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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BioMarin Leadership and External Medical Experts to Discuss 7 Product Candidates in Clinical Development as well as ROCTAVIAN and VOXZOGO New Indication Opportunities R&D Day to be Webcast Beginning at 8:00 a.m. Eastern Time September 12 th SAN RAFAEL, Calif.

BioMarin to Present Pipeline at Upcoming R&D Day on Tuesday, September 12th

First Person Treated in Germany; Reimbursement Discussions on Track in France and Italy In the U.S., ROCTAVIAN Now Commercially Available and Hemophilia Treatment Centers Have Begun Screening Eligible Individuals SAN RAFAEL, Calif. , Aug. 30, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that an individual in Germany with severe hemophilia A was treated with ROCTAVIAN ®  (valoctocogene roxaparvovec-rvox), marking the first time that the gene therapy has been given commercially in Europe.

BioMarin Announces First Person Treated Commercially with ROCTAVIAN® (valoctocogene roxaparvovec-rvox) for Severe Hemophilia A in Europe

BioMarin's 2Q23 revenue rose 12% YoY to $595 million, with adjusted EBITDA at $135 million. Revenues of the mucopolysaccharidosis drug portfolio decreased 6% YoY, but Voxzogo's quarterly sales were $113 million, up 29% QoQ. Voxzogo and ValRox remain the biggest BMRN's revenue drivers.

BioMarin: Q2 2023 Confirmed My Bullish View

BioMarin (BMRN) beats on earnings and sales estimates in the second quarter on the back of robust demand for Voxzogo.

BioMarin (BMRN) Q2 Earnings & Sales Top, Voxzogo Drives Sales

Although the revenue and EPS for BioMarin (BMRN) give a sense of how its business performed in the quarter ended June 2023, it might be worth considering how some key metrics compare with Wall Street estimates and the year-ago numbers.

BioMarin (BMRN) Q2 Earnings: Taking a Look at Key Metrics Versus Estimates

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 Richter - Goldman Sachs Geoffrey Meacham - BofA Securities Christopher Raymond - Piper Sandler Robyn Karnauskas - Truist Securities Phil Nadeau - TD Cowen Joseph Schwartz - Leerink Partners Paul Matteis - Stifel Gena Wang - Barclays Operator Thank you for joining BioMarin Second Quarterly Results Conference Call. Hosting today's call from BioMarin is Traci McCarty, Head of Investor Relations at BioMarin.

Biomarin Pharmaceutical, Inc. (BMRN) Q2 2023 Earnings Call Transcript

BioMarin Pharmaceutical (BMRN) came out with quarterly earnings of $0.54 per share, beating the Zacks Consensus Estimate of $0.47 per share. This compares to earnings of $0.59 per share a year ago.

BioMarin Pharmaceutical (BMRN) Tops Q2 Earnings and Revenue Estimates

VOXZOGO® Growth Continued in the Second Quarter Driven by Global Demand Resulting in Increased Full Year 2023 Guidance Pivotal Program with VOXZOGO in New, Potential Second Indication, Hypochondroplasia, to Begin in the Fourth Quarter of 2023 U.S. Approval of ROCTAVIAN™ Received in the Second Quarter and Commercial Launch Underway; Commercial Launch in Europe Making Progress Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended June 30, Six Months Ended June 30, 2023 2022 % Change 2023 2022 % Change Total Revenues $        595.3 $        533.8 12 % $     1,191.7 $    1,053.2 13 % Total Enzyme Product Revenues (1) $        420.8 $        425.7 (1) % $        868.8 $       852.3 2 % VIMIZIM® Net Product Revenues $        177.4 $        173.3 2 % $        366.6 $       356.4 3 % VOXZOGO® Net Product Revenues $        113.3 $          34.4 229 % $        201.2 $         54.0 273 % NAGLAZYME® Net Product Revenues $          90.1 $        115.8 (22) % $        213.1 $       243.8 (13) % PALYNZIQ® Net Product Revenues $          74.9 $          61.6 22 % $        137.2 $       116.5 18 % KUVAN® Net Product Revenues $          50.6 $          57.6 (12) % $        101.1 $       116.9 (14) % ALDURAZYME® Net Product Revenues $          40.3 $          37.3 8 % $          74.7 $         61.7 21 % BRINEURA® Net Product Revenues $          38.1 $          37.7 1 % $          77.2 $         73.9 4 % GAAP Net Income (2) $          56.0 $          27.7 $        106.9 $       148.5 Non-GAAP Income (3) $        105.2 $          76.8 $        221.0 $       175.7 GAAP Diluted Earnings per Share (EPS) $          0.29 $          0.15 $          0.56 $         0.79 Non-GAAP Diluted EPS (4) $          0.54 $          0.41 $          1.14 $         0.93   June 30, 2023 December 31, 2022 Total cash, cash equivalents & investments $               1,556.7 $               1,625.4 (1) Enzyme-based products include ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ, and VIMIZIM. (2) GAAP Net income in the first half of 2022 included a $89.0 million gain, net of taxes, related to the sale of the Rare Pediatric DiseasePriority Review Voucher (PRV) the Company received from the U.S. Food and Drug Administration (FDA) in connection with U.S. approvalof VOXZOGO.

