Oxford BioMedica has announced that it has initiated a Phase I/II trial of ProSavin, its 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.”