PART I—FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=5&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents REGENXBIO Inc.'s unaudited consolidated financial statements, including balance sheets, statements of operations and comprehensive loss, statements of stockholders' equity, and statements of cash flows, along with detailed notes explaining the company's accounting policies, financial instruments, and significant agreements for the periods ended June 30, 2025, and December 31, 2024 [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) Consolidated Balance Sheet Highlights (in thousands) | Metric | June 30, 2025 | December 31, 2024 | Change (vs. Dec 31, 2024) | | :-------------------------------- | :------------ | :---------------- | :------------------------ | | **Assets** | | | | | Cash and cash equivalents | $79,558 | $57,526 | +$22,032 | | Marketable securities | $243,740 | $177,161 | +$66,579 | | Total current assets | $373,752 | $278,001 | +$95,751 | | Total assets | $581,027 | $465,989 | +$115,038 | | **Liabilities & Equity** | | | | | Deferred revenue (current) | $13,977 | $115 | +$13,862 | | Royalty monetization liabilities (current) | $40,302 | $34,309 | +$5,993 | | Total current liabilities | $119,540 | $103,194 | +$16,346 | | Deferred revenue (non-current) | $23,804 | $— | +$23,804 | | Royalty monetization liabilities (non-current) | $153,693 | $25,378 | +$128,315 | | Total liabilities | $367,348 | $206,338 | +$161,010 | | Total stockholders' equity | $213,679 | $259,651 | -$45,972 | - Total assets increased by **$115.0 million**, primarily driven by increases in cash, cash equivalents, and marketable securities[17](index=17&type=chunk) - Total liabilities significantly increased by **$161.0 million**, largely due to a substantial rise in royalty monetization liabilities and deferred revenue[17](index=17&type=chunk) [Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Consolidated Statements of Operations and Comprehensive Loss Highlights (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change (YoY) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change (YoY) | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------- | :------------------------------- | :------------------------------- | :----------- | | License and royalty revenue | $18,465 | $21,846 | -$3,381 | $105,514 | $37,190 | +$68,324 | | Service revenue | $2,894 | $449 | +$2,445 | $4,857 | $727 | +$4,130 | | Total revenues | $21,359 | $22,295 | -$936 | $110,371 | $37,917 | +$72,454 | | Research and development expense | $59,500 | $48,869 | +$10,631 | $112,587 | $103,713 | +$8,874 | | General and administrative expense | $19,883 | $18,855 | +$1,028 | $40,230 | $37,146 | +$3,084 | | Loss from operations | $(63,278) | $(56,037) | -$7,241 | $(51,151) | $(119,900) | +$68,749 | | Interest expense | $(10,993) | $(449) | -$10,544 | $(19,563) | $(2,422) | -$17,141 | | Net loss | $(70,871) | $(52,989) | -$17,882 | $(64,788) | $(116,319) | +$51,531 | | Net loss per share, basic and diluted | $(1.38) | $(1.05) | -$0.33 | $(1.26) | $(2.41) | +$1.15 | - Total revenues for the three months ended June 30, 2025, decreased slightly by **$0.9 million** year-over-year, primarily due to a **$3.4 million** decrease in Zolgensma royalty revenue, partially offset by a **$2.4 million** increase in service revenue from the Nippon Shinyaku collaboration[18](index=18&type=chunk)[203](index=203&type=chunk)[204](index=204&type=chunk) - For the six months ended June 30, 2025, total revenues significantly increased by **$72.5 million**, driven by a **$70.0 million** upfront license revenue from the Nippon Shinyaku collaboration[18](index=18&type=chunk)[208](index=208&type=chunk)[209](index=209&type=chunk) - Net loss for the three months ended June 30, 2025, widened to **$(70.9) million** from **$(53.0) million** in the prior year, largely due to increased research and development expenses and a substantial increase in interest expense from royalty monetization liabilities[18](index=18&type=chunk)[205](index=205&type=chunk)[207](index=207&type=chunk) - Net loss for the six months ended June 30, 2025, improved to **$(64.8) million** from **$(116.3) million** in the prior year, primarily due to the significant increase in license and service revenues, despite higher operating and interest expenses[18](index=18&type=chunk)[208](index=208&type=chunk)[210](index=210&type=chunk)[212](index=212&type=chunk) [Consolidated Statements of Stockholders' Equity](index=7&type=section&id=Consolidated%20Statements%20of%20Stockholders'%20Equity) Changes in Stockholders' Equity (in thousands) | Metric | Three Months Ended June 30, 2025 | Six Months Ended June 30, 2025 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Balances at March 31, 2025 / Dec 31, 2024 | $274,197 / $259,651 | $259,651 | | Issuance of warrants, net of transaction costs | $1,610 | $1,610 | | Stock-based compensation expense | $8,653 | $17,190 | | Net loss | $(70,871) | $(64,788) | | Balances at June 30, 2025 | $213,679 | $213,679 | - Total stockholders' equity decreased from **$259.7 million** at December 31, 2024, to **$213.7 million** at June 30, 2025, primarily due to the net loss incurred during the period, partially offset by stock-based compensation and warrant issuance[17](index=17&type=chunk)[24](index=24&type=chunk) [Consolidated Statements of Cash Flows](index=9&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Consolidated Statements of Cash Flows Highlights (in thousands) | Metric | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change (YoY) | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------- | | Net cash used in operating activities | $(15,713) | $(100,952) | +$85,239 | | Net cash provided by (used in) investing activities | $(95,693) | $12,915 | -$108,608 | | Net cash provided by financing activities | $133,438 | $111,280 | +$22,158 | | Net increase in cash and cash equivalents and restricted cash | $22,032 | $23,243 | -$1,211 | - Net cash used in operating activities significantly decreased by **$85.2 million**, primarily due to the **$110.0 million** upfront fee from the Nippon Shinyaku collaboration[26](index=26&type=chunk)[220](index=220&type=chunk)[221](index=221&type=chunk) - Net cash used in investing activities increased by **$108.6 million**, mainly due to higher purchases of marketable debt securities in 2025 compared to 2024[26](index=26&type=chunk)[223](index=223&type=chunk)[224](index=224&type=chunk) - Net cash provided by financing activities increased by **$22.2 million**, driven by proceeds from the 2025 Royalty Bond and warrants, partially offset by royalty monetization repayments[26](index=26&type=chunk)[225](index=225&type=chunk)[226](index=226&type=chunk) [Notes to Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) [1. Nature of Business](index=10&type=section&id=1.%20Nature%20of%20Business) - REGENXBIO Inc. is a clinical-stage biotechnology company focused on gene therapy using its proprietary NAV Technology Platform, which includes exclusive rights to a large portfolio of AAV vectors[29](index=29&type=chunk) - The company has incurred cumulative losses of **$996.9 million** as of June 30, 2025, and relies on additional capital to fund operations, with current cash, cash equivalents, and marketable securities of **$363.6 million** expected to fund operations for at least the next **12 months**[30](index=30&type=chunk) [2. Summary of Significant Accounting Policies](index=10&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) - The company reclassified service revenue separately from license and royalty revenues retrospectively due to a collaboration and license agreement with Nippon Shinyaku Co., Ltd. in March 2025[36](index=36&type=chunk) - Restricted cash of **$2.03 million** as of June 30, 2025, collateralizes letters of credit for lease agreements[37](index=37&type=chunk) - Revenue recognition follows ASC 606, involving a five-step model to identify contracts, performance obligations, transaction price, allocation, and recognition, with specific considerations for variable consideration, milestones, and royalties[43](index=43&type=chunk)[44](index=44&type=chunk)[45](index=45&type=chunk)[52](index=52&type=chunk)[53](index=53&type=chunk)[54](index=54&type=chunk)[55](index=55&type=chunk) - New accounting pronouncements include ASU 2023-09 (Income Tax Disclosures, effective Jan 1, 2025) and ASU 2024-03 (Expense Disaggregation Disclosures, effective Jan 1, 2027), with the company currently evaluating their impact[60](index=60&type=chunk)[61](index=61&type=chunk) [3. Marketable Securities](index=19&type=section&id=3.%20Marketable%20Securities) Marketable Securities (Available-for-Sale Debt Securities) (in thousands) | Category | Amortized Cost (June 30, 2025) | Fair Value (June 30, 2025) | Amortized Cost (Dec 31, 2024) | Fair Value (Dec 31, 2024) | | :-------------------------------- | :----------------------------- | :------------------------- | :----------------------------- | :------------------------- | | U.S. government and agency securities | $132,032 | $132,019 | $44,281 | $44,215 | | Certificates of deposit | $245 | $245 | $1,466 | $1,462 | | Corporate bonds | $151,649 | $151,772 | $141,474 | $141,663 | | Total | $283,926 | $284,036 | $187,221 | $187,340 | - The company's marketable securities, consisting solely of available-for-sale debt securities, increased significantly from **$187.3 million** at December 31, 2024, to **$284.0 million** at June 30, 2025[62](index=62&type=chunk) - As of June 30, 2025, the company held **41** investment grade securities in an unrealized loss position, but did not identify any credit losses due to intent and ability to hold until recovery and low severity of losses[64](index=64&type=chunk) [4. Fair Value Measurements](index=21&type=section&id=4.%20Fair%20Value%20Measurements) Fair Value of Cash Equivalents and Marketable Securities (in thousands) | Category | Level 2 (June 30, 2025) | Level 2 (Dec 31, 2024) | | :-------------------------------- | :---------------------- | :--------------------- | | Money market mutual funds | $44,975 | $43,895 | | U.S. government and agency securities | $141,988 | $46,713 | | Certificates of deposit | $245 | $1,462 | | Corporate bonds | $151,772 | $141,663 | | Total cash equivalents and marketable securities | $338,980 | $233,733 | - The fair values of cash equivalents and marketable securities are primarily categorized as Level 2, based on quoted market prices or broker/dealer quotations for similar assets[65](index=65&type=chunk)[67](index=67&type=chunk) - Royalty monetization liabilities are estimated at fair value using Level 3 inputs, based on the company's estimate of future royalties, milestones, and repayment obligations[67](index=67&type=chunk) [5. Property and Equipment, Net](index=22&type=section&id=5.%20Property%20and%20Equipment,%20Net) Property and Equipment, Net (in thousands) | Category | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Laboratory and manufacturing equipment | $77,765 | $77,141 | | Leasehold improvements | $101,533 | $101,465 | | Total property and equipment, net | $111,017 | $117,589 | - Property and equipment, net, decreased from **$117.6 million** at December 31, 2024, to **$111.0 million** at June 30, 2025[69](index=69&type=chunk) - An impairment of **$0.7 million** on furniture, fixtures, and leasehold improvements was recorded in Q1 2024 due to a sublease agreement for New York office facilities[69](index=69&type=chunk) [6. Leases](index=22&type=section&id=6.%20Leases) - The company subleased its New York office space in March 2024, with the sublease term commencing in April 2024 and expiring concurrently with the main lease in April 2027[70](index=70&type=chunk)[71](index=71&type=chunk) - Sublease income of **$0.1 million** and **$0.2 million** was recognized for the three and six months ended June 30, 2025, respectively[71](index=71&type=chunk) - An impairment loss of **$2.1 million** was recognized in Q1 2024 on the long-lived asset group associated with the New York Sublease, allocated to right-of-use assets (**$1.4 million**) and property and equipment (**$0.7 million**)[73](index=73&type=chunk) [7. Royalty Monetization Liabilities](index=23&type=section&id=7.