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声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。4月24日，基因疗法龙头BioMarin Pharmaceutical 发布2024一季报：营收6.49亿美元，同比增长9%；归母净利润达0.89亿美元，增长75%；总资产增长7%至68.73亿美元。该公司已有8个商业化产品，其中支柱产品2024Q1营收：Vimizim：1.92亿美元，同比增长2%，是首个Morquio A综合征治疗药物；Naglazyme：1.06亿美元，同比下降14%，用于治疗粘多糖增多症VI；Palynziq：0.76亿美元，同比增长21%，用于治疗苯丙酮尿症成人患者。值得注意的是，于2023年6月获批上市，首个A型血友病基因疗法Roctavian 2024Q1 销售80万美元。而 Roctavian2023年销售额为350万美元。BioMarin对研发管线进行了评估，有3个项目进展迅速，分别是：针对多种生长障碍的 BMN 333、针对α-1抗胰蛋白酶缺乏症(AATD)相关肝病的 BMN 349 和针对杜氏肌营养不良症的反义寡核苷酸疗法 BMN 351。同时，BioMarin表示将停止4个研发项目的投资，分别是：BMN 355：治疗长QT综合征；BMN 365：用于PKP2基因突变引起的心律失常性心肌病的AAV基因疗法，尚未进行人体测试；BMN 331：用于遗传性血管性水肿的基因疗法，处于1/2期研究阶段；BMN 255：治疗肝脏疾病，处于早期研究阶段。受此影响，BioMarin股价大跌10%，目前市值为173.1亿美元。最后，BioMarin 表示，通过停止的4个研究，今年预计节省 5000 万~ 6000 万美元开支，不过公司已将部分资金投入到加速发展的3个项目中。预计 2024 年，公司运营费用将减少 3500 万~ 4000 万美元。关于BioMarinBioMarin成立于1997年，是一家致力于通过基因发现改变生活的全球性生物技术公司。该公司开发并商业化靶向治疗，解决遗传疾病的根本原因。BioMarin强大的研发能力已经为罕见遗传疾病患者带来了多种创新的商业疗法。研发管线参考资料：https://investors.biomarin.com/news/news-details/2024/BioMarin-Reports-Record-Financial-Results-for-the-First-Quarter-2024/default.aspx版权声明：本文转自细胞基因治疗前沿，如不希望被转载的媒体或个人可与我们联系，我们将立即删除-END-药事纵横投稿须知：稿费已上调，欢迎投稿各位朋友好，觉得本文对您有帮助，请随手点一下下方的在看，以便让你的朋友也能看到哦。

First Quarter 2024 Total Revenues of $649 Million (+9% Y/Y and +13% at Constant Currency Y/Y); GAAP Diluted Earnings per Share (EPS) of $0.46 (+70% Y/Y) and Non-GAAP Diluted Earnings per Share of $0.71 (+18% Y/Y) VOXZOGO® Net Product Revenues of $153 Million in Q1'24 (+74% Y/Y); Children Treated Increased Over 100% Y/Y For Full-year 2024 Guidance, Total Revenues Reaffirmed, Non-GAAP Operating Margin and Non-GAAP EPS Raised R&D Prioritization Results in Acceleration of Three Highest Value Programs Conference Call and Webcast Scheduled Today at 4:30 p.m. ET SAN RAFAEL, Calif., April 24, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the quarter ended March 31, 2024. "During the quarter, execution across our business led to double digit revenue growth, on a constant currency basis, and an 18% increase in non-GAAP diluted earnings per share. At the same time, we made rapid progress on advancing our strategic priorities for the year, including accelerating and maximizing the VOXZOGO opportunity, focusing R&D on the most productive assets, and increasing profitability," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We were pleased with the outcome of our strategic R&D asset review, resulting in the acceleration and prioritization of the most potentially impactful medicines for patients. By advancing and focusing on these promising programs, we have taken decisive steps to drive value creation for all our stakeholders. This approach to delivering innovative therapies is an important part of BioMarin's operational transformation, and we look forward to sharing more details at Investor Day on September 4th." Mr. Hardy continued, "Quarterly results were driven by strong demand for VOXZOGO, the only approved treatment for children with achondroplasia, and solid contributions from our established enzyme products. During the quarter, we made significant progress executing on our top priority to maximize and accelerate the reach of VOXZOGO, in achondroplasia and beyond. Globally, the number of children treated with VOXZOGO increased by over 500 from the prior quarter, and totaled over 3,100 by quarter end. In our largest single market, the U.S., we are realizing the tremendous opportunity ahead and expect U.S. uptake trends to continue. Our registration-enabling plans with VOXZOGO in hypochondroplasia, and ongoing discussions with health authorities to align on development plans for idiopathic short stature and pathway conditions are on-track, with all three studies expected to begin enrollment this year." Financial Highlights: Total Revenues for the first quarter of 2024 were $648.8 million, an increase of 9% compared to the same period in 2023. The increase in Total Revenues was primarily attributed to higher VOXZOGO sales volume driven by new patients initiating therapy across all regions. The increase was also due to higher PALYNZIQ product revenues primarily driven by sales volume growth in the U.S. The increase was partially offset by lower NAGLAZYME product revenues due to timing of orders in countries that place large government orders, particularly in the Middle East, as well as lower KUVAN product revenues attributed to continued generic competition as a result of the loss of market exclusivity. GAAP Net Income increased by $37.8 million to $88.7 million in the first quarter of 2024 compared