Laronidase

Second Quarter 2026 Total Revenues Increased 20% Year-over-year to $990 million Stronger Growth Expectations Drive Increased Guidance for Full-year 2026 Total Revenues, VOXZOGO®, and Non-GAAP Diluted Earnings Per Share (EPS) Addition of GALAFOLD® and POMBILITI® + OPFOLDA®, with Cost Synergies, Expected to Accelerate Revenue Growth, Non-GAAP Diluted EPS Accretion, Non-GAAP Operating Margin Expansion, and Operating Cash Flow through the Mid-2030s Conference Call and Webcast Scheduled Today at 4:30 p.m. ET SAN RAFAEL, Calif., Aug. 6, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the second quarter ended June 30, 2026. "This quarter, we executed strongly across our portfolio while rapidly integrating Amicus into BioMarin's operations and advancing plans to accelerate growth for GALAFOLD and POMBILITI + OPFOLDA, and extending the benefit of these medicines to more patients worldwide," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Strong global demand led us to increase full-year VOXZOGO revenue guidance to at least $1 billion in 2026. Adding to this momentum is the opportunity to advance our second potential indication with VOXZOGO, hypochondroplasia, based on recent pivotal data that exceeded our expectations." Mr. Hardy added, "With our larger, more diversified commercial portfolio of innovative medicines, we are positioned to deliver additional growth and increased profitability. We expect strong execution through the remainder of 2026, bringing together our expanded portfolio, scale and disciplined integration efforts to reach more patients living with serious genetic conditions around the world." 2026 Business and Pipeline Highlights Innovation BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch. The company plans to provide an update on the application status as part of its third quarter earnings update. In May, the company announced that the Phase 3 CANOPY-HCH-3 study of VOXZOGO in children with hypochondroplasia met its primary endpoint, with a statistically significant increase in annualized growth velocity (AGV) at week 52 versus placebo (LS mean difference +2.33 cm/yr, p<0.0001), along with significant improvements in standing height, height Z-score, and the key secondary endpoint of arm span. The full Phase 3 dataset will be shared in a late-breaking oral presentation at the European Society for Paediatric Endocrinology Annual Meeting in September. In June, at the Endocrine Society Annual Meeting (ENDO 2026), a Phase 2 investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia showed sustained improvements in growth with a favorable safety profile. Mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through year three, with mean height standard deviation score (SDS) improving 0.72 over the three years. Also at ENDO 2026, the company presented Phase 1 data for BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a single-ascending-dose study in healthy adults, BMN 333 demonstrated sustained exposure supporting weekly dosing and was well tolerated, with free CNP exposure at the highest dose more than 13-fold that of another long-acting CNP agent, reflecting its potential to become a new standard of care in achondroplasia. The Phase 2/3 study is enrolling, with a data update expected in 2027. In July, BioMarin announced that the FDA accepted its sNDA for full approval of VOXZOGO in children with achondroplasia, with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027. In the second quarter, the European Commission approved PALYNZIQ® for adolescents 12 years and older with phenylketonuria (PKU). PALYNZIQ is the only therapy that enables people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity. During the quarter, BioMarin added BMN 820 (formerly DMX-200) to its portfolio, a first-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis (FSGS) for which BioMarin holds exclusive U.S. commercialization rights. BMN 820 has the potential to treat a broad FSGS population, regardless of nephrotic syndrome status, and represents a U.S. total addressable patient population of approximately 30,000. The Phase 3 ACTION 3 trial is ongoing, with pivotal data expected in 2028. BMN 351, BioMarin's Phase 1/2 candidate for Duchenne muscular dystrophy, continued in development. The company expects to provide a program update by year-end. Following the pivotal ENERGY 3 trial results, previously announced in May, in which BMN 401 did not meet one of its two co-primary endpoints for the treatment of ENPP1 deficiency, BioMarin has now made the decision to discontinue development of BMN 401 across all indications. In July, BioMarin and the n-Lorem Foundation entered