%20Royalty%20Monetization%20Liabilities) Royalty Monetization Liabilities (in thousands) | Agreement | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | 2020 Royalty Purchase Agreement | $48,231 | $59,687 | | 2025 Royalty Bond | $145,764 | $— | | Total | $193,995 | $59,687 | | Current portion | $40,302 | $34,309 | | Non-current portion | $153,693 | $25,378 | - Total royalty monetization liabilities increased significantly from **$59.7 million** at December 31, 2024, to **$194.0 million** at June 30, 2025, primarily due to the new 2025 Royalty Bond[74](index=74&type=chunk) - The 2020 Royalty Purchase Agreement's effective interest rate was **71.4%** as of June 30, 2025, up from **65.5%** at December 31, 2024, based on estimated future Zolgensma royalty payments[80](index=80&type=chunk) - In May 2025, the company entered into a **$250.0 million** 2025 Royalty Bond with HCR, with an initial funding of **$144.5 million** (net of discounts/costs). This bond bears interest at **9.75%** plus 3-month SOFR (minimum **14.0%**) and is repaid from specified royalties and milestones[82](index=82&type=chunk)[83](index=83&type=chunk)[84](index=84&type=chunk) - In connection with the 2025 Royalty Bond, warrants to purchase **268,096** shares of common stock were issued to HCR, valued at **$1.7 million** and recorded as additional paid-in capital[86](index=86&type=chunk) [8. Commitments and Contingencies](index=27&type=section&id=8.%20Commitments%20and%20Contingencies) Expenses Related to GSK License (in thousands) | Category | Three Months Ended June 30, 2025 | Six Months Ended June 30, 2025 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Royalties on net sales of Zolgensma | $5,204 | $7,786 | | Other cost of license and royalty revenues | $5 | $243 | | General and administrative | $53 | $107 | | Total | $5,262 | $8,136 | - The company has a potential dispute with GlaxoSmithKline (GSK) regarding sublicense fees, with GSK claiming underpayment based on a broader interpretation of sublicense revenue. The company disagrees and does not believe a loss is probable[92](index=92&type=chunk) [9. Capitalization](index=29&type=section&id=9.%20Capitalization) - In March 2024, the company completed a public offering, raising **$131.1 million** net, through the sale of common stock and pre-funded warrants[93](index=93&type=chunk) - As of June 30, 2025, **1,125,440** March 2024 Pre-funded Warrants remained outstanding, with **199,300** shares exercised during the six months ended June 30, 2025[95](index=95&type=chunk) - The May 2025 Warrants, issued in connection with the 2025 Royalty Bond, allow purchase of **268,096** common shares at **$14.92/share**, classified as equity, with none exercised as of June 30, 2025[96](index=96&type=chunk) - The company has an At-the-Market (ATM) Offering Program established in December 2024 to sell up to **$150.0 million** of common stock, with no shares sold as of June 30, 2025[97](index=97&type=chunk) [10. License and Collaboration Agreements](index=30&type=section&id=10.%20License%20and%20Collaboration%20Agreements) Revenues from License and Collaboration Agreements (in thousands) | Revenue Type | Three Months Ended June 30, 2025 | Six Months Ended June 30, 2025 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Zolgensma royalties | $18,423 | $35,416 | | Nippon Shinyaku licenses | $— | $69,979 | | Other license and royalty revenue | $42 | $119 | | Total license and royalty revenue | $18,465 | $105,514 | | Nippon Shinyaku services | $2,720 | $4,494 | | Other service revenue | $174 | $363 | | Total service revenue | $2,894 | $4,857 | | Total revenues | $21,359 | $110,371 | - Unachieved milestones from license and collaboration agreements could result in aggregate payments of up to **$2.18 billion**, including **$548.2 million** for clinical trials, **$106.3 million** for regulatory approvals, and **$1.53 billion** for sales targets[99](index=99&type=chunk) - Deferred revenue as of June 30, 2025, was **$37.8 million**, primarily from the Nippon Shinyaku collaboration, expected to be satisfied over approximately **five years**[101](index=101&type=chunk) - The AbbVie Collaboration Agreement was amended in August 2025, modifying the development plan and milestone payment structure for the ABBV-RGX-314 DR program, splitting a **$200.0 million** milestone into two **$100.0 million** payments[112](index=112&type=chunk)[163](index=163&type=chunk) - The Nippon Shinyaku Collaboration Agreement, effective March 2025, granted licenses for RGX-121 and RGX-111, with an upfront fee of **$110.0 million** and potential milestones up to **$700.0 million**. The company recognized **$74.5 million** in revenue from this agreement in H1 2025[113](index=113&type=chunk)[115](index=115&type=chunk)[164](index=164&type=chunk)[165](index=165&type=chunk) [11. Stock-based Compensation](index=40&type=section&id=11.%20Stock-based%20Compensation) Stock-based Compensation Expense (in thousands) | Award Type | Three Months Ended June 30, 2025 | Six Months Ended June 30, 2025 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Stock options | $4,778 | $9,485 | | Restricted stock units | $3,713 | $7,381 | | Employee stock purchase plan | $162 | $324 | | Total | $8,653 | $17,190 | - The 2015 Equity Incentive Plan expired in June 2025, replaced by the 2025 Equity Incentive Plan, which authorized **5,500,000** shares for issuance[123](index=123&type=chunk)[124](index=124&type=chunk) - As of June 30, 2025, **$65.6 million** of unrecognized stock-based compensation expense remains, to be recognized over a weighted-average period of **2.5 years**[127](index=127&type=chunk) [12. Income Taxes](index=42&type=section&id=12.%20Income%20Taxes) - The company maintains a full valuation allowance for its net deferred tax assets due to a history of operating losses, indicating that the benefit of these assets is not likely to be realized[133](index=133&type=chunk) - The company is evaluating the impact of the recently enacted One Big Beautiful Bill Act (OBBBA) on its consolidated financial statements, which amends U.S. tax laws related to bonus depreciation and R&D expense deductions[134](index=134&type=chunk) [13. Net Loss Per Share](index=43&type=section&id=13.%20Net%20Loss%20Per%20Share) - Due to net losses, common stock equivalents (stock options, restricted stock units, employee stock purchase plan, warrants) were excluded from diluted net loss per share calculations as their effect would be anti-dilutive[135](index=135&type=chunk) Potentially Dilutive Common Stock Equivalents Excluded (in thousands) | Category | June 30, 2025 | June 30, 2024 | | :-------------------------------- | :------------ | :------------ | | Stock options issued and outstanding | 11,810 | 9,638 | | Unvested restricted stock units outstanding | 2,982 | 1,668 | | Employee stock purchase plan | 111 | 78 | | Warrants outstanding | 268 | — | | Total | 15,171 | 11,384 | [14. Segment Information](index=43&type=section&id=14.%20Segment%20Information) - The company operates as a single operating segment, focusing on the development and commercialization of gene therapies, with the CEO reviewing consolidated net income (loss) and cash, cash equivalents, and marketable securities for performance assessment and resource allocation[136](index=136&type=chunk) - As of June 30, 2025, cash, cash equivalents, and marketable securities were **$363.6 million**, up from **$244.9 million** at December 31, 2024[136](index=136&type=chunk) [15. Supplemental Disclosures](index=45&type=section&id=15.%20Supplemental%20Disclosures) Other Current Assets (in thousands) | Category | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Net cost reimbursement due from AbbVie | $16,989 | $11,304 | | Accrued interest on investments | $1,287 | $1,094 | | Other | $1,203 | $1,376 | | Total | $19,479 | $13,774 | Accrued Expenses and Other Current Liabilities (in thousands) | Category | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Accrued personnel costs | $13,801 | $17,607 | | Accrued external research and development expenses | $11,418 | $8,998 | | Accrued sublicense fees and royalties | $7,548 | $8,658 | | Accrued external general and administrative expenses | $2,015 | $2,002 | | Accrued purchases of property and equipment | $39 | $156 | | Other | $736 | $649 | | Total | $35,557 | $38,070 | - Non-cash financing activities for the six months ended June 30, 2025, included **$2.7 million** of accrued interest converted to principal balance under the 2025 Royalty Bond[144](index=144&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=46&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides a detailed discussion of REGENXBIO Inc.'s financial condition and results of operations for the three and six months ended June 30, 2025, compared to the same periods in 2024, covering business overview, product pipeline, collaboration agreements, revenue streams, operating expenses, critical accounting policies, and liquidity and capital resources [Overview](index=46&type=section&id=Overview) - REGENXBIO is a clinical-stage biotechnology company focused on gene therapy using its proprietary NAV Technology Platform to deliver functional genes for one-time treatments of various diseases[147](index=147&type=chunk)[148](index=148&type=chunk) [Overview of Product Candidates](index=46&type=section&id=Overview%20of%20Product%20Candidates) - ABBV-RGX-314 (surabgene lomparvovec, sura-vec) is being developed in collaboration with AbbVie for chronic retinal conditions (wet AMD, DR, DME) using both subretinal and suprachoroidal delivery. Topline data from pivotal trials (ATMOSPHERE and ASCENT) for wet AMD subretinal delivery are expected in 2026[148](index=148&type=chunk)[149](index=149&type=chunk) - New ALTITUDE trial data for DR showed durable safety and efficacy through **two years** with a single, in-office suprachoroidal injection, leading to plans for a pivotal two-part Phase IIb/III trial[152](index=152&type=chunk) - RGX-202 for Duchenne muscular dystrophy (Duchenne) showed positive interim safety and efficacy data from the Phase I/II AFFINITY DUCHENNE trial, with pivotal study enrollment expected to complete by October 2025 and BLA submission planned for mid-2026[154](index=154&type=chunk)[155](index=155&type=chunk)[156](index=156&type=chunk) - RGX-121 for Mucopolysaccharidosis Type II (MPS II) achieved its primary endpoint in the CAMPSIITE trial, demonstrating a reduction in cerebrospinal fluid Heparan sulfate levels. The FDA granted priority review for the BLA submitted in March 2025, with a PDUFA target action date of November 9, 2025[158](index=158&type=chunk)[159](index=159&type=chunk) - RGX-111 for Mucopolysaccharidosis Type I (MPS I) development was halted in November 2023 due to strategic pipeline prioritization, but efforts to continue development are ongoing as part of the Nippon Shinyaku partnership[161](index=161&type=chunk) [AbbVie Collaboration for ABBV-RGX-314](index=49&type=section&id=AbbVie%20Collaboration%20for%20ABBV-RGX-314) - The collaboration with AbbVie for ABBV-RGX-314 involves joint development and commercialization, with AbbVie paying an upfront fee of **$370.0 million** and potential milestones up to **$1.38 billion**[162](index=162&type=chunk) - An August 2025 amendment to the AbbVie Collaboration Agreement modified the development plan and milestone structure for the DR program, splitting a **$200.0 million** milestone into two **$100.0 million** payments[163](index=163&type=chunk) [Nippon Shinyaku Collaboration for RGX-121 and RGX-111](index=49&type=section&id=Nippon%20Shinyaku%20Collaboration%20for%20RGX-121%20and%20RGX-111) - The January 2025 collaboration with Nippon Shinyaku for RGX-121 and RGX-111 included an upfront payment of **$110.0 million** and potential milestones up to **$700.0 million**, with **$74.5 million** revenue recognized in H1 