Intercept Pharmaceuticals (ICPT) 10-Q 14 Nov 13 2013 Q3 Quarterly report Financial data

Document_And_Entity_Informatio

Document And Entity Information	9 Months Ended
	Sep. 30, 2013	Oct. 31, 2013
Document Information [Line Items]	'	'
Entity Registrant Name	'INTERCEPT PHARMACEUTICALS INC	'
Entity Central Index Key	'0001270073	'
Current Fiscal Year End Date	'--12-31	'
Entity Filer Category	'Non-accelerated Filer	'
Trading Symbol	'ICPT	'
Entity Common Stock, Shares Outstanding	'	19,335,576
Document Type	'10-Q	'
Amendment Flag	'false	'
Document Period End Date	30-Sep-13	'
Document Fiscal Period Focus	'Q3	'
Document Fiscal Year Focus	'2013	'

Condensed_Consolidated_Balance

Condensed Consolidated Balance Sheets (USD $)	Sep. 30, 2013	Dec. 31, 2012
Assets	'	'
Cash and cash equivalents	$33,492,017	$45,511,641
Investment securities, available-for-sale	123,260,775	64,682,270
Prepaid expenses and other current assets	1,601,213	1,584,308
Total current assets	158,354,005	111,778,219
Fixed assets, net	190,673	148,838
Security deposits	346,611	251,540
Total assets	158,891,289	112,178,597
Liabilities and Stockholders' Equity	'	'
Accounts payable, accrued expenses and other liabilities	5,079,581	3,745,773
Short-term portion of warrant liability	809,867	7,596,659
Short-term portion of deferred revenue	1,621,622	1,621,622
Total current liabilities	7,511,070	12,964,054
Long-term liabilities:	'	'
Long-term portion of warrant liability	51,093,479	22,762,135
Long-term portion of deferred revenue	9,324,324	10,540,543
Total liabilities	67,928,873	46,266,732
Stockholders' equity:	'	'
Common stock. 25,000,000 shares authorized; 16,526,885 and 19,300,957 shares issued and outstanding as of December 31, 2012 and September 30, 2013, respectively; par value $0.001 per share	19,301	16,527
Additional paid-in capital	264,519,497	184,100,139
Accumulated other comprehensive income (loss), net	31,106	-21,451
Accumulated deficit during development stage	-173,607,488	-118,183,350
Total stockholders’ equity	90,962,416	65,911,865
Total liabilities and stockholders’ equity	$158,891,289	$112,178,597

Condensed_Consolidated_Balance1

Condensed Consolidated Balance Sheets [Parenthetical] (USD $)	Sep. 30, 2013	Dec. 31, 2012
Common stock, par value (in dollars per share)	$0.00	$0.00
Common stock, authorized	25,000,000	25,000,000
Common stock, issued	19,300,957	16,526,885
Common stock, outstanding	19,300,957	16,526,885

Condensed_Consolidated_Stateme

Condensed Consolidated Statements of Operations and Comprehensive Loss (USD $)	3 Months Ended		9 Months Ended		133 Months Ended
	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013
Licensing revenue	$405,407	$523,191	$1,216,219	$2,040,700	$5,467,454
Costs and expenses:	'	'	'	'	'
Research and development	8,392,628	3,318,310	18,358,155	11,395,924	89,792,846
General and administrative	3,115,095	991,062	8,402,454	2,994,121	38,000,536
Total operating expenses	11,507,723	4,309,372	26,760,609	14,390,045	127,793,382
Other income (expense):	'	'	'	'	'
Revaluation of warrants	-20,756,086	-1,417,690	-30,010,672	-438,215	-53,086,248
Foreign currency loss on liquidation	0	0	0	-191,733	-191,733
Other income (expense), net	121,329	16,883	130,924	26,625	1,507,462
QTDP Grant	0	0	0	0	488,959
Total Other Nonoperating Income	-20,634,757	-1,400,807	-29,879,748	-603,323	-51,281,560
Net loss	-31,737,073	-5,186,988	-55,424,138	-12,952,668	-173,607,488
Dividends on preferred stock, not declared	0	-1,000,000	0	-2,500,000	-10,944,134
Net loss attributable to common stockholders	-31,737,073	-6,186,988	-55,424,138	-15,452,668	-184,551,622
Net loss per share, basic and diluted	($1.65)	($1.86)	($3.15)	($4.64)	'
Weighted average number of shares of common stock outstanding, basic and diluted:	19,198,923	3,329,266	17,585,531	3,329,266	'
Other comprehensive gain/(loss):	'	'	'	'	'
Unrealized gain / (loss) on investment securities	140,397	0	52,557	0	'
Foreign currency translation adjustments	0	0	0	184,500	'
Total comprehensive loss	($31,596,676)	($5,186,988)	($55,371,581)	($12,768,168)	'

Condensed_Consolidated_Stateme1

Condensed Consolidated Statements of Cash Flows (USD $)	9 Months Ended		133 Months Ended
	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013
Cash flows from operating activities:	'	'	'
Net loss	($55,424,138)	($12,952,668)	($173,607,488)
Adjustments to reconcile net loss to net cash used in operating activities:	'	'	'
Revaluation of warrants	30,010,672	438,215	53,086,248
Stock-based compensation	6,478,346	1,251,579	16,256,216
Amortization of investment premium	1,076,157	0	1,194,337
Depreciation & amortization	79,370	178,469	2,446,039
Foreign currency loss on liquidation	0	191,733	191,733
Loss from sale of assets	0	0	217,296
Impairment of bonds	0	0	151,402
Changes in:	'	'	'
Prepaid expenses and other assets	80,900	-1,110,136	-1,278,720
Accounts payable, accrued expenses, and other current liabilities	1,333,808	2,586,275	5,079,585
Deferred revenue	-1,216,219	-2,040,700	10,945,946
Interest accrued on promissory notes	0	0	91,249
Net cash used in operating activities	-17,581,104	-11,457,233	-85,226,157
Cash flows from investing activities:	'	'	'
Investments in certificates of deposit	0	120,399	-627,631
Purchases of investment securities	-86,231,665	0	-152,172,641
Sales of investment securities	26,436,682	0	27,555,757
Purchases of equipment, improvements, and furniture and fixtures	-121,205	-24,112	-1,518,443
Net cash used in investing activities	-59,916,188	96,287	-126,762,958
Cash flows from financing activities:	'	'	'
Proceeds from issuance of stock offerings, net of issuance costs	61,169,317	29,713,851	233,646,479
Proceeds from issuance of common stock warrants	0	0	7,385,897
Payments of capital lease obligation	0	-81,762	-1,335,567
Proceeds from exercise of options	4,300,250	0	4,342,955
Proceeds from exercise of warrants	8,101	0	383,101
Proceeds from issuance of convertible promissory notes payable	0	0	1,250,000
Net cash provided by financing activities	65,477,668	29,632,089	245,672,865
Effect of exchange rate changes	0	-7,233	-191,733
Net increase (decrease) in cash and cash equivalents	-12,019,624	18,263,910	33,492,017
Cash and cash equivalents - beginning of period	45,511,641	17,707,476	0
Cash and cash equivalents - end of period	33,492,017	35,971,386	33,492,017
Supplemental disclosure of cash flow information:	'	'	'
Cash paid during the year for interest	0	4,232	181,980
Supplemental disclosures of noncash activities:	'	'	'
Conversion of promissory note payable, including accrued interest of $91,250 into common shares	0	0	1,341,249
Issuance of 108,169 warrants for private placement agent fees	0	0	1,471,485
Acquisition of equipment pursuant to capital leases	0	0	1,335,567
Issuance of common stock for cashless warrant exchange	$8,455,833	$0	$9,380,323

Condensed_Consolidated_Stateme2

Condensed Consolidated Statements of Cash Flows [Parenthetical] (USD $)	133 Months Ended
	Sep. 30, 2013
Accrued interest on conversion of promissory note payable	$91,250
Issuance of warrants for private placement agent fees	108,169

Nature_of_Business_and_Basis_o

Nature of Business and Basis of Presentation	9 Months Ended
	Sep. 30, 2013
Organization, Consolidation and Presentation Of Financial Statements [Abstract]	'
Business Description and Basis of Presentation [Text Block]	'
		1	Nature of Business and Basis of Presentation
	Intercept Pharmaceuticals, Inc. (“Intercept” or the “Company”), a development stage company, is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic liver diseases utilizing its proprietary bile acid chemistry. The Company’s product candidates have the potential to treat orphan and more prevalent liver diseases for which there currently are limited therapeutic solutions.
	The Company has its administrative headquarters in New York, New York and an office in San Diego, California. Prior to April 2011, the Company operated a wholly-owned subsidiary in Italy where its bile acid receptor research was primarily conducted. In April 2011, the Company began the process of liquidating this subsidiary and has since disposed of all assets. However, the Company is continuing its early stage TGR5 (a bile acid receptor) research through its collaboration with Les Laboratories Servier and Institut de Recherches Servier, or collectively Servier. On March 15, 2013, the Company decided to remove the Italian subsidiary from the legal liquidation process, which removal was completed as of July 15, 2013, so that it could continue to act as the Company’s legal representative for its clinical trials in the European Union to satisfy European Union regulatory requirements. Intercept was incorporated in Delaware in September 2002.
	On September 13, 2012, the board of directors of the Company approved, and on September 25, 2012 the stockholders of the Company approved, a one-for-5.7778 reverse stock split of the Company’s outstanding common stock, which was effected on September 26, 2012. Stockholders entitled to fractional shares as a result of the reverse stock split received a cash payment in lieu of receiving fractional shares. Shares of common stock underlying outstanding stock options and other equity instruments were proportionately reduced and the respective exercise prices, if applicable, were proportionately increased in accordance with the terms of the agreements governing such securities. Shares of common stock reserved for issuance upon the conversion of the Company’s series A preferred stock, series B preferred stock, and series C preferred stock were proportionately reduced and the respective conversion prices were proportionately increased. All share and per share amounts in the financial statements have been retroactively adjusted for all periods presented to give effect to the reverse stock split, including reclassifying an amount equal to the reduction in par value to additional paid-in capital.
	The Company’s condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (GAAP). 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 the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates.
	The accompanying condensed interim financial statements are unaudited. The condensed interim unaudited financial statements have been prepared in accordance with GAAP on the same basis as the annual audited financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for a fair statement of the Company’s financial position, results of operations and cash flows for the dates and periods presented herein. These condensed consolidated financial statements should be read in conjunction with the consolidated financial statements and notes set forth in the Company’s Annual Report on Form 10-K for the year ended December 31, 2012 filed with the Securities and Exchange Commission on April 1, 2013. The results for the year ended December 31, 2012, the three and  nine months ended September 30, 2012 and September 30, 2013 (unaudited), and for the period from inception (September 4, 2002) through September 30, 2013 (unaudited) are not necessarily indicative of results to be expected for the year ending December 31, 2013, any other interim periods or any future year or period.

Summary_of_Significant_Account

Summary of Significant Accounting Policies	9 Months Ended
	Sep. 30, 2013
Accounting Policies [Abstract]	'
Significant Accounting Policies [Text Block]	'
		2	Summary of Significant Accounting Policies
	The Company’s significant accounting policies are described in Note 2 of the Notes to the Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2012.

