Catalyst Pharmaceuticals (CPRX) 10-Q 10 May 17 2017 Q1 Quarterly report Financial data

Document and Entity Information

Document and Entity Information - shares	3 Months Ended
	Mar. 31, 2017	May 05, 2017
Document And Entity Information [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Mar. 31, 2017
Document Fiscal Year Focus	2,017
Document Fiscal Period Focus	Q1
Trading Symbol	CPRX
Entity Registrant Name	CATALYST PHARMACEUTICALS, INC.
Entity Central Index Key	1,369,568
Current Fiscal Year End Date	--12-31
Entity Filer Category	Accelerated Filer
Entity Common Stock, Shares Outstanding		83,786,229

Balance Sheets

Balance Sheets - USD ($)	Mar. 31, 2017	Dec. 31, 2016
Current Assets:
Cash and cash equivalents	$ 9,981,044	$ 13,893,064
Short-term investments	26,545,898	26,512,753
Prepaid expenses and other current assets	929,862	1,047,944
Total current assets	37,456,804	41,453,761
Property and equipment, net	231,246	244,204
Deposits	8,888	8,888
Total assets	37,696,938	41,706,853
Current Liabilities:
Accounts payable	917,928	933,176
Accrued expenses and other liabilities	987,612	1,161,359
Total current liabilities	1,905,540	2,094,535
Accrued expenses and other liabilities, non-current	175,992	181,162
Warrants liability, at fair value	519,461	122,226
Total liabilities	2,600,993	2,397,923
Commitments and contingencies
Stockholders' equity:
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding
Common stock, $0.001 par value, 150,000,000 shares authorized; 82,972,316 shares issued and outstanding at both March 31, 2017 and December 31, 2016	82,972	82,972
Additional paid-in capital	148,128,172	147,374,028
Accumulated deficit	(113,115,199)	(108,148,070)
Total stockholders' equity	35,095,945	39,308,930
Total liabilities and stockholders' equity	$ 37,696,938	$ 41,706,853

Balance Sheets (Parenthetical)

Balance Sheets (Parenthetical) - $ / shares	Mar. 31, 2017	Dec. 31, 2016
Statement of Financial Position [Abstract]
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	150,000,000	150,000,000
Common stock, shares issued	82,972,316	82,972,316
Common stock, shares outstanding	82,972,316	82,972,316

Statements of Operations (unaud

Statements of Operations (unaudited) - USD ($)	3 Months Ended
	Mar. 31, 2017	Mar. 31, 2016
Income Statement [Abstract]
Revenues	$ 0	$ 0
Operating costs and expenses:
Research and development	2,813,929	3,546,391
General and administrative	1,865,942	2,691,145
Total operating costs and expenses	4,679,871	6,237,536
Loss from operations	(4,679,871)	(6,237,536)
Other income, net	109,977	117,943
Change in fair value of warrants liability	(397,235)	733,356
Loss before income taxes	(4,967,129)	(5,386,237)
Provision for income taxes	0	0
Net loss	$ (4,967,129)	$ (5,386,237)
Net loss per share - basic and diluted	$ (0.06)	$ (0.07)
Weighted average shares outstanding - basic and diluted	82,972,316	82,860,083

Statement of Stockholders' Equi

Statement of Stockholders' Equity (unaudited) - 3 months ended Mar. 31, 2017 - USD ($)	Total	Common Stock [Member]	Additional Paid-in Capital [Member]	Accumulated Deficit [Member]
Beginning Balance at Dec. 31, 2016	$ 39,308,930	$ 82,972	$ 147,374,028	$ (108,148,070)
Issuance of stock options for services	735,536		735,536
Amortization of restricted stock for services	18,608		18,608
Net loss	(4,967,129)			(4,967,129)
Ending Balance at Mar. 31, 2017	$ 35,095,945	$ 82,972	$ 148,128,172	$ (113,115,199)

Statements of Cash Flows (unaud

Statements of Cash Flows (unaudited) - USD ($)	3 Months Ended
	Mar. 31, 2017	Mar. 31, 2016
Operating Activities:
Net loss	$ (4,967,129)	$ (5,386,237)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation	12,958	13,198
Stock-based compensation	754,144	457,969
Change in fair value of warrants liability	397,235	(733,356)
(Increase) decrease in:
Prepaid expenses and other current assets and deposits	118,082	480,846
Increase (decrease) in:
Accounts payable	(15,248)	(542,654)
Accrued expenses and other liabilities	(178,917)	(219,984)
Net cash used in operating activities	(3,878,875)	(5,930,218)
Investing Activities:
Purchase of short-term investments	(33,145)	(65,201)
Purchase of certificates of deposit		(370)
Net cash used in investing activities	(33,145)	(65,571)
Financing Activities:
Payment of employee withholding tax related to stock-based compensation		(11,265)
Net cash (used in) provided by financing activities		(11,265)
Net increase (decrease) in cash and cash equivalents	(3,912,020)	(6,007,054)
Cash and cash equivalents - beginning of period	13,893,064	28,235,016
Cash and cash equivalents - end of period	$ 9,981,044	$ 22,227,962

