Phase I/II Trial
Oxford Biomedica PLC
13 December 2007
For Immediate Release 13 DECEMBER 2007
OXFORD BIOMEDICA INITIATES PHASE I/II TRIAL OF PROSAVIN(R) GENE-BASED TREATMENT
FOR PARKINSON'S DISEASE
Oxford, UK - 13 December 2007: Oxford BioMedica (LSE: OXB), a leading gene
therapy company, announced today that it has initiated a Phase I/II trial of
ProSavin, its novel gene-based treatment for Parkinson's disease. This follows
regulatory clearance from the French Health Products Safety Agency (AFSSAPS) of
the Company's Clinical Trial Application. Patient recruitment for the Phase I/II
trial will start immediately. The trial is being conducted at the Henri Mondor
Hospital in Creteil, which is a European centre of excellence for neurosurgery
and a member of the Assistance Publique Hopitaux de Paris (APHP) in France.
The primary objectives of the trial are to assess the safety and efficacy of
ProSavin. The analyses of patients will include the application of advanced
non-invasive neuro-imaging techniques, in collaboration with the Commissariat a
l'Energie Atomique (CEA) and Service Hospitalier Frederic Joliot (SHFJ)/
Molecular Imaging Research Centre (MIRCen) in Orsay, France.
ProSavin uses gene therapy to restore dopamine production in the brain.
Parkinson's disease is caused by the degeneration of dopamine producing nerve
cells, leading to movement impairments. The product uses the Company's
LentiVector(R) system to deliver the genes for three enzymes (tyrosine
hydroxylase, GTP-cyclohydrolase 1 and aromatic amino acid decarboxylase) that
are required for the synthesis of dopamine. ProSavin is administered locally to
the region of the brain called the striatum, converting the target cells into a
replacement dopamine factory within the brain, thus replacing the patient's own
lost source of the neurotransmitter.
Long-term efficacy data in the industry-standard preclinical model of
Parkinson's disease have shown that ProSavin induces almost complete recovery of
movement function and other behavioural measurements. In this model, the
therapeutic effect of ProSavin following a single administration has been
maintained for over 24 months with no diminishment.
Professor Alan Kingsman, Chief Executive of Oxford BioMedica, commented: 'The
strength of the preclinical safety and efficacy data with ProSavin has
established strong scientific support for the clinical development of this
pioneering product candidate for Parkinson's disease. If ProSavin's safety and
efficacy profile is replicated in humans, then the product could represent a
fundamentally new approach for the treatment of Parkinson's disease and could
significantly expand the worldwide market for existing therapies, which is
estimated to be approximately US$3 billion.'
The Phase I/II trial of ProSavin
ProSavin will be the first gene-based treatment for Parkinson's disease to be
evaluated in a European clinical trial. Patients in the trial will have been
diagnosed with Parkinson's disease and will be failing on current treatment with
L-DOPA but they will not have progressed to drug-induced dyskinesias. It is a
two-stage study. The first stage is an open-label dose escalation to evaluate
two dose levels of ProSavin in cohorts of three patients each. Oxford BioMedica
plans to report preliminary results once the first cohort of patients is
assessable, which is expected in mid-2008. In the second stage of the trial, a
further 12 patients will be recruited to confirm efficacy of the optimal dose.
Current surgical approaches to the treatment of Parkinson's disease require the
destruction of brain tissue or the permanent placement of electrodes in the case
of deep brain stimulation. These treatments have certain limitations and
side-effects. The surgical procedure for administration of ProSavin entails
stereotactic bilateral injection into the striatum under general anaesthesia
using MRI-imaging and mapping. The procedure is designed to be non-destructive
to tissue and does not leave any device in the brain.
The efficacy of ProSavin will be assessed using the Unified Parkinson's Disease
Rating Score (UPDRS). Patients will be monitored at regular intervals, with the
primary endpoint being an efficacy assessment at six months after treatment. The
secondary objective of the trial is to asses the extent to which patients'
current therapy (L-DOPA) can be reduced following administration of ProSavin.
The principal investigator for the trial is Stephane Palfi, MD, PhD, a
neuroscientist at MIRCen/SHFJ-CEA and neurosurgeon at the Henri Mondor Hospital.
Dr. Palfi commented on the start of the trial: 'Current standard therapy for
Parkinson's disease is only partially effective in the mid to late stage of
disease and can induce debilitating side-effects after long-term use. ProSavin
has the potential to address this unmet medical need, offering long-lasting
benefit from a single administration. I am very pleased to be involved in the
first clinical trial of this potentially exciting new treatment paradigm for
Parkinson's disease.'
Professor Alan Kingsman of Oxford BioMedica added: 'We are delighted to have
received a favourable review from the French authorities that allows us to
commence patient recruitment in the Phase I/II trial. This clinical trial of
ProSavin is the culmination of over ten years of research in Oxford BioMedica
and, before that, in Oxford University. It is the first trial using our
proprietary LentiVector technology and, as such, this represents a major event
for Oxford BioMedica and the future of the pipeline of products that use the
same technology.'
-Ends-
For further information, please contact:
Oxford BioMedica plc: Tel: +44 (0)1865 783 000
Professor Alan Kingsman, Chief Executive
JPMorgan Cazenove Limited: Tel: +44 (0)20 7588 2828
James Mitford/Gina Gibson
City/Financial Enquiries: Tel: +44 (0)20 7466 5000
Lisa Baderoon/ Mark Court/ Mary-Jane Johnson Buchanan
Communications
Scientific/Trade Press Enquiries: Tel: +44 (0)20 7457 2020
Gemma Price/ Holly Griffiths/ Katja Stout
College Hill Life Sciences
US Enquiries: Tel: (646) 378 2900
Thomas Fechtner
The Trout Group LLC
Notes to editors
1. Oxford BioMedica
Oxford BioMedica (LSE: OXB) is a biopharmaceutical company specialising in the
development and commercialisation of novel therapeutic vaccines and gene-based
therapies with a focus on oncology and neurotherapy. The Company was established
in 1995 as a spin-out from Oxford University, and is listed on the London Stock
Exchange.