Significant_Agreements

Significant Agreements	9 Months Ended
	Sep. 30, 2013
Significant Agreements Disclosure [Abstract]	'
Significant Agreements Disclosure [Text Block]	'
		3	Significant Agreements
	Dainippon Sumitomo Pharma Co, Ltd. (DSP)
	In March 2011, the Company entered into an exclusive license agreement with DSP to research, develop and commercialize obeticholic acid (OCA) as a therapeutic for the treatment of primary biliary cirrhosis (PBC) and nonalcoholic steatohepatitis (NASH) in Japan and China (excluding Taiwan). Under the terms of the license agreement, the Company received an up-front payment from DSP of $15.0 million and may be eligible to receive additional milestone payments up to an aggregate of approximately $30.0 million in development milestones based on the initiation or completion of clinical trials, $70.0 million in regulatory approval milestones and $200.0 million in sales milestones. The regulatory approval milestones include $15.0 million for receiving marketing approval for OCA for NASH in Japan, $10.0 million for receiving marketing approval for OCA for NASH in China, and up to $5.0 million for receiving marketing approval for OCA for PBC in the United States. The sales milestones are based on aggregate sales amounts of OCA and include $5.0 million for achieving net sales of $50.0 million, $10.0 million for achieving net sales of $100.0 million, $20.0 million for achieving net sales of $200.0 million, $40.0 million for achieving net sales of $400.0 million and $120.0 million for achieving net sales of $1.2 billion. DSP is also required to make royalty payments ranging from the tens to the twenties in percent based on net sales of OCA products in the DSP territory. DSP has the option to add several other Asian countries to its territory, including Korea and Taiwan, and to pursue OCA for additional indications. DSP will be responsible for the costs of developing and commercializing OCA in its territory.
	The Company evaluated the license agreement with DSP and determined that it is a revenue arrangement with multiple deliverables, or performance obligations. The Company’s substantive performance obligations under this license include an exclusive license to its technology, technical and scientific support to the development plan and participation on a joint steering committee. The Company determined that these performance obligations represent a single unit of accounting, since, initially, the license does not have stand-alone value to DSP without the Company’s technical expertise and steering committee participation during the development of OCA. This development period is currently estimated as continuing through June 2020 and, as such, the up-front payment is being recognized ratably over this period. During the three months ended September 30, 2012 and 2013, the Company recorded revenue of $405,000 and $405,000, respectively, and during the nine months ended September 30, 2012 and 2013, the Company recorded revenue of $1,215,000 and $1,216,000, respectively, in “Licensing Revenue” in its Consolidated Statement of Operations for the Company’s efforts under the agreement. The Company has not achieved any of the milestones relating to the agreement as of September 30, 2013 and has not recognized any revenue related to such milestones. The Company has determined that each potential future development, regulatory and sales milestone is substantive.
	Les Laboratories Servier and Institut de Recherches Servier
	In August 2011, the Company entered into a research collaboration agreement with Servier under which the Company granted Servier the exclusive license to research, develop and commercialize TGR5 agonists (other than the Company’s preclinical product candidates INT-767 and INT-777) for use in the treatment of diabetes, obesity, atherosclerosis and reperfusion injury in all countries other than the United States and Japan. The agreement expires when no payment obligations are or will become due and may be terminated earlier by the parties in certain circumstances.
	Under the terms of the agreement, the Company received an up-front payment from Servier of $1.4 million. The Company is also eligible to receive up to an aggregate of approximately €8.5 million in development milestones based on the initiation of clinical trials by Servier or the selection by Servier of product candidates for development, including a payment of €4.0 million upon the determination by Servier to initiate a Phase 3 clinical trial for the first product candidate under the agreement. The Company may also receive up to an aggregate of approximately €10.0 million in regulatory submission and approval milestones, including a payment of €5.0 million upon the first product candidate under the agreement achieving regulatory approval in the EU for its initial indication. The agreement also contemplates up to an aggregate of approximately €90.0 million in sales milestones, including a payment of €10.0 million upon the first product candidate under the agreement achieving its first commercial sale, €10.0 million upon achieving net sales of €200.0 million for a product, €20.0 million upon achieving net sales of €400.0 million for a product, €25.0 million for achieving net sales of €500.0 million for a product and €25.0 million for achieving net sales of €600.0 million for a product. Servier is also obligated to pay the Company royalties based on net sales of products developed under the agreement on a country-by-country basis.
	Intercept and Servier will jointly support the discovery effort, while Servier alone will be responsible for all costs associated with the global development, regulatory approval and commercialization of any compound selected as a lead candidate by the parties. The Company agreed to reimburse Servier up to a mid-double digit percentage of the total historical development costs incurred by Servier in relation to clinical development activities aimed at achieving regulatory approval in the European Union and the United States if the Company enters into a partnership agreement, or commences development or commercialization activities, with respect to any such compound in the United States. Servier may credit a portion of any reimbursable development costs against any milestone or royalty payments due and payable to the Company by Servier under the research collaboration agreement until all such reimbursable amounts are repaid. During the three months and nine months ended September 30, 2012 and 2013, the Company did not reimburse any development costs to Servier nor is it expected that any such costs will be reimbursed during 2013, as no such reimbursable developments costs are planned during the period.
	The Company evaluated the research collaboration agreement with Servier and determined that it is a revenue arrangement with multiple deliverables, or performance obligations. The Company’s substantive performance obligations under this research collaboration include an exclusive license to its technology, technical, scientific and intellectual property support to the research plan during the first year of the agreement and participation on an executive committee and a research and development committee. The Company determined that these performance obligations represent a single unit of accounting, since the license does not have stand-alone value to Servier without the Company’s technical expertise and committee participation during the initial 12-month period. The up-front payment was recognized ratably over the estimated 12-month performance period as the research and development and executive committee services were provided. During the three months and nine months ended September 30, 2012, the Company recorded revenue of $118,000 and $824,000, respectively, related to the Company’s efforts under the Servier arrangement, which was recorded in “Licensing Revenue” in the Company’s Consolidated Statement of Operations. As the up-front payment was fully recognized as of September 30, 2012, no revenue was recorded during the three months and nine months ended September 30, 2013 and no further revenue will be recognized for the up-front payment. The Company determined that each potential future development, regulatory and sales milestone is substantive.
	The Company is required to conduct a research program under the agreement to identify and optimize compounds that meet certain specified criteria sufficient for further development by Servier. The Company has engaged Professor Pellicciari and TES Pharma SRL (TES) to provide the research services required under the agreement, as described below. Servier is required to reimburse the Company for the full time equivalent costs incurred by the Company in the conduct of the research program, up to a set maximum amount. The Company recognizes the expense reimbursement from Servier as a reduction of research and development expenses as the Company is acting as an agent regarding these research activities. All amounts incurred by the Company for research under the Servier agreement during the three and nine months ended September 30, 2013, including the amounts incurred under the related agreements with Professor Pellicciari and TES, were covered under the Servier agreement. At December 31, 2012 and September 30, 2013, the Company recorded $496,000 and $507,000, respectively, in prepaid expenses and other assets for amounts due from Servier for such expense reimbursement.
	The research portion of the collaboration may be extended by mutual agreement by the parties for one or more additional years. In July 2012, February 2013 and August 2013, the term of the research program was extended until January 31, 2013, July 31, 2013 and September 30, 2013, respectively, on the same financial terms as the existing research program, including the reimbursement by Servier. In November 2013, the Company and Servier further extended the research program until September 30, 2015 on the same financial terms as the existing agreement, with such extension being effective as of October 1, 2013.
	Sponsored Research Agreement (SRA) with the University of Perugia and Professor Pellicciari
	The Company is engaged in a sponsored research agreement with the University of Perugia and Professor Roberto Pellicciari, a founder of the Company, to design, synthesize, optimize, scale-up, and develop pharmacologically active ligands for bile acid receptors. Under the SRA, the Company is assigned ownership of any patent and intellectual property rights arising from the research project. The Company paid the University of Perugia €100,000 quarterly commencing July 1, 2006 through 2010 and €100,000 for the fiscal year 2011. In 2012, the Company amended and restated the SRA to extend the term to the end of 2012 and paid the University of Perugia €80,000 during fiscal 2012. The Company recognized expense for the three months ended September 30, 2012 and 2013 of $16,000 and $26,000, respectively, and for the nine months ended September 30, 2012 and 2013 of $77,000 and $80,000, respectively. In April 2013, by mutual agreement of the parties, the term of this agreement was extended, effective as of January 1, 2013, until December 31, 2013 on the same financial terms as were previously in effect.
	Consulting Agreements with Professor Pellicciari
	The Company entered into an amended and restated consulting and intellectual property agreement with Professor Pellicciari on November 1, 2008, which was amended on October 27, 2010. Pursuant to this agreement, as amended, the Company was required to pay Professor Pellicciari €8,000 per month through December 31, 2010 for consulting services. The agreement also required the Company to make a lump sum payment of €172,500 and monthly payments of €12,000 through December 31, 2010 for the assignment of certain intellectual property rights. The Company entered into amended and restated consulting and intellectual property agreements with Professor Pellicciari on January 1, 2011 and January 1, 2012, pursuant to which the Company agreed to pay Professor Pellicciari an aggregate of €100,000 per year for services provided through December 31, 2012 for consulting services and intellectual property rights in relation to OCA, INT-767 and INT-777 product candidates. In April 2013, by mutual agreement of the parties, the term of this agreement was extended, effective as of January 1, 2013, until December 31, 2013 on the same financial terms as were previously in effect.
	On August 1, 2011, the Company signed a separate agreement with Professor Pellicciari for consulting services and intellectual property rights related to his services on the TGR5 program and the Servier license, pursuant to which the Company agreed to pay him an aggregate of €150,000 for his services through July 31, 2012. This agreement also provided that Professor Pellicciari will be eligible for a performance bonus of €50,000 based on the results of the research collaboration. The performance bonus is a discretionary bonus based upon the Company’s assessment of the success of the initial work performed under the collaboration, as extended. No such bonus was agreed upon by the parties as of September 30, 2013. In July 2012, February 2013 and August 2013, by mutual agreement of the parties, the term of this agreement was extended until January 31, 2013, July 31, 2013 and September 30, 2013, respectively, in conjunction with the extension of the term of the research program with Servier, on the same financial terms as the original consulting agreement with Professor Pellicciari. In November 2013, in conjunction with the extension of the research program with Servier, the parties further extended the consulting agreement until September 30, 2015 on the same financial terms as the original consulting agreement with Professor Pellicciari, with such extension being effective as of October 1, 2013.
	The Company recognized expense related to these agreements for the three months ended September 30, 2012 and 2013 of $80,000 and $68,000, respectively, and for the nine months ended September 30, 2012 and 2013 of $243,000 and $234,000, respectively.
	TES Pharma SRL (TES)
	In August 2011, the Company contracted with TES to provide research and development services for the Company’s TGR5 program through July 31, 2012 to enable the Company to uphold its obligations for providing such services under the Servier agreement described above. Professor Pellicciari is an owner of TES. The Company is required under the agreement to pay TES an aggregate amount of €250,000 each quarter during the term of the agreement. The agreement provides that any funds paid to TES that have not been expended or irrevocably committed at the expiration of the agreement will be refunded to the Company.
	The agreement had a term of one year unless the Company, in its sole discretion, extends the term of this agreement for one additional year on the same terms and conditions as the current agreement. In July 2012, February 2013 and August 2013, by mutual agreement of the parties, the term of this agreement was extended until January 31, 2013, July 31, 2013, and September 30, 2013, respectively, in conjunction with the extension of the term of the research program with Servier, on the same financial terms as the original agreement with TES.
	In November 2013, in conjunction with the extension of the research program with Servier, the parties agreed to further extend the research agreement with TES until September 30, 2015, with such extension being effective as of October 1, 2013. Pursuant to the amended agreement, the Company is required to pay TES an aggregate amount of €250,000 on a quarterly basis during the extended term. The Company also agreed to provide TES with a low single digit royalty based on worldwide net sales of products developed under the agreement on a country-by-country basis. Royalty rates are subject to reduction under the agreement in specified circumstances, including with respect to any country if sales of generic products reach a certain threshold in that country.
	The Company incurred charges related to this agreement for the three months ended September 30, 2012 and 2013 of $310,000 and $227,000, respectively, and $982,000 and $888,000 for the nine months ended September 30, 2012 and 2013, respectively.
	National Institute of Diabetes and Digestive and Kidney Disease Institute (NIDDK)
	In 2010, the Company contracted with the NIDDK of the National Institute of Health to research the effects of OCA for the treatment of patients with NASH in a Phase 2b clinical trial called the FLINT trial. Under the contract with the NIDDK, the Company made a milestone payment of $1.0 million in June 2012 following notification in June 2012 that the FLINT trial will continue based upon the results of a blinded interim analysis and a payment of $1.25 million following the completion of enrollment in the trial in November 2012. The Company does not have any additional contractual payments remaining.
	WIL Research Laboratories, LLC (WIL)
	On October 2, 2007, the Company entered into a master laboratory services agreement with WIL to perform certain research and laboratory services. The agreement was amended in October 2011. The agreement has a term ending on October 2, 2014, and automatically extends for successive one year periods, unless either party gives written notice to the other party at least 60 days prior to the end of the current term. Either the Company or WIL may terminate the agreement upon 90 days written notice. However, if a work order pertaining to the ongoing studies is outstanding, WIL may not terminate the agreement with 90 days written notice until the work order has been completed or otherwise terminated.
	On November 16, 2011, the Company finalized two work orders with WIL for FDA-required studies in mice and rats to investigate the presence or absence of carcinogenic potential of OCA. The Company agreed to pay an aggregate of $4.0 million for the studies, consisting of a combination of quarterly installment payments of approximately $300,000 and milestone payments totaling approximately $400,000 upon delivery of final result reports. If additional costs are incurred beyond the amounts specified in the work orders, the Company agreed to pay such reasonable additional costs upon receipt of proper invoice. The Company anticipates that all the studies will continue through completion, all milestones will be satisfied and that it will pay to WIL $4.0 million under this agreement. The Company recognized expense related to these contracts and other work orders for the three months ended September 30, 2012 and 2013 of $345,000 and $363,000, respectively, and for the nine months ended September 30, 2012 and 2013 of $1.2 million and $1.4 million, respectively.

