1. SAN RAFAEL, Calif., March 3, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the company has completed full enrollment in a global Phase 2 randomized, placebo-controlled study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. The 52-week study consists of approximately 70 infants and young children with achondroplasia, aged zero to less than five years old (60 months). The study will be followed by a subsequent open-label extension trial where all children receive active treatment. Children in this study will have completed a minimum three- or six-month baseline…

    SAN RAFAEL, Calif., March 3, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the company has completed full enrollment in a global Phase 2 randomized, placebo-controlled study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. The 52-week study consists of approximately 70 infants and young children with achondroplasia, aged zero to less than five years old (60 months). The study will be followed by a subsequent open-label extension trial where all children receive active treatment. Children in this study will have completed a minimum three- or six-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on growth. The company also plans to augment the height data with assessments including proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries.

    There are currently no approved pharmacological treatments for achondroplasia, with existing treatments mainly limited to surgical interventions to address a variety of symptoms. This treatment gap presents a significant unmet need. Vosoritide is an investigational therapy that seeks to directly target the root cause of achondroplasia by interrupting the pathway that slows bone growth due to the causative mutation in achondroplasia. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, such as foramen magnum compression, sleep apnea, and spinal stenosis. Some of these complications can result in the need for invasive surgeries. In addition, studies show increased mortality at every age.

    "This milestone is an important building block of a comprehensive clinical program that is methodically and responsibly studying this potential first pharmacological treatment choice for achondroplasia with the goal of further understanding the safety and efficacy in the youngest children," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "In this trial, we are studying the effects of vosoritide during the most productive time of growth. We are grateful to the children and families enrolled in this placebo-controlled study and are committed to the long-term follow up of the children in these studies."

    "This is an exciting milestone for children and families, who are interested in a treatment choice to address the basic cause of the irregular bone growth seen in achondroplasia. It represents a potential medical breakthrough that would be the first non-surgical treatment for children with achondroplasia," said John A. Phillips, III, M.D., Vanderbilt University Medical Center (David T Karzon Professor of Pediatrics) and investigator for the vosoritide clinical program. "As a treating physician, I see an urgent demand from families for a treatment option that addresses bone growth and potentially the serious complications associated with achondroplasia, especially during infancy." 

    "This milestone is a giant step towards improving the quality of medical care and options available to individuals with achondroplasia and to their families," said Munira Shamim, co-founder of Growing Stronger. Many families are eagerly awaiting a drug treatment option that could possibly decrease the risk of health issues associated with achondroplasia and increase the quality of life. We would like to recognize the committed families and participants in the placebo-controlled studies for collaborating with dedicated scientists to further scientific learning that can potentially change the lives of thousands of families."

    Regulatory Status

    In 2020, the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) accepted and validated the marketing authorization application for vosoritide for achondroplasia. The Committee for Medicinal Products for Human Use (CHMP) opinion is expected in Europe in June of 2021. The U.S. New Drug Application (NDA) for vosoritide is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of August 20, 2021. In the United States, the Company has chosen to provide the 2-year outcomes from the Phase 3 extension study to the FDA as additional data to convey the vosoritide treatment effect and long-term durability. The Company believes that supplying this additional data could result in a major amendment, resetting the current PDUFA target action date out three months to November. 

    In January 2021, the Company received notice from the FDA that the NDA for vosoritide had been granted Priority Review Designation based on the serious pediatric indication it addresses, and the lack of treatment options currently available. Consistent with FDA's policy on changes to review classification for an ongoing application review, the PDUFA action date is not affected by this designation. If approved, the vosoritide NDA may qualify for a Priority Review Voucher (PRV). A PRV confers priority review to a subsequent drug application that would not otherwise qualify for that designation. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases. 

    Upon the acceptance of the regulatory submission for vosoritide, the Agency reiterated a position raised during the Pediatric Advisory Committee (PAC) and Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) held on May 11, 2018 recommending two-year controlled trials in different age groups. BioMarin believes the highly persuasive outcomes from the one-year randomized, double-blind, placebo-controlled Phase 3 trial, coupled with data from the Phase 2 program with up to 5 years of long-term follow-up that has been compared to robust natural history data on growth and the updated 2-year data from the Phase 3 study, offers a rigorous and reliable method to assess whether vosoritide has a durable impact on the rate of endochondral bone growth that ultimately increases final adult height. 

    Vosoritide has also received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia. 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.

    Description of Phase 2 Study in Infants and Young Children Ages 0 to 5 Years

    This is a Phase 2 randomized, placebo-controlled study of vosoritide. The 52-week study consists of approximately 70 infants and young children with achondroplasia, aged zero to less than five years old (60 months). The study will be followed by a subsequent open-label extension trial when all subjects receive active treatment. Children in this study will have completed a three-to-six-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The primary objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on height. The company also plans to augment the height data with other analyses of effects on growth and assessments including proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries. 

    About Achondroplasia

    Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, including foramen magnum compression, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, spinal stenosis and recurrent ear infections. Some of these complications can result in the need for invasive surgeries such as spinal cord decompression and straightening of bowed legs. In addition, studies show increased mortality at every age.

    More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. Vosoritide is being tested in children whose growth plates are still "open", typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.  In the U.S., Europe, Latin America, the Middle East, and most of Asia Pacific, there are currently no licensed medicines for achondroplasia.

    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 such website is not incorporated by reference into this press release.

    Forward-Looking Statement

    This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin's vosoritide program generally; the potential benefits of vosoritide for infants and young children; the continued clinical development of vosoritide; the timing, design and conduct of the planned Phase 2 study in infants and young children and the expectation that topline results from this Phase 2 study will be released in mid-2022; the timing, design and conduct of other ongoing and possible future studies of vosoritide; the expected results of such studies, the ability to use the primary objectives of the Phase 2 study to support the use of vosoritide in infants and young children; the timing of actions by regulatory authorities including the expectation of the CHMP opinion for vosoritide in Europe in June of 2021; the potential for the vosoritide NDA, if approved, to qualify for a Priority Review Voucher; and the plan to submit the second year of Phase 3 data to the FDA and the potential that this could result in a major amendment, resetting the current PDUFA date out three months to November. 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 vosoritide; our ability to enroll participants into such clinical trials, our ability to successfully manufacture vosoritide; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning vosoritide; and those other risks and uncertainties detailed from time to time under the caption "Risk Factors" and elsewhere in the Company's Securities and Exchange Commission (SEC) filings, including, without limitation, BioMarin's Annual Report on Form 10-K for the year ended December 31, 2020 as such factors may be updated by any subsequent reports. 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.

    Contact:



    Investors:

    Media:

    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558

    (415) 455-7451

     

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  2. SAN RAFAEL, Calif., Feb. 25, 2021 /PRNewswire/ --

    BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

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


    Three Months Ended December 31,


    Twelve Months Ended December 31,


    2020


    2019


    % Change


    2020


    2019


    % Change













    Total Revenues

    $

    452.1



    $

    454.4



    (1)

    %


    $

    1,860.5



    $

    1,704.0



    9

    %













    Net Product Revenues Marketed by BioMarin (1)

    435.8



    412.7



    6

    %


    1,675.8



    1,563.2



    7

    %













    Vimizim Net Product Revenues

    142.5



    132.3



    8

    %


    544.4



    544.3



    %

    Kuvan Net Product Revenues

    89.0



    122.6



    (27)

    %


    457.7



    463.4



    (1)

    %

    Naglazyme Net Product Revenues

    119.7



    94.8



    26

    %


    391.3



    374.3



    5

    %

    Palynziq Net Product Revenues

    49.6



    31.7



    56

    %


    171.0



    86.9



    97

    %

    Brineura Net Product Revenues

    35.0



    25.2



    39

    %


    110.2



    72.0



    53

    %













    Aldurazyme Net Product Revenues

    1.2



    23.9



    (95)

    %


    130.1



    97.8

    SAN RAFAEL, Calif., Feb. 25, 2021 /PRNewswire/ --

    BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

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



    Three Months Ended December 31,



    Twelve Months Ended December 31,



    2020



    2019



    % Change



    2020



    2019



    % Change

























    Total Revenues

    $

    452.1





    $

    454.4





    (1)

    %



    $

    1,860.5





    $

    1,704.0





    9

    %

























    Net Product Revenues Marketed by BioMarin (1)

    435.8





    412.7





    6

    %



    1,675.8





    1,563.2





    7

    %

























    Vimizim Net Product Revenues

    142.5





    132.3





    8

    %



    544.4





    544.3





    %

    Kuvan Net Product Revenues

    89.0





    122.6





    (27)

    %



    457.7





    463.4





    (1)

    %

    Naglazyme Net Product Revenues

    119.7





    94.8





    26

    %



    391.3





    374.3





    5

    %

    Palynziq Net Product Revenues

    49.6





    31.7





    56

    %



    171.0





    86.9





    97

    %

    Brineura Net Product Revenues

    35.0





    25.2





    39

    %



    110.2





    72.0





    53

    %

























    Aldurazyme Net Product Revenues

    1.2





    23.9





    (95)

    %



    130.1





    97.8





    33

    %

























    GAAP Net Income (Loss)

    $

    22.1





    $

    15.0









    $

    859.1





    $

    (23.8)







    GAAP Net Income (Loss) per Share – Basic

    $

    0.12





    $

    0.08









    $

    4.75





    $

    (0.13)







    GAAP Net Income (Loss) per Share – Diluted

    $

    0.12





    $

    0.08









    $

    4.53





    $

    (0.13)







    Non-GAAP Income (2)

    $

    39.5





    $

    46.4









    $

    312.2





    $

    166.6







     



    December 31,

    2020



    December 31,

    2019



    Cash, cash equivalents and investments

    $

    1,350.9





    $

    1,165.8



























    (1)

    Net Product Revenues Marketed by BioMarin is the sum of revenues from Vimizim, Kuvan, Naglazyme, Palynziq, Brineura and Firdapse, 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 8 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 twelve months ending December 31, 2020; as a result of the transaction BioMarin will not recognize Net Product Revenues from Firdapse in the future.

    (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 10 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 fourth quarter and full year ended December 31, 2020.

    Net Product Revenues for the fourth quarter of 2020 were essentially flat as compared to the fourth quarter of 2019. The change in Net Product Revenues was primarily attributed to the following:

    • Kuvan Net Product Revenues decreased by $33.6 million, primarily due to the U.S. loss of market exclusivity in October 2020 resulting from generic competition; and
    • Aldurazyme Net Product Revenues decreased by $22.7 million due to timing of product fulfillment to Genzyme. Aldurazyme is marketed by Genzyme and BioMarin Aldurazyme revenues are driven by the timing of when the product is released and control is transferred to Genzyme. Revenues for the fourth quarter of 2020 were comparatively lower than 2019 due to such timing. Based on data provided to us by Genzyme, patients receiving commercial Aldurazyme increased by 10% during 2020; partially offset by
    • Naglazyme and Vimizim Net Product Revenues increased by an aggregate of $35.1 million primarily due to timing of sales in the Middle East and Latin America;
    • Palynziq Net Product Revenues increased by $17.9 million driven by a combination of revenue from U.S. patients achieving maintenance dosing and new patients initiating therapy; and
    • Brineura Net Product Revenues increased by $9.8 million driven by growth in the number of patients in all regions.

    The increase in GAAP Net Income for the fourth quarter of 2020, compared to the same period in 2019 was primarily due to the following:

    • decreased research and development (R&D) expense of $16.1 million primarily due to lower clinical activity spend for valoctocogene roxaparvovec gene therapy programs and decreased tralesinidase alfa costs as the program was licensed to a third-party in 2019; and
    • an increase in the benefit from income taxes of $37.5 million primarily due to the change in the jurisdictional mix of earnings and the related tax impact from the completion of an intra-entity transfer of certain intellectual property rights to an Irish subsidiary where the Company's ex-US regional headquarters are located during the third quarter of 2020; partially offset by
    • an increase in Cost of Sales of $30.2 million primarily due to inventory reserves and higher sales volumes of products with lower margins.

