STOCK TITAN

Ionis reports third quarter 2024 financial results

Rhea-AI Impact
(Neutral)
Rhea-AI Sentiment
(Neutral)
Tags

Ionis Pharmaceuticals reported Q3 2024 financial results, highlighting the successful launch of WAINUA in the U.S. and its approval in the UK. Total revenue was $134M for Q3, down 7% year-over-year. The company is preparing for two key product launches: olezarsen for FCS with FDA decision date December 19, 2024, and donidalorsen for HAE with PDUFA date August 21, 2025. SPINRAZA generated global sales of $381M in Q3. The company reaffirmed its 2024 P&L guidance and increased cash guidance to $2.2B following an equity offering that raised $500M.

Ionis Pharmaceuticals ha riportato i risultati finanziari per il terzo trimestre del 2024, evidenziando il lancio di successo di WAINUA negli USA e la sua approvazione nel Regno Unito. Il fatturato totale è stato di 134 milioni di dollari per il terzo trimestre, in calo del 7% rispetto all'anno precedente. L'azienda si sta preparando per il lancio di due prodotti chiave: olezarsen per FCS, con data di decisione FDA prevista per il 19 dicembre 2024, e donidalorsen per HAE, con data PDUFA fissata per il 21 agosto 2025. SPINRAZA ha generato vendite globali di 381 milioni di dollari nel terzo trimestre. L'azienda ha confermato le sue previsioni di P&L per il 2024 e ha aumentato la guida sulla liquidità a 2,2 miliardi di dollari dopo un'offerta di azioni che ha raccolto 500 milioni di dollari.

Ionis Pharmaceuticals informó los resultados financieros del tercer trimestre de 2024, destacando el exitoso lanzamiento de WAINUA en EE. UU. y su aprobación en el Reino Unido. Los ingresos totales fueron de 134 millones de dólares para el tercer trimestre, disminuyendo un 7% en comparación con el año anterior. La empresa se está preparando para el lanzamiento de dos productos clave: olezarsen para FCS, con fecha de decisión de la FDA el 19 de diciembre de 2024, y donidalorsen para HAE, con fecha PDUFA el 21 de agosto de 2025. SPINRAZA generó ventas globales de 381 millones de dólares en el tercer trimestre. La empresa reafirmó su guía de P&L para 2024 y aumentó su guía de efectivo a 2.2 mil millones de dólares tras una oferta de acciones que recaudó 500 millones de dólares.

아이오니스 제약은 2024년 3분기 재무 결과를 보고하며, 미국에서 WAINUA를 성공적으로 출시하고 영국에서 승인이 이루어졌음을 강조했습니다. 3분기 총 수익은 1억 3400만 달러로, 전년 대비 7% 감소했습니다. 이 회사는 두 가지 주요 제품 출시를 준비하고 있습니다: 2024년 12월 19일 FDA 결정일이 예정된 olezarsen (FCS용)과 2025년 8월 21일 PDUFA 날짜가 정해진 donidalorsen (HAE용)입니다. SPINRAZA는 3분기 동안 전 세계에서 3억 8100만 달러의 판매를 올렸습니다. 회사는 2024년 P&L 가이드를 재확인하고, 5억 달러의 자본 조달에 이어 현금 가이드를 22억 달러로 증가시켰습니다.

Ionis Pharmaceuticals a publié les résultats financiers du troisième trimestre 2024, soulignant le lancement réussi de WAINUA aux États-Unis et son approbation au Royaume-Uni. Le chiffre d'affaires total s'est élevé à 134 millions de dollars pour le troisième trimestre, en baisse de 7 % par rapport à l'année précédente. La société se prépare à deux lancements de produits clés : olezarsen pour le FCS, avec une date de décision de la FDA fixée au 19 décembre 2024, et donidalorsen pour l'HAE, avec une date PDUFA du 21 août 2025. SPINRAZA a généré des ventes mondiales de 381 millions de dollars au troisième trimestre. La société a réaffirmé ses prévisions de P&L pour 2024 et a augmenté ses prévisions de liquidités à 2,2 milliards de dollars après une offre d'actions ayant levé 500 millions de dollars.

