BioMarin Reports First Quarter 2025 Results and Reaffirms Full-year Guidance

First Quarter 2025 Total Revenues of $745 million (+15% Y/Y and +17% at Constant Currency Y/Y)

First Quarter 2025 GAAP Diluted Earnings Per Share (EPS) of $0.95 (+107% Y/Y)

First Quarter 2025 Non-GAAP Diluted EPS of $1.13 (+59% Y/Y)

Conference Call and Webcast Scheduled Today at 4:30 p.m. ET

SAN RAFAEL, Calif., May 1, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the first quarter ended March 31, 2025.

"During the first quarter, we saw continued high demand for our innovative medicines resulting in strong revenue growth and profitability," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Products in our pipeline also advanced according to plan. In April, we shared positive top-line results from the Phase 3 PALYNZIQ(®) study for the treatment of adolescents with phenylketonuria between the ages of 12 and 17. Also in April, we were pleased to conclude enrollment in the pivotal study in hypochondroplasia with VOXZOGO, keeping us on track to launch in 2027, should data be supportive."

Mr. Hardy continued, "For the remainder of 2025, we look forward to continued momentum in our global expansion of VOXZOGO(®) for achondroplasia. Across our Enzyme Therapies, we plan to build upon strong PALYNZIQ performance in the quarter, as well as initiatives to drive uptake of our other therapies to reach an even greater number of patients around the world. In addition to our strong financial outlook, we expect to advance multiple new indications with VOXZOGO in our CANOPY clinical program, share early clinical results from both BMN 351 for Duchenne Muscular dystrophy and BMN 333, our long-acting C-type natriuretic peptide, as well as execute on our business development strategy. We are delivering strong growth and profitability while we continue to implement BioMarin's new strategy and operating model. We look forward to seeing the benefits of this transformation flow through our results in the coming quarters and beyond."

First Quarter 2025 Financial Highlights

    --  Total Revenues for the first quarter of 2025 were $745 million, an
        increase of 15% compared to the same period in 2024, driven by strong
        40% year-over-year VOXZOGO revenue growth from new patients initiating
        therapy across all regions. In the quarter, revenues from BioMarin's
        Enzyme Therapies (ALDURAZYME(®), BRINEURA(®), NAGLAZYME(®), PALYNZIQ
        and VIMIZIM(®)) increased 8% compared to the first quarter of 2024,
        driven by a combination of increased patient demand and the timing of
        large government orders in all regions. The increase was partially
        offset by lower KUVAN(®) product revenues attributed to continued
        generic competition as a result of the loss of market exclusivity.
    --  GAAP Net Income increased by $97 million to $186 million in the first
        quarter of 2025 compared to the same period in 2024, an increase of
        109%, primarily attributed to higher gross profit driven by the factors
        noted above. The increase was also attributed to lower operating
        expenses following the termination of certain early stage development
        programs following the company's 2024 strategic portfolio review and
        focused ROCTAVIAN strategy announced in the second half of 2024. These
        increases were partially offset by higher tax provision primarily due to
        increase in taxable income.
    --  Non-GAAP Income increased by $81 million to $221 million in the first
        quarter of 2025 compared to the same period in 2024, representing 58%
        growth. The increase in Non-GAAP Income was primarily due to the factors
        noted above.

