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Biomarin Pharmaceutical Inc. - Registered Shares (BMRN)

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.

Company profile

Ticker
BMRN
Exchange
CEO
Jean-Jacques Bienaime
Employees
Incorporated
Location
Fiscal year end
SEC CIK
Subsidiaries
BioMarin Commercial Ltd • BioMarin International Ltd ...
IRS number
680397820

BMRN stock data

Press releases

From Benzinga Pro
BioMarin Announces Record Revenues in Second Quarter 2022; Increases Full-year 2022 Top and Bottom-line Guidance
3 Aug 22
VOXZOGO™ $34 Million Contribution Drives Total Revenues of $534 Million in the Quarter; Total Revenues Grew 13% Year-Over-Year, Excluding KuvanBioMarin Expects that ROCTAVIAN™ (valoctocogene roxaparvovec)
BioMarin to Host Second Quarter 2022 Financial Results Conference Call and Webcast on Wednesday, August 3, at 4:30pm ET
20 Jul 22
SAN RAFAEL, Calif., July 20, 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
BioMarin Presents Findings from Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 Congress July 9-12, Including 4 Oral and 2 Poster Presentations
11 Jul 22
Commitment to Advancing Care for People with Hemophilia A Demonstrated with Largest and Longest, Ongoing Clinical Development Program for any Gene Therapy in Hemophilia A European Commission Approval for Valoctocogene
BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A
24 Jun 22
BioMarin Announces the Ministry of Health, Labor and Welfare (MHLW) in Japan Granted Approval for VOXZOGO® (vosoritide) for Injection for the Treatment of Children with Achondroplasia, Whose Growth Plates are Not Closed
21 Jun 22

Calendar

4 Aug 22
12 Aug 22
31 Dec 22
Quarter (USD) Jun 22 Mar 22 Dec 21 Sep 21
Revenue
Cost of revenue
Operating income
Operating margin
Net income
Net profit margin
Cash on hand
Change in cash
Diluted EPS
Annual (USD) Dec 21 Dec 20 Dec 19 Dec 18
Revenue
Cost of revenue
Operating income
Operating margin
Net income
Net profit margin
Cash on hand
Change in cash
Diluted EPS
Cash burn rate (est.) Burn method: Change in cash Burn method: Operating income Burn method: FCF (opex + capex)
Last Q Avg 4Q Last Q Avg 4Q Last Q Avg 4Q
Cash on hand (at last report) 619.8M 619.8M 619.8M 619.8M 619.8M 619.8M
Cash burn (monthly) (no burn) 1.81M (no burn) (no burn) (no burn) (no burn)
Cash used (since last report) n/a 2.62M n/a n/a n/a n/a
Cash remaining n/a 617.18M n/a n/a n/a n/a
Runway (months of cash) n/a 340.8 n/a n/a n/a n/a

