Potential new approach to the treatment of systemic amyloidosis
Pentraxin Therapeutics Ltd announces publication of results, from first in human clinical trial.
In 2009 GSK and Pentraxin Therapeutics Ltd, a UCL spinout company created to develop the intellectual property of Professor Sir Mark Pepys and his team, entered into collaboration to develop the world’s first dual drug-antibody treatment for the rare disease systemic amyloidosis.
The results of the first in human clinical trial have today been published online in the New England Journal of Medicine. The publication shows the results of the first 15 patients treated with a therapeutic partnership of CPHPC (a small chemical molecule) and an anti-SAP antibody.
Amyloid is an abnormal protein material that accumulates in the tissues, damaging their structure and function and causing a rare and usually fatal disease called amyloidosis. Present treatments can stabilise some patients and substantially prolong life but about 20% of patients still die within 6 months of diagnosis.
The results of the phase I study showed that the antibody was generally well tolerated and produced rapid clearance of amyloid from various organs. Removal of amyloid from the liver was associated with improved function. Further clinical testing is in progress to establish functional improvement in other organs and a phase II trial is planned.
Professor Sir Mark Pepys FRS, Director of the UCL Wolfson Drug Discovery Unit, said “Amyloidosis is a very challenging clinical condition. It causes serious ill health and is usually fatal, despite best efforts to support the patients and to control the underlying conditions responsible for amyloid formation. Furthermore amyloidosis can present in so many different ways that, coupled with its relative rarity, the diagnosis is often long delayed. As a result patients frequently have advanced disease before they receive any treatment. We have long recognised that measures to remove amyloid from the tissues are required and have been working on this problem for over 30 years. We were finally successful for the first time in experimental models 10 years ago. The collaboration with GSK started in 2009 and it has been a privilege to work with their expertise and resources to bring our approach into clinical testing. Seeing clearance of amyloid deposits from patients’ tissues has been thrilling. It is a crucial first step on the long path towards having a medicine that could transform the outlook for people suffering from this terrible disease.”
Dr Duncan Richards, Head of GSK’s Academic Discovery Performance Unit, said “Establishing proof of mechanism is a vital milestone that needs to be determined early in the path of developing a medicine so we are pleased to have this confirmed for the first time in patients. The initial results of the therapeutic intervention of CPHPC and the anti-SAP antibody are encouraging and we are now actively planning the next stages of development to better understand its potential benefits and safety in patients.”
Mr Cengiz Tarhan, Managing Director of UCLB and Director of Pentraxin Therapeutics Ltd said “Having now established proof of mechanism, we look forward to developing this novel treatment in partnership with 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 Sir 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 both research and clinical management of amyloidosis (www.ucl.ac.uk/medicine/amyloidosis)
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.
About UCL Business PLC
UCL Business PLC (UCLB) is a leading technology transfer company that supports and commercialises research and innovations arising from UCL, one of the UK’s top research-led universities. UCLB has a successful track record and a strong reputation for identifying and protecting promising new technologies and innovations from UCL academics. UCLB has a strong track record in commercialising medical technologies and provides technology transfer services to UCL’s associated hospitals; University College London Hospitals, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital. It invests directly in development projects to maximise the potential of the research and manages the commercialisation process of technologies from laboratory to market.
For further information, please visit: www.uclb.com