2025[164](index=164&type=chunk)[165](index=165&type=chunk) - Future royalties and milestones from the Nippon Shinyaku agreement, along with other NAV Technology Platform licenses, will be used to repay principal and interest under the May 2025 Royalty Bond with HCR[166](index=166&type=chunk) [NAV Technology Licensing Platform](index=51&type=section&id=NAV%20Technology%20Licensing%20Platform) - The NAV Technology Platform is licensed to other biotechnology and pharmaceutical companies, with one commercial product (Zolgensma) and several others in preclinical and clinical development, providing additional revenue opportunities and technological validation[167](index=167&type=chunk) [Financial Overview](index=51&type=section&id=Financial%20Overview) - Revenues are primarily from licensing the NAV Technology Platform and other intellectual property rights, with Zolgensma royalties being a significant component[168](index=168&type=chunk)[171](index=171&type=chunk) - Operating expenses consist mainly of cost of license and royalty revenues, research and development (R&D), and general and administrative (G&A) expenses, with personnel costs being a significant component[172](index=172&type=chunk) - R&D expenses are expected to remain significant due to ongoing product candidate development and early-stage research[177](index=177&type=chunk) - Interest expense is primarily associated with royalty monetization liabilities, including the 2020 Royalty Purchase Agreement and the 2025 Royalty Bond with HCR[183](index=183&type=chunk) [Critical Accounting Policies and Estimates](index=56&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) - Revenue recognition under ASC 606 involves a five-step model, requiring significant judgment in identifying performance obligations, determining transaction price (including variable consideration and milestones), and allocating revenue[186](index=186&type=chunk)[187](index=187&type=chunk)[193](index=193&type=chunk)[194](index=194&type=chunk)[195](index=195&type=chunk)[196](index=196&type=chunk) - The company evaluates collaboration agreements under ASC 808 to determine if transactions are with customers (ASC 606) or collaborative arrangements, applying consistent recognition and presentation methods[191](index=191&type=chunk) [Results of Operations](index=61&type=section&id=Results%20of%20Operations) Key Financial Results (in thousands) | Metric | Three Months Ended June 30, 2025 | Change (YoY) | Six Months Ended June 30, 2025 | Change (YoY) | | :-------------------------------- | :------------------------------- | :----------- | :------------------------------- | :----------- | | Total revenues | $21,359 | -$936 | $110,371 | +$72,454 | | License and royalty revenue | $18,465 | -$3,381 | $105,514 | +$68,324 | | Service revenue | $2,894 | +$2,445 | $4,857 | +$4,130 | | Research and development expense | $59,500 | +$10,631 | $112,587 | +$8,874 | | General and administrative expense | $19,883 | +$1,028 | $40,230 | +$3,084 | | Interest expense | $(10,993) | -$10,544 | $(19,563) | -$17,141 | | Net loss | $(70,871) | -$17,882 | $(64,788) | +$51,531 | - Q2 2025 revenues decreased slightly due to lower Zolgensma royalties, while YTD 2025 revenues significantly increased due to the Nippon Shinyaku upfront payment[203](index=203&type=chunk)[204](index=204&type=chunk)[208](index=208&type=chunk)[209](index=209&type=chunk) - R&D expenses increased in both periods, driven by higher manufacturing-related expenses, clinical trial costs for ABBV-RGX-314 and RGX-202, and personnel costs[205](index=205&type=chunk)[206](index=206&type=chunk)[210](index=210&type=chunk) - Interest expense saw a substantial increase in both periods, primarily due to royalty monetization liabilities from the 2020 Royalty Purchase Agreement and the new 2025 Royalty Bond[207](index=207&type=chunk)[212](index=212&type=chunk) [Liquidity and Capital Resources](index=62&type=section&id=Liquidity%20and%20Capital%20Resources) - As of June 30, 2025, the company had **$363.6 million** in cash, cash equivalents, and marketable securities, which management believes is sufficient to fund operations for at least the next **12 months**[213](index=213&type=chunk) - Key liquidity sources include **$144.5 million** (net) from the May 2025 Royalty Bond and a **$110.0 million** upfront payment from the Nippon Shinyaku Collaboration Agreement in March 2025[213](index=213&type=chunk)[214](index=214&type=chunk)[216](index=216&type=chunk) - The company expects to incur significant R&D and G&A expenses, requiring additional capital through equity offerings, debt financings, or strategic alliances to fund operations and potential commercialization[217](index=217&type=chunk)[228](index=228&type=chunk) - Future capital requirements are dependent on clinical trial timing and results, regulatory approvals, manufacturing capacity build-out, and the success of licensing agreements[229](index=229&type=chunk)[236](index=236&type=chunk) [Cash Flows](index=64&type=section&id=Cash%20Flows) Cash Flow Summary (in thousands) | Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Net cash used in operating activities | $(15,713) | $(100,952) | | Net cash provided by (used in) investing activities | $(95,693) | $12,915 | | Net cash provided by financing activities | $133,438 | $111,280 | | Net increase in cash and cash equivalents and restricted cash | $22,032 | $23,243 | - Net cash used in operating activities decreased by **$85.2 million** year-over-year, primarily due to the **$110.0 million** upfront fee from Nippon Shinyaku, which increased deferred revenue[220](index=220&type=chunk)[221](index=221&type=chunk) - Net cash used in investing activities was **$(95.7) million**, a significant change from **$12.9 million** provided in the prior year, mainly due to increased purchases of marketable debt securities[223](index=223&type=chunk)[224](index=224&type=chunk) - Net cash provided by financing activities increased to **$133.4 million**, driven by proceeds from the 2025 Royalty Bond and warrants, partially offset by royalty monetization repayments[225](index=225&type=chunk) [Off-Balance Sheet Arrangements](index=68&type=section&id=Off-Balance%20Sheet%20Arrangements) - The company did not have any off-balance sheet arrangements during the periods presented[232](index=232&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=68&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) This section refers to the company's Annual Report on Form 10-K for detailed market risk disclosures and states that there have been no material changes to its exposure to market risk during the six months ended June 30, 2025 - No material changes to market risk exposure occurred during the six months ended June 30, 2025[233](index=233&type=chunk) [Item 4. Controls and Procedures](index=68&type=section&id=Item%204.%20Controls%20and%20Procedures) This section details the evaluation of the effectiveness of the company's disclosure controls and procedures, concluding they were effective as of June 30, 2025, and states there were no material changes in internal control over financial reporting during the quarter - Management, including the CEO and CFO, concluded that disclosure controls and procedures were effective at a reasonable assurance level as of June 30, 2025[234](index=234&type=chunk)[235](index=235&type=chunk) - There were no changes in internal control over financial reporting during the quarter ended June 30, 2025, that materially affected, or are reasonably likely to materially affect, internal control over financial reporting[237](index=237&type=chunk) PART II—OTHER INFORMATION [Item 1. Legal Proceedings](index=71&type=section&id=Item%201.%20Legal%20Proceedings) This section states that the company is not currently party to any pending legal actions that could reasonably be expected to have a material adverse effect on its business, financial condition, results of operations, or cash flows - No pending legal actions are expected to have a material adverse effect on the company's business or financial condition[241](index=241&type=chunk) [Item 1A. Risk Factors](index=71&type=section&id=Item%201A.%20Risk%20Factors) This section refers to the material risk factors disclosed in the company's Annual Report on Form 10-K for the year ended December 31, 2024, and confirms that there have been no material changes to these risk factors - No material changes to the risk factors previously disclosed in the Annual Report on Form 10-K for the year ended December 31, 2024[242](index=242&type=chunk) [Item 2. Unregistered Sales of Equity Securities, Use of Proceeds and Issuer Purchases of Equity Securities](index=71&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities,%20Use%20of%20Proceeds%20and%20Issuer%20Purchases%20of%20Equity%20Securities) This section indicates that there were no unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities during the reporting period - No unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities occurred[243](index=243&type=chunk) [Item 3. Defaults Upon Senior Securities](index=71&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This section states that there were no defaults upon senior securities during the reporting period - No defaults upon senior securities occurred[244](index=244&type=chunk) [Item 4. Mine Safety Disclosures](index=71&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This section indicates that mine safety disclosures are not applicable to the company - Mine safety disclosures are not applicable[245](index=245&type=chunk) [Item 5. Other Information](index=71&type=section&id=Item%205.%20Other%20Information) This section reports that none of the company's directors or Section 16 reporting officers adopted or terminated any Rule 10b5-1 trading arrangements or non-Rule 10b5-1 trading arrangements during the three months ended June 30, 2025 - No Rule 10b5-1 or non-Rule 10b5-1 trading arrangements were adopted or terminated by directors or Section 16 officers during Q2 2025[246](index=246&type=chunk) [Item 6. Exhibits](index=72&type=section&id=Item%206.%20Exhibits) This section provides a list of exhibits filed with the Quarterly Report on Form 10-Q, including corporate documents, equity incentive plans, the loan agreement for the 2025 Royalty Bond, and certifications - Exhibits include the 2025 Equity Incentive Plan, forms of stock option and restricted stock unit agreements, and the Loan Agreement for the 2025 Royalty Bond[247](index=247&type=chunk) - Certifications from the Chief Executive Officer and Chief Financial Officer are included as Exhibits 31.1, 31.2, and 32.1[247](index=247&type=chunk) [Signatures](index=73&type=section&id=Signatures) This section contains the signatures of the President and Chief Executive Officer, Curran Simpson, and the Chief Financial Officer, Mitchell Chan, certifying the report on behalf of REGENXBIO Inc - The report is signed by Curran Simpson, President and CEO, and Mitchell Chan, CFO, on August 7, 2025[251](index=251&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________ to ______________ Commission File Number: 001-39411 Vital Farms, Inc. (Exact name of registrant as specified in its charter) FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 29, 2025 OR WASHINGTON, D.C. 20549 Securities register ...

FORM 10-Q UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________________ to ___________________ Commission File Number: 001-39871 SAB BIOTHERAPEUTICS, INC. (Mark One) (Exact Name of Registrant as Specified in its Charter) Del ...

THE TILE SHOP REPORTS SECOND QUARTER 2025 RESULTS Exhibit 99.1 MINNEAPOLIS – August 7, 2025 – Tile Shop Holdings, Inc. (Nasdaq: TTSH) (the "Company"), a specialty retailer of natural stone, man-made and luxury vinyl tiles, today announced results for its second quarter ended June 30, 2025. Second Quarter 2025 Summary Net Sales Decreased 3.4% Comparable Store Sales Decreased 3.5% Gross Margin of 64.4% Net Income of $0.4 Million and Adjusted EBITDA of $4.9 Million No Debt Outstanding and $27.8 million of Cash ...