    Non-GAAP Income for the fourth quarter of 2020 decreased to $39.5 million, compared to Non-GAAP Income of $46.4 million for the same period in 2019. The decrease in Non-GAAP Income for the quarter, compared to the same period in 2019, was primarily attributed to lower gross profits and higher SG&A expenses, partially offset by lower R&D expenses.

    As of December 31, 2020, BioMarin had cash, cash equivalents and investments totaling $1.35 billion, which includes net proceeds of $535.8 million from the Company's May 2020 convertible debt offering, as compared to $1.17 billion as of December 31, 2019. On October 15, 2020, the Company's 1.50% senior subordinate convertible notes matured and were settled in cash for approximately $375.0 million.

    Commenting on full-year 2020 results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "Despite the impact in 2020 from the COVID-19 pandemic and a delay in the potential approval of valoctocogene roxaparvovec for severe hemophilia A, demand for our current product portfolio continued to drive steady revenue growth and expansion of our pipeline.  Excluding contributions from Kuvan, for which a generic became available during 2020, total revenues grew 13% in 2020, and generated $85 million of positive operating cash flows for the full year, underscoring the essential nature of our medicines." 

    Mr. Bienaimé continued, "The most recent Phase 3 data updates from our latest-stage development programs in achondroplasia and severe hemophilia A demonstrated significant efficacy.  In the largest gene therapy trial ever conducted for the treatment of severe hemophilia A, we were pleased that valoctocogene roxaparvovec was the first in hemophilia A to demonstrate statistically significant evidence of annualized bleed rate superiority over standard of care recombinant FVIII. Based on these results, we are very encouraged that one infusion of valoctocogene roxaparvovec gene therapy may potentially address the high treatment burden for people with severe hemophilia A.  We are targeting submission of the one-year Phase 3 results to the European Medicines Agency in the second quarter of 2021 and planning to dialog with the FDA to align on steps to obtain approval in the United States."

    "Also in 2021, we look forward to the potential approval of vosoritide, which would be the first pharmacological treatment to address the underlying cause of achondroplasia, the most common form of dwarfism.  We announced in the fourth quarter of 2020 that vosoritide demonstrated sustained growth effects for over two years in children with achondroplasia participating in our Phase 3 extension study.  In addition to the large, Phase 3 program currently in the extension phase, we have built a multi-pronged dossier of additional studies to support our understanding of the unmet medical need for children with achondroplasia and the effects of vosoritide in this condition. In addition to the highly statistically significant placebo-controlled Phase 3 results, the program includes the long-term clinical results in 5 to 18 year-olds from our Phase 2 study, natural history data, and the ongoing study of newborns through 5 years. Many families are keen to seek early treatment for their children so we are hopeful that, if approved, vosoritide will become available later in 2021 upon potential approvals."

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

    Item



    2021 Guidance *

    Total Revenues



    $1,750



    to



    $1,850

    Vimizim Net Product Revenues



    $570



    to



    $610

    Kuvan Net Product Revenues



    $250



    to



    $290

    Naglazyme Net Product Revenues



    $365



    to



    $395

    Palynziq Net Product Revenues



    $210



    to



    $250

    Brineura Net Product Revenues



    $120



    to



    $140















    Cost of Sales (% of Total Revenues)



    23%



    to



    25%

    Research and Development Expense



    $645



    to



    $695

    Selling, General and Administrative Expense



    $725



    to



    $775















    GAAP Net Loss



    ($130)



    to



    ($80)

    Non-GAAP Income (1)



    $170



    to



    $220



















    *2021 Guidance takes into consideration ongoing expected impact from the COVID-19 pandemic in 2021 assuming consistent trends experienced during 2020.





    (1)

    All Financial Guidance items are calculated based on U.S. GAAP with the exception of Non-GAAP Income/Loss. 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.

    Key Program Highlights

    • Valoctocogene roxaparvovec gene therapy for severe hemophilia A: On January 10, 2021, the Company announced positive top-line, one-year data results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. Data from the study in the pre-specified primary analysis for Annualized Bleeding Rate (ABR) showed that a single dose of valoctocogene roxaparvovec significantly reduced ABR by 84% compared with prior treatment with prophylactic FVIII infusions. These results were from a pre-specified group of participants in a non-interventional prospective baseline observational study (rollover population; N=112) with a median follow-up of 60.1 weeks after dosing with valoctocogene roxaparvovec. 80% of the rollover participants were bleed-free starting at week five after treatment.

    Additionally, 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 mean endogenous Factor VIII expression level of 42.9 (SD 45.5, median 23.9) IU/dL, as measured by the chromogenic substrate (CS) assay, supporting the marked clinical benefits observed with abrogation of bleeding episodes and Factor VIII infusion treatment rate. Factor VIII expression declined at a slower rate compared to the Phase 1/2 study, and remained in a range to provide hemostatic efficacy. 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 (SD 50.9, median 23.9) IU/dL at the end of year one to a mean of 24.4 (SD 29.2, median 14.7) IU/dL at the end of year two with continued hemostatic efficacy demonstrated by a mean ABR of 0.9 (median 0.0) bleeding episodes per year.

    Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII usage in the rollover population by 99% from 135.9 (median 128.6) to 2.0 (median 0.0) infusions per year (p-value <0.0001).

    In the U.S., the FDA recommended that the Company complete the Phase 3 study and submit two-year follow-up safety and efficacy data on all study participants. The Company plans to meet with FDA to review the two-year data request and share the Phase 3 GENEr8-1 results announced on January 10, 2021. BioMarin is targeting submission of the Marketing Authorization Application (MAA) with these results to the EMA in the second quarter of 2021 pending confirmation in presubmission meetings.

    • Vosoritide for children with achondroplasia: On December 21, 2020 the Company announced that children in the open-label long-term extension of the Phase 3 study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP), maintained an increase in Annual Growth Velocity (AGV) through the second year of continuous treatment. An analysis, comparing all children randomized and treated with vosoritide for two years (n=52) to all children from the run-in study who were randomized to receive placebo with an untreated observation period of two years (n=38), showed improvement in one-year height change in the treated group relative to the untreated group that was similar in the second year of treatment, 1.79 cm, as in the first year of treatment, 1.73 cm. The cumulative height gain over the 2-year treatment period was 3.52 cm more than the untreated children.

    In 2020, marketing applications for vosoritide were validated and accepted by EMA and FDA, respectively. The CHMP opinion is expected in Europe in June of 2021. The U.S. New Drug Application (NDA) for vosoritide is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of August 20, 2021.

    In January 2021, the Company received notice from FDA that the NDA for vosoritide had been granted Priority Review Designation. Under this designation, the vosoritide NDA may qualify for a Priority Review Voucher (PRV) upon approval.  A PRV confers priority review to a subsequent drug application that would not otherwise qualify for that designation. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.

    • Palynziq for PKU: On October 7, 2020 the Company announced that the FDA approved the supplemental Biologics License Application (sBLA) to increase the maximum allowable dose of Palynziq (pegvaliase-pqpz) Injection for treatment of adults with PKU to 60 mg daily. Previously, the maximum dose was 40 mg daily. In the Phase 3 PRISM studies, 19% of study participants required a 60 mg dose to achieve adequate response to Palynziq.

    Palynziq is indicated to reduce blood Phe concentrations in adults with PKU, who have uncontrolled blood Phe concentrations greater than 600 μmol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first and only approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe.

    • BMN 307 gene therapy product candidate for PKU: The Company announced that it plans to dose escalate in PHEarless, the Phase 1/2 study of BMN 307 based on encouraging Phe lowering and safety signals observed in study participants who were treated with the lowest dose. Both the FDA and EMA granted BMN 307 Orphan Drug Status. Additionally, the FDA has granted Fast Track status to BMN 307. Product for use in the Phase 1/2 study was made at commercial scale from BioMarin's award-winning gene therapy manufacturing facility.
    • BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): IND-enabling studies are ongoing with BMN 331, BioMarin's third gene therapy candidate, for the treatment of HAE. 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.
    • 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.
    • BMN 255 for a subset of chronic renal disease: On January 11, 2021 the Company announced that it filed an IND in 2020 for BMN 255, a small molecule for a subset of chronic renal disease. BMN 255 was driven by genetic discoveries for both mechanism and for identifying individuals for treatment.
    • BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies are underway with BMN 351, an oligonucleotide therapy that has demonstrated a high-level of protein expression in experimental animals possessing skippable dystrophic mutations and at doses that are promising in regard to safety. The Company intends to determine timing of a potential IND filing at the end of the year based on results of ongoing IND-enabling studies.

    BioMarin will host a conference call and webcast to discuss fourth quarter and full-year 2020 financial results today, Thursday, February 25, 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: 6488682

    Conference ID: 6488682

    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; 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 plan to submit complete one-year Phase 3 data for valoctocogene roxaparvovec to the EMA in the second quarter of 2021, (ii) BioMarin's plan to resubmit its MAA for valoctocogene roxaparvovec to the EMA in the second quarter of 2021 (iii) BioMarin's plans to meet with FDA to review the two-year data request and share the Phase 3 GENEr8-1 results announced on January 10, 2021, (iv) that the CHMP opinion for vosoritide is expected in Europe in the second half of 2021; and (v) the target PDUFA action date with respect to vosoritide of August 20, 2021; the potential approval and commercialization of BioMarin's product candidates, including vosoritide for the treatment of achondroplasia and valoctocogene roxaparvovec for the treatment of severe hemophilia A, including timing of such approval decisions; and the expected benefits and availability of BioMarin's product candidates, including (i) that valoctocogene roxaparvovec gene therapy may potentially address the high treatment burden for people with severe hemophilia A and (ii) BioMarin's hope that, if approved, vosoritide will become available later in 2021.

    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 September 30, 2020 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. Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

    BIOMARIN PHARMACEUTICAL INC.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    December 31, 2020 and December 31, 2019

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





    December 31,

    2020



    December 31,

    2019(1)

    ASSETS

    (unaudited)





    Current assets:







    Cash and cash equivalents

    $

    649,158





    $

    437,446



    Short-term investments

    416,228





    316,361



    Accounts receivable, net

    448,351





    377,404



    Inventory

    698,548





    680,275



    Other current assets

    129,934





    130,657



    Total current assets

    2,342,219





    1,942,143



    Noncurrent assets:







    Long-term investments

    285,473





    411,978



    Property, plant and equipment, net

    1,032,471





    1,010,868



    Intangible assets, net

    417,271





    456,580



    Goodwill

    196,199





    197,039



    Deferred tax assets

    1,432,150





    549,422



    Other assets

    142,237





    122,009



    Total assets

    $

    5,848,020





    $

    4,690,039



    LIABILITIES AND STOCKHOLDERS' EQUITY







    Current liabilities:







    Accounts payable and accrued liabilities

    $

    492,548





    $

    570,621



    Short-term convertible debt, net





    361,882



    Total current liabilities

    492,548





    932,503



    Noncurrent liabilities:







    Long-term convertible debt, net

    1,075,145





    486,238



    Long-term contingent consideration

    60,130





    50,793



    Other long-term liabilities

    114,195





    98,124



    Total liabilities

    $

    1,742,018





    $

    1,567,658



    Stockholders' equity:







    Common stock, $0.001 par value: 500,000,000 shares authorized; 181,740,999 and 179,838,114 shares issued and outstanding, respectively.

