BioMarin Pharmaceutical

BioMarin Pharmaceutical, Inc. engages in the development and commercialization of therapies for people with serious and life-threatening rare diseases and medical conditions. Its pipeline products include Valoctocogene roxaparvovec, Vosoritide, and BMN 307. The company was founded by John C. Klock, Christopher M. Starr and Grant W. Denison on March 21, 1997, and is headquartered in San Rafael, CA.

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BioMarin excels in Q4 with record revenues and gene therapy launch

BioMarin Pharmaceutical reported strong financial results in Q4 2022, achieving revenue growth, profitability, and successful introduction of the ROCTAVIAN gene therapy for hemophilia A.
The company's performance highlights its R&D capabilities, market positioning, and strategic readiness for upcoming product launches, positioning BioMarin for continued growth and innovation.
While facing challenges in reimbursement processes and regulatory requirements, BioMarin has opportunities to drive revenue expansion, market penetration, and address unmet medical needs through its innovative pipeline.

BioMarin reports strong Q1 earnings, eyes blockbuster status for ROCTAVIAN

BioMarin Pharmaceuticals had a robust Q1 2023, focusing on revenue growth, profitability, and commercial readiness for key drug launches. While facing challenges in Germany, the company is optimistic about the potential of ROCTAVIAN and VOXZOGO in driving future growth.

BioMarin excels in Q2 with record revenues and treatment milestones

BioMarin's strong Q2 performance, driven by record revenues and product demand, positions the company for growth.
Challenges like supply issues and site readiness are being addressed, while opportunities in new programs and markets are promising.
Overall, BioMarin's focus on compliance, expansion, and pipeline development sets a positive trajectory for future success.

BioMarin reports strong Q3 revenue growth, eyes Roctavian expansion

BioMarin's Q3 performance showed revenue growth driven by Voxzogo, with strong expectations for 2024 revenue.
New CEO Alexander Hardy is set to continue the core strategy, focusing on Roctavian's global launch and Voxzogo's growth potential.
Challenges include delays in Roctavian's takeoff and supply constraints for Voxzogo, but efforts are underway to address these issues.
Opportunities lie in Roctavian's revenue contribution, Voxzogo's U.S. growth potential, and leveraging CNP for stature improvements.

BioMarin posts robust Q4 earnings, eyes expansion in gene therapy

BioMarin Pharmaceutical delivered strong financial results in Q4 2023, driven by revenue growth and increased earnings per share. The company's focus on high-impact medicines and gene therapy expertise positions it well for future growth.
While facing challenges related to analyst inquiries and market adoption of key products like Roctavian and Voxzogo, BioMarin remains committed to innovation and optimizing its business model.
Opportunities lie in expanding product reach, maximizing shareholder value through strategic capital allocation, and driving innovation in transformative programs. BioMarin's long-term strategy and growth potential remain promising.

BioMarin reports record revenue, focuses on key assets for growth

BioMarin Pharmaceutical's Q1 2024 performance reflects strong revenue growth and financial stability, driven by key product demand.
While facing challenges such as revenue impact and supply constraints, the company's strategic focus on key assets and cost reduction initiatives bode well for future growth.
Opportunities lie in expanding Voxzogo's market reach, optimizing manufacturing capabilities, and maintaining a competitive edge in innovative treatments.

BioMarin Pharmaceuticals reports record revenue and profitability outlook for 2024

BioMarin Pharmaceuticals delivered strong financial performance in Q2 2024, driven by robust revenue growth and strategic decisions. The company's focus on profitability, expansion of key treatments, and progress in R&D highlight a promising outlook. Addressing analyst inquiries and leveraging upcoming events can further enhance investor confidence and market positioning.

BioMarin reports record Q3 revenues, eyes long-term growth targets

BioMarin Pharmaceuticals showcased robust financial performance and raised full-year guidance.
The company remains optimistic about its long-term growth prospects and is committed to defending its IP.
Strategic focus on expanding global reach and driving growth in enzyme therapies portfolio.

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New England Journal of Medicine Publishes Editorial: Prepare the Way for Hemophilia A Gene Therapy Significantly Reduced Bleeding and Factor VIII Utilization Compared to Baseline Factor VIII Prophylactic Therapy Mean Annualized Bleeding Rate (ABR) Reduced by 84% (p-value

BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A

BioMarin released positive financials, but investors were disappointed by some clinical trial data.