[PART I. FINANCIAL INFORMATION](index=9&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=9&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Presents unaudited condensed consolidated financial statements, including balance sheets, operations, equity, cash flows, and detailed notes [Condensed Consolidated Balance Sheets](index=9&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Summarizes the company's assets, liabilities, and equity at specific points in time **Condensed Consolidated Balance Sheets (in thousands)** | Metric | June 30, 2025 | December 31, 2024 | | :---------------------------------- | :-------------- | :---------------- | | Cash and cash equivalents | $55,067 | $56,805 | | Short-term investments | $205,380 | $248,475 | | Total current assets | $270,242 | $309,802 | | Total assets | $285,495 | $314,933 | | Total current liabilities | $11,879 | $8,767 | | Total liabilities | $21,983 | $9,506 | | Total stockholders' equity | $263,512 | $305,427 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Details the company's revenues, expenses, and net loss over specific periods **Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except per share data)** | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development expenses | $22,680 | $15,689 | $42,252 | $28,193 | | General and administrative expenses | $6,816 | $5,111 | $14,352 | $9,701 | | Total operating expenses | $29,496 | $20,800 | $56,604 | $37,894 | | Loss from operations | $(29,496) | $(20,800) | $(56,604) | $(37,894) | | Interest income | $2,764 | $2,679 | $5,809 | $4,494 | | Change in fair value of preferred stock tranche right liability | $0 | $0 | $0 | $(7,390) | | Net loss | $(26,732) | $(18,121) | $(50,795) | $(40,790) | | Net loss per share, basic and diluted | $(0.75) | $(1.70) | $(1.44) | $(6.42) | | Weighted-average common shares outstanding | 35,444,635 | 10,666,528 | 35,356,098 | 6,356,700 | | Comprehensive loss | $(26,713) | $(18,144) | $(50,349) | $(40,977) | [Condensed Consolidated Statements of Convertible Preferred Stock and Stockholders' Equity (Deficit)](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Convertible%20Preferred%20Stock%20and%20Stockholders'%20Equity%20(Deficit)) Outlines changes in convertible preferred stock and stockholders' equity over reporting periods - Total stockholders' equity decreased from **$305.4 million** at December 31, 2024, to **$263.5 million** at June 30, 2025, primarily due to net loss and stock-based compensation[30](index=30&type=chunk) - Stock-based compensation expense for the six months ended June 30, 2025, was **$8.4 million**, contributing to the change in additional paid-in capital[30](index=30&type=chunk) - The company reported a net loss of **$26.7 million** for the three months ended June 30, 2025, and **$50.8 million** for the six months ended June 30, 2025, increasing the accumulated deficit[24](index=24&type=chunk)[30](index=30&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=12&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Reports cash inflows and outflows from operating, investing, and financing activities **Condensed Consolidated Statements of Cash Flows (in thousands)** | Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------------ | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(45,307) | $(33,967) | | Net cash provided by (used in) investing activities | $43,627 | $(149,030) | | Net cash (used in) provided by financing activities | $(58) | $223,012 | | Net (decrease) increase in cash, cash equivalents, and restricted cash | $(1,738) | $40,015 | | Cash, cash equivalents, and restricted cash at end of period | $55,172 | $110,269 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=13&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Provides detailed explanations and breakdowns for figures presented in the financial statements - Rapport Therapeutics, Inc. is a clinical-stage biotechnology company focused on neurological or psychiatric disorders, incorporated in February 2022[34](index=34&type=chunk) - The company has incurred recurring losses since inception, with an accumulated deficit of **$174.5 million** as of June 30, 2025, and expects to continue generating operating losses[37](index=37&type=chunk) - Cash, cash equivalents, and short-term investments totaled **$260.4 million** as of June 30, 2025, expected to fund operations for at least 12 months from the financial statement issuance date[37](index=37&type=chunk) - The company completed its IPO and concurrent private placement in June 2024, raising net proceeds of **$157.6 million**[37](index=37&type=chunk) - Significant estimates in financial statements include research and development expenses, valuation of common stock and stock-based awards, and preferred stock tranche right liability[45](index=45&type=chunk) - Short-term investments, classified as available-for-sale debt securities, consist of U.S. Treasury bills, government securities, and government agency securities, carried at fair market value[48](index=48&type=chunk) **Short-term investments (in thousands)** | Type | June 30, 2025 Fair Value | December 31, 2024 Fair Value | | :-------------------------- | :----------------------- | :------------------------- | | U.S. Treasury bills | $26,867 | $28,723 | | Government securities | $91,979 | $136,514 | | Government agency securities | $86,534 | $83,238 | | **Total** | **$205,380** | **$248,475** | - The Series B preferred stock tranche right liability was settled in March 2024, resulting in no balance as of June 30, 2024, and no further changes in fair value recognized[73](index=73&type=chunk) **Property and equipment, net (in thousands)** | Category | June 30, 2025 | December 31, 2024 | | :-------------------------- | :-------------- | :---------------- | | Lab equipment | $4,261 | $3,716 | | Computer equipment | $93 | $67 | | Leasehold improvements | $281 | $281 | | Construction in process | $0 | $430 | | Total property and equipment | $4,635 | $4,494 | | Less: Accumulated depreciation | $(1,463) | $(965) | | **Net property and equipment** | **$3,172** | **$3,529** | **Accrued expenses and other current liabilities (in thousands)** | Category | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :-------------- | :---------------- | | Research and development | $2,974 | $1,664 | | Professional fees | $759 | $235 | | Employee related | $3,141 | $3,708 | | Accrued other | $70 | $469 | | **Total** | **$6,944** | **$6,076** | - Prior to the June 2024 IPO, all outstanding Series A and Series B convertible preferred stock converted into **22,146,816 shares** of common stock[79](index=79&type=chunk) - In June 2024, the company issued and sold **9,200,000 shares** of common stock in its IPO and **1,058,824 shares** in a concurrent private placement, generating **$140.9 million** and **$16.7 million** in net proceeds, respectively[94](index=94&type=chunk)[95](index=95&type=chunk) - As of June 30, 2025, **9,096,166 shares** of common stock were reserved for issuance under the 2022 Plan, 2024 Plan, and 2024 ESPP[96](index=96&type=chunk) - Stock-based compensation expense for stock options was **$3.8 million** and **$7.2 million** for the three and six months ended June 30, 2025, respectively[119](index=119&type=chunk) - The company entered into new operating leases in San Diego, CA (commenced Jan 2025) and Boston, MA (commenced June 2025) for laboratory and office space[121](index=121&type=chunk)[123](index=123&type=chunk) - Related party transactions with Janssen Pharmaceutical NV and Third Rock Ventures LLC decreased to zero for the six months ended June 30, 2025, from **$69 thousand** and **$0.1 million**, respectively, in 2024[124](index=124&type=chunk)[125](index=125&type=chunk) - The company entered into a Master Services Agreement with NeuroPace Inc. in November 2023 for data services related to the RAP-219 Phase 2a clinical trial, with payments up to **$3.7 million**[128](index=128&type=chunk)[131](index=131&type=chunk) - The company operates as a single operating and reportable segment, focused on developing medicines for neurological or psychiatric disorders in the United States[132](index=132&type=chunk)[133](index=133&type=chunk)[134](index=134&type=chunk) - Basic and diluted net loss per common share was **$(0.75)** and **$(1.44)** for the three and six months ended June 30, 2025, respectively[138](index=138&type=chunk) - On July 1, 2025, the company filed an S-3 registration statement for up to **$400.0 million** in securities, including an at-the-market (ATM) offering program for up to **$150.0 million** in common stock[140](index=140&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=38&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Analyzes Rapport Therapeutics' financial condition, operations, clinical progress, product candidates, and future funding needs [Overview](index=38&type=section&id=Overview) Provides a high-level summary of the company's business, lead product candidates, and financial performance - Rapport Therapeutics is a clinical-stage biotechnology company developing small molecule precision medicines for neurological or psychiatric disorders using its RAP technology platform[144](index=144&type=chunk) - RAP-219, the lead product candidate, is an AMPA receptor negative allosteric modulator (NAM) targeting TARPγ8 for focal onset seizures, bipolar disorder, and peripheral neuropathic pain[145](index=145&type=chunk) - Phase 2a proof-of-concept trial in drug-resistant focal onset seizures is on track for topline results in September 2025[147](index=147&type=chunk) - Phase 2 proof-of-concept trial in bipolar mania has been initiated, with topline results expected in the first half of 2027[147](index=147&type=chunk) - The IND for a Phase 2a trial of RAP-219 for diabetic peripheral neuropathic pain (DPNP) was placed on clinical hold by the FDA in Q4 2024, pending additional information[147](index=147&type=chunk) - The company has incurred significant operating losses since inception, with net losses of **$26.7 million** and **$50.8 million** for the three and six months ended June 30, 2025, respectively, and an accumulated deficit of **$174.5 million**[150](index=150&type=chunk) - As of June 30, 2025, cash, cash equivalents, and short-term investments totaled **$260.4 million**, expected to fund operations through the end of 2026[149](index=149&type=chunk)[155](index=155&type=chunk) [License and Collaboration Agreements](index=42&type=section&id=License%20and%20Collaboration%20Agreements) Details key agreements with third parties for product development and data services - The company holds an exclusive option and license agreement with Janssen Pharmaceutical NV for TARPγ8 AMPAR products and a non-exclusive license for nACh products[156](index=156&type=chunk) - Payments to Janssen include a **$1.0 million** upfront payment, a **$4.0 million** option fee, and potential future development and sales milestone payments up to **$76.0 million** and **$40.0 million**, respectively, for the lead TARPγ8 product, plus royalties[157](index=157&type=chunk)[158](index=158&type=chunk) - A Master Services Agreement with NeuroPace Inc. provides data services for the RAP-219 Phase 2a clinical trial in drug-resistant focal onset seizures, with payments up to **$3.7 million** over two years[160](index=160&type=chunk)[161](index=161&type=chunk) [Components of Results of Operations](index=44&type=section&id=Components%20of%20Results%20of%20Operations) Explains the primary drivers and nature of the company's operating expenses, interest income, and other financial items - Research and development expenses include personnel, external costs (CMOs, CROs), intellectual property licenses, lab supplies, and facility costs, and are expected to increase substantially[162](index=162&type=chunk)[163](index=163&type=chunk)[165](index=165&type=chunk) - General and administrative expenses cover personnel, legal, professional, office, IT, and facility costs, and are anticipated to rise due to public company operations and growth[167](index=167&type=chunk)[168](index=168&type=chunk) - Interest income is derived from cash, cash equivalents, and short-term investments[169](index=169&type=chunk) - The Series B preferred stock tranche right liability was settled in March 2024, eliminating future changes in fair value recognition[171](index=171&type=chunk) - No income tax provision was recorded for the periods, with a full valuation allowance against net deferred tax assets[172](index=172&type=chunk) [Results of Operations](index=47&type=section&id=Results%20of%20Operations) Compares the company's financial performance across different reporting periods, highlighting key changes in expenses and net loss **Results of Operations (Three Months Ended June 30, in thousands)** | Metric | 2025 | 2024 | Change | | :-------------------------- | :--- | :--- | :----- | | Research and development | $22,680 | $15,689 | $6,991 | | General and administrative | $6,816 | $5,111 | $1,705 | | Total operating expenses | $29,496 | $20,800 | $8,696 | | Loss from operations | $(29,496) | $(20,800) | $(8,696) | | Interest income | $2,764 | $2,679 | $85 | | Net loss | $(26,732) | $(18,121) | $(8,611) | **Results of Operations (Six Months Ended June 30, in thousands)** | Metric | 2025 | 2024 | Change | | :------------------------------------------------ | :--- | :--- | :----- | | Research and development | $42,252 | $28,193 | $14,059 | | General and administrative | $14,352 | $9,701 | $4,651 | | Total operating expenses | $56,604 | $37,894 | $18,710 | | Loss from operations | $(56,604) | $(37,894) | $(18,710) | | Interest income | $5,809 | $4,494 | $1,315 | | Change in fair value of preferred stock tranche right liability | $0 | $(7,390) | $7,390 | | Net loss | $(50,795) | $(40,790) | $(10,005) | - The increase in R&D expenses for both periods was primarily driven by higher RAP-219 program costs (clinical trials, manufacturing) and increased personnel-related costs, partially offset by decreased preclinical program costs[178](index=178&type=chunk)[185](index=185&type=chunk) - General and administrative expenses increased due to higher workforce expenses (salaries, bonuses, stock-based compensation) and administrative costs associated with public company operations, partially offset by reduced professional and consulting fees post-IPO[177](index=177&type=chunk)[182](index=182&type=chunk)[184](index=184&type=chunk)[186](index=186&type=chunk)[187](index=187&type=chunk) - Interest income increased due to higher cash, cash equivalents, and short-term investment balances following the IPO and Series B financing[179](index=179&type=chunk)[188](index=188&type=chunk) [Liquidity and Capital Resources](index=52&type=section&id=Liquidity%20and%20Capital%20Resources) Assesses the company's ability to meet its short-term and long-term financial obligations and its funding strategies - As of June 30, 2025, the company had **$260.4 million** in cash, cash equivalents, and short-term investments, excluding restricted cash[190](index=190&type=chunk) - Historically, operations have been funded by convertible notes, preferred stock, and common stock (IPO and private placement), totaling **$424.4 million** in gross proceeds[190](index=190&type=chunk) - The company filed an S-3 registration statement for up to **$400.0 million** in securities, including an ATM program for up to **$150.0 million** in common stock, to fund future operations[191](index=191&type=chunk) **Cash Flows Summary (Six Months Ended June 30, in thousands)** | Activity | 2025 | 2024 | | :------------------------------------------ | :--- | :--- | | Net cash used in operating activities | $(45,307) | $(33,967) | | Net cash provided by (used in) investing activities | $43,627 | $(149,030) | | Net cash (used in) provided by financing activities | $(58) | $223,012 | | Net increase (decrease) in cash, cash equivalents and restricted cash | $(1,738) | $40,015 | - Operating activities used **$45.3 million** in cash in H1 2025, an increase from **$34.0 million** in H1 2024, primarily due to net loss and increased prepaid expenses[193](index=193&type=chunk)[194](index=194&type=chunk) - Investing activities provided **$43.6 million** in cash in H1 2025, a significant shift from using **$149.0 million** in H1 2024, driven by maturities of short-term investments[195](index=195&type=chunk)[196](index=196&type=chunk) - Financing activities used **$0.1 million** in cash in H1 2025, compared to providing **$223.0 million** in H1 2024, reflecting deferred offering costs in 2025 versus IPO and preferred stock proceeds in 2024[197](index=197&type=chunk)[198](index=198&type=chunk) - Existing cash, cash equivalents, and short-term investments are projected to fund operating expenses and capital expenditure requirements through the end of 2026[199](index=199&type=chunk) - Future funding requirements are substantial and depend on the progress of product candidates, clinical trials, manufacturing, regulatory approvals, and commercialization efforts[200](index=200&type=chunk)[205](index=205&type=chunk) - Contractual obligations include **$14.2 million** in future minimum operating lease payments over a weighted average remaining lease term of 5.0 years[203](index=203&type=chunk) - The Janssen License includes potential milestone payments up to **$76.0 million** for development and **$40.0 million** for sales, plus tiered royalties[204](index=204&type=chunk) - The NeuroPace Agreement involves payments up to **$3.7 million** over approximately two years for services and milestone achievement[206](index=206&type=chunk) [Critical Accounting Policies and Estimates](index=58&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) Highlights key accounting policies and estimates that require significant judgment and can materially impact financial reporting - There were no material changes to the critical accounting policies and estimates previously disclosed in the Annual Report on Form 10-K for the year ended December 31, 2024[210](index=210&type=chunk) [Emerging Growth Company and Smaller Reporting Company Status](index=58&type=section&id=Emerging%20Growth%20Company%20and%20Smaller%20Reporting%20Company%20Status) Discusses the company's status as an emerging growth company and smaller reporting company, and the associated reduced disclosure requirements - The company qualifies as an 'emerging growth company' (EGC) and has elected to use the extended transition period for complying with new or revised accounting standards[211](index=211&type=chunk)[507](index=507&type=chunk) - As an EGC, the company benefits from reduced reporting requirements, including fewer years of audited financial statements and reduced executive compensation disclosures[505](index=505&type=chunk) - The company also qualifies as a 'smaller reporting company,' allowing for scaled disclosures[213](index=213&type=chunk)[508](index=508&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=59&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, detailed quantitative and qualitative disclosures about market risk are not required - The company is a smaller reporting company and is not required to provide the information required by this Item[213](index=213&type=chunk) [Item 4. Controls and Procedures](index=59&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls and procedures were effective as of June 30, 2025, with no material changes in internal control - Management, including the principal executive officer and principal financial officer, concluded that disclosure controls and procedures were effective as of June 30, 2025, at the reasonable assurance level[215](index=215&type=chunk) - There have been no material changes in internal control over financial reporting during the quarter ended June 30, 2025[216](index=216&type=chunk) [PART II. OTHER INFORMATION](index=60&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=60&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in legal proceedings expected to have a material adverse effect on its business - The company is not currently a party to any litigation or legal proceedings that are probable to have a material adverse effect on its business[219](index=219&type=chunk) [Item 1A. Risk Factors](index=60&type=section&id=Item%201A.%20Risk%20Factors) Outlines risks and uncertainties impacting Rapport Therapeutics, covering operations, finances, product development, third parties, and intellectual property [Risks Related to Our Limited Operating History, Financial Condition and Need for Additional Capital](index=60&type=section&id=Risks%20Related%20to%20Our%20Limited%20Operating%20History,%20Financial%20Condition%20and%20Need%20for%20Additional%20Capital) Highlights risks associated with the company's early stage, recurring losses, and substantial future funding requirements - As a clinical-stage biotechnology company formed in February 2022, Rapport Therapeutics has a limited operating history and has not generated any revenue from product sales[222](index=222&type=chunk) - The company has incurred significant net losses since inception, totaling **$50.8 million** for the six months ended June 30, 2025, and an accumulated deficit of **$174.5 million**[223](index=223&type=chunk) - Additional funding will be required to finance operations and product development, and the inability to raise capital on acceptable terms could force delays or elimination of programs[227](index=227&type=chunk)[231](index=231&type=chunk) - Raising additional capital through equity or convertible debt securities will dilute existing stockholders' ownership interests[232](index=232&type=chunk) - Obligations under the Janssen License, including substantial contingent milestone payments, could be a significant drain on cash resources or necessitate incurring debt[238](index=238&type=chunk) [Risks Related to Our Business](index=66&type=section&id=Risks%20Related%20to%20Our%20Business) Covers risks concerning product development, resource allocation, acquisitions, data security, and the use of new technologies - The business is highly dependent on the successful clinical development, regulatory approval, and commercialization of product candidates, particularly RAP-219 for focal onset seizures[239](index=239&type=chunk) - The successful development of pharmaceutical products is a lengthy, expensive, and highly uncertain process, with many product candidates failing to reach the market[242](index=242&type=chunk) - The FDA placed a clinical hold on the Phase 2a proof-of-concept trial of RAP-219 for diabetic peripheral neuropathic pain (DPNP) in Q4 2024, requesting additional information[242](index=242&type=chunk) - Due to significant resource requirements, the company must prioritize certain product candidates, potentially diverting resources from more profitable opportunities[248](index=248&type=chunk)[249](index=249&type=chunk) - Growth through acquisitions or investments in new businesses, products, or technologies carries numerous risks, including integration difficulties, unanticipated costs, and diversion of management attention[250](index=250&type=chunk)[251](index=251&type=chunk) - The company is subject to stringent and evolving privacy and data security laws (e.g., CCPA, GDPR, NIS 2), with potential for significant fines, penalties, and business disruption from non-compliance or breaches[253](index=253&type=chunk)[254](index=254&type=chunk)[260](index=260&type=chunk)[262](index=262&type=chunk)[266](index=266&type=chunk)[270](index=270&type=chunk) - Information technology systems and data are vulnerable to cyber-attacks, cybersecurity incidents, and breaches, which could result in substantial costs, revenue loss, and operational disruption[272](index=272&type=chunk)[278](index=278&type=chunk) - The use of new technologies like AI/ML introduces risks related to intellectual property, compliance with evolving regulations (e.g., EU AI Act), and potential for reputational harm or liability[281](index=281&type=chunk)[282](index=282&type=chunk) [Risks Related to the Discovery and Development of Our Current or Future Product Candidates](index=81&type=section&id=Risks%20Related%20to%20the%20Discovery%20and%20Development%20of%20Our%20Current%20or%20Future%20Product%20Candidates) Addresses challenges in regulatory approval, clinical trials, potential side effects, market acceptance, patient enrollment, and reliance on third-party manufacturing - The regulatory approval processes of the FDA, EMA, MHRA, and other authorities are lengthy, time-consuming, and inherently unpredictable, with no guarantee of approval[285](index=285&type=chunk) - Clinical trials are expensive, difficult to design, and have a high failure rate; positive results from early stages may not be replicated in later-stage trials[286](index=286&type=chunk)[293](index=293&type=chunk)[295](index=295&type=chunk) - Product candidates may cause undesirable side effects or have other properties that could delay or prevent regulatory approval or limit commercialization[303](index=303&type=chunk)[305](index=305&type=chunk) - Even if approved, product candidates may fail to achieve sufficient market acceptance by physicians, patients, and third-party payors, hindering commercial success[322](index=322&type=chunk) - Concentrating R&D efforts on nervous system disorders presents challenges due to reliance on subjective patient-reported outcomes and the placebo effect[315](index=315&type=chunk) - Difficulties in enrolling patients in clinical trials, influenced by eligibility criteria, competition, and patient drop-out rates, could significantly delay or adversely affect development activities[317](index=317&type=chunk)[318](index=318&type=chunk)[321](index=321&type=chunk) - Reliance on third parties (CROs, CMOs, NeuroPace) for clinical trials and manufacturing introduces risks related to control, timing, expense, quality, and compliance with regulatory requirements[351](index=351&type=chunk)[353](index=353&type=chunk)[354](index=354&type=chunk)[358](index=358&type=chunk)[361](index=361&type=chunk)[366](index=366&type=chunk) - Geopolitical events and trade policies, particularly concerning manufacturers located outside the United States (e.g., China), could disrupt supply chains and increase costs[365](index=365&type=chunk) - Establishing collaborations on commercially reasonable terms is challenging, and reliance on collaborators' efforts and resources poses risks to product development and commercialization[370](index=370&type=chunk)[371](index=371&type=chunk)[376](index=376&type=chunk) - Scaling up manufacturing production or changes in product candidate manufacturing methods/formulation may result in additional costs or