    182





    180



    Additional paid-in capital

    4,993,407





    4,832,707



    Company common stock held by Nonqualified Deferred Compensation Plan

    (9,839)





    (9,961)



    Accumulated other comprehensive income

    (16,139)





    20,164



    Accumulated deficit

    (861,609)





    (1,720,709)



    Total stockholders' equity

    4,106,002





    3,122,381



    Total liabilities and stockholders' equity

    $

    5,848,020





    $

    4,690,039















    (1)

    December 31, 2019 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, 2019, filed with the U.S. Securities and Exchange Commission (SEC) on February 27, 2020.



     

    BIOMARIN PHARMACEUTICAL INC.

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

    Three and Twelve Months Ended December 31, 2020 and 2019

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





    Three Months Ended

    December 31,



    Twelve Months Ended

    December 31,



    2020



    2019



    2020



    2019 (1)



    (unaudited)



    (unaudited)



    (unaudited)





    REVENUES:















    Net product revenues

    $

    437,045





    $

    436,585





    $

    1,805,861





    $

    1,661,043



    Royalty and other revenues

    15,072





    17,858





    54,594





    43,005



    Total net revenues

    452,117





    454,443





    1,860,455





    1,704,048



    OPERATING EXPENSES:















    Cost of sales

    126,138





    95,899





    524,272





    359,466



    Research and development

    156,667





    172,812





    628,116





    715,007



    Selling, general and administrative

    195,512





    187,900





    737,669





    680,924



    Intangible asset amortization and contingent consideration

    18,640





    16,994





    66,658





    74,108



    Gain on sale of nonfinancial assets





    (10,000)





    (59,495)





    (25,000)



    Total operating expenses

    496,957





    463,605





    1,897,220





    1,804,505



    LOSS FROM OPERATIONS

    (44,840)





    (9,162)





    (36,765)





    (100,457)



















    Equity in the income (loss) of BioMarin/Genzyme LLC

    1,071





    193





    (6)





    (587)



    Interest income

    3,071





    5,211





    16,610





    22,748



    Interest expense

    (4,749)





    (6,930)





    (29,309)





    (23,460)



    Other income, net

    5,262





    907





    7,148





    6,945



    LOSS BEFORE INCOME TAXES

    (40,185)





    (9,781)





    (42,322)





    (94,811)



    Benefit from income taxes

    (62,284)





    (24,805)





    (901,422)





    (70,963)



    NET INCOME (LOSS)

    22,099





    15,024





    859,100





    (23,848)



    NET INCOME (LOSS) PER SHARE, BASIC

    $

    0.12





    $

    0.08





    $

    4.75





    $

    (0.13)



    NET INCOME (LOSS) PER SHARE, DILUTED

    $

    0.12





    $

    0.08





    $

    4.53





    $

    (0.13)



    Weighted average common shares outstanding, basic

    181,435





    179,531





    180,804





    179,039



    Weighted average common shares outstanding, diluted

    184,476





    182,412





    191,678





    179,039







    (1)

    December 31, 2019 totals 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, 2019, filed with the SEC on February 27, 2020.



    The following table presents Net Product Revenues by Product:

    Net Product Revenues by Product

    (In millions of U.S. dollars)





    Three Months Ended

    December 31,



    Twelve Months Ended

    December 31,



    2020



    2019



    % Change



    2020



    2019 (1)



    % Change



    (unaudited)



    (unaudited)







    (unaudited)









    PKU franchise

    $

    138.6





    $

    154.3





    (10)

    %



    $

    628.7





    $

    550.3





    14

    %

    Vimizim

    142.5





    132.3





    8

    %



    544.4





    544.3





    %

    Naglazyme

    119.7





    94.8





    26

    %



    391.3





    374.3





    5

    %

    Brineura

    35.0





    25.2





    39

    %



    110.2





    72.0





    53

    %

    Firdapse (2)





    6.1





    (100)

    %



    1.2





    22.3





    (95)

    %

    Net Product Revenues Marketed

           by BioMarin

    435.8





    412.7









    1,675.8





    1,563.2







    Aldurazyme Net Product Revenues

          Marketed by Genzyme

    1.2





    23.9





    (95)

    %



    130.1





    97.8





    33

    %

    Total Net Product Revenues

    $

    437.0





    $

    436.6









    $

    1,805.9





    $

    1,661.0











    (1)

    December 31, 2019 totals 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, 2019, filed with the SEC on February 27, 2020.

    (2)

    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 twelve months ended months ending December 31, 2020; and as a result of the transaction BioMarin will not recognize Net Product Revenues from Firdapse in the future.

    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)

    (unaudited)





    Three Months Ended

    December 31,



    Twelve Months Ended

    December 31,



    2020



    2019



    % Change



    2020



    2019 (1)



    % Change



    (unaudited)



    (unaudited)







    (unaudited)









    Kuvan

    $

    89.0





    122.6





    (27)

    %



    $

    457.7





    463.4





    (1)

    %

    Palynziq

    49.6





    31.7





    56

    %



    171.0





    86.9





    97

    %

    Total PKU franchise

    $

    138.6





    $

    154.3





    (10)

    %



    $

    628.7





    $

    550.3





    14

    %

















































    (1)

    December 31, 2019 totals 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, 2019, filed with the SEC on February 27, 2020.



    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 (benefit from) 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

    December 31,



    Twelve Months Ended December 31,



    Guidance

    Year Ending



    2020



    2019



    2020



    2019



    December 31, 2021

























    GAAP Net Income (Loss)

    $

    22.1





    $

    15.0





    $

    859.1





    $

    (23.8)





    $

    (130.0)



    $

    (80.0)



























    Interest expense, net

    1.7





    1.7





    12.7





    0.7





    11.0



    11.0



    Benefit from income taxes

    (62.3)





    (24.8)





    (901.4)





    (71.0)





    (16.0)



    (16.0)



    Depreciation expense

    11.9





    9.5





    43.0





    51.8





    48.0



    48.0



    Amortization expense

    15.4





    16.3





    62.2





    53.5





    62.0



    62.0



    Stock-based compensation expense

    47.5





    38.0





    189.7





    159.8





    186.0



    186.0



    Contingent consideration expense

    3.2





    0.7





    4.5





    20.6





    9.0



    9.0



    Provision for inventory reserve, net (1)









    75.6













    Gain on sale of nonfinancial assets





    (10.0)





    (59.5)





    (25.0)









    Licensed In-Process R&D (2)









    26.3













    Non-GAAP Income

    $

    39.5





    $

    46.4





    $

    312.2





    $

    166.6





    $

    170.0



    $

    220.0







    (1)

    Represents a $81.2 million charge related to pre-launch valoctocogene roxaparvovec inventory, net of stock-based compensation, as a result of the unexpected delays in anticipated regulatory approvals.

    (2)

    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 December 31,



    2020



    2019







    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

    $

    126.1





    $





    $

    (5.9)





    $

    120.2





    $

    95.9





    $





    $

    (3.5)





    $

    92.4



    Research and development

    156.7





    (6.7)





    (16.5)





    133.5





    172.8





    (3.8)





    (13.5)





    155.5



    Selling, general and administrative

    195.5





    (5.2)





    (25.1)





    165.2





    187.9





    (5.7)





    (21.0)





    161.2



    Intangible asset amortization and contingent consideration

    18.6





    (15.4)





    (3.2)









    17.0





    (16.3)





    (0.7)







    Gain on sale of nonfinancial assets

















    (10.0)









    10.0







    Interest expense, net

    (1.7)





    1.7













    (1.7)





    1.7











    Benefit from income taxes

    (62.3)





    62.3













    (24.8)





    24.8











    GAAP Net Income /Non-GAAP Income

    $

    22.1





    $

    (33.3)





    $

    50.7





    $

    39.5





    $

    15.0





    $

    2.7





    $

    28.7





    $

    46.4



































     



    Twelve months ended December 31,



    2020



    2019







    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

    $

    524.3





    $





    $

    (101.9)





    $

    422.4





    $

    359.5





    $





    (16.1)





    $

    343.4



    Research and development

    628.1





    (21.9)





    (88.2)





    518.0





    715.0





    (26.9)





    (56.6)





    631.5



    Selling, general and administrative

    737.7





    (21.1)





    (101.5)





    615.1





    680.9





    (24.9)





    (87.1)





    568.9



    Intangible asset amortization and contingent consideration

    66.7





    (62.2)





    (4.5)









    74.1





    (53.5)





    (20.6)







    Gain on sale of nonfinancial assets

    (59.5)









    59.5









    (25.0)









    25.0







    Interest expense, net

    (12.7)





    12.7













    (0.7)





    0.7











    Benefit from income taxes

    (901.4)





    901.4













    (71.0)





    71.0











    GAAP Net Income (Loss)/Non-GAAP Income

    859.1





    (783.5)





    236.6





    312.2





    (23.8)





    35.0





    155.4





    166.6



     

    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:http://www.prnewswire.com/news-releases/biomarin-announces-fourth-quarter-and-record-full-year-2020-financial-results-and-corporate-updates-301236040.html

    SOURCE BioMarin Pharmaceutical Inc.

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  3. SAN RAFAEL, Calif., Feb. 24, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that management will participate in four upcoming virtual conferences.  An audio webcast of the presentations will be available live. You can access the webcast at: https://investors.biomarin.com/. An archived version of the remarks will also be available through the Company's website for a limited time following the conference.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. 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 

    (415) 455-7451

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-to-participate-in-four-upcoming-virtual-investor-conferences-301234450.html

    SOURCE BioMarin Pharmaceutical Inc.

    View Full Article Hide Full Article
  4. SAN RAFAEL, Calif., Feb. 18, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), a global leader in providing therapies for rare genetic diseases, today announced the appointment of former Goldman Sachs partner and executive in the life sciences industry, Maykin Ho, Ph.D., to its Board of Directors.   

    "We are thrilled to welcome Maykin to BioMarin's Board of Directors.  She brings a breadth and depth of experience in the healthcare industry and finance to an already exceptional board," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer at BioMarin.  "Maykin brings an important perspective to support BioMarin's growth trajectory as we strive to address the unmet medical needs of people affected by rare genetic diseases and deliver scientific breakthroughs." 

    "It is an honor to join the Board of Directors of BioMarin, a leader in the development, manufacturing and commercialization of first-in-class or best-in-class therapies for rare genetic diseases," said Dr. Ho.  "I look forward to working with the Board and the management team to continue advancing the standard of care for patients with these diseases."

    About Dr. Ho

    Dr. Maykin Ho has more than 30 years of experience in the healthcare and finance industries. She serves on the boards of Agios Pharmaceuticals Inc., FibroGen Inc., Grail Inc., Parexel, the Aaron Diamond AIDS Research Center, and the Institute for Protein Innovation. Dr. Ho is also a venture partner of Qiming Venture Partners and a member of the Biotech Advisory Panel of the Stock Exchange of Hong Kong. She is a retired partner of the Goldman Sachs Group where she served as senior biotechnology analyst, co-head of global healthcare investment research, and advisory director for healthcare investment banking. Prior to Goldman Sachs, Dr. Ho held various managerial positions in licensing, strategic planning, marketing and research at DuPont-Merck Pharmaceuticals and DuPont de Nemours & Company. She was a postdoctoral fellow at Harvard Medical School and a graduate of the Advanced Management Program at The Fuqua School of Business, Duke University. Dr. Ho received a Ph.D. in Microbiology and Immunology and a B.S. from the State University of New York, Downstate Medical Center.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. 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.

    BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.

    Contacts:







    Investors

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    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558

    (415) 455-7451

     

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    SOURCE BioMarin Pharmaceutical Inc.

