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IONIS PHARMACEUTICALS INC

For the period ended Mar 31, 2026. Filed Apr 29, 2026.

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About this filing

A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.

Mar 31, 2026 · Filed Apr 29, 2026

IONS$39.42+1.6%Stored close · as of Nov 6, 2026

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Notes to Condensed Consolidated Financial Statements (unaudited

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

March 31, 2026

  1. Organization and Basis of Presentation

Organization and Business Activity

We incorporated in California on January 10, 1989. In conjunction with our initial public offering, we reorganized as a Delaware corporation in April 1991. We are a fully integrated commercial-stage biotechnology company and a leader in the discovery and development of RNA-targeted therapeutics.

Basis of Presentation

We prepared the unaudited interim condensed consolidated financial statements for the three months ended March 31, 2026 and 2025 on the same basis as the audited financial statements for the year ended December 31, 2025. We included all normal recurring adjustments in the financial statements, which we considered necessary for a fair presentation of our financial position at such dates and our operating results and cash flows for those periods. Our operating results for the interim periods may not be indicative of what our operating results will be for the entire year. For more complete financial information, these financial statements, and notes thereto, should be read in conjunction with the audited financial statements for the year ended December 31, 2025 included in our Annual Report on Form 10-K filed with the Securities and Exchange Commission, or SEC.

In our condensed consolidated financial statements, we included the accounts of Ionis Pharmaceuticals, Inc. and the consolidated results of its wholly owned subsidiaries (“we”, “us” or “our”).

We operate as asingle segment, Ionis operations, because our chief operating decision maker, or CODM, reviews operating results on an aggregate basis and manages our operations as asingle operating segment. Refer to Note 13, Segment Information, for further details on our segment information.

Use of Estimates

We prepare our condensed consolidated financial statements in conformity with accounting principles generally accepted in the United States, or U.S., that require us to make estimates and assumptions that affect the amounts reported in our condensed consolidated financial statements and accompanying notes. Actual results could differ from our estimates.

  1. Significant Accounting Policies

Our significant accounting policies have not changed substantially from those included in our Annual Report on Form 10-K for the year ended December 31, 2025.

Recent Accounting Standards

In July 2025, the FASB issued ASU 2025-05, which amended the guidance in ASC 326 to simplify the estimation of credit losses on accounts receivable and contract assets from revenue transactions. The amended guidance allows companies to elect a practical expedient to assume that conditions as of the balance sheet date will remain unchanged for the remaining life of the asset when estimating the expected credit losses of the asset. This update is effective for annual periods beginning after December 15, 2025 and interim periods within those annual periods. Companies that elect the practical expedient are required to apply the amendments prospectively. We adopted this update in the first quarter of 2026 on a prospective basis and elected the practical expedient. The updated guidance did not have a material impact on our condensed consolidated financial statements.

In September 2025, the FASB issued ASU 2025-06, which amended and simplified the existing guidance for software costs. The amended guidance removes references to software development stages and allows companies to begin capitalizing software costs when management has authorized and committed to funding the software project and it is probable that the project will be completed with the software performing the intended function. This update is effective for annual periods beginning after December 15, 2027 and interim periods within those annual periods. Early adoption of this guidance is permitted at the beginning of an annual reporting period. The guidance may be applied on a prospective or retrospective basis. We early adopted this update in the first quarter of 2026 on a prospective basis. The updated guidance did not have a material impact on our condensed consolidated financial statements.

We do not expect any recently issued accounting standards other than the standards mentioned above and those included in our Annual Report on Form 10-K for the year ended December 31, 2025 to have a material impact to our financial results.

  1. Supplemental Financial Data

Inventories

Our inventories consisted of the following (in thousands):

March 31, 2026December 31, 2025
Raw materials$490$1,039
Work in process24,67824,051
Finished goods9901,096
Total$26,158$26,186
Reported as:
Inventories$5,557$10,048
Deposits and other assets20,60116,138
Total$26,158$26,186

We classify inventories as non-current assets when we expect the inventories to remain on hand beyond one year. We include non-current inventories in deposits and other assets in our condensed consolidated balance sheets. The amounts reported as deposits and other assets as of March 31, 2026 and December 31, 2025 consist of work in process inventory.

