BMRN BioMarin Pharmaceutical Inc.

76.44
-1.21  -2%
Previous Close 77.65
Open 77.17
52 Week Low 71.35
52 Week High 124.5199
Market Cap $14,015,446,372
Shares 183,352,255
Float 142,192,515
Enterprise Value $14,033,355,526
Volume 941,168
Av. Daily Volume 982,432
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Upcoming Catalysts

Drug Stage Catalyst Date
VOXZOGO (Vosoritide)
Achondroplasia
PDUFA
PDUFA
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Valoctocogene roxaparvovec (BMN 270)
Hemophilia A
Phase 3
Phase 3
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VOXZOGO (Vosoritide)
Children with Achondroplasia
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
BMN 307
Phenylketonuria (PKU)
Phase 1/2
Phase 1/2
Phase 1/2 dosing continues as of July 2021.
VIMIZIM (elosulfase alfa) (GALNS)
(MPS IVA) Morquio A Syndrome
Approved
Approved
Approved February 14, 2014.
BRINEURA (cerliponase alfa)
Batten Disease
Approved
Approved
PDUFA date extended by three months to April 27 2017. Approval announced April 27, 2017.
PALYNZIQ (Pegvaliase)
Phenylketonuria (PKU)
Approved
Approved
Approval announced May 24, 2018.
Valoctocogene roxaparvovec (BMN 270)
Hemophilia A
CRL
CRL
CRL announced August 19, 2020.
Kyndrisa
Duchenne Muscular Dystrophy (DMD)
CRL
CRL
CRL issued January 14, 2016.

Latest News

  1. SAN RAFAEL, Calif., July 28, 2021 /PRNewswire/ --

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



    Three Months Ended June 30,



    Six Months Ended June 30,



    2021



    2020



    % Change



    2021



    2020



    % Change

























    Total Revenues

    $

    501.7





    $

    429.5





    17

    %



    $

    987.7





    $

    931.6





    6

    %

























    Net Product Revenues Marketed by BioMarin (1)

    $

    458.6





    $

    386.8





    19

    %



    $

    876.3





    $

    820.1





    7

    %

























    Vimizim Net Product Revenues

    $

    171.7





    $

    116.7





    47

    %



    $

    329.9





    $

    253.9





    30

    %

    Naglazyme Net Product Revenues

    $

    118.8





    $

    81.0





    47

    %



    $

    226.1





    $

    195.3





    16

    %

    Kuvan Net Product Revenues

    $

    78.8





    $

    122.6





    (36)

    %



    $

    149.6





    $

    244.6





    (39)

    %

    Palynziq Net Product Revenues

    $

    59.0





    $

    40.7





    45

    %



    $

    113.0





    $

    75.3





    50

    %

    Brineura Net Product Revenues

    $

    30.3





    $

    25.8





    17

    %



    $

    57.7





    $

    49.8





    16

    %

























    Aldurazyme Net Product Revenues

    $

    28.1





    $

    32.3





    (13)

    %



    $

    78.1





    $

    88.0





    (11)

    %

























    GAAP Net Income (Loss)

    $

    12.9





    $

    (29.2)









    $

    30.3





    $

    52.2







    GAAP Net Income (Loss) per Share – Basic

    $

    0.07





    $

    (0.16)









    $

    0.17





    $

    0.29







    GAAP Net Income (Loss) per Share – Diluted

    $

    0.07





    $

    (0.16)









    $

    0.16





    $

    0.28







    Non-GAAP Income (2)

    $

    97.8





    $

    57.4









    $

    202.1





    $

    173.9







     



    June 30,

    2021



    December 31,

    2020



    Cash, cash equivalents and investments

    $

    1,473.6





    $

    1,350.9























    (1)

    Net Product Revenues Marketed by BioMarin is the sum of revenues from Vimizim, Kuvan, Naglazyme, Palynziq, Brineura for the three and six months ended June 30, 2021 and includes Firdapse for the six months ended June 30, 2020, each calculated in accordance with Generally Accepted Accounting Principles in the United States (U.S. GAAP). Sanofi Genzyme (Genzyme) is BioMarin's sole customer for Aldurazyme and is responsible for marketing and selling Aldurazyme to third parties. Refer to page 10 for a table showing Net Product Revenues by product. In January 2020, BioMarin divested the Firdapse assets to a third party in a sale transaction. The sale is reflected in the Company's consolidated financial statements for the first quarter ended March 31, 2020; as a result of the transaction BioMarin no longer recognizes Net Product Revenues from Firdapse.





    (2)

    Non-GAAP Income is defined by the Company as reported GAAP Net Income/Loss, excluding net interest expense, provision for (benefit from) income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and, in certain periods, certain other specified items. Refer to Non-GAAP Information beginning on page 11 of this press release for a complete discussion of the Company's Non-GAAP financial information and reconciliations to the comparable information reported under U.S. GAAP.

    BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) (BioMarin or the Company) today announced financial results for the second quarter ended June 30, 2021.

    "We enjoyed a strong first half of 2021, including financial results and the achievement of our key regulatory goals," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "With the positive CHMP opinion for VOXZOGO for children, 2 years of age and older, our commercial team is eagerly preparing for potential European approval and launch later this summer.  If approved, VOXZOGO would be the first pharmacological treatment option for children with achondroplasia. Following potential approval in Europe later this summer, we hope to have a positive outcome in the United States pending our November 20 PDUFA target action date. Upon potential global approvals of VOXZOGO for achondroplasia, we have a tremendous opportunity to address the interest from families seeking treatment for their children."

    Mr. Bienaimé continued, "We were pleased to receive validation of our MAA in Europe for valoctocogene roxaparvovec for the treatment of severe hemophilia A, paving the way for a CHMP opinion in the first half of 2022. Our data presentations at the International Society on Thrombosis and Haemostasis in July provided further evidence of  hemostatic efficacy following treatment with valoctocogene roxaparvovec, including significant reductions in bleeding events and Factor VIII infusions. We look forward to two year data results from our complete Phase 3 study with 134 participants in early 2022, and anticipate submitting a BLA in the second quarter of 2022, should data be supportive.  More than ever, we believe that valoctocogene roxaparvovec gene therapy will be an important treatment option for people with severe hemophilia A. Given the superior bleed control seen at one year with valoctocogene roxaparvovec, and the burden of frequent, regular infusions with standard of care prophylaxis, we believe one infusion of valoctocogene roxaparvovec gene therapy has the potential to address the unmet need for bleed control, supported by our clinical results to date."

    Financial Highlights:

    • Total Revenues for the second quarter of 2021 increased to $501.7 million, compared to $429.5 million for the same period in 2020. The increase in Total Revenues was primarily attributed to the following:
      • Higher Vimizim and Naglazyme product revenues primarily driven by timing of orders from Europe and Middle East; and
      • Higher Palynziq product revenues primarily driven by a combination of revenue from more U.S. patients achieving maintenance dosing and new patients initiating therapy; partially offset by
      • Lower Kuvan product revenues primarily due to generic competition due to the loss of exclusivity in the U.S.
    • GAAP Net Income increased to $12.9 million for the second quarter of 2021 compared to a GAAP Net Loss of  $29.2 million for the same period in 2020. The increase was primarily due to profits from higher revenues and lower research and development (R&D) expense driven by lower in-licensing expenses.
    • Non-GAAP Net Income for the second quarter of 2021 increased to $97.8 million compared to Non-GAAP Income of $57.4 million for the same period in 2020. The increase in Non-GAAP Income for the quarter, compared to the same period in 2020, was primarily attributed to higher gross profit partially offset by higher SG&A expense.

