Zevra Therapeutics (ZVRA) 10-Q 14 Aug 23 2023 Q2 Quarterly report Financial data

Document And Entity Information

Document And Entity Information - shares	6 Months Ended
	Jun. 30, 2023	Aug. 11, 2023
Document Information [Line Items]
Entity Central Index Key	0001434647
Entity Registrant Name	ZEVRA THERAPEUTICS, INC.
Amendment Flag	false
Current Fiscal Year End Date	--12-31
Document Fiscal Period Focus	Q2
Document Fiscal Year Focus	2023
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Jun. 30, 2023
Document Transition Report	false
Entity File Number	001-36913
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	20-5894398
Entity Address, Address Line One	1180 Celebration Boulevard, Suite 103
Entity Address, City or Town	Celebration
Entity Address, State or Province	FL
Entity Address, Postal Zip Code	34747
City Area Code	321
Local Phone Number	939-3416
Title of 12(b) Security	Common Stock, $0.0001 par value per share
Trading Symbol	ZVRA
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Non-accelerated Filer
Entity Small Business	true
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		33,932,639

Unaudited Condensed Consolidate

Unaudited Condensed Consolidated Balance Sheets - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Assets
Cash and cash equivalents	$ 66,196	$ 65,466
Securities at fair value	20,696	16,900
Short-term investments - other	479	481
Accounts and other receivables	14,033	8,299
Prepaid expenses and other current assets	2,023	1,877
Total current assets	103,427	93,023
Inventories	546	671
Property and equipment, net	689	794
Operating lease right-of-use assets	803	988
Long-term investments - other	0	20,000
Other long-term assets	53	53
Total assets	105,518	115,529
Liabilities and stockholders' equity
Accounts payable and accrued expenses	10,510	6,169
Current portion of operating lease liabilities	456	480
Current portion of discount and rebate liabilities	6,965	4,655
Other current liabilities	321	422
Total current liabilities	18,252	11,726
Line of credit payable	12,709	12,800
Operating lease liabilities, less current portion	627	843
Discount and rebate liabilities, less current portion	5,114	4,327
Other long-term liabilities	317	26
Total liabilities	37,019	29,722
Commitments and contingencies (Note D)
Stockholders’ equity:
Common stock, $0.0001 par value, 250,000,000 shares authorized, 35,503,697 shares issued and 33,928,005 shares outstanding as of June 30, 2023; 35,450,257 shares issued and 34,540,304 shares outstanding as of December 31, 2022	3	3
Additional paid-in capital	405,127	401,799
Treasury stock, at cost	(10,983)	(7,536)
Accumulated deficit	(325,423)	(308,572)
Accumulated other comprehensive (loss) income	(225)	113
Total stockholders' equity	68,499	85,807
Total liabilities and stockholders' equity	105,518	115,529
Undesignated Preferred Stock [Member]
Stockholders’ equity:
Undesignated preferred stock, $0.0001 par value, 10,000,000 shares authorized, no shares issued or outstanding as of June 30, 2023 or December 31, 2022	$ 0	$ 0

Unaudited Condensed Consolida_2

Unaudited Condensed Consolidated Balance Sheets (Parentheticals) - $ / shares	Jun. 30, 2023	Dec. 31, 2022
Preferred stock, shares authorized (in shares)	10,000,000	10,000,000
Common stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Common stock, shares authorized (in shares)	250,000,000	250,000,000
Common stock, shares issued (in shares)	35,503,697	35,450,257
Common stock, shares outstanding (in shares)	33,928,005	34,540,304
Undesignated Preferred Stock [Member]
Preferred stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Preferred stock, shares authorized (in shares)	10,000,000	10,000,000
Preferred stock, shares issued (in shares)	0	0
Preferred stock, shares outstanding (in shares)	0	0

Unaudited Condensed Consolida_3

Unaudited Condensed Consolidated Statements of Operations - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Revenue, net	$ 8,470	$ 1,300	$ 11,349	$ 5,265
Operating expenses:
Cost of revenue	677	51	802	59
Research and development	7,433	4,795	16,277	7,877
Selling, general and administrative	7,005	3,558	13,839	6,292
Total operating expenses	15,115	26,067	30,918	31,891
Loss from operations	(6,645)	(24,767)	(19,569)	(26,626)
Other (expense) income:
Interest expense	(197)	(36)	(379)	(41)
Fair value adjustment related to derivative and warrant liability	0	32	0	273
Fair value adjustment related to investments	131	(352)	327	(495)
Interest and other income, net	1,553	366	2,593	264
Total other income	1,487	10	2,541	1
Loss before income taxes	(5,158)	(24,757)	(17,028)	(26,625)
Income tax benefit	74	715	177	719
Net loss	$ (5,084)	$ (24,042)	$ (16,851)	$ (25,906)
Basic and diluted net loss per share of common stock:
Net loss (in dollars per share)	$ (0.15)	$ (0.70)	$ (0.49)	$ (0.75)
Weighted average number of shares of common stock outstanding:
Basic and diluted (in shares)	33,898,233	34,447,206	34,180,818	34,476,737
In Process Research and Development [Member]
Operating expenses:
Acquired in-process research and development	$ 0	$ 17,663	$ 0	$ 17,663

Unaudited Condensed Consolida_4

Unaudited Condensed Consolidated Statements of Comprehensive Income (Loss) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Net loss	$ (5,084)	$ (24,042)	$ (16,851)	$ (25,906)
Other comprehensive loss:
Foreign currency translation adjustment	(162)	0	(338)	0
Other comprehensive loss:	(162)	0	(338)	0
Comprehensive loss	$ (5,246)	$ (24,042)	$ (17,189)	$ (25,906)

Unaudited Condensed Consolida_5

Unaudited Condensed Consolidated Statements of Changes in Stockholders' Equity - USD ($) $ in Thousands	Common Stock [Member]	Additional Paid-in Capital [Member]	Treasury Stock, Common [Member]	Retained Earnings [Member]	AOCI Attributable to Parent [Member]	Total
Balance at Dec. 31, 2021	$ 4	$ 396,957	$ (2,814)		$ (267,029)	$ 127,118
Net Income (loss)	0	0	0		(1,864)	(1,864)
Stock-based compensation expense	0	918	0		0	918
Shares repurchased as part of the Share Repurchase Program	(1)	0	(4,722)		0	(4,723)
Issuance of common stock in exchange for consulting services	0	50	0		0	50
Balance at Mar. 31, 2022	3	397,925	(7,536)		(268,893)	121,499
Balance at Dec. 31, 2021	4	396,957	(2,814)		(267,029)	127,118
Net Income (loss)						(25,906)
Other comprehensive loss						0
Balance at Jun. 30, 2022	3	399,701	(7,536)		(292,935)	99,233
Balance at Mar. 31, 2022	3	397,925	(7,536)		(268,893)	121,499
Net Income (loss)	0	0	0		(24,042)	(24,042)
Stock-based compensation expense	0	1,510	0		0	1,510
Issuance of common stock in exchange for consulting services	0	50	0		0	50
Other comprehensive loss						0
Issuance of common stock as part of the Employee Stock Purchase Plan	0	216	0		0	216
Balance at Jun. 30, 2022	3	399,701	(7,536)		(292,935)	99,233
Balance at Dec. 31, 2022	3	401,799	(7,536)	$ (308,572)	113	85,807
Net Income (loss)	0	0	0	(11,767)	0	(11,767)
Stock-based compensation expense	0	591	0	0	0	591
Shares repurchased as part of the Share Repurchase Program	0	0	(3,447)	0	0	(3,447)
Issuance of common stock in exchange for consulting services	0	42	0	0	0	42
Severance expense	0	354	0	0	0	354
Other comprehensive loss	0	0	0	0	(176)	(176)
Balance at Mar. 31, 2023	3	402,786	(10,983)	(320,339)	(63)	71,404
Balance at Dec. 31, 2022	3	401,799	(7,536)	(308,572)	113	85,807
Net Income (loss)						(16,851)
Other comprehensive loss						(338)
Balance at Jun. 30, 2023	3	405,127	(10,983)	(325,423)	(225)	68,499
Balance at Mar. 31, 2023	3	402,786	(10,983)	(320,339)	(63)	71,404
Net Income (loss)	0	0	0	(5,084)	0	(5,084)
Stock-based compensation expense	0	1,103	0	0	0	1,103
Issuance of common stock in exchange for consulting services	0	25	0	0	0	25
Severance expense	0	1,048	0	0	0	1,048
Other comprehensive loss	0	0	0	0	(162)	(162)
Issuance of common stock as part of the Employee Stock Purchase Plan	0	165	0	0	0	165
Balance at Jun. 30, 2023	$ 3	$ 405,127	$ (10,983)	$ (325,423)	$ (225)	$ 68,499

