	1,015,747	478,503
Total current assets	113,888,281	40,520,144
Property and equipment, net	1,122,216	1,056,332
Prepaid research and development expenses	759,531	759,531
Deposits	52,320	52,320
Total assets	115,822,348	42,388,327
Current liabilities:
Accounts payable	1,631,722	1,565,210
Accrued bonuses	1,146,497	1,432,485
Accrued expenses	3,678,609	3,267,371
Deferred rent	74,072	68,551
Total current liabilities	6,530,900	6,333,617
Deferred rent, net of current portion	726,986	753,579
Total liabilities	7,257,886	7,087,196
Stockholders' equity:
Preferred stock; $0.001 par value; 5,000,000 shares authorized; no shares issued and outstanding at September 30, 2017 and December 31, 2016
Common stock; $0.001 par value; 100,000,000 shares authorized, 32,737,799 shares issued and outstanding at September 30, 2017; 100,000,000 shares authorized, 23,250,023 shares issued and outstanding at December 31, 2016	32,736	23,249
Additional paid-in capital	251,550,974	154,254,193
Accumulated deficit	(143,019,248)	(118,976,311)
Total stockholders' equity	108,564,462	35,301,131
Total liabilities and stockholders' equity	$ 115,822,348	$ 42,388,327

XML

Balance Sheets (Parenthetical) - $ / shares	Sep. 30, 2017	Dec. 31, 2016
Balance Sheets
Preferred stock, par value	$ 0.001	$ 0.001
Preferred stock, shares authorized	5,000,000	5,000,000
Preferred stock, shares issued	0	0
Preferred stock, shares outstanding	0	0
Common stock, par value	$ 0.001	$ 0.001
Common stock, shares authorized	100,000,000	100,000,000
Common stock, shares issued	32,737,799	23,250,023
Common stock, shares outstanding	32,737,799	23,250,023

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Statements of Operations and Comprehensive Loss - USD ($)	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Statements of Operations and Comprehensive (Loss) Income
Revenue		$ 18,500		$ 18,500
Costs and expenses:
Research and development expense	5,779,759	5,921,461	17,380,337	17,221,360
General and administrative expense	2,401,976	1,983,795	7,016,081	6,352,355
Total costs and expenses	8,181,735	7,905,256	24,396,418	23,573,715
Loss from operations	(8,181,735)	(7,886,756)	(24,396,418)	(23,555,215)
Other income	231,634	32,063	373,208	73,974
Net loss and comprehensive loss	$ (7,950,101)	$ (7,854,693)	$ (24,023,210)	$ (23,481,241)
Basic and diluted net loss per common share (in dollars per share)	$ (0.24)	$ (0.34)	$ (0.86)	$ (1.14)
Basic and diluted weighted average number of common shares (in shares)	32,724,010	23,049,347	27,814,781	20,638,129

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Statements of Cash Flows - USD ($)	9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016
Operating activities
Net loss	$ (24,023,210)	$ (23,481,241)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation	193,976	139,749
Stock-based compensation expense	2,796,301	2,238,647
Changes in assets and liabilities
Prepaid expenses and other current assets	(537,244)	(797,906)
Prepaid research and development expenses		(63,000)
Deposits		(52,320)
Accounts payable	66,512	682,504
Accrued expenses and bonuses	125,250	(3,122,258)
Deferred rent	(21,072)	679,177
Net cash used in operating activities	(21,399,487)	(23,776,648)
Investing activities
Purchases of property and equipment	(259,860)	(265,635)
Net cash used in investing activities	(259,860)	(265,635)
Financing activities
Proceeds from issuance of common stock, net of issuance costs	94,269,213	22,453,785
Proceeds from exercise of stock options	221,027	68,488
Net cash provided by financing activities	94,490,240	22,522,273
Net change in cash and cash equivalents	72,830,893	(1,520,010)
Cash and cash equivalents, beginning of period	40,041,641	46,802,560
Cash and cash equivalents, end of period	$ 112,872,534	45,282,550
Non-cash investing activities
Property acquisition included in accounts payable		$ 29,329

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Description of the Business

9 Months Ended

Sep. 30, 2017

Description of the Business

Description of the Business

GLYCOMIMETICS, INC.

Notes to Unaudited Financial Statements

1. Description of the Business

GlycoMimetics, Inc. (the Company), a Delaware corporation headquartered in Rockville, Maryland, was incorporated on April 4, 2003 and commenced operations on May 21, 2003. The Company is a clinical stage biotechnology company focused on the discovery and development of novel glycomimetic drugs to address unmet medical needs resulting from diseases in which carbohydrate biology plays a key role. Glycomimetics are molecules that mimic the structure of carbohydrates involved in important biological processes. The Company’s proprietary glycomimetics platform is based on its expertise in carbohydrate chemistry and its understanding of the role carbohydrates play in key biological processes. Using this expertise and understanding, the Company is developing a pipeline of proprietary glycomimetics designed to inhibit disease-related functions of carbohydrates, such as the roles they play in inflammation, cancer and infection.

The Company’s executive personnel have devoted substantially all of their time to date to the planning and organization of the Company, the process of hiring scientists, initiating research and development programs and securing adequate capital for anticipated growth and operations. The Company has not commercialized any of its drug candidates or commenced commercial operations. The Company is subject to a number of risks similar to those of other companies in similar development stages, including dependence on key individuals, the need to develop commercially viable drugs, competition from other companies, many of whom are larger and better capitalized, and the need to obtain adequate additional financing to fund the development of its drug candidates. The Company has incurred significant operating losses since inception and has relied on its ability to fund its operations through private and public equity financings, and management expects operating losses and negative operating cash flows to continue for the foreseeable future. As the Company continues to incur losses, profitability will be dependent upon the successful development, approval, and commercialization of its drug candidates and achieving a level of revenues adequate to support the Company’s cost structure. The Company may never achieve profitability, and unless and until it does, the Company will continue to need to raise additional capital. Management intends to fund future operations through additional public or private equity or debt offerings and may seek additional capital through arrangements with strategic partners or from other sources.  

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Summary of Significant Accounting Policies

9 Months Ended

Sep. 30, 2017

Summary of Significant Accounting Policies

Basis of Presentation and Significant Accounting Policies [Text Block]

2. Significant Accounting Policies

Basis of Accounting

The accompanying financial statements were prepared based on the accrual method of accounting in accordance with U.S. generally accepted accounting principles (GAAP).

Unaudited Financial Statements

The accompanying balance sheet as of September 30, 2017 and statements of operations and comprehensive loss for the three and nine months ended September 30, 2017 and 2016 and cash flows for the nine months ended September 30, 2017 and 2016 are unaudited. These unaudited financial statements have been prepared in accordance with the rules and regulations of the United States Securities and Exchange Commission (the SEC) for interim financial information. Accordingly, they do not include all of the information and footnotes required by GAAP for complete annual financial statements. These financial statements should be read in conjunction with the audited financial statements and the accompanying notes for the year ended December 31, 2016 contained in the Company’s Annual Report on Form 10-K filed with the SEC on March 1, 2017. The unaudited interim financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments (consisting of normal recurring adjustments) necessary to state fairly the Company’s financial position as of September 30, 2017, the results of operations for the three and nine months ended September 30, 2017 and 2016 and cash flows for the nine months ended September 30, 2017 and 2016. The December 31, 2016 balance sheet included herein was derived from audited financial statements, but does not include all disclosures including notes required by GAAP for complete annual financial statements. The financial data and other information disclosed in these notes to the financial statements related to the three and nine months ended September 30, 2017 and 2016 are unaudited. Interim results are not necessarily indicative of results for an entire year.

Use of Estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting periods. Although actual results could differ from those estimates, management does not believe that such differences would be material.

Fair Value Measurements

The Company had no assets or liabilities that were measured using quoted prices for similar assets and liabilities or significant unobservable inputs (Level 2 and Level 3 assets and liabilities, respectively) as of September 30, 2017 and December 31, 2016. The carrying value of cash held in money market funds of approximately $110.9 million and $38.0 million as of September 30, 2017 and December 31, 2016, respectively, is included in cash and cash equivalents and approximates market values based on quoted market prices (Level 1 inputs).

Concentration of Credit Risk

Credit risk represents the risk that the Company would incur a loss if counterparties failed to perform pursuant to the terms of their agreements. Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash and cash equivalents. Cash and cash equivalents consist of money market funds with major financial institutions in the United States. These funds may be redeemed upon demand and, therefore, bear minimal risk. The Company does not anticipate any losses on such balances.

Revenue Recognition

From time to time, the Company is awarded reimbursement contracts for services and development grant contracts with government and non-government entities and philanthropic organizations. Under these contracts, the Company typically is reimbursed for the costs in connection with specific development activities. The Company recognizes revenue to the extent of costs incurred in connection with performance under such grant arrangements.

