— Study meets primary endpoint: ProSavin® is safe, well-tolerated and mediates long-term improvement of motor function —
Oxford, UK – 16 April 2012: Oxford BioMedica plc (“Oxford BioMedica” or “the Company”) (LSE: OXB), the leading gene-based biopharmaceutical company, today announces that it has successfully completed a Phase I/II study to assess the safety, efficacy and dose evaluation of ProSavin® in patients with mid-stage Parkinson’s disease (PD) who are experiencing reduced benefit on L-DOPA “equivalent” therapy.
The study evaluated three ascending dose levels (1x, 2x and 5x) in a total of 15 patients with PD. Six patients received the 2x dose, the latter three of which were treated using an enhanced administration procedure which facilitates higher dosing and reduces surgery time. Six patients received the highest 5x dose. Patients were treated at two centres of excellence for neurosurgery; the Henri Mondor Hospital in Paris, France with Professor Stéphane Palfi as Principal and Coordinating Investigator, and at Addenbrookes Hospital in Cambridge, UK with Dr Roger Barker as Principal Investigator.
The primary endpoint of the Phase I/II study is safety, and the secondary endpoint is efficacy as measured by the Unified Parkinson’s Disease Rating Scale (UPDRS) assessment at six months. All six patients in the fourth and final cohort have reached their six-month assessment time point, the results of which have been independently verified.
Highlights of fourth cohort at six months (n=6 at 5x dose, enhanced administration)
1. Motor function is assessed according to the UPDRS in patients’ “OFF” state (i.e. after withdrawal of PD medication).
In summary, ProSavin® has demonstrated a long-term safety profile, now up to 48 months post-treatment for the first two patients treated with a 1x dose. All 15 patients treated have shown an improvement in motor function at the six-month efficacy endpoint relative to baseline. As previously announced on 15 December 2011, population analysis of the first nine patients (cohorts 1-3) revealed that ProSavin® significantly improves motor function relative to baseline, with improvements remaining statistically significant up to 12 months post-treatment. The study’s independent Data Monitoring Committee also confirmed that the signals of improvements in motor function with decreased oral dopaminergic therapy observed to date are encouraging, particularly at the 5x dose.
John Dawson, Chief Executive Officer of Oxford BioMedica, said: “ProSavin® is the first lentiviral vector-based treatment for Parkinson’s disease to be evaluated in a European clinical trial and the positive Phase I/II data set provides a solid basis for further clinical development. We are currently evaluating a more potent formulation of ProSavin® to ensure the greatest chance of success in randomised Phase II studies and increase the commercial opportunity for this novel product.”
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