DOCUMENT AND ENTITY INFORMATION
DOCUMENT AND ENTITY INFORMATION - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 | Feb. 28, 2024 | Jun. 30, 2023 | |
DOCUMENT AND ENTITY INFORMATION | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Fiscal Period Focus | FY | ||
Current Fiscal Year End Date | --12-31 | ||
Document Period End Date | Dec. 31, 2023 | ||
Document Transition Report | false | ||
Entity File Number | 000-29889 | ||
Entity Registrant Name | RIGEL PHARMACEUTICALS INC | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 94-3248524 | ||
Entity Address, Address Line One | 611 Gateway Boulevard, Suite 900, | ||
Entity Address, City or Town | South San Francisco | ||
Entity Address, State or Province | CA | ||
Entity Address, Postal Zip Code | 94080 | ||
City Area Code | 650 | ||
Local Phone Number | 624-1100 | ||
Title of 12(b) Security | Common Stock, par value $.001 per share | ||
Trading Symbol | RIGL | ||
Security Exchange Name | NASDAQ | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Interactive Data Current | Yes | ||
Entity Filer Category | Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
ICFR Auditor Attestation Flag | true | ||
Document Financial Statement Error Correction [Flag] | false | ||
Entity Shell Company | false | ||
Entity Central Index Key | 0001034842 | ||
Amendment Flag | false | ||
Entity Public Float | $ 222.7 | ||
Entity Common Stock, Shares Outstanding | 175,377,812 | ||
Document Fiscal Year Focus | 2023 | ||
Auditor Name | Ernst & Young LLP | ||
Auditor Firm ID | 42 | ||
Auditor Location | San Francisco, California |
BALANCE SHEETS
BALANCE SHEETS - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 32,786 | $ 24,459 |
Short-term investments | 24,147 | 33,747 |
Accounts receivable, net | 30,550 | 40,320 |
Inventories | 5,522 | 9,118 |
Prepaid and other current assets | 6,261 | 8,259 |
Total current assets | 99,266 | 115,903 |
Property and equipment, net | 165 | 857 |
Intangible asset, net | 13,878 | 14,949 |
Operating lease right-of-use asset | 861 | 1,930 |
Other assets | 3,055 | 640 |
Total assets | 117,225 | 134,279 |
Current liabilities: | ||
Accounts payable | 7,142 | 22,508 |
Accrued compensation | 8,676 | 8,866 |
Accrued research and development | 3,513 | 7,708 |
Revenue reserves and refund liability | 15,684 | 12,145 |
Other accrued liabilities | 5,334 | 6,485 |
Lease liabilities, current portion | 692 | 1,133 |
Deferred revenue | 1,355 | 1,369 |
Loans payable, net, current portion | 7,229 | |
Other long-term liabilities, current portion | 3,642 | 4,997 |
Total current liabilities | 53,267 | 65,211 |
Long-term portion of lease liabilities | 285 | 972 |
Long-term portion of loans payable, net of discount | 52,373 | 39,448 |
Other long-term liabilities | 39,944 | 42,264 |
Total liabilities | 145,869 | 147,895 |
Commitments | ||
Stockholders' deficit: | ||
Preferred stock, $0.001 par value; 10,000,000 shares authorized; none issued and outstanding as of December 31, 2023 and 2022 | ||
Common stock, $0.001 par value; 400,000,000 shares authorized; 174,825,610 and 173,398,645 shares issued and outstanding as of December 31, 2023 and 2022, respectively | 175 | 174 |
Additional paid-in capital | 1,378,723 | 1,368,822 |
Accumulated other comprehensive income (loss) | 8 | (153) |
Accumulated deficit | (1,407,550) | (1,382,459) |
Total stockholders' deficit | (28,644) | (13,616) |
Total liabilities and stockholders' deficit | $ 117,225 | $ 134,279 |
BALANCE SHEETS (Parenthetical)
BALANCE SHEETS (Parenthetical) - $ / shares | Dec. 31, 2023 | Dec. 31, 2022 |
BALANCE SHEETS | ||
Preferred stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 400,000,000 | 400,000,000 |
Common stock, shares issued | 174,825,610 | 173,398,645 |
Common stock, shares outstanding | 174,825,610 | 173,398,645 |
STATEMENTS OF OPERATIONS
STATEMENTS OF OPERATIONS - USD ($) shares in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Total revenues | $ 116,882,000 | $ 120,242,000 | $ 149,236,000 |
Costs and expenses: | |||
Cost of product sales | 7,110,000 | 1,749,000 | 1,083,000 |
Research and development | 24,522,000 | 60,272,000 | 65,237,000 |
Selling, general and administrative | 105,741,000 | 112,451,000 | 91,891,000 |
Restructuring charges | 1,320,000 | 3,521,000 | |
Total costs and expenses | 137,373,000 | 175,792,000 | 161,732,000 |
Loss from operations | (20,491,000) | (55,550,000) | (12,496,000) |
Interest income | 2,272,000 | 684,000 | 47,000 |
Interest expense | (6,872,000) | (3,707,000) | (4,860,000) |
Loss before income taxes | (25,091,000) | (58,573,000) | (17,309,000) |
Provision for income taxes | 605,000 | ||
Net loss | $ (25,091,000) | $ (58,573,000) | $ (17,914,000) |
Net loss per share, basic (in dollars per share) | $ (0.14) | $ (0.34) | $ (0.11) |
Net loss per share, diluted (in dollars per share) | $ (0.14) | $ (0.34) | $ (0.11) |
Weighted average shares used in computing net loss per share, basic (in shares) | 174,017 | 172,406 | 170,492 |
Weighted average shares used in computing net loss per share, diluted (in shares) | 174,017 | 172,406 | 170,492 |
Product sales, net | |||
Total revenues | $ 104,294,000 | $ 76,718,000 | $ 63,010,000 |
Revenues from collaborations | |||
Total revenues | 11,488,000 | 39,024,000 | 75,726,000 |
Government contracts | |||
Total revenues | $ 1,100,000 | $ 4,500,000 | $ 10,500,000 |
STATEMENTS OF COMPREHENSIVE LOS
STATEMENTS OF COMPREHENSIVE LOSS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
STATEMENTS OF COMPREHENSIVE LOSS | |||
Net loss | $ (25,091) | $ (58,573) | $ (17,914) |
Other comprehensive gain (loss): | |||
Net change in unrealized gain (loss) on short-term investments | 161 | (51) | (98) |
Comprehensive loss | $ (24,930) | $ (58,624) | $ (18,012) |
STATEMENTS OF STOCKHOLDERS' (DE
STATEMENTS OF STOCKHOLDERS' (DEFICIT) EQUITY - USD ($) $ in Thousands | Common Stock | Additional Paid-in Capital | Accumulated Other Comprehensive Income (Loss) | Accumulated Deficit | Total |
Balance at Dec. 31, 2020 | $ 169 | $ 1,339,833 | $ (4) | $ (1,305,972) | $ 34,026 |
Balance (in shares) at Dec. 31, 2020 | 169,316,782 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Net loss | (17,914) | (17,914) | |||
Net change in unrealized gain (loss) on short-term investments | (98) | (98) | |||
Issuance of common stock upon exercise of options | $ 3 | 4,772 | 4,775 | ||
Issuance of common stock upon exercise of options (in shares) | 2,285,444 | ||||
Stock-based compensation expense | 9,585 | 9,585 | |||
Balance at Dec. 31, 2021 | $ 172 | 1,354,190 | (102) | (1,323,886) | 30,374 |
Balance (in shares) at Dec. 31, 2021 | 171,602,226 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Net loss | (58,573) | (58,573) | |||
Net change in unrealized gain (loss) on short-term investments | (51) | (51) | |||
Issuance of common stock upon exercise of options | $ 2 | 2,122 | 2,124 | ||
Issuance of common stock upon exercise of options (in shares) | 1,580,169 | ||||
Issuance of common stock upon vesting of restricted stock units (RSUs) (in shares) | 216,250 | ||||
Stock-based compensation expense | 12,510 | 12,510 | |||
Balance at Dec. 31, 2022 | $ 174 | 1,368,822 | (153) | (1,382,459) | (13,616) |
Balance (in shares) at Dec. 31, 2022 | 173,398,645 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Net loss | (25,091) | (25,091) | |||
Net change in unrealized gain (loss) on short-term investments | 161 | 161 | |||
Issuance of common stock upon exercise of options | $ 1 | 1,048 | 1,049 | ||
Issuance of common stock upon exercise of options (in shares) | 991,959 | ||||
Issuance of common stock upon vesting of restricted stock units (RSUs) (in shares) | 435,006 | ||||
Stock-based compensation expense | 8,853 | 8,853 | |||
Balance at Dec. 31, 2023 | $ 175 | $ 1,378,723 | $ 8 | $ (1,407,550) | $ (28,644) |
Balance (in shares) at Dec. 31, 2023 | 174,825,610 |
STATEMENTS OF CASH FLOWS
STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Operating activities | |||
Net loss | $ (25,091) | $ (58,573) | $ (17,914) |
Adjustments to reconcile net loss to net cash (used in) provided by operating activities: | |||
Stock-based compensation expense | 8,806 | 12,385 | 9,486 |
Loss (gain) on sale and disposal of fixed assets | 266 | (138) | |
Depreciation and amortization | 1,238 | 998 | 1,162 |
Non-cash interest expense | 682 | 3,139 | |
Net amortization and accretion of discount on short-term investments and term loan | (479) | (63) | 287 |
Changes in assets and liabilities: | |||
Accounts receivable, net | 9,770 | (24,848) | 501 |
Inventories | 1,172 | (2,377) | (4,875) |
Prepaid and other current assets | 1,998 | (847) | 6,633 |
Other assets | 56 | 334 | (150) |
Right-of-use assets | 1,069 | 7,773 | 8,192 |
Accounts payable | (366) | 3,788 | 41 |
Accrued compensation | (190) | (1,824) | 1,098 |
Accrued research and development | (4,195) | (2,676) | 5,495 |
Revenue reserves and refund liability | 3,539 | 4,230 | 1,850 |
Other accrued liabilities | (1,151) | 1,709 | (24) |
Lease liability | (1,128) | (8,546) | (8,621) |
Deferred revenue | (14) | (1,227) | (422) |
Other current and long-term liabilities | (1,043) | (4,538) | |
Net cash (used in) provided by operating activities | (5,743) | (73,758) | 5,878 |
Investing activities | |||
Maturities of short-term investments | 41,650 | 101,228 | 62,050 |
Purchases of short-term investments | (31,206) | (28,894) | (141,459) |
Purchases of intangible asset | (15,000) | ||
Proceeds from sale of property and equipment | 259 | 893 | |
Purchases of property and equipment | (450) | (627) | |
Net cash (used in) provided by investing activities | (4,297) | 72,777 | (80,036) |
Financing activities | |||
Net proceeds from term loan financing | 19,950 | 19,542 | |
Net proceeds from issuances of common stock upon exercise of options and participation in Purchase Plan | 1,049 | 2,124 | 4,775 |
Cost share advance from a collaboration partner | 57,900 | ||
Cost share payments to a collaboration partner | (2,632) | (15,116) | |
Net cash provided by financing activities | 18,367 | 6,550 | 62,675 |
Net increase (decrease) in cash and cash equivalents | 8,327 | 5,569 | (11,483) |
Cash and cash equivalents at beginning of period | 24,459 | 18,890 | 30,373 |
Cash and cash equivalents at end of period | 32,786 | 24,459 | 18,890 |
Supplemental disclosure of cash flow information | |||
Interest paid | $ 5,848 | 2,495 | $ 1,500 |
Purchases of intangible asset included within accounts payable | $ 15,000 |
DESCRIPTION OF BUSINESS AND SUM
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 12 Months Ended |
Dec. 31, 2023 | |
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 1. DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Description of Business We are a biotechnology company dedicated to developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. We focus on products that address signaling pathways that are critical to disease mechanisms. Our first product approved by the FDA is TAVALISSE (fostamatinib disodium hexahydrate) tablets, the only approved oral SYK inhibitor for the treatment of adult patients with chronic ITP who have had an insufficient response to a previous treatment. The product is also commercially available in Europe and the UK (as TAVLESSE), and in Canada, Israel and Japan (as TAVALISSE) for the treatment of chronic ITP in adult patients. Our second FDA-approved product is REZLIDHIA (olutasidenib) capsules for the treatment of adult patients with R/R AML with a susceptible IDH1 mutation as detected by an FDA-approved test. We began our commercialization of REZLIDHIA in December 2022. W olutasidenib We continue to advance the development of our IRAK1/4 inhibitor program, in an open-label, Phase 1b trial to determine the tolerability and preliminary efficacy of the drug in patients with lower-risk MDS In February 2024, we entered into an Asset Purchase Agreement with Blueprint to purchase certain assets comprising the right to research, develop, manufacture and commercialize GAVRETO (pralsetinib) in the US. GAVRETO (pralsetinib) is a once daily, small molecule, oral, kinase inhibitor of wild-type RET and oncogenic RET fusions. GAVRETO is approved by the FDA for the treatment of adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA-approved test. GAVRETO is also approved under accelerated approval based on overall response rate and duration response rate, for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). We have strategic development collaborations with the MD Anderson to expand our evaluation of REZLIDHIA (olutasidenib) in AML and other hematologic cancers, and with CONNECT to conduct a Phase 2 clinical trial to evaluate REZLIDHIA (olutasidenib) in combination with temozolomide as maintenance therapy in newly diagnosed pediatric and young adult patients with HGG harboring an IDH1 mutation. We have a RIPK1 inhibitor program in clinical development with our partner Lilly. We also have product candidates in clinical development with partners BerGenBio and Daiichi. Basis of Presentation The accompanying financial statements have been prepared in accordance with United States generally accepted accounting principles (US GAAP). Any reference in these notes to applicable accounting guidance is meant to refer to the authoritative US GAAP included in the Accounting Standards Codification (ASC), and Accounting Standards Update (ASU) issued by the Financial Accounting Standards Board (FASB). We manage our operations as one business segment for purposes of assessing performance, making operating decisions, and allocating resources, and our chief operating decision maker is our chief executive officer. Liquidity As of December 31, 2023, we had approximately $56.9 million in cash, cash equivalents and short-term investments. Since inception, we have financed our operations primarily through sales of equity securities, debt financing arrangement, contract payments under our collaboration agreements and from product sales. Based on our current operating plan, we believe that our existing cash, cash equivalents, and short-term investments will be sufficient to fund our expenses and capital expenditure requirements through at least the next 12 months from the date of issuance of this Annual Report on Form 10-K. Use of Estimates The preparation of financial statements in conformity with US GAAP requires management to make certain judgments, estimates and assumptions that could affect the reported amounts of assets and liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting period. Critical accounting estimates We base our estimates and assumptions on historical experience and on various other assumptions we believe to be applicable, and evaluate them on an ongoing basis to ensure they remain reasonable under current conditions. Actual results could differ significantly from those estimates, which could have a material impact on our business, results of operations, and financial condition. Revenue Recognition We recognize revenue in accordance with ASC Topic 606, Revenue from Contracts with Customers Product Sales Revenues from product sales are recognized when the specialty distributors, who are our customers, obtain control of our product, which occurs at a point in time, upon delivery to such specialty distributors. These specialty distributors subsequently resell our products to specialty pharmacy providers, health care providers, hospitals and clinics. In addition to distribution agreements with our specialty distributors, we also have arrangements with certain specialty pharmacy providers, in-office dispensing providers, group purchasing organizations, and government entities that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of our products. Under ASC 606, we are required to estimate the transaction price, including variable consideration that is subject to a constraint, in our contracts with our customers. Variable consideration is included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur. Revenue from product sales is recorded net of certain variable consideration which includes estimated government-mandated rebates and chargebacks, distribution fees, estimated product returns and other deductions. Provisions for returns and other adjustments are provided for in the period the related revenue is recorded. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. The following are our significant categories of sales discounts and allowances: Sales Discounts . We provide certain customer a prompt payment discount that is explicitly stated in our contract. The sales discount is recorded as a reduction of revenue in the period the related product revenue is recognized. Product Returns. Government and Private Payor Rebates: Chargebacks and Discounts: Chargebacks for fees and discounts represent the estimated obligations resulting from contractual commitments to sell products to certain specialty pharmacy providers, in-office dispensing providers, group purchasing organizations, and government entities at prices lower than the list prices charged to our specialty distributors who directly purchase the product from us. These specialty distributors charge us for the difference between what they pay for the product and our contracted selling price to these specialty pharmacy providers, in-office dispensing providers, group purchasing organizations, and government entities. These reserves are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue. Actual chargeback amounts are generally determined at the time of resale to the specialty pharmacy providers, in-office dispensing providers, group purchasing organizations, and government entities by our specialty distributors. The estimated obligations arising from these chargebacks and discounts are recorded as revenue reserves within other accrued liabilities in the balance sheet. Co-Payment Assistance: Contract Revenues from Collaborations In the normal course of business, we conduct research and development programs independently and in connection with our corporate collaborators, pursuant to which we license certain rights to our intellectual property to third parties. The terms of these arrangements typically include payment to us for a combination of one or more of the following: upfront license fees; development, regulatory and commercial milestone payments; product supply services; and royalties on net sales of licensed products. Upfront License Fees: For arrangements that require us to share in the development costs but to which we do not participate in the co-development work, the portion of the upfront fee attributed to our share in the future development costs is excluded from the transaction price. If such share in the development costs is payable beyond 12 months from the delivery of the corresponding license, a significant financing component is deemed to exist. If a significant financing component is identified, we adjust the transaction price by reducing the upfront fee by the net present value of our share in future development costs over the expected commitment period. Such discounted amount will be reported as a liability in the balance sheet, with a corresponding interest expense being accreted based on a discount rate applied over the expected commitment period. Development, Regulatory or Commercial Milestone Payments: Product Supply Services: Sales-based Milestone Payments and Royalties: Government Contracts There is limited US GAAP accounting guidance for for-profit business entities that receives government assistance. We utilized other accounting standards, and have elected to analogize to International Financial Reporting Standards, specifically International Accounting Standards (IAS) 20, Accounting for Government Grants and Disclosures of Government Assistance. Disclosures by Business Entities about Government Assistance, Stock-based Compensation Share-based awards are valued at fair value on the date of grant and that fair value is recognized over the requisite service period, which is generally the vesting period of the respective award. We use the straight-line attribution method over the requisite employee service period for the entire award in recognizing stock-based compensation expense. We account for forfeitures as they occur. The fair value of each option award is estimated on the date of grant using the Black-Scholes option pricing model. The model requires management to make a number of assumptions including expected volatility, expected term, risk-free interest rate and expected dividends. A number of these assumptions are subjective, and their determination generally require judgment. We segregate option awards into the following three homogenous groups for the purposes of determining fair values of options: officers and directors, all other employees, and consultants. We determine the weighted-average valuation assumptions separately for each of these groups as follows: ● Volatility – We estimate volatility using the historical share price performance over the expected life of the option up to the point where we have historical market data. We also consider other factors, such as implied volatility, our current clinical trials and other company activities that may affect the volatility of our stock in the future. We determined that at this time historical volatility is more indicative of our expected future stock performance than implied volatility. ● Expected term – We analyze various historical data to determine the applicable expected term for each of the other option groups. This data includes: (1) for exercised options, the term of the options from option grant date to exercise date; (2) for cancelled options, the term of the options from option grant date to cancellation date, excluding non-vested option forfeitures; and (3) for options that remained outstanding at the balance sheet date, the term of the options from option grant date to the end of the reporting period and the estimated remaining term of the options. The consideration and calculation of the above data gives us reasonable estimates of the expected term for each employee group. We also consider the vesting schedules of the options granted and factors surrounding exercise behavior of the option groups, our current market price and company activity that may affect our market price. In addition, we consider the optionee type (i.e., officers and directors or all other employees) and other factors that may affect the expected term of the option. For options granted to consultants, we use the contractual term of the option, which is generally 10 years, for the initial valuation of the option and the remaining contractual term of the option for the succeeding periods. ● Risk-free interest rate – The risk-free interest rate is based on US Treasury constant maturity rates with similar terms to the expected term of the options for each option group. ● Dividend yield – The expected dividend yield is 0% as we have not paid and do not expect to pay dividends in the future. We