The Company has entered into a collaborative research and development agreement with Pfizer Inc. (Pfizer). The agreement is in the form of a license agreement (the Pfizer Agreement). The Pfizer Agreement calls for a nonrefundable up-front payment and milestone payments upon achieving significant milestone events. The Pfizer Agreement also contemplates royalty payments on future sales of an approved product. There are no performance, cancellation, termination, or refund provisions in the Pfizer Agreement that contain material financial consequences to the Company.

The primary deliverable under this arrangement is an exclusive worldwide license to the Company’s rivipansel compound, but the arrangement also includes deliverables related to research and preclinical development activities to be performed by the Company on Pfizer’s behalf.

Collaborative research and development agreements can provide for one or more of up-front license fees, research payments, and milestone payments. Agreements with multiple components (deliverables or items) are evaluated according to the provisions of Accounting Standards Codification (ASC) 605-25, Revenue Recognition—Multiple-Element Arrangements, to determine whether the deliverables can be separated into more than one unit of accounting. An item can generally be considered a separate unit of accounting if all of the following criteria are met: (1) the delivered item(s) has value to the customer on a stand-alone basis and (2) if the arrangement includes a general right of return relative to the delivered item(s) then delivery or performance of the undelivered item(s) is considered probable and substantially in control of the Company. Items that cannot be divided into separate units are combined with other units of accounting, as appropriate. Consideration received is allocated among the separate units based on selling price hierarchy. The selling price hierarchy for each deliverable is based on (i) vendor-specific objective evidence (VSOE), if available; (ii) third-party evidence (TPE) of selling price if VSOE is not available; or (iii) an estimated selling price, if neither VSOE nor TPE is available. Management was not able to establish VSOE or TPE for separate unit deliverables, as the Company does not have a history of entering into such arrangements or selling the individual deliverables within such arrangements separately. In addition, there may be significant differentiation in these arrangements, which indicates that comparable third-party pricing may not be available. Management determined that the selling price for the deliverables within the Pfizer Agreement should be determined using its best estimate of selling price. The process of determining the best estimate of selling price involved significant judgment on the Company’s part and included consideration of multiple factors such as estimated direct expenses, other costs, and available clinical development data.

Pursuant to ASC 605-25, each required deliverable under the Pfizer Agreement is evaluated to determine whether it qualifies as a separate unit of accounting. Factors considered in this determination include the research capabilities of Pfizer, the proprietary nature of the license and know-how, and the availability of the Company’s glycomimetics technology research expertise in the general marketplace. Based on all relevant facts and circumstances and, most significantly, on the proprietary nature of the Company’s technology and the related proprietary nature of the Company’s research services, management concluded that stand-alone value does not exist for the license, and therefore, the license is not a separate unit of accounting under the Pfizer Agreement and will be combined with the research and development services (including participation on a joint steering committee). The Company has satisfied the deliverables under the Pfizer Agreement.

Pursuant to ASC 605-28, Revenue Recognition—Milestone Method, at the inception of agreements that include milestone payments, the Company evaluates whether each milestone is substantive and at risk to both parties on the basis of the contingent nature of the milestone. This evaluation includes an assessment of whether (a) the consideration is commensurate with either (1) the entity’s performance to achieve the milestone, or (2) the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the entity’s performance to achieve the milestone, (b) the consideration relates solely to past performance and (c) the consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. In making this assessment, the Company evaluates factors such as scientific, regulatory, commercial and other risks that must be overcome to achieve the respective milestone, the level of effort and investment required to achieve the respective milestone and whether the milestone consideration is reasonable relative to all deliverables and payment terms in the agreement.

Non-refundable development and regulatory milestones that are expected to be achieved as a result of the Company’s efforts during the period of substantial involvement are recognized as revenue upon the achievement of the milestone, assuming all other revenue recognition criteria are met. Milestones that are not considered substantive because the Company does not contribute effort to the achievement of such milestones are generally achieved after the period of substantial involvement and are recognized as revenue upon achievement of the milestone, as there are no undelivered elements remaining and no continuing performance obligation, assuming all other revenue recognition criteria are met. In May 2014, the Company recognized $15.0 million in revenue as a result of the first non-refundable milestone payment received from Pfizer. In June 2015, the Company recognized $20.0 million in revenue as a result of Pfizer dosing the first patient in the Phase 3 clinical trial of rivipansel, which triggered the second non-refundable milestone payment.  

Accrued Liabilities

The Company is required to estimate accrued liabilities as part of the process of preparing its financial statements. The estimation of accrued liabilities involves identifying services that have been performed on the Company’s behalf, and then estimating the level of service performed and the associated cost incurred for such services as of each balance sheet date. Accrued liabilities include professional service fees, such as for lawyers and accountants, contract service fees, such as those under contracts with clinical monitors, data management organizations and investigators in conjunction with clinical trials, and fees to contract manufacturers in conjunction with the production of clinical materials. Pursuant to the Company’s assessment of the services that have been performed, the Company recognizes these expenses as the services are provided. Such assessments include: (i) an evaluation by the project manager of the work that has been completed during the period; (ii) measurement of progress prepared internally and/or provided by the third-party service provider; (iii) analyses of data that justify the progress; and (iv) the Company’s judgment.

Research and Development Costs

Except for payments made in advance of services, research and development costs are expensed as incurred. For payments made in advance, the Company recognizes research and development expense as the services are rendered. Research and development costs primarily consist of salaries and related expenses for personnel, laboratory supplies and raw materials, sponsored research, depreciation of laboratory facilities and leasehold improvements, and utilities costs related to research space. Other research and development expenses include fees paid to consultants and outside service providers including clinical research organizations and clinical manufacturing organizations.

Stock-Based Compensation

Stock-based payments are accounted for in accordance with the provisions of ASC 718, Compensation—Stock Compensation. The fair value of stock-based payments is estimated, on the date of grant, using the Black-Scholes-Merton model. The resulting fair value is recognized ratably over the requisite service period, which is generally the vesting period of the option.

The Company has elected to use the Black-Scholes-Merton option pricing model to value any options granted. The Company will reconsider use of the Black-Scholes-Merton model if additional information becomes available in the future that indicates another model would be more appropriate or if grants issued in future periods have characteristics that prevent their value from being reasonably estimated using this model.

A discussion of management’s methodology for developing some of the assumptions used in the valuation model follows:

Expected Dividend Yield—The Company has never declared or paid dividends and has no plans to do so in the foreseeable future.

Expected Volatility—Volatility is a measure of the amount by which a financial variable such as share price has fluctuated (historical volatility) or is expected to fluctuate (expected volatility) during a period. Prior to the Company’s initial public offering, there was not a market for the Company’s shares. The Company utilizes the historical volatilities of a peer group (e.g., several public entities of similar size, complexity, and stage of development), along with the Company’s historical volatility since its initial public offering, to determine its expected volatility.

Risk-Free Interest Rate—This is the U.S. Treasury rate for the week of each option grant during the year, having a term that most closely resembles the expected life of the option.

Expected Term—This is a period of time that the options granted are expected to remain unexercised. Options granted have a maximum term of 10 years. The Company estimates the expected life of the option term to be 6.25 years. The Company uses a simplified method to calculate the average expected term.

Expected Forfeiture Rate—Effective on January 1, 2017 with the adoption of ASU 2016-09, Compensation – Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting, the Company elected to account for forfeitures as they occur.

Net Loss Per Common Share

Basic net loss per common share is determined by dividing net loss by the weighted-average number of common shares outstanding during the period, without consideration of common stock equivalents. Diluted net loss per share is computed by dividing net loss by the weighted-average number of common stock equivalents outstanding for the period. The treasury stock method is used to determine the dilutive effect of the Company’s stock options, restricted stock units and warrants.

Basic and diluted net loss per common share is computed as follows:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Net loss	$	(7,950,101)	$	(7,854,693)	$	(24,023,210)	$	(23,481,241)
Basic and diluted net loss per common share	$	(0.24)	$	(0.34)	$	(0.86)	$	(1.14)
Basic and diluted weighted average common shares outstanding		32,724,010		23,049,347		27,814,781		20,638,129

The following potentially dilutive securities outstanding have been excluded from the computation of diluted weighted average common shares outstanding, as they would be anti-dilutive:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Warrants		553,868		555,412		553,868		555,412
Stock options and restricted stock units		3,421,124		2,815,166		3,421,124		2,815,166

Comprehensive Loss

Comprehensive loss comprises net loss and other changes in equity that are excluded from net loss. For the three and nine months ended September 30, 2017 and 2016, the Company’s net loss equaled comprehensive net loss and, accordingly, no additional disclosure is presented. 

