RIGEL PHARMACEUTICALS INC 10-Q

Item 1. Financial Statements ​ RIGEL PHARMACEUTICALS, INC. CONDENSED BALANCE SHEET S (In thousands) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of ​ ​ ​ June 30, 2023 December 31, 2022 (1) ​ ​ ​ (unaudited) ​ ​ ​ ​ Assets ​ ​ ​ ​ ​ ​ ​ Current assets: ​ ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 48,812 ​ $ 24,459 ​ Short-term investments ​ 15,545 ​ 33,747 ​ Accounts receivable, net ​ 20,819 ​ 40,320 ​ Inventories ​ ​ 10,782 ​ 9,118 ​ Prepaid and other current assets ​ 4,967 ​ 8,259 ​ Total current assets ​ 100,925 ​ 115,903 ​ Property and equipment, net ​ 215 ​ 857 ​ Intangible asset, net ​ ​ 14,413 ​ ​ 14,949 ​ Operating lease right-of-use assets ​ ​ 997 ​ ​ 1,930 ​ Other assets ​ 541 ​ 640 ​ Total assets ​ $ 117,091 ​ $ 134,279 ​ Liabilities and stockholders’ deficit ​ ​ ​ ​ ​ ​ ​ Current liabilities: ​ ​ ​ ​ ​ ​ ​ Accounts payable ​ $ 5,240 ​ $ 22,508 ​ Accrued compensation ​ 6,739 ​ 8,866 ​ Accrued research and development ​ 5,826 ​ 7,708 ​ Revenue reserves and refund liability ​ ​ 13,684 ​ ​ 12,145 ​ Other accrued liabilities ​ 5,013 ​ 6,485 ​ Lease liabilities, current portion ​ ​ 466 ​ ​ 1,133 ​ Deferred revenue ​ ​ 1,355 ​ ​ 1,369 ​ Other long-term liabilities, current portion ​ ​ 5,700 ​ ​ 4,997 ​ Total current liabilities ​ 44,023 ​ 65,211 ​ Long-term portion of lease liabilities ​ 628 ​ 972 ​ Loans payable, net of discount ​ ​ 59,574 ​ ​ 39,448 ​ Other long-term liabilities ​ 40,981 ​ 42,264 ​ Total liabilities ​ ​ 145,206 ​ ​ 147,895 ​ ​ ​ ​ ​ ​ ​ ​ ​ Commitments ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Stockholders’ deficit: ​ ​ ​ ​ ​ ​ ​ Preferred stock ​ — ​ — ​ Common stock ​ 175 ​ 174 ​ Additional paid-in capital ​ 1,374,330 ​ 1,368,822 ​ Accumulated other comprehensive loss ​ ( 25 ) ​ ( 153 ) ​ Accumulated deficit ​ ( 1,402,595 ) ​ ( 1,382,459 ) ​ Total stockholders’ deficit ​ ( 28,115 ) ​ ( 13,616 ) ​ Total liabilities and stockholders’ deficit ​ $ 117,091 ​ $ 134,279 ​ (1) The balance sheet as of December 31, 2022 has been derived from the audited financial statements included in Rigel’s Annual Report on Form 10-K for the year ended December 31, 2022 filed with the Securities and Exchange Commission (SEC) on March 7, 2023 . ​ See Accompanying Notes to Condensed Financial Statements ​ 3 ​ Table of Contents ​ RIGEL PHARMACEUTICALS, INC. CONDENSED STATEMENTS OF OPERATION S (In thousands, except per share amounts) (unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended June 30, ​ Six Months Ended June 30, ​ 2023 2022 2023 2022 Revenues: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Product sales, net ​ $ 23,881 ​ $ 18,550 ​ $ 47,626 ​ $ 34,747 Contract revenues from collaborations ​ ​ 2,005 ​ ​ 11,269 ​ ​ 4,330 ​ ​ 11,807 Government contract ​ ​ 1,000 ​ ​ — ​ ​ 1,000 ​ ​ — Total revenues ​ ​ 26,886 ​ ​ 29,819 ​ ​ 52,956 ​ ​ 46,554 Costs and expenses: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cost of product sales ​ ​ 1,075 ​ ​ 1,036 ​ ​ 2,052 ​ ​ 1,157 Research and development ​ 4,772 ​ 14,767 ​ ​ 14,861 ​ 30,241 Selling, general and administrative ​ 26,306 ​ 26,981 ​ ​ 54,035 ​ 54,382 Total costs and expenses ​ 32,153 ​ 42,784 ​ 70,948 ​ 85,780 Loss from operations ​ ( 5,267 ) ​ ( 12,965 ) ​ ( 17,992 ) ​ ( 39,226 ) Interest income ​ 529 ​ 42 ​ 922 ​ 63 Interest expense ​ ​ ( 1,862 ) ​ ​ ( 569 ) ​ ​ ( 3,066 ) ​ ​ ( 1,774 ) Net loss ​ $ ( 6,600 ) ​ $ ( 13,492 ) ​ $ ( 20,136 ) ​ $ ( 40,937 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net loss per share, basic and diluted ​ $ ( 0.04 ) ​ $ ( 0.08 ) ​ $ ( 0.12 ) ​ $ ( 0.24 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted average shares used in computing net loss per share, basic and diluted ​ ​ 173,748 ​ ​ 172,147 ​ ​ 173,659 ​ ​ 171,961 ​ See Accompanying Notes to Condensed Financial Statements ​ 4 ​ Table of Contents RIGEL PHARMACEUTICALS, INC. CONDENSED STATEMENTS OF COMPREHENSIVE LOSS (In thousands) (unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended June 30, ​ Six Months Ended June 30, ​ 2023 2022 2023 2022 Net loss ​ $ ( 6,600 ) ​ $ ( 13,492 ) ​ $ ( 20,136 ) ​ $ ( 40,937 ) Other comprehensive gain (loss): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net unrealized gain (loss) on short-term investments ​ 2 ​ ( 22 ) ​ 128 ​ ( 336 ) Comprehensive loss ​ $ ( 6,598 ) ​ $ ( 13,514 ) ​ $ ( 20,008 ) ​ $ ( 41,273 ) ​ See Accompanying Notes to Condensed Financial Statements ​ 5 ​ Table of Contents RIGEL PHARMACEUTICALS, INC. CONDENSED STATEMENTS OF STOCK HOLDERS’ (DEFICIT) EQUITY (In thousands, except share amounts) (unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ Accumulated Other ​ ​ ​ ​ Total ​ ​ Common Stock ​ Paid-in ​ Comprehensive ​ Accumulated ​ Stockholders’ ​ Shares Amount Capital Loss Deficit Deficit Balance as of January 1, 2023 173,398,645 ​ $ 174 ​ $ 1,368,822 ​ $ ( 153 ) ​ $ ( 1,382,459 ) ​ $ ( 13,616 ) Net loss — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 13,536 ) ​ ( 13,536 ) Net change in unrealized gain on short-term investments — ​ ​ — ​ ​ — ​ ​ 126 ​ ​ — ​ 126 Issuance of common stock upon exercise of options 952 ​ ​ — ​ ​ 1 ​ ​ — ​ ​ — ​ 1 Issuance of common stock upon vesting of restricted stock units (RSUs) ​ 266,256 ​ ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ — Stock-based compensation expense — ​ ​ — ​ ​ 2,768 ​ ​ — ​ ​ — ​ 2,768 Balance as of March 31, 2023 173,665,853 ​ ​ 174 ​ ​ 1,371,591 ​ ​ ( 27 ) ​ ​ ( 1,395,995 ) ​ ​ ( 24,257 ) Net loss — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 6,600 ) ​ ​ ( 6,600 ) Net change in unrealized gain on short-term investments — ​ ​ — ​ ​ — ​ ​ 2 ​ ​ — ​ ​ 2 Issuance of common stock upon exercise of options and participation in Purchase Plan 523,795 ​ ​ 1 ​ ​ 553 ​ ​ — ​ ​ — ​ ​ 554 Issuance of common stock upon vesting of RSUs ​ 168,750 ​ ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ — Stock-based compensation expense — ​ ​ — ​ ​ 2,186 ​ ​ — ​ ​ — ​ ​ 2,186 Balance as of June 30, 2023 ​ 174,358,398 ​ $ 175 ​ $ 1,374,330 ​ $ ( 25 ) ​ $ ( 1,402,595 ) ​ $ ( 28,115 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ Accumulated Other ​ ​ ​ ​ Total ​ ​ Common Stock ​ Paid-in ​ Comprehensive ​ Accumulated ​ Stockholders’ ​ Shares Amount Capital Loss Deficit Equity (Deficit) Balance as of January 1, 2022 171,602,226 ​ $ 172 ​ $ 1,354,190 ​ $ ( 102 ) ​ $ ( 1,323,886 ) ​ $ 30,374 Net loss — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 27,445 ) ​ ( 27,445 ) Net unrealized loss on short-term investments — ​ ​ — ​ ​ — ​ ​ ( 314 ) ​ ​ — ​ ( 314 ) Issuance of common stock upon exercise of options 420,521 ​ ​ — ​ ​ 940 ​ ​ — ​ ​ — ​ 940 Issuance of common stock upon vesting of RSUs ​ 22,500 ​ ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ — Stock-based compensation expense — ​ ​ — ​ ​ 3,243 ​ ​ — ​ ​ — ​ 3,243 Balance as of March 31, 2022 172,045,247 ​ ​ 172 ​ ​ 1,358,373 ​ ​ ( 416 ) ​ ​ ( 1,351,331 ) ​ ​ 6,798 Net loss — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 13,492 ) ​ ​ ( 13,492 ) Net unrealized loss on short-term investments — ​ ​ — ​ ​ — ​ ​ ( 22 ) ​ ​ — ​ ​ ( 22 ) Issuance of common stock upon exercise of options and participation in Purchase Plan 609,839 ​ ​ 1 ​ ​ 598 ​ ​ — ​ ​ — ​ ​ 599 Issuance of common stock upon vesting of RSUs ​ 181,250 ​ ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ — Stock-based compensation expense — ​ ​ — ​ ​ 2,440 ​ ​ — ​ ​ — ​ ​ 2,440 Balance as of June 30, 2022 ​ 172,836,336 ​ $ 173 ​ $ 1,361,411 ​ $ ( 438 ) ​ $ ( 1,364,823 ) ​ $ ( 3,677 ) ​ ​ ​ ​ ​ ​ ​ ​ See Accompanying Notes to Condensed Financial Statements ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 6 ​ Table of Contents ​ ​ RIGEL PHARMACEUTICALS, INC. CONDENSED STATEMENTS OF CASH FLOW S (In thousands) (unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended June 30, ​ ​ ​ 2023 2022 ​ Operating activities ​ ​ ​ ​ ​ ​ ​ Net loss ​ $ ( 20,136 ) ​ $ ( 40,937 ) ​ Adjustments to reconcile net loss to net cash used in operating activities: ​ ​ ​ ​ ​ ​ ​ Stock-based compensation expense ​ 4,930 ​ ​ 5,598 ​ Loss (gain) on sale and disposal of fixed assets ​ ​ 398 ​ ​ ( 253 ) ​ Depreciation and amortization ​ 653 ​ ​ 474 ​ Non-cash interest expense ​ ​ — ​ ​ 682 ​ Net amortization and accretion of discount on short-term investments and term loan ​ ​ ( 114 ) ​ ​ 107 ​ Changes in assets and liabilities: ​ ​ ​ ​ ​ ​ ​ Accounts receivable, net ​ 19,501 ​ ​ ( 1,726 ) ​ Inventories ​ ​ ( 1,640 ) ​ ​ 483 ​ Prepaid and other current assets ​ 3,292 ​ ​ ( 527 ) ​ Other assets ​ 99 ​ ​ 571 ​ Right-of-use assets ​ 933 ​ ​ 4,458 ​ Accounts payable ​ ( 2,268 ) ​ ​ ( 1,585 ) ​ Accrued compensation ​ ( 2,127 ) ​ ​ ( 3,165 ) ​ Accrued research and development ​ ( 1,882 ) ​ ​ ( 1,171 ) ​ Revenue reserves and refund liability ​ ​ 1,539 ​ ​ 2,316 ​ Other accrued liabilities ​ ( 1,472 ) ​ ​ 1,878 ​ Lease liability ​ ​ ( 1,011 ) ​ ​ ( 4,840 ) ​ Deferred revenue ​ ​ ( 14 ) ​ ​ ( 1,053 ) ​ Other current and long-term liabilities ​ 1,175 ​ 142 ​ Net cash provided by (used in) operating activities ​ 1,856 ​ ( 38,548 ) ​ Investing activities ​ ​ ​ ​ ​ ​ ​ Purchases of short-term investments ​ ( 8,780 ) ​ ​ ( 6,997 ) ​ Maturities of short-term investments ​ 27,400 ​ ​ 50,645 ​ Purchases of intangible asset ​ ( 15,000 ) ​ ​ — ​ Proceeds from sale of property and equipment ​ ​ 127 ​ ​ 264 ​ Purchases of property and equipment ​ — ​ ​ ( 188 ) ​ Net cash provided by investing activities ​ 3,747 ​ 43,724 ​ Financing activities ​ ​ ​ ​ ​ ​ ​ Cost share payments to a collaboration partner ​ ​ ( 1,754 ) ​ ​ ( 8,346 ) ​ Net proceeds from issuances of common stock upon exercise of options and participation in Purchase Plan ​ 554 ​ ​ 1,539 ​ Net proceeds from term loan financing ​ ​ 19,950 ​ ​ 9,942 ​ Net cash provided by financing activities ​ 18,750 ​ 3,135 ​ Net increase in cash and cash equivalents ​ 24,353 ​ 8,311 ​ Cash and cash equivalents at beginning of period ​ 24,459 ​ ​ 18,890 ​ Cash and cash equivalents at end of period ​ $ 48,812 ​ $ 27,201 ​ Supplemental disclosure of cash flow information ​ ​ ​ ​ ​ ​ ​ Interest paid ​ $ 2,485 ​ $ 951 ​ ​ See Accompanying Notes to Condensed Financial Statements ​ ​ 7 ​ Table of Contents Rigel Pharmaceuticals, In c. Notes to Condensed Financial Statements (unaudited) In this report, “Rigel,” “we,” “us” and “our” refer to Rigel Pharmaceuticals, Inc. 1. Organization and Summary of Significant Accounting Policies Description of Business We are a biotechnology company dedicated to discovering, 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 US Food and Drug Administration (FDA) is TAVALISSE ® (fostamatinib disodium hexahydrate) tablets, the only approved oral spleen tyrosine kinase (SYK) inhibitor for the treatment of adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The product is also commercially available in Europe and the United Kingdom (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 relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. We began our commercialization of REZLIDHIA in December 2022. W e in-licensed olutasidenib from Forma Therapeutics, Inc. (now Novo Nordisk), with exclusive, worldwide rights for its development, manufacturing and commercialization. We continue to advance the development of our interleukin receptor-associated kinase (IRAK) 1/4 inhibitor program, in an open-label, Phase 1b trial to determine the tolerability and preliminary efficacy of the drug in patients with lower-risk myelodysplastic syndrome (MDS) who are refractory or resistant to prior therapies. Our other ongoing clinical programs include a fostamatinib Phase 2/3 trial, the Accelerating COVID-19 Therapeutic Inventions and Vaccines Phase 2/3 trial (ACTIV-4 Host Tissue Trial), for the treatment of hospitalized high-risk patients with COVID-19 being conducted and sponsored by the National Institute of Health (NIH)/National Heart, Lung, and Blood Institute (NHLBI), and a receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitor program in clinical development with our partner Eli Lilly and Company (Lilly). We also have product candidates in clinical development with partners BerGenBio ASA (BerGenBio) and Daiichi Sankyo (Daiichi). Basis of Presentation Our accompanying unaudited condensed financial statements have been prepared in accordance with United States generally accepted accounting principles (US GAAP), for interim financial information and pursuant to the instructions to Form 10-Q and Article 10 of Regulation S-X of the Securities Act of 1933, as amended (Securities Act). Accordingly, they do not include all the information and notes required by US GAAP for complete financial statements. These unaudited condensed financial statements include only normal and recurring adjustments that we believe are necessary to fairly state our financial position and the results of our operations and cash flows. Interim-period results are not necessarily indicative of results of operations or cash flows for a full-year or any subsequent interim period. The balance sheet as of December 31, 2022 has been derived from audited financial statements at that date but does not include all disclosures required by US GAAP for complete financial statements. Because certain disclosures required by US GAAP for complete financial statements are not included herein, these interim unaudited condensed financial statements and the notes accompanying them should be read in conjunction with our audited financial statements and the notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2022 filed with the SEC on March 7, 2023. 8 ​ Table of Contents Use of Estimates The preparation of financial statements in conformity with US GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. We base our estimates on historical experience and on various other assumptions that we believe to be reasonable under the circumstances. Actual results could differ from these estimates. Significant Accounting Policies Our significant accounting policies are described in “Note 1 – Description of Business and Summary of Significant Accounting Policies” to our “Notes to Financial Statements” contained in “Part II, Item 8, Financial Statements and Supplementary Data” of our Annual Report on Form 10-K for the year ended December 31, 2022. There have been no material changes to these accounting policies. Liquidity As of June 30, 2023, we had approximately $ 64.4 million in cash, cash equivalents and short-term investments. Since inception, we have financed our operations primarily through sales of equity securities, debt financing, 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 for at least the next 12 months from the date of issuance of this Form 10-Q. Recently Issued Accounting Standards Recently issued accounting guidance is either not applicable or did not have, or is not expected to have, a material impact to us. 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 Employee Stock Purchase Plan (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): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended June 30, ​ Six Months Ended June 30, ​ ​ 2023 2022 ​ 2023 2022 Outstanding stock options ​ ​ 35,244 ​ ​ 33,081 ​ ​ 35,244 ​ ​ 33,081 RSUs ​ ​ 1,963 ​ ​ 1,191 ​ ​ 1,963 ​ ​ 1,191 Total ​ ​ 37,207 ​ ​ 34,272 ​ ​ 37,207 ​ ​ 34,272 ​ ​ ​ ​ ​ 9 ​ Table of Contents 3. Revenues ​ Revenues disaggregated by category were as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended June 30, ​ Six Months Ended June 30, ​ ​ 2023 2022 ​ 2023 2022 Product sales: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Gross product sales ​ $ 33,456 ​ $ 26,427 ​ $ 66,654 ​ ​ 49,045 Discounts and allowances ​ ​ ( 9,575 ) ​ ​ ( 7,877 ) ​ ​ ( 19,028 ) ​ ​ ( 14,298 ) Total product sales, net ​ ​ 23,881 ​ ​ 18,550 ​ ​ 47,626 ​ ​ 34,747 Revenues from collaborations: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ License revenues ​ ​ — ​ ​ 2,337 ​ ​ — ​ ​ 2,545 Development milestones ​ ​ — ​ ​ 5,000 ​ ​ — ​ ​ 5,000 Royalty, delivery of drug supplies and others ​ ​ 2,005 ​ ​ 3,932 ​ ​ 4,330 ​ ​ 4,262 Total revenues from collaborations ​ ​ 2,005 ​ ​ 11,269 ​ ​ 4,330 ​ ​ 11,807 Government contract ​ ​ 1,000 ​ ​ — ​ ​ 1,000 ​ ​ — Total revenues ​ $ 26,886 ​ $ 29,819 ​ $ 52,956 ​ $ 46,554 ​ Revenue from product sales are related to sales of our commercial products, TAVALISSE and REZLIDHIA, to our specialty distributors. For detailed discussions of our revenues from collaboration and government contract, see “Note 4 – Sponsored Research and License Agreements and Government Contract”. Our net product sales include gross product sales, net of chargebacks, discounts and fees, government and other rebates and returns. Of the total discounts and allowances from gross product sales for the six months ended June 30, 2023 and 2022, $ 18.6 million and $ 13.0 million, respectively, was accounted for as additions to revenue reserves and refund liability, and $ 0.4 million and $ 1.3 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 condensed balance sheet. The following tables summarize the activities in chargebacks, discounts and fees, government and other rebates and returns that were accounted for within 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 ​ ​ 14,439 ​ ​ 3,725 ​ ​ 395 ​ ​ 18,559 Credit or payments made during the period ​ ​ ( 13,139 ) ​ ​ ( 3,765 ) ​ ​ ( 116 ) ​ ​ ( 17,020 ) Balance as of June 30, 2023 $ 7,513 ​ $ 2,596 ​ $ 3,575 ​ $ 13,684 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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 ​ ​ 9,680 ​ ​ 2,711 ​ ​ 613 ​ ​ 13,004 Credit or payments made during the period ​ ​ ( 7,748 ) ​ ​ ( 2,901 ) ​ ​ ( 39 ) ​ ​ ( 10,688 ) Balance as of June 30, 2022 $ 5,336 ​ $ 