Triheptanoin

2025 total revenue of $673 million, Crysvita ® revenue of $481 million and Dojolvi ® revenue of $96 million 2026 total revenue from current products expected to be between $730 million to $760 million Initiated a strategic restructuring plan to significantly reduce and focus expenses and headcount, reiterate path to profitability in 2027 2026 catalysts include two potential approvals and expected pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif., Feb. 12, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter and year ended December 31, 2025. “The year ahead marks an important turning point for the company, as we approach two potential product launches and a pivotal data readout that, together, could significantly accelerate our commercial revenue trajectory,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “We are implementing a strategic restructuring plan to reduce our operating expenses and ensure our resources are squarely aligned with our highest-impact opportunities, while leading the future of rare disease with multiple first ever treatments.” Fourth Quarter 2025 Selected Financial Data Tables and Financial Results Revenues (dollars in millions), (unaudited) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Crysvita Product sales - Latin America and Türkiye $ 40 $ 22 $ 177 $ 135 Royalty revenue - U.S. and Canada 97 87 275 249 Royalty revenue - Europe 8 7 29 26 Total Crysvita Revenue 145 116 481 410 Dojolvi 32 31 96 88 Evkeeza 17 10 59 32 Mepsevii 13 8 37 30 Total revenues $ 207 $ 165 $ 673 $ 560 Revenues Ultragenyx reported $207 million in total revenue for the fourth quarter 2025, which represents 25% growth compared to the same period in 2024. Crysvita revenue in the fourth quarter 2025 was $145 million, which includes product sales of $40 million from Latin America and Türkiye. Dojolvi revenue in the fourth quarter 2025 was $32 million. Evkeeza revenue in the fourth quarter 2025 was $17 million. Total revenue for the year ended December 31, 2025 was $673 million, which represents 20% growth compared to the prior year. Full year 2025 Crysvita revenue was $481 million, which represents 17% growth compared to the prior year. This includes product sales of $177 million from Latin America and Türkiye, which represents 31% growth compared to the prior year. Dojolvi revenue in 2025 was $96 million, which represents 9% growth compared to the prior year. Evkeeza revenue in 2025 was $59 million, which represents 84% growth compared to the prior year, as demand continues to build following launches in the company’s territories outside of the United States. Selected Financial Data (dollars in millions, except per share amounts), (unaudited) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Total revenues $ 207 $ 165 $ 673 $ 560 Operating expenses: Cost of sales 29 17 109 77 Research and development 203 188 750 698 Selling, general and administrative 89 82 349 321 Total operating expenses 321 287 1,208 1,096 Net loss $ (129 ) $ (133 ) $ (575 ) $ (569 ) Net loss per share, basic and diluted $ (1.29 ) $ (1.39 ) $ (5.83 ) $ (6.29 ) Operating Expenses Total operating expenses for the fourth quarter 2025 were $321 million. Total operating expenses for the year ended December 31, 2025 were $1.2 billion, including $153 million of non-cash stock-based compensation. Net Loss Net loss for the fourth quarter 2025 was $129 million, or $1.29 per share basic and diluted, compared with a net loss for the fourth quarter 2024 of $133 million, or $1.39 per share basic and diluted. Net loss for the year ended December 31, 2025 was $575 million, or $5.83 per share basic and diluted, compared with a net loss the prior year of $569 million, or $6.29 per share, basic and diluted. Cash Balance and Net Cash Used in Operations Cash, cash equivalents, and marketable securities were $737 million as of December 31, 2025. For the three months ended December 31, 2025, net cash used in operations was $100 million and for the year ended December 31, 2025 was $466 million. Financial Guidance and Strategic Restructuring Plan 2026 revenue guidance Total revenues, excluding potential revenue from new product launches, in the range of $730 million to $760 million, an increase of 8% to 