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GlaxoSmithKline and UCL to develop biologic

Published on 24/03/09 at 11:52am

Researchers at University College London are to work with GlaxoSmithKline to develop a new treatment for the rare and often fatal condition amyloidosis.

The disease is 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, and the prognosis for patients is poor with new treatments urgently needed.

UCL's spinout commercialisation company Pentraxin Therapeutics will work with GSK to transfer the drug from animal testing into use in humans.

The drug candidate has emerged from work done at UCL's Centre for Amyloidosis and Acute Phase Proteins, which includes the UK National Amyloidosis Centre.

The treatment is the first of its kind, and combines a small molecule with an antibody. Professor Mark Pepys, head of the unit and of Pentraxin Therapeutics, explained: "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 was needed."

He added: "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."

The agreement will see Pentraxin receive undisclosed early-stage success-based milestones plus drug development milestones and royalties.

"We are delighted to enter into this alliance," said Mike Owen, senior VP, biopharmaceutical research at 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."

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