Ultragenyx (RARE) came out with a quarterly loss of $1.52 per share versus the Zacks Consensus Estimate of a loss of $1.64. This compares to loss of $2.25 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of 7.32%. A quarter ago, it was expected that this biotechnology company would post a loss of $1.72 per share when it actually produced a loss of $2.03, delivering a surprise of -18.02%. Over the last four quarters, the company h ...

产品管线和临床进展 - 公司致力于开发治疗罕见和超罕见遗传性疾病的创新产品[87] - 公司目前拥有4类产品:生物制品、小分子药物、AAV基因疗法和核酸产品候选药物[88] - Crysvita获批用于成人和儿童1岁及以上X联锁性低磷血症(XLH)和肿瘤性骨软化症(TIO)的治疗[91,92] - Mepsevii获批用于治疗黏多糖贮积症VII型(MPS VII)[94] - Dojolvi获批用于治疗长链脂肪酸氧化障碍(LC-FAOD)[95] - Evkeeza获批用于治疗纯合子家族性高胆固醇血症(HoFH)[96] - UX143(setrusumab)在II期临床试验中显示可显著降低成骨不全患者的骨折发生率和改善骨密度[104] - GTX-102计划启动III期临床试验,评估其治疗天使综合征的疗效[105,106] - UX111计划在2024年底或2025年初提交生物制品许可申请[107] - 公司在2024年5月宣布了用于治疗I型糖原贮积症的DTX401药物的III期临床试验取得积极的结果[108] - DTX401治疗组患者在48周时平均减少了41.3%的每日玉米淀粉摄入量,而安慰剂组仅减少10.3%(p<0.0001)[108] - 68%的DTX401治疗组患者实现了≥30%的玉米淀粉摄入量减少,37%实现了≥50%的减少,而安慰剂组分别只有13%和4%[108] - 公司正在进行为期64周的III期临床试验,评估DTX301治疗OTC缺乏症的疗效,预计2024年下半年完成患者入组[109] - 公司已经完成了UX701治疗Wilson病的剂量递增试验的3个剂量组,预计2024年下半年获得结果,并选择剂量进入下一阶段的随机安慰剂对照试验[110] 财务表现 - 公司2024年上半年总收入为2.559亿美元,同比增长23%,主要由于Crysvita、Evkeeza等产品需求增加[116] - Crysvita在拉丁美洲和欧洲、中东及非洲地区的销售增加是收入增长的主要原因[116] - 公司2024年6月30日的现金、现金等价物和可流通债务证券余额为8.745亿美元[111] - 公司2024年上半年净亏损为3.023亿美元,主要由于研发和销售费用的增加[111] - 研发费用增加主要是由于基因疗法项目的临床进展以及相关的内部和外部制造成本增加[122][123] - 生物制品和核酸项目的研发费用增加主要是由于UX143和GTX102项目的临床进展和相关的临床开发及制造费用增加[122][123] - 转化研究费用减少主要是由于IND阶段项目的制造和人员费用减少[122][123] - 获得许可、收购和里程碑费用减少主要是由于2023年6月30日三个月期间确认了UX143项目临床入组里程碑费用[122][123] - 已上市产品费用减少主要是由于Crysvita在北美的商业化责任转移给KKC,以及与Evkeeza相关的儿科和开放标签延长试验的Regeneron合作费用减少[122][123] 资金来源和现金流 - 公司截至2024年6月30日三个月和六个月的所得税准备金名义金额有所增加[142] - 公司主要通过出售股权证券、商业产品收入、出售未来特许权使用费以及战略合作安排为运营提供资金[143] - 截至2024年6月30日公司拥有8.745亿美元的现金、现金等价物和可流通债务证券[143] - 公司于2023年10月和2024年6月分别完成了两次公开发行,共募集到7.074亿美元[143] - 公司2024年上半年经营活动使用现金2.677亿美元,主要是由于净亏损和营运资金变动[146] - 公司2024年上半年投资活动提供现金1.55亿美元,主要是由于可流通债务证券净变动[147] - 公司2024年上半年筹资活动提供现金3.849亿美元,主要是由于公开发行股票[148] - 公司预计未来几年将继续产生年度亏损,需要额外资金支持研发、商业化和基础设施建设[149] - 公司预计未来现金需求将取决于临床试验进度、产品制造、新产品管线、监管审批等因素[149] - 公司预计将通过现有资金、商业收入、股权或债务融资等方式满足未来现金需求[150]

