IntraBio, co-founded by Department of Pharmacology academics Professors Fran Platt, Antony Galione and Grant Churchill, ran a successful international clinical trial for a new drug Aqneursa which showed significantly improved neurological outcomes over a 12 week period for a cohort of 60 patients, aged four years and over, with a confirmed diagnosis of NPC compared to a control group. Despite the recent proliferation of Drug Discovery Units in many Universities, it is nevertheless still rare for academics to take a new molecular entity from pre-clinical to phase III clinical trials culminating in regulatory approval and marketing for patients. The first prescriptions for Aqneursa have already been written.
NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. It is caused by changes in either the NPC1 or NPC2 gene, affecting the transport of cholesterol and other lipids out of the lysosome. As a result, cells do not function as they should, ultimately causing organ damage. On average, individuals affected by this devastating disease only live for about 13 years. The new drug, Aqneursa, is only the third drug approved for the treatment of NPC, including miglustat, which was the first drug to be approved for lysosomal storage diseases that was developed by the Platt laboratory.
Professor Fran Platt, Head of the Department of Pharmacology and academic co-founder of IntraBio, said “The effects of NPC on patients and their families is devastating. I am delighted that our pre-clinical studies, which have been taken forward by IntraBio into full clinical trials, offer hope to those suffering the impact of this rare disease. It is very gratifying to see such impact from our research.”
Antony Galione, Statutory Professor of Pharmacology and scientific co-founder of IntraBio, said “It has been an amazing and unprecented journey for our small company to so rapidly and efficiently take a new drug molecule from the lab to the clinic. It has been a steep learning curve for all those involved. Aqneursa will have real impact on the lives of patients with these rare genetic diseases, but the drug is also showing great promise for more common neurological diseases, which may have a huge impact there”.
Grant Churchill, Professor of Chemical Pharmacology and scientific co-founder of IntraBio, said “It’s incredibly gratifying at several levels: from identifying the potential and then developing a drug that didn’t fit the current pipeline model of drug discovery or tick all the potential boxes of traditional funders to discovering the crucial role of the monocarboxylate transporter in the drug’s uptake and distribution to the brain”.