It doesn't matter your age or experience: taking full advantage of the stock market and investing with confidence are common goals for all investors. Luckily, Zacks Premium offers several different ways to do both. Featuring daily updates of the Zacks Rank and Zacks Industry Rank, full access to the Zacks #1 Rank List, Equity Research reports, and Premium stock screens, the research service can help you become a smarter, more self-assured investor. It also includes access to the Zacks Style Scores. What are ...

Investors in BioMarin Pharmaceutical Inc. (BMRN) need to pay close attention to the stock based on moves in the options market lately. That is because the Sep 20, 2024 $40.00 Call had some of the highest implied volatility of all equity options today. What is Implied Volatility? Implied volatility shows how much movement the market is expecting in the future. Options with high levels of implied volatility suggest that investors in the underlying stocks are expecting a big move in one direction or the other. ...

Greg Friberg, M.D., Appointed EVP, Chief Research & Development Officer as Hank Fuchs, M.D., Will Retire After 15 Years at Company James Sabry, M.D., Ph.D., Appointed EVP, Chief Business Officer SAN RAFAEL, Calif., Aug. 21, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the appointments of Greg Friberg, M.D., as Executive Vice President, Chief Research & Development Officer, succeeding Hank Fuchs, M.D., and James Sabry, M.D., Ph.D., as Executive Vice President, Chief Busine ...

-Event to be webcast live and a replay will be available SOURCE BioMarin Pharmaceutical Inc. SAN RAFAEL, Calif., Aug. 19, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will host an Investor Day with financial analysts and institutional investors on Wednesday, September 4, 2024, in New York City from 10:30 am to 1:00 pm Eastern Time. The event will feature presentations from members of the executive management team and will highlight BioMarin's pipeline, strategic plans, growth opportuniti ...

BioMarin Pharmaceutical's (BMRN) dwarfism drug Voxzogo is driving the company's top line on the back of strong demand. The recent label expansion to Voxzogo for use in infants with achondroplasia is likely to boost sales further in the days ahead. Meanwhile, after several delays, the FDA approved BMRN's Roctavian, a gene therapy for treating hemophilia A last June. The company also has an attractive early-stage pipeline with a focus on gene therapies. Here are some reasons why investors should consider bett ...

It doesn't matter your age or experience: taking full advantage of the stock market and investing with confidence are common goals for all investors. Luckily, Zacks Premium offers several different ways to do both. The research service features daily updates of the Zacks Rank and Zacks Industry Rank, full access to the Zacks #1 Rank List, Equity Research reports, and Premium stock screens, all of which will help you become a smarter, more confident investor. It also includes access to the Zacks Style Scores ...

Investors seek growth stocks to capitalize on above-average growth in financials that help these securities grab the market's attention and produce exceptional returns. But finding a great growth stock is not easy at all. That's because, these stocks usually carry above-average risk and volatility. In fact, betting on a stock for which the growth story is actually over or nearing its end could lead to significant loss. However, the Zacks Growth Style Score (part of the Zacks Style Scores system), which look ...

BioMarin Pharmaceutical Inc. BMRN announced an update on its Roctavian business to focus commercial operations on three markets: the U.S., Germany and Italy. These markets are where the medicine is approved and reimbursed as a severe hemophilia A treatment. The company will adjust its Roctavian efforts and reduce additional investments in development and manufacturing. BioMarin anticipates reducing annual direct Roctavian expenses to approximately $60 million beginning in 2025. The company has already begun ...

文章核心观点 - 公司第二季度业绩超预期,调整后每股收益为96美分,高于市场预期的57美分 [1] - 公司总收入达7.12亿美元,同比增长20%,超出市场预期 [2] - 公司主要产品Voxzogo销售强劲,带动整体收入增长 [4] - 公司上调了2024年全年收入和利润指引 [11][12][13] 产品销售情况 - Voxzogo销售额为1.839亿美元,同比增长62%,超出市场预期 [4] - Vimizim销售额为1.78亿美元,略低于市场预期 [6] - Palynziq销售额为8830万美元,同比增长18%,超出市场预期 [7] - Naglazyme销售额为1.32亿美元,同比增长47% [8] - Kuvan销售额下降43%,受到仿制药竞争的影响 [9] - Roctavian新产品销售额为740万美元,环比增长 [9] - Aldurazyme销售额下降4% [10] 研发进展 - 公司正在推进Voxzogo在其他矮小症状适应症的临床试验 [14] - 公司正在优化管线,重点发展BMN 333、BMN 349和BMN 351等项目 [15] - 公司决定终止BMN 293基因疗法的开发 [16]

