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04-08-2025
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BioMarin Reports Strong Second Quarter 2025 Results and Raises Full-year Guidance¹ for Total Revenues, Non-GAAP Operating Margin, and Non-GAAP Diluted EPS
Second Quarter 2025 Total Revenues of $825 million (+16% Y/Y and +17% at Constant Currency Y/Y) Second Quarter 2025 GAAP Diluted Earnings Per Share (EPS) of $1.23 (+124% Y/Y) Second Quarter 2025 Non-GAAP Diluted EPS of $1.44 (+50% Y/Y) BioMarin Completes Acquisition of Inozyme in July 2025; Pivotal Data from Lead Indication Expected 1H'26 BMN 333 Exceeds Targeted Exposures of Free C-type Natriuretic Peptide (CNP) in Healthy Volunteer Study; Pivotal Phase 2/3 Study with BMN 333 in Pediatric Achondroplasia Planned to Begin 1H'26 Conference Call and Webcast Scheduled Today at 4:30 p.m. ET SAN RAFAEL, Calif., Aug. 4, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the second quarter ended June 30, 2025. "We were very pleased with our second quarter performance across all aspects of the business, including strong growth, exciting pipeline progress, and delivery of our business development strategy," said Alexander Hardy, President and Chief Executive Officer of BioMarin. Mr. Hardy continued, "In the quarter, global demand for BioMarin's innovative therapies resulted in double-digit year-over-year revenue growth and significant profitability expansion. In addition to these strong results, today, we are pleased to share early data for BMN 333, a potential new treatment option for children with achondroplasia. BMN 333, our long-acting CNP, achieved our targeted profile in the healthy volunteer study and is now expected to move into the pivotal study in 2026. Our goal is for BMN 333 to demonstrate superiority to VOXZOGO and set a new standard for the treatment of achondroplasia. "We were also pleased to have delivered on our business development strategy with the acquisition of Inozyme, which closed on July 1st," Mr. Hardy added. "The acquisition strengthens our portfolio, adding a late stage enzyme replacement therapy, BMN 401, formerly INZ-701, for the treatment of ENPP1 Deficiency. In conclusion, with the first half of the year now complete, I am pleased with our progress and remain enthusiastic about our potential to deliver for patients, employees and our shareholders through the remainder of 2025 and beyond." 1Excludes the estimated impact of acquired in-process research and development (IPR&D) charges from BioMarin's acquisition of Inozyme Pharma, Inc. (Inozyme), which was completed on July 1, 2025. IPR&D charges are expected to be recorded in BioMarin's financial results in the third quarter of 2025. Second Quarter 2025 Financial Highlights Total Revenues for the second quarter of 2025 were $825 million, an increase of 16% compared to the same period in 2024, driven by strong 20% 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 15% compared to the second quarter of 2024, driven by a combination of increased patient demand in all regions and the timing of large government orders. 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 $134 million to $241 million in the second quarter of 2025 compared to the same period in 2024, an increase of 125%, primarily attributed to higher gross profit driven by the factors noted above. The increase was also attributed to lower Selling, General and Administrative (SG&A) expense due to severance and restructuring costs incurred in 2024 and lower Research and Development (R&D) spend due to re-prioritization of investments associated with the company's portfolio strategy review announced in 2024. These increases were partially offset by higher tax provision primarily due to an increase in taxable income. Non-GAAP Income increased by $93 million to $282 million in the second quarter of 2025 compared to the same period in 2024, representing 49% growth. The increase in Non-GAAP Income was primarily due to higher gross profit driven by the factors noted above. The increase was also attributed to lower R&D spend due to re-prioritization of R&D investments following the company's portfolio strategy review announced in 2024. These increases were partially offset by higher Non-GAAP SG&A spend in 2025 due to ongoing support of business initiatives. Second Quarter 2025 Business Highlights Innovation Skeletal Conditions: BioMarin announced today that Phase 1 data in its healthy volunteer study with BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP), demonstrated area-under-the-curve (AUC) pharmacokinetic (PK) levels greater than three times the levels observed in other long-acting CNP studies. No safety signals were noted. Based on these results, BioMarin is advancing plans to initiate the