Organization and Description of

Organization and Description of Business	3 Months Ended
	Mar. 31, 2017
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Organization and Description of Business	1. Organization and Description of Business. Catalyst Pharmaceuticals, Inc. (the Company) is a development-stage biopharmaceutical company focused on developing and commercializing innovating therapies for people with rare debilitating diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndromes (CMS) and infantile spasms. Since inception, the Company has devoted substantially all of its efforts to business planning, research and development, recruiting management and technical staff, acquiring operating assets, and raising capital. The Company’s primary focus is on the development and commercialization of its drug candidates. The Company has incurred operating losses in each period from inception through March 31, 2017. The Company has been able to fund its cash needs to date through several public and private offerings of its common stock and warrants, through government grants, and through an investment by a strategic purchaser. See Note 9. Capital Resources On January 31, 2014, the Company filed a Shelf Registration Statement on Form S-3 No. 333-193699) On December 23, 2016, the Company filed a Shelf Registration Statement on Form S-3 No. 333-215315) While there can be no assurance, based on currently available information, the Company estimates that it currently has sufficient resources to support its operations for at least the next 12 months. The Company may raise required funds through public or private equity offerings, debt financings, corporate collaborations, governmental research grants or other means. The Company may also seek to raise new capital to fund additional product development efforts, even if it has sufficient funds for its planned operations. Any sale by the Company of additional equity or convertible debt securities could result in dilution to the Company’s current stockholders. There can be no assurance that any such required additional funding will be available to the Company at all or available on terms acceptable to the Company. Further, to the extent that the Company raises additional funds through collaborative arrangements, it may be necessary to relinquish some rights to the Company’s drug candidates or grant sublicenses on terms that are not favorable to the Company. If the Company is not able to secure additional funding when needed, the Company may have to delay, reduce the scope of, or eliminate one or more research and development programs, which could have an adverse effect on the Company’s business.

Basis of Presentation and Signi

Basis of Presentation and Significant Accounting Policies	3 Months Ended
	Mar. 31, 2017
Accounting Policies [Abstract]
Basis of Presentation and Significant Accounting Policies	2. Basis of Presentation and Significant Accounting Policies. a. INTERIM FINANCIAL STATEMENTS. 10-Q In the opinion of management, the accompanying unaudited interim financial statements of the Company contain all adjustments (consisting of only normal recurring adjustments) necessary to present fairly the financial position of the Company as of the dates and for the periods presented. Accordingly, these statements should be read in conjunction with the financial statements and notes thereto for the year ended December 31, 2016 included in the 2016 Annual Report on Form 10-K b. USE OF ESTIMATES. c. CASH AND CASH EQUIVALENTS. d. SHORT-TERM INVESTMENTS. e. PREPAID EXPENSES AND OTHER CURRENT ASSETS pre-commercialization pre-clinical f. FAIR VALUE OF FINANCIAL INSTRUMENTS. g. FAIR VALUE MEASUREMENTS. Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which are typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability. Fair Value Measurements at Reporting Date Using Balances as of Quoted Prices in Significant Significant Money market funds $ 9,868,419 $ 9,868,419 $ — $ — Short-term investments $ 26,545,898 $ 26,545,898 $ — $ — Warrants liability $ 519,461 $ — $ — $ 519,461 Fair Value Measurements at Reporting Date Using Balances as of Quoted Prices in Significant Significant Money market funds $ 13,395,759 $ 13,395,759 $ — $ — Short-term investments $ 26,512,753 $ 26,512,753 $ — $ — Warrants liability $ 122,226 $ — $ — $ 122,226 h. WARRANTS LIABILITY. i. STOCK-BASED COMPENSATION. As of March 31, 2017, there were outstanding stock options to purchase 6,088,333 shares of common stock, of which stock options to purchase 2,636,662 shares of common stock were exercisable as of March 31, 2017. For the three-month periods ended March 31, 2017 and 2016, the Company recorded stock-based compensation expense as follows: Three months ended March 31, 2017 2016 Research and development $ 206,352 $ 93,783 General and administrative 547,792 364,186 Total stock-based compensation $ 754,144 $ 457,969 j. COMPREHENSIVE INCOME (LOSS). k. NET LOSS PER SHARE. March 31, 2017 2016 Options to purchase common stock 6,088,333 4,345,000 Warrants to purchase common stock 2,407,663 2,407,663 Unvested restricted stock 26,667 53,334 Potential equivalent common stock excluded 8,522,663 6,805,997 Potentially dilutive options to purchase common stock as of both March 31, 2017 and 2016 have exercise prices ranging from $0.47 to $4.64. Potentially dilutive warrants to purchase common stock as of both March 31, 2017 and 2016 have exercise prices ranging from $1.04 to $2.08 and expire in periods between May 2017 and August 2017. l. RECENTLY ISSUED ACCOUNTING STANDARDS. No. 2016-02, Leases (Topic 842) 2016-02 On March 30, 2016, the FASB issued ASU No. 2016-09, Compensation—Stock Compensation (Topic 718) Improvements to Employee Share-Based Payment Accounting