Investments

Investments	9 Months Ended
	Sep. 30, 2013
Investments, Debt and Equity Securities [Abstract]	'
Investments in Debt and Marketable Equity Securities (and Certain Trading Assets) Disclosure [Text Block]	'
		4	Investments
	The following table summarizes the Company’s cash, cash equivalents and investments as of December 31, 2012 and September 30, 2013:
			As of December 31, 2012
			Amortized Cost		Gross		Gross		Fair Value
					Unrealized		Unrealized
					Gains		Losses
			(In thousands)
	Cash and cash equivalents:
	Cash and money market funds		$	45,512	$	—	$	—	$	45,512
	Investment securities:
	Commercial paper			12,971		10		-15		12,966
	Corporate debt securities			41,866		7		-23		41,850
	U.S. government and agency securities			9,861		4		—		9,865
	Total investments			64,698		21		-38		64,681
	Total cash, cash equivalents and		$	110,210	$	21	$	-38	$	110,193
	investments
			As of September 30, 2013
			Amortized		Gross		Gross
					Unrealized		Unrealized
			Cost		Gains		Losses		Fair Value
			(In thousands)
	Cash and cash equivalents:
	Cash and money market funds		$	33,492	$	—	$	—	$	33,492
	Investment securities:
	Commercial paper			4,998		1		—		4,999
	Corporate debt securities			105,905		78		-28		105,955
	U.S. government and agency securities			11,237		10		-		11,247
	Municipal government securities			1,061		—		-2		1,059
	Total investments			123,201		89		-30		123,260
	Total cash, cash equivalents and investments		$	156,693	$	89	$	-30	$	156,752
	The following table shows the fair value and gross unrealized losses of the Company’s available-for-sale investments aggregated by investment category and length of time that individual securities have been in the position:
			As of December 31, 2012
			Less Than 12 Months
			Fair Value		Gross
					Unrealized
					Holding Losses
			(In thousands)
	Commercial paper		$	10,461	$	-15
	Corporate debt securities			29,834		-23
	Available-for-sale securities		$	40,295	$	-38
			As of September 30, 2013
			Less Than 12 Months
			Fair Value		Gross
					Unrealized
					Holding Losses
			(In thousands)
	Corporate debt securities		$	31,265	$	-28
	Municipal government securities			1,059		-2
	Available-for-sale securities		$	32,324		-30

Prepaid_Expenses_and_Other_Cur

Prepaid Expenses and Other Current Assets	9 Months Ended
	Sep. 30, 2013
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract]	'
Other Assets Disclosure [Text Block]	'
		5	Prepaid Expenses and Other Current Assets
	Prepaid expenses and other current assets consisted of the following:
			December 31,		September 30,
			2012		2013
			(In thousands)
	Prepaid expenses		$	970	$	383
	Contract receivable			496		514
	Certificate of deposit			77		—
	Interest receivable			—		684
	Other receivable			—		20
	Refundable tax credits			42		—
	Prepaid expenses and other current assets		$	1,585	$	1,601

Fixed_Assets_Net

Fixed Assets, Net	9 Months Ended
	Sep. 30, 2013
Property, Plant and Equipment [Abstract]	'
Property, Plant and Equipment Disclosure [Text Block]	'
		6	Fixed Assets, Net
	Fixed assets, net consisted of the following:
			December 31,		September 30,
			2012		2013
			(In thousands)
	Office equipment		$	357	$	396
	Leasehold improvements			178		256
	Furniture and fixtures under capitalized lease			157		157
	Furniture and fixtures			121		125
	Subtotal fixed assets			813		934
	Less: accumulated depreciation and amortization			-664		-743
	Fixed assets, net		$	149	$	191
	Depreciation and amortization expense for the three months ended September 30, 2012 and 2013 was $24,000 and $27,000, respectively, and $178,000 and $79,000 for the nine months ended September 30, 2012 and 2013, respectively.

Accounts_Payable_Accrued_Expen

Accounts Payable, Accrued Expenses and Other Liabilities	9 Months Ended
	Sep. 30, 2013
Payables and Accruals [Abstract]	'
Accounts Payable and Accrued Liabilities Disclosure [Text Block]	'
		7	Accounts Payable, Accrued Expenses and Other Liabilities
	Accounts payable, accrued expenses and other liabilities consisted of the following:
			December 31,		September 30,
			2012		2013
			(In thousands)
	Accounts payable		$	1,180	$	1,384
	Accrued employee compensation			1,335		959
	Accrued contracted services and other			1,231		2,737
	Accounts payable, accrued expenses and other liabilities		$	3,746	$	5,080

Income_Taxes

Income Taxes	9 Months Ended
	Sep. 30, 2013
Income Tax Disclosure [Abstract]	'
Income Tax Disclosure [Text Block]	'
		8	Income Taxes
	The Company utilizes the asset and liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are recognized for the expected future tax consequences of temporary differences between the carrying amounts and the tax bases of assets and liabilities. A valuation allowance is established against net deferred tax assets if, based on the weight of available evidence, it is more likely than not that some or all of the net deferred tax assets will not be realized.
	Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be realized. The effect of a change in tax rates or laws on deferred tax assets and deferred tax liabilities is recognized in operations in the period that includes the enactment date of the rate change.
	The deferred tax asset or liability represents future tax return consequences of those differences, which will be taxable when the assets and liabilities are recovered or settled. The provision for income taxes may differ from the actual expense that would result from applying the federal statutory rate to income before taxes because certain expenses for financial reporting purposes are not deductible for tax purposes. At December 31, 2012 and September 30, 2013, the Company had available net operating loss carryforwards to reduce future taxable income of approximately $70.2 million and $97.6 million, respectively, for tax reporting purposes. These carryforwards expire between 2024 and 2032. The ability of the Company to utilize its net operating losses in future years is subject to limitation in accordance with provisions of Section 382 of the Internal Revenue Code due to previous ownership changes; however, these changes have not resulted in material limitations to the Company’s ability to utilize the net operating losses. The Company’s combined federal, state and city deferred tax asset of approximately $42.1 million and $52.1 million at December 31, 2012 and September 30, 2013, respectively, resulted from the tax effects of net operating losses and differences between the book and tax bases for the share-based compensation and depreciation. The Company does not have any deferred tax liabilities. Since the Company has not yet achieved sustained profitable operations, management believes its deferred tax assets do not satisfy the more-likely-than-not realization criteria and has provided an allowance for the full amount of the tax asset. As a result, the Company has not recorded any income tax benefit since its inception.

Warrants_to_Purchase_Common_St

Warrants to Purchase Common Stock	9 Months Ended
	Sep. 30, 2013
Warrants and Rights Note Disclosure [Abstract]	'
Warrants Disclosure [Text Block]	'
		9	Warrants to Purchase Common Stock
	In conjunction with various financing transactions, the Company issued warrants to purchase the Company’s common stock. Certain of the warrants include a provision that provides for a reduction in the warrant exercise price if there are subsequent issuances of additional shares of common stock for consideration per share less than the per share warrant exercise prices and the remaining warrants contain a provision that require the underlying shares to be registered upon an initial public offering (IPO). These warrants are deemed to be derivative instruments and as such, are recorded as a liability and are marked-to-market at each reporting period. The Company estimates the fair values of the warrants at each reporting period using a Black-Scholes option-pricing model. Management concluded, under the Company’s facts and circumstances, that the estimated fair values of the warrants using the Black-Scholes option-pricing model approximates, in all material respects, the values determined using a binomial valuation model. The estimates in the Black-Scholes option-pricing model and the binomial valuation model are based, in part, on subjective assumptions, including but not limited to stock price volatility, the expected life of the warrants, the risk free interest rate and the fair value of the common stock underlying the warrants, and could differ materially in the future. Changes in the fair value of the common stock warrant liability from the prior period are recorded as a component of other income and expense.
	The Company will continue to adjust the fair value of the common stock warrant liability at the end of each reporting period for changes in fair value from the prior period until the earlier of the exercise or expiration of the applicable common stock warrants or until such time that the warrants are no longer determined to be derivative instruments.
	As of September 30, 2013, the Company had outstanding warrants to purchase a total of 878,201 shares of its common stock, at a weighted average exercise price of $10.34 per share. Of these warrants, 8,494 expired in October 2013, 4,326 were converted into 3,852 shares of common stock upon “cashless” exercise in October 2013 and 865,381 will expire in January 2015, if not exercised. All of these warrants are “in the money” based on the market price of our common stock as of September 30, 2013.

