Travere Therapeutics (TVTX) 10-Q/A 9 Jul 15 2013 Q3 Quarterly report (amended) Financial data

Document and Entity Information

Document and Entity Information - shares	9 Months Ended
	Sep. 30, 2013	Jun. 30, 2015
Document And Entity Information
Entity Registrant Name	Retrophin, Inc.
Entity Central Index Key	1,438,533
Trading Symbol	rtrx
Current Fiscal Year End Date	--12-31
Entity Filer Category	Accelerated Filer
Entity Common Stock, Shares Outstanding		35,072,757
Document Type	10-Q/A
Document Period End Date	Sep. 30, 2013
Amendment Flag	false
Document Fiscal Period Focus	Q3
Document Fiscal Year Focus	2,013

CONDENSED CONSOLIDATED BALANCE

CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($)		Sep. 30, 2013		Dec. 31, 2012
Current assets:
Cash		$ 13,409,825	[1]	$ 11,388
Marketable securities, available-for-sale	[1]	2,957,376
Prepaid expenses and other current assets		480,647	[1]	21,830
Total current assets		16,847,848	[1]	33,218
Property and equipment, net		38,437	[1]	23,790
Patents pending		$ 23,793	[1]	18,093
Due from affiliate				$ 137,547
Security deposits		$ 177,547	[1]
Deposits on license agreements		2,250,000	[1]
Technology license, net		2,027,085	[1]	$ 2,178,617
Total assets		$ 21,364,710	[1]	2,391,265
Current liabilities:
Technology license liability				1,300,000
Accounts payable		$ 1,721,511	[1]	1,023,320
Accrued expenses		3,666,598	[1]	$ 2,467,796
Settlements payable		$ 1,691,400	[1]
Note payable - related party				$ 884,764
Investors' deposits				100,000
Due to related parties		$ 10,000	[1]	$ 23,200
Derivative financial instruments, at estimated fair value - warrants		23,853,325	[1]
Total current liabilities		$ 30,942,834	[1]	$ 5,799,080
Stockholders' Deficit:
Preferred stock Series A $0.001 par value; 20,000,000 shares authorized; 0 issued and outstanding
Common stock $0.0001 par value; 100,000,000 shares authorized; 18,376,363 and 8,952,905 issued and outstanding, respectively		$ 1,838	[1]	$ 895
Additional paid-in capital		46,219,593	[1]	30,203,402
Deficit accumulated during the development stage		(55,644,721)	[1]	$ (33,612,112)
Accumulated other comprehensive income		(154,834)	[1]
Total stockholders' deficit		(9,578,124)	[1]	$ (3,407,815)
Total liabilities and stockholders' deficit		$ 21,364,710	[1]	$ 2,391,265
[1]	(As Restated)

CONDENSED CONSOLIDATED BALANCE3

CONDENSED CONSOLIDATED BALANCE SHEETS (Parentheticals) - $ / shares	Sep. 30, 2013	[1]	Dec. 31, 2012
Statement of Financial Position [Abstract]
Preferred stock Series A, par value (in dollars per share)	$ 0.001		$ 0.001
Preferred stock Series A, shares authorized	20,000,000		20,000,000
Preferred stock Series A, shares issued	0		0
Preferred stock Series A, shares outstanding	0		0
Common stock, par value (in dollars per share)	$ 0.0001		$ 0.0001
Common stock, shares authorized	100,000,000		100,000,000
Common stock, shares issued	18,376,363		8,952,905
Common stock, shares outstanding	18,376,363		8,952,905
[1]	(As Restated)

UNAUDITED CONDENSED CONSOLIDATE

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($)		3 Months Ended			9 Months Ended			31 Months Ended
		Sep. 30, 2013		Sep. 30, 2012	Sep. 30, 2013		Sep. 30, 2012	Sep. 30, 2013
Operating expenses:
Compensation and related costs - inclusive of share base compensation $28,519, $4,780,383, $70,189, $7,724,150 and $17,808,002		$ 478,741	[1]	$ 5,068,707	$ 1,767,195	[1]	$ 8,371,481	$ 22,127,948	[1]
Professional fees - inclusive of share based compensation $362,126, $2,247,292, $571,370, $6,290,252 and $8,501,449		1,185,429	[1]	3,167,242	3,114,298	[1]	7,761,899	12,323,637	[1]
Research and development - inclusive of share based compensation $72,888, $0, $109,571, $0 and $109,571		1,399,875	[1]	110,656	2,113,813	[1]	286,889	3,008,326	[1]
Selling, general and administrative		$ 2,966,816	[1]	$ 113,140	6,035,941	[1]	$ 337,622	7,392,049	[1]
Technology license fee					100,000	[1]		1,800,000	[1]
Total operating expenses		$ 6,030,861	[1]	$ 8,459,745	13,131,247	[1]	$ 16,757,891	46,651,960	[1]
Operating loss		(6,030,861)	[1]	(8,459,745)	(13,131,247)	[1]	(16,757,891)	(46,651,960)	[1]
Other income (expense):
Interest income		$ 4	[1]	6,049	9	[1]	15,781	21,914	[1]
Interest expense				$ (26,761)	(41,563)	[1]	$ (70,559)	(147,480)	[1]
Registration payment obligation income		$ 360,000	[1]		360,000	[1]		360,000	[1]
Registration payment obligation expense	[1]	(360,000)			(360,000)			(360,000)
Realized gain on sale of marketable securities		59,737	[1]		59,737	[1]		59,737	[1]
Change in fair value of derivative instruments - warrants	[1]	$ (6,040,054)			(8,915,672)			(8,915,672)
Loss on transactions denominated in foreign currencies					(3,873)	[1]		(11,260)	[1]
Total other expense, net		$ (5,980,313)	[1]	$ (20,712)	(8,901,362)	[1]	$ (54,778)	(8,992,761)	[1]
Net loss		$ (12,011,174)	[1]	$ (8,480,457)	$ (22,032,609)	[1]	$ (16,812,669)	(55,644,721)	[1]
Net loss per common share, basic and diluted (in dollars per share)		$ (0.78)	[1]	$ (2.42)	$ (1.72)	[1]	$ (5.55)
Weighted average common shares outstanding, basic and diluted (in shares)		15,365,631	[1]	3,510,415	12,797,714	[1]	3,027,468
Net loss		$ (12,011,174)	[1]	$ (8,480,457)	$ (22,032,609)	[1]	$ (16,812,669)	(55,644,721)	[1]
Unrealized loss on marketable securities		(154,834)	[1]		(154,834)	[1]		(154,834)	[1]
Comprehensive Loss		$ (12,166,008)	[1]	$ (8,480,457)	$ (22,187,443)	[1]	$ (16,812,669)	$ (55,799,555)	[1]
[1]	(As Restated)

UNAUDITED CONDENSED CONSOLIDAT5

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (Parentheticals) - USD ($)	3 Months Ended			9 Months Ended			31 Months Ended
	Sep. 30, 2013	[1]	Sep. 30, 2012	Sep. 30, 2013	[1]	Sep. 30, 2012	Sep. 30, 2013	[1]
Income Statement [Abstract]
Compensation and related costs - Share based compensation	$ 28,519		$ 4,780,383	$ 70,189		$ 7,724,150	$ 17,808,002
Professional fees - Share based compensation	362,126		2,247,292	571,370		6,290,252	8,501,449
Research and development - share based compensation	$ 72,888		$ 0	$ 109,571		$ 0	$ 109,571
[1]	(As Restated)

CONDENSED CONSOLIDATED STATEMEN

CONDENSED CONSOLIDATED STATEMENT OF CHANGES IN STOCKHOLDERS' EQUITY (DEFICIT) - USD ($)		Common Stock	Additional Paid-In Capital	Receivables due from Stockholder	Accumulated other comprehensive loss	Accumulated Deficit	Total
Balance at Mar. 11, 2011
Balance (in shares) at Mar. 11, 2011
Issuance of common shares		$ 161	$ 24,839	$ (25,000)
Issuance of common shares (in shares)		1,608,300
Issuance of common shares to founders in connection with the initial capital contribution		$ 5	95				$ 100
Issuance of common shares to founders in connection with the initial capital contribution (in shares)		50,000
Incentive shares granted - employees		$ 176	(176)
Incentive shares granted - employees (in shares)		1,758,300
Incentive shares granted- non employees		$ 38	(38)
Incentive shares granted- non employees (in shares)		381,000
Share based compensation - employees			1,724,967				1,724,967
Share based compensation - non employees			254,332				254,332
Incentive shares forfeited - employees		$ (5)	5
Incentive shares forfeited - employees (in shares)		(45,835)
Issuance of shares in connection with March 2011 private placement, net of fees of $66,061		$ 25	658,914				658,939
Issuance of shares in connection with March 2011 private placement, net of fees of $66,061 (in shares)		253,750
Issuance of Series A preferred in connection with March 2011 private placement, net of fees of $1,367, recapitalization to common stock		$ 4	103,629				103,633
Issuance of Series A preferred in connection with March 2011 private placement, net of fees of $1,367, recapitalization to common stock (in shares)		36,750
Loan made to stockholder				(10,000)			(10,000)
Net loss						$ (3,268,256)	(3,268,256)
Ending Balance at Dec. 31, 2011		$ 404	2,766,567	(35,000)		(3,268,256)	(536,285)
Ending Balance (in shares) at Dec. 31, 2011		4,042,265
Prior Issuance of Series A preferred in connection with January 2012 private place, net of fees of $61,677, exchanged to common stock		$ 33	1,806,644				1,806,677
Prior Issuance of Series A preferred in connection with January 2012 private place, net of fees of $61,677, exchanged to common stock (in shares)		326,963
Prior Issuance of Series A preferred in connection with May 2012 private place, net of fees of $12,275, exchanged to common stock		$ 47	1,668,979				1,669,026
Prior Issuance of Series A preferred in connection with May 2012 private place, net of fees of $12,275, exchanged to common stock (in shares)		470,764
Shares transferred to consultants by founder for services		$ 0	4,400,000				4,400,000
Shares transferred to consultants by founder for services (in shares)		0
Shares transferred to employees by founder for services		$ 0	1,375,000				1,375,000
Shares transferred to employees by founder for services (in shares)		0
Shares issued in accordance with license agreement		$ 62	1,549,938				1,550,000
Shares issued in accordance with license agreement (in shares)		620,000
Shares outstanding at time of reverse merger date December 12, 2012		$ 259	1,142				1,401
Shares outstanding at time of reverse merger date December 12, 2012 (in shares)		2,585,583
Incentive shares granted - employees		$ 86	(86)
Incentive shares granted - employees (in shares)		866,180
Incentive shares granted- non employees		$ 9	(9)
Incentive shares granted- non employees (in shares)		87,503
Share based compensation - employees			14,637,850				14,637,850
Share based compensation - non employees			1,997,372				1,997,372
Incentive shares forfeited - employees		$ (5)	5
Incentive shares forfeited - employees (in shares)		(46,353)
Loan made to stockholder				(372,900)			(372,900)
Receivable due from stockholder charged to compensation				$ 407,900			407,900
Net loss						(30,343,856)	(30,343,856)
Ending Balance at Dec. 31, 2012		$ 895	30,203,402			$ (33,612,112)	$ (3,407,815)
Ending Balance (in shares) at Dec. 31, 2012		8,952,905
Incentive shares granted - employees		$ 14	(14)
Incentive shares granted - employees (in shares)		135,000
Share based compensation - consultants		$ 19	1,282,201				$ 1,282,220
Share based compensation - consultants (in shares)		194,000
Share based compensation - employees			179,760				179,760
Share based compensation - non employees			(710,850)				$ (710,850)
Incentive shares forfeited - employees		$ (2)	2
Incentive shares forfeited - employees (in shares)		(20,833)
Incentive shares forfeited - non employees		$ (4)	4
Incentive shares forfeited - non employees (in shares)		(37,500)
Issuance of common stock in connection with January 2013 private placement at $3.00 per share, net of fees of $0		$ 27	816,637				$ 816,664
Issuance of common stock in connection with January 2013 private placement at $3.00 per share, net of fees of $0 (in shares)		272,221
Issuance of common stock in connection with February 2013 private placement at $3.00 per share, net of fees of $928,986 and registration payment obligation of $360,000		$ 305	2,440,889				2,441,194
Issuance of common stock in connection with February 2013 private placement at $3.00 per share, net of fees of $928,986 and registration payment obligation of $360,000 (in shares)		3,045,929
Issuance of common stock in connection with August 2013 private placement at $4.50 per share, net of fees of $2,811,313 and payment made to February investors for inducement to participate in August financing of $2,238,681		$ 553	10,639,270				10,639,823
Issuance of common stock in connection with August 2013 private placement at $4.50 per share, net of fees of $2,811,313 and payment made to February investors for inducement to participate in August financing of $2,238,681 (in shares)		5,531,401
Issuance of common stock in connection with payment made to February investors for inducement to participate in August financing, 271,222 shares at $4.50 per share and 20,685 shares at $5.00 per share		$ 29	1,323,894				1,323,923
Issuance of common stock in connection with payment made to February investors for inducement to participate in August financing, 271,222 shares at $4.50 per share and 20,685 shares at $5.00 per share (in shares)		291,907
Shares issued on behalf of related party		$ 1	44,399				$ 44,400
Shares issued on behalf of related party (in shares)		6,000
Adjustment to existing shareholder		$ 1	$ (1)
Adjustment to existing shareholder (in shares)		5,333
Unrealized loss on marketable securities					$ (154,834)		$ (154,834)	[1]
Net loss						$ (22,032,609)	(22,032,609)	[1]
Ending Balance at Sep. 30, 2013	[1]	$ 1,838	$ 46,219,593		$ (154,834)	$ (55,644,721)	$ (9,578,124)
Ending Balance (in shares) at Sep. 30, 2013	[1]	18,376,363
[1]	(As Restated)

CONDENSED CONSOLIDATED STATEME7

CONDENSED CONSOLIDATED STATEMENT OF CHANGES IN STOCKHOLDERS' EQUITY (DEFICIT) (Parentheticals) - USD ($)	1 Months Ended
	Aug. 31, 2013	Feb. 28, 2013	Jan. 31, 2013	May. 30, 2012	Jan. 31, 2012	Mar. 31, 2011
Statement of Stockholders' Equity [Abstract]
Common shares issuance fees	$ 2,811,313	$ 928,986	$ 0			$ 66,061
Preferred shares issuance fees				$ 12,275	$ 61,677	$ 1,367
Registration payment obligation expense		$ 360,000
Issuance of common stock, per share amount (in dollars per share)	$ 4.50	$ 3	$ 3
Payment made to investors for inducement to participate in financing	$ 2,238,681
Shares issued to investors for inducement to participate in financing (in shares)	271,222
Issuance of common stock to investors, per share amount (in dollars per share)	$ 4.50
Shares issued to investors for inducement to participate in financing	20,685
Issuance of common stock to investors, per share amount (in dollars per share)	$ 5