registration-enabling Phase 2/3 study in the first half of 2026. BMN 333 is on track for a potential 2030 launch, should data be supportive. In May, VOXZOGO data was presented at the Pediatric Endocrine Society (PES) Annual Meeting from the Phase 2 CANOPY clinical studies in younger children that assessed the impact of treatment on tibial bowing, an orthopedic complication and significant cause of pain in children with achondroplasia. Children who received VOXZOGO had a significant reduction in the magnitude of tibial bowing compared to children who received placebo. The study found that this improvement was sustained in children who received VOXZOGO treatment for several years. These findings further reinforce the growing body of evidence supporting VOXZOGO's therapeutic benefits, including improvements in craniofacial development, foramen magnum dimensions, body proportionality, quality of life, and durable increases in growth velocity—all while maintaining bone health and a consistent safety profile. During the quarter, BioMarin continued to advance its CANOPY clinical program studying VOXZOGO in additional indications, including hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency. With VOXZOGO in hypochondroplasia, the company expects to share topline data from its pivotal study in the first half of 2026 and file submissions to global health authorities for approval in the second half of 2026, leading to a potential launch in 2027. Enzyme Therapies: In July 2025, BioMarin completed the acquisition of Inozyme, adding BMN 401 (formerly INZ-701), a potential first-in-disease treatment for ENPP1 Deficiency, to BioMarin's Enzyme Therapies portfolio. Initial pivotal data readout for the ENERGY 3 study in children ages 1–12 years is anticipated in the first half of 2026, with potential launch in 2027. BioMarin is committed to continue working with the patient and HCP communities to identify individuals with ENPP1 Deficiency who may benefit from treatment with BMN 401, in advance of a potential launch. BioMarin is progressing plans to advance BMN 401 for the treatment of ENPP1 Deficiency across additional age groups. The company is also evaluating BMN 401 for potential use in other indications. With PALYNZIQ for the treatment of adolescents between the ages of 12 and 17, BioMarin remains on track to submit applications in the second half of 2025 to expand PALYNZIQ age eligibility in the United States and Europe, with potential approval in 2026. PALYNZIQ is the only enzyme substitute therapy for phenylketonuria (PKU) that lowers physiological blood Phe levels to within the normal range as well as allow for an unrestricted diet, regardless of patient phenotype. 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. For BMN 351, the clinical study is progressing, and the company expects to share initial data by year-end. For BMN 349, the Phase 1 program is advancing, with the Phase 2 study expected to begin in the first half of 2026. During its regular evaluation of R&D programs, BioMarin determined that BMN 390, a pre-clinical candidate for the treatment of PKU, did not meet its target immunogenicity threshold for advancement. The program has been discontinued and employees working on the program have been redeployed within BioMarin. The company remains committed to developing new therapies for people with PKU, with other projects underway across the organization. Growth Total VOXZOGO revenue in the second quarter increased 20% compared to the same period in 2024, driven by strong worldwide demand. As of the end of the quarter, children with achondroplasia in 51 countries around the world were being treated with VOXZOGO, representing strong progress towards the company's target of accessing more than 60 countries by 2027. Global VOXZOGO Y/Y revenue growth in the quarter was led by U.S. contributions, with the majority of new patient starts in the 0-4 year age group. Outside of the U.S. (OUS), Q2 revenue expansion was supported by deeper penetration in previously opened markets and incremental contributions from new country access. Increasing new patient starts, execution of U.S. expansion initiatives, and ongoing OUS build-out are expected to drive higher VOXZOGO revenue in the second half of 2025 compared to the first half of 2025, and weighted to Q4. Total Enzyme Therapies revenues grew 15% in the second quarter Y/Y. PALYNZIQ revenue increased 20% Y/Y, and strength in the quarter was driven by greater numbers of patients titrating to daily maintenance dose and strong adherence. VIMIZIM revenue grew 21% Y/Y in the quarter, driven by both ongoing patient demand and order timing. Total Enzyme Therapies revenues in the second half of 2025 are expected to remain robust, despite typical quarter-to-quarter order timing