Ono Pharmaceutical Co., Ltd and Ionis Pharmaceuticals
Type: License agreement
In-Licensee: Ono Pharmaceutical Co., Ltd
Out-Licensor: Ionis Pharmaceuticals, Inc.
Focus: sapablursen
Upfront Payment: $280 million
Royalty received: Mid-teens percentage range on annual net sales
Financial impact: Ono Pharmaceutical Co., Ltd expected revenue growth with sapablursen gaining traction for its potential to address a range of hematologic disorders.
Ono Pharmaceutical Co., Ltd has signed a licensing agreement with Ionis Pharmaceuticals, Inc. for sapablursen, an investigational RNA-targeted therapy intended for the treatment of Polycythemia Vera (PV). The drug is currently being assessed in adult PV patients as part of the fully enrolled Phase 2 IMPRSSION study. Sapablursen received Fast Track designation in January 2024 and orphan drug designation in August 2024 from the U.S. Food and Drug Administration (FDA).
Under the terms of the agreement, Ono Pharmaceutical will receive exclusive global rights to develop and commercialize sapablursen. Ionis Pharmaceuticals will continue to oversee the ongoing Phase 2 IMPRSSION study, while Ono Pharmaceutical will take over responsibility for further development, regulatory submissions, and commercialization efforts. As part of the deal, Ono Pharmaceutical will make an upfront payment of $280 million and may pay up to an additional $660 million based on the achievement of development, regulatory, and commercial milestones. Ionis Pharmaceuticals will also receive royalties in the mid-teens percentage range on annual net sales of sapablursen. The agreement remains subject to the provisions of the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act).
Sapablursen
Sapablursen is an antisense oligonucleotide (ASO) designed to target the mRNA of transmembrane protease serine 6 (TMPRSS6), and is conjugated with N-acetylgalactosamine (GalNAc) to enhance liver-specific delivery. This targeted approach offers potential for regulating TMPRSS6 expression, increasing hepcidin levels, and managing iron levels—addressing iron overload and anemia associated with conditions such as beta thalassemia and myelodysplastic syndromes. When administered subcutaneously, the GalNAc component binds with high affinity to asialoglycoprotein receptors (ASGPRs) on liver cells (hepatocytes). Inside the cell, the ASO inhibits the production of TMPRSS6 protein. Lower TMPRSS6 levels reduce its suppressive effect on the bone morphogenetic protein (BMP)-SMAD signaling pathway, resulting in increased hepcidin production. Hepcidin, a key regulator of systemic iron balance, limits dietary iron absorption and the release of stored iron, ultimately lowering serum iron levels, restoring iron homeostasis, and promoting red blood cell production (erythropoiesis). In diseases such as beta thalassemia and polycythemia vera, low hepcidin levels contribute to iron overload. TMPRSS6 plays a central role in this process by negatively regulating hepcidin, making it a critical therapeutic target for managing iron dysregulation.
Five years' market revenue of Ono Pharmaceutical Co., Ltd
Ono Pharmaceuticals has demonstrated consistent revenue growth over the past five years, reflecting strong business performance and strategic execution. Building on this momentum, the recent licensing agreement with Ionis Pharmaceuticals, Inc. for sapablursen—a promising investigational therapy targeting polycythemia vera and other iron-related disorders—is expected to further strengthen Ono Pharmaceutical’s revenue trajectory. With exclusive global rights to develop and commercialize sapablursen, this deal positions Ono Pharmaceutical to expand its pipeline and capitalize on potential market opportunities in hematology and rare diseases, potentially contributing to continued financial growth in the coming years.
Five years of market revenue of Ono Pharmaceuticals Co., Ltd (2020-2024)
Fig 8: Market revenue of Ono Pharmaceutical Co., Ltd
Market Impact of This Partnership
The treatment landscape for polycythemia vera is undergoing significant transformation, driven by emerging innovations and evolving clinical strategies. Across the seven major markets, the polycythemia vera market is projected to grow from USD 1.9 billion in 2024, with a strong compound annual growth rate (CAGR) anticipated through 2034. This expansion is fueled by the development of novel therapeutic options, enhanced early diagnosis, progress in secondary care, active clinical research, and rising disease awareness.
Among the therapies generating considerable interest is sapablursen, which is gaining traction for its potential to address a range of hematologic disorders, including sickle cell disease and related conditions. Backed by promising clinical trial results and growing recognition within the medical and pharmaceutical communities, sapablursen is emerging as a noteworthy candidate in the evolving blood disorder treatment landscape.
“The polycythemia vera market is projected to grow from USD 1.9 billion in 2024, with a strong compound annual growth rate (CAGR) anticipated through 2034.”