Oxford BioMedica Announces Six-Month Efficacy Results With Low Dose of ProSavin in Phase I/II Trial in Parkinson’s Disease
- Improvements in Motor Function Maintained at Six Months -
- Data to be Presented at 38th Annual Meeting of Society for Neuroscience -
OXFORD, England, Nov. 19, 2008: Oxford BioMedica (LSE: OXB), a leading gene therapy company, announced today promising additional results from the low-dose cohort of patients in the Phase I/II trial of its novel gene therapy, ProSavin, for the treatment of Parkinson’s disease. The three patients in this cohort showed improved motor function, as measured by the Unified Parkinson’s Disease Rating Score (UPDRS) in the ‘off’ state, of an average of 30% at six months.
The robust safety profile of ProSavin has been maintained at six months with no evidence of adverse events or immunologic reactions to the treatment. In addition, all patients showed improvement in Quality of Life, as measured by the industry-standard PDQ-39 questionnaire, at six months.
The three patients that received the low dose of ProSavin demonstrated improvements in their UPDRS motor ‘off’ scores in the range of 10-50% after six months. This score measures the degree of mobility in the absence of standard of care dopaminergic therapies. One patient showed an improvement of up to 50% at six months, which was an increase from the three-month assessment, and another patient maintained an improvement of 30%. One patient showed an improvement of 10% at the six-month assessment compared to 23% at three months, although this patient’s score may have been affected by adjustments to his L-DOPA ‘equivalent’ therapy.
The clinical evaluation of the high dose of ProSavin is progressing. There have been no safety issues to date and the preliminary data from the first patient treated at the high dose are promising.
The principal investigator for the trial, Professor Stéphane Palfi from the Henri Mondor Hospital in Paris, will include the six-month data in his presentation at the 38th Annual Meeting of the Society for Neuroscience in Washington DC, USA. The presentation will be given 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).
Professor Palfi commented on the new data: "It is very encouraging that the early trend in benefit with the low dose of ProSavin has been maintained at six months. If the higher levels of efficacy are confirmed as the trial progresses, ProSavin would represent a fundamental new treatment option for patients with Parkinson’s disease."
John Dawson, Chief Executive Officer of Oxford BioMedica, added: "We are delighted by the progress of the Phase I/II trial of ProSavin. We look forward to reporting data from the high-dose group in the first half of 2009. Given the potential opportunity for ProSavin to address the unmet need in Parkinson’s disease, we are advancing our discussions with potential partners with the aim of accelerating development and commercialisation of the product."
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
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Lisa Baderoon/ Mark Court/ Mary-Jane Johnson Buchanan Communications
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Scientific/Trade Press Enquiries:
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College Hill Life Sciences
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The Trout Group LLC
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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