dynamics in the third and fourth quarters. Value Commitment In the second quarter of 2025, BioMarin delivered expanding margins and increasing profitability. Second quarter GAAP Operating Margin of 33.5% expanded 16.6 percentage points Y/Y while GAAP Diluted EPS of $1.23 increased 124% Y/Y. Second quarter Non-GAAP Operating Margin of 39.9% expanded 8.7 percentage points Y/Y while Non-GAAP Diluted EPS of $1.44 increased 50% Y/Y. These measures of profitability increased at rates faster than revenue growth, reflecting the company's implementation of operational efficiencies. During the second quarter, GAAP and Non-GAAP R&D expenses were lower Y/Y, benefiting from focused R&D investment in prioritized assets following the results of last year's strategic portfolio review. GAAP SG&A decreased Y/Y due to higher 2024 restructuring expenses associated with the company's revamped operating model, and Non-GAAP SG&A increased Y/Y to support the company's ERP implementation and investment in business unit expansion initiatives. Operating expenses are expected to increase in the second half of 2025 vs. the first half of 2025 as clinical and commercial initiatives advance. As a result of the completed acquisition of Inozyme on July 1, 2025, BioMarin expects to account for the transaction as an asset purchase and record the impact of acquired in-process research and development (IPR&D) charges in its third quarter 2025 financial results, which are subject to BioMarin's financial statement closing procedures. The company expects to provide an update on full-year 2025 guidance items, including impact of the acquired IPR&D charges, in its third quarter 2025 earnings update. The company generated operating cash flows totaling $185 million in second quarter 2025, an increase of 55% compared to the same period in 2024. Total cash and investments at the end of the second quarter were approximately $1.9 billion, and increasing operating cash flow is expected to continue, supporting BioMarin's priority of investment in innovation and future growth. Today, BioMarin raised full-year 2025 guidance for Total Revenues, Non-GAAP Operating Margin, and Non-GAAP Diluted EPS. The improved guidance reflects continued growth in patient demand across the portfolio and the company's commitment to generate increasing profitability and cash flow to support reinvestment in innovation and growth. The guidance reflects the impact of tariffs that have already been enacted, and BioMarin expects that any new tariffs would have a limited impact in 2025. 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 June 30,Six Months Ended June 30,20252024% Change20252024% Change Total Revenues $825$71216 %$1,571$1,36115 % Net Product Revenues by Product:VOXZOGO $221$18420 %$435$33729 % Enzyme Therapies:VIMIZIM $215$17821 %$404$3719 % NAGLAZYME 129132(2) %2432382 % PALYNZIQ 1068820 %19916421 % ALDURAZYME 563944 %1057442 % BRINEURA 49459 %89846 % Total Enzyme Therapies Revenue $555$48215 %$1,040$93112 % KUVAN $27$29(7) %$52$65(20) % ROCTAVIAN® $9$729 %$20$8150 % GAAP Net Income $241$107125 %$426$196117 % Non-GAAP Income (1) $282$18949 %$502$32953 % GAAP Operating Margin % (2) 33.5 %16.9 %31.9 %15.4 % Non-GAAP Operating Margin % (1) 39.9 %31.2 %37.9 %27.6 % GAAP Diluted Earnings per Share (EPS) $1.23$0.55124 %$2.19$1.01117 % Non-GAAP Diluted EPS (1) $1.44$0.9650 %$2.57$1.6754 % June 30,2025December 31,2024 Total cash, cash equivalents & investments $ 1,941$ 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) ItemProvided on May 1, 2025Updated August 4, 2025 Total Revenues (1)$3,100to$3,200$3,125to$3,200 Non-GAAP Operating Margin % (2)(4)32 %to33 %33 %to34 % Non-GAAP Diluted EPS (2)(3)(4)$4.20to$4.40$4.40to$4.55 (1) VOXZOGO contribution to full-year 2025 Total Revenues expected to be in the range of $900 million to $935 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. (4) Excludes the estimated impact of acquired IPR&D charges from BioMarin's acquisition of Inozyme, which was completed on July 1, 2025. Accounting for the Inozyme transaction will be finalized and included in the company's third quarter financial results, which are subject to BioMarin's financial statement closing procedures. The company expects to provide an update on full-year 2025 guidance items, including impact of the acquired IPR&D charges, in its third quarter 2025 earnings acquired IPR&D charges may be incurred upon execution of acquisitions, collaborations, licensing agreements, and other business development transactions, BioMarin does not forecast acquired IPR&D charges due to the uncertainty of the future occurrence, magnitude, and timing of these transactions in any given period. BioMarin will host a conference call and webcast to discuss second quarter 2025 financial