Ionis Pharmaceuticals hat die finanziellen Ergebnisse für das dritte Quartal 2024 veröffentlicht und hebt den erfolgreichen Start von WAINUA in den USA sowie die Genehmigung im Vereinigten Königreich hervor. Die Gesamteinnahmen betrugen 134 Millionen US-Dollar für das dritte Quartal, was einem Rückgang von 7 % im Jahresvergleich entspricht. Das Unternehmen bereitet sich auf die Einführung von zwei wichtigen Produkten vor: olezarsen für FCS mit Entscheidungsdatum der FDA am 19. Dezember 2024 und donidalorsen für HAE mit PDUFA-Datum am 21. August 2025. SPINRAZA erzielte im dritten Quartal weltweit Verkaufszahlen von 381 Millionen US-Dollar. Das Unternehmen hat seine P&L-Prognose für 2024 bekräftigt und die Liquiditätsprognose auf 2,2 Milliarden US-Dollar nach einer Aktienemission, die 500 Millionen US-Dollar eingebracht hat, erhöht.

Positive
  • WAINUA generated sales of $23M in Q3 2024, resulting in $5M royalty revenue
  • SPINRAZA generated global sales of $381M in Q3 2024, contributing $57M in royalty revenue
  • Cash position strengthened to $2.5B following $500M equity offering
  • Received UK approval and positive CHMP opinion for WAINUA
Negative
  • Q3 revenue decreased 7% year-over-year to $134M from $144M
  • Operating loss increased to $148M in Q3 2024 from $143M in Q3 2023
  • Operating expenses increased year-over-year for both Q3 and nine-month period

Insights

Q3 financial results show mixed performance with revenue declining 7% year-over-year to $134 million, though nine-month revenue increased 3% to $479 million. The operating loss widened slightly to $148 million for Q3. Key revenue drivers include SPINRAZA royalties generating $57 million and new WAINUA royalties contributing $5 million.

The company's strong cash position of $2.5 billion, bolstered by a recent $500 million equity offering, provides runway for multiple upcoming commercial launches and pipeline development. Operating expenses remain elevated due to commercial preparations for olezarsen and donidalorsen launches, reflecting strategic investment in growth opportunities.

The pipeline shows significant commercial potential with multiple near-term catalysts. Two key regulatory decisions are approaching: olezarsen for FCS (PDUFA: Dec 19, 2024) and donidalorsen for HAE (PDUFA: Aug 21, 2025). WAINUA's commercial launch is progressing well with UK approval and positive CHMP opinion in Europe. Phase 3 data readouts for olezarsen in sHTG and pelacarsen in cardiovascular disease expected next year could substantially expand market opportunities.

WAINUATM U.S. launch progressing well; approved in UK; positive CHMP opinion

Olezarsen FCS PDUFA December 19, 2024

Donidalorsen HAE PDUFA August 21, 2025; EU regulatory submission in process

On track to achieve 2024 P&L financial guidance; increased 2024 cash guidance

CARLSBAD, Calif., Nov. 6, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company") today reported financial results for the third quarter of 2024.

"Today, we stand on the cusp of a new era for Ionis, with our first co-commercialization launch proceeding well with WAINUA, our first planned independent launch fast approaching and continued strong progress across our rich pipeline. With an upcoming December FDA action date, we are ready to independently bring olezarsen to people with familial chylomicronemia syndrome, a serious rare disease with no approved treatments in the U.S. We are similarly well positioned for our second independent launch for donidalorsen, which we believe could become a preferred treatment choice for people with hereditary angioedema, with an FDA action date of August 21, 2025," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "In parallel, we are making great progress across the rest of our rich Phase 3 pipeline. We expect Phase 3 results supporting olezarsen's second indication in severe hypertriglyceridemia and pelacarsen in Lp(a)-driven cardiovascular disease next year, as well as Phase 3 results supporting eplontersen's second indication in ATTR cardiomyopathy in the second half of 2026. We are also advancing our next wave of potentially transformational wholly owned medicines, including ION582 for Angelman syndrome, which we expect to enter Phase 3 development in the first half of next year following our positive end of Phase 2 discussion with the FDA. Our recent achievements, together with our advancing and expanding pipeline, position Ionis to deliver on our goal to bring a steady cadence of transformational medicines to people with serious diseases."

Third Quarter 2024 Summary Financial Results(1):



Three months
ended
September 30,


Nine months
ended
September 30,



2024


2023


2024


2023



(amounts in millions)

Total revenue


$134


$144


$479


$463

Operating expenses


$282


$287


$843


$811

Operating expenses on a non-GAAP basis


$250


$261


$749


$732

Loss from operations


($148)


($143)


($364)


($348)

Loss from operations on a non-GAAP basis


($116)


($117)


($270)


($269)


(1)  Reconciliation of GAAP to non-GAAP basis contained later in this release.