to the same period in 2023. The increase was primarily due to higher gross profit driven by increased VOXZOGO sales volume and a decrease in Other income (expense), net, related to an impairment charge in the first quarter of 2023, which did not recur in the first quarter of 2024. The increases were partially offset by higher spend in research and development (R&D) programs to support both early-stage research and clinical activities, including new indications with VOXZOGO, and higher spend in Selling, General and Administrative (SG&A) due to the continued support of global VOXZOGO market expansion and incremental administrative expenses in the quarter. Non-GAAP Income increased by $23.9 million to $139.7 million in the first quarter of 2024 compared to the same period in 2023. The increased Non-GAAP Income was primarily due to higher gross profit driven by increased VOXZOGO revenues, partially offset by higher R&D expenses (as noted above). 1Q Update on 2024 Strategic Priorities In the first quarter, BioMarin executed on its four strategic priorities, first outlined in January, and focused on value creation through accelerating growth, optimizing efficiencies and driving operational excellence. Accelerate and maximize the VOXZOGO opportunity During the first quarter, the number of children being treated with VOXZOGO for achondroplasia accelerated. In the period, more than 500 additional children began treatment with VOXZOGO, as compared to an increase of approximately 300 new treatment starts in the fourth quarter of 2023. Over 3,100 children were benefiting from VOXZOGO treatment across 43 active markets, as of the end of the first quarter. This represents a 102% increase in the number of children being treated with VOXZOGO, year-over-year. In the largest single market, the U.S., more families with children under the age of 5 years sought treatment with VOXZOGO. This trend is expected to continue as real-world evidence, supported by VOXZOGO's extensive safety and efficacy profile, drives awareness and confidence for those families interested in treatment. With VOXZOGO now approved for children from infancy in most regions, families have the opportunity to benefit from longer treatment and potentially greater benefit. Enrollment in the pivotal program with VOXZOGO for the treatment of children with hypochondroplasia continued. The observational run-in study is expected to complete enrollment by year-end, and the 52-week randomized, double-blind, placebo-controlled phase of the 80-participant clinical trial, is expected to start mid-year and is expected to complete enrollment in the first half of 2025. During the quarter, BioMarin had productive engagement with the Food and Drug Administration to align on development programs in idiopathic short stature (ISS) and multiple genetic short stature pathway conditions (PC), and expects both studies to begin enrollment in the second half of 2024. Establish ROCTAVIAN opportunity During the quarter, reimbursement and market access challenges continued to impact the ability of interested patients to receive ROCTAVIAN treatment. For the remainder of 2024, BioMarin's global commercial team will continue to focus on key elements critical to supporting ROCTAVIAN uptake in the U.S., Italy and Germany. In April, BioMarin treated the first patient in Italy with ROCTAVIAN following the January price approval by the Italian Medicines Agency. Focus R&D on the most productive assets During the quarter, BioMarin completed a strategic portfolio assessment of R&D programs to determine which have the most transformative potential for patients and value creation for shareholders. With the combined focus on patient impact and commercial opportunity, three programs will be accelerated. The programs, BMN 333, long-acting CNP for multiple growth-disorders, BMN 349, a first-in-class, oral therapeutic for AATD-associated liver disease, and BMN 351, BioMarin's next-generation oligonucleotide for DMD, all met the highest bar for advancement. As a result of its prioritized portfolio, four programs will be discontinued, including BMN 331, BMN 255, BMN 355 and BMN 365. None of the programs were discontinued due to safety signals. The additional programs in BioMarin's pipeline will move forward within the revised evaluation framework that provides a high bar for consistent and ongoing assessment to determine if they fit in the company's focused portfolio. Accelerate EPS growth and expand margins Strong performance in the first quarter underscored BioMarin's execution of its financial