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NEW YORK, Sept. 4, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) hosted an Investor Day earlier today, where President and Chief Executive Officer Alexander Hardy and other members of BioMarin's leadership team provided an overview of the company's new corporate strategy to deliver sustained value creation and introduced longer-term Total Revenue and Non-GAAP Operating Margin guidance. A copy of the presentation and a replay of the webcast are available at investors.biomarin.com. "Over the last 9 months, we have undertaken the transformation of BioMarin's operations and ways of working with the goal of accelerating and delivering substantial value to all our stakeholders – patients, employees and shareholders," said Mr. Hardy. "Our new corporate strategy harnesses BioMarin's unique capabilities creating and leading new therapeutic markets around the world to deliver innovative medicines to the patients we serve. We are confident in our ability to realize the ambitious plans outlined today and excited to deliver on our new vision for BioMarin's future." Corporate Strategy The company reviewed BioMarin's differentiated strategy including its new company structure and updated organizational model, now built around three business units, Enzyme Therapies, Skeletal Conditions and ROCTAVIAN®, designed to support the focus on sustainable growth. The updated corporate strategy also includes the implementation of a $500 million cost transformation program that will contribute to Non-GAAP Operating margin targets set for 2026 and beyond. Value Commitment BioMarin provided an overview of its long-term financial outlook, based on its strategic plan to drive revenue growth and expand Non-GAAP Operating Margin. The summary table below includes the company's full year 2024 guidance, reaffirmed today, and the newly introduced outlook for 2026 with respect to Non-GAAP Operating Margin and other metrics for 2027, shared at BioMarin's Investor Day. Innovation The company highlighted its innovation strategy that supports BioMarin's pipeline of high impact medicines, including 11 anticipated product launches by 2034 and two by 2027. Building on its 25-year history developing medicines that target serious genetic conditions, several programs that are currently advancing through the pipeline, include: Phase 3 enrollment of VOXZOGO® for the treatment of hypochondroplasia, with expected data readout targeted in 2026, and potential approval in 2027 Clinical updates on four additional skeletal conditions, including idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX Deficiency, all progressing through or beginning Phase 2 studies PALYNZIQ® age label expansion to include 12-17-year-olds in the United States and 12-15-year-olds outside of the United States, with targeted filings in late 2025 and early 2026, respectively BMN 390 for the treatment of phenylketonuria (PKU) with a targeted profile that reduces immunogenicity from novel pegylation, and targeted Investigational New Drug (IND) application planned for late 2025 BMN 351 for the treatment of Duchenne Muscular Dystrophy that targets a unique novel site for exon skipping that may enable dystrophin expression up to 40%, based on observations preclinically. Target Proof of Concept (POC) is expected in 2025 BMN 349 for the treatment of Alpha-1 Antitrypsin Deficiency with a unique mechanism of action that has the potential to transform liver health and with target POC in 2026 BMN 370 for the treatment of von Willebrand Disease (vWD) has the potential to normalize bleeding events, based on results from a vWD preclinical model, and is designed to be delivered with a single subcutaneous injection. The targeted IND is planned for late 2025. Growth: The company highlighted its strategy for optimizing its growing and durable Enzyme Therapies business unit, as well as its plan for sustainable leadership across multiple Skeletal Conditions, building on the strength of VOXZOGO for the treatment of achondroplasia. The combined business units are expected to drive a targeted mid-teen Compounded Annual Growth Rate through 2034. A replay of today's event and accompanying presentation slides can be found at investors.biomarin.com. 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 unparalleled research and development capabilities have resulted in eight transformational 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, Non-GAAP Diluted EPS, Operating Cash Flow and Revenue Compound Annual Growth Rate (CAGR) for, in certain instances, the full-year 2024 and future periods and the underlying assumptions; BioMarin's new corporate strategy, including plans and expectations regarding innovation and growth, the $500 million cost optimization program, capital allocation, and organizational redesign efforts, as well as the anticipated benefits of such strategy, including BioMarin's ability to achieve top quartile biopharma revenue growth, double its Non-GAAP Operating Margin, achieve innovation and efficiencies, optimize cash flow and capital allocation, and deliver significant and sustained value creation to stakeholders; BioMarin's future strategy for ROCTAVIAN and its anticipated benefits, including BioMarin's expectations regarding reduction of annual direct ROCTAVIAN expenses beginning in 2025 and ROCTAVIAN being profitable by the end of 2025; ability of BioMarin's approved products, including VOXZOGO and BioMarin's enzyme therapies, to drive long-term revenue growth; the clinical development and commercialization of BioMarin's product candidates and commercial products, including plans and expectations regarding (i) the ability to expand BioMarin's leadership in achondroplasia with VOXZOGO and leverage VOXZOGO in other skeletal conditions, including hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency; (ii) development of BMN 333 for the treatment of achondroplasia and hypochondroplasia, (iii) expansion of PALYNZIQ for the treatment of adolescents with phenylketonuria (PKU), (iv) development of BMN 390 for the treatment of PKU, (v) development of BMN 351 for the treatment of Duchenne Muscular Dystrophy, (vi) development of BMN 349 for the treatment of alpha-1 antitrypsin deficiency, and (vii) development of BMN 370 for the treatment of von Willebrand disease; the expected benefits and availability of BioMarin's product candidates and commercial products; the timing of BioMarin's clinical development and commercial prospects, including announcements of data from clinical studies and trials; and potential growth opportunities and trends, including the assumptions and expectations regarding Total Addressable Patient Population with respect to the conditions targeted by BioMarin's product candidates and commercial products. 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, 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 Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as such factors may be updated by any subsequent reports. You should carefully consider that information before you make an investment decision. You should not place undue reliance on forward-looking statements, which speak only as of the date hereof. These forward-looking statements are based on the beliefs and assumptions of the Company's management based on information currently available to management and should be considered in connection with any written or oral forward-looking statements that the Company may issue in the future as well as other cautionary statements the Company has made and may make. Except as required by law, BioMarin does not undertake 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 This press release includes 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 weighted-average diluted shares outstanding. Non-GAAP weighted-average diluted shares outstanding is defined by the company as GAAP weighted-average diluted shares outstanding, adjusted to include any common shares issuable under the company's equity plans and convertible debt in periods when they are dilutive under Non-GAAP. 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 and Non-GAAP weighted-average diluted shares outstanding are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information for historical results 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 measures 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 may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. Forward-Looking Non-GAAP Financial Measures 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 its 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. SOURCE BioMarin Pharmaceutical Inc.

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。