a collaboration and global exclusive license agreement to develop a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies. ReNU syndrome has an expected global population of approximately 100,000. Growth BioMarin expects peak revenue for GALAFOLD to be approximately $1.4 billion by the mid-2030s and for POMBILITI + OPFOLDA to be approximately $1.2 billion by the mid-to-late-2030s. BioMarin expects these high growth therapies to benefit from its global scale and proven commercial capabilities. Metabolic Conditions (formerly Enzyme Therapies) revenue grew 25% Y/Y in the second quarter of 2026, driven by the additions of GALAFOLD and POMBILITI + OPFOLDA and continued strength from PALYNZIQ. The number of patients on therapy grew across all BioMarin-marketed therapies, both Y/Y and sequentially. Strong U.S. and global demand led to increased full-year 2026 VOXZOGO revenue guidance to a low end of $1 billion. The number of children being treated with VOXZOGO globally increased by more than 20% Y/Y in the second quarter. In the U.S., the majority of new patient starts were under two years of age, and the region drove approximately 25% of total VOXZOGO revenue during the quarter. Value Commitment As part of the acquisition of Amicus, which closed on April 27, 2026, the company identified approximately $280 million of cost reductions on a GAAP basis, and approximately $220 million of cost reductions on a Non-GAAP basis, expected to be fully realized in 2028, representing an approximately 50% reduction from Amicus-reported 2025 GAAP and Non-GAAP operating expenses, respectively. Synergies reflect a reduction of Amicus' legacy labor costs and external spend and are expected to be largely driven by general and administrative functions, with the large majority of sales and marketing capabilities retained to support continued commercial growth. GALAFOLD and POMBILITI + OPFOLDA, combined, are expected to reach over 60% Non-GAAP Operating Margin by 2030. The company is targeting gross leverage below 2.5 times by mid-year 2027, an acceleration by approximately one year of prior timeline guidance provided at deal announcement, supported by profitability growth of the combined company. Second Quarter 2026 Financial Highlights Total Revenues for the second quarter of 2026 were $990 million, an increase of $165 million compared to the same period in 2025, primarily driven by revenues from GALAFOLD and POMBILITI + OPFOLDA, which were acquired from Amicus on April 27, 2026, as well as new patients initiating VOXZOGO therapy across all regions and growth in U.S. patients treated with PALYNZIQ. These increases were partially offset by lower VIMIZIM® revenue due to the timing of large government orders outside the U.S. and lower ALDURAZYME® sales volume due to the timing of order fulfillment to Sanofi. GAAP Net Income for the second quarter of 2026 decreased to $45 million compared to $241 million for the same period in 2025. The decrease was primarily driven by the acquisition of Amicus, including integration and restructuring costs, intangible asset amortization, interest expense from debt issued to finance a portion of the transaction, and amortization of inventory fair value step-up. Other drivers included higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO and higher Research and Development (R&D) spend related to BMN 401, which was acquired in the third quarter of 2025, partially offset by higher gross profit driven by revenue growth as described above. Non-GAAP Income for the second quarter of 2026 decreased to $236 million compared to $282 million for the same period in 2025. The decrease was primarily driven by higher interest expense, higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO, and higher R&D spend related to BMN 401, partially offset by higher gross profit driven by revenue growth as described above. Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 % Change 2026 2025 % Change Total Revenues $990 $825 20 % $1,756 $1,571 12 % Net Product Revenues by Product: VOXZOGO $253 $221 14 % $472 $435 9 % Metabolic Conditions: VIMIZIM $194 $215 (10) % $405 $404 — % NAGLAZYME® 135 129 5 % 265 243 9 % PALYNZIQ 135 106 27 % 225 199 13 % GALAFOLD 106 — NM 106 — NM BRINEURA® 51 49 4 % 98 89 10 % ALDURAZYME 44 56 (21) % 80 105 (24) % POMBILITI + OPFOLDA 30 — NM 30 — NM Total Metabolic Conditions Revenue $695 $555 25 % $1,209 $1,040 16 % KUVAN® $24 $27 (11) % $48 $52 (8) % ROCTAVIAN®(1) $12 $9 33 % $14 $20 (30) % GAAP Net Income $45 $241 (81) % $150 $426 (65) % Non-GAAP Income (2) $236 $282 (16) % $385 $502 (23) % GAAP Operating Margin % (3) 11.2 % 33.5 % 13.7 % 31.9 % Non-GAAP Operating Margin % (2) 36.4 % 39.9 % 31.0 % 37.9 % GAAP Diluted EPS $0.23 $1.23 (81) % $0.77 $2.19 (65) % Non-GAAP Diluted EPS (2) $1.20 $1.44 (17) % $1.96 $2.57 (24) % NM Percentage change is not meaningful for products acquired from Amicus on April 27, 2026. (1) In 2026, the company announced that it will no longer market ROCTAVIAN. (2) 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. (3) 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. Updated 2026 Full-Year Financial Guidance (in millions, except EPS amounts) Total Revenues, VOXZOGO, and Non-GAAP Diluted EPS guidance raised, reflecting strong first-half 2026 performance and second-half 2026 revenue expectations for both Metabolic Conditions and VOXZOGO. Guidance reflects post-close contributions from Amicus beginning April 27, 2026. BioMarin will continue to include interest expense related to the Amicus financing in both GAAP and Non‑GAAP financial results. Based on current rates, interest expense associated with the financing is estimated at approximately $200 million on an annualized basis, with Term Loans and Senior Notes scheduled to mature after 2030. Item Provided on May 4, 2026 Updated August 6, 2026 Midpoint Growth (Y/Y) Total Revenues $3,825 to $3,925 $3,875 to $3,925 21 % Metabolic Conditions $2,725 to $2,775 Unchanged 31 % VOXZOGO $975 to $1,025 $1,000 to $1,050 11 % Other Revenues(1) $100 to $125 Unchanged Non-GAAP Diluted EPS (2)(3)(4) $4.85 to $5.05 $4.90 to $5.10 59 % (1) Other Revenues includes KUVAN, ROCTAVIAN, and royalties. (2) Refer to Non-GAAP Information beginning on page 10 of this press release for definition of Non-GAAP Diluted EPS. (3) Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted Shares Outstanding. (4) Non-GAAP Diluted EPS guidance assumes a combined company tax rate of 22%. BioMarin will host a conference call and webcast to discuss second quarter 2026 financial results today, Thursday, August 6, 2026, 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: 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: 3551298 Conference ID: 3551298 About BioMarin BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. 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 Diluted EPS, Non-GAAP Operating Margin, gross leverage, operating cash flow and revenue compound annual growth rate (CAGR) for, in certain instances, the full-year 2026, fourth quarter and second half of 2026, and future periods, and the underlying drivers of those results, such as the expected demand and continued growth of BioMarin's Metabolic Conditions portfolio, including PALYNZIQ, and VOXZOGO, and the expected impact of the acquisition of Amicus Therapeutics, Inc. (Amicus); the anticipated benefits of the acquisition of Amicus, including the expected amount and timing of cost synergies as well as expected revenue from the addition of GALAFOLD and POMBILITI + OPFOLDA, including BioMarin's plans and expectations to accelerate growth through mid-2030s; BioMarin's plans for investment in innovation and future growth; the timing of orders for commercial products; plans and expectations regarding the development, commercialization and commercial prospects of BioMarin's product candidates and commercial products, including the prospects and timing of actions relating to clinical studies and trials and product approvals, such as study initiations, study advancements, data readouts, submissions, filings, approvals, and label expansions; the expected benefits and availability of BioMarin's commercial products and product candidates, including with respect to the potential new indication for VOXZOGO in hypochondroplasia; and potential growth opportunities and trends, including the assumptions and expectations regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products; BioMarin's ability to realize the anticipated benefits of any acquisitions; BioMarin's ability to accurately estimate future financial performance; impacts of macroeconomic and other external factors on BioMarin's operations, regulatory uncertainty, the impact of new or increased tariffs, other trade protection measures, and escalating trade tensions; geopolitical instability, wars and military conflicts; 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 Medicines Agency, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; BioMarin's ability to meet product demand; 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, 2026, as such factors may be updated by any subsequent reports. Investors 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®, VOXZOGO®, VIMIZIM®, NAGLAZYME®, PALYNZIQ®, BRINEURA®, KUVAN®, ROCTAVIAN®, GALAFOLD®, and POMBILITI® + OPFOLDA® 