strategy to drive year-over-year Non-GAAP Operating Margin expansion and Non-GAAP EPS growth twice as fast as revenues. The company reaffirmed full-year 2024 Total Revenue guidance, and raised Non-GAAP Operating Margin and Non-GAAP Diluted EPS guidance. The improved profitability guidance is a result of the planned reduction in operating expenses for the discontinued early-stage R&D programs, in the range of $50 million and $60 million. The planned reductions in R&D expense were partially off-set by planned increases in operating expenses for the accelerated programs, resulting in planned net reductions to 2024 operating expenses of $35 million to $40 million, driving the updated guidance. BioMarin's updated full-year 2024 guidance does not reflect the impact of potential additional future business decisions that may result from its ongoing strategic business review. 2024 Full-Year Financial Guidance (in millions, except % and EPS amounts) (Updated) BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from our strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors. BioMarin will host a conference call and webcast to discuss first quarter 2024 financial results today, Wednesday, April 24, 2024, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at . About BioMarin Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's robust research and development capabilities have resulted in multiple innovative commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit . Forward-Looking Statements This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Operating Margin percentage, and Non-GAAP Diluted EPS for the full-year 2024; BioMarin's new corporate strategy, including the timing of the completion and announcement; BioMarin's ability to accelerate the VOXZOGO opportunity; the anticipated benefits from its ongoing strategic review; the timing of orders for commercial products; the timing of BioMarin's clinical development and commercial prospects, including announcements of data from clinical studies and trials; the clinical development and commercialization of BioMarin's product candidates and commercial products, including (i) the potential to leverage VOXZOGO in conditions beyond achondroplasia, such as hypochondroplasia as well as idiopathic short stature and other genetic short stature pathway conditions; the commercialization of BioMarin's products, including (i) the anticipated start and growth of commercial sales of VOXZOGO in additional countries, and (ii) the commercialization of ROCTAVIAN for the treatment of severe hemophilia A in the U.S. and Europe (including Italy and Germany); the expected benefits and availability of BioMarin's product candidates; and potential growth opportunities and trends, including BioMarin's expectation that VOXZOGO product growth will continue to expand rapidly. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products, impacts of macroeconomic and other external factors on BioMarin's operations; results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the Food and Drug Administration, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; actual sales of BioMarin's commercial products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the year ended December 31, 2023 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin®, BRINEURA®, KUVAN®, NAGLAZYME®, PALYNZIQ®, ROCTAVIAN®, VIMIZIM® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners. Non-GAAP Information The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP diluted shares outstanding. The company's presentation of percentage changes in total revenues at constant currency rates, which is computed using current period local currency sales at the prior period's foreign exchange rates, is also a Non-GAAP financial measure. This measure provides information about growth (or declines) in the company's total revenue as if foreign currency exchange rates had not changed between the prior period and the current period. BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. Because Non-GAAP Income, Non-GAAP Operating Margin percentage, Non-GAAP Diluted EPS, Non-GAAP Diluted Shares outstanding and constant currency are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the company's results from period to period and to its forward-looking guidance, and to identify operating trends in the company's principal business. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Non-GAAP Income and its components are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information: SOURCE BioMarin Pharmaceutical Inc.