Accrued Liabilities

Our accrued liabilities consisted of the following (in thousands):

March 31, 2026December 31, 2025
Clinical expenses$53,325$53,659
In-licensing expenses6,6558,588
Commercial expenses, including gross-to-net product accruals40,46815,556
Other miscellaneous expenses26,10128,286
Total accrued liabilities$126,549$106,089
  1. Revenues

During the three months ended March 31, 2026 and 2025, our revenues consisted of the following (in thousands):

Three Months Ended
March 31,
20262025
Revenue:
Commercial revenue:
Product sales, net:
TRYNGOLZA sales, net$27,100$6,287
DAWNZERA sales, net15,855-
Total product sales, net42,9556,287
Royalty revenue:
SPINRAZA royalties43,71048,010
WAINUA royalties10,7099,372
Other royalties3,9046,784
Total royalty revenue58,32364,166
Other commercial revenue6,5325,715
Total commercial revenue107,81076,168
Research and development revenue:
Collaborative agreement revenue120,66045,031
WAINUA joint development revenue17,62110,413
Total research and development revenue138,28155,444
Total revenue$246,091$131,612

Revenue Sources

The following are sources of revenue and when we typically recognize revenue.

Commercial Revenue

In December 2024, the U.S. Food and Drug Administration, or FDA, approved TRYNGOLZA for the treatment of familial chylomicronemia syndrome, or FCS. Following the approval, we launched TRYNGOLZA and began earning revenue from TRYNGOLZA sales.

In August 2025, the FDA approved DAWNZERA for prophylaxis to prevent attacks of hereditary angioedema, or HAE, in adult and pediatric patients 12 years of age and older. Following the approval, we launched DAWNZERA and began earning revenue from DAWNZERA sales.

We earn royalty payments primarily on net sales of SPINRAZA, WAINUA and QALSODY. We include QALSODY royalties within other royalties in the table above.

We earn commercial revenue from TEGSEDI and WAYLIVRA sales under our distribution agreements with Swedish Orphan Biovitrum AB, or Sobi. In addition, we receive royalties from PTC Therapeutics International Limited, or PTC, for TEGSEDI and WAYLIVRA sales. Refer to Part IV, Item 15, Note 4, Collaborative Arrangements and Licensing Agreements, of our audited financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2025 for details on our commercialization partnerships with Sobi and PTC.

Under our distribution agreements with Sobi, we concluded that our performance obligation is to provide services to Sobi over the term of the agreement, which includes supplying finished goods inventory to Sobi. We are also responsible for maintaining the marketing authorization for TEGSEDI and WAYLIVRA in major markets and for leading the global commercial strategy for each medicine. We view this performance obligation as a series of distinct activities that are substantially the same. We recognize as revenue the price Sobi pays us for the inventory when we deliver the finished goods inventory to Sobi. We also recognize distribution fee revenue based on Sobi’s net sales of TEGSEDI and WAYLIVRA. Under our agreements with Sobi, Sobi does not generally have a right of return.

Research and development revenue under collaboration agreements

We enter into collaboration agreements to license and sell our technology on an exclusive or non-exclusive basis. Our collaboration agreements typically contain multiple elements, or performance obligations, including technology licenses or options to obtain technology licenses, R&D services and manufacturing services.

For R&D services that we recognize over time, we measure our progress using an input method. The input methods we use are based on the effort we expend or costs we incur toward the satisfaction of our performance obligation. We estimate the amount of effort we expend, including the time we estimate it will take us to complete the activities, or costs we incur in a given period, relative to the estimated total effort or costs to satisfy the performance obligation. This results in a percentage that we multiply by the transaction price to determine the amount of revenue we recognize each period. This approach requires us to make numerous estimates and use judgement. If our estimates or judgements change over the course of the collaboration, they may affect the timing and amount of revenue that we recognize in the current and future periods.

Upfront payments: When we enter into a collaboration agreement and receive an upfront payment, we record the entire upfront payment as deferred revenue if our only performance obligation is for R&D services we will provide in the future. We amortize the upfront payment into revenue as we perform the R&D services. If part or all of the upfront payment is a license fee, we recognize as revenue the portion related to the license when we deliver the license to our partner because our partner has full use of the license and we do not have any additional performance obligations related to the license after delivery.

Milestone payments: We include variable consideration in the transaction price when it is probable. We typically include milestone payments for R&D services in the transaction price when they are achieved. We include these milestone payments when they are achieved because there is considerable uncertainty in the research and development processes that trigger these payments. Similarly, we include regulatory milestone payments in the transaction price once the medicine is approved by the applicable regulatory agency. We will recognize sales-based milestone payments in the period in which we achieve the milestone under the sales-based royalty exception allowed under accounting rules.

We recognize milestone payments that relate to an ongoing performance obligation over our period of performance. For example, when we achieve a milestone payment from a partner for advancing a clinical study under a collaboration agreement, we add the milestone payment to the transaction price if the milestone relates to an ongoing R&D services performance obligation and recognize revenue related to the milestone payment over our estimated period of performance. If we have partially completed our performance obligation, we record a cumulative-effect adjustment in the period we add the milestone payment to the transaction price.