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  5. SAN RAFAEL, Calif., Jan. 28, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced the Company received the top score of 100 on the Human Rights Campaign Foundation's 2021 Corporate Equality Index (CEI), the nation's foremost benchmarking survey and report measuring corporate policies and practices related to LGBTQ workplace equality.  BioMarin joins the ranks of 767 major U.S. businesses that also earned top marks this year.

    "At BioMarin, we believe that fostering diversity, equity and inclusion in our workforce helps our mission of delivering breakthrough therapies for rare disease patients," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin.  "We are very proud to receive this important recognition from the Human Rights Campaign Foundation, highlighting the long-standing commitment of the BioMarin community to advancing LGBTQ equality within and beyond our Company."

    "From the previously unimaginable impact of the COVID-19 pandemic, to a long overdue reckoning with racial injustice, 2020 was an unprecedented year.  Yet, many businesses across the nation stepped up and continued to prioritize and champion LGBTQ equality," said Alphonso David, Human Rights Campaign President.  "This year has shown us that tools like the CEI are crucial in the work to increase equity and inclusion in the workplace, but also that companies must breathe life into these policies and practices in real and tangible ways.  Thank you to the companies that understand protecting their LGBTQ employees and consumers from discrimination is not just the right thing to do—but the best business decision."

    The results of the 2021 CEI showcase how 1,142 U.S.-based companies are promoting LGBTQ-friendly workplace policies in the U.S. BioMarin is among the 57 percent of these companies with global operations, helping to advance the cause of LGBTQ inclusion in its workplaces abroad as well.  BioMarin's efforts in satisfying all of the CEI's criteria earned a 100 percent ranking and the designation as one of the Best Places to Work for LGBTQ Equality.

    The CEI rates employers providing these crucial protections to over 18 million U.S. workers and an additional 17 million abroad.  Companies rated in the CEI include Fortune magazine's 500 largest publicly traded businesses, and hundreds of publicly and privately held mid- to large-sized businesses. BioMarin is one of 27 biopharmaceutical companies listed in the 2021 CEI.

    The CEI rates companies on detailed criteria falling under four central pillars:

    • Non-discrimination policies across business entities;
    • Equitable benefits for LGBTQ workers and their families;
    • Supporting an inclusive culture; and,
    • Corporate social responsibility.

    The full report is available online at www.hrc.org/cei.

    About the Human Rights Campaign Foundation

    The Human Rights Campaign Foundation is the educational arm of America's largest civil rights organization working to achieve equality for lesbian, gay, bisexual transgender and queer people. HRC envisions a world where LGBTQ people are embraced as full members of society at home, at work and in every community.

    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.

    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:http://www.prnewswire.com/news-releases/biomarin-earns-top-marks-in-human-rights-campaigns-2021-corporate-equality-index-301217137.html

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  6. SAN RAFAEL, Calif., Jan. 26, 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 Thursday, February 25, at 4:30 p.m. ET to discuss fourth quarter and full year 2020 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: 6488682

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

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

    Conference ID: 6488682

    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

    (415) 455-7451

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-to-host-fourth-quarter-and-full-year-2020-financial-results-conference-call-and-webcast-on-thursday-february-25-at-430pm-et-301214639.html

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  7. SAN RAFAEL, Calif., Jan. 10, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A.  This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.  All participants in the study received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up. 

    Data from the GENEr8-1 Phase 3 study with a mean follow-up of 71.6 weeks showed that in the pre-specified primary analysis for Annualized Bleeding Rate (ABR) a single dose of valoctocogene roxaparvovec significantly reduced 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 <0.0001), among a pre-specified group of prior participants in a non-interventional baseline observational study (rollover population; N=112). 80% of participants were bleed-free starting at week five after treatment.  

    Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII in the rollover population by 99% from 135.9 (median 128.6) to 2.0 (median 0.0) infusions per year (p-value <0.0001).  

    Table 1:  Mean/Median Annualized Bleeding Rate (ABR) and FVIII Infusion Rate in Phase 3 GENEr8-1 Study Rollover Population (N=112) from Week 5 Through Week 52 at Nov. 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  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 descriptions for patient population information.

    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 mean endogenous Factor VIII expression level of 42.9 (SD 45.5, median 23.9) IU/dL, as measured by the chromogenic substrate (CS) assay, supporting the marked clinical benefits observed with abrogation of bleeding episodes and Factor VIII infusion rate. Factor VIII expression declined at a slower rate compared to the Phase 1/2 study, and remained in a range to provide hemostatic efficacy. 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 (SD 50.9, median 23.9) IU/dL at the end of year one to a mean of 24.4 (SD 29.2, median 14.7) IU/dL at the end of year two with continued hemostatic efficacy demonstrated by a mean ABR of 0.9 (median 0.0) bleeding episodes per year. 

    Table 2:  Factor VIII Activity Levels in 6-Month Intervals

    Median Factor

    VIII Activity,

    IU/dL

    Phase 3 Rollover

    Population

    (N=112)

     

    Mean (SD)

    Median

    Phase 3 mITT

    Subset

    Population

    (N=17*)

     

    Mean (SD)

    Median

    Phase 1/2

    6e13 vg/kg

    Cohort

     

    (N=7)

     

    Mean (SD)

    Median

    Phase 1/2

    4e13 vg/kg

    Cohort

     

    (N=6)

     

    Mean (SD)

    Median

    Week 26

    55.1 (57.4)

    38.6

    43.9 (42.1)

    33.8

    71.0 (41.6)

    61.2

    18.0 (8.7)

    18.0

    Week 52

    43.6 (45.3)

    24.2

    42.2 (50.9)

    23.9

    63.6 (36.5)

    60.3

    21.1 (12.3)

    23.8

    Week 76



    27.9 (30.6)

    15.8

    53.9 (31.2)

    50.2

    20.6 (15.4)

    21.3

    Week 104



    24.4 (29.2)

    14.7

    36.4 (26.3)

    26.2

    12.3 (8.2)

    11.6

    *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.

    Please see Figure 1: Box-and-Whiskers Plot.

    This is the first statistical evidence demonstrating ABR superiority in a gene therapy trial.  These data give us confidence in this groundbreaking alternative to existing therapies and bring us one step closer to a potential new treatment choice to fulfill an unmet medical need for people with hemophilia A," said Steven W. Pipe, MD, Professor of Pediatrics and Pathology, Coagulation Director, Special Coagulation Laboratory Laurence A. Boxer, MD Research Professor of Pediatrics and Communicable Diseases Department of Pathology Michigan Medicine at the University of Michigan and investigator in the Phase 3 study.  "This data set adds to the growing body of scientific and clinical data around valoctocogene roxaparvovec gene therapy for hemophilia A and creates the possibility for a new treatment paradigm."

    "Over the past seven years, we have conducted rigorous scientific research and clinical programs to address the unmet medical needs of people with severe hemophilia A," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "The decades-long aspirations of the hemophilia community are at the forefront of our ongoing commitment to advance this promising investigational gene therapy for the treatment of severe hemophilia A. We are very encouraged by these data and look forward to working with regulatory authorities, treating physicians, and people with hemophilia A to further understand the potential of this gene therapy."

    "Although factor replacement therapy has been shown to be a safe and effective treatment modality in hemophilia, it suffers both from incomplete prevention of joint disease and having a high treatment burden with recurring needs for intravenous infusions, which can limit important daily activities out of fear of bleeds," said Guy Young, M.D., Director, Hemostasis and Thrombosis Program at Children's Hospital Los Angeles and Professor of Pediatrics (Clinical Scholar), Keck School of Medicine of University of Southern California.  "Novel therapeutic approaches such as gene therapy offer the prospect for both complete prevention of bleeds and subsequent joint damage and eliminating the burden associated with current treatments resulting in an improved quality of life."

    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 developed inhibitors to Factor VIII, or thromboembolic events.  One participant was lost to follow-up.  Infusion-related reactions were effectively mitigated by managing infusion rates.

    Alanine aminotransferase (ALT) elevation (115 participants, 86%), a laboratory test of liver function, remained the most common adverse event (AE).  Other common adverse events were headache (51 participants, 38%), nausea (50 participants, 37%), aspartate aminotransferase (AST) elevation (47 participants, 35%), arthralgia (38 participants, 28%) and fatigue (37 participants, 27%).  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. These side effects have generally been grade 1/2 in intensity, manageable and reversible. Isolated grade 3 steroid-related sides effects (e.g., diabetes, hypertension, weight gain, bone fractures) were observed with longer-term higher dose corticosteroid administration. Corticosteroid-related grade 3 SAEs emerged as a safety issue with extended use of corticosteroids which were reversible with only one event of weight gain ongoing. 

    Overall, in the Phase 1/2 study, the safety profile of valoctocogene roxaparvovec remains consistent with previously reported data with no delayed-onset, treatment-related events.  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 and included transient 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. 

    GENEr8-1 Study Description

    The global Phase 3 GENEr8-1 study evaluates superiority of valoctocogene roxaparvovec at the 6e13 vg/kg dose compared to the current standard of care, 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 datacut.  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 datacut 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

    BioMarin is working with the U.S. Food and Drug Administration (FDA) to align on steps forward to obtain marketing approval for valoctocogene roxaparvovec gene therapy for severe hemophilia A. The FDA recommended that the Company complete the Phase 3 study and submit two-year follow-up safety and efficacy data on all study participants. Additionally, the European Medicines Agency (EMA) requested one-year results from the full Phase 3 study to inform their benefit-risk assessment. To facilitate this submission within the EMA regulatory framework, BioMarin withdrew the MAA and plans to resubmit the MAA with these data to the EMA in the second quarter of 2021 following discussions with the Agency.

    The FDA has granted valoctocogene roxaparvovec Breakthrough Therapy Designation.  BioMarin's valoctocogene roxaparvovec 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. 

    Call and Webinar to be Held Today, January 10, 2021 at 7:15 PM Eastern Time

    Join from a PC, Mac, iPad, iPhone or Android device:

    Please click here to join a live Zoom video webinar at 7:15 PM Eastern.

    Or join by phone:

    Dial (for higher quality, dial a number based on your current location):

    US: +1 669 900 6833  or +1 346 248 7799  or +1 253 215 8782  or +1 312 626 6799  or +1 929 205 6099  or +1 301 715 8592

    Zoom Webinar ID: 959 1943 2167

    International numbers available here.  

    Phase 1/2 Dose Escalation Study Description

    The Phase 1/2 dose escalation study is ongoing and continues to monitor participants long-term.  In the study, a total of 15 patients with severe hemophilia A and Factor VIII activity levels less than or equal to 1 IU/dL received a single dose of BMN 270, seven of whom were treated at a dose of 6e13 vg/kg and six of whom were treated at a lower dose of 4e13 vg/kg.  The other two participants were treated at lower doses as part of dose escalation in the study and did not achieve therapeutic efficacy.  

