Shares in Oxford BioMedica were on the rise yesterday after it announced encouraging data from a Phase I/II trials of ProSavin, backing earlier findings indicating that the drug is safe and effective in patients with Parkinson's disease.

One of the company’s flagship candidates, ProSavin is administered directly into the brain to deliver the genes for three enzymes - AADC (aromatic amino acid decarboxylase), TH (tyrosine hydroxylase) and CH1 (GTP-cyclohydrolase 1) - that transform certain cells into ones able to produce the neurotransmitter dopamine. Parkinson’s disease is characterised by the degeneration of cells that produce dopamine, and so the drug is designed to replenish stock in patients with the illness and thereby reverse some of the associated symptoms.

The drug has performed well in clinical trials to date, and these latest three-month data - from a third patient cohort of the ongoing Phase I/II study - show ProSavin to be safe and well-tolerated following treatment with a 2x dose of the drug, delivered using a new enhanced administration technique developed by the firm to cut the surgical delivery time and facilitate higher dosing.

Aside from adding weight to ProSavin's safety profile, the results also show an average motor function improvement of 26%, which is consistent with 28% improvement using the old administration technique at the 2x dose, the group said, and noted that all three patients showed improvements in at least one indicator of clinical benefit.

Going forward, an independent Data Monitoring Committee is supporting the firm's plans to test a 5x dose of the drug via its enhanced administration technique in a six-patient cohort, which it intends to kick off sometime during the first quarter of 2011, and has also given the green light for a second site in the UK at Addenbrookes Hospital, Cambridge. 

"With the excellent safety profile and sustained, positive signs of clinical benefit we have seen at the lower doses, we are confident that progressing ProSavin to the 5x dose could further enhance efficacy and therefore significantly increase the product’s value," commented Oxford BioMedica's chief executive John Dawson.