TIDMPRTC
RNS Number : 5441T
PureTech Health PLC
20 March 2023
20 March 2023
PureTech Health plc
PureTech Founded Entity Karuna Therapeutics Announces Positive
Results from Phase 3 EMERGENT-3 Trial of KarXT in Schizophrenia
Third positive registrational trial met its primary endpoint,
with KarXT demonstrating an 8.4-point reduction in PANSS total
score compared to placebo at Week 5 (p<0.0001)
KarXT was generally well tolerated, with a side effect profile
substantially consistent with previous trials of KarXT in
schizophrenia
Karuna is on track to submit a New Drug Application (NDA) to the
U.S. Food & Drug Administration (FDA) in mid-2023, with a
potential launch in the second half of 2024, if approved
Pre-NDA meeting is scheduled for early second quarter of
2023
Karuna to host conference call and webcast today at 8:00 a.m.
ET
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 positive topline results from its Phase 3
EMERGENT-3 trial evaluating the efficacy, safety, and tolerability
of its lead investigational therapy, KarXT (xanomeline-trospium) in
adults with schizophrenia. The trial met its primary endpoint, with
KarXT demonstrating a statistically significant and clinically
meaningful 8.4-point reduction in Positive and Negative Syndrome
Scale (PANSS) total score compared to placebo (-20.6 KarXT vs.
-12.2 placebo; p<0.0001) at Week 5 (Cohen's d effect size of
0.60). Consistent with prior trials, KarXT demonstrated an early
and sustained statistically significant reduction of symptoms from
Week 2 (p<0.05) through the end of the trial as assessed by
PANSS total score.
KarXT also demonstrated reductions in positive and negative
symptoms of schizophrenia as measured by PANSS positive, PANSS
negative, and PANSS negative Marder factor subscales - secondary
endpoints in the trial. KarXT demonstrated a clinically meaningful
and statistically significant 3.5-point reduction in PANSS positive
subscale compared to placebo at Week 5 (-7.1 KarXT vs. -3.6
placebo; p<0.0001). While not meeting the threshold for
statistical significance at Week 5, KarXT did demonstrate a
statistically significant reduction in PANSS negative subscale and
PANSS negative Marder factor subscale compared to placebo at Week 4
(p<0.05).
The NDA submission for KarXT in schizophrenia will incorporate
the efficacy and safety data from the three placebo-controlled
registrational trials, EMERGENT-1, EMERGENT-2, and EMERGENT-3, in
addition to long-term safety data from the ongoing EMERGENT-4 and
EMERGENT-5 trials. Karuna is on track to submit an NDA to the FDA
in mid-2023, with a potential launch in the second half of 2024, if
approved.
As of February 23, 2023, PureTech owned approximately 3.1% of
Karuna's outstanding stock. A founder of Karuna and co-inventor of
the KarXT program, PureTech has a right to royalty payments of 3%
of net sales of any commercialized product covered by the license
agreement, as well as 20% sublicense income covered by the license
agreement. The license agreement covers key territories including
the United States, European Union, and Japan. PureTech is also
eligible to receive certain milestone payments upon the achievement
of regulatory approvals.
KarXT was invented by a team at PureTech, including its Chief
Innovation Officer, Eric Elenko, Ph.D., who served as the founding
CEO of Karuna Therapeutics. PureTech recruited key members of the
current Karuna team, which subsequently executed the two successful
registration enabling studies as well as other key
advancements.
The full text of the announcement from Karuna is as follows:
Karuna Therapeutics Announces Positive Results from Phase 3
EMERGENT-3 Trial of KarXT in Schizophrenia
Third positive registrational trial met its primary endpoint,
with KarXT demonstrating an 8.4-point reduction in PANSS total
score compared to placebo at Week 5 (p<0.0001)
KarXT was generally well tolerated, with a side effect profile
substantially consistent with previous trials of KarXT in
schizophrenia
Company is on track to submit a New Drug Application (NDA) to
the U.S. Food & Drug Administration (FDA) in mid-2023, with a
potential launch in the second half of 2024, if approved
Pre-NDA meeting is scheduled for early second quarter of
2023
Conference call and webcast to take place today at 8:00 a.m.
ET
BOSTON -- Mar. 20, 2023 -- Karuna Therapeutics, Inc. (NASDAQ:
KRTX), a clinical-stage biopharmaceutical company driven to create
and deliver transformative medicines for people living with
psychiatric and neurological conditions, today announced positive
topline results from its Phase 3 EMERGENT-3 trial evaluating the
efficacy, safety, and tolerability of its lead investigational
therapy, KarXT (xanomeline-trospium) in adults with schizophrenia.
