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ProSavin®

ProSavin is a gene-based treatment for Parkinson's disease, a progressive movement disorder caused by the degeneration of dopamine producing nerve cells in the brain. In Parkinson's disease, there is degeneration of the cells in the brain that produce dopamine. ProSavin uses the Company's LentiVector system to deliver the genes for three enzymes that are required for the synthesis of dopamine. The product is administered locally to the region of the brain called the striatum, converting cells into a replacement dopamine factory within the brain, thus replacing the patient's own lost source of the neurotransmitter.

Encouraging Phase I/II results

ProSavin is being evaluated in a Phase I/II trial in patients with mid-stage Parkinson's disease. The first stage of the study is an open-label dose escalation of ProSavin in cohorts of three patients. The first two dose levels were safe and well tolerated and showed promising evidence of efficacy with a maximum improvement in motor function of 53%. If these results are confirmed, ProSavin would represent a significant advancement to current treatment options.

Enhanced administration

The next cohort is being treated using a new infusion technique that could reduce the surgery time by up to 50% and requires fewer needle tracks into the brain. The technology is capable of delivering higher doses of ProSavin and preclinical studies suggest that it potentially provides higher efficacy per dose compared to the previous administration procedure. Oxford BioMedica aims to reach the optimal dose level in 2010 to advance into randomised studies.

Market opportunity

Parkinson's disease affects approximately 4.1 million people worldwide and the prevalence is rising owing to demographic changes. None of the current treatments provide long-term relief from symptoms, yet, by 2012, sales could exceed US$4.6 billion in the major developed countries (source: Lead Discovery). ProSavin has the potential to address an unmet medical need in Parkinson’s disease, offering long-lasting benefit from a single administration with an excellent safety profile. The product could also reduce the social care burden that is associated with mid to late-stage disease.