Prescription Drug User Fee Act (PDUFA) action
date is September 26, 2024
If approved, KarXT would represent the first
new pharmacological approach to treating schizophrenia in several
decades
The application is supported by positive data
from the EMERGENT clinical trial program showing KarXT is
associated with significant improvements in schizophrenia
symptoms
PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the
"Company"), a clinical-stage biotherapeutics company, noted today
that its Founded Entity, Karuna Therapeutics, Inc. (Nasdaq: KRTX)
("Karuna") announced the U.S. Food and Drug Administration (FDA)
has accepted its New Drug Application (NDA) for KarXT
(xanomeline-trospium) for the treatment of schizophrenia in adults.
The application has been granted a Prescription Drug User Fee Act
(PDUFA) date of September 26, 2024.
The NDA submission is supported by efficacy and long-term safety
data from the EMERGENT program, the clinical program evaluating
KarXT as a treatment for schizophrenia. The EMERGENT program
includes the three completed positive EMERGENT-1, EMERGENT-2, and
EMERGENT-3 trials evaluating the efficacy and safety of KarXT
compared to placebo, and the EMERGENT-4 and EMERGENT-5 trials
evaluating the long-term safety of KarXT.
PureTech is a founder of Karuna and co-inventor of the KarXT
program. If approved, KarXT will be the third therapeutic candidate
to be taken from inception at PureTech to FDA regulatory
approval.
The full text of the announcement from Karuna is as follows:
Karuna Therapeutics Announces U.S. Food and
Drug Administration Accepts New Drug Application for KarXT for the
Treatment of Schizophrenia
Prescription Drug User Fee Act (PDUFA) action
date is September 26, 2024
If approved, KarXT would represent the first
new pharmacological approach to treating schizophrenia in several
decades
The application is supported by positive data
from the EMERGENT clinical trial program showing KarXT is
associated with significant improvements in schizophrenia
symptoms
BOSTON -- Nov. 29, 2023 -- Karuna Therapeutics, Inc.
(NASDAQ: KRTX), a biopharmaceutical company driven to discover,
develop, and deliver transformative medicines for people living
with psychiatric and neurological conditions, today announced the
U.S. Food and Drug Administration (FDA) has accepted its New Drug
Application (NDA) for KarXT (xanomeline-trospium) for the treatment
of schizophrenia in adults. The application has been granted a
Prescription Drug User Fee Act (PDUFA) date of September 26,
2024.
“We are pleased the NDA for KarXT has been accepted, and we look
forward to working with the FDA during the review process,” said
Bill Meury, president and chief executive officer of Karuna
Therapeutics. “There is a significant need for new treatment
options for serious mental illness. If approved, KarXT could be one
of the more important new product introductions in neuropsychiatry
by providing a novel pharmacological approach for the treatment of
schizophrenia.”
“Schizophrenia’s disabling symptoms pose significant challenges
to navigating crucial aspects of life, including developing
relationships, maintaining employment, and securing safe housing,”
said Gordon Lavigne, M.Ed., chief executive officer, Schizophrenia
& Psychosis Action Alliance. “Diagnosis marks the beginning of
an often long and tiresome search for effective and tolerable
treatment options. The nature and magnitude of side effects often
play a pivotal role in whether someone continues treatment, which
is often crucial to minimize the risk of relapse and realize the
life-altering benefits of long-term treatment. Potential approval
of a pharmacologically distinct treatment option would be a welcome
innovation for people living with schizophrenia.”
The NDA submission is supported by efficacy and long-term safety
data from the EMERGENT program, the clinical program evaluating
KarXT as a treatment for schizophrenia. The EMERGENT program
includes the three completed positive EMERGENT-1, EMERGENT-2, and
EMERGENT-3 trials evaluating the efficacy and safety of KarXT
compared to placebo, and the EMERGENT-4 and EMERGENT-5 trials
evaluating the long-term safety of KarXT.
In all three placebo-controlled trials, KarXT met its primary
endpoint, demonstrating statistically significant and clinically
meaningful improvements in symptoms of schizophrenia compared to
placebo as measured by Positive and Negative Syndrome Scale (PANSS)
total score. KarXT was found to be generally well tolerated, with
the most common adverse events being cholinergic in nature and mild
to moderate in severity. Notably, KarXT was not associated with
common side effects of currently available antipsychotics,
including weight gain, somnolence, and movement disorders.
“KarXT focuses on a novel pathway through muscarinic receptors
to indirectly modulate dopamine signaling in key brain circuits,
and in clinical trials completed to date KarXT has demonstrated the
much-needed combination of strong tolerability and clinically
meaningful symptom reduction,” remarked Rishi Kakar, M.D., chief
scientific officer and medical director of Segal Trials, and
investigator in the EMERGENT program. “This decision by the FDA
marks an important step in working toward a new chapter in the
standard of care for those facing the immense, daily struggle of
this serious mental illness.”
About KarXT
KarXT (xanomeline-trospium) is an investigational muscarinic
antipsychotic in development for the treatment of schizophrenia and
psychosis related to Alzheimer’s disease. Through its novel
mechanism of action, KarXT acts as a dual M1/M4 muscarinic
acetylcholine receptor agonist in the central nervous system, which
is thought to improve positive, negative, and cognitive symptoms of
schizophrenia. Unlike existing treatments, KarXT does not directly
block dopamine receptors, representing a potential new approach to
treating schizophrenia.
