Pentraxin announces licensing deal with GSK to develop medicine for rare disease

Published on 23 February 2011

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 theProceedings 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.


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:

Print friendly version of Pentraxin announces licensing deal with GSK to develop medicine for rare disease