results today, Monday, August 4, 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 U.S./Canada Dial-in Number: 800-715-9871 Replay Dial-in Number: 800-770-2030 International Dial-in Number: 646-307-1963 Replay International Dial-in Number: 609-800-9909 Conference ID: 6336054 Conference ID: 6336054 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 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 increasing growth and increasing operating expenses in the remainder of 2025, and the underlying drivers of those results, such as the revenue opportunity represented by treatments for Skeletal Conditions, namely VOXZOGO and VOXZOGO's contribution to full-year 2025 Total Revenues, the expected demand and continued growth of BioMarin's Enzyme Therapies portfolio, the anticipated benefits of BioMarin's acquisition of Inozyme Pharma, Inc. and the accounting treatment of such acquisition; 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, BMN 351 and BMN 401 (formerly INZ-701), the anticipated initial data read-out for BMN 351 by year-end, the expected Phase 2 study for BMN 349 in the first half of 2026, the anticipated initial readout for the BMN 401 ENERGY 3 study in the first quarter of 2026 and potential launch in 2027, plans to advance BMN 401 for the treatment of ENPP1 deficiency across additional age groups as well as potential use in other indications, and 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 for a potential launch in 2030; (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, with potential approval and launch in 2026; (iii) expected topline data from the VOXZOGO pivotal study in hypochondroplasia in 2026 and plans to submit applications in 2026 for a potential launch in 2027; (iv) the expectations regarding higher VOXZOGO revenue in the second half of 2025 compared to the first half of 2025 and the underlying assumptions for such expectations; and (v) plans to advance five new VOXZOGO indications with BioMarin's CANOPY clinical program; the expectation that any new tariffs would have limited impact in 2025; expectations for BMN 333's efficacy compared to VOXZOGO's and ability to set a new standard of treatment for achondroplasia; plans to expand VOXZOGO in more than 60 countries by 2027; 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, the impact of new or increased tariffs, other trade protection measures, and escalating trade tensions; 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 Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, 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 McCartyMarni Kottle BioMarin Pharmaceutical Pharmaceutical Inc. (415) 455-7558(650) 374-2803 BIOMARIN PHARMACEUTICAL CONSOLIDATED STATEMENTS OF INCOMEThree and Six Months Ended June 30, 2025 and 2024(In thousands of U.S. dollars, except per share amounts)(Unaudited) Three Months Ended June 30,Six Months Ended June 30,2025202420252024 REVENUES:Net product revenues $ 812,982$ 702,129$ 1,547,626$ 1,339,944 Royalty and other revenues 12,4289,90022,92920,918 Total revenues 825,410712,0291,570,5551,360,862 OPERATING EXPENSES:Cost of sales 150,090130,459301,648255,639 Research and development 161,308183,787320,039388,774 Selling, general and administrative 232,279263,032438,395488,938 Intangible asset amortization 4,84614,2999,69328,597 Gain on sale of nonfinancial assets ———(10,000) Total operating expenses 548,523591,5771,069,7751,151,948 INCOME FROM OPERATIONS 276,887120,452500,780208,914 Interest income 18,82719,78537,84039,150 Interest expense (2,679)(3,574)(5,542)(7,121) Other income (expense), net 4,833(4,527)2,879(3,260) INCOME BEFORE INCOME TAXES 297,868132,136535,957237,683 Provision for income taxes 57,33624,962109,73941,847 NET INCOME $ 240,532$ 107,174$ 426,218$ 195,836 EARNINGS PER SHARE, BASIC $ 1.25$ 0.56$ 2.23$ 1.03 EARNINGS PER SHARE, DILUTED $ 1.23$ 0.55$ 2.19$ 1.01 Weighted average common shares outstanding, basic 191,907190,114191,440189,490 Weighted average common shares outstanding, diluted 197,091200,505196,643200,137 BIOMARIN PHARMACEUTICAL CONSOLIDATED BALANCE SHEETSJune 30, 2025 and December 31, 2024 (In thousands of U.S. dollars, except per share amounts)(Unaudited) June 30, 2025December 31, 2024 ⁽¹⁾ ASSETSCurrent assets:Cash and cash equivalents $ 1,213,816$ 942,842 Short-term investments 218,309194,864 Accounts receivable, net 855,855660,535 Inventory 1,340,1691,232,653 Other current assets 177,183201,533 Total current assets 3,805,3323,232,427 Noncurrent assets:Long-term investments 508,592521,238 Property, plant and equipment, net 1,030,3851,043,041 Intangible assets, net 239,620255,278 Goodwill 196,199196,199 Deferred tax assets 1,427,0211,489,366 Other assets 249,192251,391 Total assets $ 7,456,341$ 6,988,940 LIABILITIES AND STOCKHOLDERS' EQUITYCurrent