grant performance-based stock options to purchase shares of our common stock which will vest upon the achievement of certain corporate performance-based milestones. We determine the fair values of these performance-based stock options using the Black-Scholes option pricing model at the date of grant. For the portion of the performance-based stock options of which the performance condition is considered probable of achievement, we recognize stock-based compensation expense on the related estimated grant date fair values of such options on a straight-line basis from the date of grant up to the date when we expect the performance condition will be achieved. For the performance conditions that are not considered probable of achievement at the grant date or upon re-evaluation at each reporting date, prior to the event actually occurring, we recognize the related stock-based compensation expense when the event occurs or when we can determine that the performance condition is probable of achievement. In those cases, we recognize the change in estimate at the time we determine the condition is probable of achievement (by recognizing stock-based compensation expense as cumulative catch-up adjustment as if we had estimated at the grant date that the performance condition would have been achieved) and recognize the remaining compensation cost up to the date when we expect the performance condition will be achieved, if any. The fair value of the RSU grant is based on the market price of our common stock on the date of grant. Accounts Receivable Accounts receivable are recorded net of customer allowances for prompt payment discounts and any allowance for doubtful accounts. We monitor the financial performance and creditworthiness of our customers so that we can properly assess and respond to changes in their credit profile. We have not historically experienced credit losses and no amounts were reserved for estimated losses as of the balance sheet dates presented. The following table summarizes the activity of our customer allowances for prompt payment discounts for the periods presented (in thousands): Year Ended December 31, 2023 2022 2021 Balance at the beginning of the year $ 136 $ 106 $ 171 Provision for prompt payment discount 686 557 609 Reduction in prompt payment discount (642) (527) (674) Balance at end of the year $ 180 $ 136 $ 106 Concentration of Credit Risk Financial instruments that potentially subject us to a concentration of credit risk are primarily cash, investment in debt securities and accounts receivable. All of our cash and investment in debt securities are maintained with financial institutions that management believes are creditworthy. By policy, we limit the concentration of credit risk by diversifying our investments among a variety of high credit-quality issuers. Due to the short-term nature of these investments, we believe we do not have a material exposure to credit risk arising from our investments. We have not historically experienced any significant credit losses related to these financial instruments and do not believe that we are exposed to any significant credit risk related to these instruments. Concentration of credit risk with respect to our accounts receivable is limited due to our small number of customers. Our accounts receivable consists mostly of outstanding invoices from our sale of our product to our specialty distributors. Accounts receivable may also include outstanding invoices from our collaboration partners with respect to the related sponsored research and license agreements and government contracts. As of December 31, 2023, 87% of our accounts receivable consisted of outstanding invoices from our specialty distributors, and the remaining 13% are outstanding invoices from our collaboration partners, mainly Grifols. As of December 31, 2022, 49% of our accounts receivable are outstanding invoices from our specialty distributors, and the remaining 51% are outstanding invoices from our collaboration partners, mainly Kissei which include a $20.0 million regulatory milestone receivable that was subsequently collected in January 2023. See “Note 3 - Revenues” for summary of revenues from each of our customers who individually accounted for 10% or more of the total net product sales and revenues from collaborations. Cash, Cash Equivalents and Short-Term Investments Our investment in debt securities consists of money market funds, US treasury bills, government- sponsored enterprise securities, and corporate bonds and commercial paper. All of our investment in debt securities are available-for-sale and are classified based on their maturities. We consider all highly liquid investments in debt securities with maturity of 90 days or less from the date of purchase to be cash equivalents. All other investments with maturity greater than 90 days from the date of purchase are classified as short-term investments. Unrealized gains (losses) are reported within the statements of stockholders’ (deficit) equity and comprehensive income (loss). The cost of securities sold is based on the specific identification method. We periodically evaluate our available-for-sale marketable debt securities for impairment. When the fair value of a marketable debt security is below its amortized cost, the amortized cost is reduced to its fair value if it is more likely than not that we are required to sell the impaired security before recovery of our amortized cost basis, or we have the intention to sell the security. If neither of these conditions are met, we determine whether the impairment is due to credit losses by comparing the present value of the expected cash flows of the security with its amortized cost basis. The amount of impairment recognized is limited to the excess of the amortized cost over the fair value of the security. An allowance for credit losses for the excess of amortized cost over the expected cash flows is recorded in other income (expense), net on the statements of operations. Impairment losses that are not credit-related are included in accumulated other comprehensive income (loss) in stockholders’ (deficit) equity. Fair Value of Financial Instruments The carrying amounts of our financial instruments, including cash, accounts receivable, accounts payable and accrued liabilities, approximate fair value due to their relatively short maturities. The carrying value of our loans payable and other long-term debt approximates fair value based on management’s estimation that a current interest rate would not differ materially from the stated rate, or the discount rate applied. The fair value of our cash equivalents and short-term investments measured at fair value on a recurring basis and are categorized based upon the lowest level of significant input to the valuations. Assets and liabilities recorded at fair value in our financial statements are categorized based upon the level of judgment associated with the inputs used to measure their fair value. Fair value is an exit price, representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. ● Level 1 – Inputs are unadjusted, quoted prices in active markets for identical assets at the reporting date. Active markets are those in which transactions for the asset or liability occur in sufficient frequency and volume to provide pricing information on an ongoing basis. The fair valued assets we hold that are generally included under this Level 1 are money market securities where fair value is based on publicly quoted prices. ● Level 2 – Inputs, other than quoted prices included in Level 1, that are either directly or indirectly observable for the asset or liability through correlation with market data at the reporting date and for the duration of the instrument’s anticipated life. The fair valued assets we hold that are generally assessed under Level 2 included government-sponsored enterprise securities, US treasury bills and corporate bonds and commercial paper. We utilize third-party pricing services in developing fair value measurements where fair value is based on valuation methodologies such as models using observable market inputs, including benchmark yields, reported trades, broker/dealer quotes, bids, offers and other reference data. We use quotes from external pricing service providers and other on-line quotation systems to verify the fair value of investments provided by our third-party pricing service providers. ● Level 3 – Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities and which reflect management’s best estimate of what market participants would use in pricing the asset or liability at the reporting date. Consideration is given to the risk inherent in the valuation technique and the risk inherent in the inputs to the model. We do not have fair valued assets classified under Level 3. Inventories and Cost of Product Sales Inventories are stated at the lower-of-cost or estimated net realizable value. We determine the cost of inventories using the standard cost method, which approximates actual cost, and is valued using the first-in, first-out method. Inventory costs primarily consist of active pharmaceutical ingredients, third-party manufacturing costs and allocated internal overhead costs. We capitalize inventory costs when the product is approved by the FDA, or when based on management’s judgment, future commercialization was considered probable, and the future economic benefits are expected to be realized. Prior to FDA approval of a product, costs to purchase active pharmaceutical ingredients including costs to manufacture a product are charged to research and development expense when incurred. Our physical inventories as of balance sheet dates include inventory quantities where costs have been previously charged to research and development expense since such costs were incurred prior to FDA approval of the product. We provide reserves for potential excess, dated or obsolete inventories based upon assumptions about future demand and market conditions, as well as product shelf life. Inventories that are not expected to be consumed beyond our normal operating cycle are classified as non-current inventories and included within other assets in the balance sheet. Cost of product sales primarily includes cost of inventories sold, and product shipping and handling costs. Further, following the approval of REZLIDHIA, we recognize the amortization expense of capitalized intangible asset and royalty expense incurred pursuant to our license agreement with Forma, within cost of sales. Property and Equipment Property and equipment are stated at cost net of accumulated depreciation. Depreciation is calculated using the straight-line method over the estimated useful lives of the assets, which range from three Research and Development Expenses Research and development expenses include costs incurred to conduct research and development, including scientific personnel wages and associated employee benefits, research and development supplies and equipment, payments to collaborative clinical research partners, consulting fees and other various research and development related costs. We have various contracts with third parties related to our research and development activities, including strategic development collaborations. Costs incurred but not billed to us as of the end of the period are accrued. We make estimates of the amounts incurred in each period based on the information available to us and our knowledge of the nature of the contractual activities generating such costs. Clinical trial contract expenses are accrued based on units of activity. Expenses related to other research and development contracts, such as research contracts, toxicology study contracts and manufacturing contracts are estimated to be incurred generally on a straight-line basis over the duration of the contracts. Raw materials and study materials not related to an approved drug are charged to research and development expenses at the time of purchase. We make significant judgments and estimates in determining the accrual balance in each reporting period. As actual costs become known, we adjust our accruals. Although we do not expect our estimates to be materially different from amounts actually incurred, such estimates for the status and timing of services performed relative to the actual status and timing of services performed may vary and could result in us reporting amounts that are too high or too low in any particular period. Variations in assumptions used to estimate accruals including, but not limited to, the number of patients enrolled, the rate of patient enrollment and the actual services performed may result in adjustments in research and development accruals in future periods. Changes in these estimates that result in material changes to our accruals could materially affect our financial condition and results of operations. Research and development expenses also include milestone payment obligations incurred prior to r egulatory approval of the product, which are accrued when the event requiring payment of the milestone occurs. See related discussions under “IPR&D/Intangible Asset” below. IPR&D/Intangible Asset In July 2022, we entered into a license and transition services agreement with Forma. The transaction was accounted for as an acquisition of asset under ASC 730, Research and Development account for m We perform an impairment review of intangible asset whenever events or changes in business circumstances indicate the carrying amount of the asset may not be fully recoverable. If events or changes in circumstances suggest that the carrying amount of the finite-lived intangible assets may not be recoverable, we will estimate the future cash flows expected to be generated by the asset from its use or eventual disposition. If the expected future undiscounted cash flows is less than the carrying amount of the assets, we will recognize an impairment loss based on the excess of the carrying amount over the fair value of the assets. Advertising Expense Advertising costs are expensed as incurred and are included within selling general and administrative expenses in the statements of operations. Advertising costs for the years ended December 31, 2023, 2022 and 2021 amounted to $3.1 million, $2.7 million, and $2.3 million, respectively. Leases We account for leases in accordance with ASU No. 2016-02 , Leases (Topic 842) . Topic 842 requires a lessee to determine if an arrangement is a lease or contains a lease at contract inception. For our sublease agreement wherein we were the lessor, we recognized sublease income on a straight-line basis over the term of the related sublease agreement. Restructuring Restructuring costs comprised severance, other termination benefit costs, stock-based compensation expense for stock award and stock option modifications related to workforce reductions and accelerated depreciation. We recognize restructuring charges when the liability is probable, and the amount is estimable. Employee termination benefits are accrued at the date management has committed to a plan of termination and affected employees have been notified of their termination date and expected severance benefits. Income Taxes We use the asset and liability method to account for income taxes. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases and operating loss and tax credit carryforwards. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities from a change in tax rates is recognized in income in the period the change is enacted. A valuation allowance is established to reduce deferred tax assets to an amount whose realization is more likely than not. Recent Accounting Pronouncements In November 2023, FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures. In December 2023, FASB issued ASU 2023-09, Improvements to Income Tax Disclosures Other recently issued accounting guidance not discussed in this Annual Report on Form 10-K are either not applicable or did not have, or are not expected to have, a material impact on us. |
NET LOSS PER SHARE
NET LOSS PER SHARE | 12 Months Ended |
Dec. 31, 2023 | |
NET LOSS PER SHARE | |
NET LOSS PER SHARE | 2. NET LOSS PER SHARE Basic net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock outstanding during the period. Diluted net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock outstanding during the period and the number of additional shares of common stock that would have been outstanding if potentially dilutive securities had been issued. Potentially dilutive securities include stock options, RSUs and shares issuable under our Purchase Plan. The dilutive effect of these potentially dilutive securities is reflected in diluted earnings per share using the treasury stock method. Under the treasury stock method, an increase in the fair market value of our common stock can result in a greater dilutive effect from potentially dilutive securities. The potential shares of common stock that were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive are as follows (in thousands): Year Ended December 31, 2023 2022 2021 Outstanding stock options 34,119 34,696 30,009 RSUs 1,866 1,104 226 Total 35,985 35,800 30,235 |
REVENUES
REVENUES | 12 Months Ended |
Dec. 31, 2023 | |
REVENUES | |
REVENUES | 3 REVENUES Revenues disaggregated by category were as follows (in thousands): Year Ended December 31, 2023 2022 2021 Product sales: Gross product sales $ 147,058 $ 108,523 $ 81,186 Discounts and allowances (42,764) (31,805) (18,176) Total product sales, net 104,294 76,718 63,010 Revenues from collaborations: License revenues — 7,932 70,553 Milestone revenue 75 25,000 1,875 Delivery of drug supplies, royalty and others 11,413 6,092 3,298 Total revenues from collaborations 11,488 39,024 75,726 Government contracts 1,100 4,500 10,500 Total revenues $ 116,882 $ 120,242 $ 149,236 Revenue from product sales is related to sales of our commercial products, TAVALISSE and REZLIDHIA, to our customers which are specialty distributors. For detailed discussions of our revenues from collaboration and government contracts, see “Note 4 – Sponsored Research and License Agreements and Government Contracts.” Our product sales revenue is net of chargebacks, discounts and fees, government and other rebates and returns. Of the total discounts and allowances from gross product sales for the years ended December 31, 2023, 2022 and 2021, $41.5 million, $30.9 million and $16.8 million, respectively, was accounted for as additions to revenue reserves and refund liability, and $1.3 million, $0.9 million and $1.4 million, respectively, as reductions in accounts receivable (as it relates to allowance for prompt pay discount) and prepaid and other current assets (as it relates to certain chargebacks and other fees that were prepaid) in the balance sheet. The following tables summarize the activities in chargebacks, discounts and fees, government and other rebates and returns that were accounted for revenue reserves and refund liability, for each of the periods presented (in thousands): Chargebacks, Government Discounts and and Other Fees Rebates Returns Total Balance as of January 1, 2023 $ 6,213 $ 2,636 $ 3,296 $ 12,145 Provision related to current period sales 32,330 8,299 869 41,498 Credit or payments made during the period (30,307) (7,418) (234) (37,959) Balance as of December 31, 2023 $ 8,236 $ 3,517 $ 3,931 $ 15,684 Chargebacks, Government Discounts and and Other Fees Rebates Returns Total Balance as of January 1, 2022 $ 3,404 $ 2,494 $ 2,017 $ 7,915 Provision related to current period sales 23,488 5,901 1,514 30,903 Credit or payments made during the period (20,679) (5,759) (235) (26,673) Balance as of December 31, 2022 $ 6,213 $ 2,636 $ 3,296 $ 12,145 The following table summarizes revenues from each of our customers who individually accounted for 10% or more (wherein * denotes less than 10%) of the total net product sales and revenues from collaborations: Year Ended December 31, 2023 2022 2021 McKesson Specialty Care Distribution Corporation 43% 31% 20% Cardinal Healthcare 25% 19% * ASD Healthcare and Oncology Supply 21% 17% 17% Kissei * 24% * Lilly * * 48% |
SPONSORED RESEARCH AND LICENSE
SPONSORED RESEARCH AND LICENSE AGREEMENTS AND GOVERNMENT CONTRACT | 12 Months Ended |
Dec. 31, 2023 | |
SPONSORED RESEARCH AND LICENSE AGREEMENTS AND GOVERNMENT CONTRACT | |