 

Financial Highlights

  • Revenue for the three and nine months ended September 30, 2024 decreased by 7% and increased by 3% compared to the same periods last year, respectively. Ionis continued to generate revenue from diverse sources, including a new source of royalty revenue with the launch of WAINUA in the U.S in the first quarter
  • Operating expenses for the three and nine months ended September 30, 2024 increased as planned compared to the same periods last year, excluding certain one-time costs in 2023, reflecting continued investments in late-stage development, including WAINUA for ATTR cardiomyopathy (ATTR-CM) and olezarsen for severe hypertriglyceridemia (sHTG), and commercialization efforts for WAINUA, olezarsen and donidalorsen
  • Reaffirmed 2024 P&L financial guidance, increased cash guidance to $2.2 billion reflecting proceeds from equity offering

Recent Marketed Medicines Highlights

  • WAINUA (WAINZUA in Europe) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) achieved multiple commercial and regulatory milestones:
    • Generated sales of $23 million and $44 million resulting in royalty revenue of $5 million and $10 million in the three and nine months ended September 30, 2024, respectively
    • Received positive Committee for Medicinal Products for Human Use (CHMP) opinion from European Medicines Agency (EMA) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
    • Approved in UK by the Medicines and Healthcare products Regulatory Agency (MHRA) with an accelerated National Institute for Health and Care Excellence (NICE) recommendation; earning $30 million from AstraZeneca
    • Launch underway in Canada, following approval and reimbursement from Health Canada
  • SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $381 million and $1.2 billion resulting in royalty revenue of $57 million and $152 million in the three and nine months ended September 30, 2024, respectively
    • Positive Phase 2/3 DEVOTE study data presented from higher dose nusinersen; global regulatory applications planned
  • QALSODY® (tofersen) granted marketing approval in China for the treatment of SOD1-ALS

Recent Late-Stage Pipeline Highlights

  • Olezarsen positioned to potentially treat two patient populations with urgent unmet need, familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG):
    • NDA for patients with FCS under FDA Priority Review with a PDUFA date of December 19, 2024
    • Marketing authorization application (MAA) under regulatory review by the EMA
    • Ongoing pivotal development program for sHTG on track for data in H2:2025
  • Donidalorsen Phase 3 data position it to potentially launch next year as the first RNA-targeted prophylactic treatment for people with hereditary angioedema (HAE):
    • FDA accepted the NDA for patients with HAE with a PDUFA date of August 21, 2025; Otsuka, Europe and Asia Pacific partner, preparing to submit MAA
    • Presented positive Phase 2 open label extension (OLE) study data in patients treated up to three years with every four weeks or every eight weeks dosing
  • Zilganersen Phase 3 study fully enrolled as a potential treatment for Alexander disease; on track for data in 2025
    • Granted Fast Track designation by the FDA
  • ION582 achieved important clinical and regulatory milestones enabling initiation of Phase 3 development in Angelman syndrome (AS) in H1:2025:
    • Presented positive Phase 1/2 data in patients with AS at Angelman Syndrome Foundation (ASF) Family Conference
    • Completed positive End-of-Phase 2 discussion with FDA, included alignment on Phase 3 design
  • Reported positive data from the Phase 2 study of IONIS-FB-LRx in patients with immunoglobulin A nephropathy (IgAN); Roche continues to advance IONIS-FB-LRx in the Phase 3 IMAGINATION study

Recent Other Pipeline Updates

  • Sapablursen for the treatment of polycythemia vera granted orphan drug designation by FDA; enrollment complete in Phase 2 IMPRSSION study with data expected in 2025
  • IONIS-MAPTRx (BIIB080) enrollment complete in Phase 2 CELIA study in patients with early Alzheimer's disease (AD); data expected in 2026
  • Initiated first in human studies with multiple medicines from neurological disease pipeline:
    • Phase 1/2 Orbit study of ION356 (PLP1) in patients with Pelizaeus-Merzbacher disease (PMD)
    • Phase 1/2 HERO study of ION269 (APP), for the potential treatment of Alzheimer's disease (AD), which is initially being evaluated in patients with Down syndrome (DS) who have a genetic risk for developing AD
    • Phase 1/2 ATTUNE study of ION440 (MECP2) in patients with MECP2 duplication syndrome