Recently Issued Accounting Standards

Adopted Accounting Standards

In March 2016, the Financial Accounting Standards Board (FASB) issued ASU No. 2016-09, Compensation – Stock Compensation  (Topic 718): Improvements to Employee Share-Based Payment Accounting. This ASU includes provisions intended to simplify various aspects related to how share-based payments are accounted for and presented in financial statements including the income tax effects of share-based payments, minimum statutory withholding requirements and forfeitures. The new guidance requires all income tax effects of awards to be recognized in the income statement when the awards vest or are settled. It also allows an employer to repurchase more of an employee’s shares than the current standard for tax withholding purposes without triggering liability accounting and to make a policy election to account for forfeitures as they occur. The Company adopted the provisions of ASU 2016-09 on January 1, 2017. The Company has elected to account for forfeitures as they occur. The Company has applied this change using a modified retrospective method through a cumulative-effect adjustment of $19,727 to accumulated deficit. Additionally, the Company recognized deferred tax assets of $98,767 for the excess tax benefits that arose directly from tax deductions related to equity compensation greater than the amounts recognized for financial reporting and also recognized an increase of an equal amount in the valuation allowance against those deferred tax assets. The Company has adopted the additional provisions in the standard and has determined these provisions do not have a material impact on the financial statements.

Accounting Standards Not Yet Adopted

On May 28, 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers  (Topic 606), requiring an entity to recognize the amount of revenue to which it expects to be entitled for the transfer of promised goods or services to customers. The updated standard will replace most existing revenue recognition guidance in GAAP when it becomes effective and permits the use of either the retrospective or cumulative effect transition method. In August 2015, the FASB issued ASU No. 2015-14, Revenue from Contracts with Customers: Deferral of the Effective Date, which deferred the effective date of the new revenue standard for periods beginning after December 15, 2016 to December 15, 2017, with early adoption permitted but not earlier than the original effective date. Accordingly, the updated standard is effective for the Company in the first quarter of fiscal 2018. The Company has evaluated the Pfizer Agreement to determine the impact of the new revenue standard on the upfront and milestone payments within the Pfizer Agreement and has determined that the transition to the new revenue standard will have no material impact on the financial statements. The Company expects to adopt the new standard on January 1, 2018 using the full retrospective transition method.

In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842), which supersedes existing guidance on accounting for leases in Leases (Topic 840) and generally requires all leases, including operating leases, to be recognized in the statement of financial position as right-of-use assets and lease liabilities by lessees. The provisions of ASU 2016-02 are to be applied using a modified retrospective approach and are effective for reporting periods beginning after December 15, 2018; early adoption is permitted. The Company is currently evaluating the effect that this ASU will have on the financial statements.

With the exception of the new standards discussed above, there have been no new accounting pronouncements that have significance, or potential significance, to the Company’s financial statements.

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Prepaid Expenses and Other Current Assets

9 Months Ended

Sep. 30, 2017

Prepaid Expenses and Other Current Assets

Prepaid Expenses and Other Current Assets

3. Prepaid Expenses and Other Current Assets

The following is a summary of the Company’s prepaid expenses and other current assets:

	September 30,		December 31,
	2017		2016
Prepaid expenses	$	940,099	$	199,195
Other receivables		75,648		268,659
Deposits		—		10,649
Prepaid expenses and other current assets	$	1,015,747	$	478,503

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Property and Equipment

9 Months Ended

Sep. 30, 2017

Property and Equipment

Property and Equipment

4. Property and Equipment

Property and equipment, net consists of the following:

	September 30,		December 31,
	2017		2016
Furniture and fixtures	$	314,024	$	262,135
Laboratory equipment		1,282,655		1,130,180
Office equipment		11,084		6,610
Computer equipment		191,825		169,423
Leasehold improvements		573,165		36,128
Construction in progress		—		508,417
Property and equipment		2,372,753		2,112,893
Less accumulated depreciation		(1,250,537)		(1,056,561)
Property and equipment, net	$	1,122,216	$	1,056,332

Depreciation expense was $70,060 and $45,808 for the three months ended September 30, 2017 and 2016, respectively, and $193,976 and $139,749 for the nine months ended September 30, 2017 and 2016, respectively.

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Accrued Expenses

9 Months Ended

Sep. 30, 2017

Accrued Expenses

Accrued Expenses

5. Accrued Expenses

The following is a summary of the Company’s accrued expenses:

	September 30,		December 31,
	2017		2016
Accrued research and development expenses	$	2,727,309	$	2,513,243
Accrued consulting and other professional fees		371,595		148,579
Other accrued expenses		187,067		315,002
Accrued employee benefits		392,638		290,547
Accrued expenses	$	3,678,609	$	3,267,371

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Operating Leases

9 Months Ended

Sep. 30, 2017

Operating Leases

Operating Leases

6. Operating Leases

The Company leases office and research space in Rockville, Maryland under an operating lease with a term through October 31, 2023 (as amended to date, the Lease) that is subject to annual rent increases. The Company has the right to sublease or assign all or a portion of the premises, subject to the conditions set forth in the Lease. The Lease may be terminated early by either the landlord or the Company in certain circumstances. In connection with the Lease, the Company received rent abatement as a lease incentive. The annual rent increases and rent abatement have been recognized as deferred rent that is being adjusted on a straight-line basis over the term of the Lease.

In March 2016, the Company amended the Lease (the Lease Amendment) to lease additional space as of June 1, 2016. In addition to the other terms of the Lease, the Lease Amendment provided for a tenant improvement allowance reflected in the Company’s financial statements as an increase in capitalized leasehold improvements as incurred and an increase in deferred rent. In May 2016, the Company also paid a security deposit of $52,320 to be held until the expiration or termination of the Company’s obligations under the Lease. The term of the Lease Amendment for the additional space continues through October 31, 2023, the same date as for the premises originally leased under the Lease, subject to the Company’s renewal option set forth in the Lease. The Company’s one-time option to terminate the Lease effective as of October 31, 2020 also applies to the additional space.

Deferred rent related to the Lease was $801,058 and $822,130 at September 30, 2017 and December 31, 2016, respectively. Total rent expense under the Company’s operating leases was $223,337 and $219,983 for the three months ended September 30, 2017 and 2016, respectively, and $667,507 and $557,262 for the nine months ended September 30, 2017 and 2016, respectively.

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Stockholders' Equity

9 Months Ended

Sep. 30, 2017

Stockholders' Equity

Disclosure of stockholders' equity and share-based compensation

7. Stockholders’ Equity

At-The-Market Equity Offering

On March 1, 2016, the Company entered into an at-the-market sales agreement with Cowen and Company, LLC to sell the Company’s securities under a shelf registration statement filed in March 2015. The at-the market sales agreement was terminated on May 23, 2017. During the nine months ended September 30, 2017, the Company issued and sold 1,388,647 shares of common stock under the at-the-market sales agreement. The shares were sold at a weighted average price per share of $5.55, for aggregate net proceeds of $7.4 million, after deducting commissions and offering expenses.

On September 28, 2017, the Company entered into a new at-the-market sales agreement with Cowen and Company, LLC to sell the Company’s securities under a shelf registration statement filed in September 2017. Subsequent to September 30, 2017, the Company issued and sold 1,600,000 shares of common stock under the at-the-market sales agreement. The shares were sold at a weighted average price per share of $12.50, for aggregate net proceeds of $19.2 million, after deducting commissions and offering expenses.

Equity Offering

In May 2017, the Company completed a public offering in which the Company sold 8,050,000 shares of its common stock at a price to the public of $11.50 per share. The Company received net proceeds of $86.8 million from this offering, after deducting underwriting discounts, commissions and other offering expenses.

2003 Stock Incentive Plan

The 2003 Stock Incentive Plan (the 2003 Plan) provided for the grant of incentives and nonqualified stock options and restricted stock awards. The exercise price for incentive stock options must be at least equal to the fair value of the common stock on the grant date. Unless otherwise stated in a stock option agreement, 25% of the shares subject to an option grant will vest upon the first anniversary of the vesting start date and thereafter at the rate of one forty-eighth of the option shares per month as of the first day of each month after the first anniversary. Upon termination of employment by reasons other than death, cause, or disability, any vested options shall terminate 60 days after the termination date. Stock options terminate 10 years from the date of grant. The 2003 Plan expired on May 21, 2013.

