13th Dec 2007 07:00
Oxford Biomedica PLC13 December 2007 For Immediate Release 13 DECEMBER 2007 OXFORD BIOMEDICA INITIATES PHASE I/II TRIAL OF PROSAVIN(R) GENE-BASED TREATMENT FOR PARKINSON'S DISEASE Oxford, UK - 13 December 2007: Oxford BioMedica (LSE: OXB), a leading genetherapy company, announced today that it has initiated a Phase I/II trial ofProSavin, its novel gene-based treatment for Parkinson's disease. This followsregulatory clearance from the French Health Products Safety Agency (AFSSAPS) ofthe Company's Clinical Trial Application. Patient recruitment for the Phase I/IItrial will start immediately. The trial is being conducted at the Henri MondorHospital in Creteil, which is a European centre of excellence for neurosurgeryand a member of the Assistance Publique Hopitaux de Paris (APHP) in France. The primary objectives of the trial are to assess the safety and efficacy ofProSavin. The analyses of patients will include the application of advancednon-invasive neuro-imaging techniques, in collaboration with the Commissariat al'Energie Atomique (CEA) and Service Hospitalier Frederic Joliot (SHFJ)/Molecular Imaging Research Centre (MIRCen) in Orsay, France. ProSavin uses gene therapy to restore dopamine production in the brain.Parkinson's disease is caused by the degeneration of dopamine producing nervecells, leading to movement impairments. The product uses the Company'sLentiVector(R) system to deliver the genes for three enzymes (tyrosinehydroxylase, GTP-cyclohydrolase 1 and aromatic amino acid decarboxylase) thatare required for the synthesis of dopamine. ProSavin is administered locally tothe region of the brain called the striatum, converting the target cells into areplacement dopamine factory within the brain, thus replacing the patient's ownlost source of the neurotransmitter. Long-term efficacy data in the industry-standard preclinical model ofParkinson's disease have shown that ProSavin induces almost complete recovery ofmovement function and other behavioural measurements. In this model, thetherapeutic effect of ProSavin following a single administration has beenmaintained for over 24 months with no diminishment. Professor Alan Kingsman, Chief Executive of Oxford BioMedica, commented: "Thestrength of the preclinical safety and efficacy data with ProSavin hasestablished strong scientific support for the clinical development of thispioneering product candidate for Parkinson's disease. If ProSavin's safety andefficacy profile is replicated in humans, then the product could represent afundamentally new approach for the treatment of Parkinson's disease and couldsignificantly expand the worldwide market for existing therapies, which isestimated to be approximately US$3 billion." The Phase I/II trial of ProSavin ProSavin will be the first gene-based treatment for Parkinson's disease to beevaluated in a European clinical trial. Patients in the trial will have beendiagnosed with Parkinson's disease and will be failing on current treatment withL-DOPA but they will not have progressed to drug-induced dyskinesias. It is atwo-stage study. The first stage is an open-label dose escalation to evaluatetwo dose levels of ProSavin in cohorts of three patients each. Oxford BioMedicaplans to report preliminary results once the first cohort of patients isassessable, which is expected in mid-2008. In the second stage of the trial, afurther 12 patients will be recruited to confirm efficacy of the optimal dose. Current surgical approaches to the treatment of Parkinson's disease require thedestruction of brain tissue or the permanent placement of electrodes in the caseof deep brain stimulation. These treatments have certain limitations andside-effects. The surgical procedure for administration of ProSavin entailsstereotactic bilateral injection into the striatum under general anaesthesiausing MRI-imaging and mapping. The procedure is designed to be non-destructiveto tissue and does not leave any device in the brain. The efficacy of ProSavin will be assessed using the Unified Parkinson's DiseaseRating Score (UPDRS). Patients will be monitored at regular intervals, with theprimary endpoint being an efficacy assessment at six months after treatment. Thesecondary objective of the trial is to asses the extent to which patients'current therapy (L-DOPA) can be reduced following administration of ProSavin. The principal investigator for the trial is Stephane Palfi, MD, PhD, aneuroscientist at MIRCen/SHFJ-CEA and neurosurgeon at the Henri Mondor Hospital.Dr. Palfi commented on the start of the trial: "Current standard therapy forParkinson's disease is only partially effective in the mid to late stage ofdisease and can induce debilitating side-effects after long-term use. ProSavinhas the potential to address this unmet medical need, offering long-lastingbenefit from a single administration. I am very pleased to be involved in thefirst clinical trial of this potentially exciting new treatment paradigm forParkinson's disease." Professor Alan Kingsman of Oxford BioMedica added: "We are delighted to havereceived a favourable review from the French authorities that allows us tocommence patient recruitment in the Phase I/II trial. This clinical trial ofProSavin is the culmination of over ten years of research in Oxford BioMedicaand, before that, in Oxford University. It is the first trial using ourproprietary LentiVector technology and, as such, this represents a major eventfor Oxford BioMedica and the future of the pipeline of products that use thesame technology." -Ends- For further information, please contact:Oxford BioMedica plc: Tel: +44 (0)1865 783 000 Professor Alan Kingsman, Chief ExecutiveJPMorgan Cazenove Limited: Tel: +44 (0)20 7588 2828 James Mitford/Gina GibsonCity/Financial Enquiries: Tel: +44 (0)20 7466 5000 Lisa Baderoon/ Mark Court/ Mary-Jane Johnson BuchananCommunicationsScientific/Trade Press Enquiries: Tel: +44 (0)20 7457 2020 Gemma Price/ Holly Griffiths/ Katja Stout College Hill Life Sciences US Enquiries: Tel: (646) 378 2900 Thomas FechtnerThe Trout Group LLC Notes to editors 1. Oxford BioMedica Oxford BioMedica (LSE: OXB) is a biopharmaceutical