Ionis enters new chapter in 2025 as commercial-stage biotech with multiple independent product launches anticipated and continued late-stage pipeline momentum
Ionis Pharmaceuticals (IONS) enters 2025 with significant momentum following the U.S. approval and launch of TRYNGOLZA™, the first-ever therapy for familial chylomicronemia syndrome (FCS). The company anticipates four independent product launches over the next three years, plus four additional launches from partnered programs. Key 2025 milestones include potential European approval for TRYNGOLZA, expected FDA decision on donidalorsen for hereditary angioedema by August 21, and continued progress with WAINUA™ in collaboration with AstraZeneca.
The company plans to advance seven clinical-stage therapies for neurological diseases, including Phase 3 trials for ION582 in Angelman syndrome and data readouts for zilganersen in Alexander disease. Ionis expects increasing product and royalty revenue to drive positive cash flow, marking its transition to a fully integrated commercial-stage biotechnology company.
Ionis Pharmaceuticals (IONS) inizia il 2025 con una notevole spinta dopo l'approvazione negli Stati Uniti e il lancio di TRYNGOLZA™, la prima terapia mai esistita per la sindrome da chilomicronemia familiare (FCS). L'azienda prevede quattro lanci indipendenti di prodotti nei prossimi tre anni, oltre a quattro ulteriori lanci da programmi in partnership. Tra i principali obiettivi per il 2025 ci sono l'approvazione potenziale in Europa per TRYNGOLZA, una decisione attesa dalla FDA su donidalorsen per l'angioedema ereditario entro il 21 agosto, e progressi continui con WAINUA™ in collaborazione con AstraZeneca.
L'azienda ha in programma di far avanzare sette terapie in fase clinica per malattie neurologiche, comprese le sperimentazioni di Fase 3 per ION582 nella sindrome di Angelman e la lettura dei dati per zilganersen nella malattia di Alexander. Ionis prevede un incremento delle entrate da prodotto e royalty che guiderà un flusso di cassa positivo, segnando la sua transizione in una compagnia biotech completamente integrata in fase commerciale.
Ionis Pharmaceuticals (IONS) comienza 2025 con un impulso significativo tras la aprobación y lanzamiento en EE. UU. de TRYNGOLZA™, la primera terapia para el síndrome de quilomicronemia familiar (FCS). La compañía anticipa cuatro lanzamientos de productos independientes en los próximos tres años, además de cuatro lanzamientos adicionales de programas asociados. Los hitos clave para 2025 incluyen la posible aprobación europea de TRYNGOLZA, una decisión de la FDA sobre donidalorsen para el angioedema hereditario esperada para el 21 de agosto, y un progreso continuo con WAINUA™ en colaboración con AstraZeneca.
La compañía planea avanzar siete terapias en etapa clínica para enfermedades neurológicas, incluidas las pruebas de Fase 3 para ION582 en el síndrome de Angelman y los resultados de los datos para zilganersen en la enfermedad de Alexander. Ionis espera que el aumento de ingresos por productos y regalías impulse un flujo de caja positivo, marcando su transición a una compañía de biotecnología comercial completamente integrada.
아이오니스 제약(Ions)은 미국에서 트랭골자(TRYNGOLZA™)의 승인과 출시를 통해 2025년을 강력한 성장세로 시작합니다. 이는 가족성 카일로미크론혈증 증후군(FCS)을 치료하는 최초의 요법입니다. 이 회사는 향후 3년 동안 4개 독립적인 제품 출시를 예상하며, 추가로 4개의 파트너 프로그램에서 출시를 계획하고 있습니다. 2025년의 주요 이정표로는 TRYNGOLZA의 유럽 승인 가능성, 8월 21일 예정된 유전자성 부종(syndrome de Angioedema) 치료에 대한 FDA 결정, 그리고 아스트라제네카와의 협업으로 진행 중인 WAINUA™의 지속적인 진전을 포함합니다.
회사는 앤젤만 신드롬에 대한 ION582의 3상 시험과 알렉산더 병에서의 zilganersen 데이터 발표를 포함해 신경 질환에 대한 7개의 임상 단계 요법을 발전시킬 계획입니다. 아이오니스는 제품 및 로열티 수익의 증가가 긍정적인 현금 흐름을 이끌어낼 것으로 기대하며, 완전히 통합된 상업 단계의 생명공학 회사로서의 전환을 기념합니다.
Ionis Pharmaceuticals (IONS) commence l'année 2025 avec un élan significatif suite à l'approbation et au lancement aux États-Unis de TRYNGOLZA™, la première thérapie contre le syndrome de chylomicronémie familiale (FCS). L'entreprise s'attend à quatre lancements de produits indépendants au cours des trois prochaines années, ainsi qu'à quatre lancements supplémentaires provenant de programmes partenaires. Parmi les jalons clés pour 2025, on trouve une éventuelle approbation européenne pour TRYNGOLZA, une décision attendue de la FDA sur le donidalorsen pour l'angioedème héréditaire d'ici le 21 août, et des avancées continues avec WAINUA™ en collaboration avec AstraZeneca.
L'entreprise prévoit de faire progresser sept thérapies en phase clinique pour les maladies neurologiques, y compris des essais de Phase 3 pour ION582 dans le syndrome d'Angelman et l'analyse des données pour le zilganersen dans la maladie d'Alexander. Ionis s'attend à ce que l'augmentation des revenus des produits et des redevances génère un flux de trésorerie positif, marquant ainsi sa transition vers une entreprise de biotechnologie entièrement intégrée en phase commerciale.
Ionis Pharmaceuticals (IONS) startet 2025 mit erheblichem Schwung nach der Genehmigung und dem Launch von TRYNGOLZA™ in den USA, der ersten Therapie gegen das familiäre Chylomikronämie-Syndrom (FCS). Das Unternehmen plant in den nächsten drei Jahren vier unabhängige Produkteinführungen sowie vier zusätzliche Einführungen aus Partnerprogrammen. Zu den wichtigen Meilensteinen für 2025 gehören eine mögliche europäische Genehmigung für TRYNGOLZA, eine erwartete FDA-Entscheidung über donidalorsen zur hereditären Angioödemen bis zum 21. August und kontinuierliche Fortschritte mit WAINUA™ in Zusammenarbeit mit AstraZeneca.
Das Unternehmen plant, sieben Therapien in klinischen Phasen für neurologische Erkrankungen weiterzuentwickeln, darunter Phase-3-Studien für ION582 bei Angelman-Syndrom und Datenauswertungen für zilganersen bei der Alexander-Krankheit. Ionis erwartet einen Anstieg der Produkt- und Lizenzgebühreneinnahmen, der zu einem positiven Cashflow führen wird und so den Übergang zu einem voll integrierten biotechnologischen Unternehmen in der kommerziellen Phase markiert.
- First-ever FDA-approved therapy for FCS (TRYNGOLZA) launched
- Four independent product launches planned in next three years
- Four additional partnered product launches expected
- Clear path to positive cash flow through increasing product and royalty revenue
- Multiple late-stage clinical programs advancing toward commercialization
- Safety concerns including hypersensitivity reactions with TRYNGOLZA
- Adverse reactions reported in >5% of TRYNGOLZA-treated patients
Insights
The transformation of Ionis into a commercial-stage biotech marks a pivotal inflection point. The recent FDA approval of TRYNGOLZA for FCS and potential approval of donidalorsen represent important revenue catalysts. The company's path to sustained positive cash flow is particularly noteworthy, supported by four independent product launches and four partnered launches over the next three years.
The pipeline momentum is robust, with multiple Phase 3 readouts expected in 2025, including the CORE trials and zilganersen for Alexander disease. The diversification across rare diseases, particularly in neurology, reduces clinical development risk. The partnership with AstraZeneca for WAINUA provides both immediate revenue potential and strategic validation.
