Interim Data from ProSavin? Phase I/II Study

RNS Number : 7078L

Oxford Biomedica PLC

04 August 2011

 

Oxford BioMedica Announces Interim Data from Highest (5x) Dose Cohort in ProSavin^® Phase I/II Study in Parkinson's Disease

-- Positive interim review of fourth patient cohort by Data Monitoring Committee --

Oxford, UK - 4 August 2011: Oxford BioMedica plc ("Oxford BioMedica" or "the Company") (LSE: OXB), the leading gene-based biopharmaceutical company, today announces positive interim data from the on-going Phase I/II trial of ProSavin^® for the treatment of Parkinson's disease (PD).  The first three patients in the current six-patient cohort were treated with a 5x dose of ProSavin^®, the scaled equivalent to the maximum dose in pre-clinical studies, and have reached their three-month assessment.

Highlights of fourth patient cohort at three months (n=3, 5x dose)

·      Favourable safety profile with no serious adverse events related to ProSavin^® or its method of administration;

·      Data Monitoring Committee (DMC) supports current planning for randomised studies;

·      Highest average motor function¹ improvement of 29% at this time point, with a maximum of 49% improvement in one patient; and

·      Reduction in average daily dose of L-DOPA "equivalent" therapy.

1.     Motor function is assessed according to the Unified Parkinson's Disease Rating Scale (UPDRS) in patients' "OFF" state (i.e. after withdrawal of PD medication).

John Dawson, Chief Executive Officer of Oxford BioMedica, said: "As expected, the interim data from the 5x dose of ProSavin^® already show improvements across several indicators of motor control and the reduction in background L-DOPA support is promising at this stage.  Importantly, the DMC has acknowledged that the improvements in motor function with decreased oral dopaminergic therapy observed to date are encouraging and clinically relevant; which further supports our preparations to progress to randomised studies.  We look forward to the full cohort 4 three-month results and the six-month primary end-point assessment later this year and are confident that ProSavin^® will continue to demonstrate its potential to transform the prospects for Parkinson's disease patients worldwide."

The on-going Phase I/II study is designed to assess the safety, efficacy and dose evaluation of ProSavin^® in patients with mid-stage PD who are experiencing reduced benefit on L-DOPA "equivalent" therapy.  The trial is being conducted at the Henri Mondor Hospital in Paris with Professor Stéphane Palfi as Principal Investigator and Coordinating Investigator, and at Addenbrookes Hospital in Cambridge, UK, with Professor Roger Barker as Principal Investigator. 

The primary efficacy end-point of the Phase I/II trial is the six-month UPDRS assessment, results from which are expected to be announced in Q4 2011 following a review of all four cohorts by the study's independent Data Monitoring Committee (DMC).  Planning is well underway for a sham-controlled Phase II study and, subject to the DMC opinion, Oxford BioMedica expects to submit regulatory applications to the EU and US agencies by the end of the year.

Summary of independently verified improvements in motor function to date:

Cohort2	Dose	Administration method	3 months (UPDRS)		6 months (UPDRS)	1 year (UPDRS)	2 years (UPDRS)
1, n=3	1x	Original	Mean 27% Max. up to 30%		Mean 30% Max. up to 48%	Mean 29% Max. up to 44%	Mean 20% Max. up to 30%
2, n=3	2x	Original	Mean 28% Max. up to 53%		Mean 34% Max. up to 53%	Mean 29% Max. up to 56%	-
3, n=3	2x	Enhanced	Mean 26% Max. up to 52%		Mean 43% Max. up to 61%	-	-
4, n=6	5x	Enhanced	n=3 of 6	Mean 29% Max. up to 49%	-	-	-

2.     Cohorts 3 and 4 had more severe mean baseline UPDRS scores than previous cohorts.

- Ends -

For further information, please contact:
Oxford BioMedica plc: Lara Mott, Head of Corporate Communications	Tel: [phone]
Media Enquiries: Emma Thompson/Mary Clark/Claire Dickinson M:Communications	Tel: [phone]

Notes to editors

1. Oxford BioMedica^®

Oxford BioMedica plc (LSE: OXB) is a biopharmaceutical company developing innovative gene-based medicines and therapeutic vaccines that aim to improve the lives of patients with high unmet medical needs.  The Company's technology platform includes a highly efficient LentiVector^® gene delivery system, which has specific advantages for targeting diseases of the central nervous system and the eye; and a unique tumour antigen (5T4), which is an ideal target for anti-cancer therapy.  Through in-house and collaborative research, Oxford BioMedica has a broad pipeline and its partners include Sanofi, Sigma-Aldrich and Pfizer.  Further information is available at www.oxfordbiomedica.co.uk.

2. LentiVector^® gene delivery technology

Oxford BioMedica's LentiVector^® gene delivery technology is one of the most advanced gene delivery systems currently available, which has many applications in product development and discovery research.  It is the system of choice for gene-based treatments addressing chronic and inherited diseases.  Oxford BioMedica has established a dominant intellectual property estate in the field of lentiviral-vector mediated gene delivery through its in-house research and from work conducted by the Company's co-founders at Oxford University.

3. Parkinson's disease

Parkinson's disease affects approximately 1.5 million patients in the seven major markets (US, Japan, UK, France, Germany, Italy and Spain) which is projected to rise to 1.7 million by 2019.  None of the current treatments provide long-term relief from symptoms, yet, by 2019, sales of these treatments could exceed US$2.8 billion in the seven major markets (source: Datamonitor, Dec-2010).  ProSavin^® has the potential to address a major unmet medical need in Parkinson's disease, offering long-lasting benefit from a single administration with an excellent safety profile.  The product could therefore also significantly reduce the social care burden that is associated with the mid to late-stage of disease.

4. ProSavin^®

ProSavin^® uses the Company's LentiVector^® gene delivery technology to deliver the genes for three enzymes - AADC (aromatic amino acid decarboxylase), TH (tyrosine hydroxylase) and CH1 (GTP-cyclohydrolase 1) - that are required for the synthesis of dopamine.  These genes re-programme transduced cells to manufacture and secrete dopamine.  The product is administered locally to the region of the brain called the striatum, converting cells into a replacement dopamine factory within the brain, thus replacing the patient's own lost source of the neurotransmitter.  ProSavin^® has the potential to address an unmet medical need in Parkinson's disease, offering long-lasting benefit from a single administration with an excellent safety profile.

 

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