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. BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED STATEMENTS OF INCOME Three and Six Months Ended June 30, 2026 and 2025 (In thousands of U.S. dollars, except per share amounts) (Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 REVENUES: Net product revenues $ 984,393 $ 812,982 $ 1,744,471 $ 1,547,626 Royalty and other revenues 5,315 12,428 11,445 22,929 Total revenues 989,708 825,410 1,755,916 1,570,555 OPERATING EXPENSES: Cost of sales 202,795 150,090 397,794 301,648 Research and development 206,952 161,308 385,748 320,039 Selling, general and administrative 395,549 232,279 653,839 438,395 Intangible asset amortization 73,492 4,846 77,975 9,693 Total operating expenses 878,788 548,523 1,515,356 1,069,775 INCOME FROM OPERATIONS 110,920 276,887 240,560 500,780 Interest income 10,480 18,827 33,040 37,840 Interest expense (63,295) (2,679) (78,253) (5,542) Other income, net 3,279 4,833 7,240 2,879 INCOME BEFORE INCOME TAXES 61,384 297,868 202,587 535,957 Provision for income taxes 16,622 57,336 52,298 109,739 NET INCOME $ 44,762 $ 240,532 $ 150,289 $ 426,218 EARNINGS PER SHARE, BASIC $ 0.23 $ 1.25 $ 0.78 $ 2.23 EARNINGS PER SHARE, DILUTED $ 0.23 $ 1.23 $ 0.77 $ 2.19 Weighted average common shares outstanding, basic 193,423 191,907 192,959 191,440 Weighted average common shares outstanding, diluted 194,467 197,091 194,147 196,643 BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED BALANCE SHEETS June 30, 2026 and December 31, 2025 (In thousands of U.S. dollars, except per share amounts) (Unaudited) June 30, 2026 December 31, 2025 ASSETS Current assets: Cash and cash equivalents $ 874,005 $ 1,311,679 Short-term investments — 248,930 Accounts receivable, net 1,061,047 908,214 Inventory 1,782,524 1,298,883 Other current assets 254,047 185,784 Total current assets 3,971,623 3,953,490 Noncurrent assets: Long-term investments — 492,242 Property, plant and equipment, net 989,994 952,508 Intangible assets, net 4,879,367 213,837 Goodwill 655,745 196,199 Deferred tax assets 888,575 1,508,697 Other assets 338,138 277,049 Total assets $ 11,723,442 $ 7,594,022 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities: Accounts payable and accrued liabilities $ 1,005,793 $ 759,031 Current portion of long-term debt, net 658,203 — Total current liabilities 1,663,996 759,031 Noncurrent liabilities: Long-term debt, net 3,527,939 597,176 Other long-term liabilities 209,815 150,816 Total liabilities 5,401,750 1,507,023 Stockholders' equity: Common stock, $0.001 par value: 500,000,000 shares authorized; 193,535,556 and 192,300,101 shares issued and outstanding, respectively 194 192 Additional paid-in capital 6,037,019 5,956,582 Company common stock held by the Nonqualified Deferred Compensation Plan (11,233) (10,508) Accumulated other comprehensive income (loss) (8,783) (13,473) Retained earnings 304,495 154,206 Total stockholders' equity 6,321,692 6,086,999 Total liabilities and stockholders' equity $ 11,723,442 $ 7,594,022 BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS Six Months Ended June 30, 2026 and 2025 (In thousands of U.S. dollars) (Unaudited) Six Months Ended June 30, 2026 2025 CASH FLOWS FROM OPERATING ACTIVITIES: Net income $ 150,289 $ 426,218 Adjustments to reconcile net income to net cash provided by operating activities: Depreciation and amortization 114,049 40,632 Non-cash interest expense 27,912 1,320 Stock-based compensation 119,152 85,231 Impairment of assets — 2,967 Deferred income taxes 2,261 61,771 Unrealized foreign exchange gains (4,046) (5,306) Other (5,534) (4,633) Changes in operating assets and liabilities, net of effects of business acquired: Accounts receivable, net (46,005) (156,124) Inventory 12,334 (72,462) Other current assets (23,589) (15,092) Other assets 9,005 (13,505) Accounts payable and accrued liabilities 26,265 3,111 Other long-term liabilities 6,667 5,537 Net cash provided by operating activities 388,760 359,665 CASH FLOWS FROM INVESTING ACTIVITIES: Purchases of property, plant and equipment (49,739) (33,869) Maturities and sales of investments 767,277 195,738 Purchases of investments (25,792) (202,433) Purchase of intangible assets (5,433) (266) Acquisition of Amicus, net of cash acquired (5,067,630) — Other 4,966 — Net cash used in investing activities (4,376,351) (40,830) CASH FLOWS FROM FINANCING ACTIVITIES: Proceeds from exercises of awards under equity incentive plans 6,655 7,707 Taxes paid related to net share settlement of equity awards (39,504) (51,089) Proceeds from borrowings 3,650,000 — Payments of debt issuance costs (65,604) — Net cash provided by (used in) financing activities 3,551,547 (43,382) Effect of exchange rate changes on cash (1,630) (4,479) NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS (437,674) 270,974 Cash and cash equivalents: Beginning of period $ 1,311,679 $ 942,842 End of period $ 874,005 $ 1,213,816 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 (Loss) excluding amortization, 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 Cost of Sales (COS), Non-GAAP R&D expenses and Non-GAAP Selling, General and Administrative (SG&A) expenses are defined by the company as GAAP COS, 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 (Loss) 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 or convertible debt in periods when they are dilutive under Non-GAAP. Projected Gross Leverage is defined by the company as undiscounted debt (total debt excluding unamortized discount and deferred offering costs) as of the balance sheet date divided by 4 quarter projected Non-GAAP Adjusted EBITDA. Non-GAAP Adjusted EBITDA is defined by the company as GAAP Income (or Loss) from Operations excluding the impact of depreciation, amortization and stock-based compensation expense. Non-GAAP synergies is defined by the company as Amicus' legacy labor and external spend cost reductions, excluding the impact of stock-based compensation. 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. 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. Because these Non-GAAP metrics 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. 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, 2026 2025 2026 2025 GAAP Reported Net Income $ 45 $ 241 $ 150 $ 426 Adjustments Stock-based compensation expense - COS 5 4 9 6 Stock-based compensation expense - R&D 16 14 28 26 Stock-based compensation expense - SG&A(2) 55 30 83 53 Amortization of intangible assets 73 5 78 10 Amortization of acquired inventory step-up(3) 12 — 12 — Acquisition-related costs(3) 84 — 84 — Severance costs (4) 3 — 12 — Loss on investments (5) — — — 3 Income tax effect of adjustments (57) (11) (70) (22) Non-GAAP Income $ 236 $ 282 $ 385 $ 502 Three Months Ended June 30, 2026 2025 COS R&D SG&A COS R&D SG&A GAAP expenses $ 203 $ 207 $ 396 $ 150 $ 161 $ 232 Adjustments Stock-based compensation expense(2) (5) (16) (55) (4) (14) (30) Amortization of acquired inventory step-up(3) (12) — — — — — Acquisition-related costs (3) — — (84) — — — Severance costs (4) — — (3) — — — Non-GAAP expenses $ 186 $ 191 $ 253 $ 146 $ 147 $ 203 Six Months Ended June 30, 2026 2025 COS R&D SG&A COS R&D SG&A GAAP expenses $ 398 $ 386 $ 654 $ 302 $ 320 $ 438 Adjustments Stock-based compensation expense(2) (9) (28) (83) (6) (26) (53) Amortization of acquired inventory step-up(3) (12) — — — — — Acquisition-related costs (3) — — (84) — — — Severance costs (4) — — (12) — — — Non-GAAP expenses $ 378 $ 358 $ 475 $ 295 $ 294 $ 385 Three Months Ended June 30, Six Months Ended June 30, 2026 Percent of GAAP Total Revenue 2025 Percent of GAAP Total Revenue 2026 Percent of GAAP Total Revenue 2025 Percent of GAAP Total Revenue GAAP Income from Operations $ 111 11.2 % $ 277 33.5 % $ 241 13.7 % $ 501 31.9 % Adjustments Stock-based compensation expense(2) 76 7.7 48 5.8 120 6.8 85 5.4 Amortization of intangible assets 73 7.4 5 0.6 78 4.4 10 0.6 Amortization of acquired inventory step-up(3) 12 1.2 — — 12 0.7 — — Acquisition-related costs (3) 84 8.5 — — 84 4.8 — — Severance costs (4) 3 0.3 — — 12 0.7 — — Non-GAAP Income from Operations $ 360 36.4 % $ 329 39.9 % $ 545 31.0 % $ 596 37.9 % Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 GAAP Diluted EPS $ 0.23 $ 1.23 $ 0.77 $ 2.19 Adjustments Stock-based compensation expense(2) $ 0.38 $ 0.24 0.60 0.43 Amortization of intangible assets $ 0.37 $ 0.03 0.39 0.05 Amortization of acquired inventory step-up(3) $ 0.06 $ — 0.06 — Acquisition-related costs(3) $ 0.42 $ — 0.42 — Severance costs (4) $ 0.02 $ — 0.06 — Loss on investments (5) $ — $ — — 0.02 Income tax effect of adjustments $ (0.29) $ (0.06) (0.35) (0.11) Non-GAAP Diluted EPS $ 1.20 $ 1.44 $ 1.96 $ 2.57 Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 GAAP Weighted-Average Diluted Shares Outstanding 194.5 197.1 194.1 196.6 Adjustments Common stock issuable under company's convertible debt (6) 4.4 — 4.4 — Non-GAAP Weighted-Average Diluted Shares Outstanding 198.9 197.1 198.5 196.6 (1) Certain amounts may not sum or recalculate due to rounding. (2) Stock-based compensation expense recorded in SG&A for the three and six months ended June 30, 2026, includes approximately $13 million related to the post-combination service period for unvested Amicus stock options. (3) These amounts represent costs resulting from the Amicus acquisition that closed on