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Oxford BioMedica announced today that it has initiated a Phase I/II trial of ProSavin, its novel gene-based treatment for Parkinson’s disease
OXFORD, UK | December 13, 2007 | Oxford BioMedica (LSE: OXB), a leading gene therapy company, announced today that it has initiated a Phase I/II trial of ProSavin, its novel gene-based treatment for Parkinson’s disease. This follows regulatory clearance from the French Health Products Safety Agency (AFSSAPS) of the Company’s Clinical Trial Application. Patient recruitment for the Phase I/II trial will start immediately. The 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 primary objectives of the trial are to assess the safety and efficacy of ProSavin. The analyses of patients will include the application of advanced non-invasive neuro-imaging techniques, in collaboration with the Commissariat à l’Energie Atomique (CEA) and Service Hospitalier Frédéric 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 nerve cells, leading to movement impairments. The product uses the Company’s LentiVector® system to deliver the genes for three enzymes (tyrosine hydroxylase, GTP-cyclohydrolase 1 and aromatic amino acid decarboxylase) that are required for the synthesis of dopamine. ProSavin is administered locally to the region of the brain called the striatum, converting the target cells into a replacement dopamine factory within the brain, thus replacing the patient's own lost source of the neurotransmitter.
Long-term efficacy data in the industry-standard preclinical model of Parkinson’s disease have shown that ProSavin induces almost complete recovery of movement function and other behavioural measurements. In this model, the therapeutic effect of ProSavin following a single administration has been maintained for over 24 months with no diminishment.
Professor Alan Kingsman, Chief Executive of Oxford BioMedica, commented: “The strength of the preclinical safety and efficacy data with ProSavin has established strong scientific support for the clinical development of this pioneering product candidate for Parkinson’s disease. If ProSavin’s safety and efficacy profile is replicated in humans, then the product could represent a fundamentally new approach for the treatment of Parkinson’s disease and could significantly expand the worldwide market for existing therapies, which is estimated 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 be evaluated in a European clinical trial. Patients in the trial will have been diagnosed with Parkinson’s disease and will be failing on current treatment with L-DOPA but they will not have progressed to drug-induced dyskinesias. It is a two-stage study. The first stage is an open-label dose escalation to evaluate two dose levels of ProSavin in cohorts of three patients each. Oxford BioMedica plans to report preliminary results once the first cohort of patients is assessable, which is expected in mid-2008. In the second stage of the trial, a further 12 patients will be recruited to confirm efficacy of the optimal dose.
Current surgical approaches to the treatment of Parkinson’s disease require the destruction of brain tissue or the permanent placement of electrodes in the case of deep brain stimulation. These treatments have certain limitations and side-effects. The surgical procedure for administration of ProSavin entails stereotactic bilateral injection into the striatum under general anaesthesia using MRI-imaging and mapping. The procedure is designed to be non-destructive to tissue and does not leave any device in the brain.
The efficacy of ProSavin will be assessed using the Unified Parkinson’s Disease Rating Score (UPDRS). Patients will be monitored at regular intervals, with the primary endpoint being an efficacy assessment at six months after treatment. The secondary 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 Stéphane Palfi, MD, PhD, a neuroscientist at MIRCen/SHFJ-CEA and neurosurgeon at the Henri Mondor Hospital. Dr. Palfi commented on the start of the trial: “Current standard therapy for Parkinson’s disease is only partially effective in the mid to late stage of disease and can induce debilitating side-effects after long-term use. ProSavin has the potential to address this unmet medical need, 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 Parkinson’s disease.”
Professor Alan Kingsman of Oxford BioMedica added: “We are delighted to have received a favourable review from the French authorities that allows us to commence patient recruitment in the Phase I/II trial. This clinical trial of ProSavin is the culmination of over ten years of research in Oxford BioMedica and, before that, in Oxford University. It is the first trial using our proprietary LentiVector technology and, as such, this represents a major event for Oxford BioMedica and the future of the pipeline of products that use the same technology.”
SOURCE: Oxford Biomedica |