Conversely, we recognize in full those milestone payments that we earn based on our partners’ activities when our partner achieves the milestone event and we do not have a remaining performance obligation.

License fees: We recognize as revenue the total amount we determine to be the relative stand-alone selling price of a license when we deliver the license to our partner because our partner has full use of the license and we do not have any additional performance obligations related to the license after delivery.

WAINUA (Eplontersen) Collaboration with AstraZeneca

In 2021, we entered into a joint development and commercialization agreement with AstraZeneca to develop and commercialize WAINUA for the treatment of transthyretin amyloidosis, or ATTR. Under the terms of the agreement, we received a $200 million upfront payment in 2021.

We evaluated our WAINUA collaboration under ASC Topic 808, Collaborative Arrangements, or ASC 808, and identified four material components: (i) the license we granted to AstraZeneca in 2021, (ii) the co-development activities that we and AstraZeneca are performing, (iii) the co-commercialization activities that we and AstraZeneca are performing and (iv) the co-medical affairs activities that we and AstraZeneca are performing.

We determined that we had a vendor-customer relationship within the scope of ASC Topic 606, Revenue from Contracts with Customers, or ASC 606, for the license we granted to AstraZeneca and as a result we hadone performance obligation. For our sole performance obligation, we determined the transaction price was the $200 million upfront payment we received. We recognized the upfront payment in full in 2021 because we did not have any remaining performance obligations after we delivered the license to AstraZeneca.

We also concluded that the co-development activities, the co-commercialization activities and the co-medical affairs activities are within the scope of ASC 808 because we and AstraZeneca are active participants exposed to the risks and benefits of the activities under the collaboration and therefore do not have a vendor-customer relationship. From inception through December 31, 2025, AstraZeneca was responsible for 55 percent of the costs associated with the ongoing global Phase 3 development program. After December 31, 2025, AstraZeneca is responsible for 75 percent of costs for development activities intended solely to support U.S. regulatory approvals and 87.5 percent of costs for development activities intended to support global regulatory approvals. Because we are leading the Phase 3 development program, we made an accounting policy election to recognize as non-customer revenue the cost-share funding from AstraZeneca, net of our share of AstraZeneca’s development expenses, in the same period we incur the related development expenses. As AstraZeneca is responsible for the majority of the commercial and medical affairs costs in the U.S. and all costs associated with bringing WAINUA to market outside the U.S., we made an accounting policy election to recognize cost-share funding we receive from AstraZeneca related to commercial and medical affairs activities as reductions of our selling, general and administrative, or SG&A, expense and R&D expense, respectively.

  1. Collaborative Arrangements and Licensing Agreements

Below, we have included our GSK, Otsuka and Roche collaborations, which were the only collaborations that had substantive changes from those included in Part IV, Item 15, Note 4, Collaborative Arrangements and Licensing Agreements, of our audited financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2025.

GSK

In 2010, we entered into a collaboration with GSK using our antisense drug discovery platform to discover and develop new medicines against targets for serious and rare diseases, including infectious diseases. Under our collaboration, GSK is developing bepirovirsen for the treatment of chronic hepatitis B, or CHB, infection. In 2019, following positive Phase 2 results, GSK licensed our CHB program. GSK is responsible for all global development, regulatory and commercialization activities and costs for the CHB program. From inception through March 31, 2026, we have received more than $105 million in an upfront payment and other payments related to the CHB program.

In the first quarter of 2026, we earned a $15 million milestone payment when the Japanese Ministry of Health, Labour and Welfare, or MHLW, accepted for review a New Drug Application, or NDA, filing for bepirovirsen. In addition, we earned a $15 million milestone payment when the European Medicines Agency, or EMA, accepted for review a Marketing Authorization Application, or MAA, filing for bepirovirsen. We recognized these milestone payments as R&D revenue in full in the first quarter of 2026 because we did not have any remaining performance obligations related to the milestone payments. We will achieve the next payment of $35 million if bepirovirsen is approved in a major country other than China.

During the three months ended March 31, 2026 and 2025, we earned the following revenue from our relationship with GSK (in thousands, except percentage amounts):

Three Months Ended
March 31,
2026 · 2025
Revenue from our relationship with GSK$30,000$-
Percentage of total revenue12%-%

We didnot have any deferred revenue from our relationship with GSK at March 31, 2026 and December 31, 2025.