liabilities:Accounts payable and accrued liabilities $ 684,247$ 606,988 Total current liabilities 684,247606,988 Noncurrent liabilities:Long-term convertible debt, net 596,162595,138 Other long-term liabilities 148,819128,824 Total liabilities 1,429,2281,330,950 Stockholders' equity:Common stock, $0.001 par value: 500,000,000 shares authorized; 192,001,650 and 190,761,349 shares issued and outstanding, respectively 192191 Additional paid-in capital 5,851,6375,802,068 Company common stock held by the Nonqualified Deferred Compensation Plan (11,674)(11,227) Accumulated other comprehensive income (loss) (44,565)61,653 Retained earnings (accumulated deficit) 231,523(194,695) Total stockholders' equity 6,027,1135,657,990 Total liabilities and stockholders' equity $ 7,456,341$ 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 CONSOLIDATED STATEMENTS OF CASH FLOWSSix Months Ended June 30, 2025 and 2024 (In thousands of U.S. dollars)(Unaudited) Six Months Ended June 30,20252024 CASH FLOWS FROM OPERATING ACTIVITIES:Net income $ 426,218$ 195,836 Adjustments to reconcile net income to net cash provided by operating activities:Depreciation and amortization 40,63253,813 Non-cash interest expense 1,3201,981 Accretion of discount on investments (2,717)(4,678) Stock-based compensation 85,231106,163 Gain on sale of nonfinancial assets —(10,000) Impairment of assets 2,96714,204 Deferred income taxes 61,7711,537 Unrealized foreign exchange gain (5,306)(19,958) Other (1,916)(858) Changes in operating assets and liabilities:Accounts receivable, net (156,124)(56,081) Inventory (72,462)(47,409) Other current assets (15,092)1,615 Other assets (13,505)(22,880) Accounts payable and accrued liabilities 3,111(54,261) Other long-term liabilities 5,5376,709 Net cash provided by operating activities 359,665165,733 CASH FLOWS FROM INVESTING ACTIVITIES:Purchases of property, plant and equipment (33,869)(47,431) Maturities and sales of investments 195,738317,649 Purchases of investments (202,433)(195,462) Proceeds from sale of nonfinancial assets —10,000 Purchase of intangible assets (266)(8,512) Net cash provided by (used in) investing activities (40,830)76,244 CASH FLOWS FROM FINANCING ACTIVITIES:Proceeds from exercises of awards under equity incentive plans 7,70736,618 Taxes paid related to net share settlement of equity awards (51,089)(66,739) Other —(60) Net cash used in financing activities (43,382)(30,181) Effect of exchange rate changes on cash (4,479)5,227 NET INCREASE IN CASH AND CASH EQUIVALENTS 270,974217,023 Cash and cash equivalents:Beginning of period $ 942,842$ 755,127 End of period $ 1,213,816$ 972,150 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 June 30,Six Months Ended June 30,2025202420252024 GAAP Reported Net Income $ 241$ 107$ 426$ 196 AdjustmentsStock-based compensation expense - COS 4467 Stock-based compensation expense - R&D 14132634 Stock-based compensation expense - SG&A 30315366 Amortization of intangible assets 5141029 Gain on sale of nonfinancial assets (2) ———(10) Severance and restructuring costs (3) —39—42 Loss on investments (4) —535 Income tax effect of adjustments (11)(24)(22)(39) Non-GAAP Income $ 282$ 189$ 502$ 329 Three Months Ended June 30,20252024DollarPercentageDollarPercentage GAAP Change in Total Revenues $ 11316 %$ 11720 % Adjustment for unfavorable impact of foreign currency exchange rates on product sales denominated in currencies other than U.S. dollars 730 Non-GAAP change in Total Revenues at Constant Currency $ 12017 %$ 14725 %Six Months Ended June 30,20252024DollarPercentageDollarPercentage GAAP Change in Total Revenues $ 21015 %$ 16914 % Adjustment for unfavorable impact of foreign currency exchange rates on product sales denominated in currencies other than U.S. dollars 2153 Non-GAAP change in Total Revenues at Constant Currency $ 23117 %$ 22219 % Three Months Ended June 30,Six Months Ended June 30,2025202420252024R&DSG&AR&DSG&AR&DSG&AR&DSG&A GAAP expenses $ 161$ 232$ 184$ 263$ 320$ 438$ 389$ 489 AdjustmentsStock-based compensation expense (14)(30)(13)(31)(26)(53)(34)(66) Severance and restructuring costs (3) ———(39)———(42) Non-GAAP expenses $ 147$ 203$ 171$ 193$ 294$ 385$ 355$ 381 Three Months Ended June 30,Six Months Ended June 30,2025 Percent of GAAP Total Revenue2024 Percent of GAAP Total Revenue2025 Percent of GAAP Total Revenue2024 Percent of GAAP Total Revenue GAAP Income from Operations $ 277 33.5 %$ 120 16.9 %$ 501 31.9 %$ 209 15.4 % AdjustmentsStock-based compensation expense 48 5.848 6.885 5.4106 7.7 Amortization of intangible assets 5 0.614 2.010 0.629 2.1 Gain on sale of nonfinancial assets (2) — —— —— —(10) (0.7) Severance and restructuring costs (3) — —39 5.5— —42 3.1 Non-GAAP Income from Operations $ 329 39.9 %$ 222 31.2 %$ 596 37.9 %$ 376 27.6 % Three