    Commercial Portfolio (Naglazyme, Vimizim, Brineura, Palynziq, Kuvan and Aldurazyme) 

    • Naglazyme, Vimizim and Brineura maintained robust patient compliance in the quarter and continue to grow based on strong underlying demand.
      • The number of patients on commercial Naglazyme and Vimizim therapy increased by over 10% each year-over-year.
      • The number of commercial patients on commercial Brineura therapy increased by over 30% year-over-year.
    • Order timing for both Naglazyme and Vimizim for the full year 2021 are expected to be more concentrated in the first half of the year.
    • Palynziq top-line results for the full year 2021 are expected to increase approximately 40% as compared to full-year 2020 results based on the mid-point of full-year 2021 guidance.  
    • Palynziq growth in the European, Middle East and African regions (EMEA) has been impacted by ongoing challenges due to COVID-19 with Palynziq revenue from EMEA expected to increase when PKU clinics have more freedom to operate and start treating additional patients.
      • The number of U.S. patients on commercial Palynziq therapy increased approximately 30% year-over-year and is expected to continue to grow as U.S. PKU clinics are expected to re-open over the coming quarters.
    • Loss of Kuvan U.S. market share to generics was consistent with expectations.

    Late-stage Regulatory Portfolio (VOXZOGO and valoctocogene roxaparvovec)

    • In Europe, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of VOXZOGO for the treatment of children, ages 2 years and older. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the third quarter of  2021.
    • The U.S. Food and Drug Administration (FDA) is reviewing the VOXZOGO New Drug Application (NDA) with the Prescription Drug User Fee Act (PDUFA) target action date of November 20, 2021.
    • The European Medicines Agency (EMA) validated BioMarin's MAA for valoctocogene roxaparvovec:  CHMP opinion anticipated in the second quarter of 2022.
    • BioMarin is targeting a Biologics License Application (BLA) submission for valoctocogene roxaparvovec in the second quarter of 2022 assuming favorable results from the two-year follow-up safety and efficacy data from the GENEr8-1 study, followed by an expected 6-month review procedure by the FDA.

    Earlier-stage Development Portfolio (BMN 307, BMN 255, BMN 331, DiNA-001, Allen Institute Collaboration)

    • BMN 307: Dose escalation in PHEarless, the Phase 1/2 study of BMN 307 continues based on encouraging Phe lowering and safety profile observed in study participants who were treated with the lowest dose.
    • BMN 255 for a subset of chronic renal disease: BMN 255 is a small molecule for the treatment of a subset of chronic renal disease. The Investigational New Drug application (IND) for BMN 255 is active and the Company is dosing subjects.
    • BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): BMN 331 is BioMarin's third gene therapy candidate. BioMarin plans to leverage its broad expertise in developing gene therapies for severe hemophilia A and PKU to improve efficiencies in the development process of BMN 331. The IND for BMN 331 was recently cleared by FDA and is active.  
    • BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies continue with BMN 351, an antisense oligonucleotide therapy that has demonstrated dystrophin expression levels of 30-50% of wild-type levels in the quadriceps in a DMD mouse model treated at 18.7 mg/kg/week for 13 weeks (measured 2 weeks following last administration). If results from the ongoing pre-clinical studies are supportive, BioMarin anticipates filing an IND for BMN 351 in the first half of 2022.
    • DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM): Pre-clinical studies are underway with DiNA-001 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR received an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales.
    • On April 28, 2021, BioMarin and the Allen Institute announced a collaboration to create new gene therapies aimed at rare genetic diseases of the central nervous system (CNS). The goal is to combine the Allen Institute's leadership in large-scale genomic science in the CNS therapeutic area with BioMarin's expertise in developing transformational gene therapies.

    2021 Full-Year Financial Guidance (in millions, except %)

    Item



    Provided April 29, 2021*



    Revised July 28, 2021*

    Total Revenues



    $1,750



    to



    $1,850



    $1,790



    to



    $1,880

    Vimizim Net Product Revenues



    $570



    to



    $610



    $580



    to



    $620

    Kuvan Net Product Revenues



    $250



    to



    $290



    $260



    to



    $290

    Naglazyme Net Product Revenues



    $365



    to



    $395



    $375



    to



    $405

    Palynziq Net Product Revenues



    $210



    to



    $250



    $220



    to



    $260

    Brineura Net Product Revenues



    $120



    to



    $140



    Unchanged



























    Cost of Sales (% of Total Revenues)



    23%



    to



    25%



    Unchanged

    Research and Development Expense



    $645



    to



    $695



    $645



    to



    $685

    Selling, General and Administrative Expense



    $725



    to



    $775



    $735



    to



    $775



























    GAAP Net Loss



    ($130)



    to



    ($80)



    ($110)



    to



    ($60)

    Non-GAAP Income (1)



    $170



    to



    $220



    $190



    to



    $240































    *2021 Guidance takes into consideration ongoing expected impact from the COVID-19 pandemic assuming consistent trends experienced during 2020 and the first two quarters of 2021.

    (1)

    All Financial Guidance items are calculated based on U.S. GAAP with the exception of Non-GAAP Income. Refer to Non-GAAP Information beginning on page 10 of this press release for a complete discussion of the Company's Non-GAAP financial information and reconciliations to the corresponding GAAP reported information.

    BioMarin will host a conference call and webcast to discuss second quarter and full-year 2021 financial results today, Wednesday, July 28, 2021 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.

    U.S./Canada Dial-in Number: 866.502.9859

    Replay Dial-in Number: 855.859.2056

    International Dial-in Number: 574.990.1362

    Replay International Dial-in Number: 404.537.3406

    Conference ID: 8363378

    Conference ID: 8363378

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of several commercial therapies and multiple clinical and preclinical product candidates.

    For additional information, please visit www.biomarin.com.

    Forward-Looking Statements

    This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the expectations of Total Revenues, Net Product Revenues, Research and Development Expense, Selling, General and Administrative Expense, Cost of Sales, GAAP Net Loss, Non-GAAP Income, and other specified income statement guidance for the full-year 2021; cash flows from operating activities; the timing of orders for commercial products; expectations regarding the ongoing impact of the COVID-19 pandemic; 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) BioMarin's plans to re-submit a BLA for valoctocogene roxaparvovec to the FDA with two-year follow-up results from all the subjects from the Phase 3 GENEr8-1 study in the second quarter of 2022, (ii) BioMarin's anticipated IND submission for BMN 351 in the first half of 2022, and (iii) BioMarin and Allen Institute collaborating to create new gene therapies; the potential approval and commercialization of BioMarin's product candidates, including VOXZOGO for the treatment of achondroplasia and valoctocogene roxaparvovec for the treatment of severe hemophilia A; the timing of such approval decisions, including (i) the expectation of a CHMP opinion in the second quarter of 2022 for valoctocogene roxaparvovec, (ii) the expectation of a European Commission Decision in the third quarter of 2021 following the receipt of the positive CHMP opinion for VOXZOGO and (iii) the extended PDUFA target action date with respect to VOXZOGO of November 20, 2021; and the expected benefits and availability of BioMarin's product candidates, including that VOXZOGO would be the first potential pharmacological therapeutic option for children with achondroplasia; and potential growth opportunities and trends, including (i) that BioMarin would have a tremendous opportunity to address the interest from families seeking treatment for their children upon potential global approval of VOXZOGO, (ii) that BioMarin believes that one infusion of valoctocogene roxaparvovec gene therapy has the potential to address the unmet need for bleed control and (iii) growth expectations regarding Palynziq.