April 27, 2026. Acquisition-related costs were included in SG&A and consisted of severance, transaction and integration costs. Amortization of acquired inventory step-up was included in COS. (4) These amounts were included in SG&A and represent charges for severance in connection with the company's plan to simplify its organizational design and strategic initiatives in the first and second quarters of 2026. (5) Represents impairment loss on non-marketable equity securities recorded in Other income, net, in the first quarter of 2025. (6) Common stock issuable under the company's convertible debt were excluded from the computation of GAAP Weighted-Average Diluted Shares Outstanding for the three and six months ended June 30, 2026 as they were anti-dilutive. Amicus Therapeutics, Inc. (1) Reconciliation of Non-GAAP Financial Measures (in thousands) (Unaudited) Twelve Months Ended December 31, 2025 Total operating expenses - as reported GAAP $ 528,492 Research and development: Share-based compensation 12,156 Selling, general and administrative: Share-based compensation 75,254 Loss on impairment of assets 1,702 Depreciation and amortization 7,460 Total operating expense adjustments to reported GAAP 96,572 Total operating expenses - as adjusted $ 431,920 (1) The above historical reconciliation is reproduced from Amicus' earnings release furnished as Exhibit 99.1 to its Current Report on Form 8-K dated February 20, 2026 and reflects Amicus' historical definitions of the applicable non-GAAP measures. Contact: Investors: Media: Traci McCarty Marni Kottle BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. (415) 455-7558 (650) 374-2803 SOURCE BioMarin Pharmaceutical Inc.

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced today that it presented new clinical data in a poster session at the 18th International Symposium on MPS and Related Lysosomal Diseases, which was held June 4-7, 2026, in Florence, Italy. “Due to the protective blood-brain barrier, lysosomal storage disorders historically have been challenging to treat due to the inability to deliver a therapy into the central nervous system. With our J-Brain Cargo® platform technology, we have the potential to address the progressive neurological symptoms associated with these rare and life-limiting diseases, many of which have inadequate treatment options or no approved therapies available,” said Hiroyuki Sonoda, Ph.D., President and Chief Scientific Officer of JCR Pharmaceuticals. “The data presented at this symposium demonstrate the safety and efficacy evidence of JR-171 (lepunafusp alfa) in people with mucopolysaccharidosis type I (MPS I) and highlight its potential. We look forward to sharing additional data from our J-Brain Cargo® platform technology as they are available. We wish to thank all of the patients who have participated in our clinical programs and their families, clinical investigators, and other partners who continue to support our work.” New Clinical Data Presentation Three-Year safety and pharmacodynamics of lepunafusp alfa (JR-171) in patients with mucopolysaccharidosis type I (MPS-I): Results from a phase I/II trial and extension study (Presentation Number: 63) Lead Author: Paul Harmatz, M.D. (UCSF Benioff Children’s Hospital, Oakland, CA) Researchers reported on three years of data describing safety and pharmacodynamics of lepunafusp alfa (JR-171) in patients with mucopolysaccharidosis type I (MPS I) who were treated in a Phase I/II trial plus a three-year extension study (NCT04227600 and NCT04453085). Patients with MPS I were randomized to weekly intravenous lepunafusp alfa at a “Low” (2.0 mg/kg; n=6) or a “High” (4.0 mg/kg; n=8) dose and followed for three years. Enrollment was open to all MPS I phenotypes (Hurler [n=7], Hurler-Scheie [n=5], Scheie [n=2]); one Low-dose Hurler patient did not continue in the extension study. Both the High‑ and Low‑dose cohorts demonstrated a favorable safety and tolerability profile, with compliance exceeding 90% in both groups. Overall, six patients experienced serious adverse events (SAEs) [5/8 in the High dose group, 1/5 in the Low dose group]. Of the 8 serious treatment-emergent adverse events (TEAEs), none were attributed to lepunafusp alfa. Adverse events of special interest — specifically anaphylaxis or infusion‑associated reactions — were uncommon, occurring in fewer than 5% of participants across both dose groups. By Week 12, cerebrospinal fluid-heparan sulfate (CSF-HS) decreased in all patients. In the High dose group, CSF-HS decreases remained stable during the three-year study period in most patients; partial rebound was observed in one patient. In the Low dose group, two