RARE Daily

Ionis Enters Licensing Agreement with Otsuka for HAE Drug in Europe

December 19, 2023

Rare Daily Staff

Otsuka Pharmaceutical agreed to license the exclusive European rights to commercialize Ionis Pharmaceuticals’ experimental therapy donidalorsen to prevent hereditary angioedema attacks.

Otsuka brings expertise in delivering rare disease medicines to patients, a robust commercial infrastructure, and deep knowledge of regional and local regulations across European countries.

Under the agreement, Ionis will receive a $65 million upfront payment and milestone payments based on achievement of regulatory and sales targets. Ionis is also eligible to earn tiered royalties ranging from 20 to 30 percent (based on aggregate annual net sales). Otsuka will be responsible for European regulatory filings and commercialization. Ionis plans to independently launch donidalorsen in the United States, if approved, as part of the company’s strategy to deliver wholly owned medicines to patients.

HAE is a rare and life-threatening genetic disease characterized by unpredictable and frequently severe swelling of the skin, gastrointestinal (GI) tract, upper respiratory system, face, and throat, which can be life-threatening. HAE is estimated to affect more than 20,000 patients in the U.S. and Europe. In the U.S., doctors frequently use prophylactic treatment approaches to prevent and reduce the severity of HAE attacks in patients.

Donidalorsen is an investigational LIgand-Conjugated Antisense (LICA) medicine designed to target the prekallikrein, or PKK, pathway. PKK plays an important role in activating inflammatory mediators associated with acute attacks of hereditary angioedema (HAE). By reducing the production of PKK, donidalorsen could be an effective prophylactic approach to preventing HAE attacks.

Ionis recently reported two-year results from the phase 2 open-label extension trial showing that donidalorsen treatment resulted in a 96 percent overall sustained mean reduction from baseline in HAE attack rates and was recently granted Orphan Drug designation in the United States.

“This agreement is aligned with our strategy to initially focus our commercialization efforts on the U.S. market,” said Brett Monia, Ionis CEO. ” We are encouraged by the strong product profile of donidalorsen to date and look forward to reporting pivotal topline phase 3 donidalorsen results in HAE in the first half of next year.”

Photo: Brett Monia, Ionis CEO

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