Warrants Liability, at Fair Val

Warrants Liability, at Fair Value	3 Months Ended
	Mar. 31, 2017
Text Block [Abstract]
Warrants Liability, at Fair Value	3. Warrants Liability, at Fair Value. 2011 Warrants The Company allocated approximately $1.3 million of proceeds from its October 2011 registered direct offering to the fair value of common stock purchase warrants issued in connection with the offering that are classified as a liability (the 2011 warrants). The 2011 warrants are classified as a liability because of provisions in such warrants that allow for the net cash settlement of such warrants in the event of certain fundamental transactions (as defined in the warrant agreement). The valuation of the 2011 warrants is determined using the Black-Scholes Model. This model uses inputs such as the underlying price of the shares issued when the warrant is exercised, volatility, risk free interest rate and expected life of the instrument. The Company has determined that the 2011 warrants liability should be classified within Level 3 of the fair value hierarchy by evaluating each input for the Black-Scholes Model against the fair value hierarchy criteria and using the lowest level of input as the basis for the fair value classification. There are six inputs: closing price of the Company’s common stock on the day of evaluation; the exercise price of the warrants; the remaining expected term of the warrants; the volatility of the Company’s common stock; annual rate of dividends; and the risk-free rate of return. Of those inputs, the exercise price of the warrants and the remaining term are readily observable in the warrants agreement. The annual rate of dividends is based on the Company’s historical practice of not granting dividends. The closing price of the Company’s common stock would fall under Level 1 of the fair value hierarchy as it is a quoted price in an active market. The risk-free rate of return is a Level 2 input, while the historical volatility is a Level 3 input in accordance with the fair value accounting guidance. Since the lowest level input is a Level 3, the Company determined the 2011 warrants liability is most appropriately classified within Level 3 of the fair value hierarchy. This liability is subject to a fair value mark-to-market The calculated value of the 2011 warrants liability was determined using the Black-Scholes Model with the following assumptions: March 31, 2017 December 31, 2016 Risk free interest rate 1.03 % 0.85 % Expected term 0.09 years 0.33 years Expected volatility 110 % 100 % Expected dividend yield 0 % 0 % Expected forfeiture rate 0 % 0 % The following table rolls forward the fair value of the Company’s warrants liability activity for the three-month periods ended March 31, 2017 and 2016: Three months ended March 31, 2017 2016 Fair value, beginning of period $ 122,226 $ 1,008,363 Issuance of warrants — — Exercise of warrants — — Change in fair value 397,235 (733,356 ) Fair value, end of period $ 519,461 $ 275,007 During both the three months ended March 31, 2017 and 2016 none of the 2011 warrants were exercised. The Company recognizes the change in the fair value of the warrants liability as a non-operating

Prepaid Expenses and Other Curr

Prepaid Expenses and Other Current Assets	3 Months Ended
	Mar. 31, 2017
Text Block [Abstract]
Prepaid Expenses and Other Current Assets	4. Prepaid Expenses and Other Current Assets. Prepaid expenses and other current assets consist of the following: March 31, 2017 December 31, 2016 Prepaid research fees $ 303,898 $ 334,565 Prepaid insurance 457,487 598,909 Prepaid pre-commercialization — 35,500 Prepaid subscription fees 91,095 22,770 Prepaid rent 19,906 19,756 Other 57,476 36,444 Total prepaid expenses and other current assets $ 929,862 $ 1,047,944

Property and Equipment, net

Property and Equipment, net	3 Months Ended
	Mar. 31, 2017
Property, Plant and Equipment [Abstract]
Property and Equipment, net	5. Property and Equipment, net. Property and equipment, net consists of the following: March 31, 2017 December 31, 2016 Computer equipment $ 27,915 $ 27,915 Furniture and equipment 177,061 177,061 Leasehold improvements 152,708 152,708 357,684 357,684 Less: Accumulated depreciation (126,438 ) (113,480 ) Total property and equipment, net $ 231,246 $ 244,204 Depreciation expense was $12,958 and $13,198, respectively, for the three-month periods ended March 31, 2017 and 2016.

