Document and Entity Information
| Document and Entity Information - shares | 3 Months Ended | |
| Mar. 31, 2018 | May 10, 2018 | |
| Document and Entity Information | ||
| Entity Registrant Name | GTX INC /DE/ | |
| Entity Central Index Key | 1,260,990 | |
| Document Type | 10-Q | |
| Document Period End Date | Mar. 31, 2018 | |
| Amendment Flag | false | |
| Current Fiscal Year End Date | --12-31 | |
| Entity Current Reporting Status | Yes | |
| Entity Filer Category | Smaller Reporting Company | |
| Entity Common Stock, Shares Outstanding | 22,529,690 | |
| Document Fiscal Year Focus | 2,018 | |
| Document Fiscal Period Focus | Q1 |
CONDENSED BALANCE SHEETS
| CONDENSED BALANCE SHEETS - USD ($) $ in Thousands | Mar. 31, 2018 | Dec. 31, 2017 |
| Current assets: | ||
| Cash and cash equivalents | $ 14,958 | $ 15,816 |
| Short-term investments | 17,186 | 28,083 |
| Prepaid expenses and other current assets | 2,067 | 2,178 |
| Total current assets | 34,211 | 46,077 |
| Property and equipment, net | 43 | 51 |
| Intangible assets, net | 105 | 108 |
| Total assets | 34,359 | 46,236 |
| Current liabilities: | ||
| Accounts payable | 1,739 | 2,604 |
| Accrued expenses and other current liabilities | 7,871 | 5,371 |
| Total current liabilities | 9,610 | 7,975 |
| Commitments and contingencies | ||
| Stockholders' equity: | ||
| Common stock, $0.001 par value: 60,000,000 shares authorized at March 31, 2018 and December 31, 2017; 22,529,690 and 21,541,909 shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively | 23 | 22 |
| Additional paid-in capital | 599,920 | 599,876 |
| Accumulated deficit | (575,194) | (561,637) |
| Total stockholders' equity | 24,749 | 38,261 |
| Total liabilities and stockholders' equity | $ 34,359 | $ 46,236 |
CONDENSED BALANCE SHEETS (Paren
| CONDENSED BALANCE SHEETS (Parenthetical) - $ / shares | Mar. 31, 2018 | Dec. 31, 2017 |
| CONDENSED BALANCE SHEETS | ||
| Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
| Common stock, shares authorized | 60,000,000 | 60,000,000 |
| Common stock, shares issued | 22,529,690 | 21,541,909 |
| Common stock, shares outstanding | 22,529,690 | 21,541,909 |
CONDENSED STATEMENTS OF OPERATI
| CONDENSED STATEMENTS OF OPERATIONS - USD ($) $ in Thousands | 3 Months Ended | |
| Mar. 31, 2018 | Mar. 31, 2017 | |
| Expenses: | ||
| Research and development expenses | $ 11,000 | $ 4,193 |
| General and administrative expenses | 2,688 | 2,087 |
| Total expenses | 13,688 | 6,280 |
| Loss from operations | (13,688) | (6,280) |
| Other income, net | 131 | 27 |
| Net loss | $ (13,557) | $ (6,253) |
| Net loss per share -- basic and diluted (in dollars per share) | $ (0.62) | $ (0.39) |
| Weighted average shares outstanding - basic and diluted (in shares) | 21,967,805 | 16,018,342 |
CONDENSED STATEMENTS OF CASH FL
| CONDENSED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 3 Months Ended | |
| Mar. 31, 2018 | Mar. 31, 2017 | |
| Cash flows from operating activities: | ||
| Net loss | $ (13,557) | $ (6,253) |
| Adjustments to reconcile net loss to net cash used in operating activities: | ||
| Depreciation and amortization | 11 | 12 |
| Share-based compensation | 646 | 663 |
| Directors' deferred compensation | 42 | 41 |
| Changes in assets and liabilities: | ||
| Prepaid expenses and other assets | 111 | 189 |
| Accounts payable | (865) | (706) |
| Accrued expenses and other liabilities | 2,500 | 852 |
| Net cash used in operating activities | (11,112) | (5,202) |
| Cash flows from investing activities: | ||
| Purchase of short-term investments, held to maturity | (5,106) | (5,200) |
| Proceeds from maturities of short-term investments, held to maturity | 16,003 | 9,419 |
| Net cash provided by investing activities | 10,897 | 4,219 |
| Cash flows from financing activities: | ||
| Tax payments related to shares withheld for vested restricted stock units | (643) | (156) |
| Net cash used in financing activities | (643) | (156) |
| Net decrease in cash and cash equivalents | (858) | (1,139) |
| Cash and cash equivalents, beginning of period | 15,816 | 8,910 |
| Cash and cash equivalents, end of period | $ 14,958 | $ 7,771 |
Business and Basis of Presentat
| Business and Basis of Presentation | 3 Months Ended |
| Mar. 31, 2018 | |
| Business and Basis of Presentation | |
| Business and Basis of Presentation | 1. Business and Basis of Presentation Business and Going Concern GTx, Inc. (“GTx” or the “Company”), a Delaware corporation incorporated on September 24, 1997 and headquartered in Memphis, Tennessee, is a biopharmaceutical company dedicated to the discovery, development and commercialization of medicines to treat serious and/or significant unmet medical conditions, including stress urinary incontinence and prostate cancer. The Company is developing selective androgen receptor modulators (“SARMs”), including its lead product candidate, enobosarm (GTx-024). SARMs are a class of drugs that the Company believes has the potential to treat serious medical conditions where unmet medical needs in muscle-related diseases may benefit from increasing muscle mass, such as stress urinary incontinence (“SUI”). In 2016, the Company initiated a Phase 2 open-label, non-placebo controlled, proof-of-concept clinical trial of enobosarm to treat postmenopausal women with SUI. Based on the results from this ongoing proof-of-concept clinical trial, the Company initiated in the third quarter of 2017 a randomized, placebo-controlled Phase 2 clinical trial to evaluate the change in the mean number of daily SUI episodes following 12 weeks of enobosarm treatment. This trial is evaluating the safety and efficacy of enobosarm (1 mg and 3 mg) compared with placebo in postmenopausal women who have demonstrated SUI symptoms for more than six months, with an average of 3 to 15 reported SUI episodes per day over a three-day period, and a positive bladder stress test. The primary endpoint for the clinical trial is the percentage of patients with at least a 50 percent reduction in mean leaks per day at week 12, compared to baseline. The trial has completed enrollment of 493 women and the Company expects top-line data early in the fourth quarter of 2018. The Company previously announced that enobosarm achieved the pre-specified primary efficacy endpoint in both dose cohorts of the Phase 2 clinical trial designed to evaluate the efficacy and safety of a 9 mg and 18 mg dose of enobosarm in patients whose advanced breast cancer is both estrogen receptor (“ER”) positive and androgen receptor (“AR”) positive. After evaluating the breast cancer environment where the treatment paradigms are shifting to immunotherapies and/or combination therapies, the Company has decided that the time and cost of conducting the necessary clinical trials for potential approval in this indication does not warrant further development of enobosarm in this indication at this time. The Company has also evaluated several SARM compounds, including enobosarm, in preclinical models of Duchenne muscular dystrophy (“DMD”) where a SARM’s ability to increase muscle mass may prove beneficial to patients suffering from DMD. In order to further the development of a SARM for the treatment of DMD, the Company will need to enter into new collaborative arrangements or other strategic transactions with third parties with expertise in DMD and orphan drug indications. In 2015, the Company entered into an exclusive license agreement with the University of Tennessee Research Foundation (“UTRF”) to develop UTRF’s proprietary selective androgen receptor degrader (“SARD”) technology which may have the potential to provide compounds that can degrade multiple forms of AR to treat those patients who do not respond or are resistant to current therapies by inhibiting tumor growth in patients with progressive castration-resistant prostate cancer (“CRPC”). The Company has ongoing mechanistic preclinical studies to select the most appropriate SARD compound to potentially move into a first-in-human clinical trial. The Company’s ability to pursue the continued development of SARMs and its SARD program is contingent upon the Company’s ability to obtain additional funding. Accordingly, the Company is actively seeking additional funds through potential collaborative, partnering or other strategic arrangements to provide the Company with the necessary resources for the development of all of its preclinical and clinical product candidates. Based on its current business plan and assumptions, the Company estimates that its current cash, cash equivalents and short-term investments together with interest thereon, will be sufficient to meet its projected operating requirements only into the first quarter of 2019. Accordingly, the Company will need to raise substantial additional capital in the near term in order to fund its operations through and beyond the first quarter of 2019 and to continue as a going concern thereafter. In addition, the Company has based its cash sufficiency estimates on its current business plan and its assumptions that may prove to be wrong. The Company could utilize its available capital resources sooner than it currently expects, and it could need additional funding to sustain its operations even sooner than currently anticipated. The Company believes, based on its current estimates of clinical trial expenditures, that the Company’s existing capital resources will be adequate to enable it to complete its ongoing Phase 2 placebo-controlled clinical trial of enobosarm in postmenopausal women with SUI, in which top line results are expected early in the fourth quarter of 2018. However, the Company’s