Beta Read what these cash burn values mean

Date Owner Security Transaction Code Indirect 10b5-1 $Price #Shares $Value #Remaining
9 Aug 22 George Eric Davis Common Stock Sell Dispose S No No 95.93 47,526 4.56M 55,251
9 Aug 22 George Eric Davis Common Stock Option exercise Acquire M No No 67.81 47,526 3.22M 102,777
9 Aug 22 George Eric Davis Common Stock Sell Dispose S No No 96.34 3,690 355.49K 55,251
9 Aug 22 George Eric Davis Stock Option (Right to buy Common Stock) Common Stock Option exercise Dispose M No No 67.81 47,526 3.22M 0
8 Aug 22 Charles Greg Guyer Common Stock Sell Dispose S No No 96.56 6,205 599.15K 42,168
5 Aug 22 Mueller Brian Common Stock Sell Dispose S No Yes 94.5 3,500 330.75K 30,726
5 Aug 22 Mueller Brian Common Stock Option exercise Acquire M No Yes 67.81 3,500 237.34K 34,226
5 Aug 22 Mueller Brian Stock Option (Right to buy Common Stock) Common Stock Option exercise Dispose M No Yes 67.81 3,500 237.34K 7,261
8 Jul 22 George Eric Davis Common Stock Sell Dispose S No Yes 90 7,398 665.82K 58,941
8 Jul 22 Mueller Brian Common Stock Sell Dispose S No Yes 89.5 7,337 656.66K 30,726
99.4% owned by funds/institutions
13F holders Current Prev Q Change
Total holders 540 541 -0.2%
Opened positions 78 103 -24.3%
Closed positions 79 55 +43.6%
Increased positions 188 179 +5.0%
Reduced positions 187 172 +8.7%
13F shares Current Prev Q Change
Total value 27.89B 16.43B +69.7%
Total shares 184.42M 186.35M -1.0%
Total puts 809.61K 1.16M -30.5%
Total calls 1.41M 1.77M -20.3%
Total put/call ratio 0.6 0.7 -12.7%
Largest owners Shares Value Change
Primecap Management 17.78M $1.37B -0.1%
Vanguard 16.5M $1.27B +0.9%
Dodge & Cox 15.03M $1.16B +1.4%
BLK Blackrock 13.96M $1.08B +0.4%
CAFBX Amcap Fund 10.18M $866.57M 0.0%
Capital Research Global Investors 9.39M $723.83M -25.9%
Baker Bros. Advisors 7.62M $587.54M 0.0%
STT State Street 5.38M $414.83M +1.2%
AMP Ameriprise Financial 5.34M $411.75M -18.5%
JHG Janus Henderson 4.36M $336.14M +12.0%
Largest transactions Shares Bought/sold Change
Capital Research Global Investors 9.39M -3.28M -25.9%
Clearbridge Advisors 2.11M -3.15M -59.9%
UBS UBS Group AG - Registered Shares 2.86M +2.7M +1645.0%
Norges Bank 0 -2.35M EXIT
AMP Ameriprise Financial 5.34M -1.21M -18.5%
Perceptive Advisors 0 -1.18M EXIT
Viking Global Investors 3.75M +882.45K +30.7%
Orbimed Advisors 1.65M +786.15K +91.3%
Citadel Advisors 2.09M +732.49K +53.9%
HealthCor Management 769.02K +609.7K +382.7%