财务数据 - 第二季度总收入为1.47亿美元，Crysvita®收入为1.14亿美元，Dojolvi®收入为1,935万美元[2] - 2024年全年总收入预计将增加至5.3亿美元至5.5亿美元[2] - 2024年上半年总收入为2.56亿美元，同比增长21.3%[31] - 2024年上半年产品销售收入为1.36亿美元，同比增长57.7%[31] - 2024年上半年专利权使用费收入为1.20亿美元，同比增长133.6%[31] - 2024年6月30日现金及可流动性资产为8.74亿美元，较2023年12月31日增加12.5%[34] - 2024年6月30日总资产为16.18亿美元，较2023年12月31日增加8.5%[34] - 2024年6月30日股东权益为4.32亿美元，较2023年12月31日增加57.1%[34] 产品收入预测 - Crysvita收入预计将达到上限375-400百万美元，包括拉丁美洲和土耳其的产品销售、欧洲的特许权使用费以及北美的特许权使用费[11] - Dojolvi收入预计将达到7500-8000万美元[11] 研发及费用 - 2024年上半年研发费用为3.40亿美元，同比增加2.8%[31] - 2024年上半年销售及管理费用为1.59亿美元，与去年同期持平[31] - 2024年上半年非现金股票激励费用为7,630万美元，同比增加14.6%[32] - 2023年6月UX143临床里程碑费用为900万美元[32] 临床试验进展 - UX143(setrusumab)治疗成骨不全的14个月数据显示骨折率大幅降低67%，骨密度持续改善[12][13] - GTX-102治疗天使综合征已完成与FDA的关键性III期临床试验设计沟通，计划于2024年底启动III期临床试验[15][16][18] - UX701基因治疗治疗Wilson病的III期临床试验第3组患者已完成给药，预计2024年下半年获得中期数据[19] - UX111基因治疗治疗粘多糖贮积症IIIA型已与FDA达成共识,可以使用脑脊液硫酸肝素作为加速批准的合理替代终点指标[20] 现金流预测 - 2024年净现金使用量预计低于4亿美元[11]

Wall Street expects a year-over-year increase in earnings on higher revenues when Ultragenyx (RARE) reports results for the quarter ended June 2024. While this widely-known consensus outlook is important in gauging the company's earnings picture, a powerful factor that could impact its near-term stock price is how the actual results compare to these estimates.The stock might move higher if these key numbers top expectations in the upcoming earnings report. On the other hand, if they miss, the stock may move ...

Ultragenyx Pharmaceutical (RARE) announced that it has reached alignment with the FDA regarding the phase III study design and endpoints for GTX-102, an antisense oligonucleotide for Angelman syndrome, following the completion of a successful end-of-phase II meeting with the regulatory body. The company expects to initiate the same by the end of 2024.In the meeting, Ultragenyx mainly discussed the positive interim data from the phase I/II study, announced in April 2024, with the FDA. The study evaluated the ...

NOVATO, Calif., June 13, 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 the pricing of its underwritten public offering of 7,435,898 shares of its common stock at a price to the public of $39.00 per share. In addition, in lieu of issuing common stock to certain investors, the company is offering pre-funded warrants to purchas ...

NOVATO, Calif., June 13, 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 the pricing of its underwritten public offering of 7,435,898 shares of its common stock at a price to the public of $39.00 per share. In addition, in lieu of issuing common stock to certain investors, the company is offering pre-funded warrants to purcha ...

Ultragenyx (RARE) shares soared 7.8% in the last trading session to close at $44.64. The move was backed by solid volume with far more shares changing hands than in a normal session. This compares to the stock's 1.1% gain over the past four weeks.The stock rallied after the company, along with its partner Mereo BioPharma, announced new positive 14-month data from the phase II portion of the phase II/III Orbit study evaluating setrusumab (UX143) in patients with osteogenesis imperfecta (OI). The data showed ...

NOVATO, Calif., June 12, 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 that it has commenced an underwritten public offering of up to $350,000,000 of shares of its common stock and, in lieu of issuing common stock to certain investors, pre-funded warrants to purchase shares of its common stock. In addition, the company is ex ...

NOVATO, Calif., June 12, 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 that it has commenced an underwritten public offering of up to $350,000,000 of shares of its common stock and, in lieu of issuing common stock to certain investors, pre-funded warrants to purchase shares of its common stock. In addition, the company is e ...