Third Quarter 2024 Financial Results

"This year has been marked by strong delivery on our pipeline and business goals, which position Ionis to deliver on our vision of bringing a steady cadence of innovative medicines to patients in need. Fully realizing these significant opportunities requires substantial investment. As a result, we recently executed an equity offering that extends our cash runway, enabling us to continue to invest in the numerous attractive opportunities ahead of us, including our near-term commercial launches with multi-billion-dollar revenue potential and our rich late and mid-stage pipeline," said Elizabeth L. Hougen, chief financial officer of Ionis. "Looking beyond this year, we will continue to invest in go-to-market preparations for our planned olezarsen and donidalorsen launches. Additionally, with our increased confidence in the potential of WAINUA and olezarsen to address broader patient populations, we plan to scale our capabilities in line with the significant potential of these important medicines. At the same time, we are investing in our next wave of medicines, including pre-commercialization activities and Phase 3 development for ION582 for Angelman syndrome and zilganersen for Alexander disease. We expect our investments today will position Ionis for sustainable growth for years to come."

Revenue

Ionis' revenue was comprised of the following:



Three months ended
September 30,


Nine months ended
September 30,



2024


2023


2024


2023

Revenue:


(amounts in millions)

     Commercial revenue:









SPINRAZA royalties


$57


$67


$152


$179

WAINUA royalties


5


-


10


-

Other commercial revenue:









TEGSEDI and WAYLIVRA revenue, net


9


8


26


25

Licensing and other royalty revenue


5


9


19


26

Total commercial revenue


76


84


207


230

Research and development revenue:









Amortization from upfront payments


28


18


105


47

Milestone payments


16


16


76


90

License fees


-


5


38


25

Other services


1


5


18


11

Collaborative agreement revenue


45


44


237


173

WAINUA joint development revenue


13


16


35


60

Total research and development revenue


58


60


272


233

Total revenue


$134


$144


$479


$463

 

Commercial revenue for the three and nine months ended September 30, 2024 included a new source of royalty revenue with the launch of WAINUA in the U.S. in late January 2024. Ionis' commercial revenue for the three and nine months ended September 30, 2024 and 2023 also included royalties from the net sales of QALSODY, which Biogen launched in the U.S. in the second quarter of 2023 and in the EU in the second quarter of 2024. SPINRAZA product sales for the three months ended September 30, 2024 compared to the same period last year increased slightly in the U.S. and decreased outside of the U.S. primarily due to an annual order from a single country that did not recur in 2024.

R&D revenue was relatively consistent for the three months ended September 30, 2024 compared to the same period last year. R&D revenue increased for the nine months ended September 30, 2024 compared to the same period last year primarily due to the amortization of upfront payments from the new collaborations with Roche and Novartis that Ionis entered into during the second half of last year. In addition, license fees increased year over year as a result of new collaborations Ionis entered into during the second quarter of 2024, including the expanded donidalorsen licensing agreement with Otsuka, which now includes the Asia-Pacific region in addition to Europe. These increases were partially offset by the decrease in WAINUA joint development revenue, which decreased as development activities relating to ATTRv-PN wound down with the launch of WAINUA for this indication.

Operating Expenses

Ionis' operating expenses, excluding one-time costs associated with a lease exit in the third quarter of 2023, increased for the three and nine months ended September 30, 2024 compared to the same periods in 2023, consistent with expectations. SG&A expenses increased year over year primarily due to the launch of WAINUA in the U.S. and launch preparation activities for olezarsen and donidalorsen, including establishing the field team for olezarsen in the second quarter of 2024. R&D expenses were essentially flat for the three and nine months ended September 30, 2024 compared to the same periods last year as several late-stage studies have ended.

Balance Sheet

As of September 30, 2024, Ionis' cash, cash equivalents and short-term investments increased to $2.5 billion compared to $2.3 billion at December 31, 2023. In September 2024, Ionis issued 11.5 million shares of its common stock at a public offering price of $43.50 per share that generated gross proceeds of $500 million, before deducting underwriting discounts and commissions and other offering expenses payable by Ionis. The Company plans to continue deploying its capital resources toward growth opportunities, and projects to end 2024 with $2.2 billion in cash, cash equivalents and short-term investments. Ionis' working capital also increased over the same period primarily due to the Company's higher cash and short-term investments balance. As a result of Ionis' advancing pipeline that has delivered several positive data readouts, Ionis expects to make increased investments in the years ahead with the goal to realize the value of these opportunities, with a focus on its wholly owned late-stage and next wave of innovative medicines.

