KalVista Pharmaceuticals Presents New Sebetralstat Data at the Western Society of Allergy, Asthma & Immunology 2025 Annual Meeting
– Claims data show
-Data from KONFIDENT-S show patients on LTP average 1.7 HAE attacks per month and symptom relief for treated attacks in median 1.3 hours -
–Results confirm sebetralstat effective for on-demand treatment of attacks regardless of type of LTP therapy used, including an all-oral regimen–
Raffi Tachdjian, MD, MPH, Associate Clinical Professor of Medicine and Pediatrics in the Division of Allergy and Clinical Immunology at the David Geffen School of Medicine at the University of
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According to a US commercial claims analysis, nearly
40% of HAE patients who initiated LTP had substantial refill gaps in claims over 12 months, with more than half of those discontinuing LTP. - For patients with substantial refill gaps, which increase the likelihood of non-adherence, on-demand claims remained unchanged before and after one year of starting LTP.
“Despite guidelines emphasizing regular assessment of patients using LTP to confirm efficacy and safety of their therapy, these data suggest many patients experience substantial lapses in refilling their LTP, which may reflect non-adherence,” said Dr. Tachdjian. “We saw no decrease in on-demand claims for patients with LTP refill gaps. These findings are important as most HAE patients in the US now receive LTP, and a greater focus on monitoring appears warranted, as LTP effectiveness is not a given. These data may also help to explain why on-demand treatment volumes in HAE have remained steady despite the advent of several effective subcutaneous and oral LTP options since 2017.”
Marc A. Riedl, MD, Professor of Medicine and Clinical Director, US Hereditary Angioedema Association Center at the University of
- Participants receiving LTP treated 382 attacks with sebetralstat (mean 1.7 attacks per month), of which more than half involved the abdomen and/or larynx.
- Sebetralstat enabled early treatment (median 6 minutes) and early symptom relief (median 1.3 hours), and was well-tolerated, regardless of LTP mechanism of action or route of administration.
“Treatment guidelines recommend that patients with HAE should consider treating all attacks regardless of LTP use and should treat attacks as early as possible,” noted Dr. Riedl. “These interim results suggest that, if approved, sebetralstat could be a safe and effective oral on-demand treatment for breakthrough attacks, regardless of severity, location, or type of LTP used.”
Michael E. Manning, MD, allergist-immunologist at Allergy, Asthma and Immunology Associates, Ltd.,
- Participants receiving berotralstat treated 178 attacks with sebetralstat (mean 1.8 attacks per month).
- Sebetralstat enabled early treatment (median 20 minutes), early symptom relief (median 1.3 hours), and was well-tolerated with no increase in gastrointestinal side effects.
“In general, patients prefer oral treatments over injectables, underscoring the strong interest in oral options for HAE,” said Dr. Manning. “These results demonstrate that sebetralstat, when used as an oral on-demand treatment for attacks occurring among patients receiving berotralstat for LTP, enabled rapid treatment and symptom improvement. If approved, sebetralstat could transform the treatment landscape for physicians and patients who prefer to manage HAE without needles.”
“Injectable on-demand treatments have side effects and logistical obstacles that hinder adequate disease control and drive an over-reliance on LTP,” said Paul Audhya, MD, MBA, Chief Medical Officer of KalVista. “LTP does not, however, always yield the anticipated reductions in attacks, possibly due to adherence issues in a chronic lifelong setting. Sebetralstat has the potential to enable early treatment of attacks, thereby halting progression at an early stage and reducing morbidity, even among patients on LTP. By overcoming the barriers imposed by current injectable on-demand therapies, sebetralstat could shift the treatment paradigm and become the foundation of HAE management.”
Links to all presentations can be found on the KalVista website under Publications.
About the KONFIDENT Phase 3 Trial
The KONFIDENT phase 3 clinical trial was a randomized, double-blind, 3-way crossover trial evaluating the safety and efficacy of sebetralstat 300 mg and 600 mg versus placebo for the on-demand treatment of HAE in adult and pediatric patients aged 12 years and older. The trial randomized 136 HAE patients from 66 clinical sites across 20 countries, making it the largest clinical trial ever conducted in HAE. In the trial, participants treated each eligible attack with up to two doses of study drug and treated up to three attacks over the course of the study. The trial included Type I and Type II HAE patients who had at least two documented HAE attacks 90 days prior to randomization and also included patients receiving long-term prophylaxis.
About the KONFIDENT-S Trial
KONFIDENT-S is an open-label extension trial with numerous real-world elements evaluating the long-term safety and efficacy of sebetralstat for the on-demand treatment of HAE attacks in adults and pediatric patients aged 12 years and older with HAE Type I or Type II. KalVista is transitioning ongoing trial participants to a novel oral disintegrating tablet (ODT) formulation to support a planned 2026 sNDA filing. If approved, the ODT formulation would provide people living with HAE with an alternative, novel option for oral, on-demand treatment.
About Sebetralstat
Sebetralstat is an investigational, novel oral plasma kallikrein inhibitor for the treatment of hereditary angioedema (HAE). We have filed multiple regulatory applications seeking approval of sebetralstat as the first oral, on-demand treatment for HAE in individuals aged 12 and older, with ongoing studies exploring its use in children aged 2 to 11. If approved, sebetralstat has the potential to become the foundational therapy for HAE management worldwide.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected. All currently approved on-demand treatment options require either intravenous or subcutaneous administration.
About KalVista Pharmaceuticals, Inc.
KalVista Pharmaceuticals, Inc., is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. Our lead investigational product is sebetralstat, a novel, oral, on-demand treatment for hereditary angioedema (HAE). Sebetralstat is under regulatory review by the
For more information about KalVista, please visit www.kalvista.com or follow us on social media at @KalVista and LinkedIn.
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Jenn Snyder
Vice President, Corporate Affairs
(617) 448-0281
jsnyder@kalvista.com
Ryan Baker
Head, Investor Relations
(617) 771-5001
ryan.baker@kalvista.com
Source: KalVista Pharmaceuticals, Inc.