Seeding Drug Discovery-funded company announces licensing deal with GSK
18 January 2011
In this latest deal, GSK have taken on Pentraxin's programme to develop small molecules that stabilise transthyretin (TTR), a blood protein linked to 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 fibres. These fibres can accumulate in organs and tissues, damaging their structure and function and causing TTR amyloidosis, a fatal and currently untreatable condition.
Pentraxin has identified a molecule, mds84, which binds to and stabilises TTR, preventing amyloid formation in experimental models of the disease. This research was published on 23 November 2010 in the ' Proceedings of the National Academy of Sciences (USA)'.
Professor Mark 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."
Professor Pepys received one of the first Seeding Drug Discovery Initiative (SDDI) awards 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 that led to this week's licensing deal.
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's research team at UCL, is commencing immediately to develop this latest novel treatment to a point where it can be taken forward into patient trials.
In an earlier deal, Pentraxin's first-in-class dual small-molecule and antibody combination treatment for amyloidosis was licensed by GSK in February 2009; it is progressing successfully toward clinical trials. This approach should be complementary to treatment with the TTR stabiliser compound for patients with TTR amyloidosis.
Pentraxin formed in 2001 as a spin-out company from UCL, holding the intellectual property and proprietary knowledge from Professor Pepys's research at UCL.