2,304 ​ $ 2,591 ​ $ 10,231 The following table summarizes the percentages of revenues from each of our customers who individually accounted for 10% or more of the total net product sales and revenues from collaborations: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended June 30, ​ ​ Six Months Ended June 30, ​ ​ 2023 2022 ​ ​ 2023 2022 McKesson Specialty Care Distribution Corporation ​ ​ 44 % ​ ​ 34 % ​ ​ ​ 45 % ​ ​ 36 % Cardinal Healthcare ​ ​ 28 % ​ ​ 16 % ​ ​ ​ 26 % ​ ​ 20 % ASD Healthcare and Oncology Supply ​ ​ 19 % ​ ​ 12 % ​ ​ ​ 21 % ​ ​ 19 % Kissei ​ ​ — ​ ​ 25 % ​ ​ ​ — ​ ​ 16 % ​ 10 ​ Table of Contents 4. Sponsored Research and License Agreements and Government Contract Sponsored Research and License Agreements We conduct research and development programs independently and in connection with our corporate collaborators. As of June 30, 2023, we are a party to collaboration agreements with Lilly to develop and commercialize R552, a RIPK1 inhibitor, for the treatment of non-central nervous system (non-CNS) diseases and collaboration aimed at developing additional RIPK1 inhibitors for the treatment of central nervous system (CNS) diseases; with Grifols S.A. (Grifols) to commercialize fostamatinib for human diseases in all indications, including chronic ITP and autoimmune hemolytic anemia (AIHA), in Grifols territory which includes Europe, the UK, Turkey, the Middle East, North Africa and Russia (including Commonwealth of Independent States); with Kissei Pharmaceutical Co., Ltd. (Kissei) to develop and commercialize fostamatinib in Kissei territory which includes Japan, China, Taiwan and the Republic of Korea; with Medison Pharma Trading AG (Medison Canada) and Medison Pharma Ltd. (Medison Israel and, together with Medison Canada, Medison) to commercialize fostamatinib in all indications, including chronic ITP and AIHA, in Medison territory which includes Canada and Israel; and with Knight Therapeutics International SA (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 (Knight territory). 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 receptor tyrosine kinase ( AXL) inhibitors in oncology, and with Daiichi to pursue research related to murine double minute 2 ( MDM2) inhibitors, a novel class of drug targets called ligases. Under the above existing agreements that we entered into in the ordinary course of business, we received or may be entitled to receive upfront cash payments, payments contingent upon specified events achieved by such partners and royalties on any net sales of products sold by such partners under the agreements. As of June 30, 2023, total future contingent payments to us under all of the above existing agreements, excluding terminated agreements, could exceed $ 1.3 billion if all potential product candidates achieved all of the payment triggering events under all of our current agreements. Of this amount, $ 279.5 million relates to the achievement of development events, $ 263.1 million relates to the achievement of regulatory events and $ 796.0 million relates to the achievement of certain commercial events. This estimated future contingent amount does not include any estimated royalties that could be due to us if the partners successfully commercialize any of the licensed products. Future events that may trigger payments to us under the agreements are based solely on our partners’ future efforts and achievements of specified development, regulatory and/or commercial events. Global Exclusive License Agreement with Lilly We have a global exclusive license agreement and strategic collaboration 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 license agreement, we granted to Lilly 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 the 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 in milestone payments upon the achievement of specified development, regulatory and commercial 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 11 ​ Table of Contents payments on net sales of CNS disease products up to low-double digits, subject to certain standard reductions and offsets. 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. Under the Lilly Agreement, we are responsible for 20 % of development costs for R552 in the US, Europe, and Japan, up to a specified cap, and 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 at two different specified times. If we exercise our first opt-out right (no later than September 30, 2023), we are required to fund our share of the R552 development activities up to a maximum funding commitment of $ 65.0 million through April 1, 2024. If we decide not to exercise our opt-out rights, we will be required to share in global development costs of up to certain amounts at a specified cap, as provided for in the Lilly Agreement. Given our rights to opt-out from the development of R552, we believe at the minimum, we have 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, adjusted for timing of expected cost share payments. No interest was accreted during the six months ended June 30, 2023, and $ 0.7 million of interest was accreted during the six months ended June 30, 2022. Through June 30, 2023, Lilly billed us $ 16.9 million for our share of development costs under this agreement, and the amount was fully paid as of June 30, 2023. As of June 30, 2023 and December 31, 2022, the outstanding financing liability to Lilly was $ 44.5 million and $ 46.2 million, respectively, and included within other long-term liabilities, current portion, and other long-term liabilities in the condensed balance sheet. 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. 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 upon delivery of the non-CNS penetrant IP to Lilly during 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, since we were required to perform additional research and development efforts before the final acceptance of the license by Lilly. 