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Karuna Receives $8 Million Wellcome Trust Award to Advance First-in-Class Phase 2 Clinical Program in Schizophrenia

Karuna
Pharmaceuticals, an affiliate of PureTech
Health plc (LSE: PRTC), focused on targeting muscarinic receptors
for the treatment of disorders marked by psychosis and cognitive
symptoms, today announced that it has been awarded approximately $8
million by the Wellcome Trust. The funding will be used to further
advance clinical development of Karuna’s lead program, KarXT
(Karuna-Xanomeline-Trospium), through a Phase 2 study in people with
schizophrenia.

“Antipsychotics are the mainstay therapy for the treatment of
schizophrenia and have all relied on the same fundamental mechanism of
action for the past 60 years. Currently available treatments have
limited efficacy – primarily addressing only positive symptoms, such as
hallucinations and delusions – and are often associated with serious,
often irreversible, side effects, leaving a significant need for new
treatments for people living with this devastating disease,” said Andrew
Miller, Ph.D., President and Chief Executive Officer of Karuna. “With
KarXT, we have an opportunity to potentially improve positive, negative,
and cognitive symptoms based on prior compelling efficacy data with
xanomeline, and without the debilitating side effects of existing
therapies. We are delighted that Wellcome is continuing to back this
important new therapeutic approach.”

Xanomeline previously demonstrated compelling efficacy in double-blind
placebo-controlled studies in schizophrenia and Alzheimer’s disease; at
that time tolerability issues associated with the activation of
muscarinic receptors outside the brain hindered xanomeline’s further
development. Karuna’s KarXT is designed to improve tolerability and
unlock the potential of muscarinic agonists by selectively targeting
M1/M4 muscarinic receptors in the brain while blocking their activation
in tissues outside the brain. KarXT has demonstrated a significant
improvement in tolerability compared to xanomeline in a double-blind,
placebo-controlled, healthy volunteer study, and Karuna is currently
completing a Phase 1 study using a proprietary co-formulation of
xanomeline and trospium. The Phase 2 acute psychosis trial in people
with schizophrenia is designed to replicate and expand on the previous
efficacy results with xanomeline and is expected to begin in the third
quarter of 2018.

Dr. Lynsey Bilsland, from Wellcome’s Innovations team said: “Karuna’s
treatment candidate has the potential to have a significant impact on
patients affected by schizophrenia and other psychotic disorders.
Research in this important area has long been under-resourced, and
doctors and patients currently rely on treatments which have existed for
decades. Wellcome is committed to supporting the innovation vital to
developing global understanding and treatment of mental illness and
improving life for patients affected.”

Wellcome previously awarded Karuna $3.8 million to fund a Phase 1
tolerability proof-of-concept study with KarXT, which reported
successful results. Both awards have been structured in the form of
unsecured convertible loans.

About SchizophreniaSchizophrenia 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 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 WellcomeWellcome exists to improve health for
everyone by helping great ideas to thrive. We’re a global charitable
foundation, both politically and financially independent. We support
scientists and researchers, take on big problems, fuel imaginations and
spark debate.

About Karuna PharmaceuticalsKaruna is a clinical-stage drug
development company targeting muscarinic receptors for the treatment of
psychosis and cognition across central nervous system (CNS) disorders,
including schizophrenia and Alzheimer’s disease. Karuna’s lead product
candidate, KarXT,
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 CNS. Karuna has demonstrated improved
tolerability of KarXT vs. xanomeline in a placebo-controlled human study
and is currently conducting a Phase 1 study using a proprietary
co-formulation of xanomeline and trospium. A Phase 2 trial of safety and
efficacy in people with schizophrenia is expected to begin in the third
quarter of 2018. Karuna, an affiliate of PureTech
Health (LSE: PRTC), has a worldwide exclusive license for xanomeline
and has a patent portfolio more broadly covering selective muscarinic
targeting enabled by the KarXT approach. For more information, visit www.karunapharma.com.

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