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Fourth Quarter and Full-year 2024 Highlights and 2025 Outlook

Innovation

• Skeletal Conditions: Recently published data highlight the importance of early treatment with VOXZOGO. As published in the December 2024 issue of MED, VOXZOGO contributes to benefits beyond height, a key area of focus for caregivers and health care professionals. With three years of follow-up, VOXZOGO-treated children had statistically significant improvements in body proportionality compared with untreated children of the same age range and gender. VOXZOGO is the only treatment for achondroplasia to have demonstrated, and been published in a peer-reviewed journal, statistically significant improvement in proportionality versus an untreated control arm.
  
  

• In January 2025, new international treatment guidelines were published in the journal Nature Reviews Endocrinology recommending early screening for achondroplasia followed by VOXZOGO treatment soon after diagnosis to provide children with the maximal opportunity for clinical benefit. These guidelines are expected to provide health care professionals and families confidence in VOXZOGO as the treatment of choice when making the important decision to pursue therapy, from infancy, for children with achondroplasia.
  
  

• Also in January 2025, dosing was initiated for the first-in-human study with BMN 333, a long-acting C-type natriuretic peptide (CNP). Initial pharmacokinetic (PK) data is expected by year-end 2025, with detailed data to be presented at a scientific conference in the first half of 2026. BioMarin is encouraged by the pre-clinical profile of BMN 333 in non-human primates, where sustained 100 pM concentrations for free CNP were observed, representing an approximate 2-3 fold increase versus published data in an analogous pre-clinical model for other long-acting CNP analogs.
  
  

• During the quarter, BioMarin also advanced development across its CANOPY clinical program with VOXZOGO in hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency. The pivotal study in hypochondroplasia is expected to complete enrollment in the first half of 2025 and remains on track to launch in 2027.
  
  

• Other Clinical Pipeline Programs: BioMarin's next generation oligonucleotide for Duchenne Muscular Dystrophy, BMN 351, is expected to report initial proof-of-concept data at a scientific congress in the second half of 2025 (including muscle dystrophin levels after 25 weeks of dosing). Results from the phase 3 study with PALYNZIQ in adolescents ages 12-17 are expected in mid-2025 to support potential submissions for age label expansions in the U.S. and Europe in the second half of the year. With BMN 349, an oral therapeutic for Alpha-1 antitrypsin deficiency (AATD)-associated liver disease, the single-ascending dose (SAD) phase of the first-in-human study is complete and dosing in the multiple-ascending dose (MAD) phase of the study began in December 2024.

Growth

• In 2024, strong global demand drove 42% fourth quarter growth and 56% full-year growth for VOXZOGO revenues, compared to the same periods in 2023. VOXZOGO's extensive safety and efficacy profile led more families to begin therapeutic intervention early to potentially improve craniofacial volume, foramen magnum area, body proportionality and quality of life, in addition to durable increases in growth velocity. As of the end of 2024, 47 countries were contributing to VOXZOGO's global revenues, and the company expects VOXZOGO to be available in more than 60 countries by 2027. With its expected growth trajectory, VOXZOGO is on track to achieve a revenue compound annual growth rate (CAGR) exceeding 25% for the period 2023 - 2027.
  
  

• To provide additional insight into the dynamics of VOXZOGO's ongoing global expansion, today BioMarin provided the percentage of actual total VOXZOGO revenues split between the U.S. and the combined contributions from outside of the U.S. (OUS). In fourth quarter 2024, VOXZOGO global revenues were $208 million, including a 26% contribution from the U.S. and a 74% contribution from OUS. In the full year 2024, VOXZOGO global revenues were $735 million, including a 24% contribution from the U.S. and a 76% contribution from OUS.
  
  

• In the U.S., the largest single market opportunity for achondroplasia, the majority of new patient starts in the quarter were for children under the age of 5 years. In 2025, launch momentum across the U.S. is expected to contribute meaningfully to the continued global expansion of VOXZOGO across all ages from infancy.
  
  

• Enzyme Therapies continue to be a significant driver of growth, with revenues increasing 9% in the fourth quarter and 12% for the full-year 2024, compared to the same periods in 2023. 2024 benefited from the timing of ALDURAZYME orders, resulting in 40% Y/Y revenue growth in ALDURAZYME. Enzyme Therapies excluding ALDURAZYME contributed 10% Y/Y growth in full year 2024. Double-digit increases in PALYNZIQ revenue throughout 2024, and solid growth from BioMarin's other enzyme treatments are expected to continue into 2025, with Enzyme Therapies on track toward its long-term growth outlook of high single-digit CAGR.

