Otsuka and Ionis Pharmaceuticals partner for  Hereditary Angioedema Treatment

Japanese pharmaceutical company Otsuka Pharmaceutical has entered into a significant agreement with California-based Ionis Pharmaceuticals, involving a payment of $65 million for the exclusive marketing rights to Ionis’ hereditary angioedema (HAE) drug candidate, donidalorsen, in the European market. This strategic collaboration is set to address the unmet medical needs of patients suffering from the rare genetic disease.

Hereditary angioedema is a serious genetic disease with symptoms including unpredictable and severe swelling, affecting various body parts, such as the skin, gastrointestinal tract, upper respiratory system, face, and throat. With potentially life-threatening consequences, this disease typically afflicts 1 in 50,000 people.

The terms of the agreement outline that Otsuka will provide an upfront payment of $65 million to Ionis, coupled with potential milestone-related payments. Furthermore, Ionis stands to receive tiered royalties, ranging between 20 and 30 percent, based on the aggregate annual net sales of donidalorsen. 

Ionis will retain responsibility for the non-clinical and clinical development of the drug, while Otsuka will undertake the task of applying for regulatory approval and subsequently commercializing the treatment throughout Europe.

Brett Monia, Chief Executive Officer of Ionis, expressed enthusiasm about the collaboration, citing Otsuka’s track record in delivering rare disease medicines to European patients. Monia emphasized that the agreement aligns with Ionis’ strategic focus on initially concentrating commercialization efforts within the United States.

Makoto Inoue, President and Representative Director of Otsuka Pharmaceutical, underscored Otsuka’s commitment to drug development for rare diseases and highlighted the potential impact of the collaboration in addressing the medical needs of HAE patients in Europe.

Donidalorsen, the investigational ligand-conjugated antisense (LICA) medicine at the core of this collaboration, targets the prekallikrein pathway, pivotal in activating inflammatory mediators linked to acute attacks of hereditary angioedema. In a Phase II open-label study, donidalorsen showcased promising results, reducing the HAE attack rate by an impressive 96 percent over two years, with minimal side effects reported by patients.

Currently, Ionis is advancing two Phase III multicenter studies for donidalorsen: a double-blind, placebo-controlled study as well as an open-label, global study in North America & Europe respectively. Anticipated topline Phase III results for HAE are expected in the first half of the coming year.

In light of this collaboration, Otsuka Pharmaceutical’s upfront payment secures exclusive European licensing rights for donidalorsen. Ionis, in return, retains control over clinical development and potential U.S. launch activities, positioning the companies for a joint effort in bringing this promising therapeutic option to patients in need. 

Analysts view this deal as an incremental positive for Ionis, emphasizing the notable royalty rate and the company’s strategic approach to focusing on rare disease drug launches in the U.S. while seeking international partnerships.

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