Unaudited Condensed Consolida_6

Unaudited Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Cash flows from operating activities:
Net loss	$ (16,851)	$ (25,906)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation expense	1,694	2,428
Severance expense	1,402	0
Depreciation and amortization expense	157	246
Fair value adjustment related to derivative and warrant liability	0	(273)
Fair value adjustment related to investments	(327)	495
Loss on sublease and disposal of property and equipment	0	9
Consulting fees paid in common stock	67	100
Gain on foreign currency exchange	(138)	0
Change in assets and liabilities:
Accounts and other receivables	(5,734)	(1,292)
Prepaid expenses and other assets	(146)	(1,892)
Inventories	125	39
Operating lease right-of-use assets	161	(24)
Accounts payable and accrued expenses	3,338	630
Discount and rebate liability	3,097	496
Operating lease liabilities	(216)	(37)
Other liabilities	622	(339)
Net cash used in operating activities	(12,749)	(7,657)
Cash flows from investing activities:
Acquisitions, net	0	(14,090)
Purchases of property and equipment	(52)	(31)
Purchases of investments	(17,467)	(23,832)
Maturities of investments	34,000	1,025
Net cash provided by (used in) investing activities	16,481	(36,928)
Cash flows from financing activities:
Proceeds from issuance of debt	12,800	12,800
Repayment of debt	(13,007)	0
Proceeds from insurance financing arrangements	1,256	1,273
Proceeds from Employee Stock Purchase Plan	166	216
Payments of principal on insurance financing arrangements	(564)	(469)
Payments to repurchase shares as part of the Share Repurchase Program	(3,447)	(4,723)
Payment of offering costs	0	(68)
Repayment of principal on finance lease liabilities	(3)	(11)
Net cash (used in) provided by financing activities	(2,799)	9,018
Effect of exchange rate changes on cash and cash equivalents	(203)	0
Net increase (decrease) in cash and cash equivalents	730	(35,567)
Cash and cash equivalents, beginning of period	65,466	112,346
Cash and cash equivalents, end of period	66,196	76,779
Supplemental cash flow information:
Cash paid for interest	261	41
In Process Research and Development [Member]
Adjustments to reconcile net loss to net cash used in operating activities:
Acquired in-process research and development	$ 0	$ 17,663

Note A - Description of Busines

Note A - Description of Business, Basis of Presentation and Significant Transactions	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Business Description and Basis of Presentation [Text Block]	A. Description of Business, Basis of Presentation, and Significant Transactions Organization Zevra Therapeutics, Inc. (the "Company") is a rare disease company melding science, data and patient need to create transformational therapies for diseases with limited or no first KP1077 eing developed as a treatment for idiopathic hypersomnia ("IH"), a rare neurological sleep disorder, and narcolepsy. KP1077 KP1077 February 21, 2023. March 1, 2023, The accompanying unaudited condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America ("U.S. GAAP") for interim financial information and with the instructions 10 8 03 X. not three six June 30, 2023, not may December 31, 2023. This interim information should be read in conjunction with the audited financial statements included in the Company’s Annual Report on Form 10 December 31, 2022, March 7, 2023. Basis of Presentation The Company prepared the unaudited condensed consolidated financial statements in accordance with U.S. GAAP and the rules and regulations of the SEC and, in the Company's opinion, reflect all adjustments, including normal recurring items that are necessary. Arimoclomol Acquisition On May 15, 2022, February 21, 2023) ( May 31, 2022. The Company accounted for the arimoclomol acquisition as an asset acquisition as the majority of the value of the assets acquired related to the arimoclomol acquired in-process research and development (“IPR&D”) asset. The intangible asset associated with IPR&D relates to arimoclomol. The estimated fair value of $17.7 million was determined using the excess earnings valuation method, a variation of the income valuation approach. The excess earnings valuation method estimates the value of an intangible asset equal to the present value of the incremental after-tax cash flows attributable to that intangible asset over its remaining economic life. Some of the more significant assumptions utilized in the Company's asset valuations included projected revenues, probability of commercial success, and the discount rate. The fair value using the excess earnings valuation method was determined using an estimated weighted average cost of capital of 42%, which reflects the risks inherent in future cash flow projections and represents a rate of return that a market participant would expect for this asset. This fair value measurement was based on significant inputs not 3 In accordance with Accounting Standards Codification ("ASC"), Subtopic 730 10 25, Accounting for Research and Development Costs not no d a $0.8 million income tax benefit for the year ended December 31, 2022, The following represents the consideration paid and purchase price allocation for the acquisition of arimoclomol (in thousands): Cash $ 12,800 Assumed reserve liability 5,200 Total consideration $ 18,000 Total consideration $ 18,000 Direct transaction costs associated with the acquisition (1) 1,290 Total purchase price to be allocated $ 19,290 Property and equipment, inventory and assembled workforce acquired $ 1,627 IPR&D (2) 17,663 Total allocated purchase price $ 19,290 ( 1 ( 2 Amendment to Registration Statement on Form S- 3 On January 25, 2022, 1 No. 333 250945 3 January 2021 February 1, 2022. Entry into 2021 ATM Agreement On July 2, 2021, "2021 may 2021 3. may 415 1933, may 2021 3 2021 July 12, 2021. June 30, 2023, 2021 Share Repurchase Program On December 20, 2021, may December 31, 2023. may 10b 18 1934, not may June 30, 2023, hased 1,575,692 shares of its common stock for approximately $11.0 million under the Share Repurchase Program. Reclassifications Certain reclassifications were made to the 2022 2023. no

