Ionis Pharmaceuticals (IONS) and Ono Pharmaceutical announced that the two companies have entered into a license agreement in which Ono obtains exclusive global rights for the development and commercialization of sapablursen, an investigational RNA-targeted medicine for polycythemia vera, or PV, a rare and potentially life-threatening hematologic disease. Sapablursen is currently being evaluated in adults living with PV in the fully enrolled Phase 2 IMPRSSION study. Sapablursen was granted Fast Track designation and orphan drug designation in 2024 by the FDA. Under the terms of the agreement, Ionis will receive a $280M upfront payment, with the potential to earn up to $660M in additional payments based on the achievement of development, regulatory and sales milestones. Ionis is also eligible to earn royalties in the mid-teen percentage range on annual net sales of sapablursen. Ionis will be responsible for the completion of the ongoing Phase 2 IMPRSSION study, while Ono will be solely responsible for subsequent development, regulatory filings and commercialization. The transaction is subject to the requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 1976.
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