13% compared to 2025 Crysvita revenue in the range of $500 million to $520 million, reflecting growing underlying global demand partially offset by expected timing of ordering patterns in Brazil Dojolvi revenue in the range of $100 million to $110 million Strategic restructuring plan and path to profitability in 2027 Ultragenyx has initiated a strategic restructuring plan designed to reduce its headcount and expenses and focus resources on its largest value drivers. The significant reduction and partial reinvestment of expenses, and the planned growth in revenue from current and new product launches, are designed to keep the company on its path to profitability in 2027. Today, in connection with the restructuring plan, the company announced a 10% workforce reduction, impacting approximately 130 employees. Based on the progression of the business and the reductions from the restructuring plan: In 2026, combined R&D and SG&A expenses are expected to be flat to down low-single digits versus 2025. This includes the impact of spend reductions and approximately $50 million for severance, manufacturing, and other non-recurring restructuring charges. In 2027, R&D expenses are expected to decrease from 2025 levels by 38%, or approximately $280 million, driven by the completion of multiple phase 3 studies and reduction of early-stage research efforts. SG&A expenses are expected to increase in support of new product launches and existing approved products. On a combined basis, 2027 R&D and SG&A expenses are expected to decrease at least 15% versus 2025. 2026 Clinical and Regulatory Catalysts DTX401 (pariglasgene brecaparvovec) AAV8 gene therapy for glycogen storage disease type Ia (GSDIa): Biologics License Application (BLA) rolling submission completed in December 2025, with an anticipated Prescription Drug User Fee Act (PDUFA) action date in the third quarter of 2026. UX111 (rebisufligene etisparvovec) AAV9 gene therapy for Sanfilippo syndrome type A (MPS IIIA): The BLA was resubmitted in January 2026 and included substantial longer-term data, that were recently presented at the 2026 WORLD Symposium , on multiple measures of neurologic benefit to support an intermediate clinical endpoint for accelerated approval supported further by CSF heparan sulfate and other biomarker data, as agreed with the FDA during the last clinical review. Earlier today the company received an Incomplete Response Letter (IRL) regarding its resubmitted BLA. The IRL requests additional supportive documentation related to its CRL CMC responses, which the company will provide in a resubmission. GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): Data from the fully enrolled, pivotal, Phase 3 Aspire study in patients with a genetically confirmed diagnosis of UBE3A deletion is expected in the second half of 2026. Enrollment in the Phase 2/3 Aurora study is also underway in other genotypes and ages, with the first patient dosed in October 2025. UX701 (rivunatpagene miziparvovec) AAV9 gene therapy for Wilson disease: Enrollment is complete for the fourth cohort in the ongoing, dose-finding stage of the pivotal Cyprus2+ study. Data from this stage are expected in 2026. Conference Call and Webcast Information Ultragenyx will host a conference call today, Thursday, February 12, 2026, at 2 p.m. PT/5 p.m. ET to discuss the fourth quarter and full year 2025 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at . The replay of the call will be available for three months. About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: . Forward-Looking Statements and Use of Digital Media Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, including the company’s expectations for profitability in 2027, anticipated cost or expense reductions, including the company’s expectations related to benefits and savings from the strategic restructuring plan, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, the components and timing of regulatory submissions, the company’s ability to provide the requested documentation and address the comments in the CRL for UX111 to the satisfaction of the FDA, the timing of FDA review of the company’s BLA submissions, the timing and outcome of any FDA inspections related to UX111 or other clinical