About Schizophrenia
Schizophrenia is a persistent and often disabling mental illness
impacting how a person thinks, feels, and behaves, and affects
nearly 24 million people worldwide, including 2.8 million people in
the U.S. It is characterized by three symptom domains: positive
symptoms (hallucinations and delusions), negative symptoms
(difficulty enjoying life and withdrawal from others), and
cognitive impairment (deficits in memory, concentration, and
decision-making). In part due to limitations with current
treatments, people living with schizophrenia often struggle to
maintain employment, live independently, and manage relationships.
While current treatments can be effective in managing select
symptoms, approximately 30% of people do not respond to therapy,
with an additional 50% experiencing only a partial improvement in
symptoms or unacceptable side effects.
About Karuna
Karuna Therapeutics is a biopharmaceutical company driven to
discover, develop, and deliver transformative medicines for people
living with psychiatric and neurological conditions. At Karuna, we
understand there is a need for differentiated and more effective
treatments that can help patients navigate the challenges presented
by serious mental illness. Utilizing our extensive knowledge of
neuroscience, we are harnessing the untapped potential of the brain
in pursuit of novel pathways to develop medicines that make
meaningful differences in peoples’ lives. For more information,
please visit www.karunatx.com.
Forward-Looking Statements
This press release contains forward looking statements within
the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended, including statements regarding our goals to develop and
commercialize our product candidates, and other statements
identified by words such as “could,” “expects,” “intends,” “may,”
“plans,” “potential,” “should,” “will,” “would,” or similar
expressions and the negatives of those terms. Forward-looking
statements are not promises or guarantees of future performance and
are subject to a variety of risks and uncertainties, many of which
are beyond our control, and which could cause actual results to
differ materially from those contemplated in such forward-looking
statements. These factors include risks related to our limited
operating history, our ability to obtain necessary funding, our
ability to generate positive clinical trial results for our product
candidates and other risks inherent in clinical development, the
timing and scope of regulatory approvals, changes in laws and
regulations to which we are subject, competitive pressures, our
ability to identify additional product candidates, risks relating
to business interruptions, and other risks set forth under the
heading “Risk Factors” of our Annual Report on Form 10-K for the
year ended December 31, 2022 and in our subsequent filings with the
Securities and Exchange Commission. Our actual results could differ
materially from the results described in or implied by such
forward-looking statements. Forward-looking statements speak only
as of the date hereof, and, except as required by law, we undertake
no obligation to update or revise these forward-looking
statements.
About PureTech Health
PureTech is a clinical-stage biotherapeutics company dedicated
to giving life to new classes of medicine to change the lives of
patients with devastating diseases. The Company has created a broad
and deep pipeline through its experienced research and development
team and its extensive network of scientists, clinicians and
industry leaders that is being advanced both internally and through
its Founded Entities. PureTech's R&D engine has resulted in the
development of 27 therapeutics and therapeutic candidates,
including two (Plenity® and EndeavorRx®) that have received both US
FDA clearance and European marketing authorization and a third
(KarXT) that has been filed for FDA approval. A number of these
programs are being advanced by PureTech or its Founded Entities in
various indications and stages of clinical development, including
registration enabling studies. All of the underlying programs and
platforms that resulted in this pipeline of therapeutic candidates
were initially identified or discovered and then advanced by the
PureTech team through key validation points.
For more information, visit www.puretechhealth.com or connect
with us on X (formerly Twitter) @puretechh.
Cautionary Note Regarding Forward-Looking Statements
This press release contains statements that are or may be
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. All statements contained
in this press release that do not relate to matters of historical
fact should be considered forward-looking statements, including
without limitation those related to the acceptance of a New Drug
Application (NDA) to the U.S. Food and Drug Administration (FDA)
for KarXT (xanomeline-trospium) for the treatment of schizophrenia
and Karuna’s and PureTech’s future prospects, developments and
strategies. The forward-looking statements are based on current
expectations and are subject to known and unknown risks,
uncertainties and other important factors that could cause actual
results, performance and achievements to differ materially from
current expectations, including, but not limited to, those risks,
uncertainties and other important factors described under the
caption "Risk Factors" in our Annual Report on Form 20-F for the
year ended December 31, 2022 filed with the SEC and in our other
regulatory filings. These forward-looking statements are based on
assumptions regarding the present and future business strategies of
the Company and the environment in which it will operate in the
future. Each forward-looking statement speaks only as at the date
of this press release. Except as required by law and regulatory
requirements, we disclaim any obligation to update or revise these
forward-looking statements, whether as a result of new information,
future events or otherwise.
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version on businesswire.com: https://www.businesswire.com/news/home/20231129469347/en/
PureTech Public Relations
publicrelations@puretechhealth.com Investor Relations
IR@puretechhealth.com EU Media Ben Atwell, Rob Winder +44
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Nichole Sarkis +1 774 278 8273
nichole@tenbridgecommunications.com
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