A summary of the Company’s stock option activity under the 2003 Plan for the nine months ended September 30, 2017 is as follows:

				Weighted-Average
				Remaining	Aggregate
	Outstanding	Weighted-Average		Contractual Term	Intrinsic Value
	Options	Exercise Price		(Years)	(In thousands)
Outstanding as of December 31, 2016	729,819	$	1.26	3.2
Options exercised	(16,608)		1.65
Options forfeited	—		—
Outstanding as of September 30, 2017	713,211		1.25	2.4	$	9,089
Vested as of September 30, 2017	713,211		1.25	2.4	$	9,089
Exercisable as of September 30, 2017	713,211		1.25	2.4	$	9,089

As of September 30, 2017, the options under the 2003 Plan were fully expensed. Total intrinsic value of the options exercised during the nine months ended September 30, 2017 and 2016 was $103,638 and $95,221, respectively, and total cash received for options exercised was $27,358 and $40,668 during the nine months ended September 30, 2017 and 2016, respectively. The total fair value of shares underlying options which vested in the nine months ended September 30, 2017 and 2016 was $1,573 and $14,450, respectively.

2013 Equity Incentive Plan

The Company’s board of directors adopted, and its stockholders approved, its 2013 Equity Incentive Plan (the 2013 Plan) effective on January 9, 2014. The 2013 Plan provides for the grant of incentive stock options within the meaning of Section 422 of the Internal Revenue Code to the Company’s employees and its parent and subsidiary corporations’ employees, and for the grant of nonstatutory stock options, restricted stock awards, restricted stock unit awards, stock appreciation rights, performance stock awards and other forms of stock compensation to its employees, including officers, consultants and directors. The 2013 Plan also provides for the grant of performance cash awards to the Company’s employees, consultants and directors. Unless otherwise stated in a stock option agreement, 25% of the shares subject to an option grant will typically vest upon the first anniversary of the vesting start date and thereafter at the rate of one forty-eighth of the option shares per month as of the first day of each month after the first anniversary. Upon termination of employment by reasons other than death, cause, or disability, any vested options will terminate 90 days after the termination date, unless otherwise set forth in a stock option agreement. Stock options generally terminate 10 years from the date of grant.

Authorized Shares

The maximum number of shares of common stock that initially could be issued under the 2013 Plan was    1,000,000 shares, plus any shares subject to stock options or similar awards granted under the 2003 Plan that expire or terminate without having been exercised in full or are forfeited to or repurchased by the Company. The number of shares of common stock reserved for issuance under the 2013 Plan automatically increases on January 1 of each year until January 1, 2023, by 3% of the total number of shares of common stock outstanding on December 31 of the preceding calendar year, or a lesser number of shares as may be determined by the Company’s board of directors. The maximum number of shares that may be issued pursuant to exercise of incentive stock options under the 2013 Plan is 20,000,000 shares. As of January 1, 2017, the number of shares of common stock that may be issued under the 2013 Plan was automatically increased by 697,500 shares, representing 3% of the total number of shares of common stock outstanding on December 31, 2016, increasing the number of shares of common stock available for issuance under the 2013 Plan to 2,837,201 shares.

Shares issued under the 2013 Plan may be authorized but unissued or reacquired shares of common stock. Shares subject to stock awards granted under the 2013 Plan that expire or terminate without being exercised in full, or that are paid out in cash rather than in shares, will not reduce the number of shares available for issuance under the 2013 Plan. Additionally, shares issued pursuant to stock awards under the 2013 Plan that the Company repurchases or that are forfeited, as well as shares reacquired by the Company as consideration for the exercise or purchase price of a stock award or to satisfy tax withholding obligations related to a stock award, will become available for future grant under the 2013 Plan.

A summary of the Company’s stock option activity under the 2013 Plan for the nine months ended September 30, 2017 is as follows:

				Weighted-Average
				Remaining	Aggregate
	Outstanding	Weighted-Average		Contractual Term	Intrinsic Value
	Options	Exercise Price		(Years)	(In thousands)
Outstanding as of December 31, 2016	2,066,105	$	7.41	7.9
Options granted	674,738		7.03
Options exercised	(32,521)		5.96
Options forfeited	(22,159)		7.35
Outstanding as of September 30, 2017	2,686,163		7.33	7.6	$	17,884
Vested or expected to vest as of September 30, 2017	2,686,163		7.33	7.6	$	17,884
Exercisable as of September 30, 2017	1,453,937		7.84	6.7	$	9,003

The weighted-average fair value of the options granted during the nine months ended September 30, 2017 and 2016 was $4.76 per share and $3.38 per share, respectively, applying the Black-Scholes-Merton option pricing model utilizing the following weighted-average assumptions:

	Nine Months Ended	Nine Months Ended
	September 30, 2017	September 30, 2016
Expected term	6.25 years	6.25 years
Expected volatility	75.20%	68.86%
Risk-free interest rate	2.08%	1.69%
Expected dividend yield	0%	0%

As of September 30, 2017, there was $4,854,266 of total unrecognized compensation expense related to unvested options under the 2013 Plan that will be recognized over a weighted-average period of approximately 2.2 years. Total intrinsic value of the options exercised during the nine months ended September 30, 2017 and 2016 was $228,291 and $2,275, respectively, and total cash received for options exercised was $193,669 and $27,825 during the nine months ended September 30, 2017 and 2016, respectively. The total fair value of shares underlying options which vested in the nine months ended September 30, 2017 and 2016 was $2,872,004 and $2,364,683, respectively.

A restricted stock unit (RSU) is a stock award that entitles the holder to receive shares of the Company’s common stock as the award vests. The fair value of each RSU is based on the closing price of the Company’s stock on the date of grant. The Company has granted RSUs with service conditions (service RSUs) that vest in three equal annual installments provided that the employee remains employed with the Company. As of September 30, 2017, there was $30,763 of unrecognized compensation costs related to unvested service RSUs.

The following is a summary of RSU activity under the 2013 Plan for the nine months ended September 30, 2017:

			Weighted-Average
		Number	Grant Date
	of Shares			Fair Value
Unvested at December 31, 2016		16,916	$	5.04
Granted		—		—
Forfeited		—		—
Vested		4,833		4.61
Unvested at September 30, 2017		12,083		5.21

Stock-based compensation expense was classified on the statement of operations as follows for the three and nine months ended September 30, 2017 and 2016:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Research and development expense	$	327,133	$	260,882	$	953,821	$	772,163
General and administrative expense		635,212		478,531		1,842,480		1,466,484
Total stock-based compensation expense	$	962,345	$	739,413	$	2,796,301	$	2,238,647

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Income Taxes

9 Months Ended

Sep. 30, 2017

Income Taxes

Income Taxes

8. Income Taxes

The Company has not recorded any tax provision or benefit for the nine months ended September 30, 2017 and 2016. The Company has provided a valuation allowance for the full amount of its net deferred tax assets since realization of any future benefit from deductible temporary differences, net operating loss carryforwards and research and development credits is not more-likely-than-not to be realized at September 30, 2017 and December 31, 2016.

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Research and License Agreements

9 Months Ended

Sep. 30, 2017

Revenue Recognition [Abstract]

Research and License Agreement

9. Research and License Agreements

In October 2011, the Company and Pfizer entered into the Pfizer Agreement that provides Pfizer an exclusive worldwide license to rivipansel for vaso-occlusive crisis associated with sickle cell disease and for other diseases for which the drug candidate may be developed. The Company was responsible for completion of the Phase 2 clinical trial, after which Pfizer assumed all further development and commercialization responsibilities. Upon execution of the Pfizer Agreement, the Company received an up-front payment of $22.5 million. The Pfizer Agreement also provides for potential milestone payments of up to $115.0 million upon the achievement of specified development milestones, including the dosing of the first patients in Phase 3 clinical trials for up to two indications and the first commercial sale of a licensed product in the United States and selected European countries for up to two indications; potential milestone payments of up to $70.0 million upon the achievement of specified regulatory milestones, including the acceptance of our filings for regulatory approval by regulatory authorities in the United States and Europe for up to two indications; and potential milestone payments of up to $135.0 million upon the achievement of specified levels of annual net sales of licensed products. Pfizer has the right to terminate the Pfizer Agreement by giving prior written notice.

The Company has determined that each potential future clinical, development and regulatory milestone is substantive. Although sales-based milestones are not considered substantive, they are still recognized upon achievement of the milestone (assuming all other revenue recognition criteria have been met) because there are no undelivered elements that would preclude revenue recognition at that time. The Company is also eligible to receive royalties on future sales contingent upon annual net sales thresholds. In addition, the Company and Pfizer have formed a joint steering committee that will oversee and coordinate activities as set forth in the research program. The $22.5 million up-front payment was recognized over a period of 1.5 years. In May 2014, Pfizer made a $15.0 million non-refundable milestone payment to the Company, which was recognized as revenue by the Company in May 2014 when earned.  In June 2015, Pfizer dosed the first patient in the Phase 3 clinical trial of rivipansel, which triggered a non-refundable milestone payment to the Company of $20.0 million, which the Company recognized as revenue in June 2015. The Company did not recognize any revenue under the Pfizer Agreement during the three and nine months ended September 30, 2017 or 2016.