Months Ended June 30,Six Months Ended June 30,2025202420252024 GAAP Diluted EPS $ 1.23$ 0.55$ 2.19$ 1.01 AdjustmentsStock-based compensation expense 0.240.240.430.53 Amortization of intangible assets 0.030.070.050.14 Gain on sale of nonfinancial assets (2) ———(0.05) Severance and restructuring costs (3) —0.20—0.21 Loss on investments (4) —0.020.020.02 Income tax effect of adjustments (0.06)(0.12)(0.11)(0.19) Non-GAAP Diluted EPS (5) $ 1.44$ 0.96$ 2.57$ 1.67 (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 impairment loss on non-marketable equity securities recorded in Other income (expense), net. (5) Non-GAAP Weighted-Average Diluted Shares Outstanding were 197.1 million and 200.5 million shares for the three months ended June 30, 2025 and 2024, respectively, and 196.6 million and 200.1 million shares for the six months ended June 30, 2025 and 2024, respectively, which were equal to the respective GAAP Weighted-Average Diluted Shares Outstanding in the periods presented. View original content to download multimedia: SOURCE BioMarin Pharmaceutical Inc.
Yahoo
23-05-2025
- Business
- Yahoo
Biotech Stock Roundup: BMRN to Acquire INZY, NVAX Gains on Vaccine Approval & More
Mergers and acquisitions grabbed the spotlight in the biotech sector once again this week after BioMarin BMRN agreed to acquire Inozyme Pharma INZY. Meanwhile, other pipeline and regulatory updates were in focus. BioMarin Pharmaceutical announced that it has entered into a definitive agreement to acquire all outstanding shares of the clinical-stage company Inozyme Pharma for $4.00 per share in cash, totaling nearly $270 million. Shares of INZY soared on the news. The deal, approved by the board of directors of both companies, is expected to be closed in the third quarter of 2025. BioMarin will add Inozyme's lead asset, INZ-701, to its pipeline, following the completion of this acquisition. This investigational enzyme replacement therapy (ERT) is currently being evaluated in a pivotal late-stage study for the treatment of a rare genetic disorder, ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency, in children. The deal is a strategic fit for BioMarin, which already markets five first-in-disease enzyme therapies, namely Aldurazyme, Brineura, Naglazyme, Palynziq and Vimizim. The addition of Inozyme's lead candidate will expand BioMarin's Enzyme Therapies portfolio and further diversify its revenue stream. Novavax NVAX announced that the FDA approved its protein-based COVID-19 vaccine, Nuvaxovid. Shares of the company surged on this long-awaited approval from the FDA, albeit with a narrower label than initially expected. While the agency granted full approval to Nuvaxovid for use in older adults aged 65 and above, it restricted the vaccine's use in individuals aged 12-64 with at least one underlying condition that puts them at high risk for severe outcomes from COVID-19. Additionally, the agency requires Novavax to conduct a new Phase IV post-marketing study in individuals aged 50 to 64 without high-risk conditions for severe COVID-19. The full approval is based on data from a pivotal late-stage study, which showed that Nuvaxovid was safe and effective for the prevention of COVID-19. Until now, the vaccine had been available for use under the FDA's emergency use the full approval, Nuvaxovid is the only non-mRNA-based COVID-19 vaccine available in the country. Moderna MRNA announced that it has voluntarily withdrawn a regulatory filing seeking the FDA's approval for mRNA-1083, its investigational mRNA-based combination vaccine against influenza and COVID-19. Per the company, the decision was taken in consultation with the agency. The stock was down on the announcement. This setback derailed the company's initial plans to secure a potential FDA approval for the vaccine before this year's end. Moderna plans to resubmit this filing later this year after vaccine efficacy data from the ongoing phase III study on its investigational seasonal influenza vaccine, mRNA-1010, is available. Interim data from this study is expected this summer. The initial FDA filing, which sought approval for use in older adults aged 50 years and above, was supported by data from a phase III study announced last year. Data from this study showed that mRNA-1083 elicited higher immune responses against influenza and COVID-19 compared with licensed standalone influenza and COVID-19 vaccines. Moderna currently carries a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. Prime Medicine, Inc. PRME announced a strategic restructuring of its business. Shares of the company declined on the news. The restructuring includes the deprioritization of its Chronic Granulomatous Disease ('CGD') programs, as well as a cost and workforce reduction to focus on its liver franchise