Karuna Announces First Patient Dosed in Phase 2 Study of Lead Product Candidate KarXT for the Treatment of Schizophrenia
15 October 2018 - 5:00PM
Business Wire
Phase 2 study aims to reproduce significant
efficacy previously observed in schizophrenia trial with xanomeline
alone
Company also announces successful Phase 1 study
of proprietary xanomeline and trospium chloride co-formulation,
which will be used in Phase 2 study
Karuna Pharmaceuticals, Inc. (“Karuna”), focused on targeting
muscarinic cholinergic receptors for the treatment of
neuropsychiatric disorders marked by psychosis and cognitive
impairment, today announced the initiation of a Phase 2 study
evaluating the efficacy and safety of its lead product candidate,
KarXT (Karuna-Xanomeline-Trospium), for the treatment of psychosis
in schizophrenia. The study will use a co-formulation of KarXT,
which was well-tolerated at dose levels exceeding those shown to be
efficacious in previous xanomeline studies. Top-line data results
from the Phase 2 study are expected at the end of 2019.
“We are excited to progress our development of KarXT, which has
the potential to be the first antipsychotic drug with a unique
mechanism in over 60 years and one which could be effective in
treating not only positive symptoms but also the disabling negative
and cognitive symptoms of the disease. Our Phase 2 study uses the
same fundamental design as the successful efficacy study conducted
previously with xanomeline alone,” said Steve Paul, M.D., Chief
Executive Officer of Karuna. “We have designed KarXT as a novel
approach to reduce the cholinergic sides effects related to the
activation of peripheral muscarinic receptors that were observed in
previous studies by Eli Lilly. We have now demonstrated the
improved tolerability in two Phase 1 studies, including with the
proprietary co-formulation of xanomeline and trospium.”
Karuna’s KarXT was evaluated in a Phase 1 dose-ranging study
that enrolled 70 healthy volunteers and successfully demonstrated
tolerability at dose levels exceeding those shown to be efficacious
in previous studies of xanomeline alone. The co-formulation also
achieved exposure levels equivalent to or higher than the separate
dosage forms used previously, and the results supported dose
selection to be carried forward into Phase 2. There were no severe
or serious adverse events reported in the co-formulation study.
Side effects associated with KarXT were mild-to-moderate and
transient in nature, often only lasting a few hours, and they were
consistent with the previous KarXT study that used separate dosage
forms for xanomeline and trospium.
The Phase 2 study is a double-blind, placebo-controlled study
designed to evaluate the efficacy and safety of KarXT in
approximately 160 patients with schizophrenia. The primary endpoint
is total change from baseline Positive and Negative Syndrome Scale
(PANSS) score compared to placebo. Additional endpoints will assess
cognitive and negative symptoms in addition to general
symptomology. The study employs a flexible dose design where
patients are randomized in a 1:1 ratio to receive either KarXT or
placebo for 5 weeks. Patients assigned to the KarXT arm will be
treated with 100/20 mg xanomeline/trospium with the option to
increase the dose to 125 mg/30mg xanomeline/trospium after the
first week of the study.
About Schizophrenia
Schizophrenia affects more than 20 million people worldwide and
is characterized by profound disruptions to daily life. Symptoms
are grouped within three domains: positive, negative, and
cognitive. Positive symptoms are generally associated with
psychotic behaviors, including hallucinations and delusions.
Negative symptoms refer to disruptions in behavior and emotions and
can manifest as reduced social engagement and motivation. Cognitive
symptoms are marked by changes in memory and attention. The
prognosis for schizophrenia remains poor as only 30 percent of
patients live independently and only 10 to 20 percent maintain
full-time employment. There is a desperate need for new treatments
in schizophrenia that not only address positive, negative, and
cognitive symptoms of the disease, but are also safer than existing
medicines.
About KarXT
KarXT (Karuna-Xanomeline-Trospium) is Karuna’s lead
investigational product candidate for the treatment of psychosis in
schizophrenia. It consists of xanomeline, a novel muscarinic
acetylcholine receptor agonist that has demonstrated efficacy in
placebo-controlled human trials in schizophrenia and Alzheimer’s
disease, and trospium chloride, an FDA-approved and
well-established muscarinic receptor antagonist that has been shown
not to enter the central nervous system (CNS). KarXT is designed to
selectively target M1/M4 muscarinic receptors in the brain while
blocking their activation in peripheral tissues to significantly
improve tolerability. Results from a Phase 1 study demonstrating
the improved tolerability of KarXT vs. xanomeline alone were
announced in 2016, and a more recent Phase 1 study completed in
2018 supported the development of a co-formulation of KarXT that is
now being evaluated in a Phase 2 study.
About Karuna Pharmaceuticals
Karuna is a clinical-stage drug development company targeting
muscarinic cholinergic receptors for the treatment of psychosis and
cognitive impairment across central nervous system (CNS) disorders,
including schizophrenia and Alzheimer’s disease, as well as pain.
Karuna’s lead product candidate, KarXT
(Karuna-Xanomeline-Trospium), is being evaluated in a Phase 2 study
in people with schizophrenia, with top-line results anticipated at
the end of 2019. Karuna, which was founded by PureTech
Health (LSE: PRTC), has a worldwide exclusive license for
xanomeline and has an intellectual property portfolio more broadly
covering selective muscarinic targeting enabled by the KarXT
approach. For more information, visit
www.karunapharma.com.
Forward Looking Statement
This press release contains statements that are or may be
forward-looking statements, including statements that relate to the
company's future prospects, developments and strategies. The
forward-looking statements are based on current expectations and
are subject to known and unknown risks and uncertainties that could
cause actual results, performance and achievements to differ
materially from current expectations, including, but not limited
to, those risks and uncertainties described in the risk factors
included in the regulatory filings for PureTech Health plc. 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,
neither the company nor any other party intends 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/20181014005040/en/
Karuna Pharmaceuticals, Inc.InvestorsAllison Mead Talbot,
+1 617-651-3156amt@puretechhealth.comorU.S. mediaTom
Donovan, +1 857-559-3397tom@tenbridgecommunications.com
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