Note B - Summary of Significant

Note B - Summary of Significant Accounting Policies	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Significant Accounting Policies [Text Block]	B. Summary of Significant Accounting Policies Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires the Company to make estimates and assumptions that affect the amounts reported in the unaudited condensed consolidated financial statements and accompanying notes. Actual results could differ from those estimates. On an ongoing basis, the Company evaluates its estimates and assumptions, including those related to revenue recognition, the useful lives of property and equipment, the recoverability of long-lived assets, the incremental borrowing rate for leases, and assumptions used for purposes of determining stock-based compensation, income taxes, the fair value of investments and the fair value of the derivative and warrant liability and discount and rebate liabilities, among others. The Company bases its estimates on historical experience and on various other assumptions that it believes to be reasonable, the results of which form the basis for making judgments about the carrying value of assets and liabilities. Investments The Company maintains investment securities that are classified as available-for-sale securities for which the Company has elected the fair value option under ASC 825, Financial Instruments June 30, 2023, December 31, 2022, $20 .7 $0.3 m 20.5 million as of December 31, 2022, December 31, 2022. May 2023. Revenue Recognition The Company recognizes revenue in accordance with the provisions of ASC 606, Revenue from Contracts with Customers 606” five 1 2 3 4 5 Arimoclomol Early Access Program Net revenue includes revenue from the sale of arimoclomol for the treatment of NPC under the remunerated early access compassionate use program in France (“French nATU”). An early access compassionate use program is a program giving specific patients access to a drug, which is not no In accordance with ASC 606, The French Health Authorities and the manufacturer have agreed to a price for sales during the French nATU, but the final transaction price depends on the terms and conditions in the contracts with the French Health Authorities and is subject to price negotiations with the French Health Authorities, following market approval. Any excess in the price charged the manufacturer compared to the price agreed with the health authorities once the drug product is approved in France must be repaid. The repayment is considered in the clawback liability (rebate). An estimate of net revenue and clawback liability are recognized using the ‘expected value’ method. Accounting for net revenue and clawback liability requires determination of the most appropriate method for the expected final transaction price. This estimate also requires assumptions with respect to inputs into the method, including current pricing of comparable marketed products within the rare disease area in France. Management has considered the expected final sales price as well as the price of similar drug products. The Company is operating within a rare disease therapeutic area where there is unmet treatment need and hence a limited number of comparable commercialized drugs products. The limited available relevant market information for directly comparable commercialized drugs within rare disease increases the uncertainty in management's estimate. For the three six June 30, 2023, million, respectively, which is net of a clawback liability o ion, respectively, and other gross to net adjustments. For the three six June 30, 2022, , which is net of a clawback liability of llion during the same periods. As part of the Arimoclomol Purchase Agreement the Company assumed an estimated reserve liability of $5.2 million related to revenue generated from the Arimoclomol EAP in France. The total estimate reserve liability as of June 30, 2023, six June 30, 2023, illion. The total reserve liabili December 31, 2022, June 30, 2023, December 31, 2022, Licensing Agreements The Company enters into licensing agreements with licensees that fall under the scope of ASC 606. The terms of the Company’s licensing agreements typically include one may As part of the accounting for these agreements, the Company must develop estimates and assumptions that require judgment to determine the underlying stand-alone selling price for each performance obligation which determines how the transaction price is allocated among the performance obligations. Generally, the estimation of the stand-alone selling price may Up-front Fees: Milestone Payments: not not not may AZSTARYS License Agreement In September 2019, In April 2021, No. 1 Commave also agreed to be responsible for and reimburse the Company for all of the development, commercialization and regulatory expenses incurred on the licensed products, subject to certain limitations as set forth in the AZSTARYS License Agreement. As part of this agreement, the Company is obligated to perform consulting services on behalf of Commave related to the licensed products. For these consulting services, Commave has agreed to pay the Company a set rate per hour on any consulting services performed on behalf of Commave for the benefit of the licensed products. In accordance with the terms of the Company’s March 20, 2012 KP1077 The AZSTARYS License Agreement is within the scope of ASC 606, not 606, third two $10.0 The Company is entitled to additional payments from Commave conditioned upon the achievement of specified regulatory milestones related to AZSTARYS and the achievement of certain U.S. sales milestones. Further, Commave will pay the Company quarterly, tiered royalty payments based on a range of percentage of Net Sales (as defined in the AZSTARYS License Agreement). The Company concluded that these regulatory milestones, sales milestones and royalty payments each contain a significant uncertainty associated with a future event. As such, these milestone and royalty payments are constrained at contract inception and are not not not ents. At the end of each reporting period, the Company updates its assessment of whether the milestone and royalty payments are constrained by considering both the likelihood and magnitude of the potential revenue reversal. For the three six June 30, 2023, June 2023. three six June 30, 2022, June 30, 2023, December 31, 2022. Consulting Arrangements The Company enters into consulting arrangements with third 606. may third Corium Consulting Agreement In July 2020, Under the Corium Consulting Agreement, the Company was entitled to receive payments from Corium of up to $15.6 million, $13.6 million of which was paid in quarterly installments through March 31, 2022. first 2022. 606, not third third not not not The Company determined that the performance of consulting services, including reimbursement of third third 606 For the six June 30, 2023, $0.2 mi six June 30, 2022, $2.0 three June 30, 2023, 2022. June 30, 2023, December 31, 2022, March 31, 2023. Foreign currency Assets and liabilities are translated into the reporting currency using the exchange rates in effect on the unaudited consolidated condensed balance sheet dates. Equity accounts are translated at historical rates, except for the change in retained earnings during the year, which is the result of the income statement translation process. Revenue and expense accounts are translated using the weighted average exchange rate during the period. The cumulative translation adjustments associated with the net assets of foreign subsidiaries are recorded in accumulated other comprehensive income/loss in the accompanying unaudited condensed consolidated statements of stockholders’ equity. Accounts and Other Receivables Accounts and other receivables consist of receivables under the AZSTARYS License Agreement and Arimoclomol EAP, as well as receivables related to consulting arrangements, income tax receivables and other receivables due to the Company. Receivables under the AZSTARYS License Agreement are recorded for amounts due to the Company related to reimbursable third June 30, 2023, $5.9 $5.7 $1.1 $1.3 m December 31, 2022, $0.2 m $0.9 million and other receivables of $0.4 m June 30, 2023, December 31, 2022, no

Note C - Debt Obligations

Note C - Debt Obligations	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Debt Disclosure [Text Block]	C. Debt Obligations Line of Credit On May 31, 2022, 0.00%. The revolving facility under the Line of Credit is secured by a perfected security interest in deposit accounts. The revolving facility under the Line of Credit is subject to customary affirmative and negative covenants. The latest maturity date of the loans under the Line of Credit was May 31, 2025. December 31, 2022, December 31, 2022. December 31, 2022. not January 31, 2023, May 27, 2023. On January 26, 2023, ts are used as collateral for the loan and the amount the Company is able to borrow is limited to 80 90% June 30, 2023,

Note D - Commitments and Contin

Note D - Commitments and Contingencies	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Commitments and Contingencies Disclosure [Text Block]	D. Commitments and Contingencies From time to time, the Company is involved in various legal proceedings arising in the normal course of business. For some matters, a liability is not not On May 31, 2023, October 25, 2018. six June 30, 2023, October 2023. December 31, 2022

Note E - Stock and Warrants

Note E - Stock and Warrants	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Equity [Text Block]	E. Stock and Warrants Authorized, Issued, and Outstanding Common Shares As of June 30, 2023 December 31, 2022 s, 35,503,697 and 35 ,450,257 June 30, 2023, December 31, 2022, d 33,928,005 and 3 4,540,304 June 30, 2023 December 31, 2022 As of June 30, 2023 December 31, 2022 June 30, 2023 December 31, 2022 Outstanding awards under equity incentive plans 7,027,739 2,456,407 Outstanding common stock warrants 4,252,490 4,252,600 Possible future issuances under equity incentive plans 2,731,788 4,421,508 Possible future issuances under employee stock purchase plans 1,375,175 1,417,365 Total common shares reserved for future issuance 15,387,192 12,547,880 Common Stock Activity The following table summarizes common stock activity for the six June 30, 2023 : Shares of Common Stock Balance as of January 1, 2023 34,540,304 Common stock issued as compensation to third parties 7,129 Common stock repurchased as a result of the Stock Repurchase Plan (665,739 ) Common stock issued as a result of stock warrants exercised 110 Balance as of March 31, 2023 33,881,804 Common stock issued as a result of the Employee Stock Purchase Plan 42,190 Common stock issued as compensation to third parties 4,011 Balance as of June 30, 2023 33,928,005 Authorized, Issued, and Outstanding Preferred Stock As of June 30, 2023, December 31, 2022, none Warrants to Purchase Common Stock In prior periods, the Company issued warrants to purchase common stock to various third June 30, 2023, The remaining 31,250 warrants are not four 815 815 three six June 30. 2023, Of the outstanding and exercisable warrants, 120,192 qualify as participating securities under ASC Topic 260, Earnings per Share may three six June 30. 2023,

Note F - Stock-based Compensati

Note F - Stock-based Compensation	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Share-Based Payment Arrangement [Text Block]	F. Stock-Based Compensation The Company maintains a stock-based compensation plan (the “Incentive Stock Plan”) that governs stock awards made to employees and directors prior to completion of the IPO. In November 2014, April 2015, 2014 “2014 April 2015. 2014 June 2021, 2014 2014 April 2021, January 1, 2031 2014 162 may 2014 is 8,271,497 as of June 30, 2023. 2014 January 1 January 1, 2016, January 1, 2031, December 31 2014 January 1, 2023, 2014 During the three six June 30, 2023, 2022, In June 2021, April 2021. may first October 1, 2021, first May 31, 2022. June 30, 2023, 124,825 shar In January 2023, 2023 "2023 may 2023 In May 2023, 2014 Stock-based compensation expense recorded under the Incentive Stock Plan, A&R 2014 2023 Three months ended June 30, Six months ended June 30, 2023 2022 2023 2022 Research and development $ 731 $ 422 $ 1,303 $ 733 Selling, general and administrative 372 1,088 391 1,695 Total stock-based compensation expense $ 1,103 $ 1,510 $ 1,694 $ 2,428 There was no stock-based compensation expense related to performance-based awards recognized during the three six June 30, 2023. three six June 30, 2022. As a result of the Mickle Transition Agreement and the Pascoe Transition Agreement, as further discussed in Note J, certain stock options were modified, resulting in a net decrease in stock-based compensation expense of $0.2 million and $1.2 million for the three six June 30, 2023,

