Ionis Pharmaceuticals Partners With Japan-Based Ono Pharmaceutical For Rare Blood Disease Drug In Over $800 Million Deal

Zinger Key Points

Ionis will receive $280M upfront from Ono, with potential milestone payments of up to $660M and mid-teen percentage royalties on net sales.
Sapablursen, an investigational RNA therapy for polycythemia vera, is in a fully enrolled Phase 2 trial.
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On Tuesday, Ionis Pharmaceuticals Inc IONS and Ono Pharmaceutical Co., Ltd 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 (PV).

PV is a rare hematologic disease characterized by the overproduction of red blood cells, significantly increasing the risk of serious blood clots, especially in critical organs like the lungs, heart and brain.

Sapablursen is currently being evaluated in adults living with PV in the fully enrolled Phase 2 IMPRSSION study.

Also Read: Ionis Pharmaceuticals Q4 Revenue Growth Exceeds Guidance, Forecasts Over $600 Million In 2025 Sales

Sapablursen received FDA Fast Track designation and orphan drug designation in 2024.

Under the agreement, Ionis will receive a $280 million upfront payment, potentially earning up to $660 million 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 completing the ongoing Phase 2 IMPRSSION study, while Ono will be solely responsible for subsequent development, regulatory filings, and commercialization.

“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,” said Brett P. Monia, Ph.D., CEO of Ionis.

Earlier this week, the European Union approved Ionis Pharmaceutical and AstraZeneca plc’s AZN Wainzua (eplontersen) for hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN.

Price Action: At the last check on Wednesday, IONS stock was up 1.24% at $33.54.

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