BioMarin: Keep Waiting For The Growth

While the company broadly met expectations for the fourth quarter, investors seemed concerned about its 2022 guidance.

Why BioMarin Pharmaceutical Tumbled on Thursday

Biomarin Pharmaceutical's (BMRN) fourth-quarter earnings and sales beat estimates. Stock declines

BioMarin (BMRN) Q4 Earnings Top Estimates, Sales Fall Y/Y

BioMarin Pharmaceutical Inc. (BMRN) CEO J.J. Bienaime on Q4 2021 Results - Earnings Call Transcript

BioMarin (BMRN) delivered earnings and revenue surprises of 250% and 1.22%, respectively, for the quarter ended December 2021. Do the numbers hold clues to what lies ahead for the stock?

BioMarin Pharmaceutical (BMRN) Q4 Earnings and Revenues Top Estimates

SAN RAFAEL, Calif., Feb. 23, 2022 /PRNewswire/ -- Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)     Three Months Ended December 31, Twelve Months Ended December 31, 2021 2020 % Change 2021 2020 % Change Total Revenues $      449.8 $      452.1 (1)% $   1,846.3 $   1,860.5 (1)% Net Product Revenues Marketed by BioMarin (1) $      414.9 $      435.8 (5)% $   1,660.7 $   1,675.8 (1)% Vimizim Net Product Revenues $      156.3 $      142.5 10% $      623.1 $      544.4 14% Naglazyme Net Product Revenues $        83.1 $      119.7 (31)% $      380.4 $      391.3 (3)% Kuvan Net Product Revenues $        68.5 $        89.0 (23)% $      285.8 $      457.7 (38)% Palynziq Net Product Revenues $        63.8 $        49.6 29% $      237.5 $      171.0 39% Brineura Net Product Revenues $        37.4 $        35.0 7% $      128.0 $      110.2 16% Voxzogo Net Product Revenues $           5.8 $            — n/a $           5.9 $            — n/a Aldurazyme Net Product Revenues $        20.3 $           1.2 nm(2) $      122.8 $      130.1 (6)% GAAP Net Income (Loss) $       (57.9) $        22.1 $       (64.1) $      859.1 GAAP Net Income (Loss) per Share – Basic $       (0.32) $        0.12 $       (0.35) $        4.75 GAAP Net Income (Loss) per Share – Diluted $       (0.32) $        0.12 $       (0.35) $        4.53 Non-GAAP Income (3) $           7.1 $        39.5 $      242.8 $      312.2   December 31, 2021 December 31, 2020 Cash, cash equivalents and investments $                 1,521.7 $                 1,350.9 (1) Net Product Revenues Marketed by BioMarin is the sum of revenues from Vimizim, Naglazyme, Kuvan, Palynziq, Brineura and Voxzogo (formerly known as vosoritide) for the three and twelve months ended December 31, 2021, each calculated in accordance with Generally Accepted Accounting Principles in the United States (U.S. GAAP).

BioMarin Announces Fourth Quarter and Full Year 2021 Financial Results and Corporate Updates

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BioMarin Pharmaceutical Inc (NASDAQ: BMRN) said that the FDA had requested additional information regarding the clinical hold on PHEARLESS Phase 1/2 study of BMN 307 issued in September 2021.  BMN 307 is an AAV5-human phenylalanine hydroxylase (hPAH) gene therapy studied in adults with phenylketonuria (PKU).

FDA Asks For More Data To Resolve BioMarin's Gene Therapy Clinical Hold

SAN RAFAEL, Calif., Feb. 17, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced updates on its investigational gene therapy programs in clinical development.

BioMarin Provides Updates on Progress in Gene Therapy Programs

SAN RAFAEL, Calif., Feb. 16, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in two upcoming virtual conferences.

BioMarin to Participate in Two Upcoming Virtual Investor Conferences

SAN RAFAEL, Calif., Feb. 10, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday, February 23rd, at 4:30 p.m.