The trial met its primary endpoint, with KarXT demonstrating a
statistically significant and clinically meaningful 8.4-point
reduction in Positive and Negative Syndrome Scale (PANSS) total
score compared to placebo (-20.6 KarXT vs. -12.2 placebo;
p<0.0001) at Week 5 (Cohen's d effect size of 0.60). Consistent
with prior trials, KarXT demonstrated an early and sustained
statistically significant reduction of symptoms from Week 2
(p<0.05) through the end of the trial as assessed by PANSS total
score.
"KarXT has now demonstrated a robust and consistent reduction of
symptoms across all three registrational trials, providing a
compelling picture of the potential of KarXT in schizophrenia. With
these data, we are one step closer to a potential treatment option
that could provide the first new mechanism of action to treat
schizophrenia in several decades," said Bill Meury, president and
chief executive officer of Karuna Therapeutics. "We look forward to
working closely with the FDA as we focus our attention on the
regulatory process, including our upcoming pre-NDA meeting in early
second quarter, and remain on track for an NDA submission in
mid-2023. I would like to thank all of the participants in the
EMERGENT trials and the study investigators - without their
commitment and trust, none of this would be possible."
"Schizophrenia is a persistent and disabling condition that
presents with symptoms which are often difficult to treat and
manage. Despite the number of currently available treatments, there
remains a significant need for new treatment options for the 21
million people living worldwide with schizophrenia," said David
Walling, Ph.D., chief clinical officer at Cenexel - CNS and
investigator on the EMERGENT-3 trial. "The results from the
EMERGENT-3 trial add to the growing body of data which suggest
KarXT could address the symptoms of schizophrenia without the
common side effects we see with current treatment options."
KarXT also demonstrated reductions in positive and negative
symptoms of schizophrenia as measured by PANSS positive, PANSS
negative, and PANSS negative Marder factor subscales - secondary
endpoints in the trial. KarXT demonstrated a clinically meaningful
and statistically significant 3.5-point reduction in PANSS positive
subscale compared to placebo at Week 5 (-7.1 KarXT vs. -3.6
placebo; p<0.0001). While not meeting the threshold for
statistical significance at Week 5, KarXT did demonstrate a
statistically significant reduction in PANSS negative subscale and
PANSS negative Marder factor subscale compared to placebo at Week 4
(p<0.05).
KarXT was generally well tolerated, with a side effect profile
substantially consistent with prior trials of KarXT. The overall
discontinuation rate in the trial was 33% (37% KarXT vs. 29%
placebo). The overall treatment emergent adverse event (TEAE) rates
for KarXT and placebo were 70% and 50%, respectively.
Discontinuation rates related to TEAEs were similar between
treatment arms (6% KarXT vs. 5% placebo), consistent with the
EMERGENT-1 and EMERGENT-2 trials. The only serious TEAE reported in
the KarXT arm was related to gastroesophageal reflux disease (acid
reflux) and deemed not to be related to study drug. There were no
serious TEAEs reported in the placebo group. The most common KarXT
TEAEs (>5%) were nausea, dyspepsia, vomiting, constipation,
headache, hypertension, diarrhea, and insomnia, which were all
rated mild or moderate in severity. There were no discontinuations
due to TEAEs of hypertension. Mean blood pressure measures were
similar between KarXT and placebo, and no syncopal events were
observed. Similar to prior trials, an increase in heart rate was
associated with KarXT treatment and decreased in magnitude by the
end of the trial. Measures of weight gain, somnolence, and
extrapyramidal symptoms of KarXT were similar to placebo,
consistent with prior trials of KarXT in schizophrenia.
The NDA submission for KarXT in schizophrenia will incorporate
the efficacy and safety data from the three placebo-controlled
registrational trials, EMERGENT-1, EMERGENT-2, and EMERGENT-3, in
addition to long-term safety data from the ongoing EMERGENT-4 and
EMERGENT-5 trials. The Company is on track to submit an NDA to the
FDA in mid-2023, with a potential launch in the second half of
2024, if approved.
Conference Call and Webcast Information
Karuna will hold a webcast and conference call this morning at
8:00 a.m. ET to share topline results from its Phase 3 EMERGENT-3
trial of KarXT in schizophrenia.
