Ono Pharmaceutical Acquires Global Rights to Ionis’ Sapablursen for Polycythemia Vera in $940M Deal

Ono Pharmaceutical has obtained exclusive global rights to develop and commercialize sapablursen, an investigational RNA-targeted medicine for polycythemia vera (PV), from Ionis Pharmaceuticals1 2.

The deal includes a $280 million upfront payment to Ionis, with potential additional payments of up to $660 million based on development, regulatory, and sales milestones, totaling $940 million1 2 3.

Ionis will also receive mid-teen percentage royalties on annual net sales of sapablursen if approved1 2 3.

Sapablursen is currently being evaluated in the fully enrolled Phase 2 IMPRSSION study for adult patients with PV1 6.

The drug received FDA Fast Track designation in January 2024 and orphan drug designation in August 20241 6.

Ionis will complete the ongoing Phase 2 IMPRSSION study, while Ono will be responsible for subsequent development, regulatory filings, and commercialization1 6.

Polycythemia vera is a rare and potentially life-threatening hematologic disease characterized by the overproduction of red blood cells, increasing the risk of serious blood clots1 6.

Sapablursen is designed to reduce the production of TMPRSS6, resulting in increased expression of hepcidin, which regulates iron homeostasis1 6.

The transaction is subject to the requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 19761.

This licensing deal aligns with Ionis' strategy to streamline its portfolio and focus on near and mid-term commercial opportunities2 9.