    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, including BioMarin's projected impact of the COVID-19 pandemic on its global revenue sources, including due to demand interruptions such as missed patient infusions and delayed treatment starts for new patients; results and timing of current and planned preclinical studies and clinical trials, as well as the potential impact of the COVID-19 pandemic on (i) BioMarin's ability to continue such preclinical studies and clinical trials and (ii) the timing of such 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 FDA, the European Commission and other regulatory authorities concerning each of the described products and product candidates, including the potential impact of the COVID-19 pandemic on the regulatory authorities' abilities to issue such decisions and the timing of such decisions; the market for each of these products; actual sales of BioMarin's commercial products and the impact that the COVID-19 pandemic may have on such sales; the introduction of generic versions of BioMarin's commercial products, in particular generic versions of Kuvan; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), 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, 2021 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® and Vimizim® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. VOXZOGO™ is our trademark. 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 BALANCE SHEETS

    June 30, 2021 and December 31, 2020

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





    June 30, 2021



    December 31,

    2020(1)

    ASSETS

    (unaudited)





    Current assets:







    Cash and cash equivalents

    $

    641,533





    $

    649,158



    Short-term investments

    481,864





    416,228



    Accounts receivable, net

    424,419





    448,351



    Inventory

    710,975





    698,548



    Other current assets

    99,046





    129,934



    Total current assets

    2,357,837





    2,342,219



    Noncurrent assets:







    Long-term investments

    350,237





    285,473



    Property, plant and equipment, net

    1,026,579





    1,032,471



    Intangible assets, net

    394,298





    417,271



    Goodwill

    196,199





    196,199



    Deferred tax assets

    1,431,683





    1,432,150



    Other assets

    141,663





    142,237



    Total assets

    $

    5,898,496





    $

    5,848,020



    LIABILITIES AND STOCKHOLDERS' EQUITY







    Current liabilities:







    Accounts payable and accrued liabilities

    $

    411,132





    $

    492,548



    Short-term contingent consideration

    32,212







    Total current liabilities

    443,344





    492,548



    Noncurrent liabilities:







    Long-term convertible debt, net

    1,077,110





    1,075,145



    Long-term contingent consideration

    30,760





    60,130



    Other long-term liabilities

    105,711





    114,195



    Total liabilities

    $

    1,656,925





    $

    1,742,018



    Stockholders' equity:







    Common stock, $0.001 par value: 500,000,000 shares authorized; 183,321,820 and 181,740,999 shares issued and outstanding, respectively.

    183





    182



    Additional paid-in capital

    5,083,831





    4,993,407



    Company common stock held by Nonqualified Deferred Compensation Plan

    (10,207)





    (9,839)



    Accumulated other comprehensive loss

    (939)





    (16,139)



    Accumulated deficit

    (831,297)





    (861,609)



    Total stockholders' equity

    4,241,571





    4,106,002



    Total liabilities and stockholders' equity

    $

    5,898,496





    $

    5,848,020











    (1)

     December 31, 2020 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, 2020, filed with the U.S. Securities and Exchange Commission (SEC) on February 26, 2021.

     

     

    BIOMARIN PHARMACEUTICAL INC.

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

    Three and Six Months Ended June 30, 2021 and 2020

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





    Three Months Ended

    June 30,



    Six Months Ended

    June 30,



    2021



    2020



    2021



    2020



    (unaudited)



    (unaudited)



    (unaudited)



    (unaudited)

    REVENUES:















    Net product revenues

    $

    486,670





    $

    419,032





    $

    954,439





    $

    908,075



    Royalty and other revenues

    15,023





    10,453





    33,284





    23,479



    Total net revenues

    501,693





    429,485





    987,723





    931,554



    OPERATING EXPENSES:















    Cost of sales

    127,062





    97,967





    247,228





    209,341



    Research and development

    161,107





    182,139





    309,832





    324,396



    Selling, general and administrative

    184,161





    175,412





    358,479





    362,707



    Intangible asset amortization and contingent consideration

    17,691





    14,912





    35,426





    30,589



    Gain on sale of nonfinancial assets













    (59,495)



    Total operating expenses

    490,021





    470,430





    950,965





    867,538



    INCOME (LOSS) FROM OPERATIONS

    11,672





    (40,945)





    36,758





    64,016



















    Equity in the loss of BioMarin/Genzyme LLC

    (175)





    (79)





    (1,526)





    (156)



    Interest income

    4,471





    4,291





    6,910





    9,535



    Interest expense

    (3,817)





    (8,048)





    (7,621)





    (14,963)



    Other income, net

    2,005





    2,508





    2,863





    647



    INCOME (LOSS) BEFORE INCOME TAXES

    14,156





    (42,273)





    37,384





    59,079



    Provision for (benefit from) income taxes

    1,215





    (13,090)





    7,072





    6,881



    NET INCOME (LOSS)

    12,941





    (29,183)





    $

    30,312





    $

    52,198



    NET INCOME (LOSS) PER SHARE, BASIC

    $

    0.07





    $

    (0.16)





    $

    0.17





    $

    0.29



    NET INCOME (LOSS) PER SHARE, DILUTED

    $

    0.07





    $

    (0.16)





    $

    0.16





    $

    0.28



    Weighted average common shares outstanding, basic

    182,844





    180,729





    182,311





    180,314



    Weighted average common shares outstanding, diluted

    185,427





    180,729





    185,089





    184,344



     

     

    BIOMARIN PHARMACEUTICAL INC.

    CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

    Six Months Ended June 30, 2021 and 2020

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





    Six Months Ended June 30,



    2021



    2020



    (unaudited)



    (unaudited)

    CASH FLOWS FROM OPERATING ACTIVITIES:







    Net income

    $

    30,312





    $

    52,198



    Adjustments to reconcile net income to net cash used in operating activities:







    Depreciation and amortization

    55,214





    51,180



    Non-cash interest expense

    2,082





    9,534



    Amortization of premium on investments

    1,878





    38



    Stock-based compensation

    104,346





    91,956



    Gain on sale of nonfinancial assets





    (59,495)



    Deferred income taxes

    1,204





    (5,864)



    Unrealized foreign exchange loss (gain)

    (1,004)





    2,201



    Non-cash changes in the fair value of contingent consideration

    4,488





    (584)



    Other

    (376)





    (650)



    Changes in operating assets and liabilities:







    Accounts receivable, net

    17,420





    (8,028)



    Inventory

    6,379





    (46,298)



    Other current assets

    34,331





    20,441



    Other assets

    321





    (5,599)



    Accounts payable and accrued liabilities

    (60,884)





    (94,425)



    Other long-term liabilities

    585





    5,938



    Net cash provided by operating activities

    196,296





    12,543



    CASH FLOWS FROM INVESTING ACTIVITIES:







    Purchases of property, plant and equipment

    (48,106)





    (66,716)



    Maturities and sales of investments

    348,941





    170,111



    Purchases of available-for-sale securities

    (484,572)





    (321,684)



    Proceeds from sale of nonfinancial assets





    67,159



    Purchase of intangible assets

    (6,400)





    (10,786)



    Investment in convertible note





    (8,709)



    Other

    (908)