Value Commitment

• In 2024, BioMarin delivered record performance across the business. Total revenues for the full-year 2024 grew 18%. BioMarin's full-year GAAP Operating Margin of 17.0% expanded 9.3 percentage points Y/Y while GAAP Diluted EPS of $2.21 increased 154% Y/Y. Full-year Non-GAAP Operating Margin of 28.6% expanded 9.2 percentage points Y/Y while Non-GAAP Diluted EPS of $3.52 increased 69% Y/Y. These measures of profitability increased at rates faster than revenue growth, representing the company's focus on operational efficiency.
  
  

• In 2025, the company will continue to implement additional components of the $500 million cost transformation program announced in September of 2024, with full realization of benefits expected in 2026. This ongoing integration of efficiencies in 2025 is expected to enable BioMarin to realize 40% Non-GAAP Operating Margin in 2026.
  
  

• In 2025, full year Total Revenues guidance represents 10% Y/Y growth at the mid-point, expected to be driven by VOXZOGO's continued global expansion and solid demand across Enzyme Therapies. The company is on track to deliver its previously targeted Total Revenues of approximately $4 billion in 2027. Non-GAAP Operating Margin is expected to grow 14% (3.9 percentage points) Y/Y at the mid-point of guidance and Non-GAAP Diluted EPS is expected to increase 22% Y/Y at the mid-point of guidance, as a result of continued strong commercial execution and focus on operational efficiencies across the business.
  
  

• The company generated operating cash flows totaling $573 million in full year 2024, an increase of 260% compared to full year 2023. Total cash and investments at the end of the fourth quarter were approximately $1.7 billion, and with anticipated increasing profitability, BioMarin is positioned to generate increasing operating cash flow into the future, targeting more than $1.25 billion operating cash flows per year starting in 2027.

Forward-Looking Non-GAAP Financial Information

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.

2025 Full-Year Financial Guidance (in millions, except % and EPS amounts)

BioMarin will host a conference call and webcast to discuss fourth quarter and full-year 2024 financial results today, Wednesday, February 19, 2025, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.

About BioMarin

BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin pursues treatments that offer new possibilities for patients and families around the world navigating rare or difficult to treat genetic conditions. To learn more, please visit www.biomarin.com.

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 and Operating Cash Flow for, in certain instances, the full-year 2025 and future periods, as well as double-digit revenue and profitability growth in 2025, and the underlying drivers of those results, such as the revenue opportunity represented by treatments for Skeletal Conditions, namely VOXZOGO, the expected demand and continued growth potential of BioMarin's Enzyme Therapies portfolio, including PALYNZIQ, and the expectation regarding the full realization of the benefits of BioMarin's cost transformation program and the integration of efficiencies; plans regarding BioMarin's revamped corporate strategy and operating model in 2025 and beyond, including expected growth in the Skeletal Conditions business unit and execution of BioMarin's business development strategy, and its anticipated benefits; expectations regarding the Revenue Compound Annual Growth Rate (CAGR) of VOXZOGO and Enzyme Therapies for future periods; the timing of orders for commercial products; BioMarin's ability to meet product demand; 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) expected advancements of pipeline candidates, including BMN 333, BMN 349 and BMN 351, and expected early clinical data read-outs in 2025 for BMN 333 and BMN 351; (ii) expected results from Phase 3 PALYNZIQ study for the treatment of adolescents with phenylketonuria between the ages of 12 and 17 in 2025 and plans to submit applications to expand age eligibility in the U.S. and Europe in the second half of 2025; (iii) plans for the pivotal study in hypochondroplasia to complete enrollment in the first half of 2025 and launch in 2027; (iv) the expectations regarding global expansion of VOXZOGO and that VOXZOGO will be available in more than 60 countries by 2027; (v) plans to advance five new VOXZOGO indications with BioMarin's CANOPY clinical program; and (vi) plans to reach greater number of patients around the world across BioMarin's Enzyme Therapies; the expected benefits and availability of BioMarin's commercial products and product candidates; and potential growth opportunities and trends.

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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 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 R&D expenses and Non-GAAP SG&A expenses are defined by the company as GAAP R&D expenses and GAAP SG&A expenses, respectively, excluding stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. 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. 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 R&D expenses, Non-GAAP SG&A expenses, Non-GAAP Operating Margin percentage, Non-GAAP Diluted EPS, Non-GAAP Weighted-Average 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 financial 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 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:

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SOURCE BioMarin Pharmaceutical Inc.