    Robust Clinical Program

    BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A.  In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company recently began 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 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 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 individuals 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., including without limitation, statements about the development of BioMarin's valoctocogene roxaparvovec program generally, and the Phase 3 results particularly; the impact of valoctocogene roxaparvovec gene therapy for treating patients with severe hemophilia A, the potential for valoctocogene roxaparvovec to reduce or eliminate bleeds, reduce the number of Factor VIII infusions, and the ongoing clinical programs generally.  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 decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture the product candidate for the preclinical and clinical trials;  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 Annual and quarterly Reports on Forms 10-K and 10-Q, 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:







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    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558

    (415) 455-7451

     

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  8. SAN RAFAEL, Calif., Jan. 5, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that management will participate in two upcoming virtual conferences.  An audio webcast of the presentations will be available live. You can access the webcast at: https://investors.biomarin.com/. An archived version of the remarks will also be available through the Company's website for a limited time following the conference.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. 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

    (415) 455-7451

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-to-participate-in-two-upcoming-virtual-investor-conferences-301200463.html

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  9. SAN RAFAEL, Calif., Dec. 21, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that children in the open-label long-term extension of the Phase 3 study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP), maintained an increase in Annual Growth Velocity (AGV) through the second year of continuous treatment.  These analyses are the result of the combination of data of the same patients enrolled in three consecutive trials.  In the first trial, a "run in" period consisted of longitudinal measurement of height in all patients prior to receiving treatment.  After at least six months observation in the run-in trial, 121 patients were randomized 1:1 to receive either placebo…

    SAN RAFAEL, Calif., Dec. 21, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that children in the open-label long-term extension of the Phase 3 study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP), maintained an increase in Annual Growth Velocity (AGV) through the second year of continuous treatment.  These analyses are the result of the combination of data of the same patients enrolled in three consecutive trials.  In the first trial, a "run in" period consisted of longitudinal measurement of height in all patients prior to receiving treatment.  After at least six months observation in the run-in trial, 121 patients were randomized 1:1 to receive either placebo or vosoritide at a dose of 15 ug/kg/day.  One year later, patients previously receiving placebo were crossed over to receive vosoritide in an open-label treatment extension study, while those patients previously on vosoritide remained on treatment. 

    A first analysis, comparing all children randomized and treated with vosoritide for two years (n=52) to all children from the run-in study who were randomized to receive placebo with an untreated observation period of two years (n=38), showed improvement in one-year height change in the treated group relative to the untreated group that was similar in the second year of treatment, 1.79 cm, as in the first year of treatment, 1.73 cm. The cumulative height gain over the 2-year treatment period was 3.52 cm compared to untreated children, which is the sum of the first year (1.73) and the second year (1.79). 

    Yearly change in standing height (cm)

    Year 1

    Year 2

    Untreated (N=38*), mean (SD)

    3.96 (0.92)

    3.82 (0.99)

    Vosoritide (N=52**), mean (SD)

    5.69 (0.97)

    5.61 (1.09)

    Treatment effect (95% CI)

    1.73 (1.33 - 2.14)

    1.79 (1.35 - 2.24)

    P-value***

    <0.0001

    <0.0001



    *38 participants were enrolled in the run-in study more than 12 months and were at least 5 years of age at that point of time in advance of randomization and therefore contribute at least two years of evaluation of height in the absence of treatment.

     

     **Data from 6 patients were unavailable due to patient withdrawals during Year 2 (n=2) and due to restrictions in study conduct because of Covid-19 (n=4).  The patients whose data are not available due to Covid-19 are still on treatment.

     

    *** p-value for unadjusted treatment effect.

    A second supportive analysis evaluated the treatment effect of vosoritide administered continuously for over two years, including all children regardless of the duration of prior observation (N=119; 58 treated and 61 untreated children). This analysis showed a mean improvement in AGV  in the vosoritide treated group of 1.69 cm/year, compared with untreated subjects, calculated over the entire observation period.  A similar method was used in the analysis of the effect of one year's treatment with vosoritide previously published in The Lancet on Sept. 5, 2020, demonstrating a placebo-adjusted improvement in AGV of 1.57 cm/year.  (Table 2 in Lancet publication, DOI:  https://doi.org/10.1016/S0140-6736(20)31541-5 ). 

    In the vosoritide treated group, AGV declined by -0.14 cm/year during the second year of vosoritide treatment compared to the baseline AGV established in the 6 months prior to the first year of treatment. This decline is similar to the annual AGV decline with age that has been observed in natural history studies, as well as during one year of treatment with placebo (-0.12 cm/year), further supporting the maintenance of treatment effect. 

    Retention of subjects on treatment was high with 93% of patients originally randomized to receive vosoritide remaining on treatment two years later.

    Vosoritide was generally well tolerated with no new safety concerns.  Serious adverse events observed in the trial were representative of common childhood illnesses and were deemed unrelated to vosoritide.  No new safety findings have emerged, and clinically inconsequential blood pressure changes were mild, transient and self-limiting.

    "BioMarin is committed to the long-term follow up of children participating in vosoritide studies and the overall health of people with achondroplasia.  We look forward to sharing more data on wider health measures that either require a longer treatment period or starting treatment at a younger age.  We are also specifically studying the impact of vosoritide in infants at risk of serious and potentially fatal medical complications related to achondroplasia," said Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin.  "We are grateful for the support of the children and their families who are in these studies, the clinical trial investigators and their staffs, BioMarin employees, and the community.  We look forward to sharing more detailed information at an upcoming medical meeting and further contributing to the scientific body of knowledge about vosoritide and its potential impact over time."

    "Follow up data from extension studies are critical to expanding our understanding of the wider impact of achondroplasia," said Melita Irving, Clinical Geneticist at Guy's and St Thomas' NHS Foundation Trust, London, UK and investigator for the vosoritide clinical program at the Evelina London Children's Hospital.  "BioMarin has developed a comprehensive clinical program designed to address the effects on health and day to day living by evaluating proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension."

    Vosoritide Safety

    The 2-year data demonstrated that vosoritide, administered at 15ug/kg/day was generally well tolerated with no new safety findings.  The majority of adverse events (AEs) were mild and no serious adverse events were reported as study drug-related.  Injection site reactions were the most common drug-related AEs, and all were transient.   No clinically significant blood pressure decreases or new safety findings were observed. 

    Regulatory Status

    BioMarin's marketing applications for vosoritide are currently under review by both the Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and if approved would be the first therapy for achondroplasia in the U.S. and Europe.  The FDA's Prescription Drug User Fee Act target action date is August 20, 2021.  The Committee for Medicinal Products for Human Use (CHMP) opinion is expected in Europe in the second half of 2021.

    Vosoritide has also received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia. 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

    Description of Phase 3 Extension Study

    This is an ongoing open-label long-term extension study to a completed pivotal, double-blind, placebo-controlled study of vosoritide in children with achondroplasia. A total of 119 children were enrolled in the extension study after completion of the pivotal phase 3 study and are receiving open-label treatment with vosoritide 15 mcg/kg daily. Vosoritide is being tested in children whose growth plates are still "open."  This is approximately 25% of people with achondroplasia.  The extension study is evaluating safety, AGV, and cumulative annual height gain until participants reach final adult height.  A wide range of secondary and exploratory endpoints include anthropometric measures such as height Z-score, body and limb proportionality and joint geometry; biochemical, biomarker and radiological assessments of bone growth and health; and evaluations of health-related quality of life (HRQoL), developmental status, and functional independence. These additional endpoints address the overall impact vosoritide has on achondroplasia.

    Description of Phase 2 Study for Children at Risk of Life-Threatening Foramen Magnum Compression

    This is a Phase 2 randomized, controlled, open-label clinical trial with an open-label extension to investigate the safety of vosoritide in infants with achondroplasia at risk of requiring cervicomedullary decompression surgery to alleviate compression at the foramen magnum, the opening in the base of the skull through which the spinal cord passes. In addition, the study will also measure a secondary endpoint to evaluate the effect of vosoritide on growth of the foramen magnum volume through MRI scans.  Within a month of study initiation in November, two of the planned 20 (10%) participants enrolled. 

    Foramen magnum compression is the foremost life-threatening complication of achondroplasia in infants. This study investigates the safety of vosoritide in infants aged 0 -1 years of age with achondroplasia who have evidence of foramen magnum compression at-risk for requiring cervicomedullary decompression surgery. Those infants are under close observation for the appearance of neurological signs of progressive foramen magnum compression, and the current standard of care is palliative with many eventually requiring surgery. The study aims to enroll approximately 20 infants, who will be randomized to either current standard of care plus vosoritide treatment or current standard of care alone for a two-year period.  After the two-year randomized period, children in the study would be eligible to receive vosoritide in an open-label, 3-year additional extension period. The study will examine the incidence of adverse events between the two groups, volume MRI measurements of the foramen magnum, skull and spine, and progression to requiring decompression surgery.

    Description of Phase 2 Study in Infants and Young Children Ages 0 to 5 Years

    This is a Phase 2 randomized, placebo-controlled study of vosoritide in approximately 70 infants and young children with achondroplasia, aged zero to less than 60 months, for a period of 52 weeks.  The study will be followed by a subsequent open-label extension trial when all subjects receive active treatment. Children in this study will have completed a three-to-six month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The primary objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on height Z-scores, which is the number of standard deviations in relation to the mean height of age- and gender-matched, average stature children.  The company also plans to augment the height Z-score data with assessments including proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries.  Currently, cohorts 1 and 2 are fully enrolled and cohort 3 is 85% (17/20)  enrolled.  The remaining study participants to enroll are in the observational period and are expected to be dosed in 1Q 2021.

    About Achondroplasia

    Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, including foramen magnum compression, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, spinal stenosis and recurrent ear infections. Some of these complications can result in the need for invasive surgeries such as spinal cord decompression and straightening of bowed legs. In addition, studies show increased mortality at every age.

    More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation.  The worldwide incidence rate of achondroplasia is about one in 25,000 live births.  Vosoritide is being tested in children whose growth plates are still "open", typically those under 18 years of age. This is approximately 25% of people with achondroplasia.  In the U.S., Europe, Latin America, the Middle East, and most of Asia Pacific, there are currently no licensed medicines for achondroplasia.

    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 such website is not incorporated by reference into this press release.

    Forward-Looking Statement

    This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin's vosoritide development program generally and specifically about the results of the extension of the Phase 3 trial, the maintenance of AGV after two years, the similarity of AGV and height gain in the first and second years of the Phase 3 study, the similarity of AGV and height gain to earlier studies, the continued clinical development of vosoritide and the timing and conduct of such clinical program; the enrollment expectations for ongoing clinical trials; the possible results of such studies, the timing of decisions by health authorities about marketing applications, and the Company's plans to discuss provision of the two-year data with health authorities. 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: final analysis of the extension of the Phase 3 data, results and timing of current and planned preclinical studies and clinical trials of vosoritide; our ability to enroll participants into such clinical trials, our ability to record data during a global pandemic, our ability to successfully manufacture vosoritide; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning vosoritide; and those other risks and uncertainties detailed from time to time under the caption "Risk Factors" and elsewhere in the BioMarin's Securities and Exchange Commission (SEC) filings, including, without limitation, BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, and future SEC 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:http://www.prnewswire.com/news-releases/biomarin-announces-benefit-maintained-for-over-two-years-in-children-with-achondroplasia-treated-with-vosoritide-in-phase-3-extension-study-301196598.html

    SOURCE BioMarin Pharmaceutical Inc.

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  10. NEW YORK, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Nasdaq (NASDAQ:NDAQ) today announced the results of the annual reconstitution of the Nasdaq-100 Index® (NASDAQ:NDX), which will become effective prior to market open on Monday, December 21, 2020.

    The following six companies will be added to the Index: American Electric Power Company, Inc. (NASDAQ:AEP), Marvell Technology Group Ltd. (NASDAQ:MRVL), Match Group, Inc. (NASDAQ:MTCH), Okta, Inc. (NASDAQ:OKTA), Peloton Interactive, Inc. (NASDAQ:PTON), Atlassian Corporation Plc (NASDAQ:TEAM).

    The Nasdaq-100 Index is composed of the 100 largest non-financial companies listed on The Nasdaq Stock Market® and dates to January 1985 when it was launched along with the Nasdaq Financial-100 Index®, which is comprised…

    NEW YORK, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Nasdaq (NASDAQ:NDAQ) today announced the results of the annual reconstitution of the Nasdaq-100 Index® (NASDAQ:NDX), which will become effective prior to market open on Monday, December 21, 2020.

