KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), today announced
the submission of a New Drug Application (NDA) for U.S. Food and
Drug Administration (FDA) review of sebetralstat, a novel
investigational oral plasma kallikrein inhibitor for the on-demand
treatment of hereditary angioedema (HAE) attacks in adults and
pediatric patients aged 12 years and older.
“This NDA submission represents a pivotal moment not only for
our company, but for the entire HAE community as we seek to bring
an important therapeutic advancement through the first-ever, oral
on-demand treatment for HAE,” said Ben Palleiko, Chief Executive
Officer of KalVista. “This achievement is the culmination of the
entire KalVista team’s long-term efforts, with the critical support
of people living with HAE, as well as our partnerships with the HAE
scientific community, the HAEA and HAEi patient advocacy
organizations, regulators and other stakeholders, that demonstrate
our commitment to addressing persistent unmet needs for people
living with this rare disease. If approved, we believe sebetralstat
could become a foundational treatment that will transform the way
people living with HAE treat their disease.”
The NDA submission is based on previously disclosed clinical
trial results, including data from the KONFIDENT phase 3 clinical
trial and the KONFIDENT-S extension trial. Sebetralstat met the
primary endpoint in the 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). The median time to beginning of symptom relief was
1.61 hours with sebetralstat 300 mg (CI 1.28, 2.27), 1.79 hours
with sebetralstat 600 mg (CI 1.33, 2.27) and 6.72 hours with
placebo (CI 2.33, >12).
Consistent with previous studies, sebetralstat was
well-tolerated, with a safety profile no different than placebo.
There were no treatment-related serious adverse events (SAEs)
observed. This favorable safety profile has been consistently
observed in all clinical studies for sebetralstat to date.
The FDA has a 60-day filing review period to determine whether
the NDA is complete and accepted for review. The Company currently
anticipates receiving notification from the FDA on the status of
the submission in September. KalVista intends to submit additional
marketing authorization applications to other global health
authorities throughout 2024.
If approved, sebetralstat would be the first oral, on-demand
therapy for people living with HAE. The NDA filing includes
patients aged 12-17 years, whom the Company believes have a
particularly high level of unmet need because of the challenges of
using injectable medications in that age group. KalVista plans to
commence a pediatric trial (KONFIDENT-KID) in Q3 2024 that, if
successful, would enable a future filing to extend this coverage to
patients aged 2-11 years.
KalVista intends to present additional data from the KONFIDENT
and KONFIDENT-S trials at the 2024 Annual Scientific Conference of
the American College of Allergy, Asthma, and Immunology (ACAAI)
Conference on October 24-28, 2024, in Boston, Massachusetts.
About the KONFIDENT Phase 3 Trial
The KONFIDENT phase 3 trial was a randomized, double blind,
3-way crossover clinical trial evaluating the safety and efficacy
of sebetralstat 300 mg and 600 mg versus placebo for the on-demand
treatment of HAE in adults and pediatrics 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.
About the KONFIDENT-S Trial
The KONFIDENT-S trial is an open label extension study
evaluating the long-term safety and efficacy of sebetralstat for
on-demand treatment of HAE attacks in adults and pediatrics aged 12
years and older with HAE Type I or Type II. KalVista is planning to
transition ongoing participants in the trial to an oral
disintegrating tablet (ODT) formulation in Q4 2024 to support a
potential 2026 sNDA approval 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 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 European
Medicines Agency (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 disclosed
positive phase 3 data for the KONFIDENT trial for its oral,
on-demand therapy sebetralstat in February 2024. The Company
expects to file for approval in the UK, Europe, and Japan later in
2024.
For more information about KalVista, please visit
www.kalvista.com.
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 or other international
regulatory agencies, our expectations about safety and efficacy of
our product candidates, our ability to obtain regulatory approvals
for sebetralstat and other candidates in development within our
expected timelines or at all, our success in engaging with
potential commercial partners, the success of any efforts to
commercialize sebetralstat, the ability of sebetralstat and other
candidates in development to treat HAE or other diseases, our
ability to commence pediatric trials of sebetralstat and develop an
ODT formulation, the future progress and potential success of our
oral Factor XIIa program, our ability to reduce spending on
discovery and preclinical activities, and our expectation to become
cash flow positive. 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, 2023, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240618507569/en/
KalVista Pharmaceuticals, Inc. Jarrod Aldom Vice
President, Corporate Communications (201) 705-0254
jarrod.aldom@kalvista.com
Ryan Baker Head, Investor Relations (617) 771-5001
ryan.baker@kalvista.com
KalVista Pharmaceuticals (NASDAQ:KALV)
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