Note G - Fair Value of Financia

Note G - Fair Value of Financial Instruments	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Fair Value Disclosures [Text Block]	G. Fair Value of Financial Instruments The accounting standard for fair value measurements provides a framework for measuring fair value and requires disclosures regarding fair value measurements. Fair value is defined as the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date, based on the Company’s principal or, in absence of a principal, most advantageous market for the specific asset or liability. The Company uses a three three ● Level 1—Observable ● Level 2—Observable ● Level 3—Unobservable no The carrying amounts of certain financial instruments, including cash and cash equivalents, investments and accounts payable and accrued expenses, approximate their respective fair values due to the short-term nature of such instruments. Assets and Liabilities Measured at Fair Value on a Recurring Basis The Company evaluates its financial assets and liabilities subject to fair value measurements on a recurring basis to determine the appropriate level in which to classify them for each reporting period. This determination requires significant judgments to be made. The following table summarizes the conclusions reached regarding fair value measurements as of June 30, 2023, December 31, 2022 ( Balance as of June 30, 2023 Quoted Prices in Active Markets for Identical Assets (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Securities: U.S. government-sponsored agency securities $ 7,325 $ — $ 7,325 $ — U.S. Treasury securities 13,371 13,371 — — Total assets $ 20,696 $ 13,371 $ 7,325 $ — Balance as of December 31, 2022 Quoted Prices in Active Markets for Identical Assets (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Securities: U.S. government-sponsored agency securities $ 7,189 $ — $ 7,189 $ — U.S. Treasury securities 9,711 9,711 — — Total assets $ 16,900 $ 9,711 $ 7,189 $ —

Note H - Net Loss Per Share

Note H - Net Loss Per Share	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Earnings Per Share [Text Block]	H. Net Loss Per Share For all periods presented herein, the Company did not two not two Under the two No no two two two As noted above, for all periods presented herein, the Company did not two no The following securities, presented on a common stock equivalent basis, have been excluded from the calculation of weighted average number of shares of common stock outstanding because their effect is anti-dilutive: Three months ended June 30, Six months ended June 30, 2023 2022 2023 2022 Awards under equity incentive plans 7,027,739 2,529,569 7,027,739 2,529,569 Common stock warrants 4,252,490 4,252,600 4,252,490 4,252,600 Total securities excluded from the calculation of weighted average number of shares of common stock outstanding 11,280,229 6,782,169 11,280,229 6,782,169 A reconciliation from net loss to basic and diluted net loss per share of common stock for the three six June 30, 2023, 2022, Three months ended June 30, Six months ended June 30, 2023 2022 2023 2022 Basic and diluted net loss per share of common stock: Net loss, basic and diluted $ (5,084 ) $ (24,042 ) $ (16,851 ) $ (25,906 ) Weighted average number of shares of common stock outstanding, basic and diluted 33,898 34,447 34,181 34,477 Basic and diluted net loss per share of common stock $ (0.15 ) $ (0.70 ) $ (0.49 ) $ (0.75 )

Note I - Leases

Note I - Leases	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Lease Disclosure [Text Block]	I. Leases The Company has operating and finance leases for office space, laboratory facilities and various laboratory equipment, furniture and office equipment and leasehold improvements. The Company determines if an arrangement is a lease at contract inception. Lease assets and lease liabilities are recognized based on the present value of lease payments over the lease term at the commencement date. The Company does not 12 not Effective June 1, 2021, one two February 2026. The components of lease expense were as follows (in thousands): Three months ended June 30, Six months ended June 30, Lease Cost 2023 2022 2023 2022 Finance lease cost: Amortization of right-of-use assets $ 32 $ 32 $ 64 $ 64 Interest on lease liabilities — — — 1 Total finance lease cost 32 32 64 65 Operating lease cost 114 99 227 190 Short-term lease cost 55 52 110 102 Variable lease cost 13 13 26 26 Less: sublease income (39 ) (39 ) (78 ) (78 ) Total lease costs $ 175 $ 157 $ 349 $ 305 Supplemental cash flow information related to leases was as follows (in thousands): Six months ended June 30, 2023 2022 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from finance leases $ — $ 1 Financing cash flows from finance leases 3 11 Operating cash flows from operating leases 284 241 Operating cash flows from short-term leases 110 102 Operating cash flows from variable lease costs 26 26 Right-of-use assets obtained in exchange for lease liabilities: Finance leases $ — $ — Operating leases — 146 Supplemental balance sheet information related to leases was as follows (in thousands, except weighted average remaining lease term and weighted average discount rate): June 30, December 31, 2023 2022 Finance Leases Property and equipment, at cost $ 1,031 $ 1,031 less: accumulated depreciation and amortization (844 ) (780 ) Property and equipment, net $ 187 $ 251 Other current liabilities $ 3 $ 6 Other long-term liabilities — — Total finance lease liabilities $ 3 $ 6 Operating Leases Operating lease right-of-use assets $ 803 $ 988 Total operating lease right-of-use assets $ 803 $ 988 Current portion of operating lease liabilities $ 456 $ 480 Operating lease liabilities, less current portion 627 843 Total operating lease liabilities $ 1,083 $ 1,323 Weighted Average Remaining Lease Term Finance leases 1 year 1 year Operating leases 3 years 3 years Weighted Average Discount Rate Finance leases 14.3 % 14.3 % Operating leases 7.5 % 7.3 % Maturities of lease liabilities were as follows (in thousands): Finance Operating Year Ending December 31, Leases Leases 2023 (excluding the six months ended June 30, 2023) $ 3 $ 275 2024 — 488 2025 — 389 2026 — 30 2027 — — Total lease payments 3 1,182 Less: future interest expense 0 (99 ) Lease liabilities $ 3 $ 1,083

Note J - Significant Events

Note J - Significant Events	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Significant Events [Text Block]	J. Significant Events On January 6, 2023, 2023 April 25, 2023. January 6, 2023, January 6, 2023, In connection with Mr. Pascoe’s appointment as the Company’s Chief Executive Officer, the Company and Mr. Pascoe entered into an amendment to the employment agreement, dated November 5, 2021, 2014 January 9, 2023. first January 6, 2024 ( In connection with the management transition, the Company entered into (i) a transition agreement with Dr. Mickle (the “Mickle Transition Agreement”) and (ii) a consulting agreement with Dr. Mickle (the “Consulting Agreement”). Pursuant to the terms of the Mickle Transition Agreement, subject to his timely delivering a release of claims in the Company’s favor, Dr. Mickle will receive severance payments and benefits consisting of (i) continued payment of his base salary for 18 months following the date on which Dr. Mickle’s employment with the Company ends (the “Separation Date”), (ii) up to 18 months of continued medical, dental and vision coverage pursuant to COBRA and (iii) a one may 18 first 2023 April 25, 2023. 2014 the six June 30, 2023. No three June 30, 2023, June 30, 2023, of approximately $1.0 million in connection with the Mickle Transition Agreement. At the Annual Meeting, each of John B. Bode, Douglas W. Calder, and Corey Watton was elected as a director of the Company and each of Richard W. Pascoe, Christopher A. Posner, and David S. Tierney ceased serving on the Company's Board of Directors. After the Annual Meeting, the Company's Board of Directors accepted the resignation of Richard W. Pascoe from his role as Chief Executive Officer on May 5, 2023, June 1, 2023, 12 12 nine The severance benefits consisted of personnel and other related charges of approximately $0.8 million and stock compensation expense of approximately $1.0 million related to the acceleration of vesting on unvested shares subject to certain stock options and the extension of the exercise period for certain stock options. These severance benefits are presented in selling, general and administrative expenses in the unaudited condensed consolidated statement of operations for the three six June 30, 2023. June 30, 2023, In addition, on May 3, 2023, not 2024 May 2023, June 1, 2023. August 7, 2023, 2024 August 7, 2023,