First Quarter 2025 Business Highlights

Innovation

    --  Skeletal Conditions: In March 2025, BioMarin presented new data
        demonstrating favorable safety and strong adherence in real-world
        clinical practice with VOXZOGO in children with achondroplasia under the
        age of 3 years old at the 2025 American College of Medical Genetics and
        Genomics (ACMG) Annual Meeting. No treatment-related adverse events nor
        any dose interruptions were reported among 63 children followed for up
        to 23.7 months. These real-world findings further validate VOXZOGO's
        established safety profile and reinforce the therapeutic benefit seen in
        clinical studies. The study's safety results, including in infants as
        young as 1 month old, add to the growing body of evidence supporting
        early treatment initiation with VOXZOGO, consistent with new
        international treatment guidelines published in the journal Nature
        Reviews Endocrinology earlier this year.
    --  In April 2025, BioMarin completed enrollment in its pivotal Phase 3
        study with VOXZOGO in hypochondroplasia and the company is on track to
        share topline data in 2026, with a potential launch in 2027. BioMarin
        plans to leverage its multiyear track record treating children with
        achondroplasia, a related condition, to raise awareness and treat
        children with hypochondroplasia across the globe. The CANOPY clinical
        program is continuing to advance VOXZOGO in additional new indications,
        including idiopathic short stature, Noonan syndrome, Turner syndrome,
        and SHOX deficiency.
    --  With BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP),
        the company enrolled multiple cohorts of healthy volunteers in its
        first-in-human study, with initial pharmacokinetic (PK) data expected by
        year-end. Detailed data from this study is expected to be presented at a
        scientific forum in the first half of 2026. Pre-clinical data with BMN
        333 demonstrated sustained 100 pM concentrations for free CNP,
        representing an approximate 2-3 fold increase versus published data in
        an analogous pre-clinical model for other long-acting CNP analogs.
    --  Additionally, BioMarin recently met with FDA and reached agreement on an
        overall clinical development plan for BMN 333 in achondroplasia.
        Assuming the Phase 1 data are supportive, the company plans to initiate
        a registration-enabling study in 2026, supporting a previously disclosed
        target for 2030 approval. BioMarin plans to seek similar agreements with
        additional global regulators in the coming months.
    --  The company announced in April that its pivotal study with PALYNZIQ for
        the treatment of adolescents between the ages of 12 and 17 met its
        primary efficacy endpoint, demonstrating a statistically significant
        lowering in blood Phe levels. These data will support the planned
        submission of applications in the second half of 2025 to expand PALYNZIQ
        age eligibility in the United States and Europe.
    --  Other Clinical Pipeline Programs: BMN 351, BioMarin's next generation
        oligonucleotide for Duchenne Muscular Dystrophy, and BMN 349, an oral
        therapeutic for Alpha-1 antitrypsin deficiency (AATD)-associated liver
        disease, continue to advance. Initial data for BMN 351 is anticipated to
        be presented at a scientific congress in the second half of 2025
        (including muscle dystrophin levels from the 6 mg/kg cohort after 25
        weeks of dosing).
    --  During a recent strategic portfolio assessment of R&D programs, BioMarin
        determined that the evolving profile for BMN 370, a pre-clinical
        candidate for the treatment of von Willebrand disease, did not meet its
        threshold for further development and commercialization. The program has
        been discontinued and impacted employees have been redeployed within
        BioMarin.

Growth

    --  Total VOXZOGO revenue in the first quarter increased 40% compared to the
        same period in 2024, representing continued strong global demand since
        its commercial launch in 2021. As of the end of the quarter, children
        with achondroplasia in 49 countries around the world were being treated
        with VOXZOGO.
    --  In the U.S., BioMarin is investing in focused initiatives to drive
        continued expansion. These efforts include increasing field personnel to
        broaden the prescriber base and adding awareness platforms to drive
        adoption of VOXZOGO treatment. This is expected to begin increasing the
        rate of U.S. expansion in the second half of the year. Outside of the
        U.S. (OUS), from where the majority of VOXZOGO revenue is generated,
        uneven ordering patterns, consistent with BioMarin's other brands, were
        observed. This OUS dynamic is expected to result in VOXZOGO full-year
        revenues being more weighted towards the second half of 2025.
    --  Enzyme Therapies revenues grew 8% in the first quarter Y/Y, driven by
        strong continued demand for PALYNZIQ. Strong PALYNZIQ performance as
        well as solid growth from BioMarin's other enzyme treatments are
        expected to continue throughout 2025.