Fair_Value_Measurements

Fair Value Measurements	9 Months Ended
	Sep. 30, 2013
Fair Value Disclosures [Abstract]	'
Fair Value Disclosures [Text Block]	'
	10.          Fair Value Measurements
	The Company categorizes its financial instruments into a three-level fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value into three broad levels. The fair value hierarchy gives the highest priority to quoted prices in active markets for identical assets or liabilities (Level 1) and the lowest priority to unobservable inputs (Level 3).
	Financial assets and liabilities recorded at fair value on the Company’s consolidated balance sheets are categorized as follows:
		⋅	Unadjusted Quoted Prices — The fair value of an asset or liability is based on unadjusted quoted prices in active markets for identical assets or liabilities (Level 1).
		⋅	Pricing Models with Significant Observable Inputs — The fair value of an asset or liability is based on information derived from either an active market quoted price, which may require further adjustment based on the attributes of the financial asset or liability being measured, or an inactive market transaction (Level 2).
		⋅	Pricing Models with Significant Unobservable Inputs — The fair value of an asset or liability is primarily based on internally derived assumptions surrounding the timing and amount of expected cash flows for the financial instrument. Therefore, these assumptions are unobservable in either an active or inactive market (Level 3).
	The following table presents the Company’s financial assets and liabilities, measured at fair value based on the hierarchy above:
					Fair Value Measurements Using
			Total		Quoted Prices		Significant		Significant
					in		Other		Unobservable
					Active		Observable		Inputs
					Markets for		Inputs		(Level 3)
					Identical		(Level 2)
					Assets or
					Liabilities
					(Level 1)
					(In thousands)
	31-Dec-12
	Assets:
	Money market funds		$	24,862	$	24,862	$	—	$	—
	Available for sale securities:
	Commercial paper		$	12,966	$	—	$	12,966	$	—
	Corporate debt securities			41,850		—		41,850		—
	U.S. government and agency securities			9,865		—		9,865		—
	Total financial assets		$	89,543	$	24,862	$	64,681	$	—
	Liabilities:
	Warrants to purchase common stock		$	-30,359	$	—	$	—	$	-30,359
	Total financial liabilities		$	-30,359	$	—	$	—	$	-30,359
	30-Sep-13
	Assets:
	Money market funds		$	16,426	$	16,426	$	—	$	—
	Available for sale securities:
	Commercial paper		$	4,999	$	—	$	4,999	$	—
	Corporate debt securities			105,955		—		105,955		—
	U.S. government and agency securities			11,247		—		11,247		—
	Municipal securities			1,059		—		1,059		—
	Total financial assets		$	139,686	$	16,426	$	123,260	$	—
	Liabilities:
	Warrants to purchase common stock		$	-51,903	$	—	$	—	$	-51,903
	Total financial liabilities		$	-51,903	$	—	$	—	$	-51,903
	Level 3 Valuation
	Financial assets or liabilities are considered Level 3 when their fair values are determined using pricing models, discounted cash flow methodologies or similar techniques and at least one significant model assumption or input is unobservable. The following table provides a summary of the changes in fair value of the Company’s financial liabilities measured at fair value on a recurring basis using significant unobservable inputs (Level 3) during the nine-month period ended September 30, 2013.
			Warrant
			Liability
			(In thousands)
	Level 3
	Balance at December 31, 2012		$	30,359
	Losses recognized in earnings			30,011
	Exercises			-8,467
	Balance at September 30, 2013		$	51,903
	The Company determines the fair value of its warrant liability based on the Black-Scholes pricing model based on the Company’s stock price at the measurement date, exercise price of the warrant, risk free interest rate and historical volatility.
	The estimated fair values of marketable debt securities (commercial paper, corporate debt securities, U.S. government and agency securities, and municipal securities) as of September 30, 2013, by contractual maturity, are as follows:
			Fair Value
			(In thousands)
	Due in one year or less		$	55,923
	Due after one year through two years			67,337
	Total investments in debt securities		$	123,260
	Actual maturities may differ from contractual maturities because issuers may have the right to call or prepay obligations without call or prepayment penalties.

Stockholders_Equity_and_Prefer

Stockholders' Equity and Preferred Stock	9 Months Ended
	Sep. 30, 2013
Equity [Abstract]	'
Stockholders' Equity Note Disclosure [Text Block]	'
		11	Stockholders’ Equity and Preferred Stock
	Common Stock
	In September 2002, the Company issued 949,035 shares of common stock at a price of $0.03 per share to the founders of the Company (Founders’ shares).
	In November 2002, the Company issued 60,576 shares of common stock at a price of $0.03 per share to the principal investigators and other researchers of the Company pursuant to an authorization by the Board of Directors to issue and sell these shares by subscription to the named parties in conjunction with the signing of certain research agreements.
	In October 2003, the Company issued 112,498 shares of common stock at a price of $0.03 per share to the two principal investigators pursuant to an authorization by the Board of Directors to issue and sell these shares by subscription.
	In October 2003, the Company repurchased and canceled 550,960 Founders’ shares from certain founders of the Company at a price of $0.03 per share.
	From October 2003 through May 2004, pursuant to a private placement agreement dated October 2003, the Company issued an aggregate of 392,163 shares of common stock at a price of $7.22 per share, receiving net proceeds of $2.4 million after $474,000 in related offering costs. In addition, Class A warrants to purchase 137,251 shares of common stock and Class B warrants to purchase 117,640 shares of common stock were issued to the placement agent and its assigns as additional placement agent commission under the terms of the placement agent agreement.
	In November 2005, the Company issued 51,922 shares of common stock, warrants with a two-year term to purchase 51,922 shares of common stock at an exercise price of $7.22 per share and warrants with a five-year term to purchase 86,538 shares of common stock at an exercise price of $7.22 per share, all pursuant to a private subscription agreement with two outside investors, receiving net proceeds of $375,000.
	In May 2006, pursuant to a private placement agreement, the Company issued 2,087,091 shares of common stock at a price of $9.82 per share, receiving net proceeds of $19.5 million, after $1.0 million in related offering costs. Also in May 2006, the Company’s 6% convertible promissory notes that were issued in February 2005 with a face amount of $1.3 million, along with $91,000 of accrued interest, were converted into 160,637 shares of common stock at a price of $8.35 per share pursuant to the mandatory conversion terms of the notes.
	In October 2012, the Company completed the IPO of its common stock pursuant to a registration statement on Form S-1. In the IPO, the Company sold an aggregate of 5,750,000 shares of common stock under the registration statement at a public offering price of $15.00 per share. Net proceeds were approximately $78.7 million, after deducting underwriting discounts and commissions and offering expenses payable by the Company. Upon the closing of the IPO, all outstanding shares of the Company’s preferred stock (described below) were converted into 7,403,817 shares of common stock. Additionally, upon completion of the IPO, the Company is now authorized to issue 25,000,000 shares of common stock, $0.001 par value per share, and 5,000,000 shares of preferred stock, $0.001 par value per share.
	In June 2013, the Company completed a public offering of 1,989,500 shares of its common stock at a public offering price of $33.01 per share. The shares were registered pursuant to a registration statement on Form S-1. Net proceeds were approximately $61.2 million, after deducting underwriting discounts and commissions and offering expenses payable by the Company.
	Dividends
	The holders of common stock are entitled to receive dividends from time to time as declared by the Board of Directors. No cash dividend may be declared or paid to common stockholders until paid on each series of outstanding preferred stock in accordance with their respective terms.
	Voting
	The holders of shares of common stock are entitled to one vote for each share held with respect to all matters voted on by the stockholders of the Company.
	Preferred Stock
	In May 2008, to effectuate the sale of Series A preferred stock, the Company amended and restated its Certificate of Incorporation in its entirety to increase the number of shares of preferred stock it was authorized to issue to 13,888,889 shares and to designate such shares as Series A preferred stock. In May 2008, 13,888,889 shares of Series A preferred stock were sold to Genextra, S.p.A. for net proceeds of $24.3 million, after $749,000 in related offering costs. In connection with this financing, the Company issued warrants with a five-year term to purchase 108,169 shares of common stock at $10.40 per share to the placement agent.
	In January 2010, the Company further amended and restated its Certificate of Incorporation in its entirety to increase the number of shares of preferred stock it was authorized to issue to 27,777,778 shares and designated 13,888,889 of such shares as Series B preferred stock. In January 2010, 13,888,889 shares of Series B preferred stock and a warrant with a five-year term to purchase 865,381 shares of common stock at $10.40 per share were sold to Genextra for $24.9 million, after $112,000 in related offering costs.
	In August 2012, the Company further amended and restated its Certificate of Incorporation in its entirety to increase the number of shares of preferred stock it was authorized to issue to 52,777,778 shares and designated 25,000,000 of such shares as Series C preferred stock. In August 2012, 15,000,000 shares of Series C preferred stock were sold to Genextra and OrbiMed Advisors LLC for $29.7 million, after $300,000 in related offering costs.
	Upon the completion of the IPO, all outstanding shares of the Company’s preferred stock were converted into 7,403,817 shares of common stock and all accrued dividends on the preferred stock were eliminated.

2003_Stock_Incentive_Plan_and_

2003 Stock Incentive Plan and 2012 Stock Plan	9 Months Ended
	Sep. 30, 2013
Disclosure Of Compensation Related Costs, Share-Based Payments [Abstract]	'
Disclosure of Compensation Related Costs, Share-based Payments [Text Block]	'
		12	2003 Stock Incentive Plan and 2012 Stock Plan
	In 2003, the Board of Directors and the stockholders of the Company approved the Amended and Restated 2003 Stock Incentive Plan (2003 Plan) which provided for the granting of restricted stock, stock options and other stock-related awards to officers, directors, employees, advisors, and consultants of the Company. Stock options were granted at exercise prices not less than the fair market value of the Company’s common stock at the dates of grant. In May 2006, June 2008 and January 2010, the number of common shares available was increased to 519,228, 865,381, and 1,384,610, respectively. Most options are scheduled to vest over a period of up to four years. The 2003 Plan was terminated upon the pricing of the IPO in October 2012, and 555,843 shares available under the 2003 Plan were added to the 2012 Plan. All outstanding options issued under the 2003 Plan as of the date of termination remained outstanding and are subject to their respective terms and the terms of the 2003 Plan.
	In September 2012, the Company’s board of directors and stockholders approved the 2012 Equity Incentive Plan (2012 Plan), which became effective upon the pricing of the Company’s IPO in October 2012. The 2012 Plan will expire on September 13, 2022. Under the 2012 Plan, the Company may grant incentive stock options, non-qualified stock options, restricted and unrestricted stock awards and other stock-based awards. On January 1, 2013, the numbers of shares reserved for issuance under the 2012 Plan was increased by 661,075 shares as a result of the automatic increase in shares reserved pursuant to the terms thereof. As of September 30, 2013, there were 470,714 shares of common stock authorized for future issuance under the 2012 Plan.
	The following table summarizes stock option activity during the nine months ended September 30, 2013:
			Number of		Weighted
			Shares		Average
					Exercise Price
	Outstanding, December 31, 2012			1,526,150	$	10.67
	Granted			522,950		35.14
	Exercised			-505,173		9.78
	Forfeited			-8,589		11.87
	Outstanding, September 30, 2013			1,535,338	$	19.29
	Exercisable, September 30, 2013			798,655	$	9.92
	The following table summarizes stock-based compensation expense for employee, director and consultant stock option grants and restricted stock grants:
			Nine Months Ended
			September 30,
			2012		2013
			(In thousands)
	Stock option expense:
	Employees and directors		$	1,138	$	3,134
	Consultants			113		1,758
				1,251		4,892
	Restricted stock expense:
	Employees and directors			—		1,315
	Consultants			—		271
				—		1,586
	Total		$	1,251	$	6,478
	The following table summarizes the activity related to the Company’s restricted stock units for the nine month period ended September 30, 2013.
			Number of		Weighted
			Shares		Average
					Grant Date
					Fair
					Value
	Outstanding, December 31, 2012			176,188	$	21.23
	Granted			24,690		31.9
	Vested and converted			-66,309		21.23
	Forfeited			—		—
	Outstanding, September 30, 2013			134,569	$	23.19
	On November 16, 2012 and May 7, 2013, the Company granted to employees and directors restricted stock units (“RSUs”) for 173,592 and 24,690 shares of common stock, respectively, under the 2012 Plan. During the three months ended June 30, 2013, the Company issued 36,168 shares of its common stock to settle the vesting of 54,646 RSUs, of which 44,296 RSUs were net settled by withholding 18,478 shares, which represented the employees' minimum statutory obligation for each such employee's applicable income and other employment taxes, and remitted cash totaling $639,000 to the appropriate tax authorities. The Company does not have a policy for net settlement of RSUs but elected to offer to do this on a one-time basis to its employees for the first vesting of the RSUs on April 9, 2013. The amount remitted for the employees' tax obligation to the tax authorities was reflected as a financing activity within the Company’s consolidated statements of cash flows. These shares withheld by the Company as a result of the net settlement of the RSUs are no longer considered issued and outstanding, thereby reducing the Company’s shares outstanding used to calculate earnings per share.

