Tovorafenib

Earned $9 million milestone upon U.S. Food and Drug Administration’s approval of Day One’s OJEMDA™ (tovorafenib); XOMA is entitled to receive a mid-single digit royalty on OJEMDA™ sales Acquired Kinnate Pharmaceuticals, adding at least $9.5 million in non-dilutive capital to XOMA’s balance sheet Expanded the commercial royalty and milestone portfolio with the acquisitions of economic interests in DSUVIA® (sufentanil sublingual tablet) and XACIATO™ (clindamycin phosphate) vaginal gel 2%, as well as two Phase 3 assets Launched XOMA’s first stock repurchase program for up to $50 million EMERYVILLE, Calif., May 09, 2024 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its first quarter 2024 financial results and highlighted recent activities. “We continue to build the foundation for accelerating value creation with a disciplined approach to capital deployment,” stated Owen Hughes, Chief Executive Officer of XOMA. “In recent months, we’ve completed the acquisition of Kinnate, acquired economic interests in two commercial assets, as well as in two first-in-class Phase 3 assets, and initiated our first share buyback program on the heels of securing the VABYSMO® royalty-backed loan with Blue Owl. Lastly, and most importantly, the FDA approved OJEMBA™ (tovorafenib), Day One Biopharmaceuticals’ type II RAF inhibitor for patients with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement or BRAF V600 mutation, ushering in an important new treatment for children living with relapsed or refractory pLGG.” Key First Quarter Events PartnerEventTalpheraXOMA added another commercial asset to its royalty portfolio with an economic interest in DSUVIA®, which is marketed by Alora Pharmaceuticals. XOMA receives 100 percent of the DSUVIA® economics until a threshold is achieved; thereafter, XOMA retains the 15 percent royalty associated with DSUVIA® commercial sales. The 75 percent royalties from Department of Defense purchases and remaining milestone payments will be shared equally between XOMA and Talphera.Kinnate PharmaceuticalsXOMA initiated the acquisition of Kinnate Pharmaceuticals for $2.5879 in cash per share, plus a non-tradeable contingent value right (CVR) representing the right to receive 85 percent of the net proceeds from the out license or sale of Kinnate assets effected on or before April 2, 2025, and 100% of the net proceeds received from Pierre-Fabre.Zevra TherapeuticsU.S. Food and Drug Administration (FDA) accepted the arimoclomol NDA resubmission for review and set a Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024. XOMA paid a $1 million milestone to LadRx based upon the achievement of this milestone.MedexusFDA approved the pediatric label expansion application for IXINITY® [coagulation factor IX (recombinant)].TakedaReported positive topline results from Phase 2 study evaluating mezagitamab (TAK-079), a potential best-in-class anti-CD38 monoclonal antibody for primary immune thrombocytopenia (ITP).iLG Chem (AVEO Oncology)Dosed the first patient in the ficlatuzumab Phase 3 study, resulting in a $1 million milestone payment to XOMA.CompugenReceived a $1 million milestone payment from Compugen. Subsequent Events PartnerEventDay One BiopharmaceuticalsFDA approved Day One’s OJEMDA™ (tovorafenib) for use in pediatric patients with pediatric low-grade glioma (pLGG). XOMA earned a $9 million milestone upon the approval and is entitled to receive mid-single digit royalties from OJEMDA sales.Daré BioscienceXOMA added economic interests to three best- or first-in-category assets to its portfolio. XACIATO™ vaginal gel 2% is commercially available and marketed by Organon. Bayer holds the U.S. rights to commercialize Ovaprene®, a hormone-free monthly intravaginal contraceptive, currently in Phase 3 clinical trials. XOMA also acquired a synthetic royalty in Sildenafil Cream, 3.6%, a Phase 3-ready asset for female sexual arousal disorder.RezoluteDosed first patient in its Phase 3 trial of RZ358; XOMA earned a $5.0 million milestone associated with the event. Anticipated 2024 Events of Note PartnerEventZevra TherapeuticsSeptember 21, 2024 – FDA PDUFA action date for arimoclomol NDATakedaIn its press release dated March 13, 2024, Takeda announced plans to initiate a global Phase 3 trial of mezagitamab in ITP in fiscal year 2024.ii First Quarter 2024 Financial Results XOMA recorded total revenues of $1.5 million for the first quarter of 2024, which included a $1.0 million milestone payment received from AVEO Oncology, as compared with $0.4 million in the first quarter of 2023. Research and development (R&D) expenses were $33,000 and $54,000, respectively, for the first quarters of 2024 and 2023. Upon closing the Kinnate merger in the second quarter of 