Value Commitment

    --  In the first quarter of 2025, BioMarin delivered strong results across
        the business. Total revenues for the first quarter grew 15% Y/Y. First
        quarter GAAP Operating Margin of 30.0% expanded 16.4 percentage points
        Y/Y while GAAP Diluted EPS of $0.95 increased 107% Y/Y. First quarter
        Non-GAAP Operating Margin of 35.7% expanded 11.9 percentage points Y/Y
        while Non-GAAP Diluted EPS of $1.13 increased 59% Y/Y. These measures of
        profitability increased at rates faster than revenue growth,
        representing the company's focus on operational efficiency.
    --  During the quarter, BioMarin continued to realize the benefits of cost
        transformation initiatives implemented in 2024, resulting in a decrease
        in GAAP and Non-GAAP R&D and SG&A expenses Y/Y. Throughout the remainder
        of 2025, BioMarin expects to increase investments in VOXZOGO indication
        expansion, clinical pipeline development, and commercialization
        initiatives supporting the company's Skeletal Conditions and Enzyme
        Therapies business units.
    --  The company generated operating cash flows totaling $174 million in
        first quarter 2025, an increase of 271% compared to first quarter 2024.
        Total cash and investments at the end of the first quarter were
        approximately $1.8 billion, and with anticipated increasing
        profitability, BioMarin is positioned to generate increasing operating
        cash flow into the future.
    --  Today, the company reaffirmed its previously communicated 2025 full-year
        financial guidance, which reflects the impact of tariffs that have
        already been enacted but does not reflect the impact of potential future
        pharmaceutical tariffs. BioMarin has immaterial exposure to U.S. tariffs
        for China, Mexico and Canada across its global supply chain operations
        and product sales.


     
              Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)




                                                                                                                Three Months Ended

                                                                                                      
         
            March 31,


                                                                                              2025         2024                    % Change





     
              Total Revenues                                                               $745         $649                        15 %







     
              Net Product Revenues by Product:



     
              VOXZOGO                                                                      $214         $153                        40 %





     
              Enzyme Therapies:



     VIMIZIM                                                                                 $188         $193                       (3) %



     NAGLAZYME                                                                                114          106                         8 %



     PALYNZIQ                                                                                  93           76                        22 %



     ALDURAZYME                                                                                49           35                        40 %



     BRINEURA                                                                                  40           39                         3 %



     
              Total Enzyme Therapies Revenue                                               $484         $449                         8 %





     
              KUVAN                                                                         $25          $36                      (31) %



     
              ROCTAVIAN(R)                                                                  $11           $1                     1,000 %





     GAAP Net Income                                                                         $186          $89                       109 %



     Non-GAAP Income (1)                                                                     $221         $140                        58 %



     GAAP Operating Margin % (2)                                                           30.0 %      13.6 %



     Non-GAAP Operating Margin % (1)                                                       35.7 %      23.8 %



     GAAP Diluted Earnings per Share (EPS)                                                  $0.95        $0.46                       107 %



     Non-GAAP Diluted EPS (1)                                                               $1.13        $0.71                        59 %


                                                 March 31, December 31,
                                                      2025          2024



     Total cash, cash equivalents & investments    $1,779        $1,659


     (1)   Refer to Non-GAAP Information beginning on page 10 of this press release for definitions of Non-GAAP Income, Non-GAAP Operating Margin
              percentage and Non-GAAP Diluted EPS along with the related reconciliations to the comparable information reported under U.S. GAAP.




     (2) 
     GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations divided by Total Revenues.

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)



     
                Item                      2025 Guidance Reaffirmed



     Total Revenues (1)              $3,100      to          $3,200



     Non-GAAP Operating Margin % (2)   32 %     to            33 %



     Non-GAAP Diluted EPS (2)(3)      $4.20      to           $4.40


     (1)   VOXZOGO contribution to full-year 2025 Total Revenues expected to be in the range of $900 million to $950 million.



     (2)   Refer to Non-GAAP Information beginning on page 10 of this press release for definitions of Non-GAAP Operating Margin
              and Non-GAAP Diluted EPS.




     (3) 
     Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted Shares Outstanding.

BioMarin will host a conference call and webcast to discuss first quarter 2025 financial results today, Thursday, May 1, 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.



