Von Willebrand Diseases

NEW YORK--( BUSINESS WIRE )--Pathway to Cures (P2C) announces two new members to its board of directors, Sharon Richardson, PhD , a Director on the National Bleeding Disorders Foundation Board, and Philip M. Gattone, M.Ed. , President and CEO of the National Bleeding Disorders Foundation. Pathway to Cures is the venture philanthropy fund for the National Bleeding Disorders Foundation (NBDF) focused on investing in emerging biotech companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community. Inheritable blood and bleeding disorders include rare and ultra-rare blood disorders, hemophilia A and B, von Willebrand disease, Sickle Cell disease, anemia, clotting disorders, and other hematological disorders, affecting more than 20 million people worldwide. Sharon Richardson, PhD, is a result-driven strategist with 28 years of experience in the pharmaceutical industry as a medical affairs leader committed to creating healthier communities through scientific exchange, patient advocacy, and community engagement. “P2C offers a unique opportunity for the National Bleeding Disorders Foundation to leverage their 75 years of serving people living with blood and bleeding disorders,” said Sharon Richardson. “It is a privilege to lend my time and assistance to advance the science and treatment options available to our community and to work alongside this talented and dedicated group of staff and volunteers.” “Sharon has led a life of selfless service to others and has used her strong leadership skills to make a difference for our community.” said Phil Gattone “We are thrilled to have Sharon join the Pathway to Cures Board and look forward to working with her to make a positive impact in more lives.” Philip M. Gattone, M.Ed., joined the National Bleeding Disorders Foundation as president and chief executive officer earlier this year. Gattone is a seasoned leader dedicated to amplifying the impact of nonprofit organizations through his distinctive blend of leadership and strategic insight. He has served as a senior executive in local, regional, national, and global nonprofit organizations and healthcare companies. “We’re honored and delighted to have Sharon and Phil supporting our efforts for the inheritable blood and bleeding disorders community!” said Teri Willey, Managing Director of the Venture Philanthropy Fund “Their knowledge and experience will enhance our ability to ensure there are more therapies and cures available for our community.” About Pathway to Cures Pathway to Cures (P2C) is the venture philanthropy fund of the National Bleeding Disorders Foundation created to accelerate the development of cures across all inheritable blood and bleeding disorders. In collaboration with other organizations, P2C invests in innovative therapies and technologies, leveraging the deep resources and scientific community relationships of the National Bleeding Disorders Foundation. By reinvesting proceeds from investments back into the Fund, P2C will amplify the investment impact, support promising biotech companies, and build a portfolio of investments that further the mission of the National Bleeding Disorders Foundation. For more information, visit www.pathwaytocures.org or contact Renee Peck at rpeck@pathwaytocures.org . About National Bleeding Disorders Foundation The National Bleeding Disorders Foundation (NBDF) is dedicated to finding cures for inheritable blood and bleeding disorders and addressing and preventing these disorders' complications through research, education, and advocacy, enabling people and families to thrive. Today, NBDF serves people across the United States with all bleeding disorders, including hemophilia, von Willebrand disease, rare factor deficiencies, and platelet disorders. The foundation also supports a network of 50 chapters across the country. To learn more, visit www.bleeding.org and follow NBDF across social media @nbd_foundation.

BioMarin will revamp its structure to divide into three separate business units of skeletal conditions, enzyme therapies and gene therapy Roctavian. In his nine months as CEO, BioMarin’s new chief Alexander Hardy has so far overseen shake-ups in the company’s leadership team, a slim-down of its R&D program and decisive action on the future of hemophilia A gene therapy Roctavian. But the company still has work to do to achieve what Hardy says could be “one of the most exciting growth stories in biotech.”At an investor event Wednesday, the former Genentech veteran divulged an ambitious plan to quadruple the California-based drugmaker’s patient reach over the next decade and land $4 billion in revenue by 2027.The $4 billion goal would double 2023’s total revenue of $2.4 billion and put BioMarin in the top quartile of biopharma revenue growth, Hardy said.To get there, BioMarin will depend on three separate but connected pillars. The growth aspect largely centers on the company’s Voxzogo, which is already its top growth driver with its sole indication for achondroplasia, or dwarfism. With five more indications planned through 2031, Voxzogo could prove a $5 billion opportunity with a total addressable patient population of around 420,000 across its global markets, according to BioMarin. The next expansion for Voxzogo would be to hypochondroplasia in 2027, which would make it the first approved medicine for the genetic disease, followed by what could be a lucrative idiopathic short stature nod in 2030 and then indications for Noonan syndrome, Turner syndrome and SHOX deficiency on the docket for 2031.The patient pool for the drug’s current dwarfism label is 24,000, but it’s still “only the beginning” for Voxzogo in that indication with more than 80% of the market yet to be penetrated, Hardy pointed out. Even so, the company’s confidence is bolstered by the drug’s standard-of-care status, high compliance rates and a broad age range that can treat patients from infancy, Chief Commercial Officer Cristin Hubbard explained.“Voxzogo is clearly the most exciting multi-indication product in our portfolio and one of the most exciting in the entire industry,” Hardy added. Before Voxzogo came about, much of the company’s revenue was drawn from bread-and-butter enzyme therapies, starting with its very first commercial launch in 2005 of Naglazyme. These days, BioMarin’s five enzyme therapies still bring in more than $1 billion annually and remain the standards of care for some otherwise untreated conditions the drugs target in approximately 80 countries, Hubbard explained.Still, BioMarin looks to further expand the franchise’s reach with efforts to identify more patients, maintain an over 90% patient adherence and invest in emerging markets such as the Middle East and Latin America, Hubbard said.Meanwhile, in business structure, the company has divided itself into three business units: skeletal conditions, enzyme therapies and Roctavian. Gene therapy Roctavian had already been a separate beast from the rest of BioMarin’s portfolio and presented more challenges as well. With a slower-than-expected launch, the company recently decided to cut costs around the drug and focus its efforts in only three markets with no further plans to expand. As a result, Roctavian support staff was separated from the rest of the company’s portfolio, Hardy said during BioMarin’s second-quarter earnings call in August.The wider business structure shuffle, coupled with the Roctavian changes, is translating into 225 layoffs representing 7% of BioMarin’s global workforce, the company announced a few days ago. Another 170 positions were on the chopping block as part of a layoff round disclosed in May.Outside of its approved medicines, BioMarin looks to launch two products by 2027 or 11 by 2034. The company’s pipeline targets diseases such as Duchenne muscular dystrophy and von Willebrand disease, to name a few.As for the shorter-term outlook, BioMarin expects its 2024 revenues to total between $2.75 billion and $2.82 billion. 

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.