    The following six companies will be added to the Index: American Electric Power Company, Inc. (NASDAQ:AEP), Marvell Technology Group Ltd. (NASDAQ:MRVL), Match Group, Inc. (NASDAQ:MTCH), Okta, Inc. (NASDAQ:OKTA), Peloton Interactive, Inc. (NASDAQ:PTON), Atlassian Corporation Plc (NASDAQ:TEAM).

    The Nasdaq-100 Index is composed of the 100 largest non-financial companies listed on The Nasdaq Stock Market® and dates to January 1985 when it was launched along with the Nasdaq Financial-100 Index®, which is comprised of the 100 largest financial stocks on Nasdaq®. These indexes act as benchmarks for financial products such as options, futures, and funds. The Nasdaq-100 is reconstituted each year in December, timed to coincide with the quadruple witch expiration Friday of the quarter.

    The Nasdaq-100 Index is the basis of the Invesco QQQ Trust (NASDAQ:QQQ) which aims to provide investment results that, before expenses, correspond with the Nasdaq-100 Index performance. In addition, options, futures and structured products based on the Nasdaq-100 Index and the Invesco QQQ Trust trade on various exchanges.

    As a result of the reconstitution, the following six companies will be removed from the Index: BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), Citrix Systems, Inc. (NASDAQ:CTXS), Expedia Group, Inc. (NASDAQ:EXPE), Liberty Global plc (NASDAQ:LBTYA), Take-Two Interactive Software, Inc. (NASDAQ:TTWO), Ulta Beauty, Inc. (NASDAQ:ULTA).

    Information

    For information about the six companies to be added to the Nasdaq-100 Index, please visit the following respective company websites:

    American Electric Power Company, Inc. – https://www.aep.com/

    Marvell Technology Group Ltd. – https://www.marvell.com/

    Match Group, Inc. – https://www.mtch.com/

    Okta, Inc. – https://www.okta.com/

    Peloton Interactive, Inc. – https://www.onepeloton.com/

    Atlassian Corporation Plc – https://www.atlassian.com/

    About Nasdaq Global Indexes

    Nasdaq Global Indexes has been creating innovative, market-leading, transparent indexes since 1971. Today, our index offering spans geographies and asset classes and includes diverse families such as the Dividend and Income (includes Dividend Achievers), Dorsey Wright, Fixed Income (includes BulletShares®), Global Equity, Green Economy, Nordic and Commodity indexes. We continuously offer new opportunities for financial product sponsors across a wide-spectrum of investable products and for asset managers to measure risk and performance. Nasdaq also provides exchange listing, custom index and design solutions to financial organizations worldwide.

    About Nasdaq

    Nasdaq (NASDAQ:NDAQ) is a global technology company serving the capital markets and other industries. Our diverse offering of data, analytics, software and services enables clients to optimize and execute their business vision with confidence. To learn more about the company, technology solutions and career opportunities, visit us on LinkedIn, on Twitter @Nasdaq, or at www.nasdaq.com.

    Media Relations Contact

    Emily Pan

    (646) 637-3964

    Issuer & Investor Contact

    Index Client Services

    The information contained above is provided for informational and educational purposes only, and nothing contained herein should be construed as investment advice, either on behalf of a particular financial product or an overall investment strategy. Neither Nasdaq, Inc. nor any of its affiliates makes any recommendation to buy or sell any financial product or any representation about the financial condition of any company or fund. Statements regarding Nasdaq's proprietary indexes are not guarantees of future performance. Actual results may differ materially from those expressed or implied. Past performance is not indicative of future results. Investors should undertake their own due diligence and carefully evaluate companies before investing. ADVICE FROM A SECURITIES PROFESSIONAL IS STRONGLY ADVISED.

    -NDAQG-



    Primary Logo

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  11. SAN RAFAEL, Calif., Dec. 1, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that management will participate in two upcoming virtual conferences.  An audio webcast of the presentations will be available live. You can access the webcast at: https://investors.biomarin.com/. An archived version of the remarks will also be available through the Company's website for a limited time following the conference.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. 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                                               

    (415) 455-7451

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-to-participate-in-two-upcoming-virtual-investor-conferences-301182457.html

    SOURCE BioMarin Pharmaceutical Inc.

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  12. SAN RAFAEL, Calif. and TORONTO, Nov. 17, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) and Deep Genomics today announced that the companies have entered into a preclinical collaboration that will use Deep Genomics' artificial intelligence drug discovery platform (The AI Workbench) to identify oligonucleotide drug candidates in four rare disease indications with high unmet need.  Deep Genomics will receive an undisclosed upfront payment and is eligible to receive development milestones as a part of the collaboration.  BioMarin will receive an exclusive option to obtain Deep Genomics' rights to each program for development and commercialization. The companies did not disclose financial terms.

    SAN RAFAEL, Calif. and TORONTO, Nov. 17, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) and Deep Genomics today announced that the companies have entered into a preclinical collaboration that will use Deep Genomics' artificial intelligence drug discovery platform (The AI Workbench) to identify oligonucleotide drug candidates in four rare disease indications with high unmet need.  Deep Genomics will receive an undisclosed upfront payment and is eligible to receive development milestones as a part of the collaboration.  BioMarin will receive an exclusive option to obtain Deep Genomics' rights to each program for development and commercialization. The companies did not disclose financial terms.

    In the collaboration, Deep Genomics will use its AI Workbench to identify and validate target mechanisms and lead candidates, and BioMarin will advance them into preclinical and clinical development. The AI Workbench enables rapid exploration of novel targetable mechanisms and therapeutic candidates.  It combines deep learning, automation, advanced biomedical knowledge and massive amounts of in vitro and in vivo data to accurately identify targetable molecular mechanisms and guide the discovery and development of oligonucleotide therapies.

    "We are thrilled to collaborate with Deep Genomics, a leader in AI-facilitated discovery and development of potential oligonucleotide-based therapeutics, and to tap into their AI Workbench to unlock the potential of exciting new drug targets for rare diseases," said Lon Cardon, Chief Scientific Strategy Officer and Senior Vice President at BioMarin.  "We believe the combination of Deep Genomics' experience in using artificial intelligence to creatively modulate targets coupled with our proven track record in developing transformational medicines for patients with rare diseases will speed BioMarin's trajectory into new biological frontiers."

    "We share BioMarin's pioneering spirit in drug discovery and are delighted to partner with them," said Brendan Frey, Founder and Chief Executive Officer of Deep Genomics. "Our second generation AI Workbench continues to unlock a rapidly growing number of therapeutic opportunities for patients with genetically defined disorders.  BioMarin is an industry leader in developing transformational therapies for patients with rare diseases, and we look forward to working with them to expand their clinical pipeline."

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for 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.

    About Deep Genomics

    Deep Genomics is a therapeutics company founded on computational biology and artificial intelligence. It's AI-based systems, datasets, processes and culture enable the intentional design of effective and highly safe genetic medicines with a speed and a success rate that far exceed what was previously possible. The AI, genome biology, software engineering and preclinical research team is located in the heart of Toronto, Canada, next to the AI research labs of Google, Uber, the Vector Institute and the University of Toronto, where deep learning was invented. The clinical and business development teams are based in Boston, Massachusetts.  For more information, visit www.deepgenomics.com and follow us on Twitter at @deepgenomics.

    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







    Michael Lampe



    Deep Genomics



    (484) 575-5040



     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-and-deep-genomics-to-collaborate-on-advancing-programs-identified-using-artificial-intelligence-301174203.html

    SOURCE BioMarin Pharmaceutical Inc.

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  13. SAN RAFAEL, Calif., Nov. 12, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that management will participate in three upcoming virtual conferences.  An audio webcast of the presentations will be available live. You can access the webcast at: https://investors.biomarin.com/. An archived version of the remarks will also be available through the Company's website for a limited time following the conference.

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. 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

    (415) 455-7451

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/biomarin-to-participate-in-three-upcoming-virtual-investor-conferences-301171792.html

    SOURCE BioMarin Pharmaceutical Inc.

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  14. SAN RAFAEL, Calif., Nov. 9, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the Company is expanding its clinical program for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), with two new Phase 2 studies.  The first study is sponsored by BioMarin to investigate the safety of vosoritide in infants with achondroplasia at risk of life-threatening foramen magnum compression.  The second study is an investigator-initiated study sponsored by Children's National Hospital in Washington, D.C. to investigate vosoritide in children with selected genetic forms of short stature, which together represent addressable patient populations of approximately 275,000.

    Phase 2 Study for Children at Risk

    SAN RAFAEL, Calif., Nov. 9, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the Company is expanding its clinical program for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), with two new Phase 2 studies.  The first study is sponsored by BioMarin to investigate the safety of vosoritide in infants with achondroplasia at risk of life-threatening foramen magnum compression.  The second study is an investigator-initiated study sponsored by Children's National Hospital in Washington, D.C. to investigate vosoritide in children with selected genetic forms of short stature, which together represent addressable patient populations of approximately 275,000.

    Phase 2 Study for Children at Risk of Life-Threatening Foramen Magnum Compression

    BioMarin announced today that the Company has enrolled the first child in a Phase 2 randomized, controlled, open-label clinical trial with an open-label extension to investigate the safety of vosoritide in infants with achondroplasia at risk of requiring cervicomedullary decompression surgery to alleviate compression at the foramen magnum, the opening in the base of the skull through which the spinal cord passes. In addition, the study will also measure a secondary endpoint to evaluate the effect of vosoritide on growth of the foramen magnum volume through MRI scans. 

    Foramen magnum compression is the foremost life-threatening complications of achondroplasia in infants. This study investigates the safety of vosoritide in infants aged 0 -1 years of age with achondroplasia who have evidence of foramen magnum compression at-risk for requiring cervicomedullary decompression surgery. Those infants are under close observation for the appearance of neurological signs of progressive foramen magnum compression, and the current standard of care is palliative with many eventually requiring surgery. The study aims to enroll approximately 20 infants, who will be randomized to either current standard of care plus vosoritide treatment or current standard of care alone for a two-year period.  After the two-year randomized period, children in the study would be eligible to receive vosoritide in an open-label, 3-year additional extension period. The study will examine the incidence of adverse events between the two groups, volume MRI measurements of the foramen magnum, skull and spine, and progression to requiring decompression surgery.

    "We continue to expand our clinical program of vosoritide for the treatment of achondroplasia with the first study specifically looking at the safety of vosoritide administered to infants at risk of serious and potentially fatal medical complications resulting from achondroplasia," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin.  "We are committed to understanding whether vosoritide can safely reduce the number of adverse outcomes in this high-risk population and are pleased to support this study as part of our commitment  to advancing the standard of care for children affected by achondroplasia." 

    Investigator-Initiated Phase 2 Study by Children's National Hospital to Explore Vosoritide in Selected Genetic Forms of Short Stature

    In addition, Children's National Hospital in Washington, D.C. the sponsor of the investigator-initiated study, and BioMarin announced today that the first children have been enrolled in a Phase 2 study of the investigational treatment of vosoritide in children with certain genetic forms of short stature. 

    This investigator-initiated study will enroll approximately 35 children with short stature in specific genetic categories and will follow them for a six-month observation period to obtain baseline growth velocity, safety profile and quality of life assessment.  Study participants will then be treated with vosoritide for 12 months and will be assessed for safety and improvement in growth outcomes.  The primary endpoints include incidence of treatment emergent adverse events, change from baseline in annualized growth velocity and change from baseline in height Z-score.

    "Many patients who present with short stature likely have genetic mutations in genes involved in growth plate physiology.  Those patients with selected causes of short stature that interact with the CNP pathway may benefit from treatment with vosoritide, which directly targets the growth plate," said Andrew Dauber, M.D., MMSc, Chief of Endocrinology at Children's National Hospital, Washington D.C. and lead investigator of this clinical study.  "In this study, our goal is to understand if vosoritide may be a safe and effective treatment option for certain genetically defined short stature syndromes."