UNAUDITED CONDENSED CONSOLIDAT8

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($)		9 Months Ended			31 Months Ended
		Sep. 30, 2013		Sep. 30, 2012	Sep. 30, 2013
Cash Flows From Operating Activities:
Net loss		$ (22,032,609)	[1]	$ (16,812,669)	$ (55,644,721)	[1]
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization		159,128	[1]	$ 73,417	284,368	[1]
Gain on securities available for sale		$ (59,737)	[1]		(59,737)	[1]
Compensation in lieu of receivable					407,900	[1]
Share based compensation - consultants		$ 1,282,220	[1]		1,282,220	[1]
Share based compensation - employees		179,760	[1]	$ 7,724,150	17,917,577	[1]
Share based compensation - non-employees		(710,850)	[1]	$ 6,290,252	5,940,854	[1]
Registration payment obligation expense	[1]	360,000			360,000
Reversal of registration payment obligation liability	[1]	$ (360,000)			(360,000)
Share based payment - Technology license contingent fee					1,550,000	[1]
Change in estimated fair value of derivative financial instruments - warrants	[1]	$ 8,915,672			8,915,672
Changes in operating assets and liabilities
Prepaid expenses		$ (458,817)	[1]	$ (30,431)	(480,647)	[1]
Other assets				$ (8,781)
Technology license fees					150,000	[1]
Settlement payable		$ 1,691,400	[1]		1,691,400	[1]
Accounts payable and accrued expenses		1,841,391	[1]	$ 675,251	5,330,186	[1]
Net cash used in operating activities		(9,192,442)	[1]	(2,088,811)	(12,714,928)	[1]
Cash Flows From Investing Activities:
Purchase of fixed assets		(22,243)	[1]	(8,471)	(49,889)	[1]
Purchase of intangible assets		(5,700)	[1]	$ (1,158,418)	(1,173,793)	[1]
Payments for security deposits for exclusivity of certain licenses		(2,250,000)	[1]		(2,250,000)	[1]
Increase in security deposit		(40,000)	[1]		(40,000)	[1]
Repayment of technology license liability		(1,300,000)	[1]		(1,300,000)	[1]
Purchase of marketable securities, available-for-sale		(3,430,418)	[1]		(3,430,418)	[1]
Proceeds from the sale of marketable securities, available-for-sale		$ 377,945	[1]		377,945	[1]
Cash received in merger transaction					$ 3,721	[1]
Due from related parties				$ (2,800)
Payments made on behalf of affiliate					$ (137,547)	[1]
Loans made to stockholder				$ (399,329)	(382,900)	[1]
Net cash used in investing activities		$ (6,670,416)	[1]	$ (1,569,018)	(8,382,881)	[1]
Cash Flows From Financing Activities:
Proceeds from related parties					56,500	[1]
Repayment of net amounts due to related parties		$ (13,200)	[1]	$ (30,000)	(46,500)	[1]
Proceeds from note payable - related party				930,000	930,000	[1]
Repayment of note payable - related party		$ (884,764)	[1]	$ (15,236)	(930,000)	[1]
Investors' deposits					100,000	[1]
Proceeds received from issuance of common stock, net		$ 31,105,455	[1]	$ 2,765,201	35,343,830	[1]
Payment to investors participated in August 2013 financing		(946,196)	[1]		(946,196)	[1]
Net cash provided by financing activities		29,261,295	[1]	$ 3,649,965	34,507,634	[1]
Net increase in cash		13,398,437	[1]	(7,864)	$ 13,409,825	[1]
Cash, beginning of period		11,388	[1]	10,053
Cash, end of period		13,409,825	[1]	2,189	$ 13,409,825	[1]
Supplemental Disclosure of Cash Flow Information
Cash paid for interest		28,263	[1]	$ 9,764	43,027	[1]
Non-cash investing and financing activities:
Unrealized loss on marketable securities		$ (154,834)	[1]		(154,834)	[1]
Forfeiture of subscription receivable					$ 25,000	[1]
Reclassification of due from related parties				$ 500
Technology license liability				$ 1,300,000
Shares issued on behalf of related party		$ 44,400	[1]		$ 44,400	[1]
Affiliate receivable applied to security deposit		$ 137,547	[1]		$ 137,547	[1]
[1]	(As Restated)

DESCRIPTION OF BUSINESS

DESCRIPTION OF BUSINESS	9 Months Ended
	Sep. 30, 2013
Notes to Financial Statements
DESCRIPTION OF BUSINESS	NOTE 1. DESCRIPTION OF BUSINESS Organization and Description of Business Retrophin, Inc. (the “Company”) is an emerging biotechnology company dedicated to developing drugs for rare and life-threatening diseases. The Company’s primary business objective is to develop and commercialize therapies for orphan diseases, such as Duchenne muscular dystrophy, or DMD, focal segmental glomerulosclerosis, and pantothenate kinase-associated neurodegeneration. The Company is considered to be a development stage company and, as such, the Company’s financial statements are prepared in accordance with the Accounting Standards Codification (“ASC”) 915 “Development Stage Entities.” The Company is subject to all of the risks and uncertainties associated with development stage companies.

BASIS OF PRESENTATION

BASIS OF PRESENTATION	9 Months Ended
	Sep. 30, 2013
Organization, Consolidation and Presentation of Financial Statements [Abstract]
BASIS OF PRESENTATION	NOTE 2. BASIS OF PRESENTATION The accompanying unaudited condensed consolidated financial statements of the Company should be read in conjunction with the audited consolidated financial statements and notes thereto included in the Company’s Annual Report on Form 10-K/A for the year ended December 31, 2012 (the “2012 10-K/A”) filed with the SEC on September 13, 2013. The accompanying condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”) for interim financial information, the instructions to Form 10-Q and the rules and regulations of the SEC. Accordingly, since they are interim statements, the accompanying condensed consolidated financial statements do not include all of the information and notes required by U.S. GAAP for annual financial statements, but reflect all adjustments consisting of normal, recurring adjustments, that are necessary for a fair presentation of the financial position, results of operations and cash flows for the interim periods presented. Interim results are not necessarily indicative of results for a full year. The December 31, 2012 balance sheet information was derived from the audited financial statements as of that date.

LIQUIDITY, FINANCIAL CONDITION

LIQUIDITY, FINANCIAL CONDITION AND MANAGEMENT PLANS	9 Months Ended
	Sep. 30, 2013
Organization, Consolidation and Presentation of Financial Statements [Abstract]
LIQUIDITY, FINANCIAL CONDITION AND MANAGEMENT PLANS	NOTE 3. LIQUIDITY, FINANCIAL CONDITION AND MANAGEMENT PLANS The Company incurred a net loss of approximately $55.6 million, including stock-based compensation charge of approximately $26 million for the period from March 11, 2011 (inception) to September 30, 2013. At September 30, 2013, the Company had a cash balance of approximately $13.4 million and a working capital deficit of approximately $14.1 million; however, the working capital deficit includes a derivative liability of approximately $23.9 million for warrants issued in financing transactions. The Company’s accumulated deficit amounted to approximately $55.6 million at September 30, 2013. The Company has principally financed its operations from inception using proceeds from sales of its equity securities in a series of private placement transactions (see Note 12). The Company to date has no revenues, significantly limited capital resources and is subject to all of the risks and uncertainties that are typical of a development stage enterprise. Significant uncertainties include, among others, whether it will be able to raise the additional capital it needs to finance the start of its planned operations and whether such operations, if launched, will enable the Company to become a profitable enterprise. On August 14, 2013, the Company and the investors who participated in the private placement transaction that the Company completed on February 14, 2013, entered into the first amendment to the registration rights agreement (the “Amended Registration Rights Agreement”) associated with that transaction. The Amended Registration Rights Agreement provides, among other things, for (i) a waiver of any and all liquidated damages that the Company incurred for its inability to cause the a registration statement to be declared effective within certain contractually defined time-frames stipulated in the original agreement; (ii) a commitment on the part of the investors in the February private placement to participate in a private placement transaction that the Company completed on August 15, 2013, and (iii) a covenant on the part of the Company to proceed with the sale of shares that were issued under the August 15, 2013 private placement transaction. In exchange, the Company paid an aggregate fee to these investors of $2,495,256 consisting of (i) 73,710 shares of the Company’s common stock with an aggregate fair value of $331,695 (based on the selling price of $4.50 per share in the August financing transaction); (ii) cash in the amount of $1,835,000; and (iii) warrants to purchase 98,756 shares of common stock with a fair value of $328,561. The investors were also given the option to purchase shares of the Company’s common stock at $4.50 as a use of the cash portion of the payment arrangement. Accordingly, $946,196 of the cash portion of the fee was settled in cash and the remainder was settled by the issuance of 197,512, shares. Additionally, the Company paid $103,425 to an investor to whom the Company sold shares in a private placement transaction in January 2013 and who participated in the August 2013 private placement transaction. This payment was settled entirely by the issuance of 20,685 shares of the Company’s common stock at a value of $5.00 per share (see Note 12) On August 16, 2013, the Company announced that it had signed an agreement with a major pharmaceutical company for the exclusive right to negotiate a royalty-bearing U.S. license for a product to be developed for the treatment of Autism and Schizophrenia. Pursuant to the exclusivity agreement, the Company paid the major pharmaceutical company a non-refundable upfront fee of $2 million and will have an exclusive period of 120 days to negotiate a license agreement (see Note 14). The Company is in active negotiations to consummate a license agreement. If a definitive license agreement is consummated, the Company will apply the upfront fee to the license and will make a determination as to whether it should be treated as an asset or research and development expense. If no definitive agreement is consummated, the upfront fee will be expensed. On September 20, 2013, the Company signed an agreement with an individual for the exclusive right to negotiate a royalty-bearing U.S. license for a product to be developed for the treatment of central nervous system disorders. Pursuant to the exclusivity agreement, the Company paid the individual a non-refundable upfront fee of $250,000 and will have an exclusive period ending on December 31, 2013 (see Note 14). The Company is in active negotiations to consummate a license agreement. If a definitive license agreement is consummated, the Company will apply the upfront fee to the license and will make a determination as to whether it should be treated as an asset or research and development expense. If no definitive agreement is consummated, the upfront fee will be expensed. Effective October 1, 2013, the Company signed a Sponsored Research Agreement (“SRA”) with St. Jude Children’s Research Hospital (“St. Jude”). Unless otherwise terminated by operation of law or by acts of the parties in accordance with the terms of the agreement, the SRA shall be in full force and effect for a period of two (2) years and shall expire on October 1, 2015. The term may be extended by written agreement between the parties. The Company and St. Jude will collaborate on research focused on the study of PKAN disease and other infectious diseases (see Note 14 and Note 15). In the second quarter of 2013, the Company, its Chief Executive Officer and a related party became parties to a series of agreements to settle up to $2,284,511 of liabilities, which Company management believes are the primary obligation of the related party. The Company paid $593,111 of these settlements in the second quarter on behalf of the related party and had outstanding liabilities of $1,691,400 as of September 30, 2013, which the Company paid as of the date of this filing. Concurrent with the execution and payment of such settlement agreements, the Company entered into indemnification agreements and received promissory notes from the related party whereby the related party agreed to pay the Company the principal amount of $2,284,511 plus interest at an annualized rate of 5% as reimbursement of payments that the Company made to settle a portion of the agreements. The Chief Executive Officer also agreed to deliver or cause to be delivered 47,128 shares of common stock to one of the counter parties as a separate component of one of these agreements. Accordingly, the Company does not believe it is required to record a liability for the shared-based component of this specific agreement during the third quarter ended September 30, 2013. There is uncertainty as to whether the related party will have sufficient liquidity to repay the Company or fund the indemnification agreements should it become necessary (see Note 11). In addition, on August 29, 2013, the Company entered into and paid an additional settlement agreement for $300,000. On September 18, 2013, the Company made a proposal to the board of directors of Transcept Pharmaceuticals, Inc. (“Transcept”) to acquire all of the outstanding shares of Transcept’s common stock for $4.00 per share in cash. The proposal has been rejected by Transcept’s board of directors. The Company has invested approximately $3 million and acquired approximately 4.96% of the outstanding common stock of Transcept as part of the proposal process. If Transcept accepts the Company’s proposal to acquire all of its outstanding shares of common stock, the Company will need to obtain additional equity or debt financing to consummate the acquisition and consolidation (see Note 15). Management believes the Company’s ability to continue its operations depends on its ability to raise capital. The Company’s future depends on the costs, timing, and outcome of regulatory reviews of its product candidates and the costs of commercialization activities, including product marketing, sales and distribution. During the first quarter of 2013, the Company raised an aggregate of approximately $9.95 million in certain private placement transactions. During the third quarter of 2013, the Company raised an additional $24.92 million in aggregate proceeds in connection with a private placement transaction. The Company expects to continue to finance its cash needs through additional private equity offerings and debt financings, corporate collaboration and licensing arrangements and grants from patient advocacy groups, foundations and government agencies. Although management believes that the Company has access to capital resources, there are no commitments for financing in place at this time, nor can management provide any assurance that such financing will be available on commercially acceptable terms, if at all. These conditions raise substantial doubt about the Company’s ability to continue as a going concern. These unaudited condensed consolidated financial statements do not include any adjustments relating to the recovery of assets or the classification of liabilities that might be necessary should the Company be unable to continue as a going concern.