Financial report summary

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Risks
  • The COVID-19 pandemic could continue to materially adversely affect our business, results of operations, and financial condition.
  • Because the target patient populations for our products are small, we must achieve significant market share and maintain high per-patient prices for our products to achieve and maintain profitability.
  • If we fail to obtain and maintain an adequate level of coverage and reimbursement for our products by third-party payers, the sales of our products would be adversely affected or there may be no commercially viable markets for our products.
  • If we fail to compete successfully with respect to product sales, we may be unable to generate sufficient sales to recover our expenses related to the development of a product program or to justify continued marketing of a product and our revenues could be adversely affected.
  • Changes in methods of treatment of disease could reduce demand for our products and adversely affect revenues.
  • If we fail to develop new products and product candidates or compete successfully with respect to acquisitions, joint ventures, licenses or other collaboration opportunities, our ability to continue to expand our product pipeline and our growth and development would be impaired.
  • The sale of generic versions of Kuvan by generic manufacturers has adversely affected and will continue to adversely affect our revenues and may cause a decline in Kuvan revenues faster than expected.
  • If we do not achieve our projected development goals in the timeframes we announce and expect, the commercialization of our product candidates may be delayed and the credibility of our management may be adversely affected and, as a result, our stock price may decline.
  • We have in the past and may in the future enter into licensing arrangements, and we may not realize the benefits of such licensing arrangements.
  • If we fail to obtain regulatory approval to commercially market and sell our product candidates, or if approval of our product candidates is delayed, we will be unable to generate revenues from the sale of these product candidates, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will increase.
  • Any product for which we have obtained regulatory approval, or for which we obtain approval in the future, is subject to, or will be subject to, extensive ongoing regulatory requirements by the FDA, the EMA and other comparable international regulatory authorities, and if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products, we may be subject to penalties, we will be unable to generate revenues from the sale of such products, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will be increased.
  • To obtain regulatory approval to market our products, preclinical studies and costly and lengthy clinical trials are required and the results of the studies and trials are highly uncertain. Likewise, preliminary, initial or interim data from
  • clinical trials should be considered carefully and with caution because the final data may be materially different from the preliminary, initial or interim data, particularly as more patient data become available.
  • Government price controls or other changes in pricing regulation could restrict the amount that we are able to charge for our current and future products, which would adversely affect our revenues and results of operations.
  • Government healthcare reform could increase our costs and adversely affect our revenues and results of operations.
  • If we fail to obtain or maintain orphan drug exclusivity for some of our products, our competitors may obtain approval to sell the same drugs to treat the same conditions and our revenues will be reduced.
  • We may face competition from biosimilars approved through an abbreviated regulatory pathway.
  • Changes in funding for the FDA, the EMA, other comparable international regulatory authorities and other government agencies or government shutdowns could hinder the ability of such agencies to hire and retain key leadership and other personnel or otherwise prevent those agencies from performing normal functions on which the operation of our business may rely, which could negatively impact our business.
  • Our valoctocogene roxaparvovec program is based on a gene therapy approach, which, as a novel technology, presents additional development and treatment risks in relation to our other, more traditional drug development programs.
  • As compared to our other, more traditional products, our gene therapy product candidate valoctocogene roxaparvovec, if approved, may present additional problems with respect to the pricing, coverage, and reimbursement and acceptance of the product candidate.
  • We have implemented data access plans for our main clinical trials of valoctocogene roxaparvovec, which restrict our management’s review of emerging key efficacy data from these trials. Without access to this ongoing data, management does not have the ability to adjust the trials based on such emerging data, which could adversely impact the ultimate outcome of these trials.
  • If we continue to incur operating losses or are unable to sustain positive cash flows for a period longer than anticipated, we may be unable to continue our operations at planned levels and be forced to reduce our operations.
  • If we fail to obtain the capital necessary to fund our operations, our financial results and financial condition will be adversely affected and we will have to delay or terminate some or all of our product development programs.
  • We have incurred substantial indebtedness that may decrease our business flexibility, access to capital, and/or increase our borrowing costs, which may adversely affect our operations and financial results.
  • In addition, our ability to refinance our indebtedness will depend on the capital markets and our financial condition at such time.
  • If we fail to comply with manufacturing regulations, our financial results and financial condition will be adversely affected.
  • If we are unable to successfully develop and maintain manufacturing processes for our product candidates to produce sufficient quantities at acceptable costs, we may be unable to support a clinical trial or be forced to terminate a program, or if we are unable to produce sufficient quantities of our products at acceptable costs, we may be unable to meet commercial demand, lose potential revenue, have reduced margins or be forced to terminate a program.
  • Supply interruptions may disrupt our inventory levels and the availability of our products and product candidates and cause delays in obtaining regulatory approval for our product candidates, or harm our business by reducing our revenues.
  • If our Manufacturing, Marketing and Sales Agreement with Sanofi were terminated, we could be prevented from continuing to commercialize Aldurazyme or our ability to successfully commercialize Aldurazyme would be delayed or diminished.
  • We conduct a significant amount of our sales and operations outside of the U.S., which subjects us to additional business risks that could adversely affect our revenues and results of operations.
  • A significant portion of our international sales are made based on special access programs, and changes to these programs could adversely affect our product sales and revenues in these countries.
  • U.S. export control and economic sanctions may adversely affect our business, financial condition and operating results. Moreover, compliance with such regulatory requirements may increase our costs and negatively impact our ability to sell our products and collect cash from customers.
  • Failure to comply with applicable anti-corruption legislation could result in fines, criminal penalties and materially adversely affect our business, financial condition and results of operations.
  • Our international operations pose currency risks, which may adversely affect our operating results and net income.
  • We face credit risks from government-owned or sponsored customers outside of the U.S. that may adversely affect our results of operations.
  • If we are unable to protect our intellectual property, we may not be able to compete effectively or preserve our market shares.
  • Competitors and other third parties may have developed intellectual property that could limit our ability to market and commercialize our products and product candidates, if approved.
  • Our stock price has been and may in the future be volatile, and an investment in our stock could suffer a decline in value.
  • Conversion of the Notes will dilute the ownership interest of existing stockholders, including holders who had previously converted their Notes, or may otherwise depress the price of our common stock.
  • Anti-takeover provisions in our charter documents and under Delaware law may make an acquisition of us, which may be beneficial to our stockholders, more difficult.
  • The fundamental change repurchase feature of the Notes may delay or prevent an otherwise beneficial attempt to take us over.
  • Our amended and restated bylaws designate the Court of Chancery of the State of Delaware and the federal district courts of the U.S. as the exclusive forums for the adjudication of certain disputes, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors, officers, or employees.
  • We depend upon our key personnel and our ability to attract and retain qualified employees.
  • Our success depends on our ability to manage our growth.
  • New tax laws or regulations that are enacted or existing tax laws and regulations that are interpreted, modified or applied adversely to us or our customers may have a material adverse effect on our business and financial condition.
  • If we are found in violation of healthcare laws or privacy and data protection laws, we may be required to pay penalties, be subjected to scrutiny by regulators or governmental entities, or be suspended from participation in government healthcare programs, which may adversely affect our business, reputation, financial condition and results of operations.
  • If product liability lawsuits are successfully brought against us, we may incur substantial liabilities.
  • We rely significantly on information technology systems and any failure, inadequacy, interruption or security lapse of that technology, including any cybersecurity incidents, could harm our ability to operate our business effectively and have a material adverse effect on our business, reputation, financial condition, and results of operations.
  • If a natural disaster, terrorist or criminal activity or other unforeseen event caused significant damage to our facilities or those of our third-party manufacturers and suppliers or significantly disrupted our operations or those of our third-party manufacturers and suppliers, we may be unable to meet demand for our products and lose potential revenue, have reduced margins, or be forced to terminate a program.
  • Our business is affected by macroeconomic conditions.
Management Discussion
  • Item 2.    Management’s Discussion and Analysis of Financial Condition and Results of Operations
  • and life-threatening rare diseases and medical conditions. We select product candidates for diseases and conditions that represent
  • significant benefit over existing products.