BioMarin to Host Fourth Quarter and Full Year 2021 Financial Results Conference Call and Webcast on Wednesday, February 23 at 4:30pm ET

BioMarin Pharmaceutical Inc BMRN has agreed to sell the Rare Pediatric Disease Priority Review Voucher (PRV) for $110 million to an undisclosed purchaser. Get the Inside Access Traders Are Using to Profit More and Win Bigger.

BioMarin Sells Priority Review Voucher For $110M

SAN RAFAEL, Calif., Feb. 9, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that it has entered into a definitive agreement with an undisclosed purchaser to sell the Rare Pediatric Disease Priority Review Voucher (PRV) it obtained in November 2021 for a lump sum payment of $110,000,000.

BioMarin Sells Priority Review Voucher for $110 Million

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SAN RAFAEL, Calif., Feb. 4, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Company presented positive results from a two-year analysis of the Phase 3 GENEr8-1 study and an overall safety update of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the 15th Annual Virtual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).

BioMarin Announces Oral Presentation of 2-Year Analysis of Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A at 15th Annual Congress of European Association for Haemophilia and Allied Disorders (EAHAD) 2-4 February

Wilmington, Delaware--(Newsfile Corp. - February 3, 2022) - Rigrodsky Law, P.A. is investigating potential claims against the officers and directors of BioMarin Pharmaceutical Inc. ("BioMarin") (NASDAQ: BMRN) on behalf of stockholders.On October 25, 2021, a class action complaint was filed against BioMarin. The complaint alleges that, between January 13, 2020 and September 3, 2021, defendants made materially false and misleading statements regarding BioMarin's business, operations, and compliance policies. Specifically, defendants made false and/or...

SHAREHOLDER ALERT: Rigrodsky Law, P.A. Announces Investigation of BioMarin Pharmaceutical Inc.

SAN RAFAEL, Calif., Feb. 2, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Company will be presenting data in several presentations on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A at the 15th Annual Virtual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) February 2-4.

BioMarin Demonstrates Enduring Commitment to Advancing the Standard of Care in Hemophilia A at 15th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) February 2-4

BioMarin achieves primary endpoint of 2-year annualized bleeding rate in phase 3 GENEr8-1 study using the gene therapy valoctocogene roxaparvovec for severe Hemophilia A patients. BLA filing and European reviews of valoctocogene roxaparvovec for severe Hemophilia A patients are expected in 1st half of 2022.

BioMarin's Win In Phase 3 Hemophilia Study Makes Its Investment Case Stronger

Shares of BioMarin Pharmaceutical Inc. BMRN, -1.10% gained 5.6% in premarket trading on Monday, the day after the company announced promising data from a Phase 3 clinical study of its investigational gene therapy for adults with severe hemophilia A. Biomarin said the treatment reduced the annualized bleeding rate among the 134 participants in the study.

Biomarin plans to resubmit its experimental gene therapy for hemophilia A to the FDA in the second quarter

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) announced results from its ongoing Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, investigational gene therapy for severe hemophilia A.  Annualized Bleeding Rate (ABR) was significantly reduced by 4.1 treated bleeds per year in the pre-specified primary analysis in participants from a prior non-interventional study.

BioMarin's Hemophilia Gene Therapy Meets Efficacy Endpoints At Two Year Analysis

SAN RAFAEL, Calif., Jan. 9, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive 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.

BioMarin Announces Stable and Durable Annualized Bleed Control in the Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A; 134-Participant Study Met All Primary and Secondary Efficacy Endpoints at Two Year Analysis

BioMarin to Present at Two Investor Conferences in January 2022 SAN RAFAEL, Calif., Jan. 4, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will present at two virtual conferences in January.

BioMarin to Participate in Two Virtual Investor Conferences in January

Securities Litigation Partner James (Josh) Wilson Encourages Investors Who Suffered Losses Exceeding $100,000 In BioMarin To Contact Him Directly To Discuss Their OptionsNew York, New York--(Newsfile Corp. - January 2, 2022) - Faruqi & Faruqi, LLP, a leading national securities law firm, is investigating potential claims against BioMarin Pharmaceutical Inc. ("BioMarin" or the "Company") (NASDAQ: BMRN) and reminds investors of the December 22, 2021 deadline to seek the role of lead plaintiff in a federal securities class action that has been filed against the Company.If you suffered losses exceeding $100,000 investing in BioMarin stock or options between...

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