• Second Quarter 2024 Total Revenues of $712 million (+20% Y/Y and +25% at Constant Currency Y/Y); First Half 2024 Total Revenues of $1.36 billion (+14% Y/Y and +19% at Constant Currency Y/Y)
• Second Quarter 2024 GAAP Diluted EPS of $0.55 (+90% Y/Y); First Half 2024 GAAP Diluted EPS of $1.01 (+80% Y/Y)
• Second Quarter 2024 Non-GAAP Diluted Earnings per Share (EPS) of $0.96 (+78% Y/Y); First Half 2024 Non-GAAP Diluted EPS of $1.67 (+46% Y/Y)
• Strong VOXZOGO^® Demand in the Quarter Resulted in 73% Y/Y Increase in the Number of Children Receiving Treatment
• Conference Call and Webcast Scheduled Today at 4:30 p.m. ET

SAN RAFAEL, Calif., Aug. 5, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the quarter and six months ended June 30, 2024.

"Strong execution across our business resulted in record double-digit revenue growth in the second quarter and first half of 2024. These top-line results, along with a focus on operational efficiency, resulted in a 78% year-over-year improvement in quarterly non-GAAP earnings per share," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Strong global demand for VOXZOGO led to nearly 900 new patient starts in the first half of 2024, the highest in VOXZOGO's history. Record VOXZOGO contributions in the quarter, driven by patient growth in all geographies, combined with double-digit growth from our enzyme therapies drove today's increased 2024 full-year guidance."

Mr. Hardy added, "During the quarter, global demand for VOXZOGO continued to increase as more families sought treatment with the only approved and genetically-targeted medicine for achondroplasia. Approximately 3,500 children were receiving VOXZOGO by the end of the second quarter, with more than half of new treatment starts in the United States for children under the age of 5. We are seeing strong interest from families in the United States seeking VOXZOGO treatment for their children and we expect this market to continue to drive significant expansion over the coming quarters." Mr. Hardy continued, "With VOXZOGO's safety and efficacy well-established, based on nearly 6,000 patient years of demonstrated and durable evidence, we are confident in our rapidly expanding leadership. Enrollment in our pivotal study with VOXZOGO for the treatment of hypochondroplasia is proceeding well, and enrollment in our separate studies for idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency are advancing this year as planned, following alignment with health authorities."

Financial Highlights:

• Total Revenues for the second quarter of 2024 were $712.0 million, an increase of 20%, compared to the same period in 2023, driven by strong VOXZOGO contributions from new patient starts, high compliance rates, and customer stock levels normalizing facilitated by ample supply. In the quarter, demand across BioMarin's enzyme therapies (VIMIZIM^®, NAGLAZYME^®, ALDURAZYME^®, BRINEURA^® and PALYNZIQ^®) drove 15% growth compared to the second quarter of 2023. This increase was partially driven by NAGLAZYME product revenues due to the timing of large government orders in certain regions outside the United States and higher PALYNZIQ revenues in the United States driven by new patient starts. Partially offsetting the increase were lower KUVAN^® product revenues attributed to continued generic competition as a result of the loss of market exclusivity.
  
  
• GAAP Net Income increased by $51.2 million to $107.2 million in the second quarter of 2024 compared to the same period in 2023. The increase was primarily due to higher gross profit driven by the factors noted above. The increase was partially offset by higher spend in Selling, General and Administrative (SG&A), primarily due to severance and other restructuring costs associated with the Company's portfolio strategy review and the associated organizational redesign efforts announced in the second quarter of 2024.
  
  
• Non-GAAP Income increased by $83.7 million to $188.9 million in the second quarter of 2024 compared to the same period in 2023. The increase in Non-GAAP Income was primarily due to higher gross profit, partially offset by higher SG&A expenses primarily related to sales and marketing activities for VOXZOGO, higher partner distribution fees, incremental administrative expenses, as well as higher Research and Development (R&D) expenses related to expansion into new VOXZOGO indications and our prioritized pipeline products.

2Q Update on 2024 Strategic Priorities

In the second quarter, BioMarin continued to deliver 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 second quarter, the number of children with achondroplasia benefiting from VOXZOGO treatment increased to approximately 3,500 across 44 countries. Global access to VOXZOGO from infancy had a significant impact on uptake as families pursued maximum therapeutic benefit by starting treatment early.
  
  
• In the U.S., the largest potential market, the majority of new patient starts in the quarter were for children under the age of 5 years. VOXZOGO's extensive safety and efficacy profile led more families to begin therapeutic intervention early to potentially impact greater improvements in craniofacial growth, foramen magnum compression, body proportionality and quality of life, in addition to durable increases in growth velocity.
  
  
• During the quarter, new, multi-year data demonstrating that treating achondroplasia with VOXZOGO improved health related quality of life and proportionality, which are important benefits, beyond height, for affected children and their families. These results were presented at the Pediatric Endocrine Society (PES) annual meeting (press release), and the 2024 International Conference on Children's Bone Health (ICCBH) (press release). In addition, positive results with VOXZOGO from Phase 2 in children with Noonan Syndrome, idiopathic short stature and other growth-related conditions were presented.
  