SUMMARY OF SIGNIFICANT ACCOUNTI

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES	9 Months Ended
	Sep. 30, 2013
Accounting Policies [Abstract]
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES	NOTE 4. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES A summary of the significant accounting policies applied in the preparation of the accompanying condensed consolidated financial statements follows: Principles of Consolidation The unaudited condensed consolidated financial statements represent the consolidation of the accounts of the Company and its subsidiary in conformity with U.S. GAAP. All intercompany accounts and transactions have been eliminated in consolidation. Cash For purposes of the statement of cash flows, the Company considers cash instruments with maturities of less than three months when purchased to be cash equivalents. There are no cash equivalents as of the balance sheet dates. Marketable Securities The Company accounts for marketable securities held as “available-for-sale” pursuant to ASC 320 Investments — Debt and Equity Securities (“ASC 320”). The Company classifies these investments as current assets and carry them at fair value. Unrealized gains and losses are recorded as a separate component of stockholders’ equity as accumulated other comprehensive income. Realized gains or losses on marketable security transactions are reported in earnings and computed using the specific identification of cost basis. Marketable securities are maintained at one financial institution and are governed by the Company’s investment policy as approved by our Board of Directors. Fair values of marketable securities are based on quoted market prices. Valuation of marketable securities are further describe in Note 6. The Company’s current investment policy generally limit security investments for purposes of strategic acquisitions. Based on the liquidity position of the Company, the CEO and CFO are authorized to make various investment transaction decisions for prudent investment of the Company’s excess funds. The ability to conduct investments is limited to the CEO and CFO. The current policy limit marketable securities investments with a maturity, credit quality, and concentration that is authorized only by the CEO and CFO. Employee Stock-Based Compensation The Company accounts for stock-based compensation in accordance with ASC 718 Compensation — Stock Compensation (“ASC 718”). ASC 718 addresses all forms of share-based payment (“SBP”) awards including shares issued under employee stock purchase plans and stock incentive shares. Under ASC 718 awards result in a cost that is measured at fair value on the awards’ grant date, based on the estimated number of awards that are expected to vest and will result in a charge to operations. Non-Employee Stock-Based Compensation The Company accounts for equity instruments issued to non-employees in accordance with the provisions of ASC 505, “Equity Based Payments to Non-Employees”, (“ASC 505”) and ASC 718 which requires that such equity instruments are recorded at their fair value on the measurement date. The measurement of stock-based compensation is subject to periodic adjustment as the underlying equity instruments vest. Non-employee stock-based compensation charges are being amortized over their respective contractual vesting periods. Use of Estimates In preparing financial statements in conformity with U.S. GAAP, management is required to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported amounts of expenses during the reporting period. Due to inherent uncertainty involved in making estimates, actual results reported in future periods may be affected by changes in these estimates. On an ongoing basis, the Company evaluates its estimates and assumptions. These estimates and assumptions include valuing equity securities in share-based payments, estimating fair value of equity instruments recorded as derivative liabilities, estimating the useful lives of depreciable and amortizable assets and estimating the fair value of long-lived assets to assets whether impairment charges may apply. Research and Development Costs Research and development costs are charged to operations as incurred and consist primarily of consulting costs, contract research and development costs, and compensation costs. For the three and nine months ended September 30, 2013 and 2012, and for the period from March 11, 2011 (inception) through September 30, 2013, the Company recognized $1,399,875, $110,656, $2,113,813, $286,889, and $3,008,326, respectively, of research and development costs. Patents The Company capitalized external cost, such as filing fees and associated attorney fees, incurred to obtain issued patents and patent applications pending. The Company expense cost associated with maintaining and defending patents subsequent to their issuance in the period incurred. The Company amortizes patent cost once issued on a straight-line basis over the estimate useful lives of the patents. The Company assess the potential impairment to all capitalized patent cost when events or changes in circumstances indicate that the carrying amount of our patent may not be recoverable. The Company accounts for patent costs in accordance with ASC Topic 350, “Goodwill and Other Intangible Assets” (“ASC 350”) and ASC Topic 805, “Business Combinations” (“ASC 805”). Basic and diluted Net Loss Per Share Basic and diluted net loss per share has been computed by dividing net loss by the weighted average number of common shares outstanding during the period. All potentially dilutive common shares have been excluded since their inclusion would be anti-dilutive. An aggregate of 4,462,426 and 0 warrants were excluded from the computation of diluted net loss per common share for the three and nine months ended September 30, 2013 and 2012, respectively, because their inclusion would have an anti-dilutive effect for the periods presented. An aggregate of 210,000 and 0 stock options were excluded from the computation of diluted net loss per common share for the three and nine months ended September 30, 2013 and 2012, respectively, because they would have an anti-dilutive effect for the periods presented. An aggregate of 211,073 and 927,310 incentive shares were excluded from the computation of diluted net loss per common share for the three and nine months ended September 30, 2013 and 2012, respectively, because they were contingent shares subject to recall. Derivative Instruments The Company does not use derivative instruments to hedge exposures to cash flow, market or foreign currency risks. The Company evaluates all of its financial instruments to determine if such instruments are derivatives or contain features that qualify as embedded derivatives. For derivative financial instruments that are accounted for as liabilities, the derivative instrument is initially recorded at its fair value and is then revalued at each reporting date, with changes in the fair value reported in the statements of operations. For stock-based derivative financial instruments, the Company calculates the fair value of the financial instruments using a probability-weighted Black-Scholes option pricing model, which is comparable to the Binomial Lattice options pricing model at inception and on each subsequent valuation date. The classification of derivative instruments, including whether such instruments should be recorded as liabilities or as equity, is evaluated at the end of each reporting period (see Note 7 and Note 8). Joint and Several Liability Assessment The Company measures obligations resulting from joint and several liability arrangements as the sum of the amount that the Company has a) contractually agreed to pay, and b) any additional amounts that the Company expects to pay on behalf of its co-obligors. Financial Instruments and Fair Value ASC Topic 820, “Fair Value Measurements and Disclosures,” (“ASC Topic 820”) establishes a fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. The hierarchy gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1 measurements) and the lowest priority to unobservable inputs (Level 3 measurements). The three levels of the fair value hierarchy under ASC Topic 820 are described below: L evel 1 Level 2 Level 3 In estimating the fair value of the Company’s marketable securities available-for-sale, the Company used quoted prices in active markets (see Note 6 and Note 8). In estimating the fair value of the Company’s derivative liabilities, the Company used a probability-weighted Black-Scholes option pricing model (see Note 7 and Note 8). Financial assets with carrying values approximating fair value include cash as well as marketable securities, deposits on license agreements, prepaid expenses and other current assets. Financial liabilities with carrying values approximating fair value include accounts payable and accrued expenses. Registration Payment Arrangement The Company accounted for registration rights agreements in accordance with ASC 825-20, “Registration Payment Arrangements.” ASC 825-20 addresses an issuer’s accounting for registration payment arrangements. This pronouncement specifies that the contingent obligation to make future payments or otherwise transfer consideration under a registration payment arrangement, whether issued as a separate agreement or included as a provision of a financial instrument, should be separately recognized and accounted for as a contingency in accordance with ASC 450-20 “Loss Contingencies”. Reclassifications Certain prior year financial statement balances have been reclassified to conform to the current year presentation. These reclassifications had no effect on the recorded net loss. Subsequent Events The Company follows the provisions of ASC Topic 855-10, “Subsequent Events,” relating to subsequent events. This guidance establishes principles and requirements for subsequent events. This guidance defines the period after the balance sheet date during which events or transactions that may occur would be required to be disclosed in a company’s financial statements. The Company has evaluated subsequent events up to the date of issuance of this report.

RESTATEMENT OF PREVIOUSLY ISSUE

RESTATEMENT OF PREVIOUSLY ISSUED FINANCIAL STATEMENTS	9 Months Ended
	Sep. 30, 2013
Restatements Of Previously Issued Consolidated Financial Statements Abstract [Abstract]
RESTATEMENT OF PREVIOUSLY ISSUED FINANCIAL STATEMENTS	NOTE 5. RESTATEMENT OF PREVIOUSLY ISSUED FINANCIAL STATEMENTS (1) In September 2014, the Company’s board of directors requested that the Company’s outside legal counsel conduct an investigation (the “Investigation”) into the circumstances surrounding the negotiation and execution by the former Chief Executive Officer of the Company, Martin Shkreli, of certain consulting and settlement agreements entered into by the Company. The Investigation also covered additional agreements and other matters involving Mr. Shkreli during his tenure as the Chief Executive Officer of the Company. In January 2015, the Company’s board of directors appointed an Oversight Committee of the board of directors (the “Oversight Committee”), consisting of Gary Lyons and Jeffrey Meckler, each of whom was not a member of the Company’s board of directors during the period of time covered by the Investigation. The Company’s board of directors delegated to the Oversight Committee the independent and plenary authority to oversee and direct the Investigation and make findings and decisions related to the Investigation. The following information is the Oversight Committee’s conclusions, which specifically relates to the financial statements for the three and nine month periods ended September 30, 2013: · On September 30, 2013, the Company entered into a consulting agreement and release with an individual who was an investor in investment funds previously managed by Mr. Shkreli (the “MSMB Entities”), or that otherwise had financial dealings with Mr. Shkreli. The agreement provided for the issuance of a total of 331,500 shares of common stock of the Company. The Oversight Committee concluded that the Company should not continue to treat this agreement as a consulting arrangement because its predominant purpose appears to have been to settle and release claims against the MSMB Entities or Mr. Shkreli personally, and not to provide meaningful and sustained consulting services to the Company. The Oversight Committee concluded that certain of the transactions described above were consummated without specific approval of the Company’s board of directors or without the Company’s board of directors knowing all of the relevant facts. The Oversight Committee also determined that the Company made approximately $135,000 of payments to certain third party service providers on behalf of the MSMB Entities, which were charged to results of operations during the nine months ended September 30, 2013. The Related Party Transactions note has been updated to include this disclosure. As a result of the matters that emerged from the investigation, we have determined that the financial statements contained in the Company’s Form 10-Q for the three months ended September 30, 2013 (the “2013 Q3 Form 10-Q”) contained errors relating to the matters described above. Specifically, the Company previously recognized expense related to the stock issued pursuant to the consulting agreement as a non-employee share-based payment arrangement. Had the Company accounted for this arrangement as a settlement, the Company would have recorded, as of the date of the agreement, an expense and a settlement liability related to the entire amount of the stock to be issued under such agreement. The settlement liability would have been revalued at each reporting period based on changes in the Company’s stock price until the stock had been entirely issued. On February 19, 2015, the Company’s board of directors concluded that as a result of the errors related to this agreement, the financial statements contained in the September 30, 2013 Form 10-Q should no longer be relied upon. The Company has corrected such errors, including any related disclosures. The following table sets forth the effects (in thousands) of the matters identified by the Oversight Committee on affected items within the Company’s previously reported Condensed Consolidated Balance Sheet for the quarter ended September 30, 2013, had the adjustments been made: September 30, 2013 As Revised* As Restated Current liabilities $ 28,788 $ 30,943 Additional paid in Capital $ 47,498 $ 46,220 The following table sets forth the effects (in thousands) of the matters identified by the Company during the fourth quarter of 2013 and the Oversight Committee in January 2014 on affected items within the Company’s previously reported Condensed Consolidated Statement of Operations for the three months and nine months ended September 30, 2013, had the adjustments been made in the corresponding quarters: Three months ended September 30, 2013 Nine months ended September 30, 2013 As Revised* As Restated As Revised* As Restated Selling, general and administrative $ 3,755 $ 4,631 $ 10,141 $ 11,017 Operating loss $ (5,155 ) $ (6,031 ) $ (12,255 ) $ (13,131 ) Net loss $ (11,135 ) $ (12,011 ) $ (21,156 ) $ (22,033 ) Net loss per share, basic and diluted $ (0.72 ) $ (0.78 ) $ (1.65 ) $ (1.72 ) *For matters identified during the fourth quarter of 2013 described in (2) below. (2) As previously discussed in the Form 10-K for the year ended 2013, in the fourth quarter of 2013, the Company discovered that certain warrants issued to a placement agent in connection with our February Private Placement were not recorded and certain expenses were misstated in our condensed consolidated financial statements for the first, second and third quarters of 2013. The misstatement of expenses include costs related to the February 14, 2013 Private Placement Offering, certain accruals, consulting fees and changes in the fair value of the unrecorded warrants, which required liability classification. The financial statements included herein have been revised to include the effects of these matters. The following table (in thousands) sets forth the effects of revisions we made to our previously reported Condensed Consolidated Balance Sheet as of September 30, 2013 with respect to the errors we identified during the fourth quarter of 2013: September 30, 2013 As Reported As Revised Derivative financial instruments, warrants $ 22,234 $ 23,853 Total liabilities 27,169 28,788 Additional paid in Capital 48,650 47,498 Deficit accumulated during the development stage (54,301 ) (54,768 ) Stockholders’ deficit $ (5,804 ) $ (7,423 ) The following table (in thousands) sets forth the effects of revisions we made to our previously reported Condensed Consolidated Statement of Operations for the three and nine months ended September 30, 2013, with respect to the errors we identified during the fourth quarter of 2013: Three months ended September 30, Nine months ended September 30, As Reported As Revised As Reported As Revised Selling, general and administrative $ 3,755 $ 3,755 $ 10,391 $ 10,141 Operating loss (5,155 ) (5,155 ) (12,505 ) (12,255 ) Change in fair value of derivative financial instruments-warrants (5,803 ) (6,040 ) (8,199 ) (8,916 ) Net loss (10,898 ) (11,135 ) (20,689 ) (21,156 ) Net loss per share, basic and diluted $ (0.71 ) $ (0.72 ) $ (1.62 ) $ (1.65 ) The cumulative effect of all errors discussed in (1) and (2) on our results of operations for the period from March 11, 2011 through September 30, 2013 for purposes of development stage reporting is that our cumulative loss in the amount of $54.3 million, as reported, is now $55.6 million, as restated. Recently Issued Accounting Pronouncements In February 2013, the FASB issued Accounting Standards Updated (“ASU”) 2013-04 “Obligations Resulting from Joint and Several Liability Arrangements for Which the Amount at the Reporting Date is Fixed”) (“ASU 2013-04”). The guidance in this update is effective for fiscal years beginning after December 15, 2013 with early adoption permitted. The guidance in this update requires companies to measure obligations resulting from joint and several liability arrangements as the sum of the amount the entity has a) contractually agreed to pay, and b) any additional amounts that the entity expects to pay on behalf of its co-obligors. The Company early adopted this guidance in the second quarter of 2013 (Note 3 and Note 11). Except as noted above, management does not believe that any recently issued, but not yet effective accounting pronouncements, if adopted, would have a significant effect on the accompanying consolidated financial statements.

MARKETABLE SECURITIES

MARKETABLE SECURITIES	9 Months Ended
	Sep. 30, 2013
Marketable Securities [Abstract]
MARKETABLE SECURITIES	NOTE 6. MARKETABLE SECURITIES The Company measures marketable securities on a recurring basis. Generally, the types of securities the Company invests in are traded on a market such as the NASDAQ Global Market, which the Company considers to be Level 1 inputs. Marketable securities at September 30, 2013 consisted of the following: Unrealized Unrealized Estimated Fair Cost Gains Losses Value Marketable securities available-for-sale: $ 3,112,210 $ - $ 154,834 $ 2,957,376 The Company’s marketable securities is comprised entirely of the common stock of Transcept.