     U.S./Canada Dial-in Number: 888-596-4144      Replay Dial-in Number: 800-770-2030



     International Dial-in Number:  646-968-2525   Replay International Dial-in Number: 609-
                                                     800-9909



     Conference ID:  4327591                     
     Conference ID: 4327591

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 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 of BioMarin's Enzyme Therapies portfolio, including PALYNZIQ, and the expectation regarding the full realization of the benefits of BioMarin's cost transformation program; 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; 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, the anticipated initial data read-out for BMN 351 in the second half of 2025, the expected data and data presentation for BMN 333 in the first half of 2026 and plans to initiate a registration-enabling study for BMN 333 in 2026, as well as plans to seek similar agreements with additional global regulators in the coming months; (ii) plans to submit applications to expand PALYNZIQ age eligibility for the treatment of adolescents with phenylketonuria between the ages of 12 and 17 in the U.S. and Europe in the second half of 2025; (iii) expected topline data from the pivotal study in hypochondroplasia in 2026 and launch in 2027; (iv) the expectations regarding global expansion of VOXZOGO for achondroplasia and expected increase in the rate of U.S. expansion in the second half of 2025; (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, such as trade wars and potential future pharmaceutical tariffs; 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 U.S. 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 fiscal year ended December 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.



     
     Contact:



     
     Investors:                                    Media:



     
     Traci McCarty                           Marni Kottle



     
     BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical
                                                        Inc.



     
     (415) 455-7558                        (650) 374-2803


                                     
              
                BIOMARIN PHARMACEUTICAL INC.


                              
              
                CONDENSED CONSOLIDATED STATEMENTS OF INCOME


                        
              
                Three Months Ended March 31, 2025
                 and 2024


                       
              
                (In thousands of U.S. dollars, except per share amounts)


                                            
              
                (Unaudited)




                                                                                                                   Three Months Ended

                                                                                                        
           
         March 31,


                                                                                                              2025                2024





     
                REVENUES:



     Net product revenues                                                                                $734,644            $637,815



     Royalty and other revenues                                                                            10,501              11,018



     Total revenues                                                                                       745,145             648,833



     
                OPERATING EXPENSES:



     Cost of sales                                                                                        151,558             125,180



     Research and development                                                                             158,731             204,987



     Selling, general and administrative                                                                  206,116             225,906



     Intangible asset amortization                                                                          4,847              14,298



     Gain on sale of nonfinancial assets                                                                        -           (10,000)



     Total operating expenses                                                                             521,252             560,371



     
                INCOME FROM OPERATIONS                                                                  223,893              88,462





     Interest income                                                                                       19,013              19,365



     Interest expense                                                                                     (2,863)            (3,547)



     Other income (expense), net                                                                          (1,954)              1,267



     
                INCOME BEFORE INCOME TAXES                                                              238,089             105,547



     Provision for income taxes                                                                            52,403              16,885



     
                NET INCOME                                                                             $185,686             $88,662



     
                EARNINGS PER SHARE, BASIC                                                                 $0.97               $0.47



     
                EARNINGS PER SHARE, DILUTED                                                               $0.95               $0.46



     Weighted average common shares outstanding, basic                                                    190,967             188,866



     Weighted average common shares outstanding, diluted                                                  196,474             199,262


                                                                                                                         
              
                BIOMARIN PHARMACEUTICAL INC.


                                                                                                                     
              
                CONDENSED CONSOLIDATED BALANCE SHEETS


                                                                                                                     
              
                March 31, 2025
                 and 2024


                                                                                                           
              
                (In thousands of U.S. dollars, except per share amounts)


                                                                                                                                  
              
                (Unaudited)




                                                                                                                                                                                            March 31, 2025 December 31, 2024
                                                                                                                                                                                                                  ?(1)?


                                                                                                                                   
              
                ASSETS



     Current assets:



     Cash and cash equivalents                                                                                                                                                                 $1,048,803           $942,842



     Short-term investments                                                                                                                                                                       223,532            194,864



     Accounts receivable, net                                                                                                                                                                     739,177            660,535



     Inventory                                                                                                                                                                                  1,274,848          1,232,653



     Other current assets                                                                                                                                                                         181,545            201,533



     Total current assets                                                                                                                                                                       3,467,905          3,232,427



     Noncurrent assets:



     Long-term investments                                                                                                                                                                        506,724            521,238



     Property, plant and equipment, net                                                                                                                                                         1,032,613          1,043,041



     Intangible assets, net                                                                                                                                                                       247,346            255,278



     Goodwill                                                                                                                                                                                     196,199            196,199