Accrued Expenses and Other Liab

Accrued Expenses and Other Liabilities	3 Months Ended
	Mar. 31, 2017
Payables and Accruals [Abstract]
Accrued Expenses and Other Liabilities	6. Accrued Expenses and Other Liabilities. Accrued expenses and other liabilities consist of the following: March 31, 2017 December 31, 2016 Accrued pre-clinical $ 571,624 $ 623,855 Accrued professional fees 124,653 102,673 Accrued compensation and benefits 85,534 264,237 Accrued license fees 178,750 152,500 Deferred rent and lease incentive 19,328 18,094 Other 7,723 — Current accrued expenses and other liabilities 987,612 1,161,359 Deferred rent and lease incentive - non-current 175,992 181,162 Non-current 175,992 181,162 Total accrued expenses and other liabilities $ 1,163,604 $ 1,342,521

Commitments and Contingencies

Commitments and Contingencies	3 Months Ended
	Mar. 31, 2017
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingencies	7. Commitments and Contingencies. a. LICENSE AGREEMENT WITH NORTHWESTERN UNIVERSITY. CPP-115. Under the license agreement with Northwestern, the Company is responsible for continued research and development of any resulting product candidates. As of March 31, 2017, the Company has paid $411,590 in connection with the license and has accrued license fees of $178,750 in the accompanying March 31, 2017 balance sheet for expenses, maintenance fees and milestones. In addition, the Company is obligated to pay certain milestone payments in future years relating to clinical development activities with respect to CPP-115, CPP-115 b. LICENSE AGREEMENT WITH NEW YORK UNIVERSITY AND THE FEINSTEIN INSTITUTE FOR MEDICAL RESEARCH. c. LICENSE AGREEMENT WITH BIOMARIN ® ® ® As part of the License Agreement, the Company agreed: (i) to pay BioMarin royalties for seven years from the first commercial sale of Firdapse ® ® ® ® ® d. AGREEMENTS FOR DRUG DEVELOPMENT, PRE-CLINICAL

Income Taxes

Income Taxes	3 Months Ended
	Mar. 31, 2017
Income Tax Disclosure [Abstract]
Income Taxes	8. Income Taxes. The Company is subject to income taxes in the U.S. federal jurisdiction and various states jurisdictions. Tax regulations within each jurisdiction are subject to the interpretation of the related tax laws and regulations and require significant judgment to apply. The Company is not subject to U.S. federal, state and local tax examinations by tax authorities for any years before 2013. If the Company were to subsequently record an unrecognized tax benefit, associated penalties and tax related interest expense would be reported as a component of income tax expense.

Stockholders' Equity

Stockholders' Equity	3 Months Ended
	Mar. 31, 2017
Equity [Abstract]
Stockholders' Equity	9. Stockholders’ Equity. 2014 Shelf Registration Statement On January 31, 2014, the Company filed a Shelf Registration Statement on Form S-3 No. 333-193699) (a) On April 3, 2014, the Company filed a prospectus supplement and offered for sale 13,023,750 shares of its common stock at a price of $2.21 per share in an underwritten public offering. The Company received gross proceeds in the public offering of approximately $28.8 million before underwriting commission and incurred expenses of approximately $2.1 million. (b) On February 4, 2015, the Company filed a prospectus supplement and offered for sale 11,500,000 shares of its common stock at a price of $3.25 per share in an underwritten public offering. The Company received gross proceeds in the public offering of approximately $37.4 million before underwriting commission and incurred expenses of approximately $2.5 million. 2016 Shelf Registration Statement On December 23, 2016, the Company filed a Shelf Registration Statement on Form S-3 No. 333-215315) Warrant Exercises No warrants were exercised during both the three months ended March 31, 2017 and 2016.

Stock Compensation

Stock Compensation	3 Months Ended
	Mar. 31, 2017
Disclosure of Compensation Related Costs, Share-based Payments [Abstract]
Stock Compensation	10. Stock Compensation. Stock Options During the three-month periods ended March 31, 2017 and 2016, the Company granted seven-year options to purchase an aggregate of 1,520,000 and 155,000 shares, respectively, of the Company’s common stock to employees and directors. The Company recorded stock-based compensation related to stock options totaling $735,536 and $439,206 respectively, during the three-month periods ended March 31, 2017 and 2016. During the three-month periods ended March 31, 2017 and 2016, respectively, 255,000 and 56,665 options vested. No stock options were exercised during both the three months ended March 31, 2017 and 2016. As of March 31, 2017, there was approximately $2,820,000 of unrecognized compensation expense related to non-vested Restricted Stock Units No restricted stock units were granted during the three months ended March 31, 2017 and 2016. The Company recorded stock-based compensation related to restricted stock units totaling $18,608 and $18,763, respectively, during the three-month periods ended March 31, 2017 and 2016. As of March 31, 2017, there was approximately $47,000 of total restricted stock unit compensation expense related to non-vested

Subsequent Events

Subsequent Events	3 Months Ended
	Mar. 31, 2017
Subsequent Events [Abstract]
Subsequent Events	11. Subsequent Events. Subsequent to quarter-end,