DERIVATIVE FINANCIAL INSTRUMENT

DERIVATIVE FINANCIAL INSTRUMENTS	9 Months Ended
	Sep. 30, 2013
Investments, All Other Investments [Abstract]
DERIVATIVE FINANCIAL INSTRUMENTS	NOTE 7. DERIVATIVE FINANCIAL INSTRUMENTS In accordance with ASC Topic 815-40, “Derivative and Hedging – Contracts in Entity’s Own Equity” (“ASC Topic 815-40”), instruments which do not have fixed settlement provisions are deemed to be derivative instruments. Based upon the Company’s analysis of the criteria contained in ASC Topic 815-40, the warrants issued in connection with the sale of the common stock during the period ended September 30, 2013 that do not have fixed settlement provisions, are not indexed to Company’s own stock. The fair value of the warrants are classified as derivative liabilities due to a ratchet provision that allows for a favorable adjustment to the exercise price if the Company issues additional equity instruments in the future at an effective price per share less than the exercise price then in effect. The warrants are re-measured at each balance sheet date based on estimated fair value. Changes in estimated fair value are recorded as non-cash valuation adjustments within other income (expense) in the Company’s results of operations. The Company recorded a loss on a change in the estimated fair value of warrants of $6,040,054 and $8,915,672 for the three and nine months ended September 30, 2013, respectively. The Company calculated the fair value of the warrants using a probability-weighted Black-Scholes option pricing model which is comparable to the Binomial Lattice pricing model. The assumptions used at the date of issuance and at September 30, 2013 are noted in the following table: As of Date of issuance Date of issuance Date of issuance September 30, Fair market price of common stock $3.75 $4.50 $4.50 $6.50 Contractual term 5 years 5 years 5 years 4.38 – 4.87 years Risk-free interest rate 0.86% 1.48% 1.48% 1.41% Expected volatility 101% 106% 106% 96%-102% Expected volatility is based on historical stock volatilities of several comparable publicly-traded companies over a period equal to the expected terms of the warrants, as the Company does not have a long trading history to estimate the volatility of its own common stock. The warrants have a transferability provision. Based on guidance provided in SEC Staff Accounting Bulletin No. 107 (“SAB 107”) for options issued with such a provision, the Company used the full contractual term as the initial expected term of the warrants. The risk free interest rate is based on the U.S. Treasury security rates for the remaining term of the warrants at the measurement date.

FAIR VALUE MEASUREMENTS

FAIR VALUE MEASUREMENTS	9 Months Ended
	Sep. 30, 2013
Fair Value Disclosures [Abstract]
FAIR VALUE MEASUREMENTS	NOTE 8. FAIR VALUE MEASUREMENTS The following table presents the Company’s asset and liability that is measured and recognized at fair value on a recurring basis classified under the appropriate level of the fair value hierarchy as of September 30, 2013: Fair Value Measurements at September 30, 2013 Significant other Significant Total carrying value at Quoted prices in active observable inputs unobservable inputs September 30, 2013 markets (Level 1) (Level 2) (Level 3) Asset: Marketable securities, available-for-sale $ 2,957,376 $ 2,957,376 $ - $ - Liability: Derivative liability related to warrants $ 23,853,325 $ - $ - $ 23,853,325 Settlement agreement $ 2,154,750 $ 2,154,750 $ - $ The following table sets forth a summary of changes in the estimated fair value of the Company’s Level 3 liability for the nine months ended September 30, 2013: Fair Value Measurements of Common Stock Warrants Using Significant Unobservable Inputs (Level 3) Balance at January 1, 2013 $ - Issuance of common stock warrants: February 14, 2013 5,407,605 August 14, 2013 328,561 August 15, 2013 9,201,487 Total value upon issuance 14,937,653 Change in fair value of common stock warrant liability 8,915,672 Balance at September 30, 2013 $ 23,853,325 A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. At each reporting period, the Company performs a detailed analysis of the assets and liabilities that are subject to ASC Topic 820. At each reporting period, all assets and liabilities for which the fair value measurement is based on significant unobservable inputs or instruments which trade infrequently and therefore have little or no price transparency are classified as Level 3.

LICENSE AGREEMENT

LICENSE AGREEMENT	9 Months Ended
	Sep. 30, 2013
Notes to Financial Statements
LICENSE AGREEMENT	NOTE 9. LICENSE AGREEMENT On February 16, 2012 the Company entered into an agreement pursuant to which a biotech company (the “Sublicensor”) with license rights to certain drug technologies agreed to grant us a worldwide sublicense for the development, manufacture and commercialization of a drug technology which is referred to as DARA, which is an Angiotesin Receptor Blocker (“ARB”) and Endothelin Receptor Antagonist (“ERA”) which the Company is initially using in connection with the treatment of focal segmental glomerulosclerosis (“FSGS”) and which we refer to as RE-021. The sublicense agreement also enables the Company to sell the licensed technology as a research product or sublicense the technology to other third parties as potential sources of revenue. Under the license agreement, Sublicensor is obligated to transfer to the Company certain information, records, regulatory filings, materials and inventory controlled by Sublicensor and relating to or useful for developing RE-021. The Company must use commercially reasonable efforts to develop and commercialize RE-021 in specified major market countries and other countries in which the Company believes it is commercially reasonable to develop and commercialize such products. The agreement shall continue until neither party has any obligations under the agreement to make payments to the other party. In accordance with the agreement as amended most recently as of January 7, 2013, the Company made two non-refundable payments totaling $2,550,000, the first payment of $1,150,000 made upon execution and the second payment of $1,400,000 was made in February 2013, which includes a $250,000 fee payable to the sublicensee in exchange for extended due date of this payment from October 1, 2012 to February 2013. As of September 30, 2013, the Company has recognized $2,300,000 for the cost of the License Agreement which is presented in the accompanying condensed consolidated balance sheet as an intangible asset that is being amortized on a straight-line basis over the term of the License Agreement which expires on September 30, 2023. The $250,000 of extension fees were expensed to operations in February 2013. In addition, the Company issued 620,000 common shares to Ligand valued at $1,550,000 as a result of the merger transaction, the amount of which was expensed to operations in December 2012. For the three and nine months ended September 30, 2013 and 2012, and for the period from March 11, 2011 (inception) through September 30, 2013, the Company recognized amortization expense of the license related to this agreement of $51,065, $34,885, $151,531, $71,466, and $272,914 respectively. The Company’s sublicense agreement with Ligand specifically provides rights for the global development, manufacture, and commercial utilization of certain compounds, for any and all medical applications. The Company purchased this license in a bargained exchange transaction that was conducted at arm’s length with an unrelated party. ASC 305 states that “Intangible assets that are acquired individually or with a group of assets in a transaction other than a business combination or an acquisition by a not-for-profit entity may meet asset recognition criteria in FASB Concepts Statement No. 5, Recognition and Measurement in Financial Statements of Business Enterprises.” The sublicense agreement provides broad and exclusive rights to use a technology based intangible that is a legally protected, separable, and has a stand-alone value independent of the Company or any other holder. The Company specifically considered the nature of the license, the rights to its use and whether the license itself meets the legal contractual and/or separability criterion described in under ASC 805 “Business Combinations.” The specific technology licensed under this agreement is known as DARA. This technology is fully developed, protected by issued patents and pending patent applications; the technology is the product of a long-term development effort that cannot be replicated. The DARA technology embodies unique know-how, including all biological, chemical, pharmacological toxicological, clinical, manufacturing assay and related data. This technology is also not generally known. Under ASC 805, intangible assets whose future economic benefits are legally protected are deemed to have met the legal contractual criteria and would therefore be accounted for as a separately identifiable intangible asset. In addition to the above, the technology has a stand-alone value that makes it capable of being separated or divided from the Company or any other acquirer. The technology can be sold, transferred, licensed, rented, or exchanged either individually or together with a related contract, identifiable asset, or liability. The Company believes that the purchase of the license and the fact that the Company or any other market participant in possession of this license or a similar type of license would be able to sell, license, or otherwise exchange this technology for something else of value, provides evidence of its separability. Further, the licensed technology could have use in other stand-alone broad applications outside of the initial intended use for FSGS, including hypertension and other nephrotic conditions. In connection with the acquired rights, the Company also acquired the right to enter into future sublicense agreements and has the right to transfer/sell this right to other third parties. Accordingly, the right to enter into sublicense agreements could be divided from other assets and independently sold. Consequently, this right meets the separability criterion specified in ASC 805-20-55- 3 (e.g. it is an acquired intangible asset that is capable of being licensed).

NOTES PAYABLE

NOTES PAYABLE	9 Months Ended
	Sep. 30, 2013
Debt Disclosure [Abstract]
NOTES PAYABLE	NOTE 10. NOTES PAYABLE Note Payable - related party On February 1, 2012, the Company entered into a secured promissory note with a related party in the amount of $900,000, with an interest rate of 12% per annum, compounded monthly. The outstanding principal and interest balance of this note was fully repaid during the first quarter of 2013. Total interest expense recognized for the three and nine months ended September 30, 2013 and 2012, and for the period from March 11, 2011 (inception) through September 30, 2013 amounted to $0, $26,761, $41,563, $70,559 and $147,480, respectively.

RELATED PARTY TRANSACTIONS

RELATED PARTY TRANSACTIONS	9 Months Ended
	Sep. 30, 2013
Related Party Transactions [Abstract]
RELATED PARTY TRANSACTIONS	NOTE 11. RELATED PARTY TRANSACTIONS On December 8, 2011, the Company received advances of funds aggregating $8,500 from entities related through common ownership. Such advances were repaid during the first quarter of 2013. In August 2012, the Company paid a security deposit on behalf of an affiliate of $137,547 in connection with a building lease entered into by such affiliate. The Company assumed the lease from its affiliate in April 2013, whereby the security deposit was assigned to the Company. The Company leases approximately 4,216 square feet of office space for approximately $275,000 annual base rent plus rent escalations, common area maintenance, insurance, and real estate taxes, under a lease agreement expiring August 2016. In the second quarter of 2013, the Company, its Chief Executive Officer and a related party, which is a former investor in the Company that was previously managed by the Company’s Chief Executive officer, became party to a series of agreements to settle up to $2,284,511 of liabilities, which Company management believes are the primary obligation of the related party. The Company and the related party have entered into indemnification agreements whereby the related party has agreed to defend and hold the Company harmless against all such obligations and amounts, whether paid or unpaid, arising from these agreements. Notwithstanding the indemnification, the Company recorded a $2,284,511 charge to operations during the quarter ended September 30, 2013 that was offset by a corresponding liability of $1,691,400 for the difference between (a) the aggregate amount of all such settlements, and (b) $593,111 of cash and non-cash consideration that the Company paid to immediately settle a portion of the agreement on behalf of the related party. The $1,691,400 is entirely paid as of the date of this filing. In addition, the Chief Executive Officer also agreed to provide one of the counter parties with 47,128 shares of his common stock in the Company as a separate component of one of the these settlement agreements. Accordingly, the Company does not believe it is required to record a liability for the shared-based component of this specific agreement during the third quarter ended September 30, 2013. There is uncertainty as to whether the related party will have sufficient liquidity to repay the Company or fund the indemnification agreements should it become necessary. Concurrent with the execution of such settlement agreements, the Company received promissory notes from the related party whereby the related party agreed to pay the Company the principal amount of $593,111 plus interest at an annualized rate of 5% as reimbursement of the payments that the Company made to settle a portion of the agreements. During the nine months ended September 30, 2013, the Company also made approximately $135,000 of other payments to third party service providers to settle liabilities on behalf of MSMB Entities,that were charged to results of operations during the nine months ended September 30, 2013. In October 2013, the Company paid $1,655,000 in cash and 5,000 shares of common stock valued at $36,400 to settle agreements on behalf of a related party. Upon payment, the Company and the related party entered into promissory notes whereby the related party agreed to pay the Company the principal amount of $1,691,400 plus interest at an annualized rate of 5% as reimbursement of payments that the Company made to settle a portion of the agreements. The Company applied the accounting guidance provided in ASU 2013-04. The guidance in this update is effective for fiscal years beginning after December 15, 2013 with early adoption permitted. The guidance in this update requires companies to measure obligations resulting from joint and several liability arrangements as the sum of the amount that the entity has a) contractually agreed to pay, and b) any additional amounts that the entity expects to pay on behalf of its co-obligors. The Company has recorded the full amount of the settlements as a charge to its operations due to uncertainty as to whether the related party will have sufficient liquidity to repay the Company or fund the indemnification agreements should it become necessary. Any amounts that the Company may recover under the note due from the related party or under the terms of the indemnification agreement, if in fact any amounts are recovered at all, would be characterized as a capital contribution at the date such payments are received. On August 15, 2013, the Company closed a private placement and sold 5,531,401 shares of the Company’s common stock, at a purchase price of $4.50 per share, or $24,891,303 in the aggregate, and warrants to purchase up to an aggregate of 2,765,701 shares of common stock with an exercise price of $6.00 per share underlying each warrant. Members of the Company’s management purchased an aggregate of 10,522 shares of common stock and warrants to purchase up to an aggregate of 5,261 shares of common stock in such private placement. The Warrants are deemed to be derivative instruments due to a ratchet provision that adjusts the exercise price if the Company issues additional equity instruments in the future at an effective price per share less than the exercise price then in effect. The issuance of the shares of common stock in such private placement was not registered under the Securities Act as such issuance was exempt from registration under Section 4(2) of the Securities Act and Regulation D promulgated thereunder.

