GlaxoSmithKline has hooked up with University College London spinout firm Pentraxin Therapeutics to develop a promising new treatment for the rare condition amyloidosis.

Amyloidosis is a potentially fatal disease caused by excess abnormal amyloid proteins in the body tissues, leading to organ failure. The condition is rare, with around 500 new cases in the UK diagnosed each year, but the prognosis for patients is poor, highlighting the urgent need for new effective therapies.

In what UCL and GSK claim is a “world first”, the groups are working together to develop a dual treatment for the disease that combines the power of a small molecule therapy, called CPHPC, with an antibody.

Explaining the background behind project, Professor Mark Pepys FRS, Head of Pentraxin, said while CHPHC had “promising results” in the treatment of early stages of the disease, the effect was less beneficial in patients with advanced disease. “We then combined CPHPC treatment with an antibody that seeks out the amyloid deposits in the organs in mice [and] this combination triggered a rapid clearance of the deposits.”

Research teams from UCL and GSK will join forces to convert the mouse antibody into one that can be used in humans in with CPHPC, in the hope that a combination of GSKs clinical development capabilities and Prof Pepys’ knowledge of the disease will help replicate the benefits in patients with the disease.

Under the terms of the deal, Pentraxin will receive undisclosed early stage success-based and drug development milestones and royalties. No further details were disclosed.