A live webcast of the presentation will be available on the
Investor Relations page of Karuna's website at
investors.karunatx.com. A replay of the webcast will also be
archived for up to 30 days on Karuna's website following the
conference.
About the EMERGENT-3 Trial
The Phase 3 EMERGENT-3 trial is a double-blind,
placebo-controlled, five-week, inpatient trial evaluating the
efficacy, safety, and tolerability of our lead investigational
therapy, KarXT, compared to placebo in adults with schizophrenia in
the United States and Ukraine. The primary endpoint was change from
baseline in Positive and Negative Syndrome Scale (PANSS) total
score, a scale for measuring schizophrenia symptom severity, of
KarXT compared to placebo at Week 5. Prespecified secondary
endpoints included change from baseline in PANSS positive, PANSS
negative and PANSS negative Marder factor subscale of KarXT
compared to placebo at Week 5.
A total of 256 adults (between the ages of 18-65 years) with
schizophrenia enrolled in the trial. Enrolled patients had a
confirmed diagnosis of schizophrenia and were experiencing symptoms
of psychosis at the time of enrollment.
Patients were randomized 1:1 to receive either a flexible dose
of KarXT or placebo two times a day (BID) for five weeks. On Days
1-2, patients received a dose of 50/20 KarXT (50mg xanomeline/20mg
trospium) BID or matching placebo. On Day 3, patients escalated to
a dose of 100/20 BID, and on Day 8, patients had the option to
increase to 125/30 BID based on tolerability. In the trial, 79% of
patients on KarXT compared to 91% on placebo titrated to the
highest dose level (125/30 BID).
About KarXT
KarXT (xanomeline-trospium) is an oral, investigational
M1/M4-preferring muscarinic agonist in development for the
treatment of psychiatric and neurological conditions, including
schizophrenia and psychosis in Alzheimer's disease. KarXT is the
first potential medicine of its kind with a truly new and unique
dual mechanism of action. Unlike current therapies, KarXT does not
rely on the dopaminergic or serotonergic pathways, and it is
designed to harness the therapeutic potential of xanomeline while
managing peripheral side effects through trospium. This approach
has the potential to provide a differentiated therapy, and, if
approved, to beneficially impact the lives of millions of people
with serious mental illness.
About Schizophrenia
Schizophrenia is a persistent and often disabling mental illness
affecting how a person thinks, feels, and behaves. It is
characterized by positive symptoms (hallucinations and delusions),
negative symptoms (difficulty enjoying life and withdrawal from
others), and cognitive impairment (deficits in memory,
concentration, and decision-making) - all of which can severely
impact functioning, with only 10% of people gainfully employed and
many struggling to meet adult milestones such as living
independently. The life expectancy of people living with
schizophrenia is reduced by 10-20 years compared to the general
population. Schizophrenia affects more than 21 million people
worldwide and is most commonly treated with antipsychotics.
Unfortunately, many people with schizophrenia continue to
experience limited efficacy or problematic side effects while on
antipsychotic therapy, and approximately 75% of patients
discontinue medication before 18 months. When schizophrenia
treatment is discontinued, it can lead to impacts on health
including relapse, hospitalization, and longer time to
remission.
About Karuna Therapeutics
Karuna Therapeutics is a clinical-stage biopharmaceutical
company driven to create 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 expectations about the
timing of our ongoing and planned clinical trials and regulatory
filings, our goals to develop and commercialize our product
candidates, our liquidity and capital resources, 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 resulting from the coronavirus (COVID-19) pandemic,
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.
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 biotherapeutics company dedicated to changing the
treatment paradigm for devastating diseases. The Company has
created a broad and deep pipeline through the expertise of its
experienced research and development team and its extensive network
of scientists, clinicians and industry leaders. This pipeline,
which is being advanced both internally and through PureTech's
Founded Entities, is comprised of 26 therapeutics and therapeutic
candidates, including two (Plenity(R) and EndeavorRx(R)) that have
received both U.S. FDA clearance and European marketing
authorization and a third (KarXT) that will soon be filed for FDA
approval, as of the most recent update by the Company. 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
based on unique insights in immunology and drug development.
For more information, visit www.puretechhealth.com or connect with us on Twitter @puretechh.
Cautionary Note Regarding Forward-Looking Statements
This press release contains 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, including without limitation
those related to the Phase 3 EMERGENT-3 results of Karuna's KarXT
in adults with schizophrenia and expectations related to timing of
the potential New Drug Application and potential commercial launch
of the therapeutic, and Karuna's and PureTech's future prospects,
development plans 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, 2021 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.
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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