Last updated on April 17, 2014 at 21:23 EDT

Kareus Therapeutics Announces Phase I Trial for Product in Alzheimer’s Disease

January 3, 2013

LA CHAUX-DE-FONDS, Switzerland, January 3, 2013 /PRNewswire/ –

~ Following US IND approval, Phase I trial to assess safety, tolerability

and pharmacokinetics of KU-046 in healthy young volunteers ~

Kareus Therapeutics SA (“Kareus” or the “Company”), the private biotechnology company
focused on the development of novel therapeutics for the unmet treatment of age-related
and chronic diseases, today announces the start of a Phase I clinical study following the
Investigational New Drug (IND) approval from the US Food and Drug Administration for the
development of KU-046, a first-in-class disease modifying new chemical entity discovered
for the treatment of Alzheimer’s disease using Kareus’ proprietary bioenergetics and
KARLECT platforms.

The Phase I clinical trial is a randomised, double-blind, placebo-controlled two-part
study to assess the safety, tolerability and pharmacokinetics of single ascending oral
doses and of multiple ascending oral doses of KU-046 in 54 healthy young volunteers. The
study is being conducted by Quintiles, the world’s leading biopharmaceutical service
provider, at its Phase I Unit at Overland Park in Kansas, US.

Kareus has developed a pipeline of novel molecules targeting diseases of the central
nervous system based on its proprietary KARLECT chemistry and drug discovery platforms
which target dysfunctional energy production in neurons. KU-046 targets bioenergetics
pathways upstream from the increased abeta peptide production found in Alzheimer’s
disease. It has demonstrated significant improvement in cognition in a number of
pre-clinical models.

Commenting on the news, Patrick Doyle, Chief Business and Corporate Development at
Kareus Therapeutics, said: “We are excited to have obtained IND approval that will bring
KU-046 into clinical development. This is a positive milestone, demonstrating the power of
Kareus’ innovation platform, which has already shown significant improvement in cognition
in a number of pre-clinical models.” He added: “We now believe we are well positioned to
partner KU-046 to take it into the next stages of its development.”

Dr. Uday Saxena, Chief Research and Development at Kareus Therapeutics, commented:
“Achieving our objective to reach IND approval for KU-046 within threeyears and in a
cost-efficient manner is strong testament to the commitment and hard work of our team.” He
added: “Alzheimer’s disease is the most common form of dementia which affects over 35
million people worldwide and for which there are currently no disease modifying approved
treatments. We are therefore hugely excited and encouraged by the potential of KU-046 to
treat patients with this devastating condition. The successful discovery of KU-046 has
prompted us to use our bioenergetics and KARLECT platformsto discover drug candidates for
other CNS diseases such as Parkinson’s and Huntington’s disease which have similar energy

About Kareus Therapeutics SA

Kareus Therapeutics SA is a private biotechnology company focused on the development
of novel therapeutics for the unmet treatment of age-related and chronic diseases. Kareus’
pipeline consists of pre-clinical stage assets in Alzheimer’s, Metabolic Diseases, Pain &
Inflammation, where the company employs two different approaches to drug discovery and
development: i) KARLECT, a unique enabling chemistry platform that combines currently
marketed drugs and GRAS molecules to produce new chemical entities for CNS disorders, and
ii) novel small molecule drug discovery in metabolic diseases, pain & inflammation.
Kareus’ business strategy is to form strategic alliances with leading pharmaceutical
companies, which have the complementary skills to maximize the business and therapeutic
potential of its molecules. In June 2011, Kareus entered into a strategic drug development
alliance with Quintiles to progress the development of two of Kareus’ pre-clinical
programs (Alzheimer’s & Type-2 diabetes) through clinical trials.

For further information, please visit http://www.kareustherapeutics.com.

About Alzheimer’s disease

Alzheimer’s disease, the most common form of dementia, causes a progressive decline in
memory and other aspects of cognition. It is unknown what causes Alzheimer’s disease and
there are currently no approved treatments shown to slow the progression of the disease.
Alzheimer’s Disease International (ADI) estimates that there are currently over 36 million
people with dementia worldwide, with 7.7 million new cases each year, or one new case
every four seconds.[1] The number of people affected is estimated to be over 115 million
by 2050 Alzheimer’s Disease International. World Alzheimer Report 2009. London :
Alzheimer’s Disease International, 2009.

About Investigational New Drug Application (IND)

IND is an application that a drug sponsor must submit to U.S. Food and Drug
Administration (FDA) before beginning tests of a new drug on humans. The IND contains the
plan for the study and is supposed to give a complete picture of the drug, including its
structural formula, animal test results, and manufacturing information. The equivalent in
Europe is called an Investigational Medicinal Product Dossier (IMPD).


1. World Health Organization. Dementia: a public health priority. Geneva : World
Health Organization, 2012. ISBN 978-92-4-156445-8.

        For more information, please contact:

        Kareus Therapeutics
        Patrick Doyle
        T: +44(0)7871-309-056
        E: pdoyle@kareustherapeutics.com

        Claire Dickinson
        T: +44(0)20-7920-2360
        E: kareus@mcomgroup.com

SOURCE Kareus Therapeutics

Source: PR Newswire