    "We are pleased to support the clinical research by Dr. Dauber at Children's National Hospital.  We applaud his efforts to study vosoritide for specific types of short stature beyond achondroplasia," Fuchs added.  "Vosoritide is currently under development for the treatment of achondroplasia, the most common form of skeletal dysplasia.  We have a good foundation of safety data to help inform a clinical study in other potential indications. We look forward to supporting this research to understand if the CNP pathway represents a target in forms of short stature with specific genetic mutations, where Dr. Dauber's study is the first step to advance science in the genetic short stature space."

    About Achondroplasia

    Achondroplasia, the most common form of skeletal dysplasia in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a mutation in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, including foramen magnum compression, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, spinal stenosis and recurrent ear infections. Some of these complications can result in the need for invasive surgeries such as spinal cord decompression and straightening of bowed legs. In addition, studies show increased mortality at every age.

    More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation.  The worldwide incidence rate of achondroplasia is about one in 25,000 live births.  Vosoritide is being tested in children whose growth plates are still "open", typically those under 18 years of age. This is approximately 25% of people with achondroplasia.  In the U.S., Europe, Latin America, the Middle East, and most of Asia Pacific, there are currently no approved medicines for achondroplasia.

    About Genetic Forms of Short Stature

    Short stature can be caused by a number of genetic etiologies, many of which directly affect the growth plate. The CNP/NPR2 pathway is central to growth of the chondrocyte, therefore patients with selected genetic causes of short stature that interact with this pathway could benefit from treatment with vosoritide, which directly targets the growth plate. This study specifically focuses on five different genes related to the CNP/NPR2 pathway, including patients with deficiency in CNP itself or defects in its receptor encoded by the NPR2 gene; patients with hypochondroplasia; patients with defects in the SHOX gene; and patients with a Rasopathy (a group of genetic disorders that includes Noonan Syndrome and Neurofibromatosis type 1 amongst other disorders).  Collectively, these categories of genetic short stature affect approximately 275,000 of the addressable patient population globally based on incidence rate, diagnosis rate, and age under 18.

    About Children's National Hospital

    Children's National Hospital, based in Washington, D.C., celebrates 150 years of pediatric care, research and commitment to community. Volunteers opened the hospital in 1870 with 12 beds for children displaced after the Civil War. Today, 150 years stronger, it is among the nation's top 10 children's hospitals. It is ranked No. 1 for newborn care for the fourth straight year and ranked in the top 10 for endocrinology by U.S. News & World Report. Children's National is transforming pediatric medicine for all children. In 2020, construction will be complete on the Children's National Research & Innovation Campus, the first in the nation dedicated to pediatric research. Children's National has been designated twice as a Magnet® hospital, demonstrating the highest standards of nursing and patient care delivery. This pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty outpatient centers in the D.C., metropolitan area, including the Maryland and Northern Virginia suburbs. Children's National is home to the Children's National Research Institute and Sheikh Zayed Institute for Pediatric Surgical Innovation and is the nation's seventh-highest NIH-funded children's hospital. It is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. 

    About BioMarin

    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.

    For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

    Forward-Looking Statement for BioMarin

    This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin's vosoritide program generally, whether vosoritide can reduce the number of adverse outcomes in this high-risk population, the potential benefits and possible new indications of vosoritide for children with short stature that is genetically defined and involves the CNP/NPR2 pathway, the timing, design and conduct of the two new Phase 2 studies and the expected results of such studies. 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 vosoritide; our ability to enroll participants into such clinical trials, our ability to successfully manufacture vosoritide; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning vosoritide; and those other risks and uncertainties detailed from time to time under the caption "Risk Factors" and elsewhere in the Company's Securities and Exchange Commission (SEC) filings, including, without limitation, BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, and future SEC 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.

    Contact:



    Investors:

    Media:

    Traci McCarty

    Debra Charlesworth

    BioMarin Pharmaceutical Inc.

    BioMarin Pharmaceutical Inc.

    (415) 455-7558 

    (415) 455-7451

     

    Cision View original content:http://www.prnewswire.com/news-releases/biomarin-expands-vosoritide-clinical-program-301168464.html

    SOURCE BioMarin Pharmaceutical Inc.

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  15. SAN RAFAEL, Calif., Nov. 5, 2020 /PRNewswire/ --

    BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

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


    Three Months Ended September 30,


    Nine Months Ended September 30,


    2020


    2019


    % Change


    2020


    2019


    % Change













    Total Revenues

    $

    476.8



    $

    461.1



    3

    %


    $

    1,408.3



    $

    1,249.6



    13

    %













    Net Product Revenues Marketed by BioMarin (1)

    419.8



    428.1



    (2)

    %


    1,239.9



    1,150.6



    8

    %













    Vimizim Net Product Revenues

    147.9



    163.5



    (10)

    %


    401.8



    412.0



    (2)

    %

    Kuvan Net Product Revenues

    124.1



    120.6



    3

    %


    368.7



    340.8



    8

    %

    Naglazyme Net Product Revenues

    76.3



    94.4



    (19)

    %


    271.6



    279.5



    (3)

    %

    Palynziq Net Product Revenues

    46.1



    24.1



    91

    %


    121.4



    55.2



    120

    %

    Brineura Net Product Revenues

    25.4



    19.8



    28

    %


    75.2



    46.8



    61

    %













    Aldurazyme Net Product Revenues

    40.9



    22.8



    79

    %


    128.9



    73.9

    SAN RAFAEL, Calif., Nov. 5, 2020 /PRNewswire/ --

    BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

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



    Three Months Ended September 30,



    Nine Months Ended September 30,



    2020



    2019



    % Change



    2020



    2019



    % Change

























    Total Revenues

    $

    476.8





    $

    461.1





    3

    %



    $

    1,408.3





    $

    1,249.6





    13

    %

























    Net Product Revenues Marketed by BioMarin (1)

    419.8





    428.1





    (2)

    %



    1,239.9





    1,150.6





    8

    %

























    Vimizim Net Product Revenues

    147.9





    163.5





    (10)

    %



    401.8





    412.0





    (2)

    %

    Kuvan Net Product Revenues

    124.1





    120.6





    3

    %



    368.7





    340.8





    8

    %

    Naglazyme Net Product Revenues

    76.3





    94.4





    (19)

    %



    271.6





    279.5





    (3)

    %

    Palynziq Net Product Revenues

    46.1





    24.1





    91

    %



    121.4





    55.2





    120

    %

    Brineura Net Product Revenues

    25.4





    19.8





    28

    %



    75.2





    46.8





    61

    %

























    Aldurazyme Net Product Revenues

    40.9





    22.8





    79

    %



    128.9





    73.9





    74

    %

























    GAAP Net Income (Loss)

    $

    784.8





    $

    55.0









    $

    837.0





    $

    (38.9)







    GAAP Net Income (Loss) per Share – Basic

    $

    4.33





    $

    0.31









    $

    4.63





    $

    (0.22)







    GAAP Net Income (Loss) per Share – Diluted

    $

    4.01





    $

    0.30









    $

    4.39





    $

    (0.22)







    Non-GAAP Income (2)

    $

    98.7





    $

    78.1









    $

    272.6





    $

    120.1







     



    September 30,

    2020



    December 31,

    2019

    Cash, cash equivalents and investments

    $

    1,770.8





    $

    1,165.8







    (1)

    Net Product Revenues Marketed by BioMarin is the sum of revenues from Vimizim, Kuvan, Naglazyme, Palynziq, Brineura and Firdapse, 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 8 for a table showing Net Product Revenues by product, including Firdapse. 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 three and nine months ending September 30, 2020; as a result of the transaction BioMarin will not recognize Net Product Revenues from Firdapse in the future.

    (2)

    Non-GAAP Income is defined by the Company as reported GAAP Net Income, 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 9 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 third quarter ended September 30, 2020.

    Net Product Revenues for the third quarter of 2020 increased to $460.7 million, compared to $450.9 million in the third quarter of 2019. The increase in Net Product Revenues was primarily attributed to the following:

    • Palynziq Net Product Revenues increased by $22.0 million driven by a combination of revenue from U.S. patients achieving maintenance dosing and new patients initiating therapy;
    • Aldurazyme Net Product Revenues increased by $18.1 million due to higher sales volume to Genzyme;
    • Naglazyme and Vimizim Net Product Revenues decreased by an aggregate of $33.7 million primarily due to timing of orders placed from Latin America as well as the impact of missed infusions resulting from the COVID-19 pandemic.

    The increase in GAAP Net Income for the third quarter of 2020, compared to the same period in 2019 was primarily due to the following:

    • an increase in the benefit from income taxes of $800.8 million primarily due the completion of an intra-entity transfer of certain intellectual property (IP) rights to an Irish subsidiary where the Company's Ex-US regional headquarters are located and has significant manufacturing and commercial operations, to better align ownership of IP rights with how the business operates resulting in a tax benefit of $835.1 million based on the fair value of the transferred IP rights; and
    • decreased research and development (R&D) expense primarily resulting from decreased clinical manufacturing costs for BMN 307 and lower clinical activity spend for valoctocogene roxaparvovec gene therapy programs; partially offset by
    • an increase in Cost of Sales of $91.8 million primarily attributed to the $81.2 million reserve of valoctocogene roxaparvovec pre-launch inventory due to delays in anticipated regulatory approvals; and
    • higher selling, general and administrative (SG&A) expense related to pre-commercialization activities for valoctocogene roxaparvovec.

    Non-GAAP Income for the third quarter of 2020 increased to $98.7 million, compared to Non-GAAP Income of $78.1 million for the same period in 2019. The increase in Non-GAAP Income for the quarter, compared to the same period in 2019, was attributed to decreased R&D expense and higher gross profit, excluding the $81.2 million pre-launch inventory charge, partially offset by higher SG&A expense.

    As of September 30, 2020, BioMarin had cash, cash equivalents and investments totaling approximately $1.8 billion, which includes net proceeds of $535.8 million from the Company's May 2020 convertible debt offering, as compared to $1.2 billion on December 31, 2019. On October 15, 2020, the Company's 1.50% senior subordinate convertible notes matured and were settled in cash for approximately $375.0 million.

    Commenting on third quarter 2020 results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "While the impact of COVID-19 continued through the third quarter, BioMarin employees remained focused on our mission to serve patients and ensure the steady supply of our critically-important medicines. In these continued unpredictable times, the essential nature of our products to the people who rely on them remains constant."

    Mr. Bienaimé continued, "In the third quarter we received unexpected news on the status of our application with valoctocogene roxaparvovec for hemophilia A from health authorities, which resulted in a delay in potential approval timelines. However, we remain confident in our valoctocogene roxaparvovec gene therapy and its potential to redefine the treatment paradigm for people with hemophilia A. We continue to work with the health authorities to align on next steps toward approval. Our 134-subject Phase 3 study with valoctocogene roxaparvovec will complete one-year of observation in all subjects later this month, and we anticipate sharing top-line results comprising 1 to 2 years of follow-up from that study, in the first quarter of 2021. We also plan to submit the complete one-year Phase 3 data to the EMA in the second quarter of 2021."

    "Vosoritide for the treatment of achondroplasia is advancing as planned with applications under review in both the U.S. and Europe with potential regulatory approvals anticipated in 2021. The significant unmet medical need for children with achondroplasia has enabled BioMarin to build a multi-pronged dossier of clinical studies. It includes the highly statistically significant placebo-controlled Phase 3 results, long-term clinical results in 5 to 18 year-olds from our Phase 2 study, natural history data, and the ongoing study of newborns through 5 years, which is nearing enrollment completion. The positive results from our vosoritide clinical programs bolster our confidence in the potential for this drug to be the first pharmacological treatment to address the underlying cause of achondroplasia. Interest in our clinical studies with vosoritide has been extremely robust, demonstrating that many families are keen to seek early treatment for their children."