    (722)



    Net cash used in investing activities

    (191,045)





    (171,347)



    CASH FLOWS FROM FINANCING ACTIVITIES:







    Proceeds from exercises of awards under equity incentive plans

    27,640





    46,927



    Taxes paid related to net share settlement of equity awards

    (38,731)





    (36,645)



    Proceeds from convertible senior subordinated note offering, net





    585,782



    Repurchase of common stock





    (50,000)



    Principal repayments of financing leases

    (1,941)





    (2,047)



    Other

    (381)







    Net cash provided by (used in) financing activities

    (13,413)





    544,017



    Effect of exchange rate changes on cash

    537





    (3,759)



    NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS

    $

    (7,625)





    $

    381,454



    Cash and cash equivalents:







    Beginning of period

    $

    649,158





    $

    437,446



    End of period

    $

    641,533





    $

    818,900



     



    The following table presents Net Product Revenues by Product:

    Net Product Revenues by Product

    (In millions of U.S. dollars)





    Three Months Ended

    June 30,



    Six Months Ended

    June 30,



    2021



    2020



    % Change



    2021



    2020



    % Change



    (unaudited)



    (unaudited)







    (unaudited)



    (unaudited)





    Vimizim

    $

    171.7





    $

    116.7





    47

    %



    $

    329.9





    $

    253.9





    30

    %

    PKU franchise

    137.8





    163.3





    (16)

    %



    262.6





    319.9





    (18)

    %

    Naglazyme

    118.8





    81.0





    47

    %



    226.1





    195.3





    16

    %

    Brineura

    30.3





    25.8





    17

    %



    57.7





    49.8





    16

    %

    Firdapse (1)









    %







    1.2





    (100)

    %

    Net Product Revenues Marketed by BioMarin

    $

    458.6





    $

    386.8









    $

    876.3





    $

    820.1







    Aldurazyme Net Product Revenues Marketed by Genzyme

    28.1





    32.3





    (13)

    %



    78.1





    88.0





    (11)

    %

    Total Net Product Revenues

    $

    486.7





    $

    419.1









    $

    954.4





    $

    908.1







     

    (1)

    In January 2020, BioMarin divested the Firdapse assets to a third party in a sale transaction. The sale is reflected in the Company's consolidated financial statements for the six months ending June 30, 2020; and as a result of the transaction BioMarin no longer generates Net Product Revenues from Firdapse.

    The following table presents Net Product Revenues for the PKU Franchise by Product:

    Net Product Revenues by Product for the PKU Franchise

    (In millions of U.S. dollars)





    Three Months Ended

    June 30,



    Six Months Ended

    June 30,



    2021



    2020



    % Change



    2021



    2020



    % Change



    (unaudited)



    (unaudited)







    (unaudited)



    (unaudited)





    Kuvan

    $

    78.8





    $

    122.6





    (36)

    %



    $

    149.6





    $

    244.6





    (39)

    %

    Palynziq

    59.0





    40.7





    45

    %



    113.0





    75.3





    50

    %

    Total PKU franchise

    $

    137.8





    $

    163.3





    (16)

    %



    $

    262.6





    $

    319.9





    (18)

    %

     



    Non-GAAP Information

    The results presented in this press release include both GAAP information and Non-GAAP information. As used in this release, Non-GAAP Income is defined by the Company as GAAP Net Income/Loss excluding net interest expense, provision for income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and, in certain periods, certain other specified items, as detailed below when applicable. In addition, BioMarin includes in this press release the effects of these adjustments on certain components of GAAP Net Income/Loss for each of the periods presented. In this regard, Non-GAAP Income and its components, including Non-GAAP Cost of Sales, Non-GAAP Research and Development expenses, Non-GAAP Selling, General and Administrative expense, Non-GAAP Intangible Asset Amortization and Contingent Consideration, Non-GAAP Gain on the Sale of Intangible Asset and Non-GAAP Benefit From Income Taxes are statement of operations line items prepared on the same basis as, and therefore components of, the overall Non-GAAP measures.

    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 and its components are important internal measurements for BioMarin, the Company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the Company's results from period to period and to its forward-looking guidance, and to identify operating trends in the Company's principal business. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure.

    Non-GAAP Income and its components are not meant to be considered in isolation, 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 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 measures; likewise, the Company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP measures. Because of the non-standardized definitions, the Non-GAAP 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 table presents the reconciliation of GAAP Net Income (Loss) to Non-GAAP Income:

     

    Reconciliation of GAAP Net Income (Loss) to Non-GAAP Income

    (In millions of U.S. dollars)

    (unaudited)





    Three Months Ended

    June 30,



    Six Months Ended

    June 30,



    Guidance

    Year Ending



    2021



    2020



    2021



    2020



    December 31, 2021

























    GAAP Net Income (Loss)

    $

    12.9





    $

    (29.2)





    $

    30.3





    $

    52.2





    $

    (110.0)



    $

    (60.0)



























    Interest expense (income), net

    (0.7)





    3.7





    0.7





    5.4





    11.0



    11.0



    Provision for (benefit from) income taxes

    1.2





    (13.1)





    7.1





    6.9





    (16.0)



    (16.0)



    Depreciation expense

    11.8





    9.7





    24.3





    20.0





    48.0



    48.0



    Amortization expense

    15.5





    15.5





    30.9





    31.2





    62.0



    62.0



    Stock-based compensation expense

    54.9





    45.1





    104.3





    92.0





    186.0



    186.0



    Contingent consideration expense (benefit)

    2.2





    (0.6)





    4.5





    (0.6)





    9.0



    9.0



    Gain on sale of nonfinancial assets













    (59.5)









    Licensed In-Process R&D (1)





    26.3









    26.3









    Non-GAAP Income

    $

    97.8





    $

    57.4





    $

    202.1





    $

    173.9





    $

    190.0



    $

    240.0



     

    (1)

    Represents the upfront license fee paid to a third party and recognized as R&D expense in the second quarter of 2020.

    The following reconciliation of the GAAP reported to the Non-GAAP information provides the details of the effects of the Non-GAAP adjustments on certain components of the Company's operating results for each of the periods presented.

     

    Reconciliation of Certain GAAP Reported Information to Non-GAAP Information

    (In millions of U.S. dollars)

    (unaudited)





    Three months ended June 30,



    2021



    2020







    Adjustments











    Adjustments







































    GAAP

    Reported



    Interest,

    Taxes,

    Depreciation

    and

    Amortization



    Stock-Based

    Compensation,

    Contingent

    Consideration

    and Other

    Adjustments



    Non-GAAP



    GAAP

    Reported



    Interest,

    Taxes,

    Depreciation

    and

    Amortization



    Stock-Based

    Compensation,

    Contingent

    Consideration

    and Other

    Adjustments



    Non-GAAP

    Cost of sales

    $

    127.1





    $





    $

    (6.1)





    $

    121.0





    $

    97.9





    $





    $

    (4.9)





    $

    93.0



    Research and

    development

    161.1





    (7.1)





    (20.8)





    133.2





    182.1





    (4.6)





    (42.3)





    135.2



    Selling, general and

    administrative

    184.2





    (4.7)





    (28.0)





    151.5





    175.4





    (5.1)





    (24.2)





    146.1



    Intangible asset

    amortization and

    contingent

    consideration

    17.7





    (15.5)





    (2.2)









    14.9





    (15.5)





    0.6







    Interest income

    (expense), net

    0.7





    (0.7)













