Ionis Pharma expands donidalorsen licensing agreement in Asia Pacific

Ionis Pharmaceuticals has entered into a license agreement with Otsuka Pharmaceutical under which Otsuka obtains exclusive rights across the Asia-Pacific region for donidalorsen, an investigational RNA-targeted prophylactic medicine for hereditary angioedema or HAE. Ionis will maintain primary responsibility for the development of donidalorsen, while Otsuka will be responsible for territory-specific development, regulatory filings and commercialization in the Asia-Pacific region and Europe. Ionis plans to file a New Drug Application with the FDA this year, and will independently launch donidalorsen in the U.S., if approved. Ionis and Otsuka previously announced a licensing agreement for donidalorsen in Europe, and Otsuka is also preparing to submit a Marketing Authorization Application to the European Medicines Agency this year. Under the terms of the Asia-Pacific agreement, Ionis will receive a $20M upfront payment and milestone payments based on achievement of regulatory and sales targets. Ionis is also eligible to earn tiered royalties, with similar economic terms to the previous agreement for Europe. Ionis recently reported positive results from two Phase 3 studies, presented at the 2024 European Academy of Allergy and Clinical Immunology Annual Congress in Valencia, Spain and published in The New England Journal of Medicine.