SPONSORED RESEARCH AND LICENSE AGREEMENTS AND GOVERNMENT CONTRACT | 4. SPONSORED RESEARCH AND LICENSE AGREEMENTS AND GOVERNMENT CONTRACTS Sponsored Research and License Agreements We conduct research and development programs independently and in connection with our corporate collaborators. As of December 31, 2023, we are a party to collaboration agreements with Lilly to develop and commercialize R552, a RIPK1 inhibitor, for the treatment of non-CNS diseases and collaboration aimed at developing additional RIPK1 inhibitors for the treatment of CNS diseases; with Grifols to commercialize fostamatinib for human diseases in all indications, including chronic ITP and AIHA, in Grifols territory which includes Europe, the UK, Turkey, the Middle East, North Africa and Russia (including Commonwealth of Independent States); with Kissei to develop and commercialize fostamatinib in Kissei territory which includes Japan, China, Taiwan and the Republic of Korea; with Medison to commercialize fostamatinib in all indications, including chronic ITP and AIHA, in Medison territory which includes Canada and Israel; and with Knight to commercialize fostamatinib in all indications, including chronic ITP and AIHA, in Knight territory which includes Latin America, consisting of Mexico, Central and South America, and the Caribbean. Further, we are also a party to collaboration agreements, but do not have ongoing performance obligations with BerGenBio for the development and commercialization of AXL inhibitors in oncology, and with Daiichi to pursue research related to MDM2 inhibitors, a novel class of drug targets called ligases. Global Exclusive License Agreement with Lilly We have a global exclusive license and collaboration agreement with Lilly (Lilly Agreement) entered in February 2021, which became effective on March 27, 2021, upon clearance under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, to develop and commercialize R552 for the treatment of non-CNS diseases. In addition, the collaboration is aimed at developing additional RIPK1 inhibitors for the treatment of CNS diseases. Pursuant to the terms of the Lilly agreement, we granted Lilly the exclusive rights to develop and commercialize R552 and related RIPK1 inhibitors in all indications worldwide. The parties’ collaboration is governed through a joint governance committee and appropriate subcommittees. Under the terms of Lilly Agreement, we were entitled to receive a non-refundable and non-creditable upfront cash payment amounting to $125.0 million, which we received in April 2021. We are also entitled to additional milestone payments for non-CNS disease products consisting of up to $330.0 million milestone payments upon the achievement of specified development and regulatory milestones, and up to $100.0 million in sales milestone payments on a product-by-product basis. In addition, depending on the extent of our co-funding of R552 development activities, we would be entitled to receive tiered royalty payments on net sales of non-CNS disease products at percentages ranging from the mid-single digits to high-teens, subject to certain standard reductions and offsets. We are also eligible to receive milestone payments for CNS disease products consisting of up to $255.0 million in milestone payments upon the achievement of specified development, regulatory and commercial milestones, and up to $150.0 million in sales milestone payments on a product-by-product basis. We would be entitled to receive tiered royalty payments on net sales of CNS disease products up to low-double digits, subject to certain standard reductions and offsets. Under the Lilly Agreement, we are responsible for performing and funding initial discovery and identification of CNS disease development candidates. Following candidate selection, Lilly will be responsible for performing and funding all future development and commercialization of the CNS disease development candidates. We are responsible for 20% of development costs for R552 in the US, Europe, and Japan, up to a specified cap. Lilly is responsible for funding the remainder of all development activities for R552 and other non-CNS disease development candidates. Pursuant to the terms of the Lilly Agreement, we have the right to opt-out of co-funding the R552 development activities in the US, Europe and Japan at two different specified times and as a result receive lesser royalties from sales. Prior to us providing our first opt-out notice and the amendment to the Lilly Agreement as discussed below, under the Lilly Agreement, we were required to fund our share of the R552 development activities in the US, Europe, and Japan up to a maximum funding commitment of $65.0 million through April 1, 2024. On September 28, 2023, we entered into an amendment to the Lilly Agreement which provided, among others that if we exercise our first opt-out right, we have the right to opt-in to the co-funding of R552 development, upon us providing notice to Lilly within 30 days of certain events as specified in the Lilly Agreement, and as a result receive greater royalties from sales. If we decide to exercise our opt-in right, we will be required to continue to share in global development costs, and if we later exercise our second opt-out right (no later than April 1, 2025), our share in global development costs will be up to a specified cap through December 31, 2025, as provided for in the Lilly Agreement. On September 29, 2023, we provided the first opt-out notice to Lilly. We continue to fund our share of the R552 development activities up to $22.6 million through April 1, 2024, as provided for in the amended Lilly Agreement. We accounted for this agreement under ASC 606 and identified the following distinct performance obligations at inception of the agreement: (a) granting of the license rights over the non-CNS penetrant intellectual property (IP), and (b) granting of the license rights over the CNS penetrant IP which will be delivered to Lilly upon completion of the additional research and development efforts specified in the agreement. We concluded each of these performance obligations is distinct. We based our assessment on the assumption that Lilly can benefit from each of the licenses on its own by developing and commercializing the underlying product using its own resources. At the inception of the Lilly Agreement, given our rights to opt-out from the development of R552, we believed at the minimum, we had a commitment to fund the development costs up to $65.0 million as discussed above. We considered this commitment to fund the development costs as a significant financing component of the contract, which we accounted for as a reduction of the upfront fee to derive the transaction price. This financing component was recorded as a liability at its net present value of approximately $57.9 million using a 6.4% discount rate. Interest expense is accreted on such liability over the expected commitment period and adjusted for timing of expected cost share payments. No interest, $0.7 million and $2.8 million of interest, was accreted during the year ended December 31, 2023, 2022 and 2021, respectively. Through December 31, 2023, Lilly billed us $18.6 million for our share of development costs under this agreement, of which, $0.8 million was outstanding as of December 31, 2023 and was subsequently paid in January 2024. As of December 31, 2023 and 2022, the outstanding financing liability to Lilly was $43.6 million and $46.2 million, respectively, and included within other long-term liabilities, current portion, and other long-term liabilities in the balance sheet. As discussed above, following the amendment to the Lilly Agreement and us providing the first opt-out notice to Lilly, our cumulative share of the R552 development cost is now capped at $22.6 million through April 1, 2024. Although our cumulative share of the development cost is now at the specified cap that is less than our outstanding recorded liability at the balance sheet date, such excess amount has not been recognized as revenue because we cannot conclude that it is probable that a significant reversal of the amount of revenue, if recognized, will not occur until the likelihood of us exercising our opt-in right becomes remote, or when the opt-in right period lapses. At the inception, we allocated the net transaction price of $67.1 million to each performance obligation based on our best estimate of its relative standalone selling price using the adjusted market assessment approach. The transaction price allocated to the non-CNS penetrant IP of $60.4 million was recognized as revenue in the year ended December 31, 2021 upon delivery of the non-CNS penetrant IP to Lilly in the first quarter of 2021. The transaction price allocated to the CNS penetrant IP of $6.7 million was recognized as revenue from the effective date of the Lilly Agreement through the eventual acceptance by Lilly using the input method. In June 2022, Lilly provided notice of continuance pursuant to the terms of the Lilly Agreement, whereby Lilly elected its option to lead the identification and selection of CNS penetrant lead candidate. As such, we recognized the remaining outstanding deferred revenue in the second quarter of 2022. For the year ended December 31, 2022 and 2021, we recognized revenue of $0.5 million and $6.2 million, respectively, relative to the delivery of CNS penetrant IP. Further, we recognized additional $0.2 million of revenue relative to a separate delivery of CNS compound to Lilly during the year ended December 31, 2022. The remaining future variable consideration related to future milestone payments as discussed above were fully constrained because we cannot conclude that it is probable that a significant reversal of the amount of cumulative revenue recognized will not occur, given the inherent uncertainty of success with these future milestones. For sales-based milestones and royalties, we determined that the license is the predominant item to which the royalties or sales-based milestones relate. Accordingly, we will recognize revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). We will re-evaluate the transaction price in each reporting period and as uncertain events are resolved or other changes in circumstances occur. Grifols License Agreement We have a commercialization license agreement with Grifols entered in January 2019 with exclusive rights to commercialize fostamatinib for human diseases, including chronic ITP and AIHA, and non-exclusive rights to develop, fostamatinib in Grifols territory. Under the agreement, we received an upfront payment of $30.0 million, with the potential for $297.5 million in total regulatory and commercial milestones. We are also entitled to receive stepped double-digit royalty payments based on tiered net sales which may reach 30% of net sales. The agreement also required us to continue to conduct our long-term open-label extension study on patients with ITP through EMA approval of ITP in Europe or until the study ends as well as conduct the Phase 3 trial of fostamatinib in AIHA. In January 2020, the EC granted a centralized MA for fostamatinib valid throughout the EU, and in the UK after the departure of the UK from the EU, for the treatment of chronic ITP in adult patients who are refractory to other treatments. With this approval, in February 2020, we received $20.0 million non-refundable payment, consisted of a $17.5 million payment due upon MAA approval by the EMA of fostamatinib for the first indication and a $2.5 million creditable advance royalty payment, based on the terms of our collaboration agreement with Grifols. The above milestone payment was allocated to the distinct performance obligations in the collaboration agreement with Grifols. We accounted for this agreement under ASC 606 and identified the following distinct performance obligations at inception of the agreement: (a) granting of the license, (b) performance of research and regulatory services related to our ongoing long-term open-label extension study on patients with ITP, and (c) performance of research services related to our Phase 3 trial in AIHA. We allocated the transaction price to the distinct performance obligations based on our best estimate of the relative standalone selling price, and recognized the corresponding revenue in the periods we satisfied the performance obligations. As of December 31, 2023, there was no outstanding deferred revenue. For the years ended December 31, 2023, 2022 and 2021, we recognized an immaterial amount, $0.7 million, $0.9 million, respectively, of revenue associated with the remaining performance obligation to perform research and development services. The remaining future variable consideration related to future regulatory and commercial milestones were fully constrained due to the fact that it was probable that a significant reversal of cumulative revenue would occur, given the inherent uncertainty of success with these future milestones. We are recognizing revenues related the research and regulatory services throughout the term of the respective clinical programs using the input method. For sales-based milestones and royalties, we determined that the license is the predominant item to which the royalties or sales-based milestones relate. Accordingly, we will recognize revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). We will re-evaluate the transaction price in each reporting period and as uncertain events are resolved or other changes in circumstances occur. We have a Commercial Supply Agreement with Grifols entered in October 2020 to supply and sell our drug product priced at a certain markup specified in the agreement, in quantities Grifols shall order from us pursuant to and in accordance with the agreement. For the years ended December 31, 2023, 2022, and 2021, we recognized $5.6 million, $1.6 million and $2.0 million, respectively, of revenue related to delivery of drug supply to Grifols. Pursuant to our commercial license agreement with Grifols, we began recognizing royalty revenue beginning in the third quarter of 2022, and included such amounts within contract revenues from collaboration. For the years ended December 31, 2023 and 2022, we recognized $3.2 million and $0.7 million, respectively, of royalty revenue. Kissei License Agreement We have an exclusive license and supply agreement with Kissei to develop and commercialize fostamatinib in all current and potential indications in Kissei territory. Kissei is responsible for performing and funding all development activities for fostamatinib in Kissei territories. We received an upfront cash payment of $33.0 million, with the potential for up to an additional $147.0 million in development, regulatory and commercial milestone payments, and will receive mid- to upper twenty percent, tiered, escalated net sales-based payments for the supply of fostamatinib. Under the agreement, we granted Kissei the license rights to fostamatinib in Kissei territory and are obligated to supply Kissei with drug product for use in clinical trials and pre-commercialization activities. We are also responsible for the manufacture and supply of fostamatinib for all future development and commercialization activities under the agreement. We accounted for this agreement under ASC 606 and identified the following distinct performance obligations at inception of the agreement: (a) granting of the license, (b) supply of fostamatinib for clinical use and (c) material right associated with discounted fostamatinib that is supplied for use other than clinical or commercial. In addition, we will provide commercial product supply if the product is approved in the licensed territory. We concluded that each of these performance obligations is distinct. We determined that the upfront fee of $33.0 million represented the transaction price and was allocated to the performance obligations based on our best estimate of the relative standalone selling price and recognized the corresponding revenue in the period we satisfied the performance obligations. As of December 31, 2023 and 2022, the remaining deferred revenue was related to the material right associated with discounted fostamatinib supply which amounted to $1.4 million. There were no material revenue recognized associated with the remaining performance obligation during the years ended December 31, 2023, 2022 and 2021. In April 2022, Kissei announced that an NDA was submitted to Japan’s PMDA for fostamatinib in chronic ITP. With this milestone event, we received $5.0 December 31, 2022 2023 . The remaining variable consideration related to future development and regulatory milestones was fully constrained because we cannot conclude that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur, given the inherent uncertainty of success with these future milestones. We recognize revenues related to the supply of fostamatinib and material right upon delivery of fostamatinib to Kissei. For sales-based milestones and royalties, we determined that the license is the predominant item to which the royalties or sales-based milestones relate to. Accordingly, we recognize revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). We re-evaluate the transaction price in each reporting period and as uncertain events are resolved or other changes in circumstances occur. Pursuant to our supply agreement with Kissei, during the years ended December 31, 2023, 2022 and 2021, we recognized $2.2 million, $2.6 million, and $0.3 million, respectively, of revenue related to delivery of drug supplies to Kissei. Medison Commercial and License Agreements We have two exclusive commercial and license agreements with Medison entered in October 2019 for the commercialization of fostamatinib for chronic ITP in Medison territory. Pursuant to which, we received a $5.0 million upfront payment with respect to the agreement in Canada. We accounted for the agreement made with an upfront payment under ASC 606 and identified the following combined performance obligations at inception of the agreement: (a) granting of the license and (b) obtaining regulatory approval in Canada of fostamatinib in ITP. We determined that the non-refundable upfront fee represented the transaction price. However, under the agreement, we have the option to buy back all rights to the product in Canada within six months from obtaining regulatory approval for the treatment of AIHA in Canada. The buyback option precludes us from transferring control of the license to Medison under ASC 606. We believe that the buyback provision, if exercised, will require us to repurchase the license at an amount equal to or more than the upfront fee. As such this arrangement is accounted for as a financing arrangement. Interest expense was accreted on such liability over the expected buyback period. We also billed Medison for the delivery of drug supplies for clinical use which we deferred and included within the outstanding financing liability considering the buy-back provision. The decision to exercise the buyback option is dependent on many factors including management’s cost and benefit assessment and the success of obtaining regulatory approval for the treatment of AIHA in Canada. In June 2022, we reported the top-line results from our Phase 3 trial of fostamatinib in wAIHA which showed that the trial did not demonstrate statistical significance in the primary efficacy endpoint in the overall study population. We also announced in early October 2022 that filing an sNDA for wAIHA indication was remote considering the top-line data results and the guidance received from the FDA. With these developments, we assessed our options path forward, including our buyback option right with regards to the Medison license agreement. Based on management’s assessment, it was concluded that exercising the buyback option right is remote. As such, we relieved the outstanding financing liability to Medison amounting to $5.7 million in the fourth quarter of 2022 and recognized such amount as collaboration revenue in accordance with ASC 606 in the year ended December 31, 2022. No remaining outstanding financing liability from Medison as of December 31, 2023 and 2022 During the year ended December 31, 2023, we recognized $0.5 million of revenue from Medison related to the delivery of drug supplies, royalty revenue, and a milestone pursuant to the commercial and license agreement. Revenue from Medison of $5.7 million during the year ended December 31, 2022 was related to the release of financing liability as discussed above. During the year ended December 31, 2021, we recognized $0.1 million of revenue from Medison related to achievement of certain milestones. Knight Commercial License and Supply Agreement We have commercial license and supply agreements with Knight entered in May 2022 for the commercialization of fostamatinib for approved indications in Knight territory. Pursuant to such commercial license agreement, we received a $2.0 million one-time, non-refundable, and non-creditable upfront payment, with potential for up to an additional $20.0 million in regulatory and sales-based commercial milestone payments, and will receive twenty- to mid-thirty percent, tiered, escalated net-sales based royalty payments for products sold in the Knight territory. We accounted for this agreement under ASC 606 and identified that the upfront payment was a consideration for granting Knight the license to commercialize fostamatinib for approved indication in the Knight territory, and no further material deliverables associated to such upfront payment. As such, we recognized the upfront payment as revenue during the year ended December 31, 2022 circumstances occur. We are also responsible for the exclusive manufacture and supply of fostamatinib for all future development and commercialization activities under the agreement. Daiichi Collaboration Agreement Pursuant to the Amended Collaboration Agreement entered in April 2005 with Daiichi, during the year ended December 31, 2021, we recognized $1.8 million of revenue related to the achievement of a certain milestone. All deliverables under the agreement had been previously delivered, and as such the revenue was recognized in the period such milestone was achieved. Other license agreements In February 2021, we entered into a non-exclusive license agreement with an unrelated third party whereby we granted such unrelated third-party rights to a certain patent. In consideration for the license rights granted, we received a one-time fee of $4.0 million. All the deliverables under the agreement had been delivered and the one-time fee was recognized as revenue Government Contracts DOD In January 2021, we were awarded up to $16.5 million by the DOD to support our ongoing Phase 3 clinical trial to evaluate the safety and efficacy of fostamatinib for the treatment of hospitalized high-risk patients with COVID-19. For the years ended December 31, 2023, 2022 and 2021, we recognized $1.0 million, $4.5 million and $10.5 million, respectively, of revenue related to this grant. Through December 31, 2023, we received $16.0 million of the award. BARDA In August 2023, we were awarded up to $0.8 million by BARDA for our evaluation of fostamatinib in mitigating the impact of long-term respiratory distress. For the year ended December 31, 2023, we recognized $0.1 million of revenue related to this grant. License and Transition Services Agreement with Forma We have a license and transition services agreement with Forma entered in July 2022, for an exclusive license to develop, manufacture and commercialize olutasidenib, a proprietary inhibitor of mutated IDH1 (mIDH1), for any uses worldwide, including for the treatment of AML and other malignancies. Forma became a wholly owned subsidiary of Novo Nordisk following the closing of its acquisition by Novo Nordisk in October 2022. Pursuant to the terms of the license and transition services agreement, we paid an upfront fee of $2.0 million, with the potential to pay up to $67.5 million of additional payments upon achievement of specified development and regulatory milestones and up to $165.5 million of additional payments upon achievement of certain commercial milestones. In addition, subject to the terms and conditions of the license and transition services agreement, Forma would be entitled to tiered royalty payments on net sales of licensed products at percentages ranging from low-teens to mid-thirties, as well as certain portion of our sublicensing revenue, subject to certain standard reductions and offsets. The transaction was accounted for as an acquisition of asset under ASC 730, Research and Development At the acquisition date, we accounted for the upfront fee of $2.0 million Under the accounting guidance, we account for contingent cash payments when it is probable that a liability is incurred and the amount can be reasonably estimated. We account for m at the commercial stage, are recorded as intangible asset when the event requiring payment of the milestones occurs. Prior to the FDA approval of REZLIDHIA in December 2022, we achieved certain regulatory milestone which entitled Forma to receive a $2.5 million milestone payment. Because such milestone payment obligation was incurred prior to a regulatory approval of an indication associated with the acquired licensed asset, we recorded such amount as research and development expense during the year ended December 31, 2022. On December 1, 2022, the FDA approved REZLIDHIA capsules for the treatment of adult patients with R/R AML with susceptible IDH1 mutations as detected by an FDA-approved test. Following the FDA approval, we launched REZLIDHIA and made first shipments of the product to our customers in December 2022. With this FDA approval and first commercial sale of the product, Forma was entitled to receive a total of $15.0 million in milestone payments. Since such milestone payment obligations were incurred upon and after regulatory approval of the product, we recorded such amount as intangible asset on our balance sheet. Such amount was outstanding as of December 31, 2022 and included within accounts payable in our balance sheet, which was subsequently paid in the first quarter of 2023. The amount recorded as intangible asset is being amortized over the estimated useful life of the acquired licensed asset. Royalty expense related to the acquired licensed asset is recognized when incurred. For the year ended December 31, 2023 and 2022, we recognized $1.1 million and $0.1 million, respectively, of amortization of intangible asset, and $1.6 million and $0.1 million, respectively, of royalty expense related to the license and transition services agreement as discussed above. Such costs were included within cost of sales in our statements of operations. Strategic Development Collaborations with MD Anderson and CONNECT In December 2023, we entered into a Strategic Collaboration Agreement with MD Anderson, a comprehensive cancer research, treatment, and prevention center. The collaboration will expand our evaluation of REZLIDHIA (olutasidenib) in AML and other hematologic cancers. Under the collaboration, we will provide MD Anderson the study materials and $15.0 million in time-based milestone payments as compensation for services to be provided for the studies, over the five-year collaboration term, unless terminated earlier as provided for in the agreement. In December 2023, we provided $2.0 million funding to MD Anderson. In January 2024, we announced our collaboration with CONNECT, an international collaborative network of pediatric cancer centers, to conduct a Phase 2 clinical trial to evaluate REZLIDHIA (olutasidenib) in glioma. Under the collaboration, we will provide funding up to $3.0 million and study material over the four-year collaboration. We account for the funding we provide under the above research collaboration agreements as prepaid research and development in the balance sheet to the extent the payment is made in advance of services being rendered, and recognize such amount as research and development expense within the statements of operations as the collaborative partners render the services under the respective agreement. |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 12 Months Ended |
