Ionis reports third quarter 2024 financial results
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.
- 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
- 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
The company's strong cash position of
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
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
"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 | Nine months | |||||||
2024 | 2023 | 2024 | 2023 | |||||
(amounts in millions) | ||||||||
Total revenue | ||||||||
Operating expenses | ||||||||
Operating expenses on a non-GAAP basis | ||||||||
Loss from operations | ( | ( | ( | ( | ||||
Loss from operations on a non-GAAP basis | ( | ( | ( | ( |
(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 by3% 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 theU.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
reflecting proceeds from equity offering$2.2 billion
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
and$23 million resulting in royalty revenue of$44 million and$5 million in the three and nine months ended September 30, 2024, respectively$10 million - 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 from AstraZeneca$30 million - Launch underway in
Canada , following approval and reimbursement from Health Canada
- Generated sales of
- SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of
and$381 million resulting in royalty revenue of$1.2 billion and$57 million in the three and nine months ended September 30, 2024, respectively$152 million - 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 andAsia 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
- FDA accepted the NDA for patients with HAE with a PDUFA date of August 21, 2025; Otsuka,
- 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 | Nine months ended | |||||||
2024 | 2023 | 2024 | 2023 | |||||
Revenue: | (amounts in millions) | |||||||
Commercial revenue: | ||||||||
SPINRAZA royalties | ||||||||
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 |
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
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
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
Balance Sheet
As of September 30, 2024, Ionis' cash, cash equivalents and short-term investments increased to
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 (≥
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. | ||||||||
Three months ended | Nine months ended | |||||||
September 30, | September 30, | |||||||
2024 | 2023 | 2024 | 2023 | |||||
(unaudited) | ||||||||
Revenue: | ||||||||
Commercial revenue: | ||||||||
SPINRAZA royalties | ||||||||
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 | ( | ( | ( | ( | ||||
Basic and diluted net loss per share | ( | ( | ( | ( | ||||
Shares used in computing basic and diluted net | 149 | 143 | 147 | 143 |
IONIS PHARMACEUTICALS, INC.
| ||||||||
Three months ended September 30, | Nine months ended September 30, | |||||||
2024 | 2023 | 2024 | 2023 | |||||
(unaudited) | ||||||||
As reported research, development and patent | ||||||||
Excluding compensation expense related to equity awards | (22) | (19) | (67) | (58) | ||||
Non-GAAP research, development and patent | ||||||||
As reported selling, general and administrative | ||||||||
Excluding compensation expense related to equity awards | (10) | (7) | (26) | (22) | ||||
Non-GAAP selling, general and administrative | ||||||||
As reported operating expenses according to GAAP | ||||||||
Excluding compensation expense related to equity awards | (32) | (26) | (94) | (79) | ||||
Non-GAAP operating expenses | ||||||||
As reported loss from operations according to GAAP | ( | ( | ( | ( | ||||
Excluding compensation expense related to equity awards | (32) | (26) | (94) | (79) | ||||
Non-GAAP loss from operations | ( | ( | ( | ( | ||||
As reported net loss according to GAAP | ( | ( | ( | ( | ||||
Excluding compensation expense related to equity awards | (32) | (26) | (94) | (79) | ||||
Non-GAAP net loss | ( | ( | ( | ( |
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. | |||||||
September 30, | December 31, | ||||||
2024 | 2023 | ||||||
(unaudited) | |||||||
Assets: | |||||||
Cash, cash equivalents and short-term investments | |||||||
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 | |||||||
Liabilities and stockholders' equity: | |||||||
Current portion of deferred contract revenue | |||||||
| 44 | 44 | |||||
Other current liabilities | 184 | 253 | |||||
| 564 | 562 | |||||
| 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 |
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 | • | ||
Olezarsen | FCS | FDA approval decision | |
NDA filing | • | ||
EU filing | • | ||
Donidalorsen | HAE | NDA filing | • |
QALSODY | SOD1-ALS | EMA approval decision | • |
• | |||
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 | • |
Nusinersen | SMA | DEVOTE study data (higher | • |
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 | HALOS study data | • |
ION541 (ATXN2) | ALS | ALSpire study data | -- |
Sapablursen | Polycythemia vera | IMPRSSION study full | • |
IONIS-MAPTRx | Alzheimer's | CELIA study full enrollment | • |
(1) Timing expectations based on current assumptions and subject to change. |
• Milestone achieved |
-- Milestone achieved, however program discontinued |
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SOURCE Ionis Pharmaceuticals, Inc.
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