Otsuka

In 2023, we entered into an agreement with Otsuka Pharmaceutical Co., Ltd., or Otsuka, to commercialize DAWNZERA in Europe. In the second quarter of 2024, we expanded the agreement to include commercialization rights for DAWNZERA in the Asia-Pacific region. We are responsible for the ongoing development of DAWNZERA. We retained the rights to commercialize DAWNZERA in the U.S. and in the rest of the world, assuming regulatory approval. In November 2024, we entered into an agreement with Otsuka to commercialize ulefnersen, an investigational medicine for the treatment of amyotrophic lateral sclerosis, or ALS, caused by mutations in theFUS gene, worldwide. We are responsible for the ongoing development of ulefnersen. From inception through March 31, 2026, we have received more than $125 million in payments from Otsuka.

In the first quarter of 2026, we achieved a $15 million milestone payment when the European Commission approved DAWNZERA in the EU. We recognized this milestone payment as R&D revenue in full in the first quarter of 2026 because we did not have any remaining performance obligations related to the milestone payment. We will achieve the next payment of $20 million if Otsuka receives reimbursement approval inthree of thefive major European countries, which include the United Kingdom, France, Germany, Italy and Spain.

During the three months ended March 31, 2026 and 2025, we earned the following revenue from our relationship with Otsuka (in thousands, except percentage amounts):

Three Months Ended
March 31,
20262025
Revenue from our relationship with Otsuka$19,932$6,528
Percentage of total revenue8%5%

Our condensed consolidated balance sheets at March 31, 2026 and December 31, 2025 included deferred revenue of $3.4 million and $4.3 million, respectively, from our relationship with Otsuka.

Roche

We have three collaborations with Hoffmann-La Roche Inc. and F. Hoffmann-La Roche Ltd, collectively Roche: one to develop treatments for Huntington’s disease, or HD, one to develop sefaxersen for the treatment of immunoglobulin A, or IgA, nephropathy, or IgAN, andone to develop RNA-targeted programs for Alzheimer's disease, or AD, and HD. From inception through March 31, 2026, we have received more than $405 million in payments from our Roche collaborations.

In the first quarter of 2026, we earned a $50 million milestone payment when Roche initiated a Phase 1 trial for an investigational medicine for the treatment of AD. We recognized this milestone payment as R&D revenue in full in the first quarter of 2026 because we did not have any remaining performance obligations related to the milestone payment. We will achieve the next payment of $10 million if Roche initiates a Phase 2 trial for an investigational medicine for the treatment of AD under our collaboration for RNA-targeted programs.

During the three months ended March 31, 2026 and 2025, we earned the following revenue from our relationship with Roche (in thousands, except percentage amounts):

Three Months Ended
March 31,
20262025
Revenue from our relationship with Roche$50,000$1,965
Percentage of total revenue20%1%

We didnot have any deferred revenue from our relationship with Roche at March 31, 2026 and December 31, 2025.

  1. Basic and Diluted Net Loss Per Share

Basic net loss per share

We calculated our basic net loss per share for the three months ended March 31, 2026 and 2025 by dividing our net loss by our weighted-average number of common shares outstanding during the period.

Diluted net loss per share

For the three months ended March 31, 2026 and 2025, we incurred a net loss; therefore, we did not include dilutive common equivalent shares in the computation of diluted net loss per share because the effect would have been anti-dilutive. Common stock from the following would have had an anti-dilutive effect on net loss per share:

1.75 percent convertible senior notes due 2028, or 1.75% Notes due 2028;

0 percent convertible senior notes due 2026, or 0% Notes due 2026;

Note hedges related to the 0% Notes due 2026;

Warrants related to the 0% Notes due 2026;

Dilutive stock options;

Unvested restricted stock units, or RSUs;

Unvested performance restricted stock units, or PRSUs; and

Employee Stock Purchase Plan, or ESPP.

For the three months ended March 31, 2026, common stock underlying the 0 percent convertible senior notes due 2030, or 0% Notes due 2030 would also have had an anti-dilutive effect on net loss per share.

  1. Investments

The following table summarizes the contract maturity of the available-for-sale securities we held as of March 31, 2026:

One year or less67%
After one year but within two years27%
After two years but within three and a half years6%
Total100%

As illustrated above, at March 31, 2026,94 percent of our available-for-sale securities had a maturity of less thantwo years.

All of our available-for-sale debt securities are available to us for use in our current operations. As a result, we categorize all of these securities as current assets even though the stated maturity of some individual securities may be one year or more beyond the balance sheet date.

We invest in debt securities with strong credit ratings and an investment grade rating at or above A-1, P-1 or F-1 by Standard & Poor’s, Moody’s or Fitch, respectively.

At March 31, 2026, we had an equity ownership interest of less than 20 percent infour private companies andfour public companies with which we conduct business.