财务数据和关键指标变化 - 公司在第二季度实现了20%的总收入增长和78%的非GAAP每股收益增长,这证明了公司能够推动增长、实现成本效率并加快盈利能力 [18][19] - VOXZOGO收入同比增长62%至1.84亿美元,截至第二季度末已有3,500名儿童接受VOXZOGO治疗 [24][47] - 酶治疗产品组合(NAGLAZYME、PALYNZIQ等)同比增长15% [29] - 公司将2024年全年收入指引上调至27.5亿-28.25亿美元,同比增长约15% [46][55] - 公司将2024年非GAAP营业利润率指引上调至26%-27%,同比扩张7个百分点 [55] - 公司将2024年非GAAP每股收益指引上调至3.10-3.25美元 [55] 各条业务线数据和关键指标变化 - VOXZOGO在所有年龄段的患者中均实现了强劲需求,尤其是5岁以下儿童的新处方占大多数 [11] - 公司正在加快推进VOXZOGO在缺骨发育不良、特发性矮小症、努南综合征、特纳综合征和SHOX缺乏症等5个新适应症的开发 [12][37] - ROCTAVIAN在美国、德国和意大利三个重点市场取得了一定进展,第二季度实现了约700万美元的收入 [30][31][32] 各个市场数据和关键指标变化 - 在美国这个最大的潜在市场,VOXZOGO在所有年龄段的患者中均实现了强劲需求,5岁以下儿童的新处方占大多数 [11] - 在美国以外的其他重点战略市场,VOXZOGO已实现了显著渗透,但仍有进一步增长空间 [11] - ROCTAVIAN在美国、德国和意大利三个重点市场取得了一定进展,但公司将把资源集中在这三个市场,以降低ROCTAVIAN相关开支至2025年约6000万美元 [30][31][32][34][120][136] 公司战略和发展方向及行业竞争 - 公司正在加快推进VOXZOGO在缺骨发育不良、特发性矮小症、努南综合征、特纳综合征和SHOX缺乏症等5个新适应症的开发 [12] - 公司决定调整ROCTAVIAN的策略,将资源集中在美国、德国和意大利三个重点市场,以降低ROCTAVIAN相关开支,预计到2025年底ROCTAVIAN将实现盈利 [13][14][15][16] - 公司将重点推进BMN 351(杜氏肌营养不良症)、BMN 349(α1抗胰蛋白酶缺乏症)和BMN 333(长效CMP制剂)三个管线项目的开发 [43] - 公司将停止BMN 293(肥厚型心肌病基因疗法)的开发,以确保资源集中在有最大潜在影响力的项目上 [44] 管理层对经营环境和未来前景的评论 - 公司对VOXZOGO在多个生长相关疾病领域的可持续领导地位充满信心,因为其安全性概况、作用机制以及在多个属性方面的扩展数据 [12] - 公司相信ROCTAVIAN的治疗特征和在血友病A治疗中的作用,并有信心通过调整资源配置,使ROCTAVIAN在2025年底前实现盈利 [13][14][15] - 公司对未来几年的收入和利润增长以及营业利润率的提升充满信心,这一点从本季度的强劲业绩和大幅提升的全年指引中可见一斑 [19][60] 其他重要信息 - 公司获得了BRINEURA适应症扩大至3岁以下CLN2病患儿童的批准,这将有助于尽早开始治疗,改善疾病自然进程 [20] - 公司将于下月举行投资者日,届时将分享公司的新愿景和战略,包括提高运营效率和成本管理的框架,以及管线和生命周期管理计划、长期财务目标和资本配置策略等 [61] 问答环节重要的提问和回答 问题1 **Phil Nadeau 提问** 关于调整ROCTAVIAN策略,除了缩减地理覆盖范围,公司是否还调整了在重点市场的投资策略和方式? [63] **Alexander Hardy 回答** 公司将把地理重点放在美国、德国和意大利这三个市场,并将投资集中在这些市场的患者转化活动上,这是公司根据经验认为对ROCTAVIAN成功最关键的。同时公司也将减少ROCTAVIAN的研发和制造开支。这些调整使公司有信心到2025年ROCTAVIAN能够实现盈利。 [64][65][66] 问题2 **Salveen Richter 提问** 公司对2025年ROCTAVIAN实现盈利的信心来自哪些方面?是需求还是340B动态的改善等? [70] **Cristin Hubbard 回答** 公司在美国、意大利和德国这三个重点市场看到了一些积极进展,包括美国有更多治疗中心开始给患者输注,意大利和德国也取得了一些进展。加上公司将ROCTAVIAN相关开支控制在约6000万美元的水平,这些因素让公司有信心ROCTAVIAN能在2025年实现盈利。 [71][72][73] 问题3 **Cory Kasimov 提问** 公司对VOXZOGO在其他适应症的竞争格局有何预期?以及是否考虑过VOXZOGO与生长激素的联合使用? [83] **Alexander Hardy 和 Hank Fuchs 回答** 公司对VOXZOGO在缺骨发育不良及后续适应症的竞争地位充满信心,因为VOXZOGO具有良好的安全性和持久疗效,且可以尽早用于0-5岁儿童,发挥最大治疗效果。 至于联合生长激素,公司认为单一使用VOXZOGO就能将患者身高恢复到正常人的90%左右,如果需要进一步优化疗效,公司更倾向于开发长效CMP制剂BMN