existing capital resources will not be sufficient to allow the Company to complete any additional clinical studies of enobosarm, to potentially advance a SARD compound into a first-in-human clinical trial, to undertake any additional preclinical or clinical development activities related to the development of SARMs as a potential treatment for DMD, and to fund the Company’s operations and to continue as a going concern. Also, the Company’s clinical trials may encounter technical or other difficulties that could increase its development costs beyond its current estimates or delay its development timelines, and the Company could otherwise exhaust its available financial resources sooner than it expects. The Company has evaluated its capital resources and current business plans in accordance with Accounting Standards Update (ASU) No. 2014-15, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern , (“ASU 2014-15”). ASU 2014-15 requires the assessment of an entity’s ability to continue as a going concern for a period of one year after the date the entity’s financial statements are issued and to provide related footnote disclosures, if necessary. As the Company currently estimates based on its current business plan and assumptions that its current cash, cash equivalents and short-term investments, together with interest thereon, will be sufficient to meet its projected operating requirements only into the first quarter of 2019, the Company has evaluated its ability to continue as a going concern for one year after the date these condensed financial statements are issued. Pursuant to the guidance in ASU 2014-15, the Company believes that it has the ability to successfully implement plans to mitigate the conditions that may raise doubt about its ability to continue as a going concern. The Company’s plans for mitigation include reducing or delaying expenditures by postponing or discontinuing additional clinical development activity for enobosarm and implementing other cost saving measures related to other research and development and general and administrative expenditures, which plans, if implemented, would materially harm the Company’s business. If the Company is unable to raise substantial additional capital in the near term, the Company believes it is probable that these plans could be effectively implemented to successfully mitigate the considerations regarding the Company’s ability to continue as a going concern for one year after the date these condensed financial statements are issued. Therefore, these condensed financial statements do not include any adjustments or charges that might be necessary should the Company be unable to continue as a going concern, such as charges related to impairment of its assets, the recoverability and classification of assets or the amounts and classification of liabilities or other similar adjustments. Basis of Presentation The accompanying unaudited condensed financial statements reflect, in the opinion of management, all adjustments (consisting of normal recurring adjustments) necessary for a fair presentation of GTx’s financial position, results of operations and cash flows for each period presented in accordance with accounting principles generally accepted in the United States for interim financial information and with the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, information and footnote disclosures normally included in financial statements prepared in accordance with accounting principles generally accepted in the United States have been condensed or omitted from the accompanying condensed financial statements. These interim condensed financial statements should be read in conjunction with the audited financial statements and related notes thereto, which are included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 13, 2018. Operating results for the three months ended March 31, 2018 are not necessarily indicative of the results that may be expected for the entire fiscal year ending December 31, 2018. Use of Estimates The preparation of condensed financial statements in conformity with accounting principles generally accepted in the United States requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the condensed financial statements, and the reported amounts of revenues and expenses during the reporting period. Actual amounts and results could differ from those estimates. Fair Value of Financial Instruments The carrying amounts of the Company’s financial instruments (which include cash, cash equivalents, short-term investments, and accounts payable) approximate their fair values. The Company’s financial assets and liabilities are classified within a three-level fair value hierarchy that prioritizes the inputs used to measure fair value, which is defined as