Oxford BioMedica Announces Successful Completion of ProSavin® Phase I/II Study in Parkinson's Disease
-- 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)
· Favourable safety profile with no serious adverse events related to ProSavin® or the enhanced
· Average motor function1 improvement of 30%, with a maximum of 41% in one patient; and
· L-DOPA "equivalent" therapy has reduced in all six patients, in what is usually a progressively degenerative disease requiring an increase in dose.
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."
- Ends -
For further information, please contact:
Oxford BioMedica plc:
Lara Mott, Head of Corporate Communications
Tel: +44 (0)1865 783 000
Mary Clark/Sarah Macleod/Claire Dickinson
Tel: +44 (0)20 7920 2360
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 with current partners and licensees including Sanofi, Pfizer, GlaxoSmithKline, MolMed, Sigma-Aldrich, Biogen Idec, VIRxSYS, Emergent BioSolutions and ImaginAb. 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.
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.
This information is provided by RNS