The following is a summary of our investments (in thousands):

Amortized · Gross Unrealized · Estimated
March 31, 2026CostGainsLossesFair Value
Available-for-sale debt securities:
Corporate debt securities (1)$574,026$424$(288)$574,162
Debt securities issued by U.S. government agencies41,03868(11)41,095
Debt securities issued by the U.S. Treasury (1)497,599272(295)497,576
Debt securities issued by states of the U.S. and political subdivisions of the states4,9082-4,910
Total debt securities with a maturity of one year or less1,117,571766(594)1,117,743
Corporate debt securities363,131279(1,084)362,326
Debt securities issued by U.S. government agencies76,88514(350)76,549
Debt securities issued by the U.S. Treasury191,22945(704)190,570
Debt securities issued by states of the U.S. and political subdivisions of the states1,2241(1)1,224
Total debt securities with a maturity of more than one year632,469339(2,139)630,669
Total available-for-sale debt securities$1,750,040$1,105$(2,733)$1,748,412
Equity securities:
Publicly traded equity securities included in other current assets (2)$11,897$55,855$(9,802)$57,950
Privately held equity securities included in deposits and other assets (3)10,0009,816-19,816
Total equity securities21,89765,671(9,802)77,766
Total available-for-sale debt and equity securities$1,771,937$66,776$(12,535)$1,826,178
Amortized · Gross Unrealized · Estimated
December 31, 2025CostGainsLossesFair Value
Available-for-sale debt securities:
Corporate debt securities (1)$746,814$991$(27)$747,778
Debt securities issued by U.S. government agencies55,76892(5)55,855
Debt securities issued by the U.S. Treasury (1)769,034843(13)769,864
Debt securities issued by states of the U.S. and political subdivisions of the states5,70911-5,720
Total debt securities with a maturity of one year or less1,577,3251,937(45)1,579,217
Corporate debt securities435,3501,586(106)436,830
Debt securities issued by U.S. government agencies92,770100(94)92,776
Debt securities issued by the U.S. Treasury242,288595(1)242,882
Other municipal debt securities1,2194-1,223
Total debt securities with a maturity of more than one year771,6272,285(201)773,711
Total available-for-sale debt securities$2,348,952$4,222$(246)$2,352,928
Equity securities:
Publicly traded equity securities included in other current assets (2)$11,897$35$(8,920)$3,012
Privately held equity securities included in deposits and other assets (3) (2)4,90554,395(7,091)52,209
Total equity securities16,80254,430(16,011)55,221
Total available-for-sale debt and equity securities$2,365,754$58,652$(16,257)$2,408,149

(1)Includes investments classified as cash equivalents in our condensed consolidated balance sheets.

(2)Our publicly traded equity securities are included in other current assets. We recognize publicly traded equity securities at fair value. In the first quarter of 2026, one of our privately held investees became a publicly traded entity after completing an initial public offering. As a result, we recorded a $23.4 million unrealized gain in our condensed consolidated statements of operations, which reflects the difference between the carrying value of this investment as of December 31, 2025 and its fair value of $55.8 million as of March 31, 2026. This investment is subject to a 12-month contractual sale restriction that ends in January 2027. In the three months ended March 31, 2026, we recorded a $0.9 million net unrealized loss in our condensed consolidated statements of operations related to changes in the fair value of our other investments in publicly traded companies.

(3)Our privately held equity securities are included in deposits and other assets. We recognize our privately held equity securities at cost minus impairments, plus or minus changes resulting from observable price changes in orderly transactions for the identical or similar investment of the same issuer, which are Level 3 inputs. In the three months ended March 31, 2026, there were no changes to the carrying value of our privately held equity securities.

The following is a summary of our investments we consider to be temporarily impaired at March 31, 2026 (in thousands, except for number of investments):

Less than 12 Months of Temporary Impairment · More than 12 Months of Temporary Impairment · Total Temporary Impairment
Number of InvestmentsEstimated Fair ValueUnrealized LossesEstimated Fair ValueUnrealized LossesEstimated Fair ValueUnrealized Losses
Corporate debt securities267$516,847$(1,358)$4,480$(14)$521,327$(1,372)
Debt securities issued by U.S. government agencies4185,793(350)6,841(11)92,634(361)
Debt securities issued by the U.S. Treasury56417,944(999)--417,944(999)
Debt securities issued by states of the U.S. and political subdivisions of the states41,541(1)--1,541(1)
Total temporarily impaired debt securities368$1,022,125$(2,708)$11,321$(25)$1,033,446$(2,733)

We believe that the decline in value of these securities is temporary and is primarily related to the change in market interest rates since purchase rather than underlying credit deterioration for any of the issuers. We believe it is more likely than not that we will be able to hold our debt securities with declines in value to maturity. Therefore, we intend to hold these securities to maturity and anticipate full recovery of our debt securities’ amortized cost basis at maturity.

