Ionis expands Otsuka licensing deal in Asia Pacific for hereditary angioedema drug

Ionis Pharmaceuticals, Inc. has entered into a significant license agreement with Otsuka Pharmaceutical Co., Ltd., granting Otsuka exclusive rights to commercialize donidalorsen in the Asia-Pacific region. Donidalorsen is an investigational RNA-targeted prophylactic medicine aimed at treating hereditary angioedema (HAE). Under this agreement, Ionis will oversee the overall development of the drug, while Otsuka will handle region-specific development, regulatory filings, and commercialization efforts in both the Asia-Pacific region and Europe.

Ionis is also preparing to submit a New Drug Application for donidalorsen to the U.S. Food and Drug Administration (FDA) this year. If approved, Ionis plans to independently market the drug to American patients. Additionally, Otsuka is gearing up to present a Marketing Authorization Application to the European Medicines Agency within the same timeframe.

Brett P. Monia, Ph.D., CEO of Ionis, emphasized the shared commitment between Ionis and Otsuka to make donidalorsen accessible to as many HAE patients as possible. This sentiment follows the positive results from Phase 3 trials for donidalorsen, which were recently disclosed. Ionis has also been preparing for the imminent launches of donidalorsen and another drug, olezarsen, designed to treat familial chylomicronemia syndrome in the U.S.

The Asia-Pacific agreement stipulates that Ionis will receive an upfront payment of $20 million, along with potential milestone payments tied to regulatory and sales achievements. Additionally, Ionis stands to earn tiered royalties based on the commercialization success of donidalorsen. This agreement mirrors a previous arrangement between the two companies for the European market. Otsuka brings a wealth of expertise in local and regional regulations and a robust commercial infrastructure, particularly in delivering treatments for rare diseases.

Ionis recently showcased promising data from the Phase 3 OASIS-HAE and OASISplus studies at the 2024 European Academy of Allergy and Clinical Immunology Annual Congress in Valencia, Spain. The results were also published in The New England Journal of Medicine, further underscoring the potential of donidalorsen as a groundbreaking treatment for HAE.

Hereditary angioedema (HAE) is a rare genetic disorder causing severe and recurrent swelling in various body parts, including the hands, feet, genitals, stomach, face, and throat. The condition affects over 20,000 people in the U.S. and Europe. Prophylactic treatment approaches are commonly used in the U.S. to mitigate the frequency and severity of HAE attacks.

Donidalorsen is a Ligand-Conjugated Antisense (LICA) medicine designed to target prekallikrein (PKK), a protein involved in triggering inflammatory mediators responsible for acute HAE attacks. By reducing PKK levels, donidalorsen aims to offer a preventative treatment option for HAE patients.

Ionis Pharmaceuticals has been at the forefront of developing innovative RNA-targeted medicines for three decades. The company boasts a strong pipeline in neurology, cardiology, and other critical healthcare areas. Ionis is committed to advancing RNA therapies and exploring new gene-editing approaches to bring life-changing treatments to patients with serious diseases.