Strategic positioning in RNA therapeutics, coupled with next-generation technology development (MsPA backbone, siRNA platforms), strengthens the company's competitive moat. The $5.28B market cap appears to undervalue the commercial potential of the expanding product portfolio and advanced pipeline assets.
The commercial landscape for Ionis's products shows significant market opportunity. TRYNGOLZA addresses an untapped market in FCS with no existing approved therapies, enabling premium pricing and high margins. The severe hypertriglyceridemia indication expansion represents a substantially larger commercial opportunity.
The hereditary angioedema market for donidalorsen, while competitive, offers significant revenue potential given the chronic nature of treatment. WAINUA's launch trajectory in ATTRv-PN provides early validation of Ionis's commercial execution capabilities. The potential expansion into ATTR cardiomyopathy could multiply the addressable market substantially.
– Accelerating value creation with numerous commercial, regulatory and pipeline milestones –
– Ionis outlines clear path to sustained positive cash flow –
"With last month's
2025 Anticipated Highlights Include:
- Launch TRYNGOLZA, Ionis' first independent product and the first-ever therapy for
U.S. patients with familial chylomicronemia syndrome (FCS):U.S. Food and Drug Administration (FDA) Approval for FCS on December 19, 2024- Potential European Medicines Agency (EMA) approval for FCS
- Phase 3 results and
U.S. regulatory submission for olezarsen in severe hypertriglyceridemia, a large patient population with high unmet need- Phase 3 topline data for CORE, CORE 2 and ESSENCE Phase 3 trials expected in second half of 2025
- Potential approval of donidalorsen, a first-in-class RNA-targeted medicine for hereditary angioedema and Ionis' second anticipated independent launch:
U.S. FDA action date of August 21, 2025U.S. commercial field team scale-up and launch
- Continue progress with WAINUA™ (eplontersen), Ionis' first
U.S. co-commercialized medicine in collaboration with AstraZeneca:- Continued positive performance in
U.S. launch for polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) - OUS approvals for ATTRv-PN
- Continuing the fully enrolled, landmark CARDIO-TTRansform trial in ATTR cardiomyopathy, with data expected in the second half of 2026
- Continued positive performance in
- Advance the next wave of wholly owned medicines for serious neurological diseases, including seven clinical-stage therapies:
- Phase 3 trial start in first half of 2025 for ION582 for Angelman syndrome
- Phase 3 data for zilganersen in Alexander disease, a rare leukodystrophy with no approved disease-modifying therapies
- Phase 2 data for ION464 (SNCA) in multiple system atrophy
- Continue momentum with key partnered programs, including:
- Phase 3 data for pelacarsen, a potentially groundbreaking medicine for Lp(a)-driven cardiovascular disease (Novartis)
- Continue advancing next-generation technologies for RNA-targeted medicines
- Achieve clinical proof of concept for Mesyl Phosphoramidate (MsPA) backbone and siRNA platforms
- Advance first Bicycle-siRNA into clinical development
- Select first clinical candidate engineered to cross the blood brain barrier
INDICATION for TRYNGOLZA™ (olezarsen)
TRYNGOLZA™ (olezarsen) was approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred.
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur.
ADVERSE REACTIONS
The most common adverse reactions (incidence >
Please see full Prescribing Information for TRYNGOLZA.
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
About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six 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 Statements
This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of TRYNGOLZA, Ionis' technologies and other products in development. 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 but not limited to those related to our commercial products and the medicines in our pipeline, 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. 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 Dec. 31, 2023, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at www.Ionis.com.
Ionis Pharmaceuticals® and TRYNGOLZA™ are trademarks of Ionis Pharmaceuticals, Inc. WAINUA™ is a registered trademark of the AstraZeneca plc.
Ionis Investor Contact:
D. Wade Walke, Ph.D.
IR@ionis.com
760-603-2331
Ionis Media Contact:
Hayley Soffer
media@ionis.com
760-603-4679
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SOURCE Ionis Pharmaceuticals, Inc.
FAQ
When did IONS receive FDA approval for TRYNGOLZA?
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