Net_Loss_Per_Share

Net Loss Per Share	9 Months Ended
	Sep. 30, 2013
Earnings Per Share [Abstract]	'
Earnings Per Share [Text Block]	'
		13	Net Loss Per Share
	The following table presents the historical computation of basic and diluted net loss per share:
			Three Months Ended				Nine Months Ended
			September 30,				September 30,
			2012		2013		2012		2013
			(In thousands, except share and per share amounts)
	Historical net loss per share
	Numerator:
	Net loss attributable to common stockholders		$	-6,187	$	-31,737	$	-15,453	$	-55,424
	Denominator:
	Weighted average number of shares of common stock			3,329,266		19,198,923		3,329,266		17,585,531
	outstanding, basic and diluted:
	Net loss per share, basic and diluted		$	-1.86	$	-1.65	$	-4.64	$	-3.15
	The following potentially dilutive securities have been excluded from the computations of diluted weighted average shares outstanding:
			As of September 30,
			2012		2013
			(In thousands)
	Shares issuable upon conversion of preferred stock			7,404		-
	Shares issuable pursuant to accumulated preferred stock dividend			1,037		-
	Options			1,322		1,535
	Warrants to purchase common stock			1,233		878
	Restricted stock units			-		135
	Total			10,996		2,548

Recent_Accounting_Pronouncemen

Recent Accounting Pronouncements	9 Months Ended
	Sep. 30, 2013
New Accounting Pronouncements and Changes in Accounting Principles [Abstract]	'
New Accounting Pronouncements and Changes in Accounting Principles [Text Block]	'
		14	Recent Accounting Pronouncements
	In February 2013, the Financial Accounting Standards Board (“FASB”) issued ASU 2013-02, “Comprehensive Income: Reporting of Amounts Reclassified Out of Accumulated Other Comprehensive Income.” This accounting standard is an update to existing guidance on the presentation of comprehensive income. This update requires companies to report the effect of significant reclassifications out of accumulated other comprehensive income (AOCI) by component. For significant items reclassified out of AOCI to net income in their entirety during the reporting period, companies must report the effect on the line items in the statement when net income is presented. For significant items not reclassified to net income in their entirety during the period, companies must provide cross references in the notes to other disclosures that already provide information about those amounts. The Company adopted this amendment effective January 1, 2013 and it did not have a material impact on the results of operations or financial position of the Company.

Subsequent_Events

Subsequent Events	9 Months Ended
	Sep. 30, 2013
Subsequent Events [Abstract]	'
Subsequent Events [Text Block]	'
		15	Subsequent Events
	In October 2013, the Company completed a secondary offering of common stock sold by the Company’s two largest stockholders. The selling stockholders, both of which are affiliated with certain of the Company’s directors, sold an aggregate of 1,665,000 shares of common stock in the offering (inclusive of 165,000 shares of common stock from the partial exercise of the option granted to the underwriters to purchase additional shares from the selling stockholders). The Company did not receive any proceeds from the sale of shares by the selling stockholders. The anticipated offering expenses for the secondary offering are approximately $275,000, which includes legal and accounting costs and various other fees associated with the offering.
	In October 2013, the Company entered into a lease agreement to provide the Company with 11,124 rentable square feet (the Primary Space) in New York City for its new corporate headquarters. The rent for the Primary Space (without giving effect to any rent abatements) for the first year will be approximately $801,000 and will increase by two percent annually until the end of the fifth lease year, at which point the rent will increase to approximately $940,000 and subsequently increase by two percent annually until July 2024, the termination date.  The Company also has a pre-negotiated commitment to take an additional 9,502 rentable square feet (the Additional Space) under a co-terminus lease term expected to commence in September 2014, although the Company may in its sole discretion opt not to take the Additional Space before April 30, 2014 upon the payment of a termination fee of up to approximately $171,000. The rent for the Additional Space (without giving effect to any rent abatements) for the first year will be approximately $684,000 and will increase by two percent annually until the end of the fifth lease year, at which point the rent will increase to approximately $803,000 and subsequently increase by two percent annually until the termination date. The lease requires the Company to provide the landlord with a letter of credit for one year’s annual rent.
	In November 2013, the Company entered into an amendment to its collaboration with Servier, together with amendments to its related agreements with Professor Pellicciari and TES, in each case, effective as of October 1, 2013. See Note 3.

Investments_Tables

Investments (Tables)	9 Months Ended
	Sep. 30, 2013
Investments, Debt and Equity Securities [Abstract]	'
Schedule of Available-for-sale Securities Reconciliation [Table Text Block]	'
	The following table summarizes the Company’s cash, cash equivalents and investments as of December 31, 2012 and September 30, 2013:
		As of December 31, 2012
		Amortized Cost		Gross		Gross		Fair Value
				Unrealized		Unrealized
				Gains		Losses
		(In thousands)
	Cash and cash equivalents:
	Cash and money market funds	$	45,512	$	—	$	—	$	45,512
	Investment securities:
	Commercial paper		12,971		10		-15		12,966
	Corporate debt securities		41,866		7		-23		41,850
	U.S. government and agency securities		9,861		4		—		9,865
	Total investments		64,698		21		-38		64,681
	Total cash, cash equivalents and	$	110,210	$	21	$	-38	$	110,193
	investments
		As of September 30, 2013
		Amortized		Gross		Gross
				Unrealized		Unrealized
		Cost		Gains		Losses		Fair Value
		(In thousands)
	Cash and cash equivalents:
	Cash and money market funds	$	33,492	$	—	$	—	$	33,492
	Investment securities:
	Commercial paper		4,998		1		—		4,999
	Corporate debt securities		105,905		78		-28		105,955
	U.S. government and agency securities		11,237		10		-		11,247
	Municipal government securities		1,061		—		-2		1,059
	Total investments		123,201		89		-30		123,260
	Total cash, cash equivalents and investments	$	156,693	$	89	$	-30	$	156,752
Available-for-sale Securities [Table Text Block]	'
	The following table shows the fair value and gross unrealized losses of the Company’s available-for-sale investments aggregated by investment category and length of time that individual securities have been in the position:
		As of December 31, 2012
		Less Than 12 Months
		Fair Value		Gross
				Unrealized
				Holding Losses
		(In thousands)
	Commercial paper	$	10,461	$	-15
	Corporate debt securities		29,834		-23
	Available-for-sale securities	$	40,295	$	-38
		As of September 30, 2013
		Less Than 12 Months
		Fair Value		Gross
				Unrealized
				Holding Losses
		(In thousands)
	Corporate debt securities	$	31,265	$	-28
	Municipal government securities		1,059		-2
	Available-for-sale securities	$	32,324		-30

Prepaid_Expenses_and_Other_Cur1

Prepaid Expenses and Other Current Assets (Tables)	9 Months Ended
	Sep. 30, 2013
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract]	'
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Table Text Block]	'
	Prepaid expenses and other current assets consisted of the following:
		December 31,		September 30,
		2012		2013
		(In thousands)
	Prepaid expenses	$	970	$	383
	Contract receivable		496		514
	Certificate of deposit		77		—
	Interest receivable		—		684
	Other receivable		—		20
	Refundable tax credits		42		—
	Prepaid expenses and other current assets	$	1,585	$	1,601

Fixed_Assets_Net_Tables

Fixed Assets, Net (Tables)	9 Months Ended
	Sep. 30, 2013
Property, Plant and Equipment [Abstract]	'
Property, Plant and Equipment [Table Text Block]	'
	Fixed assets, net consisted of the following:
		December 31,		September 30,
		2012		2013
		(In thousands)
	Office equipment	$	357	$	396
	Leasehold improvements		178		256
	Furniture and fixtures under capitalized lease		157		157
	Furniture and fixtures		121		125
	Subtotal fixed assets		813		934
	Less: accumulated depreciation and amortization		-664		-743
	Fixed assets, net	$	149	$	191

Accounts_Payable_Accrued_Expen1

Accounts Payable, Accrued Expenses and Other Liabilities (Tables)	9 Months Ended
	Sep. 30, 2013
Payables and Accruals [Abstract]	'
Schedule of Accounts Payable and Accrued Liabilities [Table Text Block]	'
	Accounts payable, accrued expenses and other liabilities consisted of the following:
		December 31,		September 30,
		2012		2013
		(In thousands)
	Accounts payable	$	1,180	$	1,384
	Accrued employee compensation		1,335		959
	Accrued contracted services and other		1,231		2,737
	Accounts payable, accrued expenses and other liabilities	$	3,746	$	5,080

Fair_Value_Measurements_Tables

Fair Value Measurements (Tables)	9 Months Ended
	Sep. 30, 2013
Fair Value Disclosures [Abstract]	'
Fair Value, Liabilities Measured on Recurring and Nonrecurring Basis [Table Text Block]	'
	The following table presents the Company’s financial assets and liabilities, measured at fair value based on the hierarchy above:
				Fair Value Measurements Using
		Total		Quoted Prices		Significant		Significant
				in		Other		Unobservable
				Active		Observable		Inputs
				Markets for		Inputs		(Level 3)
				Identical		(Level 2)
				Assets or
				Liabilities
				(Level 1)
				(In thousands)
	31-Dec-12
	Assets:
	Money market funds	$	24,862	$	24,862	$	—	$	—
	Available for sale securities:
	Commercial paper	$	12,966	$	—	$	12,966	$	—
	Corporate debt securities		41,850		—		41,850		—
	U.S. government and agency securities		9,865		—		9,865		—
	Total financial assets	$	89,543	$	24,862	$	64,681	$	—
	Liabilities:
	Warrants to purchase common stock	$	-30,359	$	—	$	—	$	-30,359
	Total financial liabilities	$	-30,359	$	—	$	—	$	-30,359
	30-Sep-13
	Assets:
	Money market funds	$	16,426	$	16,426	$	—	$	—
	Available for sale securities:
	Commercial paper	$	4,999	$	—	$	4,999	$	—
	Corporate debt securities		105,955		—		105,955		—
	U.S. government and agency securities		11,247		—		11,247		—
	Municipal securities		1,059		—		1,059		—
	Total financial assets	$	139,686	$	16,426	$	123,260	$	—
	Liabilities:
	Warrants to purchase common stock	$	-51,903	$	—	$	—	$	-51,903
	Total financial liabilities	$	-51,903	$	—	$	—	$	-51,903
Schedule of Fair Value, Assets and Liabilities Measured on Recurring Basis [Table Text Block]	'
	The following table provides a summary of the changes in fair value of the Company’s financial liabilities measured at fair value on a recurring basis using significant unobservable inputs (Level 3) during the nine-month period ended September 30, 2013.
		Warrant
		Liability
		(In thousands)
	Level 3
	Balance at December 31, 2012	$	30,359
	Losses recognized in earnings		30,011
	Exercises		-8,467
	Balance at September 30, 2013	$	51,903
Schedule Of Available For Sale Securities Debt Maturities [Table Text Block]	'
	The estimated fair values of marketable debt securities (commercial paper, corporate debt securities, U.S. government and agency securities, and municipal securities) as of September 30, 2013, by contractual maturity, are as follows:
		Fair Value
		(In thousands)
	Due in one year or less	$	55,923
	Due after one year through two years		67,337
	Total investments in debt securities	$	123,260