2024, XOMA assumed operations of Kinnate’s pipeline, as well as ongoing Phase 1 study, and will incur increased R&D costs until winddown activities are complete. General and administrative (“G&A”) expenses were $8.5 million for the first quarter of 2024, compared to $6.2 million for the first quarter of 2023. The increase of $2.3 million was primarily due to a $1.3 million increase in stock-based compensation and a $0.7 million increase in consulting and legal expenses. The increase in stock-based compensation expenses was largely due to the appointment of Mr. Hughes as our full-time Chief Executive Officer in January 2024. In the first quarter of 2024, G&A expenses included $2.9 million in non-cash stock-based compensation expense, compared with $1.6 million in the first quarter of 2023. The increase in stock-based compensation expenses was largely due to the PSU grant associated with the appointment of Mr. Hughes as our full-time Chief Executive Officer in January 2024 combined with PSUs granted in May 2023. Interest expense in the first quarter of 2024 was $3.6 million, representing interest related to the Blue Owl Loan established in December 2023. The Company reported total other income, net, of $2.0 million in the first quarter of 2024, as compared to total other income, net, of $0.4 million in the corresponding period of 2023. The $1.6 million increase reflects a $1.3 million increase in investment income due to higher cash balances and higher market interest rates on our investments, as well as the change in the market price of Rezolute’s common stock. Net loss for the first quarter of 2024 was $8.6 million, compared to a net loss of $9.8 million for the first quarter of 2023. On March 31, 2024, XOMA had cash and cash equivalents of $142.4 million (including $6.2 million in restricted cash). On December 31, 2023, XOMA had cash and cash equivalents of $159.6 million (including $6.3 million in restricted cash). During the first quarter of 2024, XOMA received $9.8 million in cash from royalty and milestone payments and deployed $8 million to acquire new royalty and milestone economic interests. Net cash used in operating activities during the quarter was $4.9 million. On April 15, 2024, the Company paid a total of $1.4 million in cash dividends on the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) and the 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO). About XOMA CorporationXOMA is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA acquires the potential future economics associated with pre-commercial and commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit www.xoma.com or follow the Company on LinkedIn. Forward-Looking Statements/Explanatory NotesCertain statements contained in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, including statements regarding the timing and amount of potential commercial payments to XOMA and other developments related to VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], DSUVIA® (sufentanil sublingual tablet), and arimoclomol; the potential occurrences of the events listed under “Anticipated 2024 Events of Note”; the anticipated timings of regulatory filings and approvals related to assets in XOMA’s portfolio; and the potential of XOMA’s portfolio of partnered programs and licensed technologies generating substantial milestone and royalty proceeds over time. In some cases, you can identify such forward-looking statements by terminology such as “anticipate,” “intend,” “believe,” “estimate,” “plan,” “seek,” “project,” “expect,” “may,” “will”, “would,” “could” or “should,” the negative of these terms or similar expressions. These forward-looking statements are not a guarantee of XOMA’s performance, and you should not place undue reliance on such statements. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks inherent in the biotechnology industry, including those related to the fact that our product candidates subject to out-license agreements are still being developed, and our licensees may require substantial funds to continue development which may not be available; we do not know whether there will be, or will continue to be, a viable market for the products in which we have an ownership or royalty interest; if the therapeutic product candidates to which we have a royalty interest do not receive regulatory approval, our third-party licensees will not be able to market them; and the impact to the global economy as a result of the COVID-19 pandemic. Other potential risks to XOMA meeting these expectations are described in more detail in XOMA's most recent filing on Form 10-Q and in other filings with the Securities and Exchange Commission. Consider such risks carefully when considering XOMA's prospects. Any forward-looking statement in this press release represents XOMA's beliefs and assumptions only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA disclaims any obligation to update any forward-looking statement, except as required by applicable law. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. XOMA CORPORATION CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands, except share and per share amounts) March 31, December 31, 2024 2023 ASSETS(unaudited) Current assets: Cash and cash equivalents$136,225 $153,290 Short-term restricted cash 160 160 Short-term equity securities 413 161 Trade and other receivables, net 3 1,004 Short-term royalty and commercial payment receivables 9,819 14,215 Prepaid expenses and other current assets 270 483 Total current assets 146,890 169,313 Long-term restricted cash 6,016 6,100 Property and equipment, net 40 25 Operating lease right-of-use assets 364 378 Long-term royalty and commercial payment receivables 65,577 57,952 Other assets - long term 533 533 Total assets$219,420 $234,301 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable$1,515 $653 Accrued and other liabilities 1,299 2,768 Contingent consideration under RPAs, AAAs and CPPAs 3,000 7,000 Operating lease liabilities 55 54 Unearned revenue recognized under units-of-revenue method 2,159 2,113 Preferred stock dividend accrual 1,368 1,368 Current portion of long-term debt 6,144 5,543 Total current liabilities 15,540 19,499 Unearned revenue recognized under units-of-revenue method – long-term 6,692 7,228 Long-term operating lease liabilities 319 335 Long-term debt 114,528 118,518 Total liabilities 137,079 145,580 Stockholders’ equity: Preferred Stock, $0.05 par value, 1,000,000 shares authorized: 8.625% Series A cumulative, perpetual preferred stock, 984,000 shares issued and outstanding at March 31, 2024 and December 31, 2023 49 49 8.375% Series B cumulative, perpetual preferred stock, 1,600 shares issued and outstanding at March 31, 2024 and December 31, 2023 — — Convertible preferred stock, 5,003 issued and outstanding at March 31, 2024 and December 31, 2023 — — Common stock, $0.0075 par value, 277,333,332 shares authorized, 11,636,355 and 11,495,492 shares issued and outstanding at March 31, 2024 and December 31, 2023, respectively 87 86 Additional paid-in capital 1,314,036 1,311,809 Accumulated deficit (1,231,831) (1,223,223) Total stockholders’ equity 82,341 88,721 Total liabilities and stockholders’ equity$219,420 $234,301 XOMA CORPORATION CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (in thousands, except share and per share amounts) Three Months Ended March 31, 2024 2023 Revenues: Revenue from contracts with customers$1,000 $- Revenue recognized under units-of-revenue method 490 437 Total revenues 1,490 437 Operating expenses: Research and development 33 54 General and administrative 8,461 6,196 Arbitration settlement costs - 4,132 Amortization of intangible assets - 225 Total operating expenses 8,494 10,607 Loss from operations (7,004) (10,170) Other income (expense) Interest expense (3,551) - Other income (expense), net 1,960 357 Net loss and comprehensive loss$(8,595) $(9,813) Less: accumulated dividends on Series A and Series B preferred stock (1,368) (1,368) Net loss and comprehensive loss attributable to common stockholders, basic and diluted$(9,963) $(11,181) Basic and diluted net loss per share attributable to common stockholders$(0.86) $(0.98) Weighted average shares used in computing basic and diluted net loss per share attributable to common stockholders 11,580 11,460 XOMA CORPORATIONCONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS(in thousands) Three Months Ended March 31, 2024 2023 Cash flows from operating activities: Net loss$(8,595) $(9,813)Adjustments to reconcile net loss to net cash used in operating activities: Stock-based compensation expense 2,856 1,570 Common stock contribution to 401(k) 118 123 Amortization of intangible assets — 225 Depreciation 2 1 Accretion of long-term debt discount and debt issuance costs 306 — Non-cash lease expense 14 47 Change in fair value of equity securities (252) 24 Changes in assets and liabilities: Trade and other receivables, net 1,001 (5)Prepaid expenses and other assets 213 269 Accounts payable and accrued liabilities (105) 3,122 Operating lease liabilities (15) (50)Unearned revenue recognized under units-of-revenue method (490) (437) Net cash used in operating activities (4,947) (4,924) Cash flows from investing activities: Payments of consideration under RPAs, AAAs and CPPAs (15,000) (9,600)Receipts under RPAs, AAAs and CPPAs 7,771 2,366 Purchase of property and equipment (17) — Net cash used in investing activities (7,246) (7,234) Cash flows from financing