KalVista's Sebetralstat: Successful Phase 3 KONFIDENT Trial for Hereditary Angioedema

KalVista Pharmaceuticals, a company dedicated to developing innovative pharmaceuticals, has reported encouraging outcomes from the phase 3 KONFIDENT clinical trial. The trial evaluated sebetralstat, a potential oral treatment for hereditary angioedema (HAE), a rare and often debilitating genetic disorder. The study, which was the largest of its kind in HAE, included a diverse patient group and demonstrated that sebetralstat significantly outperformed placebo in providing rapid symptom relief.

The KONFIDENT trial was a comprehensive, randomized, double-blind, crossover study that enrolled 136 HAE patients from 20 countries. The trial's design allowed for a robust assessment of sebetralstat's efficacy and safety in treating acute HAE attacks. The results showed that patients who took 300 mg or 600 mg of sebetralstat experienced a faster onset of symptom relief compared to those on placebo, with a median time of 1.61 hours for 300 mg and 1.79 hours for 600 mg, versus 6.72 hours for placebo.

Sbetralstat was found to be well-tolerated, with a safety profile comparable to that of placebo. The rate of treatment-related adverse events was low, with no serious adverse events attributed to the drug. This is a significant finding, as it suggests that sebetralstat could offer a safe and effective treatment option for HAE patients.

The trial's success is a major milestone for KalVista Pharmaceuticals and the HAE community. The company is planning to submit a new drug application for sebetralstat to the U.S. FDA in the first half of 2024, with further submissions planned for the EU and Japan later in the year. If approved, sebetralstat would be the first oral, on-demand therapy for HAE, offering a new and potentially life-changing treatment option for patients.

The KONFIDENT trial's principal investigator, Dr. Danny Cohn, highlighted the significance of the results, stating that sebetralstat's rapid symptom relief could transform the management of HAE. Dr. Marc A. Riedl, another investigator, emphasized the potential value of an oral, on-demand treatment in reducing the burden of current injectable treatments.

KalVista Pharmaceuticals is committed to advancing the treatment of HAE and other diseases through its portfolio of oral, small molecule protease inhibitors. The company's positive phase 3 data for sebetralstat is a testament to its ongoing efforts to meet significant unmet medical needs.

The KONFIDENT trial's detailed findings will be presented at the American Academy of Allergy Asthma and Immunology's annual meeting in February 2024, providing further insights into the potential of sebetralstat as a groundbreaking treatment for HAE.