    (3.7)





    3.7











    Provision for (benefit

    from) income taxes

    1.2





    (1.2)













    (13.1)





    13.1











    GAAP Net Income

    (Loss) / Non-GAAP

    Income

    $

    12.9





    $

    27.8





    $

    57.1





    $

    97.8





    $

    (29.2)





    $

    15.8





    $

    70.8





    $

    57.4



     

     



    Six months ended June 30,



    2021



    2020







    Adjustments











    Adjustments







































    GAAP

    Reported



    Interest,

    Taxes,

    Depreciation

    and

    Amortization



    Stock-Based

    Compensation,

    Contingent

    Consideration

    and Other

    Adjustments



    Non-GAAP



    GAAP

    Reported



    Interest,

    Taxes,

    Depreciation

    and

    Amortization



    Stock-Based

    Compensation,

    Contingent

    Consideration

    and Other

    Adjustments



    Non-GAAP

    Cost of sales

    $

    247.2





    $





    $

    (12.6)





    $

    234.6





    $

    209.3





    $





    $

    (10.0)





    $

    199.3



    Research and

    development

    309.8





    (14.6)





    (38.3)





    256.9





    324.4





    (9.4)





    (56.0)





    259.0



    Selling, general and

    administrative

    358.5





    (9.7)





    (53.4)





    295.4





    362.7





    (10.6)





    (52.3)





    299.8



    Intangible asset

    amortization and

    contingent

    consideration

    35.4





    (30.9)





    (4.5)









    30.6





    (31.2)





    0.6







    Gain on sale of

    nonfinancial assets

    0.0

















    (59.5)









    59.5







    Interest expense, net

    (0.7)





    0.7













    (5.4)





    5.4











    Provision for income

    taxes

    7.1





    (7.1)













    6.9





    (6.9)











    GAAP Net Income /

    Non-GAAP Income

    $

    30.3





    $

    63.0





    $

    108.8





    $

    202.1





    $

    52.2





    $

    63.5





    $

    58.2





    $

    173.9



     

    Contact:





    Investors:



    Media:

    Traci McCarty



    Debra Charlesworth

    BioMarin Pharmaceutical Inc.



    BioMarin Pharmaceutical Inc.

    (415) 455-7558



    (415) 455-7451

     

     

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-announces-second-quarter-2021-financial-results-and-corporate-updates-301343556.html

    SOURCE BioMarin Pharmaceutical Inc.

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  2. SAN RAFAEL, Calif., July 21, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today new data for valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A, from its open-label Phase 1/2 study during an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.   

    Five-year and four-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts, respectively, shows a sustained treatment benefit of valoctocogene roxaparvovec.  All participants in both cohorts remain off prophylactic Factor VIII treatment. 

    Annualized Bleed Rate

    The 6e13 vg/kg dose cohort of the Phase 1/2 study (N=7), with a mean follow-up of 266.1 weeks (5.1 years), showed that a single dose of valoctocogene roxaparvovec after week 4 reduced mean ABR by 95% from 16.3 (median 16.5) at baseline to 0.8 (median 0.0) bleeding episodes per year among the 6 participants previously treated with FVIII prophylaxis.  In year 5, 86% (6 of 7) of study participants in the 6e13 vg/kg dose cohort had no treated bleeds.

    The 4e13 vg/kg dose cohort of the Phase 1/2 study (N=6), with a mean follow-up of 218.6 weeks (4.2 years) showed that a single dose of valoctocogene roxaparvovec reduced mean ABR by 92% from 12.2 (median 8.0) at baseline to 1.0 (median 0.5) bleeding episodes per year.  In year 4, 50% (3/6) of study participants in the 4e13 vg/kg dose cohort had no treated bleeds.

    Factor VIII Utilization

    In the 6e13 vg/kg dose cohort after week 4, the mean annualized Factor VIII utilization was  reduced by 96% from 135.6 (median 136.5) to 5.2 (median 0.1) infusions per year among the six participants previously treated with FVIII prophylaxis.  (This excludes one study participant receiving on-demand Factor VIII prophylaxis at baseline.)

    In the 4e13 vg/kg dose cohort after week 4, the mean annualized Factor VIII utilization was reduced by 95% from 142.8 (median 155.8) to 7.8 (median 1.4) infusions per year.  During follow-up, in both cohorts, exogenous Factor VIII was used as treatment for bleeding, surgery or procedures, and as one-time prophylaxis.

    Factor VIII Expression and Rate of Change Over Time Comparable in Phase 1/2 and Pivotal Phase 3 Studies

    For the 6e13 vg/kg and 4e13 vg/kg cohorts, study participants continued to have clinically meaningful levels in endogenous Factor VIII expression (Table 1). Mean Factor VIII activity levels over five and four years, respectively, support the observed reductions in bleed rates and annualized Factor VIII usage.

    Table 1:  Factor VIII Activity Levels of 4e13 and 6e13 vg/kg dose cohorts for 4 and 5 Years respectively as measured by CS and OS assays

    Factor VIII Activity, IU/dL

    Chromogenic Substrate

    Assay

     

    Mean (SD)

    Median

     

     

    One-Stage Assay

     

     

    Mean (SD)

    Median

    4e13 vg/kg Dose Cohort at

    4 Years (N=6)

     

    5.6 (5.6)

     

    4.8

     

     

    9.5 (7.0)

     

    7.5

    6e13 vg/kg Dose Cohort at

    5 Years (N=7)

     

    11.6 (12.2)

     

    8.2

     

     

    18.7 (17.5)

     

    15.7

     

    Factor VIII activity levels for participants in the 6e13 and 4e13 vg/kg vg/kg dose cohorts, was highest in year 1, and the rate of Factor VIII decline in years 4 and 5 were commensurate with that observed in previous years.  Earlier, results from the pivotal Phase 3 GENEr8-1 study were presented at ISTH.  The mean Factor VIII activity from a subset of the population that had been dosed at least two years prior to the data cut date (N=17) falls between Factor VIII activity of the 6e13 and 4e13 vg/kg dose cohorts in the Phase 1/2 study and is of help in understanding the potential future trajectory of Factor VIII activity in the GENEr8-1 study.  In the subset of the GENEr8-1 study that had been dosed at least two years prior to the data cut date, Factor VIII expression declined from a mean of 42.2 (median 23.9) IU/dL at the end of year one to 24.4 (median 14.7) IU/dL at the end of year two with continued hemostatic efficacy as measured by the chromogenic substrate (CS) assay.

    Individual Participant ABR, Factor VIII Infusion Rate, and Factor VIII Activity

    In the 6e13 and 4e13 vg/kg dose cohorts, after week 4, all participants continued to experience a reduction in ABR compared to their baseline values, even participants with low Factor VIII activity.  No participants chose to resume routine prophylaxis.

    In the most recent year of observation in  the 6e13 vg/kg dose cohort, six of the seven participants were in the mild to moderate hemophilia range, and one participant was below the lower limit of quantification as measured by the CS assay.  Measuring with the one-stage (OS) assay, one participant was in the non-hemophilic range, four were in the mild range, and one was in the moderate range.

    In the 4e13 vg/kg dose cohort, four of the six participants were in the mild to moderate hemophilia range, and two participants were below the lower limit of quantification as measured by the CS assay.  Measuring with the OS assay, all six participants were in the mild to moderate range. 