Dec. 31, 2023 | |
STOCK-BASED COMPENSATION | |
STOCK-BASED COMPENSATION | 5. STOCK-BASED COMPENSATION Total stock-based compensation expense related to all of our stock-based awards was as follows (in thousands): Year Ended December 31, 2023 2022 2021 Selling, general and administrative $ 6,712 $ 10,217 $ 7,337 Research and development 2,094 2,168 1,700 Restructuring charges — — 449 Total stock-based compensation expense $ 8,806 $ 12,385 $ 9,486 Stock-based compensation expense for the years ended December 31, 2023, 2022 and 2021 include incremental stock-based compensation charges of approximately $0.5 million, $1.4 million and $0.4 million, respectively, as a result of modifications of certain stock option grants. The stock option modifications during the years ended December 31, 2023 and 2022 were related to acceleration of vesting of certain stock option grants and extension of exercise period of vested stock options granted to former officers and board of directors. Such amounts were included within selling, general and administrative expenses and research and development expenses. The stock option modification in 2021 was related to the reduction of workforce wherein certain affected employees were provided extension to exercise certain vested stock options. Such amount was recorded within restructuring charges. Equity Incentive Plans n May 2023, our stockholders approved an amendment to our 2018 Plan, to, among other items, add an additional 4,000,000 shares to the number of shares of common stock authorized for issuance under our 2018 Plan Stock Options and RSUs The following table summarizes stock options and RSUs activity, and shares available for grant under our Equity Incentive Plans for the periods presented: Stock Options RSUs Weighted Weighted Weighted Average Shares Available Number of Average Intrinsic Value Number of Grant Date For Grant Shares Exercise Price (in thousands) Shares Fair Value Outstanding as of December 31, 2022 10,612,618 34,696,273 $ 2.74 $ 1,605 1,103,653 $ 2.39 Authorized for grant 5,019,700 Granted (5,669,944) 3,671,800 $ 1.69 1,387,600 $ 1.80 Exercised/Released — (50,161) $ 0.97 (435,006) $ 2.32 Cancelled and forfeited 3,908,921 (4,199,147) $ 3.29 (190,356) $ 2.20 Outstanding as of December 31, 2023 13,871,295 34,118,765 $ 2.56 $ 1,453 1,865,891 $ 1.98 Vested and expected to vest as of December 31, 2023 31,373,765 $ 2.55 $ 1,349 Exercisable as of December 31, 2023 24,378,890 $ 2.70 $ 403 Of the total stock options outstanding as of December 31, 2023, 2,745,000 shares outstanding are performance-based stock options wherein the achievements of the corresponding corporate-based milestones were not probable. Accordingly, the related grant date fair value for these performance-based stock options of $5.1 million has not been recognized as stock-based compensation expense as of December 31, 2023. For the years ended December 31, 2023, 2022 and 2021, stock options vested were 4,346,977 shares, 5,280,235 shares and 4,765,814 shares, respectively, with weighted-average exercise price of $2.34 per share, $2.60 per share, and $2.67 per share, respectively. The aggregate intrinsic values of stock options outstanding, vested and expected to vest, and exercisable For the years ended December 31, 2023, 2022 and 2021, we granted options to purchase 3,671,800 shares, 8,179,113 shares and 6,997,981 shares, respectively, of common stock, with The following table summarizes the weighted-average assumptions relating to stock options granted during the periods presented: Year Ended December 31, 2023 2022 2021 Risk-free interest rate 3.9 % 2.6 % 1.0 % Expected term (in years) 6.8 6.3 6.4 Dividend yield 0.0 % 0.0 % 0.0 % Expected volatility 84.0 % 74.8 % 70.5 % As of December 31, 2023, there was approximately $10.6 million of unrecognized stock-based compensation cost which is expected to be recognized over the remaining weighted-average period of 2.45 Details of our stock options by exercise price are as follows as of December 31, 2023: Options Outstanding Options Exercisable Weighted-Average Number of Remaining Contractual Weighted-Average Number of Weighted-Average Exercise Price Shares Life (in years) Exercise Price Shares Exercise Price $0.90 - $1.87 6,712,896 8.61 $ 1.43 1,991,518 $ 1.44 $1.96 - $2.11 5,063,237 4.44 $ 2.03 5,063,237 $ 2.03 $2.14 - $2.40 3,940,279 2.89 $ 2.26 3,717,256 $ 2.26 $2.42 - $2.42 5,816,323 6.81 $ 2.42 3,794,160 $ 2.42 $2.44 - $3.54 6,852,637 5.14 $ 3.16 4,473,712 $ 3.06 $3.59 - $4.49 5,542,956 3.95 $ 3.97 5,215,904 $ 3.99 $4.50 - $4.50 190,437 7.29 $ 4.50 123,103 $ 4.50 $0.90 - $4.50 34,118,765 5.56 $ 2.56 24,378,890 $ 2.70 Employee Stock Purchase Plan Our Purchase Plan permits eligible employees to purchase common stock at a discount through payroll deductions during defined offering periods. Our Purchase Plan provides for a 24-month offering period comprised four six-month purchase periods with a look-back option. A look-back option is a provision in our Purchase Plan under which eligible employees can purchase shares of our common stock at a price per share equal to the lesser of 85% of the fair market value on the first day of the offering period or 85% of the fair market value on the purchase date. Our Purchase Plan also includes a feature that provides for a new offering period to begin when the fair market value of our common stock on any purchase date during an offering period falls below the fair market value of our common stock on the first day of such offering period. This feature is called a “reset.” Participants are automatically enrolled in the new offering period. Our previous 24-month offering period under our Purchase Plan ended on June 30, 2022, and a new 24-month offering period started on July 1, 2022. The fair value of awards under our Purchase Plan is estimated on the date of our new offering period using the Black-Scholes option pricing model, which is being amortized over the requisite service periods. The table below summarizes the weighted-average assumptions related to our Purchase Plan for periods presented. Expected volatilities for our Purchase Plan are based on the two-year historical volatility of our stock. Expected term represents the weighted- average of the purchase periods within the offering period. The risk-free interest rate for periods within the expected term is based on US Treasury constant maturity rates. Year Ended December 31, 2023 2022 2021 Risk-free interest rate * 3.1 % * Expected term (in years) * 1.3 * Dividend yield * 0.0 % * Expected volatility * 121.0 % * Weighted average grant date fair value (dollar per share) * $0.78 * * Not a measurement period. As of December 31, 2023, unrecognized stock-based compensation cost related to our Purchase Plan amounted to $0.1 million, which is expected to be recognized over the remaining weighted average period of 0.49 For the years ended December 31, 2023, 2022 and 2021, there were 941,798 shares, 1,146,851 shares and 932,018 shares, respectively, of common stock purchased under the Purchase Plan, at an average price of $1.06 per share, $1.01 per share and $1.51 per share, respectively. As of December 31, 2023, there were 2,495,835 shares reserved for future issuance under the Purchase Plan. |
INVENTORIES
INVENTORIES | 12 Months Ended |
Dec. 31, 2023 | |
INVENTORIES | |
INVENTORIES | 6 INVENTORIES The following table summarizes inventories, net (in thousands): As of December 31, 2023 2022 Raw materials $ 4,609 $ 4,555 Work in process 1,876 2,659 Finished goods 1,508 1,904 Total $ 7,993 $ 9,118 Reported as: Inventories $ 5,522 $ 9,118 Other assets 2,471 — Total $ 7,993 $ 9,118 Inventories as of December 31, 2023 and 2022 include inventories acquired from Forma pursuant to the license and transition agreement. As of December 31, 2023 and 2022, zero and $0.8 million, respectively, of advance payments to the manufacturer of our raw materials were included within prepaid and other current assets in the balance sheet. Non-current inventories consist of active pharmaceutical ingredient classified as raw materials which have multi-year shelf life, as well as certain work in process and finished goods inventories that are not expected to be consumed beyond our normal operating cycle. |
CASH, CASH EQUIVALENTS AND SHOR
CASH, CASH EQUIVALENTS AND SHORT-TERM INVESTMENTS | 12 Months Ended |
Dec. 31, 2023 | |
CASH, CASH EQUIVALENTS AND SHORT-TERM INVESTMENTS | |
CASH, CASH EQUIVALENTS AND SHORT-TERM INVESTMENTS | 7. CASH, CASH EQUIVALENTS AND SHORT-TERM INVESTMENTS Cash, cash equivalents and short-term investments consisted of the following (in thousands): As of December 31, 2023 2022 Cash $ 8,247 $ 6,264 Money market funds 9,685 4,155 US treasury bills 12,594 5,225 Government-sponsored enterprise securities 11,233 15,796 Corporate bonds and commercial paper 15,174 26,766 $ 56,933 $ 58,206 Reported as: Cash and cash equivalents $ 32,786 $ 24,459 Short-term investments 24,147 33,747 $ 56,933 $ 58,206 Cash equivalents and short-term investments include the following securities with gross unrealized gains and losses (in thousands): Gross Gross Amortized Unrealized Unrealized As of December 31, 2023 Cost Gains Losses Fair Value US treasury bills $ 12,591 $ 3 $ — $ 12,594 Government-sponsored enterprise securities 11,230 7 (4) $ 11,233 Corporate bonds and commercial paper 15,172 5 (3) 15,174 Total $ 38,993 $ 15 $ (7) $ 39,001 Gross Gross Amortized Unrealized Unrealized As of December 31, 2022 Cost Gains Losses Fair Value US treasury bills $ 5,251 $ — $ (26) $ 5,225 Government-sponsored enterprise securities 15,882 1 (87) 15,796 Corporate bonds and commercial paper 26,807 — (41) 26,766 Total $ 47,940 $ 1 $ (154) $ 47,787 As of December 31, 2023 and 2022, our cash equivalents and short-term investments had a weighted-average time to maturity of approximately 82 days and 89 days, respectively. Our short-term investments are classified as available-for-sale securities. Accordingly, we have classified certain securities as short-term investments on our balance sheets as they are available for use in the current operations. The following table shows the fair value and gross unrealized losses of our investments in individual securities that are in an unrealized loss position, aggregated by investment category (in thousands): As of December 31, 2023 Fair Value Unrealized Losses US treasury bills $ 1,000 $ — Government-sponsored enterprise securities 2,980 (4) Corporate bonds and commercial paper 6,561 (3) Total $ 10,541 $ (7) |
FAIR VALUE
FAIR VALUE | 12 Months Ended |
Dec. 31, 2023 | |
FAIR VALUE | |
FAIR VALUE | 8. FAIR VALUE Assets at Fair Value as of December 31, 2023 Level 1 Level 2 Level 3 Total Money market funds $ 9,685 $ — $ — $ 9,685 US treasury bills — 12,594 — 12,594 Government-sponsored enterprise securities — 11,233 — 11,233 Corporate bonds and commercial paper — 15,174 — 15,174 Total $ 9,685 $ 39,001 $ — $ 48,686 Assets at Fair Value as of December 31, 2022 Level 1 Level 2 Level 3 Total Money market funds $ 4,155 $ — $ — $ 4,155 US treasury bills — 5,225 — 5,225 Government-sponsored enterprise securities — 15,796 — 15,796 Corporate bonds and commercial paper — 26,766 — 26,766 Total $ 4,155 $ 47,787 $ — $ 51,942 |
OTHER BALANCE SHEET COMPONENTS
OTHER BALANCE SHEET COMPONENTS | 12 Months Ended |
Dec. 31, 2023 | |
OTHER BALANCE SHEET COMPONENTS | |
OTHER BALANCE SHEET COMPONENTS | 9. OTHER BALANCE SHEET COMPONENTS Property and equipment Property and equipment consist of the following (in thousands): As of December 31, 2023 2022 Laboratory equipment $ 1,470 $ 7,435 Computer and software 363 2,048 Leasehold improvements, furniture and equipment — 2,107 Others 36 74 Total property and equipment 1,869 11,664 Less accumulated depreciation and amortization (1,704) (10,807) Property and equipment, net $ 165 $ 857 Depreciation and amortization expense was $0.2 million, $0.9 million and $1.2 million for the years ended December 31, 2023, 2022 and 2021, respectively. Certain property and equipment were either sold, retired or disposed of, which related costs and accumulated depreciation were removed from the balance sheet, and any resulting gain or loss were reflected in statements of operations in the period realized. Intangible asset Intangible asset consist of the following (in thousands): As of December 31, 2023 2022 Intangible asset cost $ 15,000 $ 15,000 Accumulated amortization (1,122) (51) Intangible asset, net $ 13,878 $ 14,949 Intangible asset pertain to amortized cost of capitalized milestone payment to Forma, incurred upon and after regulatory approval of an acquired product. See “Note 4 - Sponsored Research and License Agreements and Government Contracts” for related discussions. Such cost is being amortized on a straight-line basis over the estimated useful life of approximately 14 years . Amortization expense recorded within cost of sales in the statements of operations were $1.1 million and $0.1 million for the years ended December 31, 2023 and 2022, respectively. The following table presents the estimated future amortization expense of intangible asset (in thousands): For the year ending December 31, 2024 $ 1,071 2025 1,071 2026 1,071 2027 1,071 2028 1,071 Thereafter 8,523 $ 13,878 Other accrued liabilities Other accrued liabilities consist of the following (in thousands): As of December 31, 2023 2022 Accrued commercial expenses $ 1,852 $ 3,144 Accrued other expenses 3,482 3,341 Total other accrued liabilities $ 5,334 $ 6,485 |
DEBT
DEBT | 12 Months Ended |
Dec. 31, 2023 | |
DEBT | |
DEBT | 10. DEBT The following table summarizes loans payable, net (in thousands): As of December 31, 2023 2022 Principal outstanding $ 60,000 $ 40,000 Unamortized debt issuance costs (398) (552) Principal outstanding, net of unamortized debt issuance costs $ 59,602 $ 39,448 Reported as: Loans payable, net, current portion $ 7,229 $ - Long-term portion of loans payable, net 52,373 39,448 $ 59,602 $ 39,448 The outstanding loans payable as of the periods presented was related to our Credit Agreement with MidCap entered on September 27, 2019 (Closing Date) and amended on March 29, 2021 (First Amendment), February 11, 2022 (Second Amendment) and July 27, 2022 (Third Amendment). The Credit Agreement provides for a $60.0 million term loan credit facility. At the Closing Date, $10.0 million was funded (Tranche 1), in May 2020, an additional $10.0 million was funded (Tranche 2), at the Second Amendment, an additional $10.0 million was funded (Tranche 3), at the Third Amendment, an additional $10.0 million was funded (Tranche 4), and in March 2023, an additional $20.0 million was funded (Tranche 5). As of December 31, 2023, the outstanding principal balance of the loan was $60.0 million, and no remaining funds are available for draw under the term loan credit facility. The First Amendment to the Credit Agreement extended the period through which Tranche 3 was available to us. The Second Amendment to the Credit Agreement, among other things, amended the applicable funding conditions, applicable commitments and certain other terms relating to available credit facilities (Tranches 3 and 4), added additional term loan credit facility (Tranche 5), and revised certain terms related to the financial covenants. Following the Third Amendment, the maturity date for the term loans is on September 1, 2026, and the interest-only period is through October 1, 2024. The interest rate applicable to the term loans under the amended Credit Agreement is the sum of one-month , plus an adjustment of 0.11448% , subject to 1.50% applicable floor, plus applicable margin of 5.65% . A or a comparable applicable index rate determined pursuant to the Credit Agreement if the LIBOR was no longer available, We The amendment to the Credit Agreement in 2022 was accounted for as debt modification. As such, fees paid to Midcap of $0.4 million were recorded as additional debt discount and added to the unamortized debt discount that are being amortized as interest expense through maturity using the effective interest rate method. Debt issuance costs are recorded as a direct deduction from the outstanding principal balance of the term loan. Interest expense, including amortization of the debt discount and accretion of the final fees related to the Credit Agreement for the years ended December 31, 2023, 2022 and 2021 were $6.8 million, $3.0 million and $1.7 million, respectively. Accrued interest of $1.5 million and $0.8 million as of December 31, 2023 and 2022, respectively, was included within other accrued liabilities in the balance sheet. The following table presents the future minimum principal payments of the outstanding loan as of December 31, 2023 (in thousands): For the year ending December 31, 2024 $ 7,500 2025 30,000 2026 22,500 Principal amount (Tranches 1, 2, 3 and 4) $ 60,000 The amended Credit Agreement contains certain covenants which, among others, require us to deliver financial reports at designated times of the year and maintain minimum unrestricted cash and trailing net revenues. As of December 31, 2023, we were not in violation of any covenants. |
LEASES
LEASES | 12 Months Ended |
Dec. 31, 2023 | |
LEASES | |
LEASES | 11. LEASES We have a sublease agreement with Atara entered in October 2022 to sublease an office space located in South San Francisco, California. Subject to the terms of the sublease agreement, the lease term commenced in November 2022 and shall expire in May 2025. This leased facility is currently held as our new Headquarters following the expiration of our previous leased facility in January 2023. 1.42 We previously leased our prior headquarter space located in South San Francisco, California with Healthpeak Properties, Inc. (formerly known as HCP BTC, LLC), and had a sublease agreement with an unrelated third-party to sublet a portion of the leased facility. Both the lease and the sublease expired in January 2023. The components of our operating lease expense were as follows (in thousands): Year Ended December 31, 2023 2022 2021 Fixed operating lease expense $ 1,109 $ 5,470 $ 5,360 Variable operating lease expense 134 818 910 Total operating lease expense $ 1,243 $ 6,288 $ 6,270 Supplemental information related to our operating lease expense was as follows (in thousands): Year Ended December 31, 2023 2022 2021 Cash payments included in the measurement of operating lease liabilities $ 1,534 $ 10,485 $ 10,082 Supplemental information related to our operating sublease was as follows (in thousands): Year Ended December 31, 2023 2022 2021 Fixed sublease expense $ 365 $ 4,381 $ 4,381 Variable sublease expense 77 911 917 Sublease income (442) (5,292) (5,298) Net $ — $ — $ — The following table presents the future minimum lease payments of our operating lease liabilities as of December 31, 2023 (in thousands): For year ending December 31, 2024 $ 739 2025 301 Total minimum payments required $ 1,040 |
STOCKHOLDERS' EQUITY
STOCKHOLDERS' EQUITY | 12 Months Ended |
Dec. 31, 2023 | |
STOCKHOLDERS' EQUITY | |
STOCKHOLDERS' EQUITY | 12. STOCKHOLDERS’ EQUITY Preferred Stock We are authorized to issue 10,000,000 shares of preferred stock. As of December 31, 2023 and 2022, there were no issued and outstanding shares of preferred stock. Our board of directors is authorized to fix or alter the designation, powers, preferences and rights of the shares of each series of preferred shares, and the qualifications, limitations or restrictions of any wholly unissued shares, to establish from time to time the number of shares constituting any such series, and to increase or decrease the number of shares, if any. Common Stock Our Certificate of Incorporation as amended and restated in May 2018, authorizes us to issue 400,000,000 shares of common stock. Open Market Sale Agreement In August 2020, we entered into an Open Market Sale Agreement with Jefferies, as a sole agent, pursuant to which we may sell from time to time, through Jefferies, shares of our common stock in sales deemed to be “at-the-market offerings” as defined in Rule 415 under the Securities Act, subject to conditions specified in the Open Market Sale Agreement, including maintaining an effective registration statement covering the sale of shares under the Open Market Sale Agreement. We have a shelf registration statement filed with the SEC that was declared effective on May 3, 2022, which registered, among other securities, |