2003_Stock_Incentive_Plan_and_1

2003 Stock Incentive Plan and 2012 Stock Plan (Tables)	9 Months Ended
	Sep. 30, 2013
Disclosure Of Compensation Related Costs, Share-Based Payments [Abstract]	'
Schedule of Share-based Compensation, Stock Options, Activity [Table Text Block]	'
	The following table summarizes stock option activity during the nine months ended September 30, 2013:
		Number of		Weighted
		Shares		Average
				Exercise Price
	Outstanding, December 31, 2012		1,526,150	$	10.67
	Granted		522,950		35.14
	Exercised		-505,173		9.78
	Forfeited		-8,589		11.87
	Outstanding, September 30, 2013		1,535,338	$	19.29
	Exercisable, September 30, 2013		798,655	$	9.92
Schedule of Compensation Cost for Share-based Payment Arrangements, Allocation of Share-based Compensation Costs by Plan [Table Text Block]	'
	The following table summarizes stock-based compensation expense for employee, director and consultant stock option grants and restricted stock grants:
		Nine Months Ended
		September 30,
		2012		2013
		(In thousands)
	Stock option expense:
	Employees and directors	$	1,138	$	3,134
	Consultants		113		1,758
			1,251		4,892
	Restricted stock expense:
	Employees and directors		—		1,315
	Consultants		—		271
			—		1,586
	Total	$	1,251	$	6,478
Schedule of Share-based Compensation, Restricted Stock Units Award Activity [Table Text Block]	'
	The following table summarizes the activity related to the Company’s restricted stock units for the nine month period ended September 30, 2013.
		Number of		Weighted
		Shares		Average
				Grant Date
				Fair
				Value
	Outstanding, December 31, 2012		176,188	$	21.23
	Granted		24,690		31.9
	Vested and converted		-66,309		21.23
	Forfeited		—		—
	Outstanding, September 30, 2013		134,569	$	23.19

Net_Loss_Per_Share_Tables

Net Loss Per Share (Tables)	9 Months Ended
	Sep. 30, 2013
Earnings Per Share [Abstract]	'
Schedule of Earnings Per Share, Basic and Diluted [Table Text Block]	'
	The following table presents the historical computation of basic and diluted net loss per share:
		Three Months Ended				Nine Months Ended
		September 30,				September 30,
		2012		2013		2012		2013
		(In thousands, except share and per share amounts)
	Historical net loss per share
	Numerator:
	Net loss attributable to common stockholders	$	-6,187	$	-31,737	$	-15,453	$	-55,424
	Denominator:
	Weighted average number of shares of common stock		3,329,266		19,198,923		3,329,266		17,585,531
	outstanding, basic and diluted:
	Net loss per share, basic and diluted	$	-1.86	$	-1.65	$	-4.64	$	-3.15
Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share [Table Text Block]	'
	The following potentially dilutive securities have been excluded from the computations of diluted weighted average shares outstanding:
		As of September 30,
		2012		2013
		(In thousands)
	Shares issuable upon conversion of preferred stock		7,404		-
	Shares issuable pursuant to accumulated preferred stock dividend		1,037		-
	Options		1,322		1,535
	Warrants to purchase common stock		1,233		878
	Restricted stock units		-		135
	Total		10,996		2,548

Nature_of_Business_and_Basis_o1

Nature of Business and Basis of Presentation (Details Textual)	12 Months Ended
	Dec. 31, 2012
Stockholders' Equity, Reverse Stock Split	'one-for-5.7778

Significant_Agreements_Details

Significant Agreements (Details Textual)

3 Months Ended

9 Months Ended

133 Months Ended

1 Months Ended

3 Months Ended

9 Months Ended

1 Months Ended

3 Months Ended

9 Months Ended

1 Months Ended

3 Months Ended

9 Months Ended

12 Months Ended

9 Months Ended

12 Months Ended

1 Months Ended

3 Months Ended

9 Months Ended

3 Months Ended

9 Months Ended

12 Months Ended

54 Months Ended

1 Months Ended

3 Months Ended

7 Months Ended

9 Months Ended

12 Months Ended

Sep. 30, 2013

Sep. 30, 2012

Sep. 30, 2013

Sep. 30, 2012

Sep. 30, 2013

Dec. 31, 2012

Mar. 31, 2011

Sep. 30, 2013

Sep. 30, 2012

Sep. 30, 2013

Sep. 30, 2012

Mar. 31, 2011

Mar. 31, 2011

Mar. 31, 2011

Mar. 31, 2011

Mar. 31, 2011

Mar. 31, 2011

Mar. 31, 2011

Mar. 31, 2011

Aug. 31, 2011

Aug. 31, 2011

Sep. 30, 2012

Sep. 30, 2012

Sep. 30, 2013

Dec. 31, 2012

Aug. 31, 2011

Aug. 31, 2011

Aug. 31, 2011

Aug. 31, 2011

Aug. 31, 2011

Aug. 31, 2011

Sep. 30, 2013

Sep. 30, 2012

Sep. 30, 2013

Sep. 30, 2012

Dec. 31, 2012

Sep. 30, 2013

Dec. 31, 2012

Nov. 30, 2011

Sep. 30, 2013

Sep. 30, 2012

Sep. 30, 2013

Sep. 30, 2012

Sep. 30, 2013

Sep. 30, 2012

Sep. 30, 2013

Sep. 30, 2012

Dec. 31, 2012

Dec. 31, 2011

Dec. 31, 2010

Aug. 31, 2011

Sep. 30, 2013

Sep. 30, 2012

Jul. 31, 2012

Sep. 30, 2013

Sep. 30, 2012

Dec. 31, 2012

Dec. 31, 2010

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Dainippon Sumitomo Pharma Co Ltd [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

Les Laboratories Servier and Institut De Recherches Servier [Member]

TES Pharma SRL [Member]

TES Pharma SRL [Member]

TES Pharma SRL [Member]

TES Pharma SRL [Member]

TES Pharma SRL [Member]

TES Pharma SRL [Member]

National Institute Of Diabetes and Digestive and Kidney Disease Institute [Member]

WIL Research Laboratories LLC [Member]

WIL Research Laboratories LLC [Member]

WIL Research Laboratories LLC [Member]

WIL Research Laboratories LLC [Member]

WIL Research Laboratories LLC [Member]

University Of Perugia and Professor Roberto Pellicciari [Member]

University Of Perugia and Professor Roberto Pellicciari [Member]

University Of Perugia and Professor Roberto Pellicciari [Member]

University Of Perugia and Professor Roberto Pellicciari [Member]

University Of Perugia and Professor Roberto Pellicciari [Member]

University Of Perugia and Professor Roberto Pellicciari [Member]

University Of Perugia and Professor Roberto Pellicciari [Member]

Professor Roberto Pellicciari [Member]

Professor Roberto Pellicciari [Member]

Professor Roberto Pellicciari [Member]

Professor Roberto Pellicciari [Member]

Professor Roberto Pellicciari [Member]

Professor Roberto Pellicciari [Member]

Professor Roberto Pellicciari [Member]

Professor Roberto Pellicciari [Member]

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

Criteria One [Member]

Criteria Two [Member]

Criteria Three [Member]

Criteria Four [Member]

Criteria Five [Member]

Japan [Member]

China [Member]

United States [Member]

USD ($)

EUR (€)

USD ($)

USD ($)

USD ($)

USD ($)

Criteria One [Member]

Criteria Two [Member]

Criteria Three [Member]

Criteria Four [Member]

Clinical Trial For First Product [Member]

First Product Candidate [Member]

USD ($)

USD ($)

USD ($)

USD ($)

EUR (€)

Subsequent Event [Member]

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

EUR (€)

EUR (€)

EUR (€)

EUR (€)

USD ($)

USD ($)

EUR (€)

USD ($)

USD ($)

EUR (€)

EUR (€)

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

USD ($)

EUR (€)

EUR (€)

EUR (€)

EUR (€)

EUR (€)

EUR (€)

EUR (€)

License Agreement Up Front Payment

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$15,000,000

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License Agreement Maximum Additional Milestone Payment Development Milestones

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30,000,000

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License Agreement Maximum Additional Milestone Payment Regulatory Approval Milestones

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70,000,000

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15,000,000

10,000,000

5,000,000

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License Agreement Maximum Additional Milestone Payment Sales Milestones

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200,000,000

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20,000,000

40,000,000

120,000,000

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License Agreement Sales Milestones Criteria One

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50,000,000

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License Agreement Sales Milestones Criteria Two

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100,000,000

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License Agreement Sales Milestones Criteria Three

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200,000,000

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License Agreement Sales Milestones Criteria Four

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400,000,000

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License Agreement Sales Milestones Criteria Five

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1,200,000,000

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Licensing revenue

405,407

523,191

1,216,219

2,040,700

5,467,454

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405,000

405,000

1,216,000

1,215,000

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118,000

824,000

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Research Collaboration Agreement Up Front Payment

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1,400,000

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Research Collaboration Agreement Milestone Payment Development Milestones

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8,500,000

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4,000,000

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Research Collaboration Agreement Milestone Payment Regulatory Submission and Approval Milestones

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10,000,000

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5,000,000

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Research Collaboration Agreement Milestone Payment Sales Milestones

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90,000,000

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20,000,000

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25,000,000

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10,000,000

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Research Collaboration Agreement Sales Milestones Criteria One

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200,000,000

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Research Collaboration Agreement Sales Milestones Criteria Two

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400,000,000

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Research Collaboration Agreement Sales Milestones Criteria Three

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500,000,000

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Research Collaboration Agreement Sales Milestones Criteria Four

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600,000,000

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Prepaid expenses and other current assets

1,601,213

'

1,601,213

'

1,601,213

1,584,308

'

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'

'

'

'

'

'

'

'

'

'

'

'

'

507,000

496,000

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Sponsored Research Agreement Payment

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

80,000

100,000

100,000

'

'

'

'

'

'

'

'

Research and development

8,392,628

3,318,310

18,358,155

11,395,924

89,792,846

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

227,000

310,000

888,000

982,000

'

'

1,250,000

'

363,000

345,000

1,400,000

1,200,000

26,000

16,000

80,000

77,000

'

'

'

'

