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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SAN RAFAEL, Calif., Oct. 12, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday, October 27, at 4:30 p.m.

BioMarin to Host Third Quarter 2021 Financial Results Conference Call and Webcast on Wednesday, October 27 at 4:30pm ET

SAN RAFAEL, Calif., Oct. 4, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Company hired two industry veterans Harold S.

BioMarin Hires Industry Veterans to Fill Key Roles in R&D and Technical Operations

New York, New York--(Newsfile Corp. - September 21, 2021) - Pomerantz LLP is investigating claims on behalf of investors of BioMarin Pharmaceutical Inc. ("BioMarin" or the "Company") (NASDAQ: BMRN). Such investors are advised to contact Robert S. Willoughby at newaction@pomlaw.com or 888-476-6529, ext. 7980.The investigation concerns whether BioMarin and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices. [Click here for information about...

SHAREHOLDER ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of BioMarin Pharmaceutical Inc. - BMRN

Stifel has upgraded BioMarin Pharmaceutical Inc's (NASDAQ:BMRN) to Buy from Hold with a price target of $96, up from $86.  After the stock's "significant underperformance" since August 2020, analyst Paul Matteis is expecting a turnaround with potential Vosoritide FDA approval and a "strong launch after that.

Stifel Upgrades This Pharma Stock On Prospective FDA Approval For Dwarfism Drug

BioMarin's (BMRN) phase I/II study on gene therapy candidate, BMN 307, has been placed under a clinical hold by the FDA possibly on malignancy concerns.

BioMarin (BMRN) Phenylketonuria Gene Therapy on Clinical Hold

​​​​​​BioMarin Pharmaceutical Inc (NASDAQ: BMRN) faces another setback in its gene therapy development program after the FDA placed the phenylketonuria (PKU) gene therapy trial on hold. The Phase 1/2 Phearless study is evaluating BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy for PKU.

FDA Slaps Clinical Hold on BioMarin's Gene Therapy For Genetic Metabolism Disorder

SAN RAFAEL, Calif., Sept. 6, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S Food and Drug Administration (FDA) placed a clinical hold on the BMN 307 Phearless Phase 1/2 study.

U.S. FDA Placed a Clinical Hold on BMN 307 Phearless Phase 1/2 Gene Therapy Study in Adults with PKU Based on Interim Pre-clinical Study Findings

Biomarin Pharmaceutical's (BMRN) Voxzogo is the first medicine in Europe to be approved to treat achondroplasia, the most common form of dwarfism.

BioMarin's (BMRN) Dwarfism Drug Gets Approval in Europe

BioMarin (BMRN) reported earnings 30 days ago. What's next for the stock?

Why Is BioMarin (BMRN) Up 0.4% Since Last Earnings Report?

The European Commission (EC) has approved BioMarin Pharmaceutical Inc's (NASDAQ: BMRN) Voxzogo (vosoritide) once-daily injection to treat achondroplasia. The approval covers children from 2 years until growth plates are closed, which occurs after puberty when children reach final adult height.

Drug For Short-Limbed Dwarfism From Biomarin Wins European Approval

SAN RAFAEL, Calif., Aug. 27, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Commission (EC) has granted marketing authorization for VOXZOGO® (vosoritide), a once daily injection to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height.

European Commission Approves BioMarin's VOXZOGO® (vosoritide) for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close

The value of the current BioMarin's business + Voxzogo is $104 per share.

BioMarin: More Opportunities Ahead

SAN RAFAEL, Calif., Aug. 25, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in three upcoming virtual investor conferences.

BioMarin to Participate in Three Upcoming Virtual Investor Conferences

All it will take is an FDA approval and a partial repeat of history.

Get Ready for a Potential Double in This Biopharmaceutical Stock in the Next 6 to 12 Months

BMRN has an upcoming PDUFA, and probably another for Roctavian next year. They recently raised full year guidance on the back of better than expected revenue from the last quarter.

BioMarin Is Highly Attractive For Longer-Term Investors

BioMarin (BMRN) beats on both earnings and revenues in the second quarter of 2021.

BioMarin (BMRN) Beats on Q2 Earnings, Ups 2021 Guidance

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) reported a 17% Y/Y increase in revenue to $501.7 million in Q2, beating the consensus of $448.8 million, attributable to Vimizim, Naglazyme, and Palynziq product revenues, partially offset by lower Kuvan product revenues.  Vimizim and Naglazyme sales increased 47% to $171.7 million and $118.8 million, respectively, primarily driven by the timing of orders from Europe and the Middle East.

BioMarin Bounces Back With Solid Q2 Earnings, Nudges Up 2021 Guidance

BioMarin Pharmaceutical Inc. (BMRN) CEO J.J. Bienaimé on Q2 2021 Results - Earnings Call Transcript

A strong quarterly sales report, and a new product approval, brighten the outlook for the maker of rare-disease treatments.

BioMarin Bounces Back With Solid Results

Shares of Biomarin Pharmaceutical (NASDAQ:BMRN) were flat in after-market trading after the company reported Q2 results. Quarterly Results Earnings per share rose 143.75% over the past year to $0.07, which beat the estimate of ($0.11).

Biomarin Pharmaceutical: Q2 Earnings Insights

SAN RAFAEL, Calif., July 28, 2021 /PRNewswire/ -- Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended June 30, Six Months Ended June 30, 2021 2020 % Change 2021 2020 % Change Total Revenues $ 501.7 $ 429.5 17 % $ 987.7 $ 931.6 6 % Net Product Revenues Marketed by BioMarin (1) $ 458.6 $ 386.8 19 % $ 876.3 $ 820.1 7 % Vimizim Net Product Revenues $ 171.7 $ 116.7 47 % $ 329.9 $ 253.9 30 % Naglazyme Net Product Revenues $ 118.8 $ 81.0 47 % $ 226.1 $ 195.3 16 % Kuvan Net Product Revenues $ 78.8 $ 122.6 (36) % $ 149.6 $ 244.6 (39) % Palynziq Net Product Revenues $ 59.0 $ 40.7 45 % $ 113.0 $ 75.3 50 % Brineura Net Product Revenues $ 30.3 $ 25.8 17 % $ 57.7 $ 49.8 16 % Aldurazyme Net Product Revenues $ 28.1 $ 32.3 (13) % $ 78.1 $ 88.0 (11) % GAAP Net Income (Loss) $ 12.9 $ (29.2) $ 30.3 $ 52.2 GAAP Net Income (Loss) per Share – Basic $ 0.07 $ (0.16) $ 0.17 $ 0.29 GAAP Net Income (Loss) per Share – Diluted $ 0.07 $ (0.16) $ 0.16 $ 0.28 Non-GAAP Income (2) $ 97.8 $ 57.4 $ 202.1 $ 173.9   June 30, 2021 December 31, 2020 Cash, cash equivalents and investments $ 1,473.6 $ 1,350.9 (1) Net Product Revenues Marketed by BioMarin is the sum of revenues from Vimizim, Kuvan, Naglazyme, Palynziq, Brineura for the three and six months ended June 30, 2021 and includes Firdapse for the six months ended June 30, 2020, each calculated in accordance with Generally Accepted Accounting Principles in the United States (U.S. GAAP).

BioMarin Announces Second Quarter 2021 Financial Results and Corporate Updates

BioMarin (BMRN) might move higher on growing optimism about its earnings prospects, which is reflected by its upgrade to a Zacks Rank #1 (Strong Buy).

BioMarin (BMRN) Upgraded to Strong Buy: Here's What You Should Know

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) has announced new data from open-label Phase 1/2 trial of valoctocogene roxaparvovec, an investigational gene therapy candidate for severe hemophilia A. Data were presented at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.

BioMarin Shares 5-Year Data From Open-Label Trial Of Hemophilia Gene Therapy

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

BioMarin Announces Oral Presentation at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress with 5 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec in Adults with Severe Hemophilia A, Demonstra

SAN RAFAEL, Calif., July 19, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced new data for valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, in its positive pivotal study, GENEr8-1, during an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.

BioMarin Announces Oral Presentation of Positive One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congres

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