follows: Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly Level 3 — Inputs that are unobservable for the asset or liability As the Company has the positive intent and ability to hold its certificates of deposit classified as short-term investments until maturity, these investments have been classified as held to maturity investments and are stated at cost, which approximates fair value. The Company considers these to be Level 2 investments as the fair values of these investments are determined using third-party pricing sources, which generally utilize observable inputs, such as interest rates and maturities of similar assets. Research and Development Expenses Research and development expenses include, but are not limited to, the Company’s expenses for personnel, supplies, and facilities associated with research activities, screening and identification of product candidates, formulation and synthesis activities, manufacturing, preclinical studies, toxicology studies, clinical trials, regulatory and medical affairs activities, quality assurance activities and license fees. The Company expenses these costs in the period in which they are incurred. The Company estimates its liabilities for research and development expenses in order to match the recognition of expenses to the period in which the actual services are received. As such, accrued liabilities related to third party research and development activities are recognized based upon the Company’s estimate of services received and degree of completion of the services in accordance with the specific third party contract. Cash, Cash Equivalents and Short-term Investments The Company considers highly liquid investments with initial maturities of three months or less to be cash equivalents. At March 31, 2018 and December 31, 2017, short-term investments consisted of Federal Deposit Insurance Corporation insured certificates of deposit with original maturities of greater than three months and less than one year. Income Taxes The Company accounts for deferred taxes by recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. A valuation allowance is provided when it is more likely than not that some portion or all of the deferred tax assets will not be realized. Accordingly, at March 31, 2018 and December 31, 2017, net of the valuation allowance, the net deferred tax assets were reduced to zero. Income taxes are described more fully in Note 8 to the Company’s financial statements included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 13, 2018. Other Income, net Other income, net consists of foreign currency transaction gains and losses, interest earned on the Company’s cash, cash equivalents and short-term investments, and other non-operating income or expense. Recent Accounting Pronouncements In February 2016, the Financial Accounting Standards Board issued Accounting Standard Update (“ASU”) 2016-02, Leases (Topic 842). This ASU requires that lessees recognize assets and liabilities on the balance sheet for the present value of the rights and obligations created by all leases with terms of more than 12 months. The ASU also will require disclosures designed to give financial statement users information on the amount, timing, and uncertainty of cash flows arising from leases. This new guidance will be effective for the Company as of January 1, 2019. The Company does not expect the adoption of the standard update to have a significant impact on its financial position or results of operations. Subsequent Events The Company has evaluated all events or transactions that occurred after March 31, 2018 up through the date the condensed financial statements were issued. There were no material recognizable or nonrecognizable subsequent events during the period evaluated. |
Share-Based Compensation
| Share-Based Compensation | 3 Months Ended |
| Mar. 31, 2018 | |
| Share-Based Compensation | |
| Share-Based Compensation | 2. Share-Based Compensation Share-based payments include stock option grants and restricted stock units, or RSUs, under the Company’s stock option and equity incentive plans and deferred compensation arrangements for the Company’s non-employee directors. The Company recognizes compensation expense for its share-based payments based on the fair value of the awards over the period during which an employee or non-employee director is required to provide service in exchange for the award. The Company’s share-based compensation plans are described more fully in Note 3 to the Company’s financial statements included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 13, 2018. The following table summarizes share-based compensation expense included within the condensed statements of operations for the three months ended March 31, 2018 and 2017: Three Months Ended 2018 2017 Research and development