company specialising in thedevelopment and commercialisation of novel therapeutic vaccines and gene-basedtherapies with a focus on oncology and neurotherapy. The Company was establishedin 1995 as a spin-out from Oxford University, and is listed on the London StockExchange. The Company has a platform of gene delivery technologies, which are based onhighly engineered viral systems. Oxford BioMedica also has in-house clinical,regulatory and manufacturing know-how. In oncology, the lead product candidateis TroVax(R), an immunotherapy for multiple solid cancers, which is licensed tosanofi-aventis for global development and commercialisation. A Phase III trialof TroVax in renal cancer is ongoing and two Phase III trials in colorectalcancer are planned. Oxford BioMedica has two other anti-cancer productcandidates in Phase II development for melanoma and pancreatic cancerrespectively. In neurotherapy, the Company has initiated a Phase I/II trial ofits gene-based treatment for Parkinson's disease, ProSavin(R). The neurotherapypipeline also includes preclinical gene-based therapeutics for vision loss,motor neuron disease and nerve repair. The Company is underpinned by over 80 patent families, which represent one ofthe broadest patent estates in the field. The Company has a staff ofapproximately 80 split between its main facilities in Oxford and its whollyowned subsidiary, BioMedica Inc, in San Diego, California. Corporate partnersinclude sanofi-aventis for TroVax and Wyeth for an anti-cancer targeted antibodytherapy. The Company also has collaborations with Sigma-Aldrich, MolMed andVirxsys. Technology licensees include Biogen Idec, Merck & Co, GlaxoSmithKlineand Pfizer. Further information is available at www.oxfordbiomedica.co.uk 2. AFSSAPS, Agence francaise de securite sanitaire des produits de sante The French Health Products Safety Agency (AFSSAPS) was created by French law on1 July 1998 to strengthen existing monitoring and health safety systems, and toprovide an improved response to the increasing diversity and interaction ofhealth safety issues relating to healthcare products. AFSSAPS benefits not onlyfrom the skills of its forerunner in France, the Medicinal Products Agency, butalso from the fact that its scope of activity has been extended to cover thewhole range of healthcare products. It serves the interests of French publichealth thanks to the high level of expertise it can call upon, both internallyand externally. It is thus able to produce not only scientific analyses andrecommendations, but also tens of thousands of decisions a year, taken on behalfof the State by the Agency's Director General. Its mission is to guarantee thesafety, quality and proper use of health products for human use. 3. Henri Mondor Hospital Henri Mondor Hospital, based in Creteil, is one of the largest hospitals in theGreater Paris area. This teaching and university hospital has become a referencefor treatments, research and teaching not only locally but also for doctors andpatients elsewhere in France and abroad. The hospital's main activities are inneuroscience, cardiology, oncology, genetics and rare diseases. The hospital has17 medicine and intensive care departments and eight surgery departments. 4. SHFJ and MIRCen Imaging Centres of the French Atomic Agency (CEA) The Molecular Imaging Research Centre (MIRCen) brings together skills inmolecular biology, cell biology, electrophysiology and behavioural science withradio-isotope and anatomic functional imaging techniques. The centre designs,develops and validates new therapeutic strategies for the treatment of varioushuman disorders including neurodegenerative diseases such as Parkinson'sdisease. It is based in the Commissariat a l'Energie Atomique (CEA) centre atFontenay-aux-Roses (Paris region). The CEA's Frederic Joliot Hospital Service(SHFJ) based in Orsay (Paris region) is one of the few centres in Europe thatemploy non-invasive methods of human functional exploration (single photonemission tomography, nuclear magnetic resonance imaging and spectroscopy,positron emission tomography) in research as well as clinical departments. 5. ProSavin(R) ProSavin is Oxford BioMedica's novel gene-based therapeutic for the treatment ofParkinson's disease. The product is administered directly to the striatum in thebrain. It delivers three genes required to convert cells that normally do notproduce dopamine into cells that do, thereby replacing the dopamine synthesisingcells lost during the course of the disease. ProSavin utilises OxfordBioMedica's proprietary LentiVector(R) system to deliver the genes AADC(aromatic amino acid decarboxylase), TH (tyrosine hydroxylase) and CH1(GTP-cyclohydrolase 1). These genes reprogramme transduced cells to manufactureand secrete dopamine. 6. LentiVector(R) LentiVector is a highly effective gene delivery system. It is based on anengineered lentiviral vector, which is harmless to humans. The LentiVectorsystem has been shown to express its genetic payload efficiently and stably inmultiple tissue types, and is particularly effective in targeting non-dividingcells, such as neurons in the brain. In addition, genes delivered with aLentiVector system have shown long-term stable expression and, hence, offerlong-term therapeutic benefit. 7. Parkinson's Disease Parkinson's disease is a progressive movement disorder that requires care over aperiod of 10-15 years. It is caused by the degeneration of dopamine producingnerve cells in the brain. Dopamine is a neurotransmitter involved in controllingmovement and coordination. As patients' dopamine levels decrease, they exhibitprogressive inability to initiate and control physical movements. The diseaseaffects 1% of the over 50 population and about 10% of over 60s, which equates toabout one million people in the USA. The current worldwide market forParkinson's disease products is estimated to be approximately US$3 billion. Noneof the current treatments provide long-term relief from symptoms. This information is provided by RNS The company news service from the London Stock ExchangeRelated Shares:
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