Webcast 

Management will host a conference call and webcast to discuss Ionis' third quarter 2024 results at 11:30 a.m. Eastern time on Wednesday, November 6, 2024. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. To access the Company's third quarter 2024 earnings slides click here.

For more information about SPINRAZA and QALSODY, visit https://www.spinraza.com/ and https://www.qalsody.com/, respectively. QALSODY is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval may be contingent upon verification of clinical benefit in confirmatory trial(s).

INDICATION for WAINUA™ (eplontersen)
WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

IMPORTANT SAFETY INFORMATION for WAINUA™ (eplontersen)

WARNINGS AND PRECAUTIONS
Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.

ADVERSE REACTIONS
Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).

Please see link to U.S. Full Prescribing Information for WAINUA. 

About Ionis Pharmaceuticals, Inc.

For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram.

Ionis' Forward-looking Statement

This press release includes forward-looking statements regarding Ionis' business, financial guidance and the therapeutic and commercial potential of our commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' 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 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. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. 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. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2023, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.

In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics® is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI® is a registered trademark of Akcea Therapeutics, Inc. WAYLIVRA® is a registered trademark of Akcea Therapeutics, Inc. SPINRAZA® and QALSODY® are registered trademarks of Biogen. WAINUATM is a registered trademark of the AstraZeneca group of companies.

Ionis Investor Contact:
D. Wade Walke, Ph.DD.
IR@ionis.com
760-603-2331

Ionis Media Contact:
Hayley Soffer
media@ionis.com 
760-603-4679

 

IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)




Three months ended


Nine months ended



September 30,


September 30,



2024


2023


2024


2023



(unaudited)

Revenue:









     Commercial revenue:









SPINRAZA royalties


$57


$67


$152


$179

WAINUA royalties


5


-


10


-

Other commercial revenue


14


17


45


51

Total commercial revenue


76


84


207


230

Research and development revenue:









Collaborative agreement revenue


45


44


237


173

WAINUA joint development revenue


13


16


35


60

Total research and development revenue


58


60


272


233

Total revenue


134


144


479


463

Expenses:









      Cost of sales


1


2


7


6

    Research, development and patent


220


215


656


643

    Selling, general and administrative


61


70


180


162

Total operating expenses


282


287


843


811

Loss from operations


(148)


(143)


(364)


(348)










Other income (expense):









Interest expense related to the sale of future royalties


(19)


(18)


(55)


(51)

Other income, net


23


20


66


68

Loss before income tax benefit (expense)


(144)


(141)


(353)


(331)










Income tax benefit (expense)


4


(6)


3


(26)










Net loss


($140)


($147)


($350)


($357)










Basic and diluted net loss per share


($0.95)


($1.03)


($2.38)


($2.50)

Shares used in computing basic and diluted net
loss per share


149


143


147


143

 

IONIS PHARMACEUTICALS, INC.
Reconciliation of GAAP to Non-GAAP Basis:
Condensed Consolidated Operating Expenses, Loss From Operations, and Net Loss
(In Millions)

 








Three months ended

September 30,


Nine months ended

September 30,



2024


2023


2024


2023



(unaudited)

As reported research, development and patent
expenses according to GAAP


$220


$215


$656


$643

    Excluding compensation expense related to equity awards      


(22)


(19)


(67)


(58)

Non-GAAP research, development and patent
expenses


$198


$196


$589


$585










 

As reported selling, general and administrative
expenses according to GAAP


$61


$70


$180


$162

    Excluding compensation expense related to equity awards      


(10)


(7)


(26)


(22)

Non-GAAP selling, general and administrative
expenses


$51


$63


$154


$140










As reported operating expenses according to GAAP


$282


$287


$843


$811

Excluding compensation expense related to equity awards      


(32)


(26)


(94)


(79)

Non-GAAP operating expenses


$250


$261


$749


$732










As reported loss from operations according to GAAP


($148)


($143)


($364)


($348)

    Excluding compensation expense related to equity awards      


(32)


(26)


(94)


(79)

Non-GAAP loss from operations


($116)


($117)


($270)


($269)










As reported net loss according to GAAP


($140)


($147)


($350)


($357)

    Excluding compensation expense related to equity awards
     and related tax effects              


(32)


(26)


(94)


(79)

Non-GAAP net loss


($108)


($121)


($256)


($278)

Reconciliation of GAAP to Non-GAAP Basis

As illustrated in the Selected Financial Information in this press release, non-GAAP operating expenses, non-GAAP loss from operations, and non-GAAP net loss were adjusted from GAAP to exclude compensation expense related to equity awards and the related tax effects. Compensation expense related to equity awards are non-cash. These measures are provided as supplementary information and are not a substitute for financial measures calculated in accordance with GAAP. Ionis reports these non-GAAP results to better enable financial statement users to assess and compare its historical performance and project its future operating results and cash flows. Further, the presentation of Ionis' non-GAAP results is consistent with how Ionis' management internally evaluates the performance of its operations.