    "The consistent and impressive bleed control in the majority of the study participants out to five years in this study, which is the longest duration of clinical experience for any gene therapy in hemophilia A, which increases our understanding of the interplay between Factor VIII expression, ABR, and Factor VIII infusion rate as it relates to hemostatic efficacy," said Professor Michael Laffan, faculty of Medicine, Department of Immunology and Inflammation at Imperial College London, Director of the Hammersmith Hospital Haemophilia Centre, and Chief Investigator for the valoctocogene roxaparvovec Phase 1/2 study.  "These data show that most of the study participants have not had a bleed or had to infuse Factor VIII in the last five years after one infusion of valoctocogene roxaparvovec, which has the potential to provide a treatment choice that addresses many of the unmet needs in hemophilia."

    "We are optimistic that ABR may be maintained acceptably low through years three, four, and five after treatment with valoctocogene roxaparvovec in the GENEr8-1 study given the predictable, and not sudden or dramatic, change in Factor VIII expression in the later years following treatment in the Phase 1/2 study," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin.  "The data show excellent hemostatic efficacy in the 6e13 and 4e13 vg/kg dose cohorts, which is maintained into year five and four, respectively.  We look forward to sharing topline two-year data from all participants in the pivotal Phase 3 GENEr8-1 study in early 2022."

    Safety Summary

    Overall, the safety profile of valoctocogene roxaparvovec in the Phase 1/2 study remains consistent with previously reported data with no delayed-onset treatment related adverse events.  All participants continue to remain off corticosteroids since the first year.  No participants developed inhibitors to Factor VIII, and no participants withdrew from the study.  No participants have developed thrombotic events.  The most common adverse events associated with valoctocogene roxaparvovec occurred early after a single infusion and included short-lived infusion-associated reactions and transient, asymptomatic, and mild to moderate rise in the levels of certain proteins and enzymes measured in liver function tests with no long-lasting clinical sequelae.

    Regulatory Status

    The European Medicines Agency (EMA) validated BioMarin's resubmission of a Marketing Authorization Application (MAA) on July 15, 2021.  In May 2021, the EMA granted the Company's request for accelerated assessment.  Accelerated assessment potentially reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a MAA for an Advanced Therapy Medicinal Product (ATMP), although an application initially designated for accelerated assessment can revert to the standard procedure during the review for a variety of reasons. The decision to grant accelerated assessment has no impact on the eventual CHMP and CAT opinion on whether a marketing authorization should be granted.  A CHMP and CAT opinion is anticipated in the first half of 2022. 

    The MAA submission includes safety and efficacy data from the 134 subjects enrolled in the Phase 3 GENEr8-1 study, all of whom have been followed for at least one year after treatment with valoctocogene roxaparvovec, as well as four and three years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts, respectively, in the ongoing Phase 1/2 dose escalation study.

    In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase 3 GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.

    The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are intended to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received in 2017. 

    In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. 

    Robust Clinical Program

    BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A.  In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is actively enrolling participants in a Phase 3b, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A.  The Company is running a Phase 1/2 Study with the 6e13kg/vg dose of valoctocogene roxaparvovec in approximately 10 participants with pre-existing AAV5 antibodies, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors. 

    About Hemophilia A

    People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints.  Individuals with the most severe form of hemophilia A make up approximately 45 to 50% of the hemophilia A population.  People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much-reduced propensity to bleed.  The standard of care for adults with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year.  Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

    Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A.

    About ISTH

    Founded in 1969, the ISTH is the leading worldwide not-for-profit organization dedicated to advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders. The ISTH is an international professional membership organization with more than 7,700 clinicians, researchers and educators working together to improve the lives of patients in more than 110 countries around the world. Among its highly regarded activities and initiatives are education and standardization programs, research activities, meetings and congresses, peer-reviewed publications, expert committees and World Thrombosis Day on 13 October.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

    Forward-Looking Statements

    This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including without limitation, statements about: (i) the development of BioMarin's valoctocogene roxaparvovec program generally, (ii) the impact and potential of valoctocogene roxaparvovec gene therapy for treating patients with severe hemophilia A, (iii) plans to submit two-year follow-up safety and efficacy data on all study participants from the GENEr8-1 study in response to FDA's request for these data to support their benefit-risk assessment of valoctocogene roxaparvovec, (iv) our target Biologics License Application (BLA) submission date in the second quarter of 2022 assuming favorable study results, followed by an expected six-month review procedure by the FDA, (v) the potential approval and commercialization of valoctocogene roxaparvovec for the treatment of severe hemophilia A, including timing of such approval decisions, and (vi) sharing topline two-year data from all participants in the pivotal Phase 3 GENEr8-1 study in early 2022.

    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: results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec, including final analysis of the above interim data; any potential adverse events observed in the continuing monitoring of the patients in the Phase 1/2 trial; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities, including the potential impact of the COVID-19 pandemic on the regulatory authorities' abilities to issue such decisions and the timing of such decisions; the content and timing of decisions by local and central ethics committees regarding the clinical trials; BioMarin's ability to successfully manufacture valoctocogene roxaparvovec for the clinical trials and commercially, if approved; and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

    BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.

     

    Contacts:



    Investors

    Media

    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558 

    (415) 455-7451

     

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-announces-oral-presentation-at-international-society-on-thrombosis-and-haemostasis-isth-2021-virtual-congress-with-5-years-of-clinical-data-from-ongoing-phase-12-study-of-valoctocogene-roxaparvovec-in-adults-with-sever-301338345.html

    SOURCE BioMarin Pharmaceutical Inc.

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  3. SAN RAFAEL, Calif., July 19, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced new data for valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, in its positive pivotal study, GENEr8-1, during an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.  The pivotal study demonstrated superiority to Factor VIII prophylaxis in key clinical efficacy endpoints.  With 134 participants, this is the largest global Phase 3 study to date for gene therapy in hemophilia.  All participants in the study received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up.  Top-line one-year…

    SAN RAFAEL, Calif., July 19, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced new data for valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, in its positive pivotal study, GENEr8-1, during an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.  The pivotal study demonstrated superiority to Factor VIII prophylaxis in key clinical efficacy endpoints.  With 134 participants, this is the largest global Phase 3 study to date for gene therapy in hemophilia.  All participants in the study received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up.  Top-line one-year results from this study were previously communicated in January 2021.

    New data presented at ISTH include more details on annualized bleeding rate (ABR) in all study participants and annualized Factor VIII utilization rate, in terms of international units per kilogram per year (IU/kg/year) of replacement Factor VIII.  Over 90 percent (N=134) of all participants in the GENEr8-1 study had an annualized bleed rate (ABR) of zero or a lower bleed rate than baseline after week 4 after treatment with valoctocogene roxaparvovec. 

    New data presented at ISTH also include information on Factor VIII utilization after treatment with valoctocogene roxaparvovec.  Mean annualized Factor VIII utilization rate, among a pre-specified group of prior participants in a non-interventional baseline observational study (rollover population; N=112) decreased from baseline on Factor VIII prophylaxis by 99% from 3961.2 (median 3754.4) to 56.9 (median 0) IU/kg/year after week 4 after treatment with valoctocogene roxaparvovec (p-value <0.001). 

    As previously shared in January 2021, data from the pre-specified rollover population (N=112) in the GENEr8-1 study with a mean follow-up of 71.6 weeks demonstrated that in the pre-specified primary analysis for ABR, calculated through each subject's last assessment, a single dose of valoctocogene roxaparvovec significantly reduced mean ABR by 84% from a prospectively collected 4.8 (median 2.8) at baseline to 0.8 (median 0.0) bleeding episodes per year (p-value <0.001).