Note K - Subsequent Events

Note K - Subsequent Events	6 Months Ended
	Jun. 30, 2023
Notes to Financial Statements
Subsequent Events [Text Block]	K. Subsequent Events The Company evaluated events and transactions occurring subsequent to June 30, 2023, August 14, 2023, August 7, 2023, no

Significant Accounting Policies

Significant Accounting Policies (Policies)	6 Months Ended
	Jun. 30, 2023
Accounting Policies [Abstract]
Use of Estimates, Policy [Policy Text Block]	Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires the Company to make estimates and assumptions that affect the amounts reported in the unaudited condensed consolidated financial statements and accompanying notes. Actual results could differ from those estimates. On an ongoing basis, the Company evaluates its estimates and assumptions, including those related to revenue recognition, the useful lives of property and equipment, the recoverability of long-lived assets, the incremental borrowing rate for leases, and assumptions used for purposes of determining stock-based compensation, income taxes, the fair value of investments and the fair value of the derivative and warrant liability and discount and rebate liabilities, among others. The Company bases its estimates on historical experience and on various other assumptions that it believes to be reasonable, the results of which form the basis for making judgments about the carrying value of assets and liabilities.
Investment, Policy [Policy Text Block]	Investments The Company maintains investment securities that are classified as available-for-sale securities for which the Company has elected the fair value option under ASC 825, Financial Instruments June 30, 2023, December 31, 2022, $20 .7 $0.3 m 20.5 million as of December 31, 2022, December 31, 2022. May 2023.
Revenue from Contract with Customer [Policy Text Block]	Revenue Recognition The Company recognizes revenue in accordance with the provisions of ASC 606, Revenue from Contracts with Customers 606” five 1 2 3 4 5 Arimoclomol Early Access Program Net revenue includes revenue from the sale of arimoclomol for the treatment of NPC under the remunerated early access compassionate use program in France (“French nATU”). An early access compassionate use program is a program giving specific patients access to a drug, which is not no In accordance with ASC 606, The French Health Authorities and the manufacturer have agreed to a price for sales during the French nATU, but the final transaction price depends on the terms and conditions in the contracts with the French Health Authorities and is subject to price negotiations with the French Health Authorities, following market approval. Any excess in the price charged the manufacturer compared to the price agreed with the health authorities once the drug product is approved in France must be repaid. The repayment is considered in the clawback liability (rebate). An estimate of net revenue and clawback liability are recognized using the ‘expected value’ method. Accounting for net revenue and clawback liability requires determination of the most appropriate method for the expected final transaction price. This estimate also requires assumptions with respect to inputs into the method, including current pricing of comparable marketed products within the rare disease area in France. Management has considered the expected final sales price as well as the price of similar drug products. The Company is operating within a rare disease therapeutic area where there is unmet treatment need and hence a limited number of comparable commercialized drugs products. The limited available relevant market information for directly comparable commercialized drugs within rare disease increases the uncertainty in management's estimate. For the three six June 30, 2023, million, respectively, which is net of a clawback liability o ion, respectively, and other gross to net adjustments. For the three six June 30, 2022, , which is net of a clawback liability of llion during the same periods. As part of the Arimoclomol Purchase Agreement the Company assumed an estimated reserve liability of $5.2 million related to revenue generated from the Arimoclomol EAP in France. The total estimate reserve liability as of June 30, 2023, six June 30, 2023, illion. The total reserve liabili December 31, 2022, June 30, 2023, December 31, 2022, Licensing Agreements The Company enters into licensing agreements with licensees that fall under the scope of ASC 606. The terms of the Company’s licensing agreements typically include one may As part of the accounting for these agreements, the Company must develop estimates and assumptions that require judgment to determine the underlying stand-alone selling price for each performance obligation which determines how the transaction price is allocated among the performance obligations. Generally, the estimation of the stand-alone selling price may Up-front Fees: Milestone Payments: not not not may AZSTARYS License Agreement In September 2019, In April 2021, No. 1 Commave also agreed to be responsible for and reimburse the Company for all of the development, commercialization and regulatory expenses incurred on the licensed products, subject to certain limitations as set forth in the AZSTARYS License Agreement. As part of this agreement, the Company is obligated to perform consulting services on behalf of Commave related to the licensed products. For these consulting services, Commave has agreed to pay the Company a set rate per hour on any consulting services performed on behalf of Commave for the benefit of the licensed products. In accordance with the terms of the Company’s March 20, 2012 KP1077 The AZSTARYS License Agreement is within the scope of ASC 606, not 606, third two $10.0 The Company is entitled to additional payments from Commave conditioned upon the achievement of specified regulatory milestones related to AZSTARYS and the achievement of certain U.S. sales milestones. Further, Commave will pay the Company quarterly, tiered royalty payments based on a range of percentage of Net Sales (as defined in the AZSTARYS License Agreement). The Company concluded that these regulatory milestones, sales milestones and royalty payments each contain a significant uncertainty associated with a future event. As such, these milestone and royalty payments are constrained at contract inception and are not not not ents. At the end of each reporting period, the Company updates its assessment of whether the milestone and royalty payments are constrained by considering both the likelihood and magnitude of the potential revenue reversal. For the three six June 30, 2023, June 2023. three six June 30, 2022, June 30, 2023, December 31, 2022. Consulting Arrangements The Company enters into consulting arrangements with third 606. may third Corium Consulting Agreement In July 2020, Under the Corium Consulting Agreement, the Company was entitled to receive payments from Corium of up to $15.6 million, $13.6 million of which was paid in quarterly installments through March 31, 2022. first 2022. 606, not third third not not not The Company determined that the performance of consulting services, including reimbursement of third third 606 For the six June 30, 2023, $0.2 mi six June 30, 2022, $2.0 three June 30, 2023, 2022. June 30, 2023, December 31, 2022, March 31, 2023.
Foreign Currency Transactions and Translations Policy [Policy Text Block]	Foreign currency Assets and liabilities are translated into the reporting currency using the exchange rates in effect on the unaudited consolidated condensed balance sheet dates. Equity accounts are translated at historical rates, except for the change in retained earnings during the year, which is the result of the income statement translation process. Revenue and expense accounts are translated using the weighted average exchange rate during the period. The cumulative translation adjustments associated with the net assets of foreign subsidiaries are recorded in accumulated other comprehensive income/loss in the accompanying unaudited condensed consolidated statements of stockholders’ equity.
Receivable [Policy Text Block]	Accounts and Other Receivables Accounts and other receivables consist of receivables under the AZSTARYS License Agreement and Arimoclomol EAP, as well as receivables related to consulting arrangements, income tax receivables and other receivables due to the Company. Receivables under the AZSTARYS License Agreement are recorded for amounts due to the Company related to reimbursable third June 30, 2023, $5.9 $5.7 $1.1 $1.3 m December 31, 2022, $0.2 m $0.9 million and other receivables of $0.4 m June 30, 2023, December 31, 2022, no

Note A - Description of Busin_2

Note A - Description of Business, Basis of Presentation and Significant Transactions (Tables)	6 Months Ended
	Jun. 30, 2023
Notes Tables
Asset Acquisition [Table Text Block]	Cash $ 12,800 Assumed reserve liability 5,200 Total consideration $ 18,000 Total consideration $ 18,000 Direct transaction costs associated with the acquisition (1) 1,290 Total purchase price to be allocated $ 19,290 Property and equipment, inventory and assembled workforce acquired $ 1,627 IPR&D (2) 17,663 Total allocated purchase price $ 19,290