INCOME TAXES
INCOME TAXES | 12 Months Ended |
Dec. 31, 2023 | |
INCOME TAXES | |
INCOME TAXES | 13. INCOME TAXES We have no provision for income taxes recorded for the years ended December 31, 2023 and 2022 due to our pre-tax book losses and a full valuation allowance was recorded against our deferred tax assets. For the year ended December 31, 2021, we recognized provision for income tax of $0.6 million. This provision for income tax was related to the state tax liability primarily due to revenue recognized for the Lilly Agreement. We did not have federal income taxes due to the sufficient NOL carryforwards that were generated prior to the enactment of the Tax Act, as well as significant research and development credit carryforwards. We continue to record a full valuation allowance on our deferred tax assets considering our cumulative losses in prior years and forecasted losses in the future. Deferred income taxes reflect the net tax effects of temporary differences between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. Significant components of our deferred tax assets are as follows (in thousands): As of December 31, 2023 2022 Deferred tax assets Net operating loss carryforwards $ 229,967 $ 230,373 Orphan drug and research and development credits 62,457 68,646 Capitalized research and development credits 21,017 15,680 Deferred revenue 11,223 11,234 Deferred compensation 10,365 9,620 Lease liabilities 244 504 Other, net 3,654 2,494 Deferred tax liabilities Operating lease right-of-use asset (215) (461) Others (81) (439) Total net deferred tax assets 338,631 337,651 Less: valuation allowance (338,631) (337,651) Deferred tax assets, net of allowance $ — $ — The reconciliation of the statutory federal income tax rate to the effective tax rate was as follows: Year Ended December 31, 2023 2022 2021 Federal statutory tax rate (21.0) % (21.0) % (21.0) % State, net of federal benefit 0.1 % 0.0 % 2.8 % Valuation allowance 13.9 % 20.2 % 27.5 % Stock compensation 5.3 % 2.5 % 5.6 % Orphan drug and research and development credits 0.2 % (2.6) % (14.0) % Other, net 1.5 % 1.0 % 2.7 % Effective tax rate 0.0 % 0.1 % 3.6 % In general, under Section 382 of the Internal Revenue Code (Section 382), a corporation that undergoes an ownership change is subject to limitations on its ability to utilize its pre-change NOL carryovers and tax credits to offset future taxable income. Our existing NOL carryforwards and tax credits are subject to limitations arising from ownership changes which occurred in previous periods. We finalized our analysis of potential ownership changes and concluded our Section 382 owner shift analysis during the year ended December 31, 2012. We have updated our NOL carryforwards to reflect the results of the Section 382 owner shift analysis as of December 31, 2023. We did not experience any significant changes in ownership in the periods presented. Future changes in our stock ownership, some of which are outside of our control, could result in an ownership change under Section 382 and result in additional limitations. As of December 31, 2023, we had NOL carryforwards for federal income tax purposes of approximately $972.6 million. Of the federal NOL carryforward, $833.8 million, which expire beginning in the year 2025 and the remaining NOL carryforwards can be carried forward indefinitely, subject to annual limitation of 80% of taxable income. We also had state NOL carryforwards of approximately $388.5 million, which expire beginning in the year 2028. We have general business credits of approximately $45.1 million, which will expire beginning in 2024, if not utilized, and is consisted of research and development credits and orphan drug credits. We also have state research and development tax credits of approximately $31.8 million, which have no expiration date. Realization of deferred tax assets is dependent upon future earnings, if any, the timing and amount of which are uncertain. Accordingly, our deferred tax assets have been fully offset by valuation allowance considering our cumulative losses in prior years and forecasted losses in the future. The following table summarizes the activity related to our gross unrecognized tax benefits (in thousands): Year Ended December 31, 2023 2022 2021 Balance at the beginning of the year $ 9,426 $ 9,186 $ 8,901 Decrease related to prior year tax positions (843) — — Increase related to current year tax positions 89 240 285 Balance at the end of the year $ 8,672 $ 9,426 $ 9,186 During the years ended December 2023, 2022 and 2021, the amount of unrecognized tax benefits increased due to additional research and development and orphan drug credits generated during those years. During 2023, we engaged our tax consultant to perform an orphan credits study for years 2015 to 2020. The results of the study decreased the unrecognized tax benefits by $4.7 million. The reversal of the uncertain tax benefits would not affect our effective tax rate to the extent that we continue to maintain a full valuation allowance against our deferred tax assets. We are subject to federal income tax and various state taxes. Because of NOL and research credit carryovers, substantially all of our tax years remain open to examination. Our policy is to recognize interest and penalties accrued on any unrecognized tax benefits as a component of income tax expense. We currently have no |
RESTRUCTURING CHARGES
RESTRUCTURING CHARGES | 12 Months Ended |
Dec. 31, 2023 | |
RESTRUCTURING CHARGES. | |
RESTRUCTURING CHARGES | 14. RESTRUCTURING CHARGES In October 2022, we announced a reduction in our workforce primarily in our development and administration groups. In November 2021, we announced a reduction in our workforce primarily in the research organization. We recorded restructuring charges of $3.5 million in the statements of operations for the year ended December 31, 2021, comprised $2.9 million cash severance, bonus and related employee benefits and taxes of affected employees, $0.4 million of stock-based compensation expense related to option modification and $0.1 million impairment of certain property and equipment which was recorded within depreciation expense. |
SUBSEQUENT EVENTS
SUBSEQUENT EVENTS | 12 Months Ended |
Dec. 31, 2023 | |
SUBSEQUENT EVENTS | |
SUBSEQUENT EVENTS | 15. SUBSEQUENT EVENTS On February 22, 2024, we entered into an Asset Purchase Agreement with Blueprint to purchase certain assets comprising the right to research, develop, manufacture and commercialize GAVRETO (pralsetinib) in the US. Such assets include, among other things, applicable intellectual property related to pralsetinib in the United States, including patents, copyrights and trademarks, as well as clinical regulatory and commercial data and records. The potential regulatory milestones include full regulatory approval of pralsetinib (or related compounds) for the treatment of adult RET-fusion positive thyroid cancer, and maintenance of the current regulatory approval of pralsetinib for the treatment of adult RET-fusion positive thyroid cancer during the period beginning on February 22, 2024 and ending on the third anniversary of the first commercial sale of pralsetinib subject to certain conditions. |
DESCRIPTION OF BUSINESS AND S_2
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 12 Months Ended |
Dec. 31, 2023 | |
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |
Basis of Presentation | Basis of Presentation The accompanying financial statements have been prepared in accordance with United States generally accepted accounting principles (US GAAP). Any reference in these notes to applicable accounting guidance is meant to refer to the authoritative US GAAP included in the Accounting Standards Codification (ASC), and Accounting Standards Update (ASU) issued by the Financial Accounting Standards Board (FASB). We manage our operations as one business segment for purposes of assessing performance, making operating decisions, and allocating resources, and our chief operating decision maker is our chief executive officer. |
Liquidity | Liquidity As of December 31, 2023, we had approximately $56.9 million in cash, cash equivalents and short-term investments. Since inception, we have financed our operations primarily through sales of equity securities, debt financing arrangement, contract payments under our collaboration agreements and from product sales. Based on our current operating plan, we believe that our existing cash, cash equivalents, and short-term investments will be sufficient to fund our expenses and capital expenditure requirements through at least the next 12 months from the date of issuance of this Annual Report on Form 10-K. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with US GAAP requires management to make certain judgments, estimates and assumptions that could affect the reported amounts of assets and liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting period. Critical accounting estimates We base our estimates and assumptions on historical experience and on various other assumptions we believe to be applicable, and evaluate them on an ongoing basis to ensure they remain reasonable under current conditions. Actual results could differ significantly from those estimates, which could have a material impact on our business, results of operations, and financial condition. |
Revenue Recognition | Revenue Recognition We recognize revenue in accordance with ASC Topic 606, Revenue from Contracts with Customers Product Sales Revenues from product sales are recognized when the specialty distributors, who are our customers, obtain control of our product, which occurs at a point in time, upon delivery to such specialty distributors. These specialty distributors subsequently resell our products to specialty pharmacy providers, health care providers, hospitals and clinics. In addition to distribution agreements with our specialty distributors, we also have arrangements with certain specialty pharmacy providers, in-office dispensing providers, group purchasing organizations, and government entities that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of our products. Under ASC 606, we are required to estimate the transaction price, including variable consideration that is subject to a constraint, in our contracts with our customers. Variable consideration is included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur. Revenue from product sales is recorded net of certain variable consideration which includes estimated government-mandated rebates and chargebacks, distribution fees, estimated product returns and other deductions. Provisions for returns and other adjustments are provided for in the period the related revenue is recorded. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. The following are our significant categories of sales discounts and allowances: Sales Discounts . We provide certain customer a prompt payment discount that is explicitly stated in our contract. The sales discount is recorded as a reduction of revenue in the period the related product revenue is recognized. Product Returns. Government and Private Payor Rebates: Chargebacks and Discounts: Chargebacks for fees and discounts represent the estimated obligations resulting from contractual commitments to sell products to certain specialty pharmacy providers, in-office dispensing providers, group purchasing organizations, and government entities at prices lower than the list prices charged to our specialty distributors who directly purchase the product from us. These specialty distributors charge us for the difference between what they pay for the product and our contracted selling price to these specialty pharmacy providers, in-office dispensing providers, group purchasing organizations, and government entities. These reserves are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue. Actual chargeback amounts are generally determined at the time of resale to the specialty pharmacy providers, in-office dispensing providers, group purchasing organizations, and government entities by our specialty distributors. The estimated obligations arising from these chargebacks and discounts are recorded as revenue reserves within other accrued liabilities in the balance sheet. Co-Payment Assistance: Contract Revenues from Collaborations In the normal course of business, we conduct research and development programs independently and in connection with our corporate collaborators, pursuant to which we license certain rights to our intellectual property to third parties. The terms of these arrangements typically include payment to us for a combination of one or more of the following: upfront license fees; development, regulatory and commercial milestone payments; product supply services; and royalties on net sales of licensed products. Upfront License Fees: For arrangements that require us to share in the development costs but to which we do not participate in the co-development work, the portion of the upfront fee attributed to our share in the future development costs is excluded from the transaction price. If such share in the development costs is payable beyond 12 months from the delivery of the corresponding license, a significant financing component is deemed to exist. If a significant financing component is identified, we adjust the transaction price by reducing the upfront fee by the net present value of our share in future development costs over the expected commitment period. Such discounted amount will be reported as a liability in the balance sheet, with a corresponding interest expense being accreted based on a discount rate applied over the expected commitment period. Development, Regulatory or Commercial Milestone Payments: Product Supply Services: Sales-based Milestone Payments and Royalties: |
Government Contracts | Government Contracts There is limited US GAAP accounting guidance for for-profit business entities that receives government assistance. We utilized other accounting standards, and have elected to analogize to International Financial Reporting Standards, specifically International Accounting Standards (IAS) 20, Accounting for Government Grants and Disclosures of Government Assistance. Disclosures by Business Entities about Government Assistance, |
Stock-based Compensation | Stock-based Compensation Share-based awards are valued at fair value on the date of grant and that fair value is recognized over the requisite service period, which is generally the vesting period of the respective award. We use the straight-line attribution method over the requisite employee service period for the entire award in recognizing stock-based compensation expense. We account for forfeitures as they occur. The fair value of each option award is estimated on the date of grant using the Black-Scholes option pricing model. The model requires management to make a number of assumptions including expected volatility, expected term, risk-free interest rate and expected dividends. A number of these assumptions are subjective, and their determination generally require judgment. We segregate option awards into the following three homogenous groups for the purposes of determining fair values of options: officers and directors, all other employees, and consultants. We determine the weighted-average valuation assumptions separately for each of these groups as follows: ● Volatility – We estimate volatility using the historical share price performance over the expected life of the option up to the point where we have historical market data. We also consider other factors, such as implied volatility, our current clinical trials and other company activities that may affect the volatility of our stock in the future. We determined that at this time historical volatility is more indicative of our expected future stock performance than implied volatility. ● Expected term – We analyze various historical data to determine the applicable expected term for each of the other option groups. This data includes: (1) for exercised options, the term of the options from option grant date to exercise date; (2) for cancelled options, the term of the options from option grant date to cancellation date, excluding non-vested option forfeitures; and (3) for options that remained outstanding at the balance sheet date, the term of the options from option grant date to the end of the reporting period and the estimated remaining term of the options. The consideration and calculation of the above data gives us reasonable estimates of the expected term for each employee group. We also consider the vesting schedules of the options granted and factors surrounding exercise behavior of the option groups, our current market price and company activity that may affect our market price. In addition, we consider the optionee type (i.e., officers and directors or all other employees) and other factors that may affect the expected term of the option. For options granted to consultants, we use the contractual term of the option, which is generally 10 years, for the initial valuation of the option and the remaining contractual term of the option for the succeeding periods. ● Risk-free interest rate – The risk-free interest rate is based on US Treasury constant maturity rates with similar terms to the expected term of the options for each option group. ● Dividend yield – The expected dividend yield is 0% as we have not paid and do not expect to pay dividends in the future. We grant performance-based stock options to purchase shares of our common stock which will vest upon the achievement of certain corporate performance-based milestones. We determine the fair values of these performance-based stock options using the Black-Scholes option pricing model at the date of grant. For the portion of the performance-based stock options of which the performance condition is considered probable of achievement, we recognize stock-based compensation expense on the related estimated grant date fair values of such options on a straight-line basis from the date of grant up to the date when we expect the performance condition will be achieved. For the performance conditions that are not considered probable of achievement at the grant date or upon re-evaluation at each reporting date, prior to the event actually occurring, we recognize the related stock-based compensation expense when the event occurs or when we can determine that the performance condition is probable of achievement. In those cases, we recognize the change in estimate at the time we determine the condition is probable of achievement (by recognizing stock-based compensation expense as cumulative catch-up adjustment as if we had estimated at the grant date that the performance condition would have been achieved) and recognize the remaining compensation cost up to the date when we expect the performance condition will be achieved, if any. The fair value of the RSU grant is based on the market price of our common stock on the date of grant. |
Accounts Receivable | Accounts Receivable Accounts receivable are recorded net of customer allowances for prompt payment discounts and any allowance for doubtful accounts. We monitor the financial performance and creditworthiness of our customers so that we can properly assess and respond to changes in their credit profile. We have not historically experienced credit losses and no amounts were reserved for estimated losses as of the balance sheet dates presented. The following table summarizes the activity of our customer allowances for prompt payment discounts for the periods presented (in thousands): Year Ended December 31, 2023 2022 2021 Balance at the beginning of the year $ 136 $ 106 $ 171 Provision for prompt payment discount 686 557 609 Reduction in prompt payment discount (642) (527) (674) Balance at end of the year $ 180 $ 136 $ 106 |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject us to a concentration of credit risk are primarily cash, investment in debt securities and accounts receivable. All of our cash and investment in debt securities are maintained with financial institutions that management believes are creditworthy. By policy, we limit the concentration of credit risk by diversifying our investments among a variety of high credit-quality issuers. Due to the short-term nature of these investments, we believe we do not have a material exposure to credit risk arising from our investments. We have not historically experienced any significant credit losses related to these financial instruments and do not believe that we are exposed to any significant credit risk related to these instruments. Concentration of credit risk with respect to our accounts receivable is limited due to our small number of customers. Our accounts receivable consists mostly of outstanding invoices from our sale of our product to our specialty distributors. Accounts receivable may also include outstanding invoices from our collaboration partners with respect to the related sponsored research and license agreements and government contracts. As of December 31, 2023, 87% of our accounts receivable consisted of outstanding invoices from our specialty distributors, and the remaining 13% are outstanding invoices from our collaboration partners, mainly Grifols. As of December 31, 2022, 49% of our accounts receivable are outstanding invoices from our specialty distributors, and the remaining 51% are outstanding invoices from our collaboration partners, mainly Kissei which include a $20.0 million regulatory milestone receivable that was subsequently collected in January 2023. See “Note 3 - Revenues” for summary of revenues from each of our customers who individually accounted for 10% or more of the total net product sales and revenues from collaborations. |
Cash, Cash Equivalents and Short-Term Investments | Cash, Cash Equivalents and Short-Term Investments Our investment in debt securities consists of money market funds, US treasury bills, government- sponsored enterprise securities, and corporate bonds and commercial paper. All of our investment in debt securities are available-for-sale and are classified based on their maturities. We consider all highly liquid investments in debt securities with maturity of 90 days or less from the date of purchase to be cash equivalents. All other investments with maturity greater than 90 days from the date of purchase are classified as short-term investments. Unrealized gains (losses) are reported within the statements of stockholders’ (deficit) equity and comprehensive income (loss). The cost of securities sold is based on the specific identification method. We periodically evaluate our available-for-sale marketable debt securities for impairment. When the fair value of a marketable debt security is below its amortized cost, the amortized cost is reduced to its fair value if it is more likely than not that we are required to sell the impaired security before recovery of our amortized cost basis, or we have the intention to sell the security. If neither of these conditions are met, we determine whether the impairment is due to credit losses by comparing the present value of the expected cash flows of the security with its amortized cost basis. The amount of impairment recognized is limited to the excess of the amortized cost over the fair value of the security. An allowance for credit losses for the excess of amortized cost over the expected cash flows is recorded in other income (expense), net on the statements of operations. Impairment losses that are not credit-related are included in accumulated other comprehensive income (loss) in stockholders’ (deficit) equity. |