IONIS PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
(In Millions)    






September 30,


December 31,




2024


2023




(unaudited)




Assets:






  Cash, cash equivalents and short-term investments


$2,483


$2,331


  Contracts receivable


18


98


  Other current assets


213


213


  Property, plant and equipment, net


83


71


  Right-of-use assets


164


172


  Other assets


120


105


     Total assets


$3,081


$2,990








Liabilities and stockholders' equity:






  Current portion of deferred contract revenue


$76


$151


  0.125% convertible senior notes, net – short-term


44


44


  Other current liabilities


184


253


  1.75% convertible senior notes, net


564


562


  0% convertible senior notes, net


628


625


  Liability related to sale of future royalties, net


538


514


  Long-term lease liabilities


168


171


  Long-term obligations, less current portion


43


42


  Long-term deferred contract revenue


174


241


  Total stockholders' equity


662


387


    Total liabilities and stockholders' equity


$3,081


$2,990


Key 2024 Value Driving Events(1) 

New Product Launches

Program

Indication

Achieved

WAINUA

ATTRv-PN

Olezarsen

FCS


QALSODY (EU)

SOD1-ALS


Regulatory Actions

Program

Indication

Regulatory Action

Achieved

Eplontersen

ATTRv-PN

Additional OUS filings

EMA approval decision


Additional OUS approval
decision(s)

Olezarsen

FCS

FDA approval decision


NDA filing

EU filing

Canada filing


Donidalorsen

HAE

NDA filing

QALSODY

SOD1-ALS

EMA approval decision

China approval


Key Phase 3 Clinical Data Events

Program

Indication

Event

Achieved

Olezarsen

FCS

Balance study full data

Donidalorsen

HAE

OASIS-HAE topline data

Donidalorsen

HAE

OASIS-HAE full data

Donidalorsen

HAE

OASIS-Plus: OLE + Switch
data

Nusinersen

SMA

DEVOTE study data (higher
dose)


Key Phase 2 Clinical Data Events

Program

Indication

Event

Achieved

Donidalorsen

HAE

3-year Phase 2 OLE data

IONIS-FB-LRx

IgAN

Phase 2 data

IONIS-FB-LRx

GA

GOLDEN study data

--

ION224 (DGAT2)

NASH

Phase 2 data

ION582 (UBE3A)

Angelman
syndrome

HALOS study data

ION541 (ATXN2)

ALS

ALSpire study data

--

Sapablursen
(TMPRSS6)

Polycythemia vera

IMPRSSION study full
enrollment

IONIS-MAPTRx
(Tau)

Alzheimer's
disease

CELIA study full enrollment


(1)  Timing expectations based on current assumptions and subject to change.

•         Milestone achieved

--     Milestone achieved, however program discontinued

 

Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/ionis-reports-third-quarter-2024-financial-results-302297131.html

SOURCE Ionis Pharmaceuticals, Inc.

FAQ

What was Ionis (IONS) revenue in Q3 2024?

Ionis reported total revenue of $134 million in Q3 2024, representing a 7% decrease from $144 million in Q3 2023.

When is the FDA decision date for Ionis (IONS) olezarsen?

The FDA PDUFA date for olezarsen in FCS treatment is December 19, 2024.

How much revenue did WAINUA generate for Ionis (IONS) in Q3 2024?

WAINUA generated sales of $23 million in Q3 2024, resulting in $5 million in royalty revenue for Ionis.

What is Ionis (IONS) cash guidance for 2024?

Ionis increased its cash guidance to $2.2 billion for 2024 following a successful equity offering.

Ionis Pharmaceuticals, Inc.

NASDAQ:IONS

IONS Rankings

IONS Latest News

IONS Stock Data

6.14B
157.71M
0.78%
98.13%
6.26%
Biotechnology
Pharmaceutical Preparations
Link
United States of America
CARLSBAD