     Deferred tax assets                                                                                                                                                                        1,460,566          1,489,366



     Other assets                                                                                                                                                                                 235,654            251,391



     Total assets                                                                                                                                                                              $7,147,007         $6,988,940


                                                                                                                    
              
                LIABILITIES AND STOCKHOLDERS' EQUITY



     Current liabilities:



     Accounts payable and accrued liabilities                                                                                                                                                    $628,213           $606,988



     Total current liabilities                                                                                                                                                                    628,213            606,988



     Noncurrent liabilities:



     Long-term convertible debt, net                                                                                                                                                              595,650            595,138



     Other long-term liabilities                                                                                                                                                                  129,685            128,824



     Total liabilities                                                                                                                                                                          1,353,548          1,330,950



     Stockholders' equity:



     Common stock, $0.001 par value: 500,000,000 shares authorized; 191,755,802 and 190,761,349 shares issued and outstanding, respectively                                                           192                191



     Additional paid-in capital                                                                                                                                                                 5,794,302          5,802,068



     Company common stock held by the Nonqualified Deferred Compensation Plan                                                                                                                    (11,177)          (11,227)



     Accumulated other comprehensive income (loss)                                                                                                                                                 19,151             61,653



     Accumulated deficit                                                                                                                                                                          (9,009)         (194,695)



     Total stockholders' equity                                                                                                                                                                 5,793,459          5,657,990



     Total liabilities and stockholders' equity                                                                                                                                                $7,147,007         $6,988,940


     (1) December 31, 2024 balances were derived from the audited Consolidated Financial Statements included in the company's Annual Report on Form 10-K
            for the year ended December 31, 2024, filed with the SEC on February 24, 2025.


                                                              
              
                BIOMARIN PHARMACEUTICAL INC.


                                                    
              
                CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS


                                                
              
                Three Months Ended March 31, 2025
                 and 2024


                                                             
              
                (In thousands of U.S. dollars)


                                                                    
              
                (Unaudited)




                                                                                                                                           Three Months Ended March 31,


                                                                                                                                      2025               2024



     
                CASH FLOWS FROM OPERATING ACTIVITIES:



     Net income                                                                                                                  $185,686            $88,662



     Adjustments to reconcile net income to net cash provided by operating activities:



     Depreciation and amortization                                                                                                 22,069             27,350



     Non-cash interest expense                                                                                                        660                990



     Accretion of discount on investments                                                                                         (1,362)           (2,502)



     Stock-based compensation                                                                                                      37,700             58,249



     Gain on sale of nonfinancial assets                                                                                                -          (10,000)



     Impairment of assets                                                                                                           2,967



     Deferred income taxes                                                                                                         28,429                285



     Unrealized foreign exchange gain                                                                                            (10,026)          (10,804)



     Other                                                                                                                        (1,267)               127



     Changes in operating assets and liabilities:



     Accounts receivable, net                                                                                                    (57,590)           (3,386)



     Inventory                                                                                                                   (24,335)          (16,820)



     Other current assets                                                                                                         (6,327)          (17,353)



     Other assets                                                                                                                 (1,624)          (12,130)



     Accounts payable and accrued liabilities                                                                                     (2,655)          (59,006)



     Other long-term liabilities                                                                                                    2,069              3,309



     Net cash provided by operating activities                                                                                    174,394             46,971



     
                CASH FLOWS FROM INVESTING ACTIVITIES:



     Purchases of property, plant and equipment                                                                                  (16,768)          (26,104)



     Maturities and sales of investments                                                                                           77,804            131,533



     Purchases of investments                                                                                                    (89,274)         (121,665)



     Proceeds from sale of nonfinancial assets                                                                                          -            10,000



     Purchase of intangible assets                                                                                                      -           (8,000)



     Net cash used in investing activities                                                                                       (28,238)          (14,236)



     
                CASH FLOWS FROM FINANCING ACTIVITIES:



     Proceeds from exercises of awards under equity incentive plans                                                                     -             7,197



     Taxes paid related to net share settlement of equity awards                                                                 (38,779)          (49,948)



     Other                                                                                                                              -              (42)



     Net cash used in financing activities                                                                                       (38,779)          (42,793)



