Third quarter 2024 total revenue grew 42% versus prior year to $139 million, including Crysvita® revenue of $98 million and Dojolvi® revenue of $21 million Reaffirmed 2024 expected total revenue guidance of $530 million to $550 million Breakthrough Designation granted for setrusumab (UX143) in osteogenesis imperfecta DTX401 Phase 3 follow-up data demonstrated higher and faster 62% mean reduction in cornstarch in crossover patients with glycogen storage disease type Ia (GSDIa) NOVATO, Calif., Nov. 05, 2024 ( ...

NOVATO, Calif., Oct. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today reported that the Phase 1/2/3 Cyprus2+ study of its UX701 gene therapy has demonstrated meaningful clinical activity as well as improvements in copper metabolism in Stage 1. Multiple responders have completely tapered off of standard-of-care treatment with responses seen in all three dose cohorts. The company plans to enroll an additional cohort in Stage 1 at a moderately increased dose and with an optimize ...

NOVATO, Calif., Sept. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it will present seven abstracts related to its ongoing late-stage program evaluating setrusumab (UX143) and osteogenesis imperfecta (OI), including a late-breaker oral presentation of the 14- month data from the Phase 2/3 Orbit study, at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting. The meeting is being held September 27-30, 2024, in Toronto, Canada. "Presen ...

Morgan Stanley 22nd Annual Global Healthcare Conference on September 4 Cantor Global Healthcare Conference on September 17 Bank of America Global Healthcare Conference on September 18 NOVATO, Calif., Aug. 29, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced participation in three upcoming investor conferences. Morgan Stanley 2 ...

Andrzej Rostek Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops innovative therapies for rare and ultra-rare genetic diseases. Its commercial portfolio includes Crysvita, Mepsevii, Dojolvi, and Evkeeza, which address unmet medical needs in conditions like X-linked hypophosphatemia [XLH], Mucopolysaccharidosis VII [MPS VII], long-chain fatty acid oxidation disorders [LC-FAOD], and homozygous familial hypercholesterolemia [HoFH]. Additionally, RARE's pipeline targets severe diseases such as osteogenesis ...

When the human genome was fully sequenced in 2003, the prospects of genetic medicine ushered in a new era of human health. Today, scientists and drug developers have a better understanding of the genetic drivers of disease than ever before. As such, genomics stocks present a new opportunity for investors to buy into the potential future of medicine, as nearly all modern therapies rely on understanding patient genetics to be as efficient as possible. Moreover, genomics companies differ from traditional healt ...

财务数据和关键指标变化 - 公司在第二季度实现总收入1.47亿美元,较上一季度有所反弹 [20][21] - Crysvita在北美、拉美和欧洲的收入合计1.14亿美元,是公司最大的收入来源 [23] - Dojolvi收入1900万美元,Evkeeza收入800万美元,Mepsevii收入600万美元 [24] - 总运营费用2.63亿美元,其中研发费用1.62亿美元,销售及管理费用8100万美元 [24] - 第二季度净亏损1.32亿美元,每股亏损1.52美元 [25] - 截至6月30日,公司现金及等价物余额8.74亿美元,包括6月融资3.81亿美元 [25] - 全年净现金流支出预计低于4亿美元 [26] 各条业务线数据和关键指标变化 - Crysvita在美国需求保持强劲,成人患者占比约60% [14] - Crysvita在拉美地区新增约60名患者,累计超620名获得报销治疗 [15] - Dojolvi在美国新增约30名患者,累计520名获得报销治疗 [17] - Dojolvi在欧洲和中东地区主要通过临时性销售获得收入 [18] - Evkeeza在EMEA地区新增约60名患者,在日本已获批并开始销售 [19] 各个市场数据和关键指标变化 - Crysvita在北美收入6700万美元,在拉美和土耳其收入4000万美元,在欧洲收入600万美元 [23] - Dojolvi在欧洲和中东地区的收入主要来自临时性销售 [18] - Evkeeza在EMEA地区和日本市场开始销售,为公司带来新的收入来源 [19] 公司战略和发展方向及行业竞争 - 公司在罕见病药物研发和商业化方面取得重大进展,管线有多个重要临床数据读出和监管进展 [8][9][10] - 公司有望在未来12-18个月内提交2个新药申请,并获得UX143的关键III期临床数据 [43] - 公司有望在未来6-18个月内取得多个重要监管和临床里程碑,展现了强大的执行能力 [12] - 公司在罕见病领域的管线和商业化能力在行业中处于领先地位 [12] 管理层对经营环境和未来前景的评论 - 公司在商业化和临床开发方面的强劲表现,使公司有信心超过年初设定的收入指引区间 [7][21][27] - 公司有望在未来12-18个月内提交2个新药申请,并获得多个重要临床数据读出,展现了强大的执行能力 [37][43] - 公司在罕见病领域的管线和商业化能力在行业中处于领先地位,前景看好 [12] 其他重要信息 - 公司已完成与FDA关于UX-111和GTX-102的关键临床试验设计的沟通,为后续提交新药申请奠定基础 [10][34] - 公司计划在2024年底或2025年初提交UX-111和DTX401的新药申请 [43] - 公司正在积极推进GTX-102的III期临床试验,预计年内启动 [34][35] - 公司正在评估UX143的中期分析结果,如达到预设标准可能会提前停止试验 [39][40] 问答环节重要的提问和回答 问题1 **Gena Wang 提问** 询问公司对竞争对手Ionis和Roche Angelman综合征药物数据的看法,以及对公司GTX-102临床试验设计的影响 [46][47] **Eric Crombez 回答** 公司对竞争对手的数据有所关注,但认为自身长期数据更为出色,显示了药物的持续疗效。公司已与FDA达成一致,确定了III期试验的设计和关键终点指标 [48][49] 问题2 **Dae Gon Ha 提问** 询问公司在GTX-102临床试验设计上如何最大化产品差异化,以及是否考虑使用脊髓腔注射装置等生命周期管理策略 [57] **Emil Kakkis 回答** 公司的长期数据显示了药物的持续疗效优势,且给药剂量远低于竞争对手,体现了产品的优势。公司未来会考虑使用脊髓腔注射等生命周期管理策略,以进一步提升疗效和患者体验 [58][59][60] 问题3 **Tazeen Ahmad 提问** 询问公司UX701 Wilson病临床试验数据推迟的原因,以及后续的发展计划 [62] **Emil Kakkis 回答** 完成最后一组病人的6个月随访时间较预期有所延迟,公司需要更长时间观察药物对铜代谢的影响。公司对早期数据结果感到鼓舞,未来将继续推进III期临床试验 [63]

Ultragenyx Pharmaceutical (RARE) incurred a second-quarter 2024 loss of $1.52 per share, narrower than the Zacks Consensus Estimate of a loss of $1.64. The company had reported a loss of $2.25 per share in the yearago quarter. Ultragenyx's total revenues amounted to $147 million in the reported quarter, up 36% year over year. The top line also beat the Zacks Consensus Estimate of $123 million on the back of higher product revenues. The company markets four drugs, namely Crysvita, Mepsevii, Dojolvi and Evkee ...

业绩总结 - 2024年预计总收入为5.3亿至5.5亿美元，同比增长22%至27%[16] - Crysvita在2024年的收入指导为3.75亿至4亿美元，增长率为14%至22%[17] - Dojolvi在2024年的收入指导为7500万至8000万美元，增长率为6%至13%[17] - 截至2024年6月30日，现金及现金等价物为8.74亿美元[24] - 2024年预计运营现金使用量将低于4亿美元[24] 临床试验与研发进展 - 2024年预计临床成功率为82%[12] - 从临床试验到批准的平均时间为5.5年[13] - UX143在治疗成骨不全症的临床试验中，观察到腰椎骨密度的显著增加，基线至12个月的变化率为22.25%[30] - UX143治疗后，年化骨折率减少67%[32] - GTX-102在Cohorts A & B的临床改善在多个领域表现出快速且具有临床意义的提升，Day 170的净响应为+2，p值<0.0001[34][37] - DTX401在第48周显示出每日玉米淀粉摄入量显著减少41%，p值<0.0001，同时维持了血糖控制[43][45] - 在DTX401组中，68%的患者实现了≥30%的玉米淀粉摄入减少，而安慰剂组仅有13%[45] - UX701在Wilson病的Cohort 1中，5名患者中有4名减少了标准治疗，2名患者完全停止了螯合剂和/或锌治疗[42] - GTX-102在Day 758的Bayley-4认知评分中显示出持续的临床显著改善，Cohorts 4-7的患者表现超出自然历史数据[35][36] - DTX401组在夜间玉米淀粉摄入量上，平均减少0.4克，p值为0.0410[48] - GTX-102在Phase 2安全性数据中未出现与治疗相关的严重不良事件[39] - DTX401在关键次要终点中，低血糖范围内的葡萄糖值变化显示出非劣效性，p值<0.0001[43] - DTX401的基线平均值为87.4（标准差37.0），而安慰剂组为100（标准差74.4）[49] 未来展望与产品开发 - GTX-102的Phase 3研究计划预计在2024年底启动，Aurora研究计划在2025年启动[40][41] - 预计到2024年底，Angelman综合症的Phase 3 Aspire研究将启动[51] - DTX301（OTC缺乏症）的Phase 3入组预计在2024年下半年完成[51] - UX701（威尔逊病）的第一阶段安全性和初步有效性数据预计在2024年下半年发布[51] - Ultragenyx预计将实现多个重要产品的批准，支持收入增长和盈利能力的路径[53] 知识产权与市场潜力 - DTX401的美国知识产权权利和特许权使用费预计到2034年到期[72] - UX143（成骨不全症）的抗体特许权使用费为双位数的分层特许权使用费[72] - Evkeeza的潜在监管和销售里程碑高达6300万美元[70] - MEPSEVII的生物制剂专有权预计到2029年到期[82]

Ultragenyx (RARE) reported $147.03 million in revenue for the quarter ended June 2024, representing a yearover-year increase of 35.8%. EPS of -$1.52 for the same period compares to -$2.25 a year ago. The reported revenue compares to the Zacks Consensus Estimate of $123.42 million, representing a surprise of +19.13%. The company delivered an EPS surprise of +7.32%, with the consensus EPS estimate being -$1.64. While investors scrutinize revenue and earnings changes year-over-year and how they compare with Wa ...