    In addition, the mean annualized Factor VIII infusion rate was reduced by 99% from 135.9 (median 128.6) to 2.0 (median 0.0) infusions per year (p-value <0.001).  

    Table 1:  Mean/Median Annualized Bleeding Rate (ABR) and FVIII Infusion Rate in Phase 3 GENEr8-1 Study Rollover Population (N=112) after Week 4 Through Week 52 at November 2020 Cut Off



    Phase 3

    Rollover Population*

    On Factor VIII prophylaxis, before

    valoctocogene roxaparvovec infusion

    N=112

    Phase 3

    Rollover Population*

    After valoctocogene roxaparvovec infusion

    N=112



    Mean (SD)

    Median (IQR)

    Mean (SD)

    Median (IQR)

    Annualized Bleeding

    Rate (bleeding

    episodes per year)

    4.8 (6.5)

    2.8 (0.0, 7.6)

    0.8 (3.0)

    0.0 (0.0, 0.0)

    Annualized

    FVIII Utilization Rate
     (IU

    per kg per year)
     

    3961.2 (1751.5) 

    3754.4 (2799.5, 4706.8) 

    56.9 (194.6) 

    0.0 (0.0, 22.1) 

    Annualized FVIII

    Infusion Rate

    (infusions per year)

    135.9 (52.0)

    128.6 (104.1, 159.9)

    2.0 (6.4)

    0.0 (0.0, 0.9)



    *See study description for patient population information.

    Study participants also experienced a clinically meaningful increase in endogenous Factor VIII expression. At the end of the first year post-infusion with valoctocogene roxaparvovec, participants in the modified intent-to-treat (mITT) population (N=132) had a significant increase in mean endogenous Factor VIII expression level from an imputed baseline of 1 IU/dL to 42.9 IU/dL (median 23.9) (p-value <0.001) as measured by the chromogenic substrate (CS) assay, supporting the marked clinical benefits observed with abrogation of bleeding episodes and Factor VIII utilization and infusion rates.  In a subset of the mITT population that had been dosed at least two years prior to the data cut date (N=17), Factor VIII expression declined from a mean of 42.2 (median 23.9) IU/dL at the end of year one to 24.4 (median 14.7) IU/dL at the end of year two with continued hemostatic efficacy.

    Table 2:  Factor VIII Activity Levels in 12-Month Intervals

    Median Factor

    VIII Activity,

    IU/dL

    Phase 3 mITT Population*

    (N=132)

    Mean (SD)

    Median

    Phase 3 mITT Subset Population**

    (N=17)

    Mean (SD)

    Median

    Week 52

    42.9 (45.5)

    23.9 (11.9, 62.3)

    42.2 (50.9)

    23.9 (11.2, 55.0

    Week 104

    N/A

    24.4 (29.2)

    14.7 (6.4, 28.6)



    * mITT = modified intent-to-treat population, which excludes 2 HIV- positive subjects dosed 2 or more years prior to November 2020 data cut.

    **Includes only HIV-negative subjects dosed 2 or more years prior to Nov 2020 data cut date. One participant was lost to follow-up at 66.1 weeks and was henceforth imputed to have a Factor VIII activity of 0 IU/dL through 104 weeks.

    "The demonstrated bleed control at 52 weeks and beyond in this pivotal study supports our thesis that gene therapy can play an important role in the treatment of severe hemophilia A and potentially creates the possibility for a new treatment paradigm," said Margareth C. Ozelo, MD, PhD, Director, INCT do Sangue Hemocentro UNICAMP, University of Campinas and Lead Principal Investigator of the GENEr8-1 Study. "It is encouraging to see meaningful endogenous Factor VIII expression and decreases in bleeding and Factor VIII infusions for people in this study.  These pivotal results contribute to the growing body of data to increase understanding of the safety and efficacy of gene therapy treatment over time." 

    "From the start of our valoctocogene roxaparvovec program, our goal remains to advance treatment options for people with severe hemophilia A in light of the unmet need in bleed control. Current prophylactic therapies for hemophilia A cannot maintain Factor VIII levels for sustained periods, leading to the need for frequent, regular infusions or injections while still having a risk of ongoing, unpredictable bleeds and unavoidable, irreversible joint damage even with standard of care treatment," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "These data build upon the foundation for a potential transformative treatment option that addresses the root cause of severe hemophilia A.  Later at ISTH, we look forward to sharing five years of clinical data from the ongoing Phase 1/2 study with the longest duration of clinical experience, which complements this pivotal Phase 3 study, the largest study of a gene therapy in hemophilia A."

    Valoctocogene Roxaparvovec Safety

    Overall, in the Phase 3 study, valoctocogene roxaparvovec has been well tolerated by the 134 participants who received a single 6e13 vg/kg dose. No participants withdrew due to adverse events.  No participants developed inhibitors to Factor VIII, or experienced thromboembolic events.  One participant was lost to follow-up.  Infusion reactions were defined as any AEs occurring within 48 hours post-infusion.  The most common infusion reactions were nausea (14.2%), fatigue (7.5%), and headache (6.0%).  Systemic hypersensitivity during or following infusion was mitigated by slowing or pausing infusion and treating with supportive medications, as indicated.  All four (3.0%) participants with an interruption due to infusion-related symptoms were able to complete their infusion.  Twenty-two (16.4%) participants experienced a total of 43 serious adverse events (SAEs), and all SAEs resolved. 

    Common, steroid-related side effects can occur with temporary use of corticosteroid (or alternative immunosuppressants) to manage ALT elevation.  ALT elevation was the most common AE.  Overall, 79% of participants received corticosteroids per protocol as treatment for ALT elevation.  The average duration of corticosteroid treatment was 33 weeks.  Overall, 72% of participants who used any corticosteroids reported AEs attributed to their use, most commonly acne, insomnia, cushingoid changes, and weight increased.  Three participants reported SAEs attributed to corticosteroids.  Other immunosuppressants were used by 29% of participants for ALT elevation due to contraindication, side effects, or poor or no response to corticosteroid treatment.  No Grade 4 ALT elevations occurred, and no participants met Hy's law criteria for drug-induced liver injury.

    GENEr8-1 Study Description

    The global Phase 3 GENEr8-1 study evaluates superiority of valoctocogene roxaparvovec at the 6e13 vg/kg dose compared to FVIII prophylactic therapy.  All study participants had severe hemophilia A at baseline, defined as less than or equal to 1 IU/dL of Factor VIII activity.  The study included 134 total participants, all of whom had a minimum of 12 months of follow-up at the time of the data cut.  The first 22 participants were directly enrolled into the Phase 3 study, 17 of whom were HIV-negative and dosed at least 2 years prior to the data cut date (referred to as the subset).  The remaining 112 participants (rollover population) completed at least six months in a separate non-interventional study to prospectively assess bleeding episodes, Factor VIII use, and health-related quality of life while receiving Factor VIII prophylaxis prior to rolling over to receive a single infusion of valoctocogene roxaparvovec in the GENEr8-1 study.

    Regulatory Status

    The European Medicines Agency (EMA) validated BioMarin's resubmission of a Marketing Authorization Application (MAA) on July 15, 2021.  In May 2021, the EMA granted the Company's request for accelerated assessment.  Accelerated assessment potentially reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a MAA for an Advanced Therapy Medicinal Product (ATMP), although an application initially designated for accelerated assessment can revert to the standard procedure during the review for a variety of reasons. The decision to grant accelerated assessment has no impact on the eventual CHMP and CAT opinion on whether a marketing authorization should be granted.  A CHMP and CAT opinion is anticipated in the first half of 2022. 