    Mr. Bienaimé concluded, "Despite the impact from COVID-19 and the timing set-back on the potential approval of valoctocogene roxaparvovec, we remain confident in our business. BioMarin fundamentals are strong, driven by our global base business of essential medicines and cash position, but our people and pursuit and development of innovative therapies will always be our most important assets."

    2020 Full-Year Financial Guidance (in millions, except %) 

    Item



    Provided August 4, 2020



    Revised November 5, 2020

    Total Revenues (1)



    $1,850

    to

    $1,950



    $1,810

    to

    $1,870

    Vimizim Net Product Revenues



    $530

    to

    $570



    $515

    to

    $545

    Kuvan Net Product Revenues



    $430

    to

    $480



    Unchanged

    Naglazyme Net Product Revenues



    $360

    to

    $400



    $370

    to

    $400

    Palynziq Net Product Revenues



    $160

    to

    $190



    Unchanged

    Brineura Net Product Revenues



    $85

    to

    $115



    $90

    to

    $110



















    Cost of Sales (% of Total Revenues) (2)



    20%

    to

    21%



    26%

    to

    28%

    Research and Development Expense



    $675

    to

    $725



    $630

    to

    $670

    Selling, General and Administrative Expense



    $780

    to

    $830



    $725

    to

    $765



















    GAAP Net Income



    $720

    to

    $980



    $760

    to

    $820

    Non-GAAP Income (3)



    $260

    to

    $310



    $280

    to

    $330





    (1)

    Revenue guidance reflects BioMarin's projected impact of the COVID-19 pandemic on its global revenue sources, mostly in the form of demand interruptions such as missed patient infusions and delayed treatment starts for new patients. Total Revenue guidance also reflects the impact of the valoctocogene roxaparvovec FDA Complete Response Letter whereby the Company no longer expects any revenue from valoctocogene roxaparvovec in 2020 and the previously anticipated October 2020 loss of market exclusivity for Kuvan in the U.S. Management also notes that the impact of COVID-19 on revenues is expected to persist into 2021 due primarily to the effect of delays in new patients initiating therapy.

    (2)

    Revised Cost of Sales guidance for 2020 reflects the incremental charge of $81.2 million during the third quarter of 2020 related to valoctocogene roxaparvovec pre-launch inventory reserves.

    (3)

    All Financial Guidance items are calculated based on U.S. GAAP with the exception of Non-GAAP Income/Loss. Refer to Non-GAAP Information beginning on page 9 of this press release for a complete discussion of the Company's Non-GAAP financial information and reconciliations to the corresponding GAAP reported information.

    Key Program Highlights

    • Valoctocogene roxaparvovec gene therapy for severe hemophilia A: BioMarin is working with the U.S. Food and Drug Administration to align on steps forward to obtain marketing approval following the August 18, 2020 Complete Response Letter to the Company's Biologics License Application for valoctocogene roxaparvovec gene therapy for severe hemophilia A. The FDA recommended that the Company complete the Phase 3 study and submit two-year follow-up safety and efficacy data on all study participants. The Phase 3 study was fully enrolled in November 2019 and will complete one-year of follow-up of all patients in November 2020. The Company intends to share the one-year top-line Phase 3 data in the first quarter of 2021.



      Additionally, the EMA recently requested the 52-week results from the full Phase 3 study cohort of 134 subjects to inform their benefit-risk assessment. To facilitate this submission within the EMA regulatory framework, BioMarin recently withdrew the MAA and plans to resubmit the MAA with these data to the EMA in the second quarter of 2021.



    • Vosoritide for children with achondroplasia: Marketing applications for vosoritide were recently validated and accepted, by EMA and FDA, respectively. The CHMP opinion is expected in Europe in the second half of 2021. The U.S. New Drug Application for vosoritide is under Standard review by the FDA with a Prescription Drug User Fee Act target action date of August 20, 2021. Vosoritide is an investigational, once daily injection of an analog of C-type Natriuretic Peptide. If approved, vosoritide would be the only therapeutic treatment available for children with achondroplasia.



    • Palynziq for Phenylketonuria (PKU): On October 7, 2020 the Company announced that the FDA approved the supplemental Biologics License Application (sBLA) to increase the maximum allowable dose of Palynziq (pegvaliase-pqpz) Injection for treatment of adults with Phenylketonuria (PKU) to 60 mg daily. Previously, the maximum dose was 40 mg daily. In the Phase 3 PRISM studies, 19% of study participants required a 60 mg dose to achieve adequate response to Palynziq.



      Palynziq is indicated to reduce blood Phe concentrations in adults with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 μmol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first and only approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe.



    • BMN 307 gene therapy product candidate for phenylketonuria (PKU): On September 24, 2020, the Company announced that it began dosing participants in PHEARLESS, the Phase 1/2 study of BMN 307. Both the FDA and EMA granted BMN 307 Orphan Drug Status. Additionally, the FDA has granted Fast Track status to BMN 307. Product for use in the Phase 1/2 study was made at commercial scale from BioMarin's award-winning gene therapy manufacturing facility.



    • BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): IND-enabling studies are ongoing with BMN 331, BioMarin's third gene therapy candidate, for the treatment of Hereditary Angioedema (HAE). 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.



    • DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM): Pre-clinical studies are underway with DiNA-001 following a collaboration announced in May 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.

    BioMarin will host a conference call and webcast to discuss third quarter 2020 financial results today, Thursday, November 5, 2020 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: 3291898

    Conference ID: 3291898

    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 Income, Non-GAAP Income, and other specified income statement guidance for the full-year 2020; the timing of BioMarin's clinical development and commercial prospects, including announcements of data from clinical studies and trials, including that the Company anticipates sharing top-line results from its Phase 3 study with valoctocogene roxaparvovec in early 2021; the clinical development and commercialization of BioMarin's product candidates and commercial products, including (i) BioMarin's plan to submit complete one-year Phase 3 data to the EMA in the second quarter of 2021, (ii) BioMarin's plan to resubmit its MAA for valoctocogene roxaparvovec to the EMA in the second quarter of 2021,and (iii) the potential approval and commercialization of BioMarin's product candidates, including vosoritide for the treatment of achondroplasia and valoctocogene roxaparvovec for the treatment of severe hemophilia A, including timing of such approval decisions.

    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; 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 June 30, 2020 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. Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

    Contact:





    Investors:



    Media:

    Traci McCarty



    Debra Charlesworth

    BioMarin Pharmaceutical Inc.



    BioMarin Pharmaceutical Inc.

    (415) 455-7558



    (415) 455-7451

     

    BIOMARIN PHARMACEUTICAL INC.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    September 30, 2020 and December 31, 2019

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





    September 30,

    2020



    December 31,

    2019(1)

    ASSETS

    (unaudited)





    Current assets:







    Cash and cash equivalents

    $

    1,015,675





    $

    437,446



    Short-term investments

    489,998





    316,361



    Accounts receivable, net

    411,712





    377,404



    Inventory

    700,847





    680,275



    Other current assets

    120,747





    130,657



    Total current assets

    2,738,979





    1,942,143



    Noncurrent assets:







    Long-term investments

    265,122





    411,978



    Property, plant and equipment, net

    1,015,062





    1,010,868



    Intangible assets, net

    427,172





    456,580



    Goodwill

    196,199





    197,039



    Deferred tax assets

    1,396,547





    549,422



    Other assets

    119,009





    122,009



    Total assets

    $

    6,158,090





    $

    4,690,039



    LIABILITIES AND STOCKHOLDERS' EQUITY







    Current liabilities:







    Accounts payable and accrued liabilities

    $

    480,403





    $

    570,621



    Short-term convertible debt, net

    374,290





    361,882



    Total current liabilities

    854,693





    932,503



    Noncurrent liabilities:







    Long-term convertible debt, net

    1,074,164





    486,238



    Long-term contingent consideration

    54,103





    50,793



    Other long-term liabilities

    121,237





    98,124



    Total liabilities

    $

    2,104,197





    $

    1,567,658



    Stockholders' equity:







    Common stock, $0.001 par value: 500,000,000 shares authorized; 181,492,344 and 179,838,114 shares issued and outstanding, respectively.

    181





    180



    Additional paid-in capital

    4,937,791





    4,832,707



    Company common stock held by Nonqualified Deferred Compensation Plan

    (10,756)





    (9,961)



    Accumulated other comprehensive income

    10,385





    20,164



    Accumulated deficit

    (883,708)





    (1,720,709)



    Total stockholders' equity

    4,053,893





    3,122,381



    Total liabilities and stockholders' equity

    $

    6,158,090





    $

    4,690,039







    (1)

    December 31, 2019 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, 2019, filed with the U.S. Securities and Exchange Commission (SEC) on February 27, 2020.



     

    BIOMARIN PHARMACEUTICAL INC.

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

    Nine Months Ended September 30, 2020 and 2019

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





    Three Months Ended

    September 30,



    Nine Months Ended

    September 30,



    2020



    2019



    2020



    2019



    (unaudited)



    (unaudited)



    (unaudited)



    (unaudited)

    REVENUES:















    Net product revenues

    $

    460,741





    $

    450,900





    $

    1,368,816





    $

    1,224,458



    Royalty and other revenues

    16,043





    10,197





    39,522





    25,147



    Total net revenues

    476,784





    461,097





    1,408,338





    1,249,605



    OPERATING EXPENSES:















    Cost of sales

    188,793





    96,949





    398,134





    263,567



    Research and development

    147,053





    172,963





    471,449





    542,195



    Selling, general and administrative

    179,450





    170,112





    542,157





    493,024



    Intangible asset amortization and contingent consideration

    17,429





    17,063





    48,018





    57,114



    Gain on sale of nonfinancial assets









    (59,495)





    (15,000)



    Total operating expenses

    532,725





    457,087





    1,400,263





    1,340,900



    INCOME (LOSS) FROM OPERATIONS

    (55,941)





    4,010





    8,075





    (91,295)



















    Equity in the loss of BioMarin/Genzyme LLC

    (921)





    (551)





    (1,077)





    (780)



    Interest income

    4,004





    5,340





    13,539





    17,537



    Interest expense

    (9,597)





    (2,937)





    (24,560)





    (16,530)



    Other income, net

    1,239





    3,960





    1,886





    6,038



    INCOME (LOSS) BEFORE INCOME TAXES

    (61,216)





    9,822





    (2,137)





    (85,030)



    Benefit from income taxes

    (846,019)





    (45,214)





    (839,138)





    (46,158)



    NET INCOME (LOSS)

    784,803





    55,036





    837,001





    (38,872)



    NET INCOME (LOSS) PER SHARE, BASIC

    $

    4.33





    $

    0.31





    $

    4.63





    $

    (0.22)



    NET INCOME (LOSS) PER SHARE, DILUTED

    $

    4.01





    $

    0.30





    $

    4.39





    $

    (0.22)



    Weighted average common shares outstanding, basic

    181,142





    179,289





    180,592





    178,873



    Weighted average common shares outstanding, diluted

    197,674





    185,924





    194,959





    178,873



    The following table presents Net Product Revenues by Product:

    Net Product Revenues by Product

    (In millions of U.S. dollars)





    Three Months Ended

    September 30,



    Nine Months Ended

    September 30,



    2020



    2019



    % Change



    2020



    2019



    % Change



    (unaudited)



    (unaudited)







    (unaudited)



    (unaudited)





    PKU franchise

    $

    170.2





    $

    144.7





    18

    %



    $

    490.1





    $

    396.0





    24

    %

    Vimizim