IONS Ionis Pharmaceuticals

For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Its scientific innovation began and continues with the knowledge that sick people depend on it, which fuels its vision of becoming one of the most successful biotechnology companies.

Company profile

Brett Monia
Fiscal year end
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5 May 21
3 Aug 21
31 Dec 21
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Financial data from company earnings reports.

Cash burn rate (estimated) Burn method: Change in cash Burn method: Operating income/loss Burn method: FCF (opex + capex)
Last Q Avg 4Q Last Q Avg 4Q Last Q Avg 4Q
Cash on hand (at last report) 414.16M 414.16M 414.16M 414.16M 414.16M 414.16M
Cash burn (monthly) (positive/no burn) 6M 30.66M 16.91M 18.22M 931.5K
Cash used (since last report) n/a 24.82M 126.73M 69.9M 75.32M 3.85M
Cash remaining n/a 389.34M 287.43M 344.25M 338.84M 410.3M
Runway (months of cash) n/a 64.9 9.4 20.4 18.6 440.5

Beta Read what these cash burn values mean

Date Owner Security Transaction Code Indirect 10b5-1 $Price #Shares $Value #Remaining
15 Jul 21 Hayden Michael R Common Stock Option exercise Aquire M No No 0 1,778 0 12,445
15 Jul 21 Hayden Michael R Common Stock Option exercise Aquire M No No 0 5,333 0 10,667
15 Jul 21 Hayden Michael R RSU Common Stock Option exercise Dispose M No No 0 1,778 0 12,441
15 Jul 21 Hayden Michael R RSU Common Stock Option exercise Dispose M No No 0 5,333 0 14,219
15 Jul 21 Allene M. Diaz RSU Common Stock Grant Aquire A No No 0 8,000 0 8,000
15 Jul 21 Berthelsen Spencer R Common Stock Option exercise Aquire M No No 0 1,778 0 109,649
15 Jul 21 Berthelsen Spencer R Common Stock Option exercise Aquire M No No 0 666 0 107,871
15 Jul 21 Berthelsen Spencer R Common Stock Option exercise Aquire M No No 0 889 0 107,205
15 Jul 21 Berthelsen Spencer R Common Stock Option exercise Aquire M No No 0 5,333 0 106,316
15 Jul 21 Berthelsen Spencer R RSU Common Stock Option exercise Dispose M No No 0 1,778 0 9,775

Data for the last complete 13F reporting period. To see the most recent changes to ownership, click the ownership history button above.