In February 2004, the Company entered into a research services agreement (the Research Agreement) with the University of Basel (the University) for biological evaluation of selectin antagonists. Certain patents covering the rivipansel compound are subject to provisions of the Research Agreement. Under the terms of the Research Agreement, the Company will owe to the University 10% of all future milestone and royalty payments received from Pfizer with respect to rivipansel. There were no milestone payments due to the University for the three and nine months ended September 30, 2017 or 2016.

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Summary of Significant Accounting Policies (Policies)

9 Months Ended

Sep. 30, 2017

Summary of Significant Accounting Policies

Basis of Accounting

Basis of Accounting

The accompanying financial statements were prepared based on the accrual method of accounting in accordance with U.S. generally accepted accounting principles (GAAP).

Unaudited Financial Statements

Unaudited Financial Statements

The accompanying balance sheet as of September 30, 2017 and statements of operations and comprehensive loss for the three and nine months ended September 30, 2017 and 2016 and cash flows for the nine months ended September 30, 2017 and 2016 are unaudited. These unaudited financial statements have been prepared in accordance with the rules and regulations of the United States Securities and Exchange Commission (the SEC) for interim financial information. Accordingly, they do not include all of the information and footnotes required by GAAP for complete annual financial statements. These financial statements should be read in conjunction with the audited financial statements and the accompanying notes for the year ended December 31, 2016 contained in the Company’s Annual Report on Form 10-K filed with the SEC on March 1, 2017. The unaudited interim financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments (consisting of normal recurring adjustments) necessary to state fairly the Company’s financial position as of September 30, 2017, the results of operations for the three and nine months ended September 30, 2017 and 2016 and cash flows for the nine months ended September 30, 2017 and 2016. The December 31, 2016 balance sheet included herein was derived from audited financial statements, but does not include all disclosures including notes required by GAAP for complete annual financial statements. The financial data and other information disclosed in these notes to the financial statements related to the three and nine months ended September 30, 2017 and 2016 are unaudited. Interim results are not necessarily indicative of results for an entire year.

Use of Estimates

Use of Estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting periods. Although actual results could differ from those estimates, management does not believe that such differences would be material.

Fair Value Measurements

Fair Value Measurements

The Company had no assets or liabilities that were measured using quoted prices for similar assets and liabilities or significant unobservable inputs (Level 2 and Level 3 assets and liabilities, respectively) as of September 30, 2017 and December 31, 2016. The carrying value of cash held in money market funds of approximately $110.9 million and $38.0 million as of September 30, 2017 and December 31, 2016, respectively, is included in cash and cash equivalents and approximates market values based on quoted market prices (Level 1 inputs).

Concentration of Credit Risk

Concentration of Credit Risk

Credit risk represents the risk that the Company would incur a loss if counterparties failed to perform pursuant to the terms of their agreements. Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash and cash equivalents. Cash and cash equivalents consist of money market funds with major financial institutions in the United States. These funds may be redeemed upon demand and, therefore, bear minimal risk. The Company does not anticipate any losses on such balances.

Revenue Recognition

Revenue Recognition

From time to time, the Company is awarded reimbursement contracts for services and development grant contracts with government and non-government entities and philanthropic organizations. Under these contracts, the Company typically is reimbursed for the costs in connection with specific development activities. The Company recognizes revenue to the extent of costs incurred in connection with performance under such grant arrangements.

The Company has entered into a collaborative research and development agreement with Pfizer Inc. (Pfizer). The agreement is in the form of a license agreement (the Pfizer Agreement). The Pfizer Agreement calls for a nonrefundable up-front payment and milestone payments upon achieving significant milestone events. The Pfizer Agreement also contemplates royalty payments on future sales of an approved product. There are no performance, cancellation, termination, or refund provisions in the Pfizer Agreement that contain material financial consequences to the Company.

The primary deliverable under this arrangement is an exclusive worldwide license to the Company’s rivipansel compound, but the arrangement also includes deliverables related to research and preclinical development activities to be performed by the Company on Pfizer’s behalf.

Collaborative research and development agreements can provide for one or more of up-front license fees, research payments, and milestone payments. Agreements with multiple components (deliverables or items) are evaluated according to the provisions of Accounting Standards Codification (ASC) 605-25, Revenue Recognition—Multiple-Element Arrangements, to determine whether the deliverables can be separated into more than one unit of accounting. An item can generally be considered a separate unit of accounting if all of the following criteria are met: (1) the delivered item(s) has value to the customer on a stand-alone basis and (2) if the arrangement includes a general right of return relative to the delivered item(s) then delivery or performance of the undelivered item(s) is considered probable and substantially in control of the Company. Items that cannot be divided into separate units are combined with other units of accounting, as appropriate. Consideration received is allocated among the separate units based on selling price hierarchy. The selling price hierarchy for each deliverable is based on (i) vendor-specific objective evidence (VSOE), if available; (ii) third-party evidence (TPE) of selling price if VSOE is not available; or (iii) an estimated selling price, if neither VSOE nor TPE is available. Management was not able to establish VSOE or TPE for separate unit deliverables, as the Company does not have a history of entering into such arrangements or selling the individual deliverables within such arrangements separately. In addition, there may be significant differentiation in these arrangements, which indicates that comparable third-party pricing may not be available. Management determined that the selling price for the deliverables within the Pfizer Agreement should be determined using its best estimate of selling price. The process of determining the best estimate of selling price involved significant judgment on the Company’s part and included consideration of multiple factors such as estimated direct expenses, other costs, and available clinical development data.

Pursuant to ASC 605-25, each required deliverable under the Pfizer Agreement is evaluated to determine whether it qualifies as a separate unit of accounting. Factors considered in this determination include the research capabilities of Pfizer, the proprietary nature of the license and know-how, and the availability of the Company’s glycomimetics technology research expertise in the general marketplace. Based on all relevant facts and circumstances and, most significantly, on the proprietary nature of the Company’s technology and the related proprietary nature of the Company’s research services, management concluded that stand-alone value does not exist for the license, and therefore, the license is not a separate unit of accounting under the Pfizer Agreement and will be combined with the research and development services (including participation on a joint steering committee). The Company has satisfied the deliverables under the Pfizer Agreement.

Pursuant to ASC 605-28, Revenue Recognition—Milestone Method, at the inception of agreements that include milestone payments, the Company evaluates whether each milestone is substantive and at risk to both parties on the basis of the contingent nature of the milestone. This evaluation includes an assessment of whether (a) the consideration is commensurate with either (1) the entity’s performance to achieve the milestone, or (2) the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the entity’s performance to achieve the milestone, (b) the consideration relates solely to past performance and (c) the consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. In making this assessment, the Company evaluates factors such as scientific, regulatory, commercial and other risks that must be overcome to achieve the respective milestone, the level of effort and investment required to achieve the respective milestone and whether the milestone consideration is reasonable relative to all deliverables and payment terms in the agreement.

Non-refundable development and regulatory milestones that are expected to be achieved as a result of the Company’s efforts during the period of substantial involvement are recognized as revenue upon the achievement of the milestone, assuming all other revenue recognition criteria are met. Milestones that are not considered substantive because the Company does not contribute effort to the achievement of such milestones are generally achieved after the period of substantial involvement and are recognized as revenue upon achievement of the milestone, as there are no undelivered elements remaining and no continuing performance obligation, assuming all other revenue recognition criteria are met. In May 2014, the Company recognized $15.0 million in revenue as a result of the first non-refundable milestone payment received from Pfizer. In June 2015, the Company recognized $20.0 million in revenue as a result of Pfizer dosing the first patient in the Phase 3 clinical trial of rivipansel, which triggered the second non-refundable milestone payment.  

Accrued Liabilities

Accrued Liabilities

The Company is required to estimate accrued liabilities as part of the process of preparing its financial statements. The estimation of accrued liabilities involves identifying services that have been performed on the Company’s behalf, and then estimating the level of service performed and the associated cost incurred for such services as of each balance sheet date. Accrued liabilities include professional service fees, such as for lawyers and accountants, contract service fees, such as those under contracts with clinical monitors, data management organizations and investigators in conjunction with clinical trials, and fees to contract manufacturers in conjunction with the production of clinical materials. Pursuant to the Company’s assessment of the services that have been performed, the Company recognizes these expenses as the services are provided. Such assessments include: (i) an evaluation by the project manager of the work that has been completed during the period; (ii) measurement of progress prepared internally and/or provided by the third-party service provider; (iii) analyses of data that justify the progress; and (iv) the Company’s judgment.

Research and Development Costs

Research and Development Costs

Except for payments made in advance of services, research and development costs are expensed as incurred. For payments made in advance, the Company recognizes research and development expense as the services are rendered. Research and development costs primarily consist of salaries and related expenses for personnel, laboratory supplies and raw materials, sponsored research, depreciation of laboratory facilities and leasehold improvements, and utilities costs related to research space. Other research and development expenses include fees paid to consultants and outside service providers including clinical research organizations and clinical manufacturing organizations.