Basis of Presentation and Sig18

Basis of Presentation and Significant Accounting Policies (Policies)	3 Months Ended
	Mar. 31, 2017
Accounting Policies [Abstract]
INTERIM FINANCIAL STATEMENTS	a. INTERIM FINANCIAL STATEMENTS. 10-Q In the opinion of management, the accompanying unaudited interim financial statements of the Company contain all adjustments (consisting of only normal recurring adjustments) necessary to present fairly the financial position of the Company as of the dates and for the periods presented. Accordingly, these statements should be read in conjunction with the financial statements and notes thereto for the year ended December 31, 2016 included in the 2016 Annual Report on Form 10-K
USE OF ESTIMATES	b. USE OF ESTIMATES.
CASH AND CASH EQUIVALENTS	c. CASH AND CASH EQUIVALENTS.
SHORT-TERM INVESTMENTS	d. SHORT-TERM INVESTMENTS.
PREPAID EXPENSES AND OTHER CURRENT ASSETS	e. PREPAID EXPENSES AND OTHER CURRENT ASSETS pre-commercialization pre-clinical
FAIR VALUE OF FINANCIAL INSTRUMENTS	f. FAIR VALUE OF FINANCIAL INSTRUMENTS.
FAIR VALUE MEASUREMENTS	g. FAIR VALUE MEASUREMENTS. Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which are typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability. Fair Value Measurements at Reporting Date Using Balances as of Quoted Prices in Significant Significant Money market funds $ 9,868,419 $ 9,868,419 $ — $ — Short-term investments $ 26,545,898 $ 26,545,898 $ — $ — Warrants liability $ 519,461 $ — $ — $ 519,461 Fair Value Measurements at Reporting Date Using Balances as of Quoted Prices in Significant Significant Money market funds $ 13,395,759 $ 13,395,759 $ — $ — Short-term investments $ 26,512,753 $ 26,512,753 $ — $ — Warrants liability $ 122,226 $ — $ — $ 122,226
WARRANTS LIABILITY	h. WARRANTS LIABILITY.
STOCK-BASED COMPENSATION	i. STOCK-BASED COMPENSATION. As of March 31, 2017, there were outstanding stock options to purchase 6,088,333 shares of common stock, of which stock options to purchase 2,636,662 shares of common stock were exercisable as of March 31, 2017. For the three-month periods ended March 31, 2017 and 2016, the Company recorded stock-based compensation expense as follows: Three months ended March 31, 2017 2016 Research and development $ 206,352 $ 93,783 General and administrative 547,792 364,186 Total stock-based compensation $ 754,144 $ 457,969
COMPREHENSIVE INCOME (LOSS)	j. COMPREHENSIVE INCOME (LOSS).
NET LOSS PER SHARE	k. NET LOSS PER SHARE. March 31, 2017 2016 Options to purchase common stock 6,088,333 4,345,000 Warrants to purchase common stock 2,407,663 2,407,663 Unvested restricted stock 26,667 53,334 Potential equivalent common stock excluded 8,522,663 6,805,997 Potentially dilutive options to purchase common stock as of both March 31, 2017 and 2016 have exercise prices ranging from $0.47 to $4.64. Potentially dilutive warrants to purchase common stock as of both March 31, 2017 and 2016 have exercise prices ranging from $1.04 to $2.08 and expire in periods between May 2017 and August 2017.
RECENTLY ISSUED ACCOUNTING STANDARDS	l. RECENTLY ISSUED ACCOUNTING STANDARDS. No. 2016-02, Leases (Topic 842) 2016-02 On March 30, 2016, the FASB issued ASU No. 2016-09, Compensation—Stock Compensation (Topic 718) Improvements to Employee Share-Based Payment Accounting

Basis of Presentation and Sig19

Basis of Presentation and Significant Accounting Policies (Tables)	3 Months Ended
	Mar. 31, 2017
Accounting Policies [Abstract]
Fair Value Measurement Specific to Assets or Liability	The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability. Fair Value Measurements at Reporting Date Using Balances as of Quoted Prices in Significant Significant Money market funds $ 9,868,419 $ 9,868,419 $ — $ — Short-term investments $ 26,545,898 $ 26,545,898 $ — $ — Warrants liability $ 519,461 $ — $ — $ 519,461 Fair Value Measurements at Reporting Date Using Balances as of Quoted Prices in Significant Significant Money market funds $ 13,395,759 $ 13,395,759 $ — $ — Short-term investments $ 26,512,753 $ 26,512,753 $ — $ — Warrants liability $ 122,226 $ — $ — $ 122,226
Stock-Based Compensation Expense	For the three-month periods ended March 31, 2017 and 2016, the Company recorded stock-based compensation expense as follows: Three months ended March 31, 2017 2016 Research and development $ 206,352 $ 93,783 General and administrative 547,792 364,186 Total stock-based compensation $ 754,144 $ 457,969
Potential Shares Excluded from Determination of Basic and Diluted Net Loss Per Share	k. NET LOSS PER SHARE. March 31, 2017 2016 Options to purchase common stock 6,088,333 4,345,000 Warrants to purchase common stock 2,407,663 2,407,663 Unvested restricted stock 26,667 53,334 Potential equivalent common stock excluded 8,522,663 6,805,997

