pipeline
Sebetralstat is a novel, oral plasma kallikrein inhibitor and the most advanced investigational medicine in our portfolio of drug candidates for the treatment of hereditary angioedema (HAE).
Discovered by KalVista, sebetralstat is an investigational novel, orally-administered therapy for the on-demand treatment of HAE. Sebetralstat works by targeting the kallikrein-kinin system (KKS) cascade, selectively inhibiting plasma kallikrein and its uncontrolled activity that drive HAE attacks.
Sebetralstat is designed to offer people living with HAE a medication that can be taken as a discreet oral dose to readily treat HAE attacks on-demand, including at the earliest signs before the attack fully develops. All currently approved on-demand treatment options for HAE are administered by injection or infusion. Due to the complexities and treatment burden associated with these current treatments, many people with HAE delay or withhold treatment, undermining treatment efficacy.
In 2022, KalVista initiated the KONFIDENT Phase 3 clinical trial, evaluating the safety and efficacy of sebetralstat as an on-demand therapy for HAE attacks. The KONFIDENT phase 3 trial was a randomized, double blind, event-driven, crossover clinical trial evaluating the efficacy and safety of sebetralstat 300 mg and 600 mg versus placebo for the oral on-demand treatment for hereditary angioedema (HAE). The trial enrolled a total of 136 adult and adolescent HAE patients from 66 clinical sites across 20 countries, making it the largest clinical trial ever conducted in HAE based on number of subjects. In the study, patients self-administered each eligible HAE attack with up to two doses of study drug, and each patient could treat up to three HAE attacks over the course of the study. The trial included type 1 and type 2 HAE patients who had at least two documented HAE attacks within three months prior to enrollment.
We reported positive topline data from the Phase 3 KONFIDENT trial in February 2024, and, we expect to be able to file a New Drug Application (NDA) with the U.S. FDA in the first half of 2024. Sebetralstat has received a Fast Track designation from the U.S. Food and Drug Administration and an approved Pediatric Investigational Plan from the European Medicines Agency (EMA).