Stock-Based Compensation

Stock-Based Compensation

Stock-based payments are accounted for in accordance with the provisions of ASC 718, Compensation—Stock Compensation. The fair value of stock-based payments is estimated, on the date of grant, using the Black-Scholes-Merton model. The resulting fair value is recognized ratably over the requisite service period, which is generally the vesting period of the option.

The Company has elected to use the Black-Scholes-Merton option pricing model to value any options granted. The Company will reconsider use of the Black-Scholes-Merton model if additional information becomes available in the future that indicates another model would be more appropriate or if grants issued in future periods have characteristics that prevent their value from being reasonably estimated using this model.

A discussion of management’s methodology for developing some of the assumptions used in the valuation model follows:

Expected Dividend Yield—The Company has never declared or paid dividends and has no plans to do so in the foreseeable future.

Expected Volatility—Volatility is a measure of the amount by which a financial variable such as share price has fluctuated (historical volatility) or is expected to fluctuate (expected volatility) during a period. Prior to the Company’s initial public offering, there was not a market for the Company’s shares. The Company utilizes the historical volatilities of a peer group (e.g., several public entities of similar size, complexity, and stage of development), along with the Company’s historical volatility since its initial public offering, to determine its expected volatility.

Risk-Free Interest Rate—This is the U.S. Treasury rate for the week of each option grant during the year, having a term that most closely resembles the expected life of the option.

Expected Term—This is a period of time that the options granted are expected to remain unexercised. Options granted have a maximum term of 10 years. The Company estimates the expected life of the option term to be 6.25 years. The Company uses a simplified method to calculate the average expected term.

Expected Forfeiture Rate—Effective on January 1, 2017 with the adoption of ASU 2016-09, Compensation – Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting, the Company elected to account for forfeitures as they occur.

Net Loss Per Common Share

Net Loss Per Common Share

Basic net loss per common share is determined by dividing net loss by the weighted-average number of common shares outstanding during the period, without consideration of common stock equivalents. Diluted net loss per share is computed by dividing net loss by the weighted-average number of common stock equivalents outstanding for the period. The treasury stock method is used to determine the dilutive effect of the Company’s stock options, restricted stock units and warrants.

Basic and diluted net loss per common share is computed as follows:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Net loss	$	(7,950,101)	$	(7,854,693)	$	(24,023,210)	$	(23,481,241)
Basic and diluted net loss per common share	$	(0.24)	$	(0.34)	$	(0.86)	$	(1.14)
Basic and diluted weighted average common shares outstanding		32,724,010		23,049,347		27,814,781		20,638,129

The following potentially dilutive securities outstanding have been excluded from the computation of diluted weighted average common shares outstanding, as they would be anti-dilutive:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Warrants		553,868		555,412		553,868		555,412
Stock options and restricted stock units		3,421,124		2,815,166		3,421,124		2,815,166

Comprehensive Loss

Comprehensive Loss

Comprehensive loss comprises net loss and other changes in equity that are excluded from net loss. For the three and nine months ended September 30, 2017 and 2016, the Company’s net loss equaled comprehensive net loss and, accordingly, no additional disclosure is presented.

Recently Issued Accounting Standards

Recently Issued Accounting Standards

Adopted Accounting Standards

In March 2016, the Financial Accounting Standards Board (FASB) issued ASU No. 2016-09, Compensation – Stock Compensation  (Topic 718): Improvements to Employee Share-Based Payment Accounting. This ASU includes provisions intended to simplify various aspects related to how share-based payments are accounted for and presented in financial statements including the income tax effects of share-based payments, minimum statutory withholding requirements and forfeitures. The new guidance requires all income tax effects of awards to be recognized in the income statement when the awards vest or are settled. It also allows an employer to repurchase more of an employee’s shares than the current standard for tax withholding purposes without triggering liability accounting and to make a policy election to account for forfeitures as they occur. The Company adopted the provisions of ASU 2016-09 on January 1, 2017. The Company has elected to account for forfeitures as they occur. The Company has applied this change using a modified retrospective method through a cumulative-effect adjustment of $19,727 to accumulated deficit. Additionally, the Company recognized deferred tax assets of $98,767 for the excess tax benefits that arose directly from tax deductions related to equity compensation greater than the amounts recognized for financial reporting and also recognized an increase of an equal amount in the valuation allowance against those deferred tax assets. The Company has adopted the additional provisions in the standard and has determined these provisions do not have a material impact on the financial statements.

Accounting Standards Not Yet Adopted

On May 28, 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers  (Topic 606), requiring an entity to recognize the amount of revenue to which it expects to be entitled for the transfer of promised goods or services to customers. The updated standard will replace most existing revenue recognition guidance in GAAP when it becomes effective and permits the use of either the retrospective or cumulative effect transition method. In August 2015, the FASB issued ASU No. 2015-14, Revenue from Contracts with Customers: Deferral of the Effective Date, which deferred the effective date of the new revenue standard for periods beginning after December 15, 2016 to December 15, 2017, with early adoption permitted but not earlier than the original effective date. Accordingly, the updated standard is effective for the Company in the first quarter of fiscal 2018. The Company has evaluated the Pfizer Agreement to determine the impact of the new revenue standard on the upfront and milestone payments within the Pfizer Agreement and has determined that the transition to the new revenue standard will have no material impact on the financial statements. The Company expects to adopt the new standard on January 1, 2018 using the full retrospective transition method.

In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842), which supersedes existing guidance on accounting for leases in Leases (Topic 840) and generally requires all leases, including operating leases, to be recognized in the statement of financial position as right-of-use assets and lease liabilities by lessees. The provisions of ASU 2016-02 are to be applied using a modified retrospective approach and are effective for reporting periods beginning after December 15, 2018; early adoption is permitted. The Company is currently evaluating the effect that this ASU will have on the financial statements.

With the exception of the new standards discussed above, there have been no new accounting pronouncements that have significance, or potential significance, to the Company’s financial statements.

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Significant Accounting Policies - 10-Q (Tables)

9 Months Ended

Sep. 30, 2017

Net Loss Per Share of Common Stock

Computation of Basic and Diluted Earnings Per Share

Basic and diluted net loss per common share is computed as follows:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Net loss	$	(7,950,101)	$	(7,854,693)	$	(24,023,210)	$	(23,481,241)
Basic and diluted net loss per common share	$	(0.24)	$	(0.34)	$	(0.86)	$	(1.14)
Basic and diluted weighted average common shares outstanding		32,724,010		23,049,347		27,814,781		20,638,129

Potentially Dilutive Securities Outstanding

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Net loss	$	(7,950,101)	$	(7,854,693)	$	(24,023,210)	$	(23,481,241)
Basic and diluted net loss per common share	$	(0.24)	$	(0.34)	$	(0.86)	$	(1.14)
Basic and diluted weighted average common shares outstanding		32,724,010		23,049,347		27,814,781		20,638,129

The following potentially dilutive securities outstanding have been excluded from the computation of diluted weighted average common shares outstanding, as they would be anti-dilutive:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Warrants		553,868		555,412		553,868		555,412
Stock options and restricted stock units		3,421,124		2,815,166		3,421,124		2,815,166

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Prepaid Expenses and Other Current Assets (Tables)

9 Months Ended

Sep. 30, 2017

Prepaid Expenses and Other Current Assets

Summary of Prepaid Expenses and Other Current Assets

The following is a summary of the Company’s prepaid expenses and other current assets:

	September 30,		December 31,
	2017		2016
Prepaid expenses	$	940,099	$	199,195
Other receivables		75,648		268,659
Deposits		—		10,649
Prepaid expenses and other current assets	$	1,015,747	$	478,503

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Property and Equipment (Tables)

9 Months Ended

Sep. 30, 2017

Property and Equipment

Summary of Property and Equipment, Net

Property and equipment, net consists of the following:

	September 30,		December 31,
	2017		2016
Furniture and fixtures	$	314,024	$	262,135
Laboratory equipment		1,282,655		1,130,180
Office equipment		11,084		6,610
Computer equipment		191,825		169,423
Leasehold improvements		573,165		36,128
Construction in progress		—		508,417
Property and equipment		2,372,753		2,112,893
Less accumulated depreciation		(1,250,537)		(1,056,561)
Property and equipment, net	$	1,122,216	$	1,056,332

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Accrued Expenses (Tables)

9 Months Ended

Sep. 30, 2017

Accrued Expenses

Summary of Accrued Expenses

	September 30,		December 31,
	2017		2016
Accrued research and development expenses	$	2,727,309	$	2,513,243
Accrued consulting and other professional fees		371,595		148,579
Other accrued expenses		187,067		315,002
Accrued employee benefits		392,638		290,547
Accrued expenses	$	3,678,609	$	3,267,371

