Interim data for ProSavin

RNS Number : 9954C

Oxford Biomedica PLC

09 September 2008

 



For Immediate Release	9 SEPTEMBER 2008

OXFORD BIOMEDICA ANNOUCES PROMISING INTERIM PHASE I/II RESULTS OF PROSAVIN® IN PARKINSON'S DISEASE

- Interim Data Show Improvements in Motor Function of up to 30% and DMC Recommends Continuation of Trial to Higher Dose -

- Data to be Presented at 16th ESGCT Annual Congress 13-16 November 2008 -

Oxford, UK - 9 September 2008: Oxford BioMedica (LSE: OXB), a leading gene therapy company, announced today promising interim results from the Phase I/II trial of its novel gene therapy, ProSavin, for the treatment of Parkinson's disease. The three patients in the first-dose cohort 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. An independent Data Monitoring Committee (DMC) has reviewed the data, as required by the study protocol, and recommended that the trial continues to evaluate the higher dose of ProSavin.

There were no serious adverse events associated with the surgical procedure for administration of ProSavin into the brain. All three patients were ambulatory within 48 hours of the procedure. At three months post ProSavin administration, patients showed an improvement of 23-30% as measured by the UPDRS  'off' score. This measures the degree of mobility in the absence of standard of care dopaminergic therapies. Two patients showed improvements in Quality of Life measures (QoL) that are specific to patients with Parkinson's disease, and one patient's QoL was unchanged. 

The primary efficacy endpoint of the study is based on the UPDRS Part III (motor score) assessment at six months after treatment. The principal investigator for the trial, Professor Stéphane Palfi from the Henri Mondor Hospital in Paris, intends to report the detailed interim results of the study, which may include the six-month UPDRS data and the imaging study performed at the Commissariat à l'Energie Atomique and Service Hospitalier Frédéric Joliot  in Orsay, at the 16th Annual Congress of the European Society of Gene and Cell Therapy in Bruges, Belgium, on 13-16 November 2008. 

The independent DMC has reviewed the three-month data and recommended that the trial advances to the evaluation of the higher dose of ProSavin. The first patient of this second cohort is expected to be treated in September 2008. Assuming the trial progresses as planned, the Company anticipates preliminary data from patients at the higher dose and the start of the second stage of the trial in the first quarter of 2009.

Professor Palfi commented on the interim data: '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 lower dose level. I look forward to confirming this trend over the next few months and also advancing the study to evaluate patients receiving the 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, Acting Chief Executive Officer of Oxford BioMedica, added: 'We are pleased by the progress of the first cohort of patients and we look forward to the maturing data over the coming months. The favourable recommendation from the DMC to proceed to the higher dose of ProSavin is an important milestone for the trial. 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.'

-Ends-

For further information, please contact:
Oxford BioMedica plc:  John Dawson, Acting Chief Executive Officer Nick Woolf, Chief Business Officer	Tel: [phone]
JPMorgan Cazenove Limited: James Mitford/ Gina Gibson	Tel: [phone]
City/Financial Enquiries: Lisa Baderoon/ Mark Court/ Mary-Jane Johnson Buchanan Communications	Tel: [phone]
Scientific/Trade Press Enquiries: Claire Mosley/ Holly Griffiths/ Katja Stout College Hill Life Sciences	Tel: [phone]
US Enquiries: Thomas Fechtner The Trout Group LLC	Tel: [phone]

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®, an immunotherapy 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

2. ProSavin®

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 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. 

4. LentiVector®

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.

 

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