The Company has a platform of gene delivery technologies, which are based on
highly engineered viral systems. Oxford BioMedica also has in-house clinical,
regulatory and manufacturing know-how. In oncology, the lead product candidate
is TroVax(R), an immunotherapy for multiple solid cancers, which is licensed to
sanofi-aventis for global development and commercialisation. A Phase III trial
of TroVax in renal cancer is ongoing and two Phase III trials in colorectal
cancer are planned. Oxford BioMedica has two other anti-cancer product
candidates in Phase II development for melanoma and pancreatic cancer
respectively. In neurotherapy, the Company has initiated a Phase I/II trial of
its gene-based treatment for Parkinson's disease, ProSavin(R). The neurotherapy
pipeline also includes preclinical gene-based therapeutics for vision loss,
motor neuron disease and nerve repair.
The Company is underpinned by over 80 patent families, which represent one of
the broadest patent estates in the field. The Company has a staff of
approximately 80 split between its main facilities in Oxford and its wholly
owned subsidiary, BioMedica Inc, in San Diego, California. Corporate partners
include sanofi-aventis for TroVax and Wyeth for an anti-cancer targeted antibody
therapy. The Company also has collaborations with Sigma-Aldrich, MolMed and
Virxsys. Technology licensees include Biogen Idec, Merck & Co, GlaxoSmithKline
and Pfizer.
Further information is available at www.oxfordbiomedica.co.uk
2. AFSSAPS, Agence francaise de securite sanitaire des produits de sante
The French Health Products Safety Agency (AFSSAPS) was created by French law on
1 July 1998 to strengthen existing monitoring and health safety systems, and to
provide an improved response to the increasing diversity and interaction of
health safety issues relating to healthcare products. AFSSAPS benefits not only
from the skills of its forerunner in France, the Medicinal Products Agency, but
also from the fact that its scope of activity has been extended to cover the
whole range of healthcare products. It serves the interests of French public
health thanks to the high level of expertise it can call upon, both internally
and externally. It is thus able to produce not only scientific analyses and
recommendations, but also tens of thousands of decisions a year, taken on behalf
of the State by the Agency's Director General. Its mission is to guarantee the
safety, quality and proper use of health products for human use.
3. Henri Mondor Hospital
Henri Mondor Hospital, based in Creteil, is one of the largest hospitals in the
Greater Paris area. This teaching and university hospital has become a reference
for treatments, research and teaching not only locally but also for doctors and
patients elsewhere in France and abroad. The hospital's main activities are in
neuroscience, cardiology, oncology, genetics and rare diseases. The hospital has
17 medicine and intensive care departments and eight surgery departments.
4. SHFJ and MIRCen Imaging Centres of the French Atomic Agency (CEA)
The Molecular Imaging Research Centre (MIRCen) brings together skills in
molecular biology, cell biology, electrophysiology and behavioural science with
radio-isotope and anatomic functional imaging techniques. The centre designs,
develops and validates new therapeutic strategies for the treatment of various
human disorders including neurodegenerative diseases such as Parkinson's
disease. It is based in the Commissariat a l'Energie Atomique (CEA) centre at
Fontenay-aux-Roses (Paris region). The CEA's Frederic Joliot Hospital Service
(SHFJ) based in Orsay (Paris region) is one of the few centres in Europe that
employ non-invasive methods of human functional exploration (single photon
emission tomography, nuclear magnetic resonance imaging and spectroscopy,
positron emission tomography) in research as well as clinical departments.
5. ProSavin(R)
ProSavin is Oxford BioMedica's novel gene-based therapeutic for the treatment of
Parkinson's disease. The product is administered directly to the striatum in the
brain. It delivers three genes required to convert cells that normally do not
produce dopamine into cells that do, thereby replacing the dopamine synthesising
cells lost during the course of the disease. ProSavin utilises Oxford
BioMedica's proprietary LentiVector(R) system to deliver the genes AADC
(aromatic amino acid decarboxylase), TH (tyrosine hydroxylase) and CH1
(GTP-cyclohydrolase 1). These genes reprogramme transduced cells to manufacture
and secrete dopamine.
6. LentiVector(R)
LentiVector is a highly effective gene delivery system. It is based on an
engineered lentiviral vector, which is harmless to humans. The LentiVector
system has been shown to express its genetic payload efficiently and stably in
multiple tissue types, and is particularly effective in targeting non-dividing
cells, such as neurons in the brain. In addition, genes delivered with a
LentiVector system have shown long-term stable expression and, hence, offer
long-term therapeutic benefit.
7. Parkinson's Disease
Parkinson's disease is a progressive movement disorder that requires care over a
period of 10-15 years. It is caused by the degeneration of dopamine producing
nerve cells in the brain. Dopamine is a neurotransmitter involved in controlling
movement and coordination. As patients' dopamine levels decrease, they exhibit
progressive inability to initiate and control physical movements. The disease
affects 1% of the over 50 population and about 10% of over 60s, which equates to
about one million people in the USA. The current worldwide market for
Parkinson's disease products is estimated to be approximately US$3 billion. None
of the current treatments provide long-term relief from symptoms.
This information is provided by RNS
The company news service from the London Stock Exchange
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