TIDMPRTC
PureTech Health PLC
28 September 2023
28 September 2023
PureTech Health plc
KarXT, invented at PureTech, submitted for FDA Approval in
Schizophrenia
Founded Entity Karuna Therapeutics submits New Drug Application
to U.S. Food and Drug Administration for KarXT for the treatment of
schizophrenia
If approved, KarXT will be the first new mechanism in over 50
years for patients living with schizophrenia
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 submission of a New Drug Application (NDA)
to the U.S. Food and Drug Administration (FDA) for KarXT
(xanomeline-trospium) for the treatment of schizophrenia.
"We are thrilled to note this additional exciting milestone from
the KarXT program, following the achievement of three successful,
registration enabling studies in schizophrenia. KarXT, which was
invented at PureTech, showcases how we invent and advance novel
medicines by giving new life to drugs that have validated
pharmacology but had previously not reached patients due to an
issue that is now addressable with our innovations. This NDA
submission is also an important milestone for patients, as it could
represent the first major advance for treating schizophrenia in
over 50 years if approved by the FDA," said Eric Elenko, Chief
Innovation Officer at PureTech.
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 ongoing 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 a statistically significant and clinically meaningful
reduction in Positive and Negative Syndrome Scale (PANSS) total
score compared to placebo. KarXT also demonstrated reductions in
both positive and negative symptoms of schizophrenia as measured by
PANSS positive, PANSS negative, and PANSS negative Marder factor
subscales, which were secondary endpoints in the trials.
As a founder of Karuna and co-inventor of the KarXT program,
PureTech retains an equity ownership and has rights to receive
milestone payments upon the achievement of certain regulatory
approvals and 20% of sublicense income.(1)
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 Submits New Drug Application to U.S. Food
and Drug Administration for KarXT for the Treatment of
Schizophrenia
KarXT, a dual M1/M4 muscarinic agonist, represents the first new
mechanism of action to treat schizophrenia in several decades, if
approved
Submission is supported by data from three positive
registrational trials demonstrating consistent and robust
reductions of schizophrenia symptoms
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 submission of a New Drug
Application (NDA) to the U.S. Food and Drug Administration (FDA)
for KarXT (xanomeline-trospium) for the treatment of
schizophrenia.
"Schizophrenia is a serious mental illness that affects how one
thinks, feels, and behaves, with symptoms often appearing in
patients in early adulthood, during the prime years of their
lives," said Bill Meury, president and chief executive officer of
Karuna Therapeutics. "While current therapies have made a
difference for many patients, they are not without limitations due
to lack of full symptom relief or side effects that may lead to
treatment discontinuation. KarXT, if approved, will represent the
first novel pharmacological approach to treating schizophrenia in
several decades and provide a new treatment option for patients and
their physicians."
"The NDA submission represents an important step toward helping
patients in need. It is also a defining moment for Karuna
Therapeutics. It represents the culmination of years of
pre-clinical and clinical development, and a great deal of skill
and hard work by our R&D organization. Our priorities over the
next year are the regulatory review process, our ongoing
development efforts, and building the platform to introduce KarXT
to the medical community. I believe we are uniquely positioned to
make a positive impact on how neuropsychiatric conditions are
treated," Meury added.
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 ongoing 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 a statistically significant and clinically meaningful
reduction in Positive and Negative Syndrome Scale (PANSS) total
score compared to placebo. KarXT also demonstrated reductions in
both positive and negative symptoms of schizophrenia as measured by
PANSS positive, PANSS negative, and PANSS negative Marder factor
subscales, which were secondary endpoints in the trials.
KarXT was found to be generally well-tolerated, with the most
common adverse events being cholinergic in nature and rated mild to
moderate in severity. Discontinuation rates due to treatment
emergent adverse events were low and similar between KarXT and
placebo across all trials. Notably, KarXT was not associated with
common side effects of currently available antipsychotics,
including changes in metabolic function, weight gain, somnolence,
and extrapyramidal symptoms.
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 mediate 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 to 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.
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(R) and EndeavorRx(R)) 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.
(1) As of July 31, 2023, PureTech's percentage ownership of
Karuna was approximately 2.8% on an outstanding voting share basis.
As of March 22, 2023, PureTech has sold its right to receive a 3%
royalty from Karuna to Royalty Pharma on net sales up to $2 billion
annually, after which threshold PureTech will receive 67% of the
royalty payments and Royalty Pharma will receive 33%. PureTech
retains its equity ownership in Karuna. Additionally, under its
license agreement with Karuna, PureTech retains the right to
receive milestone payments upon the achievement of certain
regulatory approvals and 20% of sublicense income.
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 submission 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
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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.
Contact:
PureTech
Public Relations
publicrelations@puretechhealth.com
Investor Relations
IR@puretechhealth.com
EU Media
Ben Atwell, Rob Winder
+44 (0) 20 3727 1000
ben.atwell@FTIconsulting.com
U.S. Media
Nichole Sarkis
+1 774 278 8273
nichole@tenbridgecommunications.com
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