delays[379](index=379&type=chunk)[380](index=380&type=chunk) [Risks Related to Government Regulation](index=111&type=section&id=Risks%20Related%20to%20Government%20Regulation) Examines regulatory approval complexities, ongoing compliance, expedited pathways, funding impacts, healthcare laws, reimbursement, and international regulations - Obtaining regulatory approval in one jurisdiction does not guarantee approval in others, and foreign regulatory requirements can be more extensive and time-consuming[381](index=381&type=chunk)[382](index=382&type=chunk) - Approved products are subject to ongoing regulatory obligations, including manufacturing, labeling, safety reporting, and compliance with cGMP and GCP, with potential penalties for non-compliance[383](index=383&type=chunk)[386](index=386&type=chunk) - While the company may seek expedited approval pathways (e.g., Fast Track, Breakthrough Therapy), there is no assurance of obtaining such designations or realizing their intended benefits[389](index=389&type=chunk)[390](index=390&type=chunk)[391](index=391&type=chunk)[392](index=392&type=chunk) - Accelerated approval pathways require confirmatory trials, and failure to verify clinical benefit or comply with post-marketing requirements could lead to withdrawal of approval[395](index=395&type=chunk)[396](index=396&type=chunk) - Inadequate funding for the FDA or other government agencies could hinder their ability to review and approve new products in a timely manner, negatively impacting the business[398](index=398&type=chunk)[401](index=401&type=chunk) - Relationships with healthcare providers and payors are subject to anti-kickback, fraud and abuse, and other healthcare laws, risking criminal sanctions, civil penalties, and reputational harm[403](index=403&type=chunk)[405](index=405&type=chunk) - Coverage and adequate reimbursement from third-party payors are critical for product success, but may be limited or unavailable, making profitable sales difficult[406](index=406&type=chunk)[408](index=408&type=chunk)[409](index=409&type=chunk) - Ongoing healthcare legislative and regulatory reforms, such as the Inflation Reduction Act and OBBBA, could reduce Medicare/Medicaid spending, impact drug pricing, and adversely affect revenue and commercial prospects[414](index=414&type=chunk)[415](index=415&type=chunk)[416](index=416&type=chunk)[418](index=418&type=chunk) - Employee misconduct or off-label use of product candidates could lead to regulatory sanctions, product liability suits, and reputational harm[422](index=422&type=chunk)[423](index=423&type=chunk) - EU drug marketing and reimbursement regulations, including governmental price controls and anti-bribery laws, could materially affect the ability to market and receive coverage for products[424](index=424&type=chunk)[425](index=425&type=chunk)[428](index=428&type=chunk) - The company is subject to export/import controls, economic sanctions, and anti-corruption laws (e.g., FCPA), with potential for criminal liability and other serious consequences for violations[429](index=429&type=chunk) - Failure to comply with environmental, health, and safety laws by the company or third-party manufacturers could result in fines, penalties, or liabilities[430](index=430&type=chunk)[433](index=433&type=chunk) [Risks Related to Our Intellectual Property](index=126&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) Details risks concerning in-licensed IP, patent protection, challenges, foreign laws, term extensions, infringement claims, trade secrets, and trademarks - The company depends on in-licensed intellectual property, and failure to comply with license obligations could lead to the loss of important rights[435](index=435&type=chunk) - Inability to obtain and maintain broad patent protection for product candidates, or challenges to existing patents, could allow competitors to commercialize similar products[437](index=437&type=chunk)[438](index=438&type=chunk)[439](index=439&type=chunk)[443](index=443&type=chunk) - Changes to patent law in the United States and foreign jurisdictions could diminish the value of patents and impair the ability to protect product candidates[455](index=455&type=chunk)[456](index=456&type=chunk) - The company may not be able to protect its intellectual property rights throughout the world, as foreign laws may offer less extensive protection[457](index=457&type=chunk)[459](index=459&type=chunk) - Failure to obtain patent term extension for product candidates could allow competitors to enter the market sooner[460](index=460&type=chunk) - Third parties may initiate legal proceedings alleging infringement or misappropriation of their intellectual property, leading to substantial damages, injunctions, or costly licensing requirements[461](index=461&type=chunk)[462](index=462&type=chunk)[465](index=465&type=chunk) - Intellectual property litigation is expensive, time-consuming, and can divert management's attention, potentially harming the business[471](index=471&type=chunk) - Inability to protect the confidentiality of trade secrets would harm the business and competitive position[473](index=473&type=chunk)[474](index=474&type=chunk) - Claims challenging inventorship or ownership of patents and other intellectual property could be expensive and time-consuming[472](index=472&type=chunk) - Inadequate protection of trademarks and trade names could hinder name recognition and adversely affect the business[476](index=476&type=chunk) - European patents and applications could be challenged in the Unified Patent Court (UPC), potentially leading to a loss of patent protection across multiple European countries[477](index=477&type=chunk) [Risks Related to Ownership of Our Common Stock](index=138&type=section&id=Risks%20Related%20to%20Ownership%20of%20Our%20Common%20Stock) Addresses stock price volatility, fluctuating operating results, analyst coverage, significant stockholder control, dilution, dividend policy, and anti-takeover provisions - The trading price of the common stock may be volatile due to factors such as clinical trial results, regulatory decisions, competition, and general market conditions[480](index=480&type=chunk)[481](index=481&type=chunk) - Quarterly and annual operating results may fluctuate significantly, making future predictions difficult and potentially causing results to fall below expectations[482](index=482&type=chunk) - Inaccurate or unfavorable research, or cessation of coverage by securities or industry analysts, could lead to a decline in stock price and trading volume[484](index=484&type=chunk) - Executive officers, directors, and principal stockholders own a significant percentage of common stock, allowing them to exert substantial control over corporate actions[487](index=487&type=chunk) - Sales of a substantial number of shares of common stock in the public market could cause the stock price to fall[488](index=488&type=chunk) - Future issuances of additional capital stock in connection with financings, acquisitions, or incentive plans will dilute existing stockholders' ownership interests[492](index=492&type=chunk) - The company does not currently intend to pay dividends on its common stock, meaning investment return will depend solely on stock price appreciation[493](index=493&type=chunk) - Provisions in corporate charter documents and Delaware law could make an acquisition of the company more difficult and prevent attempts to replace current directors and management[494](index=494&type=chunk)[495](index=495&type=chunk) - Bylaw provisions designating specific courts as the sole forum for certain actions may limit stockholders' ability to choose favorable judicial forums[496](index=496&type=chunk)[497](index=497&type=chunk) - Failure to continue satisfying Nasdaq listing requirements could lead to delisting, impairing stockholders' ability to trade common stock and affecting its market price[500](index=500&type=chunk) [Other General Risks](index=148&type=section&id=Other%20General%20Risks) Covers risks from global economic conditions, business interruptions, public company operations, internal controls, tax law changes, and various litigations - Unfavorable global economic conditions, political instability, and geopolitical events (e.g., conflicts, trade tensions, inflation) could adversely affect the business, financial condition, and stock price[501](index=501&type=chunk)[502](index=502&type=chunk)[503](index=503&type=chunk) - Natural disasters, public health crises, or other business interruptions could severely disrupt operations and delay clinical trials or regulatory approvals[504](index=504&type=chunk) - The company's status as an 'emerging growth company' and 'smaller reporting company' with reduced reporting requirements may make its common stock less attractive to investors[505](index=505&type=chunk)[506](index=506&type=chunk) - Operating as a public company incurs significant legal, accounting, and compliance costs, diverting management time and potentially increasing net loss[511](index=511&type=chunk)[512](index=512&type=chunk) - Failure to establish and maintain an effective system of internal control over financial reporting could lead to inaccurate financial reporting, fraud, and loss of investor confidence[513](index=513&type=chunk)[515](index=515&type=chunk) - The ability to use net operating loss carryforwards and other tax attributes may be limited by ownership changes or regulatory changes, even if profitability is achieved[517](index=517&type=chunk)[518](index=518&type=chunk) - Changes in tax law, such as the OBBBA or Section 174 of the Code, could adversely affect the business and financial condition[519](index=519&type=chunk) - Clinical trial and product liability lawsuits could divert resources, result in substantial liabilities, and limit commercialization of any developed products[520](index=520&type=chunk)[521](index=521&type=chunk) - General litigation, including securities class action lawsuits, could be expensive, divert management's attention, and harm the business[522](index=522&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=157&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) Confirms no unregistered equity sales and no material changes to IPO and private placement proceeds use - No unregistered sales of equity securities occurred during the period[524](index=524&type=chunk) - There has been no material change in the planned use of proceeds from the initial public offering and concurrent private placement[525](index=525&type=chunk) [Item 3. Defaults Upon Senior Securities](index=157&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) Reports no defaults upon senior securities during the period - No defaults upon senior securities were reported[527](index=527&type=chunk) [Item 4. Mine Safety Disclosures](index=157&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the registrant - This item is not applicable to the registrant[528](index=528&type=chunk) [Item 5. Other Information](index=157&type=section&id=Item%205.%20Other%20Information) No directors or officers adopted, modified, or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements - None of the directors or officers adopted, modified, or terminated a Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement during the quarter ended June 30, 2025[529](index=529&type=chunk) [Item 6. Exhibits](index=158&type=section&id=Item%206.%20Exhibits) Lists all exhibits filed as part of the Form 10-Q, including corporate governance documents, agreements, and certifications - Key exhibits filed include the Third Amended and Restated Certificate of Incorporation, Amended and Restated Bylaws, Specimen Common Stock Certificate, Amended and Restated Investors' Rights Agreement, and a Lease Agreement[531](index=531&type=chunk) - Certifications from the Principal Executive Officer and Principal Financial Officer pursuant to the Securities Exchange Act of 1934 and Sarbanes-Oxley Act of 2002 are included[531](index=531&type=chunk) [Signatures](index=159&type=section&id=Signatures) Contains required signatures of the CEO and CFO, certifying the accuracy and completeness of the quarterly report - The report is duly signed on August 7, 2025, by Abraham N. Ceesay, Chief Executive Officer and Director, and Troy Ignelzi, Chief Financial Officer (Principal Financial and Accounting Officer)[537](index=537&type=chunk)

Second-Quarter 2025 Detailed Supplemental Information | | | | 2024 | | | | 2025 | | | | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | | | 1st Qtr | 2nd Qtr | 3rd Qtr | 4th Qtr | Full Year | 1st Qtr | 2nd Qtr | 3rd Qtr 4th Qtr | YTD | | $ Millions, Except as Indicated | | | | | | | | | | | CONSOLIDATED INCOME STATEMENT | | | | | | | | | | | Revenues and Other Income | | | | | | | | | | | Sales and other operating revenues | 13,848 | 13,620 | 13,041 | 14,236 | 54,745 | 16,517 | 14,004 | | 30,521 ...

Exhibit 99.1 Nuvation Bio Reports Second Quarter 2025 Financial Results and Provides Business Update Received U.S. Food and Drug Administration (FDA) approval for IBTROZI™ (taletrectinib), a next-generation oral treatment for advanced ROS1- positive (ROS1+) non-small cell lung cancer (NSCLC) on June 11, 2025 Successfully started 70 patients on IBTROZI as of July 31, 2025, approximately seven weeks after U.S. FDA approval National Comprehensive Cancer Network® added taletrectinib (IBTROZI) as a Preferred Opt ...