patients, both with Hurler syndrome, had a subsequent rebound in HS levels. Rebound did not correlate with presence/absence of antibodies. The somatic symptoms (including serum HS levels, and liver and spleen volume) remained stable, or improved, in patients in both doses who received previous laronidase and decreased in the treatment-naïve patient. With three years of follow-up, researchers concluded that weekly lepunafusp alfa is safe and well tolerated in a broad spectrum of patients with MPS I, with no serious TEAEs attributable to the study drug. Pharmacodynamic response of CSF-HS demonstrates that lepunafusp alfa crosses the blood-brain barrier (BBB), with a trend toward greater reduction in CSF-HS in the High dose cohort (4mg/kg). The long-term safety and clinical efficacy warrants evaluation in a larger clinical trial. Encore Clinical Data Presentations The following encore presentations provide additional evidence and context for the use of JR-141 (pabinafusp alfa) in the treatment of MPS II and were shared at the 22nd Annual WORLDSymposium™ 2026 (February 2-6, 2026). Sustained cognitive and adaptive behavior outcomes of long-term treatment with pabinafusp alfa in patients with severe or attenuated mucopolysaccharidosis type II (Presentation Number: 61) Lead Author: Roberto Giugliani, M.D., Ph.D. (Federal University of Rio Grande do Sul, Brazil) In a longitudinal, pooled, post hoc analysis of patients with MPS II receiving pabinafusp alfa across five open-label trials with up to five years of follow-up, researchers reported on sustained cognitive and adaptive behavior outcomes of long-term treatment with pabinafusp alfa in patients with severe or attenuated MPS II. Researchers concluded that long-term treatment with pabinafusp alfa was well tolerated and associated with stabilization or continued skill acquisition in many patients with severe or attenuated MPS II, suggesting that, with timely initiation prior to the onset of irreversible neurodegeneration, treatment with pabinafusp alfa may provide a benefit to patients with MPS II. Long-term somatic efficacy of pabinafusp alfa across a broad spectrum of age groups and phenotypes in patients with mucopolysaccharidosis type II (Presentation Number: 62) Lead Author: Ana Maria Martins, M.D., Ph.D. (Federal University of São Paulo) In a longitudinal, pooled, post hoc analysis of patients with MPS II receiving pabinafusp alfa in open-label trials with up to five years of follow-up, researchers reported on the somatic effects of pabinafusp alfa in a heterogenous population of patients with MPS II who initiated treatment at different ages. Researchers concluded that long-term treatment with pabinafusp alfa was well tolerated and provided positive somatic effects to a broad spectrum of severe and attenuated patients with MPS II. About the International Symposium on MPS and Related Lysosomal Diseases The International Symposium on MPS and Related Lysosomal Diseases brings together healthcare professionals, researchers, and industry leaders to accelerate progress in mucopolysaccharidoses (MPS) and related lysosomal storage disorders through collaborative innovation. We unite diverse perspectives to advance early diagnosis through cutting-edge technologies, develop revolutionary therapies, and ensure equitable global access to care. Our mission is to foster the next generation of rare disease advocates and professionals while creating sustainable partnerships that transform scientific breakthroughs into real-world improvements in patient outcomes worldwide. For more information, please visit https://mps2026.com/. About the J-Brain Cargo® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo®, to bring biotherapeutics into the central nervous system (CNS). The first drug developed based on this technology is IZCARGO™ (INN: pabinafusp alfa), which is approved in Japan for the treatment of a lysosomal storage disorder (LSD). With J-Brain Cargo®, JCR seeks to address the unresolved clinical challenges of LSDs by delivering the enzyme to both the body and the brain. About Mucopolysaccharidosis Type I (Hurler, Hurler-Scheie, Scheie Syndrome) Mucopolysaccharidosis type I (MPS I) is an autosomal recessive lysosomal storage disorder (LSD) caused by a deficiency of α-L-iduronidase, an enzyme that breaks down glycosaminoglycans (mucopolysaccharides) in the body. The current worldwide prevalence of MPS I is estimated to be approximately 3,000-4,000 (according to JCR internal research). MPS I gives rise to a wide range of somatic and neurological symptoms. A major limitation of current enzyme replacement therapy (ERT) is that it does not address central