  1. Fair Value Measurements

The following tables present the major security types we held at March 31, 2026 and December 31, 2025 that we regularly measure and carry at fair value. The following tables segregate each security type by the level within the fair value hierarchy of the valuation techniques we utilized to determine the respective security’s fair value (in thousands):

At · Quoted Prices in Active Markets · Significant Other Observable Inputs
March 31, 2026 · (Level 1) · (Level 2)
Cash equivalents (1)$85,534$85,534$-
Corporate debt securities (2)936,488-936,488
Debt securities issued by U.S. government agencies (3)117,644-117,644
Debt securities issued by the U.S. Treasury (3)688,146688,146-
Debt securities issued by states of the U.S. and political subdivisions of the states (3)6,134-6,134
Publicly traded equity securities included in other current assets (4)57,95057,950-
Total$1,891,896$831,630$1,060,266
At · Quoted Prices in Active Markets · Significant Other Observable Inputs
December 31, 2025 · (Level 1) · (Level 2)
Cash equivalents (1)$213,579$213,579$-
Corporate debt securities (5)1,184,608-1,184,608
Debt securities issued by U.S. government agencies (3)148,631-148,631
Debt securities issued by the U.S. Treasury (3)1,012,7461,012,746-
Debt securities issued by states of the U.S. and political subdivisions of the states (3)6,943-6,943
Publicly traded equity securities included in other current assets (4)3,0123,012-
Total$2,569,519$1,229,337$1,340,182

The following footnotes reference lines in our condensed consolidated balance sheets:

(1)Included in cash and cash equivalents.

(2)$2.7 million was included in cash and cash equivalents, with the difference included in short-term investments.

(3)Included in short-term investments.

(4)Included in other current assets.

(5)$47.8 million was included in cash and cash equivalents, with the difference included in short-term investments.

Convertible Notes

Our 0% Notes due 2030,1.75% Notes due 2028 and 0% Notes due 2026 had a fair value of$802.6 million, $869.0 million and $564.2 million at March 31, 2026, respectively. Our 0% Notes due 2030,1.75% Notes due 2028 and 0% Notes due 2026 had a fair value of $830.3 million, $918.1 million and $594.6 million at December 31, 2025, respectively. We determine the fair value of our notes based on quoted market prices for these notes, which are Level 2 measurements because the notes do not trade regularly.

  1. Stock-based Compensation Expense

The following table summarizes stock-based compensation expense for the three months ended March 31, 2026 and 2025 (in thousands):

Three Months Ended
March 31,
20262025
Cost of sales$147$100
Research, development and patent expense24,70620,298
Selling, general and administrative expense18,4229,320
Stock-based compensation expense, net of amounts capitalized43,27529,718
Capitalized stock-based compensation expense18491
Total stock-based compensation expense$43,293$30,209

As of March 31, 2026, total unrecognized estimated stock-based compensation expense related to non-vested stock options, RSUs and PRSUs was $69.5 million, $190.3 million and $27.3 million, respectively. Our actual expenses will likely differ from these estimates because we will adjust our unrecognized stock-based compensation expense for future forfeitures, including any PRSUs that do not vest. We expect to recognize the cost of stock-based compensation expense related to our non-vested stock options, RSUs and PRSUs over a weighted average amortization period of 1.3 years,1.8 years and 1.9 years, respectively.

For the three months ended March 31, 2026 and 2025, we used the following weighted-average assumptions in our Black-Scholes calculations:

Employee Stock Options:

Three Months Ended
March 31,
20262025
Risk-free interest rate3.9%4.5%
Dividend yield0.0%0.0%
Volatility43.1%41.9%
Expected life6.3 years6.3 years

ESPP:

Three Months Ended
March 31,
20262025
Risk-free interest rate3.6%4.3%
Dividend yield0.0%0.0%
Volatility51.7%41.3%
Expected life6 months6 months

Stock Options:

The weighted-average grant date fair value of stock options granted to employees for thethree months ended March 31, 2026 and 2025 was $38.13 and $16.62 per share, respectively.

RSUs:

The weighted-average grant date fair value of RSUs granted to employees for the three months ended March 31, 2026 and 2025 was $77.21 and $33.21 per share, respectively.

PRSUs:

Under the terms of the PRSUs we granted in 2026 and 2025, the PRSUs may vest at the end of thethree-year performance period based on our relative total shareholder return, or TSR, as compared to a peer group of companies and as measured at the end of the performance period. Under the terms of the grants, no number of PRSUs is guaranteed to vest and the actual number of PRSUs that will vest at the end of each performance period may be anywhere from zero to 200 percent of the target number depending on our relative TSR.

The weighted-average grant date fair value of PRSUs we granted to our executive officers for the three months ended March 31, 2026 and 2025 was $115.05 and $48.81 per share, respectively.

