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Pentraxin announces licensing deal with GSK to develop medicine for rare disease
University College London spin-out company Pentraxin Therapeutics Ltd has licensed a second drug development programme to pharmaceutical giant GlaxoSmithKline (GSK).
Pentraxin was established by
Professor Mark Pepys FRS, Director of the UCL Centre for Amyloidosis and
Acute Phase Proteins, to hold all the IP arising from his research. In
this latest licensing agreement, Pentraxin has entered into a
collaboration with GSK to develop Prof Pepys’s invention of novel small
molecules that stabilise transthyretin (TTR), a blood protein which can
cause a rare but fatal disease called amyloidosis.
The TTR protein is a normal component
of blood. As people age, or when there are mutations in the TTR gene,
the protein can become unstable and develop into an abnormal insoluble
form known as amyloid fibrils. These fibres accumulate in the organs
and tissues as amyloid deposits, damaging their structure and function,
and causing TTR amyloidosis, a fatal and currently untreatable
condition.
One of the novel molecules created by
the UCL team is mds84, which is bound irreversibly by TTR and prevents
the protein from forming amyloid.. This research was published on 23
November 2010 in the Proceedings of the National Academy of Science
(USA) (link).
Pepys and his colleagues received a
Seeding Drug Discovery Initiative award from the Wellcome Trust in 2007
to work on strategies for targeting TTR to treat and prevent TTR
amyloidosis. The £2.5 million award supported some of the research which
led to this recent licensing deal.
Prof Pepys, who led the research at
UCL, commented: “The creation of mds84 involved cutting edge science and
some serendipity. The subsequent generous support of the Wellcome Trust
for this early phase drug design programme created the opportunity for
further progression and evaluation. Now GSK will bring its drug
discovery and development expertise to work with the team on developing
the potential of these small molecules.”
Dr Rick Davis, Business Development
Manager at the Wellcome Trust, commented: “We are pleased that the
promising findings of Mark Pepys and his team towards tackling this rare
but untreatable disease will be accelerated with the support of this
latest deal with GSK.”
Work at GSK, in close collaboration
with Professor Pepys’ research team at UCL, is commencing immediately,
to develop a compound to the point where it can be tested for the first
time in patients.
In an earlier deal, Pepys’s first in
class, dual small molecule and antibody combination treatment for
amyloidosis was licensed to GSK by Pentraxin in February 2009 and is now
progressing towards clinical trials. This approach should be
complementary to treatment with the TTR stabiliser compound for patients
with TTR amyloidosis.
Reference
Simon E. Kolstoe et al. Trapping of
palindromic ligands within native transthyretin prevents amyloid
formation. Proceedings of the National Academy of Science,23 November
2010. Vol. 107 (47); pp 20483-20488. Click here to read in
full.
About Pentraxin Therapeutics Ltd
Pentraxin Therapeutics Ltd is a
company spun out of UCL by UCL Business (UCLB). Formed in 2001, it holds
all the intellectual property and proprietary knowledge emanating from
the research of Professor Mark Pepys at UCL. Major IP within Pentraxin
covers treatments for amyloidosis and amyloid-related diseases such as
Alzheimer's disease and type II diabetes, as well as targeting the
pathogenic effects of C?reactive protein in cardiovascular disease and
inflammatory diseases.
About the Wellcome Trust
The Wellcome Trust is a global
charitable foundation dedicated to achieving extraordinary improvements
in human and animal health. It supports the brightest minds in
biomedical research and the medical humanities. The Trust’s breadth of
support includes public engagement, education and the application of
research to improve health. It is independent of both political and
commercial interests. Click here
for further information.
Further Information
Dr Joanna Davidge, R&D Programme Manager, Pentraxin Therapeutics Ltd. Email: j.davidge@medsch.ucl.ac.uk