expenses $ $ General and administrative expenses Total share-based compensation $ $ Share-based compensation expense recorded as general and administrative expense for the three months ended March 31, 2018 and 2017 included share-based compensation expense related to deferred compensation arrangements for the Company’s non-employee directors of $42 and $41, respectively. The Company uses the Black-Scholes Model to value stock options. The expected life of options is determined by calculating the average of the vesting term and the contractual term of the options. The expected price volatility is based on the Company’s historical stock price volatility. The risk-free interest rate is determined using U.S. Treasury rates where the term is consistent with the expected life of the stock options. Expected dividend yield is not considered as the Company has not made any dividend payments and has no plans of doing so in the foreseeable future. The fair value of options granted was estimated using the following assumptions for the periods presented: Three Months Ended 2018 2017 Expected price volatility % % Risk-free interest rate % % Weighted average expected life in years 7 years 7 years The following is a summary of stock option transactions for all of the Company’s stock option and equity incentive plans since the Company’s most recent fiscal year end: Number of Shares Weighted Average Options outstanding at December 31, 2017 $ Options granted Options forfeited or expired — — Options outstanding at March 31, 2018 The Company estimates the fair value of RSUs using the closing price of its stock on the grant date. The fair value of RSUs is amortized on a straight-line basis over the requisite service period of the awards. The following is a summary of the RSU transactions for all of the Company’s equity incentive plans since the Company’s most recent fiscal year end: Number of Shares Nonvested RSUs outstanding at December 31, 2017 RSUs granted — RSUs vested ) RSUs forfeited — Nonvested RSUs outstanding at March 31, 2018 |
Basic and Diluted Net Income (L
| Basic and Diluted Net Income (Loss) Per Share | 3 Months Ended |
| Mar. 31, 2018 | |
| Basic and Diluted Net Income (Loss) Per Share | |
| Basic and Diluted Net Income (Loss) Per Share | 3. Basic and Diluted Net Income (Loss) Per Share Basic and diluted net income (loss) per share attributable to common stockholders is calculated based on the weighted average number of common shares outstanding during the period. Diluted net income (loss) per share gives effect to the dilutive potential of common stock consisting of stock options, unvested RSUs and common stock warrants. Weighted average potential shares of common stock of 11,862,772 and 8,257,283 for the three months ended March 31, 2018 and 2017, respectively, were excluded from the calculations of diluted income (loss) per share as inclusion of the potential shares would have had an anti-dilutive effect on the net income (loss) per share for the periods. |
Stockholders' Equity
| Stockholders' Equity | 3 Months Ended |
| Mar. 31, 2018 | |
| Stockholders' Equity | |
| Stockholders' Equity | 4. Stockholders’ Equity On February 9, 2018, the Company entered into an At-the-Market Equity Offering SM Sales Agreement (the “ATM Sales Agreement”) with Stifel, Nicolaus & Company, Incorporated, as sales agent (“Stifel”), pursuant to which the Company may offer and sell, from time to time, through Stifel, shares of the Company’s common stock, having an aggregate offering price of up to $50,000. As of March 31, 2018, no sales had been made by the Company under the ATM Sales Agreement. On September 29, 2017, the Company completed a private placement of units consisting of an aggregate of 5,483,320 shares of common stock and warrants to purchase an aggregate of 3,289,988 shares of its common stock for net proceeds of $45,648, after deducting placement agent fees and other offering expenses. The purchasers in the registered direct offering consisted solely of accredited investors that included certain institutional and existing stockholders, including a member of the Company’s board of directors. The warrants, which have five year terms expiring on September 29, 2022, are immediately exercisable and have a per share exercise price of $9.02. The Company assessed whether the warrants require accounting as derivatives. The Company determined that the warrants were indexed to the Company’s own stock. As such, the Company has concluded the warrants meet the scope exception for determining whether the instruments require accounting as derivatives and are classified in stockholders’ equity. The fair value of the warrants was estimated at $21,069 using the Black-Scholes Model with the following assumptions: expected volatility of 97%, risk free interest rate