Note E - Stock and Warrants (Ta

Note E - Stock and Warrants (Tables)	6 Months Ended
	Jun. 30, 2023
Notes Tables
Schedule of Authorized Shares of Common Stock Reserved for Future Issuance [Table Text Block]	June 30, 2023 December 31, 2022 Outstanding awards under equity incentive plans 7,027,739 2,456,407 Outstanding common stock warrants 4,252,490 4,252,600 Possible future issuances under equity incentive plans 2,731,788 4,421,508 Possible future issuances under employee stock purchase plans 1,375,175 1,417,365 Total common shares reserved for future issuance 15,387,192 12,547,880
Schedule of Common Stock Outstanding Roll Forward [Table Text Block]	Shares of Common Stock Balance as of January 1, 2023 34,540,304 Common stock issued as compensation to third parties 7,129 Common stock repurchased as a result of the Stock Repurchase Plan (665,739 ) Common stock issued as a result of stock warrants exercised 110 Balance as of March 31, 2023 33,881,804 Common stock issued as a result of the Employee Stock Purchase Plan 42,190 Common stock issued as compensation to third parties 4,011 Balance as of June 30, 2023 33,928,005

Note F - Stock-based Compensa_2

Note F - Stock-based Compensation (Tables)	6 Months Ended
	Jun. 30, 2023
Notes Tables
Share-Based Payment Arrangement, Expensed and Capitalized, Amount [Table Text Block]	Three months ended June 30, Six months ended June 30, 2023 2022 2023 2022 Research and development $ 731 $ 422 $ 1,303 $ 733 Selling, general and administrative 372 1,088 391 1,695 Total stock-based compensation expense $ 1,103 $ 1,510 $ 1,694 $ 2,428

Note G - Fair Value of Financ_2

Note G - Fair Value of Financial Instruments (Tables)	6 Months Ended
	Jun. 30, 2023
Notes Tables
Schedule of Fair Value, Assets and Liabilities Measured on Recurring Basis [Table Text Block]	Balance as of June 30, 2023 Quoted Prices in Active Markets for Identical Assets (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Securities: U.S. government-sponsored agency securities $ 7,325 $ — $ 7,325 $ — U.S. Treasury securities 13,371 13,371 — — Total assets $ 20,696 $ 13,371 $ 7,325 $ — Balance as of December 31, 2022 Quoted Prices in Active Markets for Identical Assets (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Securities: U.S. government-sponsored agency securities $ 7,189 $ — $ 7,189 $ — U.S. Treasury securities 9,711 9,711 — — Total assets $ 16,900 $ 9,711 $ 7,189 $ —

Note H - Net Loss Per Share (Ta

Note H - Net Loss Per Share (Tables)	6 Months Ended
	Jun. 30, 2023
Notes Tables
Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share [Table Text Block]	Three months ended June 30, Six months ended June 30, 2023 2022 2023 2022 Awards under equity incentive plans 7,027,739 2,529,569 7,027,739 2,529,569 Common stock warrants 4,252,490 4,252,600 4,252,490 4,252,600 Total securities excluded from the calculation of weighted average number of shares of common stock outstanding 11,280,229 6,782,169 11,280,229 6,782,169
Schedule of Earnings Per Share, Basic and Diluted [Table Text Block]	Three months ended June 30, Six months ended June 30, 2023 2022 2023 2022 Basic and diluted net loss per share of common stock: Net loss, basic and diluted $ (5,084 ) $ (24,042 ) $ (16,851 ) $ (25,906 ) Weighted average number of shares of common stock outstanding, basic and diluted 33,898 34,447 34,181 34,477 Basic and diluted net loss per share of common stock $ (0.15 ) $ (0.70 ) $ (0.49 ) $ (0.75 )

Note I - Leases (Tables)

Note I - Leases (Tables)	6 Months Ended
	Jun. 30, 2023
Notes Tables
Lease, Cost [Table Text Block]	Three months ended June 30, Six months ended June 30, Lease Cost 2023 2022 2023 2022 Finance lease cost: Amortization of right-of-use assets $ 32 $ 32 $ 64 $ 64 Interest on lease liabilities — — — 1 Total finance lease cost 32 32 64 65 Operating lease cost 114 99 227 190 Short-term lease cost 55 52 110 102 Variable lease cost 13 13 26 26 Less: sublease income (39 ) (39 ) (78 ) (78 ) Total lease costs $ 175 $ 157 $ 349 $ 305
Schedule of Leases Cash Flow Information [Table Text Block]	Six months ended June 30, 2023 2022 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from finance leases $ — $ 1 Financing cash flows from finance leases 3 11 Operating cash flows from operating leases 284 241 Operating cash flows from short-term leases 110 102 Operating cash flows from variable lease costs 26 26 Right-of-use assets obtained in exchange for lease liabilities: Finance leases $ — $ — Operating leases — 146
Schedule of Leases Balance Sheet Information [Table Text Block]	June 30, December 31, 2023 2022 Finance Leases Property and equipment, at cost $ 1,031 $ 1,031 less: accumulated depreciation and amortization (844 ) (780 ) Property and equipment, net $ 187 $ 251 Other current liabilities $ 3 $ 6 Other long-term liabilities — — Total finance lease liabilities $ 3 $ 6 Operating Leases Operating lease right-of-use assets $ 803 $ 988 Total operating lease right-of-use assets $ 803 $ 988 Current portion of operating lease liabilities $ 456 $ 480 Operating lease liabilities, less current portion 627 843 Total operating lease liabilities $ 1,083 $ 1,323 Weighted Average Remaining Lease Term Finance leases 1 year 1 year Operating leases 3 years 3 years Weighted Average Discount Rate Finance leases 14.3 % 14.3 % Operating leases 7.5 % 7.3 %
Operating and Finance Lease, Liability, Maturity [Table Text Block]	Finance Operating Year Ending December 31, Leases Leases 2023 (excluding the six months ended June 30, 2023) $ 3 $ 275 2024 — 488 2025 — 389 2026 — 30 2027 — — Total lease payments 3 1,182 Less: future interest expense 0 (99 ) Lease liabilities $ 3 $ 1,083

Note A - Description of Busin_3

Note A - Description of Business, Basis of Presentation and Significant Transactions (Details Textual) - USD ($) $ in Thousands				3 Months Ended	6 Months Ended	12 Months Ended
		May 15, 2022	Jul. 12, 2021	Mar. 31, 2023	Jun. 30, 2023	Dec. 31, 2022	Dec. 20, 2021	Jul. 02, 2021
Sale of Stock, Authorized Amount			$ 350,000
Stock Repurchase Program, Authorized Amount							$ 50,000
Share Repurchase Program [Member]
Treasury Stock, Common, Shares (in shares)					1,575,692
Treasury Stock, Common, Value					$ 11,000
JMP and RBCCM [Member] | ATM Agreement Sales [Member]
Equity Distribution Agreement, Maximum Aggregate Offering Price								$ 75,000
Equity Distribution Agreement, Commission Fee, Percentage of Gross Sales								3%
Stock Issued During Period, Value, New Issues			$ 75,000
Stock Issued During Period, Shares, New Issues (in shares)				0
Arimoclomol Purchase Agreement [Member]
Payments to Acquire Productive Assets, Total		$ 12,800
Asset Acquisition, Consideration Transferred, Estimated Reserve Liability		$ 5,200			$ 12,100	$ 9,000
Estimated Weighted Average Cost of Capital		42%
Effective Income Tax Rate Reconciliation, Tax Credit, Research, Amount						$ 800
Arimoclomol Purchase Agreement [Member] | In Process Research and Development [Member]
Asset Acquisition, Recognized Identifiable Assets, Finite-Lived Intangibles	[1]	$ 17,663
[1]The primary asset acquired, the IPR&D asset, was expensed and the allocated transaction related costs were included with and expensed with this asset.

