There were no serious adverse events associated with the surgical procedure for administration of ProSavin into the brain. All three patients were ambulatory within 48 hours of the procedure. At three months post ProSavin administration, patients showed an improvement of 23-30% as measured by the UPDRS ‘off’ score. This measures the degree of mobility in the absence of standard of care dopaminergic therapies. Two patients showed improvements in Quality of Life measures (QoL) that are specific to patients with Parkinson’s disease, and one patient’s QoL was unchanged.
The primary efficacy endpoint of the study is based on the UPDRS Part III (motor score) assessment at six months after treatment. The principal investigator for the trial, Professor Stéphane Palfi from the Henri Mondor Hospital in Paris, intends to report the detailed interim results of the study, which may include the six-month UPDRS data and the imaging study performed at the Commissariat à l’Energie Atomique and Service Hospitalier Frédéric Joliot in Orsay, at the 16th Annual Congress of the European Society of Gene and Cell Therapy in Bruges, Belgium, on 13-16 November 2008.
The independent DMC has reviewed the three-month data and recommended that the trial advances to the evaluation of the higher dose of ProSavin. The first patient of this second cohort is expected to be treated in September 2008. Assuming the trial progresses as planned, the Company anticipates preliminary data from patients at the higher dose and the start of the second stage of the trial in the first quarter of 2009.
Professor Palfi commented on the interim data: "The initial data suggest that ProSavin has an excellent safety profile. I am encouraged by the early trend in patients’ UPDRS measurements, particularly given that this is the lower dose level. I look forward to confirming this trend over the next few months and also advancing the study to evaluate patients receiving the higher dose. ProSavin has the potential to address an unmet medical need in Parkinson’s disease, offering long-lasting benefit from a single administration. I am very pleased to be involved in the first clinical trial of this potentially exciting new treatment paradigm for patients with Parkinson’s disease."
John Dawson, Acting Chief Executive Officer of Oxford BioMedica, added: "We are pleased by the progress of the first cohort of patients and we look forward to the maturing data over the coming months. The favourable recommendation from the DMC to proceed to the higher dose of ProSavin is an important milestone for the trial. This is the first trial using our proprietary LentiVector technology and, as such, the preliminary safety conclusions add value not only to ProSavin, but also to our other development candidates that use the same technology."
