– Claims data show 40% of patients on long-term
prophylaxis have gaps in prescription refills, leading to greater
on-demand use and higher rate of LTP discontinuation-
-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–
KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), today announced
the presentation of novel data related to long-term prophylaxis and
sebetralstat at the Western Society of Allergy, Asthma &
Immunology (WSAAI) 2025 Annual Meeting taking place in Waimea, HI
from February 9-13, 2025.
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 California, Los Angeles (UCLA), presented data on the Impact
of Long-Term Prophylaxis (LTP) Adherence in Hereditary Angioedema
Patients: Results of a Claims Database Analysis.
- 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
California, San Diego, presented data on the Safety and
Effectiveness of Sebetralstat in Patients with Hereditary
Angioedema Receiving Long-Term Prophylaxis: Interim Analysis from
the KONFIDENT-S Open-Label Study.
- 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., Scottsdale, Arizona and
Past President of WSAAI, presented Sebetralstat for the
Treatment of HAE Attacks in Patients Receiving Berotralstat:
Interim Analysis from the KONFIDENT-S Open-Label Study.
- 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 U.S. FDA, with a
PDUFA goal date of June 17, 2025. In addition, we have completed
Marketing Authorization Applications for sebetralstat to the
European Medicines Agency and multiple other global regulatory
authorities.
For more information about KalVista, please visit
www.kalvista.com or follow us on social media at
@KalVista and LinkedIn.
Forward-Looking Statements This press release contains
"forward-looking" statements within the meaning of the safe harbor
provisions of the U.S. Private Securities Litigation Reform Act of
1995. Forward-looking statements can be identified by words such
as: "anticipate," "intend," "plan," "goal," "seek," "believe,"
"project," "estimate," "expect," "strategy," "future," "likely,"
"may," "should," "will" and similar references to future periods.
These statements are subject to numerous risks and uncertainties
that could cause actual results to differ materially from what we
expect. Examples of forward-looking statements include, among
others, timing or outcomes of communications with the FDA, our
expectations about safety and efficacy of our product candidates
and timing of clinical trials and its results, our ability to
commence clinical studies or complete ongoing clinical studies,
including our KONFIDENT-S and KONFIDENT-KID trials, and to obtain
regulatory approvals for sebetralstat and other candidates in
development, the success of any efforts to commercialize
sebetralstat, the ability of sebetralstat and other candidates in
development to treat HAE or other diseases, and the future progress
and potential success of our oral Factor XIIa program. Further
information on potential risk factors that could affect our
business and financial results are detailed in our filings with the
Securities and Exchange Commission, including in our annual report
on Form 10-K for the year ended April 30, 2024, our quarterly
reports on Form 10-Q, and our other reports that we may make from
time to time with the Securities and Exchange Commission. We
undertake no obligation to publicly update any forward-looking
statement, whether written or oral, that may be made from time to
time, whether as a result of new information, future developments
or otherwise.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20250210755734/en/
Jenn Snyder Vice President, Corporate Affairs (617) 448-0281
jsnyder@kalvista.com
Ryan Baker Head, Investor Relations (617) 771-5001
ryan.baker@kalvista.com
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