and programs funded through external partnerships. Prime Medicine is currently advancing in vivo programs to cure two of the largest genetic liver diseases, Wilson's Disease and Alpha-1 Antitrypsin Deficiency ('AATD'). Initial clinical data from both programs are expected in 2027. An investigational new drug (IND) and/or clinical trial application (CTA) is expected to be filed for its Wilson's Disease program in the first half of 2026 and for its AATD program in mid-2026. Initial data from both programs are expected in 2027. Prime Medicine is exploring options for the continued clinical development of PM359 and ceasing further efforts in X-linked CGD. In addition, PRME is reducing its headcount by approximately 25%. The headcount reduction, along with its cost reduction measures and other restructuring efforts, is expected to reduce operating expenses and cash burn, lowering anticipated cash needs by almost half through 2027. Prime Medicine announced that it has recently engaged in binding arbitration proceedings with Beam Therapeutics, Inc. regarding its collaboration agreement. Prime Medicine continues to expect that its cash, cash equivalents and investments, as of March 31, 2025, will be sufficient to fund its operations and capital expenditure requirements into the first half of 2026. The Nasdaq Biotechnology Index has gained 4.56% in the past five trading sessions and GILD's shares have gained 9.49%. In the past six months, shares of MRNA have plunged 32.55%. (See the last biotech stock roundup here: Biotech Stock Roundup: BMY Down on Study Data, Updates From REGN & More) Image Source: Zacks Investment Research Stay tuned for more pipeline updates. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report Moderna, Inc. (MRNA) : Free Stock Analysis Report Novavax, Inc. (NVAX) : Free Stock Analysis Report Prime Medicine, Inc. (PRME) : Free Stock Analysis Report Inozyme Pharma, Inc. (INZY) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research
Business Wire
21-05-2025
- Business
- Business Wire
INOZYME PHARMA INVESTOR ALERT by the Former Attorney General of Louisiana: Kahn Swick & Foti, LLC Investigates Adequacy of Price and Process in Proposed Sale of Inozyme Pharma, Inc.
NEW YORK & NEW ORLEANS--(BUSINESS WIRE)--Former Attorney General of Louisiana Charles C. Foti, Jr., Esq. and the law firm of Kahn Swick & Foti, LLC ('KSF') are investigating the proposed sale of Inozyme Pharma, Inc. (NasdaqGS: INZY) to BioMarin Pharmaceutical Inc. (NasdaqGS: BMRN). Under the terms of the proposed transaction, shareholders of Inozyme will receive $4.00 in cash for each share of Inozyme that they own. KSF is seeking to determine whether this consideration and the process that led to it are adequate, or whether the consideration undervalues the Company. If you believe that this transaction undervalues the Company and/or if you would like to discuss your legal rights regarding the proposed sale, you may, without obligation or cost to you, e-mail or call KSF Managing Partner Lewis S. Kahn ( toll free at any time at 855-768-1857, or visit to learn more. Please note that the transaction is structured as a tender offer, such that time may be of the essence. To learn more about KSF, whose partners include the Former Louisiana Attorney General, visit
Yahoo
20-05-2025
- Business
- Yahoo
BioMarin Offers to Buy Inozyme for $270M to Boost Enzyme Therapy Biz
BioMarin Pharmaceutical BMRN announced that it has entered into a definitive agreement to acquire all outstanding shares of the clinical-stage company Inozyme Pharma INZY for $4.00 per share in cash, totaling nearly $270 million. The deal, approved by the board of directors of both companies, is expected to be closed in the third quarter of 2025. Following the completion of this acquisition, BioMarin will add Inozyme's lead asset, INZ-701, to its pipeline. This investigational enzyme replacement therapy (ERT) is currently being evaluated in a pivotal late-stage study for the treatment of a rare genetic disorder, ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency, in children. This condition affects blood vessels, soft tissues and bones, and is associated with increased cardiovascular mortality risk. Interim results from this late-stage study are expected in early 2026. If this data is positive, a regulatory approval for INZ-701 is expected in 2027, making it the first-in-disease treatment for this indication. Inozyme is also evaluating the ERT in a pivotal study for infants with ENPP1 and plans to initiate a supportive study for adolescents and adults with the disease. Inozyme is also exploring the potential of INZ-701 in separate clinical studies for two more rare disorders, namely ABCC6 Deficiency and calciphylaxis. Like ENPP1, there are currently no approved