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

3 August 2011


— 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
3 months
6 months
1 year
2 years
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 –