    The MAA submission includes safety and efficacy data from the 134 subjects enrolled in the Phase 3 GENEr8-1 study, all of whom have been followed for at least one year after treatment with valoctocogene roxaparvovec, as well as four and three years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts, respectively, in the ongoing Phase 1/2 dose escalation study.

    In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase 3 GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.

    The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are intended to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received in 2017. 

    In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. 

    Robust Clinical Program

    BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A.  In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is actively enrolling participants in a Phase 3b, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with hemophilia A.  The Company is also running a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with pre-existing AAV5 antibodies, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors. 

    About Hemophilia A

    People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints.  Individuals with the most severe form of hemophilia A make up approximately 45 to 50 percent of the hemophilia A population.  People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much-reduced propensity to bleed.  The standard of care for adults with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year.  Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

    Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A.

    About ISTH

    Founded in 1969, the ISTH is the leading worldwide not-for-profit organization dedicated to advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders. The ISTH is an international professional membership organization with more than 7,700 clinicians, researchers and educators working together to improve the lives of patients in more than 110 countries around the world. Among its highly regarded activities and initiatives are education and standardization programs, research activities, meetings and congresses, peer-reviewed publications, expert committees and World Thrombosis Day on 13 October.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

    Forward-Looking Statements

    This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including without limitation, statements about (i) the development of BioMarin's valoctocogene roxaparvovec program generally, (ii) the anticipated timing of a CHMP and CAT opinion in the first half of 2022, (iii) BioMarin's intention to submit to the U.S. Food and Drug Administration (FDA) two-year follow-up safety and efficacy data on all study participants from the GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, (iv) BioMarin targeting resubmission of a Biologics License Application in the second quarter of 2022 assuming favorable study results, followed by an expected six-month review procedure by the FDA,  (v) the anticipated Phase 1/2 study to be presented later at ISTH and (vi) the timing of the regulatory activities in the U.S and Europe, including validation and timing of potential approvals and the expected review procedures.  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: results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec, including final analysis of the above data and additional data from the continuation of these trials; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the EMA and other regulatory authorities; the content and timing of decisionsby local and central ethics committees regarding the clinical trials; our ability to successfully manufacture valoctocogene roxaparvovec for the clinical trials and commercially, if approved; and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, and future filings and reports by BioMarin... BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

    BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.

    Contacts:



    Investors

    Media

    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558

    (415) 455-7451

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-announces-oral-presentation-of-positive-one-year-results-from-phase-3-pivotal-trial-with-valoctocogene-roxaparvovec-gene-therapy-in-adults-with-severe-hemophilia-a-at-international-society-on-thrombosis-and-haemostasis-i-301336202.html

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  4. SAN RAFAEL, Calif., July 15, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.  With today's validation the MAA review can now commence.  A CHMP opinion is anticipated in the first half of 2022.

    The submission includes safety and efficacy data from the 134 subjects enrolled in the Phase 3 GENEr8-1 study, all of whom have been followed for at least one year after treatment with valoctocogene roxaparvovec, as well as four and three years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts, respectively, in the ongoing Phase 1/2 dose escalation study. 

    "We look forward to working with the agency as it evaluates the robust data set in this application, which we believe address the requests made during the prior MAA review.  This pivotal study demonstrated superiority of valoctocogene roxaparvovec compared to the standard of care, prophylactic Factor VIII replacement," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin.  "The extensive data set for valoctocogene roxaparvovec is supported by decades of scientific and clinical research in the field of gene therapy.  We continue to contribute to the body of scientific knowledge and will be sharing results from our Phase 1/2 and Phase 3 studies with the community at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Congress next week.  We believe that this gene therapy has the potential to fulfill the unmet medical needs in the community."

    In May 2021, the EMA granted the Company's request for accelerated assessment of the application. Applications are eligible for accelerated assessment if the CHMP and CAT decide the product is of major interest for public health, particularly from the point of view of therapeutic innovation. Evaluating a MAA under the EMA centralized procedure can take up to 210 days, not counting clock stops when applicants are requested to provide additional information. On request, the CHMP and CAT can reduce the time frame to 150 days if the applicant provides sufficient justification for an accelerated assessment, although an application initially designated for accelerated assessment can revert to the standard procedure during the review for a variety of reasons. The decision to grant accelerated assessment has no impact on the eventual CHMP and CAT opinion on whether a marketing authorization should be granted.

    Regulatory Status

    BioMarin resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) on June 25, 2021In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase 3 GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.

    The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are intended to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received in 2017. 

    In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. 

    Robust Clinical Program

    BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A.  In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is actively enrolling participants in a Phase 3b, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with hemophilia A.  The Company is also running a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with pre-existing AAV5 antibodies, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors. 

    About Hemophilia A

    People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints.  Individuals with the most severe form of hemophilia A make up approximately 45 to 50 percent of the hemophilia A population.  People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much-reduced propensity to bleed.  The standard of care for adults with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year.  Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

    Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

    Forward-Looking Statements

    This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about (i) the development of BioMarin's valoctocogene roxaparvovec program generally, (ii) the anticipated timing of a CHMP opinion in the first half of 2022, (iii) BioMarin's intention to submit to the U.S. Food and Drug Administration (FDA) two-year follow-up safety and efficacy data on all study participants from the GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, (iv) BioMarin targeting resubmission of a Biologics License Application in the second quarter of 2022 assuming favorable study results, followed by an expected six-month review procedure by the FDA,  and (v) the timing of the regulatory activities in the U.S and Europe, including validation and timing of potential approvals and the expected review procedures.  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: results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec; additional data from the continuation of the Phase 1/2 trial and the Phase 3 trial, any potential adverse events observed in the continuing monitoring of the participants in the clinical trials; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture valoctocogene roxaparvovec for the clinical trials and commercially, if approved; and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

    BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.

    Contacts:

    Investors

    Media

    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558

    (415) 455-7451

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/european-medicines-agency-validates-biomarins-marketing-authorization-application-for-valoctocogene-roxaparvovec-to-treat-severe-hemophilia-a-301334635.html

    SOURCE BioMarin Pharmaceutical Inc.

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  5. SAN RAFAEL, Calif., July 14, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday, July 28, at 4:30 p.m. ET to discuss second quarter 2021 financial results and provide a general business update.

    Dial-in Number 

    U.S. / Canada Dial-in Number: (866) 502-9859

    International Dial-in Number: (574) 990-1362

    Conference ID: 8363378

    Replay Dial-in Number: (855) 859-2056

    Replay International Dial-in Number: (404) 537-3406

    Conference ID: 8363378

    Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.biomarin.com. A replay of the call will be archived on the site for one week following the call.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare genetic diseases. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.  For additional information, please visit www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

    Contacts:

    Investors 

    Media

    Traci McCarty 

    Debra Charlesworth

    BioMarin Pharmaceutical Inc. 

     BioMarin Pharmaceutical Inc.

    (415) 455-7558 

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    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-to-host-second-quarter-2021-financial-results-conference-call-and-webcast-on-wednesday-july-28-at-430pm-et-301333393.html

    SOURCE BioMarin Pharmaceutical Inc.

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