BioMarin Pharmaceutical Inc. (BMRN) develops treatments for serious life-threatening medical conditions, mainly affecting children. The company’s top line is currently being driven by strong demand for its marketed products, especially new dwarfism drug, Voxzogo. Also, the recent approval of Roctavian (valoctocogene roxaparvovec, or valrox), a gene therapy for treating adults with severe hemophilia A, has opened up a key sales opportunity.BioMarin has an attractive early-stage pipeline primarily focusing on ...

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The great advances and improvements in the treatment of diseases and the effectiveness of these medicines are undoubtedly due to technological progress and how biotech companies take advantage of it for their growth and development. Without the work of these companies, we would still be using very old and ineffective medicines. This new year brings with it very good opportunities to invest in companies in this sector. Here are these top biotech stocks to consider adding to your portfolio. Let’s take a look ...

Biomarin Pharmaceutical, Inc. (NASDAQ:BMRN) Q3 2023 Earnings Conference Call November 1, 2023 4:30 PM ET Company Participants Traci McCarty - Head of Investor Relations Jean-Jacques Bienaime - Chairman and Chief Executive Officer Jeffrey Ajer - Chief Commercial Officer Henry Fuchs - President, Worldwide Research and Development Brian Mueller - Chief Financial Officer Greg Guyer - Chief Technical Officer, Manufacturing and Technical Operations Conference Call Participants Salveen Richter - Goldman Sachs Geof ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ______________________________________ ______________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 Or☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number: 000-26727 ______________________________________ BioMarin Pharmaceutical Inc ...

Securities registered pursuant to Section 12(b) of the Act: Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, par value $0.001 BMRN The Nasdaq Global Select Market ______________________________________ Form 10-Q Commission File Number: 000-26727 ______________________________________ BioMarin Pharmaceutical Inc. (Exact name of registrant as specified in its charter) ______________________________________ Delaware 68-0397820 (State or other jurisdiction of incorpo ...

Biomarin Pharmaceutical, Inc. (NASDAQ:BMRN) Q2 2023 Earnings Conference Call July 31, 2023 4:30 PM ET Company Participants Traci McCarty - Head of Investor Relations Jean-Jacques Bienaime - Chairman and Chief Executive Officer Jeffrey Ajer - Chief Commercial Officer Henry Fuchs - President, Worldwide Research and Development Brian Mueller - Chief Financial Officer Greg Guyer - Chief Technical Officer, Manufacturing and Technical Operations Conference Call Participants Akash Tewari - Jefferies Salveen Richte ...

| --- | --- | --- | --- | --- | --- | --- | |----------------------------------|-------------|---------------------------------------------------------------------------------------|-------|---------------------------------------------|-------|--------| | Assets: | Significant | Fair Value Measurements as of December 31, 2022 \n Other Observable Inputs (Level 2) | | Significant Unobservable Inputs (Level 3) | | Total | | Other current assets: | | | | | | | | NQDC Plan assets | $ | 2,654 | $ | — | $ | 2,654 ...

Biomarin Pharmaceutical, Inc. (NASDAQ:BMRN) Q1 2023 Earnings Conference Call April 26, 2023 4:30 PM ET Company Participants Traci McCarty - Group VP, IR Jean-Jacques Bienaime - Chairman & CEO Jeffrey Ajer - Chief Commercial Officer Henry Fuchs - President, Worldwide Research & Development Brian Mueller - CFO & EVP Conference Call Participants Salveen Richter - Goldman Sachs Group Geoffrey Meacham - Bank of America Merrill Lynch Christopher Raymond - Piper Sandler & Co. Timothy Lugo - William Blair & Company ...

Financial Data and Key Metrics Changes - BioMarin achieved total revenues of $2.1 billion for the full year 2022, marking a record financial achievement for the company [33] - The company reported a revenue growth of close to 20% in Q4 2022, with total revenues of $538 million [34] - GAAP net income and non-GAAP income growth were achieved despite recognizing approximately $23 million in charges to SG&A expenses in 2022, primarily in Q4 [27] Business Line Data and Key Metrics Changes - VOXZOGO generated full-year global revenue of $169 million in 2022, with guidance for 2023 set between $330 million and $380 million, indicating over 100% growth from the midpoint compared to 2022 [12] - PALYNZIQ therapy in the U.S. and Europe saw net product revenue growth of 7% in 2022, totaling $255 million [15] - ROCTAVIAN is projected to generate revenues between $100 million and $200 million in 2023, contingent on U.S. approval [38] Market Data and Key Metrics Changes - As of the end of Q4 2022, only 6% of the total addressable patient population had received VOXZOGO treatment, indicating significant growth potential [12] - In Germany, a major payer agreement has been established for ROCTAVIAN, although the commercial ramp has been hindered by lengthy negotiations for additional outcomes-based agreements [39] Company Strategy and Development Direction - The company aims for over 15% top-line growth in 2023, with a focus on sustainable profitability and leveraging its enzyme products alongside new market opportunities from VOXZOGO and ROCTAVIAN [33][43] - BioMarin is actively pursuing regulatory approvals to expand VOXZOGO's label to younger patients, which could significantly increase the addressable market [13][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the interest from prescribers and patients for ROCTAVIAN, despite the lengthy reimbursement process in Germany [32] - The company anticipates increased demand for all brands in 2023, including enzyme products, VOXZOGO, and ROCTAVIAN, with a commitment to growth and sustainable profitability [43] Other Important Information - The company is transitioning to a new methodology for calculating non-GAAP income to better align with its profitable business model and peer group [75] - BioMarin is investing in its largest early-stage research pipeline to fuel growth throughout the decade [74] Q&A Session Summary Question: What is the expected timeline for revenue flow from ROCTAVIAN in Germany? - Management confirmed that the 10 patients undergoing companion diagnostic testing are in Germany and emphasized the importance of these tests as a step towards treatment [80][81] Question: What is the status of the three-year data submission for ROCTAVIAN? - The company indicated that the assessment of whether the submission constitutes a major amendment is subjective and cannot provide specific guidance at this time [85] Question: How many U.S. treatment centers are expected to be ready for ROCTAVIAN at launch? - The company plans to target a small number of the largest and most capable hemophilia treatment centers in the U.S. for the launch of ROCTAVIAN [96] Question: What are the implications of the outcomes-based agreements in Germany? - Management explained that while these agreements will be superseded by federal reimbursement, they still provide value in facilitating patient access to therapy [89][116]