Kaken, Kalvista partner on HAE treatment sebetralstat in Japan
Kaken gains licensing rights to on-demand therapy
Kaken Pharmaceutical obtained the rights to commercialize sebetralstat, an on-demand treatment for hereditary angioedema (HAE), in Japan under a licensing agreement with the HAE treatment’s developer, Kalvista Pharmaceuticals.
Under the terms of the agreement, Kalvista will be eligible to receive an upfront payment of $11 million, with the possibility of additional payments once certain regulatory and commercial milestones are met, as well as royalties based on sales.
An application seeking sebetralstat’s approval for HAE patients 12 and older is under review in Japan. If approved by Japan’s Ministry of Health, Labour and Welfare, sebetralstat would be the country’s first oral, on-demand therapy.
“We are pleased to partner with Kaken, whose expertise and demonstrated success in the region make them well-suited to work alongside our exceptional team to bring sebetralstat to the HAE community in Japan,” Ben Palleiko, CEO of Kalvista, said in a company press release. “This collaboration is an important part of our strategy to expand the global reach of sebetralstat as we prepare for several commercial launches starting this year.”
In HAE, an enzyme called kallikrein goes into overdrive, resulting in the overproduction of a signaling molecule called bradykinin. High bradykinin levels cause blood vessels to leak fluid into nearby tissues, leading to swelling.
HAE treatment under review in other regions
Sebetralstat is designed to ease the swelling attacks that characterize HAE. It works by blocking the activity of kallikrein in the bloodstream and preventing bradykinin levels from rising too high.
Unlike available on-demand HAE treatments that are administered through into-the-vein or under-the-skin injections, sebetralstat is given as an oral tablet.
Kalvista has submitted applications seeking sebetralstat’s approval in the U.S., the European Union, Australia, Singapore, Switzerland, and the U.K.
Applications were backed by data from the Phase 3 KONFIDENT trial (NCT05259917) and its extension study KONFIDENT-S (NCT05505916) showing that on-demand treatment with sebetralstat rapidly led to symptom relief, with patients starting to see their attack symptoms ease within two hours of taking sebetralstat. In many cases, a single dose of the therapy was sufficient to kickstart symptom relief.
More recent data from KONFIDENT-S showed sebetralstat was also effective in treating swelling attacks occurring in the throat or larynx (voice box), which can be life-threatening as they may impede breathing.
Another Phase 3 trial, KONFIDENT-KID (NCT06467084), is underway to assess sebetralstat’s efficacy and safety in children ages 2 to 11 with HAE. Kalvista recently announced that the study had completed enrollment a year ahead of schedule.
Japan’s health ministry designated sebetralstat an orphan drug, a status that provides financial benefits and faster review to therapies for rare diseases. This designation aims to incentivize the development of treatments that might otherwise be less profitable because of a small market.
Regulatory agencies in the U.S. and EU have also granted orphan drug designation to sebetralstat and agreed to review Kalvista’s applications requesting the therapy’s approval. The U.S. Food and Drug Administration is expected to announce its decision in June.
Palleiko hailed the partnership with Kraken as an important step in making sebetralstat available internationally.
“Our focus remains on delivering a safe and effective oral on-demand therapy that we believe will make a meaningful difference for people living with HAE worldwide,” he said.
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