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2 February, 2017 - 13:01 By Kate Sweeney

University spin-out raises £14m to develop haemophilia drug

haemophilia, cambridge institute for medical research

A Cambridge University spin-out developing a drug to tackle haemophilia has raised £14 million Series A funding to target a $10 billion market.

ApcinteX is developing a novel treatment based on the work of Professor Jim Huntington (Cambridge Institute for Medical Research) and Dr Trevor Baglin (Cambridge University Hospitals), world-renowned experts in blood clotting disorders.

The duo were behind XO1, based on their discovery of an anticoagulent which has the potential to prevent heart attack and stroke without causing bleeding.

The founders described the antibody – ichorcumab  – as “the blood of the gods” and a “one in a billion” medical breakthrough, comparing it to Fleming’s discovery of penicillin 85 years ago. Global pharma giant Janssen bought XO1 in 2015 for an undisclosed sum not long after the fledgling venture – then Business Weekly’s Startup Company of the Year – had raised $11m startup cash.

The medical pioneers have high hopes that their latest enterprise ApcinteX will have similar impact as the drug they propose would tackle all types of haemophilia. Around 400,000 individuals globally are affected by haemophilia, a genetic disorder that causes uncontrolled bleeding as a result of patients having a deficiency in proteins required for normal blood clotting.

Currently, the standard treatment is administration of the missing clotting factor, but this requires regular intravenous injections and is not completely effective.

Also, about one quarter of patients develop inhibitory antibodies to the administered clotting factor which renders further treatment ineffective.

ApcinteX says its treatment seeks to turn down the activity of a key natural anticoagulant pathway to produce normal blood clotting in patients with haemophilia.

This means the drug could potentially treat patients with all types of haemophilia, including those who develop antibodies to replacement factors. Nor does the drug cause anti-clotting antibodies to form so it could be administered fortnightly by simple injection under the skin.

Medicxi and Touchstone Innovations have co-led the funding round. Cambridge Enterprise, the university’s commercialisation arm, helped in ApcinteX’s formation – licensing key intellectual property to the company – and has co-invested.

Dr Baglin said: “Bearing in mind that the majority of people in the world with haemophilia have no access to effective therapy, a stable, easily administered, long-acting, drug that can be used in all patients, regardless of the type of haemophilia, could bring treatment to a great deal many more people who suffer from haemophilia.”

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