of 1.92%, expected life of five years and no dividends. The net proceeds from the private placement were allocated to the common stock and warrants based upon their relative fair values. On November 14, 2014, the Company completed a private placement of units consisting of an aggregate of 6,431,111 shares of common stock and warrants to purchase an aggregate of 6,430,948 shares of its common stock for net proceeds of $42,814, after deducting offering expenses. The net proceeds from the private placement were allocated to the common stock and warrants based upon the fair value method. Similarly, the offering expenses were allocated between the common stock and warrants with the portion allocated to common stock offset against the proceeds allocated to stockholders’ equity, whereas the portion allocated to the warrants was expensed immediately. The warrants have a per share exercise price of $8.50, became exercisable on May 6, 2015 and will continue to be exercisable for four years thereafter. During the three months ended March 31, 2018, certain holders of warrants issued in November 2014 exercised 1,111,082 warrants in a cashless exercise for which the Company issued an aggregate of 674,579 shares of common stock upon exercise. |
University of Tennessee Researc
| University of Tennessee Research Foundation License Agreements | 3 Months Ended |
| Mar. 31, 2018 | |
| University of Tennessee Research Foundation License Agreements | |
| University of Tennessee Research Foundation License Agreements | 5. University of Tennessee Research Foundation License Agreements The Company and UTRF are parties to a consolidated, amended and restated license agreement (the “SARM License Agreement”) pursuant to which the Company was granted exclusive worldwide rights in all existing SARM technologies owned or controlled by UTRF, including all improvements thereto, and exclusive rights to future SARM technology that may be developed by certain scientists at the University of Tennessee or subsequently licensed to UTRF under certain existing inter-institutional agreements with The Ohio State University. Under the SARM License Agreement, the Company is obligated to pay UTRF annual license maintenance fees, low single-digit royalties on net sales of products and mid-single-digit royalties on sublicense revenues. The Company and UTRF also entered into a license agreement (the “SARD License Agreement”) in March 2015 pursuant to which the Company was granted exclusive worldwide rights in all existing SARD technologies owned or controlled by UTRF, including all improvements thereto. Under the SARD License Agreement, the Company is obligated to employ active, diligent efforts to conduct preclinical research and development activities for the SARD program to advance one or more lead compounds into clinical development. The Company is also obligated to pay UTRF annual license maintenance fees, low single-digit royalties on net sales of products and additional royalties on sublicense revenues, depending on the state of development of a clinical product candidate at the time it is sublicensed. |
Business and Basis of Present11
| Business and Basis of Presentation (Policies) | 3 Months Ended |
| Mar. 31, 2018 | |
| Business and Basis of Presentation | |
| Basis of Presentation | Basis of Presentation The accompanying unaudited condensed financial statements reflect, in the opinion of management, all adjustments (consisting of normal recurring adjustments) necessary for a fair presentation of GTx’s financial position, results of operations and cash flows for each period presented in accordance with accounting principles generally accepted in the United States for interim financial information and with the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, information and footnote disclosures normally included in financial statements prepared in accordance with accounting principles generally accepted in the United States have been condensed or omitted from the accompanying condensed financial statements. These interim condensed financial statements should be read in conjunction with the audited financial statements and related notes thereto, which are included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 13, 2018. Operating results for the three months ended March 31, 2018 are not necessarily indicative of the results that may be expected for the entire fiscal year ending December 31, 2018. |
| Use of Estimates | Use of Estimates The preparation of condensed financial statements in conformity with accounting principles generally accepted in the United States requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the condensed financial statements, and the reported amounts of revenues and expenses during the reporting period. Actual amounts and results could differ from those estimates. |
