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First Parkinson's Gene Therapy Planned by Oxford Biomedica By Nuala Moran BioWorld International Correspondent LONDON - Oxford Biomedica plc said the first patient is within days of being treated with ProSavin, its gene therapy treatment for Parkinson's disease. The neurosurgery required to administer it has been booked, but the company has agreed with the Henri Mondor Hospital in Paris where the trial is taking place not to disclose the actual date. It is the first European trial of a gene therapy to treat Parkinson's disease and also the first use of Oxford BioMedica's LentiVector gene delivery system in humans. ProSavin delivers genes for the three enzymes, tyrosine hydroxylase, GTP-cyclohydrolase 1 and aromatic amino acid decarboxylase, that are required for the synthesis of the neurotransmitter dopamine. The product is administered by microinjection into the striatum, where it works to convert the target cells into a replacement dopamine factory. In animal models, that translates to improvements in movement within one to two weeks and full recovery within five to eight weeks. To date the effect of a single injection has been maintained for 27 months. "Essentially this is a cure with a single administration," said Alan Kingsman, CEO. "This is remarkable by any standards. We are not aware of any other product that does so well in this model, other than L-Dopa." But while L-dopa is effective in the early stages of Parkinson's disease, as the substantia nigra where it is processed into dopamine is destroyed by the disease, it becomes ineffective. The 18 patients in the Phase I/II trial will be failing on L-Dopa. First data are expected around the middle of this year, and if the effect in animals is replicated Kingsman said Prosavin will go into a Phase III trial in late 2009. Oxford BioMedica also said it has completed recruitment of the first Phase III trial of its cancer immunotherapeutic TroVax in treating advanced renal cancer, putting it on track to get approval before the end of 2009 and triggering a substantial milestone payment from partner Sanofi-Aventis SA. The company has agreed on a special protocol assessment with the FDA, which means it needs only to confirm that the trial meets its primary endpoint of an increase in survival. The analysis will be carried out once the 309th death occurs in the 700 patient trial, an event that is expected to occur in the first half of 2009. "We are tracking our data very carefully: 82 percent of case reports are in the data center. That is a very high proportion of data to be live at this time," said Chief Medical Officer Mike McDonald. "Once we have 309 events, we can close in 10 weeks. It leaves us with one data point to discuss with the FDA, and that is the primary endpoint." At the same time, Sanofi-Aventis is in the final stages of preparing for a Phase III trial in metastatic colorectal cancer, which will recruit 1,300 patients. "The protocol has been submitted, and there have been positive discussions with the FDA. No fundamental issues have been raised," McDonald said. Another 3,000 patient UK Phase III trial of TroVax as an adjuvant treatment in early stage colorectal cancer is due to kick off in mid-2008. "TroVax is making steady progress to commercialization," he said. The product targets the 5T4 antigen that is expressed on the surface of all solid tumors apart from melanoma and does not occur elsewhere in the body. The amount of antigen increases as the tumor develops. To date TroVax has an impeccable safety record, with no patient choosing to withdraw from treatment, and more than 90 percent mounting an immune response. Antibody levels generated are similar to that reported for other cancer vaccines, but TroVax also elicits a cellular immune response. "That's what distinguishes it from other [cancer vaccines] out there," McDonald said. In all but one trial there was a direct correlation between the strength of the immune response and the increase in survival. Oxford BioMedica outlined the progress with the portfolio as it reported its 2007 financial results. Those show how the ?518 million (US$794.4 million) deal with Sanofi-Aventis signed a year ago, has transformed the fortunes of the Oxford, UK-based company. "We were cash generative in 2007 for the first time, and at the year end had £38 million cash," Kingsman said. He added that 2007 "was a great year, we really enjoyed it." Unfortunately, that rosy assessment has not been reflected in the market, and the company's share price has halved in the past 12 months. The results and progress report prompted a further fall of £0.75 to £27.25. Rayilyn Brown Board Member AZNPF Arizona Chapter National Parkinson's Foundation [log in to unmask] ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn