Pharma Focus Asia

Ionis and Otsuka Expand Licensing Agreement for Hereditary Angioedema Drug Donidalorsen in Asia Pacific

Wednesday, June 19, 2024

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has finalized a licensing agreement with Otsuka Pharmaceutical Co., Ltd. (Otsuka) granting Otsuka exclusive rights to donidalorsen in the Asia-Pacific region. Donidalorsen is an investigational RNA-targeted therapy intended to prevent hereditary angioedema (HAE) attacks. Ionis will continue to oversee the development of donidalorsen, while Otsuka will be responsible for its development, regulatory approvals, and commercialization in the Asia-Pacific region and Europe.

Ionis plans to submit a New Drug Application to the U.S. Food and Drug Administration (FDA) this year and aims to launch donidalorsen independently in the U.S. if it receives approval. An earlier agreement with Otsuka already covers Europe, where Otsuka is getting ready to submit a Marketing Authorization Application to the European Medicines Agency.

"Ionis and Otsuka are committed to making donidalorsen accessible to as many patients with HAE as possible," stated Brett P. Monia, Ph.D., CEO of Ionis. "We look forward to working with Otsuka on the regulatory approvals in Europe and the Asia-Pacific region following our favorable Phase 3 trial results for donidalorsen, which were recently presented. In the U.S., we have established a strong commercial infrastructure in preparation for the upcoming launches of olezarsen for familial chylomicronemia syndrome and donidalorsen for HAE."

Under the terms of the agreement for the Asia-Pacific region, Ionis will receive a $20 million upfront payment, along with milestone payments based on achieving certain regulatory and sales milestones. Ionis is also eligible for tiered royalties, mirroring the economic terms of the earlier agreement for Europe. Otsuka's deep understanding of regional regulatory requirements and its expertise in bringing rare disease treatments to market will be valuable in this collaboration.

Ionis has reported positive results from the Phase 3 OASIS-HAE and OASISplus studies, which were presented at the 2024 European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress and published in The New England Journal of Medicine.

Hereditary Angioedema (HAE) is a rare genetic disorder that causes recurring episodes of severe swelling in various body parts, such as the hands, feet, genitals, stomach, face, and throat. It affects over 20,000 patients in the U.S. and Europe. Preventive treatments are commonly used in the U.S. to prevent and reduce the severity of HAE attacks.

Donidalorsen is an experimental Ligand-Conjugated Antisense (LICA) therapy designed to target prekallikrein (PKK), a protein that triggers the inflammation associated with HAE attacks. By lowering PKK levels, donidalorsen may offer an effective preventive treatment for these attacks.



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