product candidates, the timing of future regulatory interactions related to the company’s clinical product candidates are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercialization of Crysvita in certain major markets, including the U.S. and Canada, and for our commercial supply of Crysvita in those markets, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, , smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, our ability to successfully manage the expansion of our company, delays or unexpected costs and other adverse effects related to the strategic restructuring plan, competition from other therapies or products, regulatory scrutiny of the company’s products and product candidates, the company’s limited experience as a company in operating its own manufacturing facility, market acceptance of our products, uncertainty related to insurance coverage and reimbursement, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidate. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 5, 2025, and its subsequent periodic reports filed with the SEC. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website ( ) and LinkedIn website ( ). Ultragenyx Pharmaceutical Inc. Selected Statement of Operations Financial Data (in millions, except per share amounts) (unaudited) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Statement of Operations Data: Revenues: Product sales $ 102 $ 72 $ 369 $ 285 Royalty revenue 105 $ 93 304 275 Total revenues 207 165 673 560 Operating expenses: Cost of sales 29 17 109 77 Research and development 203 188 750 698 Selling, general and administrative 89 82 349 321 Total operating expenses 321 287 1,208 1,096 Loss from operations (114 ) (122 ) (535 ) (536 ) Non-cash interest expense on liabilities for sales of future royalties (19 ) (15 ) (62 ) (63 ) Other income, net 5 4 26 32 Loss before income taxes (128 ) (133 ) (571 ) (567 ) Provision for income taxes (1 ) — (4 ) (2 ) Net loss $ (129 ) $ (133 ) $ (575 ) $ (569 ) Net loss per share, basic and diluted $ (1.29 ) $ (1.39 ) $ (5.83 ) $ (6.29 ) Shares used in computing net loss per share, basic and diluted 99.9 95.7 98.6 90.5 Ultragenyx Pharmaceutical Inc. Selected Activity included in Operating Expenses (in millions) (unaudited) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Non-cash stock based compensation $ 38 $ 40 $ 153 $ 158 GTX-102 clinical milestone — $ 30 — $ 30 Ultragenyx Pharmaceutical Inc. Selected Balance Sheet Financial Data (in millions) (unaudited) December 31, December 31, 2025 2024 Balance Sheet Data: Cash, cash equivalents, and marketable securities $ 737 $ 745 Working capital 567 473 Total assets 1,532 1,503 Total stockholders' equity (deficit) (80 ) 255 Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jess Rowlands media@ultragenyx.com

香港交易及結算所有限公司及香港聯合交易所有限公司對本公告的內容概不負責，對其準確性 或完整性亦不發表任何聲明，並明確表示，概不對因本公告全部或任何部分內容而產生或因倚 賴該等內容而引致的任何損失承擔任何責任。 EDDING GENOR GROUP HOLDINGS LIMITED 億騰嘉和醫藥集團有限公司 （前稱嘉和生物藥業（開曼）控股有限公司） （於開曼群島註冊成立的有限公司） （股份代號：6998） EDP167 II期臨床試驗完成首例受試者給藥 本公告由億騰嘉和醫藥集團有限公司（「本公司」，連同其附屬公司統稱「本集團」） 自願作出，以告知本公司股東（「股東」）及潛在投資者有關本集團最新業務更新。 本公司董事（「董事」）會（「董事會」）欣然宣佈，旗下小核酸創新藥EDP167 II期臨 床試驗成功完成首例受試者給藥。 EDP167 II期臨床試驗是一項針對純合子家族性高膽固醇血症（「HoFH」）成人患者 的多中心、劑量探索、開放標籤的試驗，旨在評估EDP167在HoFH患者中的療效 和安全性，其主要終點為首次給藥24周後低密度脂蛋白膽固醇（「LDL-C」）水平相 較基線的變化，預計於2026年第四季度完成主要終點的評估。 關於EDP167 EDP167是一款創新性siRNA療法，專為治療血脂異常而設，其開發乃為配合 本公司聚焦於心血管疾病的戰略重點。該藥物靶向肝臟血管生成素樣蛋白3 （「ANGPTL3」），ANGPTL3由肝臟分泌，可同時抑制脂蛋白脂酶和內皮脂酶，從 而調控多種致動脈粥樣硬化脂蛋白的代謝。基因研究證實，ANGPTL3功能缺失突 變的人群，其患動脈粥樣硬化性心血管疾病的風險明顯降低。 EDP167透過精準且持續地抑制肝臟中ANGPTL3的表達，可實現對LDL-C和三酸 甘油酯（「TG」）水平的雙重強力降低。尤為重要的是，其作用機制獨立於LDL受 體（「LDLR」）途徑，突破了傳統降脂藥物（包括他汀和PCSK9抑制劑）依賴LDLR 功能的局限性。EDP167的獨特特性為血脂異常患者（特別是HoFH或混合型血脂 異常患者）提供了重要的創新治療選擇，展現廣闊的臨床應用前景。 在啟動針對HoFH患者的II期臨床試驗前，本公司已完成一項在健康及輕度血脂異 常人群中進行的隨機、雙盲、安慰劑對照、單次劑量遞增的I期臨床研究。該研究 系統評估了不同劑量下EDP167的安全性、耐受性、藥代動力學和藥效學特徵。結 果顯示EDP167展現出良好的安全性及耐受性特徵。相關數據將於2026年舉行的 醫學會議中披露。 關於本公司 本公司是一家綜合型的專業生物製藥公司，專注腫瘤學、自體免疫疾病、心血管 疾病、呼吸系統疾病及抗感染治療領域。通過向跨國醫藥公司（「跨國公司」）收購 品牌藥物資產，以及從全球生物製藥公司授權引進創新專利藥物的開發及商業化 權利，本公司已建立富競爭力的原研藥及創新藥組合。本公司已成功完成多款創 新藥在中國上市，彰顯其強大的臨床開發及管理能力。此外，本公司於其過往資 產收購中，獲跨國公司轉讓生產設施及管理體系，通過營運該等生產設施及管理 體系，本公司展示了高品質的製造、供應鏈管理、技術轉移及質量控制體系。 承董事會命 億騰嘉和醫藥集團有限公司 主席兼執行董事 倪昕先生 香港，2026年2月9日 於本公告日期，董事會成員包括執行董事倪昕先生及翟婧女士；非執行董事David Guowei Wang博士及于鐵銘先生；及獨立非執行董事許慶博士、陳文先生及鄭晶 晶女士。