STOCKHOLDERS' DEFICIT

STOCKHOLDERS' DEFICIT	9 Months Ended
	Sep. 30, 2013
Equity [Abstract]
STOCKHOLDERS' DEFICIT	NOTE 12. STOCKHOLDERS’ DEFICIT Issuances Common Stock In January 2013, the Company sold an aggregate of 272,221 shares of common stock, at a purchase price of $3.00 per share in certain private placement transactions, for an aggregate purchase price of $816,664 in cash. The issuance of such shares of common stock was not registered under the Securities Act as such issuance was exempt from registration under Section 4(2) of the Securities Act and Regulation D promulgated thereunder. On February 14, 2013, the Company closed a private placement (the “February Private Placement”) of 3,045,929 shares of common stock, at a purchase price of $3.00 per share, or $9,137,787 in the aggregate, and warrants (the “Warrants”) to purchase up to an aggregate of 1,597,969 shares of common stock with an exercise price of $3.60 per such share underlying any Warrant. The Warrants are deemed to be derivative instruments due to a ratchet provision that adjusts the exercise price if the Company issues additional equity instruments in the future at an effective price per share less than the exercise price then in effect. Upon issuance of the warrants, the Company recorded a liability of $4,505,605 to derivative financial instruments in its balance sheet. The issuance of such shares of common stock was not registered under the Securities Act as such issuance was exempt from registration under Section 4(2) of the Securities Act and Regulation D promulgated thereunder. On August 15, 2013, the Company closed a private placement and sold 5,531,401 shares of the Company’s common stock, at a purchase price of $4.50 per share, or $24,891,303 in the aggregate, and warrants to purchase up to an aggregate of 2,765,701 shares of common stock with an exercise price of $6.00 per share underlying each warrant. The Warrants are deemed to be derivative instruments due to a ratchet provision that adjusts the exercise price if the Company issues additional equity instruments in the future at an effective price per share less than the exercise price then in effect. Upon issuance of the warrants, the Company recorded a liability of $9,201,487 to derivative financial instruments in its balance sheet. The issuance of the shares of common stock in such private placement was not registered under the Securities Act as such issuance was exempt from registration under Section 4(2) of the Securities Act and Regulation D promulgated thereunder. February Registration Rights Agreement On February 14, 2013, in connection with the closing of the February Private Placement, the Company entered into a Registration Rights Agreement (the “Registration Rights Agreement”) with the purchasers in the February Private Placement (the “Purchasers”), which sets forth the rights of the Purchasers to have their shares of common stock purchased in the February Private Placement and shares of common stock issuable upon exercise of the Warrants registered with the SEC for public resale. Pursuant to the Registration Rights Agreement, the Company was required to file a Registration Statement on Form S-1 (the “Registration Statement”) with the SEC within 30 days of the date of the Registration Rights Agreement registering the total number of shares of common stock purchased in the February Private Placement and shares of common stock issuable upon exercise of the Warrants. The Company further agreed to use its reasonable efforts to have the Registration Statement declared effective within 60 days after the date of the Registration Rights Agreement (or, in the event of a “full review” by the SEC, within 90 days after the date of the Registration Rights Agreement). The Company has also agreed to use reasonable efforts to maintain the effectiveness of the Registration Statement until all of the securities covered by the Registration Statement have or may be sold by investors under Rule 144 of the Securities Act, without volume or manner-of-sale restrictions. The Registration Rights Agreement provided that in the event the Registration Statement was not filed or declared effective within the prescribed time period or if the Company failed to maintain the effectiveness of the Registration Statement as required for specified time periods, the Company shall pay to the holders of registrable securities, on the date of each such event and on each monthly anniversary thereof until the applicable event is cured, partial liquidated damages equal to 2.0% of the aggregate purchase price paid by such Purchaser in the February Private Placement, up to a maximum of 10.0% of such aggregate purchase price. If the Company fails to pay any partial liquidated damages pursuant to this Section in full within seven days after the date payable, the Company will pay interest thereon at a rate of 18% per annum (or such lesser maximum amount that is permitted to be paid by applicable law) to the Purchaser, accruing daily from the date such partial liquidated damages are due until such amounts, plus all such interest thereon, are paid in full. The Company determined, as of the date of the financing transaction, that it was probable that it would not be in a position to cause the registration statement to be declared effective within the contractually defined time period. Accordingly, the Company allocated approximately $360,000 of the proceeds to a registration payment arrangement liability on the date that the financing transaction closed, in accordance with the guidelines of ASC 825-20. As described in Note 3, the Company and the investors who are parties to the registration payment arrangement entered into an the Amended Registration Rights Agreement which provides, among other things, for a waiver of the liquidated damages that the Company incurred under the original terms of the registration payment arrangement described herein. The Company recognized $360,000 as income upon the waiver of the liquidated damages. First Amendment to the February Registration Rights Agreement As described in Note 3, the Company and the investors who participated in the private placement transaction that the Company completed on February 14, 2013 entered into the Amended Registration Rights Agreement which provides, among other things, for (i) a waiver of any and all liquidated damages that the Company incurred for its inability to cause the a registration statement to be declared effective within certain contractually defined time-frames stipulated in the original agreement; (ii) a commitment on the part of the investors in the February private placement to participate in the private placement transaction that the Company completed on August 15, 2013; and (iii) a covenant on the part of the Company to proceed with the sale of shares that were issued in the August 15, 2013 private placement transaction. In exchange, the Company paid an aggregate fee of $2,495,256 to these investors consisting of (i) 73,710 shares of the Company’s common stock with an aggregate fair value of $331,695 (based on the selling price of $4.50 per share in the August financing transaction); (ii) cash in the amount of $1,835,000; and (iii) warrants to purchase 98,756 shares of common stock with a fair value of $328,561 that were classified as derivative liability instruments. The investors were also given the option to purchase shares of the Company’s common stock at $4.50 per share as a use of the cash portion of the payment arrangement. Accordingly, $946,196 of the cash portion of the fee was settled in cash and the remainder was settled by the issuance of 197,512 shares. Additionally, the Company paid $103,425 to an investor to whom the Company sold shares in a private placement transaction in January 2013 and who participated in the August 2013 private placement transaction. This payment was settled entirely by the issuance of 20,685 shares of the Company’s common stock at a value of $5.00 per share. The Company recorded the aggregate amount of the payments made to the investors by to allocating approximately $360,000 to the waiver of the original registration payment obligation taken as a charge to operations and the remaining amount of $2,238,681 is treated as reduction of the proceeds received in the August financing transaction. August Registration Rights Agreement On August 15, 2013, in connection with the closing of the August 15, 2013 private placement (the “August Private Placement”), the Company entered into a Registration Rights Agreement (the “Registration Rights Agreement”) with the purchasers in the August Private Placement (the “Purchasers”), which sets forth the rights of the Purchasers to have their shares of common stock purchased in the Private Placement and shares of common stock issuable upon exercise of the Warrants registered with the SEC for public resale. Pursuant to the Registration Rights Agreement, the Company was required to file a Registration Statement on Form S-1 (the “Registration Statement”) with the SEC within 30 days of the date of the Registration Rights Agreement registering the total number of shares of common stock purchased in the August Private Placement and shares of common stock issuable upon exercise of the Warrants. The Company further agreed to use its reasonable efforts to have the Registration Statement declared effective within 60 days after the date of the Registration Rights Agreement (or, in the event of a “full review” by the SEC, within 120 days after the date of the Registration Rights Agreement). The Company has also agreed to use reasonable efforts to maintain the effectiveness of the Registration Statement until all of the securities covered by the Registration Statement have or may be sold by investors under Rule 144 of the Securities Act, without volume or manner-of-sale restrictions. The Registration Rights Agreement provided that in the event the Registration Statement was not filed or declared effective within the prescribed time period or if the Company failed to maintain the effectiveness of the Registration Statement as required for specified time periods, the Company shall pay to the holders of registerable securities, on the date of each such event and on each monthly anniversary thereof until the applicable event is cured, partial liquidated damages equal to 2.0% of the aggregate purchase price paid by such Purchaser in the August Private Placement, up to a maximum of 10.0% of such aggregate purchase price. If the Company fails to pay any partial liquidated damages pursuant to this Section in full within seven days after the date payable, the Company will pay interest thereon at a rate of 18% per annum (or such lesser maximum amount that is permitted to be paid by applicable law) to the Purchaser, accruing daily from the date such partial liquidated damages are due until such amounts, plus all such interest thereon, are paid in full. On September 13, 2013, the Company submitted the Registration Statement to the SEC on a confidential basis. The Company determined, as of the date of the financing transaction, that it was probable that it would be in a position to cause the registration statement to be declared effective within the contractually defined time period. Stock Options On May 13, 2013, the Company issued options (the “Options”) to purchase 120,000 shares of common stock in connection with an employment agreement with Horacio Plotkin, M.D. (the “Plotkin Employment Agreement”) pursuant to which Dr. Plotkin was appointed as Chief Medical Officer of the Company. The options vest quarterly in pro rata portions during the 3 years following the effective date of July 1, 2013. The Company valued these Options using the Black-Scholes options pricing model and the following assumption terms: risk-free interest rate of .83% (based on the US Treasury note yield), expected term (in years) of 5.81 (based on guidance provided in SAB 107 that allows the Company to use the simplified method for “plain vanilla” options for this calculation), expected volatility of 98.56% (based on historical stock volatilities of several comparable publicly-traded companies over a period equal to the expected term of the options, as the Company does not have a long trading history to estimate the volatility of its own common stock), and an exercise price equal to the fair value of the stock on the date of issuance of $8.70 per share. For the three and nine months ended September 30, 2013 the Company recognized $64,716 and $101,399 as compensation expense related to the Options. At September 30, 2013, the unrecognized compensation expense, remaining amortization period, intrinsic value and remaining contract life of the Options are $703,333, 2.5 years, $0 and 9.62 years, respectively. On September 9, 2013, the Company issued options to purchase 90,000 shares of common stock to two employees. The options vest quarterly in pro rata portions during the 3 years following the effective date of July 1, 2013. The Company valued these options using the Black-Scholes options pricing model and the following assumption terms: risk-free interest rate of 1.71% (based on the US Treasury note yield), expected term (in years) of 5.81 (based on guidance provided in SAB 107 that allows the Company to use the simplified method for “plain vanilla” options for this calculation), expected volatility of 104.78% (based on historical stock volatilities of several comparable publicly-traded companies over a period equal to the expected term of the options, as the Company does not have a long trading history to estimate the volatility of its own common stock), and an exercise price equal to the fair value of the stock on the date of issuance of $6.20 per share. For the three and nine months ended September 30, 2013 the Company recognized $9,606 as compensation expense related to the options. At September 30, 2013, the unrecognized compensation expense, remaining amortization period, intrinsic value and remaining contract life of the options are $438,748, 2.5 years, $0 and 9.94 years, respectively.

INCENTIVE SHARES

INCENTIVE SHARES	9 Months Ended
	Sep. 30, 2013
Disclosure of Compensation Related Costs, Share-based Payments [Abstract]
INCENTIVE SHARES	NOTE 13. INCENTIVE SHARES At September 30, 2013, the Company did not have any active share-based compensation plans available for grants to employees, non-employee directors and consultants. Since its inception, the Company has granted incentive shares. For the three and nine months ended September 30, 2013 and 2012, and for the period from March 11, 2011 (inception) through September 30, 2013, the Company recognized $1,741,908, $7,027,675, $2,029,505, $14,014,402 and $26,419,022 as compensation expense related to incentive shares granted in the consolidated statements of operations, respectively. Share compensation for non-employee awards subject to vesting is being accrued at current fair value. As of September 30, 2013, there was approximately $1,294,232, of unrecognized compensation cost related to incentive shares issued. This amount is expected to be recognized over a weighted average of 2.25 years. Employee- Non- Total Weighted Average Unvested December 31, 2011 1,281,225 321,165 1,602,390 $ 4.00 Granted 866,180 87,503 953,683 12.89 Vested (2,048,280 ) (193,672 ) (2,241,952 ) 7.34 Forfeited (46,353 ) - (46,353 ) 9.06 Unvested December 31, 2012 52,772 214,996 267,768 5.79 Granted 135,000 - 135,000 6.24 Vested (17,918 ) (115,445 ) (133,363 ) 5.05 Forfeited (20,833 ) (37,500 ) (58,333 ) 4.00 Unvested September 30, 2013 149,021 62,051 211,072 $ 6.43 All of the Company’s share base payments were originally issued as Retrophin LLC Class B incentive units that represent a profits interest up through the date of Retrophin LLC’s conversation to a C Corporation, which was structured as a tax free exchange transaction. Shares granted as incentive shares were originally subject to certain conditions at the time of grant. Such conditions specified that upon the occurrence of a Termination Event, as defined in the amended operating agreement the Company shall have the right, but not the obligation, to repurchase, all, of the vested incentive shares owned by such incentive shareholder, at a purchase price based on the fair market value of the incentive shares determined in good faith by the Board of Directors. The aforementioned repurchase option was rescinded upon the Company’s conversion to a corporation. Effective May 20, 2013, the Company entered into an employment agreement with Marc L. Panoff (the “Panoff Employment Agreement”) pursuant to which Mr. Panoff was appointed as Chief Financial Officer and Chief Accounting Officer of the Company. In accordance with the terms of the Panoff Employment Agreement, Mr. Panoff will be granted 120,000 units of restricted common stock of the Company, a pro rata portion of which will vest quarterly beginning on December 31, 2013 during the 3 years following the effective date. On July 1, 2013, the Company granted 15,000 units of restricted common stock of the Company to an employee. The stock will vest quarterly in pro rata portions beginning September 30, 2013 during the 3 years following the grant date.

COMMITMENTS AND CONTINGENCIES

COMMITMENTS AND CONTINGENCIES	9 Months Ended
	Sep. 30, 2013
Commitments and Contingencies Disclosure [Abstract]
COMMITMENTS AND CONTINGENCIES	NOTE 14. COMMITMENTS AND CONTINGENCIES Leases The Company assumed a building lease from an affiliate in April 2013 for office space at its principal offices in New York, New York and is responsible for rent of approximately $275,000 annually plus rent escalations through August 2016 (see Note 11). On October 1, 2013, the Company entered into building lease for office space in Cambridge, Massachusetts and is responsible for rent of approximately $216,000 annually plus rent escalations through September 2016. On October 8, 2013, the Company entered into an amended lease agreement for additional office space at its principal offices in New York, New York and is responsible for additional rent of approximately $225,000 annually plus rent escalations through August 2016. Exclusivity Agreements On August 16, 2013, the Company announced that it had signed an agreement with a major pharmaceutical company for the exclusive right to negotiate a royalty-bearing U.S. license for a product to be developed for the treatment of Autism and Schizophrenia. Pursuant to the exclusivity agreement, the Company paid the major pharmaceutical company a non-refundable upfront fee of $2 million and will have an exclusive period of 120 days to negotiate a license agreement (see Note 3). The Company is in active negotiations to consummate a license agreement. If a definitive license agreement is consummated, the Company will apply the upfront fee to the license and will make a determination as to whether it should be treated as an asset or research and development expense. If no definitive agreement is consummated, the upfront fee will be expensed. On September 20, 2013, the Company signed an agreement with an individual for the exclusive right to negotiate a royalty-bearing U.S. license for a product to be developed for the treatment of central nervous system disorders. Pursuant to the exclusivity agreement, the Company paid the individual a non-refundable upfront fee of $250,000 and will have an exclusive right until December 31, 2013. If an agreement is not executed on or before December 31, 2013, the Company will receive a partial refund of the upfront fee. Upon execution of a license agreement, the Company would receive the exclusive right to the intellectual property to develop, manufacture and sell the product worldwide and would pay additional fees to the major pharmaceutical company. The Company is in active negotiations to consummate a license agreement. If a definitive license agreement is consummated, the Company will apply the upfront fee to the license and will make a determination as to whether it should be treated as an asset or research and development expense. If no definitive agreement is consummated, the upfront fee will be expensed. For the three and nine months ended September 30, 2013, the Company has recorded $2,250,000 as exclusivity agreement deposits to secure license exclusivity under current assets (see Note 3). Research Agreement Effective October 1, 2013, the Company signed a Sponsored Research Agreement (“SRA”) with St. Jude (see Note 15).

