财务数据和关键指标变化 - 2022年公司全球收入超过3.5亿美元 预计2023年收入将在4.2亿至4.5亿美元之间 [4] - 2022年公司净亏损7.07亿美元 每股亏损10.12美元 年末现金及等价物和可交易证券约为9亿美元 [34] - Crysvita在Ultragenyx区域的收入为2.58亿美元 其中北美利润分享区域为2.15亿美元 其他区域净产品销售额为4300万美元 [16] - Dojolvi 2022年收入为5600万美元 Mepsevii收入为2100万美元 [58] 各条业务线数据和关键指标变化 - Crysvita在2022年实现了35%的同比增长 全球销售额超过10亿美元 [53] - Dojolvi在2022年完成了约425份启动表格 约有375名患者接受报销治疗 处方医生数量增长至约190名 [14] - Mepsevii在2022年实现了29%的增长 [53] - Evkeeza在美国以外地区获得商业化许可 预计将推动全球业务增长 [5] 各个市场数据和关键指标变化 - 北美Crysvita在第四季度新增约110份启动表格 约90名患者接受报销治疗 新增50名处方医生 [12] - 拉丁美洲Crysvita持续增长 预计2023年收入将在3.25亿至3.4亿美元之间 同比增长16%至22% [31] - 巴西Dojolvi已获批准 正在争取财政部的全额报销批准 [32] 公司战略和发展方向和行业竞争 - 公司完成了基因治疗制造工厂的建设 预计今年春季开始生产 这将增强对基因治疗生产时间、成本和规模的控制 [6][7] - 公司收购了UX111和GeneTx 进一步巩固了在基因治疗领域的地位 [7][8] - 公司将继续专注于运营效率 积极管理费用和人员 以推动商业发布的价值并执行最重要的临床项目 [10] 管理层对经营环境和未来前景的评论 - 公司预计现有产品仍处于商业化的早期阶段 收入将保持稳定增长 [4] - 公司对2023年的前景持乐观态度 预计收入将增长20%以上 [15] - 公司预计2023年现金净使用量将显著减少 随着收入增长和运营效率提升 未来几年将实现盈利 [59] 其他重要信息 - 公司完成了UX143的关键II/III期研究 并启动了UX701的剂量探索研究 [9][27] - 公司预计将在2023年上半年与FDA会面 讨论基于生物标志物终点的加速批准计划 [36] - 公司计划在2023年中期分享UX143的II期数据 并过渡到III期研究 [35] 问答环节所有的提问和回答 问题: 关于成骨不全症的研究进展 - 公司正在比较不同剂量在儿童和成人中的效果 预计将在2023年中期分享数据 [40][41] 问题: 关于与Enozyme的合作 - 公司目前没有计划将更多产品引入其产品组合 但将继续与其他公司合作寻找患者 [44] 问题: 关于GTX-102的剂量选择 - 公司尚未公布具体剂量 但已确定了一些具有适当效益风险的剂量 将在未来公布更多信息 [66] 问题: 关于UX111的竞争格局 - 目前市场上没有其他有效的AAV基因治疗 公司认为UX111是领先项目 [75] 问题: 关于Evkeeza的反馈 - 医生对Evkeeza的反馈非常积极 预计在欧洲将有强劲增长 [77] 问题: 关于UX053的数据预期 - 公司将主要关注生物标志物和葡萄糖控制的变化 预计将在2023年上半年获得数据 [78] 问题: 关于GTX-102的临床活动 - 公司继续看到GTX-102在患者中的临床活动 预计将在今年晚些时候提供更多数据 [89] 问题: 关于Crysvita的季节性影响 - Q4和Q1通常会有一些季节性影响 但公司对潜在需求保持信心 [112] 问题: 关于Sanfilippo综合征的FDA会议 - 公司计划与FDA会面 讨论加速批准的可能性 预计将在2023年上半年进行 [108]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-K ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 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 No. 001-36276 Ultragenyx Pharmaceutical Inc. (Exact name of registrant as specified in its charter) Delaware 27-2546083 (State or other jurisdiction of (I. ...

400 0 1 Cash used in operations, Gene Therapy Manufacturing Facility (GTMF) Capital Expenses and select non-recurring uses of cash 2 2021: ~$55M GTMF; 2022: ~$90M GTMF, $75M GeneTx Acquisition, $30M Evkeeza License; 2023: ~$20M GTMF 3 Estimated cash, cash equivalents, and available-for-sale investments as of December 31, 2022 (unaudited) 9 Confidential and Proprietary Abnormal bone metabolism leads to increased bone resorption, inadequate bone production | --- | --- | --- | --- | --- | --- | --- | |-------- ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q3 2022 Earnings Conference Call November 2, 2022 5:00 PM ET Company Participants Joshua Higa - Head of Investor Relations Emil Kakkis - Founder, President and Chief Executive Officer Erik Harris - Executive Vice President and Chief Commercial Officer Mardi Dier - Executive Vice President and Chief Financial Officer Camille Bedrosian - Executive Vice President and Chief Medical Officer Conference Call Participants Gena Wang - Barclays Joori Park - SVB Securities ...

Corporate Deck July 2022 Legal Warning Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations and projections regarding our future operating results and financial performance, anticipated cost or expense reductions, plans with respect to commercializing our product and product candidates, our translational research program, expectations regarding our manufacturing capabilities, the expe ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q2 2022 Earnings Conference Call July 28, 2022 5:00 PM ET Company Participants Joshua Higa - Executive Director & Head, Investor Relations Emil Kakkis - Chief Executive Officer & President Erik Harris - Chief Commercial Officer Mardi Dier - Chief Financial Officer Camille Bedrosian - Chief Medical Officer Conference Call Participants Gena Wang - Barclays Joel Beatty - Baird Dae Gon Ha - Stifel Tazeen Ahmad - Bank of America Cory Kasimov - JPMorgan Yaron Werber - ...

Corporate Deck May 2022 Legal Warning Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations and projections regarding our future operating results and financial performance, anticipated cost or expense reductions, plans with respect to commercializing our product and product candidates, our translational research program, expectations regarding our manufacturing capabilities, the expec ...

Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) Q1 2022 Earnings Conference Call May 5, 2022 5:00 PM ET Company Participants Joshua Higa - Director, IR & Corporate Communications Emil Kakkis - President, CEO & Director Erik Harris - EVP & Chief Commercial Officer Mardi Dier - EVP & CFO Camille Bedrosian - Chief Medical Officer & EVP Conference Call Participants Huidong Wang - Barclays Bank Yigal Nochomovitz - Citigroup Tazeen Ahmad - Bank of America Merrill Lynch Joel Beatty - Robert W. Baird & Co. Maurice Ra ...

ultrageny% Corporate Deck February 2022 Legal Warning Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations and projections regarding our future operating results and financial performance, anticipated cost or expense reductions, plans with respect to commercializing our product and product candidates, our translational research program, expectations regarding our manufacturing capabil ...

ultrageny% Corporate Deck February 2022 Legal Warning Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations and projections regarding our future operating results and financial performance, anticipated cost or expense reductions, plans with respect to commercializing our product and product candidates, our translational research program, expectations regarding our manufacturing capabil ...