     Effect of exchange rate changes on cash                                                                                      (1,416)             1,927



     
                NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS                                                            105,961            (8,131)



     Cash and cash equivalents:



     Beginning of period                                                                                                         $942,842           $755,127



     End of period                                                                                                             $1,048,803           $746,996

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:


         
              
                Reconciliation of GAAP Reported Information to Non-GAAP Information (1)


                   
              
                (In millions of U.S. dollars, except per share data)


                                       
              
                (unaudited)




                                                                                                              Three Months Ended

                                                                                                              March 31,


                                                                                                         2025      2024





     
                GAAP Reported Net Income                                                              $186       $89



     Adjustments



     Stock-based compensation expense - COS                                                                2         3



     Stock-based compensation expense - R&D                                                               12        21



     Stock-based compensation expense - SG&A                                                              23        34



     Amortization of intangible assets                                                                     5        14



     Gain on sale of nonfinancial assets (2)                                                               -     (10)



     Severance and restructuring costs (3)                                                                 -        3



     Loss on investments (4)                                                                               3



     Income tax effect of adjustments                                                                   (10)     (15)



     Non-GAAP Income                                                                                    $221      $140


                                                                                                                                                     
       
                Three Months Ended

                                                                                                                                                       
         
                March 31,


                                                                                                                                                             2025                                 2024


                                                                                                                                              Dollar      Percentage                     Dollar  Percentage



     
                GAAP Change in Total Revenues                                                                                                 $96             15 %                        $52          9 %



     Adjustment for unfavorable impact of foreign currency exchange rates on product sales denominated in currencies other than U.S. dollars     14                                          23



     Non-GAAP change in Total Revenues at Constant Currency                                                                                    $110             17 %                        $75         13 %


                                                 
       
             Three Months Ended

                                                   
       
                March 31,


                                                       2025                              2024


                                             R&D          SG&A                     R&D        SG&A





     
                GAAP expenses            $159           $206                     $205         $226



     Adjustments



     Stock-based compensation expense      (12)          (23)                    (21)        (34)



     Severance and restructuring costs (3)                                                    (3)



     Non-GAAP expenses                     $147           $183                     $184         $188


                                                                    Three Months Ended

                                                      
         
                March 31,


                                               2025         Percent         2024               Percent
                                                    of GAAP                            of GAAP
                                                     Total                              Total
                                                    Revenue                            Revenue





     
                GAAP Income from Operations $224          30.0 %          $88                13.6 %



     Adjustments



     Stock-based compensation expense           37             5.0            58                   9.0



     Amortization of intangible assets           5             0.7            14                   2.2



     Gain on sale of nonfinancial assets (2)                              (10)                (1.5)



     Severance and restructuring costs (3)                                   3                   0.5



     Non-GAAP Income from Operations          $266          35.7 %         $154                23.8 %


                                                     Three Months Ended

                                                     March 31,


                                                2025     2024





     
                GAAP Diluted EPS            $0.95    $0.46



     Adjustments



     Stock-based compensation expense          0.19     0.29



     Amortization of intangible assets         0.03     0.07



     Gain on sale of nonfinancial assets (2)         (0.05)



     Severance and restructuring costs (3)             0.02



     Loss on investments (4)                   0.02



     Income tax effect of adjustments        (0.05)  (0.08)



     Non-GAAP Diluted EPS (5)                 $1.13    $0.71


     (1) 
     Certain amounts may not sum or recalculate due to rounding.



     (2)   Represents a payment triggered by a third party's attainment of a regulatory approval milestone related to previously sold intangible assets.



     (3)   These amounts were included in SG&A and represent severance and restructuring costs related to the Company's 2024 corporate initiatives and the
              associated organizational redesign efforts.



     (4) 
     Represents an impairment loss on non-marketable equity securities recorded in Other income (expense), net.



     (5)   Non-GAAP Weighted-Average Diluted Shares Outstanding were 196.5 million and 199.3 million shares for the three months ended March 31, 2025 and
              2024, respectively, which were equal to the respective GAAP Weighted-Average Diluted Shares Outstanding in the periods presented.

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SOURCE BioMarin Pharmaceutical Inc.