SUBSEQUENT EVENTS

SUBSEQUENT EVENTS	9 Months Ended
	Sep. 30, 2013
Subsequent Events [Abstract]
SUBSEQUENT EVENTS	NOTE 15. SUBSEQUENT EVENTS On October 1, 2013 the Company entered into a building lease for approximately 4,232 square feet of office space located in Cambridge, MA. The Company is responsible for approximately $216,000 annual base rent plus rent escalations, common area maintenance, insurance, and real estate taxes, under a lease agreement expiring in September 2016. On October 8, 2013, the Company amended its lease agreement for its principal office located in New York City. The Company expanded its principal office and is responsible for additional rent of approximately $225,000 annual base rent plus rent escalations, common area maintenance, insurance, and real estate taxes, under a lease agreement expiring in September 2016. Research Agreement Effective October 1, 2013, the Company signed a SRA with St. Jude. The Company is responsible for a total of $780,674 payable in four equal installments on October 19, 2013, March 19, 2014, September 19, 2014, and March 19, 2015. Unless otherwise terminated by operation of law or by acts of the parties in accordance with the terms of the agreement, the SRA shall be in full force and effect for a period of two (2) years and shall expire on October 1, 2015. The term may be extended by written agreement between the parties. Settlement Payments In October 2013, the Company paid $1,655,000 in cash and 5,000 shares of common stock valued at $36,400 to settle agreements on behalf of a related party. Upon payment, the Company and the related party entered into promissory notes whereby the related party agreed to pay the Company the principal amount of $1,691,400 plus interest at an annualized rate of 5% as reimbursement of payments that the Company made to settle a portion of the agreements. The Company applied the accounting guidance provided in ASU 2013-04. The guidance in this update is effective for fiscal years beginning after December 15, 2013 with early adoption permitted. The guidance in this update requires companies to measure obligations resulting from joint and several liability arrangements as the sum of the amount that the entity has a) contractually agreed to pay, and b) any additional amounts that the entity expects to pay on behalf of its co-obligors. The Company has recorded the full amount of the settlements as a charge to its operations due to uncertainty as to whether the related party will have sufficient liquidity to repay the Company or fund the indemnification agreements should it become necessary. Any amounts that the Company may recover under the note due from the related party or under the terms of the indemnification agreement, if in fact any amounts are recovered at all, would be characterized as a capital contribution at the date such payments are received. On September 18, 2013, the Company made a proposal to the board of directors of Transcept Pharmaceuticals, Inc. (“Transcept”) to acquire all of the outstanding shares of Transcept’s common stock for $4.00 per share in cash. Although Transcept’s board of directors rejected the Company’s proposal, the Company informed Transcept that it will be willing to continue to discuss the proposal. If Transcept accepts the Company’s proposal to acquire all of its outstanding shares of common stock, the Company will need to obtain additional equity or debt financing to consummate the acquisition and consolidation (see Note 3).

SUMMARY OF SIGNIFICANT ACCOUN24

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies)	9 Months Ended
	Sep. 30, 2013
Summary Of Significant Accounting Policies
Principles of Consolidation	Principles of Consolidation The unaudited condensed consolidated financial statements represent the consolidation of the accounts of the Company and its subsidiary in conformity with U.S. GAAP. All intercompany accounts and transactions have been eliminated in consolidation.
Cash	Cash For purposes of the statement of cash flows, the Company considers cash instruments with maturities of less than three months when purchased to be cash equivalents. There are no cash equivalents as of the balance sheet dates.
Marketable Securities	Marketable Securities The Company accounts for marketable securities held as “available-for-sale” pursuant to ASC 320 Investments — Debt and Equity Securities (“ASC 320”). The Company classifies these investments as current assets and carry them at fair value. Unrealized gains and losses are recorded as a separate component of stockholders’ equity as accumulated other comprehensive income. Realized gains or losses on marketable security transactions are reported in earnings and computed using the specific identification of cost basis. Marketable securities are maintained at one financial institution and are governed by the Company’s investment policy as approved by our Board of Directors. Fair values of marketable securities are based on quoted market prices. Valuation of marketable securities are further describe in Note 6. The Company’s current investment policy generally limit security investments for purposes of strategic acquisitions. Based on the liquidity position of the Company, the CEO and CFO are authorized to make various investment transaction decisions for prudent investment of the Company’s excess funds. The ability to conduct investments is limited to the CEO and CFO. The current policy limit marketable securities investments with a maturity, credit quality, and concentration that is authorized only by the CEO and CFO.
Employee Stock-Based Compensation	Employee Stock-Based Compensation The Company accounts for stock-based compensation in accordance with ASC 718 Compensation — Stock Compensation (“ASC 718”). ASC 718 addresses all forms of share-based payment (“SBP”) awards including shares issued under employee stock purchase plans and stock incentive shares. Under ASC 718 awards result in a cost that is measured at fair value on the awards’ grant date, based on the estimated number of awards that are expected to vest and will result in a charge to operations.
Non-Employee Stock-Based Compensation	Non-Employee Stock-Based Compensation The Company accounts for equity instruments issued to non-employees in accordance with the provisions of ASC 505, “Equity Based Payments to Non-Employees”, (“ASC 505”) and ASC 718 which requires that such equity instruments are recorded at their fair value on the measurement date. The measurement of stock-based compensation is subject to periodic adjustment as the underlying equity instruments vest. Non-employee stock-based compensation charges are being amortized over their respective contractual vesting periods.
Use of Estimates	Use of Estimates In preparing financial statements in conformity with U.S. GAAP, management is required to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported amounts of expenses during the reporting period. Due to inherent uncertainty involved in making estimates, actual results reported in future periods may be affected by changes in these estimates. On an ongoing basis, the Company evaluates its estimates and assumptions. These estimates and assumptions include valuing equity securities in share-based payments, estimating fair value of equity instruments recorded as derivative liabilities, estimating the useful lives of depreciable and amortizable assets and estimating the fair value of long-lived assets to assets whether impairment charges may apply.
Research and Development Costs	Research and Development Costs Research and development costs are charged to operations as incurred and consist primarily of consulting costs, contract research and development costs, and compensation costs. For the three and nine months ended September 30, 2013 and 2012, and for the period from March 11, 2011 (inception) through September 30, 2013, the Company recognized $1,399,875, $110,656, $2,113,813, $286,889, and $3,008,326, respectively, of research and development costs.
Patents	Patents The Company capitalized external cost, such as filing fees and associated attorney fees, incurred to obtain issued patents and patent applications pending. The Company expense cost associated with maintaining and defending patents subsequent to their issuance in the period incurred. The Company amortizes patent cost once issued on a straight-line basis over the estimate useful lives of the patents. The Company assess the potential impairment to all capitalized patent cost when events or changes in circumstances indicate that the carrying amount of our patent may not be recoverable. The Company accounts for patent costs in accordance with ASC Topic 350, “Goodwill and Other Intangible Assets” (“ASC 350”) and ASC Topic 805, “Business Combinations” (“ASC 805”).
Basic and diluted Net Loss Per Share	Basic and diluted Net Loss Per Share Basic and diluted net loss per share has been computed by dividing net loss by the weighted average number of common shares outstanding during the period. All potentially dilutive common shares have been excluded since their inclusion would be anti-dilutive. An aggregate of 4,462,426 and 0 warrants were excluded from the computation of diluted net loss per common share for the three and nine months ended September 30, 2013 and 2012, respectively, because their inclusion would have an anti-dilutive effect for the periods presented. An aggregate of 210,000 and 0 stock options were excluded from the computation of diluted net loss per common share for the three and nine months ended September 30, 2013 and 2012, respectively, because they would have an anti-dilutive effect for the periods presented. An aggregate of 211,073 and 927,310 incentive shares were excluded from the computation of diluted net loss per common share for the three and nine months ended September 30, 2013 and 2012, respectively, because they were contingent shares subject to recall.
Derivative Instruments	Derivative Instruments The Company does not use derivative instruments to hedge exposures to cash flow, market or foreign currency risks. The Company evaluates all of its financial instruments to determine if such instruments are derivatives or contain features that qualify as embedded derivatives. For derivative financial instruments that are accounted for as liabilities, the derivative instrument is initially recorded at its fair value and is then revalued at each reporting date, with changes in the fair value reported in the statements of operations. For stock-based derivative financial instruments, the Company calculates the fair value of the financial instruments using a probability-weighted Black-Scholes option pricing model, which is comparable to the Binomial Lattice options pricing model at inception and on each subsequent valuation date. The classification of derivative instruments, including whether such instruments should be recorded as liabilities or as equity, is evaluated at the end of each reporting period (see Note 7 and Note 8).
Joint and Several Liability Assessment	Joint and Several Liability Assessment The Company measures obligations resulting from joint and several liability arrangements as the sum of the amount that the Company has a) contractually agreed to pay, and b) any additional amounts that the Company expects to pay on behalf of its co-obligors.
Financial Instruments and Fair Value	Financial Instruments and Fair Value ASC Topic 820, “Fair Value Measurements and Disclosures,” (“ASC Topic 820”) establishes a fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. The hierarchy gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1 measurements) and the lowest priority to unobservable inputs (Level 3 measurements). The three levels of the fair value hierarchy under ASC Topic 820 are described below: L evel 1 Level 2 Level 3 In estimating the fair value of the Company’s marketable securities available-for-sale, the Company used quoted prices in active markets (see Note 6 and Note 8). In estimating the fair value of the Company’s derivative liabilities, the Company used a probability-weighted Black-Scholes option pricing model (see Note 7 and Note 8). Financial assets with carrying values approximating fair value include cash as well as marketable securities, deposits on license agreements, prepaid expenses and other current assets. Financial liabilities with carrying values approximating fair value include accounts payable and accrued expenses.
Registration Payment Arrangement	Registration Payment Arrangement The Company accounted for registration rights agreements in accordance with ASC 825-20, “Registration Payment Arrangements.” ASC 825-20 addresses an issuer’s accounting for registration payment arrangements. This pronouncement specifies that the contingent obligation to make future payments or otherwise transfer consideration under a registration payment arrangement, whether issued as a separate agreement or included as a provision of a financial instrument, should be separately recognized and accounted for as a contingency in accordance with ASC 450-20 “Loss Contingencies”.
Reclassifications	Reclassifications Certain prior year financial statement balances have been reclassified to conform to the current year presentation. These reclassifications had no effect on the recorded net loss.
Subsequent Events	Subsequent Events The Company follows the provisions of ASC Topic 855-10, “Subsequent Events,” relating to subsequent events. This guidance establishes principles and requirements for subsequent events. This guidance defines the period after the balance sheet date during which events or transactions that may occur would be required to be disclosed in a company’s financial statements. The Company has evaluated subsequent events up to the date of issuance of this report.

RESTATEMENT OF PREVIOUSLY ISS25

RESTATEMENT OF PREVIOUSLY ISSUED FINANCIAL STATEMENTS (Tables)	9 Months Ended
	Sep. 30, 2013
Restatements Of Previously Issued Consolidated Financial Statements Abstract [Abstract]
Schedule of effects of restatement on affected items within previously reported Consolidated Balance Sheets	The following table sets forth the effects (in thousands) of the matters identified by the Oversight Committee on affected items within the Company’s previously reported Condensed Consolidated Balance Sheet for the quarter ended September 30, 2013, had the adjustments been made: September 30, 2013 As Revised* As Restated Current liabilities $ 28,788 $ 30,943 Additional paid in Capital $ 47,498 $ 46,220 The following table (in thousands) sets forth the effects of revisions we made to our previously reported Condensed Consolidated Balance Sheet as of September 30, 2013 with respect to the errors we identified during the fourth quarter of 2013: September 30, 2013 As Reported As Revised Derivative financial instruments, warrants $ 22,234 $ 23,853 Total liabilities 27,169 28,788 Additional paid in Capital 48,650 47,498 Deficit accumulated during the development stage (54,301 ) (54,768 ) Stockholders’ deficit $ (5,804 ) $ (7,423 )
Schedule of effects of restatement on affected items within previously reported Consolidated Statement of Operations	The following table sets forth the effects (in thousands) of the matters identified by the Company during the fourth quarter of 2013 and the Oversight Committee in January 2014 on affected items within the Company’s previously reported Condensed Consolidated Statement of Operations for the three months and nine months ended September 30, 2013, had the adjustments been made in the corresponding quarters: Three months ended September 30, 2013 Nine months ended September 30, 2013 As Revised* As Restated As Revised* As Restated Selling, general and administrative $ 3,755 $ 4,631 $ 10,141 $ 11,017 Operating loss $ (5,155 ) $ (6,031 ) $ (12,255 ) $ (13,131 ) Net loss $ (11,135 ) $ (12,011 ) $ (21,156 ) $ (22,033 ) Net loss per share, basic and diluted $ (0.72 ) $ (0.78 ) $ (1.65 ) $ (1.72 ) *For matters identified during the fourth quarter of 2013 described in (2) below. (2) As previously discussed in the Form 10-K for the year ended 2013, in the fourth quarter of 2013, the Company discovered that certain warrants issued to a placement agent in connection with our February Private Placement were not recorded and certain expenses were misstated in our condensed consolidated financial statements for the first, second and third quarters of 2013. The misstatement of expenses include costs related to the February 14, 2013 Private Placement Offering, certain accruals, consulting fees and changes in the fair value of the unrecorded warrants, which required liability classification. The financial statements included herein have been revised to include the effects of these matters. The following table (in thousands) sets forth the effects of revisions we made to our previously reported Condensed Consolidated Statement of Operations for the three and nine months ended September 30, 2013, with respect to the errors we identified during the fourth quarter of 2013: Three months ended September 30, Nine months ended September 30, As Reported As Revised As Reported As Revised Selling, general and administrative $ 3,755 $ 3,755 $ 10,391 $ 10,141 Operating loss (5,155 ) (5,155 ) (12,505 ) (12,255 ) Change in fair value of derivative financial instruments-warrants (5,803 ) (6,040 ) (8,199 ) (8,916 ) Net loss (10,898 ) (11,135 ) (20,689 ) (21,156 ) Net loss per share, basic and diluted $ (0.71 ) $ (0.72 ) $ (1.62 ) $ (1.65 )

MARKETABLE SECURITIES (Tables)

MARKETABLE SECURITIES (Tables)	9 Months Ended
	Sep. 30, 2013
Marketable Securities Tables
Schedule of marketable securities	Unrealized Unrealized Estimated Fair Cost Gains Losses Value Marketable securities available-for-sale: $ 3,112,210 $ - $ 154,834 $ 2,957,376

