Otsuka pays $65M cash for European rights to Ionis’ phase 3 rare disease med

Otsuka Pharmaceutical is paying $65 million upfront for exclusive European licensing rights to Ionis Pharmaceutical’s late-phase investigational treatment for a rare genetic disease known as hereditary angioedema.

California-based Ionis will also have the chance to earn additional milestone payments and tiered royalties ranging from 20% to 30%, according to the companies.

The deal centers around donidalorsen, a prophylactic treatment that recently snagged an orphan drug designation from the FDA. Hereditary angioedema is a life-threatening genetic disease characterized by unpredictable swelling.

Otsuka now has exclusive rights to European regulatory filings and commercialization activities for donidalorsen, formerly known as IONIS-PKK-LRx, while Ionis will maintain responsibility for clinical development and potential U.S. launch activities. Ionis said it plans to share topline data from a phase 3 trial evaluating the ligand-conjugated antisense medicine in the first half of 2024.

William Blair analysts dubbed the deal as “an incremental positive for Ionis,” citing the notable royalty rate in a Dec. 18 note.

The analyst group called Ionis’ move to focus on its own rare disease drug launch efforts in the U.S. while seeking partners outside of the country as “a prudent use of resources,” and that it sees “sound commercial potential for donidalorsen.”