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Stockholders Equity (Tables)

9 Months Ended

Sep. 30, 2017

Stock-Based Compensation Expense

Stock-based compensation expense was classified on the statement of operations as follows for the three and nine months ended September 30, 2017 and 2016:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2017		2016		2017		2016
Research and development expense	$	327,133	$	260,882	$	953,821	$	772,163
General and administrative expense		635,212		478,531		1,842,480		1,466,484
Total stock-based compensation expense	$	962,345	$	739,413	$	2,796,301	$	2,238,647

2003 Stock Incentive Plan

Company's Stock Option Activity

A summary of the Company’s stock option activity under the 2003 Plan for the nine months ended September 30, 2017 is as follows:

				Weighted-Average
				Remaining	Aggregate
	Outstanding	Weighted-Average		Contractual Term	Intrinsic Value
	Options	Exercise Price		(Years)	(In thousands)
Outstanding as of December 31, 2016	729,819	$	1.26	3.2
Options exercised	(16,608)		1.65
Options forfeited	—		—
Outstanding as of September 30, 2017	713,211		1.25	2.4	$	9,089
Vested as of September 30, 2017	713,211		1.25	2.4	$	9,089
Exercisable as of September 30, 2017	713,211		1.25	2.4	$	9,089

2013 Equity Incentive Plan

Company's Stock Option Activity

A summary of the Company’s stock option activity under the 2013 Plan for the nine months ended September 30, 2017 is as follows:

				Weighted-Average
				Remaining	Aggregate
	Outstanding	Weighted-Average		Contractual Term	Intrinsic Value
	Options	Exercise Price		(Years)	(In thousands)
Outstanding as of December 31, 2016	2,066,105	$	7.41	7.9
Options granted	674,738		7.03
Options exercised	(32,521)		5.96
Options forfeited	(22,159)		7.35
Outstanding as of September 30, 2017	2,686,163		7.33	7.6	$	17,884
Vested or expected to vest as of September 30, 2017	2,686,163		7.33	7.6	$	17,884
Exercisable as of September 30, 2017	1,453,937		7.84	6.7	$	9,003

Weighted-Average Fair Value of Options Granted

	Nine Months Ended	Nine Months Ended
	September 30, 2017	September 30, 2016
Expected term	6.25 years	6.25 years
Expected volatility	75.20%	68.86%
Risk-free interest rate	2.08%	1.69%
Expected dividend yield	0%	0%

Summary of RSU Activity

The following is a summary of RSU activity under the 2013 Plan for the nine months ended September 30, 2017:

			Weighted-Average
		Number	Grant Date
	of Shares			Fair Value
Unvested at December 31, 2016		16,916	$	5.04
Granted		—		—
Forfeited		—		—
Vested		4,833		4.61
Unvested at September 30, 2017		12,083		5.21

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Summary of Significant Accounting Policies (Details) - USD ($)	1 Months Ended		3 Months Ended		9 Months Ended		18 Months Ended
	Jun. 30, 2015	May 31, 2014	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016	Mar. 31, 2013	Dec. 31, 2016
Share-based Compensation
Expected term					6 years 3 months	6 years 3 months
Level 1
Fair Value Measurements
Carrying value of cash held in money market fund			$ 110,900,000		$ 110,900,000			$ 38,000,000
Level 2
Fair Value Measurements
Assets measured at fair value levels 2 or 3			0		0			0
Liabilities measured at fair value levels 2 or 3			0		0			0
Level 3
Fair Value Measurements
Assets measured at fair value levels 2 or 3			0		0			0
Liabilities measured at fair value levels 2 or 3			0		$ 0			$ 0
Maximum
Share-based Compensation
Expiration period					10 years
Pfizer | License Agreement
Revenue Recognition
Milestone revenue			$ 0	$ 0	$ 0	$ 0
Pfizer | License Agreement | Up-Front Payment Arrangement
Revenue Recognition
Upfront payment revenue recognized							$ 22,500,000
Recognition period of up-front payment							1 year 6 months
Achievement Of Specified Development Milestone | Pfizer | License Agreement
Revenue Recognition
Milestone revenue	$ 20,000,000	$ 15,000,000

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Significant Accounting Policies - Computation of Basic and Diluted Earnings Per Share - 10Q (Detail) - USD ($)	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Summary of Significant Accounting Policies
Net loss	$ (7,950,101)	$ (7,854,693)	$ (24,023,210)	$ (23,481,241)
Earnings Per Share, Basic and Diluted	$ (0.24)	$ (0.34)	$ (0.86)	$ (1.14)
Basic and diluted weighted average common shares outstanding	32,724,010	23,049,347	27,814,781	20,638,129

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Significant Accounting Policies - Common Stock Equivalents Included in Calculation of Diluted Net Income Per Common Share -10-Q (Details) - shares	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Warrants [Member]
Anti-dilutive securities	553,868	555,412	553,868	555,412
Stock Options and Restricted Stock Units [Member]
Anti-dilutive securities	3,421,124	2,815,166	3,421,124	2,815,166

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Significant Accounting Policies - Recently Issued Accounting Standards (Details) - ASU 2016-09	Jan. 01, 2017 USD ($)
New accounting pronouncement, modified retrospective method
Deferred tax assets for equity compensation	$ 98,767
Deferred tax assets, valuation allowance	98,767
Accumulated Deficit
New accounting pronouncement, modified retrospective method
Cumulative effect adjustment for modified retrospective transition to ASU 2016-09	$ 19,727

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Prepaid Expenses and Other Current Assets - Summary of Prepaid Expenses and Other Current Assets (Detail) - USD ($)	Sep. 30, 2017	Dec. 31, 2016
Prepaid Expenses and Other Current Assets
Prepaid expenses	$ 940,099	$ 199,195
Other receivables	75,648	268,659
Deposits		10,649
Prepaid expenses and other current assets	$ 1,015,747	$ 478,503

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Property and Equipment (Detail) - USD ($)	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016	Dec. 31, 2016
Property, Plant and Equipment [Line Items]
Property and equipment	$ 2,372,753		$ 2,372,753		$ 2,112,893
Less accumulated depreciation	(1,250,537)		(1,250,537)		(1,056,561)
Property and equipment, net	1,122,216		1,122,216		1,056,332
Depreciation of property and equipment	70,060	$ 45,808	193,976	$ 139,749
Furniture and Fixtures
Property, Plant and Equipment [Line Items]
Property and equipment	314,024		314,024		262,135
Laboratory Equipment
Property, Plant and Equipment [Line Items]
Property and equipment	1,282,655		1,282,655		1,130,180
Office Equipment
Property, Plant and Equipment [Line Items]
Property and equipment	11,084		11,084		6,610
Computer Equipment
Property, Plant and Equipment [Line Items]
Property and equipment	191,825		191,825		169,423
Leasehold Improvements
Property, Plant and Equipment [Line Items]
Property and equipment	$ 573,165		$ 573,165		36,128
Construction in Progress
Property, Plant and Equipment [Line Items]
Property and equipment					$ 508,417

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Accrued Expenses - Summary of Accrued Expenses (Detail) - USD ($)	Sep. 30, 2017	Dec. 31, 2016
Accrued Expenses
Accrued research and development expenses	$ 2,727,309	$ 2,513,243
Accrued consulting and other professional fees	371,595	148,579
Other accrued expenses	187,067	315,002
Accrued employee benefits	392,638	290,547
Accrued expenses	$ 3,678,609	$ 3,267,371

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Operating Leases (Details) - USD ($)	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016	Dec. 31, 2016	May 31, 2016
Deferred Rent Credit	$ 801,058		$ 801,058		$ 822,130
Operating leases, rent expense	223,337	$ 219,983	667,507	$ 557,262
Security deposit paid	$ 52,320		$ 52,320		$ 52,320
Rockville
Security deposit paid						$ 52,320

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Stockholders' Equity - Equity Offerings (Detail) - USD ($) $ / shares in Units, $ in Millions		1 Months Ended	9 Months Ended
	Oct. 01, 2017	May 31, 2017	Sep. 30, 2017
At The Market Equity Offering | Subsequent Event
Equity offerings
Issuance of common stock, net of issuance costs, shares	1,600,000
Weighted average price per share issued	$ 12.50
Aggregate net proceeds from stock sale	$ 19.2
At The Market Equity Offering | Common Stock
Equity offerings
Issuance of common stock, net of issuance costs, shares			1,388,647
Weighted average price per share issued			$ 5.550
Aggregate net proceeds from stock sale			$ 7.4
Public Equity Offering | Common Stock
Equity offerings
Issuance of common stock, net of issuance costs, shares		8,050,000
Weighted average price per share issued		$ 11.50
Aggregate net proceeds from stock sale		$ 86.8