nervous system (CNS) symptoms because of the enzyme’s inability cross the blood-brain barrier (BBB). MPS I is the only LSD in which hematopoietic stem cell transplantation (HSCT) is part of the standard of case for the severe form of the disease. Significant unmet medical need persists in all forms of MPS I. About JR-171 JR-171 (lepunafusp alfa) is a recombinant fusion protein of an antibody against the human transferrin receptor and α-L-iduronidase, the enzyme that is missing or malfunctioning in patients with mucopolysaccharidosis type I (MPS I).1,2 By crossing the blood brain-barrier (BBB) through transferrin receptor mediated transcytosis, it is expected to be effective against central nervous system (CNS) signs and symptoms of the disease thereby addressing a significant unmet need for the treatment of MPS I. JR-171 previously was granted Fast Track designation by the US Food and Drug Administration (FDA). About Mucopolysaccharidosis Type II (Hunter Syndrome) Mucopolysaccharidosis type II (MPS II, or Hunter syndrome) is an X-linked recessive lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase, an enzyme that breaks down complex carbohydrates called glycosaminoglycans (GAGs, also known as mucopolysaccharides) in the body. Hunter syndrome, which affects an estimated 2,000-3,000 individuals worldwide (according to JCR research), gives rise to a wide range of somatic and neurological symptoms. The current standard of care for Hunter syndrome is enzyme replacement therapy. Central nervous system symptoms related to MPS II have been unmet medical needs so far. About JR-141 JR-141 (pabinafusp alfa) is a recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase, the enzyme that is missing or malfunctioning in subjects with Hunter syndrome. It incorporates J-Brain Cargo®, JCR’s proprietary blood-brain barrier (BBB)-penetrating technology, to cross the BBB through transferrin receptor-mediated transcytosis, and its uptake into cells is mediated through the mannose-6-phosphate receptor. This novel mechanism of action is expected to make pabinafusp alfa effective against the central nervous system (CNS) symptoms of Hunter syndrome. In pre-clinical trials, JCR has confirmed both high-affinity binding of pabinafusp alfa to transferrin receptors and passage across the BBB into neuronal cells. In addition, JCR has confirmed enzyme uptake in various brain tissues. The company has also confirmed a reduction of substrate accumulation in the CNS and peripheral organs in an animal model of Hunter syndrome.3,4 In several clinical trials of pabinafusp alfa, JCR obtained evidence of reducing heparan sulfate (HS) concentrations in the cerebrospinal fluid (CSF), a biomarker for assessing effectiveness against CNS symptoms; these results were consistent with those obtained in pre-clinical studies.5 Clinical studies have also demonstrated the positive effects of pabinafusp alfa on CNS symptoms.6,7,8 Pabinafusp alfa was approved in Japan by the Ministry of Health, Labour and Welfare and marketed since May 2021 under the brand name “IZCARGO™ I.V. Infusion 10mg.” About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world’s most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including partners, patients and employees. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit JCR’s global website: https://jcrpharm.com/. Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future. References 1: Kida S, et al. Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice. Mol Ther Methods Clin Dev. 2023; 29: 439-449. 2: Harmatz P, et al. α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial. Mol Ther. 2024; 32(3): 609-618. 3: Sonoda, et al. A blood-brain-barrier-penetrating anti-human transferrin receptor antibody fusion protein for neuronopathic mucopolysaccharidosis II. Mol. Ther. 2018; 26(5): 1366-1374. 4: Morimoto, et al. Clearance of heparin sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice. Mol. Ther. 2021; 29(5): 1853-1861. 5: Okuyama, et al. Iduronate-2-sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. Mol Ther. 2020; 27(2): 456-464. 6: Okuyama, et al. A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II. Mol Ther. 2021; 29(2): 671-679. 7: Giugliani, et al. Iduronate-2-sulfatase fused with anti-human transferrin receptor antibody, pabinafusp alfa, for treatment of neuronopathic and non-neuronopathic mucopolysaccharidosis II: Report of a phase 2 trial in Brazil. Mol Ther. 2021; 29(7): 2378-2386. 8: Giugliani, et al. Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II; an Integrated Analysis of Preclinical and Clinical Data. Int. J. Mol. Sci. 2021, Volume 22, Issue 20, 10938.