activities: Principal payments – debt (3,616) — Debt issuance costs and loan fees paid in connection with long-term debt (581) — Payment of preferred stock dividends (1,368) (1,368)Repurchases of common stock (13) — Proceeds from exercise of options and other share-based compensation 1,956 — Taxes paid related to net share settlement of equity awards (1,334) — Net cash used in financing activities (4,956) (1,368) Net decrease in cash, cash equivalents and restricted cash (17,149) (13,526)Cash, cash equivalents and restricted cash at the beginning of the period 159,550 57,826 Cash, cash equivalents and restricted cash at the end of the period$142,401 $44,300 Supplemental Cash Flow Information: Cash paid for interest$3,780 $— Non-cash investing and financing activities: Accrual of contingent consideration under the Affitech CPPA$3,000 $— Preferred stock dividend accrual$1,368 $1,368 Investor contact: Media contact:Juliane SnowdenKathy VincentXOMAKV Consulting & Management+1-646-438-9754+1-310-403-8951juliane.snowden@xoma.comkathy@kathyvincent.com i https://www.takeda.com/newsroom/newsreleases/2024/takeda-announces-positive-topline-results-from-phase-2-study-evaluating-mezagitamab-TAK-079-a-potential-best-in-class-anti-CD38-monoclonal-antibody-for-primary-immune-thrombocytopenia/

OJEMDA™ (tovorafenib) launch underway following U.S. FDA accelerated approval for relapsed or refractory BRAF-altered Pediatric Low-Grade Glioma (pLGG) First prescriptions received in the U.S. BRISBANE, Calif., May 06, 2024 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals (Nasdaq: DAWN) (“Day One” or the “Company”), a commercial-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced its first quarter 2024 financial results and highlighted recent corporate achievements. “We are excited that OJEMDA is now approved and available here in the U.S., and we are grateful to the members of the pediatric brain tumor community whose support of the program has been invaluable,” said Jeremy Bender, Ph.D., chief executive officer of Day One. “Our team is focused on executing our U.S. launch, on advancing our other programs, and on exploring opportunities to expand our pipeline.” Program Highlights In April 2024, the Company received U.S. Food and Drug Administration (FDA) accelerated approval of OJEMDA (tovorafenib), the first and only FDA approved therapy for the treatment of pediatric patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. With OJEMDA now available and the first prescriptions written, patients have begun enrolling in EveryDay Support From Day One™, a comprehensive program that offers personalized services for OJEMDA patients and their care teams, including insurance coverage support, financial assistance options and educational resources throughout the treatment journey. The pivotal Phase 3 FIREFLY-2/LOGGIC clinical trial evaluating tovorafenib as a front-line therapy in patients aged 6 months to 25 years with pLGG continues to enroll in the United States, Canada, Europe, Australia and Asia, with more than 90 sites activated. Patient enrollment continues in the Phase 1b/2 substudy (102b) of the FIRELIGHT-1 trial evaluating the combination of tovorafenib with the Company’s investigational MEK inhibitor, pimasertib. Corporate Highlights and Upcoming Milestones The Company received a rare pediatric disease priority review voucher from the FDA upon OJEMDA’s approval. Results from the FIRELIGHT-1 Phase 1b and next steps are expected in the second half of 2024. First Quarter 2024 Financial Highlights Cash Position: Cash, cash equivalents and short-term investments totaled $317.9 million on March 31, 2024. Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into 2026. R&D Expenses: Research and development expenses were $40.2 million for the first quarter of 2024 compared to $27.8 million for the first quarter of 2023. The increase was primarily due to additional employee compensation costs, a payment for the buyback of priority review voucher obligation, and increased clinical trial and manufacturing activities related to Day One’s lead product, OJEMDA. G&A Expenses: General and administrative expenses were $26.6 million for the first quarter of 2024 compared to $18.0 million for the first quarter of 2023. The increase was primarily due to additional employee compensation costs, ongoing commercial buildout, and increased professional service expenses to support company growth. Net Loss: Net loss totaled $62.4 million for the first quarter of 2024 with non-cash stock compensation expense of $12.6 million, compared to $42.4 million for the first quarter of 2023 with non-cash stock compensation expense of $9.4 million. Upcoming Events 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, May 