Ionis Pharmaceuticals Strikes $940M Licensing Deal with Ono for Polycythemia Vera Drug

Ionis Pharmaceuticals has entered into a significant licensing agreement with Japanese firm Ono Pharmaceutical for its investigational antisense oligonucleotide drug, sapablursen. The deal, potentially worth up to $940 million, grants Ono exclusive global rights to develop and commercialize the drug candidate for polycythemia vera, a rare blood disease.

Deal Structure and Financial Terms

Under the terms of the agreement, Ionis will receive an upfront payment of $280 million from Ono. The deal also includes the potential for up to $660 million in additional milestone payments tied to development, regulatory, and commercial achievements. Should sapablursen reach the market, Ionis stands to earn mid-teen percentage royalties on sales.

The licensing agreement, which is still subject to anti-trust clearance, represents a strategic move for both companies. For Ionis, it provides substantial funding and "financial flexibility" to pursue its commercial opportunities and revenue growth, according to CEO Brett Monia. Ono, in turn, gains access to a promising candidate in the competitive rare disease market.

Sapablursen: Mechanism and Clinical Progress

Sapablursen is designed to treat polycythemia vera, a rare blood disorder characterized by the overproduction of red blood cells. The condition can lead to severe complications, including life-threatening blood clots, organ damage, and iron deficiency.

The drug works by lowering the expression of the TMPRSS6 gene, which in turn boosts the production of hepcidin, a protein crucial for iron homeostasis regulation. By improving hepcidin expression, sapablursen aims to alleviate the severity of polycythemia vera.

Currently, sapablursen is being evaluated in the Phase II IMPRSSION study, which completed enrollment late last year. Topline data from this trial are expected later this year. As part of the licensing agreement, Ionis will complete the IMPRSSION trial before handing over further development responsibilities to Ono.

Strategic Implications and Market Outlook

This deal aligns with Ionis' broader strategy of partnering select assets while also developing wholly owned medicines. The company expects to launch three more independent products in the next three years, including the recently approved Tryngolza for a rare triglyceride disorder.

The success of sapablursen could have significant implications for the treatment of polycythemia vera. While another hepcidin-targeting drug, rusfertide (developed by Protagonist Therapeutics and Takeda), recently showed positive results in a Phase 3 study, analysts believe there is still room for sapablursen in the market, potentially offering more convenient dosing options.

William Blair analyst Myles Minter views the Ionis-Ono deal as "positive for both sides," noting that it provides Ionis with capital to fund its internal pipeline while giving Ono an "attractive" option for polycythemia vera patients and physicians.

As the pharmaceutical industry continues to focus on rare diseases and innovative treatment modalities, deals like this underscore the ongoing interest in RNA-targeting therapies and the potential for significant returns on investment in the rare disease space.