[Executive Summary & Key Announcements](index=1&type=section&id=Executive%20Summary%20%26%20Key%20Announcements) WhiteHorse Finance, Inc. announced its Q2 2025 financial results, CEO insights on portfolio performance, and declared its quarterly distribution [Second Quarter 2025 Earnings Announcement](index=1&type=section&id=Second%20Quarter%202025%20Earnings%20Results%20and%20Declares%20Quarterly%20Distribution) WhiteHorse Finance, Inc. announced its financial results for the second quarter ended June 30, 2025, and declared a quarterly distribution of $0.385 per share - WhiteHorse Finance, Inc. announced its financial results for the second quarter ended June 30, 2025[1](index=1&type=chunk) - The Company's board of directors declared a distribution of **$0.385 per share** for the quarter ended June 30, 2025[1](index=1&type=chunk) [CEO Commentary](index=1&type=section&id=Stuart%20Aronson%2C%20WhiteHorse%20Finance%27s%20Chief%20Executive%20Officer%2C%20commented) CEO Stuart Aronson noted continued portfolio-specific challenges but expressed confidence in the broader portfolio's stability, highlighting efforts to resolve underperforming investments and a disciplined approach to new originations - Second-quarter results continued to reflect portfolio-specific challenges, though the Company remains confident in the underlying stability of the broader portfolio[2](index=2&type=chunk) - Steady progress is being made in resolving underperforming investments with the support of an experienced restructuring team[2](index=2&type=chunk) - Origination activity is in line with seasonal norms, favoring selectivity over volume, with a focus on credits with strong fundamentals to navigate the current environment and support long-term shareholder value[2](index=2&type=chunk) [Quarterly Distribution Declaration](index=1&type=section&id=Distributions) The Company's board declared a distribution of $0.385 per share for Q2 2025, payable on October 3, 2025, marking the fifty-first consecutive quarter with distributions at or above $0.355 per share Q2 2025 Quarterly Distribution Details | Metric | Value | | :----- | :---- | | Per Share | $0.385 | | Payable Date | October 3, 2025 | | Record Date | September 19, 2025 | - Distributions declared at or above **$0.355** for the fifty-first consecutive quarter since the Company's initial public offering[8](index=8&type=chunk) - Distributions are paid from taxable earnings and may include return of capital and/or capital gains, with specific tax characteristics reported on Form 1099-DIV[9](index=9&type=chunk) [Financial Performance](index=1&type=section&id=Financial%20Performance) This section analyzes WhiteHorse Finance, Inc.'s Q2 2025 financial results, covering investment income, expenses, net investment income, and net asset value [Summary Financial Results (QoQ)](index=1&type=section&id=Summary%20Information%20as%20of%20June%2030%2C%202025%20and%20March%2031%2C%202025%20%28unaudited%29) For Q2 2025, total investment income slightly increased by 0.2% QoQ to $18.838 million, while total expenses rose by 2.7% to $12.276 million, leading to a 4.1% decrease in net investment income to $6.562 million, or $0.282 per share Summary Financial Information (QoQ) | Metric ($ in thousands except per share data) | June 30, 2025 | March 31, 2025 | Change ($ in thousands) | Change (%) | | :------------------------------------------ | :------------ | :------------- | :---------------------- | :--------- | | Total investment income | 18,838 | 18,801 | 37 | 0.2 | | Total expenses, including excise tax | 12,276 | 11,958 | 318 | 2.7 | | Net investment income and core net investment income | 6,562 | 6,843 | (281) | (4.1) | | Net investment income and core net investment income per share | 0.282 | 0.294 | (0.012) | (4.1) | | Net realized gains (losses) | (22,045) | (402) | (21,643) | NM | | Net unrealized appreciation (depreciation) | 17,733 | (2,177) | 19,910 | NM | | Net increase (decrease) in net assets resulting from operations | 2,250 | 4,264 | (2,014) | (47.2) | | Total investments, at fair value | 629,264 | 650,999 | (21,735) | (3.3) | | STRS JV total investments, at fair value | 330,187 | 310,160 | 20,027 | 6.5 | | Net asset value per share | 11.82 | 12.11 | (0.29) | (2.4) | [Net Investment Income (GAAP vs. Non-GAAP)](index=3&type=section&id=SCHEDULE%201) Core net investment income, a non-GAAP measure, was $6.562 million ($0.282 per share) for Q2 2025, consistent with GAAP net investment income for the period, representing a decrease from $9.292 million ($0.400 per share) in Q2 2024 - Core net investment income is a non-GAAP financial measure that provides useful information by reflecting the Company's financial performance excluding specific costs associated with refinancing, capital gains incentive fees, and related excise/income taxes[14](index=14&type=chunk)[17](index=17&type=chunk) Reconciliation of Net Investment Income to Core Net Investment Income | Metric ($ in thousands, except per share data) | June 30, 2025 Amount | June 30, 2025 Per Share | June 30, 2024 Amount | June 30, 2024 Per Share | | :------------------------------------------- | :------------------- | :---------------------- | :------------------- | :---------------------- | | Net investment income | $6,562 | 0.282 | $9,292 | 0.400 | | Net impact of costs associated with refinancing of indebtedness | - | - | - | - | | Accrual for capital gains incentive fee | - | - | - | - | | Net impact of excise tax expense related to net realized gains and losses | - | - | - | - | | **Core net investment income** | **$6,562** | **0.282** | **$9,292** | **0.400** | - There were no excise or other income taxes related to net realized gains and losses for the quarters ended June 30, 2025, and June 30, 2024[17](index=17&type=chunk) [Portfolio and Investment Activity](index=2&type=section&id=Portfolio%20and%20Investment%20Activity) This section outlines WhiteHorse Finance, Inc.'s Q2 2025 investment portfolio composition, yield, and transaction activities [Portfolio Composition and Yield](index=2&type=section&id=Portfolio%20Composition%20and%20Yield) As of June 30, 2025, the investment portfolio's fair value was $629.3 million, a 3.3% decrease, predominantly comprising first lien secured loans (77.6%) with a weighted average effective yield of 11.9% Portfolio Snapshot (June 30, 2025) | Metric | Value | | :------------------------------------ | :-------------------- | | Fair Value of Investment Portfolio | $629.3 million | | Number of Positions | 132 | | Number of Companies | 71 | | Weighted Average Effective Yield (debt) | 11.9% | | Average Debt Investment Size | $5.0 million | Portfolio Composition by Asset Type (June 30, 2025) | Asset Type | Percentage of Portfolio | | :-------------------------- | :---------------------- | | First Lien Secured Loans | 77.6% | | Second Lien Secured Loans | 0.5% | | Unsecured Loans | 0.2% | | Equity | 4.8% | | Investments in STRS JV | 16.9% | - Almost all loans (**98.7%**) were variable rate investments (primarily indexed to SOFR) with fixed rate securities representing only **1.3%** of loans at fair value, and nearly all performing floating rate investments have interest rate floors[4](index=4&type=chunk) [Investment Transactions](index=2&type=section&id=Investment%20Activity) During Q2 2025, WhiteHorse Finance made $33.1 million in new investments and $5.9 million in additions, while proceeds from sales and repayments totaled $36.2 million, including full repayments from two companies Investment Activity (Three Months Ended June 30, 2025) | Activity | Amount ($ millions) | | :------------------------------------ | :------------------ | | Investments in new portfolio companies | 33.1 | | Additions to existing portfolio companies | 5.9 | | Net fundings to revolver loans | 0.3 | | Proceeds from sales and repayments | 36.2 | | Assets transferred to STRS JV | 22.8 | - Proceeds from sales and repayments were driven by full repayments from CleanChoice Energy, Inc. and FGI Acquisition Corp[5](index=5&type=chunk) - Assets comprising three new and one existing portfolio companies, totaling **$22.8 million**, were transferred to STRS JV in exchange for cash[6](index=6&type=chunk) [Corporate Information](index=2&type=section&id=Corporate%20Information) This section provides WhiteHorse Finance, Inc.'s corporate overview, management details, conference call information, forward-looking statements, and contact details [About WhiteHorse Finance, Inc.](index=2&type=section&id=About%20WhiteHorse%20Finance%2C%20Inc.) WhiteHorse Finance, Inc. is a business development company (BDC) that originates and invests in loans to privately held, lower middle market companies, managed by H.I.G. WhiteHorse Advisers, LLC - WhiteHorse Finance is a business development company that originates and invests in loans to privately held, lower middle market companies[12](index=12&type=chunk) - Investment activities are managed by H.I.G. WhiteHorse Advisers, LLC, an affiliate of H.I.G. Capital, LLC[12](index=12&type=chunk) - H.I.G. Capital is a leading global alternative asset manager with **$70 billion** of capital under management[12](index=12&type=chunk) [Conference Call Information](index=2&type=section&id=Conference%20Call) WhiteHorse Finance will host a conference call on Thursday, August 7, 2025, at 2:00 p.m. ET to discuss its Q2 2025 results, with details for live access and replay options provided - A conference call to discuss Q2 2025 results will be held at **2:00 p.m. ET** on **Thursday, August 7, 2025**[10](index=10&type=chunk) - Access to the live teleconference is available via domestic (800-267-6316) or international (+1 203-518-9783) dial-in, referencing ID WHFQ225, or through the investor relations website[10](index=10&type=chunk) - A replay will be available approximately two hours after the call's completion through August 7, 2025, via teleconference (800-934-7879 or +1 402-220-6986) and webcast on the Company's website[11](index=11&type=chunk) [Forward-Looking Statements](index=3&type=section&id=Forward-Looking%20Statements) The press release contains forward-looking statements subject to risks and uncertainties, and actual results may differ materially, with the Company undertaking no duty to update these statements - This press release may contain 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995[13](index=13&type=chunk) - Actual results may differ materially from forward-looking statements due to various factors, including those described in SEC filings[13](index=13&type=chunk) - The Company undertakes no duty to update any forward-looking statement made herein[13](index=13&type=chunk) [Contacts](index=4&type=section&id=Contacts) Contact information for WhiteHorse Finance, Inc. (Stuart Aronson, Joyson Thomas) and investor relations (Robert Brinberg of Rose & Company) is provided for inquiries - Contact information is provided for Stuart Aronson (CEO), Joyson Thomas (WhiteHorse Finance, Inc.), and Robert Brinberg (Rose & Company) for investor inquiries[19](index=19&type=chunk) [Non-GAAP Financial Measures Definition](index=3&type=section&id=%281%29%20%22Core%20net%20investment%20income%22%20is%20a%20non-GAAP%20financial%20measure.) 'Core net investment income' is a non-GAAP financial measure that adjusts GAAP net investment income by excluding specific non-recurring costs, offering a clearer view of ongoing financial performance - 'Core net investment income' is a non-GAAP financial measure intended to provide useful information by reflecting financial performance excluding specific non-recurring or non-operational items[14](index=14&type=chunk)[17](index=17&type=chunk) - Adjustments include the net impact of costs associated with refinancing indebtedness, the accrual of capital gains incentive fees, and excise or other income taxes related to net realized gains and losses (net of incentive fees)[14](index=14&type=chunk)[17](index=17&type=chunk) - This non-GAAP measure is not meant to be considered in isolation or as a substitute for financial results prepared in accordance with GAAP[14](index=14&type=chunk)

