– If approved, sebetralstat will be the first,
oral on-demand treatment for HAE –
– FDA PDUFA goal date of June 17, 2025 –
KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), today announced
that the U.S. Food and Drug Administration (FDA) has accepted its
New Drug Application (NDA) for sebetralstat, a novel,
investigational oral plasma kallikrein inhibitor for the on-demand
treatment of hereditary angioedema (HAE) attacks in adult and
pediatric patients aged 12 years and older. The FDA has set a
Prescription Drug User Fee Act (PDUFA) goal date of June 17, 2025.
If approved, sebetralstat would be the first oral, on-demand
treatment for HAE in adult and pediatric patients aged 12 years and
older. The FDA is not currently planning to hold an advisory
committee meeting to discuss the application.
“We are thrilled with the FDA’s acceptance of our NDA for
sebetralstat as it moves us one step closer to bringing a
potentially transformative therapy to the HAE community,” said Ben
Palleiko, Chief Executive Officer at KalVista. “We understand that
people living with HAE and their families carry a tremendous burden
every day as they don’t know when the next attack may occur or if
the attack could cause life-threatening consequences. The
compelling data included in our NDA package show that sebetralstat
has the potential to significantly alter the way people treat and
manage their disease. Given that it could be the first, oral
on-demand treatment for HAE, we continue to receive strong support
and hear a sense of urgency among healthcare providers, advocates,
patients and their families for sebetralstat. I am proud of the
team at KalVista for their dedication to achieving this milestone
and deeply grateful for the support of patients living with HAE,
their families, the HAE scientific community, and the HAEA and HAEi
patient advocacy organizations.”
The NDA submission was supported by previously disclosed
results, including data from the KONFIDENT phase 3 clinical trial
and ongoing KONFIDENT-S open label extension trial. Sebetralstat
met the primary endpoint for its phase 3 trial with both 300 mg and
600 mg formulations achieving the beginning of symptom relief
significantly faster than placebo (p<0.0001 for 300 mg, p=0.0013
for 600 mg) and was well-tolerated, with a safety profile similar
to placebo. In KONFIDENT-S, sebetralstat has enabled patients to
treat attacks early with a median time from attack onset to
treatment of 9 minutes, demonstrated a consistent safety and
efficacy profile with KONFIDENT, and included a median time to
beginning of symptom relief for laryngeal attacks of 1.3 hours.
KalVista’s KONFIDENT-KID clinical trial, designed to evaluate
the safety and efficacy of sebetralstat in a pediatric population
aged 2-11 years, was initiated ahead of schedule in June 2024 and
has since started dosing patients.
In addition to the NDA acceptance, KalVista recently announced
that the European Medicines Agency (EMA) validated the submission
of the Marketing Authorization Application (MAA) for sebetralstat.
KalVista expects to file for approval in the UK, Japan, and other
countries later in 2024.
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 a total of 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 1 and type 2 HAE patients who had at least two
documented HAE attacks in 90 days prior to randomization, including
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 on-demand treatment of HAE attacks in adults and
pediatric patients aged 12 years and older with HAE Type I or Type
II. KalVista plans to transition ongoing participants in the trial
to an oral disintegrating tablet (ODT) formulation in Q4 2024 to
support a planned 2026 sNDA filing of this additional formulation.
If approved, the ODT formulation would provide people living with
HAE an alternative, novel option for oral, on-demand treatment.
About the KONFIDENT-KID Trial
KONFIDENT-KID is an open label trial enrolling approximately 24
children aged 2 to 11 years across seven countries in North
America, Europe and Asia. KONFIDENT-KID will collect safety,
pharmacokinetic, and efficacy data for each patient for up to one
year and will feature a proprietary pediatric oral disintegrating
tablet (ODT) formulation of sebetralstat. If approved, sebetralstat
would be the first oral on-demand therapy for this age group, and
only the second FDA-approved on-demand therapy of any type for this
population.
About Sebetralstat
Discovered and developed entirely by the scientific team at
KalVista, sebetralstat is a novel, investigational oral plasma
kallikrein inhibitor for the on-demand treatment of hereditary
angioedema (HAE). Sebetralstat received Fast Track and Orphan Drug
Designations from the U.S. FDA, as well as Orphan Drug Designation
and an approved Pediatric Investigational Plan from the EMA.
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 location
affected. All currently approved on-demand treatment options
require either intravenous or subcutaneous administration.
About KalVista Pharmaceuticals, Inc.
KalVista Pharmaceuticals, Inc. is a global pharmaceutical
company focused on the development and delivery of oral medicines
for diseases with significant unmet need. KalVista announced
positive phase 3 data from the KONFIDENT trial for its oral,
on-demand therapy, sebetralstat for HAE in February 2024. The
Company’s NDA for sebetralstat has been accepted by the FDA with a
PDUFA goal date of June 17, 2025. In addition, KalVista received
validation of its MAA from the EMA in August 2024. KalVista expects
to file for approval in the UK, Japan, and other countries later in
2024.
For more information about KalVista, please visit
www.kalvista.com or follow 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/20240903673442/en/
Media: Jenn Snyder Vice President, Corporate Affairs
(857) 356-0479 jennifer.snyder@kalvista.com
Investors: Ryan Baker Head, Investor Relations (617)
771-5001 ryan.baker@kalvista.com
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