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 three and six months ended June 30, 2022, we recognized $ 0.3 million and $ 0.5 million, respectively, of revenue associated with the delivery of CNS penetrant IP. No such revenue was recognized in the three and six months ended June 30, 2023. 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. 12 ​ Table of Contents Grifols License Agreement We have an exclusive 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 European Medicines Agency (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 European Commission (EC) granted a centralized Marketing Authorization (MA) for fostamatinib valid throughout the European Union (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, composed of a $ 17.5 million payment due upon Marketing Authorization Application (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 long-term open-label extension study on patients with ITP, and (c) performance of research services related to our Phase 3 study in AIHA. We allocated the transaction price to the distinct performance obligations in our collaboration agreement based on our best estimate of the relative standalone selling price, and recognized the corresponding revenue in the periods we satisfied the performance obligations. During the three and six months ended June 30, 2022, we recognized $ 0.2 million and $ 0.5 million, respectively, of revenue associated with the remaining performance obligation to perform research services. No such revenue was recognized during the three and six months ended June 30, 2023. ​ The remaining variable consideration related to future regulatory and commercial milestones were 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 are recognizing revenues related to 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 entered into a Commercial Supply Agreement with Grifols in October 2020 to supply and sell our drug product priced at a certain markup specified in the agreement, in quantities Grifols order from us pursuant to and in accordance with the agreement. Prior to the Commercial Supply Agreement, we had a Drug Product Purchase Agreement with Grifols entered in December 2019. During the three and six months ended June 30, 2023, we recognized $ 1.2 million and $ 2.8 million, respectively, of revenue related to delivery of drug supply to Grifols. D uring the three and six months ended June 30, 2022, we recognized $ 1.2 million of revenue related to delivery of drug supply to Grifols . ​ We began recognizing royalty revenue from Grifols beginning in the third quarter of 2022. For the three and six months ended June 30, 2023, we recognized $ 0.8 million and $ 1.5 million, respectively, of royalty revenue from Grifols, and such amount was included within contract revenues from collaboration. No such revenue was recognized for the three and six months ended June 30, 2022. ​ 13 ​ Table of Contents Kissei License Agreement ​ We have an exclusive license and supply agreement with Kissei entered in October 2018, to develop and commercialize fostamatinib in all current and potential indications in Kissei’s territory. Kissei is responsible for performing and funding all development activities for fostamatinib in the above-mentioned 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’s 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 June 30, 2023 and December 31, 2022, the remaining deferred revenue was related to the material right associated with discounted fostamatinib supply which amounted to $ 1.4 million. ​ For the three and six months ended June 30, 2022, we recognized $ 2.5 million and $ 2.6 million, respectively, of revenue related to the delivery of fostamatinib supply to Kissei mainly for commercial use. No such revenue was recognized during the three and six months ended June 30, 2023. ​ In April 2022, Kissei announced that an NDA was submitted to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for fostamatinib in chronic ITP. With this milestone event, we received $ 5.0 million non-refundable and non-creditable payment from Kissei pursuant to the terms of our collaboration agreement, and such amount was recognized as revenue in the three and six months ended June 30, 2022 . In December 2022, Kissei announced that Japan’s PMDA approved the NDA for fostamatinib in chronic ITP. With this milestone event, we were entitled to receive $ 20.0 million non-refundable and non-creditable payment from Kissei pursuant to the terms of our collaboration agreement, which we recognized as revenue in the fourth quarter of 2022 . The amount was subsequently collected in January 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. For sales-based milestones and tiered, escalated net sales-based payments for the supply of fostamatinib, we determined that the license is the predominant item to which the sales-based milestones relate to. 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 allocated costs for the tiered, escalated net sales-based payments 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. 