68,000

80,000

'

234,000

243,000

'

'

Consulting And Intellectual Property Agreement Fees For Consulting Services

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

150,000

'

'

100,000

'

Consulting And Intellectual Property Agreement Monthly Fees For Consulting Services

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

8,000

Consulting And Intellectual Property Agreement For Consulting Services Maturity Date

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

1-Jan-13

'

'

'

Consulting And Intellectual Property Agreement Lump Sum Payment

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

172,500

Consulting And Intellectual Property Agreement Monthly Payment For Assignment Of Intellectual Property Rights

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

12,000

Consulting and Intellectual Property Agreement Performance Bonus

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

50,000

'

'

'

'

'

'

'

Research and Development Service Agreement Quarterly Payment

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

250,000

250,000

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Milestone Payment

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

1,000,000

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Master Laboratory Service Agreement Aggregate Payment

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

4,000,000

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Master Laboratory Service Agreement Quarterly Installment Payment

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

300,000

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Master Laboratory Service Agreement Milestone Payment

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

$400,000

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Investments_Details

Investments (Details) (USD $)	9 Months Ended	12 Months Ended
In Thousands, unless otherwise specified	Sep. 30, 2013	Dec. 31, 2012
Investment Securities	'	'
Total investments, Amortized Cost	$123,201	$64,698
Total investments, Gross Unrealized Gains	89	21
Total investments, Gross Unrealized Losses	-30	-38
Total investments, Fair Value	123,260	64,681
Total cash and cash equivalents and investments, Amortized Cost	156,693	110,210
Total cash and cash equivalents and investments, Gross Unrealized Gains	89	21
Total cash and cash equivalents and investments, Gross Unrealized Losses	-30	-38
Total cash and cash equivalents and investments, Fair Value	156,752	110,193
Commercial paper [Member]	'	'
Investment Securities	'	'
Total investments, Amortized Cost	4,998	12,971
Total investments, Gross Unrealized Gains	1	10
Total investments, Gross Unrealized Losses	0	-15
Total investments, Fair Value	4,999	12,966
Corporate debt securities [Member]	'	'
Investment Securities	'	'
Total investments, Amortized Cost	105,905	41,866
Total investments, Gross Unrealized Gains	78	7
Total investments, Gross Unrealized Losses	-28	-23
Total investments, Fair Value	105,955	41,850
U.S. government and agency securities [Member]	'	'
Investment Securities	'	'
Total investments, Amortized Cost	11,237	9,861
Total investments, Gross Unrealized Gains	10	4
Total investments, Gross Unrealized Losses	0	0
Total investments, Fair Value	11,247	9,865
Municipal government securities [Member]	'	'
Investment Securities	'	'
Total investments, Amortized Cost	1,061	'
Total investments, Gross Unrealized Gains	0	'
Total investments, Gross Unrealized Losses	-2	'
Total investments, Fair Value	1,059	'
Cash and money market funds [Member]	'	'
Cash and cash equivalents:	'	'
Cash and Cash Equivalents, Amortized Cost	33,492	45,512
Cash and cash equivalents, Gross Unrealized Gains	0	0
Cash and cash equivalents, Gross Unrealized Losses	0	0
Cash and cash equivalents, Fair Value	$33,492	$45,512

Investments_Details_1

Investments (Details 1) (USD $)	9 Months Ended	12 Months Ended
In Thousands, unless otherwise specified	Sep. 30, 2013	Dec. 31, 2012
Available for sale securities, Less than 12 months Fair Value	$32,324	$40,295
Available for sale securities Less than 12 months, Gross Unrealized Holding Losses	-30	-38
Commercial Paper [Member]	'	'
Available for sale securities, Less than 12 months Fair Value	'	10,461
Available for sale securities Less than 12 months, Gross Unrealized Holding Losses	'	-15
Corporate Debt Securities [Member]	'	'
Available for sale securities, Less than 12 months Fair Value	31,265	29,834
Available for sale securities Less than 12 months, Gross Unrealized Holding Losses	-28	-23
Municipal government securities [Member]	'	'
Available for sale securities, Less than 12 months Fair Value	1,059	'
Available for sale securities Less than 12 months, Gross Unrealized Holding Losses	($2)	'

Prepaid_Expenses_and_Other_Cur2

Prepaid Expenses and Other Current Assets (Details) (USD $)	Sep. 30, 2013	Dec. 31, 2012
Prepaid expenses	$383,000	$970,000
Contract receivable	514,000	496,000
Certificate of deposit	0	77,000
Interest receivable	684,000	0
Other receivable	20,000	0
Refundable tax credits	0	42,000
Prepaid expenses and other current assets	$1,601,213	$1,584,308

Fixed_Assets_Net_Details

Fixed Assets, Net (Details) (USD $)	Sep. 30, 2013	Dec. 31, 2012
Fixed assets	$934,000	$813,000
Less: accumulated depreciation and amortization	-743,000	-664,000
Fixed assets, net	190,673	148,838
Office equipment [Member]	'	'
Fixed assets	396,000	357,000
Leasehold improvements [Member]	'	'
Fixed assets	256,000	178,000
Furniture and fixtures under capitalized lease [Member]	'	'
Fixed assets	157,000	157,000
Furniture and fixtures [Member]	'	'
Fixed assets	$125,000	$121,000

Fixed_Assets_Net_Details_Textu

Fixed Assets, Net (Details Textual) (USD $)	3 Months Ended		9 Months Ended
	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013	Sep. 30, 2012
Depreciation and Amortization	$27,000	$24,000	$79,000	$178,000

Accounts_Payable_Accrued_Expen2

Accounts Payable, Accrued Expenses and Other Liabilities (Details) (USD $)	Sep. 30, 2013	Dec. 31, 2012
Accounts payable	$1,384,000	$1,180,000
Accrued employee compensation	959,000	1,335,000
Accrued contracted services and other	2,737,000	1,231,000
Accounts payable, accrued expenses and other liabilities	$5,079,581	$3,745,773

Income_Taxes_Details_Textual

Income Taxes (Details Textual) (USD $)	9 Months Ended
In Millions, unless otherwise specified	Sep. 30, 2013	Dec. 31, 2012
Operating Loss Carryforwards	$97.60	$70.20
Deferred Tax Assets, Other	$52.10	$42.10
Operating Losses Carry forwards Expiration Dates	'between 2024 and 2032	'

Warrants_to_Purchase_Common_St1

Warrants to Purchase Common Stock (Details Textual)	1 Months Ended	9 Months Ended
	Oct. 31, 2012	Sep. 30, 2013
Warrants to purchase common stock	'	878,201
Warrants exercised weighted average exercise price	'	10.34
Stock Issued During Period, Shares, New Issues	5,750,000	1,989,500
October 2013 [Member]	'	'
Warrants to purchase common stock	'	8,494
January 2015 [Member]	'	'
Warrants to purchase common stock	'	4,326
Warrants exercised weighted average exercise price	'	865,381
Stock Issued During Period, Shares, New Issues	'	3,852

Fair_Value_Measurements_Detail

Fair Value Measurements (Details) (USD $)	Sep. 30, 2013	Dec. 31, 2012
In Thousands, unless otherwise specified
Assets:	'	'
Money market funds	$16,426	$24,862
Available for sale securities:	'	'
Commercial paper	4,999	12,966
Corporate debt securities	105,955	41,850
U.S. government and agency securities	11,247	9,865
Municipal securities	1,059	'
Total financial assets	139,686	89,543
Liabilities:	'	'
Warrants to purchase common stock	-51,903	-30,359
Total financial liabilities	-51,903	-30,359
Fair Value, Inputs, Level 1 [Member]	'	'
Assets:	'	'
Money market funds	16,426	24,862
Available for sale securities:	'	'
Commercial paper	0	0
Corporate debt securities	0	0
U.S. government and agency securities	0	0
Municipal securities	0	'
Total financial assets	16,426	24,862
Liabilities:	'	'
Warrants to purchase common stock	0	0
Total financial liabilities	0	0
Fair Value, Inputs, Level 2 [Member]	'	'
Assets:	'	'
Money market funds	0	0
Available for sale securities:	'	'
Commercial paper	4,999	12,966
Corporate debt securities	105,955	41,850
U.S. government and agency securities	11,247	9,865
Municipal securities	1,059	'
Total financial assets	123,260	64,681
Liabilities:	'	'
Warrants to purchase common stock	0	0
Total financial liabilities	0	0
Fair Value, Inputs, Level 3 [Member]	'	'
Assets:	'	'
Money market funds	0	0
Available for sale securities:	'	'
Commercial paper	0	0
Corporate debt securities	0	0
U.S. government and agency securities	0	0
Municipal securities	0	'
Total financial assets	0	0
Liabilities:	'	'
Warrants to purchase common stock	-51,903	-30,359
Total financial liabilities	($51,903)	($30,359)

Fair_Value_Measurements_Detail1

Fair Value Measurements (Details 1) (Fair Value, Inputs, Level 3 [Member], USD $)	9 Months Ended
In Thousands, unless otherwise specified	Sep. 30, 2013
Fair Value, Inputs, Level 3 [Member]	'
Balance at December 31, 2012	$30,359
Losses recognized in earnings	30,011
Exercises	-8,467
Balance at September 30, 2013	$51,903

Fair_Value_Measurements_Detail2

Fair Value Measurements (Details 2) (USD $)	Sep. 30, 2013	Dec. 31, 2012
In Thousands, unless otherwise specified
Due in one year or less	$55,923	'
Due after one year through two years	67,337	'
Total investments in debt securities	$123,260	$64,681

Stockholders_Equity_and_Prefer1

Stockholders' Equity and Preferred Stock (Details Textual) (USD $)

1 Months Ended

9 Months Ended

133 Months Ended

1 Months Ended

1 Months Ended

12 Months Ended

1 Months Ended

8 Months Ended

1 Months Ended

Oct. 31, 2012

Jan. 31, 2010

Nov. 30, 2005

Sep. 30, 2013

Sep. 30, 2012

Sep. 30, 2013

Dec. 31, 2012

Aug. 31, 2012

31-May-08

Jan. 31, 2010

Aug. 31, 2012

Feb. 05, 2005

31-May-06

Feb. 28, 2005

Oct. 31, 2003

Nov. 30, 2002

Sep. 30, 2002

Dec. 31, 2010

Dec. 31, 2008

31-May-04

31-May-06

31-May-04

31-May-04

31-May-04

Nov. 30, 2005

Nov. 30, 2005

Series A Preferred Stock [Member]

Series B Preferred Stock [Member]

Series C Preferred Stock [Member]

Convertible Notes Payable [Member]

Private Placement [Member]

Common Stock [Member]

Common Stock [Member]

Common Stock [Member]

Common Stock [Member]

Common Stock [Member]

Common Stock [Member]

Common Stock [Member]

Common Stock [Member]

Common Stock [Member]

Common Stock [Member]

Common Stock [Member]

Warrant One [Member]

Warrant Two [Member]

Private Placement [Member]

Private Placement [Member]

Private Placement [Member]

Private Placement [Member]

Class A Warrants [Member]

Class B Warrants [Member]

Stock Issued During Period Shares Issued For Services To Founders

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

949,035

'

'

'

'

'

'

'

'

'

Stock Issued During Period Exercise Price Of Shares Issued For Services To Founders

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

$0.03

'

'

'

'

'

'

'

'

'

Stock Issued During Period Shares Issued For Services To Principal Investigators and Researchers

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

60,576

'

