Triheptanoin

Second quarter total revenue of $214 million, Crysvita® revenue of $156 million and Dojolvi® revenue of $27 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and combined R&D and SG&A expenses to be flat to slightly down versus 2025; remain on path to profitability in 2027 Catalysts in second half of 2026 include two PDUFA dates and pivotal data readout from GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif., Aug. 04, 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 ended June 30, 2026 and reaffirmed its financial guidance for 2026. “In the second quarter we generated the highest quarterly revenue in the history of the company, supporting our full-year revenue guidance, and keeping us on track toward profitability in 2027,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “As we look to the second half of the year, we are entering a transformative period with multiple important catalysts. We are ready to launch two gene therapy products and are preparing for a pivotal Phase 3 GTX-102 data readout in Angelman syndrome. This puts us in position to broaden our patient impact with sustained growth in the years to come.” Second Quarter 2026 Revenue Highlights and 2026 Revenue Guidance Total revenue in the second quarter of 2026 was $214 million. The company reaffirms its full year 2026 total revenue guidance of $730 million to $760 million, which excludes revenue from potential new product launches. Crysvita revenue in the second quarter of 2026 was $156 million, consistent with expected seasonality in the U.S. and Canada and ordering patterns in Latin America. The company reaffirms its full year 2026 Crysvita revenue guidance of $500 million to $520 million. Dojolvi revenue in the second quarter 2026 was $27 million. The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million. Evkeeza® revenue in the second quarter 2026 was $21 million, driven by increased demand from new country launches and early access. Mepsevii® revenue in the second quarter 2026 was $10 million. Milestones and Upcoming Catalysts Dojolvi for the treatment of LC-FAOD: In May 2026, Dojolvi was listed on the National Health Insurance (NHI) drug price list and was launched in Japan following the receipt of manufacturing and marketing approval under the Conditional Approval System for Pharmaceuticals on March 23, 2026. DTX401 (pariglasgene brecaparvovec) AAV8 gene therapy for the treatment of glycogen storage disease type Ia (GSDIa): In February 2026, the U.S. Food and Drug Administration (FDA) accepted for review the Biologics License Application (BLA) seeking approval of DTX401 as a treatment for GSDIa and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026. UX111 (rebisufligene etisparvovec) AAV9 gene therapy for the treatment of Sanfilippo syndrome type A (MPS IIIA): In April 2026, the FDA accepted for review the resubmitted BLA seeking accelerated approval for UX111 as a treatment for MPS IIIA and assigned a PDUFA action date of September 19, 2026. GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): The Phase 3 Aspire study, in patients with a full maternal UBE3A gene deletion, enrolled 129 patients, randomized 1:1 to GTX-102 or sham. Data from this study are expected in the September or October timeframe. Enrollment in the open-label Phase 2/3 Aurora study, evaluating GTX-102 in other genotypes and ages, began in October 2025 and is expected to complete in the second half of 2026. UX701 (rivunatpagene miziparvovec) AAV9 gene therapy for the treatment of 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 the fourth quarter of 2026. UX016 novel prodrug for sialic acid used as a substrate replacement therapy for the treatment of GNE myopathy: The FDA cleared the Investigational New Drug (IND) application for UX016 and an externally funded Phase 1/2 study is expected to begin in the second half of 2026. DTX301 (avalotcagene ontaparvovec) AAV8 gene therapy for the treatment of Ornithine Transcarbamylase, or OTC, deficiency: The Phase 3 Enh3ance study continues with patients in both treatment and cross-over groups progressing through 64 weeks of follow-up. Data from the second primary endpoint, which evaluates reduction in treatment burden, including use of ammonia scavengers and dietary management, are expected in the first half of 2027. Summary of Second Quarter 2026 Financial Results Selected