therapies for either of these disorders. Following this news on Friday, shares of Inozyme surged 178% and those of BioMarin gained about 2%. Year to date, shares of BMRN have lost 10%, while those of INZY have risen 43%. During the same timeframe, the industry has declined 7%. Image Source: Zacks Investment Research In our opinion, the deal is a strategic fit for BioMarin, which already markets five first-in-disease enzyme therapies, namely Aldurazyme, Brineura, Naglazyme, Palynziq and Vimizim. The addition of Inozyme's lead candidate will expand BioMarin's Enzyme Therapies portfolio and further diversify its revenue stream. The deal also benefits INZY as it lacks the commercial infrastructure and expertise needed to bring a drug to market, areas where BioMarin is already well established. BioMarin has reaffirmed its full-year 2025 guidance of $3.1-$3.2 billion for sales and $4.20-$4.40 for adjusted EPS. BioMarin currently carries a Zacks Rank #3 (Hold). BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote A couple of better-ranked stocks from the industry are Adaptive Biotechnologies ADPT and Agenus AGEN, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. In the past 60 days, estimates for Adaptive Biotechnologies' 2025 loss per share have improved from 92 to 87 cents. During the same timeframe, estimates for 2026 loss per share have narrowed from 69 to 65 cents. Adaptive Biotechnologies' earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 21.38%. Shares of ADPT have surged 51% year to date. Estimates for Agenus' 2025 loss per share have narrowed from $7.05 to $2.78 over the past 60 days, and the same for 2026 loss has improved from $7.14 to $5.26. Agenus' earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 22.71%. Year to date, its shares have gained 27%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report Agenus Inc. (AGEN) : Free Stock Analysis Report Adaptive Biotechnologies Corporation (ADPT) : Free Stock Analysis Report Inozyme Pharma, Inc. (INZY) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
19-05-2025
- Business
- Yahoo
BioMarin agrees to acquire Inozyme for $270m
BioMarin Pharmaceutical has agreed to acquire Inozyme Pharma for $4.00 per share in a $270m all-cash transaction. This will enhance BioMarin's enzyme therapy portfolio by adding a late-stage enzyme replacement therapy, INZ-701. Unanimously approved by both companies' boards, the transaction will close in the third quarter of 2025, subject to regulatory clearance, tender offer completion and other customary closing conditions. INZ-701 is being evaluated to treat ectonucleotide pyrophosphatase/ phosphodiesterase 1 (ENPP1) deficiency, a rare genetic condition that impacts blood vessels, bones and soft tissues. This leads to increased cardiovascular mortality risk, particularly in infants, and severe rickets and osteomalacia in the paediatric and adult population. The first Phase III pivotal trial data for the therapy in children is anticipated in early 2026. Regulatory approval is expected in 2027. BioMarin will commence a cash tender offer to purchase all outstanding shares of Inozyme common stock. The board of directors of Inozyme has unanimously recommended that its stockholders tender their shares. The tender offer's completion is contingent upon standard closing conditions, including a majority of the outstanding Inozyme shares being tendered, the Hart-Scott-Rodino Antitrust Improvements Act waiting period expiry or termination, and other customary conditions. After the tender offer, a subsidiary of BioMarin will merge with Inozyme, and the remaining shares not tendered will be converted into the right to obtain the same $4.00 per share in cash. The acquisition is not dependent on financing. Goldman Sachs & Co is the exclusive financial advisor to BioMarin, with Cooley as legal counsel. Centerview Partners is Inozyme's exclusive financial advisor, and Goodwin Procter provides legal counsel. BioMarin Pharmaceutical CEO and president Alexander Hardy stated: "This acquisition brings to BioMarin an important medicine that has the potential to be the first treatment for children and adults with ENPP1 deficiency, improving care for people living with this serious condition. 'As BioMarin continues our transformation and delivers on our corporate strategy, we will continue to evaluate external innovation alongside internal innovation.' In 2024, the US Food and Drug Administration expanded the approval of BioMarin's supplemental biologics licence application (sBLA) for Brineura, an enzyme replacement therapy, to include children under three years of age with neuronal ceroid lipofuscinosis type 2 (CLN2) disease. "BioMarin agrees to acquire Inozyme for $270m" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