  1. Liability Related to Sale of Future Royalties

In 2023, we entered into a royalty purchase agreement with Royalty Pharma Investments, or Royalty Pharma, to monetize a portion of our future SPINRAZA and pelacarsen royalties we are entitled to under our arrangements with Biogen and Novartis, respectively. As a result, we received an upfront payment of $500 million and we are eligible to receive up to $625 million in additional milestone payments. Under the terms of the agreement, Royalty Pharma will receive 25 percent of our SPINRAZA royalty payments from 2023 through 2027, increasing to 45 percent of royalty payments in 2028, on up to $1.5 billion in annual sales. In addition, Royalty Pharma will receive 25 percent of any future royalty payments on pelacarsen, our medicine in development to treat patients with elevated lipoprotein(a)-driven cardiovascular disease. Royalty Pharma’s royalty interest in SPINRAZA will revert to us after total SPINRAZA royalty payments to Royalty Pharma reach either $475 million or $550 million, depending on the timing and occurrence of FDA approval of pelacarsen.

We recorded the upfront payment of $500 million as a liability related to the sale of future royalties, net of transaction costs of $10.4 million, which we are amortizing over the estimated life of the arrangement using the effective interest rate method. We recognize royalty revenue in the period in which the counterparty sells the related product and recognizes the related revenue. We record royalty payments made to Royalty Pharma as a reduction of the liability.

We determine the effective interest rate used to record interest expense under this agreement based on an estimate of future royalty payments to Royalty Pharma. As of March 31, 2026 and 2025, the estimated effective interest rate under the agreement was 12.0 percent and 13.5 percent, respectively.

The following table sets forth information on our liability related to sale of future royalties (in thousands):

Liability related to sale of future royalties, net as of December 31, 2025$563,042
Royalty payments to Royalty Pharma(11,689)
Interest expense related to sale of future royalties17,184
Amortization of issuance costs related to sale of future royalties153
Liability related to sale of future royalties, net as of March 31, 2026$568,690
Less: Current portion (1)(10,925)
Liability related to sale of future royalties, net as of March 31, 2026 – Non-current$557,765

(1)Included in other current liabilities in our condensed consolidated balance sheet.

There are numerous factors, most of which are not within our control, that could materially impact the amount and timing of royalty payments from Biogen and Novartis, and result in changes to our estimate of future royalty payments to Royalty Pharma. Such factors include, but are not limited to, the commercial sales of SPINRAZA, the regulatory approval and commercial sales of pelacarsen, competing products or other significant events.

  1. Convertible Debt

0 Percent Convertible Senior Notes due 2030

In November 2025, we completed a $770.0 million offering of our 0% Notes due 2030.

At March 31, 2026, we had the following 0% Notes due 2030 outstanding (in millions except interest rate and price per share data):

Outstanding principal balance$770.0
Unamortized debt issuance costs$17.6
Maturity dateDecember 2030
Interest rate0%
Effective interest rate0.5%
Conversion price per share$98.10
Total shares of common stock subject to conversion7.8

1.75 Percent Convertible Senior Notes due 2028

In 2023, we completed a $575.0 million offering of our 1.75% Notes due 2028.

At March 31, 2026, we had the following 1.75% Notes due 2028 outstanding (in millions except interest rate and price per share data):

Outstanding principal balance$575.0
Unamortized debt issuance costs$6.5
Maturity dateJune 2028
Interest rate1.75%
Effective interest rate2.3%
Conversion price per share$53.73
Total shares of common stock subject to conversion10.7

0 Percent Convertible Senior Notes due 2026 and Call Spread

In 2021, we completed a $632.5 million offering of our 0% Notes due 2026.

At March 31, 2026, we had the following 0% Notes due 2026 outstanding (in millions except interest rate and price per share data):

Outstanding principal balance$432.5
Unamortized debt issuance costs$-
Maturity dateApril 2026
Interest rate0%
Effective interest rate0.5%
Conversion price per share$57.84
Effective conversion price per share with call spread$76.39
Total shares of common stock subject to conversion7.5

In November 2025, we used $267.6 million of the net proceeds from the issuance of the 0% Notes due 2030 to repurchase $200.0 million in principal of our 0% Notes due 2026 at a premium, which we subsequently held in treasury. As of March 31, 2026, we had an escrow deposit of $632.5 million, which we recorded as a current asset in our condensed consolidated balance sheet. In April 2026, we used $432.5 million to repay the remaining outstanding balance of the 0% Notes due 2026 and used the remaining $200.0 million to extinguish the 0% Notes due 2026 held in treasury. In the second quarter of 2026, this $200.0 million was returned to us and will be reflected in our cash, cash equivalents and short-term investments.

