Otsuka and Ionis enter into licensing agreement covering Europe for hereditary angioedema
LONDON, UK, 19 December 2023 – Otsuka Pharmaceutical Europe Ltd. (Otsuka) today announces that its parent company Otsuka Pharmaceutical Co., Ltd. and Ionis Pharmaceuticals, Inc. (Ionis), a leader in RNA-targeted therapy based in California, have entered into a licence agreement for Otsuka to acquire exclusive marketing rights to the Ionis hereditary angioedema (HAE) drug candidate, donidalorsen (generic name), in Europe.
Donidalorsen is an investigational ligand-conjugated antisense (LICA) medicine designed to target the prekallikrein, or PKK, pathway.1 PKK plays an important role in activating inflammatory mediators associated with acute attacks of hereditary angioedema.2 . The Phase 2, open-label, extension study demonstrated donidalorsen reduced HAE attack rate by a mean of 96%, from 2.70 attacks per month to 0.06 attacks per month over 2 years with treatment being well tolerated.3,4 Ionis is currently conducting two Phase 3 multicentre studies; a double-blind, placebo-controlled study in North America and Europe and an open-label, global study. Donidalorsen has received Orphan Drug Designation in the US in 2023.
Makoto Inoue, president and representative director of Otsuka Pharmaceutical Co., Ltd. commented, “Otsuka is committed to the development of drugs for rare diseases such as autosomal dominant polycystic kidney disease (ADPKD). Through this collaboration with Ionis, if regulatory approval is received, we look forward to bringing donidalorsen to patients in Europe to address the unmet medical needs of patients with HAE.”
Under the terms of the agreement, Otsuka will pay Ionis an upfront payment of $65 million, plus payments based on achievement of regulatory and sales milestones. Otsuka will apply for regulatory approval and exclusively commercialise the product in Europe.
“We are excited to collaborate with Otsuka given their proven results in bringing rare disease medicines to patients in Europe,” said Brett P. Monia, Ph.D., Ionis chief executive officer. “This agreement is aligned with our strategy to initially focus our commercialisation efforts on the US market. We are encouraged by the product profile of donidalorsen to date and look forward to reporting pivotal topline Phase 3 results in HAE in the first half of next year.”
About hereditary angioedema
HAE is a rare, autosomal-dominant genetic disease characterised by unpredictable and frequently severe swelling of the skin, gastrointestinal (GI) tract, upper respiratory system, face, and throat, which can be life-threatening.5,6 The disease onset age for most cases occurs during the teenage years, and the worldwide prevalence of this rare disease is estimated to be 1 in 50,000.5,7
About Otsuka
Otsuka Pharmaceutical is a global healthcare company with the corporate philosophy “Otsuka-people creating new products for better health worldwide”. Otsuka researches, develops, manufactures, and markets innovative products, focusing on pharmaceutical products to meet unmet medical needs and nutraceutical products for the maintenance of everyday health. In pharmaceuticals, Otsuka is a leader in the challenging area of mental health and has research programmes in several under-addressed diseases including tuberculosis, a significant global public health issue.
Otsuka Europe employs around 550 people and focuses on psychiatric and neurologic disorders, infectious disease, nephrology, oncology, and digital medicines.
Otsuka Pharmaceutical Europe Ltd. is a part of Otsuka Pharmaceutical Company, Ltd., a subsidiary of Otsuka Holdings Co., Ltd. headquartered in Tokyo, Japan.
The Otsuka group of companies employed 47,000 people worldwide with consolidated sales of approximately €12.4 billion and a spend of €1.67 billion on research and development in 2022. For further information on Otsuka, please visit www.otsuka-europe.com.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted therapy, pioneering new markets and changing standards of care. Ionis currently has four marketed medicines and a promising late-stage pipeline highlighted by cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become the leader in genetic medicine, utilising a multi-platform approach to discover, develop and deliver life-transforming therapies.
MEDIA CONTACT
OTSUKA PHARMACEUTICAL EUROPE LTD.
Alison Ross
Otsuka Pharmaceutical Europe Ltd.
+44 (0)7768 337 128
References
- Donidalorsen. Ionis Pharmaceuticals, Inc. Available online: https://www.ionispharma.com/medicines/ionis-pkk-l/. Last accessed: December 2023.
- Schmaier AH. Plasma Prekallikrein: Its Role in Hereditary Angioedema and Health and Disease. Front Med (Lausanne). 2018 Jan 25;5:3.
- Cohn DM, Newman KB, Bordone L, et al. Two-Year Analysis of the Donidalorsen Phase 2 Open-Label Extension Study in Patients With Hereditary Angioedema. E-poster presented at the American College of Allergy, Asthma & Immunology 2023 Annual Scientific Meeting; November 9–13; Anaheim, CA. Available online: https://epostersonline.com/acaai2023/poster/p053?view=true. Last accessed: December 2023.
- ClinicalTrials.gov. An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema. NCT04307381. Available online: https://clinicaltrials.gov/study/NCT04307381. Last accessed: December 2023.
- Ghazi A, Grant JA. Hereditary angioedema: epidemiology, management, and role of icatibant. Biologics. 2013;7:103-113
- Valerieva A, Nedeva D, Yordanova V, et al. Therapeutic management of hereditary angioedema: past, present, and future. Balkan Med J. 2021 Mar;38(2):89-103.
- Maurer M, Magerl M, Betschel S, et al. The international WAO/EAACI guideline for the management of hereditary angioedema-The 2021 revision and update. Allergy. 2022 Jul;77(7):1961-1990.
