Skip to Content

Oxford BioMedica Announces Presentation of New Clinical Data from ProSavin Phase I/II Study in Parkinson's Disease

-- Six-month data from third cohort presented at ASGCT 14th Annual Meeting --

-- Highest efficacy results to date with 43% average motor function improvement --

Oxford, UK – 23 May 2011: Oxford BioMedica plc (“Oxford BioMedica” or “the Company”) (LSE: OXB), a leading gene therapy company, announces that new data from the on-going Phase I/II trial of ProSavin® for the treatment of Parkinson’s disease (PD) were presented at the American Society of Gene & Cell Therapy (ASGCT) 14th Annual Meeting held in Seattle, USA by Professor Stéphane Palfi, Principal Investigator at the Henri Mondor Hospital in Paris, on Saturday 21 May 2011.

Highlights of third cohort at six months (2x dose, enhanced administration)

* Average motor function1 improvement of 43%, with a maximum of 61% in one patient; * Patient diary data2 show an increase in functional “ON” time (when PD symptoms are not present) of approximately 3 hours; * Patient diary data2 show a decrease in “OFF” time (after withdrawal of PD medication) of approximately 4 hours; * Daily dose of L-DOPA “equivalent” therapy has either reduced or remained stable; * Quality of life has either improved or remained stable on the PDQ-39 questionnaire3 and these findings are consistent with the UPDRS activities of daily living (ADL) subscore4; and * Continued favourable safety profile with no serious adverse events related to ProSavin® or the enhanced administration procedure developed by the Company.

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). 2. Patient diary data only available for n=2 patients at six months. 3. Quality of life is assessed based on a standard measure of clinical benefit using a patient questionnaire known as PDQ-39. 4. The activities of daily living (ADL) subscore of the UPDRS captures the impact of PD on daily function.

Commenting on the six-month third cohort results, Professor Stéphane Palfi, Principal Investigator at the Henri Mondor Hospital in Paris, said: “Cohort 3 results at six months confirmed the safety profile of ProSavin® and the enhanced delivery method which permits a significant reduction of the surgical time. The encouraging efficacy data on Parkinsonian motor symptoms obtained in cohort 3 patients show that we are definitely approaching the right dose for the randomised Phase II study.”


Stuart Naylor, Chief Scientific Officer of Oxford BioMedica, said: “The ProSavin® data set is extremely promising in terms of the improvements we are seeing across multiple endpoints. With patient diary measures further supporting the positive impact on patients’ lives, these data underline the potential for this novel approach to address the motor symptoms of Parkinson’s disease. Our LentiVector® platform technology is designed to treat chronic, degenerative diseases and the ProSavin® results to date demonstrate the long-term benefits associated with a single administration.”


Tom Isaacs, President and Co-Founder of The Cure Parkinson’s Trust and person with Parkinson’s disease, said: “ProSavin® is one of the more advanced of the prospective gene therapy products in development for Parkinson’s and is unique in its aim to achieve dopamine replacement. These results demonstrate it also has the potential to make a huge difference to those of us living with this terrible condition. For 40 years, people with Parkinson’s have struggled with the complexities and side-effects of oral L-DOPA. These statistics indicate that, at last, there might be an effective and enduring alternative means of re-asserting control over the movement of your own body. People with Parkinson’s everywhere should take heart.” 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 two centres of excellence for neurosurgery; the Henri Mondor Hospital in Paris with Professor Stéphane Palfi as Principal and Coordinating Investigator, and at Addenbrookes Hospital in Cambridge, UK, with Dr Roger Barker as Principal Investigator.

Two dose levels (1x and 2x) have been evaluated in nine patients to date. Six patients received the 2x dose, the latter three of which were treated using an enhanced administration procedure that has been shown to reduce the surgical delivery time by 50%; facilitates higher dosing; and has the potential to provide better reproducibility of administration as study centres expand. Pre-clinical evidence suggests that the enhanced procedure may also improve the distribution and, consequently, may improve efficacy of ProSavin®, which is further supported by the new data reported today.


Summary of independently verified improvements in motor function to date:




Administration method

3 months (UPDRS)

6 months


1 year


2 years


1, n=3



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



Mean 28%

Max. up to 53%

Mean 34%

Max. up to 53%

Mean 29%

Max. up to 56%


3, n=3



Mean 26%

Max. up to 52%

Mean 43%

Max. up to 61%




A further higher (5x) dose of ProSavin® is being assessed in the current six-patient cohort; the scaled equivalent to the optimal dose in pre-clinical studies. Three-month results from the first three patients in the 5x dose cohort are expected mid-2011 and will be announced in H2 2011 following a review by the study’s independent Data Monitoring Committee (DMC). Planning is on-going for a sham-controlled Phase II study that will recruit up to 50 patients. Depending on the results from the 5x dose cohort and the independent opinion from the study’s DMC, a randomised Phase II trial of ProSavin® could be initiated in the EU/US in 2012.



For further information, please contact:


Oxford BioMedica plc: Lara Mott, Head of Corporate Communications

Tel: +44 (0)1865 783 000

Media/Financial Enquiries: Emma Thompson/Katja Toon/Amber Bielecka M:Communications

Tel: +44 (0)20 7920 2342



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-aventis, Sigma-Aldrich and Pfizer. Further information is available at<>.

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.

5. The Cure Parkinson’s Trust The Cure Parkinson’s Trust funds innovative science and inspirational scientists. It supports and galvanises pilot studies of novel therapies. It believes that the key ingredients to achieving its only goal – “a cure” - are teamwork, communication and urgency. People with Parkinson’s were integral to its formation and continue to be the focal point for every decision made.<> The Cure Parkinson’s Trust, The Vestry, 1, St Clement's Court, London, EC4N 7HB Tel: 020 7929 7656 Email:





Posted: May 2011