31-June 4, 2024 Abstract #10036 titled “Type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma (pLGG): Reversible decreases in growth velocity in the phase 2 FIREFLY-1 trial” will be presented in a poster session on Saturday, June 1 from 1:30-4:30 pm CDT in Hall A Goldman Sachs 45th Annual Global Healthcare Conference, June 10-13, 2024 21st International Symposium on Pediatric Neuro-Oncology (ISPNO), June 28-29, 2024 About OJEMDA™ OJEMDA (tovorafenib) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. OJEMDA is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Tovorafenib is under evaluation as a therapy for patients with pLGG requiring front-line treatment (Phase 3 FIREFLY-2/LOGGIC). It is also being studied in combination with the MEK inhibitor pimasertib for adolescent and adult patient populations with recurrent or progressive solid tumors with MAPK pathway alterations (FIRELIGHT-1). Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma. For more information, please visit About Day One Biopharmaceuticals Day One Biopharmaceuticals is a commercial-stage biopharmaceutical company that believes when it comes to pediatric cancer, we can do better. The Company was founded to address a critical unmet need: the dire lack of therapeutic development in pediatric cancer. Inspired by “The Day One Talk” that physicians have with patients and their families about an initial cancer diagnosis and treatment plan, Day One aims to re-envision cancer drug development and redefine what’s possible for all people living with cancer—regardless of age—starting from Day One. Day One partners with leading clinical oncologists, families, and scientists to identify, acquire, and develop important targeted cancer treatments. The Company’s pipeline includes tovorafenib (OJEMDA™) and pimasertib. Day One is based in Brisbane, California. For more information, please visit or find the Company on LinkedIn or X. Cautionary Note Regarding Forward-Looking Statements This press release contains “forward-looking” statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: Day One’s plans to develop and commercialize cancer therapies, expectations from current clinical trials, the execution of the Phase 2 and Phase 3 clinical trial for tovorafenib as designed, any expectations about safety, efficacy, timing and ability to complete clinical trials, release data results and to obtain regulatory approvals for tovorafenib and other candidates in development, and the ability of tovorafenib to treat pLGG or related indications. Statements including words such as “believe,” “plan,” “continue,” “expect,” “will,” “develop,” “signal,” “potential,” or “ongoing” and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. Forward-looking statements are subject to risks and uncertainties that may cause Day One’s actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties in this press release and other risks set forth in our filings with the Securities and Exchange Commission, including Day One’s ability to develop, obtain and retain regulatory approval for or commercialize any product candidate, Day One’s ability to protect intellectual property, the potential impact of global business or macroeconomic conditions, including as a result of inflation, rising interest rates, instability in the global banking system, geopolitical conflicts and the sufficiency of Day One’s cash, cash equivalents and investments to fund its operations. These forward-looking statements speak only as of the date hereof and Day One specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law. Day One Biopharmaceuticals, Inc. Condensed Statements of Operations (unaudited) (in thousands, except shares) Three Months Ended March 31, 2024 2023 Operating expenses: Research and development $ 40,210 $ 27,828 General and administrative 26,557 18,027 Total operating expenses 66,767 45,855 Loss from operations (66,767 ) (45,855 ) Investment income, net 4,365 3,466 Other expense, net (10 ) (4 ) Net loss attributable to common stockholders (62,412 ) (42,393 ) Net loss per share, basic and diluted $ (0.72 ) $ (0.59 ) Weighted-average number of common shares used in computing net loss per share, basic and diluted 86,679,282 71,972,888 Day One Biopharmaceuticals, Inc. Selected Consolidated Balance Sheet Data (unaudited) (in thousands) March 31, 2024 December 31, 2023 Cash, cash equivalents and short-term investments $ 317,944 $ 366,347 Total assets 326,645 376,048 Total liabilities 29,839 29,508 Accumulated deficit (520,997 ) (458,585 ) Total stockholders’ equity 296,806 346,540 DAY ONE MEDIA Laura Cooper, Head of Communications media@dayonebio.com DAY ONE INVESTORS LifeSci Advisors, PJ Kelleher pkelleher@lifesciadvisors.com