Fair Value of Financial Instruments | Fair Value of Financial Instruments The carrying amounts of our financial instruments, including cash, accounts receivable, accounts payable and accrued liabilities, approximate fair value due to their relatively short maturities. The carrying value of our loans payable and other long-term debt approximates fair value based on management’s estimation that a current interest rate would not differ materially from the stated rate, or the discount rate applied. The fair value of our cash equivalents and short-term investments measured at fair value on a recurring basis and are categorized based upon the lowest level of significant input to the valuations. Assets and liabilities recorded at fair value in our financial statements are categorized based upon the level of judgment associated with the inputs used to measure their fair value. Fair value is an exit price, representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. ● Level 1 – Inputs are unadjusted, quoted prices in active markets for identical assets at the reporting date. Active markets are those in which transactions for the asset or liability occur in sufficient frequency and volume to provide pricing information on an ongoing basis. The fair valued assets we hold that are generally included under this Level 1 are money market securities where fair value is based on publicly quoted prices. ● Level 2 – Inputs, other than quoted prices included in Level 1, that are either directly or indirectly observable for the asset or liability through correlation with market data at the reporting date and for the duration of the instrument’s anticipated life. The fair valued assets we hold that are generally assessed under Level 2 included government-sponsored enterprise securities, US treasury bills and corporate bonds and commercial paper. We utilize third-party pricing services in developing fair value measurements where fair value is based on valuation methodologies such as models using observable market inputs, including benchmark yields, reported trades, broker/dealer quotes, bids, offers and other reference data. We use quotes from external pricing service providers and other on-line quotation systems to verify the fair value of investments provided by our third-party pricing service providers. ● Level 3 – Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities and which reflect management’s best estimate of what market participants would use in pricing the asset or liability at the reporting date. Consideration is given to the risk inherent in the valuation technique and the risk inherent in the inputs to the model. We do not have fair valued assets classified under Level 3. |
Inventories and Cost of Product Sales | Inventories and Cost of Product Sales Inventories are stated at the lower-of-cost or estimated net realizable value. We determine the cost of inventories using the standard cost method, which approximates actual cost, and is valued using the first-in, first-out method. Inventory costs primarily consist of active pharmaceutical ingredients, third-party manufacturing costs and allocated internal overhead costs. We capitalize inventory costs when the product is approved by the FDA, or when based on management’s judgment, future commercialization was considered probable, and the future economic benefits are expected to be realized. Prior to FDA approval of a product, costs to purchase active pharmaceutical ingredients including costs to manufacture a product are charged to research and development expense when incurred. Our physical inventories as of balance sheet dates include inventory quantities where costs have been previously charged to research and development expense since such costs were incurred prior to FDA approval of the product. We provide reserves for potential excess, dated or obsolete inventories based upon assumptions about future demand and market conditions, as well as product shelf life. Inventories that are not expected to be consumed beyond our normal operating cycle are classified as non-current inventories and included within other assets in the balance sheet. Cost of product sales primarily includes cost of inventories sold, and product shipping and handling costs. Further, following the approval of REZLIDHIA, we recognize the amortization expense of capitalized intangible asset and royalty expense incurred pursuant to our license agreement with Forma, within cost of sales. |
Property and Equipment | Property and Equipment Property and equipment are stated at cost net of accumulated depreciation. Depreciation is calculated using the straight-line method over the estimated useful lives of the assets, which range from three |
Research and Development Expenses | Research and Development Expenses Research and development expenses include costs incurred to conduct research and development, including scientific personnel wages and associated employee benefits, research and development supplies and equipment, payments to collaborative clinical research partners, consulting fees and other various research and development related costs. We have various contracts with third parties related to our research and development activities, including strategic development collaborations. Costs incurred but not billed to us as of the end of the period are accrued. We make estimates of the amounts incurred in each period based on the information available to us and our knowledge of the nature of the contractual activities generating such costs. Clinical trial contract expenses are accrued based on units of activity. Expenses related to other research and development contracts, such as research contracts, toxicology study contracts and manufacturing contracts are estimated to be incurred generally on a straight-line basis over the duration of the contracts. Raw materials and study materials not related to an approved drug are charged to research and development expenses at the time of purchase. We make significant judgments and estimates in determining the accrual balance in each reporting period. As actual costs become known, we adjust our accruals. Although we do not expect our estimates to be materially different from amounts actually incurred, such estimates for the status and timing of services performed relative to the actual status and timing of services performed may vary and could result in us reporting amounts that are too high or too low in any particular period. Variations in assumptions used to estimate accruals including, but not limited to, the number of patients enrolled, the rate of patient enrollment and the actual services performed may result in adjustments in research and development accruals in future periods. Changes in these estimates that result in material changes to our accruals could materially affect our financial condition and results of operations. Research and development expenses also include milestone payment obligations incurred prior to r egulatory approval of the product, which are accrued when the event requiring payment of the milestone occurs. See related discussions under “IPR&D/Intangible Asset” below. |
IPR&D/Intangible Asset | IPR&D/Intangible Asset In July 2022, we entered into a license and transition services agreement with Forma. The transaction was accounted for as an acquisition of asset under ASC 730, Research and Development account for m We perform an impairment review of intangible asset whenever events or changes in business circumstances indicate the carrying amount of the asset may not be fully recoverable. If events or changes in circumstances suggest that the carrying amount of the finite-lived intangible assets may not be recoverable, we will estimate the future cash flows expected to be generated by the asset from its use or eventual disposition. If the expected future undiscounted cash flows is less than the carrying amount of the assets, we will recognize an impairment loss based on the excess of the carrying amount over the fair value of the assets. |
Advertising Expense | Advertising Expense Advertising costs are expensed as incurred and are included within selling general and administrative expenses in the statements of operations. Advertising costs for the years ended December 31, 2023, 2022 and 2021 amounted to $3.1 million, $2.7 million, and $2.3 million, respectively. |
Leases | Leases We account for leases in accordance with ASU No. 2016-02 , Leases (Topic 842) . Topic 842 requires a lessee to determine if an arrangement is a lease or contains a lease at contract inception. For our sublease agreement wherein we were the lessor, we recognized sublease income on a straight-line basis over the term of the related sublease agreement. |
Restructuring | Restructuring Restructuring costs comprised severance, other termination benefit costs, stock-based compensation expense for stock award and stock option modifications related to workforce reductions and accelerated depreciation. We recognize restructuring charges when the liability is probable, and the amount is estimable. Employee termination benefits are accrued at the date management has committed to a plan of termination and affected employees have been notified of their termination date and expected severance benefits. |
Income Taxes | Income Taxes We use the asset and liability method to account for income taxes. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases and operating loss and tax credit carryforwards. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities from a change in tax rates is recognized in income in the period the change is enacted. A valuation allowance is established to reduce deferred tax assets to an amount whose realization is more likely than not. |
Recently Issued Accounting Standards | Recent Accounting Pronouncements In November 2023, FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures. In December 2023, FASB issued ASU 2023-09, Improvements to Income Tax Disclosures Other recently issued accounting guidance not discussed in this Annual Report on Form 10-K are either not applicable or did not have, or are not expected to have, a material impact on us. |
DESCRIPTION OF BUSINESS AND S_3
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |
Summary of customer allowances for prompt payment discounts | The following table summarizes the activity of our customer allowances for prompt payment discounts for the periods presented (in thousands): Year Ended December 31, 2023 2022 2021 Balance at the beginning of the year $ 136 $ 106 $ 171 Provision for prompt payment discount 686 557 609 Reduction in prompt payment discount (642) (527) (674) Balance at end of the year $ 180 $ 136 $ 106 |
NET LOSS PER SHARE (Tables)
NET LOSS PER SHARE (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
NET LOSS PER SHARE | |
Schedule of antidilutive securities | The potential shares of common stock that were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive are as follows (in thousands): Year Ended December 31, 2023 2022 2021 Outstanding stock options 34,119 34,696 30,009 RSUs 1,866 1,104 226 Total 35,985 35,800 30,235 |
REVENUES (Tables)
REVENUES (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
REVENUES | |
Schedule of revenues disaggregated by category | Revenues disaggregated by category were as follows (in thousands): Year Ended December 31, 2023 2022 2021 Product sales: Gross product sales $ 147,058 $ 108,523 $ 81,186 Discounts and allowances (42,764) (31,805) (18,176) Total product sales, net 104,294 76,718 63,010 Revenues from collaborations: License revenues — 7,932 70,553 Milestone revenue 75 25,000 1,875 Delivery of drug supplies, royalty and others 11,413 6,092 3,298 Total revenues from collaborations 11,488 39,024 75,726 Government contracts 1,100 4,500 10,500 Total revenues $ 116,882 $ 120,242 $ 149,236 |
Schedule of product revenue allowance and reserve categories | Chargebacks, Government Discounts and and Other Fees Rebates Returns Total Balance as of January 1, 2023 $ 6,213 $ 2,636 $ 3,296 $ 12,145 Provision related to current period sales 32,330 8,299 869 41,498 Credit or payments made during the period (30,307) (7,418) (234) (37,959) Balance as of December 31, 2023 $ 8,236 $ 3,517 $ 3,931 $ 15,684 Chargebacks, Government Discounts and and Other Fees Rebates Returns Total Balance as of January 1, 2022 $ 3,404 $ 2,494 $ 2,017 $ 7,915 Provision related to current period sales 23,488 5,901 1,514 30,903 Credit or payments made during the period (20,679) (5,759) (235) (26,673) Balance as of December 31, 2022 $ 6,213 $ 2,636 $ 3,296 $ 12,145 |
Schedule of revenues from product sales disaggregated by customers | Year Ended December 31, 2023 2022 2021 McKesson Specialty Care Distribution Corporation 43% 31% 20% Cardinal Healthcare 25% 19% * ASD Healthcare and Oncology Supply 21% 17% 17% Kissei * 24% * Lilly * * 48% |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Schedule of stock-based compensation | Total stock-based compensation expense related to all of our stock-based awards was as follows (in thousands): Year Ended December 31, 2023 2022 2021 Selling, general and administrative $ 6,712 $ 10,217 $ 7,337 Research and development 2,094 2,168 1,700 Restructuring charges — — 449 Total stock-based compensation expense $ 8,806 $ 12,385 $ 9,486 |
Schedule of option activity under equity incentive plans | Stock Options RSUs Weighted Weighted Weighted Average Shares Available Number of Average Intrinsic Value Number of Grant Date For Grant Shares Exercise Price (in thousands) Shares Fair Value Outstanding as of December 31, 2022 10,612,618 34,696,273 $ 2.74 $ 1,605 1,103,653 $ 2.39 Authorized for grant 5,019,700 Granted (5,669,944) 3,671,800 $ 1.69 1,387,600 $ 1.80 Exercised/Released — (50,161) $ 0.97 (435,006) $ 2.32 Cancelled and forfeited 3,908,921 (4,199,147) $ 3.29 (190,356) $ 2.20 Outstanding as of December 31, 2023 13,871,295 34,118,765 $ 2.56 $ 1,453 1,865,891 $ 1.98 Vested and expected to vest as of December 31, 2023 31,373,765 $ 2.55 $ 1,349 Exercisable as of December 31, 2023 24,378,890 $ 2.70 $ 403 |
Schedule of stock options by exercise price | Options Outstanding Options Exercisable Weighted-Average Number of Remaining Contractual Weighted-Average Number of Weighted-Average Exercise Price Shares Life (in years) Exercise Price Shares Exercise Price $0.90 - $1.87 6,712,896 8.61 $ 1.43 1,991,518 $ 1.44 $1.96 - $2.11 5,063,237 4.44 $ 2.03 5,063,237 $ 2.03 $2.14 - $2.40 3,940,279 2.89 $ 2.26 3,717,256 $ 2.26 $2.42 - $2.42 5,816,323 6.81 $ 2.42 3,794,160 $ 2.42 $2.44 - $3.54 6,852,637 5.14 $ 3.16 4,473,712 $ 3.06 $3.59 - $4.49 5,542,956 3.95 $ 3.97 5,215,904 $ 3.99 $4.50 - $4.50 190,437 7.29 $ 4.50 123,103 $ 4.50 $0.90 - $4.50 34,118,765 5.56 $ 2.56 24,378,890 $ 2.70 |
Employee Stock Option [Member] | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Summary of weighted-average assumptions relating to options granted | Year Ended December 31, 2023 2022 2021 Risk-free interest rate 3.9 % 2.6 % 1.0 % Expected term (in years) 6.8 6.3 6.4 Dividend yield 0.0 % 0.0 % 0.0 % Expected volatility 84.0 % 74.8 % 70.5 % |
Purchase Plan | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Summary of weighted-average assumptions used to calculate fair value of purchase rights granted under Employee Stock Purchase Plan | Year Ended December 31, 2023 2022 2021 Risk-free interest rate * 3.1 % * Expected term (in years) * 1.3 * Dividend yield * 0.0 % * Expected volatility * 121.0 % * Weighted average grant date fair value (dollar per share) * $0.78 * |
INVENTORIES (Tables)
INVENTORIES (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
INVENTORIES | |
Schedule of Inventories | The following table summarizes inventories, net (in thousands): As of December 31, 2023 2022 Raw materials $ 4,609 $ 4,555 Work in process 1,876 2,659 Finished goods 1,508 1,904 Total $ 7,993 $ 9,118 Reported as: Inventories $ 5,522 $ 9,118 Other assets 2,471 — Total $ 7,993 $ 9,118 |
CASH, CASH EQUIVALENTS AND SH_2
CASH, CASH EQUIVALENTS AND SHORT-TERM INVESTMENTS (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
CASH, CASH EQUIVALENTS AND SHORT-TERM INVESTMENTS | |
Schedule of cash, cash equivalents and short-term investments | Cash, cash equivalents and short-term investments consisted of the following (in thousands): As of December 31, 2023 2022 Cash $ 8,247 $ 6,264 Money market funds 9,685 4,155 US treasury bills 12,594 5,225 Government-sponsored enterprise securities 11,233 15,796 Corporate bonds and commercial paper 15,174 26,766 $ 56,933 $ 58,206 Reported as: Cash and cash equivalents $ 32,786 $ 24,459 Short-term investments 24,147 33,747 $ 56,933 $ 58,206 |
Schedule of cash equivalents and short-term investments including securities with unrealized gains and losses | Cash equivalents and short-term investments include the following securities with gross unrealized gains and losses (in thousands): Gross Gross Amortized Unrealized Unrealized As of December 31, 2023 Cost Gains Losses Fair Value US treasury bills $ 12,591 $ 3 $ — $ 12,594 Government-sponsored enterprise securities 11,230 7 (4) $ 11,233 Corporate bonds and commercial paper 15,172 5 (3) 15,174 Total $ 38,993 $ 15 $ (7) $ 39,001 Gross Gross Amortized Unrealized Unrealized As of December 31, 2022 Cost Gains Losses Fair Value US treasury bills $ 5,251 $ — $ (26) $ 5,225 Government-sponsored enterprise securities 15,882 1 (87) 15,796 Corporate bonds and commercial paper 26,807 — (41) 26,766 Total $ 47,940 $ 1 $ (154) $ 47,787 |
Schedule of fair value and gross unrealized losses of investments in unrealized loss position | The following table shows the fair value and gross unrealized losses of our investments in individual securities that are in an unrealized loss position, aggregated by investment category (in thousands): As of December 31, 2023 Fair Value Unrealized Losses US treasury bills $ 1,000 $ — Government-sponsored enterprise securities 2,980 (4) Corporate bonds and commercial paper 6,561 (3) Total $ 10,541 $ (7) |
FAIR VALUE (Tables)
FAIR VALUE (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
FAIR VALUE | |
Schedule of financial assets measured at fair value on a recurring basis | Assets at Fair Value as of December 31, 2023 Level 1 Level 2 Level 3 Total Money market funds $ 9,685 $ — $ — $ 9,685 US treasury bills — 12,594 — 12,594 Government-sponsored enterprise securities — 11,233 — 11,233 Corporate bonds and commercial paper — 15,174 — 15,174 Total $ 9,685 $ 39,001 $ — $ 48,686 Assets at Fair Value as of December 31, 2022 Level 1 Level 2 Level 3 Total Money market funds $ 4,155 $ — $ — $ 4,155 US treasury bills — 5,225 — 5,225 Government-sponsored enterprise securities — 15,796 — 15,796 Corporate bonds and commercial paper — 26,766 — 26,766 Total $ 4,155 $ 47,787 $ — $ 51,942 |
OTHER BALANCE SHEET COMPONENTS
OTHER BALANCE SHEET COMPONENTS (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
OTHER BALANCE SHEET COMPONENTS | |
Schedule of property and equipment | Property and equipment consist of the following (in thousands): As of December 31, 2023 2022 Laboratory equipment $ 1,470 $ 7,435 Computer and software 363 2,048 Leasehold improvements, furniture and equipment — 2,107 Others 36 74 Total property and equipment 1,869 11,664 Less accumulated depreciation and amortization (1,704) (10,807) Property and equipment, net $ 165 $ 857 |
Schedule of intangible assets | Intangible asset consist of the following (in thousands): As of December 31, 2023 2022 Intangible asset cost $ 15,000 $ 15,000 Accumulated amortization (1,122) (51) Intangible asset, net $ 13,878 $ 14,949 |
Schedule of estimated future amortization expense of intangible asset | The following table presents the estimated future amortization expense of intangible asset (in thousands): For the year ending December 31, 2024 $ 1,071 2025 1,071 2026 1,071 2027 1,071 2028 1,071 Thereafter 8,523 $ 13,878 |
Schedule of other accrued liabilities | Other accrued liabilities consist of the following (in thousands): As of December 31, 2023 2022 Accrued commercial expenses $ 1,852 $ 3,144 Accrued other expenses 3,482 3,341 Total other accrued liabilities $ 5,334 $ 6,485 |
DEBT (Tables)
DEBT (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
DEBT | |
Summary of loans payable, net | The following table summarizes loans payable, net (in thousands): As of December 31, 2023 2022 Principal outstanding $ 60,000 $ 40,000 Unamortized debt issuance costs (398) (552) Principal outstanding, net of unamortized debt issuance costs $ 59,602 $ 39,448 Reported as: Loans payable, net, current portion $ 7,229 $ - Long-term portion of loans payable, net 52,373 39,448 $ 59,602 $ 39,448 |
Schedule of future minimum payments | The following table presents the future minimum principal payments of the outstanding loan as of December 31, 2023 (in thousands): For the year ending December 31, 2024 $ 7,500 2025 30,000 2026 22,500 Principal amount (Tranches 1, 2, 3 and 4) $ 60,000 |
LEASES (Tables)
LEASES (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
LEASES | |
Schedule of components of operating lease expense | The components of our operating lease expense were as follows (in thousands): Year Ended December 31, 2023 2022 2021 Fixed operating lease expense $ 1,109 $ 5,470 $ 5,360 Variable operating lease expense 134 818 910 Total operating lease expense $ 1,243 $ 6,288 $ 6,270 |
Schedule of supplemental information related to operating lease | Supplemental information related to our operating lease expense was as follows (in thousands): Year Ended December 31, 2023 2022 2021 Cash payments included in the measurement of operating lease liabilities $ 1,534 $ 10,485 $ 10,082 |
Schedule of operating sublease information | Supplemental information related to our operating sublease was as follows (in thousands): Year Ended December 31, 2023 2022 2021 Fixed sublease expense $ 365 $ 4,381 $ 4,381 Variable sublease expense 77 911 917 Sublease income (442) (5,292) (5,298) Net $ — $ — $ — |
Schedule of future minimum lease payments | The following table presents the future minimum lease payments of our operating lease liabilities as of December 31, 2023 (in thousands): For year ending December 31, 2024 $ 739 2025 301 Total minimum payments required $ 1,040 |
