Oxford BioMedica Announces Two Scientific Presentations of ProSavin in Parkinson's Disease
– 16th Annual Congress of European Society of Gene and Cell Therapy –
– 38th Annual Meeting of Society for Neuroscience –
Oxford, UK – 14 November 2008: Oxford BioMedica (LSE: OXB), a leading gene therapy company, announced today that the principal investigator for the Phase I/II trial of the Company’s novel gene-based therapeutic, ProSavin, in Parkinson’s disease is presenting an overview of the programme at two scientific meetings:
1) 16th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) in Bruges, Belgium, on 13-16 November 2008. Presentation at 9.40am CET on Friday, 14 November (session: Progress in Clinical Gene Therapy; presentation title: ProSavin: A gene therapy for Parkinson’s disease).
2) 38th Annual Meeting of the Society for Neuroscience in Washington DC, USA, on 15-19 November 2008 (http://www.sfn.org/). Presentation at 3.00pm EST on Wednesday, 19 November (session: Parkinson’s Disease Interventions in Animal Models and Humans; presentation title: A Phase I/II clinical trial for Parkinson’s disease using ProSavin).
The presenter and principal investigator for the trial is Professor Stéphane Palfi, Head of Neurosurgery at the Henri Mondor Hospital in Paris, France. In his presentations, Professor Palfi will review the preclinical studies of ProSavin and will provide an update on the ongoing Phase I/II trial.
The preclinical data to be presented include new results from a recent study that suggest ProSavin may prevent dyskinesias as well as providing sustained recovery of movement and behavioural function. The Phase I/II trial has progressed to evaluation of the second dose level of ProSavin. Professor Palfi will present initial data from the three patients treated with the low dose that indicate ProSavin is safe and well tolerated. All three patients were ambulatory within 48 hours of the procedure. All three patients have been assessed at three months following treatment. There have been no adverse events related to ProSavin and no exacerbations of dyskinesia. In addition, none of the patients has shown unexpected immunological responses to their treatment.
The low dose of ProSavin is potentially below the range that is expected to yield a substantive therapeutic effect in patients. However, the three patients treated showed improvements in disease symptoms, as measured by the Unified Parkinson’s Disease Rating Score (UPDRS) in the ‘off’ state, of up to 30% at three months. The UPDRS ‘off’ score measures the degree of patients’ mobility in the absence of standard of care dopaminergic therapies.
The first patient of the second cohort at the higher dose was treated in September 2008. Assuming the trial progresses as planned, the Company anticipates preliminary data from the three patients at the higher dose and the start of the second stage of the trial in the first half of 2009.
Professor Palfi commented on the ProSavin trial: "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 low dose level. I look forward to evaluating the product’s therapeutic potential as we progress with the evaluation of a 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, Chief Executive Officer of Oxford BioMedica, added: "We welcome the support of Professor Palfi for our ProSavin programme and the opportunity to update the scientific community with our progress. We are pleased by the initial results of the Phase I/II trial and we look forward to further data over the coming months. 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."
For further information, please contact:
Oxford BioMedica plc:
John Dawson, Chief Executive Officer
Nick Woolf, Chief Business Officer
Tel: +44 (0)1865 783 000
JPMorgan Cazenove Limited:
James Mitford/ Gina Gibson
Tel: +44 (0)20 7588 2828
Lisa Baderoon/ Mark Court/ Mary-Jane Johnson
Tel: +44 (0)20 7466 5000
Scientific/Trade Press Enquiries:
Katja Stout/ Holly Griffiths/ John McIntyre
College Hill Life Sciences
Tel: +44 (0)20 7457 2020
The Trout Group LLC
Tel: (646) 378 2900
Notes to editors
1. Oxford BioMedica
Oxford BioMedica (LSE: OXB) is a biopharmaceutical company specialising in cancer immunotherapy and gene-based therapies. The Company was established in 1995, as a spin-out from Oxford University, and is listed on the London Stock Exchange. The Company has a platform of gene delivery technologies, which are based on highly engineered viral systems. Oxford BioMedica also has in-house clinical, regulatory and manufacturing know-how. The lead product candidate is TroVax®, a therapeutic vaccine for multiple solid cancers, which is licensed to sanofi-aventis for global development and commercialisation. TroVax is in Phase III development. Oxford BioMedica has three other products in clinical development, including ProSavin®, a novel gene-based treatment for Parkinson’s disease, in a Phase I/II trial. The Company is underpinned by over 80 patent families, which represent one of the broadest patent estates in the field. Oxford BioMedica has collaborations with sanofi-aventis, Wyeth, Sigma-Aldrich, MolMed and Virxsys. Technology licensees include Biogen Idec, Merck & Co, GlaxoSmithKline and Pfizer. Further information is available at www.oxfordbiomedica.co.uk
ProSavin is Oxford BioMedica’s novel gene-based therapeutic for the treatment of Parkinson's disease. The product is administered directly to the striatum in the brain. It delivers three genes required to convert cells that normally do not produce dopamine into cells that do, thereby replacing the dopamine synthesising cells lost during the course of the disease. ProSavin utilises Oxford BioMedica’s proprietary LentiVector® system to deliver the genes AADC (aromatic amino acid decarboxylase), TH (tyrosine hydroxylase) and CH1 (GTP-cyclohydrolase 1). These genes reprogramme transduced cells to manufacture and secrete dopamine.
3. Phase I/II trial of ProSavin
The Phase I/II trial is being conducted at the Henri Mondor Hospital in Créteil, which is a European centre of excellence for neurosurgery and a member of the Assistance Publique Hôpitaux de Paris (APHP) in France. The study is designed to assess the safety and efficacy of ProSavin in patients with Parkinson’s disease who are failing on current treatment with L-DOPA but have not progressed to experiencing marked drug-induced movement disorders called dyskinesias. The first stage is an open-label dose escalation to evaluate at least two dose levels of ProSavin in cohorts of three patients each. In the second stage of the trial, a further 12 patients will be recruited, some of which will act as a control group and only receive “sham” surgery. Efficacy is assessed at regular intervals using the Unified Parkinson’s Disease Rating Score (UPDRS). The primary endpoints of the study are: 1) the number and severity of any adverse events associated with the administration of ProSavin, including the incidence of dyskinesias; and 2) efficacy based on the UPDRS assessment at six months after treatment. The secondary objectives of the trial include the extent to which patients’ current L-DOPA therapy can be reduced or removed following administration of ProSavin.
LentiVector is a highly effective gene delivery system. It is based on an engineered lentiviral vector, which is harmless to humans. The LentiVector system has been shown to express its genetic payload efficiently and stably in multiple tissue types, and is particularly effective in targeting non-dividing cells, such as neurons in the brain. In addition, genes delivered with a LentiVector system have shown long-term stable expression and, hence, offer long-term therapeutic benefit.
5. Parkinson’s Disease
Parkinson's disease is a progressive movement disorder that requires care over a period of 10-15 years. It is caused by the degeneration of dopamine producing nerve cells in the brain. Dopamine is a neurotransmitter involved in controlling movement and coordination. As patients’ dopamine levels decrease, they exhibit progressive inability to initiate and control physical movements. The disease affects 1% of the over 50 population and about 10% of over 60s, which equates to about one million people in the USA. The current worldwide market for Parkinson's disease products is estimated to be approximately US$3 billion. None of the current treatments provide long-term relief from symptoms.
Posted: November 2008