Ono Pharmaceutical has signed a licensing agreement with Ionis Pharmaceuticals to acquire exclusive global rights for the development and commercialisation of sapablursen.
Sapablursen is an investigational RNA-targeted medicine designed for treating polycythemia vera (PV), a rare haematologic condition marked by excessive red blood cell production.
Under the licensing agreement, Ono will obtain exclusive rights to develop and commercialise sapablursen worldwide.
Ionis will receive an upfront payment of $280m and is eligible for additional payments of up to $660m based on achieving development, regulatory, and sales milestones.
In addition, Ono will pay Ionis royalties in the mid-teens on annual net sales of sapablursen.
The licensing deal is subject to the Hart-Scott-Rodino Antitrust Improvements Act.
ONO president and COO Toichi Takino said: “We are delighted to enter into this collaboration with Ionis Pharmaceuticals, a pioneer in RNA-targeted medicines, for sapablursen.
“This partnership aligns with our strategy to strengthen our pipeline in haematology. We expect sapablursen to become a new treatment option for PV patients worldwide.”
The drug received the US Food and Drug Administration Fast Track designation in January 2024 and orphan drug designation in August 2024.
It is currently being developed in the Phase 2 IMPRSSION study in adult PV patients.
Ionis will complete the ongoing Phase 2 IMPRSSION study, while Ono will be responsible for further development, regulatory submissions, and commercialisation efforts.
Ionis CEO Brett Monia said: “We are pleased to entrust sapablursen to Ono, whose unique capabilities will help maximise its value by ensuring broad access for people living with PV.
“Ionis remains committed to advancing the wholly owned medicines we choose to commercialise 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.”
Recently, Ionis and AstraZeneca secured the EC approval for Wainzua (eplontersen) to treat hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults with polyneuropathy.
