Ionis and Ono announce global license agreement for sapablursen in polycythemia vera
Ionis Pharmaceuticals (IONS) and Ono Pharmaceutical have entered into a global license agreement for sapablursen, an RNA-targeted medicine for polycythemia vera (PV). Under the agreement, Ono obtains exclusive global rights for development and commercialization of the drug.
The deal includes a $280 million upfront payment to Ionis, with potential additional payments of up to $660 million based on development, regulatory and sales milestones. Ionis will also receive mid-teen percentage royalties on annual net sales.
Sapablursen is currently in Phase 2 IMPRSSION study and has received Fast Track designation and orphan drug designation from the FDA in 2024. Ionis will complete the ongoing Phase 2 study, while Ono will handle subsequent development, regulatory filings, and commercialization.
Ionis Pharmaceuticals (IONS) e Ono Pharmaceutical hanno stipulato un accordo di licenza globale per sapablursen, un farmaco mirato all'RNA per la policitemia vera (PV). In base all'accordo, Ono ottiene diritti esclusivi a livello globale per lo sviluppo e la commercializzazione del farmaco.
L'accordo prevede un pagamento iniziale di 280 milioni di dollari a Ionis, con potenziali pagamenti aggiuntivi fino a 660 milioni di dollari basati su traguardi di sviluppo, regolatori e di vendita. Ionis riceverà anche royalties percentuali a due cifre sui ricavi netti annuali.
Attualmente, sapablursen è in fase 2 dello studio IMPRSSION e ha ricevuto la designazione Fast Track e la designazione di farmaco orfano dalla FDA nel 2024. Ionis completerà lo studio di fase 2 in corso, mentre Ono si occuperà dello sviluppo successivo, delle pratiche regolatorie e della commercializzazione.
Ionis Pharmaceuticals (IONS) y Ono Pharmaceutical han firmado un acuerdo de licencia global para sapablursen, un medicamento dirigido al ARN para la policitemia vera (PV). Según el acuerdo, Ono obtiene derechos exclusivos a nivel mundial para el desarrollo y la comercialización del fármaco.
El acuerdo incluye un pago inicial de 280 millones de dólares a Ionis, con pagos adicionales potenciales de hasta 660 millones de dólares basados en hitos de desarrollo, regulatorios y de ventas. Ionis también recibirá regalías porcentuales de dos dígitos sobre las ventas netas anuales.
Actualmente, sapablursen se encuentra en el estudio IMPRSSION de fase 2 y ha recibido la designación de Fast Track y la designación de medicamento huérfano por parte de la FDA en 2024. Ionis completará el estudio de fase 2 en curso, mientras que Ono se encargará del desarrollo posterior, las presentaciones regulatorias y la comercialización.
아이오니스 제약(Ions)과 오노 제약이 사파블루르센에 대한 글로벌 라이선스 계약을 체결했습니다. 이는 진성적혈구증가증(PV)을 위한 RNA 표적 의약품입니다. 계약에 따라 오노는 이 약물의 개발 및 상용화에 대한 독점적인 글로벌 권리를 확보합니다.
이번 거래에는 아이오니스에 대한 2억 8천만 달러의 선불 지급이 포함되며, 개발, 규제 및 판매 이정표에 따라 최대 6억 6천만 달러의 추가 지급이 가능성 있습니다. 아이오니스는 연간 순매출의 중간 10대 비율의 로열티도 받게 됩니다.
사파블루르센은 현재 2상 IMPRSSION 연구 중이며, 2024년 FDA로부터 패스트 트랙 및 희귀 약물 지정을 받았습니다. 아이오니스는 진행 중인 2상 연구를 완료하고, 오노는 이후 개발, 규제 제출 및 상용화를 담당할 것입니다.
Ionis Pharmaceuticals (IONS) et Ono Pharmaceutical ont conclu un accord de licence mondial pour sapablursen, un médicament ciblant l'ARN pour la polycythémie vera (PV). En vertu de cet accord, Ono obtient des droits exclusifs mondiaux pour le développement et la commercialisation du médicament.
L'accord comprend un paiement initial de 280 millions de dollars à Ionis, avec des paiements supplémentaires potentiels pouvant atteindre 660 millions de dollars en fonction des jalons de développement, réglementaires et de vente. Ionis recevra également des redevances à deux chiffres sur les ventes nettes annuelles.
Le sapablursen est actuellement dans l'étude IMPRSSION de phase 2 et a reçu la désignation Fast Track et la désignation de médicament orphelin de la FDA en 2024. Ionis complétera l'étude de phase 2 en cours, tandis qu'Ono s'occupera du développement ultérieur, des dépôts réglementaires et de la commercialisation.