Content analysis

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Positive
Negative
Uncertain
Constraining
Legalese
Litigous
Readability
H.S. junior Avg
New words: alleviate, compression, construed, cord, Cycle, ENDO, Endocrine, endpoint, feedback, hemostatic, imaging, interindividual, intermediate, interventional, Japanese, longest, magnetic, MRI, multinational, nonqualified, opposition, permission, reply, resonance, secondary, skull, spinal, surgery, threshold, Yen
Removed: addressing, Aid, aim, deductibility, favor, informally, newly, NQDC, Relief, repealed, TCJA, utilization

Patents

Utility
Targeted Therapeutic Lysosomal Enzyme Fusion Proteins, Associated Formulations and Uses Thereof
11 Aug 22
The present disclosure relates in general to therapeutic lysosomal enzyme fusion proteins useful for treating lyso-somal storage diseases, liquid formulations comprising such fusion proteins and associated methods useful for treating lysosomal storage diseases in mammals.
Utility
Adeno-associated virus factor VIII vectors
9 Aug 22
The present disclosure provides methods of generating multiplexed genetically modified animals, for example, porcine endogenous retrovirus (PERV)-inactivated pigs.
Utility
Glycolate oxidase inhibitors for the treatment of disease
12 Jul 22
Described herein are compounds, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or disorders associated with the enzyme glycolate oxidase (GO).
Utility
N-(1-hydroxy-3-(pyrrolidinyl)propan-2-yl)pyrrolidine-3-carboxamide derivatives as glucosylceramide synthase inhibitors
14 Jun 22
Described herein are compounds of Formula I, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or conditions associated with the enzyme glucosylceramide synthase (GCS).
Utility
Lysosomal targeting peptides and uses thereof
7 Jun 22
The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology.