Ono pays $280M to license Ionis rare disease drug

Dive Brief:

• Ionis Pharmaceuticals is licensing out another experimental medicine, entering an agreement with Ono Pharmaceutical that offers $280 million upfront and as much as $660 million more for reaching certain development, regulatory and sales goals.
• The drug, sapablursen, is designed to treat polycythemia vera, a rare disease that can cause life-threatening blood clots. Ionis is currently testing sapablursen in a Phase 2 trial and will continue to be responsible for that study.
• Ono will handle development after the completion of the Phase 2 study, as well as regulatory filings and commercialization. If sapablursen reaches the market, Ionis is in line for royalties in the “mid-teen” percentage range, the companies said Tuesday.

Dive Insight:

Ionis has a long history of discovering medicines and then finding partners for them, but recently has expressed interest in developing and commercializing more drugs on its own, taking on additional risk for the possibility of bigger profits. The first test of that new strategy will be the launch of Tryngolza, which was approved in December, for a rare disease that causes dangerous buildups of triglycerides in the body.

The commitment to advancing wholly owned medicines hasn’t changed, Ionis CEO Brett Monia said in a statement. Rather, deals like the Ono agreement allow the company “financial flexibility” to pursue commercial opportunities and revenue growth, he said. Ionis expects to undertake three more independent launches in the next three years, he said.

Sapablursen is an RNA-targeting medicine designed to boost levels of a hormone called hepcidin that plays a key role in regulating iron in the body. Another drug that harnesses hepcidin, Protagonist Therapeutics and Takeda’s rusfertide, recently succeeded in a Phase 3 study.

While sapablursen works in a different way than rusfertide, the successful study helps make the case for targeting hepcidin, William Blair analyst Myles Minter said in a note to clients. And there’s still room for treatment options like sapablursen that may offer more convenient dosing than rusfertide, he said.

The Ionis-Ono deal is “positive for both sides,” Minter said. It gives Ionis cash to continue pursuing the development of wholly owned drugs, while giving Ono an experimental medicine that’s poised to offer an “attractive” option to doctors and patients who have with polycythemia vera, he said.

Source:
https://www.biopharmadive.com/news/ionis-ono-sapablursen-polycythemia-vera-licensing-deal/742269/

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