Ono Pharma secures global rights to Ionis’ Sapablursen for polycythemia vera

JAPAN—Ono Pharmaceutical Co., Ltd. has secured the global rights to develop and commercialise sapablursen, an innovative RNA-targeting therapy developed by Ionis Pharmaceuticals.

This agreement aims to treat polycythemia vera (PV), a rare and potentially life-threatening blood disorder characterized by the overproduction of red blood cells.  PV increases the risk of serious blood clots in critical organs like the lungs, heart, and brain.

Under the agreement, Ono will pay Ionis an upfront payment of US$280 million, with the potential for additional payments of up to US$660 million based on the achievement of specific development, regulatory, and sales milestones.

Furthermore, once sapablursen reaches the market, Ionis will be eligible to receive royalties in the mid-teen percentage range on annual net sales.

This financial arrangement not only provides Ionis with substantial financial benefits but also gives Ono the exclusive rights to develop and commercialize sapablursen worldwide.

Sapablursen is currently being evaluated in a fully enrolled Phase 2 study, known as the IMPRSSION study, which is focused on adults living with PV.

 Ionis will continue to oversee the completion of this ongoing trial, while Ono will take over subsequent development, regulatory filings, and commercialization efforts once the study is concluded.

This collaborative approach ensures that the drug’s development process is streamlined and efficient.

Polycythemia vera is a rare hematologic disease that affects the bone marrow, leading to an excessive production of red blood cells.

This condition significantly increases the risk of blood clots, particularly in critical organs such as the lungs, heart, and brain.

Patients often experience symptoms like headaches, dizziness, and fatigue, although the disease is frequently discovered during routine blood tests.

 Despite its rarity, PV has several FDA-approved treatments, including antimetabolite medications and janus kinase (JAK) inhibitors.

However, there remains a need for innovative therapies that can offer new treatment options for patients.

Sapablursen targets the transmembrane protease serine 6 (TMPRSS6) mRNA, a negative regulator of hepcidin, a hormone crucial for iron regulation in the body.

By increasing hepcidin production, sapablursen has the potential to impact blood diseases like PV positively.

Notably, the FDA granted sapablursen Fast Track and orphan drug designations in 2024, reflecting its potential as a groundbreaking treatment for PV.

If approved, it would be the first PV therapy to target the disease at the RNA level, offering a novel approach to managing this complex condition.

Although sapablursen has shown promise in treating PV, its development has not been without challenges.

 Ionis previously investigated the therapy for beta thalassemia, another blood disorder, but terminated a Phase 2 trial in 2023 due to disappointing mid-phase efficacy data.

However, Ono remains optimistic about sapablursen’s potential in treating PV, aligning with its strategy to strengthen its pipeline in hematology.

The partnership between Ono and Ionis is part of Ono’s broader strategy to enhance its presence in the hematology sector.

Ono’s Chief Operating Officer, Toichi Takino, expressed confidence in sapablursen’s potential to become a new treatment option for PV patients worldwide.

This deal follows Ono’s previous licensing agreement for an antibody-drug conjugate from LigaChem Biosciences, demonstrating Ono’s commitment to expanding its therapeutic offerings.

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