'

'

'

'

'

'

'

'

'

Stock Issued During Period Exercise Price Of Shares Issued For Services To Principal Investigators and Researchers

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

$0.03

'

'

'

'

'

'

'

'

'

'

Stock Issued During Period Shares Issued For Services To Two Principal Investigators

'

'

'

'

'

'

'

'

'

'

'

'

'

'

112,498

'

'

'

'

'

'

'

'

'

'

'

Stock Issued During Period Exercise Price Of Shares Issued For Services To Two Principal Investigators

'

'

'

'

'

'

'

'

'

'

'

'

'

'

$0.03

'

'

'

'

'

'

'

'

'

'

'

Stock Repurchased and Retired During Period Shares

'

'

'

'

'

'

'

'

'

'

'

'

'

'

550,960

'

'

'

'

'

'

'

'

'

'

'

Stock Repurchased and Retired During Period Price Per Share

'

'

'

'

'

'

'

'

'

'

'

'

'

'

$0.03

'

'

'

'

'

'

'

'

'

'

'

Stock Issued During Period Shares Issued In Private Placement October Two Thousand and Three

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

392,163

'

'

'

'

Stock Issued During Period Shares Issued In Private Placement In May Two Thousand and Six

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

2,087,091

'

'

'

'

'

Stock Issued During Period Exercise Price New Issues

'

'

'

'

'

'

'

'

'

'

$15,000,000

'

'

'

'

'

'

'

'

'

$9.82

$7.22

'

'

'

'

Costs associated with issuance of stock

'

'

'

'

'

'

'

'

$749,000

$112,000

$300,000

'

'

'

'

'

'

'

'

'

$1,000,000

'

'

'

'

'

Proceeds from issuance of stock offerings, net of issuance costs

78,700,000

'

'

61,169,317

29,713,851

233,646,479

'

'

'

'

'

'

19,500,000

'

'

'

'

'

'

'

'

2,400,000

'

'

'

'

Stock Issued During Period, Shares, New Issues

5,750,000

'

'

1,989,500

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Warrants Issued During Period Number Of Warrants

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

865,381

108,169

'

'

'

137,251

117,640

51,922

86,538

Warrant Expiration Period

'

'

'5 years

'

'

'

'

'

'

'5 years

'

'

'

'

'

'

'

'

'5 years

'

'

'

'

'

'2 years

'5 years

Warrant Exercise Price

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

$10.40

$10.40

'

'

'

'

'

$7.22

$7.22

Debt Instrument, Interest Rate, Stated Percentage

'

'

'

'

'

'

'

'

'

'

'

6.00%

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Stock Issued During Period, Shares, Conversion of Convertible Securities

'

'

'

'

'

'

'

'

'

'

'

'

'

160,637

'

'

'

'

'

'

'

'

'

'

'

'

Debt Instrument, Convertible, Conversion Price

'

'

'

'

'

'

'

'

'

'

'

'

'

$8.35

'

'

'

'

'

'

'

'

'

'

'

'

Preferred stock, authorized

5,000,000

27,777,778

'

'

'

'

'

52,777,778

13,888,889

13,888,889

25,000,000

1,300,000

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Preferred stock, issued

'

'

'

'

'

'

'

'

'

13,888,889

'

91,000

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Net Proceeds from Issuance of Preferred Stock, Preference Stock, and Warrants

'

24,900,000

'

'

'

'

'

'

24,300,000

'

29,700,000

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Number Of Common Shares Attributable To Conversion Of Preferred Stock

7,403,817

'

'

'

'

'

'

7,403,817

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Stock Issued During Period Value Issued In Private Placement In November Two Thousand And Five

'

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'

'

'

'

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375,000

'

Equity Issuance, Per Share Amount (in dollars per share)

$15

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'

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'

'

'

'

Common stock, authorized

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'

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25,000,000

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25,000,000

25,000,000

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'

'

'

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'

'

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'

'

'

'

'

'

'

'

Common stock, par value (in dollars per share)

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$0.00

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$0.00

$0.00

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'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

'

Preferred stock, par value (in dollars per share)

$0.00

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'

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Preferred Units, Offering Costs

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$474,000

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Shares Issued, Price Per Share

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$33.01

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$33.01

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'

2003_Stock_Incentive_Plan_and_2

2003 Stock Incentive Plan and 2012 Stock Plan (Details) (USD $)	9 Months Ended
	Sep. 30, 2013	Sep. 30, 2012
Outstanding, Number of Shares, Beginning Balance	1,526,150	1,322,000
Granted - Number of Shares	522,950	'
Exercised - Number of Shares	-505,173	'
Forfeited - Number of Shares	-8,589	'
Outstanding, Number of Shares, Ending Balance	1,535,338	1,322,000
Exercisable - Number of Shares	798,655	'
Outstanding - Weighted Average Exercise Price - Beginning Balance	$10.67	'
Granted - Weighted Average Exercise Price	$35.14	'
Exercised - Weighted Average Exercise Price	$9.78	'
Forfeited - Weighted Average Exercise Price	$11.87	'
Outstanding - Weighted Average Exercise Price - Ending Balance	$19.29	'
Exercisable - Weighted Average Exercise Price	$9.92	'

2003_Stock_Incentive_Plan_and_3

2003 Stock Incentive Plan and 2012 Stock Plan (Details 1) (USD $)	9 Months Ended		133 Months Ended
	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013
Stock options expense	$4,892,000	$1,251,000	'
Restricted stock expense	1,586,000	0	'
Total	6,478,346	1,251,579	16,256,216
Employees and directors [Member]	'	'	'
Stock options expense	3,134,000	1,138,000	'
Restricted stock expense	1,315,000	0	'
Consultants [Member]	'	'	'
Stock options expense	1,758,000	113,000	'
Restricted stock expense	$271,000	$0	'

2003_Stock_Incentive_Plan_and_4

2003 Stock Incentive Plan and 2012 Stock Plan (Details 2) (USD $)	9 Months Ended
	Sep. 30, 2013
Outstanding, Number of Shares, Beginning Balance	176,188
Granted - Number of Shares	24,690
Vested and converted - Number of Shares	-66,309
Forfeited - Number of Shares	0
Outstanding, Number of Shares, Ending Balance	134,569
Outstanding - Weighted Average Exercise Price - Beginning Balance	$21.23
Granted - Weighted Average Exercise Price	$31.90
Vested and converted - Weighted Average Exercise Price	$21.23
Forfeited - Weighted Average Exercise Price	$0
Outstanding - Weighted Average Exercise Price - Ending Balance	$23.19

2003_Stock_Incentive_Plan_and_5

2003 Stock Incentive Plan and 2012 Stock Plan (Details Textual) (USD $)	1 Months Ended	9 Months Ended							3 Months Ended	12 Months Ended
	Oct. 31, 2012	Sep. 30, 2013	Sep. 30, 2013	Mar. 31, 2013	Oct. 31, 2012	Jan. 31, 2010	Jun. 30, 2008	31-May-06	Jun. 30, 2013	Dec. 31, 2012
			Plan 2012 [Member]	Plan 2012 [Member]	Plan 2012 [Member]	Plan 2003 [Member]	Plan 2003 [Member]	Plan 2003 [Member]	Employee and Director [Member]	Employee and Director [Member]
Share-based Compensation Arrangement by Share-based Payment Award, Number of Shares Available for Grant (in shares)	'	'	'	'	555,843	1,384,610	865,381	519,228	'	'
Share-based Compensation Arrangement by Share-based Payment Award, Number of Shares Authorized	'	'	470,714	661,075	'	'	'	'	'	'
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Grants in Period	'	24,690	'	'	'	'	'	'	24,690	173,592
Stock Issued During Period, Shares, New Issues	5,750,000	1,989,500	'	'	'	'	'	'	36,168	'
Share Based Compensation Arrangement By Share Based Payment Award Equity Instruments Other Than Options Settled Net	'	'	'	'	'	'	'	'	44,296	'
Share Based Compensation Arrangement By Share Based Payment Award Equity Instruments Other Than Options Withheld	'	'	'	'	'	'	'	'	18,478	'
Employees Minimum Statutory Obligation	'	'	'	'	'	'	'	'	$639,000	'
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Vested in Period	'	66,309	'	'	'	'	'	'	54,646	'

Net_Loss_Per_Share_Details

Net Loss Per Share (Details) (USD $)	3 Months Ended		9 Months Ended		133 Months Ended
	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013
Numerator:	'	'	'	'	'
Net loss attributable to common stockholders	($31,737,073)	($6,186,988)	($55,424,138)	($15,452,668)	($184,551,622)
Denominator:	'	'	'	'	'
Weighted average number of shares of common stock outstanding, basic and diluted (in shares)	19,198,923	3,329,266	17,585,531	3,329,266	'
Net loss per share, basic and diluted (in dollars per share)	($1.65)	($1.86)	($3.15)	($4.64)	'

Net_Loss_Per_Share_Details_1

Net Loss Per Share (Details 1)	Sep. 30, 2013	Dec. 31, 2012	Sep. 30, 2012
Shares issuable upon conversion of preferred stock	0	'	7,404,000
Shares issuable pursuant to accumulated preferred stock dividend	0	'	1,037,000
Options	1,535,338	1,526,150	1,322,000
Warrants to purchase common stock	878,000	'	1,233,000
Restricted stock units	135,000	'	0
Total	2,548,000	'	10,996,000

Subsequent_Events_Details_Text

Subsequent Events (Details Textual) (USD $)	1 Months Ended	9 Months Ended
	Oct. 31, 2012	Sep. 30, 2013
Stock Issued During Period, Shares, New Issues	5,750,000	1,989,500
Subsequent Event [Member] | New York City Primary Space [Member]	'	'
Capital Leases Future Minimum Payments Due Current	'	801,000
Capital Leases, Future Minimum Payments Due, Total	'	940,000
Net Rentable Area	'	11,124
Lease Expiration Date	'	31-Jul-24
Capital Leases of Lessee, Contingent Rentals, Description of Variable Rate Basis	'	'The rent for the Primary Space (without giving effect to any rent abatements) for the first year will be approximately $801,000 and will increase by two percent annually until the end of the fifth lease year, at which point the rent will increase to approximately $940,000 and subsequently increase by two percent annually until July 2024,
Subsequent Event [Member] | Additional Space [Member]	'	'
Gain (Loss) on Contract Termination	'	171,000
Subsequent Event [Member] | New York City Additional Space [Member]	'	'
Net Rentable Area	'	9,502
Capital Leases of Lessee, Contingent Rentals, Description of Variable Rate Basis	'	'The rent for the Additional Space (without giving effect to any rent abatements) for the first year will be approximately $684,000 and will increase by two percent annually until the end of the fifth lease year, at which point the rent will increase to approximately $803,000 and subsequently increase by two percent annually until the termination date.
Subsequent Event [Member] | Primary Space [Member]	'	'
Capital Leases Future Minimum Payments Due Current	'	684,000
Subsequent Event [Member] | Primary Space [Member] | Fifth Year [Member]	'	'
Capital Leases, Future Minimum Payments Due, Total	'	803,000
Subsequent Event [Member] | Two Largest Stock Holders [Member]	'	'
Stock Issued During Period, Shares, New Issues	'	1,665,000
Operating Costs and Expenses, Total	'	275,000
Subsequent Event [Member] | Two Largest Stock Holders [Member] | Common Stock [Member]	'	'
Stock Issued During Period, Shares, New Issues	'	165,000