Note A - Description of Busin_4

Note A - Description of Business, Basis of Presentation and Significant Transactions - Consideration Paid and Purchase Price Allocation for the Acquisition of Arimoclomol (Details) - Arimoclomol Purchase Agreement [Member] - USD ($) $ in Thousands			6 Months Ended	12 Months Ended
		May 15, 2022	Jun. 30, 2023	Dec. 31, 2022
Cash		$ 12,800
Assumed reserve liability		5,200	$ 12,100	$ 9,000
Total consideration		18,000
Total consideration		18,000
Direct transaction costs associated with the acquisition (1)	[1]	1,290
Total purchase price to be allocated		19,290
Property and equipment, inventory and assembled workforce acquired		1,627
Total allocated purchase price		19,290
In Process Research and Development [Member]
IPR&D (2)	[2]	$ 17,663
[1]As a result of the asset acquisition accounting, the transaction costs associated with the acquisition should be included in the costs of the assets acquired and allocated amongst qualifying assets using the relative fair value basis. The transaction costs primarily included financial advisor fees, legal expenses and auditor expenses.[2]The primary asset acquired, the IPR&D asset, was expensed and the allocated transaction related costs were included with and expensed with this asset.

Note B - Summary of Significa_2

Note B - Summary of Significant Accounting Policies (Details Textual) - USD ($) $ in Thousands			1 Months Ended	3 Months Ended			6 Months Ended		12 Months Ended
	May 15, 2022	Mar. 20, 2012	Jun. 30, 2023	Jun. 30, 2023	Jun. 30, 2022	Mar. 31, 2022	Jun. 30, 2023	Jun. 30, 2022	Dec. 31, 2022	May 07, 2021	Jul. 31, 2020
Investments, Fair Value Disclosure			$ 20,700	$ 20,700			$ 20,700		$ 16,900
Unrealized Gain (Loss) on Investments							(300)		(600)
Certificates of Deposit, at Carrying Value									20,500
Revenue from Contract with Customer, Including Assessed Tax				8,470	$ 1,300		11,349	$ 5,265
Accounts Receivable, after Allowance for Credit Loss, Current			1,300	1,300			1,300
Income Taxes Receivable			1,100	1,100			1,100		900
Accounts Receivable, Allowance for Credit Loss, Ending Balance			0	0			0		0
Consulting Services [Member]
Revenue from Contract with Customer, Including Assessed Tax				0	0		200	3,900
Deferred Revenue, Total			0	0			0		0
Accounts Receivable, after Allowance for Credit Loss, Current									400
GPC Member | License [Member]
Revenue Recognition, Milestone Method, Additional Revenue to be Recognized										$ 590,000
Deferred Revenue, Total			0	0			0		0
Accounts Receivable, after Allowance for Credit Loss, Current			5,700	5,700			5,700		500
Aquestive Therapeutics [Member] | License [Member]
Contract with Customer, Liability, Revenue Recognized			5,000
Asset Acquisition, Consideration Transferred, Estimated Reserve Liability							6,300
Royalty Revenue, Percent		10%
Revenue from Contract with Customer, Including Assessed Tax				5,700	100			200
Corium, Inc [Member]
Accounts Receivable, after Allowance for Credit Loss, Current									200
Corium, Inc [Member] | Consulting Services [Member]
Revenue from Contract with Customer, Including Assessed Tax						$ 2,000
Consulting Agreement, Maximum Amount to be Received											$ 15,600
Contract with Customer, Asset, after Allowance for Credit Loss, Total											13,600
Consulting Agreement, Conditional Milestone Achievement to be Received											$ 2,000
Arimoclomol EAP [Member]
Accounts Receivable, after Allowance for Credit Loss, Current			$ 5,900	5,900			5,900		6,300
Arimoclomol Purchase Agreement [Member]
Contract with Customer, Liability, Revenue Recognized				2,800	900		4,800	900
Contract with Customer, Clawback Liability				$ 1,600	$ 500		3,100	$ 500
Asset Acquisition, Consideration Transferred, Estimated Reserve Liability	$ 5,200						$ 12,100		$ 9,000

Note C - Debt Obligations (Deta

Note C - Debt Obligations (Details Textual) - USD ($) $ in Millions				9 Months Ended
	Jan. 31, 2023	Jan. 26, 2023	May 31, 2022	Sep. 30, 2022	Jun. 30, 2023	Dec. 31, 2022
Line of Credit Facility, Maximum Borrowing Capacity			$ 20			$ 20
Line of Credit Facility, Maximum Amount Outstanding During Period				$ 12.8
Certificates of Deposit, at Carrying Value						20.5
Line of Credit Facility, Remaining Borrowing Capacity						7.2
Wells Fargo Bank, N.A. [Member]
Long-Term Line of Credit					$ 12.7
Revolving Credit Facility [Member] | Ameris Bank [Member]
Repayments of Lines of Credit	$ 12.8
Asset Pledged as Collateral [Member]
Certificates of Deposit, at Carrying Value						$ 12.8
Secured Overnight Financing Rate (SOFR) Overnight Index Swap Rate [Member]
Debt Instrument, Basis Spread on Variable Rate			1.60%
Prime Rate [Member] | Wells Fargo Bank, N.A. [Member]
Debt Instrument, Basis Spread on Variable Rate		2.25%

Note D - Commitments and Cont_2

Note D - Commitments and Contingencies (Details Textual) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Loss Contingency Accrual	$ 900	$ 0

Note E - Stock and Warrants (De

Note E - Stock and Warrants (Details Textual) - shares	Jun. 30, 2023	Mar. 31, 2023	Dec. 31, 2022	Dec. 31, 2020	Oct. 25, 2018
Common Stock, Shares Authorized (in shares)	250,000,000		250,000,000
Common Stock, Shares, Issued (in shares)	35,503,697		35,450,257
Common Stock, Shares, Outstanding (in shares)	33,928,005	33,881,804	34,540,304
Preferred Stock, Shares Authorized (in shares)	10,000,000		10,000,000
Warrants to Purchase Common Stock [Member]
Class of Warrant or Right, Outstanding (in shares)	4,252,490
Class of Warrant or Right, Exercisable During Period (in shares)	4,221,240
Warrant Issued to KVK [Member]
Class of Warrant or Right, Number of Securities Called by Warrants or Rights (in shares)					31,250
Deerfield Warrant [Member] | Common Stock [Member]
Class of Warrant or Right, Number of Securities Called by Warrants or Rights (in shares)				120,192

Note E - Stock and Warrants - R

Note E - Stock and Warrants - Reserved Authorized Shares of Common Stock for Future Issuance (Details) - shares	Jun. 30, 2023	Dec. 31, 2022
Common shares reserved for future issuance (in shares)	15,387,192	12,547,880
Warrant [Member]
Common shares reserved for future issuance (in shares)	4,252,490	4,252,600
Share-Based Payment Arrangement [Member]
Common shares reserved for future issuance (in shares)	7,027,739	2,456,407
Possible Future Issuances Under Equity Incentive Plans [Member]
Common shares reserved for future issuance (in shares)	2,731,788	4,421,508
Possible Future Issuances Under Employee Stock Purchase Plans [Member]
Common shares reserved for future issuance (in shares)	1,375,175	1,417,365

Note E - Stock and Warrants - C

Note E - Stock and Warrants - Common Stock Activity (Details) - shares	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Mar. 31, 2023	Jun. 30, 2023
Balance (in shares)	33,881,804	34,540,304	34,540,304
Common stock issued as compensation to third parties (in shares)	4,011	7,129
Common stock repurchased as a result of the Stock Repurchase Plan (in shares)		(665,739)
Common stock issued as a result of stock warrants exercised (in shares)		110
Common stock issued as a result of the Employee Stock Purchase Plan (in shares)	42,190		124,825
Balance (in shares)	33,928,005	33,881,804	33,928,005

Note F - Stock-based Compensa_3

Note F - Stock-based Compensation (Details Textual) - USD ($) $ in Thousands		1 Months Ended	3 Months Ended		6 Months Ended
	Jan. 01, 2023	Jun. 30, 2021	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022	Jan. 31, 2023
Employee Stock Purchase Plan, Maximum Number of Issuable Shares (in shares)		1,500,000
Stock Issued During Period, Shares, Employee Stock Purchase Plans (in shares)			42,190		124,825
Share-Based Payment Arrangement, Expense			$ 1,103	$ 1,510	$ 1,694	$ 2,428
Severance Costs Recognized			200		1,200
Performance Shares [Member]
Share-Based Payment Arrangement, Expense			$ 0	$ 400	$ 0	$ 400
2014 Equity Incentive Plan [Member]
Share-based Compensation Arrangement by Share-based Payment Award, Number of Additional Shares Authorized (in shares)		4,900,000
Share-Based Compensation Arrangement by Share-Based Payment Award, Number of Shares Authorized (in shares)			8,271,497		8,271,497
Share-based Compensation Arrangement by Share-based Payment Award, Number of Shares Reserved for Issuance, Incremental Percentage of Capital Stock Outstanding		4%
Increase in Common Stock Reserved for Issuance (in shares)	1,381,612
Stock Issued During Period, Shares, Restricted Stock Award, Net of Forfeitures, Total (in shares)			0	0	0	0
The 2023 Plan [Member]
Share-Based Compensation Arrangement by Share-Based Payment Award, Number of Shares Authorized (in shares)							1,500,000