INCOME TAXES (Tables)
INCOME TAXES (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
INCOME TAXES | |
Schedule of components of the entity's deferred tax assets | As of December 31, 2023 2022 Deferred tax assets Net operating loss carryforwards $ 229,967 $ 230,373 Orphan drug and research and development credits 62,457 68,646 Capitalized research and development credits 21,017 15,680 Deferred revenue 11,223 11,234 Deferred compensation 10,365 9,620 Lease liabilities 244 504 Other, net 3,654 2,494 Deferred tax liabilities Operating lease right-of-use asset (215) (461) Others (81) (439) Total net deferred tax assets 338,631 337,651 Less: valuation allowance (338,631) (337,651) Deferred tax assets, net of allowance $ — $ — |
Schedule of reconciliation of the statutory federal income tax rate to the effective tax rate | Year Ended December 31, 2023 2022 2021 Federal statutory tax rate (21.0) % (21.0) % (21.0) % State, net of federal benefit 0.1 % 0.0 % 2.8 % Valuation allowance 13.9 % 20.2 % 27.5 % Stock compensation 5.3 % 2.5 % 5.6 % Orphan drug and research and development credits 0.2 % (2.6) % (14.0) % Other, net 1.5 % 1.0 % 2.7 % Effective tax rate 0.0 % 0.1 % 3.6 % |
Schedule of activity related to the entity's gross unrecognized tax benefits | The following table summarizes the activity related to our gross unrecognized tax benefits (in thousands): Year Ended December 31, 2023 2022 2021 Balance at the beginning of the year $ 9,426 $ 9,186 $ 8,901 Decrease related to prior year tax positions (843) — — Increase related to current year tax positions 89 240 285 Balance at the end of the year $ 8,672 $ 9,426 $ 9,186 |
DESCRIPTION OF BUSINESS AND S_4
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Basis of Presentation and Liquidity (Details) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 USD ($) segment | Dec. 31, 2022 USD ($) | |
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | ||
Number of business segments | segment | 1 | |
Cash, cash equivalents and short-term investments | $ | $ 56,933 | $ 58,206 |
DESCRIPTION OF BUSINESS AND S_5
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Stock-based Compensation (Details) | 12 Months Ended |
Dec. 31, 2023 item | |
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |
Number of homogenous groups for purposes of determining fair values of options | 3 |
Dividend yield (as a percent) | 0% |
DESCRIPTION OF BUSINESS AND S_6
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Accounts Receivable (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |||
Balance at the beginning of the year | $ 136 | $ 106 | $ 171 |
Provision for prompt payment discount | 686 | 557 | 609 |
Reduction in prompt payment discount | (642) | (527) | (674) |
Balance at end of year | $ 180 | $ 136 | $ 106 |
DESCRIPTION OF BUSINESS AND S_7
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Concentration of Credit Risk (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Accounts receivable | $ 30,550 | $ 40,320 |
Kissei | ||
Accounts receivable | $ 20,000 | |
Accounts Receivable | Customer Concentration Risk | Three specialty distributors | ||
Percentage | 87% | 49% |
Accounts Receivable | Customer Concentration Risk | US Government and from other collaboration partners | ||
Percentage | 13% | 51% |
DESCRIPTION OF BUSINESS AND S_8
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Property and Equipment (Details) | Dec. 31, 2023 |
Minimum | |
Property and equipment | |
Estimated useful life | 3 years |
Maximum | |
Property and equipment | |
Estimated useful life | 7 years |
DESCRIPTION OF BUSINESS AND S_9
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Advertising Expense (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |||
Advertising expense | $ 3.1 | $ 2.7 | $ 2.3 |
NET LOSS PER SHARE (Details)
NET LOSS PER SHARE (Details) - shares shares in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Antidilutive securities excluded from the computation of diluted net loss per share | |||
Total | 35,985 | 35,800 | 30,235 |
Employee Stock Option [Member] | |||
Antidilutive securities excluded from the computation of diluted net loss per share | |||
Total | 34,119 | 34,696 | 30,009 |
RSUs | |||
Antidilutive securities excluded from the computation of diluted net loss per share | |||
Total | 1,866 | 1,104 | 226 |
REVENUES - Disaggregated (Detai
REVENUES - Disaggregated (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Disaggregation of Revenue [Line Items] | |||
Total revenues | $ 116,882,000 | $ 120,242,000 | $ 149,236,000 |
Gross product sales | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 147,058,000 | 108,523,000 | 81,186,000 |
Discounts and allowances | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | (42,764,000) | (31,805,000) | (18,176,000) |
Product sales, net | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 104,294,000 | 76,718,000 | 63,010,000 |
License revenues | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 7,932,000 | 70,553,000 | |
Milestone | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 75,000 | 25,000,000 | 1,875,000 |
Delivery of drug supplies, royalty and others | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 11,413,000 | 6,092,000 | 3,298,000 |
Revenues from collaborations | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 11,488,000 | 39,024,000 | 75,726,000 |
Government contracts | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | $ 1,100,000 | $ 4,500,000 | $ 10,500,000 |
REVENUES - Activity (Details)
REVENUES - Activity (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
SEC Schedule, 12-09, Valuation and Qualifying Accounts Disclosure [Line Items] | |||
Balance | $ 12,145 | $ 7,915 | |
Provision related to current period sales | 41,498 | 30,903 | $ 16,800 |
Credit or payments made during the period | (37,959) | (26,673) | |
Balance | 15,684 | 12,145 | 7,915 |
Other accrued liabilities | 5,334 | 6,485 | |
Accounts receivable and prepaid and other current assets | |||
SEC Schedule, 12-09, Valuation and Qualifying Accounts Disclosure [Line Items] | |||
Provision related to current period sales | 1,300 | 900 | 1,400 |
Chargebacks, Discounts and Fees | |||
SEC Schedule, 12-09, Valuation and Qualifying Accounts Disclosure [Line Items] | |||
Balance | 6,213 | 3,404 | |
Provision related to current period sales | 32,330 | 23,488 | |
Credit or payments made during the period | (30,307) | (20,679) | |
Balance | 8,236 | 6,213 | 3,404 |
Government and Other Rebates | |||
SEC Schedule, 12-09, Valuation and Qualifying Accounts Disclosure [Line Items] | |||
Balance | 2,636 | 2,494 | |
Provision related to current period sales | 8,299 | 5,901 | |
Credit or payments made during the period | (7,418) | (5,759) | |
Balance | 3,517 | 2,636 | 2,494 |
Returns | |||
SEC Schedule, 12-09, Valuation and Qualifying Accounts Disclosure [Line Items] | |||
Balance | 3,296 | 2,017 | |
Provision related to current period sales | 869 | 1,514 | |
Credit or payments made during the period | (234) | (235) | |
Balance | $ 3,931 | $ 3,296 | $ 2,017 |
REVENUES - Percentage by Custom
REVENUES - Percentage by Customer (Details) - Sales - Customer Concentration Risk | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
McKesson Specialty Care Distribution Corporation | |||
Disaggregation of Revenue [Line Items] | |||
Percentage | 43% | 31% | 20% |
Cardinal Healthcare | |||
Disaggregation of Revenue [Line Items] | |||
Percentage | 25% | 19% | |
ASD Healthcare and Oncology Supply | |||
Disaggregation of Revenue [Line Items] | |||
Percentage | 21% | 17% | 17% |
Kissei | |||
Disaggregation of Revenue [Line Items] | |||
Percentage | 24% | ||
Lilly | |||
Disaggregation of Revenue [Line Items] | |||
Percentage | 48% |
SPONSORED RESEARCH AND LICENS_2
SPONSORED RESEARCH AND LICENSE AGREEMENTS AND GOVERNMENT CONTRACT (Details) $ in Thousands | 1 Months Ended | 12 Months Ended | 36 Months Ended | |||||||||||
Dec. 31, 2023 USD ($) | Aug. 31, 2023 USD ($) | Jan. 31, 2023 USD ($) | Feb. 28, 2021 USD ($) | Jan. 31, 2021 USD ($) | Feb. 29, 2020 USD ($) | Oct. 31, 2019 USD ($) | Jan. 31, 2019 USD ($) | Oct. 31, 2018 USD ($) | Dec. 31, 2023 USD ($) agreement period | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | Dec. 31, 2023 USD ($) | Jan. 31, 2024 USD ($) | |
Collaborations | ||||||||||||||
Right to opt out of agreement, different specified times | period | 2 | |||||||||||||
Upfront payment received | $ 125,000 | |||||||||||||
Financing liability with accreted interest expense | $ 46,200 | |||||||||||||
Accretion expense | $ 700 | 2,800 | ||||||||||||
Payment of cost share to collaboration partner | 2,632 | 15,116 | ||||||||||||
Accounts receivable | $ 30,550 | 30,550 | 40,320 | $ 30,550 | ||||||||||
Research and development | 24,522 | 60,272 | 65,237 | |||||||||||
Intangible asset | 13,878 | 13,878 | 14,949 | 13,878 | ||||||||||
Accounts payable | 7,142 | 7,142 | 22,508 | 7,142 | ||||||||||
Amortization of intangible assets | 1,100 | 100 | ||||||||||||
Collaborative arrangement | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | 1,300,000 | |||||||||||||
License agreement with unrelated third party | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | 4,000 | |||||||||||||
Proceeds from license fees received | $ 4,000 | |||||||||||||
One-time fee received from license rights granted | $ 4,000 | |||||||||||||
Specified Development Events | Collaborative arrangement | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | 279,500 | |||||||||||||
Specified Regulatory Events | Collaborative arrangement | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | 263,100 | |||||||||||||
Specified Product Launch Events | Collaborative arrangement | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | 796,000 | |||||||||||||
Development and regulatory milestones by non-CNS disease products | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | 330,000 | |||||||||||||
Development and regulatory milestones by non-CNS disease products | Milestone payments on a product-by-product basis | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | 100,000 | |||||||||||||
Development and regulatory milestones by CNS disease products | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | 255,000 | |||||||||||||
Development and regulatory milestones by CNS disease products | Milestone payments on a product-by-product basis | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | 150,000 | |||||||||||||
Delivery of drug supply for commercialization | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | 200 | |||||||||||||
Grifols | ||||||||||||||
Collaborations | ||||||||||||||
Upfront payment received | $ 30,000 | |||||||||||||
Deferred revenue | 0 | 0 | 0 | |||||||||||
Grifols | Delivery of drug supply for commercialization | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | 5,600 | 1,600 | 2,000 | |||||||||||
Grifols | Commercial milestones | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | $ 297,500 | |||||||||||||
Kissei | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | $ 147,000 | |||||||||||||
Upfront payment received | 33,000 | |||||||||||||
Revenue, remaining performance obligation | $ 33,000 | |||||||||||||
Revenue recognized | $ 2,200 | 2,600 | 300 | |||||||||||
Medison | Commercial and license agreements | ||||||||||||||
Collaborations | ||||||||||||||
Number of agreements | agreement | 2 | |||||||||||||
Knight | Commercial and license agreements | ||||||||||||||
Collaborations | ||||||||||||||
Upfront payment received | 2,000 | |||||||||||||
Revenue recognized | 2,000 | |||||||||||||
Knight | Minimum | Commercial and license agreements | ||||||||||||||
Collaborations | ||||||||||||||
Royalty payment as a percentage of net sales | 20% | |||||||||||||
Knight | Maximum | Commercial and license agreements | ||||||||||||||
Collaborations | ||||||||||||||
Contingent payments | 20,000 | |||||||||||||
Royalty payment as a percentage of net sales | 30% | |||||||||||||
Daiichi Sankyo | Collaborative arrangement | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | 1,800 | |||||||||||||
Forma | ||||||||||||||
Collaborations | ||||||||||||||
Upfront fee | 2,000 | |||||||||||||
Research and development | 2,000 | |||||||||||||
Forma | Commercial milestones | ||||||||||||||
Collaborations | ||||||||||||||
Potential payments | 165,500 | |||||||||||||
Forma | Development and regulatory milestones | ||||||||||||||
Collaborations | ||||||||||||||
Potential payments | 67,500 | |||||||||||||
Forma | Achievement of certain near-term regulatory milestone. | ||||||||||||||
Collaborations | ||||||||||||||
Research and development | 2,500 | |||||||||||||
fostamatinib | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | $ 1,000 | 4,500 | 10,500 | 16,000 | ||||||||||
fostamatinib | Maximum | ||||||||||||||
Collaborations | ||||||||||||||
Government contract | $ 16,500 | |||||||||||||
fostamatinib | Grifols | ||||||||||||||
Collaborations | ||||||||||||||
Collaborative payment received | $ 20,000 | |||||||||||||
fostamatinib | Grifols | Upon EMA approval of fostamatinib for treatment of chronic ITP | ||||||||||||||
Collaborations | ||||||||||||||
Collaborative payment received | 17,500 | |||||||||||||
fostamatinib | Grifols | Creditable advance royalty payment | ||||||||||||||
Collaborations | ||||||||||||||
Collaborative payment received | $ 2,500 | |||||||||||||
fostamatinib | Grifols | Maximum | ||||||||||||||
Collaborations | ||||||||||||||
Royalty payment as a percentage of net sales | 30% | |||||||||||||
fostamatinib | Kissei | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | $ 20,000 | $ 5,000 | ||||||||||||
Deferred revenue | 1,400 | 1,400 | 1,400 | 1,400 | ||||||||||
Collaborative payment received | 5,000 | |||||||||||||
Accounts receivable | 20,000 | 20,000 | 20,000 | |||||||||||
fostamatinib | Medison | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | 500 | |||||||||||||
fostamatinib | Medison | Financing arrangement | ||||||||||||||
Collaborations | ||||||||||||||
Upfront payment received | $ 5,000 | |||||||||||||
Financing component liability | 0 | 0 | 0 | 0 | ||||||||||
Revenue recognized | 5,700 | |||||||||||||
fostamatinib | Medison | Achievement of milestones | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | 100 | |||||||||||||
fostamatinib | BARDA | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | $ 100 | |||||||||||||
fostamatinib | BARDA | Maximum | ||||||||||||||
Collaborations | ||||||||||||||
Potential payments | $ 800 | |||||||||||||
R552 | ||||||||||||||
Collaborations | ||||||||||||||
Company's percentage of development costs | 20% | |||||||||||||
Financing component liability | 57,900 | $ 57,900 | 57,900 | |||||||||||
Financing liability interest accretion discount rate | 6.40% | |||||||||||||
Financing liability with accreted interest expense | 43,600 | $ 43,600 | 43,600 | |||||||||||
Development costs due to collaboration partner | 800 | 800 | 800 | |||||||||||
Payment of cost share to collaboration partner | $ 18,600 | |||||||||||||
Revenue, remaining performance obligation | 67,100 | |||||||||||||
R552 | Amended Lilly Agreement | ||||||||||||||
Collaborations | ||||||||||||||
Agreement opt-out right, exercise period | 30 days | |||||||||||||
Funding commitment | 22,600 | $ 22,600 | 22,600 | |||||||||||
R552 | Maximum | ||||||||||||||
Collaborations | ||||||||||||||
Funding commitment | 65,000 | 65,000 | 65,000 | |||||||||||
Non-CNS penetrant IP | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | 60,400 | |||||||||||||
Non-CNS penetrant IP | Licensed Rights | ||||||||||||||
Collaborations | ||||||||||||||
Revenue, remaining performance obligation | 6,700 | |||||||||||||
CNS penetrant IP | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | 500 | 6,200 | ||||||||||||
Research and development services | Grifols | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | 700 | $ 900 | ||||||||||||
Royalty | Grifols | ||||||||||||||
Collaborations | ||||||||||||||
Revenue recognized | 3,200 | 700 | ||||||||||||
REZLIDHIA | Strategic collaboration agreement with MD Anderson | ||||||||||||||
Collaborations | ||||||||||||||
Development costs due to collaboration partner | 15,000 | $ 15,000 | $ 15,000 | |||||||||||
Payment of cost share to collaboration partner | $ 2,000 | |||||||||||||
Collaboration period | 5 years | |||||||||||||
REZLIDHIA | Strategic collaboration agreement with CONNECT | ||||||||||||||
Collaborations | ||||||||||||||
Collaboration period | 4 years | |||||||||||||
REZLIDHIA | FDA approval and first commercial sale of product | ||||||||||||||
Collaborations | ||||||||||||||
Accounts payable | 15,000 | |||||||||||||
REZLIDHIA | Forma | ||||||||||||||
Collaborations | ||||||||||||||
Amortization of intangible assets | $ 1,100 | 100 | ||||||||||||
Royalty expense | $ 1,600 | $ 100 | ||||||||||||
Subsequent event | REZLIDHIA | Strategic collaboration agreement with CONNECT | ||||||||||||||
Collaborations | ||||||||||||||
Development costs due to collaboration partner | $ 3,000 |
STOCK-BASED COMPENSATION (Detai
STOCK-BASED COMPENSATION (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Stock-based compensation expense related to stock-based awards | |||
Total stock-based compensation expense | $ 8,806 | $ 12,385 | $ 9,486 |
Incremental stock-based compensation expense | 500 | 1,400 | 400 |
Selling, general and administrative | |||
Stock-based compensation expense related to stock-based awards | |||
Total stock-based compensation expense | 6,712 | 10,217 | 7,337 |
Research and development | |||
Stock-based compensation expense related to stock-based awards | |||
Total stock-based compensation expense | $ 2,094 | $ 2,168 | 1,700 |
Restructuring charges | |||
Stock-based compensation expense related to stock-based awards | |||
Total stock-based compensation expense | $ 449 |
STOCK-BASED COMPENSATION - Plan
STOCK-BASED COMPENSATION - Plans (Details) | 1 Months Ended | 12 Months Ended |
May 31, 2023 shares | Dec. 31, 2023 plan shares | |
STOCK-BASED COMPENSATION | ||
Number of active equity plans | plan | 2 | |
Employee Stock Option [Member] | ||
STOCK-BASED COMPENSATION | ||
Options exercised during the period (in shares) | 50,161 | |
2018 Equity Incentive Plan | ||
STOCK-BASED COMPENSATION | ||
Additional shares approved | 4,000,000 | |
2018 Equity Incentive Plan | Maximum | ||
STOCK-BASED COMPENSATION | ||
Expiration period | 10 years | |
Inducement Plan | ||
STOCK-BASED COMPENSATION | ||
Additional shares approved | 1,019,700 |
STOCK-BASED COMPENSATION - Opti
STOCK-BASED COMPENSATION - Options and RSUs (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Additional disclosures | |||
Total unrecognized compensation costs | $ 10,600 | ||
Weighted-average recognition period of unrecognized compensation cost | 2 years 5 months 12 days | ||
Employee stock options and Restricted Stock Units | |||
Shares Available For Grant | |||
Outstanding, beginning of period (in shares) | 10,612,618 | ||
Authorized for grant (in shares) | 5,019,700 | ||
Granted (in shares) | (5,669,944) | ||
Cancelled and forfeited (in shares) | 3,908,921 | ||
Outstanding, end of period (in shares) | 13,871,295 | 10,612,618 | |
Employee Stock Option [Member] | |||
Number of Shares | |||
Outstanding, beginning of period (in shares) | 34,696,273 | ||
Granted (in shares) | 3,671,800 | ||
Exercised/Released (in shares) | (50,161) | ||
Cancelled and forfeited (in shares) | (4,199,147) | ||
Outstanding, end of period (in shares) | 34,118,765 | 34,696,273 | |
Vested and expected to vest (in shares) | 31,373,765 | ||
Exercisable (in shares) | 24,378,890 | ||
Weighted-Average Exercise Price | |||
Outstanding, beginning of period (in dollars per share) | $ 2.74 | ||
Granted (in dollars per share) | 1.69 | ||
Exercised/Released (in dollars per share) | 0.97 | ||
Cancelled and forfeited (in dollars per share) | 3.29 | ||
Outstanding, end of period (in dollars per share) | 2.56 | $ 2.74 | |
Vested and expected to vest (in dollars per share) | 2.55 | ||
Exercisable (in dollars per share) | $ 2.70 | ||
Weighted Intrinsic Value (in thousands) | |||
Outstanding (in thousands) | $ 1,453 | $ 1,605 | |
Vested and Expected to Vest (in thousands) | 1,349 | ||
Exercisable (in thousands) | $ 403 | ||
Number of Shares | |||
Granted (in shares) | 3,671,800 | 8,179,113 | 6,997,981 |
Additional disclosures | |||
Number of shares vested | 4,346,977 | 5,280,235 | 4,765,814 |
Shares vested, weighted average exercise price | $ 2.34 | $ 2.60 | $ 2.67 |
Aggregate intrinsic value of options exercised | $ 20 | $ 200 | $ 2,100 |
RSUs | |||
Number of Shares | |||
Outstanding, beginning of period (in shares) | 1,103,653 | ||
Granted (in shares) | 1,387,600 | ||
Exercised/Released (in shares) | (435,006) | ||
Cancelled and forfeited (in shares) | (190,356) | ||
Outstanding, end of period (in shares) | 1,865,891 | 1,103,653 | |
Weighted-Average Grant Date Fair Value | |||
Outstanding, beginning of period (in dollars per share) | $ 2.39 | ||
Granted (in dollars per share) | 1.80 | ||
Exercised/Released (in dollars per share) | 2.32 | ||
Cancelled and forfeited (in dollars per share) | 2.20 | ||
Outstanding, end of period (in dollars per share) | $ 1.98 | $ 2.39 | |
Performance shares | |||
Number of Shares | |||
Outstanding, end of period (in shares) | 2,745,000 | ||
Additional disclosures | |||
Total unrecognized compensation costs | $ 5,100 |
STOCK-BASED COMPENSATION - Assu
STOCK-BASED COMPENSATION - Assumptions (Details) - USD ($) $ / shares in Units, $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Weighted-average assumptions | |||
Dividend yield (as a percent) | 0% | ||
Additional disclosures | |||
Total unrecognized compensation costs | $ 10.6 | ||
Weighted-average recognition period of unrecognized compensation cost | 2 years 5 months 12 days | ||
Purchase Plan | |||
Weighted-average assumptions | |||
Risk-free interest rate (as a percent) | 3.10% | ||
Expected term (in years) | 1 year 3 months 18 days | ||
Dividend yield (as a percent) | 0% | ||
Expected volatility (as a percent) | 121% | ||
Weighted-average grant date fair value (in dollars per share) | $ 0.78 | ||
Additional disclosures | |||
Weighted-average recognition period of unrecognized compensation cost | 5 months 26 days | ||
Employee Stock Option [Member] | |||
Weighted-average assumptions | |||
Risk-free interest rate (as a percent) | 3.90% | 2.60% | 1% |
Expected term (in years) | 6 years 9 months 18 days | 6 years 3 months 18 days | 6 years 4 months 24 days |
Dividend yield (as a percent) | 0% | 0% | 0% |
Expected volatility (as a percent) | 84% | 74.80% | 70.50% |
Weighted-average grant date fair value (in dollars per share) | $ 1.28 | $ 1.29 | $ 2.34 |
STOCK-BASED COMPENSATION - Stoc
STOCK-BASED COMPENSATION - Stock Options by Exercise Price (Details) - Employee Stock Option [Member] - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Stock options by exercise price | |||