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 this agreement 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 of $ 5.0 million 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 believed that the buyback provision, if exercised, will require us to repurchase the license at an amount equal to or more than the upfront $ 5.0 million. As such, this arrangement was accounted for as a financing arrangement. Interest expense 14 ​ Table of Contents was accreted on such liability over the expected buyback period. We also billed Medison for the delivery of fostamatinib supplies for clinical use which we previously deferred and included within the outstanding financing liability considering the buy-back provision. The decision to exercise the buyback option is dependent of many factors including management’s cost and benefit assessments 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 warm autoimmune hemolytic anemia (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 we will not file a supplemental new drug application (sNDA) for wAIHA indication 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, the likelihood of exercising the buy-back option right was remote. As such, during the fourth quarter of 2022, we relieved the outstanding financing liability to Medison amounting to $ 5.7 million and recognized such amount as collaboration revenue in accordance with ASC 606. There was no outstanding financing liability to Medison as of June 30, 2023 and December 31, 2022. Knight Commercial License and Supply Agreement We have a 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 three and six months ended June 30, 2022. Variable consideration related to future 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. 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 will recognize revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all 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 are also responsible for the exclusive manufacture and supply of fostamatinib for all future development and commercialization activities under agreement. Government Contract - US Department of Defense’s JPEO-CBRND In January 2021, we were awarded up to $ 16.5 million by the US Department of Defense 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. The amount of award we will receive from the US Department of Defense is subject to submission of proper documentation as evidence of completion of certain clinical trial events or milestones as specified in the agreement, and approval by the US Department of Defense that such events or milestones have been met. We record government contract revenue in the statement of operations in the period when it is probable that we will receive the award, which is when we comply with the conditions associated with the award and obtain approval from the US Department of Defense that such conditions have been met. For the three and six months ended June 30, 2023, we recognized $ 1.0 million of revenue related to this grant upon achievement of certain milestones. Through June 30, 2023, we received $ 16.0 million of the awards which we recognized as revenue in the respective periods, with remaining $ 0.5 million available, subject to us meeting certain milestone and approval by the US Department of Defense that such milestone has been met, as specified in the agreement. 15 ​ Table of Contents License and Transition Services Agreement with Forma (now Novo Nordisk) ​ We have a license and transition services agreement with Forma (now Novo Nordisk) 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 (now Novo Nordisk) 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 . In accordance with the guidance, in a transaction accounted for as an asset acquisition, any acquired IPR&D that does not have alternative future use is charged to expense at the acquisition date. At the acquisition date, the acquired license asset was accounted for as IPR&D, and we anticipated no other economic benefit to be derived from such acquired licensed asset other than the primary indications. As such, we accounted for the upfront fee of $ 2.0 million as IPR&D and recorded such cost within research and development expense in the condensed statements of operations in the third quarter of 2022. 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 ilestone payment obligations incurred at development stage and prior to a regulatory approval of an indication associated with the acquired licensed asset as research and development expense when the event requiring payment of the milestone occurs. Milestone payment obligations incurred upon and after a regulatory approval of an indication associated with the acquired licensed asset, and at the commercial stage, are recorded as intangible asset when the event requiring payment of the milestones occurs. The amount recorded as intangible asset is amortized over the estimated useful life of the acquired licensed asset. Royalty payments related to the acquired licensed asset is recorded as cost of sales when incurred. Prior to the FDA approval of REZLIDHIA in December 2022, we achieved certain regulatory milestone which entitled Forma (now Novo Nordisk) 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 in the fourth quarter of 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 (now Novo Nordisk) was entitled to receive a total of $ 15.0 million 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 condensed balance sheet in the fourth quarter of 2022. The $ 15.0 million milestone payment obligation was outstanding as of December 31, 2022 and included within accounts payable in our condensed balance sheet. Such amount was paid in the first quarter of 2023 . During the three and six months ended June 30, 2023, we recognized $ 0.3 million and $ 0.5 million, respectively, of amortization of intangible asset, and $ 0.4 million and $ 0.6 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 condensed statements of operations. No such expenses were recognized during the three and six months ended June 30, 2022. 