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Oxford BioMedica Announces Presentation of New Clinical Data From ProSavin(R) Phase I/II Study in Parkinson’s Disease

May 23, 2011

OXFORD, England, May 23, 2011 /PRNewswire-FirstCall/ –

– Six-Month Data From Third Cohort Presented at ASGCT 14th Annual Meeting
-

– Highest Efficacy Results to Date With 43% Average Motor Function
Improvement -

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(R) 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 Stephane 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(R) 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 Stephane
Palfi
, Principal Investigator at the Henri Mondor Hospital in Paris, said:
“Cohort 3 results at six months confirmed the safety profile of ProSavin(R)
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(R) 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(R) platform technology is designed to
treat chronic, degenerative diseases and the ProSavin(R) 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(R) 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(R) 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 Stephane 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(R), which is further supported by the new data reported today.

    Summary of independently verified improvements in motor function to date:

    Cohort Dose Administration 3 months        6 months 1 year     2 years
                method         (UPDRS)
                                               (UPDRS)  (UPDRS)    (UPDRS)
    1, n=3 1x   Original       Mean 27%        Mean 30% Mean 29%   Mean 20%
                               Max. up to 30%  Max. up  Max. up to Max. up to
                                               to 48%   44%        30%

    2, n=3 2x   Original       Mean 28%        Mean 34% Mean 29%   -
                               Max. up to 53%  Max. up  Max. up to
                                               to 53%   56%

    3, n=3 2x   Enhanced       Mean 26%        Mean 43% -          -
                               Max. up to 52%  Max. up
                                               to 61%

A further higher (5x) dose of ProSavin(R) 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(R) could be initiated in the EU/US in 2012.

Notes to editors

1. Oxford BioMedica(R)

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(R) 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
http://www.oxfordbiomedica.co.uk.

2. LentiVector(R) gene delivery technology

Oxford BioMedica’s LentiVector(R) 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(R) 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(R)

ProSavin(R) uses the Company’s LentiVector(R) 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(R)
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.
http://www.cureparkinsons.org.uk

The Cure Parkinson’s Trust, The Vestry, 1, St Clement’s Court, London,
EC4N 7HB Tel: +44(0)20-7929-7656 Email: cptinfo@cureparkinsons.org.uk
Registered charity number: 1111816

    For further information, please contact:
    Oxford BioMedica plc:
    Lara Mott, Head of Corporate Communications  Tel: +44(0)1865-783-000

    Media/Financial Enquiries:                  Tel: +44(0)20-7920-2342
    Emma Thompson/Katja Toon/Amber Bielecka
    M:Communications

SOURCE Oxford Biomedica Plc


Source: newswire