Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Ono Pharmaceutical Co., Ltd. (Ono) have entered into a new partnership, granting Ono exclusive global rights to develop and commercialize sapablursen, a promising RNA-targeted therapy for polycythemia vera (PV). PV is a rare and potentially life-threatening blood disorder, and sapablursen is currently being evaluated in adults as part of the fully enrolled Phase 2 IMPRSSION study. The U.S. Food and Drug Administration (FDA) recognized the drug’s potential in 2024 by granting it both Fast Track and orphan drug designations.

As part of the agreement, Ionis will receive an upfront payment of $280 million and could earn up to $660 million in additional payments based on key development, regulatory, and sales milestones. The company will also receive royalties in the mid-teen percentage range on future global sales of sapablursen. While Ionis will see the Phase 2 study through to completion, Ono will take the lead on further development, regulatory approvals, and bringing the drug to market. The agreement is still subject to regulatory clearance under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act).

Brett P. Monia, Ph.D., CEO of Ionis, expressed confidence in Ono’s ability to maximize sapablursen’s impact and ensure it reaches patients who need it most. He emphasized Ionis’ focus on advancing its own portfolio of innovative medicines, including its first independent product launch currently underway and three more expected over the next three years. According to Monia, streamlining the company’s efforts allows for greater financial flexibility, enabling Ionis to invest in near- and mid-term opportunities while driving significant revenue growth.

Toichi Takino, president and COO of Ono, shared his enthusiasm for the partnership, calling it an important step in strengthening Ono’s hematology pipeline. He highlighted sapablursen’s potential to become a valuable new treatment option for PV patients around the world, reinforcing Ono’s commitment to advancing innovative therapies.

Source: https://ir.ionis.com/news-releases/news-release-details/ionis-and-ono-announce-global-license-agreement-sapablursen