Warrants Liability, at Fair V20

Warrants Liability, at Fair Value (Tables)	3 Months Ended
	Mar. 31, 2017
Summary of Company's Warrants Liability Activity	The following table rolls forward the fair value of the Company’s warrants liability activity for the three-month periods ended March 31, 2017 and 2016: Three months ended March 31, 2017 2016 Fair value, beginning of period $ 122,226 $ 1,008,363 Issuance of warrants — — Exercise of warrants — — Change in fair value 397,235 (733,356 ) Fair value, end of period $ 519,461 $ 275,007
Warrants Liability, at Fair Value [Member]
Summary of Assumptions Used in Black-Scholes Model to Calculate Fair Value of Warrants Liability	The calculated value of the 2011 warrants liability was determined using the Black-Scholes Model with the following assumptions: March 31, 2017 December 31, 2016 Risk free interest rate 1.03 % 0.85 % Expected term 0.09 years 0.33 years Expected volatility 110 % 100 % Expected dividend yield 0 % 0 % Expected forfeiture rate 0 % 0 %

Prepaid Expenses and Other Cu21

Prepaid Expenses and Other Current Assets (Tables)	3 Months Ended
	Mar. 31, 2017
Text Block [Abstract]
Prepaid Expenses and Other Current Assets	Prepaid expenses and other current assets consist of the following: March 31, 2017 December 31, 2016 Prepaid research fees $ 303,898 $ 334,565 Prepaid insurance 457,487 598,909 Prepaid pre-commercialization — 35,500 Prepaid subscription fees 91,095 22,770 Prepaid rent 19,906 19,756 Other 57,476 36,444 Total prepaid expenses and other current assets $ 929,862 $ 1,047,944

Property and Equipment, net (Ta

Property and Equipment, net (Tables)	3 Months Ended
	Mar. 31, 2017
Property, Plant and Equipment [Abstract]
Property and Equipment, Net	Property and equipment, net consists of the following: March 31, 2017 December 31, 2016 Computer equipment $ 27,915 $ 27,915 Furniture and equipment 177,061 177,061 Leasehold improvements 152,708 152,708 357,684 357,684 Less: Accumulated depreciation (126,438 ) (113,480 ) Total property and equipment, net $ 231,246 $ 244,204

Accrued Expenses and Other Li23

Accrued Expenses and Other Liabilities (Tables)	3 Months Ended
	Mar. 31, 2017
Payables and Accruals [Abstract]
Schedule of Accrued Expenses and Other Liabilities	Accrued expenses and other liabilities consist of the following: March 31, 2017 December 31, 2016 Accrued pre-clinical $ 571,624 $ 623,855 Accrued professional fees 124,653 102,673 Accrued compensation and benefits 85,534 264,237 Accrued license fees 178,750 152,500 Deferred rent and lease incentive 19,328 18,094 Other 7,723 — Current accrued expenses and other liabilities 987,612 1,161,359 Deferred rent and lease incentive - non-current 175,992 181,162 Non-current 175,992 181,162 Total accrued expenses and other liabilities $ 1,163,604 $ 1,342,521

Organization and Description 24

Organization and Description of Business - Additional Information (Detail)	3 Months Ended
	Mar. 31, 2017Offerings	Dec. 23, 2016USD ($)	Jan. 31, 2014USD ($)
Organization And Description Of Business [Line Items]
Maximum dollar amount of common stock to be issued under shelf registration statement			$ 100,000,000
Number of direct offering under 2014 shelf registration | Offerings	2
Maximum dollar amount of common stock to be issued under shelf registration statement		$ 33,800,000
2014 Shelf Registration Statement [Member]
Organization And Description Of Business [Line Items]
Maximum dollar amount of common stock to be issued under shelf registration statement			$ 100,000,000
Expiry date of common stock remaining under the shelf registration statement	Mar. 19, 2017

Basis of Presentation and Sig25

Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) - USD ($)	1 Months Ended	3 Months Ended
	Oct. 31, 2011	Mar. 31, 2017	Mar. 31, 2016	Dec. 31, 2016
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Maximum maturity period of cash and cash equivalent		Three months
Number of options outstanding		6,088,333
Number of options exercisable		2,636,662
Stock option exercise price range, Minimum		$ 0.47	$ 0.47
Stock option exercise price range, Maximum		$ 4.64	$ 4.64
Trading Securities [Member] | Other Income, Net [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Unrealized gain		$ 29,430	$ 58,861
October 28, 2011 Warrants [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Warrants issued in offering	1,523,370
Warrants outstanding		763,913		763,913
Warrants expired date		May 2, 2017
Minimum [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Minimum amortization period of compensation cost on straight line basis		1 year
Potentially dilutive warrants to purchase common stock		$ 1.04	$ 1.04
Maximum [Member]
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items]
Minimum amortization period of compensation cost on straight line basis		3 years
Potentially dilutive warrants to purchase common stock		$ 2.08	$ 2.08