In conjunction with the 2021 offering, we entered into a call spread transaction, which was comprised of purchasing note hedges and selling warrants, to minimize the impact of potential economic dilution upon conversion of our 0% Notes due 2026 by increasing the effective conversion price on our 0% Notes due 2026. We increased our effective conversion price to $76.39 with the same number of underlying shares as our 0%Notes due 2026. The call spread cost us $46.9 million, of which $136.7 million was for the note hedge purchase, offset by $89.8 million we received for selling the warrants. Similar to our 0% Notes due 2026, our note hedges were subject to adjustment. Additionally, our note hedges were exercisable upon conversion of the 0% Notes due 2026. The note hedges were exercised upon maturity of the 0% Notes due 2026 in April 2026. The warrants will begin to expire in July 2026. The note hedges and warrants are separate transactions and are not part of the terms of our 0% Notes due 2026. The holders of the 0% Notes due 2026 do not have any rights with respect to the note hedges and warrants.

We recorded the amount we paid for the note hedges and the amount we received for the warrants in additional paid-in capital in our condensed consolidated balance sheets. Refer to Part IV, Item 15, Note 1, Organization and Significant Accounting Policies, of our audited financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2025 for our Call Spread accounting policy. We reassess our ability to continue to classify the note hedges and warrants in shareholders’ equity at each reporting period.

Other Terms of Convertible Senior Notes

The 0% Notes due 2030 and 1.75% Notes due 2028 are convertible under certain conditions, at the option of the note holders. We can settle conversions of the notes, at our election, in cash, shares of our common stock or a combination of both. We may not redeem the notes prior to maturity, and we do not have to provide a sinking fund for them. Holders of the notes may require us to purchase some or all of their notes upon the occurrence of certain fundamental changes, as set forth in the indentures governing the notes, at a purchase price equal to 100 percent of the principal amount of the notes to be purchased, plus any accrued and unpaid interest. The 0% Notes due 2026 were subject to similar terms.

  1. Legal Proceedings

From time to time, we are involved in legal proceedings arising in the ordinary course of our business. Periodically, we evaluate the status of each legal matter and assess our potential financial exposure. If we consider the potential loss from any legal proceeding to be probable and we can reasonably estimate the amount, we accrue a liability for the estimated loss. The outcome of any proceeding is not determinable in advance. Therefore, we are required to use significant judgment to determine the probability of a loss and whether the amount of the loss is reasonably estimable. Our assessment of a potential liability and the amount of accruals we recorded are based only on the information available to us at the time. As additional information becomes available, we reassess the potential liability related to the legal proceeding and may revise our estimates.

On September 11, 2025, we sued Arrowhead Pharmaceuticals, Inc., in the Central District of California asserting that Arrowhead's announced intention to commercialize plozasiran in November 2025 would infringe our patent US9,593,333. Arrowhead is now commercializing plozasiran and the lawsuit is ongoing. Arrowhead has filed its Answer to our Complaint and Counterclaims asserting that our patent US9,593,333 is invalid or not infringed by use of plozasiran.

  1. Segment Information

We operate as asingle operating segment, Ionis operations, focused on the research, development and commercialization of our RNA-targeted medicines to bring better futures to people with serious diseases. The CODM, our Chief Executive Officer, manages our company, reviews operating results, assesses performance and allocates resources on an aggregate basis using consolidated net income or loss as the key measure of segment profit or loss. As such, results of our operations are reported on a consolidated basis for purposes of management and segment reporting.

Ionis operations derives its revenues from commercial and R&D revenue sources. Refer to Note 4, Revenues, for further details on our sources of revenue.

The following table sets forth information on segment profit or loss, including significant segment expenses (in thousands):

Three Months Ended
March 31,
20262025
Revenue$246,091$131,612
Less:
Cost of sales2,8361,363
Drug discovery28,55726,989
Drug development104,182112,946
Medical affairs9,9895,642
Manufacturing and development chemistry20,30814,281
R&D support22,43120,553
Selling, general and administrative131,93766,980
Other segment items (1)18,37929,796
Consolidated net loss$(92,528)$(146,938)

(1)Other segment items include stock-based compensation expense, investment income, interest expense, gain or loss on investments, other income or expense and income tax expense or benefit.

ITEM 2. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS

In this Report on Form 10-Q, unless the context requires otherwise, “Ionis,” the “Company,” “we,” “our,” and “us,” means Ionis Pharmaceuticals, Inc. and its subsidiaries.

Forward-Looking Statements

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 our commercial medicines, additional medicines in development, technologies and our expectations regarding development and regulatory milestones. 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 and particularly 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, 2025, 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. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements.

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