DERIVATIVE FINANCIAL INSTRUME27

DERIVATIVE FINANCIAL INSTRUMENTS (Tables)	9 Months Ended
	Sep. 30, 2013
Investments, All Other Investments [Abstract]
Schedule of assumptions for valuation of warrants	As of Date of issuance Date of issuance Date of issuance September 30, Fair market price of common stock $3.75 $4.50 $4.50 $6.50 Contractual term 5 years 5 years 5 years 4.38 – 4.87 years Risk-free interest rate 0.86% 1.48% 1.48% 1.41% Expected volatility 101% 106% 106% 96%-102%

FAIR VALUE MEASUREMENTS (Tables

FAIR VALUE MEASUREMENTS (Tables)	9 Months Ended
	Sep. 30, 2013
Fair Value Disclosures [Abstract]
Schedule of fair value on a recurring basis	Fair Value Measurements at September 30, 2013 Significant other Significant Total carrying value at Quoted prices in active observable inputs unobservable inputs September 30, 2013 markets (Level 1) (Level 2) (Level 3) Asset: Marketable securities, available-for-sale $ 2,957,376 $ 2,957,376 $ - $ - Liability: Derivative liability related to warrants $ 23,853,325 $ - $ - $ 23,853,325 Settlement agreement $ 2,154,750 $ 2,154,750 $ - $
Schedule of summary of changes in the estimated fair value of the Company's Level 3 liability	Fair Value Measurements of Common Stock Warrants Using Significant Unobservable Inputs (Level 3) Balance at January 1, 2013 $ - Issuance of common stock warrants: February 14, 2013 5,407,605 August 14, 2013 328,561 August 15, 2013 9,201,487 Total value upon issuance 14,937,653 Change in fair value of common stock warrant liability 8,915,672 Balance at September 30, 2013 $ 23,853,325

INCENTIVE SHARES (Tables)

INCENTIVE SHARES (Tables)	9 Months Ended
	Sep. 30, 2013
Incentive Shares Tables
Schedule of incentive shares	Employee- Non- Total Weighted Average Unvested December 31, 2011 1,281,225 321,165 1,602,390 $ 4.00 Granted 866,180 87,503 953,683 12.89 Vested (2,048,280 ) (193,672 ) (2,241,952 ) 7.34 Forfeited (46,353 ) - (46,353 ) 9.06 Unvested December 31, 2012 52,772 214,996 267,768 5.79 Granted 135,000 - 135,000 6.24 Vested (17,918 ) (115,445 ) (133,363 ) 5.05 Forfeited (20,833 ) (37,500 ) (58,333 ) 4.00 Unvested September 30, 2013 149,021 62,051 211,072 $ 6.43

LIQUIDITY, FINANCIAL CONDITIO30

LIQUIDITY, FINANCIAL CONDITION AND MANAGEMENT PLANS (Detail Textuals) - USD ($)	Aug. 15, 2013	Aug. 14, 2013	Jan. 31, 2013	Sep. 30, 2013		Sep. 30, 2012	Sep. 30, 2013		Sep. 30, 2012	Dec. 31, 2011	Dec. 31, 2012	Sep. 30, 2013
Liquidity And Financial Condition And Managements Plans Details Narrative
Net Loss				$ (12,011,174)	[1]	$ (8,480,457)	$ (22,032,609)	[1]	$ (16,812,669)	$ (3,268,256)	$ (30,343,856)	$ (55,644,721)	[1]
Stock-based compensation (Approximately)												26,000,000
Cash				13,409,825	[1]		13,409,825	[1]			11,388	13,409,825	[1]
Working Capital deficit (Approximately)				14,100,000			14,100,000					14,100,000
Retained Earnings (Accumulated Deficit)				(55,644,721)	[1]		(55,644,721)	[1]			$ (33,612,112)	(55,644,721)	[1]
Derivative financial instruments, at estimated fair value - warrants				$ 23,853,325	[1]		$ 23,853,325	[1]				$ 23,853,325	[1]
Private Placement
Subsidiary, Sale of Stock [Line Items]
Sale of Stock, Description of Transaction		(i) a waiver of any and all liquidated damages that the Company incurred for its inability to cause the a registration statement to be declared effective within certain contractually defined time-frames stipulated in the original agreement; (ii) a commitment on the part of the investors in the February private placement to participate in a private placement transaction that the Company completed on August 15, 2013, and (iii) a covenant on the part of the Company to proceed with the sale of shares that were issued under the August 15, 2013 private placement transaction. In exchange, the Company paid an aggregate fee to these investors of $2,495,256 consisting of (i) 73,710 shares of the Company’s common stock with an aggregate fair value of $331,695 (based on the selling price of $4.50 per share in the August financing transaction); (ii) cash in the amount of $1,835,000; and (iii) warrants to purchase 98,756 shares of common stock with a fair value of $328,561.
Aggregate fee paid to investors		$ 2,495,256
Number of shares issued in transaction	5,531,401	73,710
Common stock with an aggregate fair value		$ 331,695
Selling price per share	$ 4.50	$ 4.50
Cash paid on Transaction		$ 1,835,000
Number of common stock called by warrants	2,765,701	98,756
Fair value of warrants	$ 24,891,303	$ 328,561
Remaining fee settled in cash		$ 946,196
Number of shares issue for remaining fee settled		197,512	103,425
Shares issued to investors for inducement to participate in financing		20,685
Fair value of shares, per share		$ 5
[1]	(As Restated)

LIQUIDITY AND FINANCIAL CONDITI

LIQUIDITY AND FINANCIAL CONDITION AND MANAGEMENT'S PLANS (Detail Textuals 1) - USD ($)	Oct. 01, 2013	Sep. 20, 2013	Sep. 18, 2013	Aug. 29, 2013	Aug. 16, 2013	Sep. 30, 2013	Jun. 30, 2013	Mar. 31, 2013
Related Party Transaction [Line Items]
Interest rate							5.00%
Payment for additional settlement agreement				$ 300,000
Company raised capital from issuance of private placement						$ 24,920,000		$ 9,950,000
Transcept
Related Party Transaction [Line Items]
Business acquisition, share price			$ 4
Percentage of voting interests acquired			4.96%
Payments to acquire businesses			$ 3,000,000
Subsequent event | Research Agreement
Related Party Transaction [Line Items]
Effective period of SRA	2 years
Pharmaceutical company
Related Party Transaction [Line Items]
Non-refundable upfront fee for license					$ 2,000,000
Period For License Agreement					120 days
Individual
Related Party Transaction [Line Items]
Non-refundable upfront fee for license		$ 250,000
Chief Executive Officer
Related Party Transaction [Line Items]
Secured promissory note issued to related party							$ 2,284,511
Cash or non cash consideration paid to settle portion of agreement							$ 593,111
Interest rate							5.00%
Outstanding liabilities on settlement payable							$ 2,284,511
Number of share due to cause to be delivered							47,128
Chief Executive Officer | Second payment
Related Party Transaction [Line Items]
Outstanding liabilities on settlement payable							$ 1,691,400

SUMMARY OF SIGNIFICANT ACCOUN32

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Detail Textuals) - USD ($)	3 Months Ended			9 Months Ended			31 Months Ended
	Sep. 30, 2013		Sep. 30, 2012	Sep. 30, 2013		Sep. 30, 2012	Sep. 30, 2013	[1]
Summary Of Significant Accounting Policies Details Narrative
Research and development costs	$ 1,399,875	[1]	$ 110,656	$ 2,113,813	[1]	$ 286,889	$ 3,008,326
Warrants
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Computation of diluted net loss per common share	4,462,426		0	4,462,426		0
Stock options
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Computation of diluted net loss per common share	210,000		0	210,000		0
Incentive shares
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Computation of diluted net loss per common share	211,073		927,310	211,073		927,310
[1]	(As Restated)

RESTATEMENT OF PREVIOUSLY ISS33

RESTATEMENT OF PREVIOUSLY ISSUED FINANCIAL STATEMENTS (Details) - USD ($)		Sep. 30, 2013		Dec. 31, 2012	Dec. 31, 2011
Error Corrections and Prior Period Adjustments Restatement [Line Items]
Current liabilities		$ 30,942,834	[1]	$ 5,799,080
Additional paid in Capital		46,219,593	[1]	30,203,402
Deficit accumulated during the development stage		(55,644,721)	[1]	(33,612,112)
Stockholders' deficit		(9,578,124)	[1]	$ (3,407,815)	$ (536,285)
As Revised
Error Corrections and Prior Period Adjustments Restatement [Line Items]
Derivative financial instruments, warrants		23,853,000
Total liabilities		28,788,000
Current liabilities	[2]	28,788,000
Additional paid in Capital	[2]	47,498,000
Deficit accumulated during the development stage		(54,768,000)
Stockholders' deficit		(7,423,000)
As Restated
Error Corrections and Prior Period Adjustments Restatement [Line Items]
Current liabilities		30,943,000
Additional paid in Capital		46,220,000
As Reported
Error Corrections and Prior Period Adjustments Restatement [Line Items]
Derivative financial instruments, warrants		22,234,000
Total liabilities		27,169,000
Additional paid in Capital		48,650,000
Deficit accumulated during the development stage		(54,301,000)
Stockholders' deficit		$ (5,804,000)
[1]	(As Restated)
[2]	For matters identified during the fourth quarter of 2013 described in (2) below.

RESTATEMENT OF PREVIOUSLY ISS34

RESTATEMENT OF PREVIOUSLY ISSUED FINANCIAL STATEMENTS (Details 1) - USD ($)		3 Months Ended			9 Months Ended			10 Months Ended	12 Months Ended	31 Months Ended
		Sep. 30, 2013		Sep. 30, 2012	Sep. 30, 2013		Sep. 30, 2012	Dec. 31, 2011	Dec. 31, 2012	Sep. 30, 2013
Error Corrections and Prior Period Adjustments Restatement [Line Items]
Selling, general and administrative		$ 2,966,816	[1]	$ 113,140	$ 6,035,941	[1]	$ 337,622			$ 7,392,049	[1]
Operating loss		(6,030,861)	[1]	(8,459,745)	(13,131,247)	[1]	(16,757,891)			(46,651,960)	[1]
Change in fair value of common stock warrant liability	[1]	(6,040,054)			(8,915,672)					(8,915,672)
Net Loss		$ (12,011,174)	[1]	$ (8,480,457)	$ (22,032,609)	[1]	$ (16,812,669)	$ (3,268,256)	$ (30,343,856)	$ (55,644,721)	[1]
Net loss per common share - basic and diluted		$ (0.78)	[1]	$ (2.42)	$ (1.72)	[1]	$ (5.55)
As Revised
Error Corrections and Prior Period Adjustments Restatement [Line Items]
Selling, general and administrative	[2]	$ 3,755,000			$ 10,141,000
Operating loss	[2]	(5,155,000)			(12,255,000)
Change in fair value of common stock warrant liability		(6,040,054)			8,916,000
Net Loss	[2]	$ (11,135,000)			$ (21,156,000)
Net loss per common share - basic and diluted	[2]	$ (0.72)			$ (1.65)
As Restated
Error Corrections and Prior Period Adjustments Restatement [Line Items]
Selling, general and administrative		$ 4,631,000			$ 11,017,000
Operating loss		(6,031,000)			(13,131,000)
Net Loss		$ (12,011,000)			$ (22,033,000)
Net loss per common share - basic and diluted		$ (0.78)			$ (1.72)
As Reported
Error Corrections and Prior Period Adjustments Restatement [Line Items]
Selling, general and administrative		$ 3,755,000			$ 10,391,000
Operating loss		(5,155,000)			(12,505,000)
Change in fair value of common stock warrant liability		(5,803,000)			(8,199,000)
Net Loss		$ (10,898,000)			$ (20,689,000)
Net loss per common share - basic and diluted		$ (0.71)			$ (1.62)
[1]	(As Restated)
[2]	For matters identified during the fourth quarter of 2013 described in (2) below.

RESTATEMENT OF PREVIOUSLY ISS35

RESTATEMENT OF PREVIOUSLY ISSUED FINANCIAL STATEMENTS (Detail Textuals) - USD ($)	9 Months Ended			31 Months Ended
	Sep. 30, 2013		Sep. 30, 2012	Sep. 30, 2013		Dec. 31, 2012
Related Party Transaction [Line Items]
Cash payments	$ 13,200	[1]	$ 30,000	$ 46,500	[1]
Deficit accumulated during the development stage	55,644,721	[1]		55,644,721	[1]	$ 33,612,112
As Reported
Related Party Transaction [Line Items]
Deficit accumulated during the development stage	$ 54,301,000			$ 54,301,000
Mr. Shkreli
Related Party Transaction [Line Items]
Issuance of common shares, Shares	331,500
Cash payments	$ 135,000
[1]	(As Restated)

MARKETABLE SECURITIES (Details)

MARKETABLE SECURITIES (Details) - Marketable securities available-for-sale	Sep. 30, 2013USD ($)
Cost	$ 3,112,210
Unrealized Gains
Unrealized Losses	$ 154,834
Estimated Fair value	$ 2,957,376

DERIVATIVE FINANCIAL INSTRUME37

DERIVATIVE FINANCIAL INSTRUMENTS (Details) - 9 months ended Sep. 30, 2013 - $ / shares	Total
Fair market price of common stock	$ 6.50
Expected option term	4.38 - 4.87 years
Risk-free interest rate	1.41%
Expected volatility	96%-102
Date of issuance February 14, 2013
Fair market price of common stock	$ 3.75
Expected option term	5 years
Risk-free interest rate	0.86%
Expected volatility	101.00%
Date of issuance August 14, 2013
Fair market price of common stock	$ 4.50
Expected option term	5 years
Risk-free interest rate	1.48%
Expected volatility	106.00%
Date of issuance August 15, 2013
Fair market price of common stock	$ 4.50
Expected option term	5 years
Risk-free interest rate	1.48%
Expected volatility	106.00%

DERIVATIVE FINANCIAL INSTRUME38

DERIVATIVE FINANCIAL INSTRUMENTS (Detail Textuals) - Sep. 30, 2013 - USD ($)		Total	Total	Total
Investments, All Other Investments [Abstract]
Change in fair value of common stock warrant liability	[1]	$ (6,040,054)	$ (8,915,672)	$ (8,915,672)
[1]	(As Restated)

FAIR VALUE MEASUREMENTS (Detail

FAIR VALUE MEASUREMENTS (Details) - USD ($)		Sep. 30, 2013		Dec. 31, 2012
Marketable securities, available-for-sale	[1]	$ 2,957,376
Derivative liability related to warrants		23,853,325	[1]
Settlement agreement		2,154,750
Quoted prices in active markets (Level 1)
Marketable securities, available-for-sale		$ 2,957,376
Derivative liability related to warrants
Settlement agreement		$ 2,154,750
Significant other observable inputs (Level 2)
Marketable securities, available-for-sale
Derivative liability related to warrants
Settlement agreement
Significant unobservable inputs (Level 3)
Marketable securities, available-for-sale
Derivative liability related to warrants		$ 23,853,325
[1]	(As Restated)

FAIR VALUE MEASUREMENTS (Deta40

FAIR VALUE MEASUREMENTS (Details 1) - Sep. 30, 2013 - USD ($)		Total	Total	Total
Fair Value Measurements Details 2
Balance at January 1, 2013
February 14, 2013			$ 5,407,605
August 14, 2013			328,561
August 15, 2013			9,201,487
Total value upon issuance			14,937,653
Change in fair value of common stock warrant liability	[1]	$ 6,040,054	8,915,672	$ 8,915,672
Balance at September 30, 2013	[1]	$ 23,853,325	$ 23,853,325	$ 23,853,325
[1]	(As Restated)

LICENSE AGREEMENT (Detail Textu

LICENSE AGREEMENT (Detail Textuals) - USD ($)	Jan. 07, 2013	Feb. 28, 2013	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013	Sep. 30, 2012	Dec. 31, 2012	Sep. 30, 2013
License Agreement [Line Items]
Value of issued common shares							$ 1,550,000
Common Stock
License Agreement [Line Items]
Common shares issued to Ligand							620,000
Value of issued common shares							$ 62
License Agreement
License Agreement [Line Items]
Non-refundable payments	$ 2,550,000
Extension fee expensed to operations		$ 250,000
License agreement			$ 2,300,000		$ 2,300,000			$ 2,300,000
Amortization expense of the license			$ 51,065	$ 34,885	$ 151,531	$ 71,466		$ 272,914
License Agreement | First payment
License Agreement [Line Items]
Non-refundable payments	$ 1,150,000
License Agreement | Second payment
License Agreement [Line Items]
Non-refundable payments		1,400,000
Fee payable to sublicense		$ 250,000

NOTES PAYABLE (Detail Textuals)

NOTES PAYABLE (Detail Textuals) - USD ($)	3 Months Ended			9 Months Ended			31 Months Ended
	Sep. 30, 2013	[1]	Sep. 30, 2012	Sep. 30, 2013	[1]	Sep. 30, 2012	Sep. 30, 2013	[1]	Jun. 30, 2013	Feb. 01, 2012
Short-term Debt [Line Items]
Interest rate									5.00%
Interest expense			$ 26,761	$ 41,563		$ 70,559	$ 147,480
Secured promissory note
Short-term Debt [Line Items]
Secured promissory note issued to related party										$ 900,000
Interest rate										12.00%
[1]	(As Restated)

RELATED PARTY TRANSACTIONS (Det

RELATED PARTY TRANSACTIONS (Detail Textuals)	1 Months Ended		3 Months Ended	9 Months Ended			31 Months Ended
	Oct. 31, 2013USD ($)shares	Aug. 31, 2012USD ($)ft²	Jun. 30, 2013USD ($)shares	Sep. 30, 2013USD ($)		Sep. 30, 2012USD ($)	Sep. 30, 2013USD ($)		Dec. 31, 2012USD ($)	Dec. 08, 2011USD ($)
Related Party Transaction [Line Items]
Due to related parties				$ 10,000	[1]		$ 10,000	[1]	$ 23,200	$ 8,500
Payments made on behalf of affiliate		$ 137,547					137,547	[1]
Office space | ft²		4,216
Annual base rent		$ 275,000
Liabilities settled to series of agreements				$ 2,284,511			$ 2,284,511
Rate of reimbursement payments			5.00%
Subsequent event | Settlement Agreement
Related Party Transaction [Line Items]
Outstanding liabilities on settlement payable	$ 1,691,400
Rate of reimbursement payments	5.00%
Payment for settlement	$ 1,655,000
Issuance of common shares, Shares | shares	5,000
Issuance of common shares	$ 36,400
Chief Executive Officer
Related Party Transaction [Line Items]
Principal amount of promissory note			$ 2,284,511
Outstanding liabilities on settlement payable			2,284,511
Cash or non cash consideration paid to settle portion of agreement			$ 593,111
Number of share due to cause to be delivered | shares			47,128
Rate of reimbursement payments			5.00%
Chief Executive Officer | Second payment
Related Party Transaction [Line Items]
Outstanding liabilities on settlement payable			$ 1,691,400
MSMB Entities
Related Party Transaction [Line Items]
Other payments				$ 135,000
[1]	(As Restated)

RELATED PARTY TRANSACTIONS (D44

RELATED PARTY TRANSACTIONS (Detail Textuals 1) - USD ($) $ / shares in Units, $ in Thousands	Aug. 15, 2013	Aug. 14, 2013	Sep. 30, 2013	Mar. 31, 2013
Related Party Transaction [Line Items]
Company raised capital from issuance of private placement			$ 24,920	$ 9,950
Private Placement
Related Party Transaction [Line Items]
Number of shares issued in transaction	5,531,401	73,710
Purchase price of common stock issued	$ 4.50	$ 4.50
Number of common stock called by warrants	2,765,701	98,756
Exercise price of warrant	$ 6
Number of common stock purchase by management	10,522
Number of common stock called by warrants in private placement	5,261

STOCKHOLDERS' DEFICIT (Detail T

STOCKHOLDERS' DEFICIT (Detail Textuals) - USD ($)	Aug. 15, 2013	Aug. 14, 2013	Feb. 14, 2013	Sep. 30, 2013	Mar. 31, 2013	Sep. 30, 2013	Dec. 31, 2011	Aug. 31, 2013	Feb. 28, 2013	Jan. 31, 2013
Stockholders Equity [Line Items]
Issuance of common stock in connection with January 2013 private placement at $3.00 per share, net of fees of $0						$ 816,664
Issuance of common stock, per share amount (in dollars per share)								$ 4.50	$ 3	$ 3
Company raised capital from issuance of private placement				$ 24,920,000	$ 9,950,000
Private Placement
Stockholders Equity [Line Items]
Fair value of warrants	$ 24,891,303	$ 328,561
Purchase price of common stock issued	$ 4.50	$ 4.50
Number of common stock called by warrants	2,765,701	98,756
Exercise price of warrant	$ 6
Private Placement | Roth Capital Partners
Stockholders Equity [Line Items]
Issuance of common shares, Shares	5,531,401		3,045,929
Company raised capital from issuance of private placement	$ 24,891,303		$ 9,137,787
Purchase price of common stock issued	$ 4.50		$ 3
Number of common stock called by warrants	2,765,701		1,597,969
Exercise price of warrant	$ 6		$ 3.60
Derivative liability	$ 9,201,487		$ 4,505,605
Partial liquidated damages equal to aggregate purchase price			2.00%
Maximum liquidated damages equal to aggregate purchase price			10.00%
Interest rate on failure of partial liquidation damages			18.00%
Proceeds from registration payment arrangement liability			$ 360,000
Income upon waiver of liquidated damages			$ 360,000
Common Stock
Stockholders Equity [Line Items]
Issuance of common stock in connection with January 2013 private placement at $3.00 per share, net of fees of $0 (in shares)						272,221
Issuance of common stock in connection with January 2013 private placement at $3.00 per share, net of fees of $0						$ 27
Issuance of common stock, per share amount (in dollars per share)										$ 3
Issuance of common shares, Shares							1,608,300

STOCKHOLDERS' DEFICIT (Detail46

STOCKHOLDERS' DEFICIT (Detail Textuals 1) - USD ($)	Aug. 15, 2013	Aug. 14, 2013	Aug. 31, 2013	Jan. 31, 2013
Stockholders Equity [Line Items]
Issuance of common stock to investors, per share amount (in dollars per share)			$ 5
Private Placement
Stockholders Equity [Line Items]
Aggregate fee paid to investors		$ 2,495,256
Number of shares issued in transaction	5,531,401	73,710
Common stock with an aggregate fair value		$ 331,695
Purchase price of common stock issued	$ 4.50	$ 4.50
Cash or non cash consideration paid to settle portion of agreement		$ 1,835,000
Number of common stock called by warrants	2,765,701	98,756
Fair value of warrants	$ 24,891,303	$ 328,561
Remaining fee settled in cash		$ 946,196
Number of shares issue for remaining fee settled		197,512		103,425
Shares issued to investors for inducement to participate in financing		20,685
Issuance of common stock to investors, per share amount (in dollars per share)		$ 5
Waiver of original registration payment obligation		$ 360,000
Reduction of proceeds received in financing transaction		$ 2,238,681

STOCKHOLDERS' DEFICIT (Detail47

STOCKHOLDERS' DEFICIT (Detail Textuals 2) - USD ($)	Sep. 09, 2013	May. 13, 2013	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013
Stockholders Equity [Line Items]
Compensation expense related to options			$ 1,741,908	$ 7,027,675	$ 2,029,505	$ 14,014,402	$ 26,419,022
September 9, 2013
Stockholders Equity [Line Items]
Share-based payment award, vesting period	3 years
Compensation expense related to options			9,606		9,606
Risk-free interest rate	1.71%
Expected term	5 years 9 months 22 days
Expected volatility	104.78%
Exercise price equal to fair value of stock	$ 6.20
Unrecognized compensation expense			$ 438,748		438,748		438,748
Remaining amortization period of options			2 years 6 months
Intrinsic value of options			$ 0		0		0
Remaining contract life of Options			9 years 11 months 8 days
September 9, 2013 | Common Stock
Stockholders Equity [Line Items]
Number of options issued to purchase common stock	90,000
Horacio Plotkin Employment Agreement | May 13, 2013
Stockholders Equity [Line Items]
Share-based payment award, vesting period		3 years
Compensation expense related to options			$ 64,716		101,399
Risk-free interest rate		0.83%
Expected term		5 years 9 months 22 days
Expected volatility		98.56%
Exercise price equal to fair value of stock		$ 8.70
Unrecognized compensation expense			$ 703,333		703,333		703,333
Remaining amortization period of options			2 years 6 months
Intrinsic value of options			$ 0		$ 0		$ 0
Remaining contract life of Options			9 years 7 months 13 days
Horacio Plotkin Employment Agreement | May 13, 2013 | Common Stock
Stockholders Equity [Line Items]
Number of options issued to purchase common stock		120,000

INCENTIVE SHARES (Details)

INCENTIVE SHARES (Details) - $ / shares	9 Months Ended	12 Months Ended
	Sep. 30, 2013	Dec. 31, 2012	Dec. 31, 2011
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unvested	267,768	1,602,390
Granted	135,000	953,683
Vested	(133,363)	(2,241,952)
Forfeited	(58,333)	(46,353)
Unvested	211,072	267,768	1,602,390
Weighted Average Fair Value, Unvested	$ 5.79	$ 4
Weighted Average Fair Value Granted	6.24	12.89
Weighted Average Fair Value, Vested	5.05	7.34
Weighted Average Fair Value, Forfeited	4	9.06
Weighted Average Fair Value, Unvested	$ 6.43	$ 5.79	$ 4
Employee
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unvested	52,772	1,281,225
Granted	135,000	866,180
Vested	(17,918)	(2,048,280)
Forfeited	(20,833)	(46,353)
Unvested	149,021	52,772	1,281,225
Non Employee
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unvested	214,996	321,165
Granted		87,503
Vested	(115,445)	(193,672)
Forfeited	(37,500)
Unvested	62,051	214,996	321,165

INCENTIVE SHARES (Detail Textua

INCENTIVE SHARES (Detail Textuals) - USD ($)	1 Months Ended		3 Months Ended		9 Months Ended		12 Months Ended	31 Months Ended
	Jul. 31, 2013	May. 20, 2013	Sep. 30, 2013	Sep. 30, 2012	Sep. 30, 2013	Sep. 30, 2012	Dec. 31, 2012	Sep. 30, 2013
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Compensation expense related to options			$ 1,741,908	$ 7,027,675	$ 2,029,505	$ 14,014,402		$ 26,419,022
Number of options granted					135,000		953,683
Unrecognized compensation cost related to incentive shares			$ 1,294,232		$ 1,294,232			$ 1,294,232
Expected to be recognized over a weighted average			2 years 3 months
Restricted Stock
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of options granted	15,000
Share-based payment award, vesting period	3 years
Restricted Stock | Marc L. Panoff | Panoff employment agreement
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of options granted		120,000
Share-based payment award, vesting period		3 years

COMMITMENTS AND CONTINGENCIES (

COMMITMENTS AND CONTINGENCIES (Detail Textuals) - USD ($)	Oct. 08, 2013	Oct. 01, 2013	Sep. 20, 2013	Aug. 16, 2013	Aug. 31, 2012	Sep. 30, 2013	Sep. 30, 2013
Loss Contingencies [Line Items]
Annual base rent					$ 275,000
Payment of non-refundable upfront fee			$ 250,000	$ 2,000,000
Lease Agreement | Massachusetts | Subsequent event
Loss Contingencies [Line Items]
Annual base rent		$ 216,000
Lease Agreement | New York
Loss Contingencies [Line Items]
Annual base rent							$ 275,000
Lease Agreement | New York | Subsequent event
Loss Contingencies [Line Items]
Annual base rent	$ 225,000
Exclusivity Agreements
Loss Contingencies [Line Items]
Payments for exclusive deposits						$ 2,250,000	$ 2,250,000

SUBSEQUENT EVENTS (Detail Textu

SUBSEQUENT EVENTS (Detail Textuals)	Oct. 08, 2013USD ($)	Oct. 01, 2013USD ($)Square_FeetInstallment	Oct. 31, 2013USD ($)shares	Aug. 31, 2012USD ($)Square_Feet	Sep. 30, 2013USD ($)	Sep. 18, 2013$ / shares	Jun. 30, 2013
Subsequent Event [Line Items]
Office space | Square_Feet				4,216
Annual base rent				$ 275,000
Rate of reimbursement payments							5.00%
Lease Agreement | New York
Subsequent Event [Line Items]
Annual base rent					$ 275,000
Subsequent event | Lease Agreement | Massachusetts
Subsequent Event [Line Items]
Office space | Square_Feet		4,232
Annual base rent		$ 216,000
Subsequent event | Lease Agreement | New York
Subsequent Event [Line Items]
Annual base rent	$ 225,000
Subsequent event | Research Agreement
Subsequent Event [Line Items]
Payment of research agreement		$ 780,674
Number of installments | Installment		4
Effective period of SRA		2 years
Subsequent event | Settlement Agreement
Subsequent Event [Line Items]
Payment for settlement			$ 1,655,000
Issuance of common shares, Shares | shares			5,000
Issuance of common shares			$ 36,400
Rate of reimbursement payments			5.00%
Outstanding liabilities on settlement payable			$ 1,691,400
Subsequent event | Settlement Agreement | Transcept Pharmaceuticals, Inc. ("Transcept")
Subsequent Event [Line Items]
Business acquisition, share price | $ / shares						$ 4