Note F - Stock-based Compensa_4

Note F - Stock-based Compensation - Stock-based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Stock based compensation expense	$ 1,103	$ 1,510	$ 1,694	$ 2,428
Research and Development Expense [Member]
Stock based compensation expense	731	422	1,303	733
General and Administrative Expense [Member]
Stock based compensation expense	$ 372	$ 1,088	$ 391	$ 1,695

Note G - Fair Value of Financ_3

Note G - Fair Value of Financial Instruments - Assets and Liabilities Measured at Fair Value on Recurring Basis (Details) - Fair Value, Recurring [Member] - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Total assets	$ 20,696	$ 16,900
Fair Value, Inputs, Level 1 [Member]
Total assets	13,371	9,711
Fair Value, Inputs, Level 2 [Member]
Total assets	7,325	7,189
Fair Value, Inputs, Level 3 [Member]
Total assets	0	0
US Government Agencies Debt Securities [Member]
Total assets	7,325	7,189
US Government Agencies Debt Securities [Member] | Fair Value, Inputs, Level 1 [Member]
Total assets	0	0
US Government Agencies Debt Securities [Member] | Fair Value, Inputs, Level 2 [Member]
Total assets	7,325	7,189
US Government Agencies Debt Securities [Member] | Fair Value, Inputs, Level 3 [Member]
Total assets	0	0
US Treasury Securities [Member]
Total assets	13,371	9,711
US Treasury Securities [Member] | Fair Value, Inputs, Level 1 [Member]
Total assets	13,371	9,711
US Treasury Securities [Member] | Fair Value, Inputs, Level 2 [Member]
Total assets	0	0
US Treasury Securities [Member] | Fair Value, Inputs, Level 3 [Member]
Total assets	$ 0	$ 0

Note H - Net Loss Per Share - A

Note H - Net Loss Per Share - Anti-dilutive Securities (Details) - shares	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Antidilutive securities (in shares)	11,280,229	6,782,169	11,280,229	6,782,169
Share-Based Payment Arrangement [Member]
Antidilutive securities (in shares)	7,027,739	2,529,569	7,027,739	2,529,569
Warrants to Purchase Common Stock [Member]
Antidilutive securities (in shares)	4,252,490	4,252,600	4,252,490	4,252,600

Note H - Net Loss Per Share - E

Note H - Net Loss Per Share - Earnings Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Net loss	$ (5,084)	$ (24,042)	$ (16,851)	$ (25,906)
Weighted average number of shares of common stock outstanding, basic and diluted (in shares)	33,898,233	34,447,206	34,180,818	34,476,737
Basic and diluted net loss per share of common stock (in dollars per share)	$ (0.15)	$ (0.70)	$ (0.49)	$ (0.75)

Note I - Leases (Details Textua

Note I - Leases (Details Textual)	6 Months Ended
	Jun. 30, 2023
Lessee, Lease, Option to Extend, Maximum Term (Year)	5 years
Lessee, Lease, Option to Terminate, Term (Year)	1 year
Minimum [Member]
Lessee, Lease, Remaining Term of Contract (Year)	1 year
Maximum [Member]
Lessee, Lease, Remaining Term of Contract (Year)	3 years

Note I - Leases - Lease Cost (D

Note I - Leases - Lease Cost (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Amortization of right-of-use assets	$ 32	$ 32	$ 64	$ 64
Interest on lease liabilities	0	0	0	1
Total finance lease cost	32	32	64	65
Operating lease cost	114	99	227	190
Short-term lease cost	55	52	110	102
Variable lease cost	13	13	26	26
Less: sublease income	(39)	(39)	(78)	(78)
Total lease costs	$ 175	$ 157	$ 349	$ 305

Note I - Leases - Supplement Ca

Note I - Leases - Supplement Cash Flow Information Related to Leases (Details) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Operating cash flows from finance leases	$ 0	$ 1
Financing cash flows from finance leases	3	11
Operating cash flows from operating leases	284	241
Operating cash flows from short-term leases	110	102
Operating cash flows from variable lease costs	26	26
Finance leases	0	0
Operating leases	$ 0	$ 146

Note I - Leases - Supplement Ba

Note I - Leases - Supplement Balance Sheet Information Related to Lease (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Lease liabilities, finance leases	$ 3	$ 6
Operating lease right-of-use assets	803	988
Total operating lease right-of-use assets	803	988
Current portion of operating lease liabilities	456	480
Operating lease liabilities, less current portion	627	843
Total operating lease liabilities	$ 1,083	$ 1,323
Finance leases (Year)	1 year	1 year
Operating leases (Year)	3 years	3 years
Finance leases	14.30%	14.30%
Operating leases	7.50%	7.30%
Property and Equipment, At Cost [Member]
Property and equipment, at cost	$ 1,031	$ 1,031
Accumulated Depreciation and Amortization [Member]
less: accumulated depreciation and amortization	(844)	(780)
Property and Equipment, Net [Member]
Property and equipment, net	187	251
Other Current Liabilities [Member]
Other current liabilities	3	6
Other Noncurrent Liabilities [Member]
Other long-term liabilities	0	0
Operating Lease Right-of-Use Assets [Member]
Operating lease right-of-use assets	803	988
Total operating lease right-of-use assets	803	988
Current Portion of Operating Lease Liabilities [Member]
Current portion of operating lease liabilities	456	480
Operating Lease Liabilities, Less Current Portion [Member]
Operating lease liabilities, less current portion	$ 627	$ 843

Note I - Leases - Maturities of

Note I - Leases - Maturities of lease Liabilities (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
2023, finance leases	$ 3
2023, operating leases	275
2024, finance leases	0
2024, operating leases	488
2025, finance leases	0
2025, operating leases	389
2026, finance leases	0
2026, operating leases	30
2027, finance leases	0
2027, operating leases	0
Total lease payments, finance leases	3
Total lease payments, operating leases	1,182
Less: future interest expense, finance leases	0
Less: future interest expense, operating leases	(99)
Lease liabilities, finance leases	3	$ 6
Lease liabilities, operating leases	$ 1,083	$ 1,323

Note J - Significant Events (De

Note J - Significant Events (Details Textual)			6 Months Ended
	Jan. 09, 2023 shares	Jan. 06, 2023 USD ($) shares	Jun. 30, 2023 USD ($)	Jun. 30, 2022 USD ($)
Severance Costs			$ 1,402,000	$ 0
Mr Pascoe [Member]
Severance Costs			800,000
Employee Severance [Member]
Restructuring Charges			400,000
Restructuring Reserve			1,000,000
Employee Severance [Member] | Mr Pascoe [Member]
Restructuring Charges			1,000,000
Restructuring Reserve			800,000
Chief Executive Officer [Member] | 2014 Equity Incentive Plan [Member]
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Grants in Period, Gross (in shares) | shares	700,000
Share-Based Compensation Arrangement by Share-Based Payment Award, Award Vesting Number of Installments	4
Company's President and Former Chief Executive Officer [Member]
Transition Agreement, Continued Payment of Base Salary, Period (Month)		18 months
Transition Agreement, Continued Medical, Dental and Vision Coverage, Period (Month)		18 months
Transition Agreement, Monthly Consulting Fees		$ 40,000
Severance Costs			$ 1,000,000
Company's President and Former Chief Executive Officer [Member] | Restricted Stock Units (RSUs) [Member]
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Grants in Period (in shares) | shares		547,945