[Executive Summary & Business Update](index=1&type=section&id=Executive%20Summary%20%26%20Business%20Update) Precision BioSciences reported Q2 2025 results, focusing on advancing PBGENE-HBV and PBGENE-DMD programs and extending cash runway to H2 2027 through expense reduction [Company Overview and Strategic Highlights](index=1&type=section&id=Company%20Overview%20and%20Strategic%20Highlights) Precision BioSciences, a clinical-stage gene editing company, focuses on advancing lead programs and extending its cash runway to H2 2027 through expense reduction - Focus on advancing clinical-stage PBGENE-HBV and PBGENE-DMD programs[3](index=3&type=chunk) - Extended expected cash runway to the **second half of 2027** through significant reduction in non-program related annual operating expenses[3](index=3&type=chunk)[5](index=5&type=chunk) - Actions expected to enable commencement of a Phase 2 study for PBGENE-HBV and a potential pivotal trial for PBGENE-DMD[3](index=3&type=chunk) [Pipeline Update](index=1&type=section&id=Pipeline%20Update) The company provided updates on wholly-owned and partnered gene editing programs, including PBGENE-HBV, PBGENE-DMD, iECURE-OTC, and Azer-Cel, while pausing PBGENE-3243 [Wholly Owned Portfolio](index=1&type=section&id=Wholly%20Owned%20Portfolio) Updates on wholly-owned programs, including PBGENE-HBV, PBGENE-DMD, and the paused PBGENE-3243 [PBGENE-HBV (Viral Elimination Program)](index=1&type=section&id=PBGENE-HBV%20(Viral%20Elimination%20Program)) PBGENE-HBV, a viral elimination program for chronic Hepatitis B, showed promising Phase 1 safety and antiviral activity, with Cohort 3 initiation recommended - PBGENE-HBV is a global first-in-human clinical trial designed to eliminate cccDNA and inactivate integrated HBV DNA for chronic Hepatitis B[4](index=4&type=chunk) - Phase 1 ELIMINATE-B trial Cohort 1 demonstrated substantial antiviral activity in all patients, with HBsAg reductions of **47-69%** and durable reduction in one patient **7 months post-dose**[5](index=5&type=chunk)[8](index=8&type=chunk) - PBGENE-HBV was well-tolerated in Cohort 1 with no Grade 2+ treatment-related adverse events, serious adverse events, or dose-limiting toxicity[7](index=7&type=chunk) - Initial safety data from Cohort 2 showed no adverse events above Grade 2, and the Data Monitoring Committee recommended initiation of Cohort 3[9](index=9&type=chunk) - Company expects to provide a data update later in 2025[10](index=10&type=chunk) [PBGENE-DMD (Muscle Targeted Excision Program)](index=2&type=section&id=PBGENE-DMD%20(Muscle%20Targeted%20Excision%20Program)) PBGENE-DMD, a muscle-targeted excision program for Duchenne Muscular Dystrophy, received FDA designations and showed significant preclinical functional improvement - PBGENE-DMD is designed to improve function for over **60% of DMD patients** by excising exons 45-55 of the dystrophin gene to restore near-full length functional dystrophin protein[11](index=11&type=chunk) - Granted FDA Rare Pediatric Disease Designation (June 2025) and Orphan Drug Designation (July 2025) for DMD[5](index=5&type=chunk)[12](index=12&type=chunk) - Preclinical data showed significant and durable functional improvement in a humanized DMD mouse model, restoring dystrophin protein expression across multiple muscle types[13](index=13&type=chunk)[14](index=14&type=chunk) - IND/CTA filing targeted by end of 2025, with initial clinical data expected in 2026[5](index=5&type=chunk)[15](index=15&type=chunk) [PBGENE-3243 (Mutant Mitochondrial DNA Elimination Program)](index=3&type=section&id=PBGENE-3243%20(Mutant%20Mitochondrial%20DNA%20Elimination%20Program)) Development of PBGENE-3243, a mutant mitochondrial DNA elimination program, has been paused to prioritize lead programs - Development of PBGENE-3243 paused to prioritize PBGENE-HBV and PBGENE-DMD[16](index=16&type=chunk) [Partnered In Vivo Gene Editing Programs](index=3&type=section&id=Partnered%20In%20Vivo%20Gene%20Editing%20Programs) Updates on partnered in vivo gene editing programs, including iECURE-OTC for OTC deficiency and PBGENE-NVS for hemoglobinopathies [iECURE-OTC (Gene Insertion Program)](index=3&type=section&id=iECURE-OTC%20(Gene%20Insertion%20Program)) iECURE-OTC, a gene insertion program for neonatal onset OTC deficiency, demonstrated a complete clinical response in a Phase 1/2 trial - ECUR-506 demonstrated a complete clinical response from three months post-exposure to six months in a Phase 1/2 trial for neonatal onset OTC deficiency[17](index=17&type=chunk) - Second infant with severe OTC deficiency dosed in H1 2025[17](index=17&type=chunk) - iECURE expects to complete enrollment in 2025 and anticipates complete data in H1 2026[18](index=18&type=chunk) [PBGENE-NVS (Gene Insertion Program)](index=3&type=section&id=PBGENE-NVS%20(Gene%20Insertion%20Program)) PBGENE-NVS is a collaboration with Novartis to develop an ARCUS nuclease for in vivo gene insertion to treat hemoglobinopathies - Collaboration with Novartis to develop a custom ARCUS nuclease for in vivo gene insertion to treat hemoglobinopathies[19](index=19&type=chunk) [Non-Core Ex Vivo Programs](index=3&type=section&id=Non-Core%20Ex%20Vivo%20Programs) Update on non-core ex vivo program Azer-Cel, an allogeneic CAR T treatment for cancer, showing promising response rates [Azer-Cel (Allogeneic CAR T treatment for cancer)](index=3&type=section&id=Azer-Cel%20(Allogeneic%20CAR%20T%20treatment%20for%20cancer)) Azer-Cel, an allogeneic CAR T treatment, achieved a 75% overall response rate and 55% complete response rate in DLBCL patients - Azer-Cel achieved a **75% best overall response rate** and **55% complete response rate** in relapsed/refractory DLBCL patients[20](index=20&type=chunk) - Imugene expects to request an end of Phase 1 meeting with the FDA in Q4 2025 to discuss pivotal trial designs[20](index=20&type=chunk) [Other Corporate Announcements](index=3&type=section&id=Other%20Corporate%20Announcements) Dr. Mark Sulkowski was appointed Head Clinical Development Advisor to support clinical strategy for PBGENE-HBV and later-stage trials - Dr. Mark Sulkowski appointed Head Clinical Development Advisor to support clinical strategy for PBGENE-HBV and later-stage trials[21](index=21&type=chunk) [Financial Results for Second Quarter 2025](index=4&type=section&id=Financial%20Results%20for%20Second%20Quarter%202025) This section details Precision BioSciences' financial performance for Q2 2025, including operating expenses, revenue, and net loss, alongside an updated cash runway [Financial Summary and Cash Runway](index=4&type=section&id=Financial%20Summary%20and%20Cash%20Runway) The company reduced Q2 operating expenses by $3.9 million and initiated an efficiency program to cut annual cash operating expenses by $25 million, extending its cash runway to H2 2027 - Total operating expenses decreased by **$3.9 million** in Q2 2025 compared to Q2 2024[22](index=22&type=chunk) - Initiated an operating efficiency program to reduce annual cash operating expenses by approximately **$25 million** in 2026 and 2027 compared to 2025 levels[22](index=22&type=chunk) - Expected cash runway extended to the **second half of 2027**[5](index=5&type=chunk)[22](index=22&type=chunk) Cash, Cash Equivalents, and Restricted Cash | As of | Amount (in millions) | | :---- | :------------------- | | June 30, 2025 | $84.8 | | December 31, 2024 | $108.5 | [Detailed Financial Performance](index=4&type=section&id=Detailed%20Financial%20Performance) Detailed financial performance shows a significant revenue decrease due to prior non-cash recognition, reduced R&D expenses, slightly increased G&A, and a net loss for Q2 2025 [Revenues](index=4&type=section&id=Revenues) Total revenues for Q2 2025 significantly decreased compared to Q2 2024, primarily due to a non-cash deferred revenue recognition in the prior period Total Revenues (QoQ) | Period | Revenue (in millions) | Change | | :----- | :-------------------- | :----- | | Q2 2025 | < $0.1 | -$49.8M | | Q2 2024 | $49.9 | | - Decrease primarily due to **$48.2 million** of non-cash deferred revenue recognized in Q2 2024 related to the Prevail Therapeutics Agreement[23](index=23&type=chunk) [Operating Expenses](index=4&type=section&id=Operating%20Expenses) Operating expenses saw a decrease in R&D due to program adjustments and pauses, while general and administrative expenses slightly increased Research and Development Expenses (QoQ) | Period | R&D Expenses (in millions) | Change | | :----- | :------------------------- | :----- | | Q2 2025 | $12.8 | -$4.4M | | Q2 2024 | $17.2 | | - Decrease in R&D primarily due to lower manufacturing/toxicology expenses for PBGENE-HBV and decreased expenses for paused PBGENE-3243, offset by increased PBGENE-DMD expenses[24](index=24&type=chunk) General and Administrative Expenses (QoQ) | Period | G&A Expenses (in millions) | Change | | :----- | :------------------------- | :----- | | Q2 2025 | $9.1 | +$0.6M | | Q2 2024 | $8.5 | | - Increase in G&A due to higher employee-related costs, including non-cash costs[25](index=25&type=chunk) [Net Loss and EPS](index=4&type=section&id=Net%20Loss%20and%20EPS) The company reported a net loss and negative EPS for Q2 2025, a shift from net income in the prior year Net Income/Loss and EPS (QoQ) | Period | Net (Loss) Income (in millions) | EPS (Basic) | | :----- | :------------------------------ | :---------- | | Q2 2025 | ($23.5) | ($2.13) | | Q2 2024 | $32.7 | $4.70 | [Company Information](index=4&type=section&id=Company%20Information) This section provides an overview of Precision BioSciences as a clinical-stage gene editing company and details its proprietary ARCUS® genome editing platform [About Precision BioSciences](index=4&type=section&id=About%20Precision%20BioSciences) Precision BioSciences is a clinical-stage gene editing company developing in vivo gene editing therapies using its proprietary ARCUS® platform - Precision BioSciences is a clinical-stage gene editing company focused on in vivo gene editing therapies[27](index=27&type=chunk) - Utilizes its novel and proprietary ARCUS® genome editing platform[27](index=27&type=chunk) [ARCUS® Platform](index=6&type=section&id=ARCUS%C2%AE%20Platform) The ARCUS® platform facilitates advanced gene edits, including insertion, elimination of viral or mutant mitochondrial DNA, and excision of defective gene portions - ARCUS® platform supports gene insertion, elimination (viral DNA, mutant mitochondrial DNA), and excision (large defective gene portions)[28](index=28&type=chunk) [Forward-Looking Statements](index=6&type=section&id=Forward-Looking%20Statements) This section outlines forward-looking statements regarding future expectations, plans, and potential outcomes, acknowledging inherent risks and uncertainties [Forward-Looking Statements](index=6&type=section&id=Forward-Looking%20Statements) This section details forward-looking statements concerning program development, regulatory processes, financial projections, and collaborations, emphasizing inherent risks and uncertainties - Statements cover expectations for ARCUS advantages, program development (PBGENE-HBV, PBGENE-DMD), regulatory timelines, cash runway, and partner activities[29](index=29&type=chunk) - Forward-looking statements are subject to known and unknown risks, uncertainties, and assumptions, including funding, operating expenses, program success, regulatory approvals, and competition[30](index=30&type=chunk)[31](index=31&type=chunk) [Financial Statements](index=8&type=section&id=Financial%20Statements) This section presents the condensed financial statements, including statements of operations and balance sheet data, for the specified periods [Statements of Operations](index=8&type=section&id=Statements%20of%20Operations) Condensed statements of operations for Q2 2025 and 2024 are presented, detailing revenues, operating expenses, and net income or loss Statements of Operations (Three Months Ended June 30) | (In thousands, except share and per share amounts) | 2025 | 2024 | | :------------------------------------------------ | :--- | :--- | | Revenue | $18 | $49,898 | | Research and development | 12,768 | 17,225 | | General and administrative | 9,127 | 8,527 | | Total operating expenses | 21,895 | 25,752 | | Operating (loss) income | (21,877) | 24,146 | | Total other (expense) income | (1,643) | 8,603 | | Net (loss) income | $(23,520) | $32,749 | | Net (loss) income per share - Basic | $(2.13) | $4.70 | | Net (loss) income per share - Diluted | $(2.13) | $4.67 | | Weighted-average shares of common stock outstanding - Basic | 11,046,401 | 6,966,680 | | Weighted-average shares of common stock outstanding - Diluted | 11,046,401 | 7,011,630 | [Balance Sheets Data](index=9&type=section&id=Balance%20Sheets%20Data) Key balance sheet data as of June 30, 2025, and December 31, 2024, including cash, working capital, assets, liabilities, and equity, is provided Balance Sheets Data (As of) | (In thousands, except share amounts) | June 30, 2025 | December 31, 2024 | | :---------------------------------- | :------------ | :---------------- | | Cash, cash equivalents, and restricted cash | $84,806 | $108,468 | | Working capital | 56,691 | 80,009 | | Total assets | 108,928 | 136,388 | | Total liabilities | 74,874 | 79,995 | | Total stockholders' equity | $34,054 | $56,393 | | Common stock outstanding | 11,636,981 | 8,202,715 |

Exhibit 99.1 "Looking ahead to the balance of the year, assuming a normalized retail margin environment, and further improvement and growth at GREW led by Diversegy and Genie Solar, we continue to expect that Genie will generate $40 to $50 million of consolidated Adjusted EBITDA in 2025." Second Quarter 2025 Highlights (Unless otherwise noted, 2Q25 results are compared to 2Q24, and results of the former Genie Retail Energy International (GREI) segment are included in discontinued operations for all periods. ...