16 ​ Table of Contents 5. Stock-Based Compensation ​ Stock-based compensation for the periods presented was as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended June 30, ​ Six Months Ended June 30, ​ ​ 2023 2022 ​ 2023 2022 Selling, general and administrative ​ $ 1,796 ​ $ 1,933 ​ $ 3,531 ​ $ 4,672 Research and development ​ ​ 376 ​ ​ 458 ​ ​ 1,399 ​ ​ 926 Total stock-based compensation expense ​ $ 2,172 ​ $ 2,391 ​ $ 4,930 ​ $ 5,598 ​ Stock-based compensation expense within research and development in the six months ended June 30, 2023 include an incremental charge of approximately $ 0.5 million from stock option modifications recorded in the first quarter of 2023 related to the acceleration of vesting and extension of exercise period of vested stock option grants made to a former officer whose employment ended in March 2023. Stock-based compensation expense within selling, general and administrative in the six months ended June 30, 2022 include an incremental charge of approximately $ 0.8 million from stock option modifications recorded in the first quarter of 2022 related to the extension of the exercise period of the stock option grants made to our two former Board of Directors whose terms expired in May 2022. During the six months ended June 30, 2023, we granted stock options to purchase 2,938,600 shares of common stock with weighted-average grant-date fair value of $ 1.37 per share, and 15,557 stock options were exercised. As of June 30, 2023, there were 35,244,121 stock options outstanding, of which, 2,870,000 are outstanding performance-based stock options wherein the achievement of the corresponding corporate-based milestones were not considered probable as of June 30, 2023. Accordingly, none of the $ 5.3 million grant date fair value for these awards has been recognized as stock-based compensation expense as of June 30, 2023. The fair value of each option award is estimated on the date of grant using the Black-Scholes option pricing model. The following table summarizes the weighted-average assumptions relating to options granted pursuant to our Equity Incentive Plans (2018 Equity Incentive Plan and Inducement Plan) for the periods presented: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended June 30, Six Months Ended June 30, ​ 2023 2022 2023 2022 Risk-free interest rate ​ 3.7 % 2.9 % ​ 3.7 % 1.9 % Expected term (in years) ​ 6.7 ​ 6.5 ​ ​ 7.0 ​ 6.5 ​ Dividend yield ​ 0.0 % 0.0 % ​ 0.0 % 0.0 % Expected volatility ​ 86.0 % 70.3 % ​ 83.1 % 70.0 % ​ During the six months ended June 30, 2023, we granted 1,387,600 RSUs with a grant-date weighted-average fair value of $ 1.80 per share, and 435,006 RSUs were released. The RSUs granted generally vest over 4 years . As of June 30, 2023, there were 1,963,487 RSUs outstanding. As of June 30, 2023, there was approximately $ 14.6 million of unrecognized stock-based compensation cost which is expected to be recognized over a remaining weighted-average period of 2.68 years, related to time-based stock options, performance-based stock options wherein achievement of the corresponding corporate-based milestones was considered as probable, and RSUs. In April 2023, our Board of Directors approved additional 108,600 shares of common stock reserved for issuance under our Inducement Plan. In 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. As of June 30, 2023, there were 12,186,098 shares of common stock available for future grant under our Equity Incentive Plans. ​ 17 ​ Table of Contents Employee Stock Purchase Plan ​ Our Purchase Plan provides for a 24 -month offering period comprises 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 twenty-four-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. As of June 30, 2023, unrecognized stock-based compensation cost related to our Purchase Plan amounted to $ 0.4 million, which is expected to be recognized over the remaining weighted average period of 0.74 years. ​ During the six months ended June 30, 2023, there were 509,190 shares purchased under the Purchase Plan. As of June 30, 2023, there were 2,928,443 shares reserved for future issuance under the Purchase Plan. 6. Inventories Inventories for the periods presented consist of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of ​ ​ June 30, 2023 December 31, 2022 Raw materials ​ $ 6,878 ​ $ 4,555 Work in process ​ ​ 2,046 ​ ​ 2,659 Finished goods ​ ​ 1,858 ​ ​ 1,904 Total ​ $ 10,782 ​ $ 9,118 ​ Inventories as of June 30, 2023 and December 31, 2022 include inventories acquired from Forma (now Novo Nordisk) pursuant to the license and transition agreement. As of June 30, 2023 and December 31, 2022, we have $ 0.7 million and $ 0.8 million, respectively, in advance payments to the manufacturer of our raw materials, which were included within prepaid and other current assets in the condensed balance sheet. 7. Cash, Cash Equivalents and Short-Term Investments Cash, cash equivalents and short-term investments for the periods presented consist of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of ​ ​ June 30, 2023 December 31, 2022 Cash ​ $ 8,794 ​ $ 6,264 Money market funds ​ 21,109 ​ 4,155 US treasury bills ​ 8,953 ​ 5,225 Government-sponsored enterprise securities ​ 15,584 ​ 15,796 Corporate bonds and commercial paper ​ 9,917 ​ 26,766 ​ ​ $ 64,357 ​ $ 58,206 Reported as: ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 48,812 ​ $ 24,459 Short-term investments ​ 15,545 ​ 33,747 ​ ​ $ 64,357 ​ $ 58,206 ​ 18 ​ Table of Contents 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 June 30, 2023 ​ Cost ​ Gains ​ Losses ​ Fair Value US treasury bills ​ $ 8,951 ​ $ 2 ​ $ — ​ $ 8,953 ​ Government-sponsored enterprise securities ​ ​ 15,607 ​ ​ 2 ​ ​ ( 25 ) ​ $ 15,584 ​ Corporate bonds and commercial paper ​ 9,921 ​ — ​ ( 4 ) ​ 9,917 ​ Total ​ $ 34,479 ​ $ 4 ​ $ ( 29 ) ​ $ 34,454 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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 June 30, 2023 and December 31, 2022, our cash equivalents and short-term investments had a weighted-average time to maturity of approximately 72 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 condensed balance sheets as they are available for use in the current operations. As of June 30, 2023, we had no investments that had been in a continuous unrealized loss position for more than 12 months. As of June 30, 2023, a total of 19 individual securities had been in an unrealized loss position for 12 months or less, and the losses were determined to be temporary. No significant facts or circumstances have arisen to indicate that there has been any significant deterioration in the creditworthiness of the issuers of the securities held by us. Based on our review of these securities, including the assessment of the duration and severity of the unrealized losses, we have no t recognized any credit losses on these securities as of June 30, 2023 and December 31, 2022. 