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Stockholders' Equity - Incentive Plans (Detail) - USD ($)			9 Months Ended
	Jan. 01, 2017	Jan. 09, 2014	Sep. 30, 2017	Sep. 30, 2016	Dec. 31, 2016
Maximum
Incentive plans
Expiration period			10 years
2003 Stock Incentive Plan
Incentive plans
Period from date of termination that any vested options shall expire			60 days
Expiration period			10 years
Intrinsic value of options exercised			$ 103,638	$ 95,221
Cash proceeds from exercise of stock options			$ 27,358	40,668
Options exercised			16,608
Fair value of shares vested			$ 1,573	14,450
2003 Stock Incentive Plan | Upon first anniversary of start date
Incentive plans
Percent of shares subject to option grant that will vest			25.00%
2003 Stock Incentive Plan | Each month after the first anniversary
Incentive plans
Percent of shares subject to option grant that will vest			2.083%
2013 Equity Incentive Plan
Incentive plans
Period from date of termination that any vested options shall expire		90 days
Expiration period		10 years
Unrecognized compensation expense related to unvested options			$ 4,854,266
Period for unrecognized compensation expense related to unvested options yet has not been recognized			2 years 2 months 12 days
Intrinsic value of options exercised			$ 228,291	2,275
Cash proceeds from exercise of stock options			$ 193,669	27,825
Options exercised			32,521
Fair value of shares vested			$ 2,872,004	$ 2,364,683
2013 Equity Incentive Plan | Upon first anniversary of start date
Incentive plans
Percent of shares subject to option grant that will vest		25.00%
2013 Equity Incentive Plan | Each month after the first anniversary
Incentive plans
Percent of shares subject to option grant that will vest		2.083%
Common Stock | 2013 Equity Incentive Plan
Authorized shares
Common stock authorized under 2013 plan	2,837,201	1,000,000
Automatic increase in number of shares reserved for issuance as a percentage of the total common stock outstanding at the end of the prior year		3.00%			3.00%
Maximum number of shares that may be issued pursuant to exercise of incentive stock		20,000,000
Increase in number of shares of common stock	697,500

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Stockholders' Equity - Company's Stock Option Activity (Detail) - USD ($) $ / shares in Units, $ in Thousands	9 Months Ended	12 Months Ended
	Sep. 30, 2017	Dec. 31, 2016
2003 Stock Incentive Plan
OUTSTANDING OPTIONS
Outstanding at beginning of period	729,819
Options exercised	(16,608)
Outstanding at end of period	713,211	729,819
WEIGHTED-AVERAGE EXERCISE PRICE
Outstanding at beginning of period	$ 1.26
Options exercised	1.65
Outstanding at end of period	$ 1.25	$ 1.26
VESTED AND EXPECTED TO VEST
Outstanding options	713,211
Weighted-Average Exercise Price	$ 1.25
Weighted-Average Remaining Contractual Term	2 years 4 months 24 days
Aggregate Intrinsic Value (in thousands)	$ 9,089
ADDITIONAL DISCLOSURES
Outstanding options exercisable at end of period	713,211
Weighted-average exercise price exercisable at end of period	$ 1.25
Weighted-average contractual term (years) exercisable at end of period	2 years 4 months 24 days
Aggregate intrinsic value (in thousands) exercisable at end of period	$ 9,089
Weighted-average contractual term outstanding	2 years 4 months 24 days	3 years 2 months 12 days
Aggregate intrinsic value (in thousands) outstanding at end of period	$ 9,089
2013 Equity Incentive Plan
OUTSTANDING OPTIONS
Outstanding at beginning of period	2,066,105
Options granted	674,738
Options exercised	(32,521)
Options forfeited	(22,159)
Outstanding at end of period	2,686,163	2,066,105
WEIGHTED-AVERAGE EXERCISE PRICE
Outstanding at beginning of period	$ 7.41
Options granted	7.03
Options exercised	5.96
Options forfeited	7.35
Outstanding at end of period	$ 7.33	$ 7.41
VESTED AND EXPECTED TO VEST
Outstanding options	2,686,163
Weighted-Average Exercise Price	$ 7.33
Weighted-Average Remaining Contractual Term	7 years 7 months 6 days
Aggregate Intrinsic Value (in thousands)	$ 17,884
ADDITIONAL DISCLOSURES
Outstanding options exercisable at end of period	1,453,937
Weighted-average exercise price exercisable at end of period	$ 7.84
Weighted-average contractual term (years) exercisable at end of period	6 years 8 months 12 days
Aggregate intrinsic value (in thousands) exercisable at end of period	$ 9,003
Weighted-average contractual term outstanding	7 years 7 months 6 days	7 years 10 months 24 days
Aggregate intrinsic value (in thousands) outstanding at end of period	$ 17,884

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Stockholders' Equity - Weighted-Average assumptions (Detail) - $ / shares	9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016
Weighted-average assumptions
Expected term	6 years 3 months	6 years 3 months
2013 Equity Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted-average fair value of the options granted	$ 4.76	$ 3.38
Weighted-average assumptions
Expected volatility	75.20%	68.86%
Risk-free interest rate	2.08%	1.69%
Expected dividend yield	0.00%	0.00%

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Stockholders' Equity - Summary of RSU Activity (Detail) - RSU [Member] - 2013 Equity Incentive Plan	9 Months Ended
	Sep. 30, 2017 USD ($) installment $ / shares shares
Restricted Stock Units information
Unrecognized compensation expense related to unvested service RSUs | $	$ 30,763
Number of vesting installments | installment	3
NUMBER OF SHARES
Unvested at beginning of period | shares	16,916
Granted | shares	0
Forfeited | shares	0
Vested | shares	4,833
Unvested at end of period | shares	12,083
WEIGHTED-AVERAGE GRANT DATE FAIR VALUE
Unvested at beginning of period | $ / shares	$ 5.04
Granted | $ / shares	0.00
Forfeited | $ / shares	0.00
Vested | $ / shares	4.61
Unvested at end of period | $ / shares	$ 5.21

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Stockholders' Equity - Stock-Based Compensation Expense (Detail) - USD ($)	3 Months Ended		9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016	Sep. 30, 2017	Sep. 30, 2016
Stock-based compensation expense
Total stock-based compensation expense	$ 962,345	$ 739,413	$ 2,796,301	$ 2,238,647
Research and Development
Stock-based compensation expense
Total stock-based compensation expense	327,133	260,882	953,821	772,163
General and Administrative Expense
Stock-based compensation expense
Total stock-based compensation expense	$ 635,212	$ 478,531	$ 1,842,480	$ 1,466,484

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Income Taxes - 10-Q (Details) - USD ($)	9 Months Ended
	Sep. 30, 2017	Sep. 30, 2016
Income Taxes
Income tax (benefit) provision	$ 0	$ 0

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Research and License Agreements - Additional Information (Detail)	1 Months Ended			3 Months Ended		9 Months Ended		18 Months Ended
	Jun. 30, 2015 USD ($)	May 31, 2014 USD ($)	Oct. 31, 2011 USD ($) item	Sep. 30, 2017 USD ($)	Sep. 30, 2016 USD ($)	Sep. 30, 2017 USD ($)	Sep. 30, 2016 USD ($)	Mar. 31, 2013 USD ($)
Research and License Agreements
Research and Development Expense				$ 5,779,759	$ 5,921,461	$ 17,380,337	$ 17,221,360
License Agreement | Pfizer
Research and License Agreements
Milestone revenue				0	0	$ 0	0
License Agreement | Pfizer | Up-Front Payment Arrangement
Research and License Agreements
Upfront payment received			$ 22,500,000
Upfront payment revenue recognized								$ 22,500,000
Recognition period of up-front payment								1 year 6 months
Research Services Agreement | University Of Basel
Research and License Agreements
Royalty payment due against license fees received (as a percent)						10.00%
Milestone payment obligation				$ 0	$ 0	$ 0	$ 0
Achievement Of Specified Development Milestone | License Agreement | Pfizer
Research and License Agreements
Milestone revenue	$ 20,000,000	$ 15,000,000
Achievement Of Specified Development Milestone | License Agreement | Pfizer | Maximum
Research and License Agreements
Potential milestone payments			$ 115,000,000
Number of indications | item			2
Achievement Of Specified Regulatory Milestone | License Agreement | Pfizer | Maximum
Research and License Agreements
Potential milestone payments			$ 70,000,000
Number of indications | item			2
Achievement Of Specified Levels Of Annual Net Sales Of Licensed Products | License Agreement | Pfizer | Maximum
Research and License Agreements
Potential milestone payments			$ 135,000,000