Otsuka acquires Asia-Pacific rights to HAE treatment donidalorsen

Developer Ionis to seek U.S. approval this year

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Otsuka Pharmaceutical has obtained exclusive rights to commercialize donidalorsen for hereditary angioedema (HAE) in the Asia-Pacific region.

The deal is an extension of a previous license agreement that granted Otsuka the rights to commercialize the therapy in Europe. Ionis Pharmaceuticals, the therapy’s original developer, retains primary responsibility for developing donidalorsen.

Both companies will file applications seeking regulatory approval of donidalorsen as a prophylactic (preventive) treatment for HAE in the U.S. and Europe this year, following positive data from the Phase 3 trials OASIS-HAE and OASISplus.

“Ionis and Otsuka share a steadfast commitment to bring donidalorsen to as many people living with HAE as possible,” Brett P. Monia, PhD, CEO of Ionis, said in a company press release. “We look forward to working alongside Otsuka to advance regulatory discussions across Europe and the Asia-Pacific region based on our positive Phase 3 results for donidalorsen, which were presented late last month.”

In HAE, abnormally high levels of a signaling molecule called bradykinin cause blood vessels to widen and become more permeable, allowing fluid to leak out. As a result, patients experience swelling attacks. Bradykinin is produced from precursor molecules with the help of an enzyme called kallikrein.

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Trial shows HAE treatment reduces attacks

Donidalorsen is an antisense oligonucleotide — a short, lab-made molecule made of genetic material — designed to lower the production of prekallikrein, a kallikrein precursor. The treatment is administered once monthly, via a subcutaneous (under-the-skin) injection.

In the OASIS-HAE trial (NCT05139810), treatment with donidalorsen, either once every four weeks or once every eight weeks for about six months, was associated with significant reductions in HAE attack rates compared with a placebo.

Reductions in HAE attack rates seen in OASIS-HAE were sustained for up to a year in patients who entered in long-term OASISplus study (NCT05392114) after completing the main trial. 

A group of 64 HAE patients who had been treated for at least three months with other preventive treatments, such as C1 esterase inhibitors, Orladeyo (berotralstat), or Takhzyro (lanadelumab), were included in OASISplus study. In a little more than four months, these patients saw their mean monthly attack rates drop by a 62% relative to their previous treatment.

The new license agreement gives Ionis a $20 million upfront payment. The company is also eligible to receive additional milestone payments tied to the achievement of certain regulatory, and commercial targets, as well as royalties.