UCL teams up with GlaxoSmithKline to develop drug for rare disease

Collaboration

UCL and GlaxoSmithKline are to collaborate to develop a treatment for the rare and often fatal disease amyloidosis. The agreement focuses on the work of Mark Pepys, founder of spin-out Pentraxin Therapeutics, who has spent 30 years researching the disease.

Amyloidosis is caused by build up of amyloid proteins 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.

Pepys previously had a research agreement with Roche to develop a small molecule drug, CHPHC, for treating amyloidosis, but promising preclinical work did not play out in clinical trials. Under the new collaboration the aim is to link CHPHC to an antibody that will target it directly to abnormal amyloid in affected organs.

Pepys, who is head of the UCL Centre for Amyloidosis and Acute Phase Proteins, said combining CPHPC with a targeting antibody has been shown to trigger a rapid clearance of amyloid deposits in mouse models of the disease.

Under the new agreement, research teams from UCL and GSK will work to convert the mouse antibody into one for use in humans in combination with CPHPC.

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