| Fair Value of Financial Instruments | Fair Value of Financial Instruments The carrying amounts of the Company’s financial instruments (which include cash, cash equivalents, short-term investments, and accounts payable) approximate their fair values. The Company’s financial assets and liabilities are classified within a three-level fair value hierarchy that prioritizes the inputs used to measure fair value, which is defined as follows: Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly Level 3 — Inputs that are unobservable for the asset or liability As the Company has the positive intent and ability to hold its certificates of deposit classified as short-term investments until maturity, these investments have been classified as held to maturity investments and are stated at cost, which approximates fair value. The Company considers these to be Level 2 investments as the fair values of these investments are determined using third-party pricing sources, which generally utilize observable inputs, such as interest rates and maturities of similar assets. |
| Research and Development Expenses | Research and Development Expenses Research and development expenses include, but are not limited to, the Company’s expenses for personnel, supplies, and facilities associated with research activities, screening and identification of product candidates, formulation and synthesis activities, manufacturing, preclinical studies, toxicology studies, clinical trials, regulatory and medical affairs activities, quality assurance activities and license fees. The Company expenses these costs in the period in which they are incurred. The Company estimates its liabilities for research and development expenses in order to match the recognition of expenses to the period in which the actual services are received. As such, accrued liabilities related to third party research and development activities are recognized based upon the Company’s estimate of services received and degree of completion of the services in accordance with the specific third party contract. |
| Cash, Cash Equivalents and Short-term Investments | Cash, Cash Equivalents and Short-term Investments The Company considers highly liquid investments with initial maturities of three months or less to be cash equivalents. At March 31, 2018 and December 31, 2017, short-term investments consisted of Federal Deposit Insurance Corporation insured certificates of deposit with original maturities of greater than three months and less than one year. |
| Income Taxes | Income Taxes The Company accounts for deferred taxes by recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. A valuation allowance is provided when it is more likely than not that some portion or all of the deferred tax assets will not be realized. Accordingly, at March 31, 2018 and December 31, 2017, net of the valuation allowance, the net deferred tax assets were reduced to zero. Income taxes are described more fully in Note 8 to the Company’s financial statements included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 13, 2018. |
| Other Income, net | Other Income, net Other income, net consists of foreign currency transaction gains and losses, interest earned on the Company’s cash, cash equivalents and short-term investments, and other non-operating income or expense. |
| Recent Accounting Pronouncements | Recent Accounting Pronouncements In February 2016, the Financial Accounting Standards Board issued Accounting Standard Update (“ASU”) 2016-02, Leases (Topic 842). This ASU requires that lessees recognize assets and liabilities on the balance sheet for the present value of the rights and obligations created by all leases with terms of more than 12 months. The ASU also will require disclosures designed to give financial statement users information on the amount, timing, and uncertainty of cash flows arising from leases. This new guidance will be effective for the Company as of January 1, 2019. The Company does not expect the adoption of the standard update to have a significant impact on its financial position or results of operations. |
| Subsequent Events | Subsequent Events The Company has evaluated all events or transactions that occurred after March 31, 2018 up through the date the condensed financial statements were issued. There were no material recognizable or nonrecognizable subsequent events during the period evaluated. |
Share-Based Compensation (Table
| Share-Based Compensation (Tables) | 3 Months Ended |
| Mar. 31, 2018 | |
| Share-Based Compensation | |
| Summary of share-based compensation expense | Three Months Ended 2018 2017 Research and development expenses $ $ General and administrative expenses Total share-based compensation $ $ |