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Financial report summary

  • Our business could be materially adversely affected by the effects of health epidemics. To date, we believe the impacts of the recent COVID-19 pandemic on our business are limited and manageable.
  • We have limited experience as a company in commercializing medicines, and we may have to invest significant resources to develop these capabilities. If we are unable to establish effective marketing, sales and distribution capabilities or enter into agreements with third parties to market, sell and distribute our medicines, we may not be able to generate revenue from our medicines.
  • If the market does not accept our medicines, including SPINRAZA, TEGSEDI and WAYLIVRA, and our medicines in development, we are not likely to generate substantial revenues or become consistently profitable.
  • If we or our partners fail to compete effectively, our medicines, including SPINRAZA, TEGSEDI and WAYLIVRA, and our medicines in development, will not contribute significant revenues.*
  • Certain of our medicines may compete with our other medicines, which could reduce our expected revenues.
  • Our medicines could be subject to regulatory limitations following approval.*
  • We depend on our collaboration with Biogen for the development and commercialization of SPINRAZA.
  • We are relying on third parties to market, sell and distribute TEGSEDI and WAYLIVRA.*
  • Our operations are subject to additional healthcare laws.
  • If government or other third-party payers fail to provide adequate coverage and payment rates for our medicines, including SPINRAZA, TEGSEDI and WAYLIVRA, and our medicines in development, our revenue will be limited.
  • If we cannot manufacture our medicines or contract with a third party to manufacture our medicines at costs that allow us to charge competitive prices to buyers, we cannot market our products profitably.
  • If we or our partners fail to obtain regulatory approval for our medicines and additional approvals for SPINRAZA, TEGSEDI and WAYLIVRA, we or our partners cannot sell them in the applicable markets.
  • If the results of clinical testing indicate that any of our medicines are not suitable for commercial use, we may need to abandon one or more of our drug development programs.*
  • Even if our medicines are successful in preclinical and human clinical studies, the medicines may not be successful in late-stage clinical studies.*
  • We depend on third parties to conduct our clinical studies for our medicines and any failure of those parties to fulfill their obligations could adversely affect our development and commercialization plans.*
  • Since corporate partnering is a significant part of our strategy to fund the advancement and commercialization of our development programs, if any of our collaborative partners fail to fund our collaborative programs, or if we cannot obtain additional partners, we may have to delay or stop progress on our drug development programs.*
  • Even with funding from corporate partners, if our partners do not effectively perform their obligations under our agreements with them, it would delay or stop the progress of our drug development and commercial programs.
  • If we do not progress in our programs as anticipated, the price of our securities could decrease.*
  • We have incurred losses, and our business will suffer if we fail to consistently achieve profitability in the future.
  • If we fail to obtain timely funding, we may need to curtail or abandon some of our programs.
  • If we cannot protect our patent rights or our other proprietary rights, others may compete more effectively against us.
  • Intellectual property litigation could be expensive and prevent us from pursuing our programs.
  • If we fail to successfully integrate Akcea’s business and operations, it may adversely affect our future results.
  • If our management transition is not successful our business could suffer.
  • The loss of key personnel, or the inability to attract and retain highly skilled personnel, could make it more difficult to run our business and reduce our likelihood of success.
  • Our ability to use our net operating loss carryovers and certain other tax attributes may be limited.*
  • Our future taxable income could be impacted by changes in tax laws, regulations and treaties.
  • We could be subject to additional tax liabilities.
  • If the price of our securities continues to be highly volatile, this could make it harder for you to liquidate your investment and could increase your risk of suffering a loss.*
  • Provisions in our certificate of incorporation, convertible notes documents, call spread hedge transaction documents and Delaware law may prevent stockholders from receiving a premium for their shares.
  • Future sales of our common stock in the public market could adversely affect the trading price of our securities.
  • We are exposed to potential product liability claims, and insurance against these claims may not be available to us at a reasonable rate in the future or at all.
  • We are dependent on information technology systems, infrastructure and data, which exposes us to data security risks.
  • Because we use biological materials, hazardous materials, chemicals and radioactive compounds, if we do not comply with laws regulating the protection of the environment and health and human safety, our business could be adversely affected.
  • If a natural or man-made disaster strikes our research, development or manufacturing facilities or otherwise affects our business, it could delay our progress developing and commercializing our medicines.
  • Our business is subject to changing regulations for corporate governance and public disclosure that has increased both our costs and the risk of noncompliance.
  • Negative conditions in the global credit markets and financial services and other industries may adversely affect our business.
  • The impact on us of the vote by the United Kingdom to leave the European Union cannot be predicted.
Management Discussion
  • In this Report on Form 10-Q, unless the context requires otherwise, “Ionis,” “Company,” “we,” “our,” and “us,” means Ionis Pharmaceuticals, Inc. and its wholly owned affiliate, Akcea Therapeutics, Inc.
  • In addition to historical information contained in this Report on Form 10-Q, the Report includes forward-looking statements regarding our business and the therapeutic and commercial potential of SPINRAZA (nusinersen), TEGSEDI (inotersen), WAYLIVRA (volanesorsen) and our technologies and products in development. Any statement describing our goals, expectations, financial or other projections, intentions or beliefs, is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including those related to the impact COVID-19 could have on our business, and including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Our forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause our results to differ materially from those expressed or implied by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in this report and described in additional detail in our annual report on Form 10-K for the year ended December 31, 2020, which is on file with the U.S. Securities and Exchange Commission and is available from us, and those identified within Part II Item 1A. Risk Factors of this Report. Although our forward-looking statements reflect the good faith judgment of our management, these statements are based only on facts and factors currently known by us. As a result, you are cautioned not to rely on these forward-looking statements.
  • We are a leader in RNA-targeted therapy and believe our medicines are pioneering new markets, changing standards of care and transforming the lives of people with devastating diseases. Our clinical pipeline of potential first-in-class and/or best-in-class medicines address a broad range of diseases. We are primarily focused on two core franchises: neurology and cardiometabolic. Our commercial products SPINRAZA, TEGSEDI and WAYLIVRA, are approved in major markets around the world. Within our late-stage pipeline, we have six Phase 3 studies ongoing with five medicines: tofersen for SOD1-ALS, IONIS-TTR-LRx for transthyretin, or TTR, amyloidosis, IONIS-APOCIII-LRx for familial chylomicronemia syndrome, or FCS, pelacarsen for lipoprotein(a), or Lp(a), driven cardiovascular disease and ION363 for amyotrophic lateral sclerosis, or ALS, with mutations in the fused in sarcoma gene, or FUS.
Content analysis
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