BioMarin Announces Strong Second Quarter 2023 Results and Record Breaking Revenues for the First Half of 2023, Including 13% Year Over Year Growth Year-to-date

BioMarin's stocks have underperformed the S&P500 index recently due to the investor rotation from defensive sectors to riskier ones and potential slowdown in M&A activity in the biotechnology sector. Despite this, BioMarin's business remains attractive with approvals for its two flagship drugs, Voxzogo and Roctavian, and potential inclusion in the S&P500 index could lead to a significant increase in stock price. I remain my bullish view on BMRN's shares with fair value of $118.

BioMarin: More Growth Is Coming

SAN RAFAEL, Calif. , July 12, 2023 /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 Monday, July 31, at 4:30 p.m.

BioMarin to Host Second-Quarter 2023 Financial Results Conference Call and Webcast on Monday, July 31, at 4:30pm ET

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BioMarin NASDAQ: BMRN shares have been under pressure due to delays in approving its hemophilia A drug Roctavian. That is over.

Is BioMarin Ready To Rebound After FDA Approval Of Roctavian?

BioMarin Pharmaceutical Inc. received FDA approval for ROCTAVIAN for adults with severe Hemophilia A. Net product revenues of VOXZOGO grew by 346% year-over-year to $87.8 million. In the coming months U.S. and European Union will decide if VOXZOGO should be expanded to treat another 1,000 achondroplasia patients.

BioMarin: FDA Approval Of ROCTAVIAN And Massive VOXZOGO Sales Increase

Following FDA's approval, BioMarin's (BMRN) Roctavian becomes the first gene therapy for treating severe hemophilia A.

BioMarin (BMRN) Gets FDA Nod for Hemophilia A Gene Therapy

BioMarin Pharmaceutical Inc. BMRN, -3.57% received Food and Drug Administration approval of its gene therapy Roctavian to treat hemophilia A, the agency said late Thursday. The FDA said Roctavian was the first gene therapy to treat adults with severe hemophilia A, a serious genetic disorder that affects the gene that makes the protein that casues blood to clot, increasing the risk of uncontrolled bleeding.

FDA approves BioMarin's gene therapy drug for hemophilia

The U.S. Food and Drug Administration on Thursday approved BioMarin Pharmaceutical's gene therapy for hemophilia A, the company said, giving patients with the inherited bleeding disorder an option to reduce regular blood infusions.

US FDA approves BioMarin's gene therapy for hemophilia A

ROCTAVIAN is a One-Time, Single-Dose Treatment Available for Adults with Severe Hemophilia A to Control Bleeds ROCTAVIAN's Approval was Based on Durability, Efficacy and Safety Results from the Largest and Longest Phase 3 Study for a Gene Therapy for Hemophilia Majority of Patients Continued to Respond to ROCTAVIAN Treatment over 3 Years Conference Call and Webcast to be Held at 4:30 p.m. Eastern Time on Thursday, June 29, 2023  SAN RAFAEL, Calif.

U.S. Food and Drug Administration Approves BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec-rvox), the First and Only Gene Therapy for Adults with Severe Hemophilia A

A major catalyst may arrive tomorrow for BioMarin as the FDA announces whether it will approve hemophilia gene therapy Roctavian. The drug is already approved in Europe, although pricing disputes are hindering sales. That ought not to be a problem in the US. BioMarin generated >$2bn of revenues in 2022 and is guiding for ~$2.4bn in 2023. Newly approved achondroplasia therapy VOXZOGO is expected to become a blockbuster.

BioMarin: Decision Day For Hemophilia Gene Therapy Upcoming - Approval Likely

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New Data to be Presented for ROCTAVIAN from Phase 3 GENEr8-1 Study, Including Data on the Impact of ROCTAVIAN on Quality of Life and M usculoskeletal Health  for Adults with Severe Hemophilia A SAN RAFAEL, Calif. , June 22, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced it will present new data from the ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy clinical trial program.

BioMarin to Present ROCTAVIAN™ (valoctocogene roxaparvovec) Data from Longest and Largest Hemophilia Gene Therapy Clinical Trial Program at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress

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

Why Is BioMarin (BMRN) Down 4% Since Last Earnings Report?

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