Financial Data (dollars in millions, except per share amounts), (unaudited) Operating Expenses Total operating expenses for the second quarter 2026 were $289 million, including $34 million of non-cash stock-based compensation. The company reaffirms its full year 2026 and 2027 guidance for combined R&D and SG&A operating expenses: compared to 2025, combined R&D and SG&A expenses in 2026 are expected to be flat to down low-single digits, and combined R&D and SG&A expenses in 2027 are expected to decrease by at least 15%. Net Loss Net loss for the second quarter 2026 was $92 million, or $0.90 per share basic and diluted, compared with a net loss for the second quarter 2025 of $115 million, or $1.17 per share basic and diluted. Cash Balance and Net Cash Used in Operations Cash, cash equivalents, and marketable securities were $436 million as of June 30, 2026. For the three months ended June 30, 2026, net cash used in operations was $97 million. Conference Call and Webcast Information Ultragenyx will host a conference call today, Tuesday, August 4, 2026, at 2 p.m. PT/5 p.m. ET to discuss the second quarter financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at https://ir.ultragenyx.com/events-presentations. 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: www.ultragenyx.com. 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 regarding Ultragenyx’s expectations and projections concerning its future operating results and financial performance, including its 2026 revenue guidance for total revenue, Crysvita and Dojolvi, its anticipated R&D and SG&A expenses in 2026 and 2027, anticipated benefits and savings from its strategic restructuring plan, and the timing and sustainability of profitability; the timing, progress, results and plans for its clinical programs and studies, including the anticipated timing and outcome of the Phase 3 Aspire study of GTX-102, enrollment in the Aurora study, data from the UX701 and DTX301 studies, and initiation of the UX016 study; the FDA’s review of the BLAs for DTX401 and UX111, including the anticipated PDUFA action dates, the potential approval of either product candidate, whether the FDA may require additional information, studies or manufacturing changes, and the timing and outcome of regulatory inspections; Ultragenyx’s manufacturing and commercial readiness and the timing and success of any potential launches of DTX401 and UX111, if approved; and the potential patient impact, commercial opportunity and growth associated with Ultragenyx’s products and 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 May 6, 2026, 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 (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/). Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jess Rowlands media@ultragenyx.com

从“自主制造的王冠”到“合规风暴的中心”。 在罕见病和超罕见病的新药研发领域，基因治疗被视作许多绝症患者“一生一次”的最后希望。我们这次解读的就是一家专注于罕见病领域的明星生物制药公司——Ultragenyx Pharmaceutical Inc.在2025年接受FDA检查的483。通过回顾这间原本被寄予厚望的基因治疗工厂在2025年的经历，希望能为整个行业的生产与质量管理带来一些深刻的启示。 Ultragenyx成立于2010年，一直致力于为全球存在极大未满足医疗需求的罕见病患者提供创新疗法（其获批的传统产品如 Dojolvi 和 Mepsevii 已在全球多地区进行商业化销售）。为了解决其高度依赖外部合同定制生产机构（CDMO），导致产能受限且质量难以实现绝对把控的痛点，Ultragenyx斥巨资在距离大波士顿研发中心不远的Bedford地区，买下了一块10.7英亩的土地，建立起一座占地超11万平方英尺的先进基因治疗制造工厂，并于2023年6月正式落成启用，定位为药品制造商，同时具备药物原液和药物制剂的端到端生产能力。 随着 Ultragenyx 的核心在研 AAV9 基因疗法 UX111（用于治疗A型圣菲利普综合征）进入上市冲刺阶段，2025年4月21日至4月25日期间，FDA对该设施进行了批准前检查，最终出具了6条缺陷的483报告，覆盖数据完整性、设施维护、偏差管理、质量监督、无菌工艺、污染控制六大领域，最终导致2025年7月11日，FDA 针对 UX111 的生物制品许可申请（BLA）正式下发了完整回复函（CRL），在函件中明确承认该疗法在临床神经发育结果和生物标志物数据上的显著有效性，但由于化学、制造和控制问题，特别是受到刚刚结束的 Bedford 工厂制造设施检查结果的拖累，FDA拒绝了该药物的上市批准。 对于基因治疗公司而言，产品上市的延期往往意味着资金链的极度承压。由于获批时间被迫推迟至2026年，Ultragenyx 的财务状况受到了实质性影响。为了削减开支并在漫长的整改期内存活下来，公司不得不在2026年2月宣布了一项痛苦的重组计划：裁减全球约10%的员工（约130人），以将有限的资源集中于应对 FDA 的整改要求和维持高价值项目。好在，经过大半年的痛定思痛、彻查数据漏洞以及全面提升 Bedford 工厂的质量管理体系，公司完成了对 CMC 和检查缺陷的整改。2026年4月，FDA 终于重新接受了其里程碑式基因疗法的 BLA 申请，并将最终审批日期（PDUFA）定档在2026年8月23日。 Ultragenyx Bedford 工厂的这段经历，告诉每一位从业者，仅仅拥有顶尖的科学技术是远远不够的——复杂而严苛的生产合规与质量控制，往往才是决定一款救命药能否真正走向市场的“胜负手”。 设施名称：Ultragenyx Pharmaceutical Inc 地址：170 Middlesex Turnpike, Bedford, MA 01730 USA FEI：3025268614 设施类型：药品制造商 检查时间：04/21/2025-04/25/2025 检察官：Massoud Motamed, CSO, Lead Inspector Kula; N. Jha, Biological Reviewer Jacob; L. Bitterman, Biological Reviewer Timothy Kamaldinov, Biological Reviewer 01           通过受控文件管理来确保数据完整性的系统存在缺陷。 具体如下： …… 欲知详情，请加入海螺企业VIP 具体操作流程参考： 【新VIP会员招募】 添加小编vx：gmpclub08，注明：VIP