Ionis Pharmaceuticals (IONS) und Ono Pharmaceutical haben einen globalen Lizenzvertrag für sapablursen abgeschlossen, ein RNA-gerichtetes Medikament für Polycythaemia vera (PV). Im Rahmen des Vertrags erhält Ono die exklusiven globalen Rechte für die Entwicklung und Vermarktung des Medikaments.
Der Deal umfasst eine Vorauszahlung von 280 Millionen US-Dollar an Ionis, mit potenziellen zusätzlichen Zahlungen von bis zu 660 Millionen US-Dollar, die auf Entwicklungs-, Regulierungs- und Verkaufsmeilensteinen basieren. Ionis wird auch mittlere zweistellige Prozentsätze an Lizenzgebühren auf den jährlichen Nettoumsatz erhalten.
Sapablursen befindet sich derzeit in der Phase 2 der IMPRSSION-Studie und hat 2024 von der FDA die Fast Track- und Orphan Drug-Designation erhalten. Ionis wird die laufende Phase-2-Studie abschließen, während Ono die nachfolgende Entwicklung, regulatorische Einreichungen und Vermarktung übernehmen wird.
- Substantial upfront payment of $280M strengthens IONS cash position
- Potential for additional $660M in milestone payments
- Mid-teen percentage royalties on future sales
- FDA Fast Track and orphan drug designations enhance market potential
- Deal reduces development costs while maintaining revenue potential
- IONS gives up control and commercialization rights for sapablursen
- Future revenue dependent on Ono's development and commercialization success
Insights
Ionis Pharmaceuticals has secured a $280 million upfront payment plus potential milestone payments of up to $660 million and mid-teen percentage royalties in a global licensing deal with Ono Pharmaceutical for sapablursen, its RNA-targeted treatment for polycythemia vera (PV).
This transaction represents a significant financial win for Ionis, with the upfront payment alone constituting approximately
The strategic rationale aligns perfectly with management's stated portfolio prioritization strategy. By monetizing sapablursen while maintaining future economics, Ionis can redirect resources toward their wholly-owned pipeline assets, including their first independent commercial launch currently underway and three additional anticipated launches over the next three years.
Sapablursen's FDA Fast Track designation and orphan drug status enhance its regulatory pathway, potentially accelerating approval timelines and providing market exclusivity - advantages that likely contributed to the deal's attractive terms. For Ono, the acquisition strengthens their hematology portfolio with an asset already in Phase 2 development.
From a financial perspective, this deal significantly enhances Ionis' cash position without diluting shareholders, providing flexibility to execute their commercial strategy while reducing future R&D and commercial expenditure requirements. The milestone structure creates multiple potential future catalysts while the royalty component allows Ionis to participate meaningfully in commercial success without bearing associated costs.
Under the terms of the agreement, Ionis will receive a
“We are pleased to entrust sapablursen to Ono, whose unique capabilities will help maximize its value by ensuring broad access for people living with PV,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “Ionis remains committed to advancing the wholly owned medicines we choose to commercialize ourselves, which includes our first independent launch currently underway and three additional anticipated launches in the next three years. Streamlining our Ionis-owned portfolio provides financial flexibility, supporting our commitment to invest in and focus on our near and mid-term commercial opportunities and generate substantial revenue growth.”
“We are delighted to enter into this collaboration with Ionis Pharmaceuticals, a pioneer in RNA-targeted medicines, for sapablursen. This partnership aligns with our strategy to strengthen our pipeline in hematology. We expect sapablursen to become a new treatment option for PV patients worldwide,” said Toichi Takino, president and COO, Ono.
About Polycythemia Vera (PV)
PV is a rare and potentially life-threatening hematologic disease characterized by the overproduction of red blood cells, which significantly increases the risk of serious blood clots, especially in critical organs like the lungs, heart and brain. Patients with PV also experience severe iron deficiency and commonly have symptoms of fatigue.
About Sapablursen
Sapablursen is designed to reduce the production of TMPRSS6 resulting in increased expression of hepcidin, which is the key regulator of iron homeostasis. By increasing production of hepcidin, sapablursen has the potential to positively impact blood diseases such as PV.
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 select 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.
About Ono Pharmaceutical Co., Ltd.
Ono Pharmaceutical Co., Ltd., headquartered in
For further information visit: https://www.ono-pharma.com/en
Ionis Forward-Looking Statements
This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of sapablursen, 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, 2024, 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.
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Ionis Investor Contact:
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IR@ionis.com
760-603-2331
Ionis Media Contact:
Hayley Soffer
media@ionis.com
760-603-4679
Source: Ionis Pharmaceuticals, Inc.
FAQ
What are the financial terms of the Ionis-Ono sapablursen licensing deal?
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