  
  • At PES on May 4, results from a Phase 3 extension study in children with achondroplasia who began VOXZOGO treatment at 10 years of age or older, demonstrated that meaningful height gains were observed despite the age of participants at treatment initiation. Results from a Phase 2 extension study showed that VOXZOGO maintained positive effects on linear growth over 4 years in children who began treatment under age five. Results from an investigator-sponsored Phase 2 in children 3-11 years old with several genetic growth-related conditions demonstrated marked improvement in annualized growth velocity and height standard deviation across all conditions studied.
    
    
  • At ICCBH on June 17, a new investigator-led study showed VOXZOGO significantly increased bone length while maintaining bone strength through 5 years of observation in children with achondroplasia, an essential functional outcome for children receiving multi-year treatment. Phase 2 and Phase 3 data on VOXZOGO demonstrated durable safety and efficacy, and improvements on proportionality and health-related quality of life in children with achondroplasia.
    
    
• Building on its leadership in achondroplasia, BioMarin's clinical programs across multiple new growth-related conditions are underway. The pivotal study with VOXZOGO for hypochondroplasia is actively enrolling. Clinical programs in idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency are all on track for enrollment later this year.
  
  
• During the second quarter, through extensive supply chain efforts, BioMarin secured ample VOXZOGO supply to support patient demand worldwide. As of the end of the quarter, the company was able to provide VOXZOGO supply to new patients immediately, as identified. Additionally, the company has ample supply capacity to support all ongoing VOXZOGO clinical programs in hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency, as well as expected commercial demand.

Establish ROCTAVIAN^® opportunity

• Today, the company announced its updated strategy for ROCTAVIAN. The strategy will enable ROCTAVIAN to contribute to BioMarin's long-term profitability. By focusing commercial, research and manufacturing programs in three prioritized countries, including the United States, Germany and Italy, BioMarin anticipates reducing annual direct ROCTAVIAN expenses to approximately $60 million, beginning in 2025. The company has already begun to operationalize the reduction of ROCTAVIAN expenses this year to achieve $60 million in expenses beginning in full-year 2025. As a result of these changes, the company expects ROCTAVIAN to be profitable by the end of 2025.
  
  
• During the quarter, BioMarin treated 3 patients in the U.S. and 2 in Italy, generating $7 million in revenue. BioMarin's global commercial team will continue to focus on key elements critical to supporting ROCTAVIAN uptake in with the U.S., Germany and Italy.

Focus R&D on the most promising assets

• During the quarter, BioMarin progressed development of its prioritized pipeline products: BMN 351, BMN 349, and BMN 333. BMN 351, BioMarin's next generation oligonucleotide for Duchenne Muscular Dystrophy, has completed enrollment of the first dose cohort, with data expected by year-end. With BMN 349, a potential best-in-class, oral therapeutic for Alpha-1 antitrypsin deficiency (AATD)-associated liver disease, the company will begin enrolling its first-in-human study with healthy volunteers later this year. BMN 333, a long-acting C-type natriuretic peptide (CNP) for multiple growth disorders, is completing IND-enabling activities and is expected to enter the clinic in early 2025.
  
  
• During the quarter, the company chose to discontinue development of BMN 293, a gene therapy for hypertrophic cardiomyopathy. Applying its focused approach to investing in only those assets that have the highest potential impact for patients, the time and resources anticipated to bring BMN 293 through development and to market no longer met BioMarin's high bar for advancement.

Accelerate EPS growth and expand margins

• Sustained strong performance in the second quarter highlighted BioMarin's ongoing execution of its financial strategy to drive year-over-year Non-GAAP Operating Margin expansion and Non-GAAP EPS growth faster than revenues.
  
  
• The company raised full-year 2024 guidance for Total Revenues, Non-GAAP Operating Margin, and Non-GAAP Diluted EPS. The improved guidance reflects the underlying strength of enzyme products and continued high demand for VOXZOGO, along with BioMarin's commitment to accelerate profitability while continuing to invest in innovation. 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.

Investor Day to be held Wednesday, September 4th. Event details will be available later in August.

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 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.

BioMarin will host a conference call and webcast to discuss second quarter 2024 financial results today, Monday, August 5, 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 www.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 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 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, and Non-GAAP Diluted EPS for the full-year 2024 and the underlying drivers of those results; 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 and the associated organizational redesign efforts; BioMarin's updated 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; 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) the potential to leverage VOXZOGO in conditions beyond achondroplasia, such as hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome, SHOX deficiency and other genetic short stature pathway conditions, (ii) the expected expansion of VOXZOGO in the U.S. and the anticipated start and growth of commercial sales of VOXZOGO in additional countries, (iii) the commercialization of ROCTAVIAN for the treatment of severe hemophilia A in the U.S., Germany and Italy, (iv) BioMarin's expectation to receive data regarding the first dose cohort for BMN 351 by year-end, (v) BioMarin's plans to enroll its first-in-human study with BMN 349 later this year, and (vi) BioMarin's plans to initiate a clinical program with BMN 333 in early 2025; 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 Quarterly Report on Form 10-Q for the quarter ended March 31, 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 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: