Treatment for amyloidosis a step closer

UCL spin out company, Pentraxin Therapeutics Ltd, has hit the first milestone in its collaboration with pharmaceutical giant GlaxoSmithKline.

Pentraxin owns the IP and proprietary knowledge of Professor Mark Pepys FRS, including his invention of a new therapy for systemic amyloidosis. Systemic amyloidosis is a serious and usually fatal disease that can affect virtually any organ in the body. It is the cause of death in one per thousand of the population in developed countries and effective treatments are urgently needed.

In 2009 GSK licensed Professor Pepys’s new therapy and is developing it in collaboration with Pentraxin for early clinical trials.

The first published description of the underlying research appeared online last week and will be in print in the prestigious scientific journal Nature on 4 November 2010. The paper demonstrates efficacy of this first in class, dual small molecule?antibody therapy in an experimental model.

Amyloid is composed of abnormal protein fibres that are deposited in the body’s tissues, damaging their structure and function. Diagnosis is often difficult and delayed so that, by the time the disease is recognised, most patients already have irreversible organ damage. The SAP protein from the blood accumulates in amyloid deposits and contributes to their formation and persistence. In work supported by the MRC spanning 30 years, Professor Pepys and his team from UCL discovered the role of SAP in amyloidosis and have been developing new treatments aimed at it. Initially they partnered with Roche to develop a small molecule drug, called CPHPC, which removes SAP from the blood but only partly clears it from amyloid deposits. This treatment stopped the accumulation of new amyloid but did not clear the existing deposits.

The latest development uses CPHPC to first remove SAP from the blood so that antibodies to SAP can then be safely given to target the residual SAP in the amyloid deposits. In experimental models closely resembling human disease, this treatment swiftly eliminated all the amyloid. The next stage will be to test the treatment in patients and, as mentioned above, work towards clinical trials is proceeding well in collaboration with GlaxoSmithKline.

Professor Pepys, Director of the UCL Centre for Amyloidosis and Acute Phase Proteins, said: “Our findings open up the prospect of a successful treatment for patients with amyloidosis and we are looking forward to continuing the development of the drugs for testing in the first human studies with our commercial partner GSK.”

About Pentraxin Therapeutics
Pentraxin Therapeutics Ltd is a company spun out from University College London (UCL) by UCL Business PLC (UCLB) to hold and develop the intellectual property of Professor Mark Pepys and his colleagues in the UCL Centre for Amyloidosis and Acute Phase Proteins. This clinical and basic science research centre houses the UK NHS National Amyloidosis Centre and it leads the world in research and clinical management of amyloidosis (www.ucl.ac.uk/medicine/amyloidosis).

About GlaxoSmithKline
GlaxoSmithKline – one of the world’s leading research-based pharmaceutical and healthcare companies – is committed to improving the quality of human life by enabling people to do more, feel better and live longer.

For company information, visit GlaxoSmithKline at: www.gsk.com.

Further Information
Professor Mark Pepys FRS , UCL/Pentraxin, 020 7433 2786