UCL and GSK join forces to develop combined small molecule-antibody treatment for rare disease

UCL (University College London) and GlaxoSmithKline join forces to develop combined small molecule-antibody treatment for rare disease

A collaboration to develop a world first drug-antibody dual treatment for the rare and often fatal condition amyloidosis has been formed between the University College London spinout company Pentraxin Therapeutics Ltd and GlaxoSmithKline (GSK).

Amyloidosis is a disease caused by build up of abnormal proteins (amyloid) in body tissues, leading to organ failure. The heart, kidneys, liver and almost any other organ can be affected. Around 500 new cases are diagnosed each year in the UK. Despite the best available therapy, the prognosis for patients with amyloidosis is poor and new treatments are urgently needed.

"We initially developed the small molecule drug, CHPHC, and while we had promising early results, they were not enough to benefit patients with advanced disease. Something more dramatic is needed," explained Professor Mark Pepys FRS, the head of Pentraxin and the UCL Centre for Amyloidosis and Acute Phase Proteins which includes the UK National Amyloidosis Centre.

"We then combined CPHPC treatment with an antibody that seeks out the amyloid deposits in the organs in mice. This combination triggered a rapid clearance of the deposits."

With this new agreement, the research teams from UCL and GSK will work together to convert the mouse antibody into one that can be used in humans in combination with CPHPC. The aim is to find out if the benefits seen in the animal model can be replicated in patients with amyloidosis.

The collaboration brings together UCL's clinical and science expertise and the development expertise of GSK's Academic Discovery Performance and Biopharm Units.

"We are delighted to enter into this alliance," said Mike Owen, Senior Vice President, Biopharmaceutical Research, GSK. "Our biopharmaceutical and clinical development capabilities and Prof Pepys's team's knowledge of the disease provide a synergistic collaboration that will greatly enhance our chances of success."

Under the terms of the agreement, Pentraxin will receive undisclosed early stage success-based milestones plus drug development milestones and royalties.

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 both research and clinical management of amyloidosis (www.ucl.ac.uk/medicine/amyloidosis)

Founded in 1826, UCL was the first English university established after Oxford and Cambridge, the first to admit students regardless of race, class, religion or gender, and the first to provide systematic teaching of law, architecture and medicine. UCL is the seventh-ranked university in the 2008 THES-QS World University Rankings, and the third-ranked UK university in the 2008 league table of the top 500 world universities produced by the Shanghai Jiao Tong University. UCL alumni include Marie Stopes, Jonathan Dimbleby, Lord Woolf, Alexander Graham Bell, and members of the band Coldplay. UCL currently has over 12,000 undergraduate and 8,000 postgraduate students. Its annual income is over £600 million.

UCL Business PLC (UCLB) is the organisation responsible for commercialising research across all disciplines, generated from within UCL and associated organisations. It is primarily responsible for protecting inventions and transacting commercial activity including options, licences and collaborative commercial research. UCLB also has responsibility for creating and spinning-out companies from UCL. UCLB is wholly-owned by UCL and operates as an independent company with its own Board of Directors.

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.

For more information contact:
UCL/Pentraxin: Professor Mark Pepys 020 7433 2786
GlaxoSmithKline: Janet Morgan 020 8996 2417