# PureTech Health New England Journal of Medicine Publication of Data from EMERGENT-1 Phase 2 Trial

- Link: https://www.directorstalkinterviews.com/puretech-health-new-england-journal-of-medicine-publication-of-data-from-emergent-1-phase-2-trial/412963843
- Published: 2021-02-25T09:15:45+00:00
- Author: Giles

**PureTech Health plc** (Nasdaq: PRTC, LON:PRTC), a clinical-stage biotherapeutics
company dedicated to discovering, developing and commercializing highly differentiated
medicines for devastating diseases, has noted that its Founded Entity, **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, announced that results from the EMERGENT-1 Phase 2 
clinical trial evaluating KarXT for the treatment of schizophrenia were published
in the_ New England Journal of Medicine (NEJM)._

The full text of the announcement from Karuna is as follows:

**Karuna Therapeutics Announces New England Journal of Medicine Publication of Data
from EMERGENT-1 Phase 2 Trial Evaluating KarXT in Schizophrenia**

KarXT demonstrated statistically significant and clinically meaningful improvements
in primary and key secondary efficacy measures compared with placebo

Incidences of somnolence, weight gain and extrapyramidal symptoms were similar in
KarXT and placebo treatment groups

KarXT is in Phase 3 clinical development for the treatment of psychosis in adults
with schizophrenia

**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 that results from 
the EMERGENT-1 Phase 2 clinical trial evaluating KarXT for the treatment of schizophrenia
were published in the_ New England Journal of Medicine (NEJM)._

“The publication of the EMERGENT-1 Phase 2 trial results in the peer-reviewed _New
England Journal of Medicine_ reinforces earlier clinical and preclinical data that
KarXT’s modulation of muscarinic receptor function in the brain improves the symptoms
of psychosis in schizophrenia, and underscores the potential of KarXT to offer a
novel approach to treating this serious and disabling condition,” said Steve Brannan,
M.D., chief medical officer of Karuna Therapeutics and lead author of the manuscript.“
These findings support the potential for KarXT to treat symptoms of psychosis in
schizophrenia without producing the common problematic side effects of current therapies,
such as weight gain and extrapyramidal symptoms. Given these encouraging results,
we have advanced KarXT into Phase 3 clinical development in our efforts to provide
a meaningful, new, non-dopaminergic treatment option for this serious neuropsychiatric
disorder affecting more than 21 million people worldwide.”

The double-blind, placebo-controlled, five-week inpatient EMERGENT-1 Phase 2 trial
enrolled 182 adults with an acute exacerbation of schizophrenia. In this trial, 
a twice-daily, flexible-dose treatment with KarXT demonstrated a statistically significant
and clinically meaningful 11.6-point reduction in the Positive and Negative Syndrome
Scale (PANSS) total score compared to placebo at Week 5, the primary outcome measure
of the study, with an effect size of 0.75 (p<0.0001). Results published in _NEJM_
also include data for pre-specified secondary outcome measures. Four of the five
pre-specified secondary outcome measures, including PANSS positive symptom subscore,
PANSS negative symptom subscore, PANSS Marder negative symptom subscore, and Clinical
Global Impression – Severity frequency counts, showed statistically significant 
reductions following treatment with KarXT compared to placebo at Week 5 (p<0.001).

KarXT was generally well-tolerated, with similar discontinuation rates between KarXT
and placebo arms, both overall (20% vs. 21%) and due to treatment-emergent adverse
events (2% in both arms). The most common adverse events associated with KarXT, 
including constipation, nausea, dry mouth, dyspepsia and vomiting, were mild-to-
moderate in severity and were not associated with treatment discontinuation. Rates
of nausea, vomiting, and dry mouth decreased over the course of the trial, while
rates of constipation remained essentially constant. Incidences of somnolence, weight
gain and extrapyramidal symptoms, which are common problematic side effects of current
antipsychotic therapies, were similar in the placebo and KarXT treatment groups.

“Many people living with schizophrenia have persistent symptoms, experience poor
quality of life and impaired ability to function, despite treatment with current
antipsychotic drugs,” said Jeffrey Lieberman, M.D., professor and chairman of the
Department of Psychiatry, Columbia University, College of Physicians and Surgeons,
member of Karuna’s scientific advisory board, and co-author of the manuscript. “
The clinical trial results with KarXT highlight its potential to be a differentiated
treatment option with a completely new mechanism of action for people living with
schizophrenia, offering relief from acute psychotic symptoms without the debilitating
side effects associated with the current standard of care.”

About KarXT

KarXT, a proprietary oral modulator of muscarinic cholinergic receptors, is Karuna’s
lead product candidate. It combines xanomeline, a novel muscarinic agonist, with
trospium, an FDA-approved muscarinic antagonist that does not appreciably cross 
the blood-brain-barrier, to preferentially stimulate muscarinic receptors in the
central nervous system. This novel product candidate, if approved, has the potential
to usher in a new treatment paradigm and dramatically impact patients with schizophrenia
and other psychotic disorders by providing a differentiated mechanism of action 
relative to current D2 dopamine and serotonin receptor-targeting antipsychotic drugs.

About Schizophrenia

Schizophrenia is a chronic and often disabling brain condition affecting how a person
thinks, feels and behaves. It is estimated to affect more than 21 million people
worldwide, and has a young age at onset, typically presenting during late adolescence
to early adulthood. Symptoms of schizophrenia generally fall into three categories–
positive (delusions, hallucinations, and difficulty organizing and expressing thoughts),
negative (difficulty enjoying life and withdrawal from others) and cognitive (deficits
in memory, concentration and decision making). Given the nature of these symptoms,
schizophrenia can affect all areas of patients’ lives. Many people living with schizophrenia
have difficulty finding an effective treatment and continue to experience distressing
symptoms. With the help of a dedicated treatment team, it is possible for people
with schizophrenia to live full lives. New therapeutic options with different mechanisms
of action will enable more patients to find an effective and safe treatment regimen.

About Karuna

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
these severe and disabling disorders. 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.

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