| Sugars
are fundamental to human biochemistry:
glucose is our energy molecule,
ribose holds our DNA together, and
other sugars are crucial building
blocks in our cellular membranes,
enzymes, and organelles. One
of the members of our Scientific
Advisory Board and Board of Directors,
University of Oxford Glycobiology
Institute Founder and also Chairman
of the Biochemistry Department,
Professor Raymond A. Dwek, F.R.S.,
is one of the foremost experts on
iminosugars, a class of small molecules
that act like sugars but are created
synthetically. In particular,
Professor Dwek discovered a way
to use iminosugars so that they
have impressive therapeutic benefits.
The
first therapeutic iminosugars used
by Professor Dwek were called alpha-glucosidase
inhibitors. This kind of molecule
was found to be quite effective
in treating Gaucher’s Disease and
other glycolipid storage disorders
and was licensed exclusively to
the U.K. company Oxford Glycosciences
via Searle/Monsanto.
More
recently, Professor Dwek and his
colleagues have discovered a new
set of therapeutic iminosugars that
in laboratory tests were found to
be effective at reversing the symptoms
of infection by flaviviruses.
There are four well-known diseases
caused by flaviviruses: hepatitis
B, hepatitis C, dengue fever, and Japanese
encephalitis. The exclusive
worldwide rights to use these therapeutic
iminosugars were licensed to United
Therapeutics. We are now actively
engaged in:
-
Producing clinical trial quantities
of our lead investigational iminosugar,
UT-231B, as well asarranging
for the production of yet larger
quantities;
-
Ensuring the safety of UT-231B
via required preclinical tests;
-
Conducting clinical studies in
man; and
-
Continuing research and development
efforts to produce additional
iminosugars with favorable safety
and efficacy profiles.
Our
iminosugars have a novel mechanism
of action against viruses that is
different from conventional antiviral
therapies. Our iminosugars
are able to slip through cellular
membranes and take up residence
in the endoplasmic reticulum, where
many viruses are assembled.
Many of the glycoproteins, and possibly
the proteins, in the viruses interact
with iminosugars, such as our investigational
drug UT-231B. In the specific
case of hepatitis C, our data suggest
that UT-231B alters the assembly
of the viruses and thereby prevents
them from being able to continue
infecting and replicating other
cells.
Recent published reports estimate
that as many as 12% of the world
population may be infected with
hepatitis C. In addition,
the plague of dengue fever has been
spreading rapidly. We believe our
iminosugar product line offers tremendous
potential for combating these major
worldwide health threats. |