Basis of Presentation and Sig26

Basis of Presentation and Significant Accounting Policies - Fair Value Measurement Specific to Assets or Liability (Detail) - USD ($)	Mar. 31, 2017	Dec. 31, 2016
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Short-term investments	$ 26,545,898	$ 26,512,753
Warrants liability	519,461	122,226
Money Market Funds [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Money market funds	9,868,419	13,395,759
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Short-term investments	26,545,898	26,512,753
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] | Money Market Funds [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Money market funds	9,868,419	13,395,759
Significant Unobservable Inputs (Level 3) [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Warrants liability	$ 519,461	$ 122,226

Basis of Presentation and Sig27

Basis of Presentation and Significant Accounting Policies - Stock-Based Compensation Expense (Detail) - USD ($)	3 Months Ended
	Mar. 31, 2017	Mar. 31, 2016
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Total stock-based compensation	$ 754,144	$ 457,969
Research and Development [Member]
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Total stock-based compensation	206,352	93,783
General and Administrative [Member]
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items]
Total stock-based compensation	$ 547,792	$ 364,186

Basis of Presentation and Sig28

Basis of Presentation and Significant Accounting Policies - Potential Shares Excluded from Determination of Basic and Diluted Net Loss Per Share (Detail) - shares	3 Months Ended
	Mar. 31, 2017	Mar. 31, 2016
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential equivalent common stock excluded	8,522,663	6,805,997
Options to Purchase Common Stock [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential equivalent common stock excluded	6,088,333	4,345,000
Warrants Liability, at Fair Value [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential equivalent common stock excluded	2,407,663	2,407,663
Unvested Restricted Stock [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential equivalent common stock excluded	26,667	53,334

Warrants Liability, at Fair V29

Warrants Liability, at Fair Value - Additional Information (Detail) - USD ($)	1 Months Ended	3 Months Ended
	Oct. 31, 2011	Mar. 31, 2017	Mar. 31, 2016
Class of Warrant or Right [Line Items]
Recognition of warrants fair value at date of issuance		$ 0	$ 0
Number of warrants exercised during the period		0	0
October 28, 2011 Warrants [Member] | Warrants Liability, at Fair Value [Member]
Class of Warrant or Right [Line Items]
Recognition of warrants fair value at date of issuance	$ 1,300,000
Number of warrants exercised during the period		0	0

Warrants Liability, at Fair V30

Warrants Liability, at Fair Value - Summary of Assumptions Used in Black-Scholes Model to Calculate Fair Value of Warrants Liability (Detail) - Warrants Liability, at Fair Value [Member]	3 Months Ended	12 Months Ended
	Mar. 31, 2017	Dec. 31, 2016
Fair Value Inputs, Liabilities, Quantitative Information [Line Items]
Risk free interest rate	1.03%	0.85%
Expected term	1 month 2 days	3 months 29 days
Expected volatility	110.00%	100.00%
Expected dividend yield	0.00%	0.00%
Expected forfeiture rate	0.00%	0.00%

Warrants Liability, at Fair V31

Warrants Liability, at Fair Value - Summary of Company's Warrants Liability Activity (Detail) - USD ($)	3 Months Ended
	Mar. 31, 2017	Mar. 31, 2016
Fair Value Disclosures [Abstract]
Fair value, beginning of period	$ 122,226	$ 1,008,363
Issuance of warrants	0	0
Exercise of warrants	0	0
Change in fair value	397,235	(733,356)
Fair value, end of period	$ 519,461	$ 275,007

Prepaid Expenses and Other Cu32

Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($)	Mar. 31, 2017	Dec. 31, 2016
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract]
Prepaid research fees	$ 303,898	$ 334,565
Prepaid insurance	457,487	598,909
Prepaid pre-commercialization fees		35,500
Prepaid subscription fees	91,095	22,770
Prepaid rent	19,906	19,756
Other	57,476	36,444
Total prepaid expenses and other current assets	$ 929,862	$ 1,047,944

Property and Equipment, Net - P

Property and Equipment, Net - Property and Equipment, Net (Detail) - USD ($)	Mar. 31, 2017	Dec. 31, 2016
Property, Plant and Equipment [Line Items]
Property and equipment, gross	$ 357,684	$ 357,684
Less: Accumulated depreciation	(126,438)	(113,480)
Total property and equipment, net	231,246	244,204
Computer Equipment [Member]
Property, Plant and Equipment [Line Items]
Property and equipment, gross	27,915	27,915
Furniture and Equipment [Member]
Property, Plant and Equipment [Line Items]
Property and equipment, gross	177,061	177,061
Leasehold Improvements [Member]
Property, Plant and Equipment [Line Items]
Property and equipment, gross	$ 152,708	$ 152,708

Property and Equipment, net - A

Property and Equipment, net - Additional Information (Detail) - USD ($)	3 Months Ended
	Mar. 31, 2017	Mar. 31, 2016
Property, Plant and Equipment [Abstract]
Depreciation expenses	$ 12,958	$ 13,198