Range of exercise prices, low end of the range (in dollars per share) | $ 0.90 | ||
Range of exercise prices, high end of the range (in dollars per share) | $ 4.50 | ||
Options Outstanding | |||
Number of Outstanding Options (in shares) | 34,118,765 | ||
Weighted-Average Remaining Contractual Life (in years) | 5 years 6 months 21 days | ||
Weighted-Average Exercise Price (in dollars per share) | $ 2.56 | ||
Options Exercisable | |||
Number of Options (in shares) | 24,378,890 | ||
Weighted Average Exercise Price (in dollars per share) | $ 2.70 | ||
Additional disclosures | |||
Aggregate intrinsic value of stock options vested and expected to vest | $ 1,349 | ||
Aggregate intrinsic value of options exercised | $ 20 | $ 200 | $ 2,100 |
$0.90 - $1.87 | |||
Stock options by exercise price | |||
Range of exercise prices, low end of the range (in dollars per share) | $ 0.90 | ||
Range of exercise prices, high end of the range (in dollars per share) | $ 1.87 | ||
Options Outstanding | |||
Number of Outstanding Options (in shares) | 6,712,896 | ||
Weighted-Average Remaining Contractual Life (in years) | 8 years 7 months 9 days | ||
Weighted-Average Exercise Price (in dollars per share) | $ 1.43 | ||
Options Exercisable | |||
Number of Options (in shares) | 1,991,518 | ||
Weighted Average Exercise Price (in dollars per share) | $ 1.44 | ||
$1.96 - $2.11 | |||
Stock options by exercise price | |||
Range of exercise prices, low end of the range (in dollars per share) | 1.96 | ||
Range of exercise prices, high end of the range (in dollars per share) | $ 2.11 | ||
Options Outstanding | |||
Number of Outstanding Options (in shares) | 5,063,237 | ||
Weighted-Average Remaining Contractual Life (in years) | 4 years 5 months 8 days | ||
Weighted-Average Exercise Price (in dollars per share) | $ 2.03 | ||
Options Exercisable | |||
Number of Options (in shares) | 5,063,237 | ||
Weighted Average Exercise Price (in dollars per share) | $ 2.03 | ||
$2.14 - $2.40 | |||
Stock options by exercise price | |||
Range of exercise prices, low end of the range (in dollars per share) | 2.14 | ||
Range of exercise prices, high end of the range (in dollars per share) | $ 2.40 | ||
Options Outstanding | |||
Number of Outstanding Options (in shares) | 3,940,279 | ||
Weighted-Average Remaining Contractual Life (in years) | 2 years 10 months 20 days | ||
Weighted-Average Exercise Price (in dollars per share) | $ 2.26 | ||
Options Exercisable | |||
Number of Options (in shares) | 3,717,256 | ||
Weighted Average Exercise Price (in dollars per share) | $ 2.26 | ||
$2.42 - $2.42 | |||
Stock options by exercise price | |||
Range of exercise prices, low end of the range (in dollars per share) | 2.42 | ||
Range of exercise prices, high end of the range (in dollars per share) | $ 2.42 | ||
Options Outstanding | |||
Number of Outstanding Options (in shares) | 5,816,323 | ||
Weighted-Average Remaining Contractual Life (in years) | 6 years 9 months 21 days | ||
Weighted-Average Exercise Price (in dollars per share) | $ 2.42 | ||
Options Exercisable | |||
Number of Options (in shares) | 3,794,160 | ||
Weighted Average Exercise Price (in dollars per share) | $ 2.42 | ||
$2.44 - $3.54 | |||
Stock options by exercise price | |||
Range of exercise prices, low end of the range (in dollars per share) | 2.44 | ||
Range of exercise prices, high end of the range (in dollars per share) | $ 3.54 | ||
Options Outstanding | |||
Number of Outstanding Options (in shares) | 6,852,637 | ||
Weighted-Average Remaining Contractual Life (in years) | 5 years 1 month 20 days | ||
Weighted-Average Exercise Price (in dollars per share) | $ 3.16 | ||
Options Exercisable | |||
Number of Options (in shares) | 4,473,712 | ||
Weighted Average Exercise Price (in dollars per share) | $ 3.06 | ||
$3.59 - $4.49 | |||
Stock options by exercise price | |||
Range of exercise prices, low end of the range (in dollars per share) | 3.59 | ||
Range of exercise prices, high end of the range (in dollars per share) | $ 4.49 | ||
Options Outstanding | |||
Number of Outstanding Options (in shares) | 5,542,956 | ||
Weighted-Average Remaining Contractual Life (in years) | 3 years 11 months 12 days | ||
Weighted-Average Exercise Price (in dollars per share) | $ 3.97 | ||
Options Exercisable | |||
Number of Options (in shares) | 5,215,904 | ||
Weighted Average Exercise Price (in dollars per share) | $ 3.99 | ||
$4.50 - $4.50 | |||
Stock options by exercise price | |||
Range of exercise prices, low end of the range (in dollars per share) | 4.50 | ||
Range of exercise prices, high end of the range (in dollars per share) | $ 4.50 | ||
Options Outstanding | |||
Number of Outstanding Options (in shares) | 190,437 | ||
Weighted-Average Remaining Contractual Life (in years) | 7 years 3 months 14 days | ||
Weighted-Average Exercise Price (in dollars per share) | $ 4.50 | ||
Options Exercisable | |||
Number of Options (in shares) | 123,103 | ||
Weighted Average Exercise Price (in dollars per share) | $ 4.50 |
STOCK-BASED COMPENSATION - Empl
STOCK-BASED COMPENSATION - Employee Stock Purchase Plan (Details) $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 USD ($) $ / shares shares | Dec. 31, 2022 $ / shares shares | Dec. 31, 2021 $ / shares shares | |
Stock Based Compensation | |||
Weighted-average recognition period of unrecognized compensation cost | 2 years 5 months 12 days | ||
Purchase Plan | |||
Stock Based Compensation | |||
Purchase price expressed as a percentage of fair market value of common stock on the first day of the offering period | 85% | ||
Purchase price expressed as a percentage of fair market value of common stock on the purchase date | 85% | ||
Number of shares of common stock issued | 941,798 | 1,146,851 | 932,018 |
Average price of shares issued (in dollars per share) | $ / shares | 1.06 | 1.01 | 1.51 |
Number of shares of common stock available for future issuance | 2,495,835 | ||
Award offering period | 24 months | ||
Unrecognized compensation cost related to purchase plan | $ | $ 0.1 | ||
Weighted-average recognition period of unrecognized compensation cost | 5 months 26 days |
INVENTORIES (Details)
INVENTORIES (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
INVENTORIES | ||
Raw Materials | $ 4,609 | $ 4,555 |
Work in process | 1,876 | 2,659 |
Finished goods | 1,508 | 1,904 |
Reported as: | ||
Inventories | 5,522 | 9,118 |
Other assets | 2,471 | |
Total | 7,993 | 9,118 |
Advance payments for raw materials | $ 0 | $ 800 |
CASH, CASH EQUIVALENTS AND SH_3
CASH, CASH EQUIVALENTS AND SHORT-TERM INVESTMENTS (Details) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 USD ($) security | Dec. 31, 2022 USD ($) | |
Cash, cash equivalent and short term investments | ||
Cash and cash equivalents | $ 32,786 | $ 24,459 |
Short-term Investments | 24,147 | 33,747 |
Cash, cash equivalents and short-term investments | 56,933 | 58,206 |
Available-For-Sale Securities Reconciliation | ||
Amortized Cost | 38,993 | 47,940 |
Fair Value | $ 39,001 | $ 47,787 |
Weighted-average time to maturity of cash equivalents and available-for-sale securities | 82 days | 89 days |
Number of investments in continuous unrealized loss position for more than 12 months | security | 0 | |
Fair value and gross unrealized losses of investments in individual securities in unrealized loss position | ||
Number of individual securities in unrealized loss position for 12 months or less | security | 10 | |
Fair Value | $ 10,541 | |
Unrealized Losses | (7) | |
Money market funds | ||
Cash, cash equivalent and short term investments | ||
Cash, cash equivalents and short-term investments | 9,685 | $ 4,155 |
US treasury bills | ||
Cash, cash equivalent and short term investments | ||
Cash, cash equivalents and short-term investments | 12,594 | 5,225 |
Available-For-Sale Securities Reconciliation | ||
Amortized Cost | 12,591 | 5,251 |
Fair Value | 12,594 | 5,225 |
Fair value and gross unrealized losses of investments in individual securities in unrealized loss position | ||
Fair Value | 1,000 | |
Government-sponsored enterprise securities | ||
Cash, cash equivalent and short term investments | ||
Cash, cash equivalents and short-term investments | 11,233 | 15,796 |
Available-For-Sale Securities Reconciliation | ||
Amortized Cost | 11,230 | 15,882 |
Fair Value | 11,233 | 15,796 |
Fair value and gross unrealized losses of investments in individual securities in unrealized loss position | ||
Fair Value | 2,980 | |
Unrealized Losses | (4) | |
Corporate bonds and commercial paper | ||
Cash, cash equivalent and short term investments | ||
Cash, cash equivalents and short-term investments | 15,174 | 26,766 |
Available-For-Sale Securities Reconciliation | ||
Amortized Cost | 15,172 | 26,807 |
Fair Value | 15,174 | 26,766 |
Fair value and gross unrealized losses of investments in individual securities in unrealized loss position | ||
Fair Value | 6,561 | |
Unrealized Losses | (3) | |
Gross Unrealized Gains | ||
Available-For-Sale Securities Reconciliation | ||
Gross Unrealized Gains (Losses) | 15 | 1 |
Gross Unrealized Gains | US treasury bills | ||
Available-For-Sale Securities Reconciliation | ||
Gross Unrealized Gains (Losses) | 3 | |
Gross Unrealized Gains | Government-sponsored enterprise securities | ||
Available-For-Sale Securities Reconciliation | ||
Gross Unrealized Gains (Losses) | 7 | 1 |
Gross Unrealized Gains | Corporate bonds and commercial paper | ||
Available-For-Sale Securities Reconciliation | ||
Gross Unrealized Gains (Losses) | 5 | |
Gross Unrealized Losses | ||
Available-For-Sale Securities Reconciliation | ||
Gross Unrealized Gains (Losses) | (7) | (154) |
Gross Unrealized Losses | US treasury bills | ||
Available-For-Sale Securities Reconciliation | ||
Gross Unrealized Gains (Losses) | (26) | |
Gross Unrealized Losses | Government-sponsored enterprise securities | ||
Available-For-Sale Securities Reconciliation | ||
Gross Unrealized Gains (Losses) | (4) | (87) |
Gross Unrealized Losses | Corporate bonds and commercial paper | ||
Available-For-Sale Securities Reconciliation | ||
Gross Unrealized Gains (Losses) | (3) | (41) |
Cash | ||
Cash, cash equivalent and short term investments | ||
Cash, cash equivalents and short-term investments | $ 8,247 | $ 6,264 |
FAIR VALUE (Details)
FAIR VALUE (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Fair Value | ||
Investments at fair value | $ 39,001 | $ 47,787 |
Fair Value Measurements Recurring | ||
Fair Value | ||
Investments at fair value | 48,686 | 51,942 |
Fair Value Measurements Recurring | Money market funds | ||
Fair Value | ||
Investments at fair value | 9,685 | 4,155 |
Fair Value Measurements Recurring | US treasury bills | ||
Fair Value | ||
Investments at fair value | 12,594 | 5,225 |
Fair Value Measurements Recurring | Government-sponsored enterprise securities | ||
Fair Value | ||
Investments at fair value | 11,233 | 15,796 |
Fair Value Measurements Recurring | Corporate bonds and commercial paper | ||
Fair Value | ||
Investments at fair value | 15,174 | 26,766 |
Fair Value Measurements Recurring | Level 1 | ||
Fair Value | ||
Investments at fair value | 9,685 | 4,155 |
Fair Value Measurements Recurring | Level 1 | Money market funds | ||
Fair Value | ||
Investments at fair value | 9,685 | 4,155 |
Fair Value Measurements Recurring | Level 2 | ||
Fair Value | ||
Investments at fair value | 39,001 | 47,787 |
Fair Value Measurements Recurring | Level 2 | US treasury bills | ||
Fair Value | ||
Investments at fair value | 12,594 | 5,225 |
Fair Value Measurements Recurring | Level 2 | Government-sponsored enterprise securities | ||
Fair Value | ||
Investments at fair value | 11,233 | 15,796 |
Fair Value Measurements Recurring | Level 2 | Corporate bonds and commercial paper | ||
Fair Value | ||
Investments at fair value | $ 15,174 | $ 26,766 |
OTHER BALANCE SHEET COMPONENT_2
OTHER BALANCE SHEET COMPONENTS - Property and Equipment (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
PROPERTY AND EQUIPMENT | |||
Total property and equipment | $ 1,869 | $ 11,664 | |
Less accumulated depreciation and amortization | (1,704) | (10,807) | |
Property and equipment, net | 165 | 857 | |
Depreciation and amortization expense | 200 | 900 | $ 1,200 |
Laboratory equipment | |||
PROPERTY AND EQUIPMENT | |||
Total property and equipment | 1,470 | 7,435 | |
Computer and software | |||
PROPERTY AND EQUIPMENT | |||
Total property and equipment | 363 | 2,048 | |
Leasehold improvements, furniture and equipment | |||
PROPERTY AND EQUIPMENT | |||
Total property and equipment | 2,107 | ||
Others | |||
PROPERTY AND EQUIPMENT | |||
Total property and equipment | $ 36 | $ 74 |
OTHER BALANCE SHEET COMPONENT_3
OTHER BALANCE SHEET COMPONENTS - Intangible Asset (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
OTHER BALANCE SHEET COMPONENTS | ||
Intangible asset cost | $ 15,000 | $ 15,000 |
Accumulated amortization | (1,122) | (51) |
Intangible asset, net | $ 13,878 | 14,949 |
Estimated useful life | 14 years | |
Amortization expense | $ 1,100 | $ 100 |
OTHER BALANCE SHEET COMPONENT_4
OTHER BALANCE SHEET COMPONENTS - Estimated Future Amortization Expense Of Intangible Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
OTHER BALANCE SHEET COMPONENTS | ||
2024 | $ 1,071 | |
2025 | 1,071 | |
2026 | 1,071 | |
2027 | 1,071 | |
2028 | 1,071 | |
Thereafter | 8,523 | |
Intangible asset, net | $ 13,878 | $ 14,949 |
OTHER BALANCE SHEET COMPONENT_5
OTHER BALANCE SHEET COMPONENTS - Other Accrued Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
OTHER BALANCE SHEET COMPONENTS | ||
Accrued commercial expenses | $ 1,852 | $ 3,144 |
Accrued other expenses | 3,482 | 3,341 |
Total other accrued liabilities | $ 5,334 | $ 6,485 |
Debt (Summary of Loans Payable)
Debt (Summary of Loans Payable) (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
DEBT | ||
Principal outstanding | $ 60,000 | $ 40,000 |
Unamortized debt issuance costs | (398) | (552) |
Principal outstanding, net of unamortized debt issuance costs | 59,602 | 39,448 |
Reported as: | ||
Loans payable, net, current portion | 7,229 | |
Long-term portion of loans payable, net | 52,373 | 39,448 |
Total | $ 59,602 | $ 39,448 |
DEBT (Narrative) (Details)
DEBT (Narrative) (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Mar. 31, 2023 | |
Debt Instrument [Line Items] | ||||
Interest expense | $ 6,872 | $ 3,707 | $ 4,860 | |
Accrued interest | 1,500 | 800 | ||
Credit Facility with MidCap | ||||
Debt Instrument [Line Items] | ||||
Maximum borrowing capacity | $ 60,000 | |||
Final payment fee, percentage of principal | 2.50% | |||
Outstanding balance | $ 60,000 | |||
Debt issuance costs being amortized ratably | 400 | |||
Interest expense | $ 6,800 | $ 3,000 | $ 1,700 | |
Credit Facility with MidCap | SOFR | ||||
Debt Instrument [Line Items] | ||||
Rate adjustment | 0.11448% | |||
Basis spread on variable rate | 5.65% | |||
Floor rate | 1.50% | |||
Credit Facility with MidCap | LIBOR | ||||
Debt Instrument [Line Items] | ||||
Basis spread on variable rate | 5.65% | |||
Floor rate | 1.50% | |||
Credit Facility with MidCap | Tranche 1 | ||||
Debt Instrument [Line Items] | ||||
Maximum borrowing capacity | $ 10,000 | |||
Credit Facility with MidCap | Tranche 2 | ||||
Debt Instrument [Line Items] | ||||
Maximum borrowing capacity | 10,000 | |||
Credit Facility with MidCap | Tranche 3 | ||||
Debt Instrument [Line Items] | ||||
Maximum borrowing capacity | 10,000 | |||
Credit Facility with MidCap | Tranche 4 | ||||
Debt Instrument [Line Items] | ||||
Maximum borrowing capacity | $ 10,000 | |||
Credit Facility with MidCap | Tranche 5 | ||||
Debt Instrument [Line Items] | ||||
Maximum borrowing capacity | $ 20,000 |
DEBT - Future Minimum Payments
DEBT - Future Minimum Payments (Details) $ in Thousands | Dec. 31, 2023 USD ($) |
Future minimum payments | |
2025 | $ 7,500 |
2026 | 30,000 |
2027 | 22,500 |
Principal amount (Tranches 1, 2, 3 and 4) | $ 60,000 |
LEASES (Details)
LEASES (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Sublease Agreement | ||
Right-of-use assets | $ 861 | $ 1,930 |
Weighted average remaining lease term | 1 year 5 months 1 day |
LEASES - Lease Expense (Details
LEASES - Lease Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
LEASES | |||
Fixed operating lease expense | $ 1,109 | $ 5,470 | $ 5,360 |
Variable operating lease expense | 134 | 818 | 910 |
Total operating lease expense | $ 1,243 | $ 6,288 | $ 6,270 |
LEASES - Cash Flow Information
LEASES - Cash Flow Information (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
LEASES | |||
Cash payments included in the measurement of operating lease liabilities | $ 1,534 | $ 10,485 | $ 10,082 |
LEASES - Sublease Information (
LEASES - Sublease Information (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Operating sublease information | |||
Fixed sublease expense | $ 365 | $ 4,381 | $ 4,381 |
Variable sublease expense | 77 | 911 | 917 |
Sublease income | (442) | (5,292) | (5,298) |
Net |
LEASES - Future Minimum Lease P
LEASES - Future Minimum Lease Payments (Details) $ in Thousands | Dec. 31, 2023 USD ($) |
Operating Lease | |
2024 | $ 739 |
2025 | 301 |
Total minimum payments required | $ 1,040 |
STOCKHOLDERS' EQUITY - Preferre
STOCKHOLDERS' EQUITY - Preferred Stock (Details) - shares | Dec. 31, 2023 | Dec. 31, 2022 |
Preferred Stock | ||
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
STOCKHOLDERS' EQUITY - Common S
STOCKHOLDERS' EQUITY - Common Stock and Sale Agreement (Details) - USD ($) $ in Millions | 12 Months Ended | |||
May 03, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | May 18, 2018 | |
Controlled Equity Offering | ||||
Authorized number of shares of common stock | 400,000,000 | 400,000,000 | ||
Common stock, shares issued | 174,825,610 | 173,398,645 | ||
Common stock, shares outstanding | 174,825,610 | 173,398,645 | ||
Open Market Sales Agreement | ||||
Controlled Equity Offering | ||||
Maximum value of shares available under Equity Offering | $ 250 | |||
Common Stock | ||||
Controlled Equity Offering | ||||
Authorized number of shares of common stock | 400,000,000 | |||
Common Stock | Open Market Sales Agreement | ||||
Controlled Equity Offering | ||||
Maximum value of shares available under Equity Offering | $ 100 | |||
Number of shares of common stock sold (in shares) | 0 |
INCOME TAXES - Provision (Detai
INCOME TAXES - Provision (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2021 USD ($) | |
INCOME TAXES | |
Provision for income taxes | $ 605 |
INCOME TAXES (Details)
INCOME TAXES (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Deferred tax assets | |||
Net operating loss carryforwards | $ 229,967 | $ 230,373 | |
Orphan drug and research and development credits | 62,457 | 68,646 | |
Capitalized research and development credits | 21,017 | 15,680 | |
Deferred revenue | 11,223 | 11,234 | |
Deferred compensation | 10,365 | 9,620 | |
Lease liabilities | 244 | 504 | |
Other, net | 3,654 | 2,494 | |
Deferred tax liabilities | |||
Operating lease right-of-use asset | (215) | (461) | |
Others | (81) | (439) | |
Total net deferred tax assets | 338,631 | 337,651 | |
Less: valuation allowance | (338,631) | (337,651) | |
Deferred tax assets, net of allowance | |||
Reconciliation of the statutory federal income tax rate to the effective tax rate | |||
Federal statutory tax rate (as a percent) | (21.00%) | (21.00%) | (21.00%) |
State, net of federal benefit (as a percent) | 0.10% | 0% | 2.80% |
Valuation allowance (as a percent) | 13.90% | 20.20% | 27.50% |
Stock compensation (as a percent) | 5.30% | 2.50% | 5.60% |
Orphan drug and research and development credits (as a percent) | 0.20% | (2.60%) | (14.00%) |
Other, net (as a percent) | 1.50% | 1% | 2.70% |
Effective tax rate (as a percent) | 0% | 0.10% | 3.60% |
INCOME TAXES - Operating Loss C
INCOME TAXES - Operating Loss Carryforwards (Details) $ in Millions | Dec. 31, 2023 USD ($) |
Federal | |
Operating loss carryforwards | |
Net operating loss carryforwards | $ 972.6 |
Federal | Expire beginning in the year 2025 | |
Operating loss carryforwards | |
Net operating loss carryforwards | 833.8 |
State | |
Operating loss carryforwards | |
Net operating loss carryforwards | $ 388.5 |
INCOME TAXES - Tax Credits (Det
INCOME TAXES - Tax Credits (Details) - Research [Member] $ in Millions | Dec. 31, 2023 USD ($) |
Federal | |
Tax credit carryforward | |
Amount of tax credit carryforward | $ 45.1 |
State | |
Tax credit carryforward | |
Amount of tax credit carryforward | $ 31.8 |
INCOME TAXES - Valuation Allowa
INCOME TAXES - Valuation Allowance (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
INCOME TAXES | |||
Increase in valuation allowance | $ 1 | $ 15.3 | $ 6.6 |
INCOME TAXES - Unrecognized Tax
INCOME TAXES - Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Gross unrecognized tax benefits | |||
Balance at the beginning of the year | $ 9,426 | $ 9,186 | $ 8,901 |
Decrease related to prior year tax positions | (843) | ||
Increase related to current year tax positions | 89 | 240 | 285 |
Balance at the end of the year | 8,672 | $ 9,426 | $ 9,186 |
Unrecognized tax benefits decrease, evaluation of prior orphan drug credits | 4,700 | ||
Tax positions subject to interest or penalties | $ 0 |
RESTRUCTURING CHARGES (Details)
RESTRUCTURING CHARGES (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Restructuring Charges | ||
Restructuring charges | $ 1,320 | $ 3,521 |
Severance, bonus and related employee benefits and taxes | ||
Restructuring Charges | ||
Restructuring charges | 2,900 | |
Accrued restructuring liability | $ 500 | |
Stock-based compensation expense related to option modification | ||
Restructuring Charges | ||
Restructuring charges | 400 | |
Property and equipment impairment | ||
Restructuring Charges | ||
Restructuring charges | $ 100 |
SUBSEQUENT EVENTS (Details)
SUBSEQUENT EVENTS (Details) - Subsequent event - GAVRETO (pralsetinib) - Blueprint $ in Millions | Feb. 22, 2024 USD ($) |
Subsequent Event [Line Items] | |
Asset acquisition, expected price | $ 15 |
Minimum | |
Subsequent Event [Line Items] | |
Potential tiered royalties (as a percentage) | 10% |
Maximum | |
Subsequent Event [Line Items] | |
Potential tiered royalties (as a percentage) | 30% |
First commercial sale | |
Subsequent Event [Line Items] | |
Asset acquisition, expected price | $ 10 |
First anniversary of the closing date | |
Subsequent Event [Line Items] | |
Asset acquisition, expected price | 5 |
Commercial milestones | |
Subsequent Event [Line Items] | |
Potential milestone payments | 97.5 |
Regulatory milestones | |
Subsequent Event [Line Items] | |
Potential milestone payments | $ 5 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Pay vs Performance Disclosure | |||
Net Income (Loss) | $ (25,091) | $ (58,573) | $ (17,914) |
Insider Trading Arrangements
Insider Trading Arrangements | 12 Months Ended |
Dec. 31, 2023 | |
Trading Arrangements, by Individual | |
Rule 10b5-1 Arrangement Adopted | false |
Non-Rule 10b5-1 Arrangement Adopted | false |
Rule 10b5-1 Arrangement Terminated | false |
Non-Rule 10b5-1 Arrangement Terminated | false |