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 June 30, 2023 Fair Value Unrealized Losses Government-sponsored enterprise securities ​ $ 10,576 ​ $ ( 26 ) ​ Corporate bonds and commercial paper ​ ​ 7,950 ​ ​ ( 3 ) ​ Total ​ $ 18,526 ​ $ ( 29 ) ​ ​ ​ ​ ​ 19 ​ Table of Contents 8. Fair Value ​ The table below summarizes 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 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets at Fair Value as of June 30, 2023 ​ Level 1 Level 2 Level 3 Total Money market funds ​ $ 21,109 ​ $ — ​ $ — ​ $ 21,109 US treasury bills ​ ​ — ​ ​ 8,953 ​ ​ — ​ ​ 8,953 Government-sponsored enterprise securities ​ — ​ 15,584 ​ — ​ 15,584 Corporate bonds and commercial paper ​ — ​ 9,917 ​ — ​ 9,917 Total ​ $ 21,109 ​ $ 34,454 ​ $ — ​ $ 55,563 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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 ​ ​ ​ 9. Debt We have a Credit and Security Agreement (Credit Agreement) with MidCap Financial Trust (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 June 30, 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 Secured Overnight Financing Rate (SOFR) , plus an adjustment of 0.11448% , subject to 1.50 % applicable floor, plus applicable margin of 5.65 % . A final payment fee of 2.5 % of principal is due at maturity date of the term loans. Prior to the Third Amendment, the outstanding principal balance of the loan bore interest at an annual rate of one-month London Interbank Offered Rate (LIBOR), or a comparable applicable index rate, plus applicable margin of 5.65 % , subject to a LIBOR floor of 1.50 % and is payable monthly in arrears. We may make voluntary prepayments, in whole or in part, subject to certain prepayment premiums and additional interest payments. The Credit Agreement also contains certain provisions, such as event of default and change in control provisions, which, if triggered, would require us to make mandatory prepayments on the term loan, which are subject to certain prepayment premiums and additional interest payments. The obligations under the amended Credit Agreement are secured by a perfected security interest in all of our assets including our intellectual property. Debt issuance costs are recorded as a direct deduction from the outstanding principal balance of the term loan. As of June 30, 2023 and December 31, 2022, the unamortized issuance costs and debt discounts amounted to $ 0.4 million and $ 0.6 million, respectively. 20 ​ Table of Contents Interest expense, including amortization of the debt discount and accretion of the final fees related to the Credit Agreement for the three months ended June 30, 2023 and 2022 was $ 1.9 million and $ 0.6 million, respectively, and for the six months ended June 30, 2023 and 2022 was $ 3.1 million and $ 1.1 million, respectively. Accrued interest of $ 1.2 million was included within other accrued liabilities in the condensed balance sheet as of June 30, 2023. The following table presents the future minimum principal payments of the outstanding loan as of June 30, 2023 (in thousands): ​ ​ ​ ​ Remainder of 2023 ​ $ — 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 June 30, 2023, we were not in violation of any covenants. 10. Leases We have a sublease agreement with Atara Biotherapeutics, Inc. (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 previously leased facility in January 2023. At lease measurement date in the fourth quarter of 2022, we recognized the operating lease right-of-use asset and lease liability of approximately $ 1.3 million. As of June 30, 2023, we recorded $ 0.2 million of lease incentives from our sublease with Atara, which we recorded as a reduction to operating lease right-of-use asset and lease liability until the lease ends and the asset is transferred. The weighted average remaining term of our leases as of June 30, 2023 was 1.92 years. We had a lease agreement with Healthpeak Properties, Inc. (formerly known as HCP BTC, LLC), to occupy research and office space located in South San Francisco, California and a sublease agreement with an unrelated third-party to sublet a portion of the leased facility. Both leases expired in January 2023. The components of our operating lease expense were as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended June 30, ​ Six Months Ended June 30, ​ ​ 2023 ​ 2022 ​ 2023 ​ 2022 Fixed operating lease expense $ 166 $ 1,340 ​ $ 778 $ 2,680 Variable operating lease expense (net credit) ​ ​ ( 42 ) ​ ​ 125 ​ ​ 30 ​ ​ 391 Total operating lease expense $ 124 $ 1,465 ​ $ 808 $ 3,071 ​ Supplemental information related to our operating lease were as follow (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended June 30, ​ Six Months Ended June 30, ​ ​ 2023 ​ 2022 ​ 2023 ​ 2022 Cash payments included in the measurement of operating lease liabilities ​ $ 178 ​ $ 2,630 ​ $ 1,174 ​ $ 5,226 ​ 21 ​ Table of Contents Supplemental information related to our operating sublease was as follow (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended June 30, ​ Six Months Ended June 30, ​ ​ 2023 ​ 2022 ​ 2023 ​ 2022 Fixed sublease expense $ — $ 1,095 ​ $ 365 $ 2,190 Variable sublease expense ​ ​ — ​ ​ 206 ​ ​ 77 ​ ​ 450 Sublease income ​ ​ — ​ ​ ( 1,301 ) ​ ​ ( 442 ) ​ ​ ( 2,640 ) Net $ — $ — ​ $ — $ — ​ The following table presents the future lease payments as of June 30, 2023 (in thousands): ​ ​ ​ ​ ​ Remainder of 2023 ​ $ 360 2024 ​ ​ 739 2025 ​ ​ 301 Total minimum payments required ​ $ 1,400 ​ ​ ​ ​ ​ 22 ​ Table of Contents