Accrued Expenses and Other Li35

Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($)	Mar. 31, 2017	Dec. 31, 2016
Payables and Accruals [Abstract]
Accrued pre-clinical and clinical trial expenses	$ 571,624	$ 623,855
Accrued professional fees	124,653	102,673
Accrued compensation and benefits	85,534	264,237
Accrued license fees	178,750	152,500
Deferred rent and lease incentive	19,328	18,094
Other	7,723
Current accrued expenses and other liabilities	987,612	1,161,359
Deferred rent and lease incentive - non-current	175,992	181,162
Non-current accrued expenses and other liabilities	175,992	181,162
Total accrued expenses and other liabilities	$ 1,163,604	$ 1,342,521

Commitments and Contingencies -

Commitments and Contingencies - Additional Information (Detail) - USD ($)	3 Months Ended
	Mar. 31, 2017	Dec. 31, 2016	Oct. 26, 2012
Commitments [Line Items]
Accrued license fees	$ 178,750	$ 152,500
Northwestern License Agreement [Member]
Commitments [Line Items]
License fee paid	411,590
Accrued license fees	178,750
Future milestone payment	$ 300,000
License Agreement with BioMarin [Member]
Commitments [Line Items]
Date on which strategic collaboration is entered into	Oct. 26, 2012
Investment pursuant to strategic collaboration			$ 5,000,000
Royalty agreement period	7 years
Net sales royalty threshold	$ 100,000,000
Milestone payment due upon NDA acceptance	2,600,000
Milestone payment due upon NDA approval	7,200,000
Costs paid for Firdapse joint studies	$ 3,800,000
License Agreement with BioMarin [Member] | Minimum [Member]
Commitments [Line Items]
Percentage of royalty on net sales	7.00%
License Agreement with BioMarin [Member] | Maximum [Member]
Commitments [Line Items]
Percentage of royalty on net sales	10.00%

Stockholders' Equity (2014 Shel

Stockholders' Equity (2014 Shelf Registration Statement) - Additional Information (Detail) - USD ($)	Feb. 04, 2015	Apr. 03, 2014	Mar. 31, 2017	Jan. 31, 2014
Stockholders' Equity [Line Items]
Maximum dollar amount of common stock to be issued under shelf registration statement				$ 100,000,000
2014 Shelf Registration Statement [Member]
Stockholders' Equity [Line Items]
Maximum dollar amount of common stock to be issued under shelf registration statement				$ 100,000,000
Expiry date of common stock remaining under the shelf registration statement			Mar. 19, 2017
Underwritten Public Offering [Member]
Stockholders' Equity [Line Items]
Number of common stock sold in offering	11,500,000	13,023,750
Common stock issued, price per share	$ 3.25	$ 2.21
Gross proceeds from issuance of common stock	$ 37,400,000	$ 28,800,000
Offering expenses	$ 2,500,000	$ 2,100,000

Stockholders' Equity (2016 Shel

Stockholders' Equity (2016 Shelf Registration Statement) - Additional Information (Detail) - USD ($)	Dec. 23, 2016	Jan. 31, 2014
Stockholders' Equity [Line Items]
Maximum dollar amount of common stock to be issued under shelf registration statement		$ 100,000,000
2016 Shelf Registration Statement [Member]
Stockholders' Equity [Line Items]
Maximum dollar amount of common stock to be issued under shelf registration statement	$ 33,800,000

Stockholders' Equity (Warrant E

Stockholders' Equity (Warrant Exercises) - Additional Information (Detail) - shares	3 Months Ended
	Mar. 31, 2017	Mar. 31, 2016
Equity [Abstract]
Number of warrants exercised during the period	0	0

Stock Compensation - Additional

Stock Compensation - Additional Information (Detail) - USD ($)	3 Months Ended
	Mar. 31, 2017	Mar. 31, 2016
Restricted Stock Units (RSUs) [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation	$ 18,608	$ 18,763
Expected remaining weighted average vesting period	7 months 13 days
Restricted stock unit granted	0	0
Total compensation expenses related to non-vested awards not yet recognized	$ 47,000
Options to Purchase Common Stock [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock option granted, contractual term	7 years	7 years
Stock options granted	1,520,000	155,000
Stock-based compensation	$ 735,536	$ 439,206
Stock option vested during the period	255,000	56,665
Number of stock options exercised	0	0
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan	$ 2,820,000
Expected remaining weighted average vesting period	1 year 8 months 9 days

Subsequent Events - Additional

Subsequent Events - Additional Information (Detail) - USD ($) $ in Millions	Apr. 01, 2017	Mar. 31, 2017	Mar. 31, 2016
Subsequent Event [Line Items]
Number of warrants exercised during the period		0	0
October 28, 2011 Warrants [Member]
Subsequent Event [Line Items]
Warrants expired date		May 2, 2017
Subsequent Event [Member]
Subsequent Event [Line Items]
Number of warrants exercised during the period	813,913
Issuance of common stock for warrant exercises	813,913
Proceeds from exercise of warrants	$ 1