| Schedule of assumptions used to estimate fair value of options granted | Three Months Ended 2018 2017 Expected price volatility % % Risk-free interest rate % % Weighted average expected life in years 7 years 7 years |
| Summary of stock option transactions | Number of Shares Weighted Average Options outstanding at December 31, 2017 $ Options granted Options forfeited or expired — — Options outstanding at March 31, 2018 |
| Summary of RSU transactions | Number of Shares Nonvested RSUs outstanding at December 31, 2017 RSUs granted — RSUs vested ) RSUs forfeited — Nonvested RSUs outstanding at March 31, 2018 |
Business and Basis of Present13
| Business and Basis of Presentation (Details) - USD ($) $ in Thousands | Mar. 31, 2018 | Dec. 31, 2017 |
| Income Taxes | ||
| Net deferred tax assets | $ 0 | $ 0 |
Share-Based Compensation - Shar
| Share-Based Compensation - Share-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | |
| Mar. 31, 2018 | Mar. 31, 2017 | |
| Share-based compensation expense | ||
| Total share-based compensation | $ 688 | $ 704 |
| Share-based compensation expense related to deferred compensation arrangements for non-employee directors | 42 | 41 |
| Research and development expenses | ||
| Share-based compensation expense | ||
| Total share-based compensation | 281 | 233 |
| General and administrative expenses | ||
| Share-based compensation expense | ||
| Total share-based compensation | 407 | 471 |
| Director | General and administrative expenses | ||
| Share-based compensation expense | ||
| Share-based compensation expense related to deferred compensation arrangements for non-employee directors | $ 42 | $ 41 |
Share-Based Compensation - Stoc
| Share-Based Compensation - Stock Option Activity (Details) - $ / shares | 3 Months Ended | |
| Mar. 31, 2018 | Mar. 31, 2017 | |
| Stock-based compensation options granted | ||
| Expected price volatility (as a percent) | 93.10% | 88.40% |
| Risk-free interest rate (as a percent) | 2.30% | 2.20% |
| Weighted average expected life in years | 7 years | 7 years |
| Number of Shares | ||
| Options outstanding at the beginning of the period (in shares) | 1,900,496 | |
| Options granted (in shares) | 429,000 | |
| Options outstanding at the end of the period (in shares) | 2,329,496 | |
| Weighted Average Exercise Price Per Share | ||
| Options outstanding at the beginning of the period (in dollars per share) | $ 13.84 | |
| Options granted (in dollars per share) | 12.84 | |
| Options outstanding at the end of the period (in dollars per share) | $ 13.66 | |
Share-Based Compensation - RSU
| Share-Based Compensation - RSU Activity - (Details) - Restricted Stock Units (RSUs) | 3 Months Ended |
| Mar. 31, 2018shares | |
| Number of Shares | |
| Nonvested RSUs outstanding at the beginning of the period (in shares) | 380,500 |
| RSUs vested (in shares) | (363,833) |
| Nonvested RSUs outstanding at the end of the period (in shares) | 16,667 |
Basic and Diluted Net Income 17
| Basic and Diluted Net Income (Loss) Per Share (Details) - shares | 3 Months Ended | |
| Mar. 31, 2018 | Mar. 31, 2017 | |
| Basic and Diluted Net Income (Loss) Per Share | ||
| Weighted average potential shares of common stock excluded from calculation of diluted net income (loss) per share | 11,862,772 | 8,257,283 |
Stockholders' Equity (Details)
| Stockholders' Equity (Details) - USD ($) $ / shares in Units, $ in Thousands | Sep. 29, 2017 | May 06, 2015 | Nov. 14, 2014 | Mar. 31, 2018 | Feb. 09, 2018 |
| At-The-Market Equity Offering | |||||
| Common Stock | |||||
| Common stock value that may be sold under the At-the-Market Equity Offering Sales Agreement | $ 50,000 | ||||
| Value of sales made | $ 0 | ||||
| Private Placement, September 2017 | |||||
| Common Stock | |||||
| Shares issued under private placement | 5,483,320 | ||||
| Warrants issued to purchase shares under private placement | 3,289,988 | ||||
| Net proceeds from private placement after deducting offering expenses | $ 45,648 | ||||
| Exercise price (in dollars per share) | $ 9.02 | ||||
| Warrant term | 5 years | ||||
| Estimated fair value of warrants | $ 21,069 | ||||
| Expected volatility of warrants (as a percent) | 97.00% | ||||
| Risk-free interest rate of warrants (as a percent) | 1.92% | ||||
| Expected life of warrants | 5 years | ||||
| Expected dividends on warrants (in dollars per share) | $ 0 | ||||
| Private Placement, November 2014 | |||||
| Common Stock | |||||
| Shares issued under private placement | 6,431,111 | ||||
| Common stock issued upon exercise of warrants | 674,579 | ||||
| Warrants issued to purchase shares under private placement | 6,430,948 | ||||
| Net proceeds from private placement after deducting offering expenses | $ 42,814 | ||||
| Exercise price (in dollars per share) | $ 8.50 | ||||
| Warrant term | 4 years | ||||
| Warrants exercised | 1,111,082 |